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<?xml version="1.0" encoding="UTF-8"?><rss version="2.0" xmlns:content="http://purl.org/rss/1.0/modules/content/" xmlns:wfw="http://wellformedweb.org/CommentAPI/" xmlns:dc="http://purl.org/dc/elements/1.1/" xmlns:atom="http://www.w3.org/2005/Atom" xmlns:sy="http://purl.org/rss/1.0/modules/syndication/" xmlns:slash="http://purl.org/rss/1.0/modules/slash/" > <channel> <title>Vial</title> <atom:link href="https://vial.com/feed/" rel="self" type="application/rss+xml" /> <link>https://vial.com/</link> <description></description> <lastBuildDate>Wed, 15 May 2024 21:46:30 +0000</lastBuildDate> <language>en-US</language> <sy:updatePeriod> hourly </sy:updatePeriod> <sy:updateFrequency> 1 </sy:updateFrequency> <generator>https://wordpress.org/?v=6.5.3</generator> <image> <url>https://vial.com/wp-content/uploads/2021/09/cropped-favicon_512-32x32.png</url> <title>Vial</title> <link>https://vial.com/</link> <width>32</width> <height>32</height></image> <item> <title>Medidata vs. Vial | Pros and Cons</title> <link>https://vial.com/blog/articles/medidata-vs-vial-pros-and-cons/</link> <comments>https://vial.com/blog/articles/medidata-vs-vial-pros-and-cons/#respond</comments> <dc:creator><![CDATA[Owen Allen]]></dc:creator> <pubDate>Wed, 15 May 2024 20:50:27 +0000</pubDate> <category><![CDATA[Articles]]></category> <category><![CDATA[Biotech]]></category> <category><![CDATA[clinical trials]]></category> <category><![CDATA[CRO]]></category> <category><![CDATA[CRO's]]></category> <category><![CDATA[CROs]]></category> <guid isPermaLink="false">https://vial.com/?p=48930</guid> <description><![CDATA[Medidata vs. Vial | Pros and Cons The unprecedented consequences of the COVID-19 pandemic gave rise to the advent of decentralized clinical trials (DCTs), which are studies that use telemedicine and remote or local healthcare professionals to enable participants to join from different locations. As a result, the clinical research industry has slowly been shifting […]]]></description> <content:encoded><![CDATA[<p><strong>Medidata vs. Vial | Pros and Cons</strong></p> <p>The unprecedented consequences of the COVID-19 pandemic gave rise to the advent of decentralized <a href="https://vial.com/glossary/clinical-trial/">clinical trials</a> (DCTs), which are studies that use telemedicine and remote or local healthcare professionals to enable participants to join from different locations. As a result, the <a href="https://vial.com/glossary/clinical-research/">clinical research</a> industry has slowly been shifting from traditional randomized clinical trials (RCTs) to DCTs. To help facilitate this shift as smoothly as possible, technology <a href="https://vial.com/glossary/cro-contract-research-organization/">contract research organizations (CROs)</a> like Medidata and Vial CRO have been at the forefront of this transition, providing innovative digital solutions and tools. In this article, we will take a comparative look between both these leading companies to shed light on their experience and their suite of clinical trial technology. Read on to find out whether Medidata or Vial CRO is a better fit for your next study!</p> <p><strong>Medidata: A Journey of Innovation</strong></p> <p>Medidata Solutions, headquartered in New York City, was founded in 1999 by Tarek Sherif and Glen de Vries, who established the company to transform clinical trials with technology. This was a result of Sherif and de Vries noticing a significant unmet need for efficient <a href="https://vial.com/glossary/data-management/">data management</a> systems given the industry’s heavy reliance on time-consuming, costly, and error-prone processes. With this vision, they launched Medidata Rave <a href="https://vial.com/glossary/edc/">EDC (Electronic Data Capture)</a>, a cloud-based clinical data management system that has since revolutionized the industry by streamlining data collection, storage, and analysis. The long-standing worldwide success of Medidata Rave resulted in Medidata expanding their areas of focus, which now includes delivering solutions for study design, randomization, trial management, and patient engagement.</p> <p>In 2019, the company was acquired by Dassault Systèmes, a French software company, for $5.8 billion and now functions as a subsidiary. Today, Medidata’s digital solutions boast a staggering footprint in the clinical research industry. Of note, their Rave EDC is currently used by 19 of the top 20 global pharmaceutical companies, and it has powered more than 30,000 clinical studies involving over 9 million patients across nearly every country in the world. The 2023 report by Industry Standard Research also named Medidata’s Rave EDC as the most preferred EDC system used across the industry.</p> <p><strong>Vial CRO: A New Era in Clinical Research</strong></p> <p>Vial, on the other hand, was established in 2020 by Simon Burns and Andrew Brackin to serve as a platform to reimagine clinical trials by deploying technology at every step across multiple therapeutic areas. Headquartered in San Francisco, California, the rapidly growing company is supported by a robust team of nearly 200 employees, all of whom combine expertise ranging from clinical trial management and medicine to software engineering and product development. Recognizing the potential of digital tools in enhancing trial efficiency and data accuracy, Vial developed an eClinical platform that integrates all aspects of a trial including onboarding, patient enrollment, and data collection processes, providing a single, streamlined system. At the heart of this platform lies <a href="https://vial.com/edc/?utm_source=organic">Vial EDC</a>, a system integrated with Vial <a href="https://vial.com/esource/?utm_source=organic">eSource</a> (<a href="https://vial.com/glossary/electronic-source/">electronic Source</a>) and <a href="https://vial.com/epro/?utm_source=organic">ePRO</a> (<a href="https://vial.com/glossary/epro-electronic-patient-reported-outcome/">electronic Patient-Reported Outcomes</a>); this suite was designed to provide customizability and flexibility to meet the unique needs of different studies.</p> <p>In addition to its innovative technology, Vial places a strong emphasis on data security and regulatory compliance. The company’s software is fully encrypted and adheres to the latest regulatory guidelines, including the United States <a href="https://vial.com/glossary/food-and-drug-administration-fda/">Food and Drug Administration’s</a> <a href="https://vial.com/glossary/21-cfr-part-11-2/?utm_source=organic">21 CFR Part 11</a>, the <a href="https://vial.com/glossary/health-insurance-portability-and-accountability-act-hipaa-privacy-rule/">Health Insurance Portability and Accountability Act (HIPAA)</a>, and the General Data Protection Regulation (GDPR). In 2023 alone, they also announced several exciting updates to their ongoing vendor and sponsor partnerships, including the following:</p> <ul><li><strong>Nielsen BioSciences, Inc</strong>: A San Diego-based biopharmaceutical company conducting a Phase III study on the safety and efficacy of CANDIN® for the treatment of common warts.</li> <li><strong>RecensMedical, Inc</strong>: A South Korea-based medical device company evaluating their OcuCool system to deliver cooling anesthesia for painless intravitreal injection therapy, for which Vial EDC has been enabling seamless patient enrolment.</li></ul> <p>With its tech-driven approach, commitment to security and compliance, and focus on meeting the unique needs of biopharma and biotech sponsors, Vial represents a new era in clinical research.</p> <p><strong>Medidata vs. Vial: A Matter of Fit</strong></p> <p><em>Decentralized Clinical Trial Capabilities</em></p> <p>Although both Medidata and Vial offer platforms which have considerable potential to support DCTs, the former stands out in this aspect with its dedicated DCT Program. Specifically, Medidata’s Rave Clinical Cloud platform utilizes a cloud-based platform that enables remote data access, allowing sponsors and CROs to conduct trials without geographical limitations. Their end-to-end DCT solution encompasses a wide range of tools [<a href="https://www.medidata.com/en/clinical-trial-solutions/virtual-clinical-trials/">14</a>]:</p> <ul><li>Patient engagement: <a href="https://vial.com/glossary/econsent/">eConsent</a>, <a href="https://vial.com/glossary/ecoa/">eCOA (electronic clinical outcome assessment)</a>, Patient Insights, etc.</li> <li>Remote data oversight: Rave <a href="https://vial.com/glossary/what-is-a-ctms/">CTMS (clinical trial management system)</a>, Rave TSDV (targeted <a href="https://vial.com/glossary/source-data-verification-sdv/">source data verification</a>), Medidata Risk Management, etc.</li> <li>A real-time direct-to-patient (DtP) function to deliver medication to patients with ease</li></ul> <p>Although it does not cover the clinical trial lifecycle quite as extensively as Medidata, Vial’s clinical trial technology suite also offers similar support for DCTs. Its EDC platform enables real-time data capture from electronic <a href="https://vial.com/glossary/case-report-form-crf/">case report forms</a> (eCRFs), not only expediting trial performance, but also mitigating the risk of data loss like Medidata Rave. However, one of Vial’s most unique offerings relative to Medidata is its eSource tool. With its seamless integration into the Vial EDC system, clinical research sites can significantly reduce their time spent on data entry, eliminating double data entry required by systems like Rave EDC, while further minimizing human errors. Therefore, the Vial platform delivers additional measures to bolster data quality, on top of enabling similar remote data monitoring and source verification capabilities as Medidata. Learn more <a href="https://vial.com/blog/articles/comparing-vial-cros-edc-to-industry-giants/?utm_source=organic">here</a> about how Vial CRO’s EDC compares to those of industry giants.</p> <p><em>Operation Across Therapeutic Areas</em></p> <p>Medidata is fully focused on delivering top-notch digital solutions and products across a variety of different disease indications for large biopharma companies, medical device sponsors, as well as academic and not-for-profit organizations. Similarly, Vial is a full-service CRO that provides digital tools for facilitating efficient clinical trial experiences for sponsors, sites, and patients alike. However, it also offers separate teams of experts and <a href="https://vial.com/glossary/clinops-clinical-operations/">ClinOps (Clinical Operations)</a> professionals for each of their nine specialty CROs: <a href="https://vial.com/cro/oncology/">Oncology</a>, <a href="https://vial.com/cro/dermatology/">Dermatology</a>, <a href="https://vial.com/cro/ophthalmology/">Ophthalmology</a>, <a href="https://vial.com/cro/gastroenterology/">Gastroenterology</a>, <a href="https://vial.com/cro/neurology/">CNS (Central Nervous System)</a>, <a href="https://vial.com/cro/cardiology/">Cardiology</a>, <a href="https://vial.com/cro/medical-device/">Medical Device</a>, <a href="https://vial.com/cro/rare-disease/">Rare Disease</a>, and <a href="https://vial.com/cro/digital-therapeutics/">Digital Therapeutics</a>. It is worth noting that, given Medidata’s vast experience in the industry, their products have been used in trials across several therapeutic areas over the years, but not with the same level of in-house tailored medical expertise as provided by Vial. Overall, both companies offer vast coverage of different conditions, but Vial may be a better fit for sponsors looking for additional specialized guidance in specific disease indications.</p> <p><em>Management and Other Features</em></p> <p>The management team at Medidata comprises accomplished professionals with rich experience in the healthcare and technology sectors. The company also offers the following key features:</p> <ul><li>Risk-based monitoring, which optimizes the use of resources and improves data quality</li> <li>Advanced randomization and trial supply management, which ensures the right patients receive the right treatments at the right time</li> <li>A robust patient portal, which enhances patient engagement and enables patient-centered trials</li></ul> <p>On the other hand, Vial is made up of a unique blend of innovators, engineers, and ClinOps leaders. They also provide thorough end-to-end audit trails to ensure data transparency and accountability, as well as extensive data export capabilities to support data sharing and convenient analysis.</p> <p><strong>Conclusion</strong></p> <p>Medidata and Vial CRO are two leading clinical trial technology companies that have been pivotal in the transition from traditional RCTs to hybrid and decentralized clinical research. Whereas Medidata offers robust technology solutions for the entire clinical trial lifecycle, in addition to its long-standing reputation in the industry, Vial is a newer player with its innovative EDC technology that delivers efficiency, security, and affordability all at once. Medidata stands as a reliable choice for sponsors seeking solutions that have stood the test of time with a robust portfolio of success. However, Vial represents a promising prospect in the field, providing similar features with greater cost-effectiveness and user-friendliness with its customizable platform. Ultimately, the decision between these two companies and their plethora of digital tool offerings will depend on the specific needs, resources, and strategic direction of the sponsor and their clinical trial team.</p> <p><strong>Vial: The CRO for Biotech, Powered by Technology</strong></p> <p><em>Our mission at <a href="https://vial.com/cro">Vial</a> as a next-generation, tech-first CRO is to empower scientists to discover groundbreaking scientific therapeutics that help people live happier, healthier lives. <a href="https://vial.com/contact-us/">Contact a Vial representative today</a> to discover how we can make a difference for your next clinical trial!</em></p>]]></content:encoded> <wfw:commentRss>https://vial.com/blog/articles/medidata-vs-vial-pros-and-cons/feed/</wfw:commentRss> <slash:comments>0</slash:comments> </item> <item> <title>First in Human Episode #62 featuring Josh Mandel-Brehm</title> <link>https://vial.com/blog/podcast/first-in-human-episode-62-featuring-josh-mandel-brehm/</link> <comments>https://vial.com/blog/podcast/first-in-human-episode-62-featuring-josh-mandel-brehm/#respond</comments> <dc:creator><![CDATA[Owen Allen]]></dc:creator> <pubDate>Tue, 14 May 2024 20:36:42 +0000</pubDate> <category><![CDATA[Podcast]]></category> <category><![CDATA[clinical trials]]></category> <category><![CDATA[CRO]]></category> <category><![CDATA[Drug Discovery]]></category> <guid isPermaLink="false">https://vial.com/?p=48879</guid> <description><![CDATA[Amy Del Medico: Hi everyone, I’m Amy del Medico and I’m here today with Josh Mandel-Brehm from CAMP4 Therapeutics. Josh, welcome, would you like to give a little introduction on yourself? Josh Mandel-Brehm: First of all, thank you very much for having me, Amy. It’s a pleasure to join you today. I’m Josh Mendel-Brehm. I’m […]]]></description> <content:encoded><![CDATA[<p><strong>Amy Del Medico:</strong> Hi everyone, I’m Amy del Medico and I’m here today with Josh Mandel-Brehm from <a href="https://www.camp4tx.com/">CAMP4 Therapeutics</a>. Josh, welcome, would you like to give a little introduction on yourself?</p> <p><strong>Josh Mandel-Brehm:</strong> First of all, thank you very much for having me, Amy. It’s a pleasure to join you today. I’m Josh Mendel-Brehm. I’m the CEO of CAMP4. Prior to CAMP4, I was at Biogen and Genzyme in business development and strategy roles. I’m really looking forward to talking with you today about CAMP4.</p> <p><strong>Amy Del Medico:</strong> Great, thank you. Could you give us some background on CAMP4 Therapeutics and maybe touch on the meaning of the unusual name?</p> <p><strong>Josh Mandel-Brehm:</strong> Yeah, absolutely. When people ask that question. Camp 4 is the last camp before the top of Everest. It’s also a sacred ground in Yosemite National Park, where all the world’s best rock climbers come to push the boundaries of what’s possible. Aspirationally, we think of CAMP4 as pushing the boundaries of what’s possible in order to make better treatments for patients.</p> <p>That was something where we felt very strongly that it wasn’t grounded in a scientific name, but more of an aspirational name, because we have big intentions for what we can do with our platform. On that note what is CAMP4? CAMP4, in its simplest form, is using what’s called antisense oligonucleotides, a form of chemistry, to very specifically increase the expression of genes.</p> <p>How we do this is an area of biology called regulatory RNAs. These are RNAs that come out of our non-coding genome. So they come out of enhancers and promoters. They have two really important features. One is they act on nearby protein coding genes to control the expression of those genes and they act within what we say a physiological range. They allow for increases or decreases to genes. In a range that the cell can handle. A safe range, if you will. Avoiding toxicity.</p> <p>I like to think of regulatory RNAs as built in specificity. What we have discovered at CAMP4 is that when we find a regulatory RNA that is influencing the expression of a gene tied to disease, we can drug that regulatory RNA with an antisense oligonucleotide in a very specific way.</p> <p>We can increase the expression of the remaining healthy gene, which then allows us to go after a range of diseases that have a genetic basis where you’re missing a little bit of protein and the difference between being sick or healthy can be putting that little bit of missing protein back into the system.</p> <p><strong>Amy Del Medico:</strong> Interesting, thank you for the background, it sounds like it could be applied to a broad range of different indications.</p> <p><strong>Josh Mandel-Brehm:</strong> The biology itself extends to any different tissue in the body. I referred to this before, but, there’s an entire category of diseases that are either haploinsufficient, meaning one of your genes no longer works, but one is still remaining to be healthy. You’re missing 50 percent of protein to be healthy or parcel lots of function. You have a mutated enzyme that kind of works, but not enough. </p> <p>Essentially there’s hundreds of these diseases where there are no approved treatments. What we’ve chosen to do is apply our platform at first into metabolic diseases. That is the liver based diseases or certain parts of the brain. The reason for that is we know we can deliver this type of chemistry and to since all the nucleotides safely and effectively to those two tissues, which have a lot of diseases that have a genetic basis and are still in need of treatments.</p> <p><strong>Amy Del Medico:</strong> You’ve touched on it a little bit, but can you walk us through in more detail how the RAP platform works and how it amplifies mRNA and increases healthy gene expression?</p> <p><strong>Josh Mandel-Brehm:</strong> Yeah, absolutely. Our bread and butter is really what we call mapping cells. What this means is we take a human cell type. For example, in the liver, we’ll take Hepatocytes. We will apply a whole range of different next generation sequencing technologies that will generate billions of different wet lab biological data points.</p> <p>To make sense of that information, we actually have a data science team that has built algorithms that can then take all that information and turn human gene expression into an in silico exercise. What I mean by that is once we map a cell. Any gene that is being expressed, so the liver has maybe 11,000 protein coding genes, whatever gene you’re interested in, we can very rapidly show you how the DNA is controlling that gene.</p> <p>From that information, we can pick out the RNA, the regulatory RNA that is controlling a gene of interest. That’s step one, when we apply our RAP platform, which is the RNA actuating platform. Once we have a target gene in mind, so a gene that has an underlying disease where we want to increase its expression to put more protein back into the body, we can then screen the sequence of that gene, that regulatory RNA using antisense oligonucleotide. </p> <p>This is a very tried and true chemistry. It was pioneered by the likes of Ionis and Alnylam, two very established, successful companies that have approved drugs on the market. We’re using the same type of chemistry that allows us to make a very programmable and rationally designed antisense oligonucleotide that we can then deliver as a therapeutic to the patient where we want to increase gene expression to essentially shift them from an unhealthy state to a healthy state.</p> <p><strong>Amy Del Medico:</strong> I know with your development program, you’re focusing on urea cycle disorders, which can be quite complex and severe conditions. I wonder why you decided to choose that particular therapeutic area.</p> <p><strong>Josh Mandel-Brehm:</strong> Not [00:05:00] surprisingly, there are lots of different diseases we could theoretically apply our platform to, again, with a genetic basis. What are the things we think about when we choose diseases to work on? We’d like to work on as many as possible, of course, but we are a smaller biotech, and so we have to be judicious with our resources.</p> <p>One thing we think about is, “Okay, what is the genetic basis of the disease? Is there strong evidence that if we increase the expression of a target gene, even by a little bit, it’ll have a dramatic effect in helping patients?” The second thing we think about is, “The path to the clinic and how quickly and efficiently can we actually prove out our thesis in the human setting?”</p> <p>And that’s really important because sometimes the science is beautiful, but you can’t find the patients, or the science is beautiful, but you have to do a very long study to prove it out, or you need to find thousands of patients, which you’d wish to do, but again, as a smaller company, you have to be really thoughtful.</p> <p>We try and find diseases where there’s an unmet need. There’s a strong genetic basis, and we think we can at least do the first clinical study to show that our drug works. And then the last piece is, of course, is there a commercial market there? That’s really a lineup of what’s the unmet need. What do we think our drug can do in terms of its transformative impact?</p> <p> What does competition look like? In the case of urea cycle disorders, this is a disease that has many different subtypes. Meaning there are six enzymes that work together to essentially break down ammonia. For all of us that are walking around healthy, we have an intact healthy urea cycle.</p> <p>If you’re unlucky enough to have a mutated enzyme in this cycle, although you may retain some partial activity, it’s not enough to prevent the buildup of ammonia, which leads to all kinds of neurological side effects. It can lead to death very young. The idea for <a href="https://www.camp4tx.com/">CAMP4</a> would be if we can increase the rate at which the cycle can break down ammonia to urea we can have a massive impact for these patients and not just one subtype, but almost all the subtypes of urea cycle disorder patients. </p> <p>We have a strong belief based on the genetics that a small increase could lead to a big impact. We know that we can do a pretty efficient, healthy volunteer study, even though they’re not sick, but we have an assay we can look at to measure the rate of ureagenesis. We know that will allow us to essentially set parameters to go directly into patient studies. And, that this is a disease, although it’s rare 5-10,000 patients in the U. S. have it, it’s a really crummy disease. And these patients are badly in need of a therapeutic. We like that from a commercial perspective where we think our drug can become a backbone therapy for these patients that so badly need new medicines.</p> <p><strong>Amy Del Medico:</strong> And are your clinical trials focusing on just one of the subtypes? So you mentioned it could potentially work on all, are you looking at multiple subtypes?</p> <p><strong>Josh Mandel-Brehm:</strong> We’re looking at multiple cell types. That’s the grand vision for the drug. The way that we think about that is, in this case, it is a partial loss of function. It’s not a haploinsufficient disease, meaning most of the patients, regardless of their mutation, they retain some activity. So we know there’s an opportunity to boost that activity.</p> <p>What we’ve done is we’ve chosen a rate limiting enzyme. So the first enzyme in the urea cycle that’s rarely mutated is that if you increase that enzyme, the first thing it does is immediately allows more ammonia to be converted. And the second thing it does is it actually boosts the expression or the mRNA approaching of additional downstream enzymes. We get a double impact from working on that particular enzyme. That, theoretically, allows us to go up to the great majority of patients, despite having different mutations, if you will. </p> <p>One other comment, it would seem obvious to go directly into patients with our drug. However, we felt that actually going into healthy volunteers. Normal people who have a healthy intact urea cycle would be the way to go because 1: it would allow us to ensure the drug is safe. These patients have very fragile levers. The second thing is, it would allow us to actually be able to deploy a new type of assay, a relatively new assay called the ureagenesis rate test.</p> <p>That is a way of measuring the activity to convert ammonia to urea, which is a stable isotope. You want to get out of your body. Essentially, it would allow us, even though it takes a step before you get in patients in the long run, it allows us to have tools that we think can facilitate regulatory discussions that can allow us to better measure the activity of our drug and patients and essentially move more quickly towards an approvable product in the future. Because that’s our ultimate goal to get there as quickly as possible for patients in a safe way.</p> <p><strong>Amy Del Medico:</strong> Interesting. So you’re almost starting to develop your own endpoint.</p> <p><strong>Josh Mandel-Brehm:</strong> So, there is an approvable endpoint by the regulatory agencies. That is the reduction of ammonia, which would make sense because that’s the culprit that ultimately leads to symptoms. But the more tools that we have to show a benefit in patients and help patients, the better it is in terms of our own probability of success and making sure we’re running the best studies.</p> <p>Whether or not it becomes an approvable endpoint tool, we’ll see, but at the very least, it’s another tool we can use to the benefit of ourselves as well as patients.</p> <p><strong>Amy Del Medico:</strong> I wondered if you could explain to our audience the dark genome and how it’s being used to develop treatments for diseases.</p> <p><strong>Josh Mandel-Brehm:</strong> Disclaimer: I did not come up with a dark genome. I think that is a phrase that others have used. We’re happy to be included in that. [00:10:00] I’d say it’s a very broad term and said simply, of our DNA 2% give or take of our DNA encodes for genes that make proteins. The other 98% of our genome does not make proteins. It makes all different types of important functional outputs that control the expression of genes. The more and more we dig deeper into that is the dark genome. The more we study it, the more we find it’s a very important part of how our bodies control cell fade, cell differentiation, gene expression. </p> <p>For our particular purposes, we are studying the areas of the dark genome that are enhancers or promoters, specifically. That actually makes a type of regulatory RNAs. It does not code for proteins. These RNA’s turn out to have a very important function in controlling the expression of nearby protein coding genes. The 2%, if you will, of genes in our body. A fun fact is although we only have 20 to 25,000 protein coding genes, the same gene can be expressed in different cells. How is that? The way cells get specific gene expression is because of enhancers. We have many more enhancers compared to the genes themselves and the enhancers are where the RNAs come out of.</p> <p>That turns out to be a very important part of the dark genome. There are other companies that are studying it, because if there are mutations in those regions, they can lead to disease. We agree with that as well. There’s all types of opportunities leading to new targets or new ways of interpreting disease by studying the dark genome despite the fact that diseases are typically looked at as mutations, in particular.</p> <p><strong>Amy Del Medico:</strong> It sounds like a complex and quite novel approach. I imagine there were quite a lot of challenges in developing your therapeutic CMP-CPS-001. How do you address those challenges? And can you pick out any in particular that have been a problem for you?</p> <p><strong>Josh Mandel-Brehm:</strong> I always thought problems create opportunities. We always try to look at it in that way. Part of what we’re doing is novel in this. That’s our big part of it. We’re the first company that’s really taking antisense oligonucleotides and instead of down regulating or degrading proteins or enzymes that are muted and you don’t want in your body, we’re doing the opposite.</p> <p>We’re using that technology to actually act on healthy genes and increase gene expression. That’s a completely novel way of thinking about the technology and really opens the aperture for a technology that has been proven to be safe and effective in approved products and is in many products in the clinic. But again, only in one half side of the coin, that is the downregulation aspect of it. Now, all of a sudden, we’re taking something that is proven to be a therapeutic and we’re teaching it a whole new way that it could be used to treat disease. </p> <p>The way we thought about it was, we can’t get tenfold increases in gene expression. There are diseases where you really have to put a lot back in the system. That seems like a problem. On the other hand, there are hundreds of genetic diseases where, actually, you only want to increase the gene a small amount, and over-expressing the gene could lead to toxicity.</p> <p>This area of biology does not really want to allow for overexpression. It’s like a built-in safety feature. So, in this case, we took that challenge into an opportunity set, which is, this is perfect for all these haplodeficiency diseases. We don’t have to worry about overexpression.</p> <p>That’s not a major area of concern. We’re much more focused on designing a drug that is specific to the RNA that controls the gene. We’re highly confident that if we could make a version that is safe, it should have a big impact in the clinic, but that’s what we’ll be proving out. I say that based on our preclinical data. That’s an example. I think of a challenge where we took what the biology was doing. Instead of trying to make it do something it didn’t want to do, we just directed it into a set of diseases where it fit perfectly from our perspective. That’s just one example.</p> <p><strong>Amy Del Medico:</strong> Excellent. Thank you. We’ve mentioned that there’s a potential range of therapeutic applications. I wondered if beyond the UCD space, are there any other genetic diseases that CAMP4 is exploring? </p> <p><strong>Josh Mandel-Brehm:</strong> Yeah, absolutely. In the metabolic space, our first program is for urea cycle disorders, and we are in the clinic administering that drug. Our next program is for a very interesting target. It’s the LDL receptor. This is a very competitive area. Actually, there are many approved drugs in this area, for example, statins, therapies, there’s also an RNAi that’s been approved for it, as well. There are millions of people walking around some of the genetic basis that have LDL levels that essentially put them at high risk for cardiac events. Now, in a decent amount of those patients statins have been a very effective treatment.</p> <p>PCSK9 is another effective way to do that. Those are more recent, therapeutics that are based on genetics, by the way. Yet, still, there are many patients that their LDL levels are remaining way too high. Or they’re not responding or they have side effects from those other drugs I mentioned.</p> <p>And so maybe they can take 1, but not the other. So essentially, there remains a pretty big opportunity, despite the fact they’re approved drugs that if you can come up with other ways to remove LDL, you could help millions of people. Our approach is, in fact, to directly upregulate the receptor that ingests that LDL and removes it from the system.</p> <p>It’s a very clever way to essentially go about solving the problem. We think that could be a really [00:15:00] important treatment regimen in the context of the other treatments that are out there as well. Again, that’s because there’s still many people that need to lower those LDL levels. That’s another example of a metabolic program, and both those programs are subcutaneous delivery. They’re not infusion. They’re injection. Which is really important, especially given the fact that many of these patients don’t want to go through infusions. very cumbersome for them. That creates an opportunity.</p> <p>Now, in the central nervous system, one area that we’re very keen to work on is, genetic epilepsies. There are many different types of genetic epilepsies, for example, Gervais syndrome, sYNGAP, SCN2A, where these are haploinsufficient, one gene is no longer functioning, one gene is still functioning. Essentially you’re missing 50% of protein to be healthy. </p> <p>We have pretty high confidence that if you can shift expression, even by just 50%, meaning you put people back up to the 75% level based on genetics. But this is going to have a pretty big impact for patients, and we would call it disease modifying. Most of these diseases have no approved treatments. Some do, but a lot don’t. These patients are really suffering. The caregivers are suffering. Probably because nobody’s really ever found a way to address these diseases. Either because not all modalities get into the CNS. Or, it’s very hard to do this with small molecules.</p> <p>Our approach of identifying a regulatory RNA that can control these genes that are underwriting the diseases using antisense oligonucleotides delivered intrathecally, that is through the lumbar puncture, just like the approved job spin browser for spinal muscle atrophy and diversion for ALS is a really nice way to essentially get the drug to the target where these patients have no other treatments and essentially are suffering from all types of different comorbidities, including seizures. That’s the next category of diseases we’re building in the brain behind those metabolic programs. </p> <p><strong>Amy Del Medico:</strong> Josh, thanks for the in depth answer. Really appreciate it. I’ve got one last question for you. Looking ahead, I wondered what excites you the most about mRNA amplifying therapeutics?</p> <p><strong>Josh Mandel-Brehm:</strong> The entire purpose of our company is to make drugs for patients. The more we get into this, the more we learn about different diseases where we think our technology could be applicable. Yeah. And so what really excites me is that if we’re able to convince people that this is not only a fundamental area of biology that we can take advantage of to make many new drugs, but that CAMP4 has the ability to make those drugs.</p> <p>We know we can’t do it all on our own. We will absolutely shepherd things forward using our capital and resources. But, I hope that it brings to bear other partnerships with bigger and smaller companies so that we can take advantage of our platform and essentially bring more drugs to patients CAMP4 can do on its own.</p> <p>I think that’s really our big vision here. We want to create the next great platform pipeline company. We know we can’t do it on our own, but, we think if we continue to show promising data that will bring to bear other ways of creating opportunities to bring drugs to patients.</p> <p><strong>Amy Del Medico:</strong> Amazing. Thank you, Josh, so much for your time and for the very interesting discussion. Appreciate it.</p> <p><strong>Josh Mandel-Brehm:</strong> Thank you. It was a real pleasure to speak with you today.</p>]]></content:encoded> <wfw:commentRss>https://vial.com/blog/podcast/first-in-human-episode-62-featuring-josh-mandel-brehm/feed/</wfw:commentRss> <slash:comments>0</slash:comments> </item> <item> <title>Decoding Clinical Trial Costs and Efficiency Roadblocks</title> <link>https://vial.com/blog/articles/decoding-clinical-trial-costs-and-efficiency-roadblocks/</link> <comments>https://vial.com/blog/articles/decoding-clinical-trial-costs-and-efficiency-roadblocks/#respond</comments> <dc:creator><![CDATA[Owen Allen]]></dc:creator> <pubDate>Mon, 13 May 2024 20:51:33 +0000</pubDate> <category><![CDATA[Articles]]></category> <guid isPermaLink="false">https://vial.com/?p=48869</guid> <description><![CDATA[Introduction The biopharma industry is poised to make revolutionary advances in and redefine drug development, and the current climate for innovation appears ideal. However, progress from molecule to approved drug is hampered by extremely high costs and lengthy clinical trials, and approximately 90% of drugs that reach clinical trials fail. We explore the causes underlying […]]]></description> <content:encoded><![CDATA[<h2 class="wp-block-heading">Introduction</h2> <p>The biopharma industry is poised to make revolutionary advances in and redefine drug development, and the current climate for innovation appears ideal. However, progress from molecule to approved drug is hampered by extremely high costs and lengthy <a href="https://vial.com/glossary/clinical-trial">clinical trials</a>, and approximately 90% of drugs that reach clinical trials fail. We explore the causes underlying the high costs associated with drug development and what value <a href="https://vial.com/glossary/cro-contract-research-organization">contract research organizations</a> (CROs) bring to sponsors in addressing these issues.</p> <h2 class="wp-block-heading">Clinical Trials: Slow and Remarkably Expensive</h2> <p>A recent<a href="https://www.mckinsey.com/industries/life-sciences/our-insights/accelerating-clinical-trials-to-improve-biopharma-r-and-d-productivity"> McKinsey analysis</a> found that although research and development (R&D) spending increased 44% from $170 billion in 2012 to $247 billion in 2022, the number of US novel drug approvals remained flat at 43 per year. These findings indicate that R&D productivity, measured by the number of US novel drug approvals vs. R&D spending, is decreasing, and an estimated cost per novel asset is as high as $2.8 billion. According to Rodríguez-Molinero et al., only 5% of molecules in oncology <a href="https://vial.com/glossary/phase-i">Phase I</a> trials reach the market taking, on average, 7.5 years.</p> <h2 class="wp-block-heading">Length and Cost of Clinical Trials</h2> <p><a href="https://www.mckinsey.com/industries/life-sciences/our-insights/accelerating-clinical-trials-to-improve-biopharma-r-and-d-productivity">McKinsey’s analysis</a> compared the average length of Phase II clinical trials between 2011 – 2015, when it was 41 months, and 2016 – 2021, when it lengthened to 44 months. For Phase II trials, the average clinical trial lengthened from 37 to 41 months between the two periods.</p> <p>A 2016 study by <a href="https://pubmed.ncbi.nlm.nih.gov/26908540/">Sertkaya</a> et al. estimated the average cost of a Phase I study in the US to range from $1.4 million (pain and anesthesia) to $6.6 million (immunomodulation); Phase II study cost ranged from $7.0 million (cardiovascular) to $19.6 million (hematology); and <a href="https://vial.com/glossary/phase-iii">Phase III</a> study cost ranged from $11.5 million (<a href="https://vial.com/glossary/dermatology/">dermatology</a>) to $52.9 million (pain and anesthesia). The study found that therapeutic area is a significant cost determinant. Across all study phases, the top cost drivers were costs associated with clinical procedure, admin staff, and site monitoring.</p> <p>In 2021, a review by <a href="https://pubmed.ncbi.nlm.nih.gov/35170336/">Rennane et al.</a> found that the R&D cost per drug ranged from $113 million to just over $6 billion (in 2018 dollars) for all new drugs, new molecular entities, and drugs in specific therapeutic classes. For new molecular entities, the per-drug cost ranged from $318 million to $2.8 billion. A World Health Organization (WHO) 2022 study estimated that the average cost to develop a new drug ranges from $43.4 million to $4.2 billion.</p> <h2 class="wp-block-heading">What contributed to the cost being so high</h2> <p>Cost drivers of R&D of new drugs include the <a href="https://vial.com/blog/articles/drug-development-costs-explained-why-is-it-so-expensive/?https://vial.com/blog/articles/top-5-challenges-in-running-global-multi-region-clinical-trials/">industrial, regulatory, and policy environment, process development, and manufacturing cost contributions</a>. In addition, an estimated 90% of clinical drug development efforts fail, and R&D costs are affected by investment size in different development phases, duration, and phase success rates. Further, the duration and success rates depend on the type of technology used and therapeutic indication. Another cost driver is capitalization, whereby R&D costs are adjusted using a cost of capital rate to reflect the return required for investment.</p> <p>The McKinsey analysis found that R&D productivity remains low. Several factors contributing to low R&D productivity were identified, including</p> <ul><li>low success rates, as only approx. 13% of assets that enter Phase I progress to eventual launch.</li> <li>Development costs remain high (60 – 70% of total costs).</li> <li>Development cycles are long (on average, 12 years to develop a novel medicine) and getting longer.</li> <li>An estimated 80% of clinical trials do not finish on time.</li></ul> <p>As indicated by Chertman, the cost of running randomized controlled trials (RCTs) has increased due to the lengthening of trial durations as <a href="https://vial.com/glossary/clinical-research">clinical research</a> is increasingly focused on chronic diseases, the fragmentation among vendors and service providers, e.g., tech solutions companies, and overcollection of data.</p> <h2 class="wp-block-heading">What are CROs doing to deliver for sponsors?</h2> <p>Many sponsors needing to accelerate large trials and reduce costs outsource to <a href="https://vial.com/blog/articles/why-do-pharma-companies-outsource-to-cros/">CROs with established protocols, global reach, and therapeutic area expertise.</a> CROs tackle clinical trial management challenges by providing sponsors with strategic site identification, selection, and activation, in addition to efficient patient recruitment and onboarding. Throughout the trial, CROs provide comprehensive on-site management and robust data quality and management while facilitating clear communication and collaboration among stakeholders. To improve efficiency and R&D productivity, CROs also stay up-to-date with and integrate appropriate tech. CROs bring their expertise in regulatory compliance and are adept at adapting to changing environments. Some CROs believe that widespread risk-based monitoring would substantially reduce study costs and suggest that the US <a href="https://vial.com/glossary/food-and-drug-administration-fda">Food and Drug Administration</a> (FDA) encourage its use.</p> <p>The McKinsey study highlights opportunities for improving R&D productivity – pointing out that durations are typically fixed and study startup and close-out account for only a small part of the overall duration. As such, the researchers suggest that the most promising opportunity to accelerate trials is to improve the speed and efficiency of trial <a href="https://vial.com/glossary/enrollment/">enrollment</a>. CROs can deliver value to sponsors by:</p> <ul><li>Applying innovative trial designs, e.g., using statistical innovation and predictive modeling to reduce the total study participants required</li> <li>using real-world evidence (RWE) to supplement participants’ data</li> <li>using patient-burden protocol assessments, predictive models, and patient and investigator panels or surveys to make informed choices, optimize study design, and develop simpler, patient-centric protocols.</li> <li>using data and analytics for site selection and management as McKinsey analysis indicates that AI-driven models can typically identify opportunities to accelerate recruitment by 15 – 20%</li> <li>increasing participant convenience and adjusting the point of delivery for trials through, e.g., hybrid trial design, which includes home visits, telemedicine, pharmacy visits, and mobile units.</li> <li>Improving site experience, which, according to McKinsey’s data, is positively correlated with trial enrollment and patient retention</li></ul> <h2 class="wp-block-heading">How is Vial specifically working to reduce the length and cost of clinical trials</h2> <h3 class="wp-block-heading">Pricing</h3> <p>Understanding sponsors’ challenges and financial risks, Vial CRO offers <a href="https://vial.com/white-papers/one-pager-vial-fixed-pricing">fixed-fee pricing</a>, a pricing model whereby sponsors are charged a flat fee for the entire clinical trial. At Vial, a fixed-fee pricing model applies to all contracts and helps sponsors stay on budget and free of costly unanticipated change orders.</p> <h3 class="wp-block-heading">Study Startup</h3> <p>The McKinsey study reported that best-in-class timelines for study startup are about two months. <a href="https://vial.com/blog/articles/study-startup-optimization">Vial CRO has developed a centralized study startup optimization process that can expedite trial initiation</a>, where the goal is to have all study sites activated within 30 days of feasibility. Vial CRO’s Site Startup Platform enables sites to seamlessly onboard to trials, eliminating administrative burdens and increasing progress transparency.</p> <h3 class="wp-block-heading">Improving site experience</h3> <p>The Vial Technology Platform leverages connected systems and intuitive design to run global trials efficiently at scale. The connected system comprises the Site Startup Platform, Electronic Source (<a href="https://vial.com/glossary/electronic-source">eSource</a>) Platform, in-house <a href="https://vial.com/glossary/edc">electronic data capture</a> (EDC), and <a href="https://vial.com/glossary/epro-electronic-patient-reported-outcome">electronic patient-reported outcomes</a> (ePRO).</p> <p><a href="https://vial.com/contact-us/">Contact us today</a> to discover how we can make a difference for your next clinical trial!</p>]]></content:encoded> <wfw:commentRss>https://vial.com/blog/articles/decoding-clinical-trial-costs-and-efficiency-roadblocks/feed/</wfw:commentRss> <slash:comments>0</slash:comments> </item> <item> <title>Medrio vs Vial | Pros and Cons</title> <link>https://vial.com/blog/articles/medrio-vs-vial-pros-and-cons-vial/</link> <comments>https://vial.com/blog/articles/medrio-vs-vial-pros-and-cons-vial/#respond</comments> <dc:creator><![CDATA[Owen Allen]]></dc:creator> <pubDate>Wed, 08 May 2024 21:26:20 +0000</pubDate> <category><![CDATA[Articles]]></category> <category><![CDATA[#clinicaltrials]]></category> <category><![CDATA[#cro]]></category> <category><![CDATA[#cros]]></category> <category><![CDATA[clinicaltrial]]></category> <guid isPermaLink="false">https://vial.com/?p=48814</guid> <description><![CDATA[As technological advancements drive the pharmaceutical industry forward, the shift towards digitalization, particularly with electronic data capture (EDC) systems, has become increasingly prominent. The concept of EDC, a computerized system designed to replace paper-based data collection methods, has significantly evolved since its emergence in the early 2000s. These systems are often used by contract research […]]]></description> <content:encoded><![CDATA[<p>As technological advancements drive the pharmaceutical industry forward, the shift towards digitalization, particularly with <a href="https://vial.com/glossary/edc/">electronic data capture</a> (EDC) systems, has become increasingly prominent.</p> <p>The concept of EDC, a computerized system designed to replace paper-based data collection methods, has significantly evolved since its emergence in the early 2000s. These systems are often used by <a href="https://vial.com/glossary/cro-contract-research-organization/">contract research organizations</a> (CROs) facilitate the collection, verification, and management of electronic data from various sources, including electronic medical records (EMRs), laboratory systems, and <a href="https://vial.com/glossary/epro-electronic-patient-reported-outcome/">electronic patient-reported outcomes</a> (ePROs).</p> <p>The widespread adoption of EDC technology has revolutionized <a href="https://vial.com/glossary/clinops-clinical-operations/?utm_source=organic">clinical operations</a>, offering benefits such as improved data management, reduced trial timelines, remote monitoring capabilities, and enhanced data security. This article offers a comparative analysis of <a href="https://vial.com/?utm_source=organic">Vial</a> and Medrio, two leading companies at the forefront of EDC innovation.</p> <h1 class="wp-block-heading">A Brief Introduction</h1> <p>Medrio was founded in 2005 with the mission to revolutionize clinical research with the creation of the first cloud-based EDC system, designed to simplify data collection without requiring advanced programming skills. Over the years, Medrio has expanded its global presence and continued to innovate, offering a diverse range of solutions to its customers. Today, Medrio is recognized for its user-friendly EDC platform and its commitment to innovation, also fostering collaboration through its CRO Partner Program.</p> <p>Established in 2020, Vial has rapidly emerged as a leading technology-driven CRO. Vial seeks to transform <a href="https://vial.com/glossary/clinical-research/">clinical research</a> by delivering faster, superior, and more cost-effective <a href="https://vial.com/glossary/clinical-trial/">clinical trial</a> outcomes. Vial Technology Platform leverages connected data and intuitive systems to streamline trial management, drawing on 150 years of collective experience and a network of over 35 research sites. With a track record of over 750 completed trials, Vial has demonstrated its expertise and capability in the field of clinical research.</p> <p>Vial Technology Platform offers a comprehensive suite of eClinical solutions, including EDC, (ePRO), <a href="https://vial.com/glossary/ecoa/">electronic clinical outcome assessment</a> (eCOA), and eConsent.</p> <h1 class="wp-block-heading">Therapeutic Area Coverage</h1> <p>Medrio’s solutions can be applied across all therapeutic areas. Nonetheless, the company excels in:</p> <ul><li>Oncology</li> <li>Central nervous system</li> <li>Dermatology</li> <li>Infectious disease</li> <li>Cardiovascular</li> <li>Rare disease</li> <li>Gastrointestinal</li> <li>Pain</li> <li>Endocrinology</li> <li>Veterinary</li></ul> <p>Vial offers therapeutic expertise in the following areas:</p> <ul><li><a href="https://vial.com/glossary/oncology/">Oncology</a></li> <li><a href="https://vial.com/glossary/dermatology/">Dermatology</a></li> <li><a href="https://vial.com/glossary/ophthalmology/">Ophthalmology</a></li> <li><a href="https://vial.com/glossary/gastroenterology/">Gastroenterology</a></li> <li><a href="https://vial.com/glossary/central-nervous-system-cns/">Central nervous system</a></li> <li><a href="https://vial.com/glossary/cardiology/">Cardiology</a></li> <li>Medical device</li> <li>Rare disease</li> <li>Digital therapeutics</li></ul> <h1 class="wp-block-heading">Solutions</h1> <p>Both Vial and Medrio provide EDC technologies as core offerings to sponsors. EDC is a system used in clinical trials to electronically record and manage subject data. It offers several benefits, including improved efficiency, reduced errors, and enhanced data quality, helping organizations to streamline operations and reduce timelines and costs while navigating the complexities of modern clinical research.</p> <h2 class="wp-block-heading">Medrio’s integrated EDC platform</h2> <p>Medrio’s EDC technology is known for its reliability, scalability, data integrity, and security features, ensuring the delivery of high-quality study data. It can quickly adapt to changing study requirements, with study builds completed in less than three weeks, and allows for seamless mid-study modifications in as little as a day, providing real-time insight into trial data. Medrio supports direct data capture (DDC), enabling the collection of study data directly at its source, regardless of internet connectivity limitations.</p> <p>Medrio’s solutions include:</p> <p><strong>ePRO/electronic clinical outcome assessment</strong></p> <p>Medrio offers an intuitive ePRO with configurable solutions to enhance participant experience, streamline site processes, and ensure data quality from protocol development to post-market analysis. Users can easily build and reuse assessment templates, facilitating rapid and consistent setup of clinical trials. Medrio’s <a href="https://vial.com/glossary/ecoa/">electronic clinical outcome assessment</a> (eCOA) solutions for clinical offer rapid setup of fully configurable tools in just a few days, without the need for custom coding. Participants are engaged through intuitive ePRO accessible on any web-enabled device. These solutions also integrate wearables and external data with the backend database, supporting various electronic clinical outcome assessments.</p> <p><strong>eConsent</strong></p> <p>Medrio <a href="https://vial.com/glossary/econsent/?utm_source=organic">eConsent</a> prioritizes patient-centricity, exemplified by a consent process that leverages patients’ own devices, ensuring convenience and accessibility. The platform includes remote consent options that encourage questions and incorporates patient education techniques, enhancing comprehension of trial participation. Medrio eConsent supports a range of consent methods, including remote or in-clinic, paper or web-based formats, suitable for patients, caregivers, and legally authorized representatives alike.</p> <p><strong>Randomization and trial supply management</strong></p> <p>Medrio’s all-in-one <a href="https://vial.com/glossary/rtsm/?utm_source=organic">randomization and trial supply management</a> (RTSM) solution empowers study teams with enhanced oversight and access to high-quality, reliable data, ultimately saving time and money. Additionally, it integrates with Medrio’s EDC, allowing for the secure transfer of randomized participant information to a centralized data repository.</p> <h2 class="wp-block-heading">Vial’s intuitive EDC interface</h2> <p>Unlike traditional clinical trial systems, which often rely on separate tools, Vial offers a unified and compliant solution for real-time, high-quality data collection, making the process faster, simpler, and more cost-effective.</p> <p><a href="https://vial.com/edc/?utm_source=organic">Vial’s EDC solution</a>, built on a cloud-native infrastructure, provides a user-friendly experience for data capture and review, ensuring compliance with regulatory standards such as 21 CFR Part 11, HIPAA, and GDPR. It offers fast database and eCRF build timelines, typically completed within four weeks, along with robust functionalities for user and patient management, intuitive data input, and comprehensive data management capabilities.</p> <p>Key features of Vial EDC include rapid database and electronic case report form (eCRF) build timelines (approximately 4 weeks), user management with robust permissions and data access controls, streamlined patient management, intuitive eCRF data input with built-in validations and rules logic, comprehensive audit trail functionality, and a full suite of <a href="https://vial.com/glossary/data-management/?utm_source=organic">data management</a> tools including queries, <a href="https://vial.com/glossary/source-data-verification-sdv/?utm_source=organic">source data verification</a> (SDV), reviews, and data export capabilities.</p> <p>Vial’s eClinical platform, powered by Vial EDC, enables integration with third-party systems including interactive response technology (IRT), pre-integrated IRT solution, as well as custom integrations. This platform offers a comprehensive suite of clinical trial technologies:</p> <p><strong><a href="https://vial.com/onboarding/?utm_source=organic">Onboarding App</a></strong></p> <p>This innovative tool streamlines and accelerates site activation by adopting a digital-first approach to managing startups, from RegPacket to clinical trial agreement (CTA). The Vial Site Onboarding App offers a seamless, self-service experience entirely conducted online, capable of achieving site onboarding in just 30 days. It facilitates real-time editing and provides a comprehensive overview of site startup statuses, streamlining the entire process for efficient management. Moreover, the Vial Site Onboarding App integrates with Vial’s preferred <a href="https://vial.com/glossary/etmf/">electronic Trial Master File</a> (eTMF), Egnyte eTMF, ensuring quick and compliant document structuring and storage.</p> <p><strong><a href="https://vial.com/esource/?utm_source=organic">eSource</a></strong></p> <p>Vial eSource is an intuitive platform that allows to integrate technology into trial management, ensuring real-time data capture and review within a unified system. Vial eSource comprises: Vial DDC, which eliminates the need for double data entry at sites and provides an intuitive user interface for immediate data input; Vial Tablets, which are conveniently shipped directly to sites and can be managed via the cloud by Vial; and the Vial Data Platform, a robust system capable of organizing data from various sources into Vial EDC. Additionally, it offers customizable integrations with laboratories, imaging facilities, EMR, RTSMs, and EDCs (including Medrio), as well as supports flexible automatic data ingestion through various formats such as API, .csv, flat file, STFP, and email.</p> <p><strong>ePRO, eCOA, and eConsent</strong></p> <p>Leveraging the same engine as Vial’s DDC technology, Vial offers user-friendly and compliant solutions for ePRO, eCOA, and eConsent. <a href="https://vial.com/epro/?utm_source=organic">Vial ePRO</a> provides patients with a compliant and customizable mobile experience. With a user-friendly interface, accessible via mobile or desktop, Vial ePRO offers interactive features, built-in data validation, and skip logic for efficient and compliant data capture. Seamlessly connected with Vial EDC, it ensures effortless, accurate, and real-time data recording and storage. <a href="https://vial.com/blog/articles/clinical-trial-basics-what-is-ecoa/?utm_source=organic">Vial eCOA</a> offers several advantages over traditional paper-based methods and eliminates inefficiencies associated with in-person visits.</p> <p><strong>Study Analytics Dashboards</strong></p> <p>Vial Study Analytics Dashboards offer real-time insights into trial progress and outcomes, drawing data from Vial’s digital-first data capture approach and internal systems. Key include Study Milestone Tracking, which monitors site and study startup progress, subject enrollment, protocol deviations, and adverse event monitoring, as well as Study Data Monitoring, which tracks subject visit statuses, data cleaning progress, query statuses, and more. Vial Study Analytics Dashboards are highly customizable to meet the specific needs and key performance indicators of each study, and can be accessed 24/7 via Vial’s dashboard web portal.</p> <h1 class="wp-block-heading">Adopt Vial EDC in Your Next Trial</h1> <p>The Vial Technology Platform is a game-changer in global clinical trials, leveraging connected data and intuitive systems for efficient trial management. With a focus on streamlining processes, Vial’s technology suite includes the site startup portal, digital visit capture, and study analytics dashboard, developed by a dedicated team of experts. Vial’s EDC solution addresses the limitations presented by existing industry giants by offering a comprehensive platform that integrates seamlessly with external tools like ePRO and digital wearables, enabling real-time data capture. This integrated approach is particularly advantageous for smaller companies with limited budgets or technical expertise, offering a user-friendly yet powerful alternative to complex ecosystems.</p> <p><strong>To learn how Vial’s CRO can support your clinical trial, please visit our website or <em><a href="http://www.vial.com/contact-us">get in touch with a Vial representative today</a>!</em></strong></p>]]></content:encoded> <wfw:commentRss>https://vial.com/blog/articles/medrio-vs-vial-pros-and-cons-vial/feed/</wfw:commentRss> <slash:comments>0</slash:comments> </item> <item> <title>First in Human Episode #61 featuring William Korinek</title> <link>https://vial.com/blog/podcast/first-in-human-episode-61-featuring-william-korinek/</link> <comments>https://vial.com/blog/podcast/first-in-human-episode-61-featuring-william-korinek/#respond</comments> <dc:creator><![CDATA[Owen Allen]]></dc:creator> <pubDate>Tue, 07 May 2024 16:07:07 +0000</pubDate> <category><![CDATA[Podcast]]></category> <guid isPermaLink="false">https://vial.com/?p=48810</guid> <description><![CDATA[Amy Del Medico: [00:00:00] Hello, everyone. I’m Amy del Medico from Vial. I am here today with Bill Korinek from Astrocyte Pharmaceuticals. Bill, would you like to give us an introduction and perhaps some background on yourself? William Korinek: Hello, Amy. Nice to meet you. Thank you for having me on the podcast here. I’m […]]]></description> <content:encoded><![CDATA[<p><strong>Amy Del Medico:</strong> [00:00:00] Hello, everyone. I’m Amy del Medico from Vial. I am here today with Bill Korinek from Astrocyte Pharmaceuticals. Bill, would you like to give us an introduction and perhaps some background on yourself?</p> <p><strong>William Korinek:</strong> Hello, Amy. Nice to meet you. Thank you for having me on the podcast here. I’m the CEO at Astrocyte Pharmaceuticals. We have a small molecule drug that’s moving into phase two clinical trials. I was a scientist first with a PhD in molecular biology from Harvard. I quickly went to the business side, did a number of years of strategic consulting to a broad range of biotech and pharma companies, with just great experience, got a lot of the business side of things, financials. and then I went to Pfizer and I was there at Pfizer for about 10 years.</p> <p>It was great for learning a lot of how big companies industrialize pharmaceutical drug development. I was fortunate to not just be in one small group. I was in a group that was able to work across the company. I was able to work with leadership teams on early discovery through late stage developments across all our therapeutic areas, across the most small and large molecules.</p> <p>So it was a great background for seeing, I think, both the kind of the science and business side of things and also working at a very strategic level and operational. It’s a good backdrop to move into a CEO role in a biotech company.</p> <p><strong>Amy Del Medico:</strong> It sounds like you have the perfect CV for the role. Thank you for the background. I wondered if you could share some insights into how you developed Astrocyte pharmaceuticals and any challenges that you faced along the way.</p> <p><strong>William Korinek:</strong> Starting any company and particularly a biotech company is definitely hard. (laughs) There’s a lot of hazards, a lot of ways to mess up forming a biotech company. The biggest things that you can rely on really are your network. One of the things about the biotech industry, even though I think the pharmaceutical world has a bad reputation overall, there’s a lot of great people in biotech.</p> <p>If you have a network from your graduate school, or earliest jobs, or just networking from conferences, people like to help other people. They want to see companies succeed. They want to see more therapeutics advancing and clinical trials. They want to see more treatments and cures advance. If you have a network and you’re able to reach out, usually there’s a lot of helpful people.</p> <p>That was great for us in the beginning with Astrocyte. We were able to contact people that pointed us towards lawyers that are very experienced at starting up biotechs, financial people that can help you set up your accounts, and the right banks and ways to do things, help get licenses set up with universities. </p> <p> That network was hugely important when we were able to get to experts that would help us refine our early discovery strategy, our early PK plans and things that we needed to have for a more sound business plan. As we apply for grants to have those applications that really made sense with all steps of the science and all the disciplines that you need.</p> <p>Definitely, forming a company, challenging, leveraging the network, and then, of course, the funding, the other biggest challenge for every biotech company is the funding. Some companies are able to catch the venture capital from the very beginning and have a robust capital plan. But I’d say the vast majority of biotechs are in that scrappy category using friends and family, using grants, using angel networks. </p> <p>Astrocyte’s always been in that category. We’ve been fortunate to get a lot of grant funding over the years. Probably about a third of our funding has come from grants. But it’s always up and down. You get enough money to get some good results. And those results you leverage to get some more capital. But, part of the challenge is you really have to reach out to that network, get a lot of experts to help you, and then find a way to get the funding and convince people that this is a worthwhile opportunity.</p> <p><strong>Amy Del Medico:</strong> Yeah, you can certainly never sit back, can you?</p> <p><strong>William Korinek:</strong> Unfortunately, no. (laughter)</p> <p><strong>Amy Del Medico:</strong> I agree with what you said about people. I think they want to make a difference, and I think everybody is really happy to share their expertise as well. That is very helpful when you’re starting up a smaller company. </p> <p>The next question is about the unmet need in neuroprotection, specifically concerning stroke, TBI, and concussion. I just wondered how the latest advancements in neuroprotection therapies are addressing those needs?</p> <p><strong>William Korinek:</strong> In terms of an area of unmet need and the level of R&D research dollars that are going into it. I think this might be one of the biggest disparities out there in the world of pharmaceuticals. I’m curious if someone has a greater example, but you think about, stroke in particular, in the U.S. is bouncing between the third, fourth, fifth leading cause of death.</p> <p>But, worldwide, it’s the number two cause of death. It’s one of the greatest causes of disability. There’s more years lost to disability from stroke and all other neuroscience indications combined, Alzheimer’s, Huntington’s, Parkinson’s, everything, nursing homes are filled with people who’ve had strokes.</p> <p>Think about that tremendous need that’s out there, and the billions of dollars that go into health care for people who’ve had strokes, and there’s very little R&D dollars. It’s just been an area that’s not guarded much venture investment or pharmaceutical research. It’s up and down in that realm.</p> <p>That disparity is [00:05:00] staggering. And then right now, I think about a dozen companies that have a true interventional therapeutics advancing in a phase two, phase three type trial, compare that to Oncology or Alzheimer’s or many of the other indications that have hundreds and hundreds of shots on goal to provide a new therapeutic.</p> <p>Stroke has still just been an area where there’s only a few companies pushing it forward. Now, there’s going to be a breakthrough in the next five, 10 years, you’re going to see some great advances. Hopefully we’re one of them. But it’s a huge opportunity given what’s changed in the last decade.</p> <p>Again, it’s a tremendous unmet need. It’s unfortunate there’s not more, R&D dollars up there trying to help these patients.</p> <p><strong>Amy Del Medico:</strong> It’s crazy that there’s such a disparity. Do you think some of it may be in part due to the fact that acute trials, particularly stroke trials, are really difficult to conduct?</p> <p><strong>William Korinek:</strong> It’s two things. One, it is challenging to conduct the trials, but it’s not impossible. There’s lots of challenging clinical trials where people find ways to make them work. I think the bigger things we need to look at the economics. There were a number of attempts in some big trials that failed in the 90s and early 2000s.</p> <p> With those failures, people backed away. They just said that it’s too hard to do a clinical trial and stroke. It’s just too random in terms of what the actual underlying injury is and what the outcomes are going to be. It’s too hard, therefore we just wanted to try.</p> <p>Once you step away from a disease, you lose your experts, you lose touch with the developments and what’s happening. It’s harder to come back in and realize what’s changed. We’ve actually seen the device companies have really made a lot of progress in this space. They’ve been the ones blazing the trail, which is, usually, drug companies lead some of the device companies, but in this one, the device companies have really pioneered how to do clinical trials in this space, which has been fantastic. It opened the door for therapeutic companies now to follow their path.</p> <p><strong>Amy Del Medico:</strong> Interesting. So, you can learn the lessons from the device trials.</p> <p><strong>William Korinek:</strong> Exactly.</p> <p><strong>Amy Del Medico:</strong> Very helpful. For listeners that maybe are unfamiliar with astrocytes, could you give a high level overview of their critical function in the brain and why targeting them holds so much promise for neuroprotective therapies?</p> <p><strong>William Korinek:</strong> For the last 100 years, neuroscience and the central nervous system has really been all about neurons, there are the exciting cells, signals go back and forth with electrical currents, and the rest of the brain has always been thought of as this gap filler glial cells. And it’s only been the last kind of 20, 30 years where the research has started to explore more: why are all these other cells so important? Why are they conserved? Maybe I can share two quick stories. When Albert Einstein died they actually did a study of his brain.</p> <p>They thought: was there something different about his brain relative to the normal human brain? Was it bigger? Was it more dense? Was there, “Something?” And in all aspects, his brain looked like the typical human brain. Except for one little side note finding, which was the ratio of astrocytes to neurons was higher.</p> <p>He had a higher density of astrocytes in his brain than typical. People didn’t make much of that at the time, but if I could give you a second anecdote, too. The ratio of astrocytes to neurons in the frontal cortex of your brain is about 4 to 1. Astrocytes are the most common cell there. They’re very conserved and in some species are actually higher.</p> <p>Whales and dolphins have astrocytes to neuron ratios that are in the 7 to 1 range. It’s very interesting because these are animals that people think of as on the smartest end of the spectrum. At the same time, they can dive underwater for many minutes, and an hour in some cases. That relates to how we treat strokes and where there’s stress in the brain, low oxygen environments. </p> <p>These animals with higher ratios of astrocytes to neurons actually can survive longer in those low oxygen environments. There’s a lot of interesting things about astrocytes that people are starting to study and understand their role as a more important central caretaker cell in the brain.</p> <p>Now we know that there’s several large pharma that have entire programs now that are about astrocyte dysfunction, glial cell dysfunction, and how it’s really associated with many of the neurological diseases that we see out there. Only in the last 20, 30 years, though, because it’s an overlooked area and now it’s definitely starting to explode a bit.</p> <p><strong>Amy Del Medico:</strong> Fascinating. That was so interesting. I didn’t know that. Your asset is, AST-004. It works by targeting the astrocytes that we’ve just been discussing. Why has this approach been overlooked, historically, in neuroprotection research? What do you think is exciting about its potential?</p> <p><strong>William Korinek:</strong> Clearing out astrocytes here, we like to think of the analogy of parents and children that neurons are like children. They’re just very specialized in what they do. They, for the most part, are sending an axon signal.</p> <p>They really don’t care for themselves. They’re really dependent upon the astrocytes for a lot of the nutrients, metabolism, recycling And when there’s a stressful situation, like, during a stroke or traumatic brain injury. Things go haywire, and the kids just go crazy, they just start firing the signals more than they actually have recurring depolarizations. They tire themselves out, they exhaust themselves and actually, the cells can basically end up living and dying because of it. </p> <p>So the astrocytes, as the caretaker cells, can calm things down. One of the things that the astrocytes are known for is they clear glutamate from the synaptic cleft, so it stops that excitatory signal. [00:10:00] But if your assets aren’t functioning, they won’t do that. That causes the neurons to depolarize and tire themselves quicker. The caretaker cells do have mechanisms to help. It’s that clearing that glutamate, providing more glutathione as a substrate to the neurons as well.</p> <p>There’s things that they can be doing, but eventually tire out. So what is our drug AST-004? It’s an adenosine agonist. Adenosine ends up being a distress signal in the brain. With our molecule, we’re able to tell the astrocytes to prolong that distress signal. Tell them to keep using their energy stores, alternate energy stores, to keep fighting in a way to keep caring for the neurons.</p> <p>We can see the cells are able to recover their ion channels and start repolarizing control cellular level edema. We see they’re able to help the neurons greater. If the astrocytes survive, the neurons in the area can survive. There’s lots of benefits we have through this mechanism, which really is leveraging some of the intrinsic healing mechanisms that the brain does have. </p> <p><strong>Amy Del Medico:</strong> We know that with stroke, one of the key issues is being able to treat patients quickly. There’s a really narrow timeframe after onset of the disease. I just wondered how AST-004 can potentially address that particular challenge and improve outcomes for patients.</p> <p><strong>William Korinek:</strong> In stroke, the mantra is ” time is brain” and the longer people have a stroke, or have a blockage, the more the brain is lost. For the current treatments, one is TPA (alteplase) or tenecteplase. It’s a thrombolytic that helps chew up the clot, in a simple way, but it has safety liabilities, it has to be given within the first four and a half hours. With strokes, sometimes, you find someone that woke up with a stroke, you’re not sure how long it’s been going on. They often will not administer that just because you’re not sure how long it’s been. </p> <p> With surgery, it’s interesting, the time clock has really changed in stroke in the last decade, such that it’s starting to not be defined by the hours on the clock and more so by the state of the tissue. Some people have strokes that really evolve very fast. If they don’t have much collateral circulation in the area, much blood flow at all, so the cells die pretty quickly and the lesions grow, really, in the first, 6 to 10 hours, the stroke can be over. </p> <p>Other people, maybe it’s fortunate the way their brain developed has more collateral circulation in the area, the lesions grow slower. There’s more stability. And 20 hours into that stroke, they could still have a brain that has a number areas that’s at risk. But it’s still evolving, still salvageable. With the second method of treatment now, the device thrombectomy procedure, they look at the state of the tissue. If there is tissue to be salvaged, they’ll go in. By 8 hours, there might be nothing left to salvage. But in some of the people at 20 hours, there’s still plenty of salvage, and they will go in and do the surgery. </p> <p>We think that as long as there’s brain left to salvage, our drug can have a benefit. We’ve seen that even in a mouse experiment as late as 24 hours. If we start treating at 24 hours, we freeze the damage at that point. It doesn’t go further. If you treat earlier, it’s better. You can stop it earlier. But there’s a late window here where you can still be treating. The other aspect that’s an advantage of our drug is that we see benefit in both ischemic side of stroke and the hemorrhagic.</p> <p>There’s two types of stroke, both the blockage of a clot or rupture of a blood vessel. And those two treatments of thrombolytics, TPA and surgery only work when it’s a clot. They cannot even be considered until the patient gets to the hospital, they get an image, and they know what’s a clot and where it’s at, then they can do it. </p> <p>For us, because we don’t need to differentiate, we can be administered earlier. Even emergency responders can be showing up, they can tell us a stroke, they see hemiparesis, and they can start administering our drug way earlier, in the ambulance with an IV. Versus waiting until they get to the E. R. We see our approaches and that advantage of potentially starting earlier and still being relevant later if a patient shows up late.</p> <p><strong>Amy Del Medico:</strong> That’s amazing that you’ve got the option. You can treat those patients earlier, but that doesn’t happen, there’s potential for benefit later as well. Very exciting. We talked about the emerging understanding of how astrocytes work and what they do. I wondered how the landscape for neuroprotective clinical trials has evolved thinking in particularly in terms of clinical trial design and drug development.</p> <p><strong>William Korinek:</strong> This has probably been the biggest change. In many other diseases, you’ve seen more personalized trials, more stratification of patients, some ways to get more sophisticated and trial design. cancer is the prime example where they shifted from breast cancer and prostate cancer to using different biomarkers, triple negative breast cancer.</p> <p> You’ve been able to define the underlying disease a lot better. In stroke, that wasn’t available in the 90s and 2000s. (laughs) when people were coming in with a stroke, they were enrolling people in clinical studies that may have been 24 hours or 48 hours into a stroke. The damage is done. There’s nothing left to really do or save at that point. They’re testing people that had very large vessel occlusions, and people with very small vessel occlusions which are gonna have very different outcomes. People had the clots in very different locations in the brain, which leads to different, clinical outcomes, as well.</p> <p>So it was a wild west of types of patients that they were enrolling in these studies. And it makes sense that these [00:15:00] people, regardless of intervention, some people, looked like they were doing quite well and were still coherent, and then they would be dead in 24 hours. There’s other people that were comatose almost, and then, were fine 24 hours later. </p> <p>It’s a challenging population, or at least the way it was studied back then. That really changed around 2010. People started using the standard imaging, which now everybody uses, you get a code stroke, someone that’s got rushed into the ER, they always image these people now to see whether they have a clot or not. You can use that imaging to see what’s actually happening in the brain, and pick people that are having a similar type of stroke. </p> <p>The big advance was now using that imaging to say, “Hey, we’re just going to do a study with people that have large vessel occlusions, strokes that are in that middle cerebral artery, and, the lesion is only about a third at most of the at risk tissue.” So there’s a big mismatch between what could be saved and what’s already dead. If they have that mismatch, now, we’re going to try to do an intervention, which in the 2010s, it was really about thrombectomy devices and this endovascular treatment, but they’re picking people that had, again, the same injury at the same location at the same stage of progression, and they had more similar outcomes.</p> <p>You could finally tell if your intervention was having a benefit or not. So this is tremendous. It went from a lot of clinical trials that are very noisy, big spread of outcomes to now a much more homogenous population, and we’d be able to see the benefit from those device trials. Now, the newest generation of companies that are looking at neuroprotective treatments are leveraging this approach, and are doing clinical trials now based on imaging based patient selection. </p> <p>You can have that homogenous population to do these initial studies. Now, eventually, you want to work and, almost all stroke patients are broader population, but at least for these kind of critical phase 2 proof of concept type trials, you can get that more modest group and reduce the number of variables and reduce that risk, so you can actually see whether or not the drug is working or not </p> <p><strong>Amy Del Medico:</strong> So, you can reduce the noise by using the imaging to really target your patient population.</p> <p><strong>William Korinek:</strong> Exactly. That’s true in traumatic brain injury, as well. It’s an area where there has been probably even less clinical trials, but you can be looking at the level of edema, you can be looking at whether there’s clear contusion, etc . But, imaging based patient selection has been a revolution now in these areas.</p> <p><strong>Amy Del Medico:</strong> I know you’re developing other therapies beyond AST-004. Are there any other areas that you’re exploring in the field of neuroprotection?</p> <p><strong>William Korinek:</strong> We have been looking at multiple areas. We have some great data, not only in stroke, but in traumatic brain injury and spinal cord injury, and in the concussion model. In terms of most exciting, I probably put concussion. Concussion is something that, like the world’s really learned about in the last, kind of 10, 15 years, now, and have become aware of not only the acute symptoms and challenges, but, the long-term, consequences of multiple concussions as well. </p> <p>The statistics are that just 1.7 million traumatic brain injuries each year in the U.S. go and present to the emergency room. The majority of those are mild traumatic brain injuries, but there’s an estimate about 5 to 10 million more that are happening that is concussions that people don’t go to the E.R. because there’s nothing’s going to happen there except a big medical bill, perhaps, because there’s no treatments out there for concussion.</p> <p>Our drug, fortunately, has been very safe. We’ve gone through our regulatory toxicology studies, two different phase one studies, we have a very safe profile. In addition to treating the severe injuries of stroke and traumatic brain injury, we see our drug is having a lot of potential for treating concussion.</p> <p>We’ve been fortunate, the U.S. Department of defense has really looked at our data that we’ve done in various animal models and been impressed with it in traumatic brain injury. They’ve actually given us grants for the progression of drugs and to help make an oral tablet version. Our main hospital version is an IV that you get to the stroke patients or TBI patients, but we now have oral tablets that can be in weather resistant blister packs that you can imagine in a soldier’s backpack or on the sideline of a football or soccer game. When someone gets a concussion, you can take these tablets, and it dissolves in the tongue in less than four seconds without any water. We see that as having similar benefits to help, with that near term acute treatment to help with brain swelling, help with symptoms.</p> <p>We would hope that if you’re able to help in the acute phase, and help people return to the sport, or return to work, or return sooner without symptoms, that’s a win. If it can help with that immediate cleanup, so that there’s less chance of long term inflammation consequences, that would be tremendous as well. We’ve seen some evidence of that in long term mouse studies, but that’s a longer term goal for retreating people. But we see that as an area that is a tremendous need. We think we have a potential to help.</p> <p><strong>Amy Del Medico:</strong> Concussion can be quite debilitating, can’t it? I’ve had it several times and, you can imagine there’s quite a high economic impact with days off of work.</p> <p><strong>William Korinek:</strong> Exactly. Right now people just tell you to just go rest because the brain can heal itself, but, 20 percent of people are still feeling symptoms 30 days later, and so there’s a need.</p> <p><strong>Amy Del Medico:</strong> [00:20:00] Absolutely. And so this is the last question and more of a sort of forward looking question, but I just wondered what excites you most about the future of neuroprotection therapies and where do you see the field heading in the next decade?</p> <p><strong>William Korinek:</strong> Neuroscience is probably one of the most exciting places to be. It was an area that was behind the curve and really taking advantage of the genomics revolution that occurred. Oncology was ahead of it because they were taking biopsies and understanding what’s happening between pathogenic cells and normal cells. Neuroscience was much later in understanding the cells involved, the genomics implications and so I think that really started to play out in the 2010s where people really understood all the genes and cells that are really driving disease states and a lot of the neuroscience indications.</p> <p>We’re finally seeing some progress in Alzheimer’s disease, some other areas. It’s going to be the next decade here is really about neuroscience and in terms of neuroprotection, we’ll see some wins here in that acute space where people have concussions, migraine injuries, strokes, but, if you’re able to help the brain do better in those more severe situations there’s a lot of potential here to help it in the more mild situations as well. You start thinking about the stress from Alzheimer’s, or repetitive concussions. I think there’s gonna be a lot of potential here for neural protection, neural restoration, over time.</p> <p>So you think about Alzheimer’s, CTE, even aging, there’s lots of benefits that can come from enhancing glial cell function, astrocyte function, and in brain health. So that gets me excited. I think it’ll be much better brain health here in the decades to come. Hopefully, our research will contribute to that.</p> <p><strong>Amy Del Medico:</strong> Bill, thank you so much for talking with me. It’s been absolutely fascinating learning more about astrocytes. I really appreciate your time today. Thank you.</p> <p><strong>William Korinek:</strong> Thank you again, Amy. </p>]]></content:encoded> <wfw:commentRss>https://vial.com/blog/podcast/first-in-human-episode-61-featuring-william-korinek/feed/</wfw:commentRss> <slash:comments>0</slash:comments> </item> <item> <title>Castor vs. Vial | Pros and Cons</title> <link>https://vial.com/blog/articles/castor-vs-vial-pros-and-cons/</link> <comments>https://vial.com/blog/articles/castor-vs-vial-pros-and-cons/#respond</comments> <dc:creator><![CDATA[Owen Allen]]></dc:creator> <pubDate>Wed, 01 May 2024 19:09:30 +0000</pubDate> <category><![CDATA[Articles]]></category> <category><![CDATA[#clinicaltrial]]></category> <category><![CDATA[#clinicaltrials]]></category> <category><![CDATA[CRO]]></category> <category><![CDATA[CROs]]></category> <guid isPermaLink="false">https://vial.com/?p=48790</guid> <description><![CDATA[The landscape of medical research is currently in the midst of an exciting transition wherein technology has rapidly become a critical tool for clinical trial management and patient data collection. For example, the frequency of clinical trials using digital health technologies, such as wearables and telemedicine platforms, increased from 0.7% in 2010 to 11.4% in […]]]></description> <content:encoded><![CDATA[<p>The landscape of medical research is currently in the midst of an exciting transition wherein technology has rapidly become a critical tool for <a href="https://vial.com/glossary/clinical-trial/">clinical trial</a> management and patient data collection. For example, the frequency of clinical trials using digital health technologies, such as wearables and telemedicine platforms, increased from 0.7% in 2010 to 11.4% in 2020 to enable continuous remote monitoring of real-world patient health data. Other technologies like electronic data capture (<a href="https://vial.com/glossary/edc/">EDC</a>) are also deeply integrated into modern clinical trials, but these have been around since the 1990s, known then as ‘remote data capture’ (RDC).</p> <p>As far back as 2009, 41% of clinical trials were estimated to be relying on an EDC system; since then, EDC software have evolved into a user-friendly experience with significant electronic interoperability with electronic patient reported outcomes (<a href="https://vial.com/glossary/epro-electronic-patient-reported-outcome/">ePRO</a>), electronic consent (<a href="https://vial.com/glossary/econsent/">eConsent</a>), electronic <a href="https://vial.com/glossary/case-report-form-crf/">case report forms</a> (eCRFs), and electronic source (<a href="https://vial.com/glossary/edc/">eSource</a>) platforms. Among the leading <a href="https://vial.com/glossary/cro-contract-research-organization/">contract research organizations (CROs)</a> providing these technologies are Castor, with its established reputation for reliable EDC technology, and Vial CRO, a newer player quickly becoming well-known for its digital innovation. In this article, we deep dive into the products offered by each of these tech-driven CROs and provide insight into their benefits and drawbacks.</p> <p><strong>About Castor</strong></p> <p>Although many experimental drugs hold significant promise for many patients, the harsh reality is that 93% of these painstakingly developed drugs fail during clinical trials and 35% of trials do not see the light of day due to a lack of patient enrollment. Castor recognizes these discouraging numbers and has made it their mission to make research data reusable, thereby saving sponsors their time, effort, and resources. The company has a history of strongly advocating for clinical trials driven by artificial intelligence (AI) using research data captured in a secure, compliant cloud platform to help expedite the drug development process. With this in mind, Castor was founded in 2012 to provide researchers with a platform that could capture and integrate high-quality data from various sources into one compliant platform. Today, with its team of over 100 professionals, the company is relied on by thousands of medical device, biotech, and academic researchers around the globe to accelerate over 1000 studies.</p> <p><strong>Overview of Castor: Castor EDC, eConsent, and ePRO</strong></p> <p><em>EDC</em></p> <p>The company’s flagship product, <a href="https://www.castoredc.com/electronic-data-capture-system/">Castor EDC</a>, is renowned as the top-ranked EDC platform, boasting over 147,000 users and has been utilized in more than 14,000 studies [<a href="https://www.castoredc.com/about-us/">4</a>]. In addition to its well-established reputation for consistently delivering a reliable and efficient platform, the use of Castor EDC also spans over 90 countries having impacted more than 6 million patients. The company offers a comprehensive suite of tools that integrates seamlessly into the Castor EDC platform, namely eConsent and ePRO.</p> <p><em>ePRO</em></p> <p>The <a href="https://www.castoredc.com/epro/">Castor ePRO module</a> enables capturing data right from the patients themselves, helping improve their trial experience by providing secure, accessible questionnaires through email and the Castor Connect mobile application. With this system, sponsors can expect secure data capture directly from participant mobile devices, real-time survey progress tracking, and automated scheduling of questionnaires.</p> <p><em>eConsent</em></p> <p><a href="https://www.castoredc.com/econsent/">Castor’s eConsent platform</a> offers a range of electronic solutions that enhance efficiency from recruitment and screening to data capture and analysis throughout a clinical trial. This platform allows participants to have a seamless onboarding experience with a customized recruitment portal and pre-screening questionnaires. Sites also benefit from the ability to conduct video-based remote consent visits, enabling participants to engage from the comfort of their homes.</p> <p><strong>About Vial CRO</strong></p> <p>Vial CRO is a San Francisco-based company revolutionizing clinical trials by offering faster, more efficient trial results at a significantly lower cost for biotech sponsors. Founded in 2020, Vial was created to serve as a platform to reimagine clinical trials by deploying technology at every step across multiple therapeutic areas, including <a href="https://vial.com/cro/dermatology/">Dermatology</a>, <a href="https://vial.com/cro/gastroenterology/">Gastroenterology</a>, <a href="https://vial.com/cro/oncology/">Oncology</a>, and more. Vial’s state-of-the-art technology platform integrates all aspects of a trial from onboarding, patient enrollment, site communication, to data collection processes, providing a single, streamlined system. The team at Vial is a blend of <a href="https://vial.com/glossary/clinops-clinical-operations/">ClinOps (Clinical Operations)</a> expertise and software product and engineering expertise, who have worked together to develop Vial’s technology platform and raise over $100M. The funds have enabled the company to successfully become a leading global, full-service CRO reshaping the future of clinical trials.</p> <p><strong>Overview of Vial CRO: Vial EDC, eSource, and ePRO</strong></p> <p><em>EDC</em></p> <p><a href="https://vial.com/edc/?">Vial EDC</a> was developed in-house by Vial’s software engineering team to create a custom platform that streamlines study startup and build processes, minimizes vendor dependencies, and allows for flexible data integration. Housed within a cloud-native infrastructure, Vial EDC boasts a user-friendly interface that has been deployed to over 30 <a href="https://vial.com/glossary/clinical-research/">clinical research</a> sites, impacting more than 1500 enrolled participants around the United States (US). It also maintains strict compliance with regulatory guidelines like the US <a href="https://vial.com/glossary/food-and-drug-administration-fda/">Food and Drug Administration’s</a> <a href="https://vial.com/glossary/21-cfr-part-11-2/?utm_source=organic">21 CFR Part 11</a>, <a href="https://vial.com/glossary/health-insurance-portability-and-accountability-act-hipaa-privacy-rule/">HIPAA</a> (Health Insurance Portability and Accountability Act), and GDPR (General Data Protection Regulation). <a href="https://vial.com/blog/articles/comparing-vial-cros-edc-to-industry-giants/?">Read more here</a> about how Vial’s EDC system compares with that of industry giants.</p> <p><em>eSource</em></p> <p><a href="https://vial.com/esource/?">Vial eSource</a> is an innovative tool that replaces paper-based data capture and integrates with Vial EDC, eliminating double data entry, strengthening data compliance and quality, and enabling centralized remote monitoring. It assures data quality through an intelligent, real-time data capture system, built on CDISC standards using SDTM-controlled terminology for quality on-entry. The company’s eSource interface is powered by standardized data types, data validation, and range rules on entry, required fields, and skip logic, among others.</p> <p><em>ePRO</em></p> <p>Lastly, <a href="https://vial.com/epro/">Vial’s ePRO</a> platform provides a consumer-grade, mobile patient experience that is compliant and customizable for patients in the trial. It features a seamless, user-friendly interface easily accessible by either mobile or desktop, as well as built-in data validation, skip logic, and interactive functions for quick and compliant data capture. The Vial ePRO module also allows for real-time monitoring of patients and improved reporting, as well as accurate, real-time data recording and storage with its connected data flow with Vial ED.</p> <p><strong>Castor versus Vial: A Matter of Fit</strong></p> <p>Both companies offer an intuitive technology suite with the capacity to capture and integrate high-quality data from various sources into a single compliant platform, eliminating the need for double data entry. However, with the inclusion of an eConsent module, Castor’s platform may offer a more well-rounded option for sponsors looking for an all-in-one trial management tool, particularly for decentralized clinical trials (DCTs). Despite these advantages, the emphasis on data integration and the advanced nature of Castor’s technology suite might call for a deeper level of technical expertise from research sites. This increases the burden of staff training for the sponsor or CRO in charge of running the clinical trial.</p> <p>On the other hand, Vial has the advantage of providing a more convenient experience for site staff with its unique eSource tool, which addresses a significant pain point for <a href="https://vial.com/glossary/clinical-research-or-study-coordinator-crc/">clinical research coordinators (CRCs)</a> looking to reduce data entry burden after patient visits. Furthermore, because Vial’s EDC is developed in-house, their system minimizes vendor dependencies and allows for more flexible data integration. However, Vial’s relatively recent emergence in the field might be a point of concern for some sponsors, who may prefer working with more established platforms with a longer track record.</p> <p>Choosing between Castor and Vial CRO ultimately boils down to a matter of fit; therefore, sponsors must carefully consider their specific needs and objectives when making this decision. For those seeking a platform with a strong track record and a focus on data integration, Castor might be the ideal choice considering the company’s established reputation, as well as their advocacy for data reuse and AI-driven DCTs. In contrast, sponsors who prioritize a similarly streamlined all-in-one data entry system that requires a more user-friendly approach might find Vial CRO’s platform more suitable. Despite its relatively new entry into the CRO industry, Vial has shown strong potential with its tech-first approach to clinical trials and can hold its own against established companies like Castor.</p> <p><strong>Conclusion</strong></p> <p>In conclusion, both Castor and Vial CRO offer innovative solutions designed to expedite and streamline the clinical trial process. Although each platform comes with its unique strengths and limitations, the choice between the two will largely depend on the specific requirements and objectives of the sponsor. As clinical trials continue to evolve in the face of advancing technology, clinical trial management tools like those offered by these leading CROs will undoubtedly play a crucial role in shaping a new era of drug development.</p> <p><strong>Vial: The CRO for Biotech, Powered by Technology</strong></p> <p><em>Our mission at <a href="https://vial.com/cro">Vial</a> as a next-generation, tech-first CRO is to empower scientists to discover groundbreaking scientific therapeutics that help people live happier, healthier lives. <a href="https://vial.com/contact-us/">Contact a Vial representative today</a> to discover how we can make a difference for your next clinical trial!</em></p>]]></content:encoded> <wfw:commentRss>https://vial.com/blog/articles/castor-vs-vial-pros-and-cons/feed/</wfw:commentRss> <slash:comments>0</slash:comments> </item> <item> <title>Syneos vs Vial | Pros and Cons</title> <link>https://vial.com/blog/articles/syneos-vs-vial-pros-and-cons/</link> <comments>https://vial.com/blog/articles/syneos-vs-vial-pros-and-cons/#respond</comments> <dc:creator><![CDATA[Owen Allen]]></dc:creator> <pubDate>Wed, 24 Apr 2024 20:16:08 +0000</pubDate> <category><![CDATA[Articles]]></category> <guid isPermaLink="false">https://vial.com/?p=48780</guid> <description><![CDATA[Introduction In a landscape where many contract research organizations (CROs) are available, Vial is breaking through with its promise to deliver on its vision to empower scientists to cure all human diseases by reimagining clinical trials. The global, full-service Vial CRO stands out on multiple fronts, and we explore how it compares with a large […]]]></description> <content:encoded><![CDATA[<h2 class="wp-block-heading">Introduction</h2> <p>In a landscape where many <a href="https://vial.com/glossary/cro-contract-research-organization">contract research organizations</a> (CROs) are available, <a href="https://vial.com/">Vial</a> is breaking through with its promise to deliver on its vision to empower scientists to cure all human diseases by reimagining <a href="https://vial.com/glossary/clinical-trial">clinical trials</a>. The global, full-service Vial CRO stands out on multiple fronts, and we explore how it compares with a large leading CRO like <a href="https://www.syneoshealth.com/">Syneos Health</a>.</p> <h2 class="wp-block-heading">Therapeutic Area Coverage</h2> <p>Clinical trials are essential for developing new interventions and advancing patient care. The Vial CRO services encompass various therapeutic areas, including <a href="https://vial.com/glossary/oncology">oncology</a>, <a href="https://vial.com/glossary/dermatology">dermatology</a>, <a href="https://vial.com/glossary/ophthalmology">ophthalmology</a>, <a href="https://vial.com/glossary/gastroenterology">gastroenterology</a>, <a href="https://vial.com/glossary/neurology">neurology</a>, <a href="https://vial.com/glossary/cardiology">cardiology</a>, medical devices, rare diseases, and digital therapeutics (DTx). In addition to the areas above (aside from DTx), Syneos Health offers <a href="https://vial.com/glossary/biosimilars">biosimilars</a>, <a href="https://vial.com/glossary/cell-and-gene-therapies">cell and gene therapy</a>, endocrine and metabolic, <a href="https://vial.com/glossary/immunology">immunology</a> and inflammation, infectious diseases, pediatrics, respiratory, and women’s health services. Digital therapeutics is not explicitly listed as a therapeutic area of expertise for Syneos Health.</p> <h3 class="wp-block-heading">Oncology</h3> <p>Despite significant progress in <a href="https://vial.com/glossary/cancer">cancer</a> research, many patients face limited treatment options. CROs like Vial and Syneos Health are continuously innovating in the oncology space. The <a href="https://vial.com/cro/oncology">Vial Oncology CRO</a> offers sponsors solutions to support their research, including optimized study design, innovative patient recruitment strategies, and advanced data analytics. By partnering with Vial, sponsors conducting oncology clinical trials can accelerate getting innovative therapies to market. Syneos Health offers diverse and measurable experience across cancer treatment, from <a href="https://vial.com/glossary/immuno-oncology">immuno-oncology</a> and targeted therapies to novel and emerging therapies, including cell and gene therapies.</p> <h3 class="wp-block-heading">Dermatology</h3> <p><a href="https://vial.com/glossary/phase-i/">Phase I</a> Units are critical to advancing care and validating treatments. The <a href="https://vial.com/cro/dermatology">Vial Dermatology CRO</a> Phase I unit provides a unified full-service solution to early clinical development. ****Syneos Health has completed more than 200 dermatology trials for indications that include <a href="https://vial.com/glossary/psoriasis">psoriasis</a>, atopic dermatitis, <a href="https://vial.com/glossary/skin-cancer">skin cancer</a>, rare skin disorders, and skincare and medical aesthetic products.</p> <h3 class="wp-block-heading">Ophthalmology</h3> <p>The <a href="https://vial.com/cro/ophthalmology">Vial Ophthalmology CRO</a> understands the nuances of running ophthalmology trials and has formed a team of in-house ophthalmologists with decades of experience across anterior and <a href="https://vial.com/glossary/posterior-segment">posterior segments</a>. Syneos Health’s experience in ophthalmology clinical studies includes drug and device combinations, cell and gene therapies, orphan and rare diseases, inherited retinal diseases, pediatrics, and biosimilars.</p> <h3 class="wp-block-heading">Gastroenterology</h3> <p>Gastrointestinal (GI) diseases are a significant global health burden, increasing healthcare costs and decreasing productivity. The <a href="https://vial.com/cro/gastroenterology">Vial Gastroenterology CRO</a> offers sponsors full-service CRO solutions to support their GI research, from study design optimization to innovative patient recruitment strategies and advanced data analytics. The Syneos Health gastroenterology clinical study experience includes anal fissures, <a href="https://vial.com/glossary/celiac-disease">celiac disease</a>, colorectal disorders, diverticulitis, <a href="https://vial.com/glossary/gastroenteritis">gastroenteritis</a>, gastroesophageal reflux disease (GERD), hepatic cirrhosis/fibrosis, <a href="https://vial.com/glossary/inflammatory-bowel-disease-ibd">inflammatory bowel disease</a> (IBD), and <a href="https://vial.com/glossary/nonalcoholic-fatty-liver-disease-nafld">non-alcoholic fatty liver disease</a> (NAFLD)/<a href="https://vial.com/glossary/non-alcoholic-steatohepatitis-nash">non-alcoholic steatohepatitis</a> (NASH), among others.</p> <h3 class="wp-block-heading">Neurology</h3> <p>The <a href="https://vial.com/cro/neurology">Vial Neurology CRO</a> experienced CRO executive team works closely with Vial’s expert site operations team to continuously review execution strategies, closely monitor patient recruitment efforts, and mitigate key study risks for neurology clinical trials. The Neuroscience therapeutic area of expertise at Syneos Health includes analgesia, neurology, psychiatry, and clinical surveillance and training.</p> <h3 class="wp-block-heading">Cardiology</h3> <p>Cardiovascular diseases are a leading cause of global deaths and represent a significant healthcare burden. The <a href="https://vial.com/cro/cardiology">Vial Cardiology CRO</a> offers sponsors full-service solutions to support their cardiovascular research. Syneos Health has worked across the entire spectrum of cardiovascular indications, including <a href="https://vial.com/glossary/hypertension">hypertension</a>, <a href="https://vial.com/glossary/atrial-fibrillation-afib">atrial fibrillation</a>, <a href="https://vial.com/glossary/heart-failure-hf">heart failure</a>, ischemic heart disease, <a href="https://vial.com/glossary/thrombosis">thrombosis</a>, <a href="https://vial.com/glossary/dyslipidemia">dyslipidemia</a>, <a href="https://vial.com/glossary/diabetes-mellitus">diabetes</a>, and obesity.</p> <h3 class="wp-block-heading">Medical Devices</h3> <p>Medical devices can transform healthcare and improve patient outcomes; however, they require rigorous testing and evaluation to ensure their safety and <a href="https://vial.com/glossary/efficacy">efficacy</a>. With <a href="https://vial.com/cro/medical-device">Vial Medical Device CRO</a>’s best-in-class <a href="https://vial.com/glossary/clinops-clinical-operations">ClinOps</a> experts and tech platform, sponsors can conduct trials faster and more efficiently, leading to quicker regulatory approval and market access. Syneos Health’s medical device and diagnostics (MD&D) services include MD&D-specific capabilities that span the entire nonclinical and clinical development, regulatory, medical affairs, and commercial continuum to strategically accelerate the product development lifecycle.</p> <h3 class="wp-block-heading">Rare Diseases</h3> <p>Rare diseases affect a small proportion of the global population and often have few treatments. Rare diseases can be severe, life-threatening, and disabling, causing significant emotional and financial strain on affected individuals and their families. The <a href="https://vial.com/cro/rare-disease">Vial Rare Disease CRO</a> offers faster, more efficient clinical trials that allow scientists to develop effective interventions to improve patients’ quality of life, slow disease progression, or even cure the disease. Syneos Health helps companies develop rare disease therapies using novel and integrated approaches, including early engagement of key stakeholders and patient perspectives to accelerate products to market.</p> <h3 class="wp-block-heading">Digital Therapeutics</h3> <p>Digital therapeutics (DTx) are a new class of software-based therapeutic interventions for patients with chronic diseases and mental health conditions. The <a href="https://vial.com/cro/digital-therapeutics">Vial Digital Therapeutics CRO</a> offers sponsors a range of clinical trial solutions, including streamlined study design, optimized patient recruitment, and robust data analytics. With Vial’s expertise and support, DTx sponsors can bring their products to market faster and more efficiently, helping patients access the care they need. By harnessing the power of technology to develop and validate DTx, Vial enables scientists to pave the way for a new era of <a href="https://vial.com/glossary/personalized-medicine">personalized</a> and accessible healthcare. DTx is not explicitly listed as a therapeutic area of expertise for Syneos Health.</p> <h2 class="wp-block-heading">Small vs. Large CRO</h2> <p><a href="https://vial.com/blog/articles/the-benefits-of-working-with-a-small-cro-speed-agility-and-working-with-a-true-partner">Small CROs are flexible, adopt the latest tech, and can customize solutions</a> to meet the needs of sponsors, regardless of their size. Small CROs like Vial offer a high degree of specialization in specific disease areas and geographic focus.</p> <p>Smaller CROs also tend to have a much lower turnover of people compared to large CROs like Syneos Health, providing the stability and continuity sponsors need. For sponsors seeking a high-touch relationship, small CROs can offer close contact, good oversight, and a more personal touch.</p> <h2 class="wp-block-heading">Fixed-fee Pricing Structure</h2> <p><a href="https://vial.com/blog/articles/vial-cro-vs-top-5-large-cros">Unlike Vial, Syneos Health does not offer fixed-fee pricing</a>. At Vial, a fixed-fee pricing model applies to all contracts and helps sponsors stay on budget for the entirety of a project by avoiding costly unanticipated change orders. The <a href="https://vial.com/blog/articles/what-is-fixed-fee-pricing-and-how-does-it-benefit-sponsors">benefits of fixed-fee pricing include</a> transparency, <a href="https://vial.com/blog/articles/fixed-fee-pricing-and-the-importance-of-budget-predictability-in-clinical-research">predictability</a>, shared accountability on commitments, aligned incentives between CROs and sponsors, and sponsors having more control over where funds are spent.</p> <h2 class="wp-block-heading">Trial Start-up</h2> <p>Clinical trial start-up is a critical phase that can impact study timelines and costs if delayed. A recent study found that the average contract negotiation duration exceeded 100 days for industry-sponsored or investigator-initiated contracts, adversely impacting clinical trial start-up time and trial costs. An understanding of factors that affect start-up is essential to increase efficiency. With this knowledge, Vial has a streamlined site activation capability and <a href="https://vial.com/onboarding">cloud-based site start-up</a> that enables simple onboarding for sites in as few as 30 days. Cloud-based forms, uploads, signatures, and approvals allow real-time editing and a birds-eye view into site-by-site start-up statuses. <a href="https://vial.com/blog/articles/vial-cro-vs-top-5-large-cros">Syneos Health claims improved cycle times</a> but does not explicitly reference a 30-day site activation offering (or any timeline).</p> <h2 class="wp-block-heading">Technology and Tools</h2> <p>To remain relevant and offer clients a full range of value-added solutions, small CROs like Vial keep current on the latest technologies, techniques, and tools in <a href="https://vial.com/glossary/clinical-research">clinical research</a>. Small CROs with expertise and a focus in niche areas are well positioned to adopt the latest tech, identify opportunities for application, and provide insights on how tech may improve clinical trial performance. Vial offers powerful data capture and review in one connected system, including <a href="https://vial.com/glossary/electronic-source">electronic source</a> (<a href="https://vial.com/esource">eSource</a>), <a href="https://vial.com/glossary/epro-electronic-patient-reported-outcome">electronic patient-reported outcome</a> (<a href="https://vial.com/epro">ePRO</a>), and <a href="https://vial.com/glossary/edc">electronic data capture</a> (<a href="https://vial.com/edc">EDC</a>). <a href="https://vial.com/blog/articles/vial-cro-vs-top-5-large-cros">Syneos Health has multiple tech partners</a>, and their systems are spread out.</p> <h2 class="wp-block-heading">Reimagining Clinical Trials</h2> <p>Vial is a next-generation, tech-first CRO on a mission to disrupt the clinical trials industry to deliver faster, more efficient results at dramatically lower costs for sponsors. Vial is reimagining the nature of clinical trials to be more efficient and successfully deliver a superior CRO experience. By deploying technology at every step, Vial is driving efficiencies in speed and cost savings for innovative biotech companies of all sizes.</p> <p>Visit us <a href="https://vial.com/about-us/">here </a>and <a href="https://vial.com/contact-us">contact us</a> today!</p>]]></content:encoded> <wfw:commentRss>https://vial.com/blog/articles/syneos-vs-vial-pros-and-cons/feed/</wfw:commentRss> <slash:comments>0</slash:comments> </item> <item> <title>First in Human Episode #60 featuring Rachel Haines</title> <link>https://vial.com/blog/podcast/first-in-human-episode-60-featuring-rachel-haines/</link> <comments>https://vial.com/blog/podcast/first-in-human-episode-60-featuring-rachel-haines/#respond</comments> <dc:creator><![CDATA[Owen Allen]]></dc:creator> <pubDate>Tue, 23 Apr 2024 15:40:51 +0000</pubDate> <category><![CDATA[Podcast]]></category> <category><![CDATA[clinical trials]]></category> <category><![CDATA[CRO]]></category> <category><![CDATA[Drug Discovery]]></category> <guid isPermaLink="false">https://vial.com/?p=48776</guid> <description><![CDATA[Amy Del Medico: Hello everyone, I’m Amy Del Medico, VP of Therapeutic Strategy at Vial. I’m here today with Rachel Haynes from Rinri Therapeutics. Rachel, would you like to tell us a bit about yourself and Rinri?  Rachel Haines: Hi, Amy. I am the vice president of clinical development and clinical operations at Rinri Therapeutics. […]]]></description> <content:encoded><![CDATA[<p><strong>Amy Del Medico:</strong> Hello everyone, I’m Amy Del Medico, VP of Therapeutic Strategy at Vial. I’m here today with Rachel Haynes from Rinri Therapeutics. Rachel, would you like to tell us a bit about yourself and Rinri? </p> <p><strong>Rachel Haines:</strong> Hi, Amy. I am the vice president of clinical development and clinical operations at Rinri Therapeutics. We were a biotech that was established in 2018, based on some early data from Professor Marcello Revolta’s lab at the University of Sheffield, which demonstrated we could take otic neural progenitor cells, differentiate and expand them. These cells were capable of becoming mature auditory neurons. In his early work, we demonstrated auditory brainstem response recovery in a gerbil model of auditory neuropathy was possible. Rinri was founded to translate that into patient benefit. </p> <p><strong>Amy Del Medico:</strong> That’s fascinating. Thank you. What makes hearing loss such a significant global issue? How does Rinri aim to address this challenge through its research?</p> <p><strong>Rachel Haines:</strong> With hearing loss, the estimates of how many people are affected globally varies widely. But, it’s situated somewhere, probably conservatively, around 500 million people globally being affected. Any of us, once we get to middle age, notice that hearing does become more difficult, even just in social situations and when there’s lots of competing background noise.</p> <p>We’re not just thinking about hearing loss as something that affects people who are born deaf. But it really affects the majority of us, whether it’s ourselves directly, or people that we care for, or people that we love, throughout the whole life cycle. </p> <p>Not only does it affect so many people, but there’s a lot of long term consequences for hearing loss, for patients individually, but also the macroeconomic issues around hearing loss. Granted, it’s not life-limiting, but the impacts are massive in terms of people’s feeling of belonging, communicating, access to education, accessibility in the workplace, and having those meaningful relationships with people in a society that is, largely, an auditory world which we live in.</p> <p>Of course, for some individuals, the deaf community and sign language is their main mode of communication and we are absolutely not looking to solve a problem for those people for whom hearing loss is not a problem. It is a wonderful culture to be part of. But, there are people who want to be part of the hearing world because that’s the culture they grew up in. We do need to address this issue from those people who are looking for it.</p> <p>We also know, beyond being a significant issue, it’s a condition many people don’t seek treatment for. Currently, we have some great technology with hearing aids and cochlear implants which we have continued to develop in our field. But, these do not restore natural hearing and people are often very reticent to take them up.</p> <p>For whatever reason, there’s lots of stigma attached to them. At present we don’t have any licensed therapeutics for hearing or restoration of hearing. It’s something that Rinri and colleagues of ours in gene therapy have been working really hard at. At Rinri, in particular, we really believe in our approach to it because hearing loss is essentially a cellular problem. What Rinri is doing is offering a cellular solution for the cellular problem. We believe that cell therapy really is the future to treat these bigger problems of hearing loss.</p> <p><strong>Amy Del Medico:</strong> When you think about it, especially as we have an aging population, it would impact most of us in some way or another. It’s surprising that more therapeutics aren’t being developed. So, it’s really interesting to hear what you’re doing.</p> <p><strong>Rachel Haines:</strong> It is really interesting. So, in age-related hearing loss that you’re talking about, that’s what we call presbycusis, and it’s the target of the first product in our pipeline. We know that in the aging hearing model, it is the auditory neurons, which are degrading or dying. That precedes the hair cell loss. </p> <p>In hearing loss, you have two types of cells, which are important, which are the hair ,cells and the auditory neurons. When you are given a hearing aid it amplifies that sound. It makes it louder. If the hair cells are not working as well as they used to, there’s some fatigue there, or some have died off, the hearing aid is going to make that sound louder.</p> <p>That will be helpful, in terms of people with more mild or moderate hearing losses. But then once you have a more severe or profound loss, that’s where something like a cochlear implant might be [00:05:00] offered. The cochlear implant essentially replaces the hair cells and stimulates the auditory nerve directly. At present you can have a hearing aid which amplifies the sounds, or a cochlear implant, which replaces the hair cells.</p> <p>In both of those instances, if the problem is at the neural level, at the auditory nerve level, if you have populations of auditory neurons, which have died off or are dysfunctioning, neither of those are going to help. That’s what you see often, especially in an aging model where people may get a hearing aid, but they’re still really unsatisfied with using it socially because it’s not helping their speech and noise.</p> <p>It’s that neural function which is helping us do that speech and noise processing. And then equally, when you have a cochlear implant, you are replacing the hair cells, but you’re not treating any issue that might stem from the auditory neurons themselves. So it’s part of the solution. These are fantastic technologies, which have been life changing for many, but they don’t replace natural hearing. </p> <p>With that comes a very big discrepancy between outcomes of people who get hearing aids, and cochlear implants. There’s a lot of unexplained variation of how well people do with them. Also, people who do use them, (as I said) oftentimes find it very difficult to use them in situations with speech and noise, competing sound signals, and often report the big challenge of fatigue. It’s really difficult when you are listening with something less than your optimal natural hearing to do what is essentially a very complex task of listening to noise. It is very rare that we are actually not listening within a context of background noise.</p> <p><strong>Amy Del Medico:</strong> Thank you. For those that aren’t familiar, I wondered if you could elaborate on the landscape of hearing research today. Why it might be challenging to design clinical trials specifically for hearing related conditions. </p> <p><strong>Rachel Haines:</strong> The list of challenges is absolutely immense and very daunting, but extremely exciting. Also it’s a great time to be working in hearing. I remember being a postgrad student many years ago, hearing Professor Marcelo Revolta talk about some of his early work and thinking, “Wow, that’s science fiction!”</p> <p>It’s so exciting that it’s here now, and in the last three months, some colleagues of ours from gene therapy companies have been reporting some very early positive data for children with otoferlin deficiency. The gene therapy data is really encouraging that regenerative therapies for hearing are possible. They’re just there right in front of us. </p> <p>Gene therapy is going to be transformative for the patient population that’s being developed for these children with a monogenetic cause to their hearing loss. But, we know most instances of hearing loss are not monogenetic are actually acquired. For example, in the age related hearing loss and It’s not genetic factors that drive that alone. You’re going to have people who are too old to benefit from these gene therapies once they do develop a problem with hearing. That’s why we’re proposing a cellular solution for cellular hearing loss.</p> <p>What are the challenges in doing that? </p> <p>Many. </p> <p>First of all, it’s always hard being the first to do something. Second of all, one of the motivations that got me into clinical development and operations originally, coming from a background as a hearing scientist, was seeing in my field 20 years ago, we weren’t doing trials in hearing.</p> <p>Why weren’t we doing trials in hearing? Because most of our treatments were devices or interventions. The wealth of clinical trial methodology has come from a drug therapeutics background. So we don’t, even as of 2024, in our field, have tons of expertise about conducting trials.</p> <p>I’ve been absolutely privileged to be able, over the course of my career, to design and oversee the running of several hearing trials. But even for me, this is the first time I’m doing a trial, in an ATMP in this field because it hasn’t existed before. That’s absolutely amazing. Because we don’t have many trials, another challenge is outcomes. </p> <p>Even patient-reported outcome measures we may have in our field are designed to really capture more incremental changes that a hearing aid might be able to offer, a cochlear implant might be able to offer, not [00:10:00] some of the life-changing or very sensitive changes that a therapeutic could offer.</p> <p> A big challenge for us, in hearing, in terms of pushing clinical development forward, is we don’t even diagnose by etiology. We give somebody a hearing test, we measure how bad their hearing is, essentially, and decide what solution we’re going to offer them based on the magnitude of their hearing loss.</p> <p>We can’t even tell you whether somebody has hearing loss because of the hair cells or because of the auditory neurons. We don’t know. We don’t have those diagnostics, now. We just have an increasing amount of genetic data which is still a work in progress and then electrophysiological methods that are allowing us slowly to unpick this.</p> <p> Diagnostics is a big challenge for us. Another really simple thing: you’re giving somebody an advanced therapy, In our case we are going into people with profound hearing loss. So they will be getting a cochlear implant to replace the hair cells. They will be getting RinCell 1 at the same time as the cochlear implant to regenerate those auditory neurons.</p> <p><strong>Amy Del Medico:</strong> It’s really interesting what you were saying about diagnostics because it’s equivalent to giving somebody an eye test, isn’t it? And saying you’ve lost this much vision. We’re going to guess what treatment you need. I’d never really thought about it like that in terms of hearing loss. </p> <p>It’s really interesting. You’re obviously at the cutting edge, which is fascinating. I wondered how Rinri’s approach to generating cells for hearing restoration differs from traditional methods? What advantages might it offer in terms of efficacy and safety for patients? </p> <p><strong>Rachel Haines:</strong> When you are giving a hearing aid and amplifying sound, or giving a cochlear implant and electrically stimulating the auditory nerve. You’re not restoring natural hearing. A hearing aid has certain auditory processing limitations and a cochlear implant is essentially creating electric hearing. I don’t know if you’ve ever heard a simulation of a cochlear implant, a vocoded speech, really fascinating. But it’s not restoring normal hearing. </p> <p>What we are saying is, by regenerating auditory cells, we will be able to restore natural hearing. That is our end goal. That’s very different from the traditional methods, which aren’t a complete treatment. In terms of efficacy, we can hope and expect that by regenerating the cells that allow us to hear that cannot regenerate on their own in humans, we will be addressing the main complaints and challenges that patients with hearing loss face, even when they have aiding. It’s that fatigue. It’s the difficulty of being able to understand speech and noise, using communication, in those really social situations, that are so meaningful to us as humans as we connect.</p> <p>In terms of safety, we don’t have any therapeutics to compare it to. We can look at our surgical approach that we have developed at Rinri, and compare that to the standard cochlear implant surgery that has an excellent safety profile. We’ve done some real cutting edge research around developing this brand new surgical access, going through the round window into the internal auditory canal and getting the cells exactly in the place that they need to grow. </p> <p>That’s never been done before. But we’ve shown that this is safe. We’ve used synchrotron and micro ct imaging, as we’re doing that surgery scanning the temporal bones and showing how we are able to go in and not damage any of the internal structures like the facial nerve and the cochlear artery. </p> <p>We don’t feel this is any more risky than regular cochlear implantation surgery. But, we still have so much to learn. That’s really exciting, going into the first in human. It is one of the reasons that for our first in human trial, we’ve taken this approach to a randomized, open label trial. We don’t have safety data to compare it against. When we want to be evaluating the safety of what we’re doing, we want a really good data set of control participants to compare that against.</p> <p><strong>Amy Del Medico:</strong> That’s so interesting and very exciting stage to be at as well. Thank you. I just had one final question just to wrap things up. I wondered what sort of excites you the most about the future of the industry? Are there any sort of visions of the landscape that you think are coming up?</p> <p><strong>Rachel Haines:</strong> As a hearing scientist, by [00:15:00] heart, I’m so excited for all the clinical development work that we’ve done and how we’re going to keep building on that. What we can keep developing, in terms of our surgical access, our diagnostics, and our outcome measures is so fascinating scientifically, but also for how meaningful it is to patients. </p> <p>At the heart of Rinri and what we’ve been doing, is working, collaboratively with patients, families, and key opinion leaders across the globe to design, not just our trials, but that end product to be what people want it to be with the end goal in mind of reconnecting people to the auditory world. </p> <p>The clinical development that we have in the pipeline is just so exciting. I’m looking forward to us making meaningful contributions not just to developing this therapy, but for diagnostics of hearing, and, eventually, expanding our indication, for example, into monotherapies for people with more mild forms of hearing loss. We are on the precipice of something big, and it’s just an immense privilege to be part of it.</p> <p><strong>Amy Del Medico:</strong> Rachel, thank you very much for the discussion today. It was an absolute pleasure and really interesting to learn more about Rinri.</p> <p><strong>Rachel Haines:</strong> Thank you. It’s been great talking to you. I’m really excited to speak again someday about what we’ve learned from our first inhuman trial.</p> <p><strong>Amy Del Medico:</strong> Sounds good. We’d love to have you back.</p> <p><strong>Rachel Haines:</strong> Thank you so much, Amy.</p>]]></content:encoded> <wfw:commentRss>https://vial.com/blog/podcast/first-in-human-episode-60-featuring-rachel-haines/feed/</wfw:commentRss> <slash:comments>0</slash:comments> </item> <item> <title>Updates in the Ophthalmology Clinical Landscape</title> <link>https://vial.com/blog/articles/updates-in-ophthalmology-clinical-landscape/</link> <comments>https://vial.com/blog/articles/updates-in-ophthalmology-clinical-landscape/#respond</comments> <dc:creator><![CDATA[Owen Allen]]></dc:creator> <pubDate>Thu, 18 Apr 2024 18:46:35 +0000</pubDate> <category><![CDATA[Articles]]></category> <category><![CDATA[Clinical Research]]></category> <category><![CDATA[clinical trials]]></category> <category><![CDATA[CRO]]></category> <category><![CDATA[CROs]]></category> <category><![CDATA[Ophthalmology]]></category> <guid isPermaLink="false">https://vial.com/?p=48768</guid> <description><![CDATA[The field of ophthalmology is dedicated to preserving and restoring vision, recognizing its significance to human well-being. Yet, hereditary and age-related retinal diseases present challenges, often leading to progressive vision loss and potential blindness. Among these conditions, age-related macular degeneration (AMD) and diabetic retinopathy are of particular concern, with the global patient population expected to […]]]></description> <content:encoded><![CDATA[<p>The field of <a href="https://vial.com/glossary/ophthalmology/">ophthalmology</a> is dedicated to preserving and restoring vision, recognizing its significance to human well-being. Yet, hereditary and age-related retinal diseases present challenges, often leading to progressive vision loss and potential blindness. Among these conditions, age-related macular degeneration (AMD) and diabetic retinopathy are of particular concern, with the global patient population expected to exceed 400 million annually.</p> <p>The urgency for novel therapeutic approaches makes ophthalmology a rapidly growing domain of <a href="https://vial.com/glossary/clinical-research/">clinical research</a>. Market research indicates significant growth in the ophthalmic <a href="https://vial.com/glossary/clinical-trial/">clinical trials</a> market, driven by increased disease prevalence, demand for ocular treatments, and rising research funding. Specifically, the global ophthalmic clinical trials market, valued at USD 1.5 billion in 2022, anticipates steady growth at a compound annual growth rate (CAGR) of 6.6% from 2023 to 2030. Additionally, the ophthalmic drugs market, valued at USD 33.81 billion in 2022, is also expected to grow with a CAGR of 7.80% from 2023 to 2030.</p> <p>Dr. Stephen McLeod, M.D., CEO of the American Academy of Ophthalmology, discussed the evolving landscape of ophthalmology on a recent episode of <a href="https://www.realworldophthalmology.com/podcast">RWO: The Podcast</a>. He highlighted <a href="https://www.ophthalmologytimes.com/view/navigating-ophthalmology-s-changing-landscape-with-insights-from-stephen-mcleod">three pivotal forces reshaping the field</a>:</p> <ol><li>Advances in vision sciences research: Progress in treating conditions like AMD and dry eye disease, together with the potential of gene therapy for inherited retinal diseases, is enhancing patient care</li> <li>Focus on health equity: Tools like the IRIS registry enable ophthalmology to address health disparities, ensuring equitable vision outcomes for all patient populations</li> <li>Community engagement: Collaboration within the ophthalmic community strengthens advocacy efforts and supports provider wellness, addressing challenges like burnout and stress</li></ol> <p>This article explores recent developments in ophthalmology clinical trials.</p> <h2 class="wp-block-heading">The OPTIMIZE-2 Trial</h2> <p><a href="https://oculis.com/">Oculis</a>, a global biopharmaceutical company committed to preserving and enhancing vision worldwide, is at the forefront of innovation in eye care. One of its groundbreaking developments is OCS-01, a novel once-daily topical formulation of dexamethasone tailored for treating diabetic macular edema (DME) and enhancing outcomes post-cataract surgery.</p> <p>The OPTIMIZE-2 trial (<a href="https://clinicaltrials.gov/study/NCT06128369?cond=Ophthalmology&aggFilters=status:rec&page=3&rank=25">NCT06128369</a>) is currently underway to evaluate the potential of OCS-01 eye drops in managing inflammation and pain following cataract surgery. This <a href="https://vial.com/glossary/phase-iii/">Phase III</a> multi-center clinical trial, initiated on December 18, 2023, aims to enroll approximately 160 participants randomly assigned to receive either OCS-01, a dexamethasone ophthalmic suspension at a concentration of 1.5% (15 mg/mL), or a vehicle once daily. OCS-01 is administered as a single drop in the study eye once daily for 14 days, starting one day post-surgery.</p> <p>Primary outcome measures of OPTIMIZE-2 include evaluating OCS-01 efficacy in controlling inflammation, determined by the absence of anterior chamber cells on day 15 post-surgery, and assessing post-operative pain in cataract surgery subjects.</p> <p>OPTIMIZE-2 is open to individuals aged 18 and above, who are undergoing unilateral cataract surgery involving phacoemulsification and posterior chamber intraocular lens implantation in the study eye. Furthermore, participants must exhibit an anterior chamber cell score of at least 2 at the post-operative visit. Exclusions apply to individuals with known sensitivities or allergies to dexamethasone, corticosteroids, or any components of the study medication, those with monocular vision, and those showing signs of intraocular inflammation or reporting ocular pain above a specific threshold during pre-surgery assessments.</p> <p>The trial, sponsored by <a href="https://oculis.com/?_gl=1%2Aajroil%2A_ga_89PG7HJHSY%2AMTcxMjA2ODE2MC4xLjAuMTcxMjA2ODE2MC4wLjAuMA">Oculis</a>, has an estimated primary completion by July 2024 and overall completion by December 2024.</p> <h2 class="wp-block-heading">OCS-01 Achieves Primary Endpoints in Phase III OPTIMIZE Trial</h2> <p>In August 2023, Oculis <a href="https://investors.oculis.com/news-releases/news-release-details/ocs-01-first-investigational-eye-drop-front-and-back-eye-met">announced</a> positive top-line results from its OPTIMIZE trial with OCS-01 eye drops. This double-blind, placebo-controlled Phase III study included 241 patients randomized to receive once-daily OCS-01 eye drops or a vehicle for 14 days post-cataract surgery.</p> <p>The trial met both primary efficacy endpoints, showing a statistically significant decrease in inflammation absence on day 15 and pain absence on day 4 post-surgery.</p> <p>Furthermore, OCS-01 was well-tolerated, exhibiting a favorable safety profile with fewer ocular treatment emergent adverse events (TEAEs) reported compared to the vehicle group.</p> <p><em>“The results of the Phase 3 OPTIMIZE trial are exciting because once daily OCS-01 showed to be superior and highly potent in reducing inflammation and pain compared to vehicle with a favorable safety profile. This is significant for patients who have undergone cataract surgery, as they currently need to self-administer multiple daily doses of eye drops to alleviate inflammation and pain. The availability of a preservative-free treatment that requires only a once-daily eye drop could greatly benefit a large number of patients who undergo ocular surgeries worldwide,”</em> said Eric Donnenfeld, M.D., co-chair of Oculis’ Scientific Advisory Board.</p> <p>With these promising results, Oculis is advancing OCS-01 towards regulatory submission with the <a href="https://vial.com/glossary/food-and-drug-administration-fda/">U.S. Food and Drug Administration</a> (FDA), aiming to provide patients with an effective and convenient treatment option for post-operative inflammation and pain.</p> <p>Riad Sherif, MD, Chief Executive Officer of Oculis, stated: <em>“I am very pleased with the positive readout of OPTIMIZE. A once daily topical steroid eye drop has shown solid results in reduction of inflammation and pain and offers the potential of a truly simplified dosing regimen. We are on track to advance OCS-01 for inflammation and pain following ocular surgery towards an NDA submission with FDA. We now have positive Phase 3 top line results with OCS-01 preservative-free eye drops in treating front-of-the-eye inflammation and pain following ocular surgery, as well as Stage 1 Phase 3 results for back-of-the-eye diabetic macular edema (DME) from the DIAMOND program, opening for the first time ever new opportunities for topical eye drops to address highly unmet patient needs in both front- and back-of-the-eye indications.”</em></p> <h2 class="wp-block-heading">Expanding Clinical Applications</h2> <p>In addition to the OPTIMIZE and OPTIMIZE-2 trials, Oculis is conducting a Phase III double-masked, randomized, multicenter study to evaluate OCS-01 eye drops’ efficacy and safety in subjects with DME. The DIAMOND-2 trial (<a href="https://classic.clinicaltrials.gov/ct2/show/NCT06172257?term=OCS-01&draw=2&rank=3">NCT06172257</a>), initiated on December 15, 2023, counts with the collaboration of ICON plc.</p> <p>In May 2023, Oculis <a href="https://www.ophthalmologytimes.com/view/oculis-announces-positive-top-line-results-from-phase-3-trial-of-ocs-01-drops-for-diabetic-macular-edema">announced</a> positive top-line outcomes from stage 1 of its Phase III DIAMOND trial. Patients receiving OCS-01 showed a significant increase in mean best-corrected visual acuity scores, sustained up to week 12, compared to the vehicle group. Moreover, OCS-01 treatment resulted in a significant decrease in central subfield thickness, observed at week 6 and persisted through week 12. OCS-01 demonstrated a favorable safety profile, with no unexpected adverse events reported.</p> <p>“A topical agent has never demonstrated a positive result in DME,” commented Dr. Riad Sherif. “Now, OCS-01 has been validated in two different studies with consistent and repeated positive results. We remain focused on advancing with high priority the DIAMOND Phase 3 trial to Stage 2. This important milestone has the potential to bring us one step closer to providing the first treatment in the form of eye drops to patients with DME which is a devastating and blinding disease.”</p> <p>Additionally, a study sponsored by Quan Dong Nguyen, Stanford University, in collaboration with the Global Ophthalmic Research Center and Oculis, is investigating OCS-01’s performance in resolving fluid collection in the eye in patients with uveitis or those who have had eye surgery. The LEOPARD (<a href="https://classic.clinicaltrials.gov/ct2/show/NCT05608837?term=OCS-01&draw=2&rank=5">NCT05608837</a>) trial initiated on November 8, 2022, is estimated to complete by December 2024.</p> <p>Apart from OCS-01, Oculis is developing other drug candidates, including OCS-02, another topical eye drop for addressing dry eye disease and non-infectious anterior uveitis. OCS-05 represents a promising candidate for diseases like acute optic neuritis, as well as several neuro-ophthalmic conditions, with potential disease-modifying effects.</p> <h2 class="wp-block-heading">The SCOTS2 Trial</h2> <p>An ongoing interventional study initiated on January 2016 is evaluating the efficacy of autologous bone marrow derived stem cells (BMSCs) in treating retinal and optic nerve damage or diseases generally considered irreversible. The study, known as SCOTS2 (<a href="https://clinicaltrials.gov/study/NCT03011541?cond=Ophthalmology&aggFilters=status:rec&rank=7">NCT03011541</a>) is sponsored by <a href="https://www.mdstemcells.com/">MD Stem Cells</a>, a consultancy renowned for its expertise in conducting clinical trials and treatments utilizing BMSCs across diverse medical domains, including ophthalmology, <a href="https://vial.com/glossary/neurology/">neurology</a>, <a href="https://vial.com/glossary/alzheimers-disease/">Alzheimer’s disease</a>, other dementias, autism spectrum disorders, spinal cord injury, and anti-aging interventions. These cells demonstrate superior ability to navigate through the body and reach critical areas like the brain, eyes, and spinal cord, thereby enhancing their therapeutic potential.</p> <p>SCOTS2 involves administering BMSCs through various routes (retrobulbar, subtenon, intravitreal, intraocular, subretinal, and intravenous) to patients with conditions such as macular degeneration, retinitis pigmentosa, glaucoma-related optic nerve damage, among others. Patients will undergo comprehensive eye examinations and imaging over a 12-month follow-up period.</p> <p>The primary endpoint of SCOTS2 is the alteration in best-corrected visual acuity from pre-procedure to 12 months post-treatment, while secondary endpoints include changes in visual fields and optical coherence tomography parameters during the same timeframe.</p> <p>The study, projected to conclude by July 2025, anticipates enrolling approximately 500 participants. To be eligible for the study, participants must have objectively documented damage to the retina or optic nerve, either unlikely to improve or showing progressive deterioration, as well as specific visual acuity and field criteria. Those who have undergone previous ophthalmologic surgical treatment and have remained stable for at least three months post-treatment are eligible, as are individuals currently undergoing stable medical therapy for retinal or optic nerve diseases. Additionally, participants must be over the age of 18, medically stable, and must demonstrate the potential for improvement with BMSC treatment with minimal risk of harm from the procedure.</p> <p>Exclusion criteria include individuals unable to undergo adequate ophthalmologic evaluation, non-compliance with follow-up appointments, incapacity to provide informed consent, and those identified to be at significant risk to general health or visual function if they undergo the procedure.</p> <h2 class="wp-block-heading">Improvements in Patients With Dominant Optic Atrophy</h2> <p>In <a href="https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6987313/">Stem Cell Investigation</a>, SCOTS reported significant advancements in treating dominant optic atrophy (DOA), also known as Kjer’s optic neuropathy, the most prevalent inherited optic neuropathy.</p> <p>SCOTS employed minimal manipulation techniques to isolate BMSCs from aspirated autologous bone marrow using FDA-cleared medical devices. Six patients diagnosed with DOA received combinations of BMSCs via retrobulbar, subtenons, intravitreal, or subretinal placements, followed by intravenous injections, depending on their assigned study arm. The procedures exhibited no surgical complications. Importantly, of the six patients, five experienced significant visual improvements, with enhancements observed in both eyes in all cases. Among the treated eyes, 83.3% demonstrated gains in visual acuity.</p> <h2 class="wp-block-heading">The NOVA-1 Trial</h2> <p>Aerie Pharmaceuticals, a clinical-stage ophthalmic pharmaceutical company recently acquired by <a href="https://www.alcon.com/alcon-completes-acquisition-aerie">Alcon</a>, has initiated a Phase I/II study to assess the safety, efficacy, and durability of AR-14034 in treating neovascular AMD (nAMD). AR-14034 is a sustained-release (SR) retinal implant containing axitinib, a pan-VEGF inhibitor, formulated within a distinctive bio-erodible polymer blend using Aerie’s PRINT® technology.</p> <p>The NOVA-1 trial (<a href="https://clinicaltrials.gov/study/NCT05769153?cond=Ophthalmology&aggFilters=status:rec&page=3&rank=21">NCT05769153</a>), initiated on December 6, 2023, is structured into two stages:</p> <p>· Stage 1, a dose-escalation, open-label evaluation will be conducted over 48 weeks with approximately 10 subjects divided into two cohorts. Both cohorts will undergo assessments for safety and preliminary treatment effects</p> <p>· Stage 2 consists of a 56-week double-masked, active comparator, randomized, parallel group evaluation comparing AR-14034 SR with aflibercept. Approximately 130 subjects will be enrolled and randomized to different treatment groups. Participants will be assessed for safety and treatment effects over 56 weeks, followed by a 16-week open-label extension phase</p> <p>The primary outcome measure of NOVA-1 is the mean change from baseline in early treatment diabetic retinopathy study best corrected visual acuity at specified visits. Secondary outcome measures include additional assessments of visual acuity and central subfield thickness using spectral domain optical coherence tomography imaging.</p> <p>Key inclusion criteria include having an active choroidal neovascularization lesion secondary to AMD and specific visual acuity requirements. Exclusion criteria include certain ocular conditions, recent anti-VEGF treatment, uncontrolled glaucoma, and pregnancy.</p> <h2 class="wp-block-heading">Promising Findings in Preclinical Models</h2> <p>Recent findings published in <a href="https://iovs.arvojournals.org/article.aspx?articleid=2787318">Investigative Ophthalmology & Visual Science</a> support the potential of AR-14034 SR implant as a long-term treatment option for nAMD and DME, offering hope for improved therapeutic outcomes with fewer intravitreal injections.</p> <p>In the study, conducted on both rabbits and miniature swine, AR-14034 SR implants were administered intravitreally. Control groups consisted of either placebo-injected or naïve animals. The efficacy of the implant was assessed by inducing retinal blood vessel leakage with VEGF165, followed by quantification using fluorescein angiography.</p> <p>In rabbits, all dose levels of AR-14034 SR implant showed significant reductions in retinal vessel leakage compared to controls at 1- and 2-month intervals. Notably, a single dose level demonstrated sustained efficacy up to 12 months. Similarly, in miniature swine, both implant dose levels significantly reduced retinal vessel leakage at day 41.</p> <h2 class="wp-block-heading">Vial, Your Tech-Enabled Ophthalmology Partner</h2> <p>While emerging treatments offer hope, conducting ophthalmology clinical trials can be challenging. Collaboration between sponsors and <a href="https://vial.com/glossary/cro-contract-research-organization/">contract research organizations</a> (CROs) specializing in ophthalmology contribute significantly to the success of clinical trials. <a href="https://vial.com/blog/articles/what-is-a-cro/">CROs</a> offer expertise in trial design, ocular safety monitoring, patient population selection, and efficient data management.</p> <p>[Vial](<a href="https://vial.com/?)">https://vial.com/?)</a>, a next-generation, full-service CRO, is evolutionizing clinical trials to deliver faster, better, and more cost-effective results for biotech and biopharma sponsors. Our [ophthalmology CRO](<a href="https://vial.com/cro/ophthalmology/?)">https://vial.com/cro/ophthalmology/?)</a> team has extensive therapeutic knowledge to support all stages of ophthalmology drug development and clinical research.</p> <p><strong>To learn how Vial’s CRO can support your ophthalmology clinical trial, please visit our website or <em><a href="http://www.vial.com/contact-us">get in touch with a Vial representative today</a>!</em></strong></p>]]></content:encoded> <wfw:commentRss>https://vial.com/blog/articles/updates-in-ophthalmology-clinical-landscape/feed/</wfw:commentRss> <slash:comments>0</slash:comments> </item> </channel></rss> If you would like to create a banner that links to this page (i.e. this validation result), do the following:
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