Empowered Patient Podcast

Dr. Chris Bouton, the Senior VP and Head of Artificial Intelligence at Certara, is exploring applying generative AI to drug discovery and development to improve efficiency and identify novel therapeutics across various use cases. The focus is on developing specialized secure AI models to integrate internal data with the Certara platform. Generative AI is particularly useful in areas with high specificity, such as antibody design and mRNA therapeutics, where modeling the underlying biology is crucial.  Chris explains, "Certara helps pharmaceutical and biotech companies throughout their process, from early basic research to regulatory filing and beyond. And we're quite a unique company in that we are not developing our own drugs but instead helping companies develop their drugs. And it's something like over 90% of all FDA approvals are projects that, in some way, shape, or form, Certara has helped with." "Our philosophy is to start with the use case. Generative AI is an incredible novel technology. There are many different ways to apply it, but we're interested in how to turbocharge or otherwise ameliorate and create efficiencies in a wide range of the use cases that Certara works on. What we're doing is taking a look at each of those kinds of use cases, trying to identify whether generative AI is useful in those use cases. Then, build out technology-driven solutions that are a combination of gen AI plus services, plus data, plus other software for each of those use cases." #Certara #GenerativeAI #HealthAI #DrugDiscovery #DrugDevelopment certara.com Download the transcript here  

David Happel is President and CEO of Sagimet Biosciences, a company developing drugs to treat metabolic diseases by targeting the underlying cause of fat accumulation in the liver. The drug denifanstat limits the activity of fatty acid synthase (FASN), an enzyme that regulates fat accumulation. This approach is a fat inhibitor, not a fat burner, the focus of other MASH drugs.  David explains, "Sagimet was founded back in 2007 as a private company with a group of medicinal chemists that had a particular target in mind, and this particular target was called fatty acid synthase, or FASN. FASN is a rather ubiquitous enzyme throughout the body that functions as a regulating enzyme in many organs. When FASN becomes overactive or overexpressed, it sort of throws things out of balance and, in particular, certain cardiometabolic diseases, such as MASH and in the liver. So, this group of chemists, in order to resolve this overactivity, developed a portfolio of FASN inhibitors, led by our lead program denifanstat, that seek to target the underlying cause of MASH and, therefore, really work to limit the activity of FASN, try to normalize it essentially is what we do." "For us and this particular molecule, it offers a differentiated approach to treating the disease. Our molecule, denifanstat, is the only fat synthesis inhibitor in development for MASH, making it rather unique. All the rest of the molecules are fat burners, fat oxidizers, and fat mobilizers, including GLPs, FGF, and THR-beta. They all function by trying to burn fat peripherally and in the liver to a degree. They rely on that fat-burning mechanism to translate into downstream reductions in inflammation and fibrosis, which is important to treating MASH, but they’ve had varying degrees of success. Our molecule targets each pathway independently: fat, inflammation, and fibrosis, and has had a rather pronounced treatment effect as a result."  #SagimetBiosciences #MASH #FattyLiverDisease #FASN #FASNInhibitor #Fibrosis #Obesity #NASH #GLP1Agonists sagimet.com Download the transcript here

Dr. Gavin Magaha, Senior Director for External Affairs and Policy at Kalderos, describes the 340B drug discount program created in 1992 to help safety net healthcare providers obtain discounted drugs. Over time, this program has grown in complexity, and issues have arisen around duplicate discounts where Medicaid rebates are involved. The current 340B system makes it difficult for stakeholders to share data and lacks transparency. The Kalderos platform incorporates technology and AI and ML tools to address data sharing, validate claims, and prevent duplicate discounts.  Gavin explains, "Our platform primarily helps manufacturers. However, through the activities that we provide service for, we end up helping all stakeholders in a compliant manner or be more compliant with several different drug discount programs, primarily the intersection of the 340B and the Medicaid Drug Rebate programs. We work to help the states, the covered entities in the program, and the manufacturers. All come to a resolution of what's happening out there when you start looking at the data that's going through." "Top of mind right now that you're hearing in the marketplace around 340B is the concept of who is a patient of a covered entity, what does it mean to be a patient of, whether it's a hospital or a federally qualified health center. The 340B statute is silent on how it defines who a patient is. A lot of the outpatient services that are taking place with hospitals are growing and reaching out into the communities to create more access to healthcare." "And then you see this footprint of the hospitals growing and taking care of folks in the communities. And then how do you figure out who is a patient of a hospital, and are you a patient for just that one instance of care, or are you a patient of the hospital now in perpetuity? The question becomes, who's eligible for these 340B drugs? The statute, which again came around in the ’90s, doesn't really define who a patient is. It puts a burden and challenge out there for almost all stakeholders to comply with this policy." #Kalderos #DrugDiscountManagement #340BDiscount #MedicaidDrugRebateProgram #DuplicativeClaims #CoveredEntities #NoncompliantDrugDiscounts #SafetynetProviders #DrugManufacturers Kalderos.com Download the transcript here    

Chris Hart, Co-Founder, CEO, and President of Creyon Bio, is focused on engineering drugs rather than just discovering them through serendipity and traditional methods.  Creyon is developing oligonucleotide medicines for rare and common diseases by applying AI and big data models of the molecular and genetic basis of diseases to develop safe and effective drugs more quickly.  Chris elaborates, "When we think about drug engineering versus drug discovery, we’re thinking about how we take this process that currently requires us to do trial-and-error identification of molecules that may be beneficial in the clinic to a place where we can say, can I build the right machinery, the right data, the right models, the right processes to go in and understand if I want to make this modulation within a cell, here’s the molecule that will do it and I can have confidence that it will be safe." "In terms of drug engineering and the type of drug engineering that Creyon does, what made starting Creyon the right thing to do and why it’s the right time and the right time to do it is that the computing power does exist to crunch large numbers and do the analysis we need to do to build the right models. But what didn’t exist for us, and I don’t think it exists broadly in the industry, is the right data. So a major focus of what Creyon has been doing for the last four years has been on how we create the right data in an efficient way such that we can engineer drugs to have the properties we need them to have as drugs, which is to say safe and effective and not just bioactive." "Our focus is oligonucleotide medicines, which generally are antisense oligos, siRNAs, and aptamers. This is a super-modality we often think about. What characterizes them is their polymeric nucleic acids that are chemically synthesized, and that’s our primary focus. The indications we’re going after are the upside of the medicines we make: they don’t constrain themselves naturally to a therapeutic area. We can think broadly about whether or not it’s an immunological disease or a neurological disease. We can use these medicines for any type of disease, and we can use them across the board regardless of whether it’s a rare or a common disease." #CreyonBio #AIinHealthcare #BiotechInnovation #DrugDiscovery #DrugDevelopment #DrugEngineering #PersonalizedMedicine #PrecisionMedicine #RareDiseases creyonbio.com Download the transcript here

Krystyna Houser is the Founder of mBIOTA Labs, a company that develops effective and tolerable elemental diets and medical foods for treating gastrointestinal disorders such as IBS, IBD, SIBO, IMO, and Crohn's disease. While short-term liquid elemental diets have been used in the past to treat GI conditions, the formulation has been very unpalatable, resulting in high patient non-compliance. The mBIOTA clinical trials have shown the elemental diet to be more effective than antibiotics in allowing the gut to recover.  Krystyna explains, "After battling my own GI issues and successfully treating them through a combination of an elemental diet and a dietary protocol called low-fermentation eating, I became a believer in the power of functional nutrition and built mBIOTA Labs to bring these solutions along with taste and a pleasant experience to millions of people suffering with GI conditions." "Elemental diets have been around for over 50 years. And through hundreds of clinical studies, elemental diets have been proven highly effective at managing various GI conditions. But until now, physicians were unable to employ them in treatment for fear of noncompliance due to their horrible taste. mBIOTA Elemental changes the game with its truly palatable formula. An elemental diet is a short-term liquid diet that provides comprehensive nutrition in its purest, most elemental form. It's composed of proteins, fats, and carbohydrates." "Our formula is designed as a two- to four-week course of orally ingested therapy. Once ingested, it is easily absorbed into the first two to four feet of the small intestine with almost no digestive effort, which allows the gut to reset, recover, and starve off excess bacteria, causing distress and inflammation. Because the diet is so balanced, it is much easier and safer to follow than a juice cleanse or other diets. And after a few days or so, people feel fantastic on mBIOTA and crave it." #mBIOTA #SIBO #GIHealth #MedicalFood #ElementalDiets #ClinicalTrial  mbiota.com Download the transcript here

Dr. Ann Beliën, Chief Executive Officer and Founder at Rejuvenate Biomed, is addressing the underlying biology of aging including mechanisms like mitochondrial health, cell-cell communication and inflammation in order to promote healthy aging. Taking a combination therapy approach, Rejuvenate is using AI-enable drug discovery to address multiple pathways that are involved in aging. Their lead drug candidate is a treatment for sarcopenia with plans to assess impact on other age-related biomarkers. Ann explains, "The biology of aging indeed is being defined by 12 hallmarks. These 12 hallmarks are pathways where you see that the functioning is deteriorating. One of them is mitochondrial health. So, the mitochondria are there to create energy in your cells to make sure that they work to an optimal base. That’s a very important element that we need to address." "Another very important one for us is cell-cell communication. So, for instance, in sarcopenia, one of the diseases we study, it’s very important that your muscle continues to be strong and that you don’t lose too much strength during aging. Now, for the muscle to be able to do that, it’s not only important that the muscle has enough energy but that there is also a crosstalk between the neurons that signal to your muscle that it has to contract. So, that cell-cell communication is also very important." "Within Rejuvenate Biomed, when we develop medication for age-related diseases, we start from the hallmarks of aging. We have an AI-enabled in silico system that can map compounds that have existed for quite a while, for which we have checked if they’re safe for an older population and also for chronic application. That information about these compounds, how they exactly work, goes into our system. The system sees if now these compounds theoretically have the ability to have an impact on the hallmarks of aging. If this is the case, the system will predict a combination that can allow you to impact as many different elements as possible. With that combination, we then check in worms, little worms, so C. elegans, to check on health span."   #DrugDiscovery #Aging #AgeRelatedDiseases #AI #ArtificialIntelligence #HealthyAging #Longevity rejuvenatebiomed.com Download the transcript here

Eric Olson, COO and Founder of BetterWay™ by Babson Diagnostics™, reports that traditional blood testing is often seen as inconvenient and unpleasant, causing 40% of people to avoid needed blood tests. Babson has developed a more patient-friendly approach to blood collection using a device that fits on the finger and requires a minimum amount of blood. The BetterWay service allows patients or doctors to order tests where blood is drawn at a local pharmacy and samples analyzed at a central lab, providing a quick turnaround with understandable test results.  Eric explains, "Blood testing is something that most people are very familiar with. It’s a routine part of medical care. Every time you go in for your annual visit or if you’re monitoring a disease, maybe a chronic disease, you might get tested more frequently than once a year. The experience is something that a lot of people go through, which consists of having an appointment, going into some sort of patient service center, and getting blood drawn by a professional phlebotomist. They put a needle in your vein and use tubes to pull the blood out of the vein. They fill up multiple tubes of blood that get sent to a high-throughput laboratory that looks a bit like a factory for testing." "If you go to BetterWay.com, you can see the full menu and the prices of all the tests on that menu, so there are no surprise bills. Insurance, FSA, and HSA are accepted. However, the cash pay prices are often lower than many people pay, even with insurance, due to copays and deductibles, which are very high for many people." "So once you’ve got an order, you go to your local retail pharmacy to get your blood collected. We’re available in pharmacies in Austin today, on all sides of Austin, and expanding beyond Austin soon. Once you arrive at the pharmacy, you tell them that you’re there for the BetterWay test. They’ll collect a pea-sized blood sample from your fingertip after putting your hand on a hand warmer. And that enables them to collect either one or two tubes of blood, depending on the tests you’ve ordered, instead of large vials of blood from a needle in your vein." #BetterWay #BabsonDiagnostics #BloodTesting #HealthEcosystem #MedicalDeviceDevelopments BetterWay.com Download the transcript here

Marylyn Rigby, President and CEO of Bioceptive, focuses on improving women's health technologies. Bioceptive has developed a cervical suction retractor as an alternative to the 200-year-old cervical tenaculum device used to insert IUDs and perform biopsies and other intrauterine procedures. While current procedures are extremely painful, there has been a lack of innovation in understanding women's pain because women are often described as hysterical, with little clinical data to get to the source of the pain.   Marylyn explains, "I think that also has to do with what modern medicine is based on, which is a man’s body and not a woman’s body. And because of that, it’s kind of interesting to think that a man could ever possibly understand what it would feel like to have their cervix, which they don’t even have, be pierced, essentially. It is like having your ears pierced and the traumatic experience that it causes because this is done again without any type of anesthetic. So, one of the biggest issues that we see right now, regardless of what your political stance on pro-life or pro-choice, is certainly that women need better options for contraception that are more effective." "Science has indicated that the IUD is the most effective form of contraception and that is because of its efficacy, which is greater than 99%. You’re not looking to remember to take a pill. You’re not looking, you’re not having to have weight gain by having something inserted in your arm. It’s a one-stop shop that is incredibly effective and cost-effective. However, the number one reason that women do not get IUDs more often is because of the trauma and the pain that is associated with the tenaculum, which is required to insert the IUD." #WomensHealth #Gynecology #FemTech #ReproductiveRights #ReproductiveHealth #WomensRights bioceptive.com Download the transcript here

Greg Sturmer, Co-Founder and CEO of Elysium Therapeutics, a company with expertise in reducing pain and addressing opioid use disorder and fatal overdose from fentanyl. Elysium has developed O2P, Oral Overdose Protection, a technology that aims to control the impact of opioids and reduce the risk of use disorder or a fatal overdose. SOOPR is a rescue agent designed specifically for overdoses from synthetic opioids, particularly fentanyl, which can last longer and be more potent than traditional opioids. Greg explains, "Anytime there’s an epidemic, you want to control exposure. The tricky bit in the area of pain management and the use of opioids is how do you control exposure without affecting patient care. We’ve come up with a technology that maintains the unmatched efficacy of the opioids. But when somebody takes too many pills, and we’ve actually shown this in our human study, we can reduce the exposure because we use this proprietary molecular delivery system that uses the body’s natural digestive process to control how much of the opioid gets released. That’s how we’re addressing the front end of the issue, where somebody is prescribed an opioid." "But sadly, what happens today is several individuals progress along the road to opioid use disorder, and they end up using illicit drugs. That’s where our second program, SOOPR, comes into play. As you know, our streets in the US have been flooded with fentanyl. Fentanyl is a highly potent opioid. The cartels have gotten very wise in how to expand their market. They introduced fentanyl-laced pills because a lot more people are willing to take pills rather than inject. Well, that changed the game dramatically because when somebody overdoses orally on fentanyl, that puts them at risk of respiratory depression for hours where they stop breathing." "Existing rescue agents like Narcan and Opvee weren’t designed for oral synthetic opioids and overdoses of those drugs. ‎SOOPR, we believe, is the first rescue agent designed specifically for synthetic opioids. What it does is it outlasts that synthetic opioid danger zone, blocking the mu-opioid receptors and preventing that respiratory depression or rescuing somebody who’s already in respiratory depression." #ElysiumTherapeutics #Opioids #OpioidAbuse #SyntheticOpioids #OpioidUseDisorder #PainManagement #SaferMedicines Elysiumrx.com Download the transcript here

Dr. Boris Reznik, Chairman and CEO of Immorta Bio, was founded to explore longevity and life extension technologies inspired by the discovery that aged cells can be reprogrammed to become younger ones. The company has a cellular rejuvenation platform that uses the patient's cells and senolytic immunotherapy to help the immune system target and remove senescent cells. These approaches aim to leverage the body's natural mechanism for maintaining health and extending the healthspan, not just lifespan. Boris explains, "First of all, let me just touch upon aging because aging perpetually happens on a cellular level from an early age because trillions of mutations occur in our cells every single day. But the young body’s defense mechanism, such as the immune system and stem cells, effectively addresses those issues and keeps young people healthy. So everything we do is associated with the approach where we try to use young bodies’ mechanisms in dealing with all diseases and all issues associated with aging."   "As I said, young bodies have mechanisms, but all mechanisms which body uses, they have a tendency to deteriorate with age, and it starts all on cellular level. They progress from cells to tissues, from tissues to organs, from organs to systems, and, eventually, all of that results in a disease. And those diseases are what we call diseases of aging. So, actually, the disease of aging is just a specific stage of aging. So, what we created and what we focus on are the two fundamental areas of longevity technologies."  #ImmortaBio #Longevity #StemCells #Cancer #Aging #Rejuvenation  immortabio.com Download the transcript here