Empowered Patient Podcast

Michael McFadden is the CEO of Alpha Cognition, a biopharmaceutical company focused on developing treatments for brain diseases such as Alzheimer's, ALS, and traumatic brain injury. Their drug ZUNVEYL, recently approved by the FDA, is an oral treatment for mild to moderate Alzheimer's that improves symptoms such as memory loss, mental processing speed, and mood changes. A key feature of the drug is fewer gastrointestinal side effects compared to other Alzheimer's drugs coming onto the market. Michael explains, "ZUNVEYL is a class of medications known as acetylcholinesterase inhibitors. It's the second oral compound approved by the FDA to treat Alzheimer's disease in the last decade. So, it is a momentous accomplishment by our company, and it is an accomplishment that will serve patients and physicians well. It's designed to treat symptoms of mild to moderate Alzheimer's disease that many patients suffer from. These are things like memory loss, mental health processing speed, ability to solve problems or remember short-term and long-term things. Also, it affects or allows patients to manage changes in mood or personality that often accompany Alzheimer's disease. So, these are drugs that are used very commonly to treat Alzheimer's disease. We think this drug will have a lot of utility in helping patients and offering physicians a new choice." "About 55% of patients who take a drug for Alzheimer's disease will have some side effect that's so significant they have to stop taking their medicine. So side effects are a primary reason that patients cannot stay on their therapy. The side effects that are most common are GI-related side effects, GI side effects, diarrhea, nausea, or vomiting. We are excited about our compound because we believe if you can't tolerate a medicine, the medicine certainly can't be efficacious for the patients." #AlphaCognition #Alzheimers #AlzheimersDisease #AlzheimersDrug #ZUNVEYL #Dementia alphacognition.com Download the transcript here

George Pappas, CEO of Intraprise Health, works with various healthcare organizations across the healthcare industry and healthcare ecosystem to help address cybersecurity risks. While recent cyber attacks have raised awareness about the need for better cybersecurity measures, the complexity of healthcare IT systems creates significant challenges for comprehensive risk management. Proactive measures, including employee training, enhanced identity confirmation, and limiting access, are critical to mitigating the risk of cyber attacks. George explains, "The net situation is that these large technology companies that serve healthcare and hospital systems and large medical practices change are clearinghouses, revenue cycle managers, and manage real-time price transparency. What medication management companies manage is so complicated, and they have so many ways that something can be attacked. As you mentioned at the beginning, the attackers are getting very clever and sharing a lot of their methods, so you have to do a comprehensive continuous review of your entire cybersecurity posture. Because if you don't, there are invariably going to be challenges and small things that are small at the time that end up not being small and being the way that an attacker got in." "And in the case of Change, it was a multifactor authentication problem accessing a certain system through a certain technology stack that was rather old. But these things exist in companies this size. They have tens of thousands of machines and have accumulated different companies they acquired over time with different technology systems that don't all work together. So, the vulnerabilities are there, and they require comprehensive risk management and some candidly, more investment than we've seen to address fully." "I'll give you another example. So maybe it helps your listeners place this a little better. If you think about someone who has a home and is trying to protect their home from all the various threats or things that can happen. Well, their home insurance company requires them to have a smoke detector and carbon monoxide sensor. They get a credit on their policy. If they have a burglar alarm system, they might have a ring doorbell camera or any number of things like that. What you see across the industry is these large hospital systems, small hospital systems, doctor's offices, and they're doing those basic things. But here's the issue. In that same house that I mentioned, you could have 30 windows, the roof might be a little old, you might have a ground floor, and a door that has glass could be easily broken if someone tried to break in." #IntrapriseHealth #HealthcareCybersecurity #PatientDataProtection #CyberThreats #HITRUSTCompliance #RiskManagement #DataPrivacy intraprisehealth.com Download the transcript here

Ketan Mehta, Founder and CEO of Tris Pharma, is focused on developing non-addictive pain medication. Their lead drug candidate is designed to be an effective pain drug without the respiratory-depressive and addictive side effects of traditional opioid drugs. This drug works by targeting the nociceptin receptor and has also shown promise to help curb cravings and dependence on opioids. The goal is to provide doctors and patients with a non-opioid pain medication option that can help reduce reliance on highly addictive prescription opioids. Ketan explains, "Tris is focused on neuroscience-based products, especially CNS, at a high level. More specifically, we focused on ADHD and neurodevelopmental disorders and the products that treat them. And also, more recently, we have been more active in the pain space." "We're developing a drug, which is currently an investigational drug in Phase three clinical trials. It's called cebranopadol. And what we have seen is that it has an efficacy just on par with your typical traditional opioid. It's highly effective from what we have seen in more than 2,000 patients to date. But also, what we're seeing thus far is that there is no indication that it creates dependency. You can just taper off the patients, meaning you don't have to give lesser dose even after pain has subsided. And it does not give you the level of respiratory depression or failure that we see with the traditional opioid." "Right now, we're conducting our first trials in acute pain, and then we will look at any kind of moderate to severe pain. Essentially like chronic pain, lower back pain, for example, the pain in arthritis, cancer pain, other disease conditions like fibromyalgia, or just any kind of injury that gives you pretty much any nociceptive pain that one would encounter, we expect, like diabetic peripheral neuropathy and so forth." #TrisPharma #PainManagement #ClinicalTrial #Innovation #PainTherapy #Cebranopadol trispharma.com Download the transcript here

Beth Houck, Chief Executive Officer at SonarMD, works with payers and physician practices to provide proactive, value-based care for patients with chronic GI conditions. They use a digital platform to monitor self-reported patient symptoms and enable physicians to identify those patients that need attention. This approach aims to improve patient outcomes and reduce emergency room visits and hospitalizations. Beth explains, "SonarMD manages patients with chronic gut health conditions, and specifically, we started with something called inflammatory bowel disease, which is the encompassing word for patients with Crohn's and ulcerative colitis. We develop a value-based contract with a payer, so somebody at risk for the cost of care of a patient, and then we bring that cost of care program to a physician's office. In collaboration with that physician, we manage a population of patients to have better outcomes for their chronic GI conditions." "We just talk about this as a shift to proactive care from reactive care. So rather than the loudest patients being seen, we will have patients that we surface as the most important patients for them to see. So we reach out to our patients and do it very lightly with a text message or email. We tell them a little bit about the program. We talk to them about how we're doing this on behalf of their physician practice and in collaboration with them. So this isn't something that's being forced on you by a payer. We ping those patients, which is the term we use on a regular basis, to collect their reported outcomes or the symptoms they're having with their disease." #SonarMD #DigitalHealth #Healthcare #VBC #ValuebasedCare #GI #IBD SonarMD.com Download the transcript here

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