Empowered Patient Podcast

John Mulcahy, President and CEO of SiteOne Therapeutics, is developing new therapies to treat hypersensitivity disorders. Their approach focuses on targeting specific voltage-gated sodium channels. Their drug candidates are designed to treat acute pain, as well as hypersensitivity developed as a result of chronic pain. They have recently received an NIH HEAL grant to explore their drug candidate that targets one subtype of the sodium channel as a non-opioid therapeutic potential for treating different pain conditions. John explains, "At SiteOne, we're focused on a family of targets known as the voltage-gated sodium ion channels. These are proteins, ion channels, that are involved in transmitting pain signals from the site of an injury, like earlier we said, the skin or a joint, to the central nervous system, so to your spinal cord, to your brain. At SiteOne, we're basically focused on drugs that potentially dampen those signals. It kind of tunes down that pain signal before it ever reaches the central nervous system. So it's a very different approach compared to either the NSAIDs or the opioids." "We're very excited to partner with NIH and the National Institute on Drug Abuse on this program. A little bit of background on the HEAL Initiative. So HEAL stands for Helping End Addiction Long-term, and it's a major federal initiative with multiple agencies involved. The goal is to aggressively advance scientific solutions to the opioid crisis." #SiteOneTherapeutics #OpioidCrisis #Opioids #NonOpioidMedications #Pain #PainManagement #HEALInitiative siteonetherapeutics.com Download the transcript here

Dr. Ahmed Hamdy, CEO and Co-Founder of Vincerx, is focused on targeting a specific antigen found on cancer cells. With a unique enzyme, it is effective with solid tumors and hematological malignancies, releasing a kinesin spindle protein inhibitor that inhibits the division of cancer cells. Their first bioconjugate VIP236 targets a specific molecule expressed on several metastatic tumors. Ahmed explains, "At Vincerx, we have a very exciting pipeline that's designed to solve a lot of problems with the current treatments for cancer therapies. In today's world, cancer continues growing exponentially. And thankfully, there are a lot of treatments out there for different types of cancer. Yet, the current treatments come with quite a bit of morbidities. Throughout my career, the morbidity of medicine has been something that I've always been concerned about, especially for patients and their caregivers. At Vincerx, we have very exciting, unique types of treatments that can be paradigm-shifting from a safety perspective and efficacy perspective." "For solid tumors, we have a compound that's currently in dose escalation phase 1 trial that is designed for advanced metastatic tumors, where we target a specific antigen that is found on the cancer cells themselves. And with a unique enzyme, it cleaves what we're describing as a warhead or the substance that can kill the cancer cell, which is an optimized camptothecin. And the word optimize means it's a well-known topoisomerase inhibitor designed to allow for very rapid permeability or intake by the cancer cell, with very low pumping out." #VincerxPharma #VIP236 #VIP943 #ADCs #Bioconjugates #Cancer #SolidTumors #Biotech #PrecisionMedicine Vincerx.com Download the transcript here

Nasha Fitter, CEO and Co-Founder of the FOXG1 Research Foundation and Vice President of Rare and Neurological Diseases at Ciitizen. Nasha describes the characteristics of the ultra-rare disease FOXG1 Syndrome and the global network that has been built to gain a better understanding of this neurological condition. Using real-world evidence, machine learning, AI, and genetic testing are showing a path to finding a treatment. Nasha explains, "My daughter, Amara, was diagnosed at nine months of age. She began having hundreds of seizures a day. And at that point, there was information known about the FOXG1 gene. It's a really important gene for brain development. But no one was studying the syndrome and how to find a cure for it. So, I and a group of other parents, like-minded parents who, refused to just take that this is the way it is. Our children have this condition, and that's it. We started this foundation." "We've done a lot of work to get the entire world of FOXG1 to work together and then to collect patient data. That leads to my work at Ciitizen, to have all patients join the same platform, take the same surveys, and collect electronic medical records. We extract their data, so it's all on one platform. Again, that helps us understand this disease, which is critical when the population's so small." "In-person studies, which is how we collect data today, as the medical community, don't work for ultra-rare diseases. So we have to find other ways. And that's where real-world evidence comes in." "We use machine learning and AI at Ciitizen when we're looking at something as simple as extracting data from an unstructured medical record. When you look in medical records, the part that's really interesting is the clinic notes, and they're totally unstructured. They're paragraphs. So can we start using machine learning and AI to go through these paragraphs and actually extract relevant data points to scale up our ability to understand how the disease has progressed?" #FOXG1 #FOXG1ResearchFoundation #Ciitizen #GeneTherapy #RealWorldEvidence #RareDisease #GeneticTesting FOXG1Research.org Ciitizen.com Download the transcript here

David Dodd is Chairman, President, and CEO of GeoVax, which is working on vaccines that overcome restrictions of mRNA vaccines to provide longer-term, more robust protection, particularly for people who do not respond well to primary antibody stimulation. There is a strong response from T cells from this vaccine which can be stored in a non-refrigerated freeze-dried form allowing for greater access in more regions of the world. David explains, "We are targeting those individuals who have compromised immune systems. This is a category of approximately 15 million individuals in the United States and almost 250 million worldwide, who, as a result of the conditions of their body or maybe even the therapies they may be on, or other conditions. Their body does not respond appropriately or well to the existing approved vaccines for COVID-19 or to the monoclonal antibodies. It's not that the other products have no value - it's that the individuals' bodies have been depleted of the ability to mount the proper response." "We also include what is known as the nucleocapsid protein, which is highly conserved across viruses. It's always there, and it induces a very strong T cell response which drives memory in vaccines or in the human body from an immune standpoint. So, it is memory durability and also that it enables people whose bodies may have been depleted in order to mount an antibody response. They now can rely upon their T cells system or their cellular immune system to give them a protective element to reduce the severity, hospitalization, and risk of death." #GeoVax #Vaccines #VaccineFatigue #Biodefense #COVID #VaccineAccess #VaccinePreparedness #TCells GeoVax.com Download the transcript here

Chris Hoyt, CEO of KeifeRx, talks about the oral medication being developed that uses tyrosine kinase inhibitors (TKIs) and autophagy to remove unwanted proteins. Research done by Georgetown University on leukemia, led by Dr. Charbel Moussa, found that lower doses of drugs like nilotinib and bosutinib could cross the blood-brain barrier and remove toxic proteins on an intracellular basis. This research has been used to develop treatments for neurodegenerative conditions with promising results in reducing cognitive decline. Chris explains, "Essentially, the way TKIs work in the body, and particularly in the case of the neurodegenerative conditions that we're using them to treat at KeifeRx, is they trigger a mechanism called autophagy, which essentially is the cell's garbage disposal mechanism. What we're doing with TKIs is using that mechanism to remove toxic proteins. TKIs have mostly been used historically in cancer, and particularly in leukemia. The classic use would be in cancer to try to remove as much of the tumors as you can in leukemia. In that environment, use a fairly high dose of TKIs to trigger that effect. What KeifeRx has done through work at Georgetown University is find ways to utilize that same mechanism of action in the brain." "So over the course of a few years of initial work, we came to the conclusion that these drugs, in the case of specifically nilotinib and bosutinib, which are the two drugs where we have the use patent in neurodegeneration, that at much lower doses than what you see in cancer, these drugs can cross the blood-brain barrier and look to remove toxic proteins on an intracellular basis. So, it was a classic hunch, for lack of a better term, that Dr. Moussa had that he then carried out rigorous work over three years to first prove in animal models and then eventually through a series of small phase I and phase II trials at Georgetown." @KeifeRx_Thera #KeifeRx #Alzheimers #TKI #Autophagy #ALS #LewyBodyDementia KeifeRx.com Download the transcript here

Simon Allen is the CEO and Director of Anebulo Pharmaceuticals, which focuses on finding an effective treatment for acute cannabinoid intoxication or ACI, particularly in an emergency department setting. ACI is a condition where an individual has consumed or smoked too much cannabis or products that contain THC or synthetic cannabis and is experiencing symptoms of psychosis, anxiety, elevated heart rate, or is unresponsive. Their drug ANEB-001 is designed to address the cause of the symptoms without the need for traditional methods like benzodiazepines and beta blockers, which have their risks. Simon explains, "What we are seeing is individuals taking these high-potency edibles, for example, and also smoking and vaping. These are other forms that can create ACI outcomes. And essentially, they're out for several hours, if not overnight, in the hospital. This can get as serious as the escalation to a psychiatric ward if the individual is so psychotic, agitated, and anxious that the emergency department can't handle it or chooses not to handle it and pushes it to the psychiatric ward. And those expenses are in the tens of thousands of dollars to the healthcare system. Not only that, there are extremely poor patient outcomes." "What's important is that these molecules, the synthetic ones, are, unfortunately extremely strong binders to the CB1 receptor and do more to stimulate effectively. And very, very small doses of these synthetic cannabinoids can create very poor patient outcomes simply because they're much tighter binders to the CB1 receptor compared to THC. And when they do bind to that receptor, they're much more active. And so the street names like K2 and Spice are examples." @AnebuloP #AnebuloPharmaceuticals #ACI #AcuteCannabinoidIntoxication #Cannabis #Overdose Anebulo.com Download the transcript here

Nancy Lurker, CEO and Board Director of EyePoint Pharmaceuticals, aims to prevent the progression of blindness by developing technology that addresses retinal eye diseases. These back of the eye diseases are age-related and an increasing threat to populations worldwide. Current treatments require regular injections in the eye. The micro inserts developed by EyePoint will allow the drug to work for six months, allowing patients more flexibility to maintain their vision. Nancy explains, "We know that if you look at the current eye treatments on the market today, wet AMD, retinal vein occlusion, diabetic macular edema, and diabetic retinopathy, all of those are running close to $30 to $40 billion being spent to help to treat these terrible eye diseases which may lead to blindness." "So, what causes these diseases? There's a genetic component at times, but also age plays a factor. And then, of course, diabetic macular edema and diabetic retinopathy can be caused for patients who have diabetes. So. what happens is you get a proliferation of tiny, tiny micro capillaries in the eye and, in some cases, just general atrophy of these retinal epithelia. These cells help to allow you to see. And what happens is when you start to get this proliferative growth that goes on and leakage of fluid and cells into the back of the eye, it completely obstructs your vision, which grows over time." "The vast majority of patients have to get another injection every month or every other month to slow the progression or stop it. And if you miss a visit, that fluid will come right back, and remember, the fluid is very, very bad for the eye. And we can see in the large databases, it's the medical claims data from patients, millions of these patients, we can see that even though they go in and get their eyes injected, you miss visits. Why? Life happens." @EyePointPharma #EyePointPharma #Ophthalmology #RetinalEyeDiseases #WetAMD #RetinalVeinOcclusion #DiabeticMacularEdema #DiabeticRetinopathy eyepointpharma.com Download the transcript here

Dr. Gavin Samuels, Chief Business Officer at CinRx, a holding company currently with six portfolio companies, each focused on developing one drug. Providing these companies with the right resources at the right time makes for more efficient use of resources and can speed up drug development. Their strength is understanding challenges and opportunities approximately a year before an IND is filed to help shepherd the drug through the IND-enabling study and through clinical development. Gavin elaborates, "The disease areas that we're involved in, cardiovascular disease, metabolic disease, inflammatory diseases, these are problems that present a real challenge to patients and a real challenge to society and a real challenge to humanity. We want to try and address those problems by bringing innovative drugs and developing them as quickly and efficiently as possible and bringing to the patient." "The criteria we use is that it has to fit a specific unmet medical need. We have to have a large group of patients that are involved in the disease, that have the disease, and we want to make sure that we can make a meaningful difference to the way that the disease is treated as well as the quality of life for the patient. So we don't want incremental improvements. We're looking for drugs that are going to make step changes in the quality of life of the patients we are trying to bring the medicine to." #CinRx #CinRxSuperHighway #TransformationalMedicines #DrugDevelopment #Obesity #TypeOneDiabetes #T1D #Hypertension #Gastroparesis #IrritableBowelSyndrome #SolidTumors #Oncology #NeurodegenerativeDiseases cinrx.com Download the transcript here

Jennifer Quinn, Head of global pricing and health economics and outcomes research at Debiopharm, emphasizes the importance of reimbursement, not just regulatory approval, for a drug to be available to patients. With patient-centered outcomes research, drug developers can use data for risk-benefit assessment relevant to regulators that reflect how patients consider the risks and whether they will undergo a procedure. Jennifer explains, "My team really tries to make sure that, when we're thinking about a clinical development program, we make sure we're capturing those value markers throughout from a very early stage. That is so that when someone out-licenses it and becomes our partner, they have the data they need not just to get through regulatory approval but to have the drug reimbursed and made available to patients." "It's so important that patients have not just the agency in their care, but we understand how treatments are impacting patients -- impacting their lives, impacting how they feel, impacting their activities of daily living. I think that is what patient-centered outcomes research encompasses: patient preference, patient functioning, and patient feeling. If we don't do that, we are not patient-centric in our drug development because we're missing this crucial part. A drug might shrink a tumor this much, or it might give you this many fewer bleeds if you're a hemophilia patient, but what does that mean to the patient? How does that impact them?" @DebiopharmNews #Debiopharm #DrugDevelopment #Biotech #PatientCare #PatientCenteredOutcomesResearch Debiopharm.com Download the transcript here

Ohad Arazi, CEO of Clarius, a medical imaging startup with a mission to bring accurate, easy-to-use, AI-assisted, and affordable hand-held ultrasound tools to a broad range of medical professionals. Traditional ultrasound is costly, stationary, and requires specialized training. The portable Clarius device communicates with an iPad or an Android or iOS phone. The ultrasound produces highly nuanced grayscale real-time images that, with the help of artificial intelligence, can be used safely in a wider point-of-care environment to make informed decisions. Ohad explains, "We're all about bringing together high-performance ultrasound imaging, cloud data, and artificial intelligence into this powerful ecosystem that improves patient care enabled by medical imaging in every setting. The key differentiation is that handheld ultrasound is not about replacing legacy systems in places where they're already well entrenched, like radiology or cardiology. It is rather about bringing medical imaging to new care settings and putting it in the hands of new practitioners that may not have had access to an ultrasound before." "We can even extend that outside of the walls of the hospital into the ambulance. Powered by AI, we have a feature called Auto Preset AI, which, as the EMT roam through the patient's body, looking for trauma, our AI will automatically adapt the view of that image to optimize it for the organ the EMT is looking at. This lowers that threshold for knowledge to enable them to make a more informed decision. For example, to see that there might be stenosis in the carotid and they should radio the hospital and get the thrombectomy lab ready because there's a stroke patient on hand." #Clarius #MedTech #Ultrasound #MedicalImaging #AI #DigitalHealth #HandheldUltrasound clarius.com clarius.com/2023-state-of-ultrasound-report/ Download the transcript here