Empowered Patient Podcast

Dr. Robert Risinger is the Chief Medical Officer for Neuroscience at BioXcel Therapeutics, identifying molecules that have off-target indications to address unmet neurological diseases. The initial focus is on acute agitation and the molecules that will address this agitation in patients with bipolar and schizophrenia. Their fast-acting sublingual film formulation is an advancement over the current regime of intramuscular injections. The drug is also being studied for agitation related to Alzheimer's disease and at-home use to reduce the number of people who go to the emergency room for untreated agitation.

Rob explains, "For example, the medication, the active pharmaceutical ingredient dexmedetomidine, was used, for years, as an IV anesthetic. At very high doses, it can produce a level of sleep or sedation so patients can undergo surgery and medical procedures. But our algorithms identified the fact that this may affect the core causal mechanism, this kind of fight or flight reaction, that we believe is responsible for causing agitation in humans and, specifically, in patients with bipolar disorder and schizophrenia."

"The molecule itself has physical-chemical properties that allow it to get into the body quickly. We can use, essentially, very, very low doses compared to what is typically given for anesthesia purposes. So we tested this out systematically and came up with a new form and formulation, a sublingual film with mucoadhesive properties. And that allows us to diminish the agitation in patients experiencing these symptoms. The molecule itself has physical-chemical properties that allow it to get into the body quickly. We can use, essentially, very, very low doses compared to what is typically given for anesthesia purposes. So we tested this out systematically and came up with a new form and formulation, a sublingual film with mucoadhesive properties. And that allows us to diminish the agitation in patients experiencing these symptoms."

@BioXcel #Neuroscience #Schizophrenia #BipolarDisorder #AlzheimersDisease #Agitation #AcuteAgitation

bioXcelTherapeutics.com

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Dr. Robert Risinger is the Chief Medical Officer for Neuroscience at BioXcel Therapeutics, identifying molecules that have off-target indications to address unmet neurological diseases. The initial focus is on acute agitation and the molecules that will address this agitation in patients with bipolar and schizophrenia. Their fast-acting sublingual film formulation is an advancement over the current regime of intramuscular injections. The drug is also being studied for agitation related to Alzheimer's disease and at-home use to reduce the number of people who go to the emergency room for untreated agitation.

Rob explains, "For example, the medication, the active pharmaceutical ingredient dexmedetomidine, was used, for years, as an IV anesthetic. At very high doses, it can produce a level of sleep or sedation so patients can undergo surgery and medical procedures. But our algorithms identified the fact that this may affect the core causal mechanism, this kind of fight or flight reaction, that we believe is responsible for causing agitation in humans and, specifically, in patients with bipolar disorder and schizophrenia."

"The molecule itself has physical-chemical properties that allow it to get into the body quickly. We can use, essentially, very, very low doses compared to what is typically given for anesthesia purposes. So we tested this out systematically and came up with a new form and formulation, a sublingual film with mucoadhesive properties. And that allows us to diminish the agitation in patients experiencing these symptoms. The molecule itself has physical-chemical properties that allow it to get into the body quickly. We can use, essentially, very, very low doses compared to what is typically given for anesthesia purposes. So we tested this out systematically and came up with a new form and formulation, a sublingual film with mucoadhesive properties. And that allows us to diminish the agitation in patients experiencing these symptoms."

@BioXcel #Neuroscience #Schizophrenia #BipolarDisorder #AlzheimersDisease #Agitation #AcuteAgitation

bioXcelTherapeutics.com

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Neal Koller is the CEO of Alphyn Biologics, which focuses on improving therapy for skin diseases. Their first topical drug treats both atopic dermatitis and atopic dermatitis with secondary infection. The goal is to treat the recognized immune component of the disease and the bacterial component. Those most affected by this disease are ages 2 to 11 though it is a lifelong recurring problem with only a multi-drug treatment regimen available.

Neal explains, "I think we've got three advantages. First, the plant material that we're using is the microbial and small molecule defense liquid of the plant, and so it's got a number of different bioactive compounds. One advantage for us is in our drug product, we have multiple bioactive compounds, which target a disease, in our case, atopic dermatitis, in different ways. So we have multiple ways we can attack and treat the disease. That's unique and different, having multiple ways, versus a standard drug generally only has one bioactive compound and one way to treat a disease."

"The second advantage is that by using the plant-derived material in the way in which we're using it, we've had a faster path, and faster progress through our work to get to human clinical trials. That's primarily because our material, our drug substance, has a very, very strong safety profile."

"Then the third advantage so far, unless, of course, the FDA changes this, is that because our drug product is plant-derived, there isn't a generic drug approval path. You, at the moment, still can't go into the lab, mix up some chemistry experiments, keep it simple, and say, "My chemistry is similar to Alphyn's chemistry, so please give me approval."

#AlphynBiologics #AtopicDermatitis #Dermatology #SkinDisease

AlphynBiologics.com

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Neal Koller is the CEO of Alphyn Biologics, which focuses on improving therapy for skin diseases. Their first topical drug treats both atopic dermatitis and atopic dermatitis with secondary infection. The goal is to treat the recognized immune component of the disease and the bacterial component. Those most affected by this disease are ages 2 to 11 though it is a lifelong recurring problem with only a multi-drug treatment regimen available.

Neal explains, "I think we've got three advantages. First, the plant material that we're using is the microbial and small molecule defense liquid of the plant, and so it's got a number of different bioactive compounds. One advantage for us is in our drug product, we have multiple bioactive compounds, which target a disease, in our case, atopic dermatitis, in different ways. So we have multiple ways we can attack and treat the disease. That's unique and different, having multiple ways, versus a standard drug generally only has one bioactive compound and one way to treat a disease."

"The second advantage is that by using the plant-derived material in the way in which we're using it, we've had a faster path, and faster progress through our work to get to human clinical trials. That's primarily because our material, our drug substance, has a very, very strong safety profile."

"Then the third advantage so far, unless, of course, the FDA changes this, is that because our drug product is plant-derived, there isn't a generic drug approval path. You, at the moment, still can't go into the lab, mix up some chemistry experiments, keep it simple, and say, "My chemistry is similar to Alphyn's chemistry, so please give me approval."

#AlphynBiologics #AtopicDermatitis #Dermatology #SkinDisease

AlphynBiologics.com

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Brett Furst, President of HHS Technology Group, is carrying on the mission of efforts started at the beginning of the COVID outbreak to collaboratively share data to provide insights to those doing research, drug development, and addressing the impact of the pandemic on vulnerable populations. Maintaining this world's largest COVID database, HTG allows research on complex social determinants of health and how to address health equity.

Brett explains, "So we were proud to take it on and be chosen by the consortium. Today we have the largest COVID research database in the world, with over 65 billion records. It touches almost 100% of our population in the United States. We're looking at all encounters from claims and clinical data, but also other more important for predictive modeling like social determinant data. We're trying to be more thoughtful about what's the next thing that's going to hit us out of the blue. We cannot be caught so flat-footed as we were in 2020."

"There are concerns about long-term effects on those with Alzheimer's or other types of dementia, COPD, and breathing. I think what we all learned during this collaborative is that it didn't deflate everybody's competitive advantage and that sometimes regulations and rules can keep the market from actually advancing."

@HHSTechnologyGroup #HHSTechGroup #HealthTech #HealthCare #COVID #LongCOVID #Pandemic #HealthData #HealthEquity #SDOH

hhstechgroup.com

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Brett Furst, President of HHS Technology Group, is carrying on the mission of efforts started at the beginning of the COVID outbreak to collaboratively share data to provide insights to those doing research, drug development, and addressing the impact of the pandemic on vulnerable populations. Maintaining this world's largest COVID database, HTG allows research on complex social determinants of health and how to address health equity.

Brett explains, "So we were proud to take it on and be chosen by the consortium. Today we have the largest COVID research database in the world, with over 65 billion records. It touches almost 100% of our population in the United States. We're looking at all encounters from claims and clinical data, but also other more important for predictive modeling like social determinant data. We're trying to be more thoughtful about what's the next thing that's going to hit us out of the blue. We cannot be caught so flat-footed as we were in 2020."

"There are concerns about long-term effects on those with Alzheimer's or other types of dementia, COPD, and breathing. I think what we all learned during this collaborative is that it didn't deflate everybody's competitive advantage and that sometimes regulations and rules can keep the market from actually advancing."

@HHSTechnologyGroup #HHSTechGroup #HealthTech #HealthCare #COVID #LongCOVID #Pandemic #HealthData #HealthEquity #SDOH

hhstechgroup.com

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Dr. Allen Davidoff is the Founder and CEO of XORTX Therapeutics, testing the benefit of a therapeutic for autosomal dominant polycystic kidney disease, a rare progressive kidney disease with a clear genetic connection. Evidence suggests that lowering the activity of the xanthine oxidase enzyme in the kidney and lowering uric acid production via this enzyme is critical to developing a therapy that can benefit these patients.

Allen explains, "In terms of real therapies that slow the progression of kidney disease, there are very few. And so, to take the example of the rare disease, autosomal dominant polycystic kidney disease, one drug is currently approved. It was approved in 2018, that's a drug called tolvaptan, but it's only really used in about 5% of individuals. So that leaves this rare disease population largely underserved despite the fact that there's an approved drug."

"The drug that we're developing is oxypurinol. It is a xanthine oxidase inhibitor that has been studied extensively over a number of years. It is arguably the single best-tolerated xanthine oxidase inhibitor that's never been to market. We've started with that drug and reformulated it in a proprietary way to deliver it so that it will reach circulating blood concentrations that can inhibit the xanthine oxidase enzyme by 90% or more."

@XORTXinc #ADPKD #PKD #ProgressiveKidneyDisease #KidneyHealth #RareDisease

XORTX.com

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Dr. Allen Davidoff is the Founder and CEO of XORTX Therapeutics, testing the benefit of a therapeutic for autosomal dominant polycystic kidney disease, a rare progressive kidney disease with a clear genetic connection. Evidence suggests that lowering the activity of the xanthine oxidase enzyme in the kidney and lowering uric acid production via this enzyme is critical to developing a therapy that can benefit these patients.

Allen explains, "In terms of real therapies that slow the progression of kidney disease, there are very few. And so, to take the example of the rare disease, autosomal dominant polycystic kidney disease, one drug is currently approved. It was approved in 2018, that's a drug called tolvaptan, but it's only really used in about 5% of individuals. So that leaves this rare disease population largely underserved despite the fact that there's an approved drug."

"The drug that we're developing is oxypurinol. It is a xanthine oxidase inhibitor that has been studied extensively over a number of years. It is arguably the single best-tolerated xanthine oxidase inhibitor that's never been to market. We've started with that drug and reformulated it in a proprietary way to deliver it so that it will reach circulating blood concentrations that can inhibit the xanthine oxidase enzyme by 90% or more."

@XORTXinc #ADPKD #PKD #ProgressiveKidneyDisease #KidneyHealth #RareDisease

XORTX.com

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Lisa Yanez is the CEO of Aerami Therapeutics, a clinical stage biopharmaceutical company focused on the rare cardiopulmonary disease pulmonary arterial hypertension. Early studies show that using this smart breath-activated inhalation delivery system to administer AER901, inhaled Imatinib reduces side effects and requires less frequent administration by those suffering from PAH.

Lisa explains, "Our company is focused on a rare disease, pulmonary arterial hypertension. There was a drug that was an oral drug that was studied before, but due to the side effects, that trial ceased. That said, if you look at the efficacy of that drug, it was one of the best-performing drugs ever for these patients. It didn't just help with symptoms. It also helped the underlying cause of the disease. What our company has decided to do is take a very intelligent inhaled patient-proven device and couple it with this product to generate the best of both worlds. What we're trying to do is helping patients live longer, but our mission is also helping patients live better."

"One of the reasons I've fallen in love with this particular condition is because it mostly impacts women of childbearing age. They go about two years not knowing what's wrong with them. They have shortness of breath, they have a lot of fatigue, they're very dizzy, and sometimes they pass out. They go to all these different doctors, and typically it does take two years, and people think they're crazy."

"Then, once they're diagnosed, there are 15 approved therapies. Even then, there's only a five to seven-year time span in terms of survival. And so, there's a lot more that needs to be done with these particular patients."

@AeramiTx #Aerami #Cardiopulmonary #RareDisease #PulmonaryArterialHypertension #PulmonaryHypertension #PAH #PH #HeartDisease #LungDisease #AER901 #InhaledTherapeutics #AER601

aerami.com

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Lisa Yanez is the CEO of Aerami Therapeutics, a clinical stage biopharmaceutical company focused on the rare cardiopulmonary disease pulmonary arterial hypertension. Early studies show that using this smart breath-activated inhalation delivery system to administer AER901, inhaled Imatinib reduces side effects and requires less frequent administration by those suffering from PAH.

Lisa explains, "Our company is focused on a rare disease, pulmonary arterial hypertension. There was a drug that was an oral drug that was studied before, but due to the side effects, that trial ceased. That said, if you look at the efficacy of that drug, it was one of the best-performing drugs ever for these patients. It didn't just help with symptoms. It also helped the underlying cause of the disease. What our company has decided to do is take a very intelligent inhaled patient-proven device and couple it with this product to generate the best of both worlds. What we're trying to do is helping patients live longer, but our mission is also helping patients live better."

"One of the reasons I've fallen in love with this particular condition is because it mostly impacts women of childbearing age. They go about two years not knowing what's wrong with them. They have shortness of breath, they have a lot of fatigue, they're very dizzy, and sometimes they pass out. They go to all these different doctors, and typically it does take two years, and people think they're crazy."

"Then, once they're diagnosed, there are 15 approved therapies. Even then, there's only a five to seven-year time span in terms of survival. And so, there's a lot more that needs to be done with these particular patients."

@AeramiTx #Aerami #Cardiopulmonary #RareDisease #PulmonaryArterialHypertension #PulmonaryHypertension #PAH #PH #HeartDisease #LungDisease #AER901 #InhaledTherapeutics #AER601

aerami.com

Download the transcript here