Empowered Patient Podcast

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

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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

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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

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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

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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

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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

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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

Listen to the podcast 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

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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

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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

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