Empowered Patient Podcast

David Young, President of R&D at Processa Pharmaceuticals, talks about the FDA's Project Optimus draft guidance on optimal dosage regimens for specific patients. Processa is modifying existing cancer-killing molecules to improve efficacy and decrease side effects by taking into account genetic factors and the benefit-risk profile of each patient. This precision medicine approach will inform clinical trial recruitment, dosing regimens and the number of patients who can be effectively helped.

David explains, "There are a couple of things that make us unique. One is that we're taking the active cancer-killing molecules on the market. It's a given drug that's approved by the FDA, and we know it kills cancer. We've taken that molecule and either changed it slightly or administered it with another drug in some way to decrease or change the drug's metabolism and then to distribute more drug to the cancer cell."

"So, we started to learn how they look at the benefit-risk profile, and we started to develop our version of regulatory science, and that's expanded now over the last 30 years. That expansion into regulatory science now encompasses Project Optimus, which the FDA recently has put out draft guidance on in terms of optimal dosage regimens. They as well put out some documentation on the Project Optimus initiative where you go in to determine the optimal regimen for your patient. Not just any regimen, but the optimal regimen for your patients. If you get an optimal regimen for your patients, maybe that has a certain balance of efficacy and safety. You might come up with something different than we used to do five years ago in terms of the dosage regimen."

#ProcessaPharmaceuticals #ProjectOptimus #BenefitRiskProfile #Cancer #PrecisionMedicine #ClinicalTrials

processapharmaceuticals.com

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David Young, President of R&D at Processa Pharmaceuticals, talks about the FDA's Project Optimus draft guidance on optimal dosage regimens for specific patients. Processa is modifying existing cancer-killing molecules to improve efficacy and decrease side effects by taking into account genetic factors and the benefit-risk profile of each patient. This precision medicine approach will inform clinical trial recruitment, dosing regimens and the number of patients who can be effectively helped.

David explains, "There are a couple of things that make us unique. One is that we're taking the active cancer-killing molecules on the market. It's a given drug that's approved by the FDA, and we know it kills cancer. We've taken that molecule and either changed it slightly or administered it with another drug in some way to decrease or change the drug's metabolism and then to distribute more drug to the cancer cell."

"So, we started to learn how they look at the benefit-risk profile, and we started to develop our version of regulatory science, and that's expanded now over the last 30 years. That expansion into regulatory science now encompasses Project Optimus, which the FDA recently has put out draft guidance on in terms of optimal dosage regimens. They as well put out some documentation on the Project Optimus initiative where you go in to determine the optimal regimen for your patient. Not just any regimen, but the optimal regimen for your patients. If you get an optimal regimen for your patients, maybe that has a certain balance of efficacy and safety. You might come up with something different than we used to do five years ago in terms of the dosage regimen."

#ProcessaPharmaceuticals #ProjectOptimus #BenefitRiskProfile #Cancer #PrecisionMedicine #ClinicalTrials

processapharmaceuticals.com

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Dr. Juan Vera, CEO of Marker Therapeutics, a clinical-stage immuno-oncology company, is developing next-generation immunotherapy with a multi-tumor-specific T-cell platform to overcome limitations of current T-cell therapies with narrow target recognition. This approach, which recognizes multiple antigens within the tumor, helps prevent tumor resistance and adaptation. Marker is participating in the APOLLO trial testing this broad spectrum of target recognition in lymphoma patients who have relapsed after CAR-T CD19 therapy while also exploring acute myeloid leukemia and pancreatic cancer.

Juan explains, "Without a doubt, the earlier products that have been approved, such as mainly chimeric T-cell receptors, have validated the clinical power of T-cells in their ability to recognize and kill cancer cells. I think that no one will argue that, and this has been something that has been well evaluated, particularly in hematologic malignancies and particularly in lymphoma."

"However, there are limitations with these types of technologies, and some of the limitations now have become more and more obvious a few years after the approval of those initial drugs. One of the main limitations relies on the short-term clinical benefit observed in many cases once patients receive these therapies. That can be attributed largely to the narrow target recognition of these initial strategies."

"Our approach is different and seeks to overcome this particular limitation. The way that we seek to do so is by creating a product that is capable of recognizing multiple antigens present within the tumor. In that particular way, our therapy is capable of preventing this mechanism of tumor adaptation and tumor resistance. We hope that this multi-target approach will lead to clinical responses that will be long-sustained and durable in comparison to what we're seeing initially with these initial studies or initial drugs that have been approved in the market."

#MarkerTherapeutics #Oncology #AutologousCellTherapy #TCellTherapy #Lymphoma #Cancer

markertherapeutics.com

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Dr. Juan Vera, CEO of Marker Therapeutics, a clinical-stage immuno-oncology company, is developing next-generation immunotherapy with a multi-tumor-specific T-cell platform to overcome limitations of current T-cell therapies with narrow target recognition. This approach, which recognizes multiple antigens within the tumor, helps prevent tumor resistance and adaptation. Marker is participating in the APOLLO trial testing this broad spectrum of target recognition in lymphoma patients who have relapsed after CAR-T CD19 therapy while also exploring acute myeloid leukemia and pancreatic cancer.

Juan explains, "Without a doubt, the earlier products that have been approved, such as mainly chimeric T-cell receptors, have validated the clinical power of T-cells in their ability to recognize and kill cancer cells. I think that no one will argue that, and this has been something that has been well evaluated, particularly in hematologic malignancies and particularly in lymphoma."

"However, there are limitations with these types of technologies, and some of the limitations now have become more and more obvious a few years after the approval of those initial drugs. One of the main limitations relies on the short-term clinical benefit observed in many cases once patients receive these therapies. That can be attributed largely to the narrow target recognition of these initial strategies."

"Our approach is different and seeks to overcome this particular limitation. The way that we seek to do so is by creating a product that is capable of recognizing multiple antigens present within the tumor. In that particular way, our therapy is capable of preventing this mechanism of tumor adaptation and tumor resistance. We hope that this multi-target approach will lead to clinical responses that will be long-sustained and durable in comparison to what we're seeing initially with these initial studies or initial drugs that have been approved in the market."

#MarkerTherapeutics #Oncology #AutologousCellTherapy #TCellTherapy #Lymphoma #Cancer

markertherapeutics.com

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Andreas Grill, President and CEO of DepYmed, discusses protein tyrosine phosphatase-targeted drugs, a new class of drugs. With a focus on the specific enzyme PTP1B, DepYmed discovered orally bioavailable molecules that inhibit PTP1B, targeting the signal transaction pathway. They are initially testing to treat Rett syndrome, a rare disease with no current therapy while exploring the use of PTP1B inhibitors to treat inflammatory diseases, cancer, diabetes, and neurological diseases.

Andreas elaborates, "In DepYmed, we're focused on a specific enzyme. It's PTP1B. It's part of a family of enzymes called protein tyrosine phosphatases, and, in particular, we're looking at PTP1B. It's a metabolic regulatory enzyme that regulates signal transduction between cells and how cells communicate with each other. It's been worked on in the '90s and early 2000s. A couple of companies were working on the target, and they failed in the target, mainly because they couldn't create an orally bioavailable compound that would inhibit the PTP1B enzyme itself."

"So that was one of the holy grails that we were able to find, where we were able to discover molecules that were orally bioavailable and would inhibit PTP1B. It was a game-changer when it came to the therapeutics around the target of PTP1B. Much of this work came out of Dr. Nicholas Tonks' laboratory out of Cold Spring Harbor Laboratory. We are in close collaboration with Nick and his team at Cold Spring Harbor, developing this new area of PTP1B inhibitors targeting the signal transaction pathway."

#DepYmed #RettSyndrome #DPM1003 #PTP1B #Phosphatases #PhosphatasesInhibitors #RareDisease

DepYmed.com

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Andreas Grill, President and CEO of DepYmed, discusses protein tyrosine phosphatase-targeted drugs, a new class of drugs. With a focus on the specific enzyme PTP1B, DepYmed discovered orally bioavailable molecules that inhibit PTP1B, targeting the signal transaction pathway. They are initially testing to treat Rett syndrome, a rare disease with no current therapy while exploring the use of PTP1B inhibitors to treat inflammatory diseases, cancer, diabetes, and neurological diseases.

Andreas elaborates, "In DepYmed, we're focused on a specific enzyme. It's PTP1B. It's part of a family of enzymes called protein tyrosine phosphatases, and, in particular, we're looking at PTP1B. It's a metabolic regulatory enzyme that regulates signal transduction between cells and how cells communicate with each other. It's been worked on in the '90s and early 2000s. A couple of companies were working on the target, and they failed in the target, mainly because they couldn't create an orally bioavailable compound that would inhibit the PTP1B enzyme itself."

"So that was one of the holy grails that we were able to find, where we were able to discover molecules that were orally bioavailable and would inhibit PTP1B. It was a game-changer when it came to the therapeutics around the target of PTP1B. Much of this work came out of Dr. Nicholas Tonks' laboratory out of Cold Spring Harbor Laboratory. We are in close collaboration with Nick and his team at Cold Spring Harbor, developing this new area of PTP1B inhibitors targeting the signal transaction pathway."

#DepYmed #RettSyndrome #DPM1003 #PTP1B #Phosphatases #PhosphatasesInhibitors #RareDisease

DepYmed.com

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Vin Singh, Founder and CEO of Bullfrog AI, discusses how AI is being used to improve the drug development process through a precision medicine approach that looks at a combination of genetic and non-genetic attributes to predict patient response to drugs. Bullfrog AI aims to reduce the failure rate in late-stage trials by enrolling the right patients in clinical trials. Through their partnership with the Johns Hopkins Lieber Institute for Brain Development, they use AI to analyze a unique dataset of post-mortem brains to discover new drug targets and improve clinical trial design.

Vin explains, "Bullfrog is a technology-enabled drug development company. And what we're doing is using a proprietary AI to improve the drug development process, reduce development times and costs for development, and increase access, which benefits the world."

"The pharmaceutical industry is plagued with inefficiencies. It takes 10 to 15 years and one to two billion dollars to develop drugs, and the failure rates in that drug development process are incredibly high. Our goal here is to fix that formula of failure, which will ultimately benefit patients and get them access to drugs that work for them."

"Typically, when you think of AI and machine learning, you think about training the AI to identify or predict something. Typically, the thinking is you need lots and lots of data and lots of patients. With our technology, we can do a shallow and wide analysis. What that means is shallow means not thousands or millions of patients. We can do it with hundreds of patients. Wide means it could be a lot of different types of data, including genomic data. So there might be millions of areas."

#BullfrogAI #AI #DrugDevelopment #ClinicalTrials #LieberInstitute #JohnsHopkinsUniversity

Bullfrogai.com

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Vin Singh, Founder and CEO of Bullfrog AI, discusses how AI is being used to improve the drug development process through a precision medicine approach that looks at a combination of genetic and non-genetic attributes to predict patient response to drugs. Bullfrog AI aims to reduce the failure rate in late-stage trials by enrolling the right patients in clinical trials. Through their partnership with the Johns Hopkins Lieber Institute for Brain Development, they use AI to analyze a unique dataset of post-mortem brains to discover new drug targets and improve clinical trial design.

Vin explains, "Bullfrog is a technology-enabled drug development company. And what we're doing is using a proprietary AI to improve the drug development process, reduce development times and costs for development, and increase access, which benefits the world."

"The pharmaceutical industry is plagued with inefficiencies. It takes 10 to 15 years and one to two billion dollars to develop drugs, and the failure rates in that drug development process are incredibly high. Our goal here is to fix that formula of failure, which will ultimately benefit patients and get them access to drugs that work for them."

"Typically, when you think of AI and machine learning, you think about training the AI to identify or predict something. Typically, the thinking is you need lots and lots of data and lots of patients. With our technology, we can do a shallow and wide analysis. What that means is shallow means not thousands or millions of patients. We can do it with hundreds of patients. Wide means it could be a lot of different types of data, including genomic data. So there might be millions of areas."

#BullfrogAI #AI #DrugDevelopment #ClinicalTrials #LieberInstitute #JohnsHopkinsUniversity

Bullfrogai.com

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Melinda Thomas, the COO and Co-Founder of Octave Bioscience is focused on delivering a new standard of care for multiple sclerosis and other neurodegenerative diseases. The company aims to address the unmet needs of people living with MS using blood-based biomarkers, expanded imaging, and a clinical insights program. The solution includes an app, wearable sensors, and a nurse care partner to help patients and neurologists make better-informed treatment decisions.

Melinda elaborates, "What we learned when we did about 150 interviews before we even started developing anything was, in MS specifically, there was no way to see what was going on beneath the surface. You didn't have a blood test that told you what was going on in the biology. So, we developed a blood test, 18 biomarkers analytically validated, that reveal underlying activated pathways and mechanisms that allow for accurate measurement of what's going on with their disease activity."

"The tool used today is an MRI, which shows new or worsening lesions. It shows damage has occurred. So, the biomarker helps understand whether there is more disease activity that will eventually lead to these kinds of lesions. But also, if that is the gold standard, to understand if there are new or worsening lesions. We worked very hard with clinicians saying, "Where's the gap for you right now? What's the unmet need?"

#OctaveBioscience #MS #MultipleSclerosis #NeurodegenerativeDiseases #Biomarkers #BloodTests

octavebio.com

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Melinda Thomas, the COO and Co-Founder of Octave Bioscience is focused on delivering a new standard of care for multiple sclerosis and other neurodegenerative diseases. The company aims to address the unmet needs of people living with MS using blood-based biomarkers, expanded imaging, and a clinical insights program. The solution includes an app, wearable sensors, and a nurse care partner to help patients and neurologists make better-informed treatment decisions.

Melinda elaborates, "What we learned when we did about 150 interviews before we even started developing anything was, in MS specifically, there was no way to see what was going on beneath the surface. You didn't have a blood test that told you what was going on in the biology. So, we developed a blood test, 18 biomarkers analytically validated, that reveal underlying activated pathways and mechanisms that allow for accurate measurement of what's going on with their disease activity."

"The tool used today is an MRI, which shows new or worsening lesions. It shows damage has occurred. So, the biomarker helps understand whether there is more disease activity that will eventually lead to these kinds of lesions. But also, if that is the gold standard, to understand if there are new or worsening lesions. We worked very hard with clinicians saying, "Where's the gap for you right now? What's the unmet need?"

#OctaveBioscience #MS #MultipleSclerosis #NeurodegenerativeDiseases #Biomarkers #BloodTests

octavebio.com

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