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15. Targeting Misfolded SOD1 in ALS: A First-in-Class Antibody Shows Promise
This episode of BioBiz Buzz explores AL-S Pharma's pioneering approach to treating amyotrophic lateral sclerosis through AP-101, a first-in-class monoclonal antibody targeting misfolded SOD1 protein. Host Mike Ward speaks with Dr. Michael Salzmann, CEO of AL-S Pharma and COO of Neurimmune, and Professor Angela Genge, CMO of AL-S Pharma and Professor of Neurology at McGill University.
The conversation examines the devastating impact of ALS, a progressive neurodegenerative disease that typically leads to death within three to five years of symptom onset, and the profound unmet need for disease-modifying therapies. Dr. Salzmann explains how AP-101 was discovered using Neurimmune's proprietary Reverse Translational Medicine platform, which leverages human immune responses to identify antibodies that selectively bind misfolded and aggregated forms of SOD1 while sparing normally folded protein. Professor Genge discusses the intriguing evidence that misfolded SOD1 may play a role not only in familial ALS patients with SOD1 mutations but also in sporadic ALS patients without known genetic causes, potentially expanding the therapeutic opportunity to a much broader patient population.
The Phase 2 study enrolled 73 ALS patients, both sporadic and SOD1 mutation carriers, and met its co-primary safety and tolerability endpoints while demonstrating clinically meaningful changes in exploratory outcome measures, including survival, ventilation requirements, and neurofilament biomarkers after 12 months of treatment. The guests explore the unique collaborative ecosystem that enabled this progress, including the partnership between Neurimmune and TVM Capital Life Sciences, and discuss AP-101's Orphan Drug Designation from the FDA, EMA, and Swissmedic.
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By https://biobizbuzz.com/This episode of BioBiz Buzz explores AL-S Pharma's pioneering approach to treating amyotrophic lateral sclerosis through AP-101, a first-in-class monoclonal antibody targeting misfolded SOD1 protein. Host Mike Ward speaks with Dr. Michael Salzmann, CEO of AL-S Pharma and COO of Neurimmune, and Professor Angela Genge, CMO of AL-S Pharma and Professor of Neurology at McGill University.
The conversation examines the devastating impact of ALS, a progressive neurodegenerative disease that typically leads to death within three to five years of symptom onset, and the profound unmet need for disease-modifying therapies. Dr. Salzmann explains how AP-101 was discovered using Neurimmune's proprietary Reverse Translational Medicine platform, which leverages human immune responses to identify antibodies that selectively bind misfolded and aggregated forms of SOD1 while sparing normally folded protein. Professor Genge discusses the intriguing evidence that misfolded SOD1 may play a role not only in familial ALS patients with SOD1 mutations but also in sporadic ALS patients without known genetic causes, potentially expanding the therapeutic opportunity to a much broader patient population.
The Phase 2 study enrolled 73 ALS patients, both sporadic and SOD1 mutation carriers, and met its co-primary safety and tolerability endpoints while demonstrating clinically meaningful changes in exploratory outcome measures, including survival, ventilation requirements, and neurofilament biomarkers after 12 months of treatment. The guests explore the unique collaborative ecosystem that enabled this progress, including the partnership between Neurimmune and TVM Capital Life Sciences, and discuss AP-101's Orphan Drug Designation from the FDA, EMA, and Swissmedic.
● Feedback
● Subscribe (Get notified when new episodes are available. NO marketing!)
● Disclaimer