Scholar Rock submits BLA to the U.S. FDA for apitegromab as a treatment for spinal muscular atrophy
- Scholar Rock has submitted a Biologics License Application (BLA) to the U.S. FDA for apitegromab, a therapy for spinal muscular atrophy (SMA).
- The submission is based on positive data from the Phase 3 SAPPHIRE trial, which showed a significant improvement in motor function.
- Apitegromab has received Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA.
- Scholar Rock plans to initiate the Phase 2 OPAL trial in mid-2025 to study apitegromab in SMA patients under two years of age.
Read more
MindMed announces first patient dosed in Panorama, the second pivotal phase 3 study of MM120 in generalized anxiety disorder
- MindMed has dosed the first patient in the Panorama study, a Phase 3 trial for MM120 ODT in generalized anxiety disorder (GAD).
- The study will enroll approximately 250 participants across the US and Europe, evaluating MM120 ODT's efficacy and safety against placebo.
- Panorama is a 52-week study with a 12-week double-blind period followed by a 40-week open-label extension.
- The trial builds on Phase 2b results, which showed significant improvements in anxiety symptoms, and has received Breakthrough Therapy Designation from the FDA.
Read more
Lundbeck announces positive results from 12-month open-label extension of the PACIFIC trial evaluating bexicaserin
- Bexicaserin achieved a 59.3% median reduction in countable motor seizures over a 12-month period.
- The study involved participants aged 12-65 with Developmental and Epileptic Encephalopathies (DEEs).
- Favorable safety and tolerability were observed, with 92.7% of participants completing the study.
- The full results are expected to be presented at a medical conference in 2025.
Read more
