Vesalius announces multi-target strategic alliance with GSK to develop breakthrough treatments for Parkinson's disease
- Vesalius and GSK have formed a strategic alliance to develop treatments for Parkinson's disease and another neurodegenerative condition.
- GSK gains worldwide rights to a preclinical small molecule program focused on Parkinson's disease.
- Vesalius will receive $80 million upfront, with potential milestone payments up to $570 million.
- GSK will handle development and commercialization, with Vesalius eligible for royalties.
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Neurogene reports positive interim efficacy data from NGN-401 gene therapy trial for Rett syndrome
- Neurogene announced positive interim data from the low-dose cohort of its Phase 1/2 trial for NGN-401 gene therapy in pediatric Rett syndrome patients.
- The trial showed meaningful gains in skills and developmental milestones, with improvements across key clinical domains.
- The low-dose NGN-401 demonstrated a favorable safety profile with no treatment-related serious adverse events.
- Neurogene has initiated an adolescent/adult cohort to explore NGN-401's potential in a broader patient population.
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IND for ATA-200, a gene therapy for limb-girdle muscular dystrophy, cleared by FDA
- The FDA has cleared Atamyo Therapeutics' IND application for ATA-200, a gene therapy for LGMD2C/R5, to proceed to a Phase 1b/2b clinical trial.
- The trial will be a multicenter Phase 1b open-label dose escalation study evaluating safety, pharmacodynamics, efficacy, and immunogenicity in children.
- ATA-200 has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA, granting market exclusivity and recognition of its potential impact.
- The trial is funded by The Dion Foundation and has already received regulatory clearance in France and Italy, with US patient enrollment expected in Q4 2024.
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Neumora therapeutics reports third quarter 2024 financial results and provides business update
- Phase 3 data from KOASTAL-1 study of navacaprant in major depressive disorder (MDD) expected by end of 2024.
- Ongoing studies for navacaprant in bipolar depression and NMRA-511 in Alzheimer's disease agitation, with data expected in the second half of 2025.
- Strong financial position with $341.3 million in cash, supporting operations into mid-2026.
- Plans to submit an IND for the M4 PAM program in the first half of 2025.
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