September 25, 2024 - 🧬 [nGram] Today’s Neurology Scoop: FDA Approves New Drug, Novartis AI Collaboration, Spine BioPharma Phase 3

1. FDA approves new drug to treat Niemann-Pick disease, type C

   • The FDA approved Aqneursa (levacetylleucine) for treating neurological symptoms of Niemann-Pick disease type C (NPC) in patients weighing at least 15 kg.
   • NPC is a rare genetic disease causing progressive neurological symptoms and organ dysfunction, with an average life expectancy of 13 years.
   • The approval was based on a 24-week crossover study involving 60 patients, showing better outcomes in the functional SARA score for those treated with Aqneursa.
   • Aqneursa received Priority Review, Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA.
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2. Generate:Biomedicines announces multi-target collaboration with Novartis to discover and develop protein therapeutics with generative AI

   • Generate:Biomedicines and Novartis have entered a multi-target collaboration to develop protein therapeutics.
   • The collaboration leverages Generate’s generative AI platform to create novel molecules.
   • Generate will receive $65 million upfront, including $15 million in equity, and is eligible for over $1 billion in milestone payments.
   • The collaboration aims to accelerate drug discovery and development across multiple disease areas.
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3. Spine BioPharma completes enrollment for phase 3 trial of SB-01 for chronic low back pain

   • Spine BioPharma has completed enrollment for its Phase 3 MODEL trial of SB-01 for chronic low back pain.
   • The trial enrolled 417 patients across 30 U.S. sites over two years.
   • SB-01 is the first intradiscal pharmacologic treatment to enter Phase 3 for chronic low back pain due to degenerative disc disease (DDD).
   • The trial aims to establish the safety and effectiveness of SB-01 in adult patients with lumbar DDD.
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4. Aviceda Therapeutics completes enrollment of Phase 2b SIGLEC study assessing the safety and efficacy of AVD-104 for geographic atrophy secondary to AMD

   • Aviceda Therapeutics has completed enrollment for the Phase 2b SIGLEC study.
   • The study compares AVD-104 with avacincaptad pegol (ACP) in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
   • Phase 2b is a multi-center, double-masked, randomized, controlled trial with approximately 300 participants.
   • Primary endpoint: difference in growth rate of GA area at 12 months between AVD-104 and ACP.
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5. Hesperos supports phase II clinical trial with human-on-a-chip technology

   • Hesperos' Human-on-a-Chip technology aided Dianthus Therapeutics' IND submission to the FDA for a Phase II trial of DNTH103.
   • DNTH103 is a monoclonal antibody targeting the classical pathway of the complement system for treating generalized myasthenia gravis (gMG).
   • Hesperos' neuromuscular junction model showed DNTH103 improves nerve cell transmission and reduces muscle fatigue in gMG patient serum.
   • Data were presented at the AAN Annual Meeting and the EAN Congress, supporting the potential role of DNTH103 in treating gMG.
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