January 8, 2025 - 🧬 [nGram] Today’s News Scoop: FDA Alzheimer's Study, Solid Biosciences Gene Therapy, AbbVie Opt-in

1. FDA accepts final protocol for pivotal phase 3 Alzheimer’s disease study

   • Annovis Bio's updated protocol for a pivotal Phase 3 Alzheimer's study has been accepted by the FDA.
   • The study integrates a 6-month symptomatic trial and an 18-month disease-modifying trial into a single 6/18-month trial.
   • The 6-month data will focus on symptomatic effects, potentially supporting a New Drug Application (NDA) filing.
   • The trial aims to assess the long-term disease-modifying potential of the drug buntanetap.
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2. Solid Biosciences receives FDA IND clearance for dual route gene therapy for Friedreich’s ataxia

   • SGT-212 is a novel gene therapy targeting both neurologic and cardiac symptoms of Friedreich’s ataxia.
   • The therapy uses a dual route of administration, delivering treatment directly to the cerebellum and heart.
   • FDA clearance allows for a Phase 1b clinical trial expected to start in the second half of 2025.
   • The trial will assess safety and tolerability in both non-ambulatory and ambulatory adult patients.
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3. Angelini Ventures co-leads €20 million Series A financing in Neumirna Therapeutics

   • Angelini Ventures and Invivo Partners co-lead a €20 million Series A round in Neumirna Therapeutics.
   • Funding will support the clinical development of NMT.001, an ASO therapy for drug-resistant epilepsy.
   • Proceeds will also expand Neumirna's R&D capabilities and drug discovery platform.
   • Neumirna aims to develop RNA therapies for neurological disorders like epilepsy and Parkinson’s disease.
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4. Azafaros receives regulatory designations for phase 3 studies

   • Azafaros' lead asset, nizubaglustat, granted orphan drug designation in the US and EU for GM1 gangliosidosis.
   • Clinical Trial Application approved for global Phase 3 studies in GM1/GM2 gangliosidoses and Niemann-Pick Type C.
   • Phase 3 trials expected to start in Q2 2025, following positive Phase 2 results showing safety and early efficacy.
   • Orphan drug designations provide market exclusivity and reduced regulatory fees, aiding rare disease treatment development.
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5. Capsida announces AbbVie opt-in for first genetic medicine program from neurodegenerative disease collaboration

   • AbbVie has opted into the first neurodegenerative disease program with Capsida, triggering a $40 million license payment.
   • The decision was based on successful primate studies using Capsida's AAV capsids for targeted delivery to the brain.
   • AbbVie will advance the program into IND-enabling studies and clinical trials, while Capsida handles manufacturing.
   • Capsida is eligible for additional milestones and royalties as part of the ongoing collaboration.
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6. LB Pharmaceuticals announces positive topline results from Phase 2 trial of LB-102 in schizophrenia

   • The Phase 2 trial, NOVA1, involved 359 patients with acute schizophrenia and met its primary endpoint.
   • LB-102 showed statistically significant reductions in PANSS total score at all dose levels compared to placebo.
   • The 50 mg, 75 mg, and 100 mg doses demonstrated effect sizes of 0.61, 0.41, and 0.83, respectively.
   • LB-102 was generally safe and well-tolerated, with plans to advance to Phase 3 and explore additional indications.
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