September 23, 2024 - 🧬 [nGram] Today’s Neurology Scoop: Biohaven's Troriluzole, FDA Niemann-Pick Approval, Teva's Schizophrenia Data

1. Biohaven achieves positive topline results in pivotal study of troriluzole in spinocerebellar ataxia (SCA)

   • Troriluzole 200 mg met the primary endpoint in the f-SARA scale at 3 years across all genotypes.
   • Statistically significant improvements were observed at 1 and 2 years of treatment.
   • SCA patients treated with troriluzole showed a 50-70% slowing of disease progression.
   • Biohaven plans to submit a New Drug Application (NDA) to the FDA in Q4 2024, eligible for priority review.
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2. FDA approves first treatment for Niemann-Pick disease, type C

   • The FDA has approved Miplyffa (arimoclomol) for treating Niemann-Pick disease, type C (NPC).
   • Miplyffa is used in combination with miglustat to treat neurological symptoms in patients aged 2 and older.
   • The approval was based on a 12-month randomized, double-blind, placebo-controlled trial involving 50 patients.
   • Miplyffa showed efficacy in slowing disease progression as measured by the R4DNPCCSS score.
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3. Neu-REFIX Beta glucan receives rare pediatric disease and orphan drug designations from the US FDA for treatment of Duchenne muscular dystrophy

   • Neu-REFIX Beta glucan has received Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) from the US FDA for Duchenne muscular dystrophy (DMD).
   • The designations will facilitate the transition of pre-clinical and clinical studies from Japan and India to clinical trials in the USA.
   • Neu-REFIX Beta glucan has shown potential in reducing skeletal muscle inflammation, enhancing muscle regeneration, and improving plasma dystrophin levels in DMD patients.
   • The product is commercially available in Japan since 2018 and is not a drug or remedy but a food supplement.
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4. Medincell’s partner Teva presented new efficacy, safety, and tolerability data from pivotal phase 3 of olanzapine LAI for adult patients diagnosed with schizophrenia

   • Teva presented new positive efficacy, safety, and tolerability results for Phase 3 SOLARIS trial at ECNP 2024.
   • The trial evaluated Olanzapine LAI (TV-‘749 / mdc-TJK) in adult patients with schizophrenia.
   • Three doses were evaluated, aligning with the current oral olanzapine doses, simplifying the switch for physicians.
   • No new safety signals were identified, and no PDSS observed, addressing a major barrier to the use of intramuscular olanzapine LAI.
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5. Teva presents new phase 3 data from SOLARIS trial evaluating TEV-749 for schizophrenia

   • Teva announced positive efficacy, safety, and tolerability results for TEV-749 from the Phase 3 SOLARIS trial.
   • TEV-749 met the primary endpoint, showing significant improvements in PANSS total score from baseline to week 8.
   • No incidence of post-injection delirium/sedation syndrome (PDSS) was observed in the study participants.
   • The safety profile of TEV-749 was consistent with other oral olanzapine options, with no new safety signals identified.
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6. Skye Bioscience comments on Monlunabant Phase 2 top-line data and reiterates confidence in Nimacimab clinical development plan

   • Monlunabant met its primary endpoint with a 6% placebo-adjusted weight loss at 16 weeks.
   • Skye highlights the safety advantages of their large-molecule CB1 inhibitor, Nimacimab, over small-molecule approaches.
   • Nimacimab has shown minimal brain accumulation and no neuropsychiatric adverse events in preclinical and Phase 1 studies.
   • Skye's Phase 2 trial of Nimacimab in obesity, launched in August 2024, will report interim data in Q2 2025 and top-line data in Q4 2025.
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