January 16, 2025 - 🧬 [nGram] Today’s News Scoop: Anavex's Alzheimer's Data & GenSight's 5-Year Gene Therapy Outcomes


  1. Anavex Life Sciences publishes phase IIb/III data on blarcamesine for Alzheimer's
    • Anavex Life Sciences announced the publication of phase IIb/III trial results for oral blarcamesine in The Journal of Prevention of Alzheimer’s Disease.
    • The trial showed a 36.3% reduction in clinical progression of early Alzheimer's disease over 48 weeks, with a 49.8% reduction in the SIGMAR1 wild-type gene group.
    • Blarcamesine, an oral treatment, demonstrated a favorable safety profile and could serve as an alternative to injectable anti-beta amyloid drugs.
    • The data supports a Marketing Authorization Application (MAA) currently under review by the European Medicines Agency (EMA).
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  2. GenSight Biologics publishes 5-year outcomes for LUMEVOQ gene therapy
    • GenSight Biologics announced 5-year follow-up data for LUMEVOQ gene therapy in treating Leber Hereditary Optic Neuropathy (LHON).
    • The study showed sustained bilateral improvement in Best-Corrected Visual Acuity (BCVA) and a good safety profile over 5 years.
    • 66.1% of participants achieved clinically meaningful improvement in at least one eye, with 80.6% having on-chart vision.
    • The therapy was well-tolerated, with most ocular events being mild and no severe or serious events reported.
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