February 18, 2025 - 🧬 [nGram] Today’s Scoop: AlgoTx Phase 2 Results & Solid Biosciences' Gene Therapy Success


  1. AlgoTx announces results of its phase 2 'ACT' trial of ATX01 in chemotherapy-induced peripheral neuropathy
    • AlgoTx completed a 276-patient Phase 2 trial, 'ACT', for ATX01 targeting chemotherapy-induced peripheral neuropathy (CIPN).
    • The trial was double-blind and placebo-controlled, testing two concentrations of ATX01, with a focus on the 15% concentration.
    • ATX01 showed promising efficacy, particularly at 15%, but a high placebo effect in some sites affected overall results.
    • Future studies will explore strategies to mitigate placebo effects and further evaluate ATX01's efficacy.
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  2. Solid Biosciences reports positive initial clinical data from next-generation Duchenne gene therapy candidate SGT-003
    • Phase 1/2 INSPIRE DUCHENNE trial shows 110% average microdystrophin expression in first 3 participants.
    • SGT-003 well-tolerated in 6 participants with no serious adverse events; enrollment continues.
    • Potential cardiac benefits observed; company plans FDA meeting for accelerated approval in mid-2025.
    • Trial aims to dose 20 participants by Q4 2025, with additional trial sites expected by end of 2025.
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