Quince Therapeutics announces The Lancet Neurology publication of Phase 3 ATTeST clinical trial data evaluating EryDex for the treatment of ataxia-telangiectasia
- Phase 3 ATTeST trial evaluated EryDex for ataxia-telangiectasia (A-T) in 175 participants across 22 institutions in 12 countries.
- The trial was randomized, double-blind, and placebo-controlled, focusing on safety and efficacy of EryDex.
- Primary efficacy endpoint measured by change in mICARS score; high-dose EryDex showed reduced neurological symptoms but was not statistically significant overall.
- Subgroup analysis showed significant reduction in symptoms for ages six to nine, with a -2.8 and -4.4 change in mICARS in mITT and per-protocol populations, respectively.
- Quince is enrolling the Phase 3 NEAT study, with results expected in Q4 2025 and potential regulatory submissions in 2026.
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uniQure announces dosing of first patient in phase I/IIa clinical trial of AMT-191 for the treatment of Fabry disease
- uniQure has dosed the first patient in a Phase I/IIa clinical trial of AMT-191 for Fabry disease.
- The trial is a multi-center, open-label study in the U.S. with two dose-escalating cohorts.
- AMT-191 uses AAV5-based gene therapy to deliver a galactosidase alpha (GLA) transgene targeting the liver.
- The trial will assess safety, tolerability, and early signs of efficacy over a 24-month period.
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