PTC Therapeutics provided positive updates on October 8, 2024, regarding its vatiquinone Friedreich ataxia (FA) program, including statistically significant results from long-term extension studies. Analysis of the MOVE-FA long-term extension study showed a 3.7-point benefit (p<0.0001) on the modified Friedreich Ataxia Rating Scale (mFARS) over 144 weeks of treatment.
This benefit represents a clinically meaningful 50% slowing in disease progression over three years when compared to a matched natural history cohort. Additionally, an earlier open-label study in adults with FA demonstrated a 4.8-point benefit on the mFARS over 24 months (p<0.0001) relative to a matched natural history population.
The company confirmed its plan to submit the vatiquinone New Drug Application (NDA) to the FDA in December 2024. The NDA will include results from the placebo-controlled MOVE-FA study, which showed a statistically significant effect on the mFARS upright stability subscale, and confirmatory evidence from these long-term analyses.
The content on BeyondSPX is for informational purposes only and should not be construed as financial or investment advice. We are not financial advisors. Consult with a qualified professional before making any investment decisions. Any actions you take based on information from this site are solely at your own risk.