On the US stock charts, PTC Therapeutics, Inc. (NASDAQ: PTCT) shares saw a significant rise, closing the previous session at $40.77 after climbing 16.52%. This increase came after PTCT reported encouraging findings from clinical trials and progress toward obtaining regulatory clearance for vatiquinone, the company’s flagship medication candidate intended to treat Friedreich ataxia (FA).
Positive Outcomes from Clinical Studies
PTC Therapeutics (PTCT) reported significant findings from its long-term extension studies of vatiquinone, meeting pre-specified endpoints with statistically robust evidence indicating a durable treatment benefit in slowing disease progression.
The data reveals that 144 weeks of vatiquinone treatment yielded a clinically meaningful 3.7-point improvement on the modified Friedreich Ataxia Rating Scale (mFARS) compared to a matched natural history cohort from the FACOMS disease registry. This improvement translates to a 50% reduction in disease progression over three years.
Commitment to Safety and Efficacy
The extension studies corroborate the findings of the initial 72-week placebo-controlled MOVE-FA trial, demonstrating that the benefits observed in that study are sustained over the longer treatment duration. Importantly, vatiquinone maintained a strong safety profile, with no serious treatment-related adverse events reported.
This favorable safety record positions vatiquinone as a potentially significant therapeutic option, particularly for pediatric and adolescent patients, who currently lack approved treatment alternatives.
Upcoming NDA Submission and Broader Implications
PTC Therapeutics is poised to submit a New Drug Application (NDA) by December 2024, incorporating results from the placebo-controlled segment of the MOVE-FA trial. This submission will highlight significant benefits on the Upright Stability Subscale of the mFARS, the most relevant measure for younger patients.
Additionally, the NDA will feature confirmatory evidence from long-term analyses and mechanistic data showcasing the drug’s impact on disease biomarkers. Given the considerable unmet need in the Friedreich ataxia community, particularly among children, the anticipated NDA submission underscores PTC Therapeutics’ commitment to advancing treatment options for this vulnerable population.
