PTC Therapeutics stock surges 6% after positive updates on vatiquinone program

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Shares of PTC Therapeutics, Inc. (NASDAQ: PTCT) surged nearly 6% in early trading today, following the company’s announcement of positive updates on its vatiquinone Friedreich ataxia (FA) program. The company reported highly significant results from two long-term extension studies, showing a durable treatment benefit in slowing disease progression. This news sent the stock up as investors reacted positively to the development.

PTC Therapeutics’ CEO, Matthew B. Klein, M.D., expressed optimism about the results, emphasizing the strong safety profile of vatiquinone and its potential as a meaningful therapy, particularly for children and adolescents with Friedreich ataxia who currently lack approved treatment options. The company plans to submit a New Drug Application (NDA) to the FDA by the end of the year.

The 144-week MOVE-FA extension study demonstrated a 50% slowing of disease progression compared to a matched cohort, with a 3.7-point benefit on the modified Friedreich Ataxia Rating Scale (mFARS). These results build upon earlier findings from a 72-week placebo-controlled trial. The treatment was also well tolerated, with no serious adverse events reported.

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As PTC continues to align with the FDA on its submission strategy, the market has responded with increased trading activity, reflecting growing optimism about vatiquinone’s potential approval.