10:55 AM EDT, 10/08/2024 (MT Newswires) -- PTC Therapeutics ( PTCT ) said Tuesday that two long-term extension studies of vatiquinone, intended for the treatment of a rare neurodegenerative disorder called Friedreich ataxia, yielded "statistically significant" results.

According to the company, the pre-specified endpoint for the two long-term extension studies was met, with "highly statistically significant evidence of durable treatment benefit on disease progression."

In one of the extension studies, results showed that the slowing of disease progression recorded in the 72-week trial are maintained over 144 weeks of treatment, which represents a 50% slowing in disease progression over three years.

The analyses also showed that the vatiquinone treatment continues to be safe and well-tolerated.

The company said the "positive" results will be included in a new drug application to be submitted to the US Food and Drug Administration in December.

Shares of PTC were more than 8% higher in recent trading.

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