PTC Misses Phase III In Friedrich Ataxia, But Isn’t Giving Up

Company Ends Gene Therapy R&D, Cuts Headcount

 
• By Joseph Haas
  
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Pointing to data showing benefit in patients who completed 72 weeks of therapy, PTC thinks vatiquinone still could be viable for pediatric FA patients. Restructuring won’t affect Upstaza.

PTC
PTC maintains hopes in FA, exits gene therapy R&D • Source: Shutterstock

Despite missing the primary endpoint in a top-line readout of its Phase III study testing vatiquinone in Friedrich ataxia, PTC Therapeutics, Inc. thinks it has much to discuss about the dataset with regulators since the trial met multiple secondary endpoints and a pre-specified analysis of patients who completed the 72-week treatment regimen showed a 75% slowing of disease progression. But the biotech also announced on 23 May that it will end gene therapy R&D and cut staff by about 15% following a strategic portfolio review.

Key to PTC’s appeal to regulators will be the unmet medical need in FA, a physically debilitating and life-shortening neuromuscular...

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