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 disorder with an estimated patient base of about 25,000 people globally. While Reata Pharmaceuticals, Inc. obtained US Food and Drug Administration approval of Skyclarys (omaveloxolone) in February for the disease, it is indicated for patients 16 and older. FA tends to onset in childhood or adolescence, and PTC stressed on a 23 May call that its study enrolled patients between ages 7 and 21
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