PTC Misses Phase III In Friedrich Ataxia, But Isn’t Giving Up
Company Ends Gene Therapy R&D, Cuts Headcount
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.
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By agreeing to pay $7.3bn for Reata and its Friedrich’s ataxia drug Skyclarys, “recalibrating” Biogen can be more patient with launches of Alzheimer’s drug Leqembi and ALS therapy Qalsody.
PTC Therapeutics’ CEO Matthew Klein says the uncertainties of gene therapy development and commercialization have led to huge opportunity costs. After shelving PTC’s preclinical gene therapy programs, he is leveraging the company's diverse platforms and expanding its commercial efforts.
Phase III data for PTC’s sepiapterin shows statistically significant reduction of blood phenylalanine levels in patients with phenylketonuria. BioMarin has the only two approved drugs for PKU.