PTC Confident About Commercial Prospects For Gene Therapy Upstaza
One-Time Injection Gets CHMP Nod For AADC Deficiency
PTC CEO Stuart Peltz tells Scrip that cost watchdogs are likely to look favorably on the firm’s transformational gene therapy for a distressing rare brain disorder in children who could not even lift their heads before treatment.
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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.
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.
Public Company Edition: PTC will get $350m up front as part of an initial $500m commitment. Also, while Biohaven and Vaxcyte launched traditional follow-on offerings, Recursion and Agios took the private placement and royalty sale routes, while others revealed job cuts and other strategic updates.