PTC’s Phase III Data Sets Up PKU Competition With BioMarin
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.
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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.
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.
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.