PTC Eyes Transition From Capital-Raising To Money-Making Company
With $1.1bn in cash plus revenue from its existing commercial products and royalties from Roche’s sales of newly approved Evrysdi to invest in R&D, PTC Therapeutics may achieve profitability in 2023.
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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.
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.
Brazil has established an accelerated pathway for approval of drugs for ultra-rare diseases, encouraging development activity – but the definition of qualifying drugs is narrow. Expansion to include more treatments could boost clinical trials activity and companies’ overall development presence.