PTC Therapeutics

At the Cantor Healthcare Conference PTC Therapeutics reported a strong SEPHIENCE launch in PKU with over 2,000 therapy-start applications in the U.S. and a global revenue potential of more than $2 billion. The company shows over $2 billion in cash and expects cash-flow breakeven in 2026. Phase‑2 data for votoplam in Huntington’s chorea demonstrated a 52% slowing of disease progression at the higher dose; a Phase‑3 study is already ongoing. PTC is also acquiring ST-920 for Fabry disease (upfront payment $111 million), which is in the BLA stage and could have peak sales potential of more than $750 million. » Plus sur de.investing.com

At the Wells Fargo conference PTC Therapeutics outlined its strategy: shifting away from older products toward a broader rare-disease portfolio. SUFIANCE (Pegvaliase) is driving growth, has reached all 103 US centers of excellence and is expected to offer more than $2 billion in revenue potential. Management reports more than $2 billion in cash, disciplined cost reductions and expects cash-flow breakeven in 2026. Meanwhile PTC is expanding its pipeline (including Votoplam, MSH3, Vatiquinon, NLRP3) and is acquiring the gene therapy ST-920 for Fabry disease ($100–110 million) for further diversification. » Plus sur de.investing.com

PTC Therapeutics purchased ST-920 from Sangamo Therapeutics in a bankruptcy auction. The deal includes an upfront payment of $111 million and up to $100 million in regulatory approval milestone payments. PTC plans to complete the BLA filing for accelerated approval in the U.S. by Q4 2026 and then pursue international approvals using its existing infrastructure. Closing remains subject to contract documentation, bankruptcy court approval and antitrust review, and is expected in late Q3/early Q4 2026. » Plus sur de.investing.com