PTC Therapeutics

PTC Therapeutics reports robust sales driven by the drug Sephience and raises its full-year forecast. Wells Fargo reaffirms its buy rating and sets a price target of $112, implying roughly 74% upside. Management says Sephience’s market penetration is proceeding as planned. » Mere på finanzen.net

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. » Mere på 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. » Mere på de.investing.com