PTC Therapeutics to Acquire Censa Pharma for Mid-Stage Rare Disease Drug
Summary
PTC Therapeutics has reached an agreement to acquire Censa Pharmaceuticals and its lead asset, an experimental metabolic disorder therapy that is now being prepared for a pivotal study. The deal, announced after the market close Wednesday, will bring South Plainfield, NJ-based PTC (NASDAQ: PTCT) another compound for its pipeline of rare disease drugs. Wellesley, MA-based Censa in December reported that its drug, an oral formulation of synthetic sepiapterin, or CNSA-001, met the main goal of a Phase 2 study evaluating the drug as a potential treatment for signs and symptoms associated with phenylketonuria (PKU), a genetic condition in which the body is unable to break down phenylalanine, an amino acid commonly found in many foods. This year BioMarin plans to enter the clinic with a third potential PKU treatment: a gene therapy designed to normalize blood concentration levels of phenylalanine. In a statement, PTC Therapeutics CEO Stu Peltz said: “We believe that CNSA-001 has the potential to address the majority of PKU patients whose condition is not adequately managed by current treatments.” The drug is a precursor to intracellular tetrahydrobiopterin (BH4), which plays a key role in metabolism.
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