In another strategic M&A deal announced so far this month, PTC Therapeutics (Nasdaq: PTCT) has entered into an agreement to acquire fellow USA-based rare metabolic disease drug developer Censa Pharmaceuticals.
Censa is focused on the development of CNSA-001 (sepiapterin), a clinical-stage investigational therapy for orphan metabolic diseases, including phenylketonuria (PKU) and other diseases associated with defects in the tetrahydrobiopterin (BH4) biochemical pathways diagnosed at birth. The transaction was approved by the boards of both companies.
"Results from a Phase II clinical trial of CNSA-001 demonstrated significant and clinically relevant reductions in phenylanaline levels compared to current first-line treatment," said Stuart Peltz, chief executive of PTC Therapeutics, adding: "We believe that CNSA-001 has the potential to address the majority of PKU patients whose condition is not adequately managed by current treatments. We look forward to initiating a Phase III study in PKU so that patients diagnosed with this devastating condition can have a new oral treatment option as soon as possible."
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