PTC Therapeutics, Inc.. (NASDAQ: PTCT) today announced that it has successfully completed the acquisition of Censa Pharmaceuticals, Inc., a biopharmaceutical company 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.
“We’re thrilled to complete the acquisition of Censa,” said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. “This acquisition adds a well-established late-stage PKU clinical program to our growing rare disease portfolio. Given PTC’s established development and commercial capabilities, we’re well-positioned to bring this therapy to patients with unmet medical need expeditiously.”
With the acquisition, PTC adds CNSA-001 to its robust pipeline of treatments for rare disorders. CNSA-001 has been pursued as a possible treatment for orphan metabolic diseases associated with defects in the tetrahydrobiopterin biochemical pathways. CNSA-001 is an oral formulation of synthetic sepiapterin, a precursor to intracellular tetrahydrobiopterin, which is a critical enzymatic cofactor involved in the metabolism and synthesis of numerous metabolic products. The Phase 2 trial evaluating CNSA-001 for PKU met its primary and secondary endpoints in December 20191, and the program is Phase 3 ready.
1 – Bratkovic et al., A Phase 2 Randomized, Double Crossover, Open-label, Active Controlled Study Of CNSA-001 In Patients With Phenylketonuria, American College of Medical Genetics and Genomics (ACMG) Annual Meeting 2020.
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Source: biospace