PTC Therapeutics, Inc. (NASDAQ: PTCT) jumped into competition with BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) with its acquisition of Censa Pharmaceuticals, Inc.
Censa has developed 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.
CNSA-001 has been tested 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 2019, and the program is Phase 3 ready.
BioMarin already has two drugs on the market for PKU, an injectable called Palynziq and its first PKU drug, Kuvan. Last December, CNSA-001 beat out Kuvan in a Phase 2 trial, according to FierceBiotech, and met its primary and secondary endpoints, achieving a statistically significant and clinically meaningful reduction in blood Phe levels compared to both baseline and Kuvan.
PTC Therapeutics agreed to pay $10 million upfront and up to 850,000 shares of its common stock (which closed at $48.16 on May 6, the day of the announcement).
In addition to the upfront payments, potential future consideration includes $217.5 million in development and regulatory milestones for the two most advanced programs and receipt of a priority review voucher, including $30 million to be paid in either cash or PTC common stock at our option for completing the enrollment of Phase 3 clinical trial for CNSA-001 for PKU, $109 million in development and regulatory milestones for each additional indication of CNSA-001, net sales milestones up to an aggregate amount of $160 million and a contingent value payment of a percentage of annual net sales ranging from single to low double digits.
