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To evaluate CNSA-001 for the treatment of phenylketonuria (PKU)
January 5, 2018
By: Betsy Louda
Retrophin announced a joint development agreement with Censa Pharmaceuticals to evaluate CNSA-001 for the treatment of phenylketonuria (PKU). CNSA-001 is an orally bioavailable form of a natural precursor of tetrahydrobiopterin (BH4) with the potential to provide improved phenylalanine (Phe) reduction in patients with PKU when compared to BH4.
Under the terms of the agreement, Retrophin is making a $10 million upfront payment to Censa and will provide funding for development of CNSA-001 in PKU. Censa will run the development program, which will be conducted under the oversight of a joint steering committee. Retrophin will pay certain milestone payments and obtains the exclusive option to acquire Censa upon conclusion of a specified option period, pending clinical proof of concept of CNSA-001 in PKU.
“We believe Retrophin’s specialized capabilities and focus on developing and delivering therapies for rare diseases are highly differentiated and well-suited to this important program,” said Jonathan Reis, M.D., president and chief executive officer of Censa. “The pre-clinical data supporting this program are very encouraging, and suggest that treatment with CNSA-001 may lead to higher intracellular BH4 levels and improved phenylalanine reduction. We look forward to working with Retrophin to advance CNSA-001 through clinical proof of concept as expeditiously as possible.”
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