Retrophin Enters PKU Collaboration with Censa, Gets Option to Acquire Company

Rare Daily Staff

Retrophin said it will make an upfront payment of $10 million with Censa Pharmaceuticals as part of a joint development agreement to advance CNSA-001, Censa’s experimental treatment for PKU, a rare, genetic metabolic condition that can cause developmental and growth delays and cause mental impairment.

Phenylketonuria, or PKU, is caused by a genetic mutation that results in a deficiency in PAH, an enzyme that breaks down phenylalanine, or Phe, an amino acid present in most protein-containing foods. High Phe levels can lead to a range of growth and developmental problems.

CNSA-001 is an orally bioavailable form of a natural precursor of BH4, a co-factor for Phe that helps the body make better use of what available PAH and help reduce Phe levels.

Under the terms of the agreement, in addition to the upfront payment to Censa, Retrophin 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. As part of the agreement, Retrophin will pay certain milestone payments and obtains the exclusive option to acquire privately-held Censa upon conclusion of a specified option period, pending clinical proof of concept of CNSA-001 in PKU. Preclinical research has suggested CNSA-001 is currently being evaluated in a single ascending dose study and a phase 2 proof of concept study in PKU is expected to commence in mid-2018.

“This transaction represents a significant opportunity to further strengthen our product pipeline and expands Retrophin’s strategy of delivering life-changing therapies to individuals living with rare diseases who have limited treatment options,” said Stephen Aselage, CEO of Retrophin. “Importantly, the data generated by the program to date suggest that CNSA-001 has both the potential to offer a meaningful improvement in care for PKU patients, and the ability to move rapidly through the clinic upon positive proof of concept data.”

January 8, 2018

Photo: Stephen Aselage