According to a story from Xconomy, the drug company PTC Therapeutics has come to an agreement that will allow it to acquire Censa Pharmaceuticals and its lead product candidate. This drug candidate, designated CNSA-001, is in development as a treatment for the rare metabolic disorder phenylketonuria (PKU). The therapy has seen significant progress in its development so far, having successfully completed phase 2 clinical trials. Now, it is ready to proceed to phase 3.
About Phenylketonuria (PKU)
Phenylketonuria (PKU) is a type of metabolic disorder affecting the enzyme phenylalanine, which builds up in the body and can cause serious problems. Normally, phenylalanine is broken down, but a mutation affecting the PAH gene prevents this from happening in patients with the disorder. Prompt treatment is essential to avoid major complications. Symptoms of phenylketonuria include small head size, low birth weight, an unusual musty odor, pale skin, behavioral problems, intellectual disability, heart issues, seizures, and mental disorders. All of these symptoms can be prevented with proper and timely treatment; phenylketonuria is routinely screened for in newborns for this reason. The primary management strategy is a highly controlled diet that is low in phenylalanine. Supplements may also be considered. Most patients eventually go off the diet when they reach adulthood. Some older patients may still have trouble managing the condition later in life. To learn more about phenylketonuria, click here.
The agreement was paid with $10 million from PTC along with 850,000 of its common stock shares; these shares are currently valued at around $41 million in total. In addition, $217.5 million was tied into the agreement, which encompassed the current development of two of Censa’s most advanced projects as well as a priority review voucher.
CNSA-001, which is designed as an orally available formulation of synthetic sepiapterin, was able to surpass the performance of the only currently approved oral treatment for the disorder. At this juncture, there are only two approved treatments for phenylketonuria, both of which having been developed by BioMarin. This company is also in the midst of developing a third treatment. CNSA-001 is also in phase 1 trials as a possible treatment for another rare disorder: BH4 deficiency.
PTC is counting on CNSA-001 to continue to perform well in the upcoming phase 3 tests.