Praxis Precision Medicines and UCB Announce Epilepsy Research Collaboration

Praxis Precision Medicines and UCB announced a strategic collaboration based upon Praxis’ PRAX-020 program for the discovery of small molecule therapeutics as potential treatments of KCNT1 related epilepsies, an ultra-rare, infant-onset seizure disorder caused by mutations in the KCNT1 gene and for which there are currently no approved therapies.

Photo: Marcio Souza, president and CEO of Praxis

“Our internal research efforts give us confidence that small molecules can selectively inhibit the KCNT1 channel, and potentially could be an effective treatment for individuals suffering from KCNT1 related epilepsy, said Marcio Souza, president and CEO of Praxis. “The collaboration with UCB validates this approach and will allow us to accelerate efforts toward a potential treatment for KCNT1 patients.”

Under the terms of the collaboration, UCB retains an exclusive option to in-license global development and commercialization rights to any resulting KCNT1 small molecule development candidate. Praxis will receive an upfront payment from UCB, and if the option is exercised by UCB, would be eligible to receive an option fee and future success-based development and commercialization milestone payments, for a total of up to approximately $100 million, in addition to tiered royalties on net sales of any resulting products from the collaboration. Further financial details of the agreement were not disclosed.

“Praxis’ genetics-driven approach has led to compelling preclinical proof of concept, and we look forward to progressing this novel research program together for the benefit of individuals and families affected by this devastating form of epilepsy,” said Dhaval Patel, chief scientific officer of UCB.

Author: Rare Daily Staff