New collaboration could advance small molecule epilepsy therapy

UCB and Praxis Precision Medicines, Inc. will collaborate to advance Praxis’ PRAX-020 small molecule programme for KCNT1 related epilepsies.

New collaboration could advance small molecule epilepsy PRAX-020 therapy

Two biopharmaceutical companies, UCB and Praxis Precision Medicines Inc., have announced their collaboration to advance potential therapeutics of KCNT1 related epilepsies, based on Praxis’ PRAX-020 small molecule programme, enabling Praxis to be potentially eligible to around $100 million in development milestones and related payments.

Under the collaboration agreement, UCB will retain an exclusive option to in-license global development and commercialisation rights to any resulting KCNT1 small molecule development candidate. If the option is exercised by UCB, it will provide Praxis with an upfront payment, making the PRAX-020 developer to be eligible to a total of up to approximately $100 million. This sum is made up of potential success-based development and commercialisation milestone payments and an option fee, in addition to tiered royalties on net sales of any resulting products from the collaboration.

Small molecule therapeutics to treat KCNT1 related epilepsy

“We are excited to partner with UCB, as we work together toward a novel approach for the treatment of KCNT1 related epilepsy, which has no approved therapies currently,” commented Marcio Souza, President and Chief Executive Officer 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,” added Souza.


Clinical-stage biopharmaceutical company Praxis Precision Medicines is translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterised by neuronal excitation-inhibition imbalance. Praxis applies insights from genetic epilepsies to both rare and more common neurological disorders, by using its understanding of shared biological targets and circuits in the brain.

Clinical success begins early for small molecule drugs