Praxis Precision Medicines Receives Orphan Drug Designation for PRAX-562 for the Treatment of SCN2A-DEEPRAX-562 Phase 2 clinical trial for treatment of DEEs expected to initiate in first half of 2022

Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system disorders (CNS) characterized by neuronal imbalance, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to PRAX-562 for the treatment of SCN2A development and epileptic encephalopathy (SCN2A-DEE).  

“We are pleased by the FDA’s decision to grant orphan drug designation to PRAX-562 for the treatment of SCN2A-DEE,” said Marcio Souza, president and chief executive officer of Praxis. “As a company built on understanding the genetics of epilepsy, we are deeply committed to sodium channel research. The recognition by the FDA supports our comprehensive approach to developing treatment options for people living with mutations in the SCN2A gene. This now includes two differentiated and potentially complementary programs, PRAX-562 and PRAX-222, targeting SCN2A gain-of-function mutations in addition to a precision ASO targeting SCN2A loss-of-function mutations.”

PRAX-562 is a small molecule and is the first selective persistent sodium current blocker in development for the treatment of a wide range of rare CNS disorders. Praxis plans to initiate a Phase 2 clinical trial in the first half of 2022 to explore the potential for PRAX-562 to treat a range of rare pediatric DEEs, including SCN2A-DEE and SCN8A-DEE. PRAX-562 is currently being evaluated in a Phase 1 clinical trial in adult healthy volunteers. 

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