Praxis Precision Medicines, Inc.

Praxis Precision Medicines, Inc. is a clinical-stage biopharmaceutical company dedicated to developing transformative medicines for genetically defined neurological and psychiatric disorders. Headquartered in Boston, Massachusetts, the company's core business involves translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance. Their mission centers on identifying the underlying genetic drivers of complex neurological and psychiatric conditions to create highly targeted and effective treatments.

Praxis has established a diversified, multimodal CNS portfolio with four clinical-stage product candidates across movement disorders and epilepsy. Key pipeline assets include Ulixacaltamide for essential tremor, Relutrigine for SCN2A- and SCN8A-developmental and epileptic encephalopathies (DEE), Vormatrigine for adjunctive focal epilepsy and monotherapy focal epilepsy, and Elsunersen for SCN2A-DEE. The company leverages proprietary small molecule (Cerebrum™) and antisense oligonucleotide (Solidus™) platforms to discover and develop therapies for both rare and more prevalent neurological disorders.

Recent notable developments include the U.S. Food and Drug Administration (FDA) acceptance of a New Drug Application (NDA) for ulixacaltamide HCl for essential tremor, with a Prescription Drug User Fee Act (PDUFA) target action date of January 29, 2027. Ulixacaltamide also received Breakthrough Therapy Designation in December 2025. Additionally, the FDA accepted an NDA for relutrigine, granting it priority review with a PDUFA target action date of September 27, 2026, for SCN2A and SCN8A DEE. Positive results from the EMBRAVE Part A Trial of Elsunersen were announced in April 2026. Led by President and CEO Marcio Souza, Praxis is strategically positioning itself to transition from a clinical-stage company to a commercial player in precision neurology.