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Praxis Precision Medicines Provides Updates on Clinical Stage Pediatric Epilepsy Programs
BOSTON, Sept. 07, 2022 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic
About this update from Praxis Precision Medicines, Inc.
[{"type":"text","content":"BOSTON, Sept. 07, 2022 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today announced the plan to start the PRAX-222 EMBRAVE clinical study for the treatment of pediatric patients with early-seizure-onset SCN2A developmental and epileptic encephalopathy (DEE), after the U.S. Food and Drug Administration (FDA) cleared the Investigational New Drug (IND) application for the initial dose cohort. Following collection of the safety and efficacy data from the first cohort of patients in the EMBRAVE study, the data will be evaluated and submitted to the FDA to seek authorization for further dose escalation. Praxis also announced that it intends to start the PRAX-562 Phase 2 study for the treatment of pediatric patients with SCN2A and SCN8A DEEs outside of the U.S. before the end of 2022, after the FDA placed a clinical hold on its second IND application for PRAX-562. PRAX-562 has been dosed in over 130 healthy volunteers in completed and ongoing studies, including 66 in the U.S. under an initial IND for adult rare headache conditions. Following the clearance of the initial IND last year, Praxis completed the chronic and juvenile toxicology programs and submitted a second IND to the FDA. Praxis has initiated discussions with the FDA to provide clarification about the pre-clinical and clinical data packages in relation to the clinical hold correspondence. Topline results for the PRAX-562 Phase 2 study for the treatment of pediatric patients with SCN2A and SCN8A DEEs are expected in 2023. “We are eager to start dosing in the PRAX-222 EMBRAVE study and look forward to sharing plans for the upcoming PRAX-562 Phase 2 study,” said Marcio Souza, president and chief executive officer of Praxis. “SCN2A and SCN8A DEEs are devastating forms of epilepsy with no approved treatment options, and every day matters for children living with these diseases. We are proud to offer hope to those in the SCN2A and SCN8A communities and believe that precision medicines such as PRAX-562 and PRAX-222, which are specifically designed for these conditions, have the potential to transform care of rare genetic epilepsies.” PRAX-222 is an ASO ...