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Praxis Precision Medicines Announces Positive Topline Results from PRAX-628 Phase 1 Study Enabling Best-in-Class Profile
PRAX-628 demonstrated a favorable safety and tolerability profile in healthy volunteers at concentrations more than 15-fold the MES EC50; PRAX-628 predicted
About this update from Praxis Precision Medicines, Inc.
[{"type":"text","content":"PRAX-628 demonstrated a favorable safety and tolerability profile in healthy volunteers at concentrations more than 15-fold the MES EC50; PRAX-628 predicted therapeutic range at least 3-fold wider than current market leader based on MES model Phase 1 results support preclinical profile for PRAX-628 indicating potential for best-in-class efficacy PRAX-628 Phase 2 study for treatment of focal epilepsy expected to initiate in 4Q23 BOSTON, May 11, 2023 (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 positive topline results from the PRAX-628 Phase 1 healthy volunteer study evaluating the safety, tolerability and pharmacokinetics (PK) of single ascending doses (SAD) and multiple ascending doses (MAD) of PRAX-628. PRAX-628 is a next-generation, functionally selective small molecule targeting the hyperexcitable state of sodium-channels in the brain. PRAX-628 is currently being developed as a once daily, oral treatment for adult focal onset epilepsy. “Today’s data reinforce our expectations that PRAX-628 has the potential to be the best-in-class treatment for patients suffering from focal epilepsy,” said Marcio Souza, president and chief executive officer of Praxis. “The ultimate goal of an anti-seizure medication is seizure freedom for all affected patients, and a wide therapeutic window is critical to reach that goal. Today’s results support previously announced preclinical data to demonstrate that the unique functional selectivity of PRAX-628 may lead to a wider therapeutic range than current standard-of-care and other potential treatments in development.” Summary of PRAX-628 Phase 1 Study Topline Results: In the study, PRAX-628 or placebo was administered to 40 healthy participants (n=30, placebo=10)SAD cohorts evaluated PRAX-628 doses ranging from 5 mg to 45 mg and MAD cohorts evaluated PRAX-628 doses of 20 mg and 30 mg, resulting in concentrations of more than 15-fold the mouse Maximal Electroshock Seizure model (MES) EC50PRAX-628 was generally well-tolerated at all tested dosesPK data demonstrated dose-dependent exposure supporting once-daily dosing without titration to achieve potentially...