Praxis Precision hosted investor event on clinical programs

Praxis Precision (PRAX) Medicines hosted a virtual investor event on its clinical programs in developmental and epileptic encephalopathies, DEEs, on Friday, May 2, 2025. Relutrigine: Praxis shared updated data from the initial cohort of patients in the EMBOLD study, highlighting results through 11 months of patient dosing. Praxis presented a comprehensive set of pre-clinical data spanning 10 different DEE disease models, providing compelling evidence that relutrigine’s anti-seizure mechanism is well suited for broad DEEs. Praxis plans to initiate the EMERALD registrational study in mid-2025. The company also shared its expectation that relutrigine could potentially be used in combination with any of its DEE ASO programs allowing patients to receive both therapies together for broader clinical benefit. Elsunersen: EMBRAVE Part A is enrolling up to 16 patients on a 3:1 drug to sham ratio with once-monthly intrathecal dosing for six months. Recruitment is expected to be complete by mid-year with topline results in the first half of 2026. The EMBRAVE3 registrational trial, initiating in mid-2025, will initially enroll patients ages 2-18. Subsequent cohorts will support treatment initiation from birth, reflecting the early onset of SCN2A GoF DEE which can present in-utero or shortly after birth and is often diagnosed within the first three weeks of life. Solidus pre-clinical pipeline: Praxis plans to nominate a development candidate for its early-stage ASO program PRAX-100 in mid-2025. Praxis remains on track to nominate development candidates for PRAX-080 and PRAX-090 by the end of 2025. PRAX-080 is targeting PCDH19 mosaic expression and PRAX-090 is designed to address SYNGAP1 LoF mutations.