Praxis Precision Medicines, Inc. Q3 FY2024 earnings call

• Phase 3 study in Essential Tremor for ulixacaltamide continues, with interim analysis update to Q1 2025. Both studies are well-powered. - Relutrigine in Phase 2 EMBOLD trial in SCN2A and SCN8A-DEEs showed 46% reduction in motor seizures, with one-third of patients seizure-free; second registrational cohort initiated. - Vormatrigine's ENERGY clinical program includes EMPOWER study with over 1,000 patients registered, RADIANT and POWER1/2 trials on track. - Relutrigine has orphan and rare pediatric designations for DEEs, working on EMERALD study protocol. - Elsunersen dosing patients in Brazil for EMBRAVE study.

No specific product segment financials with absolute revenue and contribution % were detailed in the transcript. The focus was on program advancements across various therapeutic areas.

• 2025 has inflection points with potential NDA submissions. - Focus on execution to advance registrational programs and deliver precision therapies for CNS disorders.

Forward-looking statements made may not materialize as expected. Risks and uncertainties associated with the business are detailed in Praxis' most recent SEC filings.

Q: Ritu Baral with TD Cowen asked about relutrigine DEE cohort enrollment criteria and EMERALD timing.

A: Marcio Souza said second cohort start dose is 1 mg/kg per day vs previous 0.5 mg/kg, no major inclusion change; EMERALD protocol alignment ongoing.

Q: Yasmeen Rahimi with Piper Sandler asked about ET interim analysis timing and Parkinson's restart.

A: Marcio Souza explained interim analysis timing due to data cleaning and Study 2 alignment; Parkinson's study restart planned based on ET success.

Q: Joon Lee with Truist Securities asked about ET enrollment, relutrigine pain potential.

A: Marcio Souza discussed ET enrollment randomization rate and relutrigine pain preclinical evidence.

Q: Francois Brisebois with Oppenheimer asked about ET interim and DEE commercial potential.

A: Marcio Souza talked about ET interim scenarios and DEE commercial modeling (70% US, 30% ex-US).

Q: Yatin Suneja with Guggenheim asked about ET enrollment and Elsunersen global study.

A: Marcio Souza discussed ET enrollment randomization rate and Elsunersen safety exploration.

Q: Douglas Tsao with H.C. Wainwright asked about vormatrigine pain work and Elsunersen global study.

A: Marcio Souza talked about vormatrigine pain work progress and Elsunersen global study protocol.

Q: Kambiz Yazdi with Jefferies asked about ET placebo control and vormatrigine generalized epilepsy.

A: Marcio Souza discussed ET placebo control measures and vormatrigine generalized epilepsy potential.

Q: Ami Fadia with Needham asked about ET enrollment, vormatrigine RADIANT, DEE study assumptions.

A: Marcio Souza talked about ET enrollment, vormatrigine RADIANT patient mix, and DEE study regulatory assumptions.

Q: Joel Beatty with Baird asked about ET interim results and relutrigine DEE scope.

A: Marcio Souza answered on ET interim result timing and relutrigine DEE efficacy scope.

Q: Laura Chico with Wedbush Securities asked about ET interim, Study 2, and Parkinson's restart.

A: Marcio Souza discussed ET interim range, Study 2 influence, and Parkinson's restart rationale.