Praxis Precision Medicines (PRAX) H.C. Wainwright 27th Annual Global Investment Conference summary

Key clinical data and physician feedback

• Vormatrigene showed significant seizure reduction and favorable tolerability in the RADIANT study, generating strong excitement among clinicians at the International Epilepsy Congress.

• Physicians highlighted the unmet need for effective epilepsy treatments, with up to 60% of patients failing current therapies annually in the U.S.

• The rapid onset of effect and robust response rates, even in difficult-to-treat populations, were seen as major positives.

• Side effect profile was considered superior to existing options, with lower CNS side effects noted.

• Operational learnings from RADIANT led to improved patient counseling and lower discontinuation rates in ongoing POWER1.

Clinical development and timelines

• POWER1 recruitment is finishing this year, with POWER2 starting in Q3 and expected to enroll quickly.

• POWER2 enrollment may finish next year, potentially allowing for readouts and NDA submission soon after.

• POWER3, a monotherapy study, is in design phase and could deliver data next year, possibly supporting a broader label at launch.

• No protocol or statistical plan changes have been made to POWER1; operational improvements focus on patient engagement.

Market opportunity and commercial outlook

• The refractory epilepsy market is estimated at 1–2 million U.S. patients, but broader unmet needs exist even among “controlled” patients due to side effects.

• Many patients on monotherapy report significant psychiatric side effects and suboptimal seizure control.

• Commercial focus is shifting as clinical efficacy is now widely accepted, with expectations for significant market expansion.

• The company aims to include POWER3 data in the initial label to maximize commercial impact.