Praxis Precision Medicines (PRAX) investor relations material

Key clinical data and physician feedback

• Recent data from the Radiant study of Matrigene showed significant seizure reduction and favorable tolerability, generating strong excitement among clinicians at the International Epilepsy Congress.

• Physicians highlighted the rapid onset of effect, depth of response, and positive side effect profile, especially in a difficult-to-treat patient population.

• Operational learnings from Radiant led to improved patient counseling and lower discontinuation rates in ongoing Power One trials.

• No protocol or statistical plan changes were made to Power One, but operational adjustments improved trial execution.

• Feedback indicated a need for better discontinuation management, which is being addressed in current studies.

Market opportunity and commercial outlook

• The refractory epilepsy market is vast, with 1–2 million U.S. patients switching therapies annually and significant unmet needs even among those considered controlled.

• Many patients on monotherapy experience life-limiting side effects, highlighting a broader market beyond refractory cases.

• Commercial focus is shifting as new drugs demonstrate clear patient benefit, with expectations of significant market expansion.

• The company anticipates NDA submission for Matrigene after Power One and Power Two readouts, with Power Three potentially supporting a broader label.

• Strong site and investigator interest is accelerating trial enrollment and timelines.

Pipeline progress and regulatory milestones

• Power One is finalizing recruitment this year, Power Two is starting in Q3, and both may have readouts next year, supporting a near-term NDA submission.

• Power Three, a monotherapy study, is expected to enroll quickly and could provide unique label claims at launch.

• Religiogene, targeting rare epilepsies, showed a 90% reduction in seizures in the latest update and received FDA breakthrough designation.

• The registration cohort for Religiogene is substantially complete, potentially enabling accelerated approval discussions with the FDA.

• The company is targeting pediatric disease designation and aims to secure a priority review voucher by September 2026.