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Praxis Precision Medicines (PRAX) investor relations material

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

Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.
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H.C. Wainwright 27th Annual Global Investment Conference summary10 Sep, 2025

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.

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Frequently asked questions

Praxis Precision Medicines Inc. is a clinical-stage biopharmaceutical company focused on developing therapies for central nervous system (CNS) disorders. The company leverages genetic insights and a precision medicine approach to develop treatments for conditions such as epilepsy, movement disorders, and psychiatric conditions. Praxis is dedicated to addressing unmet medical needs through targeted therapies aimed at neurological circuits and pathways that are implicated in various CNS diseases. Its pipeline includes a range of investigational treatments designed to offer new therapeutic options for patients with these complex conditions. The company is headquartered in Boston, Massachusetts, and its shares are listed on the NASDAQ.

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