Praxis Precision Medicines
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Praxis Precision Medicines (PRAX) investor relations material

Praxis Precision Medicines Corporate presentation summary

Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.
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Corporate presentation summary12 Jan, 2026

Strategic vision and mission

  • Focused on delivering life-altering treatments for CNS disorders with urgency and repeatability.

  • Aims to capitalize on a late-stage portfolio with over $20B in commercial potential.

  • Utilizes two innovation platforms: Cerebrum (small molecules) and Solidus (ASO therapies).

Portfolio and pipeline highlights

  • Four lead assets: ulixacaltamide (essential tremor), vormatrigine (epilepsy), relutrigine (DEEs), and elsunersen (SCN2A DEE).

  • Multiple assets have received FDA Breakthrough Therapy, Orphan Drug, and Rare Pediatric Disease designations.

  • NDA submissions for ulixacaltamide, relutrigine, vormatrigine, and elsunersen planned for 2025–2026.

Clinical and commercial potential

  • Ulixacaltamide: First successful Phase 3 program in essential tremor, showing rapid, robust, and durable efficacy with favorable safety.

  • Vormatrigine: Demonstrated best-in-disease efficacy and tolerability in epilepsy, with rapid and sustained seizure reduction.

  • Relutrigine: Robust seizure reduction and disease-modifying potential in DEEs, with broad applicability across genetic etiologies.

  • Elsunersen: First-in-class ASO for SCN2A GoF DEE, showing significant seizure reduction and no serious adverse events.

Ulixacaltamide NDA readiness and timeline
Vormatrigine monotherapy strategy and market
Relutrigine broad DEE strategy and EMERALD
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Q4 20255 Mar, 2026
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Q4 20255 Mar, 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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