Praxis Precision Medicines (PRAX) Corporate presentation summary

Mission and strategic vision

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

• Aims to launch four CNS products with blockbuster potential by 2028, supported by a robust late-stage pipeline.

• Projects a portfolio peak sales potential exceeding $20B, with cash runway into 2028.

Pipeline and innovation platforms

• Pipeline includes ulixacaltamide (essential tremor), relutrigine (DEEs), elsunersen (early onset SCN2A DEE), and vormatrigine (epilepsy).

• Utilizes Cerebrum™ (small molecule) and Solidus™ (ASO) platforms for precision CNS drug discovery.

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

Clinical and commercial highlights

• Ulixacaltamide demonstrated significant functional improvement in essential tremor, with positive Phase 3 results and PDUFA date in January 2027.

• Relutrigine showed robust seizure reduction in DEEs, with NDA accepted and PDUFA in December 2026.

• Elsunersen achieved marked seizure reduction in early onset SCN2A DEE, with ongoing pivotal trial.

• Vormatrigine delivered best-in-disease efficacy in epilepsy, with broad applicability and favorable tolerability.