Praxis Precision Medicines (PRAX) Corporate presentation summary

Mission and strategic vision

• Focused on delivering life-altering treatments for patients with CNS disorders, aiming for faster and more effective solutions than previously achieved.

• Pipeline leverages two proprietary platforms: Cerebrum for small molecules and Solidus for antisense oligonucleotides.

Pipeline and development milestones

• Four late-stage CNS assets targeting essential tremor, developmental & epileptic encephalopathies (DEEs), and epilepsy, with two pending PDUFA dates in 2026 and 2027.

• Ulixacaltamide for essential tremor has Breakthrough Therapy Designation and a PDUFA date in January 2027.

• Relutrigine for SCN2A/8A DEEs has multiple regulatory designations and a PDUFA date in September 2026.

• Elsunersen, a first-in-class ASO for early onset SCN2A DEE, is in pivotal trials with strong efficacy and safety data.

• Vormatrigine targets focal onset seizures and generalized epilepsy, showing best-in-disease efficacy in clinical studies.

Market opportunity and commercial strategy

• Portfolio peak sales potential exceeds $20B, with each late-stage asset projected as a blockbuster.

• Commercial launch preparations underway for ulixacaltamide, including field force build-out and physician engagement campaigns.

• Pricing strategy informed by analogs in high unmet-need CNS indications, supporting significant value capture.