Praxis Precision Medicines (PRAX) investor relations material

Commercial strategy and market opportunity

• Essential tremor affects 2-2.5% of the U.S. population, with 1-2 million patients actively seeking treatment in a 1-3 year period.

• Current treatments for essential tremor are inadequate, creating a significant unmet need.

• Commercial launch will require a robust sales force and targeted outreach by zip code, leveraging a strong patient database and digital engagement.

• Pricing is expected in the $40,000–$60,000 per year range, with copays minimized for private payers and strategies to address Medicare complexities.

• Peak sales for ulixacaltamide are projected to exceed $6 billion, with potential for higher based on market dynamics.

Regulatory and development milestones

• Two NDAs, for ulixacaltamide and relutrigine, are on track for filing in mid-February, with both packages nearly complete.

• Breakthrough designation has been granted, and priority review is under consideration, balancing speed to market and economic benefit.

• The EMERALD study for relutrigine in developmental and epileptic encephalopathies (DEE) is progressing rapidly, with readout expected in the second half of the year.

• If positive, EMERALD could enable rapid label expansion via the STAR program, potentially broadening market access in early 2025.

Pipeline and portfolio expansion

• SCN2A and SCN8A addressable patient populations in the U.S. are estimated at 3,500–10,000, with nearly all identified due to early onset and genetic testing.

• Vormatrigine is expected to read out in the first half of the year, targeting focal onset seizures with the goal of moving from third-line to first-line therapy.

• Elsunersen is planned for NDA submission next year and is expected to complement, not cannibalize, relutrigine in SCN2A patients.

• Combination therapy is anticipated to become standard as mortality decreases and the market grows.