Praxis Precision Medicines (PRAX) Jefferies Global Healthcare Conference summary

Key program updates

• Ulixacaltamide phase III studies for essential tremor are on track for readout by year-end, with robust patient enrollment and a focus on replicating prior study populations for consistency.

• Over 50,000 patients expressed interest in the study, with more than 7,000 pre-qualifying, far exceeding initial expectations and highlighting significant unmet need.

• The phase III trial design was refined by removing unstable performance scale items, increasing study duration to 12 weeks, and maintaining endpoints that maximize the chance of demonstrating value.

• High confidence in trial success is based on strong prior data, positive pilot studies, and alignment with regulatory guidance.

• Essential tremor market is underserved, with only one widely used drug and high discontinuation rates, underscoring the need for new therapies.

Pipeline diversification and progress

• PRAX-628 demonstrated clear target engagement in epilepsy models, supporting advancement to late-stage studies in focal onset seizures, with the first study starting this year and readout expected next year.

• Plans are in place to expand PRAX-628 into generalized epilepsy and potentially pain, leveraging its selective mechanism.

• The epilepsy market remains large and inadequately served, with 30–40% of patients uncontrolled at any time.

Developmental epileptic encephalopathies (DEEs) program

• PRAX-562 is positioned as the first disease-modifying drug for severe DEEs like SCN2A and SCN8A, with a readout expected next quarter.

• Preclinical data show seizure elimination and restoration of normal function in genetic models, and early clinical experience is promising for meaningful patient impact.