Praxis Precision Medicines (PRAX) Jefferies Global Healthcare Conference 2025 summary

Clinical development updates

• Vormatrigine is being developed for focal onset and generalized epilepsies, with two ongoing studies: RADIANT (readout mid-year) and POWER1 (readout by year-end), and POWER2 expected next year.

• RADIANT is an open-label study designed to inform pharmacology and dose selection, with high screen failure rates ensuring a rigorous patient population.

• POWER1 and POWER2 leverage a unique patient recruitment initiative, with about 30% of participants sourced from a proprietary database, accelerating enrollment.

• Vormatrigine aims for a registration package completion within 12–18 months, targeting a large, undertreated adult epilepsy market.

• Safety events are expected to plateau early, with most adverse events occurring in the first two weeks of treatment.

Differentiation and market potential

• Vormatrigine and relutrigine are both potent sodium channel modulators, but vormatrigine is tailored for adults with epilepsy, offering convenient dosing and a clean drug-drug interaction profile.

• Relutrigine is developed for pediatric developmental and epileptic encephalopathies, formulated as a suspension for flexible dosing and targeting orphan drug markets.

• Both molecules have demonstrated significant seizure reduction, with EMBOLD results showing up to 90% reduction after 11 weeks.

• Inclusion of patients on background sodium channel blockers in studies is supported by preclinical and clinical data showing efficacy regardless of background therapy.

Relutrigine program highlights

• Relutrigine's initial focus was on ultra-rare SCN2A and SCN8A mutations, showing over 40% seizure reduction and improvements in communication and behavior.

• Ongoing cohorts are being added for registration purposes, with results expected by early next year.

• The EMERALD study, now enrolling globally, targets a broader and less severe patient population and is expected to enroll rapidly.

• Placebo response rates in pediatric epilepsy trials are typically very low, supporting robust efficacy assessments.