Praxis Precision Medicines, Inc. (NASDAQ:PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today provided a corporate update and reported financial results for the first quarter 2024.
"In the first quarter we made significant progress on all fronts. We strengthened our financial resources and reported positive photo paroxysmal response (PPR) results in PRAX-628, which we believe further de-risks our clinical development program and positions PRAX-628 as the first precision sodium channel modulator for focal onset seizures. The strength and consistency observed across both study arms, combined with a favorable tolerability and safety profile, builds confidence in our planned focal onset seizure studies for PRAX-628, with the first expected to initiate in the second half of 2024," said Marcio Souza, president and chief executive officer of Praxis. "Looking ahead, we are encouraged by the strong participation we are seeing in our Essential3 Phase 3 studies and remain on track to read out topline results in the second half of this year. These advancements, through patient-guided development strategies, will further position Praxis at the forefront of precision medicine for CNS disorders."
Recent Highlights and Anticipated Milestones:
Cerebrum™ Small Molecule Platform
- Ulixacaltamide for ET: Essential3, the Phase 3 program for ulixacaltamide, continues to progress through patient qualification, enrollment and trial execution, with topline results expected in the second half of 2024 to support a planned New Drug Application (NDA) submission in 2025.
- Essential3 is comprised of two simultaneous Phase 3 studies including a 12-week, parallel design study and a 12-week randomized withdrawal study for stable responders.
- Since beginning recruitment in November 2023, there have been over 50,000 pre-screening forms and over 7,000 referrals received to date meeting pre-qualifying eligibility criteria. Randomization pace is now dictated by appropriate patient selection and pre-randomization stability and severity criteria.
- At the American Academy of Neurology 2024 Annual Meeting, Praxis shared additional posters on the Essential3 study design [link] and the benefits of ulixacaltamide over propranolol as analyzed from the Essential1 study [link].
- PRAX-628 for Focal Onset Seizures: Praxis reported positive results, with 100% of patients responding in a Phase 2a proof of concept study evaluating PRAX-628 in epilepsy patients with PPR and confirmed plans to initiate two efficacy studies in focal onset seizures.
- The PPR study had two cohorts, where all patients responded.
- In the 15 mg cohort (n=5), 80% of patients achieved a complete response and 20% achieved a partial response.
- In the 45 mg cohort (n=3), 100% of patients achieved a complete response.
- Safety was consistent with the prior dose escalation study and pharmacokinetic analysis confirmed therapeutic exposures.
- Praxis plans to initiate two efficacy studies in focal onset seizures.
- The first study is expected to begin in the second half of 2024, with topline results expected in 2025.
- The second study is expected to initiate in the first half of 2025, with topline results expected in the first half of 2026.
- The PPR study had two cohorts, where all patients responded.
- PRAX-562 for SCN2A and SCN8A DEEs: Praxis expects topline results from the PRAX-562 Phase 2 EMBOLD study for the treatment of pediatric patients with DEEs in the third quarter of 2024.
- The EMBOLD study is a randomized, double-blind, placebo-controlled Phase 2 clinical study to evaluate the safety, tolerability, efficacy (motor seizure frequency) and pharmacokinetics of PRAX-562 in pediatric patients aged 2 to 18 years with DEEs, followed by an open-label extension.
Solidus™ Antisense Oligonucleotide (ASO) Platform
- Elsunersen (PRAX-222) for SCN2A Gain-of-Function (GoF) DEEs: Praxis is completing multiple global regulatory interactions in the first half of 2024 in anticipation of starting the pivotal phase of the program later in 2024.