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BOSTON, Jan. 12, 2025 (GLOBE NEWSWIRE) — Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a biopharmaceutical organization focused on developing treatments for disorders of the central nervous system (CNS), has provided an update on its pipeline developments and strategic goals for 2025. This year is set to be pivotal for the company as it anticipates significant milestones across its portfolio.
2024 proved to be a year of noteworthy advancements for Praxis, particularly marked by encouraging results in the Phase 2 photoparoxysmal response (PPR) trial of vormatrigine, which catalyzed the launch of the ENERGY program targeting common epilepsies. Highlights from the year included exceptional efficacy results from cohort 1 of the EMBOLD study focusing on relutrigine in developmental and epileptic encephalopathies (DEEs), along with the initiation of registrational cohort 2. The company has also observed an overwhelming interest in its Essential3 program. According to Marcio Souza, the company’s president and CEO, these achievements have positioned Praxis with three potential blockbuster programs in late-stage development, forecasting four product launches between 2026 and 2028.
Looking ahead, Souza stated, “We are well-positioned for a data-rich 2025, expecting results for ulixacaltamide in the Essential3 program, as well as topline results for vormatrigine in upcoming trials by the end of 2025. We are also optimistic about our NDA filing plans, fueled by our regulatory feedback.” The company is ensuring adequate funding to propel all its programs through to their final readouts, maintaining financial viability into 2028. The company is set to provide a comprehensive update during its Investor R&D Day in the second quarter of 2025.
Portfolio Milestones and Developments
Cerebrum Small Molecule Platform
Ulixacaltamide for Essential Tremor
Essential tremor (ET) represents a substantial healthcare challenge, affecting approximately seven million individuals across the U.S. The Essential3 program comprises two Phase 3 registrational studies aimed at validating ulixacaltamide’s efficacy. Since the initiation of patient recruitment in November 2023, over 100,000 individuals have expressed interest in participating.
Praxis anticipates an interim analysis of Study 1 in the first quarter of 2025, with subsequent announcements on the timeline for full data releases. The company plans to submit an NDA for ulixacaltamide later in 2025. Positive outcomes from this program may also prompt revitalization of research into ulixacaltamide’s application for Parkinson’s disease—a condition in dire need of innovative treatment options.
Vormatrigine (PRAX-628) for Common Epilepsies
With an estimated 3.5 million people living with common epilepsies in the U.S., the need for improved treatment options is critical. Existing medications have limitations in both safety and efficacy, making vormatrigine a potential game-changer as a potent sodium channel modulator. Early Phase 1 studies have shown promising outcomes, showcasing a strong safety profile and dose-dependent effects.
The ongoing ENERGY program seeks to further explore the performance of vormatrigine in both efficacy and registrational contexts. The RADIANT Phase 2 study expects to release topline results in the first half of 2025. Meanwhile, items such as the POWER1 study are also progressing, with results expected later in the year.
Relutrigine (PRAX-562) for Developmental and Epileptic Encephalopathies
Focusing on a spectrum of DEEs affecting roughly 200,000 patients in the U.S., relutrigine represents a breakthrough in sodium channel modulation. Results from the EMBOLD study highlight its efficacy in patients with specific genetic conditions, showing substantial reductions in seizure frequency and improved overall outcomes.
After receiving a rare pediatric disease designation for Dravet Syndrome, the company is moving forward with expanded evaluations in its registrational cohort 2. The EMBOLD study aims to clarify relutrigine’s effectiveness across broader patient demographics.
Solidus Antisense Oligonucleotide (ASO) Platform
Elsunersen (PRAX-222) for Early-Seizure-Onset SCN2A-DEE
Elsunersen targets SCN2A-related early seizure onset and has shown promise in preclinical models. Recent analyses suggest a significant reduction in seizure burden among participants in the EMBRAVE study. The company is on track to finalize trial designs in collaboration with global regulatory agencies, with a comprehensive update expected soon.
Additional Pipeline Highlights
As of December 2024, UCB has secured global development rights to a KCNT1 small molecule candidate, part of a previously established collaboration agreement. This marks a significant milestone, potentially unlocking up to $100 million in development and commercialization incentives as well as future royalties.
Praxismedicines continues to pursue early-stage ASO therapeutic developments, targeting several genetic conditions related to epilepsy and cognitive disorders, each representing considerable unmet medical needs.
Conclusion
Praxis Precision Medicines is uniquely positioned as it advances through critical phases of drug development for several CNS disorders. With a commitment to addressing significant gaps in treatment options, the company continues to leverage genetic insights to craft innovative therapies, directing its efforts towards the launch of multiple candidate products in the coming years.
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