vormatrigine (PRAX-628) / Praxis Precision Medicines - LARVOL DELTA

Vormatrigine Exhibits a Favorable Drug-Drug Interaction Profile Supporting Broad Combination Use with Antiseizure Medications (AES 2025) - "Key enzymes assessed included CYP1A2, 2C19, 3A4, and UGT isoforms, along with major efflux and uptake transporters.Clinically, the Phase 1 PRAX-628-102 study enrolled healthy adults and examined the pharmacokinetics (PK) of single 10 mg doses of vormatrigine alone and in combination with fluvoxamine (CYP1A2/2C19 inhibitor), itraconazole (CYP3A4 inhibitor) and probenecid (non-selective UGT inhibitor). Vormatrigine exhibits a highly favorable DDI profile, supported by both preclinical mechanistic studies and human PK data. With no clinically meaningful interactions observed with inhibitors or inducers of major CYP and UGT pathways—except a moderate effect via CYP1A2—vormatrigine is well-suited for use in multi-drug epilepsy regimens, including a broad array of ASMs without the need for dose adjustments. These findings reinforce vormatrigine's potential as a best-in-class sodium channel modulator and support its continued clinical development in the ENERGY program for..."

CNS Disorders • Epilepsy • ABCB1 • CYP1A2

Vormatrigine Rapidly Reduces Seizures in Adults with Treatment-Resistant Epilepsy: Results from the RADIANT Study (AES 2025) - P2 | "Vormatrigine delivered rapid seizure control within 1 week, with sustained response over 8 weeks alongside unprecedented seizure reduction and freedom in the setting of modern background ASMs, including ~30% on cenobamate. Topline RADIANT study results build on preclinical and early clinical data, positioning vormatrigine as a fast-acting, precision once-daily ASM with minimal drug–drug interactions and a favorable safety profile. These results strongly support accelerated advancement of Phase 3 studies in the vormatrigine ENERGY program."

Clinical • Late-breaking abstract • CNS Disorders • Epilepsy

Praxis Precision Medicines Announces Positive, Best-in-Disease Topline Results in Patients with Focal Onset Seizures from the RADIANT Study of Vormatrigine (Praxis) - "Dosing with vormatrigine over 8 weeks led to 56.3% median reduction in seizure frequency; Approximately 22% of patients reached 100% reduction in seizure frequency in the last 28 days on treatment; Rapid and sustained response, with over 54% of patients achieving 50% response in the first week; Vormatrigine was generally well tolerated and continues to demonstrate favorable safety profile...'We are well on track to complete the pivotal, 12-week POWER1 study in Q4 and, based on the results from RADIANT, expect to initiate the POWER2 study shortly.'"

New trial • P2 data • Trial completion date • CNS Disorders

Updates from the First-in-human Phase 1 Clinical Trial Evaluating the Safety, Tolerability, Pharmacokinetics and Food Effect of Vormatrigine in Healthy Participants (P11-4.006). (PubMed, Neurology) - "Petrou has received intellectual property interests from a discovery or technology relating to health care. Marcio Souza has received personal compensation for serving as an employee of Praxis Precision Medicines."

Clinical • Journal • P1 data • PK/PD data

Vormatrigine (PRAX-628) for Focal Onset Seizures and Generalized Epilepsy (GlobeNewswire) - "Praxis has initiated the RADIANT Phase 2, open-label, single-arm study for FOS and generalized epilepsy assessing seizure burden with vormatrigine treatment, with topline results expected by mid-year 2025. Praxis has also initiated the POWER1 Phase 2/3 registrational study of vormatrigine for treatment resistant FOS, with topline results anticipated in the second half of 2025. POWER2, the second registrational study for FOS, is on track to begin enrollment in the second half of 2025."

New trial • P2 data • P2/3 data • CNS Disorders • Epilepsy

Updates from the First-in-human Phase 1 Clinical Trial Evaluating the Safety, Tolerability, Pharmacokinetics and Food Effect of Vormatrigine in Healthy Participants (AAN 2025) - "Vormatrigine demonstrated consistent safety, tolerability, and PK profiles, with no significant food effect. These results support flexible dosing regimens up to 45mg in the ongoing vormatrigine ENERGY program."

Clinical • P1 data • PK/PD data

Praxis Precision Medicines to Host PRAX-628 Program Update (GlobeNewswire) - "Praxis Precision Medicines...announced that it will host a virtual program update to discuss its PRAX-628 program in epilepsy on Tuesday, March 26, 2024 at 8:00 a.m. ET."

Clinical • CNS Disorders • Epilepsy

Praxis Precision Medicines to Present at the American Academy of Neurology 2023 Annual Meeting (GlobeNewswire) - "Praxis Precision Medicines, Inc....announced that it will deliver presentations on its clinical stage programs at the upcoming American Academy of Neurology (AAN) 2023 Annual Meeting, held April 22-27, 2023 in Boston, Massachusetts....'Also, we expect to dose the first patients in our PRAX-222 EMBRAVE study and PRAX-562 EMBOLD study within the coming weeks and are excited to share data supporting these programs. Finally, we look forward to sharing new in-vivo results for PRAX-628 that highlight this program’s potential to be a best-in-class treatment for focal epilepsy patients'."

Clinical data • Preclinical • CNS Disorders • Epilepsy

Praxis Precision Medicines Provides Corporate Update and Reports Fourth Quarter and Full Year 2022 Financial Results (GlobeNewswire) - "Topline results expected for each of three clinical-stage epilepsy programs in 2023 – PRAX-222 first-in-patient EMBRAVE Study safety data mid-2023, PRAX-628 first-in-human Phase 1 data mid-2023, PRAX-562 Phase 2 EMBOLD Study results in 2H23."

P1 data • P2 data • CNS Disorders • Epilepsy

Praxis Precision Medicines to Present at the American Epilepsy Society 2022 Annual Meeting (GlobeNewswire) - "Praxis Precision Medicines, Inc...announced that it will deliver presentations on its epilepsy programs at the American Epilepsy Society (AES) 2022 Annual Meeting, held December 2-6, 2022 in Nashville, Tennessee....'It’s an incredibly exciting time for Praxis and our epilepsy portfolio, with first-in-patient studies for PRAX-222 and PRAX-562 and a first-in-human study for PRAX-628 expected to start shortly'....Together with pharmacokinetic findings demonstrating a 13-fold increase in concentrations compared to preclinical maximal electroshock seizure effects, our results are consistent with earlier work suggesting a wide therapeutic window for PRAX-562."

New trial • P1 data • Preclinical • CNS Disorders • Epilepsy

Praxis Precision Medicines Provides Corporate Update and Reports Third Quarter 2022 Financial Results (GlobeNewswire) - "Praxis expects to initiate a PRAX-628 Phase 1 study in the fourth quarter of 2022 and subsequently initiate a Phase 2 study in focal epilepsy in 2023."

New P1 trial • New P2 trial • CNS Disorders • Epilepsy

Praxis Precision Medicines Showcases Largest Targeted Epilepsy Portfolio in Industry at 2022 Epilepsy Day - "Updates from Praxis’ three most advanced epilepsy programs, each of which is expected to be clinical stage by year-end 2022: PRAX-222: Investigational New Drug (IND) submitted to the U.S. Food and Drug Administration (FDA) proposing a seamless study design for SCN2A patients with early-onset seizures; PRAX-562:...A Phase 2 clinical study in cohorts of children with SCN2A epilepsy, SCN8A epilepsy, and Tuberous Sclerosis Complex (TSC) is expected to initiate in 2H2022; PRAX-628: Expected initiation of a Phase 1 study in the fourth quarter of 2022 and a Phase 2 study in focal epilepsy in 2023; A review of the significant progress in Praxis’ early-stage epilepsy pipeline, including plans to declare antisense oligonucleotide (ASO) candidates for PRAX-080 for PCDH19 and PRAX-090 for SYNGAP1 in 2023."

New P1 trial • New P2 trial • CNS Disorders • Epilepsy