Praxis Precision Medicines has provided a corporate update and reported its financial results for the third quarter of 2024, highlighting significant progress across its clinical pipeline. The company is focused on translating genetic insights into therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance.
Ulixacaltamide for Essential Tremor
Ulixacaltamide, a selective small molecule inhibitor of T-type calcium channels, is currently in late-stage development for essential tremor (ET). The interim analysis for Study 1 of the Essential3 Phase 3 program is confirmed for Q1 2025, and an NDA filing is anticipated in 2025. Recent surveys presented at the Movement Disorder Specialist Conference indicated a high unmet need in ET, with 85% of neurologist visits focused on finding treatment and up to 80% of patients adjusting daily activities due to the disease.
Vormatrigine (PRAX-628) for Focal Epilepsy
Vormatrigine (PRAX-628), a next-generation small molecule targeting sodium channels, is being developed as a once-daily oral treatment for focal onset seizures and generalized epilepsy. The EMPOWER observational study, in partnership with the Epilepsy Study Consortium, has enrolled over 1,000 patients to better characterize seizure burden. Topline results from the RADIANT Phase 2 study are anticipated in the first half of 2025, while topline results from the POWER1 Phase 2/3 study are expected in the second half of 2025. Given vormatrigine's potency as a Nav 1.7 and 1.8 inhibitor, Praxis is also evaluating its potential in pain indications.
Relutrigine (PRAX-562) for DEEs
Relutrigine (PRAX-562) is a first-in-class small molecule in development for developmental and epileptic encephalopathies (DEEs). Positive topline results from the Phase 2 EMBOLD cohort 1 study (N=15) showed a 46% placebo-adjusted reduction in monthly motor seizures from baseline over a 16-week period. Furthermore, over 30% of patients (n=5) achieved seizure freedom. Based on these results, Praxis has initiated a second cohort of the EMBOLD study, aiming to enroll 80 patients, with topline results expected in the first half of 2026. A registrational study (EMERALD) in all DEEs is planned for initiation in the first half of 2025, pending regulatory alignment. Relutrigine has received Orphan Drug Designation (ODD) and Rare Pediatric Designation (RPD) from the FDA, and ODD from the European Medicines Agency (EMA) for the treatment of SCN2A-DEE and SCN8A-DEE.
Elsunersen (PRAX-222) for SCN2A-DEE
Elsunersen (PRAX-222), an antisense oligonucleotide (ASO), is designed to selectively decrease SCN2A gene expression in early-seizure-onset SCN2A-DEE. In Q3, Praxis dosed the first patient in Brazil as part of a continuation of Part A of the EMBRAVE study. Praxis is continuing to harmonize the registrational study protocol, with plans to expand in the U.S. and Europe. Elsunersen has previously received ODD and RPD from the FDA, and ODD and PRIME designations from the EMA for the treatment of SCN2A-DEE.
Financial Update
As of September 30, 2024, Praxis had $411.2 million in cash, cash equivalents, and marketable securities, compared to $81.3 million as of December 31, 2023. Research and development expenses were $41.9 million for the three months ended September 30, 2024, compared to $17.3 million for the same period in 2023. The company reported a net loss of $51.9 million for the three months ended September 30, 2024.
