Praxis Precision Medicines has made significant strides in advancing its pipeline, with relutrigine progressing into late-stage development. The company's third-quarter update highlights the advancement of multiple clinical programs and a strong financial position.
Relutrigine for Developmental and Epileptic Encephalopathies (DEEs)
Relutrigine, a first-in-class small molecule, is being developed for the treatment of developmental and epileptic encephalopathies (DEEs). The recent topline results from the EMBOLD cohort 1 study demonstrated unmatched seizure-freedom and reduction in SCN2A and 8A patients, along with potential disease-modifying effects. Marcio Souza, president and chief executive officer of Praxis, noted the rapid advancement of the SCN2A/8A program to the registrational stage and the expansion of studies to cover a broader range of DEEs.
Relutrigine functions as a preferential inhibitor of persistent sodium current, a key driver of seizure symptoms in early-onset SCN2A-DEE and SCN8A-DEE. The mechanism involves superior selectivity for disease-state sodium channel (NaV) hyperexcitability. Clinical trials have shown that relutrigine is generally well-tolerated and exhibits biomarker changes indicative of NaV channel blocking effects. The FDA and European Medicines Agency have granted Orphan Drug Designation (ODD) and Rare Pediatric Disease (RPD) designations for relutrigine in the treatment of SCN2A-DEE and SCN8A-DEE.
Ulixacaltamide for Essential Tremor
Ulixacaltamide, a highly selective small molecule inhibitor of T-type calcium channels, is designed to block abnormal neuronal burst firing in the Cerebello-Thalamo-Cortical (CTC) circuit associated with tremor activity. The operational plan to complete the interim analysis for the Essential3 Study 1 is finalized for mid-Q1 2025. Ulixacaltamide represents the most advanced program within Praxis’ Cerebrum™ small molecule platform and is in late-stage development for essential tremor.
Vormatrigine (PRAX-628) for Epilepsy
The ENERGY program for vormatrigine (PRAX-628) is progressing, driven by insights from the ongoing observational EMPOWER study. Vormatrigine, a next-generation, functionally selective small molecule, targets the hyperexcitable state of sodium-channels in the brain. It is being developed as a once-daily, oral treatment for adult focal onset seizures and generalized epilepsy. Preclinical data suggests that vormatrigine has superior selectivity for disease-state NaV channel hyperexcitability and unprecedented potency in the maximal electroshock seizure (MES) model. The RADIANT and POWER1 trials are on track, and Praxis is actively exploring lifecycle expansion opportunities in Parkinson’s Disease and pain.
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. The increase of $329.9 million is primarily due to net proceeds from Praxis’ January 2024 and April 2024 follow-on public offerings and net proceeds from at-the-market sales of common stock, offset by cash used in operating activities. Research and development expenses were $41.9 million for the three months ended September 30, 2024, compared to $17.3 million for the three months ended September 30, 2023. The net loss for the quarter was $51.9 million, including $12.4 million of stock-based compensation expense.
