Praxis Precision Medicines, Inc.

Praxis Precision Medicines, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for elsunersen (PRAX-222). This designation is for the treatment of seizures associated with SCN2A Developmental and Epileptic Encephalopathy (DEE) caused by Gain of Function variants in SCN2A. The BTD, which enables expedited development and regulatory review, was supported by positive topline results from the EMBRAVE Part A trial, a Phase 1/2 study.

Praxis Precision Medicines, Inc. announced its participation in the Goldman Sachs 47th Annual Global Healthcare Conference. The company will deliver a corporate overview on June 8, 2026, at 8:00am ET. A live webcast and a replay will be available for interested parties.

Praxis Precision Medicines, Inc. announced that its Compensation Committee granted restricted stock unit awards covering an aggregate of 2,754 shares of common stock. These awards were given to thirteen new non-executive employees on June 1, 2026, under the 2024 Inducement Plan. The grants serve as inducements material to their employment, in accordance with Nasdaq Listing Rule 5635(c)(4), and will vest in four equal annual installments.

Praxis Precision Medicines announced that its Phase 2/3 POWER1 study evaluating vormatrigine for focal onset seizures (FOS) did not meet its primary success measure. While the primary endpoint was missed, the study did meet its secondary measure of a 50% response rate, and higher doses showed more pronounced seizure reduction. The drug was generally well-tolerated, with less than 10% discontinuations due to adverse events, and approximately 90% of patients moved to an open-label extension. Consequently, the company is pausing enrollment in the POWER2 study to reassess the vormatrigine program.

Praxis Precision Medicines announced that its Phase 2/3 POWER1 study evaluating vormatrigine for focal onset seizures did not meet its primary success measure. However, the study did meet a secondary measure of a 50% response rate, and a more pronounced seizure reduction was observed on the higher 30 mg dose. Vormatrigine was generally well-tolerated with low discontinuation rates, and approximately 90% of patients continued into the open label extension study. Despite these encouraging signals, Praxis is pausing enrollment in the POWER2 study to reassess the vormatrigine program.

Praxis Precision Medicines announced its first quarter 2026 financial results and provided a corporate update, highlighting significant regulatory and clinical milestones. The FDA accepted New Drug Applications for ulixacaltamide in Essential Tremor and relutrigine in SCN2A and SCN8A DEEs, with PDUFA target action dates in January 2027 and September 2026, respectively. The company also reported positive topline results from the EMBRAVE Part A study for elsunersen, showing a 77% placebo-adjusted seizure reduction. Praxis ended the quarter with $1.4 billion in cash and investments, extending its runway into 2028, despite an increased net loss of $92.6 million.

Praxis Precision Medicines reported a net loss of $(92.6) million for the first quarter of 2026, an increase from $(69.3) million in Q1 2025. Operating expenses rose significantly, driven by increased research and development and general and administrative costs. However, the company substantially strengthened its financial position through a follow-on public offering, boosting cash, cash equivalents, and marketable securities to $1.4 billion, which is expected to fund operations for at least one year.

Praxis Precision Medicines announced FDA acceptance of New Drug Applications for ulixacaltamide in Essential Tremor (PDUFA Jan 29, 2027) and relutrigine in SCN2A/SCN8A DEEs (priority review, PDUFA Sep 27, 2026). The company also reported positive EMBRAVE Part A study results for elsunersen, showing a 77% placebo-adjusted reduction in monthly seizures. Praxis ended Q1 2026 with $1.4 billion in cash, extending its runway into 2028, despite an increased net loss of $92.6 million.

Praxis Precision Medicines, Inc. announced it will report its first quarter financial results for the period ended March 31, 2026, and provide a corporate update on Thursday, May 7, 2026, before financial markets open. The company will host a conference call and live webcast at 8:30 am ET to discuss these results and updates. Additionally, Praxis will present at the Bank of America Securities Health Care Conference on May 12, 2026, at 7:20 pm EDT, with a live webcast available.

Praxis Precision Medicines, Inc. announced its 2026 Annual Meeting of Stockholders will be held virtually on June 10, 2026. Key proposals include the election of three Class III directors, ratification of Ernst & Young LLP as the independent registered public accounting firm, and an advisory vote on executive compensation. The CEO highlighted meaningful progress in 2025 and a robust pipeline with transformative milestones for 2026, while also noting recent board and executive appointments.