Praxis Precision Medicines — Third Quarter 2026 Earnings Summary

Financial Performance:

• Reported a net loss of $94.2 million for the third quarter of 2026, compared to a net loss of $73.9 million in the same period of 2025.
• Research and development expenses were $64.2 million for the third quarter of 2026, a decrease of $1.6 million from $65.8 million in the third quarter of 2025.
• General and administrative expenses increased to $42.7 million for the third quarter of 2026 from $12.6 million in the third quarter of 2025.
• Cash, cash equivalents, and marketable securities totaled approximately $1.3 billion as of September 30, 2026, an increase of $359.9 million from $926.1 million as of December 31, 2025.
• The increase in cash and investments was primarily driven by net proceeds from a January 2026 follow-on public offering and interest income, partially offset by cash used in operations.
• Stock-based compensation expense was $14.9 million for the third quarter of 2026, compared to $7.4 million in the third quarter of 2025.
• Total operating expenses for the nine months ended September 30, 2026, were $309.7 million, compared to $229.2 million for the same period in 2025.
• Net loss for the nine months ended September 30, 2026, was $270.5 million, compared to $214.4 million in the prior year period.
• As of September 30, 2026, the company had 27.9 million shares of common stock outstanding.

Guidance and Future Outlook:

• Cash and investments are expected to fund operations into 2028.
• Topline results from the EMERALD study of relutrigine in broad developmental and epileptic encephalopathies (DEEs) are expected in the fourth quarter of 2026.
• Topline results for the elsunersen EMBRAVE3 registrational trial are anticipated in 2027.
• Praxis expects to initiate the POWER3 study of vormatrigine as a monotherapy in 2027.
• The ulixacaltamide sales force hiring is expected to be completed in the fourth quarter of 2026.

Business Segments and Product Lines:

• Ulixacaltamide (Essential Tremor): The New Drug Application (NDA) remains under FDA review with a PDUFA target action date of January 29, 2027. Commercial launch preparations are advancing, with the commercial leadership team in place. A strategic collaboration with Remagine Labs initiated in July 2026 aims to develop a transdermal patch formulation. The long-term safety study (LTSS) associated with the Essential3 program has been reinitiated.
• Relutrigine (DEEs): The NDA for SCN2A and SCN8A DEEs remains under FDA review with a PDUFA target action date of December 27, 2026. The commercial team is fully hired, and preparations for a potential commercial launch are well advanced.
• Vormatrigine (Focal Onset Seizures and Generalized Epilepsy): The POWER2 study has restarted following design updates incorporating learnings from the POWER1 Phase 2/3 study.
• Elsunersen (SCN2A DEE): Enrollment in the EMBRAVE3 registrational trial continues to progress strongly. The program is ongoing under a collaboration with Ionis Pharmaceuticals, Inc., and RogCon, Inc.

Market and Competitive Landscape:

• Ulixacaltamide is the first and only investigational therapy to demonstrate positive results in a Phase 3 program for Essential Tremor (ET), a condition affecting approximately seven million patients in the U.S.
• Vormatrigine is described as the most potent sodium-channel modulator ever developed for epilepsy, designed to target the hyperexcitable state of sodium-channels in adult common epilepsies.
• If approved, relutrigine will be the first therapy for SCN2A and SCN8A DEEs and will be eligible for a Pediatric Review Voucher.
• Elsunersen holds Breakthrough Therapy, Orphan Drug, and Rare Pediatric Disease Designations from the FDA.

Risks and Challenges:

• Forward-looking statements are subject to risks including uncertainties inherent in clinical trials, the expected timing of data readouts, regulatory approvals, and results of trials.
• Risks include potential delays in regulatory interactions and submissions for approval by governmental authorities.
• Specific risks regarding program and operations are detailed in the company's Annual Report on Form 10-K for the year ended December 31, 2025.

Management Commentary and Tone:

• CEO Marcio Souza described the period as "defining," highlighting two key regulatory milestones and the opportunity to become a commercial CNS company.
• Management expressed confidence in the readiness of the relutrigine team and the timeline for the ulixacaltamide sales force.
• The company plans to discuss commercial strategies, launch readiness, and the pipeline at an upcoming Investor Day.

Other Key Points:

• Praxis hosted an in-person and virtual Investor Day on November 12, 2026, featuring senior management and key opinion leaders.
• The company participated in a fireside chat at the Guggenheim Biotech Summit on November 9, 2026.
• Praxis initiated a strategic collaboration in July 2026 with Remagine Labs to develop a transdermal patch of ulixacaltamide.
• The company completed a follow-on public offering in January 2026, contributing significantly to the cash position increase.