Praxis Precision Medicines (PRAX) investor relations material

Executive summary

• Four late-stage CNS assets with over $20 billion peak sales potential, leveraging proprietary Cerebrum and Solidus platforms, and advancing multiple preclinical programs.

• FDA accepted NDAs for ulixacaltamide (Essential Tremor) and relutrigine (SCN2A/SCN8A-DEE), with PDUFA dates in January and September 2027; commercial launches targeted within eight months.

• EMBRAVE Part A study: elsunersen showed a 77% placebo-adjusted reduction in monthly seizures for early-onset SCN2A-DEE.

• Topline results for POWER1 (vormatrigine in epilepsy) expected Q2 2026; EMERALD (relutrigine in broad DEEs) topline results expected Q4 2026.

• Commercial preparations and infrastructure build underway for upcoming launches.

Financial highlights

• Q1 2026 net loss was $92.6 million, driven by increased R&D ($78 million) and G&A/SG&A ($28 million) expenses.

• Operating expenses rose to $105.9 million in Q1 2026, up from $74.7 million year-over-year.

• Cash, cash equivalents, and marketable securities totaled $1.4 billion as of March 31, 2026, up from $926 million at year-end 2025, mainly from a January 2026 follow-on public offering.

• Net cash used in operating activities was $86.2 million in Q1 2026.

• Net cash provided by financing activities was $609.4 million, primarily from a $621.2 million public offering.

Outlook and guidance

• Cash position expected to fund operations into 2028, supporting ongoing R&D, clinical trials, and commercialization.

• Commercial launches for ulixacaltamide and relutrigine planned within the next eight months, pending FDA approval.

• Key topline results expected: POWER1 (vormatrigine) in Q2 2026, EMERALD (relutrigine) in Q4 2026, POWER2 in 2027, and EMBRAVE3 in 2027.

• Additional indications for ulixacaltamide to be announced soon.

• Expenses expected to increase as clinical and commercial activities expand.