Praxis Precision Medicines (PRAX) Q3 2024 earnings summary

Executive summary

• Advanced four clinical-stage programs in CNS disorders, focusing on essential tremor, epilepsy, and DEEs, leveraging Cerebrum (small molecule) and Solidus (ASO) platforms.

• Essential3 phase III program for ulixacaltamide in ET is progressing, with interim analysis expected in Q1 2025 and NDA filing planned for 2025.

• Positive phase II EMBOLD results for relutrigine in DEEs showed 46% reduction in moderate seizures and 33%–46% seizure-free rate, leading to expanded cohort enrollment and advancement to registrational studies.

• Vormatrigine studies in epilepsy are advancing, with topline data for POWER1 and RADIANT expected in 2025 and lifecycle expansion into Parkinson's and pain indications being explored.

• Elsanursen dosing began in Brazil for EMBRAVE study, with global registration plans ongoing and protocol harmonization for U.S. and Europe.

Financial highlights

• Q3 2024 net loss was $51.9 million, up from $24.6 million in Q3 2023, driven by increased R&D ($41.9 million) and G&A ($15.3 million) expenses.

• Cash, cash equivalents, and marketable securities totaled $411.2 million as of September 30, 2024, up from $81.3 million at year-end 2023, mainly from public offerings.

• Operating cash outflow was $27.7 million in Q3 2024; net cash used in operating activities was $75.6 million for the nine months ended September 30, 2024.

• Collaboration revenue was $0.3 million in Q3 2024, down from $0.5 million in Q3 2023.

• Cash runway extends into 2027, funding all current programs through their readouts.

Outlook and guidance

• Interim analysis for ulixacaltamide Essential3 Study 1 expected in Q1 2025, with NDA submission planned for 2025.

• Topline results for POWER1 and RADIANT epilepsy trials anticipated in 2025; POWER2 to begin recruiting in H1 2025.

• EMBOLD Cohort 2 topline results for relutrigine in DEEs expected in H1 2026; EMERALD registrational study to initiate in H1 2025.

• Parkinson’s disease program for ulixacaltamide expected to re-initiate in 2025, contingent on ET study outcomes.

• Anticipates continued operating losses and increased expenses as clinical programs advance.