Praxis Precision Medicines (PRAX) Q3 2024 earnings summary

Executive summary

• Four late-stage clinical programs are advancing, including ulixacaltamide in essential tremor, relutrigine in developmental epilepsies, vormatrigine in epilepsy, and elsanursen in global confirmatory studies, representing a multi-billion dollar opportunity.

• Positive phase II results for relutrigine in SCN2A/SCN8A developmental epilepsies showed a 46% seizure reduction and up to 33% seizure-free patients, leading to rapid advancement to registrational studies.

• Vormatrigine and relutrigine studies in epilepsy are on track for topline data in 2025, with lifecycle expansion into Parkinson's and pain being explored.

• Elsanursen dosing began in Brazil for the EMBRAVE study, with global regulatory engagement and protocol harmonization ongoing.

• No product sales to date; revenue is derived from collaboration agreements.

Financial highlights

• Q3 2024 net loss was $51.9 million, compared to $24.6 million in Q3 2023, driven by increased R&D and G&A expenses as clinical programs advanced.

• Research and development expenses rose to $41.9 million in Q3 2024, mainly due to ulixacaltamide and vormatrigine programs.

• General and administrative expenses increased to $15.3 million in Q3 2024, up $6.5 million year-over-year.

• 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 and stock sales.

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

Outlook and guidance

• Interim analysis for ulixacaltamide Phase III (Essential3) expected in Q1 2025, with NDA submission anticipated in 2025.

• Topline results for RADIANT and POWER1 epilepsy trials (vormatrigine) anticipated in 2025.

• EMERALD study protocol for relutrigine in all DEEs to be finalized by year-end and initiated in 2025; EMBOLD Cohort 2 topline results expected in H1 2026.

• Parkinson’s disease program for ulixacaltamide expected to re-initiate in 2025.

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