Fulcrum Therapeutics, Inc. Q3 FY2024 earnings call

• Alex noted losmapimod Phase 3 REACH trial results led to suspension of development and 40% workforce reduction, focusing on pociredir and preclinical pipeline.
• Pociredir Phase 1b PIONEER trial progress, with plans to share 12-mg cohort data in 2025 and initiate Phase 1 trials in healthy volunteers.
• Early-stage programs in inherited aplastic anemias via CAMP4 licensing agreement, with upcoming IND-enabling study updates.
• Rachel King joined Board, Pat Horn retiring, Thomas Winkler joined as VP of Hematology Clinical Development.

As of September 30, 2024, cash, cash equivalents and marketable securities were $257.2 million compared to $236.2 million as of December 31, 2023. Collaboration revenue was $0 for the third quarter of 2024 compared to $0.8 million in 2023. R&D expenses were $14.6 million in Q3 2024 vs. $18.2 million in Q3 2023. G&A expenses were $8.4 million in Q3 2024 vs. $10 million in Q3 2023. Net loss was $21.7 million in Q3 2024 vs. $24 million in Q3 2023. Expected cash burn in 2025 is approximately $55 million to $65 million, and existing cash is sufficient to fund operations into at least 2027.

• Expect to end 2024 with ~$240 million in cash, cash equivalents, and marketable securities.
• 2025 cash burn expected to be $55 million to $65 million.
• Existing cash sufficient to fund operations into at least 2027.

• Losmapimod Phase 3 trial results not meeting endpoints, leading to program suspension.
• Potential risks in clinical trials, including regulatory feedback on healthy volunteer studies and site compliance.

Q: Matthew Biegler asked about platform tech, wholly-owned vs partnering, and disease areas.

A: Focus on preclinical pipeline, especially inherited aplastic anemias from CAMP4 deal, with potential licensing for non-US markets.

Q: Dae Gon Ha asked about FDA feedback on healthy volunteer studies and African sites.

A: FDA required additional work for healthy volunteers, and FDA accepts data from ex-US sites if GCP followed.

Q: Edward Tenthoff asked about PIONEER data rollout and registrational study transition.

A: 12-mg cohort data to be shared, 20-mg later; data will inform clinical development plan and potential registrational study.

Q: Rick Miller asked about healthy volunteer study dose and outcomes.

A: Studies focus on PK, ADME, safety; key outcomes include PK measures.

Q: Greg Renza asked about losmapimod journey informing future programs, balancing early discovery vs external assets, and 2025 spend.

A: Losmapimod experience informs risk assessment; robust balance sheet allows considering external assets judiciously; 2025 spend $55M-$65M.

Q: Joori Park asked about PIONEER site onboarding.

A: 12 sites activated, working to activate remaining, with plans for site initiation visits.

Q: Corinne Johnson asked about pociredir program funding.

A: Budget includes full development of pociredir, aiming to expand patient population after Phase 1b data.