Fulcrum Therapeutics, Inc. Q1 FY2026 earnings call

The first quarter of 2026 was highlighted by positive clinical data from the phase 1B pioneer trial of Pociradir in sickle cell disease. Pociradir showed robust increase in HBF, improvements in hemolysis and anemia, VOC reduction, and good tolerance. Initiated an open-label long-term dosing trial for patients in the Pioneer study and enrolled the first patient. Supported initiatives to improve care journey for sickle cell disease patients. Focused on next stage of clinical development for Pociradir, plan to update trial design after end-of-phase meeting with FDA and initiate registration-enabling trial in H2 2026. Welcomed Josh Lurer to board and Alan's upcoming retirement.

Research and development expenses were $14.1 million for the first quarter of 2026 compared to $13.4 million for the first quarter of 2025. The increase of $700,000 was primarily driven by higher employee compensation costs including $400,000 of increased stock-based compensation expense. General and administrative expenses were $8.1 million for the first quarter of 2026, compared to $7 million for the first quarter of 2025. The increase of $1.1 million was primarily driven by higher employee compensation costs, including $300,000 of increased stock-based compensation expense, as well as higher professional services costs. The net loss was $22.2 million for the first quarter of 2026 compared to a net loss of $20.4 million for the first quarter of 2025. We ended the first quarter of 2026 with cash, cash equivalents and marketable securities of $333.3 million compared to $352.3 million as of December 31st, 2025. The $19 million decrease was primarily due to cash used to fund our operating activities.

Plan to provide update on trial design following upcoming end-of-phase meeting with FDA. Intend to initiate registration-enabling trial in the second half of 2026. Existing cash, cash equivalents and marketable securities will be sufficient to fund operating requirements into 2029.

Risks associated with the company's business discussed in SEC filings, including uncertainties in clinical development and market dynamics.

Q: Alex, as you reflect on the experience gained through Pioneer, what are the most important things you've learned that you might not have fully appreciated going in and how will those lessons shape your phase three design and execution?

A: Alex mentioned learning about high unmet need in severe patient population and strong connection between HBF and VOC reduction, which will shape phase three design.

Q: At the sickle cell disease research symposium, will there be any new analyses that will be presented relative to the 1Q corporate update for Pioneer?

A: Ian said they expect to provide a fulsome reporting of the entire Pioneer study at a medical conference later this year but FSCDR symposium will include previously disclosed clinical data.

Q: What's the latest you've been hearing from these thought leaders about how they're thinking about sickle cell disease as an indication, the unmet need, and just receptiveness to hearing about new drug classes?

A: Alex and Ian discussed hearing about high unmet need in sickle cell disease on the Hill and positive receptiveness at FDA.

Q: Could you help us think through the role you expect Pacira to play, particularly if there's more kind of oral medications that come to market over the next couple of years?

A: Alex discussed Pacira's potential with 24-month head start over next closest competitor and market evolution towards oral HBF inducers.

Q: Just curious if you think that changes anything at all as it relates to the potential regulatory path for you. And, you know, if a drug were to be approved on VOCs, does that sort of change what the FDA may be open to approving, you know, as it relates to endpoints that are not a VOC endpoint?

A: James was told VOCs remains important clinical endpoint and literature supports HBF-VOC association.

Q: Maybe just piggybacking on some of the earlier comment that you made, Alex, I'm thinking about maybe potential future combination strategies here.

A: Alex and Ian discussed potential combination strategies with hydroxyurea and beyond, focusing on monotherapy first for registration.

Q: I know, of course, that the focus is on Pociradir, but I'm just curious when you see the time being right to potentially advance or nominate some of the discovery programs and think about the novel HBF inducers that you may have in the library. And then maybe secondarily, as we think about the FDA meetings upcoming, can you just give us an update on the engagement plans with respect to EMA and the global development?

A: Greg was told discovery focused on developing second, third, fourth gen oral HBF inducers, and will interact with EMA later this year for global development