Fulcrum Therapeutics (FULC) investor relations material

Executive summary

• Pociredir demonstrated robust increases in fetal hemoglobin, improved anemia and hemolysis markers, and encouraging reductions in vaso-occlusive crises in sickle cell disease patients, with the 20 mg dose cohort showing significant efficacy as of December 2025.

• Pociredir was generally well-tolerated, with no treatment-related serious adverse events or discontinuations reported; a long-term dosing study has been initiated.

• Plans are in place to initiate a registration-enabling trial for pociredir in the second half of 2026, pending FDA feedback.

• The company discontinued its bone marrow failure syndromes program and terminated the CAMP4 license agreement to focus on core hematology programs.

• Appointed Dr. Josh Lehrer to the Board of Directors and announced CFO Alan Musso's retirement.

Financial highlights

• Ended Q1 2026 with $333.3 million in cash, cash equivalents, and marketable securities, down from $352.3 million at year-end 2025.

• Research and development expenses were $14.1 million in Q1 2026, up from $13.4 million in Q1 2025, mainly due to higher employee compensation and stock-based compensation.

• General and administrative expenses rose to $8.1 million from $7.0 million year-over-year, driven by increased compensation and professional services.

• Net loss was $18.9 million for Q1 2026, compared to $17.7 million in Q1 2025.

• Other income, net, was $3.3 million, up from $2.7 million, due to higher average cash balances.

Outlook and guidance

• Cash runway is expected to fund operations into 2029 based on current operating plans and $333.3 million in liquidity.

• Update on next trial design anticipated in Q2 2026 after FDA meeting minutes are received.

• Full summary of PIONEER study to be presented at a medical conference later in 2026; long-term dosing data expected in 2027.

• Additional funding will be needed for future growth and commercialization.