FULC News & Events

As of March 31, cash, cash equivalents, and marketable securities were $333.3M, compared to $352.3M as of December 31, 2025. The decrease of $19M was primarily due to cash used to fund operating activities in 2026. "The strength of the clinical data presented in the first quarter further reinforce our conviction in pociredir's potential to address the underlying biology of sickle cell disease," said Alex Sapir, Fulcrum's president and CEO. "The magnitude of HbF induction and improvements in markers of hemolysis and anemia observed to date support our upcoming discussions with the FDA as we prepare for a potential registration-enabling study in the second half of 2026. With a strong balance sheet extending our cash runway into 2029, we are well positioned to advance pociredir through the next phase of clinical development."

CFO Alan Musso plans to retire later this year to spend more time with his family and other outside interests. Musso will remain in his role until a successor is named and has agreed to serve as a consultant thereafter to support a transition. Fulcrum will initiate a search to identify a successor.

Leerink thinks Agios Pharmaceuticals' (AGIO) announcement that it is filing mitapivat for accelerated approval in sickle cell disease has positive readthrough to Fulcrum Therapeutics (FULC) by demonstrating FDA flexibility ahead of a key regulatory update for the company expected Q2 2026. Recall Agios' Phase 3 RISE UP trial of mitapivat in SCD met the primary endpoint of hemoglobin response but technically missed statistical significance on the primary endpoint of annualized rate of sickle cell pain crises though a trend was shown, Leerink notes. Leerink says the move underscores continued FDA recognition of the high unmet need in SCD and willingness to work with sponsors to gain alignment. Leerink believes this is a positive sign for Fulcrum given they are seeking regulatory alignment on their next steps in SCD in the near term.