Leerink says Agios’ filing reinforces FDA flexibility ahead of Fulcrum update

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.