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Fulcrum Halts Pociredir Development After FDA Flags PRC2 Cancer Risk

The FDA concluded PRC2-targeting therapies carry similar cancer risks, leaving no viable regulatory path for pociredir.

Signage is seen outside of the Food and Drug Administration (FDA) headquarters in White Oak, Maryland, U.S., August 29, 2020. REUTERS/Andrew Kelly/File Photo

Overview

  • Fulcrum announced on Monday that it has stopped development of pociredir, its lead sickle-cell candidate, and opened a comprehensive strategic review of options including a possible sale or merger.
  • The company said the decision followed FDA end-of-phase feedback, which cited the global March withdrawal of Ipsen’s tazemetostat and concluded that drugs acting on the PRC2 complex may share a malignancy risk.
  • Pociredir had shown dose-dependent increases in fetal hemoglobin and early signs of clinical benefit in a Phase 1b trial, but Fulcrum and the FDA noted earlier preclinical malignancy signals tied to the program.
  • Investors reacted sharply: Fulcrum’s shares fell about 50% in after-hours and premarket trading, and the company said it will cut operating expenses and preserve cash while the board evaluates next steps.
  • Fulcrum reported $333.3 million in cash and marketable securities as of March 31, but the move raises broader regulatory and investment uncertainty for PRC2-targeted and other sickle-cell therapies and may slow patient access to new oral options.