RARE Daily

FDA Places Partial Hold on Foghorn Study in Relapsed and/or Refractory AML and MDS

May 20, 2022

Rare Daily Staff

Foghorn Therapeutics, a company focused on medicines that modulate gene expression through selectively targeting the chromatin regulatory system, said the U.S. Food and Drug Administration has placed the phase 1 dose escalation study of FHD-286 in two rare cancers, relapsed and/or refractory acute myelogenous leukemia (AML) and myelodysplastic syndrome (MDS), on a partial clinical hold.

The partial clinical hold was initiated by the FDA following the report of a recent death that occurred in a subject with potential differentiation syndrome. Differentiation syndrome is associated with AML/MDS therapeutics that induce differentiation, an effect that is believed to be on-target for the proposed mechanism of action for FHD-286. The FDA has requested a review of the safety database, risk mitigation strategies and a breakdown of clinical activity across dose levels.

Patients currently enrolled in the dose escalation phase 1 study of FHD-286 in AML/MDS and benefitting from treatment may continue to receive treatment, although no new patients can be enrolled until the partial clinical hold is resolved. The partial clinical hold does not apply to the FHD-286 dose escalation phase 1 study in the rare eye cancer metastatic uveal melanoma, with enrollment in that study continuing per protocol.

“Patient safety remains our top priority. We appreciate the dialogue with the FDA and will work diligently with the Agency to resolve the partial clinical hold in AML/MDS as soon as possible,” said Adrian Gottschalk, CEO of Foghorn Therapeutics.

Until Foghorn has resolved the partial clinical hold for the AML/MDS study, the Company is suspending guidance on the timing of the data release for the dose escalation phase of the FHD-286 program.

FHD-286 is a highly potent, selective, allosteric, and orally available, small-molecule, enzymatic inhibitor of BRG1 and BRM, two highly similar proteins that are the ATPases, or the catalytic engines across all forms of the BAF complex, one of the key regulators of the chromatin regulatory system. In preclinical studies, FHD-286 has shown anti-tumor activity across a broad range of malignancies including both hematologic and solid tumors.

Photo: Adrian Gottschalk, CEO of Foghorn Therapeutics

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