The Food and Drug Administration put a full clinical hold on a study of the novel drug FHD-286 in patients with acute myelogenous leukemia and myelodysplastic syndrome after study data showed an increase in a potentially fatal side effect.
The Food and Drug Administration (FDA) placed a full clinical hold on a phase 1 clinical trial evaluating FHD-286 in patients with relapsed or refractory acute myelogenous leukemia (AML) and myelodysplastic syndrome (MDS), according to Foghorn Therapeutics, the manufacturer of the drug.
The trial was first placed on a partial hold, which suspends or delays only part of the trial.
In response to the partial hold, Foghorn Therapeutics was required to submit further data to the FDA. Since these data showed additional instances of fatal differentiation syndrome — a potentially fatal drug reaction for patients with blood cancer that is the result of too many inflammatory molecules, called cytokines, being released into the blood — the FDA put the trial on a full clinical hold, stopping all of the study activity for patients with AML and MDS.
FHD-286 is an oral drug that inhibits BRG1 and BRM, which play a role in gene expression. In pre-clinical (not tested in humans) trials, FHD-286 showed anti-tumor activity in both blood cancers and solid tumors.
Approximately 50 patients are enrolled onto the trial. The primary goal of the trial assessing FHD-286 in patients with relapsed or refractory acute myelogenous leukemia (AML) and myelodysplastic syndrome is to identify the incidence of treatment-emergent side effects. Additionally, the investigators were analyzing the incidence of side effects that led to dose reductions as well as treatment discontinuations.
The study authors are also reviewing the treatment’s effect on response rates, as well as event-free survival (defined as time from first treatment to first date of documented disease relapse) and overall survival (time from first treatment to death from any cause).
The FDA will continue to allow the study of FHD-286 in patients with uveal melanoma, a rare type of cancer that occurs in the eye. However, in order for the AML and MDS portions of the trial to continue, Foghorn Therapeutics must answer additional questions and complete further analyses.
Adrian Gottschalk, Foghorn’s CEO, said in a company-issued press release that the pharmaceutical company is committed to patient safety and is going to continuously work with the FDA to address any questions the agency may have.
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