FDA Lifts Hold on Gilead’s MDS, AML Clinical Trials

FDA recently lifted the partial hold placed on clinical trials evaluating Gilead’s magrolimab combined with azacitidine in myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) patients.  

During the clinical hold, patients already enrolled in the affected Gilead magrolimab studies, including the Phase 3 ENHANCE study, continued receiving treatment. FDA based its decision on a review of the comprehensive safety data from each trial.  

Enrollment in the US can now resume for all studies investigating magrolimab combined with azacitidine in MDS and AML. Gilead stated that it will re-open enrollment in the studies placed on voluntary hold outside the US.  

The readout for the first interim analysis of the ENHANCE study remains on schedule for 2023.  

Additionally, Gilead is working with FDA regarding the remaining partial clinical hold affecting studies evaluating magrolimab in diffuse large B-cell lymphoma and multiple myeloma. Notably, the ongoing studies evaluating magrolumab in solid tumors were not subject to the clinical hold.  

“Our confidence in the risk-benefit profile of magrolimab has been unwavering, and we continue to believe in the potential for this treatment to address the unmet medical needs faced by people living with MDS and AML,” Merdad Parsey, MD, PhD, chief medical officer at Gilead, said in the announcement.  

“This is a significant milestone for Gilead and, more importantly, for patients diagnosed with these cancers. We look forward to continuing our work developing magrolimab and advancing this potential cancer treatment options,” Parsey continued.  

In September 2020, FDA granted breakthrough therapy designation to magrolumab to treat newly diagnosed MDS based on positive results in an ongoing Phase 1b study.  

The study evaluated magrolumab combined with azacytidine in previously untreated intermediate, high, and very high-risk MDS. Researchers found that 91% of patients who received the combination treatment achieved an objective response, including 42% who achieved complete remission. 

Patient responses increased over time, with a 56% complete remission rate after six months or more of follow-up. Magrolimab is currently being studied for several hematologic cancers and solid tumor malignancies.