Gilead Sciences has announced that the US Food and Drug Administration (FDA) has lifted the partial clinical hold placed on studies evaluating the company’s investigational agent magrolimab in combination with azacitidine.
The hold was removed after a review of the comprehensive safety data from each trial. As a consequence, enrolment in the US can resume for the studies into the therapies, which treat both myelodysplastic syndrome (MDS) and acute myeloid leukaemia (AML).
Throughout the partial clinical hold, patients already enrolled in the affected magrolimab studies – including the phase 3 ENHANCE study – continued receiving treatment.
MDS is a rare, often unrecognised and under-diagnosed bone marrow disorder widely considered to be a form of cancer. One in three people with the condition will progress to AML, making it one of the most common types of leukaemia in adults. In the US, AML has a fatality rate of approximately 10,000 people each year.
Gilead – in close coordination with regulatory authorities – is planning to resume participation, while the company is also working with the FDA regarding the remaining partial clinical hold affecting studies evaluating magrolimab in diffuse large B-cell lymphoma and multiple myeloma. The ongoing clinical studies evaluating the drug in solid tumours are 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,” said Merdad Parsey, chief medical officer at Gilead Sciences.
“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 option,” he added.
Prior to the trials being paused, Gilead had already met the pre-specified enrolment threshold required for the first interim analysis. Based on this, and the resumption of recruitment, the company is confident the results remain on schedule for 2023.
Magrolimab was granted breakthrough therapy designation for the treatment of newly diagnosed MDS by the FDA in 2020. The drug is an investigational product only and is not approved by any regulatory authority for any use while its safety and efficacy have not been established.