FDA Lifts Partial Clinical Hold on MDS and AML Magrolimab Studies

Rhea-AI Summary

Gilead Sciences announced that the FDA has lifted the partial clinical hold on studies evaluating its investigational drug magrolimab, allowing U.S. enrollment to resume for trials focused on myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). The decision follows a comprehensive safety review. Gilead plans to re-open enrollment in additional studies for magrolimab, which is granted Breakthrough Therapy designation for MDS. The company anticipates the first interim analysis for the pivotal ENHANCE study to remain on schedule for 2023.

Positive

• FDA lifted the partial clinical hold on magrolimab trials, allowing U.S. enrollment to resume.
• Gilead met the enrollment threshold for the interim analysis of the ENHANCE study before the hold.
• Magrolimab holds Breakthrough Therapy designation for newly diagnosed MDS, indicating significant potential.

Negative

• Ongoing partial clinical hold on magrolimab studies for diffuse large B-cell lymphoma and multiple myeloma.
• Safety and efficacy of magrolimab have not been established as it remains investigational.

– U.S. Pivotal Studies to Restart Enrollment Immediately –

– Decision Based on Review of the Comprehensive Safety Data from Each Trial –

FOSTER CITY, Calif.--(BUSINESS WIRE)-- Gilead Sciences, Inc. (Nasdaq: GILD) today announced that the U.S. Food and Drug Administration (FDA) lifted the partial clinical hold placed on studies evaluating its investigational agent magrolimab in combination with azacitidine. The FDA removed the partial clinical hold after a review of the comprehensive safety data from each trial.

With today’s decision from the FDA, enrollment in the U.S. can resume for the studies investigating magrolimab in combination with azacitidine in myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). Gilead, in close coordination with regulatory authorities, is planning to re-open enrollment in the magrolimab studies that were placed on a voluntary hold outside of the U.S. 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 magrolimab 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,” said Merdad Parsey, MD, PhD, Chief Medical Officer, 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.”

During the partial clinical hold, patients already enrolled in the affected Gilead magrolimab studies, including the pivotal, Phase 3 ENHANCE study, continued receiving treatment. Prior to the trial hold, Gilead already met the pre-specified enrollment threshold required for the first interim analysis of the ENHANCE study. Based on this, Gilead is confident the readout for the first interim analysis remains on schedule for 2023.

Magrolimab was granted Breakthrough Therapy designation for the treatment of newly diagnosed MDS by the FDA in 2020. In addition to MDS and AML, magrolimab is being developed in several hematologic cancers and solid tumor malignancies. Magrolimab is an investigational product and is not approved by any regulatory authority for any use; its safety and efficacy have not been established.

About Myelodysplastic Syndrome (MDS)

MDS is a rare, often unrecognized, under-diagnosed, bone marrow disorder widely considered to be a form of cancer. In MDS, the body’s bone marrow does not make enough mature, healthy red blood cells, white blood cells and/or platelets. While many cases may go undiagnosed, current estimates show that ~100,000 new cases are diagnosed each year. Therapeutic advancement for higher-risk MDS has been limited in the last 15 years and stem cell transplant is the only potential cure, but those older than 65 or with other conditions are often not eligible.

One in three people with MDS will progress to AML, one of the most common types of leukemia in adults. Approximately 10,000 people die every year from AML in the U.S.

About Acute Myeloid Leukemia (AML)

AML is a type of cancer that starts in the bone marrow and can quickly move to the blood and other parts of the body, including the lymph nodes, spleen and central nervous system. AML most often develops from cells that would turn into red blood cells, neutrophils, and platelets but can also evolve from other blood disorders such as myelodysplastic syndromes. Approximately 20,000 Americans will be diagnosed with AML each year.

About Magrolimab

Magrolimab is a potential, first-in-class investigational monoclonal antibody against CD47 and a macrophage checkpoint inhibitor that is designed to interfere with recognition of CD47 by the SIRPα receptor on macrophages, with the goal of blocking the “don’t eat me” signal used by cancer cells to avoid being ingested by macrophages. Magrolimab is being developed in several hematologic cancers, including myelodysplastic syndrome (MDS), acute myeloid leukemia (AML) as well as solid tumor malignancies. More information about clinical trials with magrolimab is available at www.clinicaltrials.gov.

About Gilead Sciences

Gilead Sciences, Inc. is a biopharmaceutical company that has pursued and achieved breakthroughs in medicine for more than three decades, with the goal of creating a healthier world for all people. The company is committed to advancing innovative medicines to prevent and treat life-threatening diseases, including HIV, viral hepatitis and cancer. Gilead operates in more than 35 countries worldwide, with headquarters in Foster City, California.

Forward-Looking Statements

This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks, uncertainties and other factors, including Gilead’s ability to initiate, progress or complete clinical trials within currently anticipated timelines or at all, the risk that FDA or other regulatory authorities may not remove other clinical holds currently in place on clinical trials, and the possibility of unfavorable results from ongoing or additional clinical trials, including those involved magrolimab; Gilead’s ability to submit regulatory applications in the currently anticipated timelines or at all, including those relating to magrolimab; Gilead’s ability to receive regulatory approvals in a timely manner or at all, including regulatory approvals of magrolimab, and the risk that any such approvals, if granted, may be subject to significant limitations on use; the possibility that Gilead may make a strategic decision to discontinue development of magrolimab and that, as a result, magrolimab may never be successfully commercialized; and any assumptions underlying any of the foregoing. These and other risks, uncertainties and other factors are described in detail in Gilead’s Annual Report on Form 10-K for the year ended December 31, 2021, as filed with the U.S. Securities and Exchange Commission. These risks, uncertainties and other factors could cause actual results to differ materially from those referred to in the forward-looking statements. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. The reader is cautioned that any such forward-looking statements are not guarantees of future performance and involve risks and uncertainties and is cautioned not to place undue reliance on these forward-looking statements. All forward-looking statements are based on information currently available to Gilead, and Gilead assumes no obligation and disclaims any intent to update any such forward-looking statements.

Gilead and the Gilead logo are trademarks of Gilead Sciences, Inc., or its related companies.

For more information about Gilead, please visit the company’s website at www.gilead.com, follow Gilead on Twitter (@GileadSciences) or call Gilead Public Affairs at 1-800-GILEAD-5 or 1-650-574-3000.

View source version on businesswire.com: https://www.businesswire.com/news/home/20220407005786/en/

Jacquie Ross, Investors

investor_relations@gilead.com

Marian Cutler, Media

marian.cutler1@gilead.com

Source: Gilead Sciences, Inc.

FAQ

What recent decision did the FDA make regarding Gilead's magrolimab trials?

The FDA lifted the partial clinical hold on studies evaluating magrolimab, allowing U.S. enrollment to resume.

What conditions are the magrolimab studies focused on?

The studies are focused on myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML).

What is the timeline for the interim analysis of the ENHANCE study?

Gilead is confident that the readout for the first interim analysis of the ENHANCE study remains on schedule for 2023.

What designation does magrolimab have from the FDA?

Magrolimab was granted Breakthrough Therapy designation for the treatment of newly diagnosed MDS.

What challenges does Gilead face with magrolimab's development?

Gilead faces a remaining partial clinical hold on studies for diffuse large B-cell lymphoma and multiple myeloma.