Celgene’s Phase 3 Study of CC-486 as Maintenance Therapy in Patients With Newly Diagnosed AML Met Primary and Key Secondary Endpoints

CC-486 was well-tolerated and there were no unexpected safety events in QUAZAR AML-001. This phase 3 study enrolled 472 patients, randomized 1:1 to receive either oral CC-486 300mg or placebo once daily for 14 days of a 28-day cycle plus best supportive care until disease relapse.

“AML remains a deadly blood cancer where most patients are not curable and less than 30% of patients survive five years¹,” said Jay Backstrom, M.D., M.P.H., Chief Medical Officer for Celgene. “The CC-486 QUAZAR AML-001 study is the first phase 3 trial to demonstrate that the addition of maintenance therapy has the potential to extend overall survival in a broad population of patients with newly diagnosed AML who have achieved remission with induction chemotherapy.”

Data from QUAZAR AML-001 will be submitted to a future medical meeting. Celgene also plans regulatory submissions for CC-486 beginning in the first half of 2020.

CC-486 is an investigational compound and not approved for any use in any geography.

About QUAZAR AML-001

Phase 3, randomized, double-blind, placebo-controlled study of CC-486 as AML maintenance therapy in patients who achieved first CR or complete response with incomplete blood count recovery (CRi) with induction chemotherapy (with or without consolidation) The primary endpoint of the study was overall survival. Key secondary endpoints included relapse-free survival (RFS), safety and tolerability, healthcare resource utilization and patient-reported outcomes per the FACIT-Fatigue Scale and EQ-5D questionnaire. The study enrolled 472 patients, randomized 1:1 to receive either oral CC-486 300mg or placebo once daily for 14 days of a 28-day cycle plus best supportive care until disease progression.

About CC-486

CC-486 is a cytidine nucleoside analogue and incorporates into DNA and RNA. The main mechanism of action is thought to cause DNA hypomethylation and direct cytotoxicity on abnormal hematopoietic cells in the bone marrow. Hypomethylation may restore normal function to genes that are critical for differentiation and proliferation. The antineoplastic effect of CC-486 is hypothesized to cause death of rapidly dividing cells, including cancer cells that are no longer responsive to normal growth control mechanism.

About AML

Acute myeloid leukemia (AML) is a type of cancer in which the bone marrow makes abnormal blast cells that are supposed to grow into different types of blood cells. It may present in multiple subtypes based on the maturity of the cancer cells at diagnosis.² There will be an estimated 21,450 new cases of AML in the United States this year, accounting for 1.2% of all cancer cases, with an estimated 10,920 deaths resulting from the disease. There are an estimated 61,048 people living with AML in the United States.³

About Celgene

Celgene Corporation, headquartered in Summit, New Jersey, is an integrated global biopharmaceutical company engaged primarily in the discovery, development and commercialization of innovative therapies for the treatment of cancer and inflammatory diseases through next-generation solutions in protein homeostasis, immuno-oncology, epigenetics, immunology and neuro-inflammation. For more information, please visit www.celgene.com. Follow Celgene on Social Media: @Celgene, Pinterest, LinkedIn, Facebook and YouTube.

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¹ https://seer.cancer.gov/statfacts/html/amyl.html
² https://www.cancer.gov/types/leukemia/patient/adult-aml-treatment-pdq
³ https://seer.cancer.gov/statfacts/html/amyl.html

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