Bristol-Myers Squibb Reports Data from Phase 3 QUAZAR AML-001 Study; Plans to File NDA in 2020

Dec 12, 2019 | Hematology, News, Oncology, Positive Results

Bristol-Myers Squibb Reports Data from Phase 3 QUAZAR AML-001 Study; Plans to File NDA in 2020

Bristol-Myers Squibb presented positive results from the QUAZAR AML-001 study, which evaluated CC-486 as maintenance therapy in a broad population of patients with front-line, newly diagnosed acute myeloid leukemia (AML) who have achieved remission with intensive induction chemotherapy. Data were presented during a late-breaker oral presentation at the 2019 ASH Annual Meeting in Orlando, Fla. Treatment with CC-486 in the maintenance setting resulted in statistically significant and clinically meaningful improvements in overall survival (OS) and relapse-free survival (RFS), as compared to patients treated with placebo.

QUAZAR AML-001 was an international, randomized, double-blind, placebo-controlled study of CC-486 as AML maintenance therapy in patients who achieved first complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following intensive induction chemotherapy (with or without consolidation). The study enrolled 472 patients, randomized 1:1 to receive initially either oral CC-486 300mg or placebo once daily for 14 days of a 28-day cycle plus best supportive care. Patients remained on treatment until unacceptable toxicity or disease progression. The primary endpoint of the study was overall survival (OS). 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.

At a median follow-up of 41.2 months, the primary endpoint of OS was significantly improved for patients receiving CC-486 compared to placebo. Median OS from time of randomization was 24.7 months in the CC-486 arm compared to 14.8 months for placebo. Median RFS, the key secondary endpoint, was 10.2 months for those receiving CC-486 compared to 4.8 months for those receiving placebo. Improvements in OS and RFS for those treated with CC-486 compared to placebo were demonstrated, regardless of cytogenetic risk category, prior consolidation or CR/CRi status at enrollment. The most common adverse events were nausea, vomiting and diarrhea.

Based on this data, BMS plans to file regulatory submissions in the first half of 2020.

About CC-486

CC-486 is an oral hypomethylating agent that incorporates into DNA and RNA allowing for sustained epigenetic regulation due to prolonged exposure. The main mechanism of action is thought to be hypomethylation of DNA, as well as direct cytotoxicity to abnormal hematopoietic cells in the bone marrow. Hypomethylation may restore normal function to genes that are critical for differentiation and proliferation.

About AML 

Acute myeloid leukemia (AML) is the most common type of acute leukemia. AML starts in the bone marrow but moves quickly into the blood. Unlike in normal blood cell development, in AML the rapid build up of abnormal white blood cells in the bone marrow may interfere with the production of normal blood cells, resulting in decreased healthy white blood cells, red blood cells and platelets. AML is a complex, diverse disease associated with multiple genetic mutations and usually gets worse quickly if not treated. 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.

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