First Patient Enrolled in REZLIDHIA Phase 1b/2 Trial for mIDH1 AML

SOUTH SAN FRANCISCO, Calif., Sept. 5, 2024. Rigel Pharmaceuticals, a biotechnology company that specializes in treatments for blood disorders and cancer, has announced the enrollment of the first patient in a new Phase 1b/2 clinical trial. This trial will evaluate a combination therapy involving decitabine, venetoclax, and REZLIDHIA (olutasidenib) for patients with mutated isocitrate dehydrogenase-1 (mIDH1) acute myeloid leukemia (AML). REZLIDHIA, an oral inhibitor of mIDH1, is designed to lower 2-hydroxyglutarate levels and promote normal differentiation of myeloid cells. It is already approved for use in relapsed or refractory (R/R) mIDH1 AML.

The clinical study is being conducted by The University of Texas MD Anderson Cancer Center and began enrolling patients in August 2024 under the trial identifier NCT06445959. Dr. Courtney DiNardo, a Professor of Leukemia at MD Anderson, is leading the trial. This multi-center, open-label, non-randomized study aims to assess the safety and tolerability of the combination therapy in its Phase 1b stage. The trial will then proceed to Phase 2 to evaluate the complete remission rate among 60 patients, split equally between newly diagnosed and R/R mIDH1 AML cases.

This trial marks the first project in Rigel's strategic alliance with MD Anderson to develop treatments for hematologic neoplasms involving IDH1 mutations. According to Raul Rodriguez, Rigel's president and CEO, combining REZLIDHIA with other AML treatments could offer new, entirely oral treatment options for patients who urgently need innovative therapies. Rodriguez emphasized the strength of the partnership with MD Anderson in exploring the potential of REZLIDHIA for treating AML and other blood cancers.

The alliance between Rigel and MD Anderson will also investigate the efficacy of olutasidenib in combination with other drugs for treating patients with newly diagnosed or recurrent AML, higher-risk myelodysplastic syndromes (MDS), and advanced myeloproliferative neoplasms (MPN). Additionally, the collaboration will look into the use of olutasidenib as a monotherapy for conditions like clonal cytopenia of undetermined significance (CCUS), lower-risk MDS, and as maintenance therapy for patients following hematopoietic stem cell transplants.

Acute myeloid leukemia (AML) is a fast-progressing cancer of the blood and bone marrow that impairs the development of myeloid cells into mature blood cells. Predominantly affecting adults, AML accounts for roughly 1% of all adult cancers. The American Cancer Society estimates around 20,800 new cases of AML will be diagnosed in the United States in 2024, mostly among adults. AML can relapse in about half of the patients, where the disease returns after remission. Refractory AML, which affects 10-40% of newly diagnosed patients, occurs when remission is not achieved despite intensive treatment. Each additional treatment phase for AML typically results in a decline in the patient's quality of life, highlighting the ongoing need for effective and well-tolerated therapies for relapsed or refractory AML cases.

Rigel Pharmaceuticals, Inc., based in South San Francisco, California, was founded in 1996. The company focuses on discovering, developing, and providing innovative therapies to improve the lives of patients with hematologic disorders and cancer.

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