Rigel Pharmaceuticals & MD Anderson Announce Strategic Alliance to Advance REZLIDHIA in AML & Other Cancers

Rigel Pharmaceuticals, Inc. and The University of Texas MD Anderson Cancer Center (MD Anderson) recently announced a multi-year strategic development collaboration to expand the evaluation of REZLIDHIA (olutasidenib) in acute myeloid leukemia (AML) and other hematologic cancers.

The alliance brings together MD Anderson’s clinical research expertise with Rigel’s differentiated targeted molecule. Under the strategic collaboration, Rigel and MD Anderson will evaluate the potential of olutasidenib to treat newly diagnosed and relapsed or refractory (R/R) patients with AML, higher-risk myelodysplastic syndromes (MDS), and advanced myeloproliferative neoplasms (MPN), in combination with other agents. The collaboration will also support the evaluation of olutasidenib as monotherapy in lower-risk MDS and as maintenance therapy in post-hematopoietic stem cell transplant patients.

“We are excited to enter into this strategic alliance with the exceptional team at MD Anderson to evaluate REZLIDHIA as a potential therapy for a broad range of IDH1-mutant cancers,” said Raul Rodriguez, Rigel’s President and CEO. “We believe REZLIDHIA has the potential to become a standard of care for patients in urgent need of new hematology-oncology therapies. We look forward to a close collaboration with MD Anderson to advance this as a new therapeutic option for more patients.”

REZLIDHIA is a potent, selective, oral, small-molecule inhibitor of mutated IDH1 (mIDH1) designed to bind to and inhibit mIDH1 to reduce 2-hydroxyglutarate levels and restore normal cellular differentiation of myeloid cells. REZLIDHIA is approved by the US FDA for the treatment of adult patients with R/R AML with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test.

“Based on its differentiated profile and compelling clinical data to date, REZLIDHIA has the potential, beyond its currently approved indication, to benefit patients with various cancers where mutant IDH1 is thought to play a role,” said Courtney DiNardo, MD, Professor of Leukemia. “We look forward to collaborating with Rigel to conduct in-depth studies that will determine the broader potential of REZLIDHIA in these patient populations.”

Rigel and MD Anderson will jointly lead all clinical development efforts, which will be overseen by a joint steering committee. Rigel will provide $15 million in time-based milestone payments and study material over the five-year collaboration. Rigel will retain all rights to its programs under the collaboration.

Acute myeloid leukemia (AML) is a rapidly progressing cancer of the blood and bone marrow that affects myeloid cells, which normally develop into various types of mature blood cells. AML occurs primarily in adults and accounts for about 1 percent of all adult cancers. The American Cancer Society estimates that in the United States alone, there will be about 20,380 new cases, most in adults, in 2023.

Relapsed AML affects about half of all patients who, following treatment and remission, experience a return of leukemia cells in the bone marrow.³ Refractory AML, which affects between 10 and 40 percent of newly diagnosed patients, occurs when a patient fails to achieve remission even after intensive treatment.⁴ Quality of life declines for patients with each successive line of treatment for AML, and well-tolerated treatments in relapsed or refractory disease remain an unmet need.

REZLIDHIA is indicated for the treatment of adult patients with relapsed or refractory AML with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test.

Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) is a biotechnology company dedicated to discovering, developing and providing novel therapies that significantly improve the lives of patients with hematologic disorders and cancer. Founded in 1996, Rigel is based in South San Francisco, California. For more information, visit www.rigel.com.