our programs

Acute Myeloid Leukemia

Olutasidenib

Acute myeloid leukemia (AML) is a cancer that starts in a person’s bone marrow but often quickly moves into the blood. AML develops from immature blood cells, known as myeloid cells, that are supposed to mature into white blood cells. However, the diseased myeloid cells don’t function properly. They instead multiply rapidly which causes normal blood cell production to fail.

AML occurs primarily in adults and accounts for about 1% of all adult cancers. The American Cancer Society estimates that about 19,940 new cases, most in adults, will arise in 2020 in the United States alone.1

Myeloid leukemia is considered acute when the inhibition of blood cell production progresses quickly. We’re specifically targeting a large subsection of patients with acute myeloid leukemia who are not achieving remission with existing treatment options.

A relapse is the return of cancer after treatment. Relapsed AML affects about 50% of all patients who, following treatment and remission, experience a return of leukemia cells in the bone marrow.2 Refractory AML, which affects between 10% and 40% of newly diagnosed patients, occurs when a patient fails to achieve remission even after intensive treatment.3

Quality of life declines for patients with each successive line of treatment for acute myeloid leukemia, and well-tolerated treatments in relapsed disease remain an unmet need.

~20 k

people will be diagnosed with AML in the U.S. this year

~50 %

patients experience relapsed AML

10-40 %

newly diagnosed patients have refractory AML

Quotation marks

AML is a relentless disease. There’s significant need to find treatment options for people who aren’t achieving remission or whose cancer has returned. We hope to make a meaningful difference for these patients.

Tino

EVP, head of research and development

Our selective inhibitor for cancers with IDH1 mutations.

In AML, IDH1 mutations inhibit normal cell differentiation and lead to the accumulation of immature myeloid cells. These mutations also cause the excessive production of specific oncometabolites that are linked to the impairment of stem cell differentiation and promotion of cancer cell growth. IDH1 mutations are present in 6-9% of patients with acute myeloid leukemia.4,5

Olutasidenib, an investigational agent, is a selective inhibitor for cancers with IDH1 mutations and is being studied in patients with AML and glioma.

Development Status

In an ongoing Phase 2 trial, olutasidenib yielded positive study results in patients with relapsed/refractory (R/R) AML. A New Drug Application (NDA) for olutasidenib has been filed with the FDA with a Prescription Drug User Fee Act (PDUFA) goal date of February 15, 2023. We have exclusively licensed olutasidenib to Rigel Pharmaceuticals to develop, manufacture and commercialize for any uses worldwide, including for the treatment of R/R AML. We will continue to progress the NDA filing of olutasidenib and the completion of the registrational study.

The key objectives of the Phase 1/2 trial are to evaluate the safety, efficacy and pharmacokinetics and pharmacodynamics of olutasidenib as a single agent or in combination with azacitidine. The primary cohort of this multi-arm study is evaluating olutasidenib as a single agent. Other arms include olutasidenib in combination with azacitidine for patients who have failed a prior IDH1 mutation inhibitor or those who are treatment naïve but are contraindicated for standard-of-care treatments.

Learn more about our Phase 2b acute myeloid leukemia trial
1 The American Cancer Society. (2020). Key statistics for acute myeloid leukemia (AML). Retrieved from link.
2 Leukaemia Care. (2019). Relapse in Acute Myeloid Leukaemia (AML). Retrieved from link.
3 Thol, F., Schlenk, R. F., Heuser, M., & Ganser, A. (2015). How I treat refractory and early relapsed acute myeloid leukemia. Blood, 126(3): 319-327. Retrieved from link.
4 Patel, K., Ravandi, F., Ma, D., Paladugu, A., Barkoh, B. A., Medeiros, L. J., & Luthra, R. (2011). Acute myeloid leukemia with IDH1 or IDH2 mutation: Frequency and clinicopathologic features. American Journal of Clinical Pathology, 135(1): 35-45. Retrieved from link.
5 National Comprehensive Cancer Network, Inc. (2019). NCCN clinical practice guidelines in oncology: Acute myeloid leukemia. Official Journal of the National Comprehensive Cancer Network, 17(6). Retrieved from link.