Agios Announces Initiation of Phase 1/2 Frontline Combination Study of AG-221 or AG-120 with VIDAZA® (azacitidine for injection) in Newly Diagnosed Acute Myeloid Leukemia (AML) Patients Not Eligible for Intensive Chemotherapy
“Many newly diagnosed AML patients cannot tolerate intensive chemotherapy, which limits their available treatment options,” said
“We are rapidly executing our frontline strategy for our IDH inhibitors, having now initiated a second study in newly diagnosed AML patients,” said
About the Phase 1/2 Frontline Combination Trial of AG-221 or AG-120 with VIDAZA® in Newly Diagnosed AML Patients Not Eligible for Intensive Chemotherapy
The Phase 1/2, multicenter, international, open-label clinical trial will evaluate the safety and clinical activity of AG-221 or AG-120 in combination with VIDAZA® in patients with newly diagnosed AML with an IDH2 and/or IDH1 mutation who are not eligible for intensive chemotherapy. The study consists of a Phase 1b dose-escalation stage and a Phase 2 randomized stage.
The study will evaluate AG-221 administered at an initial oral dose of 100 mg once daily in patients with an IDH2 mutation or AG-120 administered at an initial oral dose of 500 mg once daily in patients with an IDH1 mutation. AG-221 or AG-120 will be administered continuously in a 28-day cycle with VIDAZA® at the standard 75 mg/m2 daily dose for 7 days of each 28-day cycle.
The primary endpoint of the Phase 1b stage of the trial is to determine safety and tolerability and to establish the recommended Phase 2 dose of AG-221 or AG-120 in combination with VIDAZA®. The primary endpoint of the Phase 2 stage of the trial is to determine the efficacy of the combination of AG-221 or AG-120 with VIDAZA® compared with VIDAZA® alone. Secondary endpoints include evaluation of safety, characterization of pharmacokinetics and evaluation of effects on health-related quality-of-life outcomes. This study will enroll up to 150 patients.
Please refer to www.clinicaltrials.gov for additional clinical trial details.
About Acute Myelogenous Leukemia (AML)
AML, a cancer of blood and bone marrow characterized by rapid disease progression, is the most common acute leukemia affecting adults. Undifferentiated blast cells proliferate in the bone marrow rather than mature into normal blood cells. AML incidence significantly increases with age, and according to the
About IDH Mutations and Cancer
IDH1 and IDH2 are two metabolic enzymes that are mutated in a wide range of hematologic and solid tumor malignancies, including AML. Normally, IDH enzymes help to break down nutrients and generate energy for cells. When mutated, IDH increases production of an oncometabolite 2-hydroxyglutarate (2HG) that alters the cells' epigenetic programming, thereby promoting cancer. 2HG has been found to be elevated in several tumor types. Agios believes that inhibition of the mutated IDH proteins may lead to clinical benefit for the subset of cancer patients whose tumors carry them.
Agios is focused on discovering and developing novel investigational medicines to treat cancer and rare genetic metabolic disorders through scientific leadership in the field of cellular metabolism. In addition to an active research and discovery pipeline across both therapeutic areas, Agios has multiple first-in-class investigational medicines in clinical and/or preclinical development. All Agios programs focus on genetically identified patient populations, leveraging our knowledge of metabolism, biology and genomics. For more information, please visit the company's website at agios.com.
About Agios/Celgene Collaboration
VIDAZA® is a registered trademark of
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the potential benefits of Agios' product candidates targeting IDH mutations, including AG-221 and AG-120; its plans and timelines for the clinical development of AG-221 and AG-120; and the benefit of its strategic plans and focus. The words "anticipate," "believe," "estimate," "expect," "intend," "may," "plan," "predict," "project," "potential," "possible," "hope," "could," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from Agios' current expectations and beliefs. For example, there can be no guarantee that any product candidate Agios is developing will successfully commence or complete necessary preclinical and clinical development phases, or that development of any of Agios' product candidates will successfully continue. There can be no guarantee that any positive developments in Agios' business will result in stock price appreciation. Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other important factors, including: Agios' results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the U.S.
Renee Leck, Senior Manager, Investor & Public Relations Renee.Leck@agios.com 617-649-8299