CAMBRIDGE - Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, today announced the withdrawal of its European Marketing Authorization Application (MAA) for TIBSOVO (ivosidenib tablets) for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with an isocitrate dehydrogenase-1 (IDH1) mutation.

The decision is based on feedback from the European Medicine Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) that the available clinical data from the company's single arm, uncontrolled Phase 1 study do not sufficiently support a positive benefit-risk balance for the proposed indication.

'We are disappointed not to be able to bring TIBSOVO to AML patients in the EU based on our current data in the relapsed/refractory setting, given the tremendous need for new targeted treatment options for this devastating disease,' said Chris Bowden, M.D., chief medical officer at Agios. 'We continue to believe in the benefit of TIBSOVO for patients with AML and are advancing our two ongoing Phase 3 randomized controlled trials evaluating TIBSOVO combinations in newly diagnosed AML. If these trials are positive, we anticipate pursuing approvals in these indications in both the U.S. and EU.'

About TIBSOVO Clinical Development in AML

Agios is currently enrolling two Phase 3 combination trials in newly diagnosed AML: AGILE, a Phase 3 trial of TIBSOVO in combination with azacitidine in newly diagnosed AML patients who are not eligible for intensive chemotherapy

HOVON150/AMLSG29, an intergroup-sponsored Phase 3 trial evaluating TIBSOVO or IDHIFA (enasidenib) in combination with standard induction and consolidation chemotherapy in newly diagnosed AML patients who are eligible for intensive chemotherapy

The majority of AML patients with an IDH1 mutation are eligible for intensive therapy or non-intensive therapy.

About TIBSOVO (ivosidenib)

TIBSOVO is indicated for the treatment of acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test in: Adult patients with newly-diagnosed AML who are 75 years old or who have comorbidities that preclude use of intensive induction chemotherapy.

About Agios

Agios is focused on discovering and developing novel investigational medicines to treat malignant hematology, solid tumors and rare genetic diseases through scientific leadership in the field of cellular metabolism. In addition to an active research and discovery pipeline across these three therapeutic areas, Agios has two approved oncology precision medicines and multiple first-in-class investigational therapies in clinical and/or preclinical development.

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 TIBSOVO (ivosidenib tablets) and Agios' strategic plans and prospects. The words 'anticipate,' 'believe,' 'estimate,' 'expect,' 'intend,' 'may,' 'plan,' 'predict,' 'project,' 'would,' 'could,' 'potential,' 'possible,' 'hope' 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 or its collaborators 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, without limitation: risks and uncertainties related to the impact of the COVID-19 pandemic to Agios' business, operations, strategy, goals and anticipated milestones, including its ongoing and planned research activities, ability to conduct ongoing and planned clinical trials, clinical supply of current or future drug candidates, commercial supply of current or future approved products, and launching, marketing and selling current or future approved products; 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. FDA, the EMA or other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies; Agios' ability to obtain and maintain requisite regulatory approvals and to enroll patients in its planned clinical trials; unplanned cash requirements and expenditures; competitive factors; Agios' ability to obtain, maintain and enforce patent and other intellectual property protection for any product candidates it is developing; Agios' ability to maintain key collaborations and general economic and market conditions. These and other risks are described in greater detail under the caption 'Risk Factors' included in Agios' public filings with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Agios expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.###

Contact:

Holly Manning

Tel: 617-844-6630

Email: Holly.Manning@agios.com

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