Full Press Release Details
Agios Presents Updated Data from Phase 1 Dose-Escalation Study of
AG-881 in Patients with IDH Mutant Positive Advanced Glioma
Duration of 15 Months Observed in Non-Enhancing Glioma with 13 Patients Remaining on Treatment Provides Evidence of Prolonged Disease Control
Favorable Safety Profile at 10 mg and 50 mg, Doses Under Evaluation in Ongoing Glioma Perioperative Study
NEW ORLEANS, November 16, 2018 Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat
cancer and rare genetic diseases, today presented updated data from the ongoing Phase 1 study evaluating single agent AG-881 in advanced glioma. The data were featured in an oral presentation at the Society
for Neuro-Oncology (SNO) Annual Meeting in New Orleans. AG-881 is an investigational, oral, selective, potent inhibitor of the mutant isocitrate dehydrogenase-1 (IDH1)
and IDH2 enzymes and was designed for enhanced brain penetrance for development in IDH-mutant glioma.
additional follow-up, the AG-881 Phase 1 dose-escalation data continue to show a favorable safety profile at the doses selected for the perioperative study. Longer
treatment duration and a reduction in tumor growth rates are encouraging signs of clinical activity in low-grade glioma, said Ingo Mellinghoff, M.D., Memorial Sloan Kettering Cancer Center, an
investigator for the study. Ultimately, use of an IDH inhibitor in this difficult-to-treat disease has the potential to improve the current treatment paradigm by
delaying the multiple rounds of surgery, radiation and chemotherapy that many patients endure.
With no curative or approved targeted
therapies and a high frequency of IDH1 mutations in low-grade glioma, we are committed to advancing one of our IDH inhibitors to a registrational study in this disease, said Chris Bowden, M.D., chief
medical officer at Agios. We are continuing to collect clinical data for both ivosidenib and AG-881, along with feedback from regulators and the neuro-oncology community, to make an internal decision on
our glioma pivotal strategy by the end of this year.
AG-881 is being evaluated as a single agent in an ongoing Phase 1 dose-escalation trial in IDH1/2 mutant advanced solid tumors, including glioma. Enrollment
was completed in June 2017. Dose escalation data as of March 29, 2018 were presented at the 2018 American Society of Clinical Oncology (ASCO) Annual Meeting. As of the updated July 20, 2018 data cut-off, study design, enrollment and
baseline characteristics of the 52 glioma patients remain unchanged, as reported below.
Updated safety and efficacy data on the 52 patients with enhancing and non-enhancing glioma,
including an exploratory tumor volume growth rate analysis, are reported below.
The safety analysis conducted for all 52
glioma patients as of the data cut-off demonstrated that AG-881 continues to have a favorable safety profile at dose levels below 100 mg.
Efficacy data from the 52 glioma patients (22 with non-enhancing and 30 with enhancing disease) as of the data cut-off showed:
Ongoing Glioma Perioperative Study Presented in Trials in Progress Poster
A perioperative window trial with ivosidenib and AG-881 (10 mg and 50 mg) in up to 45 IDH1m non-enhancing low-grade glioma patients is ongoing and being presented today as part of a trials in progress poster. The goal of the trial is to confirm CNS penetrance and
tumor 2-HG suppression of ivosidenib and AG-881 as part of the strategy to finalize internal pivotal development plans by
Glioma presents in varying degrees of tumor aggressiveness, ranging from slower growing (low-grade glioma) to rapidly
progressing (high-grade glioma-Glioblastoma Multiforme). Common symptoms include seizures, memory disturbance, sensory impairment and neurologic deficits. The long-term prognosis is poor with a five-year survival rate of 33 percent.
Approximately 11,000 low-grade glioma patients are diagnosed annually in the U.S. and EU and approximately 80 percent have an IDH1 mutation.
Agios is focused on discovering and
developing novel investigational medicines to treat cancer and rare genetic diseases through scientific leadership in the field of cellular metabolism. In addition to an active research and discovery pipeline across both therapeutic areas, Agios has
two approved oncology precision medicines and multiple first-in-class investigational therapies 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 www.agios.com.
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 AG-881; Agios s plans for future clinical development of AG-881; and the potential benefit of
Agios s strategic plans and focus. The words could, expect, intend, may, path, plan, potential, strategy, will, and similar expressions
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 collaborator, Celgene, 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. FDA and 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, such as its agreements with Celgene and CStone Pharmaceuticals; 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.
Renee Leck, 617-649-8299
Associate Director, Investor Relations
Holly Manning, 617-844-6630
Associate Director, Corporate Communications