- Ivosidenib NDA for IDH1m R/R AML Submitted in December for Priority Review; Multiple Pivotal Trials Ongoing
or Planned for Ivosidenib in Frontline AML and Solid Tumors Including 4Q 2018 Initiation of Phase 3 Trial with Intensive
Chemotherapy -
- Two Pivotal Trials in Pyruvate Kinase Deficiency with AG-348, ACTIVATE-T to Initiate in 1Q 2018 and
ACTIVATE in 2Q 2018; Phase 2 Proof of Concept Trial of AG-348 in Thalassemia Planned for 4Q 2018 -
- MAT2A Inhibitor AG-270 Expected to Enter Phase 1 Dose-Escalation Trial in MTAP-Deleted Tumors in 1Q 2018
-
- Seventh IND Submission for a DHODH Inhibitor for the Treatment of Hematologic Malignancies Expected in 4Q
2018 -
- 2017 Year End Cash, Cash Equivalents and Marketable Securities of $568M Funds Expanded Clinical and
Research Programs Through the End of 3Q 2019 -
SAN FRANCISCO, Jan. 08, 2018 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field
of cellular metabolism to treat cancer and rare genetic diseases, today summarized key 2018 priorities in conjunction with its
presentation at the 36th Annual J.P. Morgan Healthcare Conference in San Francisco. The presentation will outline how
Agios’ clinical and research programs have the potential to provide meaningful benefit to a large number of patients. The company
will webcast its presentation today at 9:30 a.m. PT (12:30 p.m. ET) at investor.agios.com.
“We presented a significant amount of clinical data across our portfolio in 2017, including data supporting the approval of our
first internally discovered medicine, which proves that our research and development engine has the ability to deliver important
precision medicines from a discovery in the lab to patients as an approved drug,” said David Schenkein, M.D., chief executive
officer at Agios. “We are very pleased with the early launch performance of IDHIFA® and expect to repeat this success
with ivosidenib upon FDA approval in 2018, while continuing our label expansion opportunities for frontline AML and solid tumors
and bringing our next drug candidate targeting genetically defined cancers, AG-270, into the clinic. In rare genetic diseases,
AG-348 has demonstrated proof of concept in pyruvate kinase deficiency and in addition to beginning pivotal trials in this disease,
we are exploring the utility of PK activation in other anemias, starting with a Phase 2 study in thalassemia.”
The company plans to achieve the following key milestones in 2018:
Cancer:
- Potential FDA approval and commercialization of ivosidenib for relapsed/refractory (R/R) acute myeloid leukemia (AML) with an
isocitrate dehydrogenase-1 (IDH1) mutation in the United States in the second half of 2018.
- Plan to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for ivosidenib for IDH1m
R/R AML in the fourth quarter of 2018.
- Support with Celgene an intergroup sponsored, global, registration-enabling Phase 3 trial combining ivosidenib or enasidenib
and standard induction (7+3) and consolidation chemotherapy with a primary endpoint of event free survival (EFS) in frontline AML
patients with an IDH1 or IDH2 mutation in the fourth quarter of 2018.
- Initiate a perioperative ‘window’ trial with ivosidenib and AG-881 in low-grade glioma to further investigate their effects
on brain tumor tissue in the first half of 2018.
- Initiate a Phase 1 dose-escalation trial for AG-270, a first-in-class methionine adenosyltransferase 2a (MAT2A) inhibitor, in
methylthioadenosine phosphorylase (MTAP)-deleted tumors in the first quarter of 2018.
Rare Genetic Diseases:
- Initiate two global pivotal trials for AG-348 in PK deficiency:
- ACTIVATE-T: A single arm trial of approximately 20 regularly transfused patients is expected to initiate in the first
quarter of 2018.
- ACTIVATE: A 1:1 randomized, placebo-controlled trial of 80 patients who do not receive regular transfusions is expected
to initiate in the second quarter of 2018.
- Initiate a global registry for adult and pediatric patients with PK deficiency (PEAK) in the first quarter of 2018 to
increase understanding of the long-term disease burden of this chronic anemia.
- Initiate a Phase 2 proof of concept trial of AG-348 in thalassemia in the fourth quarter of 2018.
Research:
- Submit an investigational new drug (IND) application for our latest development candidate, an inhibitor of the metabolic
enzyme dihydroorotate dehydrogenase (DHODH) for the treatment of hematologic malignancies in the fourth quarter of 2018.
The company plans to host an analyst day in the first half of 2018 to review Agios’ commercial readiness and broad clinical
development programs and highlight the depth of the research portfolio across oncology, rare genetic disease and metabolic
immuno-oncology.
The company also highlighted key 2017 achievements:
- Collaborated with Celgene to achieve the U.S. Food and Drug Administration (FDA) full approval and subsequent launch of
IDHIFA® (enasidenib) for the treatment of adult patients with R/R AML with an isocitrate dehydrogenase-2 (IDH2)
mutation as detected by an FDA approved diagnostic test.
- Submitted a new drug application (NDA) to the FDA for ivosidenib for the treatment of patients with R/R AML with an IDH1
mutation.
- Initiated a global, registration-enabling Phase 3 study (AGILE) combining ivosidenib and VIDAZA® in newly
diagnosed AML patients with an IDH1 mutation ineligible for intensive chemotherapy.
- Finalized two global, pivotal trial designs evaluating AG-348 in adults with pyruvate kinase (PK) deficiency.
- Achieved FDA clearance of an IND application for AG-270, a MAT2A inhibitor, targeting MTAP-deleted tumors.
2017 Year-End Cash and Updated Guidance
Agios ended 2017 with approximately $568 million of cash, cash equivalents and marketable securities. Based on its expanded
clinical and research programs announced today, the company now expects that its existing cash, cash equivalents and marketable
securities as of December 31, 2017, together with anticipated interest income, anticipated expense reimbursements, and royalty
payments under our collaboration agreements, but excluding any additional program-specific milestone payments, will enable the
company to fund its anticipated operating expenses and capital expenditure requirements through the end of third quarter of
2019.
Presentation at 36th Annual J.P. Morgan Healthcare Conference
Agios will webcast its corporate presentation from the 36th Annual J.P. Morgan Healthcare Conference in San Francisco
on Monday, January 8, 2018 at 9:30 a.m. PT (12:30 p.m. ET). A live webcast of the presentation can be accessed under "Events &
Presentations" in the Investors section of the company's website at agios.com. A replay of the webcast will be archived on the
Agios website for at least two weeks following the presentation.
About Agios
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 an approved oncology precision medicine 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.
About Agios/Celgene Collaboration
IDHIFA® (enasidenib), AG-881 and AG-270 are
part of Agios' global strategic collaboration with Celgene Corporation. Under the terms of the 2010 collaboration agreement,
Celgene has worldwide development and commercialization rights for IDHIFA® (enasidenib). Agios continues to conduct
certain clinical development activities within the IDHIFA® (enasidenib) development program and is eligible to receive
reimbursement for those development activities and up to $95 million in remaining payments assuming achievement of certain
milestones, and royalties on any net sales. Celgene and Agios are currently co-commercializing IDHIFA® (enasidenib) in
the U.S. Celgene will reimburse Agios for costs incurred for its co-commercialization efforts. For AG-881, the companies have a
joint worldwide development and 50/50 profit share collaboration, and Agios is eligible to receive regulatory milestone payments of
up to $70 million. AG-270 is part of a 2016 global research collaboration agreement with Celgene. Celgene has the option to
participate in a worldwide cost and profit share with Agios, under which the parties will share all development costs, subject to
specified exceptions, and any profits on net sales and Agios will be eligible for up to $169 million in clinical and regulatory
milestone payments for the program.
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 Agios’ plans, strategies and expectations for its and its
collaborator’s preclinical, clinical and commercial advancement of its drug development programs including IDHIFA®,
ivosidenib, AG-881, AG-348 and AG-270; the potential benefits of Agios' product candidates; its key milestones for 2018; its
estimates regarding its balance of cash, cash equivalents and marketable securities for the year ended December 31, 2017; its
financial guidance regarding the period in which it will have capital available to fund its operations; and the potential benefit
of its strategic plans and focus. The words “anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,”
“project,” “would,” “could,” “potential,” “possible,” “hope,” “strategy,” “milestone,” “will,” 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 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 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.
Contacts
Investors:
Renee Leck, 617-649-8299
Senior Manager, Investor & Public Relations
Renee.Leck@agios.com
Media:
Holly Manning, 617-844-6630
Associate Director, Corporate Communications
Holly.Manning@agios.com