On January 8, 2018 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported key 2018 priorities in conjunction with its presentation at the 36th Annual J.P. Morgan Healthcare Conference in San Francisco (Press release, Agios Pharmaceuticals, JAN 8, 2018, View Source [SID1234522985]). 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.

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"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.