Agios Outlines Key 2018 Priorities Expanding Clinical and Research Programs to Drive Long Term Value
- 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 -
- Seventh IND Submission for a DHODH Inhibitor for the Treatment of Hematologic Malignancies Expected in 4Q 2018 -
- 2017 Year End Cash,
“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
The company plans to achieve the following key milestones in 2018:
FDAapproval and commercialization of ivosidenib for relapsed/refractory (R/R) acute myeloid leukemia (AML) with an isocitrate dehydrogenase-1 (IDH1) mutation in the United Statesin 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
Celgenean 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.
- 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
Celgeneto 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 FDAapproved diagnostic test.
- Submitted a new drug application (NDA) to the
FDAfor 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.
FDAclearance 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
Presentation at 36th Annual
Agios will webcast its corporate presentation from the 36th Annual
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
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,
Senior Manager, Investor & Public Relations
Associate Director, Corporate Communications
Source: Agios Pharmaceuticals, Inc.