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8-K
AGIOS PHARMACEUTICALS INC filed this Form 8-K on 01/09/2017
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Form 8-K

 

 

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

WASHINGTON, D.C. 20549

 

 

FORM 8-K

 

 

CURRENT REPORT

Pursuant to Section 13 or 15(d)

of the Securities Exchange Act of 1934

Date of Report (Date of earliest event reported): January 9, 2017

 

 

Agios Pharmaceuticals, Inc.

(Exact Name of Registrant as Specified in Charter)

 

 

 

Delaware   001-36014   26-0662915

(State or Other Jurisdiction

of Incorporation)

 

(Commission

File Number)

 

(IRS Employer

Identification No.)

88 Sidney Street, Cambridge, MA   02139
(Address of Principal Executive Offices)   (Zip Code)

Registrant’s telephone number, including area code: (617) 649-8600

(Former Name or Former Address, if Changed Since Last Report)

 

 

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):

 

  Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

 

  Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

 

  Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

 

  Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

 

 

 


Item 2.02 Results of Operations and Financial Condition.

Although it has not finalized its full financial results for the fourth quarter and fiscal year ended December 31, 2016, Agios Pharmaceuticals, Inc. (the “Company”) announced on January 9, 2017, that it expects to report that it had approximately $574 million of cash, cash equivalents and marketable securities as of December 31, 2016.

The information contained in Item 2.02 of this Form 8-K is unaudited and preliminary, and does not present all information necessary for an understanding of the Company’s financial condition as of December 31, 2016 and its results of operations for the three months and year ended December 31, 2016. The audit of the Company’s consolidated financial statements for the year ended December 31, 2016 is ongoing and could result in changes to the information set forth above.

The information in this Item 2.02 shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”) or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended (the “Securities Act”), or the Exchange Act, except as expressly set forth by specific reference in such a filing.

Item 7.01 Regulation FD Disclosure.

On January 9, 2017, the Company intends to make a slide presentation at the 35th Annual J.P. Morgan Healthcare Conference. A form of the slide presentation is being furnished as Exhibit 99.1 to this Current Report on Form 8-K.

The information responsive to Item 7.01 of this Form 8-K, including Exhibit 99.1, shall not be deemed “filed” for purposes of Section 18 of the Exchange Act or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act or the Exchange Act, except as expressly set forth by specific reference in such a filing.

Item 8.01 Other Events.

On January 9, 2017, the Company issued a press release outlining its 2017 milestones for its development programs and research pipeline, which will be discussed at the Company’s presentation at the 35th Annual J.P. Morgan Healthcare Conference on January 9, 2017. The full text of the press release issued in connection with this announcement is filed as Exhibit 99.2 to this Current Report on Form 8-K and is incorporated herein by reference.

Forward Looking Statements

This Current Report on Form 8-K and the exhibits attached hereto contain forward-looking statements of the Company that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this Current Report on Form 8-K and the exhibits attached hereto, are forward-looking statements. The words “anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “target,” “potential,” “will,” “would,” “could,” “should,” “continue,” “contemplate,” or the negative of these terms or other similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. These forward-looking statements include, among others, statements about: the Company’s estimates regarding its balance of cash, cash equivalents and marketable securities for the year ended December 31, 2016 and other expectations regarding its business, plans, prospects and strategies. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements that the Company makes due to a number of important factors, including those Risk Factors discussed in the Company’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2016, and other filings that the Company may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this Current Report on Form 8-K speak only as of the date hereof, and the Company 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.

Item 9.01 Financial Statements and Exhibits.

(d) The following exhibits are included in this report:

 

Exhibit No.

  

Description

99.1    Form of Presentation as of January 9, 2017.
99.2    Press release issued by Agios Pharmaceuticals, Inc. on January 9, 2017.


SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

 

    AGIOS PHARMACEUTICALS, INC.
Date: January 9, 2017     By:  

/s/ David P. Schenkein

     

    David P. Schenkein, M.D.

    President and Chief Executive Officer


EXHIBIT INDEX

 

Exhibit No.

  

Description

99.1    Form of Presentation as of January 9, 2017.
99.2    Press release issued by Agios Pharmaceuticals, Inc. on January 9, 2017.
EX-99.1

Slide 1

Agios: Delivering Our First Medicines to Patients JPMorgan Healthcare Conference David Schenkein, M.D. Chief Executive Officer January 9, 2017 Exhibit 99.1


Slide 2

Forward Looking Statements This presentation and various remarks we make during this presentation contain forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the Agios’ plans, strategies and expectations for its and its collaborator’s preclinical, clinical and commercial advancement of its drug development programs including enasidenib, AG-120, and AG-348; the potential benefits of Agios' product candidates; its key milestones for 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 presentation and various remarks we make during this presentation 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' Quarterly Report on Form 10-Q for the quarter ended September 30, 2016, and other filings that Agios may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this presentation and various remarks we make during this presentation 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.


Slide 3

Agios is passionately committed to applying our scientific leadership in the field of cellular metabolism to transform the lives of patients with cancer and rare genetic diseases. We Are Driven By a Clear Vision and Values CANCER METABOLISM  METABOLIC IMMUNO-ONCOLOGY  RARE GENETIC DISEASES  DYSREGULATED METABOLISM Building a Global, Commercial-Stage Biopharmaceutical Company


Slide 4

January 2009 5


Slide 5

Science Team Discovery 2009 6


Slide 6

January 9, 2017 Delivering Our First Medicines to Patients Science Team Discovery 7


Slide 7

PKR Continue to demonstrate leadership in PK deficiency Prepare for 1H 2018 pivotal trial initiation for wholly owned AG-348 in PK deficiency IDH Secure approval and co-commercialize enasidenib for R/R AML in the U.S. Submit NDA for wholly owned AG-120 in R/R AML by YE 2017 Initiate Phase 3 combining AG-120 and VIDAZA® in frontline AML in 1H 2017 Advance next wave of research in three areas of expertise: cancer metabolism, rare genetic diseases and metabolic immuno-oncology File IND application for MTAP pathway development candidate by YE 2017 RESEARCH 2017 Key Priorities & Expected Milestones 8


Slide 8

Delivering Our First Medicines to Patients Commercial Stage Biopharmaceutical Company Science Team Discovery 450+ employees 2 approved precision medicines in AML 2017 – 2018 4+ clinical-stage molecules 3+ pivotal trials (IDH, PKR) 100% committed to helping patients 3 areas of research expertise 9


Slide 9

IDH PKR RESEARCH 10


Slide 10

Repairing an IDH Mutant Cancer Cell IDHm Inhibitor Differentiation Mature Cell Normal Cell Death / HOMEOSTASIS Differentiation Progenitor Unchecked Cell Proliferation CANCER HSC Progenitor DIFFERENTIATION BLOCKED Me Me Me IDHm IDH Isocitrate aKG 2HG Epigenetic “Re-Wiring” Inhibitor 2HG Day 15 Day 28 Neutrophil Day 0 Leukemia Blast AG-120, EORTC, 2014 Mutation occurs early and persists throughout illness DIFFERENTIATION RESTORED


Slide 11

Repairing an IDH Mutant Cancer Cell IDHm Inhibitor Differentiation Mature Cell Normal Cell Death / HOMEOSTASIS Differentiation Progenitor Unchecked Cell Proliferation CANCER HSC Progenitor DIFFERENTIATION BLOCKED Me Me Me IDHm IDH Isocitrate aKG 2HG Epigenetic “Re-Wiring” Inhibitor 2HG AG-120, ASH, 2016 IDH1m Mutational Burden Mutation occurs early and persists throughout illness Days DIFFERENTIATION RESTORED


Slide 12

Goal All IDHm patients screened and treated with an IDHm inhibitor for the entire course of their disease Our Vision for IDHm Inhibitors A Roadmap for Speed and Breadth Now Next


Slide 13

Now Next Goal Now Relapsed/Refractory AML Enasidenib NDA submission 2016 AG-120 NDA submission by YE 2017 Our Vision for IDHm Inhibitors A Roadmap for Speed and Breadth


Slide 14

Next Frontline AML AG-120 + Vidaza® Phase 3 Enasidenib / AG-120 + (7+3) with maintenance Relapsed/Refractory AML Novel-novel combination Phase 1 studies Solid Tumors AG-120 Phase 3 cholangiocarcinoma AG-120 and AG-881 Phase 1 glioma expansion Glioma development strategy Our Vision for IDHm Inhibitors A Roadmap for Speed and Breadth


Slide 15

Incidence of AML Rising in U.S. and EU5 with Aging Population Sources: American Cancer Society: Cancer Facts and Figures Visser et. Al. Incidence, survival and prevalence of myeloid malignancies in Europe. Eur J Cancer. 2012 Nov;48(17):3257-66 Epiphany Partners Epic Oncology Decision Resources Thousands of Patients Incidence of AML (U.S. + EU5) Worldwide incidence of AML is growing in step with an aging population ~65% of incident patients in the U.S. and EU are 65 years of age or older It is estimated that ~15-23% of patients with AML will have an IDH mutation Acute Myeloid Leukemia (AML) IDH1m frequency IDH2m frequency 5-year overall survival 6-10% 9-13% 20-25% Multiple sources including market research SEER 15


Slide 16

AML Fit for Intensive Treatment (IC) Unfit for IC 7+3 HMA/LDAC Supportive care Clinical trial CR Non-CR Consolidation CTX 2nd induction Transplant if eligible CTX = Chemotherapy HMA = Hypomethylating Agent LDAC = Low dose Cytarabine Shifting the Treatment Paradigm for AML with Precision Medicine Long-term goal: treatment across multiple lines with an IDHm inhibitor


Slide 17

Randomization 1:1 Double Blinded AG-120 500mg (n=196) + Vidaza® Primary Endpoint OS Interim analyses for futility and superiority Advancing AG-120 into Frontline Setting Placebo (n=196) + Vidaza® Final OS Analyses Secondary Endpoints: CR, CR+CRh, EFS, ORR Trial on track for 1H 2017 initiation Global Phase 3 Frontline IC-Ineligible IDH1m AML IC = intensive chemotherapy Vidaza® is a registered trademark of Celgene Corporation


Slide 18

2017 2016 Building World-Class Commercial Capabilities for IDH Launches 2018 Co-commercialization with Celgene for enasidenib Established medical affairs and commercial leadership Chief Commercial Officer VP Market Access VP Sales VP Global Medical Affairs MSL deployment ongoing Field sales recruiting ongoing Buildout market access function Establish commercial supply chain Expand field sales organization for AG-120 18


Slide 19

Next Frontline AML AG-120 + Vidaza® Phase 3 Enasidenib / AG-120 + (7+3) with maintenance Relapsed/Refractory AML Novel-novel combination Phase 1 studies Solid Tumors AG-120 Phase 3 cholangiocarcinoma AG-120 and AG-881 Phase 1 glioma expansion Glioma development strategy Our Vision for IDHm Inhibitors A Roadmap for Speed and Breadth


Slide 20

Other solid tumor types include colon, melanoma, lung, ovarian. Multiple sources, including market research and SEER. Estimates will continue to evolve with additional future data *excludes primary GBM Treating Solid Tumors with an IDH1m Inhibitor Glioma Intrahepatic Cholangiocarcinoma (IHCC) Chondrosarcoma Low grade and 2ary GBM Bile ducts Cartilage Incidence (cases/year U.S.) 5K 2K – 4K 700 – 1000 IDH1m frequency 68-74% 11-24% 40-52% Treatment options Surgery, XRT Chemotherapy Surgery, Chemotherapy Liver transplantation Surgery, XRT Chemotherapy 5-year OS ~32-68%* ~9% ~10-90%


Slide 21

2:1 Double Blind Randomization (n=186) AG-120 Arm 500mg Registration-Enabling Phase 3 Cholangiocarcinoma Study Placebo Arm Primary Endpoint: PFS Crossover at time of progression Secondary Endpoints: OS, ORR, safety, QoL Trial Initiated in December 2016 Global Phase 3 Previously Treated IDH1m Cholangiocarcinoma


Slide 22

Phase 1 Dose-Escalation and Expansion (Completed) Encouraging Data with AG-120 Supports Clinical Development of IDH1m Inhibitor in Glioma AG-120 IDH1m Inhibition AG-881 High CNS Penetration, Pan-IDHm Inhibition Phase 1 Dose-Escalation (Ongoing) Potential Expansion 22 AG-120 Data Presented at SNO 2016


Slide 23

IDH PKR RESEARCH 23


Slide 24

PK Deficiency Is a Rare Genetic Disease that Affects Red Blood Cells Rare genetic disease of erythrocyte pyruvate kinase PK deficiency often presents at birth with jaundice and can cause lifelong hemolytic anemia and associated morbidities Estimated prevalence ranges from ~1:20K to ~1:485K1–4 PKR regulates a crucial step in red blood cell metabolism and when mutated causes premature death of these cells Healthy PKR ATP Glucose PK Deficiency Mutant PKR ATP Glucose Grace R et al. Am J Hematol 2015;90(9):825-30; 1Mohrenweiser HW PNAS 1981;78(8):5046-50; 2Carey PJ et al. Blood 2000;96(12):4005-6; 3Beutler E & Gelbart T Blood 2000;95(11):3585-8; 4deMedicis et al. Hum Hered 1992;42(3):179-83 Lactate Lactate AG-348


Slide 25

INFANT CHILD ADULT Supportive Treatments Complications Phototherapy Blood transfusions Removal of spleen Removal of gall bladder Blood transfusions Infection risk à lifelong prophylactic antibiotics Thrombosis risk Blood transfusions Iron overload à iron chelation therapy Disease Burden PK Deficiency Is a Lifelong Disease with Only Supportive Treatments IRON CHELATION (24%) GALLSTONES (42%) GALL BLADDER REMOVAL (37%) SPLEEN REMOVAL (68%) BLOOD CLOTS (5%) DECREASED BONE STRENGTH (6%) APLASTIC CRISIS (16%) RBC PRODUCTION IN INAPPROPRIATE ORGANS (14%) Baseline and retrospective data from 144 NHS patients | Source : R. Grace, poster ASH 2015


Slide 26

Quality of Life Impact Weighs Heavily for Both Patients & Families “If I say I’m tired, people think I need more sleep... I have fatigue so intense that it wakes me up at night. How do you get someone to understand that?!” “Think of a day when you are sick with a cold. That is me on my best day.”* *Quotes are from actual PK deficiency patients.


Slide 27

PK Deficiency Strategy Increase Understanding of Disease Burden Drive the Science Develop & Commercialize First Treatment Addressing Underlying Cause of Disease Demonstrate Commitment to PK Deficiency Community & Patients Agios Leadership in PK Deficiency


Slide 28

Compelling Proof-of-Concept for AG-348, the First Disease Modifying Therapy for PK Deficiency DRIVE PK Learnings Robust hemoglobin increases in 15 / 32 patients; 15 / 26 patients with 1 or more missense mutation Responses are rapid and sustained; median time to response of 1.4 weeks Majority of responders seen at doses ≤50 mg BID and as low as 5 mg QD Well-tolerated beyond six months of dosing Data presented at ASH 2016 52 patients enrolled; 17 completed first 24 weeks, 15 in extension


Slide 29

Key Considerations for AG-348 Pivotal Trial Design Design Element Considerations Rationale Patient Population Transfusion dependent adult (TD) Non-Transfusion dependent adult (NTD) Goal to treat all adult patients Size ~100 patients Rare disease Dose Dose titration up to optimal hemoglobin response Majority of responders seen at doses ≤50 mg BID and as low as 5 mg QD Endpoints Hemoglobin response (NTD) Reduction in transfusion frequency (TD) Patient-reported outcomes (PRO) Establish clinical benefit Control Placebo controlled Evaluate PRO Expect to initiate pivotal study in 1H 2018


Slide 30

IDH PKR RESEARCH 30


Slide 31

Agios’ Scientific Research Platform CANCER METABOLISM  RARE GENETIC DISEASES METABOLIC IMMUNO-ONCOLOGY  DYSREGULATED METABOLISM Inhibit key enzymes in cancer cell specific metabolic pathways to disrupt tumor cell proliferation and survival Restore defective metabolic pathways in disease cells that cause rare genetic disorders of metabolism Alter the metabolic state of immune cells to enhance the body’s anti-tumor response RESEARCH PLATFORM


Slide 32

Development Candidate for MTAP Pathway Selected IND Expected by Year-End Deletion of metabolic gene adjacent to tumor suppressor p16/p14 p16/p14 tumor suppressor locus deleted in 15% of all cancers Metabolic gene, MTAP, is adjacent to p16/p14 & typically co-deleted MTAP p16/p14 MTAP-deleted tumors constitute a large, genetically defined patient population ~75,000 new patients/year in U.S. with MTAP deletion across many indications chr9p21


Slide 33

Delivering Our First Medicines to Patients Commercial Stage Biopharmaceutical Company Science Team Discovery 450+ employees 2 approved precision medicines in AML 2017 – 2018 4+ clinical-stage molecules 3+ pivotal trials (IDH, PKR) 100% committed to helping patients 3 areas of research expertise 33

EX-99.2

Exhibit 99.2

 

LOGO

Agios Announces Key Upcoming Milestones to Support Evolution to a Commercial Stage Biopharmaceutical Company in 2017

- Enasidenib (AG-221) NDA Submitted for IDH2m Relapsed/Refractory (R/R) Acute Myeloid Leukemia (AML) -

- AG-120 NDA Submission for IDH1m R/R AML Planned by Year End 2017 -

- AG-348 Advancing to Pivotal Development in PK Deficiency -

- Development Candidate for MTAP Pathway Selected; IND Submission Expected by Year End 2017 -

- Company Ends 2016 in a Strong Financial Position with $574M in Cash, Cash Equivalents and Marketable Securities -

SAN FRANCISCO, January 9, 2017 — Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the fields of cancer metabolism and rare genetic metabolic diseases, today outlined key 2017 milestones in conjunction with its presentation at the 35th Annual J.P. Morgan Healthcare Conference in San Francisco. The presentation will outline important milestones as Agios evolves into a commercial stage company, including potential launches for enasidenib and AG-120 in R/R AML, pivotal development for its second wholly owned asset, AG-348 in pyruvate kinase (PK) deficiency, and an investigational new drug (IND) application submission for the company’s next development candidate, focused on MTAP deleted cancers. The company will webcast its presentation on Monday, January 9, 2017 at 10:00 a.m. PT (1:00 p.m. ET) at www.agios.com.

“This is the year Agios will evolve into a commercial-stage organization with the anticipated launch of enasidenib for patients with R/R AML, followed by the NDA submission of AG-120 and AG-348 preparing to enter a pivotal trial in PK deficiency,” said David Schenkein, M.D., chief executive officer of Agios. “We believe these milestones will enable us to achieve our vision of delivering important medicines with the potential to transform patients’ lives. Additionally, our robust research engine continues to be highly productive with an IND submission for the company’s sixth development candidate in eight years anticipated by the end of 2017.”

The company expects to achieve the following key milestones by the end of 2017:

 

    Potential approval of enasidenib in the United States for IDH2m positive R/R AML in collaboration with Celgene.

 

    Submit a new drug application (NDA) to the U.S. FDA for AG-120 by the end of 2017. AG-120 is a wholly owned, first-in-class, oral, selective, potent inhibitor of IDH1m, in IDH1m positive R/R AML.


LOGO

 

    Initiate a global, registration-enabling Phase 3 study combining AG-120 and VIDAZA® in frontline AML patients with an IDH1 mutation ineligible for intensive chemotherapy in the first half of 2017.

 

    Finalize design and operational activities for a global pivotal trial of AG-348 to initiate in the first half of 2018. AG-348 is a wholly owned, first-in-class, oral activator of both wild-type (normal) and mutated pyruvate kinase-R (PKR) enzymes, in PK deficiency.

 

    File an IND application for the MTAP pathway development candidate by the end of 2017.

The company also provided an update on the following 2016 milestones achieved in December:

 

    Supported Celgene’s submission of an NDA for enasidenib in IDH2m positive R/R AML.

 

    Initiated a global, registration-enabling randomized Phase 3 trial for AG-120 in IDH1m positive cholangiocarcinoma. The FDA also granted AG-120 Fast Track Designation for the treatment of patients with previously treated, unresectable or metastatic cholangiocarcinoma with an IDH1 mutation.

 

    Selected a development candidate focused on the MTAP pathway to enter IND-enabling studies.

2016 Year-End Cash and Guidance

Agios ended 2016 with approximately $574 million of cash, cash equivalents and marketable securities. Based on its current operating plans, the company expects that its existing cash, cash equivalents and marketable securities as of December 31, 2016, together with anticipated interest income, and anticipated expense reimbursements under its collaboration agreements with Celgene, but excluding any additional program-specific milestone payments from Celgene, will enable the company to fund its anticipated operating expenses and capital expenditure requirements through at least the end of 2018.

Presentation at 35th Annual J.P. Morgan Healthcare Conference

Agios will webcast its corporate presentation from the 35th Annual J.P. Morgan Healthcare Conference in San Francisco on Monday, January 9, 2017 at 10:00 a.m. PT (1:00 p.m. ET). A live webcast of the presentation can be accessed under “Events & Presentations” in the Investors and Media 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 metabolic 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 multiple first-in-class investigational medicines in clinical and/or preclinical

 


LOGO

 

development. All Agios programs focus on genetically identified patient populations, leveraging the company’s knowledge of metabolism, biology and genomics. For more information, please visit the company’s website at www.agios.com.

About Agios/Celgene Collaboration

Enasidenib and AG-881 are part of Agios’ global strategic collaboration with Celgene Corporation focused on cancer metabolism. Under the terms of the 2010 collaboration, Celgene has worldwide development and commercialization rights for enasidenib. Agios continues to conduct clinical development activities within the enasidenib development program and is eligible to receive up to $120 million in payments assuming achievement of certain milestones and royalties on net sales. Additionally, Agios and Celgene intend to co-commercialize enasidenib in the U.S. 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. The program focused on MTAP (methylthioadenosine phosphorylase) deleted cancers is part of a 2016 global co-development and co-commercialization agreement with Celgene focused on metabolic immuno-oncology with a worldwide 50/50 cost and profit share between Agios and Celgene, under which Agios is eligible for up to $169 million in clinical and regulatory milestone payments for the program.

Vidaza® is a registered trademark of Celgene Corporation.

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 Agios’ plans, strategies and expectations for its and its collaborator’s preclinical, clinical and commercial advancement of its drug development programs including enasidenib, AG-120, and AG-348; the potential benefits of Agios’ product candidates; its key milestones for 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

 


LOGO

 

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’ Quarterly Report on Form 10-Q for the quarter ended September 30, 2016, and other filings that Agios may make with the Securities and Exchange Commission in the future. 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:

Kendra Adams, 617-844-6407

Senior Director, Investor & Public Relations

Kendra.Adams@agios.com

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