Agios in 2016 JPMorgan Healthcare Conference

JPMorgan Healthcare Conference

2016 David Schenkein, M.D. Chief Executive Officer

Note Regarding Forward-Looking Statements

This presentation and various remarks we make during this

presentation contain forward-looking statements of Agios Pharmaceuticals, Inc. within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the potential benefits of Agios

product candidates targeting IDH1/IDH2 or pyruvate kinase-R mutations or other genetic mutations, including AG-221, AG-120, AG-881, AG-348 and AG-519; its plans and timelines for the clinical development of AG-221, AG-120, AG-881, AG-348 and AG-519;

its plans regarding future data presentations; its plans regarding its preclinical development activities; and the benefit of its strategic plans and focus. The words anticipate, believe, estimate,

expect, intend, may, plan, predict, project, potential, hope, could, would and similar expressions are intended to identify

forward-looking statements, although not all forward-looking statements contain these identifying words.

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 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 or the various remarks made 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 agreement 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, 2015, and other filings that Agios may make with the Securities and Exchange Commission in the future.

Any forward-looking statements contained in this presentation or in remarks made 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, except as required by law.

Driven By a Clear Vision and Values

RARE GENETIC DISORDERS OF METABOLISM CANCER METABOLISM DYSREGULATED

Agios is passionately committed to the fundamental transformation of patients lives through scientific leadership in the field of cancer metabolism and rare genetic disorders of

Great Sustainable Biopharmaceutical Company

Building Late-stage Development & Commercial Capabilities

Continuing to Expand Research Platform

Agios IDH Celgene AG-221 AG-348 medicine 2016 Key

Labs Discovery Partnership /AG-120 Proof of entering Priorities

Open Proof of Mechanism clinic; 200th

2009 2009 2010 2014 2014 2015

Key Priorities: Maturing into a Late-stage Company

Rapid and broad late stage clinical development for IDHm

Demonstrate clinical activity of PKR activators in patients

Advance research and initiate preclinical development of next wave research program

First-in-Class Clinical Portfolio

Early Stage Late Stage

Candidate Indication Primary Commercial Rights

Clinical Development Clinical Development

R/R AML Phase 1 Dose Escalation Expansion 5th Cohort

AG-221 Frontline AML (Fit) Phase 1b Combinations

(IDH2m inhibitor) Frontline AML (Unfit) Phase 1/2 Combinations (Q1 16)

MDS/Heme Malig Phase 1 Expansion 2nd Expansion (2016)

Solid Tumors Phase 1 Dose Escalation Agios U.S. Co-promotion and Royalty

Frontline AML Phase 3 (2H 16)

Escalation Expansion

AG-120 MDS/Heme Malig Phase 1 Expansion

(IDH1m inhibitor) Frontline AML (Fit) Phase 1b Combinations

Frontline AML (Unfit) Phase 1/2 Combinations (Q1 16) U.S. Rights EX-U.S. Rights

Solid Tumors Phase 1 Dose Escalation Expansion

IHCC Phase 2 (2H 16)

AG-881 R/R AML Phase 1

(pan-IDHm inhibitor) Solid Tumors Phase 1 Dose Escalation Joint Worldwide Collaboration

AG-348 PK Deficiency Phase 2 DRIVE PK

AG-519 PK Deficiency

Today s Key 2016 Milestone Announcements

Complete enrollment in AG-221 and

AG-120 expansion arms in 2H Initiate Phase 3 study of AG-120 in frontline AML in 2H

Initiate MDS expansion arm

Initiate randomized Phase 2 study of AG-120 in cholangiocarcinoma in 2H

Present first data from DRIVE PK trial of AG-348 in 1H

Present first data from AG-519 Phase 1 healthy volunteer study in 1H Present new findings from Natural History Study of PK deficiency in 2H

Outline clinical development plans for PKR activators in beta-thalassemia in 2H

Initiate preclinical development activities and publish on a new cancer metabolism program

Using a pill once a day to repair a cancer cell

an IDH Mutant Cancer Cell

DIFFERENTIATION RESTORED BLO

Isocitrate mIDH 2HG CANCER

HSC Progenitor Progenitor

Leukemia Blast Neutrophil

What s Possible for IDHm Patients

A Roadmap for Speed and Breadth

All IDHm patients screened and treated with

an IDHm inhibitor for the entire course of their disease

Solid tumors Frontline AML Combination trials

Other hematologic malignancies

Relapsed/ Refractory AML

Inhibitors Provide Maximum Opportunity

Potent, selective, reversible inhibitor of mutant isocitrate dehydrogenase-2 (IDH2) In Phase 3 clinical development Oral,

once daily dosing, 100mg

Potent, selective, reversible inhibitor of mutant isocitrate dehydrogenase-1 (IDH1) In Phase 2 clinical development Oral, once daily dosing, 500mg

Brain-penetrant, pan-IDH mutant inhibitor (IDH1 & IDH2) In Phase 1 clinical development Oral, once daily dosing

Mutations Occur in Multiple Cancers

~40K New Patients/Year; Mutation Occurs

Early and Easily Detected with Genomic Testing

AG-120 Represent a New Treatment Paradigm in AML

Median response duration: 5.6 months Median response duration: 6.9 months

CR PR CRI Ongoing Progression/death crp mcr first response bone marrow transplant treatment suration (months) death or

Progression transplant

First-in-Class, Oral, Potent, Selective, Reversible Inhibitors

With ~300 patients treated, AG-221 and AG-120 demonstrate favorable safety profiles Impressive single agent complete and

partial responses in relapsed/refractory IDHm AML

Neutrophil and platelet improvements observed in non-CR

responders and some patients with stable disease

Data Presented at ASH, 12/5-6/15

Programs Rapidly Defined Single-Agent Profile for IDHm Inhibitors in R/R AML

Phase 1 expansion cohorts

designed to demonstrate compelling clinical benefit with registration quality data

AG-221 Expansion Phase 2

Expansion Phase 1 Ongoing Dose Escalation

Four 25-patient Arms Completed R/R AML Completed (n=125 patients)

AG-120 Expansion Phase 1