What s Possible at Agios

What s Possible at Agios

David Schenkein, M.D.

Chief Executive Officer

Cautionary Note Regarding Forward-Looking Statements

This 2015 Research & Development Day 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,

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; 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.

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 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

June 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.

We Are Driven By a Clear Vision and Values

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 metabolism

Making a Difference for Patients & Building Long-Term Value

Team/Culture Execution

Building a Great Sustainable Biopharmaceutical Company

Commercial Capabilities Begin to Build

Platform Continues to Expand

AG-221/AG- AG-348 5th Agios

Agios Labs IDH Celgene 120 Proof of Proof of medicine

Open Discovery Partnership Concept Mechanism entering clinic;

2009 2009 2010 2014 2014 2015

Five clinical stage investigational

medicines with possibility to help a large number of genetically identified patients

medicines accelerating towards approval and commercialization

A robust and novel pre-clinical pipeline in both

A organization preparing for commercialization

A passion to help patients and follow great science

Advancing the IDHm inhibitors to

market as quickly and broadly as possible

Design and initiation of AG-221 AML Phase 3 trial (IDHENTIFY)

Design of combination trial with 7 + 3

Design of combination trial with azacitidine

Driving the PK activator program

molecule entering clinical development (AG-519)

Preclinical data to support potential new indications for PK activators Metabolic vulnerabilities as emerging cancer focus

What s Possible: Our Science

Find the Source of the Problem and Correct It

Discovering novel unprecedented targets

Tackling tough chemistry with allosteric inhibitors and activators Requiring a predictive marker prior to development candidate selection Exploiting intersection of several key fields:

metabolism, genomics, epigenetics and immunology Setting a high bar and rewarding answers

Find the Source of the Problem and Correct It

intensity 149.50 Peak (arbitrary

Find the Source of the Problem and Correct It

Patient A (R510Q/G511R)

Screening Randomization Stratified

L) Dosing period Placebo

ATP m 200 15 mg q12hr

lu t con 360 mg q12hr

Phase 1 NHV, ASH 2014

What s Possible: For Patients

What s Possible for IDHm Patients

Relapsed/ Refractory AML

What s Possible for IDHm Patients

Frontline AML Combination trials Maintenance MDS

Other hematologic malignancies

What s Possible for IDHm Patients

All IDHm patients screened and treated with an IDHm inhibitor for the entire course of their disease

Exploration of PK Activation in Other Hemolytic Anemias

Disease Molecular Lesion Red Cell Characteristics

Normal physiology None Biconcave

Pyruvate kinase deficiency PKR mutations Echinocyte

Sickle cell disease HbS mutation Sickled

Beta-thalassemia Microcytic

Mutations in spectrin,

Hereditary spherocytosis

ankyrin, protein 4.2

Novel First-in-Class Clinical Portfolio

Candidate Indication Primary Commercial Rights

Clinical Development Clinical Development

R/R AML Phase 1 Dose Escalation

Expansion 5th Cohort

AG-221 Frontline AML Phase 1b Combinations (Q4 15)

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

MDS/Heme Malig Phase 1 Expansion

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

R/R AML Phase 3 (1H 16)

Escalation Expansion Cohort

AG-120 MDS/Heme Malig Phase 1 Expansion

Frontline AML Phase 1b Combinations (Q4 15) U.S. Rights EX-U.S. Rights

Frontline AML Phase 1/2 Combinations (Q1 16)

Solid Tumors Phase 1 Dose Escalation

AG-881 R/R AML Phase 1 Dose Escalation

inhibitor) Solid Tumors Phase 1 Dose Escalation Joint Worldwide Collaboration

AG-348 PK Deficiency Phase 2

AG-519 PK Deficiency Phase 1 (Q1 2016)

Novel First-in-Class Research Portfolio

Validation Compound Optimization

Two Cancer Metabolism

Multiple Other Oncology Targets