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 11, 2016

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 7.01 Regulation FD Disclosure.
On January 11, 2016, Agios Pharmaceuticals, Inc. (the “Company”) intends to make a slide presentation at the 34th 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 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, or the Exchange Act, except as expressly set forth by specific reference in such a filing.
Item 8.01 Other Events.
On January 11, 2016, the Company issued a press release outlining its 2016 strategy and expected milestones for its development programs and research pipeline, each of which will be discussed at the Company’s presentation at the 34th Annual J.P. Morgan Healthcare Conference on January 11, 2016. 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.
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 11, 2016.	
 	 	
99.2	  	Press release issued by Agios Pharmaceuticals, Inc. on January 11, 2016.	

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 11, 2016	 	 	 	By:	 	/s/ David P. Schenkein	
 	 	 	 	 	 	David P. Schenkein, M.D.	
 	 	 	 	 	 	Chief Executive Officer	

EXHIBIT INDEX

Exhibit
No.	  	Description	
 	 	
99.1	  	Form of Presentation as of January 11, 2016.	
 	 	
99.2	  	Press release issued by Agios Pharmaceuticals, Inc. on January 11, 2016.	

 
Exhibit 99.1
Agios in 2016
JPMorgan Healthcare Conference
January 11, 2016 David Schenkein, M.D. Chief Executive Officer

 	

 
Cautionary 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.
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 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.

 	

 
We Are Driven By a Clear Vision and Values
RARE GENETIC DISORDERS OF METABOLISM CANCER METABOLISM DYSREGULATED METABOLISM
VISION
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
3

 	

 
Building a Great Sustainable Biopharmaceutical Company
Building Late-stage Development & Commercial Capabilities
Continuing to Expand Research Platform
5th Agios
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
Concept employee
2009 2009 2010 2014 2014 2015 2016
4

 	

 
Our 2016 Key Priorities: Maturing into a Late-stage Company
Rapid and broad late stage clinical development for IDHm inhibitors
Demonstrate clinical activity of PKR activators in patients
Advance research and initiate preclinical development of next wave research program
5

 	

 
Novel First-in-Class Clinical Portfolio
Early Stage Late Stage
Candidate Indication Primary Commercial Rights
Clinical Development Clinical Development
R/R AML Phase 3
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)
R/R AML Dose 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 Dose Escalation
(pan-IDHm inhibitor) Solid Tumors Phase 1 Dose Escalation Joint Worldwide Collaboration
AG-348 PK Deficiency Phase 2 DRIVE PK
(PK (R) Activator)
AG-519 PK Deficiency Phase 1
(PK (R) Activator)
Celgene agios

 	

 
Today’s Key 2016 Milestone Announcements
IDHm Inhibitors
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 for AG-221
Initiate randomized Phase 2 study of AG-120 in cholangiocarcinoma in 2H
PKR Activators
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
Research
Initiate preclinical development activities and publish on a new cancer metabolism program

 	

 
Cancer Metabolism: IDH
Using a pill once a day to repair a cancer cell
agios

 	

 
Repairing an IDH Mutant Cancer Cell
DIFFERENTIATION RESTORED BLO
Inhibitor
IDH Epigenetic
“Re-Wiring” Unchecked Cell Proliferation
Isocitrate mIDH 2HG CANCER
Differentiation aKG
HSC Progenitor Progenitor
IDHm Inhibitor
Leukemia Blast Neutrophil
Day 0 Day 15 Day 28
AG-120, EORTC, 2014
9

 	

 
What’s Possible for IDHm Patients
A Roadmap for Speed and Breadth
Goal
Next
Now
All IDHm patients screened and treated with an IDHm inhibitor for the entire course of their disease
Solid tumors Frontline AML Combination trials Maintenance MDS
Other hematologic malignancies
Relapsed/ Refractory AML
10

 	

 
Three IDHm Inhibitors Provide Maximum Opportunity
AG-221
Potent, selective, reversible inhibitor of mutant isocitrate dehydrogenase-2 (IDH2) In Phase 3 clinical development Oral, once daily dosing, 100mg
AG -120
Potent, selective, reversible inhibitor of mutant isocitrate dehydrogenase-1 (IDH1) In Phase 2 clinical development Oral, once daily dosing, 500mg
AG-881
Brain-penetrant, pan-IDH mutant inhibitor (IDH1 & IDH2) In Phase 1 clinical development Oral, once daily dosing
11

 	

 
IDH Mutations Occur in Multiple Cancers
AML
MDS
IHCC
Chondro
Other solid tumors
Glioma
~40K New Patients/Year; Mutation Occurs Early and Easily Detected with Genomic Testing
12

 	

 
AG-221 and AG-120 Represent a New Treatment Paradigm in AML
AG-221 AG-120
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
13

 	

 
Phase 1 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
Dose Escalation Three 25-patient Arms Ongoing Completed 125-patient Arm Ongoing
Expect to Complete Enrollment in Both 125-patient Expansion Arms in 2H’16
14

 	

 
Targeting Multiple Lines of Treatment in IDHm AML and Other Hematologic Malignancies
Relapsed AML Newly Diagnosed (Untreated) AML MDS / Other Heme Malig.
2nd+ Relapse Intensive Non-Intensive Frontline to R/R
Phase 1 AG-221 Expansion
Phase 1 AG-120 Expansion
Phase 3 IDHENTIFY
AG-221 vs SOC
Phase 1 Induction (7+3) + AG-221 or AG-120
Phase 1? 2 VIDAZA® + AG-221 or AG-120
(1Q’16)
Phase 1 AG-221 MDS Expansion Cohort (2016)
Phase 3
AG-120 in Frontline AML
(2H’16)
Ongoing
Planned
15

 	

 
IDH Mutations Occur in Multiple Cancers Glioma IHCC Chondro Aml mds other solid tumors ~40K New Patients/Year; Mutation Occurs Early and Easily Detected with Genomic Testing 16

 	

 
Encouraging AG-120 Phase 1 Data in Solid Tumors
Solid Tumors Glioma
Other chondrosarcoma cholangiocarcinoma treatment duration (weeks) pr sd pd unk/na ongoing progression/death
AG-120 well tolerated (no MTD) and showed signs of clinical activity Reductions in tumor volume observed in some glioma patients
Favorable PK properties, inhibition of 2HG in tumor and reduction in proliferation markers
Data Presented at AACR-NCI-EORTC, 11/8/15
17

 	

 
Clinical Development Path in IDH1m Solid Tumors Will
Be Data Driven
AG-120
IDH1m inhibition; low CNS penetration
Dose-Escalation Expansion
(Completed) (Ongoing)
Randomized IHCC Phase 2
(2H’16)
IHCC
Chondro sarcoma
Glioma
Other IDHm Solid Tumors
AG-881
Pan-IDHm inhibition; high CNS penetration
AG-881 Phase 1 Dose-Escalation (Ongoing)
Potential Expansion
18

 	

 
Rare Genetic Metabolic Disorders: PKR
Transforming a metabolic disorder with a small molecule
agios

 	

 
Pyruvate Kinase-R (PKR) Activation Has Broad Potential in Red Cell Disorders Normal Red Cell PEP wtPKR Pyruvate Cellular demand: ATP production meets demand Pyruvate Kinase Deficiency PEP mPKR Pyruvate Cellular demand: Inadequate production: ATP deficiency Other Hemolytic Anemias PEP wtPKR Pyruvate Cellular demand: Increased demand: ATP deficiency 20

 	

 
PK Deficiency: What We Know Today
Disease Overview Rare genetic disease often
presenting at birth as neonatal
Description jaundice
~2400 diagnosed in U.S. and EU5*
Caused by mutations in PK-LR
Etiology gene coding for Erythrocyte
Pyruvate Kinase
Clinical Lifelong hemolytic anemia and
Presentation associated morbidities
PKR enzyme activity and
Diagnosis genetic testing
Disease Pathophysiology
Red Blood Cell Blood Smear Normal PK Deficiency
* Based on genetic data and diagnosis rate
21
Source: Zanella. Blood Rev. 2007; 21(4):217; Blood and Bone Marrow Pathology; Wintrobe’s Clinical Hematology; Physician Interviews; Market Research.

 	

 
PKR Activation Represents Therapeutic Approach to
Treating PK Deficiency
2,3-DPG
pyruvate Glucose atp
AG-348 AG-519
Patient A (R510Q/G511R)
Pkr activity (relative to dmso) 2-3 dpg (relative to dmso) atp (relative to dmso) 24 hrs
Wt vs pkd atp levels rbcs treated with ag-348 24hrs wt control pkd patient log [ag-348 (m)]
AG-348 300 mg q12hr
R
-
PK by Optional
Screening RandomizationStratified genotype 3rd Arm
AG-348 50 mg q12hr
AG-348
L) Dosing period
m Placebo g/ 200 15 mg q12hr
ATP m ( n
i tion 60 mg q12hr a 120 mg q24hr hange 100 120 mg q12hr
c entr e c
lu t con 360 mg q12hr s o 700 mg q12hr
b od 0
A lo Mean + SD b
0 5 10 15 20
Time (day)
Phase 1 NHV, ASH 2014
22

 	

 
AG-348 Global Phase 2 DRIVE PK Study
Open and Enrolling
Transfusion-independent PK-deficient adults n=25 in each arm
Screening
Randomization
Stratified by PK-R genotype
AG-348
300 mg q12hr
Optional 3rd
Arm
AG-348
50 mg q12hr
6-month
dosing
PK
period
PD
Primary endpoints
Safety and tolerability
Secondary endpoints
PK of AG-348
PD response: ATP, 2,3-DPG
Indicators of clinical activity: hemoglobin, hematocrit, reticulocyte count, and other hematologic parameters.
First Data Expected 1H’16
23

 	

 
AG-519 Healthy Volunteer Study Open and Enrolling
AG-519
Potent, highly selective and orally bioavailable PKR activator Differentiated chemical structure versus AG-348 No activity against the aromatase enzyme AG-519 has similar activity in vitro, in vivo and ex vivo (patient samples) relative to AG-348
One protocol, two steps, healthy volunteers
Step 1: Integrated SAD/MAD
4 dose-ascending cohorts: 8 subjects per cohort (n=32) Placebo controlled (6A, 2P)
Step 2: Bioavailability and Food Effect Study
First Data Expected 1H’16
24

 	

 
AG-519 Provides Optionality for Clinical Development
AG-348 Healthy Volunteer Studies (Completed)
AG-348 DRIVE PK PK Deficiency (Ongoing)
AG-519 Healthy Volunteer Studies
(Q1’16)
Efficacy Safety PK
PD
Pivotal Trials:
Adults
Adolescents
Children Beta-thalassemia Studies (Planned)
Clinical Data From Both Trials Available in 1H’16 Will Determine Late-stage Development Path
25

 	

 
Research
Initiating the development of a new research program

 	

 
Novel First-in-Class Research Portfolio
Target Validation Compound Optimization
Target C
Two Cancer Metabolism
Multiple Other Oncology Targets
Wave
Rare Genetic
Multiple RGD Targets
Metabolic Disorders
Cancer Metabolism Multiple
Oncology Three Targets
Wave Rare Genetic Multiple RGD
Metabolic Disorders Targets
27

 	

 
Prevalent Deletions of MTAP Create Sensitivity to a Novel Pathway With Multiple Therapeutic Targets
Deletion of metabolic gene adjacent to tumor suppressor p16/p14
chr9p21 MTAP p16/p14
genetic selection markers ~75,000 new patients/year in U.S. with MTAP deletion across many indications
% mtap homozygous deletion (primary tumors)
Bladder melanoma brain lung pancreas breast esophageal carcinoma head & neck squamous kidney renal papillary colon adenocarcinoma dlbcl all mcl cml- balst crisis
MTAP-deleted Tumors Constitute a Large, Genetically Defined Patient Population
28

 	

 
Prevalent Deletions of MTAP Create Sensitivity to a Novel Pathway With Multiple Therapeutic Targets
Upstream metabolite MTA accumulates when MTAP is deleted
MTA
(MTAP substrate)
Vulnerable pathway
with multiple
Therapeutic targets
Target 1
Target 2
Target 3
Druggable by Agios inhibitors with precision medicine approach
Target MTAP- Inhibitor
deleted Tumor Xenograft Model
MTAP-WT Target
Tumor Inhibitor
Xenograft Model
Agios Has Developed Potent and Orally-bioavailable Inhibitors of an MTAP-selective Target
Fold change in mtap-deleted cells metabolites mtap selectivity score mtap-deleted tumor xenograft model mtap-wt tumor xenograft model vechicle target inhibitor days post implant s.e.m
29

 	

 
Key Takeaways
Focus on Strategic Priorities and Execution:
Continue rapid, broad late-stage clinical development for our
IDHm inhibitors
Demonstrate clinical activity of our wholly owned, PKR activators in patients
Advance research and initiate preclinical development of a new research program
Building a great sustainable biopharmaceutical company
Passionate in our vision to change patients’ lives
30

 	

Exhibit 99.2

Agios Outlines Key 2016 Goals and Priorities
- Complete Enrollment of 125-Patient Expansion Cohorts for AG-221 and AG-120 in Relapsed/Refractory Acute Myeloid Leukemia in Second Half of 2016 -
- Present First Data from Phase 2 DRIVE PK Study for AG-348 in PK Deficiency and Phase 1 Healthy Volunteer Study for AG-519 in First Half of 2016 -
- Initiate Preclinical Development of a Program from the Next Wave of Research -
- Company to Present at the 34th Annual J.P. Morgan Healthcare Conference Today at
3:30 p.m. PST -
SAN FRANCISCO, January 11, 2016 — Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the fields of cancer metabolism and rare genetic metabolic disorders, today outlined the company’s 2016 strategy and expected clinical development and research milestones in conjunction with the 34 th  Annual J.P. Morgan Healthcare Conference in San Francisco. The presentation will outline three strategic priorities for 2016: continue rapid and broad late-stage clinical development for its lead isocitrate dehydrogenase (IDH) mutant inhibitors in hematologic malignancies and solid tumors; demonstrate clinical activity of its wholly owned, global pyruvate kinase-R (PKR) activators in patients; and advance research and initiate preclinical development of a program from the next wave of research. The company will webcast its presentation on Monday, January 11, 2016 at 3:30 p.m. PST (6:30 p.m. EST) at www.agios.com.
“We expect each of our programs to achieve important catalysts in 2016 that will bring us closer to our vision of helping people with cancer and rare genetic disorders,” said David Schenkein, M.D., chief executive officer at Agios. “We believe these milestones, coupled with our growing late-stage development and commercial capabilities, set Agios firmly on the path to become a sustainable, multi-product biopharmaceutical company with a strong research core and broad pipeline of first-in-class medicines.”
IDH Mutant Inhibitors
Dr. Schenkein continued, “We remain focused on executing on our ‘speed and breadth’ clinical development strategy for AG-221 and AG-120 in hematological malignancies, with the intent to complete enrollment of both 125-patient expansion cohorts this year. Further understanding the potential of our IDH mutant inhibitors in solid tumors remains a priority with several new and ongoing trials in 2016.”
AG-221, AG-120 and AG-881 are part of Agios’ global strategic collaboration with Celgene Corporation.

 
Expected 2016 milestones for IDH mutant inhibitors in hematologic malignancies:
 
 	•	 	Complete enrollment of both 125-patient expansion cohorts for the Phase 1/2 study of AG-221 and Phase 1 study of AG-120 in patients with relapsed/refractory (R/R) acute myeloid leukemia (AML) in the second half of 2016	
 
 	•	 	Initiate a global, registration-enabling Phase 3 study of AG-120 in frontline AML patients with an IDH1 mutation in the second half of 2016	
 
 	•	 	Initiate an expansion arm in high-risk myelodysplastic syndrome patients for AG-221 in 2016	
 
 	•	 	Initiate a Phase 1/2 frontline combination study of AG-221 or AG-120 with VIDAZA® (azacitidine) in newly diagnosed AML patients not eligible for intensive chemotherapy in the first quarter of 2016	
 
 	•	 	Continue to enroll patients in the following ongoing clinical trials:	
 
 	•	 	Phase 3 IDHENTIFY study of AG-221 vs. standard of care chemotherapy in R/R AML	
 
 	•	 	Phase 1b frontline combination study of AG-221 or AG-120 with standard-of-care intensive chemotherapy in AML	
 
 	•	 	Phase 1 dose-escalation and expansion study of AG-881 in IDH mutant positive hematologic malignancies	
Expected 2016 milestones for IDH mutant inhibitors in solid tumors:
 
 	•	 	Initiate a randomized Phase 2 study of AG-120 in IDH1 mutant positive cholangiocarcinoma in the second half of 2016	
 
 	•	 	Continue to enroll patients in the following ongoing clinical trials:	
 
 	•	 	Expansion phase of the ongoing Phase 1 study of AG-120 in advanced IDH1 mutant positive solid tumors	
 
 	•	 	Phase 1 dose-escalation and expansion study of AG-881 in IDH mutant positive solid tumors	
PKR Activators
“Having initiated dosing in the Phase 1 healthy volunteer study of AG-519, we’ve completed the first of several key clinical milestones expected from our PKR activators in the first half of this year,” said Dr. Schenkein. “Notably, we expect to present the first data from this study and the Phase 2 DRIVE PK study for AG-348 in PK deficiency patients. There are currently no approved or disease-modifying treatments for PK deficiency, which drives our focus on advancing potential new treatment options for these patients.”
Milestone announced today:
 
 	•	 	Dosing was initiated in an integrated single ascending dose (SAD) and multiple ascending dose (MAD) placebo-controlled Phase 1 study of AG-519 in healthy volunteers	

 
Expected 2016 milestones for PKR activators:
 
 	•	 	Present the first data from DRIVE PK, a global Phase 2, open-label safety and efficacy trial of AG-348 in adult, transfusion-independent patients with PK deficiency in the first half of 2016	
 
 	•	 	Present data from Phase 1 study of AG-519 in healthy volunteers as well as preclinical findings about the molecule in the first half of 2016	
 
 	•	 	Outline the clinical development plans for Agios’ PKR activators in beta-thalassemia in the second half of 2016	
 
 	•	 	Present new findings from the Natural History Study of PK deficiency being conducted with Boston Children’s Hospital in the second half of 2016	
Research Programs
“We continue to focus on discovering and validating first-in-class targets that meet our high bar for development and align with our precision medicine strategy,” said Scott Biller, Ph.D., chief scientific officer at Agios. “We are excited to move the first program in our next wave of investigational medicines into preclinical development this year.”
 
 	•	 	Agios scientists have discovered a novel pathway comprised of multiple targets with a shared vulnerability in MTAP-deleted tumors and have demonstrated that this pathway can be modulated by small molecule inhibitors, resulting in robust anti-tumor activity in animal models	
 
 	•	 	MTAP (methylthioadenosine phosphorylase) is a metabolic enzyme that is deleted in approximately 15 percent of all cancers. This deletion is readily detected by a simple genomic test, thus allowing the selection of patients predicted to be sensitive to the therapy.	
Expected 2016 milestones for research:
 
 	•	 	Publish preclinical findings on a new cancer metabolism program	
 
 	•	 	Initiate preclinical development activities for the first molecule in the next wave of novel investigational medicines	
Presentation at 34th Annual J.P. Morgan Healthcare Conference
Agios will webcast its corporate presentation from the 34th Annual J.P. Morgan Healthcare Conference in San Francisco on Monday, January 11, 2016 at 3:30 p.m. PST (6:30 p.m. EST). 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 disorders 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

 
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
AG-221, AG-120 and AG-881 are part of Agios’ global strategic collaboration with Celgene Corporation. Under the terms of the collaboration, Celgene has worldwide development and commercialization rights for AG-221 (CC-90007). Agios continues to conduct clinical development activities within the AG-221 development program and is eligible to receive up to $120 million in payments on achievement of certain milestones and royalties on net sales. For AG-120, Agios retains U.S. development and commercialization rights and Celgene retains development and commercialization rights outside the U.S. Celgene is eligible to receive royalties on net sales in the U.S. Agios is eligible to receive royalties on net sales outside the U.S. and up to $120 million in payments on achievement of certain milestones. 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.
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 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; 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 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 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 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.
# # #
Agios Pharmaceuticals:
Renee Leck, 617-649-8299
Senior Manager, Investor Relations and Public Relations
Renee.Leck@agios.com