Agios Pharmaceuticals : 41st Annual J.P. Morgan Healthcare Presentation

Fueled by Connections to

Transform Rare Diseases

Brian Goff, Chief Executive Officer

January 11, 2023

1

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 potential benefits of PYRUKYND® (mitapivat), AG-946	and its PAH stabilizer; Agios'	plans,	strategies
and expectations for its preclinical, clinical and commercial advancement of its drug development, including PYRUKYND®, AG-946 and its PAH stabilizer;	Agios'	strategic
vision and goals, including its key milestones for 2023 and potential catalysts through 2026; and the potential benefits of Agios'	strategic plans and focus. The words
"anticipate," "expect," "goal," "hope," "milestone," "plan," "potential," "possible," "strategy," "will," "vision," 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 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, without limitation: risks and uncertainties related	to the impact of the COVID-19 pandemic	to Agios'	business, operations,
strategy, goals and anticipated milestones, including	its ongoing	and planned research activities,	ability to conduct ongoing	and planned	clinical trials,	clinical supply	of current
or future drug candidates,	commercial supply of current or future	approved products,	and launching, marketing and selling	current	or future	approved	products; 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, the EMA or 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;	the failure
of Agios to receive milestone	or royalty payments related to the sale of its oncology business,	the uncertainty of the timing of any receipt	of any such payments, and the
uncertainty of the results and effectiveness of the use of proceeds from the transaction with Servier; and general economic and market	conditions. These and other risks are
described	in greater detail under the caption "Risk Factors" included in Agios' public filings with the Securities and Exchange Commission. 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.

2

Unmatched expertise in cellular metabolism

The Leader in

Pyruvate Kinase (PK)

Activation

Track record of

success in discovering,

developing and commercializing therapies

DEVELOPED APPROVED THERAPIES	FIRST PYRUKYND® APPROVAL	RARE DISEASE FOCUS: POTENTIAL
ADULTS WITH PYRUVATE KINASE (PK)	APPROVALS IN THALASSEMIA AND
IN ONCOLOGY
DEFICIENCY	SICKLE CELL DISEASE
2017 & 2018	2022	By 2026

3

Leader in pyruvate kinase (PK) activation poised for significant growth

Compelling and consistent data across connected diseases

Robust clinical data set supports potential of PK activation to transform patient function, quality of life, and long-term outcomes

Meaningful commercial opportunities on the horizon

First rare disease launch building capabilities to maximize anticipated franchise expansion

Potential for two additional PYRUKYND® indications by 2026

Well capitalized to advance and expand

Strong cash position expected to support completion of ongoing programs and disciplined portfolio expansion

4

Focused on expanding from PK deficiency to other diseases with shared pathophysiology, limited treatment options, and profound unmet needs

Pediatric PK

Deficiency

No approved therapy for pediatric PK deficiency patients

Our goal: Deliver the first approved therapy for pediatric PK deficiency

Thalassemia	Sickle Cell Disease	Lower-Risk MDS
No approved therapy for	No novel oral therapy	No oral therapy
~60% of thalassemia	improves anemia and	addresses ineffective
patients	reduces sickle cell	erythropoiesis
Our goal: Deliver the first	pain crises	Our goal: Deliver the first
therapy approved for all	Our goal: Deliver a novel	oral therapy that addresses
thalassemia subtypes	oral therapy that improves	ineffective erythropoiesis
	anemia and reduces VOCs

5 PYRUKYND® is approved in the U.S., EU, and Great Britain for adult PK deficiency and is under investigation for pediatric PK deficiency, thalassemia, and sickle cell disease. AG-946 is under investigation for LR-MDS.