A timeline of innovation and patient impact

For nearly 15 years, Agios has been committed to furthering the science of cellular metabolism to identify therapies for patients with unmet needs.

Our deep and differentiated understanding of the science is foundational to our success and the connective thread that binds our people and our pipeline together.


We began with a focus on cellular metabolism and a “blank sheet of paper.” Our first foray into improving patients’ lives was in the oncology space; we discovered the science of IDH inhibition, which led to two approved precision medicines for cancer patients.

We also pioneered a second therapeutic approach — PK activation — which is the foundation of our focus on rare diseases.


Founded in Cambridge, Massachusetts, to unlock a new field of discovery in cellular metabolism



Entered into collaboration with Celgene Corporation to discover and develop novel cancer metabolism-related therapies

Together with collaborators at Memorial Sloan Kettering, published landmark paper in Cancer Cell, potentially implicating IDH mutation in acute myeloid leukemia (AML)


Closed $78 million Series C financing

Expanded research focus into rare genetic metabolic disorders


Discovered first potent inhibitors of mutant IDH that lower tumor 2-HG in vivo; published findings in American Chemical Society Medicinal Chemistry Letters

Published article in Nature reinforcing role of IDH mutations in AML


Initiated first clinical study of IDHIFA® (enasidenib)

Completed initial public offering on NASDAQ

Named Technology Pioneer by the World Economic Forum

Presented first preclinical data from rare genetic diseases research program

Published back-to-back articles in Science interrogating the roles of mutated IDH2 in leukemia and IDH1 in glioma


Presented first clinical data for IDHIFA® (enasidenib)

Initiated the first clinical trials of TIBSOVO® (ivosidenib tablets)

Initiated first clinical study of study of mitapivat and presented first clinical data


Received Orphan Drug designation from FDA for mitapivat for the treatment of PK deficiency

Presented first clinical data for TIBSOVO® (ivosidenib tablets)

Initiated Phase 2 study of mitapivat in adult, non-transfusion dependent patients with PK deficiency (DRIVE PK)


Presented first data from Phase 2 DRIVE PK study of mitapivat in patients with PK deficiency

Filed New Drug Application with FDA for IDHIFA® (enasidenib)


Received first FDA approval for IDHIFA® (enasidenib)

Filed New Drug Application with FDA for TIBSOVO® (ivosidenib tablets)


Received FDA approval for TIBSOVO® (ivosidenib tablets)

Initiated two global pivotal trials for mitapivat in PK deficiency: ACTIVATE and ACTIVATE-T

Brian Goff,

CEO of Agios

While Agios has evolved significantly since our founding, our commitment to making a positive impact for patients, employees, communities and the world remains unchanged. As we create the future of Agios together, we are building on our core values and pioneering leadership in cellular metabolism to advance our work on behalf of people with rare diseases.

A pipeline of possibility

Our unique understanding of cellular metabolism is foundational to our success and the connective thread that binds our people and our pipeline together.