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 genetically defined diseases.
Founded in Cambridge, Massachusetts, to unlock a new field of discovery in cellular metabolism
Established, for the first time, link between mutated metabolic gene IDH1 and cancer gene; published findings in Nature
David Schenkein, M.D., named as chief executive officer
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
Jackie Fouse, Ph.D. named as chief executive officer
Published results from Phase 2 DRIVE PK study of mitapivat in patients with PK deficiency in New England Journal of Medicine
Established clinical proof-of-concept for mitapivat in non-transfusion-dependent thalassemia
Established clinical proof-of-concept for mitapivat in sickle cell disease
Initiated first clinical study of novel PK activator AG-946
Received Orphan Drug designation from FDA for mitapivat for treatment of thalassemia and sickle cell disease; EMA granted Orphan Drug designation for PK deficiency
Royalty Pharma acquired rights to IDHIFA® worldwide net sales and outstanding regulatory milestone payments for $255 million
Announced positive data from ACTIVATE Phase 3 trial of mitapivat in adults with PK deficiency who are not regularly transfused
Announced agreement to sell oncology business to Servier for up to $2 billion plus royalties, enabling new strategic, singular focus on genetically defined diseases
Received approval of PYRUKYND® (mitapivat), our first rare disease medicine, in the U.S., EU and Great Britain
Brian Goff named as chief executive officer
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.