AGIO stock is moving in after-hours trading on December 16, 2019.

AGIO stock surging in after-hours trading (brown shaded area) on December 16, 2019.

Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for TIBSOVO® (ivosidenib) for the treatment of adult patients with relapsed or refractory myelodysplastic syndrome (MDS) with a susceptible IDH1 mutation as detected by an FDA-approved test. MDS is a group of bone marrow disorders that can cause severe complications, such as infections and uncontrolled bleeding, and can lead to the development of acute myelogenous leukemia (AML).

“There is a significant need for new targeted therapeutic approaches for individuals with MDS whose disease continues to progress despite treatment with standard of care,” said Chris Bowden, M.D., chief medical officer at Agios. “The Breakthrough Therapy designation is based on results from the initial 12 patients in the MDS arm of our Phase 1 study in advanced hematologic malignancies with an IDH1 mutation and recognizes the potential for single-agent treatment with TIBSOVO® to make an impact on these patients. We recently re-opened the MDS arm of this study with the goal of generating sufficient data to pursue a regulatory filing in this indication.”

The FDA’s Breakthrough Therapy designation is intended to expedite the development and review of a drug candidate that is under investigation to treat a serious or life-threatening disease or condition when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies on one or more clinically significant endpoints.

Results from the MDS arm of the ongoing TIBSOVO® Phase 1 dose-escalation and expansion study in hematologic malignancies were presented at the 7th Society of Hematologic Oncology Annual Meeting, held September 11-14, 2019, in Houston, Texas. These demonstrate that TIBSOVO® administered as a monotherapy was well tolerated and associated with durable remissions as well as the achievement and maintenance of transfusion independence in patients with relapsed or refractory MDS with an IDH1 mutation. Among the 12 patients who received 500 mg of oral TIBSOVO® daily, the median treatment duration was 11.4 months. The median age was 72.5 years, and 42% of patients were age 75 or older. As of the November 2, 2018 data cut-off, 75% (9/12) of patients had a response and 42% (5/12) had a complete response (CR). The median duration of CR had not been reached (95% CI, 2.8 months, NE). Of the patients who had a CR, 60% remained relapse-free at 12 months. In addition, 9 (75%) patients were transfusion-independent for 56 days or longer during study treatment. The most common adverse events (AEs) of any grade were back pain, diarrhea, fatigue and rash. Grade 2 IDH differentiation syndrome was observed in 1 of 12 patients. No AEs resulted in permanent discontinuation of treatment.

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