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Agios' Tibsovo Wins FDA's Breakthrough Therapy Tag for MDS

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Agios Pharmaceuticals, Inc. (AGIO - Free Report) announced that the FDA has granted Breakthrough Therapy designation to its leukemia drug Tibsovo (ivosidenib) for the treatment of relapsed/refractory myelodysplastic syndrome (MDS) in adult patients with a susceptible IDH1 mutation.

MDS represents a group of diverse blood disorders, which is developed because bone marrow cells fail to mature into healthy blood cells. This results into requirement of blood or red blood cell transfusions in patients. Per the press release, there is a significant need for bringing new targeted therapeutic approaches to people with MDS as their disease continues to progress despite standard of care treatment.

The Breakthrough Therapy status is granted to accelerate the development and review of the drugs that target serious or life-threatening conditions. This prestigious tag to Tibsovo is based on data from the initial 12 patients in the MDS arm of Agios’ phase I study for advanced hematologic malignancies with an IDH1 mutation.

Agios re-opened the MDS arm of the study in October 2019 and is focused on generating data to submit a potential regulatory filing for this indication. It will enroll up to 25 total patients in this cohort.

Shares of Agios were up 4.5% in after-hours trading following this development on Monday. However, the stock has dipped 0.5% so far this year against the industry’s rally of 15.7%.

Tibsovo is already approved by the FDA for treating adult patients suffering relapsed/refractory acute myeloid leukemia (AML) with a susceptible IDH-1 mutation. The drug is also under review in the EU for the same indication with decision pending in the second half of 2020.

Tibsovo generated sales of $40.3 million in the first nine months of 2019, reflecting a significant increase year over year. In May 2019, the drug was approved by the FDA in the first-line setting. This nod expanded the drug’s eligible patient population and should drive sales higher in the future quarters.

Meanwhile, Tibsovo is being evaluated in the phase III ClarIDHy study in previously treated patients with IDH1 mutant cholangiocarcinoma also called bile-duct cancer. The company expects to file an sNDA to get FDA approval for the above indication by 2019 end. Currently, there are no treatment options available to treat this cancer.

We remind investors that Geron Corporation GERN is developing its pipeline candidate imetelstat in the phase III IMerge study for treating MDS. In October 2019, the company initiated dosing in the phase III IMerge study.

Meanwhile, a regulatory application for Acceleron Pharma’s XLRN luspatercept is under review with the FDA seeking approval as a treatment for MDS. A decision from the FDA is awaited in April 2020. Aprea Therapeutics APRE is also developing a late-stage candidate for the treatment of MDS.

Zacks Rank

Agios currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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