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Agios' (AGIO) Mitapivat Gets Orphan Drug Tag to Treat SCD

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Agios Pharmaceuticals, Inc. (AGIO - Free Report) announced that the FDA has granted its first-in-class pyruvate kinase-R (PKR) activator mitapivat an orphan drug designation for the treatment of patients with sickle cell disease (SCD), which is a blood disorder. The company plans to begin a pivotal phase III study on mitapivat for SCD in 2021.

Notably, an orphan drug designation is usually granted to those drugs that are capable of treating rare diseases that affect less than 200,000 people in the United States. This tag also makes the company entitled to certain other benefits including tax credits related to clinical trial expenses and an exemption from the FDA user fee.

Shares of Agios were up 1.1% in after-hours trading following the above announcement on Thursday. However, the stock has lost 13.1% so far this year against the industry’s increase of 3%.

Price chart for Agios


We note that mitapivat is one of key pipeline candidates of Agios, which is being developed for treating patients with pyruvate kinase (PK) deficiency. The candidate was previously granted an orphan drug status, both by the FDA and the European Medicines Agency for addressing pyruvate kinase (PK) deficiency, a rare, debilitating, hemolytic anemia.

The regulatory body also granted an orphan drug status to mitapivat in June 2020 for addressing patients with thalassemia. Notably, Agios plans to commence a phase III study in 2021 on mitapivat for the treatment of thalassemia.

Agios is currently evaluating mitapivat in the single-arm ACTIVATE-T study for addressing PK in patients who are on regular blood transfusions with top-line results from the same expected in the first quarter of 2021. Also, the ACTIVATE study is evaluating mitapivat for treating PK deficiency in patients with no regular blood transfusions. Top-line data from the same is expected by 2020-end.

Successful development and potential commercialization of the candidate will drive the company’s prospects in the future.

Zacks Rank & Stocks to Consider

Agios currently carries a Zacks Rank #3 (Hold). Better-ranked stocks in the same sector include Owens & Minor, Inc. (OMI - Free Report) , LeMaitre Vascular, Inc. (LMAT - Free Report) and Bio-Rad Laboratories, Inc. (BIO - Free Report) all sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

Owens & Minor’s earnings estimates have been revised 71.8% upward for 2020 and 56.5% for 2021 over the past 60 days. The stock has skyrocketed 354% year to date.

LeMaitre Vascular’s earnings estimates have been revised 29.8% upward for 2020 and 16.8% for 2021 over the past 60 days. The stock has inched up 2.8% year to date.

Bio-Rad Laboratories’ earnings estimates have been revised 21.6% upward for 2020 and 7.5% for 2021 over the past 60 days. The stock has rallied 62.8% year to date.

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