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Agios Gets Orphan Drug Tag for Mitapivat to Treat Thalassemia

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Agios Pharmaceuticals, Inc. (AGIO - Free Report) announced that the FDA has granted an orphan drug designation to its first-in-class pyruvate kinase-R (PKR) activator mitapivat for the treatment of patients with thalassemia. The candidate is an investigational, oral, small molecule allosteric activator of wild-type and a variety of mutated PKR enzymes.

Notably, orphan drug designation is granted to 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 1.7% in after-hours trading following the above announcement on Monday. In fact, the stock has increased 9.4% so far this year against the industry’s decrease of 4.4%.


We remind investors that mitapivat 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.

Notably, mitapivat is one of key pipeline candidates of Agios, which is being developed for treating patients with PK deficiency. The company is conducting the single-arm ACTIVATE-T study for addressing PK in patients on regular blood transfusions, with a primary endpoint of reduction in transfusion burden over six months compared to individual historical transfusion burden over prior 12 months.

Agios is also performing the ACTIVATE study for treating PK deficiency in patients with no regular blood transfusions. The primary endpoint of the study is the proportion of patients achieving a sustained hemoglobin increase of ≥1.5 g/dL. Top-line data from both studies is awaited between the end of 2020 and mid-2021.

In January 2020, Agios announced that the phase II study on mitapivat for treating patients with non-transfusion-dependent thalassemia achieved the clinical proof-of-concept.

Zacks Rank & Other Stocks to Consider

Agios currently carries a Zacks Rank #2 (Buy). Other top-ranked stocks in the same sector include Zynex Inc. , Apyx Medical Corporation (APYX - Free Report) and DiaSorin S.p.A. (DSRLF - Free Report) , all sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Zynex’s earnings estimates have been revised 11.1% upward for 2020 and 27.8% for 2021 over the past 60 days. The stock has skyrocketed 190.9% year to date.

Apyx Medical’s loss per share estimates have been narrowed 32% for 2020 and 21.3% for 2020 over the past 60 days.

DiaSorin’s earnings estimates have been revised 5.1% upward for 2020 and 26.3% for 2021 over the past 60 days. The stock has rallied 44.5% year to date.

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