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Agios (AGIO) Rare Disease Drug NDA Gets FDA Priority Review

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Agios Pharmaceuticals, Inc. (AGIO - Free Report) announced that the FDA has accepted the new drug application for its lead pipeline candidate, mitapivat, for priority review. The NDA is seeking the approval of the candidate as a potential treatment of adult patients with pyruvate kinase (“PK”) deficiency, a rare, inherited disease. Currently, there is no treatment approved for the given indication.

The priority review has reduced the anticipated timeline for review of mitapivat NDA from 10 months to six months. The FDA will announce its decision onthepotential approval for mitapivat by Feb 17, 2022.

Shares of Agios were up nearly 3% during after-hours trading on Aug 17 with the FDA granting priority review tag to the NDA. The company’s shares have gained 3.1% so far this year compared with the industry’s increase of 3.6%.

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The NDA was based on data from two pivotal phase III studies – ACTIVATE and ACTIVATE-T – which evaluated mitapivat for treating PK deficiency in adults who are not regularly transfused and those who are regularly transfused, respectively.

A marketing authorization application, seeking approval of mitapivat for treating adults with PK deficiency in Europe,is also under review.

Upon potential approval and launch, mitapivat can provide a first potentially disease-modifying therapy for people with PK deficiency, thereby serving an area of high unmet medical need. However, the company’s operating expenses will likely go higher in the upcoming quarters to support pre-commercialization and commercialization activities for mitapivat. Moreover, the company is also conducting several clinical studies to support the approval of mitapivat in other hemolytic anemias indications.

A phase II/III study on mitapivat for sickle cell disease is expected to begin by year-end. The company also plans to initiate two phase III studies on mitapivat in thalassemia (regularly transfused as well as not regularly transfused) this year.

We remind investors that Agios completed the sale of its oncology portfolio to France-based pharmaceutical company, Servier, in March 2021. Following this sale, the company’s sole focus now remains on expanding its genetically defined disease portfolio, including the clinical development of mitapivat.

This apart, Agios is also evaluating AG-946, its next-generation pyruvate kinase-R activator, in a phase I study for the treatment of hemolytic anemia.

Zacks Rank & Stocks to Consider

Currently, Agios has a Zacks Rank #3 (Hold).

Some better-ranked stocks from the biotech/drug sector include Horizon Therapeutics (HZNP - Free Report) , Repligen Corporation (RGEN - Free Report) and Aptose Biosciences (APTO - Free Report) . While Horizon sports a Zacks Rank #1 (Strong Buy), Repligen and Aptose currently carry a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Horizon’s earnings per share estimates for 2021 have increased from $3.62 to $4.46 in the past 30 days while that for 2022 have increased from $5.18 to $5.84 over the same period. The stock has risen 44.2% in the year so far.

Repligen’s earnings per share estimates for 2021 have increased from $2.26 to $2.76 in the past 30 days while that for 2022 have increased from $2.56 to $3.02 over the same period. The stock has risen 34.1% in the year so far.

Aptose’s loss per share estimates for 2021 have narrowed from 76 cents to 68 cents in the past 30 days while that for 2022 have narrowed from 96 cents to 92 cents over the same period.