$AGIO Deficiency Drug AG-348 Gets Orphan Drug Status – Analyst Blog

Biotech Stocks

Agios’ PK Deficiency Drug AG-348 Gets Orphan Drug Status – Analyst Blog

Agios Pharmaceuticals, Inc. AGIO announced that its pyruvate kinase (PK) deficiency candidate, AG-348, has been granted orphan drug status by the FDA.

PK deficiency is a rare form of hemolytic anemia. The company estimates that there are approximately 2,400 diagnosed patients suffering from PK deficiency in the U.S. and five European countries (the UK, France, Germany, Italy and Spain).

The FDA usually grants orphan drug status to drugs or biologics that are being developed for the treatment of rare diseases/conditions affecting less than 200,000 people in the U.S. at any given time. This designation allows the product to enjoy a period of marketing exclusivity in the U.S., upon approval. Incentives that come with orphan drug status include tax credits as well as waiver of certain administrative fees.

We note that in Dec 2014, Agios had presented data on AG-348 at the annual meeting of the American Society of Hematology, exhibiting early proof-of-mechanism. Based on this data, the company intends to start a phase II study on the candidate in patients with PK deficiency in the first half of 2015.

Meanwhile, Agios has other pipeline events lined up this year. The company expects to commence a global registration program for AG-221 in hematologic malignancies in the second half of 2015.

Additionally, new data on another candidate – AG-120 — from a phase I study in patients with IDH1 mutant positive advanced hematologic malignancies will be presented at a medical conference in mid-2015. Agios has a collaboration agreement with Celgene Corporation CELG for both AG-221 and AG-120.

Agios carries a Zacks Rank #4 (Sell). Some better-ranked stocks in the health care sector include Lannett Company, Inc. LCI and Cambrex Corp. CBM. Both companies sport a Zacks Rank #1 (Strong Buy).


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