Agios Reports Favorable Phase II PK Deficiency Study Data

Agios Pharmaceuticals, Inc. (NASDAQ:AGIO) announced encouraging initial data from an ongoing open-label phase II study (DRIVE-PK) on one of its pipeline candidates, AG-348, for the treatment of patients suffering from pyruvate kinase (PK) deficiency. Data were presented at the Congress of the European Hematology Association.

The study is being conducted to evaluate the safety and efficacy of AG-348 in adult transfusion-independent patients with PK deficiency. Results showed that AG-348 achieved proof-of-concept with nine of 18 total patients (nine of 13 patients with at least one missense mutation) witnessing rapid, robust and sustained hemoglobin increases of >1.0 g/dL as of the data cut-off (Mar 27, 2016). In patients who had such hemoglobin increases, the mean maximum hemoglobin increase was 3.4 g/dL and ranged from 2.3 g/dL to 4.9 g/dL.

Moreover, pharmacokinetics were favorable and consistent with those observed in healthy volunteers. On the safety front, AG-348 was found to be well tolerated with none of the patients discontinuing treatment early and the majority of adverse events were mild to moderate (Grade 1-2) and temporary.

On the back of the favorable phase II study results, the company plans to move the candidate into late-stage development. Per the company’s press release, PK deficiency is a rare, debilitating, congenital anemia. Currently, there is no approved therapy to treat the underlying cause of PK deficiency.

We note that late last week the company’s shares took a beating when it reported initial data from a phase I integrated single ascending dose and multiple ascending dose study on its another PK deficiency candidate AG-519 in healthy volunteers. Results showed that one patient who received AG-519 experienced a low blood platelet count (Grade 2 thrombocytopenia). The study is presently ongoing to collect additional clinical safety, pharmacokinetic and pharmacodynamics data.

Agios is a Zacks Rank #3 (Hold) stock. Some better-ranked stocks in the health care sector include Retrophin, Inc. (NASDAQ:RTRX) , Bristol-Myers Squibb Company (NYSE:BMY) and Pfizer Inc. (NYSE:PFE) . All three stocks carry a Zacks Rank #1 (Strong Buy).