Agios Pharmaceuticals Inc (NASDAQ:AGIO) has edged closer to the marketing approval of its anemia drug candidate, AG-348. The company recently reported positive data from a Phase 2 trial of AG-348 in more than a dozen patients afflicted by an inherited form of anemia.

AG-348 is the most advanced of Agios Pharmaceuticals Inc (NASDAQ:AGIO)’s candidates, being developed for treatment in question. According to Agios, AG-348 significantly boosted hemoglobin levels in 50% of the patients enrolled in the mid-stage trial.

Study duration

The mid-stage study enrolled 18 patients who took the compound for duration of between 3 and 24 weeks.

Of the participating patients, nine, or 50% of the enrolled patient group, registered a stat sig improvement in hemoglobin levels. The smallest recorded increase was one gram per deciliter and the average increase was 3.4 grams per deciliter.

According to Agios’ CEO, David Schenkein, the range of hemoglobin in the blood of a healthy person is 13 to 17 grams. However, people with the rare form of inherited anemia being targeted by AG-348 have far less, usually in the range of 7 to 10 grams.

Side effects

Agios Pharmaceuticals said that its Phase 2 study of the drug didn’t reveal any serious safety issues that could lead to discontinuation of the development of the drug.

How does the compound work?

AG-348 is designed to bind to an enzyme that is involved in creating hemoglobin. As such, the compounded is said to work more efficiently, which will be the major selling point if the drug obtains regulatory clearance. Agios is hoping to develop AG-348 into a marketable product and the latest data that highlights the performance of the compound has lifted expectations as the drug navigates the development pathway.

Agios Pharmaceuticals has two compounds in late stage trials, both of which target the blood cancer market.

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