Agios rare blood disorder drug Pyrukynd sustains benefits in long term data

Agios Pharmaceuticals (NASDAQ:AGIO) said long term data suggested that Pyrukynd led to improvements in hemoglobin, iron overload, transfusion burden and patient-reported outcomes, regardless of transfusion status in adults with pyruvate kinase (PK) deficiency.

The company reported new data from an ongoing extension study of Pyrukynd (mitapivat) in patients who had participated in one of the studies, ACTIVATE and ACTIVATE-T, conducted in not regularly transfused and regularly transfused adults with PK deficiency, respectively.

PK deficiency is a rare genetic disorder characterized by the premature destruction of red bloods, which is called hemolytic anemia.

Agios said that as of the March 27 data cut-off, the median duration of hemoglobin response among the 31 hemoglobin responders from ACTIVATE and the long-term extension study was 18.3 months, with responses ongoing up to 32.9 months.

Hemoglobin response rate among patients who switched from placebo in ACTIVATE to Pyrukynd in the extension study (39.5% Hb response rate) was similar to that seen in patients treated with Pyrukynd in ACTIVATE, the company added.

Agios noted that All regularly transfused patients who achieved transfusion-free status in ACTIVATE-T with Pyrukynd maintained transfusion-free status in the extension study for up to 38.3 months.

The drug was well tolerated, and the safety profile was consistent with that seen in the two studies.

“Collectively, the data we have presented at ASH continue to demonstrate the benefits of long-term treatment with PYRUKYND® for adults with PK deficiency, including improvements in hemoglobin, transfusion burden, iron overload and patient-reported outcomes,” said Sarah Gheuens, chief medical officer and head of R&D at Agios.

Pyrukynd is approved in the U.S. and EU to treat adults with PK deficiency.

The company presented the data at the 64th American Society of Hematology (ASH) Annual Meeting.