Agios' blood disorder drug meets goals of late-stage study

[January 03, 2024]  (Reuters) -Agios Pharmaceuticals said on Wednesday its drug helped to increase hemoglobin levels in patients with an inherited blood disorder in a late-stage study, driving the company's shares up 17% in premarket trading.

The drug, mitapivat, met the main goal of the study as it showed a statistically significant increase in hemoglobin response compared to placebo in patients with either alpha- or beta-thalassemia, the two types of an inherited blood disorder in which the body makes less-than-normal hemoglobin, but were not dependent on transfusion.

The drug also met both its secondary goals in the 194-patient study, including an improvement in scores measuring fatigue in patients.

Mitapivat was approved by the U.S. Food and Drug Administration in 2022 and is sold under the brand Pyrukynd for the treatment of a type of hemolytic anemia caused by the deficiency of an enzyme called pyruvate kinase.

Agios is also testing the drug in thalassemia patients who are dependent on transfusion as well as those with sickle cell disease.

Thalassemia affects about 4.4 of every 10,000 live births, with the alpha type occurring most often in persons of African and Southeast Asian descent and the beta type occurring more commonly in people of Mediterranean, African and Southeast Asian descent, according to a paper on the American Academy of Family Physicians website.
 
 (Reporting by Leroy Leo in Bengaluru; Editing by Shilpi Majumdar)

[© 2023 Thomson Reuters. All rights reserved.]This material may not be published, broadcast, rewritten or redistributed.  Thompson Reuters is solely responsible for this content.