Agios Pharma (AGIO) Announces the Publication of Results from the Phase 3 ACTIVATE Study Demonstrating the Benefits of PYRUKYND

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Agios Pharmaceuticals, Inc. (NASDAQ: AGIO) today announced that mid-period data from the pivotal Phase 3 ACTIVATE study of PYRUKYND® (mitapivat) in adults with pyruvate kinase (PK) deficiency who do not receive regular transfusions was published on April 14, 2022 in the New England Journal of Medicine. The data from this study was previously presented at the Virtual European Hematology Association (EHA) Congress held in June 2021. PYRUKYND® is a first-in-class oral PK activator and the first and only disease-modifying therapy approved for this hemolytic anemia rare, debilitating and permanent.

“The results of the ACTIVATE study confirm that mitapivat, through its novel mechanism of activating pyruvate kinase and thus increasing ATP levels in red blood cells, successfully treated the underlying cause of ‘chronic hemolytic anemia in adults with PK deficiency with a wide range of genotypes,’ said Hanny Al-Samkari, MD, hematologist and clinical researcher at the Mass General Cancer Center and Harvard Medical School, researcher in the pivotal phase 3 ACTIVATE study and first author of this publication. “The improved patient-reported outcome measures specific to PK deficiency further support the clinical efficacy of mitapivat and its benefits on health-related quality of life and reduction in symptom severity. »

“We have pioneered the science of PK activation for over a decade and are pleased to have developed the first product approved for people with PK deficiency who previously had no modifying treatment options. of the disease,” said Sarah Gheuens, MD, Ph.D., chief medical officer at Agios. “The results of the ACTIVATE study underscore the clinical value of PYRUKYND® and support our ongoing efforts to bring this drug to as many patients as possible to benefit from it.”

As reported in the publication, the ACTIVATE study met its primary endpoint, with 40% of patients randomized to receive PYRUKYND® achieving a hemoglobin response, defined as an increase of ≥ 1.5 g/dL in the concentration of hemoglobin from baseline, sustained at two or more scheduled assessments at Weeks 16, 20, and 24 during the fixed-dose period, compared to 0 patients randomized to placebo (two-sided p

PYRUKYND® was approved in February 2022 by the United States Food and Drug Administration (FDA) for the treatment of hemolytic anemia in adults with PK deficiency. PYRUKYND® is also under review by the European Medicines Agency (EMA) as a potential treatment for adults with PK deficiency, and Agios expects a regulatory decision in the EU by end of 2022. The FDA and EMA have both granted orphan drug designation to PYRUKYND® in PK deficiency. Learn more about www.PYRUKYND.com.

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