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CENTOGENE and Agios Expand Partnership for Clinical Development of PYRUKYND® (mitapivat) to Treat Children With Rare Blood Disease
Working together on the world’s first treatment that targets the underlying causes of pyruvate kinase (“PK”) deficiency in children under the age of 18
About this update from Agios Pharmaceuticals, Inc.
[{"type":"text","content":"Working together on the world’s first treatment that targets the underlying causes of pyruvate kinase (“PK”) deficiency in children under the age of 18\nExpanded collaboration to include Phase 3 ACTIVATE-kids and ACTIVATE-kidsT studies CENTOGENE’s genetic testing and Biodatabank identify causative mutations, including the UGT1A1 and PKLR genes, and supports precise diagnosis CAMBRIDGE, Mass. and ROSTOCK, Germany and BERLIN, June 13, 2022 (GLOBE NEWSWIRE) -- Centogene N.V. (Nasdaq: CNTG), the commercial-stage essential biodata life science partner for rare and neurodegenerative diseases, today announced that it has expanded its long-standing PYRUKYND® (mitapivat) partnership with Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism pioneering therapies for genetically defined diseases. Agios’ PYRUKYND® is a first-in-class, selective, small molecule activator of the pyruvate kinase (PK) enzyme. In February 2022, Agios received approval from the U.S. Food and Drug Administration (FDA) for PYRUKYND® for the treatment of hemolytic anemia in adults with PK deficiency, which represents the first and only approved disease-modifying treatment for this rare blood disorder. Since 2015, CENTOGENE has provided genetic testing services to Agios for the clinical development of PYRUKYND®. The expanded agreement will now see CENTOGENE provide centralized lab support for Agios’ two global, pivotal Phase 3 trials, ACTIVATE-kids and ACTIVATE-kidsT, which are being conducted to evaluate the efficacy and safety of mitapivat in children with PK deficiency between the ages of 1 to 17. CENTOGENE will continue to provide genetic testing to help identify causative mutations, including the UGT1A1 and PKLR genes, in study participants. The aim of the Phase 3 ACTIVATE-kids and ACTIVATE-kidsT studies is to test the efficacy of PYRUKYND® in pediatric patients with PK deficiency who do not receive regular transfusions and those who do receive regular transfusions, respectively. Agios will be coordinating and bearing the costs for the programs; other financial details were not disclosed. “Our successful collaboration with Agios has already helped drive incredible clinical progress for this first-in-class PK activator as a potential therapy for children with pyruvate kinase deficiency, an under-served patient community urgently...