Centogene and Agios expand partnership for clinical development of PYRUKYND® (mitapivat) to treat children with rare blood disease

June 2022

Centogene N.V. 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.

Kim Stratton, CEO of Centogene said: “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 in need of treatment options. The expansion of our partnership confirms our ability to help advance novel treatment options that are able to influence health outcomes for patients globally. With our unparalleled diagnostic services and insights powered by the Centogene Biodatabank, the world’s largest real-world data repository for rare and neurodegenerative diseases, we offer the essential biodata that is often so critical in clinical development for our biotech and pharma partners.”

Sarah Gheuens, M.D., Ph.D., Chief Medical Officer at Agios, said: “As we forge ahead with our global, pivotal trials in pediatric pyruvate kinase deficiency, we are pleased to expand our partnership with Centogene to bring the first potential treatment for these patients one step closer to making an impact in the lives of these children and their families. At Agios, we know that great science requires world-class collaboration. Accelerating and streamlining our clinical trials is critical for bringing new innovations to patients as efficiently as possible, and Centogene’s solutions make them a great partner for this project.”

Within the partnership, Centogene’s proprietary CentoCard^® dried blood spot technology provides easy logistics for central diagnostic testing. Key assets to the partnership include the Centogene Biodatabank, diagnostic expertise built on over 15 years of experience, and the company’s multiomic-based platform, which is pioneering access to commercial multiomic products on the market today.

In 2015, Centogene originally entered into an agreement with Agios to provide support for Agios’ PK deficiency clinical program. Last year, the contract was expanded to focus on additional trial support in thalassemia and sickle cell disease.

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