Working together on the world\u2019s first treatment that targets the underlying causes of pyruvate kinase (\u201cPK\u201d) deficiency in children under the age of 18
<\/p>\n
CAMBRIDGE, Mass., ROSTOCK, Germany and BERLIN, June 13, 2022 (GLOBE NEWSWIRE)<\/strong> \u2013 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\u00ae<\/sup> (mitapivat) partnership with Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism pioneering therapies for genetically defined diseases.<\/p>\n Agios\u2019 PYRUKYND\u00ae<\/sup> 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\u00ae<\/sup> 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\u00ae<\/sup>.<\/p>\n The expanded agreement will now see CENTOGENE provide centralized lab support for Agios\u2019 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\u00ae<\/sup> 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.<\/p>\n \u201cOur 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,\u201d said Kim Stratton, CEO of CENTOGENE. \u201cWith our unparalleled diagnostic services and insights powered by the CENTOGENE Biodatabank, the world\u2019s 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.\u201d<\/p>\n Sarah Gheuens, M.D., Ph.D., Chief Medical Officer at Agios, said, \u201cAs 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.\u201d<\/p>\n W ramach partnerstwa opatentowana karta CentoCard firmy CENTOGENE\u00ae<\/sup> 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\u2019s multiomic-based platform, which is pioneering access to commercial multiomic products on the market today.<\/p>\n In 2015, CENTOGENE originally entered into an agreement with Agios to provide support for Agios\u2019 PK deficiency clinical program. Last year, the contract was expanded to focus on additional trial support in thalassemia and sickle cell disease.<\/p>\n PYRUKYND\u00ae<\/sup> is a pyruvate kinase activator indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency. In February 2022, Agios received approval from the U.S. Food and Drug Administration (FDA) for PYRUKYND\u00ae<\/sup> for the treatment of hemolytic anemic in adults with PK deficiency, which represents the first and only approved disease-modifying treatment for adults with this rare blood disorder. It has also received a waiver from the European Medicines Agency (EMA) in accordance with a Pediatric Investigation Plan (PIP) for the treatment of PK deficiency in the pediatric population from birth to less than 1 year of age.<\/p>\n Niedob\u00f3r kinazy pirogronianowej (PK) to rzadka, dziedziczna choroba, kt\u00f3ra objawia si\u0119 przewlek\u0142\u0105 anemi\u0105 hemolityczn\u0105, czyli przyspieszonym rozpadem czerwonych krwinek. Odziedziczone mutacje w genie PKLR powoduj\u0105 niedob\u00f3r energii kom\u00f3rkowej w czerwonych krwinkach, o czym \u015bwiadczy ni\u017csza aktywno\u015b\u0107 enzymu PKR, spadek poziomu adenozynotrifosforanu (ATP) oraz nagromadzenie metabolit\u00f3w, w tym 2,3-DPG (2,3-difosfoglicerynianu).<\/p>\n Niedob\u00f3r PK dotyka oko\u0142o 3000\u20138000 os\u00f3b w USA i UE5. Niedob\u00f3r PK mo\u017ce prowadzi\u0107 do powa\u017cnych powik\u0142a\u0144, takich jak nadci\u015bnienie p\u0142ucne, hematopoeza pozaszpikowa i osteoporoza.<\/p>\n To learn more about the ACTIVATE-kids and ACTIVATE-kidsT clinical trials, please visit: https:\/\/www.agios.com\/patients-partners\/patients-caregivers\/clinical-trials\/ <\/a><\/p>\n Agios is a biopharmaceutical company that is fueled by connections. The Agios team cultivates strong bonds with patient communities, healthcare professionals, partners and colleagues to discover, develop and deliver therapies for genetically defined diseases. In the U.S., Agios markets a first-in-class pyruvate kinase (PK) activator for adults with PK deficiency, the first disease-modifying therapy for this rare, lifelong, debilitating hemolytic anemia. Building on the company’s leadership in the field of cellular metabolism, Agios is advancing a robust clinical pipeline of investigational medicines with active and planned programs in alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency and MDS-associated anemia. In addition to its clinical pipeline, Agios has multiple investigational therapies in preclinical development and an industry-leading research team with unmatched expertise in cellular metabolism and genetics. For more information, please visit the company\u2019s website at https:\/\/www.agios.com\/<\/a><\/p>\n CENTOGENE zajmuje si\u0119 diagnostyk\u0105 i badaniami nad chorobami rzadkimi, przekszta\u0142caj\u0105c rzeczywiste dane kliniczne, genetyczne i multiomiczne w celu diagnozowania, zrozumienia i leczenia chor\u00f3b rzadkich. Naszym celem jest racjonalizacja decyzji terapeutycznych i przyspieszenie rozwoju nowych lek\u00f3w sierocych poprzez wykorzystanie naszej rozleg\u0142ej wiedzy i danych na temat chor\u00f3b rzadkich. CENTOGENE opracowa\u0142o globaln\u0105, autorsk\u0105 platform\u0119 bada\u0144 nad chorobami rzadkimi, opart\u0105 na naszym repozytorium danych dotycz\u0105cych chor\u00f3b rzadkich, obejmuj\u0105cym ponad 650 000 os\u00f3b z ponad 120 kraj\u00f3w.<\/p>\n Platforma firmy obejmuje dane epidemiologiczne, fenotypowe i genetyczne, kt\u00f3re odzwierciedlaj\u0105 globaln\u0105 populacj\u0119, a tak\u017ce biobank pr\u00f3bek krwi pacjent\u00f3w i kultur kom\u00f3rkowych. CENTOGENE uwa\u017ca, \u017ce jest to jedyna platforma skoncentrowana na kompleksowej analizie danych wielopoziomowych w celu lepszego zrozumienia rzadkich chor\u00f3b dziedzicznych. Umo\u017cliwia ona lepsz\u0105 identyfikacj\u0119 i stratyfikacj\u0119 pacjent\u00f3w oraz ich chor\u00f3b podstawowych, co umo\u017cliwia i przyspiesza odkrywanie, rozw\u00f3j i dost\u0119p do lek\u00f3w sierocych. Na dzie\u0144 31 grudnia 2021 r. firma wsp\u00f3\u0142pracowa\u0142a z ponad 30 partnerami farmaceutycznymi.<\/p>\n Obserwuj nas na LinkedIn<\/a>. <\/p>\n This press release contains \u201cforward-looking statements\u201d within the meaning of the U.S. federal securities laws. Statements contained herein that are not clearly historical in nature are forward-looking, and the words \u201canticipate,\u201d \u201cbelieve,\u201d \u201ccontinues,\u201d \u201cexpect,\u201d \u201cestimate,\u201d \u201cintend,\u201d \u201cproject,\u201d and similar expressions and future or conditional verbs such as \u201cwill,\u201d \u201cwould,\u201d \u201cshould,\u201d \u201ccould,\u201d \u201cmight,\u201d \u201ccan,\u201d and \u201cmay,\u201d are generally intended to identify forward-looking statements. Such forward-looking statements involve known and unknown risks, uncertainties, and other important factors that may cause CENTOGENE\u2019s actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, negative economic and geopolitical conditions and instability and volatility in the worldwide financial markets, possible changes in current and proposed legislation, regulations and governmental policies, pressures from increasing competition and consolidation in our industry, the expense and uncertainty of regulatory approval, including from the U.S. Food and Drug Administration, our reliance on third parties and collaboration partners, including our ability to manage growth and enter into new client relationships, our dependency on the rare disease industry, our ability to manage international expansion, our reliance on key personnel, our reliance on intellectual property protection, fluctuations of our operating results due to the effect of exchange rates, our ability to streamline cash usage, our requirement for additional financing, or other factors. For further information on the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to CENTOGENE\u2019s business in general, see CENTOGENE\u2019s risk factors set forth in CENTOGENE\u2019s Form 20-F filed on March 30, 2022, with the Securities and Exchange Commission (the \u201cSEC\u201d) and subsequent filings with the SEC. Any forward-looking statements contained in this press release speak only as of the date hereof, and CENTOGENE\u2019s specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events, or otherwise.<\/p>\n Ben LeggAbout PYRUKYND\u00ae (mitapivat)<\/h3>\n
O niedoborze PK<\/h3>\n
O Agios<\/h3>\n
O firmie CENTOGENE<\/h3>\n
O\u015bwiadczenia prognostyczne<\/h3>\n
Kontakt dla medi\u00f3w<\/h4>\n
CENTOGENE<\/h5>\n
Komunikacja korporacyjna
press(at)centogene(dot)com<\/a><\/p>\n