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Portion of RISE UP Pivotal Study in Sickle Cell Disease with Both Mitapivat Dose Arms Achieving Statistically Significant Hemoglobin Response","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"June 2023","url1":"","url2":"","graph1":"Hematology","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Alnylam Pharmaceuticals","pharmaFlowCategory":"D","amount":"$147.5 million","upfrontCash":"$17.5 million","newsHeadline":"Agios Announces Exclusive Worldwide License Agreement for Novel siRNA for the Potential Treatment of Polycythemia Vera","therapeuticArea":"Oncology","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2023","url1":"","url2":"","graph1":"Oncology","graph2":"Preclinical"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not 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Find Clinical Drug Pipeline Developments & Deals by Agios Pharmaceuticals
Pyrukynd (mitapivat sulfate) is a pyruvate kinase activator indicated for the treatment of hemolytic anemia. It is under phase 3 clinical development for the treatment of non-transfusion-dependent alpha- or beta-thalassemia.
AG-946 is an investigational pyruvate kinase (PK) activator, which is under clinical development fot the potential treatment for anemia in adults with lower-risk myelodysplastic syndromes (LR-MDS).
Under the agreement, Agios will acquire the rights to develop and commercialize Alnylam’s novel preclinical siRNA targeting TMPRSS6, as a potential disease-modifying treatment for patients with polycythemia vera (PV).
PYRUKYND (mitapivat sulfate) is a pyruvate kinase activator and indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency.
PYRUKYND® is a first-in-class, oral PK activator and the first approved disease-modifying therapy for patients in the EU with this rare, debilitating, lifelong hemolytic anemia.
AG-946, is a novel small molecule that activates the enzyme pyruvate kinase which contributes to the production of energy in the RBC. By helping to increase energy, RBC functionality and survival may be improved.
TIBSOVO® (ivosidenib) is an oral, targeted therapy approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with an isocitrate dehydrogenase-1 (IDH1) mutation with acute myeloid leukemia (AML) or cholangiocarcinoma (bile duct cancer).
PYRUKYND® (mitapivat sulfate) demonstrated a significant increase in hemoglobin in patients with PK deficiency who are not regularly transfused. 40 percent of patients randomized to PYRUKYND® achieved a hemoglobin response, compared to 0 patients randomized to placebo.
Result of study met primary endpoint, with 10 of 27 patients receiving PYRUKYND (mitapivat sulfate) achieving transfusion reduction response, defined as ≥33% reduction in transfusion burden in 24-week fixed dose period.
PYRUKYND® (mitapivat sulfate) is a first-in-class, investigational, oral, small molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase (PK) enzymes.
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