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[{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios Receives Positive Opinion on Orphan Drug Designation from the European Medicines Agency for Mitapivat","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios to Present Updated Clinical Proof-of-Concept Data from the Phase 2 Study of Mitapivat in Thalassemia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios Announces FDA Orphan Drug Designation Granted to Mitapivat for Treatment of Thalassemia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios PKR Activator Mitapivat Demonstrates Sustained Hemoglobin Responses in Non-transfusion-dependent \u03b1- and \u03b2-Thalassemia in Phase 2 Study","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios Announces FDA Orphan Drug Designation Granted to Mitapivat for Treatment of Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios' Phase 3 ACTIVATE Trial of Mitapivat Met Primary Endpoint in Adults with Pyruvate Kinase Deficiency Who Are Not Regularly Transfused","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios Announces Phase 3 ACTIVATE-T Trial of Mitapivat Achieved Primary Endpoint in Adults with Pyruvate Kinase Deficiency","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III"},{"orgOrder":0,"company":"Centogene","sponsor":"Agios Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"CENTOGENE Expands Partnership With Agios to Generate Novel Insights Into Rare Blood Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"GERMANY","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios to Present Updated Data from Clinical Trials of Mitapivat in Pyruvate Kinase Deficiency and Thalassemia at EHA 2021","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios Presents Positive Results from Phase 2 Study of Mitapivat in Non-transfusion-dependent \u03b1- and \u03b2-Thalassemia at EHA2021","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios Submits New Drug Application to FDA for Mitapivat for Treatment of Adults with Pyruvate Kinase Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Centogene","sponsor":"Agios Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Centogene and Agios Expand Partnership for Clinical Development of PYRUKYND (mitapivat) to Treat Children With Rare Blood Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"GERMANY","productType":"Small molecule","productStatus":"Approved","date":"June 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios Announces FDA Approval of PYRUKYND\u00ae (mitapivat) as First Disease-Modifying Therapy for Hemolytic Anemia in Adults with Pyruvate Kinase Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios Announces Publication of Phase 3 ACTIVATE Study in New England Journal of Medicine Demonstrating Benefits of PYRUKYND (mitapivat) for Adults with Pyruvate Kinase Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"April 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios to Present Clinical and Translational Data at the European Hematology Association Annual Congress","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"May 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios Presents New Clinical Data Supporting the Benefits of PYRUKYND (mitapivat) Treatment in Adults with PK Deficiency at European Hematology Association Annual Congress","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios Announces Publication of Phase 2 Data in The Lancet Demonstrating Safety and Efficacy of PYRUKYND\u00ae (mitapivat) in Non-transfusion-dependent \u03b1- and \u03b2-Thalassemia","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"August 2022","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios Announces Publication of Phase 3 ACTIVATE-T Data in The Lancet Haematology Demonstrating Benefits of PYRUKYND\u00ae (mitapivat) for Adults with Pyruvate Kinase Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"August 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios Receives Positive CHMP Opinion for PYRUKYND\u00ae (mitapivat) for the Treatment of Pyruvate Kinase (PK) Deficiency in Adult Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"September 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PYRUKYND\u00ae (mitapivat) Approved in the EU for the Treatment of Pyruvate Kinase (PK) Deficiency in Adult Patients","therapeuticArea":"Hematology","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"November 2022","url1":"","url2":"","graph1":"Hematology","graph2":"Approved"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios Announces Positive Results from Phase 2 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":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios Announces Phase 3 ENERGIZE Study of Mitapivat Met Primary Endpoint and Both Key Secondary Endpoints in Adults with Non-Transfusion-Dependent Alpha- or Beta-Thalassemia","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"January 2024","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios\u2019 Mitapivat Meets All Key Endpoints in Phase 3 ENERGIZE-T Study for Thalassemia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"June 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"}]

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            Details:

            Pyrukynd (mitapivat sulfate) is a pyruvate kinase activator. It is under phase 3 clinical development for the treatment of non-transfusion-dependent alpha- or beta-thalassemia.

            Lead Product(s): Mitapivat

            Therapeutic Area: Genetic Disease Product Name: Pyrukynd

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 03, 2024

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            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.

            Lead Product(s): Mitapivat

            Therapeutic Area: Rare Diseases and Disorders Product Name: Pyrukynd

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 03, 2024

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            PYRUKYND (mitapivat sulfate) is a pyruvate kinase activator and indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency.

            Lead Product(s): Mitapivat

            Therapeutic Area: Hematology Product Name: Pyrukynd

            Highest Development Status: Phase II/ Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 26, 2023

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            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.

            Lead Product(s): Mitapivat

            Therapeutic Area: Hematology Product Name: Pyrukynd

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 10, 2022

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            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.

            Lead Product(s): Mitapivat

            Therapeutic Area: Genetic Disease Product Name: Pyrukynd

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 16, 2022

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            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.

            Lead Product(s): Mitapivat

            Therapeutic Area: Genetic Disease Product Name: Pyrukynd

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 18, 2022

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            Details:

            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.

            Lead Product(s): Mitapivat

            Therapeutic Area: Rare Diseases and Disorders Product Name: Pyrukynd

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 11, 2022

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            Details:

            CENTOGENE’s proprietary CentoCard® dried blood spot technology provides easy logistics for central diagnostic testing. PYRUKYND (Mitapivat Sulfate), is a pyruvate kinase activator indicated for the treatment of hemolytic anemia in adults with pyruvate kinase deficiency.

            Lead Product(s): Mitapivat

            Therapeutic Area: Genetic Disease Product Name: Pyrukynd

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Agios Pharmaceuticals

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Expanded Collaboration June 13, 2022

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            Details:

            Across the ACTIVATE and extension studies, 28/78 (35.9%) of all patients achieved a normal hemoglobin level at least once during treatment with , a pyruvate kinase activator PYRUKYND (mitapivat).

            Lead Product(s): Mitapivat

            Therapeutic Area: Genetic Disease Product Name: Pyrukynd

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 10, 2022

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            Details:

            The Phase 3 ACTIVATE trial of mitapivat achieved its primary endpoint. Pyrukynd (mitapivat sulfate) demonstrated a statistically significant increase in hemoglobin in patients with PK deficiency who are not regularly transfused.

            Lead Product(s): Mitapivat

            Therapeutic Area: Genetic Disease Product Name: Pyrukynd

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 12, 2022

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