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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 IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 03, 2024
Details:
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).
Lead Product(s): AG-946
Therapeutic Area: Oncology Product Name: AG-946
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 20, 2023
Details:
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).
Lead Product(s): siRNA
Therapeutic Area: Oncology Product Name: Undisclosed
Highest Development Status: PreclinicalProduct Type: Large molecule
Recipient: Alnylam Pharmaceuticals
Deal Size: $147.5 million Upfront Cash: $17.5 million
Deal Type: Licensing Agreement August 03, 2023
Details:
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 IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 26, 2023
Details:
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: ApprovedProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 10, 2022
Details:
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.
Lead Product(s): AG-946
Therapeutic Area: Hematology Product Name: AG-946
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 03, 2022
Details:
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).
Lead Product(s): Ivosidenib
Therapeutic Area: Oncology Product Name: Tibsovo
Highest Development Status: ApprovedProduct Type: Small molecule
Partner/Sponsor/Collaborator: Sagard Healthcare Partners
Deal Size: $131.8 million Upfront Cash: Undisclosed
Deal Type: Agreement October 27, 2022
Details:
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: ApprovedProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 16, 2022
Details:
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: ApprovedProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 18, 2022
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 IIIProduct 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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