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Details:
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:
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:
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
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 IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Agios Pharmaceuticals
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Expanded Collaboration June 13, 2022
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 IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 10, 2022
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: ApprovedProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 12, 2022
Details:
PYRUKYND is a pyruvate kinase activator indicated for the treatment of hemolytic anemia in adults with pyruvate kinase deficiency. The results of the ACTIVATE study underscore the clinical value of PYRUKYND ® and support the efforts to deliver to as many patients as possible.
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 April 14, 2022
Details:
Approval based on results from ACTIVATE and ACTIVATE-T Phase 3 studies for Pyrukynd (mitapivat sulfate) oral PK activator, supports the treatment of adults with PK deficiency regardless of transfusion status.
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 February 17, 2022
Details:
Under the terms of the new agreement, CENTOGENE will provide genetic testing and clinical trial support for Agios’ three global, pivotal trials in thalassemia and sickle cell disease including AG-348 (Mitapivat).
Lead Product(s): Mitapivat
Therapeutic Area: Genetic Disease Product Name: AG-348
Highest Development Status: Phase IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Agios Pharmaceuticals
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Partnership November 04, 2021
Details:
An extension study for adults with PK deficiency previously enrolled in ACTIVATE or ACTIVATE-T is ongoing and designed to evaluate the long-term safety, tolerability and efficacy of treatment with mitapivat.
Lead Product(s): Mitapivat
Therapeutic Area: Genetic Disease Product Name: AG-348
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 June 21, 2021
Details:
AG-348, an allosteric activator of PK that is currently in clinical trials for treatment of PK deficiency, on RBCs and erythroid precursors from PK-deficient patients.
Lead Product(s): Mitapivat
Therapeutic Area: Genetic Disease Product Name: AG-348
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 June 11, 2021
Details:
Mitapivat is an investigational, wholly owned, first-in-class, novel, oral activator of both wild-type (normal) and mutated pyruvate kinase-R (PKR) enzymes used against thalassemia and sickle cell disease.
Lead Product(s): Mitapivat
Therapeutic Area: Genetic Disease Product Name: AG-348
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 May 12, 2021
Details:
Out of 27 patients enrolled in the study treated with mitapivat, at a 24-week fixed dose period, 37% achieved a ≥33% reduction in transfusion burden compared to individual historical transfusion burden standardized to 24 weeks.
Lead Product(s): Mitapivat
Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed
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 26, 2021
Details:
Treatment with mitapivat demonstrated a statistically significant, sustained increase in hemoglobin compared to placebo. 40 Percent of Patients Treated with Mitapivat Achieved a Sustained Hemoglobin Increase of ≥1.5 g/dL Compared to 0 Placebo Patients.
Lead Product(s): Mitapivat
Therapeutic Area: Rare Diseases and Disorders Product Name: AG-348
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 December 01, 2020
Details:
Mitapivat is being evaluated as a potential treatment for sickle cell disease under a Cooperative Research and Development Agreement (CRADA) with the NIH. Mitapivat is an oral, small molecule allosteric activator of wild-type and a variety of mutated PKR enzymes.
Lead Product(s): Mitapivat
Therapeutic Area: Genetic Disease Product Name: AG-348
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 12, 2020
Details:
The ongoing, open-label Phase 2 study is evaluating the efficacy, safety, pharmacokinetics and pharmacodynamics of mitapivat treatment in adults with non-transfusion-dependent α- and β-thalassemia who have a baseline hemoglobin (Hb) concentration of ≤10 g/dL.
Lead Product(s): Mitapivat
Therapeutic Area: Genetic Disease Product Name: Undisclosed
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 June 12, 2020
Details:
The U.S. FDA has granted orphan drug designation to the company’s first-in-class pyruvate kinase-R activator mitapivat for the treatment of patients with thalassemia. Mitapivat is an oral, small molecule allosteric activator of wild-type and a variety of mutated PKR enzymes.
Lead Product(s): Mitapivat
Therapeutic Area: Genetic Disease Product Name: Undisclosed
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 June 08, 2020
Details:
Mitapivat and Ivosidenib clinical data will be presented at the European Hematology Association (EHA) Annual Congress. Data Suppots ustility of both the drugs in treating Thalassemia.
Lead Product(s): Mitapivat
Therapeutic Area: Genetic Disease Product Name: Undisclosed
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 May 14, 2020
Details:
Mitapivat is the first and only PKR activator to receive this important designation in Europe and the first potential disease-modifying treatment for PK deficiency patients.
Lead Product(s): Mitapivat
Therapeutic Area: Genetic Disease Product Name: Undisclosed
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 March 30, 2020