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Lead Product(s): Mitapivat
Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed
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 26, 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.
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Lead Product(s): Mitapivat
Therapeutic Area: Rare Diseases and Disorders Product Name: AG-348
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 December 01, 2020
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.
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Lead Product(s): SNF472
Therapeutic Area: Rare Diseases and Disorders Product Name: SNF472
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 October 22, 2020
Details:
Phase 2b CaLIPSO study shows that SNF472 consistently slowed progression of coronary artery calcium (CAC) across key subgroups of patients on hemodialysis participating in the study.
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