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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.