On August 1st, we announced that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for Pracinostat in combination with azacitidine for the treatment of patients with newly diagnosed AML who are ≥75 years of age or unfit for intensive chemotherapy.
According to the FDA, Breakthrough Therapy Designation is intended to expedite the development and review of drugs for serious or life-threatening conditions. The criteria for Breakthrough Therapy Designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy.
The breakthrough designation speaks to pracinostat’s substantial improvement over available therapies for the treatment of AML patients who are unfit for induction chemotherapy based on the results of the Phase 2 trial Karen Potts, PhD, Senior Vice President, Regulatory Affairs
The Breakthrough Therapy Designation is supported by data from a Phase II study of Pracinostat plus azacitidine in elderly patients with newly diagnosed AML who are not candidates for induction chemotherapy. The study showed a median overall survival of 19.1 months and a complete response (CR) rate of 42% (21 of 50 patients). These data compare favorably to a Phase III study of azacitidine (AZA-001).
The combination of Pracinostat and azacitidine was generally well tolerated in the study, with no unexpected toxicities. The most common grade 3/4 treatment-emergent adverse events included febrile neutropenia, thrombocytopenia, anemia and fatigue.
In addition to Breakthrough Therapy Designation, we also announced that agreement has been reached with the FDA on the proposed design of the upcoming Phase III study. We have worked closely with the FDA to get to this point and are committed to supporting our new strategic partner to help bring Pracinostat to patients in need as quickly and efficiently as possible.