CX-01 is in development for the first-line treatment of AML. Data from a Phase 2 study presented at the 2019 ASCO conference indicated an advantage across multiple endpoints for patients treated with 0.25 mg/kg/hr CX-01. In the evaluable patient population, results of the 0.25 mg/kg/hr CX-01 arm compared favorably to the control arm: complete response rate of 89% vs. 58% (p=0.03), median event-free survival of 23.4 months vs. 9.0 months (p=0.011), and median overall survival which had not yet been reached on the CX-01 arm vs. 11.2 months (p=0.042).
Chimerix plans to initiate a Phase 3 study of CX-01 in mid-2020. CX-01 has received Orphan Drug and Fast Track designations from the U.S. Food and Drug Administration for the treatment of AML. Read more here