RG7388: Phase I data

An open-label Phase I/Ib trial in 86 patients with AML showed that the recommended Phase II dose of RG7388 as monotherapy and in combination with cytarabine is 1,200 mg daily (600 mg twice daily) for 5 days in a 28-day cycle. There was 1 DLT of prolonged myelosuppression and the most common adverse events were gastrointestinal or infection-related. In 20 patients in the Part 1 dose-escalation cohort, RG7388 as monotherapy led to 2 complete remissions (CR), 3 partial remissions (PR), 3 CRs with incomplete recovery/morphologic leukemia-free state CRi/MLFS and 4 cases of hematologic improvement (HI). Patients in the cohort received once-daily 400, 600 or 1,600 mg RG7388 as monotherapy for 5 days. In 9 patients in the Part 1 extension cohort, once-daily 1,200 mg RG7388 as monotherapy led to 1 CRi/MLFS and 2 cases of HI. Further enrollment in the extension was discontinued as induction of prolonged myelosuppression increased the risks of infection and early deaths. ...