Phase 2 Multi-center Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia

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This was a multicenter, single-blind, multiple dose study of Nevanimibe (ATR- 101) with a placebo washout component. The Screening Period was followed by a placebo, baseline lead-in period. Subjects then received the lowest dose of Nevanimibe for 14 days followed by a single-blind placebo Washout Period of 14 days. Safety and efficacy assessments occurred at the end of treatment and following the washout period. If the primary outcome measure (17-OHP 2x ULN) was not achieved, the subject proceeded to the next higher dose level. A total of 5 ATR-101 dose levels were possible. Ten patients were enrolled; nine completed the study, and 1 discontinued early due to a related serious adverse event. Two subjects met the primary endpoin, and 5 others experienced 17-hydroxyprogesterone decreases ranging from 27% to 72% during treatment. the most common side effects were gastointestinal (30%). There were no dose-related trends in adverse events. Nevanimibe (ATR-101) decreased 17-hydroxyprogesterone levels within 2 weeks of treatment. Larger studies of longer duration are needed to further evaluate its efficacy as add-on therapy for CAH.