We evaluated nevanimibe in a multicenter, single blind, intra-patient dose escalation Phase 2 clinical trial for the treatment of adult CAH. The trial objectives were to evaluate the efficacy and safety of nevanimibe in this patient population. Nevanimibe was administered at gradually increasing doses of 125, 250, 500, 750 and 1000 mg BID alternating with matching placebo in a two-weeks-on, two-weeks-off manner.
In the trial, we observed nevanimibe to be associated with clear signs of clinical activity in seven of 10 treated patients, as measured by reductions in 17-hydroxyprogesterone (17-OHP), a key measure of disease control. Two patients experienced a reduction in 17-OHP levels to ≤2 times the upper limit of normal, the primary endpoint of the trial. 70% of subjects experienced a decrease in 17-OHP of at least 50% during at least one nevanimibe treatment period. We also observed nevanimibe to have a rapid onset of action. Nevanimibe was reported to be well tolerated at all dose levels.
We initiated a Phase 2b clinical trial of nevanimibe for the treatment of CAH in the third quarter of 2018; this trial is active with enrollment currently paused. For additional information, please visit the Our Patients, of our website or www.clinicaltrials.gov and reference identifier number NCT03669549.