Nevanimibe is an investigational drug being studied for the treatment of the orphan adrenal disease classic congenital adrenal hyperplasia (CAH).
Nevanimibe in Congenital Adrenal Hyperplasia (CAH):
Based on an interim review of the nevanimibe open-label Phase 2b study in CAH no further investment in the program is currently planned.
Results from 10 subjects, nine from cohort 1 and one from cohort 2, with at least 12 weeks of treatment with nevanimibe in this open-label, continuous dose escalation study showed that one patient (10%) met the primary endpoint of achieving 17-hydroxyprogesterone (17-OHP) levels less than or equal to 2-times the upper limit of normal (ULN). Treatment under the amended protocol with dose titration starting at 500 mg BID improved tolerability of nevanimibe.