Corcept Therapeutics Hormone Drug Meets Main Trial Goal (CORT)

Corcept Therapeutics Incorporated CORT is up over 60% today after announcing positive top-line results from its Phase 3 study of CORLUX for the treatment of Cushing's Syndrome. The study evaluated the response of two patient groups to CORLUX treatment: one included patients who were glucose intolerant and one included patients who were hypertensive. Statistically significant improvement in the primary endpoint was achieved for both groups: with 60% responding in the glucose intolerant group and 43% in the hypertensive group. An initial review of safety data indicates that CORLUX was well tolerated by Cushing's Syndrome patients in this Phase 3 study. "The results of the study demonstrate that CORLUX has the potential to become an important treatment option for patients suffering from Cushing's Syndrome," said Joseph Belanoff, M.D., Chief Executive Officer of Corcept. "We remain on track to submit a New Drug Application (NDA) to the FDA for CORLUX in Cushing's Syndrome by the end of the first quarter of 2011 and continue to work toward our goal of making CORLUX available to patients with this severe disease." Primary Endpoints Met in Both Patient Groups Each group in the study had its own primary endpoint. The primary analysis is a responder analysis. In the "glucose intolerant group" (patients with diabetes or carbohydrate intolerance) a responder was defined as a patient who achieved a 25% or greater improvement in glucose tolerance as measured by a standard 2-hour glucose tolerance test at 24 weeks (or at the early termination visit) compared to baseline. In the "hypertension group" (patients with a diagnosis of hypertension) a responder was defined as a patient who achieved a 5 millimeter or greater improvement in diastolic blood pressure at 24 weeks (or at the early termination visit) compared to baseline. The protocol for the trial dictates that if a sufficient number of patients in either the glucose intolerant group or the hypertension group are responders, such that the lower limit of the exact one-sided 95% binomial confidence interval (CI) for the responder rate is greater than 20%, then the trial will have demonstrated efficacy for the treatment of Cushing's Syndrome. The calculation, which was predetermined in the study design, is based on analyzing the response rates in a modified intention to treat group (mITT) defined as those patients treated for at least 30 days (the mITT group). * 15 of 25, or 60%, of patients in the glucose intolerant group responded to treatment with CORLUX, significantly higher than the 20% hurdle rate (lower bound of the 95% CI = 41.7 which equates to p < 0.0001). * 9 of 21, or 43%, of patients in hypertension group responded to treatment with CORLUX, significantly higher than the 20% hurdle rate (lower bound of the 95% CI = 24.5 which equates to p < 0.01). CORLUX Was Well Tolerated In The Trial CORLUX was well tolerated in the trial population. Although the detailed analysis of the safety data from the study has not yet been completed, the tolerability of CORLUX in the treatment of Cushing's Syndrome in the Phase 3 study met our expectations. Adverse events related to treatment included symptoms of adrenal insufficiency, endometrial thickening, and hypokalemia, all of which were consistent with earlier published reports. The majority of the serious adverse events (SAEs) reported in the study were not related to CORLUX treatment, as determined by the clinical investigators. Of those that were related to treatment, all resolved with clinical management. We plan to present detailed safety data at scientific conferences during 2011. Ninety percent of the patients who completed the Phase 3 study opted to enter the long-term extension study. Shares of Corcept Therapeutics are up sharply in pre-market trading, currently 62% higher than yesterday's close at $6.79.