Detailed Narrative
FDA Complete Response Letter for Relacorilant in Cushing's Syndrome
Corcept Therapeutics received an unexpected Complete Response Letter (CRL) from the FDA for relacorilant as a treatment for Cushing's syndrome. Management expressed surprise, citing that the pivotal GRACE trial met its primary endpoint (p=0.02) and was supported by confirmatory evidence from the GRADIENT trial. They also highlighted relacorilant's benefits in hypertension and other Cushing's symptoms, along with a favorable safety profile compared to existing treatments. The company plans to meet with the FDA in April to understand the agency's thinking, with potential outcomes ranging from NDA resubmission to a formal appeal or conducting a new study.
Korlym Commercial Performance and Pharmacy Transition
The Cushing's Syndrome business experienced a surge in demand in 2025, with a record number of new prescriptions and first-time prescribers. However, the company faced challenges with its previous pharmacy vendor's capacity, leading to a gap between a 61% increase in new prescriptions and a 37% increase in tablets sold. A transition to a new specialty pharmacy, Curant, began in October and was completed in February. Management is confident in the new pharmacy's capabilities, citing improved performance metrics and expecting record new patient starts in February.
CATALYST and MOMENTUM Trial Impact on Cushing's Syndrome Business
The landmark CATALYST trial demonstrated that 24% of patients with uncontrolled diabetes (even on GLP-1s) had hypercortisolism, and treatment with Korlym led to significant improvements in HbA1c (1.47% mean decrease vs 0.17% for placebo), weight, and waist circumference. These findings are transforming medical practice and are expected to drive increased screening and treatment for Cushing's syndrome. The MOMENTUM trial, examining hypercortisolism prevalence in resistant hypertension, is fully enrolled, with results expected next month, potentially providing similar paradigm-shifting insights.
ROSELLA Phase III Data and Oncology Strategy
The Phase III ROSELLA trial for relacorilant in platinum-resistant ovarian cancer met both dual primary endpoints, showing a 35% reduction in the risk of death (HR 0.65, p=0.0004) and a 4.1-month increase in median overall survival. The safety profile was comparable to nab-paclitaxel alone, with a reduction in ALT rises in the combination arm. Full results will be presented at the SGO meeting in April. Management believes relacorilant could become a new standard of care, with a PDUFA date set for July 11. The company is also evaluating relacorilant in other solid tumors and in combination with other anticancer therapies, with results expected by the end of 2027.
Broader Pipeline Progress in MASH and ALS
Corcept is advancing its pipeline beyond Cushing's syndrome and ovarian cancer. The Phase IIb MONARCH study for relacorilant in Metabolic Dysfunction-Associated Steatohepatitis (MASH) is fully enrolled, with results anticipated by year-end. For ALS, the DAZALS trial with dazucorilant showed an 84% reduction in the risk of death. A dose titration study is underway to improve GI tolerability, with a pivotal trial planned to start by midyear. The company continues to discover and develop new cortisol modulators for various indications.