Breakthrough in Hypercortisolism: Relacorilant's Promising Phase 3 Results

Introduction

Corcept Therapeutics recently announced that relacorilant, a selective cortisol modulator, successfully met its primary endpoint in the second part of its phase 3 trial. This trial focused on patients with hypercortisolism, a condition commonly associated with Cushing’s syndrome.

The GRACE Trial Explained

The GRACE trial, a significant phase 3 clinical study, was divided into two parts. The first part was an open-label phase where 152 patients with Cushing’s syndrome, who also had hypertension, hyperglycemia, or both, were treated with relacorilant for 22 weeks.

The second part was a double-blind, randomized withdrawal phase. This phase assessed the effectiveness of relacorilant in maintaining blood pressure control among patients who previously responded well to the drug.

Open-Label Phase Results

In the initial phase, many patients experienced significant improvements. Specifically, 63% of patients with hypertension showed substantial reductions in both systolic and diastolic blood pressure. Those with hyperglycemia also benefited, with half of them meeting the study’s response criteria for improved glucose metabolism.

Randomized Withdrawal Phase Outcomes

During the withdrawal phase, patients who had previously responded to relacorilant were either continued on the drug or switched to a placebo. Remarkably, the likelihood of losing blood pressure control was 83% lower for those who continued with relacorilant compared to those on the placebo.

Safety and Tolerability

Safety analyses from both phases of the GRACE trial indicated that relacorilant was well-tolerated. No new safety concerns surfaced during the randomized withdrawal period, which is promising news for both physicians and patients.

Implications for Treatment

The encouraging results from the GRACE trial suggest that relacorilant could become a valuable treatment for patients with endogenous hypercortisolism. Patients experienced clinically significant improvements without a significant safety burden.

Next Steps

Corcept Therapeutics plans to submit its application for FDA approval in Q3 2024. This submission will be based on the positive results from both the open-label and randomized withdrawal phases of the GRACE trial.

Expert Opinions

Leading endocrinologists, including Rosario Pivonello, MD, PhD, have praised the trial's outcome. They emphasize that relacorilant offers a new, effective treatment option for managing hypercortisolism, especially in patients with concurrent hypertension and hyperglycemia.

Future Prospects

Corcept Therapeutics is committed to presenting detailed trial findings at upcoming medical conferences. The presentations aim to provide deeper insights into the trial results and further validate relacorilant’s efficacy and safety.

Conclusion

The positive outcomes from the GRACE trial mark a significant step forward in the treatment of hypercortisolism. Patients and healthcare providers alike have reason to be optimistic about the future availability of relacorilant as a therapeutic option.