AS-2473662
- Generic Name
- AS-2473662
Corcept Therapeutics Submits New Drug Application for Relacorilant to Treat Cushing's Syndrome
• Corcept Therapeutics has submitted a New Drug Application to the FDA for relacorilant, a selective cortisol modulator designed to treat patients with endogenous hypercortisolism (Cushing's syndrome).
• The application is supported by positive results from multiple clinical trials, including the pivotal GRACE trial, Phase 3 GRADIENT study, and long-term extension studies, demonstrating improvements in various symptoms with an acceptable safety profile.
• Relacorilant showed no instances of serious adverse events common in current treatments, such as drug-induced adrenal insufficiency, hypokalemia, or QT prolongation, positioning it as a potential new standard of care.
Clofutriben Shows Promise in Phase 2 Trial for Endogenous Cushing's Syndrome
• Clofutriben, an oral small molecule from Sparrow Pharmaceuticals, demonstrated promising results in a Phase 2 trial for endogenous Cushing's syndrome, with all patients choosing to continue treatment in an open-label extension.
• Interim results from the RESCUE trial showed clofutriben normalized free cortisol levels in the urine of adults with Cushing's syndrome caused by tumors outside the adrenal glands.
• The FDA granted clofutriben orphan drug designation, providing incentives such as tax credits and potential market exclusivity, to support its clinical development for this rare disease.
• Clofutriben inhibits HSD-1, cutting off cortisol production within cells, potentially avoiding adrenal insufficiency risks associated with current Cushing's syndrome therapies.
Sparrow Pharmaceuticals' Clofutriben Completes Phase 2 Trial and Receives FDA Orphan Drug Designation for Cushing's Syndrome
• Sparrow Pharmaceuticals has completed its Phase 2 RESCUE trial of clofutriben for ACTH-dependent endogenous Cushing’s syndrome, with all eligible patients continuing treatment in an open-label extension.
• Clofutriben, a selective HSD-1 inhibitor, offers a novel approach by lowering intracellular cortisol in key tissues, potentially mitigating the safety and tolerability issues of current therapies.
• The FDA has granted Orphan Drug Designation to clofutriben for endogenous Cushing’s syndrome, providing incentives such as tax credits and potential market exclusivity.
• Planning for the next phase of clinical trials is underway, with data presentation and trial designs being developed in collaboration with medical, scientific, and patient advisors.
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