Relacorilant

▎药明康德内容团队编辑 Corcept Therapeutics公司日前宣布，美国FDA已接受该公司为选择性皮质醇调节剂relacorilant递交的新药申请（NDA），用于治疗内源性高皮质醇症，又名库欣综合征（Cushing’s syndrome）。FDA预计在今年12月30日之前完成审评。新闻稿指出，relacorilant有潜力成为库欣综合征的标准治疗。 这项NDA是基于关键性临床试验GRACE的积极结果，来自3期临床试验GRADIENT及其长期扩展试验的确认性证据，以及针对高皮质醇症的2期临床试验数据。日前发布的GRADIENT试验结果显示，有高血压的患者接受relacorilant治疗后，在22周时其平均收缩压较基线下降6.6 mm Hg（p=0.012），达到具有统计学显著性和临床意义的改善，而接受安慰剂的患者仅下降2.1 mm Hg。 此外，GRADIENT试验中接受relacorilant治疗的患者在22周时其体重出现了具有临床意义和统计学显著性的改善，安慰剂调整后体重下降3.9公斤（p=0.0001），同时内脏脂肪质量和体积也分别获得改善，均优于接受安慰剂治疗的患者。 ▲Relacorilant分子结构式（图片来源：PubChem） Relacorilant为一种选择性皮质醇调节剂，可与糖皮质激素受体结合，但不会与体内其他激素受体发生作用。除了治疗内源性高皮质醇症，Corcept还在研究relacorilant在卵巢癌和前列腺癌等多种严重疾病中的应用。Relacorilant已获得美国FDA和欧洲药品管理局授予孤儿药资格，用于治疗库欣综合征。 高皮质醇症是由于激素皮质醇过度活跃引起的。症状多样，但大多数患者会出现以下一种或多种表现：高血压、中心性肥胖、血糖升高和难以控制的2型糖尿病、严重疲劳以及肌肉无力。易怒、焦虑、抑郁和认知障碍也较为常见。高皮质醇症可影响所有器官系统，如未能有效治疗，可能会危及生命。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放 参考资料： [1] FDA Files Corcept’s New Drug Application for Relacorilant as Treatment for Patients With Hypercortisolism. Retrieved March 4, 2025, from https://www.businesswire.com/news/home/20250303227690/en/FDA-Files-Corcept%E2%80%99s-New-Drug-Application-for-Relacorilant-as-Treatment-for-Patients-With-Hypercortisolism [2] Corcept Therapeutics Announces Third Quarter Financial Results, Positive Results From Phase 3 GRADIENT Trial in Patients With Cushing’s Syndrome and Provides Corporate Update. Retrieved March 4, 2025, from https://ir.corcept.com/news-releases/news-release-details/corcept-therapeutics-announces-third-quarter-financial-results-3 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，聚焦全球生物医药健康创新

REDWOOD CITY, CA, USA I December 30, 2024 I Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of medications to treat severe endocrinologic, oncologic, metabolic and neurologic disorders by modulating the effects of the hormone cortisol, has submitted a new drug application (NDA) to the U.S. Food and Drug Administration for its proprietary, selective cortisol modulator, relacorilant, to treat patients with endogenous hypercortisolism (Cushing’s syndrome). Corcept’s NDA is based on positive results from the pivotal GRACE trial and confirmatory evidence from the Phase 3 GRADIENT and long-term extension studies and a Phase 2 study in hypercortisolism. Patients in these studies who received relacorilant experienced improvements in a wide array of hypercortisolism’s signs and symptoms, with an acceptable safety burden. Notably, there were no instances of drug-induced adrenal insufficiency, hypokalemia or QT prolongation – serious adverse events that can arise in patients taking currently approved medications – and no adverse events associated with activity at the progesterone receptor, such as endometrial thickening or vaginal bleeding. “Relacorilant’s combination of efficacy and safety give it the potential to become the standard of care for the medical treatment of patients with hypercortisolism,” said Joseph Belanoff, MD, Corcept’s Chief Executive Officer. “Our commitment to the health of patients with hypercortisolism is unwavering. We are optimistic that relacorilant will be of great benefit to them.” About Relacorilant Relacorilant is a selective cortisol modulator that binds to the glucocorticoid receptor but not to the body’s other hormone receptors. Corcept is studying relacorilant in a variety of serious disorders in addition to endogenous hypercortisolism (Cushing’s syndrome), including ovarian, adrenal and prostate cancer. Relacorilant is proprietary to Corcept and is protected by composition of matter, method of use and other patents. Relacorilant has orphan drug designation in the United States and the European Union for the treatment of Cushing’s syndrome. About Hypercortisolism (Cushing’s Syndrome) Hypercortisolism is caused by excessive activity of the hormone cortisol. Symptoms vary, but most patients experience one or more of the following manifestations: hypertension, central obesity, elevated blood sugar and difficult-to-control type 2 diabetes, severe fatigue and weak muscles. Irritability, anxiety, depression and cognitive disturbances are common. Hypercortisolism can affect every organ system and can be lethal if not treated effectively. About Corcept Therapeutics For over 25 years, Corcept’s focus on cortisol modulation and its potential to treat patients with a wide variety of serious disorders has led to the discovery of more than 1,000 proprietary selective cortisol modulators. Corcept is conducting advanced clinical trials in patients with hypercortisolism, solid tumors, ALS and liver disease. In February 2012, the company introduced Korlym ® , the first medication approved by the U.S. Food and Drug Administration for the treatment of patients with endogenous hypercortisolism. Corcept is headquartered in Redwood City, California. For more information, visit Corcept.com. SOURCE: Corcept Therapeutics