Hydrocortisone

New data from CATALYST and MOMENTUM trials presented at American Diabetes Association’s (ADA) 86 th Scientific Sessions In the CATALYST trial, patients with hypercortisolism treated with Korlym exhibited clinically and statistically significant improvements in hemoglobin A1c (HbA1c) and clinically meaningful reductions in body weight, body mass index and waist circumference, with numerically greater benefit in those taking GLP-1 receptor agonists or tirzepatide Late-breaking data from the MOMENTUM trial confirm high prevalence of endogenous hypercortisolism in patients with type 2 diabetes and resistant hypertension REDWOOD CITY, Calif.--(BUSINESS WIRE)--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, today announced the presentation of new data from its CATALYST and MOMENTUM trials at the American Diabetes Association’s 86 th Scientific Sessions. The presentations underscore the critical role of hypercortisolism in difficult-to-control type 2 diabetes and resistant hypertension, and the potential of cortisol modulation treatment. The CATALYST trial screened 1,057 patients with difficult-to-control type 2 diabetes (HbA1c of 7.5 – 11.5 percent despite receiving multiple glucose-lowering medications) and found that 24 percent had hypercortisolism (cortisol levels of greater than 1.8 μg/dL in the 1 mg dexamethasone suppression test (DST)). In CATALYST’s treatment phase, 136 of the patients who had been found to have hypercortisolism were randomized 2:1 to receive either Korlym or placebo for 24 weeks. Patients who received Korlym exhibited a clinically meaningful and statistically significant reduction in HbA1c (1.3 percent, p-value: 10 percent) were hypokalemia, fatigue, nausea, vomiting, headache, peripheral edema, diarrhea and dizziness. The data presented at ADA described treatment outcomes in the 71 patients in CATALYST who were taking GLP-1 receptor agonists or the GLP-1/GIP agonist tirzepatide. In this group, patients who received Korlym exhibited numerically greater benefit than the overall study population with reductions in HbA1c (1.7 percent), body weight (6.1 kg), body mass index (2.0 kg/m 2 ) and waist circumference (6.5 cm), compared to patients who received placebo (all nominal p-values less than 0.04). “These new CATALYST data demonstrate the potential of cortisol modulation to improve critical metabolic parameters, even for patients who have poorly controlled type 2 diabetes despite treatment with powerful GLP-1 or GLP-1/GIP receptor agonists, such as semaglutide or tirzepatide. Excess cortisol disrupts the incretin system, impairs beta cell function, and induces insulin and incretin resistance, potentially limiting the effectiveness of these otherwise potent therapies. Screening for hypercortisolism and considering cortisol-directed treatment is a key part of managing type 2 diabetes in patients not responding to standard-of-care treatments,” said Lance Sloan, M.D., President, Texas Institute for Kidney and Endocrine Disorders. At ADA, Corcept also presented late-breaking data from its MOMENTUM trial, which screened 1,086 patients with resistant hypertension (as defined by the American Heart Association’s criteria ) and found that 27.3 percent of them had hypercortisolism . The prevalence of hypercortisolism was even higher in patients who had hemoglobin A1c (HbA1c) of 7.5 percent or higher and were taking 3 or more blood pressure medicines: 36.6 percent in CATALYST and 32.6 percent in MOMENTUM. “Data consistently show that hypercortisolism is an underlying driver of treatment resistant cardiometabolic disease, including in patients receiving best-in-class therapies like GLP-1s,” said Bill Guyer, PharmD, Corcept’s Chief Development Officer. “Data from CATALYST demonstrate that treatment with a cortisol modulator, such as Korlym, can be synergistic with GLP-1s or tirzepatide to help patients better control type 2 diabetes. It is our hope that this new research will lead to increased screening for hypercortisolism and improved treatment.” About CATALYST CATALYST is the largest and most rigorous trial ever conducted to determine the prevalence of hypercortisolism in patients with difficult-to-control type 2 diabetes and assess the effect of treating patients found to have hypercortisolism with a cortisol modulator. The initial prevalence phase of the trial screened 1,057 patients with difficult-to-control type 2 diabetes (i.e., patients with HbA1c greater than 7.5 percent despite receiving multiple glucose-lowering medications, including best-in-class therapies such as GLP-1 and GLP-1/GIP agonists) at 36 sites in the United States. Based on results from a standard 1-mg dexamethasone suppression test, 24 percent of the patients screened were found to have hypercortisolism and were eligible to enter the trial’s treatment phase, where they were randomized, 2:1, to receive either Korlym or placebo for 24 weeks. One hundred thirty-six patients enrolled in the treatment phase. About MOMENTUM MOMENTUM was the largest trial ever conducted to assess the prevalence of hypercortisolism in patients with resistant hypertension. A total of 1,086 patients were screened at 50 sites in the United States. All patients had resistant hypertension as defined by the American Heart Association’s criteria (systolic blood pressure greater or equal to 130 mmHg despite taking 3 or more blood-pressure lowering medications, including a diuretic, or taking 4 or more blood-pressure lowering medications). Using a simple, standardized 1-mg dexamethasone suppression test (DST), 27 percent of these patients were found to have hypercortisolism. About Hypercortisolism Hypercortisolism, also known as Cushing’s syndrome, 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 fatal if not treated effectively. Cardiovascular events are the most common cause of death among patients with hypercortisolism. Recent research shows that endogenous hypercortisolism is more prevalent than previously believed. INDICATION KORLYM (mifepristone) is a cortisol receptor blocker indicated to control hyperglycemia secondary to hypercortisolism in adult patients with endogenous Cushing's syndrome who have type 2 diabetes mellitus or glucose intolerance and have failed surgery or are not candidates for surgery. Limitations of use: KORLYM should not be used in the treatment of patients with type 2 diabetes unless it is secondary to Cushing’s syndrome. IMPORTANT SAFETY INFORMATION BOXED WARNING: TERMINATION OF PREGNANCY Mifepristone is a potent antagonist of progesterone and cortisol via the progesterone and glucocorticoid (GR-II) receptors, respectively. The antiprogestational effects will result in the termination of pregnancy. Pregnancy must therefore be excluded before the initiation of treatment with Korlym and prevented during treatment and for one month after stopping treatment by the use of a nonhormonal medically acceptable method of contraception unless the patient has had a surgical sterilization, in which case no additional contraception is needed. Pregnancy must also be excluded if treatment is interrupted for more than 14 days in females of reproductive potential. CONTRAINDICATIONS Pregnancy; patients taking simvastatin or lovastatin and CYP3A substrates with narrow therapeutic ranges; patients receiving systemic corticosteroids for lifesaving purposes; women with a history of unexplained vaginal bleeding or endometrial hyperplasia with atypia or endometrial carcinoma; patients with known hypersensitivity to mifepristone or to any of the product components. WARNINGS AND PRECAUTIONS Adrenal insufficiency: Patients should be closely monitored for signs and symptoms of adrenal insufficiency. See full Prescribing Information for further management instructions. Hypokalemia: Hypokalemia should be corrected prior to treatment and monitored for during treatment. Measure serum potassium 1-2 weeks after starting or increasing the Korlym dose, and periodically thereafter. Mifepristone-induced hypokalemia should be treated with potassium supplementation based on severity. If hypokalemia persists despite potassium supplementation, consider adding mineralocorticoid antagonists. Vaginal bleeding and endometrial changes: Women may experience endometrial thickening or unexpected vaginal bleeding. Use with caution if the patient also has a hemorrhagic disorder or is on anticoagulant therapy. Women who experience vaginal bleeding during treatment should be referred to a gynecologist for further evaluation. QT interval prolongation: Avoid use with QT interval-prolonging drugs, or in patients with potassium channel variants resulting in a long QT interval. Exacerbation/Deterioration of Conditions Treated with Corticosteroids: For medical conditions in which chronic corticosteroid therapy is lifesaving, Korlym is contraindicated. Use of strong CYP3A inhibitors: Concomitant use increases mifepristone plasma levels. Use only when necessary and do not exceed a Korlym dose of 900 mg per day. The labeling contains warnings and precautions for Pneumocystis jiroveci Infection and potential effects of hypercortisolemia . See full Prescribing Information for further management instructions. ADVERSE REACTIONS Most common adverse reactions in Cushing’s syndrome (≥20%): nausea, fatigue, headache, decreased blood potassium, arthralgia, vomiting, peripheral edema, hypertension, dizziness, decreased appetite, and endometrial hypertrophy. DRUG INTERACTIONS Drugs metabolized by CYP3A: Administer drugs that are metabolized by CYP3A at the lowest dose when used with Korlym. CYP3A inhibitors: Caution should be used when Korlym is used with strong CYP3A inhibitors. Use only when necessary, and do not exceed a Korlym dose of 900 mg. CYP3A inducers: Do not use Korlym with CYP3A inducers. Drugs metabolized by CYP2C8/2C9: Use the lowest dose of CYP2C8/2C9 substrates when used with Korlym. Drugs metabolized by CYP2B6: Use of Korlym should be done with caution with bupropion and efavirenz. Hormonal contraceptives: Do not use with Korlym. DOSAGE AND ADMINISTRATION Obtain a negative pregnancy test prior to initiating treatment with Korlym in females of reproductive potential, or if treatment is interrupted for more than 14 days. Administer once daily orally with a meal. The recommended starting dose is 300 mg once daily. Renal impairment: Do not exceed 600 mg once daily. Mild-to-moderate hepatic impairment: Do not exceed 600 mg once daily. Do not use in severe hepatic impairment. Based on clinical response and tolerability, the dose may be increased in 300-mg increments to a maximum of 1200 mg once daily. Do not exceed 20 mg/kg per day. Concomitant use of Korlym with a strong CYP3A inhibitor resulted in a 38% increase in mean plasma concentration of mifepristone. For patients already being treated with a strong CYP3A inhibitor, start with a Korlym dose of 300 mg per day and titrate to a maximum of 900 mg per day if clinically indicated. When a strong CYP3A inhibitor is administered to patients already receiving Korlym, adjust the dose as follows: for patients receiving a daily dose of 600 mg, reduce dose to 300 mg. For patients receiving a daily dose of 900 mg, reduce dose to 600 mg. For patients receiving a daily dose of 1200 mg, reduce dose to 900 mg. Titrate if clinically indicated and do not exceed a Korlym dose of 900 mg in combination with a strong CYP3A inhibitor. USE IN SPECIFIC POPULATIONS Lactation: Mifepristone is present in human milk, however, there are no data on the amount of mifepristone in human milk, the effects on the breastfed infant, or the effects on milk production during long term use of mifepristone. About Corcept Therapeutics For over 25 years, Corcept has focused on cortisol modulation and its potential to treat patients with a wide variety of serious disorders, leading to the discovery of more than 1,000 proprietary selective cortisol modulators and glucocorticoid receptor antagonists. Corcept is conducting advanced clinical trials in patients with Cushing’s syndrome, solid tumors, ALS and liver disease. In 2012, the company introduced Korlym ® , the first medication approved by the U.S. Food and Drug Administration (FDA) for the treatment of patients with endogenous Cushing’s syndrome, and in 2026, the company introduced Lifyorli™, approved in combination with nab-paclitaxel, the first FDA-approved selective glucocorticoid receptor antagonist for adults with platinum-resistant ovarian cancer. Corcept is headquartered in Redwood City, California. For more information, visit Corcept.com . Forward-Looking Statements Statements in this press release, other than statements of historical fact, are forward-looking statements based on our current plans and expectations and are subject to risks and uncertainties that might cause our actual results to differ materially from any future results expressed or implied by such forward-looking statements. In this press release, forward-looking statements include statements concerning: the potential for cortisol modulation treatment to improve critical metabolic parameters, even for patients with poorly controlled type 2 diabetes despite treatment with powerful GLP-1 or GLP-1/GIP receptor agonists; and our hope that the findings from the CATALYST and MOMENTUM trials will lead to increased screening for hypercortisolism and improved treatment. A further description of risks and uncertainties can be found in our SEC filings, which are available at our website and the SEC’s website. These risks and uncertainties include, but are not limited to, those related to: our ability to operate our business; our efforts to study and develop Korlym, relacorilant, miricorilant, dazucorilant, nenocorilant and our other product candidates; those molecules’ clinical attributes; regulatory approvals, mandates, oversight and other requirements imposed on our products or our business by laws, regulations or discretion of government authorities; and the scope and protective power of our intellectual property. We disclaim any intention or duty to update forward-looking statements made in this press release. Contacts Investor inquiries: ir@corcept.com Media inquiries: communications@corcept.com

一、清单概况：18个品种、27个规格、9种剂型 2026年6月3日，国家卫生健康委公示《第六批鼓励研发申报儿童药品建议清单》。该清单由国家卫健委牵头，联合工业和信息化部、国家医保局、国家药监局共同制定，专家组围绕我国儿童疾病谱以及相关企业研发生产能力，基于循证原则，针对中国大陆境内尚未注册上市且临床急需的儿童用药现状进行筛选论证。 第六批清单共纳入18个品种，涉及27个规格、9种剂型，覆盖A型血友病、抗肿瘤、抗过敏、内分泌及急救等治疗领域。 二、与前五批对比：从"大而全"到"精而深" 自2016年以来，国家已制定并发布了五批含144种药品的鼓励研发申报儿童药品清单，第六批与前五批的演进脉络呈现出明显的变化趋势： 维度 前五批 第六批 品种数量 平均每批约29种 18种（聚焦策略更精准） 覆盖范围 全面铺开（140余种） 精选品种，聚焦临床刚需 产品形态 以仿制为主 创新药与改良药并重 治疗领域 感染、呼吸、抗肿瘤等传统领域 血友病生物制剂、过敏免疫治疗等新兴领域 第六批清单的显著特点是从"大而全"向"精而深"转型，不再追求品种数量的堆砌，而是基于临床需求精准筛选最具紧迫性的方向。清单全面覆盖罕见病、肿瘤及慢性病等关键治疗领域。 三、三大核心亮点 亮点一：填补国产创新药空白 第六批清单明确指向国产空白领域。专家解读指出，部分重要药物目前仅有进口原研药，尚无国产药品注册上市。最典型的方向就是PD-1抗体的国产化。 PD-1抗体药物（纳武利尤单抗、帕博利珠单抗等）在成人肿瘤免疫治疗中已是主流方案，但儿童肿瘤患者长期面临"无药可用"或"依赖进口"的困境。清单纳入这一方向，意味着国家层面正式启动对儿童肿瘤免疫治疗药物的自主攻关——对现有的热门肿瘤靶点，开发适合儿童的剂型、规格和给药方案，推动"成人好药"向"儿童专用"下沉。 此外，第六批清单中注射剂作为核心品类，全部聚焦临床急救、重症治疗领域的精细化规格升级，改造逻辑简单、临床刚需极强，为企业提供了明确的盈利路径。 亮点二：攻克儿童适宜剂型和规格 儿童用药最突出的痛点是"用药靠掰、剂量靠猜"。大量成人药品没有适合儿童的剂型和规格，临床医生只能凭经验推算剂量，易导致不良反应或疗效不确切，这种现象被称为"用法靠猜，用量靠掰"。 第六批清单着力解决这一难题，设计了多条"精算"规格线： 氢化可的松（急危重症抢救用药）新增0.5mg、1mg、2mg、5mg四个低剂量规格，精准匹配不同年龄段患儿的体重和病情需求； 低剂量肾上腺素（过敏性休克抢救用药）新增0.15mg规格，为低体重婴幼儿提供精准急救剂量； 氢化可的松颗粒剂首次以口服剂型登录清单，改变了该药此前以注射剂为主的给药方式，为慢性肾上腺皮质功能不全等需要长期用药的患儿提供了更多选择。 清单中9种剂型全面覆盖：混悬剂、颗粒剂、口服液、低剂量注射剂、吸入制剂等，充分兼顾不同年龄段、不同病情程度的儿童用药可行性。 亮点三：填补罕见病用药空白 广州妇儿医院药学部主任莫小兰特别强调，"第六批清单持续填补罕见病用药空白"。 清单纳入艾美赛珠单抗——一款治疗A型血友病（俗称"皇家病"）的生物制剂，对于凝血功能异常的A型血友病患者具有较好的止血效果。 血友病是罕见病中相对多发的病种。中国血友病患者约10万人，其中儿童约占三分之一。该药物采用皮下注射方式，可显著减轻患儿频繁静脉输注的痛苦。清单的发布，意味着国家将从审评、医保、科技专项等多维度引导本土企业尽快落地国产化生产，降低患者用药成本。 此外，清单持续关注罕见病领域其他方向，为更多罕见病患儿带来用药希望。 四、重点药物解析 1. 抗肿瘤及免疫领域 纳武利尤单抗注射液、帕博利珠单抗注射液：PD-1类免疫检查点抑制剂，在儿童实体瘤治疗中具有广阔应用前景； 奥马珠单抗：重组人源化抗IgE单克隆抗体，用于儿童中重度过敏性哮喘和慢性特发性荨麻疹； 依那西普：TNF抑制剂，用于治疗幼年特发性关节炎等儿童自身免疫性疾病。 2. 血液与罕见病领域 艾美赛珠单抗：A型血友病治疗药物，罕见病领域重点攻关品种，皮下注射方式为患儿带来便利； 依库珠单抗：治疗阵发性睡眠性血红蛋白尿症和非典型溶血性尿毒症综合征的重磅生物药； 花生过敏原散剂（口服免疫疗法）：针对日益突出的儿童严重食物过敏问题，清单首次纳入口服脱敏治疗产品。 3. 急救与内分泌领域 肾上腺素注射液（0.15mg）：过敏反应急救的核心药物，新增低剂量规格； 氢化可的松颗粒剂（0.5mg、1mg、2mg、5mg）：肾上腺皮质功能不全等激素缺乏症的基础用药，首次以口服剂型纳入清单； 地塞米松棕榈酸酯注射液：肾上腺皮质激素类药物，补充急重症治疗所需规格。 4. 感染与代谢领域 美罗培南（注射用）：碳青霉烯类抗生素，针对儿童严重细菌感染的救命药； 左乙拉西坦（口服溶液剂）：抗癫痫药物，以儿童友好剂型补充清单； 吡非尼酮：抗肺纤维化药物，补充儿童间质性肺疾病用药目录。 以上均为核心聚焦品种。清单明确要求相关企业加快研发和申报进度，国家层面将从优先审评审批、医保准入、科技专项支持等多维度提供全链条保障。 五、前五批回顾：十年蓄势，从点上突破走向系统发力 2016年，国家卫健委等六部门首次发布《第一批鼓励研发申报儿童药品清单》，标志着我国儿童用药保障体系正式启动制度化建设。此后，每批清单均有独特定位： 第一批（2016年）：32个品种。以填补基础性临床空白为主，侧重基础品种，开启儿童用药系统化规范的第一步。 第二批（2017年）：39个品种。品种数量达到峰值，扩大病种覆盖面，为研究机构和企业提供更丰富的研发指引。 第三批（2019年）：34个品种。精细化细分治疗领域，重点解决部分临床急需品种。 第四批（2023年）：24个品种。清单规模回落，聚焦更精准的临床需求，为后续批次树立精简化模板。 第五批（2024年）：15个品种。领域覆盖全身用抗感染药、呼吸系统用药、抗肿瘤药及免疫调节剂等。 第六批（2026年）：18个品种。生物制剂与创新药成主角，标志着儿童用药保障从"仿制为主"迈向"创新引领"。 五批合计共144种儿童治疗药品，覆盖恶性肿瘤、感染、癫痫及罕见病等多领域。 六、政策背景：八部门新政——《关于改革完善儿童用药供应保障机制的实施意见》 第六批清单的出台，与2026年5月7日八部门联合发布的 《关于改革完善儿童用药供应保障机制的实施意见》 紧密衔接，形成从顶层设计到具体品种的完整政策链条。 我国儿童用药面临的四大核心问题 国家卫健委药物政策与基本药物制度司负责人解读指出，儿童用药仍存在以下亟待解决的问题： 品种少、剂型缺：儿童用药数量少，儿童适宜的剂型和规格缺乏，结构失衡； 说明书信息不全：上市药品说明书中仍存在儿童用药信息缺乏、不规范等问题； 企业积极性不足：未有效激发企业研发生产儿童用药的积极性，临床必需但用量较少的部分儿童用药保障供应能力仍显不足； 药学服务供给不足：部分医疗机构配备儿童用药种类较少。 八部门新政的核心举措 针对上述问题，《实施意见》从多个维度构建系统性解决方案，涉及从研发、审评、生产到使用的全链条： 一是破解儿童用药研发难题。 对儿童专用创新药早期介入、研审联动，允许滚动提交资料；对纳入清单的药品予以优先审评审批，优先纳入国家基本药物目录和创新药物研发国家科技重大专项，按程序纳入国家医保药品目录。 二是丰富儿童用药临床研发模式。 鼓励支持国家医学中心、国家区域医疗中心和国家临床医学研究中心等儿科研究型病房建设；探索建立全国儿童临床试验协作网；推动将已有中国成人数据的药品安全外推至中国儿科人群。 三是完善儿童用药临床应用。 牵头制定儿童用药临床应用指导原则，修订中国国家处方集（儿童版），探索适时制定国家儿童基本药物目录。2026版《国家基本药物目录管理办法》首次将"儿童药品"作为独立的遴选模块，并明确"剂型适宜"。 四是规范医疗机构儿科制剂使用。 对临床确有需要，而市场上没有供应或没有供儿童使用剂型、规格的儿童用药制定儿童常用医疗机构制剂清单。 五是加强短缺药品监测和保障供应。 支持小品种药（短缺药）集中生产基地的定点生产品种纳入更多儿童用药，丰富中央和地方两级储备，季节性传染病流行高发期间加强儿童常用药品供应保障。 六是健全支付管理体系。 推进医保支付方式改革，动态调整病种分组方案，在确定分组、系数等要素时适当向儿童倾斜；支持商业健康保险开发儿童保险保障产品，鼓励将创新药、罕见病用药纳入保障范围；在国家组织药品集中带量采购中，分组采购儿童专用药与成人用药；优化差比价规则，激励儿童适宜剂型、规格的供应。 七是创新儿童中医药研发机制。 开展符合儿童生理、病理特征的中药研究；开发儿科古代经典名方；筛选制定儿科中医优势病种清单，加速中药新药转化；加快规范完善中成药说明书中儿童相关信息。 另外，2026年医保目录调整首次引入"预申报"机制，已纳入商保创新药目录的药物可直接申报基本目录，并与第六批清单形成制度合力，为清单内的儿童创新药开辟了"先商保、后医保"的准入通道。 七、面临的挑战：从清单到患者手中，仍需打通三道关卡 清单发布只是第一步，真正让好药惠及儿童，仍需克服多重挑战： 1. 研发到上市的周期瓶颈： 即使纳入清单享受优先审评，一款创新药从研发到获批上市仍需3—5年甚至更长。而国内企业重成人、轻儿童的市场惯性仍强，清单的引导效应需持续释放。 2. 支付端的不确定风险： 部分高值生物制剂（如单克隆抗体药物）即便成功研发上市，若无医保或商保覆盖，高昂价格将严重影响可及性。2026年医保目录首次"预申报"机制和商保创新药目录的开通，能否真正将清单品种快速纳入保障体系，仍需观察。 3. 基层供应"最后一公里"： 即使药品获批上市，在基层医疗机构如何保障供应和规范使用，仍是长期课题。特别是罕见病药物、急救药物等用量较少的品种，县乡级医院的配备率有待提升。 八、展望与前景 2026年是儿童用药政策的密集兑现期。第六批清单的发布，叠加八部门新政、2026年医保目录动态调整规则、专属儿童基药目录筹备落地等多重政策加持，标志着国内儿童用药产业正式告别粗放仿制模式，全面进入精细化迭代、全链条政策扶持的高质量发展新阶段。 从产业链视角看，清单的发布为药企布局儿童细分赛道划定了明确的产品方向和盈利路径——未来儿童用药的市场竞争，将从"谁能快速仿制"转向"谁能解决真正的临床痛点和剂型适配问题"。 从社会价值视角看，随着清单的持续推进和落地，我国儿童用药"用药靠掰、剂量靠猜"的困局将逐步缓解，更多患儿将用上安全、有效、可及的专属药品。通过从研发、审评、生产到支付的全链条政策体系完善，中国儿童用药保障正加速从"被动应对"走向"系统治理"，为"健康中国2030"战略目标的实现提供重要支撑。