Miricorilant

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 it has resubmitted its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for relacorilant as a treatment for patients with Cushing’s syndrome. As requested by the FDA, the resubmission includes additional analyses of data included in the original NDA submission. Corcept expects the resubmission to receive a six-month review. “We are pleased to return relacorilant’s NDA to the review process and look forward to working with the Agency to advance relacorilant toward approval as a treatment for patients with Cushing’s syndrome,” said Joseph K. Belanoff, M.D., Corcept’s Chief Executive Officer. “Relacorilant has the potential to benefit many patients and it’s important we make it available as quickly as possible. We appreciate the FDA’s ongoing engagement with our application.” Corcept’s resubmission is based on positive data from its pivotal GRACE trial, the double-blind, placebo-controlled Phase III GRADIENT trial, relacorilant’s long-term extension study and earlier-stage development data. Corcept believes that these data show that relacorilant provides meaningful, durable improvements in the signs and symptoms of Cushing's syndrome, without causing some of the serious adverse events associated with currently approved medications – termination of pregnancy, hypokalemia, endometrial hypertrophy, vaginal bleeding, adrenal insufficiency and QT prolongation. About Hypercortisolism (Cushing’s Syndrome) 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. About Relacorilant Relacorilant, an oral therapy, is a selective glucocorticoid receptor (GR) antagonist that modulates cortisol activity by binding to the GR but not to the body's other hormone receptors. Relacorilant is proprietary to Corcept and is protected by composition of matter, method of use and other patents. It has been designated an orphan drug by the FDA and the European Commission (EC) for the treatment of hypercortisolism and by the EC for the treatment of ovarian cancer. Under the brand name Lifyorli™, relacorilant was approved in combination with nab-paclitaxel for adults with platinum-resistant ovarian cancer by the FDA in 2026. Corcept has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for relacorilant to treat patients with platinum-resistant ovarian cancer. 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: Corcept’s resubmission of its NDA to the FDA for relacorilant as a treatment for patients with Cushing’s syndrome; Corcept’s expectation that this resubmission will receive a six-month review; Corcept’s intent to work with the FDA to advance relacorilant toward approval as a treatment for patients with Cushing’s syndrome; relacorilant’s potential to benefit many patients; and Corcept’s belief regarding the importance of making relacorilant available as quickly as possible. 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

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