European Medicines Agency Recommends Orphan Drug Designation for Relacorilant to Treat Patients with Cushing’s Syndrome

May 2, 2019 at 5:05 PM EDT

MENLO PARK, Calif., May 02, 2019 (GLOBE NEWSWIRE) -- Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of drugs to treat severe metabolic, oncologic and psychiatric disorders by modulating the effects of the stress hormone cortisol, today announced that the European Medicines Agency Committee for Orphan Medicinal Products (COMP) has issued a positive opinion recommending the approval of orphan drug designation for Corcept’s proprietary, selective cortisol modulator, relacorilant, for the treatment of Cushing’s syndrome. The European Commission is expected to adopt the COMP’s recommendation in May 2019.

Orphan drug designation in the European Union (EU) provides regulatory and financial incentives for companies to develop and market therapies to treat serious disorders affecting no more than five in 10,000 persons in the EU, including ten-year marketing exclusivity in the EU upon approval, eligibility for protocol assistance, reduced fees and access to the EU’s centralized marketing authorization procedure.

To be considered for orphan designation in the EU, there must be plausible evidence of a drug candidate’s efficacy and of its potential to confer significant clinical benefit compared to already-approved treatments. The COMP letter states, “The sponsor has provided clinical data that demonstrate that the product can reduce blood pressure and improve control of hyperglycaemia in patients who were not adequately managed by currently authorised products. The Committee considered that this constitutes a clinically relevant advantage.” The medications ketoconazole, metyropone and pasireotide are approved in the EU for the treatment of one or more aetologies of Cushing’s syndrome.

Corcept based its submission on data from relacorilant’s Phase 2 trial, in which patients demonstrated improvements in the primary endpoints of glucose control and hypertension, and in a variety of secondary endpoints, including weight loss, liver function, coagulopathy, cognition, mood, insulin resistance, and quality of life. Relacorilant was well-tolerated, with the most common treatment-emergent adverse events being those associated with reduction in excess cortisol activity – headache, nausea, back pain and edema – that usually resolve with continued treatment.

Because relacorilant, unlike Corcept’s FDA-approved cortisol modulator, Korlym^®, does not bind to the progesterone receptor, patients did not experience vaginal bleeding caused by uterine thickening, a frequently-reported adverse event of Korlym. There were also no cases of drug-induced hypokalemia (low potassium), a leading cause of Korlym discontinuation.

Relacorilant’s Phase 3 GRACE trial is underway and is expected to enroll 130 patients at 60 sites in the United States, Canada, Europe and Israel.

The United States Food and Drug Administration granted relacorilant orphan drug status for Cushing’s syndrome in October 2018.

Cushing’s Syndrome

Hypercortisolism, often referred to as Cushing’s syndrome, is caused by excessive activity of the hormone cortisol. Endogenous Cushing’s syndrome is an orphan disease that most often affects adults aged 20-50. In the United States, an estimated 20,000 patients have Cushing’s syndrome, with about 3,000 new patients being diagnosed each year. Symptoms vary, but most people experience one or more of the following manifestations: high blood sugar, diabetes, high blood pressure, upper-body obesity, rounded face, increased fat around the neck, thinning arms and legs, severe fatigue and weak muscles. Irritability, anxiety, cognitive disturbances and depression are also common. Hypercortisolism can affect every organ system in the body and can be lethal if not treated effectively.

Corcept Therapeutics Incorporated

Corcept is a commercial-stage company engaged in the discovery and development of drugs that treat severe metabolic, oncologic and psychiatric disorders by modulating the effects of the stress hormone cortisol. Corcept’s approved product, Korlym^®, was the first FDA-approved treatment for patients with Cushing’s syndrome. Korlym modulates the activity of cortisol at the glucocorticoid receptor, one of the two receptors to which cortisol binds. Corcept has discovered a large portfolio of proprietary compounds that selectively modulate the effects of cortisol but not progesterone. Corcept owns extensive United States and foreign intellectual property covering the composition of its selective cortisol modulators and in the use of cortisol modulators, including Korlym, to treat a wide variety of serious disorders.

Forward-Looking Statements

Statements in this press release, other than statements of historical fact, are forward-looking statements, which are based on our current plans and expectations and are subject to risks and uncertainties that might cause actual results to differ materially from those such statements express or imply. These risks and uncertainties include, but are not limited to, our ability to generate sufficient revenue to fund our commercial operations and development programs; the availability of competing treatments, including generic versions of Korlym; our ability to obtain acceptable prices or adequate insurance coverage and reimbursement for Korlym; and risks related to the development of our product candidates, including regulatory approvals, mandates, oversight and other requirements. These and other risks are set forth in our U.S. Securities and Exchange Commission (SEC) filings, which are available at our website and the SEC’s website. In this press release, forward-looking statements include those concerning expectations that the European Commission will adopt the COMP’s recommendation in May 2019; the regulatory and financial incentives accompanying orphan drug designation; the progress, timing, design and results of our development programs, including the GRACE trial; 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.

CONTACT:
Christopher S. James, MD
Director, Investor Relations
Corcept Therapeutics
650-684-8725
cjames@corcept.com
www.corcept.com

Source: Corcept Therapeutics Incorporated

Release Details

European Medicines Agency Recommends Orphan Drug Designation for Relacorilant to Treat Patients with Cushing’s Syndrome