cept Therapeutics rporated(CORT) - 2024 Q4 - Annual Report

Product Development and Clinical Trials - Korlym (mifepristone) has been marketed in the U.S. since 2012 for treating hypercortisolism, with an authorized generic version launched in June 2024[19]. - In the CATALYST study, 23.8% of 1,057 patients with difficult-to-control diabetes were found to have hypercortisolism, with Korlym showing a significant HbA1c reduction of 1.47% compared to 0.15% in the placebo group (p-value: < 0.0001)[25]. - Relacorilant, a proprietary cortisol modulator, was submitted for FDA approval in December 2024, based on positive results from multiple clinical trials[28]. - In the GRACE trial, patients receiving relacorilant showed a mean systolic blood pressure reduction of 7.9 mm Hg and diastolic blood pressure reduction of 5.4 mm Hg at 22 weeks (p-values: <0.0001)[30]. - GRADIENT study patients receiving relacorilant experienced a placebo-adjusted reduction in fasting glucose of 22.2 mg/dL (p-value: 0.002) and a reduction in body weight of 3.9 kg (p-value: 0.0001)[37][38]. - The pivotal Phase 3 trial "ROSELLA" for relacorilant combined with nab-paclitaxel has completed enrollment with 381 women randomized 1:1[43]. - In the Phase 2 trial, patients receiving 150 mg of relacorilant intermittently had a median progression-free survival (PFS) of 5.6 months compared to 3.8 months for nab-paclitaxel alone, with a hazard ratio of 0.66[46]. - The Phase 2 trial also showed that patients receiving relacorilant intermittently had a median overall survival (OS) of 13.9 months versus 12.2 months for nab-paclitaxel alone, with a hazard ratio of 0.67[46]. - The Phase 1b trial of relacorilant plus pembrolizumab in adrenal cancer patients showed significant symptom improvement but did not slow tumor progression[48]. - A Phase 2 trial of dazucorilant in ALS did not meet its primary endpoint, but the 300 mg dazucorilant arm had 0 deaths compared to 5 deaths in the placebo group[50]. - The Phase 2b trial "MONARCH" for miricorilant in patients with metabolic dysfunction-associated steatohepatitis (MASH) has a planned enrollment of 120 patients[51]. Regulatory and Compliance Issues - Regulatory approval from the FDA is required for all product candidates, involving significant time and expense for compliance with pre- and post-approval regulations[69][70]. - The FDA's review process for a New Drug Application (NDA) aims for a response within ten months for standard review and six months for priority review[74]. - The Orphan Drug Act provides exclusivity for seven years for drugs that receive FDA approval for rare diseases, along with tax credits and waivers for certain fees[75]. - The company is subject to various healthcare regulations, including anti-kickback and false claims laws, which could result in significant penalties for violations[82][84]. - The company is subject to the Physician Payments Sunshine Act, requiring reporting of payments and transfers of value to healthcare providers, with penalties for non-compliance[88]. - The company faces significant risks related to compliance with various healthcare regulations, which could lead to penalties and affect financial results[94]. - The company is under investigation by the United States Attorney's Office for the District of New Jersey regarding the sale and promotion of Korlym, with a focus on relationships and payments to healthcare professionals[139]. - Potential violations of federal healthcare laws could lead to civil and criminal penalties, including fines and exclusion from government healthcare programs, adversely affecting financial results[140]. - Regulatory compliance is critical, as failure to maintain approvals or comply with regulations could lead to significant penalties and loss of revenue[135]. - The company is subject to increased scrutiny regarding patient assistance programs, which may reduce the financial support available to patients needing treatment for hypercortisolism[123]. Financial Performance and Revenue - Research and development expenses were $246.9 million,$184.4 million, and $131.0 million for the years ended December 31, 2024, 2023, and 2022, respectively, accounting for 46$20.84 to $74.61 during the same period[160]. - Approximately 21 percent of the company's common stock is beneficially owned by officers, directors, and principal stockholders as of February 18, 2025[160]. - The company faces risks related to stock performance, which could be influenced by the outcomes of ongoing investigations and regulatory compliance[159]. - The Board of Directors authorized a stock repurchase program of up to$200 million in January 2024, which may reduce cash reserves and impact future capital requirements[181]. - As of February 18, 2025, officers and directors beneficially owned approximately 21% of the common stock, potentially influencing corporate actions significantly[183]. Legal Matters - The company faces ongoing litigation, including antitrust claims from Teva Pharmaceuticals, which could impact financial conditions and operations[194]. - The company has faced a securities class action lawsuit that resulted in a settlement requiring a one-time payment of $14 million, fully reimbursed by insurers[197].