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What changed in CORCEPT THERAPEUTICS INC's 10-K — 2023 vs 2024

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Paragraph-level year-over-year comparison of CORCEPT THERAPEUTICS INC's 2023 and 2024 10-K annual filings, covering the Business, Risk Factors, Legal Proceedings, Cybersecurity, MD&A and Market Risk sections. Every new, removed and edited paragraph is highlighted side-by-side so you can see exactly what management changed in the 2024 report.

+373 added−360 removedSource: 10-K (2025-02-26) vs 10-K (2024-02-15)

Top changes in CORCEPT THERAPEUTICS INC's 2024 10-K

373 paragraphs added · 360 removed · 268 edited across 7 sections

Item 1. Business

Business — how the company describes what it does

77 edited+36 added−29 removed80 unchanged
Biggest changeIf the FDA approves the marketing of a new drug, such approval will be granted for particular indications and may entail limitations on the indicated uses for which such product may be marketed. The FDA may withdraw its approval at any time if compliance with regulatory standards is not maintained.
Biggest changeWhen a drug receives Fast Track designation, among other things, the manufacturer is eligible for more frequent communication with the FDA regarding the drug’s NDA, and for the FDA to review parts of the application as they are submitted, rather than waiting until every section of the NDA is completed. 8 If the FDA approves the marketing of a new drug, such approval will be granted for particular indications and may entail limitations on the indicated uses for which such product may be marketed.
Patients who received relacorilant intermittently also had a longer median duration of response (“DoR”) (5.6 months versus 3.7 months, hazard ratio: 0.36; p-value: 0.006) compared to those who received nab-paclitaxel alone. Patients who received relacorilant intermittently 3 also lived longer (median OS: 13.9 months versus 12.2 months, hazard ratio: 0.67; p-value: 0.066) compared to those who received nab-paclitaxel alone.
Patients who received relacorilant intermittently also had a longer median duration of response (“DoR”) (5.6 months versus 3.7 months, hazard ratio: 0.36; p-value: 0.006) compared to those who received nab-paclitaxel alone. Patients who received relacorilant intermittently also lived longer (median OS: 13.9 months versus 12.2 months, hazard ratio: 0.67; p-value: 0.066) compared to those who received nab-paclitaxel alone.
If our operations are found to be in violation of any of such laws or any other governmental regulations that apply to us, we may be subject to penalties, including, without limitation, civil and criminal penalties, damages, fines, the curtailment or restructuring 9 of our operations, exclusion from participation in federal and state healthcare programs and imprisonment, any of which could adversely affect our ability to operate our business and our financial results.
If our operations are found to be in violation of any of such laws or any other governmental regulations that apply to us, we may be subject to penalties, including, without limitation, civil and criminal penalties, damages, fines, the curtailment or restructuring of our operations, exclusion from participation in federal and state healthcare programs and imprisonment, any of which could adversely affect our ability to operate our business and our financial results.
Companies that must comply with the GDPR face increased compliance obligations and risk, including more 10 robust regulatory enforcement of data protection requirements and potential fines for noncompliance of up to €20 million or 4% of the annual global revenues of the noncompliant company, whichever is greater.
Companies that must comply with the GDPR face increased compliance obligations and risk, including more robust regulatory enforcement of data protection requirements and potential fines for noncompliance of up to €20 million or 4% of the annual global revenues of the noncompliant company, whichever is greater.
The Anti-Kickback Statute is subject to evolving interpretations, and in the absence of substantive 8 guidance, it is possible for future initiatives or engagements with healthcare professionals to be challenged under this Statute, which could adversely impact our operations.
The Anti-Kickback Statute is subject to evolving interpretations, and in the absence of substantive guidance, it is possible for future initiatives or engagements with healthcare professionals to be challenged under this statute, which could adversely impact our operations.
In the event that we are subject to or affected by HIPAA, the CCPA, the CPRA or other domestic privacy and data protection laws, any liability from failure to comply with the requirements of these laws could adversely affect our financial condition.
In the event that we are subject to or affected by HIPAA, the CCPA or other domestic privacy and data protection laws, any liability from failure to comply with the requirements of these laws could adversely affect our financial condition.
Some of these states also require the implementation of commercial compliance programs and impose restrictions on drug manufacturer marketing practices. Federal and state agencies continue to spend time, energy and resources combating healthcare fraud and abuse.
Some of these states also require the implementation of commercial compliance programs and impose restrictions on drug manufacturer marketing practices. 10 Federal and state agencies continue to spend time, energy and resources combating healthcare fraud and abuse.
The FDA may also require post-approval studies, referred to as “Phase 4 7 studies,” to monitor or further explore the effect of approved products, and may limit marketing of the drug based on the results of such studies.
The FDA may also require post-approval studies, referred to as “Phase 4 studies,” to monitor or further explore the effect of approved products, and may limit marketing of the drug based on the results of such studies.
These researchers have studied the potential utility of mifepristone and our proprietary selective cortisol modulators in a wide range of disorders, including central serous retinopathy, post-traumatic stress disorder, anxiety, alcoholism, cocaine addiction, Alzheimer’s disease, ALS, Cushing’s syndrome, metabolic syndrome, atherosclerosis, fatty liver disease, sarcoma, melanoma and solid tumors, including triple-negative breast, prostate, ovarian and non-small cell lung cancers.
These researchers have studied the potential utility of mifepristone and our proprietary selective cortisol modulators in a wide range of disorders, including central serous retinopathy, post-traumatic stress disorder, anxiety, alcoholism, cocaine addiction, Alzheimer’s disease, ALS, hypercortisolism, metabolic syndrome, atherosclerosis, fatty liver disease, sarcoma, melanoma and solid tumors, including triple-negative breast, prostate, ovarian and non-small cell lung cancers.
On December 29, 2023, the federal district in which we had sued Teva issued a ruling finding that Teva’s proposed product would not infringe the two patents we had asserted against it. We have appealed that decision to the Federal Circuit of Appeals. See “Part I, Item 3, Legal Proceedings” for additional information. Intellectual Property Overview.
On December 29, 2023, the federal district in which we had sued Teva issued a ruling finding 6 that Teva’s proposed product would not infringe the two patents we had asserted against it. We have appealed that decision to the Court of Appeals for the Federal Circuit. See “Part I, Item 3, Legal Proceedings” for additional information. Intellectual Property Overview.
In addition, from January 1, 2021, companies have had to comply with both the GDPR and the GDPR as incorporated into United Kingdom national law, the latter regime having the ability to separately fine up to the greater of £17.5 million or 4% of global turnover.
In addition, from January 1, 2021, companies have had to comply with both the GDPR and the GDPR as incorporated into United Kingdom national law (“UK GDPR”), the latter regime having the ability to separately fine up to the greater of £17.5 million or 4% of global turnover.
Before a new drug may be marketed in the United States, the FDA generally requires completion of preclinical laboratory and animal testing, performance of adequate and well-controlled human clinical trials to establish the safety and efficacy of the proposed drug’s 6 intended use and approval by the FDA.
Before a new drug may be marketed in the United States, the FDA generally requires completion of preclinical laboratory and animal testing, performance of adequate and well-controlled human clinical trials to establish the safety and efficacy of the proposed drug’s 7 intended use and approval by the FDA.
Study participants were randomized to one of three treatment arms: 60 women received 150 mg of relacorilant intermittently (the day before, the day of and the day after their weekly nab-paclitaxel infusion) and 58 women received a daily relacorilant dose of 100 mg per day in addition to nab-paclitaxel. Sixty women received nab-paclitaxel alone.
Phase 2 study participants were randomized to one of three treatment arms: 60 women received 150 mg of relacorilant intermittently (the day before, the day of and the day after their weekly nab-paclitaxel infusion) and 58 women received a daily relacorilant dose of 100 mg per day in addition to nab-paclitaxel. Sixty women received nab-paclitaxel alone.
Both of these drugs are approved by the FDA for the treatment of adult patients with Cushing’s disease who are not candidates for pituitary surgery or for whom surgery did not work, and both are sold by the Italian pharmaceutical company Recordati S.p.A (“Recordati”). Cushing’s disease is a subset of Cushing’s syndrome.
Both of these drugs are approved by the FDA for the treatment of adult patients with Cushing’s disease who are not candidates for pituitary surgery or for whom surgery did not work, and both are sold by the Italian pharmaceutical company Recordati S.p.A (“Recordati”). Cushing’s disease is a subset of hypercortisolism.
Even when HIPAA does not apply, according to the Federal Trade Commission or the FTC, failing to take appropriate steps to keep consumers’ personal information secure constitutes unfair acts or practices in or affecting commerce in violation of Section 5(a) of the Federal Trade Commission Act.
Even when HIPAA does not apply, according to the Federal Trade Commission (the “FTC”), failing to take appropriate steps to keep consumers’ personal information secure constitutes unfair acts or practices in or affecting commerce in violation of Section 5(a) of the Federal Trade Commission Act.
Benefits of orphan drug designation by the EC are similar, but also include protocol assistance from the European Medicines Agency (“EMA”), access to the centralized marketing authorization procedure in the European Union (“EU”) and, if we obtain approval, ten years of exclusive marketing rights in the EU for the treatment of patients with Cushing’s syndrome.
Benefits of orphan drug designation by the EC are similar, but also include protocol assistance from the European Medicines Agency (“EMA”), access to the centralized marketing authorization procedure in the European Union (“EU”) and, if we obtain approval, ten years of exclusive marketing rights in the EU for the treatment of patients with hypercortisolism.
In addition to the United States and foreign patents we own or have licensed relating to Cushing’s syndrome and various cancers, we also own U.S. and foreign patents for the use of cortisol modulators to treat ALS, AIWG, fatty liver disease, delirium, catatonia, psychosis induced by interferon-alpha therapy, migraine headaches, gastroesophageal reflux disease, neurological damage in premature infants, for the improvement of therapeutic response to electroconvulsive therapy, and in the treatment of diseases using combined steroid and GR modulator therapy.
In addition to the United States and foreign patents we own or have licensed relating to hypercortisolism and various cancers, we also own U.S. and foreign patents for the use of cortisol modulators to treat ALS, fatty liver disease, delirium, catatonia, psychosis induced by interferon-alpha therapy, migraine headaches, gastroesophageal reflux disease, neurological damage in premature infants, for the improvement of therapeutic response to electroconvulsive therapy, and in the treatment of diseases using combined steroid and GR modulator therapy.
Cortisol also suppresses the body’s immune response; activating – not suppressing – the immune system is beneficial in fighting certain cancers. Many types of solid tumors express the GR and are potential targets for cortisol modulation therapy, among them ovarian, adrenal and prostate cancer. Relacorilant in Patients with Advanced Ovarian Cancer .
Cortisol also suppresses the body’s immune response; activating – not suppressing – the immune system is beneficial in fighting certain cancers. Many types of solid tumors express the GR and are potential targets for cortisol modulation therapy, among them ovarian, adrenal and prostate cancer. Relacorilant in Patients with Platinum-Resistant Ovarian Cancer.
Accordingly, we believe we are not obligated to pay royalties relating to the use of intellectual property to any third parties except the University of Chicago, from which we have licensed certain patents, as described below. Cushing’s Syndrome . The composition of matter patent covering Korlym’s active ingredient, mifepristone, has expired.
Accordingly, we believe we are not obligated to pay royalties relating to the use of intellectual property to any third parties except the University of Chicago, from which we have licensed certain patents, as described below. Hypercortisolism. The composition of matter patent covering Korlym’s active ingredient, mifepristone, has expired.
In November 2020, the PTAB rejected Teva’s claims, affirming the validity of our patent in its entirety. Teva 5 appealed to the Federal Circuit Court of Appeals, which affirmed the PTAB’s decision.
In November 2020, the PTAB rejected Teva’s claims, affirming the validity of our patent in its entirety. Teva appealed to the Court of Appeals for the Federal Circuit, which affirmed the PTAB’s decision.
Pre-clinical data also suggest that modulating cortisol activity may lead to treatments for patients with amyotrophic lateral sclerosis (“ALS”). 1 Since 2012, we have marketed Korlym ® in the United States for the treatment of patients suffering from hypercortisolism (“Cushing’s syndrome”).
Pre-clinical and clinical data also suggest that modulating cortisol activity may lead to treatments for patients with amyotrophic lateral sclerosis (“ALS”). 1 Since 2012, we have marketed Korlym in the United States for the treatment of patients suffering from hypercortisolism (also known as “Cushing’s syndrome”).
As of December 31, 2023, we had 352 employees. We consider our employee relations to be good. Our employees are not covered by a collective bargaining agreement. We seek to hire, retain and motivate smart, ethical, hard-working employees, officers and directors. To achieve this goal, we offer a collegial work environment where creativity, collaboration and initiative are encouraged.
As of December 31, 2024, we had 500 employees. We consider our employee relations to be good. Our employees are not covered by a collective bargaining agreement. We seek to hire, retain and motivate smart, ethical, hard-working employees, officers and directors. To achieve this goal, we offer a collegial work environment where creativity, collaboration and initiative are encouraged.
We offer competitive salaries, performance bonuses and equity grants, as well as industry-leading health, retirement and childcare benefits. To align our people’s goals with Corcept’s goals, we offer annual performance-based cash bonuses and stock-based compensation. About Corcept We were incorporated in the State of Delaware on May 13, 1998. Our registered trademarks include Corcept ® and Korlym ® .
We offer competitive salaries, performance bonuses and equity grants, as well as industry-leading health, retirement and other benefits. To align our employees’ goals with Corcept’s goals, we offer annual performance-based cash bonuses and stock-based compensation. About Corcept We were incorporated in the State of Delaware on May 13, 1998. Our registered trademarks include Corcept ® and Korlym ® .
In the United States, relacorilant’s orphan designation confers tax credits, reduced regulatory fees and, provided we obtain approval for the treatment of patients with Cushing’s syndrome, seven years of exclusive marketing rights.
In the United States, relacorilant’s orphan designation confers tax credits, reduced regulatory fees and, provided we obtain approval for the treatment of patients with hypercortisolism, seven years of exclusive marketing rights.
We also own U.S. and foreign patents directed to the use of our selective cortisol modulators in the treatment of a variety of serious disorders, including Cushing’s syndrome, various cancers, fatty liver disease, AIWG, and other disorders. We continue to file patent applications in the United States and abroad.
We also own U.S. and foreign patents directed to the use of our selective cortisol modulators in the treatment of a variety of serious disorders, including hypercortisolism, various cancers, fatty liver disease, and other disorders. We continue to file patent applications in the United States and abroad.
Competition for Korlym Korlym competes with established treatments, including surgery, radiation and other medications, including “off-label” uses of drugs such as ketoconazole, an anti-fungal medication, and metyrapone, which is approved for testing hypothalamic-pituitary function. Korlym also competes with Signifor ® (pasireotide) Injection and Isturisa ® (osilodrostat).
Competition Our Products compete with established treatments, including surgery, radiation and other medications, including “off-label” uses of drugs such as ketoconazole, an anti-fungal medication, and metyrapone, which is approved for testing hypothalamic-pituitary function. Our Products also compete with Signifor ® (pasireotide) Injection and Isturisa ® (osilodrostat).
We own U.S. method of use patents directed to the use of Korlym in the treatment of patients with Cushing’s syndrome, with expiration dates ranging from 2028 to 2038.
We own U.S. method of use patents directed to the use of Korlym in the treatment of patients with hypercortisolism, with expiration dates ranging from 2028 to 2038.
Furthermore, we own U.S. compound and method of use patents using our proprietary selective cortisol modulators directed to the treatment of patients with Cushing’s syndrome, with expiration dates ranging from 2033 to 2041. We have asserted two patents directed to patients with Cushing’s syndrome in a lawsuit against Teva Pharmaceuticals USA, Inc.
Furthermore, we own U.S. composition of matter and method of use patents using our proprietary selective cortisol modulators directed to the treatment of patients with hypercortisolism, with expiration dates ranging from 2033 to 2041. We have asserted two patents directed to patients with hypercortisolism in a lawsuit against Teva Pharmaceuticals USA, Inc.
The orphan drug marketing exclusivity period for Korlym ended in February 2019, which means a competitor that receives FDA approval for a generic equivalent of Korlym may market its drug to patients with Cushing’s syndrome, provided doing so would not infringe any of our patents. We sued Teva Pharmaceuticals USA, Inc.
The orphan drug marketing exclusivity period for Korlym ended in February 2019, which means a competitor that receives FDA approval for a generic equivalent of Korlym may market its drug to patients with hypercortisolism, provided doing so would not infringe any of our patents. In January 2024, Teva launched a generic version of Korlym. We sued Teva Pharmaceuticals USA, Inc.
Foreign approvals may not be granted on a timely basis, or at all. Regulatory approval of pricing is required in most countries other than the United States, which pricing may be too low to generate an acceptable return. We are not seeking regulatory approval to market Korlym outside the United States.
Foreign approvals may not be granted on a timely basis, or at all. Regulatory approval of pricing is required in most countries other than the United States, which pricing may be too low to generate an acceptable return.
Research and Development Spending We incurred $184.4 million, $131.0 million and $113.9 million of research and development expense in the years ended December 31, 2023, 2022 and 2021, respectively, which accounted for 49 percent, 45 percent and 47 percent, respectively, of our total operating expenses in those years.
Research and Development Spending We incurred $246.9 million, $184.4 million and $131.0 million of research and development expense in the years ended December 31, 2024, 2023 and 2022, respectively, which accounted for 46 percent, 49 percent and 45 percent, respectively, of our total operating expenses in those years.
Because many people who suffer from Cushing’s syndrome are undiagnosed or inadequately treated, we have developed and continue to refine and expand programs to educate physicians and patients about screening for hypercortisolism and the role Korlym can play in treating patients with the disorder.
Because most people who suffer from hypercortisolism are undiagnosed or inadequately treated, we have developed and continue to refine and expand programs to educate physicians and patients about screening for hypercortisolism and the role our Products can play in treating patients with the disorder.
In the EU, osilodrostat is also approved as a treatment for Cushing’s syndrome. Korlym also competes with Recorlev ® (levoketoconazole), a chiral form of the commonly-prescribed cortisol synthesis inhibitor ketoconazole, that is sold by Xeris Biopharma Holdings, Inc. (“Xeris”), as a treatment for patients with Cushing’s syndrome.
In the EU, osilodrostat is also approved as a treatment for hypercortisolism. Our Products also compete with Recorlev ® (levoketoconazole), a chiral form of the commonly-prescribed cortisol synthesis inhibitor ketoconazole, that is sold by Xeris Biopharma Holdings, Inc. (“Xeris”), as a treatment for patients with hypercortisolism.
Women with a history of tumors that do not respond to initial platinum-based treatments (i.e., women with “primary platinum-refractory” disease) and those who have received more than three prior lines of therapy will be excluded. Relacorilant in Patients with Adrenal Cancer with Cortisol Excess .
Women with a history of tumors that do not respond to initial platinum-based treatments (i.e., women with “primary platinum-refractory” disease) and those who have received more than three prior lines of therapy were excluded.
The composition of our selective cortisol modulators and their methods of use in a wide range of indications are covered by U.S. and foreign patents. Our lead compounds are being evaluated in clinical trials as potential treatments for a variety of serious disorders – Cushing’s syndrome, advanced ovarian cancer, adrenal cancer with cortisol excess, prostate cancer, ALS and NASH.
The composition of these “selective” cortisol modulators and their methods of use in a wide range of indications are covered by U.S. and foreign patents. Our lead compounds are being evaluated in clinical trials as potential treatments for a variety of serious disorders – hypercortisolism, advanced ovarian cancer, prostate cancer, ALS and MASH. Hypercortisolism (Cushing ’ s Syndrome) Background.
Manufacturing Korlym We rely on contract manufacturers to produce Korlym and our product candidates. Because we have an agreement with one third-party manufacturer to supply mifepristone, the active pharmaceutical ingredient (“API”) in Korlym, we have purchased and hold significant quantities of API. We have two third-party manufacturers to produce and bottle Korlym tablets.
Manufacturing We rely on contract manufacturers to produce our Products and product candidates. We have agreements with manufacturers to supply mifepristone, the active pharmaceutical ingredient (“API”) in our Products, and to produce and bottle tablets of our Products. We have purchased and hold significant quantities of API.
The federal Anti-Kickback Statute prohibits, among other things, any person from knowingly and willfully offering, soliciting, receiving or providing remuneration, directly or indirectly, to induce either the referral of an individual, for an item or service or the purchasing or ordering of a good or service, for which payment may be made under federal healthcare programs such as the Medicare and Medicaid programs.
Foreign governments have comparable regulations, and violating these laws and regulations in any jurisdiction could result in significant criminal, civil, and administrative sanctions. 9 The federal Anti-Kickback Statute prohibits, among other things, any person from knowingly and willfully offering, soliciting, receiving or providing remuneration, directly or indirectly, to induce either the referral of an individual, for an item or service or the purchasing or ordering of a good or service, for which payment may be made under federal healthcare programs such as the Medicare and Medicaid programs.
We own U.S. patents covering methods of treating cancer using our proprietary selective cortisol modulators with expiration dates ranging from 2033 to 2041.
See “Part I, Item 3, Legal Proceedings” for additional details. Oncology . We own U.S. patents covering methods of treating cancer using our proprietary selective cortisol modulators with expiration dates ranging from 2033 to 2041.
Because Korlym’s active ingredient, mifepristone, reduces the binding of excess cortisol to the GR, it can modulate the effects of abnormal levels and release patterns of cortisol without compromising cortisol’s healthy functions and rhythms.
Cortisol activity can be modulated effectively by a drug that competes with cortisol’s binding to the glucocorticoid receptor (“GR”). Because Korlym’s active ingredient, mifepristone, reduces the binding of excess cortisol to the GR, it can modulate the effects of abnormal levels and release patterns of cortisol without compromising cortisol’s healthy functions and rhythms.
Excessive cortisol activity, known as hypercortisolism, may lead to a suppressed immune response, impaired glucose tolerance, diabetes, obesity, fatty liver disease, depressed mood, psychosis, wasting of the arms and legs, edema, fatigue, hypertension and other problems.
Excessive cortisol activity, known as hypercortisolism, may lead to hypertension, diabetes, impaired glucose tolerance, obesity, fatty liver disease, depressed mood, psychosis, wasting of the arms and legs, edema, fatigue, insomnia and other problems. Cortisol reduces a patient’s immune response to oncogenesis, shields certain cancer cells from the apoptotic effects of chemotherapy and facilitates the growth of others.
We also own or have exclusively licensed U.S. and European patents covering the use of GR modulators, including relacorilant, miricorilant, exicorilant, dazucorilant, and other of our proprietary compounds to treat a variety of disorders, including CRPC and other solid tumors.
We also own or have exclusively licensed U.S. and European patents covering the use of GR modulators, including relacorilant, miricorilant, dazucorilant, and other of our proprietary compounds to treat a variety of disorders, including CRPC and other solid tumors. Relacorilant has been designated an orphan drug in both the United States and the EU for the treatment of pancreatic cancer.
Our selective cortisol modulator dazucorilant improved motor performance and reduced neuroinflammation and muscular atrophy in animal models of ALS. Following these compelling results, we initiated a Phase 2 trial (“DAZALS”) of dazucorilant in patients with ALS in October 2022.
ALS ALS, also known as Lou Gehrig’s disease, is a devastating neuromuscular illness. Our selective cortisol modulator dazucorilant improved motor performance and reduced neuroinflammation and muscular atrophy in an animal model of ALS. Following these compelling results, we initiated a Phase 2 trial (“DAZALS”) of dazucorilant in patients with ALS.
The holder of an approved NDA must submit periodic reports to the FDA, including reports of adverse patient experiences, which could cause the FDA to impose marketing restrictions through labeling changes or remove the drug from the market.
The FDA may withdraw its approval at any time if compliance with regulatory standards is not maintained. The holder of an approved NDA must submit periodic reports to the FDA, including reports of adverse patient experiences, which could cause the FDA to impose marketing restrictions through labeling changes or remove the drug from the market.
In May 2021, we announced preliminary results from our 178-patient, controlled, multi-center, Phase 2 trial of relacorilant combined with nab-paclitaxel in patients with platinum-resistant ovarian cancer.
ROSELLA seeks to replicate the positive results of our Phase 2 trial, a 178-patient, controlled, multi-center, trial of relacorilant combined with nab-paclitaxel in patients with platinum-resistant ovarian cancer.
To help us achieve that goal, we fund our own patient support programs and donate money to independent charitable foundations that help patients pay for all aspects of their Cushing’s syndrome care, whether or not that care includes taking Korlym.
Our policy is that no patient with hypercortisolism will be denied access to our Products for financial reasons. To help us achieve that goal, we have patient support programs and donate money to independent charitable foundations that help patients pay for all aspects of their hypercortisolism care, whether or not that care includes taking our Products.
Relacorilant shares Korlym’s affinity for the GR, but, unlike Korlym, has no affinity for the PR, and so is not the “abortion pill.” It does not cause other effects associated with PR affinity, including endometrial thickening and vaginal bleeding.
We are developing our proprietary, selective cortisol modulator, relacorilant, as a treatment for patients with hypercortisolism. Relacorilant shares Korlym’s affinity for the GR but, unlike Korlym, has no affinity for the PR and so is 2 not the “abortion pill” and does not cause other effects associated with PR affinity, including endometrial thickening and vaginal bleeding.
Further, the California Consumer Privacy Act (“CCPA”) which took effect on January 1, 2020, created individual privacy rights for California consumers and increased the privacy and security obligations of entities handling certain personal information.
Further, the California Consumer Privacy Act which took effect on January 1, 2020, and was later revised and expanded by the California Privacy Rights Act (collectively, “CCPA”), created individual privacy rights for California consumers and increased the privacy and security obligations of entities handling certain personal information as well as limitations on data uses, audit requirements for higher risk data, and opt outs for certain uses of sensitive data.
Studies by Independent Investigators For many years we have advanced our understanding of cortisol modulation by supporting the work of independent academic investigators.
We continue to create new selective cortisol modulators, the most promising of which we advance towards the clinic. Studies by Independent Investigators For many years we have advanced our understanding of cortisol modulation by supporting the work of independent academic investigators.
Safety and tolerability of relacorilant plus nab-paclitaxel were comparable to nab-paclitaxel monotherapy. The final analysis from our Phase 2 trial was published in the Journal of Clinical Oncology (Colombo et al., 2023), the premiere journal of the American Society of Clinical Oncology (ASCO).
The final analysis from our Phase 2 trial was published in the Journal of Clinical Oncology (Colombo et al., 2023), the premiere journal of the American Society of Clinical Oncology (ASCO). Relacorilant in Patients with Adrenal Cancer with Cortisol Excess.
(“Teva”) in a lawsuit filed in Federal District Court. On December 29, 2023, the Court found that Teva’s proposed generic version of Korlym would not infringe either patent. We are appealing that decision. See “Part I, Item 3, Legal Proceedings” for additional details. Oncology .
(“Teva”) in a lawsuit filed in Federal District Court. On December 29, 2023, the Court found that Teva’s proposed generic version of Korlym would not infringe either patent. We are appealing that decision to the Court of Appeals for the Federal Circuit. We do not know when our appeal will be resolved.
Patients who received a higher dose of relacorilant intermittently exhibited a statistically significant improvement in median PFS (5.6 months versus 3.8 months, hazard ratio: 0.66; p-value: 0.038).
The trial’s primary endpoint was PFS. Patients in both of the relacorilant plus nab-paclitaxel treatment arms of the Phase 2 trial experienced longer PFS than did the patients who received nab-paclitaxel alone. Patients who received a higher dose of relacorilant intermittently exhibited a 4 statistically significant improvement in median PFS (5.6 months versus 3.8 months, hazard ratio: 0.66; p-value: 0.038).
Clinical Trial Agreements We typically conduct our clinical trials with the assistance of clinical research organizations (“CROs”). ICON plc is helping us conduct our GRACE and GRADIENT trials. Syneos Health is helping us conduct our ROSELLA trial. Julius Clinical is helping us conduct our DAZALS trial. Medpace Research is helping us conduct our MONARCH trial.
Clinical Trial Agreements We typically conduct our clinical trials with the assistance of clinical research organizations (“CROs”). Syneos Health is helping us conduct our ROSELLA trial. Medpace Research is helping us conduct our MONARCH trial. We may terminate our agreements with Syneos Health on 60 days’ written notice and with Medpace Research without cause at any time.
These laws include, without limitation, state and federal anti-kickback, fraud and abuse, false claims, and physicians’ sunshine (e.g. transparency) laws and regulations. Foreign governments have comparable regulations, and violating these laws and regulations in any jurisdiction could result in significant criminal, civil, and administrative sanctions.
These laws include, without limitation, state and federal anti-kickback, fraud and abuse, false claims, and physicians’ sunshine (e.g. transparency) laws and regulations.
These novel molecules are “selective” cortisol modulators: they share Korlym’s affinity for the GR, but, unlike Korlym, do not bind to the PR and therefore do not cause effects arising from antagonism of progesterone activity, such as termination of pregnancy, endometrial thickening and vaginal bleeding.
In addition to relacorilant, we have discovered more than 1,000 proprietary cortisol modulators that bind to the GR but have no affinity for the PR and therefore do not cause effects arising from antagonism of progesterone activity, such as termination of pregnancy, endometrial thickening and vaginal bleeding.
The challenge in treating a patient with Cushing’s syndrome is modulating cortisol’s effects without either inappropriately suppressing them or disrupting cortisol’s normal diurnal rhythm. Simply reducing or destroying the ability of the body to make cortisol can cause serious harm. Cortisol activity can be modulated effectively by a drug that competes with cortisol, binding to the glucocorticoid receptor (“GR”).
In June 2024, we made available an authorized generic version of Korlym for the same indication. The challenge in treating a patient with hypercortisolism is modulating cortisol’s effects without either inappropriately suppressing them or disrupting cortisol’s normal diurnal rhythm. Simply reducing or destroying the ability of the body to make cortisol can cause serious harm.
The primary endpoint is PFS; overall survival is a key secondary endpoint. Patients in ROSELLA will have received prior bevacizumab therapy, which is the standard of care for patients with platinum-resistant ovarian cancer.
ROSELLA has dual primary endpoints – progression free survival (“PFS”) and overall survival (“OS”). ROSELLA will have a statistically positive outcome if either endpoint is met. Patients enrolled in ROSELLA were required to have received prior bevacizumab therapy, which is the approved standard of care for patients with platinum-resistant ovarian cancer.
Pre-clinical and clinical data also indicate that modulating cortisol activity may improve outcomes in patients with fatty liver disease and non-alcoholic steatohepatitis (“NASH”), which are precursors of liver fibrosis and cirrhosis, and in patients at risk of weight gain caused by antipsychotic medications (referred to as antipsychotic induced weight gain, or “AIWG”).
Pre-clinical and clinical data indicate that modulating cortisol activity may improve outcomes in patients with fatty liver disease and metabolic dysfunction-associated steatohepatitis (“MASH”), which are precursors of liver fibrosis and cirrhosis.
We have applied, and will continue to apply, for patents covering the composition and method of use of our products and product candidates. See “Business – Intellectual Property” for additional information. We continue to identify selective cortisol modulators and plan to advance the most promising of them towards the clinic.
We hold U.S. and foreign patents covering these compounds and their methods of use in a wide range of indications. We have applied, and will continue to apply, for patents covering the composition and method of use of our Products and product candidates. See “Business – Intellectual Property” for additional information.
As a result, significant uncertainty exists with respect to GDPR compliance and the attendant obligations going forward as the regulatory environment is rapidly developing.
Transfers of personal information out of the European Union face a constantly shifting set of requirements, as courts in Europe have invalidated intergovernmental agreements. As a result, uncertainty exists with respect to GDPR compliance and the attendant obligations going forward as the regulatory environment is rapidly developing.
We have initiated a Phase 4 study (“CATALYST”), to determine the prevalence of Cushing’s syndrome in patients with difficult-to-control diabetes (HbA1c of 7.5 percent or higher) despite receiving standard treatment. In CATALYST’s first phase, 1,000 patients will be evaluated.
In 2024, we conducted the CATALYST study to determine the prevalence of hypercortisolism in patients with difficult-to-control diabetes (defined as HbA1c of 7.5 percent or higher) despite receiving optimum treatment. Of the 1,057 patients enrolled in the first phase of CATALYST, 23.8 percent were found to have hypercortisolism.
Depending on the type of tumor, surgery can result in a range of complications. Korlym . We sell Korlym in the United States, using experienced sales representatives to call on physicians caring for patients with Cushing’s syndrome (also called “hypercortisolism”). We also have a field-based force of medical science liaisons.
We sell Korlym and a generic version of Korlym in the United States (our “Products”), using sales representatives to call on physicians caring for patients with hypercortisolism. We also have a field-based force of medical science liaisons. We use a specialty pharmacy and a specialty distributor to distribute our Products and provide logistical support to physicians and patients.
There is no evidence that relacorilant causes hypokalemia (low potassium), a potentially serious condition that is a leading cause of patients stopping treatment with Korlym. Forty-four percent of patients in Korlym’s pivotal trial experienced hypokalemia. Unlike all other medications used to treat Cushing's syndrome, relacorilant does not prolong the heart's QT interval, a potentially deadly off-target effect.
Because relacorilant does not meaningfully increase cortisol levels, it does not cause hypokalemia (low potassium), a potentially serious condition that is a leading cause of patients stopping treatment with Korlym. Forty-four percent of patients in Korlym’s pivotal trial experienced hypokalemia.
Combining a cortisol modulator with an androgen modulator may block this escape route. Our collaborators at the University of Chicago have initiated a randomized, placebo-controlled Phase 2 trial of relacorilant plus enzalutamide in patients with prostate cancer, pre-prostatectomy.
Our collaborators at the University of Chicago have initiated a randomized, placebo-controlled Phase 2 trial of relacorilant plus enzalutamide in patients with prostate cancer, pre-prostatectomy. We are providing relacorilant and placebo for the study. Patents we have licensed from the University of Chicago cover the use of relacorilant combined with anticancer agents, including enzalutamide, to treat patients with this disease.
The CCPA provides for civil penalties for violations, as well as a private right of action for data breaches that is expected to increase data breach litigation. The CCPA may increase our compliance costs and potential liability.
The CCPA provides for civil penalties for violations, as well as a private right of action for data breaches that is expected to increase data breach litigation. It also created a new California data protection agency authorized to issue substantive regulations and could result in increased privacy and information security enforcement.
We are conducting an open-label, Phase 1b trial of relacorilant plus the PD-1 checkpoint inhibitor pembrolizumab in patients with metastatic or unresectable adrenal cancer whose tumors produce cortisol. The trial is examining whether adding relacorilant to pembrolizumab therapy reduces cortisol-activated immune suppression sufficiently to help pembrolizumab achieve its intended tumor-killing effect.
We have completed an open-label, Phase 1b trial of relacorilant plus the PD-1 checkpoint inhibitor pembrolizumab in 14 patients with metastatic or unresectable adrenal cancer whose tumors produce cortisol. Patients with this form of adrenal cancer virtually never respond to immunotherapy and their disease progresses very rapidly.
More than 1,000 of these compounds, including relacorilant, exicorilant, miricorilant and dazucorilant, potently bind to the GR but not the progesterone, estrogen or androgen receptors. We hold U.S. and foreign patents covering these compounds and their methods of use in a wide range of indications.
Development of Other Selective Cortisol Modulators Our portfolio of proprietary selective cortisol modulators consists of four structurally distinct series. More than 1,000 of these compounds, including relacorilant, miricorilant and dazucorilant, potently bind to the GR but not the progesterone, estrogen or androgen receptors.
These patients often exhibit less severe symptoms or have a more gradual course of disease than patients with other etiologies of Cushing’s syndrome, although their health outcomes are ultimately poor. Half of the patients in GRADIENT will receive relacorilant for 22 weeks and half will receive placebo. The trial’s primary endpoints are improvements in glucose metabolism and hypertension.
These patients have a more gradual decline than patients with other etiologies of hypercortisolism, although their health outcomes are ultimately poor. GRADIENT enrolled 137 patients with hypercortisolism and either hypertension, hyperglycemia or both. Patients were randomized on a double-blind basis 1:1 to receive either relacorilant or placebo for 22 weeks.
Relacorilant is also expected to treat the patients’ Cushing’s syndrome generated by their tumors’ excess production of cortisol. Relacorilant in Patients with Prostate Cancer . Androgen deprivation is the standard treatment for prostate cancer because androgens stimulate prostate tumor growth. Tumors often escape androgen deprivation therapy when cortisol’s activity at the GR stimulates tumor growth.
Relacorilant in Patients with Prostate Cancer . Androgen deprivation is the standard treatment for prostate cancer because androgens stimulate prostate tumor growth. Prostate cancer tumors eventually escape androgen deprivation therapy; one of the prime reasons is that these tumors begin to be stimulated by cortisol’s activity. Combining a cortisol modulator with an androgen modulator may block this escape route.
Nonalcoholic steatohepatitis (“NASH”) is an advanced form of nonalcoholic fatty liver disease that afflicts millions of patients and is a leading cause of liver-related mortality.
The FDA has granted dazucorilant Fast Track Designation and orphan drug status for the treatment of ALS in the United States. Metabolic Diseases Liver Disease. Metabolic dysfunction-associated steatohepatitis (“MASH”) is an advanced form of metabolic dysfunction-associated fatty liver disease that afflicts millions of patients and is a leading cause of liver-related mortality.
In the GRACE trial, each patient receives relacorilant for 22 weeks. Patients who exhibit pre-specified improvements in hypertension and/or glucose metabolism enter a 12-week, double-blind, “randomized withdrawal” phase, in which half of the patients continue receiving relacorilant and half receive placebo.
Patients who exhibited pre-specified improvements in either hypertension, hyperglycemia or both symptoms proceeded to GRACE’s second, double-blind, randomized withdrawal phase, in which half of the patients continued to receive relacorilant and half received placebo for 12 weeks.
In June 2022, we initiated a pivotal Phase 3 trial (“ROSELLA”) that seeks to replicate the positive results observed in our Phase 2 study. ROSELLA has a planned enrollment of 360 women with recurrent, platinum-resistant ovarian cancer, half of whom will receive 150 mg of relacorilant intermittently in addition to nab-paclitaxel, with the other half receiving nab-paclitaxel monotherapy.
We are conducting a pivotal Phase 3 trial (“ROSELLA”) of our proprietary, selective cortisol modulator, relacorilant combined with nab-paclitaxel as a treatment for patients with platinum-resistant ovarian cancer. Enrollment in ROSELLA is complete. Three hundred eighty-one women with recurrent, platinum-resistant ovarian cancer were randomized 1:1 to receive either 150 mg of relacorilant intermittently in addition to nab-paclitaxel or nab-paclitaxel monotherapy.
Following these compelling results, we initiated a Phase 2b trial (“MONARCH”) of miricorilant in patients with NASH in October 2023. MONARCH has a planned enrollment of 150 patients, randomized 2:1 to receive either 100 mg of miricorilant twice weekly or placebo for 48 weeks.
Cohort B has a planned enrollment of 75 patients with presumed MASH, randomized 2:1 to receive either 100 mg of miricorilant twice weekly for 6 weeks and then 200 mg of miricorilant twice weekly for 18 weeks or placebo for 24 weeks. The primary endpoint of Cohort B is reduction in liver fat.
Most people with Cushing’s syndrome have one or more of the following symptoms: 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.
Most people with hypercortisolism have one or more of the following symptoms: hypertension, diabetes, impaired glucose tolerance, obesity, fatty liver disease, wasting of the arms and legs, edema, fatigue and insomnia. 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.
Cushing’s syndrome can affect every organ system in the body and can be lethal if not treated. The preferred treatment is surgery, which, if successful, can cure the disease. In approximately half of patients, surgery is not successful because the tumor cannot be located or removed completely.
If the tumor can be found, the preferred treatment is surgery, which, if successful, can cure the disease. In approximately half of patients who receive surgery, the procedure is not successful. Depending on the type of tumor, surgery can also result in a range of complications and recurrence of the disease is common. Our Products.
We have discovered more than 1,000 proprietary cortisol modulators that bind to the GR but have no affinity for the PR and so do not cause Korlym’s PR-related side effects.
On December 30, 2024, we submitted to the United States Food and Drug Administration (“FDA”) a New Drug Application (“NDA”) for a proprietary cortisol modulator we have developed, relacorilant, that, like Korlym, modulates the effects of cortisol by binding at the GR, but has no affinity for the PR and so does not cause PR-related side effects.
Liver enzyme levels in all affected patients returned to baseline or below baseline (i.e. to healthier levels) after miricorilant was withdrawn. Our Phase 1b study has identified a dosing regimen that effectively reduced liver fat, improved liver health and key metabolic and lipid measures and was well-tolerated.
Our Phase 1b trial of the selective cortisol modulator miricorilant as a potential treatment for MASH identified a dosing regimen that reduced liver fat, improved liver health and key metabolic and lipid measures and was well-tolerated. Following these compelling results, we initiated a randomized, double-blind, placebo-controlled, Phase 2b trial (“MONARCH”) of miricorilant in patients with MASH in October 2023.
Additionally, Delaware, Indiana, Iowa, Montana, Oregon, Tennessee and Texas have adopted privacy laws, which take effect from July 1, 2024 through 2026. Further, Washington's My Health My Data Act, taking effect July 1, 2024, imposes similar requirements specific to consumer health data. As a result, additional compliance investment and potential business process changes may be required.
Similar laws have also passed in Connecticut and Nevada, which came into effect in 2023 and 2024. As a result, 11 additional compliance investment and potential business process changes may be required.
Removed
Pre-clinical and clinical data suggest that cortisol reduces a patient’s immune response to oncogenesis, shields certain cancer cells from the apoptotic effects of chemotherapy and facilitates the growth of others.
Added
Hypercortisolism is the result of a tumor that produces either cortisol or adrenal corticotropic hormone, a hormone that causes the adrenal glands to produce cortisol. Abnormally high levels of cortisol lead to overstimulation of the GR, which gives rise to a wide range of serious adverse effects.
Removed
COVID-19 Pandemic Public health restrictions and measures voluntarily undertaken by patients, physicians, hospitals and health clinics to reduce the impact of COVID-19 reduced our revenue growth and made it difficult to grow our Korlym business. The pandemic’s impact on our clinical development programs was variable.

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Item 1A. Risk Factors

Risk Factors — what could go wrong, per management

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Biggest changeNew laws, government regulations, or changes to existing laws and regulations could make it difficult or impossible for us to obtain acceptable prices or adequate insurance coverage and reimbursement for Korlym, which would adversely affect our results of operations and financial position. The commercial success of Korlym depends on the availability of acceptable pricing and adequate insurance coverage and reimbursement.
Biggest changeThis may be the case even though (i) our Products are not approved for the termination of pregnancy, (ii) we do not promote it for that use and (iii) we have taken measures to minimize the chance that it will accidentally be prescribed to a pregnant woman. 14 New laws, government regulations, or changes to existing laws and regulations could make it difficult or impossible for us to obtain acceptable prices or adequate insurance coverage and reimbursement for our Products, which would adversely affect our results of operations and financial position.
Each year, the Secretary will select for price negotiation a specified number of negotiation-eligible drugs with the highest total Part B or D expenditures over a preceding 12-month period. To be eligible for price negotiation a drug must have been on the market for at least seven years without generic competition.
Each year, the Secretary will select for price negotiation a specified number of negotiation-eligible drugs with the highest total Part B or D expenditures over the preceding 12-month period. To be eligible for price negotiation a drug must have been on the market for at least seven years without generic competition.
We and our partners are subject to federal, state and foreign laws and regulations concerning data privacy and security, including HIPAA and the EU General Data Protection Regulation (the “GDPR”). These and other regulatory frameworks are evolving rapidly as new rules are enacted and existing ones updated and made more stringent.
We and our partners are subject to federal, state and foreign laws and regulations concerning data privacy and security, including HIPAA and the EU General Data Protection Regulation (“GDPR”). These and other regulatory frameworks are evolving rapidly as new rules are enacted and existing ones updated and made more stringent.
Clinical trials may take longer to complete, cost more than expected and fail for many reasons, including: • failure to show efficacy or acceptable safety; • slow patient enrollment or delayed activation of clinical trial sites; • delays obtaining regulatory permission to start a trial, changes to the size or design of a trial or changes in regulatory requirements for a trial already underway; • inability to secure acceptable terms with vendors and an appropriate number of clinical trial sites; • delays or inability to obtain institutional review board (“IRB”) approval at prospective trial sites; • failure of patients or investigators to comply with the clinical trial protocol; • unforeseen safety issues; and • negative findings of inspections of clinical sites or manufacturing operations by us, the FDA or other authorities.
Clinical trials may take longer to complete, cost more than expected and fail for many reasons, including: • failure to show efficacy or acceptable safety; • slow patient enrollment or delayed activation of clinical trial sites; • delays obtaining regulatory permission to start a trial, changes to the size or design of a trial or changes in regulatory requirements for a trial already underway; • inability to secure acceptable terms with vendors and an appropriate number of clinical trial sites; • delays or inability to obtain institutional review board (“IRB”) approval at prospective trial sites; • failure of patients or investigators to comply with the clinical trial protocol; 19 • unforeseen safety issues; and • negative findings of inspections of clinical sites or manufacturing operations by us, the FDA or other authorities.
If we are found in violation of any of the laws described above or any other government regulations, we may be subject to civil and criminal penalties, damages, fines, exclusion from governmental health care programs, a corporate integrity 17 agreement or other agreement to resolve allegations of non-compliance, individual imprisonment, and the curtailment or restructuring of our operations, any of which could adversely affect our financial results and ability to operate.
If we are found in violation of any of the laws described above or any other government regulations, we may be subject to civil and criminal penalties, damages, fines, exclusion from governmental health care programs, a corporate integrity agreement or other agreement to resolve allegations of non-compliance, individual imprisonment, and the curtailment or restructuring of our operations, any of which could adversely affect our financial results and ability to operate.
If one of our product candidates receives marketing approval, and we or others later identify undesirable side effects or adverse events, potentially significant negative consequences could result, including but not limited to: • regulatory authorities may suspend, limit or withdraw approvals of such product; • regulatory authorities may require additional warnings on the label, including “boxed” warnings, or issue safety alerts and other safety information about the product; • we may be required to change the way the product is administered or conduct additional studies or clinical trials; 19 • we may be required to create a Risk Evaluation and Mitigation Strategy, which could include a medication guide outlining the risks of such side effects for distribution to patients, a communication plan for healthcare providers and/or other elements to assure safe use; • the product may become less competitive; • we may be subject to fines, injunctions or the imposition of criminal penalties; and • we could be sued and held liable for harm caused to patients.
If one of our product candidates receives marketing approval, and we or others later identify undesirable side effects or adverse events, potentially significant negative consequences could result, including but not limited to: 20 • regulatory authorities may suspend, limit or withdraw approvals of such product; • regulatory authorities may require additional warnings on the label, including “boxed” warnings, or issue safety alerts and other safety information about the product; • we may be required to change the way the product is administered or conduct additional studies or clinical trials; • we may be required to create a Risk Evaluation and Mitigation Strategy, which could include a medication guide outlining the risks of such side effects for distribution to patients, a communication plan for healthcare providers and/or other elements to assure safe use; • the product may become less competitive; • we may be subject to fines, injunctions or the imposition of criminal penalties; and • we could be sued and held liable for harm caused to patients.
Please see “Part IV, Item 15, Notes to Consolidated Financial Statements – Income Taxes.” Changes to existing tax laws could materially increase the amounts we pay, which would reduce our after tax net income. Research analysts may not continue to provide or initiate coverage of our common stock or may issue negative reports.
Please see “Part IV, Item 15, Notes to Consolidated Financial Statements – Income Taxes.” Changes to existing tax laws could materially increase the amounts we pay, which would reduce our after tax net income. 25 Research analysts may not continue to provide or initiate coverage of our common stock or may issue negative reports.
Importantly, under the False Claims Act the government may assert that a claim including items or services resulting from a violation of the federal Anti-Kickback Statute constitutes a false or fraudulent claim for purposes of the False Claims Act; • the federal Civil Monetary Penalties law, which prohibits, among other things, offering or transferring remuneration to a federal healthcare beneficiary that a person knows or should know is likely to influence the beneficiary’s decision to order or receive items or services reimbursable by the government from a particular provider or supplier; • HIPAA, which created federal criminal laws that prohibit executing a scheme to defraud any health care benefit program or making false statements relating to health care matters; similar to the federal Anti-Kickback Statute, a person or entity does not need to have actual knowledge of the statute or specific intent to violate it in order to have committed a violation; • federal “sunshine” laws, including the federal Physician Payment Sunshine Act (or sometimes referred to as the Open Payments TM Program), that require transparency regarding financial arrangements with health care providers, such as the reporting and disclosure requirements imposed by the ACA on drug manufacturers regarding any “transfer of value” made or distributed to physicians, certain non-physician practitioners, teaching hospitals, and ownership or investment interests held by physicians and their immediate family members; • federal consumer protection and unfair competition laws, which broadly regulate marketplace activities and activities that potentially harm consumers; • state law equivalents of each of the above federal laws, such as anti-kickback and false claims laws which may apply to items or services reimbursed by any third-party payer, including commercial insurers; state laws that require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government or otherwise restrict payments that may be made to healthcare providers; and • state laws that require drug manufacturers to report information related to payments and other transfers of value to physicians and other healthcare providers or marketing expenditures and pricing information.
Importantly, under the False Claims Act the government may assert that a claim including items or services resulting from a violation of the federal Anti-Kickback Statute constitutes a false or fraudulent claim for purposes of the False Claims Act; • the federal Civil Monetary Penalties law, which prohibits, among other things, offering or transferring remuneration to a federal healthcare beneficiary that a person knows or should know is likely to influence the beneficiary’s decision to order or receive items or services reimbursable by the government from a particular provider or supplier; • HIPAA, which created federal criminal laws that prohibit executing a scheme to defraud any health care benefit program or making false statements relating to health care matters; similar to the federal Anti-Kickback Statute, a person or entity does not need to have actual knowledge of the statute or specific intent to violate it in order to have committed a violation; • federal “sunshine” laws, including the federal Physician Payment Sunshine Act (or sometimes referred to as the Open Payments TM Program), that require transparency regarding financial arrangements with health care providers, such as the reporting and disclosure requirements imposed by the Patient Protection and Affordable Care Act (“ACA”) on drug manufacturers regarding any “transfer of value” made or distributed to physicians, certain non-physician practitioners, teaching hospitals, and ownership or investment interests held by physicians and their immediate family members; • federal consumer protection and unfair competition laws, which broadly regulate marketplace activities and activities that potentially harm consumers; • state law equivalents of each of the above federal laws, such as anti-kickback and false claims laws which may apply to items or services reimbursed by any third-party payer, including commercial insurers; state laws that require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government or otherwise restrict payments that may be made to healthcare providers; and • state laws that require drug manufacturers to report information related to payments and other transfers of value to physicians and other healthcare providers or marketing expenditures and pricing information.
Outside parties may have staffing difficulties, may undergo changes in priorities or may become financially distressed, adversely affecting their willingness or ability to conduct our clinical trials. Problems with the timeliness or quality of the work of a CRO may lead us to seek to terminate the relationship and use an alternative service 18 provider.
Outside parties may have staffing difficulties, may undergo changes in priorities or may become financially distressed, adversely affecting their willingness or ability to conduct our clinical trials. Problems with the timeliness or quality of the work of a CRO may lead us to seek to terminate the relationship and use an alternative service provider.
Failure to accomplish any of these tasks could harm our business. 21 If we lose key personnel or are unable to attract more skilled personnel, we may be unable to pursue our product development and commercialization goals. Our ability to operate successfully and manage growth depends upon hiring and retaining skilled managerial, scientific, sales, marketing and financial personnel.
Failure to accomplish any of these tasks could harm our business. If we lose key personnel or are unable to attract more skilled personnel, we may be unable to pursue our product development and commercialization goals. Our ability to operate successfully and manage growth depends upon hiring and retaining skilled managerial, scientific, sales, marketing and financial personnel.
From January 1, 2021, we have had to comply with the GDPR and separately the United Kingdom GDPR, which, together with the amended United Kingdom Data Protection Act 2018, retains the GDPR in United Kingdom national law, each regime having the ability to fine up to the greater of €20 million/ £17.5 million or 4 percent of global turnover.
From January 1, 2021, we have had to comply with the GDPR and separately the UK GDPR, which, together with the amended United Kingdom Data Protection Act 2018, retains the GDPR in United Kingdom national law, each regime having the ability to fine up to the greater of €20 million/ £17.5 million or 4 percent of global turnover.
Among other activities, we provide promotional materials and training programs to physicians covering the use of Korlym for this indication. The FDA may change its policies or enact new regulations at any time that may restrict our ability to promote our products, which could adversely impact our business.
Among other activities, we provide promotional materials and training programs to physicians covering the use of our Products for this indication. The FDA may change its policies or enact new regulations at any time that may restrict our ability to promote our Products, which could adversely impact our business.
And, although we structure our applicable business arrangements in accordance with the safe harbors, it is difficult to determine exactly how the law will be applied in specific circumstances. Accordingly, it is possible that certain practices of ours may be challenged under the federal 16 Anti-Kickback Statute.
And, although we structure our applicable business arrangements in accordance with the safe harbors, it is difficult to determine exactly how the law will be applied in specific circumstances. Accordingly, it is possible that certain practices of ours may be challenged under the federal Anti-Kickback Statute.
Recent legal developments have added complexity and compliance uncertainty regarding certain transfers of information from the EEA to the United States. Following EU court decisions, updated standard contractual clauses (“SCCs”) were adopted to account for these judicial decisions, imposing new requirements on data transfers.
Legal developments have added complexity and compliance uncertainty regarding certain transfers of information from the EEA to the United States. Following EU court decisions, updated standard contractual clauses (“SCCs”) were adopted to account for these judicial decisions, imposing new requirements on data transfers.
Any insurance we maintain against the risk of this type of loss may not be sufficient to cover actual losses or may not apply to the circumstances relating to any particular loss. 23 Changes in federal, state and local tax laws may reduce our net earnings. Our earnings are subject to federal, state and local taxes.
Any insurance we maintain against the risk of this type of loss may not be sufficient to cover actual losses or may not apply to the circumstances relating to any particular loss. Changes in federal, state and local tax laws may reduce our net earnings. Our earnings are subject to federal, state and local taxes.
Compliance with European data protection laws is a rigorous and time intensive process that may increase our cost of doing business, and despite those efforts, there is a risk that we may be subject to fines and penalties, litigation and reputational harm in connection with our European activities.
Compliance with European data protection laws is a rigorous and time intensive process that may increase our 24 cost of doing business, and despite those efforts, there is a risk that we may be subject to fines and penalties, litigation and reputational harm in connection with our European activities.
The GDPR provides that EEA member states may make their own further laws and regulations limiting the processing of health data, which could limit our ability to 22 use and share personal data or could cause our costs to increase and harm our business and financial condition.
The GDPR provides that EEA member states may make their own further laws and regulations limiting the processing of health data, which could limit our ability to use and share personal data or could cause our costs to increase and harm our business and financial condition.
Discovery of previously unknown problems with a product or product candidate, such as adverse events of unanticipated severity or frequency or deficiencies in manufacturing processes or management, as well as failure to comply with FDA or other U.S. or foreign regulatory requirements, may subject us to substantial civil and criminal penalties, injunctions, holds on clinical trials, product seizure, refusal to permit the import or export of products, restrictions on product marketing, withdrawal of the product from the market, product recalls, total or partial suspension of production, refusal to approve pending new drug applications (“NDAs”) or supplemental NDAs, and suspension or revocation of product approvals.
Discovery of previously unknown problems with a product or product candidate, such as adverse events of unanticipated severity or frequency or deficiencies in manufacturing processes or management, as well as failure to comply with current or future FDA or other U.S. or foreign regulatory requirements, may subject us to substantial civil and criminal penalties, injunctions, holds on clinical trials, product seizure, refusal to permit the import or export of products, restrictions on product marketing, withdrawal of the product from the market, product recalls, total or partial suspension of production, refusal to approve pending new drug applications (“NDAs”) or supplemental NDAs, and suspension or revocation of product approvals.
There may be others of which we are unaware that could materially harm our business or financial condition and cause the price of our stock to decline, in which case you could lose all or part of your investment.
There may be others of 12 which we are unaware that could materially harm our business or financial condition and cause the price of our stock to decline, in which case you could lose all or part of your investment.
Other federal or state enforcement authorities might act if they believe that the alleged improper promotion led to the submission and payment of claims for an unapproved use, which could result in significant fines or penalties under other statutory authorities, such as laws prohibiting false claims for reimbursement.
Federal or state enforcement authorities may act if they believe that the alleged improper promotion led to the submission and payment of claims for an unapproved use, which could result in significant fines or penalties under other statutory authorities, such as laws prohibiting false claims for reimbursement.
Even if we deem that our product candidates' clinical trial results demonstrate safety and efficacy, regulatory authorities may not agree. Failure to obtain or maintain regulatory approvals for our product candidates would prevent us from commercializing them. Clinical development is costly, time-consuming and unpredictable.
Even if we deem that our product candidates ’ clinical trial results demonstrate safety and efficacy, regulatory authorities may not agree. Failure to obtain or maintain regulatory approvals for our product candidates would prevent us from commercializing them. Clinical development is costly, time-consuming and unpredictable.
Also, the trends toward managed health care in the United States and recent laws and legislation intended to increase the public visibility of drug prices and reduce the cost of government and private insurance programs could significantly influence the purchase of health care services and products and may result in lower prices for Korlym.
Also, the trends toward managed health care in the United States and recent laws and legislation intended to increase the public visibility of drug prices and reduce the cost of government and private insurance programs could significantly influence the purchase of health care services and products and may result in lower prices for our Products.
We are dependent on revenue from the sale of Korlym and our cash reserves to fund our commercial operations and development programs. If Korlym revenue declines significantly, we may need to curtail our operations or raise funds to support our plans. We may also choose to raise funds for strategic reasons.
We are dependent on revenue from the sale of our Products and our cash reserves to fund our commercial operations and development programs. If our revenue declines significantly, we may need to curtail our operations or raise funds to support our plans. We may also choose to raise funds for strategic reasons.
On March 6, 2020, the FDA granted Recordati approval to market another cortisol synthesis inhibitor, Isturisa® (osilodrostat) tablets, to treat patients with Cushing’s disease. Osilodrostat is approved in the EU for the treatment of patients with Cushing’s syndrome.
On March 6, 2020, the FDA granted Recordati approval to market another cortisol synthesis inhibitor, Isturisa® (osilodrostat) tablets, to treat patients with Cushing’s disease. Osilodrostat is approved in the EU for the treatment of patients with hypercortisolism.
The facilities used by our vendors to manufacture and package the API and drug product for Korlym and our product candidates and distribute them to hospitals, clinics and patients, must be approved by government regulators in the United States, Europe, and elsewhere.
The facilities used by our vendors to manufacture and package the API and drug product for our Products and product candidates and distribute them to hospitals, clinics and patients, must be approved by government regulators in the United States, Europe, and elsewhere.
If government or private 13 payers cease to provide adequate and timely coverage, pricing and reimbursement for Korlym, physicians may not prescribe the medication and patients may not purchase it, even if it is prescribed, or the price we receive may be reduced, which would reduce our revenue.
If government or private payers cease to provide adequate and timely coverage, pricing and reimbursement for our Products, physicians may not prescribe the medication and patients may not purchase it, even if it is prescribed, or the price we receive may be reduced, which would reduce our revenue.
We may be subject to civil or criminal penalties if our marketing of Korlym violates FDA regulations or health care fraud and abuse laws. We are subject to FDA regulations governing the promotion and sale of medications. Although physicians are permitted to prescribe drugs for any indication they choose, manufacturers may only promote products for their FDA-approved use.
We may be subject to civil or criminal penalties if our marketing of our Products violates FDA regulations or health care fraud and abuse laws. We are subject to statutes and regulations governing the promotion and sale of medicine. Although physicians are permitted to prescribe drugs for any indication they choose, manufacturers may only promote products for their FDA-approved use.
If Optime does not adhere to its agreements with payers or does not continue to meet regulatory requirements concerning pharmacy operations, it may not be able to collect some or all of the payments due to us.
If Optime does not adhere to its agreements with payers or does not continue to meet regulatory requirements concerning pharmacy operations, it may not be able to collect on our behalf some or all of the payments due to us.
Vendors perform many of the activities necessary to carry out our clinical trials, including drug product distribution, trial management and oversight and data collection and analysis. Failure of these vendors to perform their duties or meet expected timelines may prevent or delay approval of our product candidates.
Risks Related to our Research and Development Activities Vendors perform many of the activities necessary to carry out our clinical trials, including drug product distribution, trial management and oversight and data collection and analysis. Failure of these vendors to perform their duties or meet expected timelines may prevent or delay approval of our product candidates.
Risks Related to our Research and Development Activities • Our efforts to discover, develop and commercialize our product candidates may not succeed. Clinical drug development is lengthy, expensive and often unsuccessful. Results of early studies and trials are often not predictive of later trial results. Failure can occur at any time.
Our efforts to discover, develop and commercialize our product candidates may not succeed. Clinical drug development is lengthy, expensive and often unsuccessful. Results of early studies and trials are often not predictive of later trial results. Failure can occur at any time.
If our suppliers become unable or unwilling to perform these functions and we cannot transfer these activities to replacement vendors in a timely manner, our business will be harmed.
If our vendors become unable or unwilling to perform these functions and we cannot transfer these activities to other vendors in a timely manner, our business will be harmed.
As supervisory authorities issue further guidance on personal data export mechanisms, including circumstances where the SCCs cannot be used, and/or start taking enforcement action, we could suffer additional costs, complaints and/or regulatory investigations or fines, and/or if we are otherwise unable to transfer personal data between and among countries and regions in which we operate, it could affect the manner in which we provide our services, the geographical location or segregation of our relevant systems and operations, and could adversely affect our financial results.
As supervisory authorities issue further guidance on personal data export mechanisms, and/or start taking enforcement action, we could suffer additional costs, complaints and/or regulatory investigations or fines, and/or if we are otherwise unable to transfer personal data between and among countries and regions in which we operate, it could affect the manner in which we provide our services, the geographical location or segregation of our relevant systems and operations, and could adversely affect our financial results.
If we are unable to maintain regulatory approval of Korlym or if we fail to comply with other requirements, we will be unable to generate revenue and may be subject to penalties.
If we are unable to maintain regulatory approval of our Products or if we fail to comply with other requirements, we will be unable to generate revenue and may be subject to penalties.
As of February 6, 2024, our officers, directors and principal stockholders beneficially owned approximately 20 percent of our common stock. Our stock price can experience extreme price and volume fluctuations that are unrelated or disproportionate to our operating performance or prospects. Securities class action lawsuits are often instituted against companies following periods of stock market volatility.
As of February 18, 2025, our officers, directors and principal stockholders beneficially owned approximately 21 percent of our common stock. 21 Our stock price can experience extreme price and volume fluctuations that are unrelated or disproportionate to our operating performance or prospects. Securities class action lawsuits are often instituted against companies following periods of stock market volatility.
All other uses are referred to as “off-label,” manufacturers are prohibited from engaging in any “off-label” promotion. In the United States, we market Korlym to treat hyperglycemia secondary to hypercortisolism in adult patients with endogenous Cushing’s syndrome who have type 2 diabetes mellitus or glucose intolerance and for whom surgery has failed or is not an option.
All other uses are referred to as “off-label”; manufacturers are prohibited from engaging in any “off-label” promotion. In the United States, we market our Products to treat hyperglycemia secondary to hypercortisolism in adult patients with endogenous hypercortisolism who have type 2 diabetes mellitus or glucose intolerance and for whom surgery has failed or is not an option.
If the FDA were to determine that we engaged in off-label promotion, the FDA could require us to change our practices and subject us to regulatory enforcement actions, including issuance of a public “warning letter,” untitled letter, injunction, seizure, civil fine or criminal penalties.
If the FDA or a law enforcement agency were to determine that we engaged in off-label promotion, we could be required to change our practices and be subject to regulatory enforcement actions, including issuance of a public “warning letter,” untitled letter, injunction, seizure, civil fine or criminal penalties.
Many factors could limit our Korlym revenue, including: • the preference of some physicians for competing treatments for Cushing’s syndrome, including off-label treatments and generic versions of Korlym; and • lack of availability of government or private insurance, the shift of a significant number of patients to Medicaid, which reimburses Korlym at a significantly lower price, or the introduction of government price controls or other price-reducing regulations, such as the Inflation Reduction Act of 2022, that may significantly limit Medicare reimbursement rates.
Many factors could limit our product revenue, including: • the preference of physicians or payors for competing treatments for hypercortisolism, including a lower-priced generic version of Korlym and off-label treatments; and • lack of availability of government or private insurance, the shift of a significant number of patients to Medicaid, which reimburses Korlym at a significantly lower price, or the introduction of government price controls or other price-reducing regulations, such as the Inflation Reduction Act of 2022, that may significantly limit Medicare reimbursement rates.
Competition from a generic version of Korlym may also cause our revenue to be materially less than the public guidance we have provided, which would likely cause the price of our common stock to decline. Legal action to enforce or defend intellectual property rights is complex, costly and involves significant commitments of management time.
It may also cause our revenue to be materially less than the public guidance we have provided, which would likely cause the price of our common stock to decline. Legal action to enforce or defend intellectual property rights is complex, costly and involves significant commitments of management time.
The active ingredient in Korlym, mifepristone, is approved by the FDA in another drug for the termination of early pregnancy. On June 24, 2022, the United States Supreme Court published its decision in the case of Dobbs v. Jackson Women’s Health Organization (“Dobbs”), which overturned Roe v.
The active ingredient in our Products, mifepristone, is approved by the FDA in another drug for the termination of early pregnancy. In 2022, the United States Supreme Court published its decision in the case of Dobbs v. Jackson Women’s Health Organization (“Dobbs”), which overturned Roe v.
Department of Health and Human Services (“HHS”) to negotiate Medicare prices for selected drugs and biologicals, including both physician-administered products covered under Medicare’s Part B benefit and self-administered drugs covered under the Part D benefit.
Department of Health and Human Services (“HHS”) to negotiate Medicare prices for selected drugs and biologicals, including both physician-administered products covered under Medicare’s Part B benefit and self-administered drugs such as our Products that are covered under the Part D benefit.
Factors that may cause the price of our common stock to fluctuate rapidly and widely include: • actual or anticipated variations in our operating results or changes to any public guidance we have provided; • actual or anticipated timing and results of our clinical trials; 20 • actual or anticipated regulatory approvals of our product candidates; • disputes or other developments relating to our intellectual property, including developments in Abbreviated New Drug Application litigation; • changes in laws or regulations applicable to the pricing, availability of insurance reimbursement, or approved uses of Korlym, our product candidates or our competitors’ products; • short-selling of our common stock, the publication of speculative opinions about our business or other market manipulation activities that are intended to lower our stock price or increase its volatility; • changes in estimates or recommendations by securities analysts or the failure of our performance to meet the published expectations of those analysts or public guidance we have provided; • purchases of our common stock pursuant to our Stock Repurchase Program or changes to that program; • general market and economic conditions, including the effects of the COVID-19 pandemic; • changes in the expected or actual timing of our competitors’ development programs and the approval of competing products; • purchases or sales of our common stock by our officers, directors or stockholders; • technological innovations by us, our collaborators or our competitors; • conditions in the pharmaceutical industry, including the market valuations of companies similar to ours; • additions or departures of key personnel; • announcements by us or our competitors of significant acquisitions, strategic partnerships, joint ventures, collaborations or capital commitments; and • additional financing activities.
Factors that may cause the price of our common stock to fluctuate rapidly and widely include: • actual or anticipated variations in our operating results or changes to any public guidance we have provided; • actual or anticipated timing and results of our clinical trials; • actual or anticipated regulatory approvals of our product candidates; • disputes or other developments relating to our intellectual property, including developments in generic-related litigation; • changes in laws or regulations applicable to the pricing, availability of insurance reimbursement, or approved uses of our commercialized products, our product candidates or our competitors’ products; • short-selling of our common stock, the publication of negative opinions about our business or other market manipulation activities that are intended to lower our stock price or increase its volatility; • sales of a substantial number of shares of our stock in the public market, leading to reductions in its price; • changes in estimates or recommendations by securities analysts or the failure of our performance to meet the published expectations of those analysts or public guidance we have provided; • purchases of our common stock pursuant to our stock repurchase program (the “Stock Repurchase Program”) or changes to that program; • general market and economic conditions; • changes in the expected or actual timing of our competitors’ development programs and the approval of competing products; • purchases or sales of our common stock by our officers, directors or stockholders; • technological innovations by us, our collaborators or our competitors; • conditions in the pharmaceutical industry, including the market valuations of companies similar to ours; • additions or departures of key personnel; • announcements by us or our competitors of significant acquisitions, strategic partnerships, joint ventures, collaborations or capital commitments; and • additional financing activities.
As a result of this scrutiny, these assistance programs and charities may decide to reduce or eliminate entirely the assistance they provide to patients, which could result in fewer patients receiving the financial support they need to cover the cost of their Cushing’s syndrome care, including the cost of medication, which may include Korlym.
As a result of this scrutiny, these assistance programs and charities may decide to reduce or eliminate entirely the assistance they provide to patients, which could result in fewer patients receiving the financial support they need to cover the cost of their hypercortisolism care, including the cost of medication, which may include one of our Products.
Our agreement with Optime extends to March 31, 2024, subject to customary termination provisions, including the right of Optime to terminate in the event of a material breach by us that we do not cure in a reasonable period of time after receiving written notice. In addition, we may terminate the agreement for convenience.
The agreement is subject to customary termination provisions, including the right of Optime to terminate in the event of a material breach by us that we do not cure in a reasonable period of time after receiving written notice. In addition, we may terminate the agreement for convenience.
We do not control the activities of these vendors, including whether they maintain adequate quality control and hire qualified personnel. We are dependent on them for compliance with the regulatory requirements known as current good manufacturing practices (“cGMPs”).
We do not control the activities of these vendors, including whether they maintain adequate quality control and hire qualified personnel. We are dependent on them for compliance with the regulatory requirements known as current good manufacturing practices (“cGMPs”), which are subject to change at the regulators’ discretion.
The foreign countries where we may someday operate may not protect our intellectual property to the extent the laws of the United States do. If we fail to obtain adequate patent protection in other countries, others may produce products in those countries based on our technology.
Our patents may not prevent third parties from producing competing products. The foreign countries where we may someday operate may not protect our intellectual property to the extent the laws of the United States do. If we fail to obtain adequate patent protection in other countries, others may produce products in those countries based on our technology.
The patents issued or licensed to us may be challenged at any time. Competitors may take actions we believe infringe our intellectual property, causing us to take legal action to defend our rights. Intellectual property litigation is lengthy, expensive and requires significant management attention. Outcomes are uncertain.
Patents are uncertain, involve complex legal and factual questions and are frequently the subject of litigation. The patents issued or licensed to us may be challenged at any time. Competitors may take actions we believe infringe our intellectual property, causing us to take legal action to defend our rights. Intellectual property litigation is lengthy, expensive and requires significant management attention.
Companies that are subject to unfavorable allegations, even if untrue, may have to expend a significant amount of resources to investigate such allegations and/or defend themselves, including shareholder suits against the company that may be prompted by such allegations.
Companies that are subject to unfavorable allegations, even if untrue, may have to expend a significant amount of resources to investigate such allegations and/or defend themselves, including shareholder suits against the company that may be prompted by such allegations. We have been, and may in the future be, the subject of shareholder suits prompted by allegations made by short sellers.
Our single specialty pharmacy, Optime, dispenses Korlym and performs related pharmacy and patient support services, including the collection of payments from insurers representing approximately 99 percent of our revenue.
Our single specialty pharmacy, Optime, dispenses our Products and performs related pharmacy and patient support services, including the collection of payments from insurers representing more than 99 percent of our revenue.
Acquisition of Corcept shares through our stock repurchase program will reduce our cash reserves. In January 2024, our Board of Directors authorized the repurchase of up to $200 million of our common stock pursuant to the Stock Repurchase Program.
Any acquisition of Corcept shares through our stock repurchase program or, in certain cases, pursuant to the exercise of stock options, will reduce our cash reserves. In January 2024, our Board of Directors authorized the repurchase of up to $200 million of our common stock pursuant to the Stock Repurchase Program.
In the event any of our vendors fails to perform its contractual obligations to us or is materially impaired in its performance, we may experience disruptions and delays in our supply chain and our ability to deliver Korlym to patients, which would adversely affect our business, results of operations and financial position.
In the event any of our vendors fails to perform its contractual obligations to us or is materially impaired in its performance, we may experience disruptions and delays in our ability to deliver our commercialized products to patients or investigational drugs to patients in our clinical trials, which would adversely affect our business, results of operations and financial position.
If we do not protect our intellectual property, competitors may erode our competitive advantage. Please see “Part I, Item 3, Legal Proceedings” for additional information. Our patent applications may not result in issued patents and patents issued to us may be challenged, invalidated, held unenforceable or circumvented. Our patents may not prevent third parties from producing competing products.
Outcomes are uncertain. If we do not protect our intellectual property, competitors may erode our competitive advantage. Please see “Part I, Item 3, Legal Proceedings” for additional information. Our patent applications may not result in issued patents and patents issued to us may be challenged, invalidated, held unenforceable or circumvented.
To date, we have relied on a small management team. Growth will impose significant added responsibilities on members of management, including the need to recruit and retain additional employees. Our financial performance and ability to compete will depend on our ability to manage growth effectively.
Growth will impose significant added responsibilities on members of management, including the need to recruit and retain additional employees. Our financial performance and ability to compete will depend on our ability to manage growth effectively.
Our officers, directors and principal stockholders, acting as a group, could significantly influence corporate actions. As of February 6, 2024, our officers and directors beneficially owned approximately 20 percent of our common stock.
Our officers, directors and principal stockholders, acting as a group, could significantly influence corporate actions. As of February 18, 2025, our officers and directors beneficially owned approximately 21 percent of our common stock.
Patients may be unwilling or unable to travel to clinical trial sites, for example, or clinical materials or data may be lost. Our insurance, if available at all, would likely be insufficient to cover losses resulting from disasters or other business interruptions. 15 We may not have adequate insurance to cover our exposure to product liability claims.
Patients may be unwilling or unable to travel to clinical trial sites, for example, or clinical materials or data may be lost. Our insurance, if available at all, would likely be insufficient to cover losses resulting from disasters or other business interruptions.
Sanctions could be imposed on us, including fines, injunctions, civil penalties, refusal of regulators to approve our product candidates, delays, suspensions or withdrawals of approvals, seizures or recalls of products, operating restrictions and criminal prosecutions, any of which could harm our business. The unfavorable public perception of mifepristone may limit our ability to sell Korlym.
Sanctions could be imposed on us, including fines, injunctions, civil penalties, refusal of regulators to approve our product candidates, delays, suspensions or withdrawals of approvals, seizures or recalls of products, operating restrictions and criminal prosecutions, any of which could harm our business.
Since 2012, a medication owned by the Italian pharmaceutical company Recordati-S.p.A., the somatostatin analogue Signifor ® (pasireotide) Injection, has been marketed in both the United States and the EU for adult patients with Cushing’s disease (a subset of Cushing’s syndrome).
The availability of competing treatments could limit our product revenue. Since 2012, a medication owned by the Italian pharmaceutical company Recordati-S.p.A., the somatostatin analogue Signifor® (pasireotide) Injection, has been marketed in both the United States and the EU for adult patients with Cushing’s disease (a subset of hypercortisolism).
Depending on the facts and circumstances, we could be subject to criminal penalties if we knowingly obtain, use, or disclose individually identifiable health information maintained by a HIPAA-covered entity in a manner that is not authorized or permitted by HIPAA.
Depending on the facts and circumstances, we could be subject to criminal penalties if we knowingly obtain, use, or disclose individually identifiable health information maintained by a HIPAA-covered entity in a manner that is not authorized or permitted by HIPAA. Requirements for compliance under HIPAA are also subject to change, 23 as the U.S.
There can be no assurance of a successful outcome. We have sued Teva in Federal District Court with respect to its proposed generic versions of Korlym. On December 29, 2023, the Court issued a ruling in that case finding that Teva’s generic product would not infringe the patents we have asserted against Teva.
We have sued Teva in Federal District Court with respect to its generic version of Korlym. On December 29, 2023, the Court issued a ruling in that case finding that Teva’s generic product would not infringe the patents we have asserted against it.
The Stock Repurchase Program does not require us to acquire any specific number of shares and it may be modified, suspended or discontinued at any time without notice. It is possible that other uses of our capital would have been more advantageous or that our future capital requirements increase unexpectedly.
Furthermore, the Stock Repurchase Program may be modified, suspended or discontinued at any time without notice. It is possible that other uses of our capital would have been more advantageous or that our future capital requirements increase unexpectedly.
On December 30, 2021, Xeris received FDA approval to market the cortisol synthesis inhibitor Recorlev ® (levoketoconazole) to treat patients with Cushing’s syndrome in the United States. Levoketoconazole is an enantiomer of the generic anti-fungal medication, ketoconazole, that is prescribed off-label to treat patients with Cushing’s syndrome.
On December 30, 2021, Xeris received FDA approval to market the cortisol synthesis inhibitor Recorlev® (levoketoconazole) to treat patients with hypercortisolism in the United States. Levoketoconazole is an enantiomer of the generic anti-fungal medication, ketoconazole, that is prescribed off-label to treat patients with hypercortisolism. Osilodrostat and levoketoconazole have been designated orphan drugs in both the EU and the United States.
As a result, holders of our common stock may not be able to sell shares quickly or at the current market price. During the 52-week period ended February 6, 2024, our average daily trading volume was approximately 902,095 shares and the intra-day sales prices per share of our common stock on The Nasdaq Stock Market ranged from $17.86 to $34.28.
As a result, holders of our common stock may not be able to sell shares quickly or at the current market price. During the 52-week period ended February 18, 2025, our average daily trading volume was approximately 970,395 shares and the intra-day sales prices per share of our common stock on The Nasdaq Stock Market ranged from $20.84 to $74.61.
To that end, we must: • manage our sales and marketing efforts, clinical trials, research and manufacturing activities effectively; • hire more management, clinical development, administrative and sales and marketing personnel; and • continue to develop our administrative systems and controls.
To that end, we must: • continue to add talented, experienced personnel to our endocrine, oncology and emerging markets businesses; • manage our clinical trials, research and manufacturing activities effectively; • hire more general management, clinical development, administrative and sales and marketing personnel; and • continue to develop our administrative systems and controls.
We depend on vendors to manufacture Korlym’s active ingredient, form it into tablets, package it and dispense it to patients. We also depend on vendors to manufacture the active pharmaceutical ingredient (“API”) and capsules or tablets for our product candidates.
We depend on vendors to manufacture the active pharmaceutical ingredient (“API”) and capsules or tablets for our commercialized products as well as our product candidates. We also depend on vendors to package our products and dispense them to patients.
In the event that we are subject to or affected by HIPAA, the CCPA, the CPRA or other domestic privacy and data protection laws, any liability from failure to comply with the requirements of these laws could adversely affect our financial condition.
As a result, additional compliance investment and potential business process changes may be required. In the event that we are subject to or affected by HIPAA, the CCPA or other domestic privacy and data protection laws, any liability from failure to comply with the requirements of these laws could adversely affect our financial condition.
Market entry by Sun or Hikma could materially harm our results of operations and financial condition, even if our on-going appeal is successful and they were required to withdraw its product and pay us damages. Please see “Part I, Item 3, Legal Proceedings” for additional details.
The availability of generic versions of Korlym from Sun or Hikma could materially harm our results of operations and financial condition, even if our on-going appeal against Teva is successful and Teva, Sun and Hikma were required to withdraw their products and pay us damages. Please see “Part I, Item 3, Legal Proceedings” for additional details.
Osilodrostat and levoketoconazole have been designated orphan drugs in both the EU and the United States. Physician preference for any of these medications, or for the off-label use of generic medications such as ketoconazole, to treat patients with Cushing’s syndrome could reduce our revenue materially and harm our results of operations, which would cause our stock price to decline.
Physician preference for any of these medications, or for the off-label use of generic medications such as ketoconazole, to treat patients with hypercortisolism could reduce our revenue materially and harm our results of operations, which would cause our stock price to decline.
In 2022, the FTC also began a rulemaking proceeding to develop additional data privacy rules and requirements, which may add additional complexity to compliance obligations going forward.
In 2024, the FTC also finalized its rulemaking on additional data privacy rules and requirements, which may add additional complexity to compliance obligations going forward.
Natural disasters, such as earthquakes, fires, extreme weather events or widespread outbreaks of a deadly disease such as COVID-19, could disrupt our commercial and clinical activities or damage or destroy clinical trial sites, our office spaces, the residences of our employees or the facilities or residences of our vendors, contractors or consultants, which could significantly harm our operations.
In addition, our reputation as a reliable sponsor of clinical studies would be harmed, which would make it more difficult for us to develop our drug candidates. 16 Natural disasters, such as earthquakes, fires, extreme weather events or widespread outbreaks of a deadly disease such as COVID-19, could disrupt our commercial and clinical activities or damage or destroy clinical trial sites, our office spaces, the residences of our employees or the facilities or residences of our vendors, contractors or consultants, which could significantly harm our operations.
Failure to generate sufficient Korlym revenue could prevent us from fully funding our planned commercial and clinical activities and would likely cause our stock price to decline. 12 If a generic version of Korlym is successfully commercialized, our business, results of operations and financial position would be adversely affected.
Failure to generate sufficient product revenue could prevent us from fully funding our planned commercial and clinical activities and would likely cause our stock price to decline. The availability of generic versions of Korlym could adversely affect our business, results of operations and financial position. In January 2024, Teva launched a generic version of Korlym.
Risks Related to our Commercial Activities • Failure to generate sufficient revenue from the sale of Korlym would harm our financial results and would likely cause our stock price to decline. 11 • If a generic version of Korlym is successfully commercialized, our business, results of operations and financial position would be adversely affected. • New laws, government regulations, or changes to existing laws and regulations could make it difficult or impossible for us to obtain acceptable prices or adequate insurance coverage and reimbursement for Korlym, which would adversely affect our results of operations and financial position.
Risks Related to our Commercial Activities • Failure to generate sufficient revenue from the sale of our Products would harm our financial results and would likely cause our stock price to decline. • The availability of generic versions of Korlym could adversely affect our business, results of operations and financial position. • Public perception of mifepristone or legislation limiting or barring its distribution or use for termination of early pregnancy may limit our ability to sell our Products. • New laws, government regulations, or changes to existing laws and regulations could make it difficult or impossible for us to obtain acceptable prices or adequate insurance coverage and reimbursement for our Products, which would adversely affect our results of operations and financial position.
Opportunities for investors to sell shares may be limited. • Our stock price may decline if our financial performance does not meet the guidance we have provided to the public, estimates published by research analysts or other investor expectations. General Risks • We rely on information technology to conduct our business.
Risks Related to our Stock • The price of our common stock fluctuates widely and is likely to continue to do so. Opportunities for investors to sell shares may be limited. • Our stock price may decline if our performance does not meet the guidance we have provided to the public, estimates published by research analysts or other investor expectations.
The revenue guidance we provide may be one factor they consider when determining their estimates. General Risk Factors We need to increase the size of our organization and may experience difficulties in managing growth. Our commercial and research and development efforts are constrained by our limited administrative, operational and management resources.
General Risk Factors We need to increase the size of our organization and may experience difficulties in managing growth. Our commercial and research and development efforts are constrained by our limited administrative, operational and management resources. To date, we have relied on a small management team.
A breakdown or breach of our information technology systems or our failure to protect confidential information concerning our business, patients or employees could interrupt the operation of our business and subject us to liability. Risk Factors – Discussion The following section discusses the principal risks listed above, as well as other risks we believe to be material.
General Risk Factors • We rely on information technology to conduct our business. A breakdown or breach of our information technology systems or our failure to protect confidential information concerning our business, patients or employees could interrupt the operation of our business and subject us to liability.
Further, the California Consumer Privacy Act, or the CCPA, which took effect on January 1, 2020, created individual privacy rights for California consumers and increased the privacy and security obligations of entities handling certain personal information.
Further, the California Consumer Privacy Act, which took effect on January 1, 2020, and was later revised and expanded by the California Privacy Rights Act, collectively the CCPA, created individual privacy rights for California consumers and increased the privacy and security obligations of entities handling certain personal information as well as limitation on data uses, audit requirements for higher risk data, and opt outs for certain uses of sensitive data.
These requirements include submissions of safety information, annual updates on manufacturing activities and continued compliance with FDA regulations, including cGMPs, good laboratory practices and good clinical practices (“GCPs”). The FDA enforces these regulations through inspections of us and the laboratories, manufacturers and clinical sites we use. Foreign regulatory authorities have comparable requirements and enforcement mechanisms.
These requirements include submissions of safety information, annual updates on manufacturing activities and continued compliance with FDA regulations, including cGMPs, good laboratory practices and good clinical practices (“GCPs”), all of which are subject to change without notice and at the regulators’ sole discretion. Foreign regulatory authorities have comparable requirements and enforcement mechanisms, which are also subject to change.
On June 28, 2021, the EC adopted an adequacy decision in favor of the United Kingdom, enabling data transfers from EU member states to the United Kingdom without additional safeguards. However, the United Kingdom adequacy decision will automatically expire in June 2025 unless the EC renews or extends that decision and remains under review by the Commission during this period.
However, the United Kingdom adequacy decision will automatically expire in June 2025 unless the EC renews or extends that decision and remains under review by the Commission during this period.
In the United States, we expect that there will continue to be federal and state proposals for similar controls.
In many foreign markets, drug prices and the profitability of prescription medications are subject to government control. In the United States, we expect that there will continue to be federal and state proposals for similar controls.
We do not have employment insurance covering any of our personnel. The loss of key individuals could delay our research, development and commercialization efforts. We are subject to government regulation and other legal obligations relating to privacy and data protection. Compliance with these requirements is complex and costly. Failure to comply could materially harm our business.
We do not have employment insurance covering any of our personnel. The loss of key individuals could delay our research, development and commercialization efforts. We are subject to regulations and other legal obligations relating to drug development and commercialization, the conduct of business as an issuer of publicly traded securities and individual privacy and data protection.
Risks Related to our Research and Development Activities Our efforts to discover, develop and commercialize our product candidates may not succeed. Clinical drug development is lengthy, expensive and often unsuccessful. Results of early studies and trials are often not predictive of later trial results. Failure can occur at any time.
Failure of these vendors to perform their duties or meet expected timelines may prevent or delay approval of our product candidates. • Our efforts to discover, develop and commercialize our product candidates may not succeed. Clinical drug development is lengthy, expensive and often unsuccessful. Results of early studies and trials are often not predictive of later trial results.
Even if it is determined that we are not in violation of these laws, we may receive negative publicity, incur significant expenses and be forced to devote management time to defending our position.
Even if it is determined that we are not in violation of these laws, we may receive negative publicity, incur significant expenses and be forced to devote management time to defending our position. 17 In addition to laws prohibiting off-label promotion, we are also subject to federal and state healthcare fraud and abuse laws and regulations designed to prevent fraud, kickbacks, self-dealing and other abusive practices.

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Item 1C. Cybersecurity

Cybersecurity — threats and controls disclosure

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Biggest changeSee “ Risk Factors – General Risk Factors ” for additional information about the risks to our business associated with a breach or compromise to our information security systems. ITEM 2. PROPERTIES We lease 50,777 square feet of office space in Menlo Park, California for our corporate facilities. Our current lease expires in June 2024.
Biggest changeSee “ Risk Factors – General Risk Factors ” for additional information about the risks to our business associated with a breach or compromise to our information security systems. ITEM 2. PROPERTIES We lease 50,632 square feet of office space in Redwood City, California for our corporate facilities. Our current lease expires in June 2030.

Item 3. Legal Proceedings

Legal Proceedings — active lawsuits and investigations

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Biggest changeThe complaint named our board of directors, Chief Executive Officer and current Chief Business Officer as defendants, and us as nominal defendant. The complaint alleges causes of action for breach of fiduciary duty, violation of Section 14(a) of the Exchange Act, waste of corporate assets, contribution and indemnification, aiding and abetting, and gross 26 mismanagement.
Biggest changeThese complaints named the then-existing members of our board of directors, our Chief Executive Officer and our current Chief Business Officer as defendants, and Corcept as a nominal defendant.
In November 2021, we received a records subpoena from the United States Attorney’s Office for the District of New Jersey (the “NJ USAO”) pursuant to Section 248 of the Health Insurance Portability and Accountability Act of 1996 (“HIPAA”) seeking information relating to the sale and promotion of Korlym, our relationships with and payments to health care professionals who can prescribe or recommend Korlym and prior authorizations and reimbursement for Korlym.
Records Subpoena In November 2021, we received a records subpoena from the United States Attorney’s Office for the District of New Jersey (the “NJ USAO”) pursuant to Section 248 of the Health Insurance Portability and Accountability Act of 1996 (“HIPAA”) seeking information relating to the sale and promotion of Korlym, our relationships with and payments to health care professionals who can prescribe or recommend Korlym and prior authorizations and reimbursement for Korlym.
Although the outcome of any such matters and the amount, if any, of our liability with respect to them cannot be predicted with certainty, we do not believe that they will have a material adverse effect on our business, results of operations or financial position. ITEM 4. MINE SAFETY DISCLOSURES Not applicable. 27 PART II
Although the outcome of any such matters and the amount, if any, of our liability with respect to them cannot be predicted with certainty, we do not believe that they will have a material adverse effect on our business, results of operations or financial position. ITEM 4. MINE SAFETY DISCLOSURES Not applicable. 29 PART II
ITEM 3. LEGAL PROCEEDINGS Teva Litigation In February 2018, we received a Paragraph IV Notice Letter advising that Teva Pharmaceuticals USA, Inc.
ITEM 3. LEGAL PROCEEDINGS Teva Patent Litigation In February 2018, we received a Paragraph IV Notice Letter advising that Teva Pharmaceuticals USA, Inc.
The complaint named us and certain of our executive officers as defendants asserting violations of Sections 10(b) and 20(a) of the Exchange Act and Rule 10b-5 promulgated thereunder and alleges that the defendants made false and materially misleading statements and failed to disclose adverse facts about our business, operations and prospects.
The complaint named us and certain of our executive officers as defendants asserting violations of Sections 10(b) and 20(a) of the Exchange Act and Rule 10b-5 promulgated thereunder and alleged that the defendants made false and materially misleading statements and failed to disclose adverse facts about our business, operations and prospects.
In December 2019, a second purported shareholder derivative complaint was filed in the United States District Court for the District of Delaware by Jeweltex Pension Plan, captioned Jeweltex Pension Plan v. James N. Wilson, et al. , Civil Action No. 1:19-cv-02308.
A second nearly identical lawsuit was filed in December 2019 in the United States District Court for the District of Delaware by Jeweltex Pension Plan, captioned Jeweltex Pension Plan v. James N. Wilson, et al. , Civil Action No. 1:19-cv-02308.
In September 2019, a purported shareholder derivative complaint was filed in the United States District Court for the District of Delaware by Lauren Williams, captioned Lauren Williams v. G. Leonard Baker, et al. , Civil Action No. 1:19-cv-01830. The complaint named our board of directors, Chief Executive Officer and current Chief Business Officer as defendants, and us as nominal defendant.
In September 2019, a purported shareholder derivative complaint was filed in the United States District Court for the District of Delaware by Lauren Williams, captioned Lauren Williams v. G. Leonard Baker, et al. , Civil Action No. 1:19-cv-01830.
The complaint alleges breach of fiduciary duty, violation of Section 14(a) of the Exchange Act, insider selling, misappropriation of insider information and waste of corporate assets and seeks damages in an amount to be proved at trial. In October 2019, this action was stayed pending a resolution of our motions to dismiss the Melucci litigation.
The complaints allege breach of fiduciary duty, violation of Section 14(a) of the Exchange Act, insider selling, misappropriation of insider information and waste of corporate assets and seek damages in an amount to be proved at trial. These actions had been stayed pending resolution of the Melucci litigation.
In May 2019, Teva 25 submitted to the Patent Trial and Appeal Board (“PTAB”) a petition for post-grant review (“PGR”) of U.S. Patent No. 10,195,214 (the “’214 patent”).
In May 2019, Teva submitted to the Patent Trial and Appeal Board (“PTAB”) a petition for post-grant review (“PGR”) of U.S. Patent No. 10,195,214 (the “’214 patent”). In November 2020, the PTAB issued a decision upholding the validity of the ’214 patent in its entirety, which decision the Court of Appeals for the Federal Circuit upheld. This matter is closed.
The NJ USAO has informed us that it is investigating whether any criminal or civil violations by us occurred in connection with the matters referenced in the subpoena. It has also informed us that it does not currently consider us a defendant but rather an entity whose conduct is within the scope of the government’s investigation.
The NJ USAO has informed us that it is investigating whether any criminal or civil violations by us occurred in connection with the matters referenced in the subpoena.
The complaint named our Board of Directors, Chief Executive Officer, current Chief Business Officer and President of Corcept Endocrinology as defendants, and us as nominal defendant. The complaint alleges a single cause of action for breach of fiduciary duty. The complaint seeks damages in an amount to be proved at trial.
Leonard Baker, et al. , Case No. 2022-0102-SG. The complaint named certain members of our Board of Directors, our Chief Executive Officer, our current Chief Business Officer and our President of Corcept Endocrinology as defendants, and Corcept as nominal defendant. The complaint alleges a single cause of action for breach of fiduciary duty.
On February 27, 2023, the Court denied both motions without prejudice. The patents currently at issue are the ʼ214 patent and U.S. Patent No. 10,842,800 (the “’800 patent”). Trial was held from September 26, 2023 through September 28, 2023 before Judge Renee Marie Bumb.
The patents currently at issue in the D.N.J matter are the ʼ214 patent and U.S. Patent No. 10,842,800 (the “’800 patent”). Trial was held from September 26, 2023 through September 28, 2023 before Judge Renee Marie Bumb. On December 29, 2023, Judge Bumb ruled that Teva’s proposed generic product would not infringe either the ’214 or ’800 patent.
In addition to the above-described matters, we are involved from time-to-time in other legal proceedings arising in the ordinary course of our business.
It has also informed us that it does not currently consider us a defendant but rather an entity whose conduct is within the scope of the government’s investigation. 28 In addition to the above-described matters, we are involved from time-to-time in other legal proceedings arising in the ordinary course of our business.
As required by law, we submitted the settlement agreement to the United States Federal Trade Commission and the United States Department of Justice for review. Other Matters In March 2019, a purported securities class action complaint was filed in the United States District Court for the Northern District of California by Nicholas Melucci ( Melucci v.
Other Litigation In March 2019, a purported securities class action complaint was filed in the United States District Court for the Northern District of California by Nicholas Melucci ( Melucci v. Corcept Therapeutics Incorporated, et al. , Case No. 5:19-cv-01372-LHK) (the “Melucci litigation”).
In September 2021, the case was further stayed pending a resolution of the Melucci litigation. In January 2022, a purported shareholder derivative complaint was filed in the Delaware Court of Chancery by Joel B. Ritchie, captioned Joel B. Ritchie v. G. Leonard Baker, et al. , Case No. 2022-0102-SG.
On June 21, 2024, the United States District Court for the District of Delaware lifted the stays on the Williams and Jeweltex cases and consolidated these two cases into one case. In January 2022, a purported shareholder derivative complaint was filed in the Delaware Court of Chancery by Joel B. Ritchie, captioned Joel B. Ritchie v. G.
We will vigorously enforce our intellectual property rights relating to Korlym but cannot predict the outcome of these matters. Hikma ANDA Litigation and Settlement In February 2021, we received a Paragraph IV Notice Letter advising that Hikma Pharmaceuticals USA Inc.
We have appealed that ruling to the United States Court of Appeals for the Federal Circuit. Teva launched its generic product in January 2024. We will vigorously enforce our intellectual property rights relating to Korlym but cannot predict the outcome of these matters.
The complaint seeks damages in an amount to be proved at trial. In April 2020, this action was stayed pending a resolution of our motions to dismiss the Melucci litigation. In December 2020, the case was further stayed pending a resolution of our motion to dismiss the third amended complaint in the Melucci litigation.
The complaint seeks damages in an amount to be proved at trial. On March 22, 2024, the Court lifted a previously-entered stay, which had been pending the resolution of the Melucci litigation, and on May 3, 2024, we filed a Motion to Dismiss this complaint. We cannot predict when the Court will rule on this motion.
The complaint asserts a putative class period extending from August 2, 2017 to February 5, 2019 and seeks unspecified monetary relief, interest and attorneys’ fees. In October 2019, the Court appointed a lead plaintiff and lead counsel. The lead plaintiff’s consolidated complaint was filed in December 2019.
The complaint asserted a putative class period extending from August 2, 2017 to February 5, 2019 and sought unspecified monetary relief, interest and attorneys’ fees. On June 6, 2024, Judge James Donato of the United States District Court for the Northern District of California granted final approval of a settlement resolving all claims in the Melucci litigation (the “Melucci Settlement”).
Removed
The PTAB agreed to initiate the PGR, and in November 2020, issued a decision upholding the validity of the ’214 patent in its entirety, which decision the Federal Circuit Court of Appeals upheld. This matter is closed. In D.N.J., the parties completed briefing on cross-motions for summary judgment regarding infringement of the ’214 patent in July 2021.
Added
Antitrust Litigation On June 13, 2024, Teva filed a complaint in the Northern District of California, captioned Teva Pharmaceuticals USA, Inc. v. Corcept Therapeutics, Inc., et al. (N.D. Cal.), Case No. 3:24-cv-03567-BLF (the “Teva Antitrust Litigation”). This lawsuit names Corcept and Optime Care, Inc.
Removed
On December 29, 2023, Judge Bumb issued a ruling finding that Teva’s proposed generic product would not infringe either the ‘214 or ‘800 patent. We have appealed that decision to the Federal Circuit Court of Appeals. Teva has announced the launch of its generic product.
Added
(“Optime”), our single specialty pharmacy that dispenses Korlym and the authorized generic version of Korlym and performs related pharmacy and patient support services, as defendants. The lawsuit alleges, among other things, that Corcept has violated federal and state laws related to antitrust and unfair business practices. 27 On August 26, 2024, Corcept and Optime filed motions to dismiss the complaint.
Removed
(“Hikma”) had submitted an ANDA to the FDA seeking authorization to manufacture, use or sell a generic version of Korlym in the United States. In March 2021, we filed a lawsuit in the United States District Court for the District of New Jersey against Hikma for infringement of the ’214 patent, U.S. Patent No. 10,500,216, the ’800 patent and U.S.
Added
On September 13, 2024, Teva filed a First Amended Complaint, and on October 14, 2024, Corcept and Optime moved to dismiss the First Amended Complaint.
Removed
Patent Nos. 10,842,801. On December 7, 2022, we entered into an agreement with Hikma resolving this litigation.
Added
On February 10, 2025, several named plaintiffs filed a complaint against Corcept in the Alameda County Superior Court for the State of California, captioned, Aetna Inc., Health Care Service Corporation, Humana Inc. and Molina Healthcare Inc. vs. Corcept Therapeutics, Inc ., Case No. 25CV110493.
Removed
Pursuant to the agreement, we have granted Hikma the right to sell a generic version of Korlym in the United States beginning October 1, 2034 or earlier under circumstances customary for settlement agreements of this type, including following the start of Teva's sales of a generic product.
Added
This lawsuit names Corcept as the sole defendant and includes allegations substantially similar to those made in the Teva Antitrust Litigation. Corcept’s response to the Complaint is due on March 17, 2025.
Removed
Corcept Therapeutics Incorporated, et al. , Case No. 5:19-cv-01372-LHK) (the “Melucci litigation”).
Added
As previously disclosed, the Melucci Settlement required us to make a one-time payment of $14 million for which our insurers reimbursed us in full. On September 6, 2024, Judge Donato approved the Plan of Allocation for payment of the settlement funds to eligible members of the class of plaintiffs. This matter is closed.
Removed
With respect to these allegations, we have stated, from the beginning, that we have done nothing wrong. On February 8, 2023, we reached an agreement in principle (the “Proposed Settlement”) to resolve all claims in the Melucci litigation.
Added
Given the overlapping allegations in these shareholder derivative actions, we and the individual defendants have filed a One Forum Motion in both the United States District Court for the District of Delaware and the Delaware Court of Chancery requesting that the Courts coordinate to determine in which jurisdiction (Federal or Chancery Court) these matters should first proceed.
Removed
Under the Proposed Settlement, we have agreed to make a one-time payment of $14.0 million, which will be covered in full by our insurers. The Court granted preliminary approval of the Proposed Settlement on January 4, 2024, following which we paid $14.0 million into escrow. Our insurers reimbursed us in full.
Added
The matters pending in the Federal Court have been stayed pending the Chancery Court’s ruling on our Motion to Dismiss. We will respond vigorously to the above allegations but cannot predict the outcome of these matters.
Removed
The Proposed Settlement does not include admission of liability on our part. The Proposed Settlement is subject to the final approval of the Court. The Court has issued its preliminary approval and has scheduled a hearing with respect to final approval on June 6, 2024.
Removed
In December 2020, the case was further stayed pending a resolution of our motion to dismiss the third amended complaint in the Melucci litigation. In September 2021, the case was further stayed pending a resolution of the Melucci litigation.
Removed
In April 2022, the case was further stayed pending a resolution of the Melucci litigation. As settlement of the Melucci litigation advances, we expect all stays of the above-described derivative actions to be lifted and for litigation to commence. We will respond vigorously to the above allegations but cannot predict the outcome of these matters.

Item 5. Market for Registrant's Common Equity

Market for Common Equity — stock, dividends, buybacks

5 edited+1 added−0 removed4 unchanged
Biggest change(2) We paid $0.5 million to satisfy the tax withholding obligations associated with the net-share settlement of these cashless option exercises.
Biggest changeIn December 2024, we issued 42,230 shares of common stock as part of restricted stock vesting, of which 14,303 shares were surrendered to us. (2) We paid $7.0 million to satisfy the tax withholding obligations associated with the net-share settlement of these cashless option exercises and restricted stock vesting.
The return shown in the graph below for our common stock is not necessarily indicative of future performance. We do not make or endorse any predictions as to future stockholder returns. 28 Five-Year Cumulative Total Returns of our Common Stock (CORT), the Nasdaq US Benchmark TR Index (NQUSBT) and the Nasdaq Biotechnology Index (NBI) ITEM 6. [RESERVED] 29
The return shown in the graph below for our common stock is not necessarily indicative of future performance. We do not make or endorse any predictions as to future stockholder returns. 30 Five-Year Cumulative Total Returns of our Common Stock (CORT), the Nasdaq US Benchmark TR Index (NQUSBT) and the Nasdaq Biotechnology Index (NBI) ITEM 6. [RESERVED] 31
ITEM 5. MARKET FOR REGISTRANT’S COMMON EQUITY, RELATED STOCKHOLDER MATTERS AND ISSUER PURCHASES OF EQUITY SECURITIES Market Information Our common stock is traded on The Nasdaq Capital Market under the symbol “CORT.” Stockholders of Record and Dividends As of February 6, 2024, we had 103,517,388 shares of common stock outstanding held by 379 stockholders of record.
ITEM 5. MARKET FOR REGISTRANT’S COMMON EQUITY, RELATED STOCKHOLDER MATTERS AND ISSUER PURCHASES OF EQUITY SECURITIES Market Information Our common stock is traded on The Nasdaq Capital Market under the symbol “CORT.” Stockholders of Record and Dividends As of February 18, 2025, we had 105,503,432 shares of common stock outstanding held by 522 stockholders of record.
Repurchases of Securities The following table contains information relating to the purchases of our common stock in the three months ended December 31, 2023 as part of the cashless net exercises of stock options (in thousands, except average price per share): Fiscal Period Total Number of Shares Purchased (1) Average Price Per Share Total Purchase Price of Shares (2) October 1, 2023 to October 31, 2023 1 $ 28.34 $ 22 November 1, 2023 to November 30, 2023 43 25.65 1,114 December 1, 2023 to December 31, 2023 76 29.50 2,240 Total 120 $ 28.10 $ 3,376 (1) In October 2023, we issued 1,250 shares of common stock as part of net-share settlement of cashless option exercises, of which 783 shares were surrendered to us in satisfaction of related exercise cost and tax obligations.
Repurchases of Securities The following table contains information relating to the purchases of our common stock in the three months ended December 31, 2024 as part of the cashless net exercises of stock options and vesting of restricted stock (in thousands, except average price per share): Fiscal Period Total Number of Shares Purchased (1) Average Price Per Share Total Purchase Price of Shares (2) October 1, 2024 to October 31, 2024 11 $ 46.78 $ 520 November 1, 2024 to November 30, 2024 122 54.79 6,716 December 1, 2024 to December 31, 2024 128 52.92 6,760 Total 261 $ 53.53 $ 13,996 (1) In October 2024, we issued 7,574 shares of common stock as part of net-share settlement of cashless option exercises, of which 3,448 shares were surrendered to us in satisfaction of related exercise cost and tax obligations.
In November 2023, we issued 62,115 shares of common stock as part of net-share settlement of cashless option exercises, of which 43,435 shares were surrendered to us. In December 2023, we issued 161,773 shares of common stock as part of net-share settlement of cashless option exercises, of which 75,914 shares were surrendered to us.
In November 2024, we issued 212,261 shares of common stock as part of net-share settlement of cashless option exercises, of which 104,456 shares were surrendered to us. In December 2024, we issued 197,059 shares of common stock as part of net-share settlement of cashless option exercises, of which 113,449 shares were surrendered to us.
Added
In October 2024, we issued 21,847 shares of common stock as part of restricted stock vesting, of which 7,661 shares were surrendered to us in satisfaction of related tax obligations. In November 2024, we issued 51,391 shares of common stock as part of restricted stock vesting, of which 18,122 shares were surrendered to us.

Item 7. Management's Discussion & Analysis

Management's Discussion & Analysis (MD&A) — revenue / margin commentary

56 edited+33 added−25 removed13 unchanged
Biggest changeFor the year ended December 31, 2022, we spent $21.7 million acquiring shares of our common stock, offset by $4.4 million received from the exercise of stock options, resulting in net cash used in financing activities of $17.3 million.
Biggest changeIn the year ended December 31, 2024, we spent $38.0 million acquiring shares of our common stock, comprised of $15.7 million pursuant to our Stock Repurchase Program, $17.0 million acquiring shares of our common stock in connection with the net exercise of employee and director stock options and $5.3 million to satisfy tax withholding requirements from vesting of restricted stock grants, offset by $5.5 million received in connection with our ESPP and $4.2 million net cash received from the exercise of stock options.
The IRA also permits CMS to negotiate prices for certain high-expenditure Medicare Part B or Part D drugs, imposes a one percent excise tax on certain share repurchases and introduces a 15 percent corporate alternative minimum tax on adjusted financial statement income. The corporate alternative minimum tax became effective for us on January 1, 2024.
The IRA permits CMS to negotiate prices for certain high-expenditure Medicare Part B or Part D drugs. The IRA also imposes a one percent excise tax on certain share repurchases and introduces a 15 percent corporate alternative minimum tax on adjusted financial statement income. The corporate alternative minimum tax became effective for us on January 1, 2024.
Please see the risk factor under Item 1A of this Annual Report on Form 10-K, “ New laws, government regulations, or changes to existing laws and regulations could make it difficult or impossible for us to obtain acceptable prices or adequate insurance coverage and reimbursement for Korlym, which would adversely affect our results of operations and financial position. ” Results of Operations Net Product Revenue – Net product revenue is gross product revenue from sales to our customers less deductions for estimated government rebates and chargebacks, patient co-pay assistance program, discounts provided to our specialty distributor for prompt payment and reserves for expected returns.
Please see the risk factor under Item 1A of this Annual Report on Form 10-K, “ New laws, government regulations, or changes to existing laws and regulations could make it difficult or impossible for us to obtain acceptable prices or adequate insurance coverage and reimbursement for our Products, which would adversely affect our results of operations and financial position. ” Results of Operations Net Product Revenue – Net product revenue is gross product revenue from sales to our customers less deductions for estimated government rebates and chargebacks, patient co-pay assistance program, discounts provided to our specialty distributor for prompt payment and reserves for expected returns.
Discussions of 2021 items and year-to-year comparisons between 2022 and 2021 are not included, and can be found in “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in Part II, Item 7 of the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2022.
Discussions of 2022 items and year-to-year comparisons between 2023 and 2022 are not included, and can be found in “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in Part II, Item 7 of the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2023.
In addition, beginning in 2025, the IRA will also shift a significant portion 32 of the Medicare beneficiary costs currently borne by the government and beneficiaries to manufacturers. We anticipate this provision will significantly limit the revenue we receive from Medicare patients and may materially reduce our profits.
In addition, beginning in 2025, the IRA will also shift a significant portion of the Medicare beneficiary costs currently borne by the government and beneficiaries to manufacturers. We anticipate this provision will limit the revenue we receive from Medicare patients and may materially reduce our profits.
Study participants were randomized to one of three treatment arms: 60 women received 150 mg of relacorilant intermittently (the day before, the day of and the day after their weekly nab-paclitaxel infusion) and 58 women received a daily relacorilant dose of 100 mg per day in addition to nab-paclitaxel. Sixty women received nab-paclitaxel alone.
Phase 2 study participants were randomized to one of three treatment arms: 60 women received 150 mg of relacorilant intermittently (the day before, the day of and the day after their weekly nab-paclitaxel infusion) and 58 women received a daily relacorilant dose of 100 mg per day in addition to nab-paclitaxel. Sixty women received nab-paclitaxel alone.
Cortisol also suppresses the body’s immune response; activating – not suppressing – the immune system is beneficial in fighting certain cancers. Many types of solid tumors express the GR and are potential targets for cortisol modulation therapy, among them ovarian, adrenal and prostate cancer. Relacorilant in Patients with Advanced Ovarian Cancer .
Cortisol also suppresses the body’s immune response; activating – not suppressing – the immune system is beneficial in fighting certain cancers. Many types of solid tumors express the GR and are potential targets for cortisol modulation therapy, among them ovarian, adrenal and prostate cancer. Relacorilant in Patients with Platinum-Resistant Ovarian Cancer.
Liquidity and Capital Resources Since 2015, we have relied on revenues from the sale of Korlym to fund our operations.
Liquidity and Capital Resources Since 2015, we have relied on revenues from the sale of our Products to fund our operations.
To determine the appropriate amount to reserve against these rebates, we identify Korlym sold to patients covered by government-funded programs, apply the applicable government discount to these sales, then estimate utilization of such programs by government payors.
To determine the appropriate amount to reserve against these rebates, we identify our Products sold to patients covered by government-funded programs, apply the applicable government discount to these sales, then estimate utilization of such programs by government payors.
We report any changes in the period they become known, even if they concern transactions occurring in prior periods. Government Rebates Korlym is eligible for purchase by, or qualifies for reimbursement from, Medicaid, Medicare and other government programs that are eligible for rebates on the price they pay for Korlym.
We report any changes in the period they become known, even if they concern transactions occurring in prior periods. Government Rebates Our Products are eligible for purchase by, or qualifies for reimbursement from, Medicaid, Medicare and other government programs that are eligible for rebates on the price they pay for our Products.
In addition, clinical development is subject to government oversight and regulations that may change without notice. We expect our research and development expense to be higher in 2024 than in 2023 as we add new clinical trials and our existing trials enroll more patients.
In addition, clinical development is subject to government oversight and regulations that may change without notice. We expect our research and development expense to be higher in 2025 than in 2024 as we add new clinical trials, assuming success in our existing trials, and our existing trials enroll more patients.
In the year ended December 31, 2023, we spent $154.5 million acquiring shares of our common stock, comprised of $145.4 million pursuant to our tender offer, $7.4 million acquiring shares of our common stock in connection with the net exercise of employee and director stock options and $1.7 million to satisfy tax withholding requirements from vesting of restricted stock grants, offset by $3.8 million received in connection with our ESPP and $2.0 million net cash received from the exercise of stock options, resulting in net cash used in financing activities of $148.7 million.
For the year ended December 31, 2023, we spent $154.5 million acquiring shares of our common stock, comprised of $145.4 million pursuant to our tender offer, $7.4 37 million acquiring shares of our common stock in connection with the net exercise of employee and director stock options and $1.7 million to satisfy tax withholding requirements from vesting of restricted stock grants, offset by $3.8 million received in connection with our ESPP and $2.0 million net cash received from the exercise of stock options.
Net product revenue was $482.4 million for the year ended December 31, 2023, compared to $401.9 million and $366.0 million for the years ended December 31, 2022 and 2021, respectively. Higher sales volume accounted for 57.4 percent of the increase for the year ended December 31, 2023, with the remaining growth due to a price increase effective January 1, 2023.
Net product revenue was $675.0 million for the year ended December 31, 2024, compared to $482.4 million and $401.9 million for the years ended December 31, 2023 and 2022, respectively. Higher sales volume accounted for 79.4 percent of the increase for the year ended December 31, 2024, with the remaining growth due to a price increase effective January 1, 2024.
Cost of sales – Cost of sales includes the cost of API, tableting, packaging, personnel, overhead, stability testing and distribution. Cost of sales was $6.5 million for the year ended December 31, 2023, compared to $5.4 million and $5.3 million for the years ended December 31, 2022 and 2021, respectively.
Cost of sales – Cost of sales includes the cost of API, tableting, packaging, personnel, overhead, stability testing and distribution. Cost of sales was $10.9 million for the year ended December 31, 2024, compared to $6.5 million and $5.4 million for the years ended December 31, 2023 and 2022, respectively.
As of December 31, 2023, we had retained earnings of $402.5 million. 34 Net Operating Loss Carryforwards See Note 9 , Income Taxes in our audited consolidated financial statements. Critical Accounting Policies and Estimates Our consolidated financial statements have been prepared in accordance with U.S.
As of December 31, 2024, we had retained earnings of $543.7 million. Net Operating Loss Carryforwards See Note 9 , Income Taxes in our audited consolidated financial statements. Critical Accounting Policies and Estimates Our consolidated financial statements have been prepared in accordance with U.S.
Net cash provided by operating activities for the years ended December 31, 2023, 2022 and 2021 was $127.0 million, $120.3 million and $167.9 million, respectively. The increase for the year ended December 31, 2023 compared to 2022 was primarily due to higher revenue.
Net cash provided by operating activities for the years ended December 31, 2024, 2023 and 2022 was $198.1 million, $127.0 million and $120.3 million, respectively. The increase for the year ended December 31, 2024 compared to 2023 was primarily due to higher revenue.
Because many people who suffer from Cushing’s syndrome are undiagnosed or inadequately treated, we have developed and continue to refine and expand programs to educate physicians and patients about screening for hypercortisolism and the role Korlym can play in treating patients with the disorder.
Because most people who suffer from hypercortisolism are undiagnosed or inadequately treated, we have developed and continue to refine and expand programs to educate physicians and patients about screening for hypercortisolism and the role our Products can play in treating patients with the disorder.
We expect our selling, general and administrative expense to be higher in 2024 than in 2023 due to increased commercial and administrative activities, including litigation and administrative support for increased research and development and marketing efforts.
We expect our selling, general and administrative expense to be higher in 2025 than in 2024 due to increased commercial and administrative activities to support our increased research and development and marketing efforts.
Net cash used in financing activities was $148.7 million, $17.3 million and $302.6 million for the years ended December 31, 2023, 2022 and 2021, respectively.
Net cash used in financing activities was $28.3 million, $148.7 million and $17.3 million for the years ended December 31, 2024, 2023 and 2022, respectively.
Patients who received relacorilant intermittently also had a longer median duration of response (“DoR”) (5.6 months versus 3.7 months, hazard ratio: 0.36; p-value: 0.006) compared to those who received nab-paclitaxel alone. Patients who received relacorilant intermittently also lived longer (median OS: 13.9 months versus 12.2 months, hazard ratio: 0.67; p-value: 0.066) compared to those who received nab-paclitaxel alone.
Patients who received relacorilant intermittently also had a longer median duration of response (“DoR”) (5.6 months versus 3.7 months, hazard ratio: 0.36; p-value: 0.006) compared to those who received nab-paclitaxel alone.
Net cash provided by (used in) investing activities for the years ended December 31, 2023, 2022 and 2021 was $90.9 million, $(114.3) million and $136.1 million, respectively.
Net cash (used in) provided by investing activities for the years ended December 31, 2024, 2023 and 2022 was $(177.6) million, $90.9 million and $(114.3) million, respectively.
Year Ended December 31, 2023 2022 2021 (in thousands) Development programs: Oncology $ 41,433 $ 20,987 $ 17,984 Cushing’s syndrome 41,196 30,031 28,639 Metabolic diseases 36,104 24,270 20,594 Pre-clinical and early-stage selective cortisol modulators and ALS 30,852 26,084 21,924 Unallocated activities, including manufacturing and regulatory activities 19,366 16,819 10,617 Stock-based compensation 15,402 12,800 14,106 Total research and development expense $ 184,353 $ 130,991 $ 113,864 It is difficult to predict the timing and cost of development activities, which are subject to many uncertainties and risks, including inconclusive or negative results, slow patient enrollment, adverse side effects and difficulties in the formulation or manufacture of study drugs and lack of drug-candidate efficacy.
Year Ended December 31, 2024 2023 2022 (in thousands) Development programs: Oncology $ 52,699 $ 41,433 $ 20,987 Cushing’s syndrome 65,215 41,196 30,031 Metabolic diseases 40,124 36,104 24,270 Pre-clinical and early-stage selective cortisol modulators and ALS 41,048 30,852 26,084 Unallocated activities, including manufacturing and regulatory activities 30,072 19,366 16,819 Stock-based compensation 17,729 15,402 12,800 Total research and development expense $ 246,887 $ 184,353 $ 130,991 36 It is difficult to predict the timing and cost of development activities, which are subject to many uncertainties and risks, including inconclusive or negative results, slow patient enrollment, adverse side effects and difficulties in the formulation or manufacture of study drugs and lack of drug-candidate efficacy.
Overview We are 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. Since 2012, we have marketed Korlym ® for the treatment of patients suffering from Cushing’s syndrome.
Overview We are 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. Since 2012, we have marketed Korlym in the United States for the treatment of patients suffering from hypercortisolism (also known as “Cushing’s syndrome”).
Benefits of orphan drug designation by the EC are similar, but also include protocol assistance from the European Medicines Agency (“EMA”), access to the centralized marketing authorization procedure in the European Union (“EU”) and, if we obtain approval, ten years of exclusive marketing rights in the EU for the treatment of patients with Cushing’s syndrome.
Benefits of orphan drug designation by the EC are similar, but also include protocol assistance from the EMA, access to the centralized marketing authorization procedure in the EU and, if we obtain approval, ten years of exclusive marketing rights in the EU for the treatment of patients with hypercortisolism.
Safety and tolerability of relacorilant plus nab-paclitaxel were comparable to nab-paclitaxel monotherapy. The final analysis from our Phase 2 trial was published in the Journal of Clinical Oncology (Colombo et al., 2023), the premiere journal of the American Society of Clinical Oncology (ASCO).
Safety and tolerability of relacorilant and nab-paclitaxel combination treatment were comparable to the safety and tolerability of nab-paclitaxel monotherapy. The final analysis from our Phase 2 trial was published in the Journal of Clinical Oncology (Colombo et al., 2023), the premiere journal of the American Society of Clinical Oncology (ASCO). Relacorilant in Patients with Adrenal Cancer with Cortisol Excess.
Inflation Reduction Act of 2022 The Inflation Reduction Act of 2022 (“IRA”) was enacted on August 16, 2022. The IRA includes provisions requiring manufacturers to pay a rebate to the Centers for Medicare & Medicaid Services (“CMS”) if the price of a Medicare Part B or Part D drug increases faster than the rate of inflation.
The IRA includes provisions requiring manufacturers to pay a rebate to the Centers for Medicare & Medicaid Services (“CMS”) if the price of a Medicare Part B or Part D drug increases faster than the rate of inflation.
Women with a history of tumors that do not respond to initial platinum-based treatments (i.e., women with “primary platinum-refractory” disease) and those who have received more than three prior lines of therapy will be excluded. Relacorilant in Patients with Adrenal Cancer with Cortisol Excess .
Women with a history of tumors that do not respond to initial platinum-based treatments (i.e., women with “primary platinum-refractory” disease) and those who have received more than three prior lines of therapy were excluded.
Selling, general and administrative expense for the years ended December 31, 2023, 2022 and 2021 was $184.3 million, $152.8 million and $122.4 million, respectively. The increase for the year ended December 31, 2023 compared to 2022 was primarily due to increased employee compensation expenses and legal fees.
Selling, general and administrative expense for the years ended December 31, 2024, 2023 and 2022 was $280.3 million, $184.3 million and $152.8 million, respectively. The increase for the year ended December 31, 2024 compared to 2023 was primarily due to increased employee compensation expenses and sales and marketing activities.
The change for the year ended December 31, 2023 compared to 2022 was primarily due to allocation of cash proceeds from maturities of marketable securities towards cash equivalents in anticipation of the closing of our tender offer in April 2023 and in anticipation of our share buyback program announced in January 2024.
The change for the year ended December 31, 2024 compared to 2023 was primarily due to investments in marketable securities during 2024 compared to allocation of cash proceeds from maturities of marketable securities towards cash equivalents in anticipation of the closing of our tender offer during 2023.
Interest and other income – Interest and other income for the years ended December 31, 2023, 2022 and 2021 was $17.3 million, $3.6 million and $0.5 million, respectively, and consisted primarily of interest income from marketable securities. The increase for the year ended December 31, 2023 compared to 2022 was due to market-wide increase in interest rates.
Interest and other income – Interest and other income for the years ended December 31, 2024, 2023 and 2022 was $24.5 million, $17.3 million and $3.6 million, respectively, and consisted primarily of interest income from marketable securities. The increase for the year ended December 31, 2024 compared to 2023 was due to higher cash and investment balances.
We do not expect it to significantly affect our consolidated financial statements.
We do not expect either of these provisions to significantly affect our consolidated financial statements.
We (i) deduct this reserve from revenue in the period to which the rebates relate and (ii) include in accrued expenses on our consolidated balance sheet a current liability of equal amount. Chargebacks Although we sell Korlym to the SD at full price, some of the government entities to which the SD sells receive a discount.
We (i) deduct this reserve from revenue in the period to which the rebates relate and (ii) include in accrued expenses on our consolidated balance sheet a current liability of equal amount.
The United States Food and Drug Administration (“FDA”) and the European Commission (“EC”) have designated relacorilant as an orphan drug for the treatment of Cushing’s syndrome. In the United States, relacorilant’s orphan designation confers tax credits, reduced regulatory fees and, provided we obtain approval for the treatment of patients with Cushing’s syndrome, seven years of exclusive marketing rights.
In the United States, relacorilant’s orphan designation confers tax credits, reduced regulatory fees and, provided we obtain approval for the treatment of patients with hypercortisolism, seven years of exclusive marketing rights.
As of December 31, 2023, we had cash, cash equivalents and marketable securities of $425.4 million, consisting of cash and cash equivalents of $135.6 million and marketable securities of $289.8 million, compared to cash, cash equivalents and marketable securities of $436.6 million, consisting of cash and cash equivalents of $66.3 million and marketable securities of $370.3 million as of December 31, 2022.
As of December 31, 2024, we had cash, cash equivalents and marketable securities of $603.2 million, consisting of cash and cash equivalents of $127.7 million and marketable securities of $475.5 million, compared to cash, cash equivalents and marketable securities of $425.4 million, consisting of cash and cash equivalents of $135.6 million and marketable securities of $289.8 million as of December 31, 2023.
In May 2021, we announced preliminary results from our 178-patient, controlled, multi-center, Phase 2 trial of relacorilant combined with nab-paclitaxel in patients with platinum-resistant ovarian cancer.
ROSELLA seeks to replicate the positive results of our Phase 2 trial, a 178-patient, controlled, multi-center, trial of relacorilant combined with nab-paclitaxel in patients with platinum-resistant ovarian cancer.
To help us achieve that goal, we fund our own patient support programs and donate money to independent charitable foundations that help patients pay for all aspects of their Cushing’s syndrome care, whether or not that care includes taking Korlym.
Our policy is that no patient with hypercortisolism will be denied access to our Products for financial reasons. To help us achieve that goal, we have patient support programs and donate money to independent charitable foundations that help patients pay for all aspects of their hypercortisolism care, whether or not that care includes taking our Products.
Research and development spending in future years will depend on the outcome of our pre-clinical and clinical trials and our development plans. 33 Selling, general and administrative expense – Selling, general and administrative expense includes (1) compensation of employees, consultants and contractors engaged in commercial and administrative activities, (2) the cost of vendors supporting commercial activities and (3) legal and accounting fees.
Selling, general and administrative expense – Selling, general and administrative expense includes (1) compensation of employees, consultants and contractors engaged in commercial and administrative activities, (2) the cost of vendors supporting commercial activities and (3) legal and accounting fees.
Cost of sales as a percentage of revenue was 1.3 percent for each of the years ended December 31, 2023 and 2022.
Cost of sales as a percentage of revenue was 1.6 percent, 1.3 percent and 1.3 percent for each of the years ended December 31, 2024, 2023 and 2022, respectively. The increase of cost of sales as a percentage of revenue for the year ended December 31, 2024 compared to 2023 was primarily due to increased manufacturing and distribution costs.
Our selective cortisol modulator dazucorilant improved motor performance and reduced neuroinflammation and muscular atrophy in animal models of ALS. Following these compelling results, we initiated a Phase 2 trial (“DAZALS”) of dazucorilant in patients with ALS in October 2022.
Amyotrophic Lateral Sclerosis (“ALS” ) ALS, also known as Lou Gehrig’s disease, is a devastating neuromuscular illness. Our selective cortisol modulator dazucorilant improved motor performance and reduced neuroinflammation and muscular atrophy in an animal model of ALS. Following these compelling results, we initiated a Phase 2 trial (“DAZALS”) of dazucorilant in patients with ALS.
Research and development expense was $184.4 million for the year ended December 31, 2023, compared to $131.0 million and $113.9 million for the years ended December 31, 2022 and 2021, respectively. The increase for the year ended December 31, 2023 compared to 2022 was primarily due to increased spending on our development programs and employee compensation expenses.
Research and development expense was $246.9 million for the year ended December 31, 2024, compared to $184.4 million and $131.0 million for the years ended December 31, 2023 and 2022, respectively.
Patients who received a higher dose of relacorilant intermittently exhibited a statistically significant improvement in median PFS (5.6 months versus 3.8 months, hazard ratio: 0.66; p-value: 0.038).
The trial’s primary endpoint was PFS. Patients in both of the relacorilant plus nab-paclitaxel treatment arms of the Phase 2 trial experienced longer PFS than did the patients who received nab-paclitaxel alone. Patients who received a higher dose of relacorilant intermittently exhibited a statistically significant improvement in median PFS (5.6 months versus 3.8 months, hazard ratio: 0.66; p-value: 0.038).
Income tax expense – Income tax expense for the years ended December 31, 2023, 2022, and 2021 was $18.4 million, $14.8 million, and $12.5 million, respectively. The increase for the year ended December 31, 2023 compared to 2022 was due to decreased excess tax benefits for stock option exercises.
Income tax expense – Income tax expense for the years ended December 31, 2024, 2023, and 2022 was $20.3 million, $18.4 million, and $14.8 million, respectively. The increase for the year ended December 31, 2024 compared to 2023 was primarily due to increased income before income taxes.
Relacorilant shares Korlym’s affinity for the glucocorticoid receptor (“GR”), but, unlike Korlym, has no affinity for the progesterone receptor (“PR”), and so is not the “abortion pill.” It does not cause other effects associated with PR affinity, including endometrial thickening and vaginal bleeding.
We are developing our proprietary, selective cortisol modulator, relacorilant, as a treatment for patients with hypercortisolism. Relacorilant shares Korlym’s affinity for the GR but, unlike Korlym, has no affinity for the PR and so is not the “abortion pill” and does not cause other effects associated with PR affinity, including endometrial thickening and vaginal bleeding.
The primary endpoint is PFS; overall survival is a key secondary endpoint. Patients in ROSELLA will have received prior bevacizumab therapy, which is the standard of care for patients with platinum-resistant ovarian cancer.
ROSELLA has dual primary endpoints – progression free survival (“PFS”) and overall survival (“OS”). ROSELLA will have a statistically positive outcome if either endpoint is met. Patients enrolled in ROSELLA were required to have received prior bevacizumab therapy, which is the approved standard of care for patients with platinum-resistant ovarian cancer.
We have initiated a Phase 4 study (“CATALYST”), to determine the prevalence of Cushing’s syndrome in patients with difficult-to-control diabetes (HbA1c of 7.5 percent or higher) despite receiving standard treatment. In CATALYST’s first phase, 1,000 patients will be evaluated.
In 2024, we conducted the CATALYST study to determine the prevalence of hypercortisolism in patients with difficult-to-control diabetes (defined as HbA1c of 7.5 percent or higher) despite receiving optimum treatment. Of the 1,057 patients enrolled in the first phase of CATALYST, 23.8 percent were found to have hypercortisolism.
There is no evidence that relacorilant causes hypokalemia (low potassium), a potentially serious condition that is a leading cause of patients stopping treatment with Korlym. Forty-four percent of patients in Korlym’s pivotal trial experienced hypokalemia. Unlike all other medications used to treat Cushing’s syndrome, relacorilant does not prolong the heart’s QT interval, a potentially deadly off-target effect.
Because relacorilant does not meaningfully increase cortisol levels, it does not cause hypokalemia (low potassium), a potentially serious condition that is a leading cause of patients stopping treatment with Korlym. Forty-four percent of patients in Korlym’s pivotal trial experienced hypokalemia.
Combining a cortisol modulator with an androgen modulator may block this escape route. Our collaborators at the University of Chicago have initiated a randomized, placebo-controlled Phase 2 trial of relacorilant plus enzalutamide in patients with prostate cancer, pre-prostatectomy.
Our collaborators at the University of Chicago have initiated a randomized, placebo-controlled Phase 2 trial of relacorilant plus enzalutamide in patients with prostate cancer, pre-prostatectomy. We are providing relacorilant and placebo for the study. Patents we have licensed from the University of Chicago cover the use of relacorilant combined with anticancer agents, including enzalutamide, to treat patients with this disease.
Our portfolio of proprietary selective cortisol modulators consists of four structurally distinct series totaling more than 1,000 compounds. Cushing’s Syndrome Korlym . We sell Korlym in the United States, using experienced sales representatives to call on physicians caring for patients with hypercortisolism (“Cushing’s syndrome”). We also have a field-based force of medical science liaisons.
We sell Korlym and a generic version of Korlym in the United States (our “Products”), using sales representatives to call on physicians caring for patients with hypercortisolism. We also have a field-based force of medical science liaisons. We use a specialty pharmacy and a specialty distributor to distribute our Products and provide logistical support to physicians and patients.
We are conducting an open-label, Phase 1b trial of relacorilant plus the PD-1 checkpoint inhibitor pembrolizumab in patients with metastatic or unresectable adrenal cancer whose tumors produce cortisol. The trial is examining whether adding relacorilant to pembrolizumab therapy reduces cortisol-activated immune suppression sufficiently to help pembrolizumab achieve its intended tumor-killing effect.
We have completed an open-label, Phase 1b trial of relacorilant plus the PD-1 checkpoint inhibitor pembrolizumab in 14 patients with metastatic or unresectable adrenal cancer whose tumors produce cortisol. Patients with this form of adrenal cancer virtually never respond to immunotherapy and their disease progresses very rapidly.
These patients often exhibit less severe symptoms or have a more gradual course of disease than patients with other etiologies of Cushing’s syndrome, although their health outcomes are ultimately poor. Half of the patients in GRADIENT will receive relacorilant for 22 weeks and half will receive placebo. The trial’s primary endpoints are improvements in glucose metabolism and hypertension.
These patients have a more gradual decline than patients with other etiologies of hypercortisolism, although their health outcomes are ultimately poor. GRADIENT enrolled 137 patients with hypercortisolism and either hypertension, hyperglycemia or both. Patients were randomized on a double-blind basis 1:1 to receive either relacorilant or placebo for 22 weeks.
Relacorilant is also expected to treat the patients’ Cushing’s syndrome generated by their tumors’ excess production of cortisol. 31 Relacorilant in Patients with Prostate Cancer . Androgen deprivation is the standard treatment for prostate cancer because androgens stimulate prostate tumor growth. Tumors often escape androgen deprivation therapy when cortisol’s activity at the GR stimulates tumor growth.
Relacorilant in Patients with Prostate Cancer . Androgen deprivation is the standard treatment for prostate cancer because androgens stimulate prostate tumor growth. Prostate cancer tumors eventually escape androgen deprivation therapy; one of the prime reasons is that these tumors begin to be stimulated by cortisol’s activity. Combining a cortisol modulator with an androgen modulator may block this escape route.
Nonalcoholic steatohepatitis (“NASH”) is an advanced form of nonalcoholic fatty liver disease that afflicts millions of patients and is a leading cause of liver-related mortality.
The FDA has granted dazucorilant Fast Track Designation and orphan drug status for the treatment of ALS in the United States. Metabolic Diseases Liver Disease. Metabolic dysfunction-associated steatohepatitis (“MASH”) is an advanced form of metabolic dysfunction-associated fatty liver disease that afflicts millions of patients and is a leading cause of liver-related mortality.
In the GRACE trial, each patient receives relacorilant for 22 weeks. Patients who exhibit pre-specified improvements in hypertension and/or glucose metabolism enter a 12-week, double-blind, “randomized withdrawal” phase, in which half of the patients continue receiving relacorilant and half receive placebo.
Patients who exhibited pre-specified improvements in either hypertension, hyperglycemia or both symptoms proceeded to GRACE’s second, double-blind, randomized withdrawal phase, in which half of the patients continued to receive relacorilant and half received placebo for 12 weeks.
In June 2022, we initiated a pivotal Phase 3 trial (“ROSELLA”) that seeks to replicate the positive results observed in our Phase 2 study. ROSELLA has a planned enrollment of 360 women with recurrent, platinum-resistant ovarian cancer, half of whom will receive 150 mg of relacorilant intermittently in addition to nab-paclitaxel, with the other half receiving nab-paclitaxel monotherapy.
We are conducting a pivotal Phase 3 trial (“ROSELLA”) of our proprietary, selective cortisol modulator, relacorilant combined with nab-paclitaxel as a treatment for patients with platinum-resistant ovarian cancer. Enrollment in ROSELLA is complete. Three hundred eighty-one women with recurrent, platinum-resistant ovarian cancer were randomized 1:1 to receive either 150 mg of relacorilant intermittently in addition to nab-paclitaxel or nab-paclitaxel monotherapy.
Following these compelling results, we initiated a Phase 2b trial (“MONARCH”) of miricorilant in patients with NASH in October 2023. MONARCH has a planned enrollment of 150 patients, randomized 2:1 to receive either 100 mg of miricorilant twice weekly or placebo for 48 weeks.
Cohort B has a planned enrollment of 75 patients with presumed MASH, randomized 2:1 to receive either 100 mg of miricorilant twice weekly for 6 weeks and then 200 mg of miricorilant twice weekly for 18 weeks or placebo for 24 weeks. The primary endpoint of Cohort B is reduction in liver fat.
Liver enzyme levels in all affected patients returned to baseline or below baseline (i.e., to healthier levels) after miricorilant was withdrawn. Our Phase 1b study has identified a dosing regimen that effectively reduced liver fat, improved liver health and key metabolic and lipid measures and was well-tolerated.
Our Phase 1b trial of the selective cortisol modulator miricorilant as a potential treatment for MASH identified a dosing regimen that reduced liver fat, improved liver health and key metabolic and lipid measures and was well-tolerated. Following these compelling results, we initiated a randomized, double-blind, placebo-controlled, Phase 2b trial (“MONARCH”) of miricorilant in patients with MASH in October 2023.
Removed
We use one specialty pharmacy and one specialty distributor to distribute Korlym and provide logistical support to physicians and patients. Our policy is that no patient with Cushing’s syndrome will be denied access to Korlym for financial reasons.
Added
In June 2024, we made available an authorized generic version of Korlym for the same indication. Our portfolio of proprietary selective cortisol modulators consists of four structurally distinct series totaling more than 1,000 compounds. Hypercortisolism (Cushing’s Syndrome) Our Products.
Removed
Those found to have Cushing’s syndrome may elect to enter the second phase of the study, in which patients will be randomized to receive either Korlym or placebo for 24 weeks. We expect that data from CATALYST will help physicians better identify patients with Cushing’s syndrome and determine their optimal treatment. Relacorilant .
Added
These patients were offered the chance to enter CATALYST’s second phase, in which 136 eligible patients were randomized 2:1 to receive either Korlym or placebo for 24 weeks. CATALYST’s primary endpoint was the difference in HbA1c in patients who received Korlym compared to patients who received placebo.
Removed
We are conducting two Phase 3 trials (“GRACE” and “GRADIENT”) of our proprietary, selective cortisol modulator, relacorilant, as a treatment for patients with Cushing’s syndrome. Relacorilant was well-tolerated in its Phase 1 and Phase 2 trials.
Added
Patients who received Korlym exhibited a clinically meaningful and statistically significant improvement in hemoglobin A1c, with a decrease from baseline of 1.47 percent, compared to a decrease of 0.15 percent in patients who received placebo (p-value: The CATALYST data will help physicians better identify patients with hypercortisolism and determine their optimal treatment. Relacorilant.
Removed
Patients in the Phase 2 trial exhibited meaningful improvements in glucose control, hypertension, weight, liver function, coagulopathy, cognition, mood, insulin resistance and quality of life measures.
Added
Unlike all other medications used to treat hypercortisolism, relacorilant does not prolong the heart’s QT interval, a potentially deadly off-target effect. In December 2024, we submitted an NDA to the FDA seeking approval to market relacorilant as a treatment for patients with endogenous hypercortisolism.
Removed
The trial’s primary endpoint is the rate and degree of relapse of hypertension in patients receiving placebo measured against the rate and degree of relapse of hypertension in those continuing relacorilant. GRACE has enrolled 152 patients at sites in the United States, Canada, Europe and Israel. Enrollment is now complete.
Added
The NDA is based on positive results from our pivotal trial “GRACE”, as well as confirmatory evidence from our Phase 3 “GRADIENT” trial, our Phase 3 long-term extension study and our Phase 2 study.
Removed
If successful, GRACE will provide the basis for a new drug application (“NDA”) for relacorilant as a treatment for patients with any etiology of endogenous Cushing’s syndrome. 30 Our second Phase 3 trial of relacorilant, GRADIENT, is studying patients whose Cushing’s syndrome is caused by a benign adrenal tumor.
Added
In 32 all of these trials, patients exhibited clinically meaningful improvements in a wide range of hypercortisolism signs and symptoms, including hypertension, glucose control, weight and body composition. Relacorilant was well-tolerated in all of the trials. Notably, patients did not experience some of the serious adverse events that can arise in patients taking Korlym or other currently approved treatments.
Removed
The trial’s primary endpoint was progression-free survival (i.e., the time from random assignment in a clinical trial to disease progression or death from any cause or “PFS”). Patients in both of the relacorilant plus nab-paclitaxel treatment arms experienced longer PFS than did the patients who received nab-paclitaxel alone.
Added
The GRACE trial had two-parts. The first, open-label phase enrolled 152 patients with any etiology of hypercortisolism. Each patient received relacorilant for 22 weeks.
Removed
We are providing relacorilant and placebo for the study and have licensed patents covering the use of relacorilant combined with anticancer agents such as enzalutamide in the treatment of patients with this indication. Amyotrophic Lateral Sclerosis (“ALS” ) ALS, also known as Lou Gehrig’s disease, is a devastating neuromuscular illness.
Added
GRACE’s primary endpoint was the number of patients in the relacorilant group who lost blood pressure control compared to the number who lost blood pressure control in the placebo group.
Removed
DAZALS has a planned enrollment of 198 patients, randomized 1:1:1 to receive either 150 mg or 300 mg of dazucorilant or placebo daily for 24 weeks. The primary endpoint is the difference between dazucorilant and placebo demonstrated by patients on the ALS Functional Rating Scale-Revised (ALSFRS-R). Metabolic Diseases Liver Disease.
Added
In the open-label phase, patients experienced clinically meaningful and statistically significant improvements in a wide-array of hypercortisolism signs and symptoms, including hypertension, hyperglycemia, weight, waist circumference, fat and lean body mass, cognition and Cushing’s Quality of Life score.
Removed
In April 2021, we suspended our Phase 2a trial of our selective cortisol modulator miricorilant as a potential treatment for NASH after four of the five patients who received miricorilant exhibited both elevated liver enzymes and large rapid reductions in liver fat.
Added
Rapid and sustained improvements in systolic blood pressure (“SBP”) and diastolic blood pressure (“DBP”) were observed in all patients with hypertension, with an improvement in mean SBP of 7.9 mm Hg and mean DBP of 5.4 mm Hg at 22 weeks (p-values: Glucose metabolism was measured by several diagnostic tests, including the oral glucose tolerance test (glucose area under the curve or AUCglucose), hemoglobin A1c (HbA1c) and fasting glucose.
Removed
The primary endpoint is reduction in liver fat, with NASH resolution and fibrosis improvement as key secondary endpoints. Antipsychotic-Induced Weight Gain (“AIWG” ). In the United States, six million people take antipsychotic medications such as olanzapine and risperidone to treat illnesses such as schizophrenia, bipolar disorder and depression.
Added
In the open-label phase, clinically meaningful and statistically significant improvements in glucose metabolism were observed in patients with diabetes or impaired glucose tolerance (i.e., pre-diabetes), with reductions in AUCglucose of 3.3 h*mmol/L, HbA1c of 0.3 percent and fasting glucose of 12.4 mg/dL at 22 weeks (p-values: GRACE met its primary endpoint.
Removed
While these drugs are very effective, they often cause rapid and sustained weight gain, other metabolic disturbances and, ultimately, cardiovascular disease. Patients taking these medications experience a 10 to 25-year reduction in life expectancy, due largely to increased cardiovascular events, such as heart attacks and strokes.
Added
Patients with hypertension who were switched to placebo in the randomized withdrawal phase were significantly more likely to lose blood pressure control than were patients who continued to receive relacorilant (odds ratio: 0.17; p-value: 0.02).

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Item 7A. Quantitative and Qualitative Disclosures About Market Risk

Market Risk — interest-rate, FX, commodity exposure

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Biggest changeITEM 7A. QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK The primary objective of our investment activities is to preserve capital. As of December 31, 2023, the fair value of our cash and cash equivalents and marketable securities was $425.4 million. Our marketable securities consisted of corporate notes, commercial paper, asset-backed securities, U.S.
Biggest changeITEM 7A. QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK The primary objective of our investment activities is to preserve capital. As of December 31, 2024, the fair value of our cash and cash equivalents and marketable securities was $603.2 million. Our marketable securities consisted of corporate notes, commercial paper, asset-backed securities, U.S.
FINANCIAL STATEMENTS AND SUPPLEMENTARY DATA The consolidated financial statements required by this item are set forth beginning at page F-1 and are incorporated herein by reference. ITEM 9. CHANGES IN AND DISAGREEMENTS WITH ACCOUNTANTS ON ACCOUNTING AND FINANCIAL DISCLOSURE None.
FINANCIAL STATEMENTS AND SUPPLEMENTARY DATA The consolidated financial statements required by this item are set forth beginning at page F-1 and are incorporated herein by reference. ITEM 9. CHANGES IN AND DISAGREEMENTS WITH ACCOUNTANTS ON ACCOUNTING AND FINANCIAL DISCLOSURE None. 38
Due to the short-term nature and high liquidity of these instruments, an increase or decrease in market interest rates by 25 basis points would not have a material impact on the total value of our portfolio as of December 31, 2023. ITEM 8.
Due to the short-term nature and high liquidity of these instruments, an increase or decrease in market interest rates by 25 basis points would not have a material impact on the total value of our portfolio as of December 31, 2024. ITEM 8.

Other CORT 10-K year-over-year comparisons