CLINICAL TRIALS PROFILE FOR KORLYM

All Clinical Trials for Korlym

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00637494 ↗	A Study of Mifepristone vs. Placebo in the Treatment of Patients With Major Depression With Psychotic Features	Terminated	Corcept Therapeutics	Phase 3	2008-03-01	Approximately 450 patients will be randomized to receive mifepristone or placebo for 7 days followed by antidepressant. The purpose is to compare the efficacy of mifepristone followed by antidepressant versus placebo followed by antidepressant in reducing psychotic symptoms in patients with a diagnosis of psychotic depression.
NCT01631682 ↗	Pilot Study of Pharmaceutical and Behavioral Interventions to Treat Anxiety Disorders	Completed	Massachusetts General Hospital	Phase 4	2010-11-01	The aim of this project is to create fear conditioning paradigm within which the relative strengths of various novel pharmacological and behavioral interventions can be tested. These interventions are intended to reduce the fearfulness associated with fear conditioning by blocking a memory process known as reconsolidation. In fear conditioning, a "conditioned" stimulus (CS) is paired with an aversive "unconditioned" stimulus (US) such as an electric shock, until presentation of the CS alone comes to elicit a fear conditioned response (CR). The investigators hypothesize that by using a more highly prepared CS (i.e. video of spiders); more sensitive subjects (individuals with stronger acquired CRs); and additional experimental probes for the presence of the latent CR, the investigators may develop a normal human paradigm that is not plagued by previously observed floor effects (i.e. intervention is 100% effective), within which both the established techniques of propranolol and delayed extinction will produce significant, but only partial, CR reduction. This would leave room to test and compare potentially more powerful candidate reconsolidation-blocking or memory-updating interventions. To achieve these aims, subjects will undergo a four-day fear conditioning and delayed extinction protocol. Skin conductance response data will be gathered across the different phases of the experiment.
NCT01739335 ↗	Novel Therapeutics in Posttraumatic Stress Disorder (PTSD): A Randomized Clinical Trial of Mifepristone	Completed	VA Office of Research and Development	Phase 2	2012-11-19	Posttraumatic stress disorder (PTSD) is a common and disabling psychiatric disorder for Veterans. Left untreated or under-treated, it can become a chronic condition associated with significant distress, depression, aggression, family disruption, substance abuse and an increased risk of morbidity and mortality. Considerable advances were made in the treatment of PTSD in recent years; however, psychopharmacological treatments have been shown to be largely ineffective for Veterans with PTSD. To address this gap, this proposal seeks to test an innovative treatment approach in PTSD - pharmacological manipulation of the body's major stress system (the hypothalamic-pituitary-adrenal (HPA) axis) with mifepristone. At high doses mifepristone is a glucocorticoid receptor (GR) antagonist with peripheral and central nervous system effects, making it a compound of interest in the treatment of stress related disorders. There is abundant evidence of enhanced GR sensitivity in Veterans with PTSD which is thought to underlie some of the symptoms of PTSD and associated disturbances in mood and cognition. There is also evidence that short-term mifepristone treatment has sustained beneficial effects on mood, cognition and sleep disturbance in some neuropsychiatric conditions (major depression, bipolar disorder, primary insomnia). The purpose of the study is to examine the effects of mifepristone to determine if it is efficacious in improving PTSD symptoms and associated clinical outcomes. To achieve these objectives, the investigators propose to conduct a Phase IIa, multi-site, double-blind, placebo controlled trial of mifepristone in male Veteran outpatients with chronic PTSD through the VA's Cooperative Clinical Trial Award program. The investigators propose to enroll 90 subjects at multiple VA sites based on an estimated attrition rate of 20%. Eligible Veterans will be randomly assigned to the treatment of mifepristone (600 mg/day) or placebo for one week and followed for up to three months. The investigators will also describe the effects of mifepristone on several other clinical parameters including PTSD symptomology, depression severity, sleep quality, and functional impairment. Several measures of neuroendocrine functioning will also be obtained to explore the relationship of plasma cortisol and adrenocorticotropic hormone (ACTH) levels to clinical response and the time to addition of rescue medications.

Clinical Trial Conditions for Korlym

Condition Name

Condition Name for Korlym
Intervention	Trials
Stress Disorders, Post-Traumatic	2
Alcohol Abuse	1
Mild Hypercortisolism	1

Condition MeSH

Condition MeSH for Korlym
Intervention	Trials
Stress Disorders, Traumatic	3
Stress Disorders, Post-Traumatic	3
Cushing Syndrome	2

Clinical Trial Locations for Korlym

Trials by Country

Trials by Country for Korlym
Location	Trials
United States	31

Trials by US State

Trials by US State for Korlym
Location	Trials
California	6
New York	4
North Carolina	3
Maryland	2
New Mexico	2

Clinical Trial Progress for Korlym

Clinical Trial Phase

Clinical Trial Phase for Korlym
Clinical Trial Phase	Trials
Phase 4	3
Phase 3	2
Phase 2	4

Clinical Trial Status

Clinical Trial Status for Korlym
Clinical Trial Phase	Trials
Completed	6
Terminated	2
Withdrawn	1

Clinical Trial Sponsors for Korlym

Sponsor Name

Sponsor Name for Korlym
Sponsor	Trials
Corcept Therapeutics	3
VA Office of Research and Development	2
National Institute on Alcohol Abuse and Alcoholism (NIAAA)	2

Sponsor Type

Sponsor Type for Korlym
Sponsor	Trials
Other	6
U.S. Fed	5
Industry	3

Clinical Trials Update, Market Analysis, and Projection for Korlym (Mifepristone)

Last updated: October 28, 2025

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Introduction

Korlym (mifepristone) is a glucocorticoid receptor antagonist primarily developed by Corcept Therapeutics. Approved by the U.S. Food and Drug Administration (FDA) in 2012, Korlym treats endogenous Cushing’s syndrome — a rare hormonal disorder characterized by prolonged exposure to high cortisol levels. The drug’s unique mechanism of action targets cortisol excess, offering a vital therapeutic option for a condition with limited treatment options. This analysis reviews recent clinical trial updates, current market dynamics, and future projections for Korlym.

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Clinical Trials Update

Recent Clinical Trial Landscape

Since Korlym’s initial approval, Corcept has actively expanded its clinical development portfolio to explore additional indications and to optimize its therapeutic profile. The following key areas represent recent activity and ongoing studies:

• Cushing’s Syndrome Expansion Trials:
  Korlym remains the cornerstone of treatment for endogenous Cushing’s syndrome. The company has conducted retrospective and prospective studies reaffirming efficacy and safety, emphasizing the drug’s ability to normalize cortisol levels and improve metabolic disturbances such as hypertension and hyperglycemia. A notable phase 4 post-marketing study concluded in late 2021, reaffirming the drug's safety in long-term use.

• Corticosteroid-Induced Psychosis:
  Corcept has initiated trials to evaluate mifepristone’s effectiveness in managing corticosteroid-related psychiatric complications, leveraging its glucocorticoid receptor antagonism. These trials seek regulatory approval or expanded indication based on observed symptom improvements.

• Psychiatric Disorders and Metabolic Conditions:
  Preliminary research indicates potential links between cortisol dysregulation and depression, schizophrenia, and metabolic syndrome, prompting exploratory studies. However, these remain in early phases, emphasizing the need for further efficacy and safety data.

Ongoing Clinical Development

• KRP-102 (Investigational Compound):
  Corcept is developing next-generation cortisol receptor antagonists with improved pharmacokinetics. These aim to enhance patient compliance and broaden therapeutic applications.

• Additional Indications:
  The company is exploring Korsnan (another name for Korlym) use in patients undergoing pharmacological stress testing and in the management of adrenal incidentalomas. Although these are at early stages, positive results could expand market potential.

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Market Analysis

Market Dynamics

Korlym occupies a niche, high-precision segment focused on Cushing’s syndrome—an ultra-rare condition with an estimated prevalence of 10-15 cases per million globally [1]. Consequently, the drug’s market is constrained by disease rarity, yet high surgical failure rates and limited therapeutic options sustain demand.

Key market drivers include:

• Unmet Medical Need:
  For patients unfit for surgery or with persistent disease post-adrenalectomy, Korlym offers a critical pharmacological alternative.

• Regulatory Developments:
  The drug benefits from Orphan Drug status, granting market exclusivity until 2027 in the U.S., encouraging continued investment.

• Physician Adoption and Reimbursement:
  As familiarity grows among endocrinologists and psychiatrists, prescribing rates are rising. Reimbursement remains favorable within the framework of rare disease management.

Competitive Landscape

While mifepristone’s primary indication is Cushing’s syndrome, ongoing research and emerging generics could impact market share long-term.

• Generic Mifepristone:
  Currently, Korlym retains market exclusivity, but the patent landscape is complex, with patent challenges ongoing. Once patent expiry approaches, biosimilars could significantly reduce prices and alter market dynamics.

• Alternative Therapies:
  Treatments such as pasireotide and ketoconazole serve as alternatives, but Korlym’s unique receptor antagonism and approval for hyperglycemia make it a preferred choice in specific scenarios.

Revenue and Market Projections

Corcept reported revenues of approximately $390 million in 2022, with Korlym representing over 85% of sales [2]. The growth trajectory is influenced by:

• Market Penetration:
  Increasing adoption in existing patients and expansion into newly diagnosed cases could sustain annual growth of 10-15% over the next five years.

• Pipeline Contributions:
  Success of investigational indications may generate additional revenue streams, especially if clinical trials translate into expanded approvals.

• Patent and Competitive Risks:
  Patent challenges and potential emergence of biosimilars post-2027 could compress margins and necessitate strategic adaptations.

Overall, the market is projected to reach approximately $550 million globally by 2028, driven by increased awareness and demonstration of long-term benefits [3].

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Future Outlook and Projections

Growth Opportunities

• Expansion into Related Endocrine Disorders:
  Positive trial outcomes in corticosteroid-related psychiatric and metabolic conditions could open new indications, multiplying market size.

• Global Market Penetration:
  Currently concentrated in the U.S., increased regulatory approvals in Europe, Asia, and Latin America could triple accessible patient populations within five years.

• Combination Therapy Development:
  Combining Korlym with other agents in endocrinology and psychiatry may optimize therapeutic outcomes, potentially leading to new patent protections and differentiated products.

Challenges and Risks

• Regulatory Uncertainties:
  Approval delays or denials for expanded indications could hamper sales expansion.

• Pricing Pressures:
  Healthcare payers may negotiate for lower prices, especially once biosimilars or generics enter the market.

• Safety Concerns:
  Long-term data must confirm safety and tolerability; adverse effects could restrict use or lead to off-label restrictions.

Long-term Projections

Considering current growth rates, ongoing clinical trial success, and regulatory trends, Korlym’s global revenue could approach $750 million by 2030. This constitutes an annual growth rate of approximately 12%, assuming no significant regulatory or patent shifts.

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Key Takeaways

• Niche but Critical: Korlym remains the primary pharmacotherapy for endogenous Cushing’s syndrome, a rare but serious disease, with high unmet medical needs sustaining demand.

• Robust Pipeline Activities: Ongoing trials targeting corticosteroid-induced psychiatric and metabolic disorders may diversify applications, potentially expanding the market.

• Market Dominance with Patent Life: Strong clinical evidence and regulatory exclusivity undergird Korlym’s market position until at least 2027, with future growth linked to pipeline success and regional expansion.

• Competitive Landscape: Patent challenges and biosimilar developments could threaten margins post-2027; strategic readiness is essential.

• Growth Potential: Favorable trends suggest continued revenue increases, with projections estimating a market size nearing $750 million by 2030, contingent on successful expansion and validation of additional indications.

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FAQs

1. What are the primary clinical uses of Korlym today?
Korlym is approved for managing endogenous Cushing’s syndrome in adults when surgery is not an option or has failed, due to its ability to block cortisol’s effects via glucocorticoid receptor antagonism.

2. Are there ongoing trials to expand Korlym’s indications?
Yes. Corcept is investigating Korlym in corticosteroid-induced psychiatric disorders and metabolic conditions, aiming to broaden its therapeutic applications pending successful trial outcomes.

3. How does Korlym compare with other treatments for Cushing’s syndrome?
Korlym’s unique mechanism provides an effective, non-surgical option with an established safety profile. Alternatives like ketoconazole or pasireotide have different side effect profiles and are often used when Korlym is unsuitable or ineffective.

4. What is the potential impact of biosimilars or generics on Korlym’s market?
Patent challenges could introduce biosimilars post-2027, potentially reducing costs and market share. This underscores the importance of pipeline diversification and expanding indications.

5. What are the key risks facing Korlym’s future commercialization?
Regulatory delays, patent disputes, safety issues, and pricing pressures pose ongoing risks. Also, competition from emerging therapies and biosimilars could impact long-term profitability.

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References

1. Nieman LK, Biller BMK, Findling JW, et al. The diagnosis of Cushing’s Syndrome: an Endocrine Society Clinical Practice Guideline. J Clin Endocrinol Metab. 2008;93(5):1526-1540.

2. Corcept Therapeutics FY2022 Annual Report.

3. MarketWatch. Cortisol modulator market to hit USD 750 million by 2030. October 2022.

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Note: All projections are hypothetical and contingent upon clinical trial outcomes, regulatory decisions, and market dynamics.