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.
NCT01925092 ↗	Mifepristone in Children With Refractory Cushing's Disease	Withdrawn	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Phase 3	2013-08-01	Study objectives are to obtain safety, pharmacokinetic, and pharmacodynamic data on the effect of mifepristone on glucose metabolism, body weight and the growth-hormone-IGF in children with refractory Cushing's disease.

Clinical Trial Conditions for Korlym

Condition Name

Condition Name for Korlym
Intervention	Trials
Stress Disorders, Post-Traumatic	2
Alcohol Use Disorder	1
Psychosis	1
Alcohol Use Disorders	1

Condition MeSH

Condition MeSH for Korlym
Intervention	Trials
Stress Disorders, Traumatic	3
Stress Disorders, Post-Traumatic	3
Adrenocortical Hyperfunction	2
Diabetes Mellitus, Type 2	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
Not yet recruiting	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
NIH	3

KORLYM (mifepristone) Clinical Trials Update, Market Analysis, and Projection (2024-2035)

Last updated: April 27, 2026

What is KORLYM and how is it positioned in the market?

KORLYM is mifepristone, a glucocorticoid receptor antagonist indicated in the US for endogenous Cushing’s syndrome patients with:

• persistent hyperglycemia and/or diabetes, and
• elevated UFC (urinary free cortisol) despite surgery and/or who are not candidates for pituitary surgery, and
• mifepristone is used as a medical therapy for the disease course.

In practice, KORLYM’s market position depends on a narrow, label-driven population: patients with active endogenous Cushing’s syndrome who require medical control and who fit the insurer- and label-aligned clinical pathways. The therapy is most relevant where:

• surgery is delayed, incomplete, or not feasible, and
• clinicians seek a non-sterile surgical route that can control cortisol biology without relying on adrenal steroidogenesis inhibitors.

Where does KORLYM’s clinical pipeline stand?

KORLYM’s label is anchored to Cushing’s syndrome programs and does not have an expansive late-stage expansion engine versus larger endocrine franchises. The most actionable “clinical trials update” for business decisions is therefore twofold: (1) ongoing or newly reported evidence tied to Cushing’s syndrome management and (2) real-world adoption signals that influence continuation of prescribing and payer coverage.

Based on publicly available trial literature and regulatory information, KORLYM’s core evidence base is centered on clinical studies leading to approval for endogenous Cushing’s syndrome, with the most widely cited pivotal program being in the Cushing’s syndrome setting. The ongoing commercial question is not whether mifepristone “works” in the condition class; it is whether the market can expand through improved patient identification, payer comfort with long-term therapy, and physician comfort in managing drug-related effects (including cortisol rebound dynamics and monitoring).

Clinical program map (high level)

• Indication class: Endogenous Cushing’s syndrome (hyperglycemia/diabetes and elevated UFC subpopulation aligned to labeling and typical use-cases)
• Mechanism: Glucocorticoid receptor antagonism (counter cortisol action at the receptor level)
• Commercial implication: Adoption depends on long-term monitoring requirements and clinician familiarity with steroid receptor blockade management.

What is the current competitive landscape for endogenous Cushing’s syndrome?

KORLYM’s competitive set spans:

• Steroidogenesis inhibitors (adrenal-directed cortisol synthesis suppression)
• Pituitary-directed medical therapies in selected cases (especially where etiology supports it)
• Bilateral adrenalectomy and repeat surgery as non-pharmacologic alternatives in eligible patients

KORLYM competes on:

• oral administration and clinician willingness to manage chronic receptor blockade
• its utility where steroidogenesis inhibition is not preferred or is insufficient
• differentiating clinical profiles versus adrenal-directed approaches

The market structure remains concentrated: the total addressable population in major markets is small, and the disease diagnosis pathway often delays treatment initiation. That favors therapies that are:

• label-clear,
• reimbursable,
• and supported by clinician experience with safety monitoring.

What is the KORLYM market size and demand drivers?

Because endogenous Cushing’s syndrome is a rare endocrine disorder with long diagnostic latency and substantial heterogeneity in etiology, the practical TAM is capped by:

• diagnosed cases that meet label-like clinical criteria (persistent hyperglycemia/diabetes and elevated UFC),
• ineligible or not-ready candidates for definitive surgery, and
• payer acceptance and clinician willingness to treat chronically.

Demand drivers

1. Clinical need in medically managed patients
   • A meaningful subset of patients cannot proceed to surgery quickly or completely.
2. Physician selection
   • Endocrinologists select therapies based on comorbid burden (diabetes, obesity, hypertension) and expected monitoring burden.
3. Payer policy
   • Coverage criteria influence time to treatment initiation and persistence.
4. Therapy persistence and switching
   • In rare disease, discontinuation due to tolerability or monitoring challenges can materially affect realized revenue.

What could 2024-2035 revenue trajectory look like for KORLYM?

The highest-leverage forecast variables are not “market growth” in the broad sense but:

• dose persistence (patients remaining on therapy long enough to sustain revenues),
• new starts (diagnosed medically managed patients who accept KORLYM versus alternative medical routes),
• pricing and access (net price after rebates and patient assistance dynamics), and
• competitive switching (especially if alternative agents gain preference in guidelines or clinical practice).

Scenario framework (base, upside, downside)

Below is a projection structure that ties revenue to patient starts and persistence. This is the most decision-relevant approach for a rare disease product where annual revenue swings can follow small absolute patient changes.

Key modeling levers

• Annual patient starts (diagnosed medically managed Cushing’s patients)
• Average duration on therapy (persistence)
• Average net price per patient-year (net revenue per treated patient)

Projected growth profile (directional)

• Base case: Stable to modest growth driven by incremental adoption and persistence, constrained by rarity and competitive switching.
• Upside case: Faster adoption through improved treatment algorithms and payer comfort, potentially modestly increasing starts and persistence.
• Downside case: Competitive substitution and payer tightening reduce starts and persistence, pushing revenue flat to declining.

Because endogenous Cushing’s syndrome is rare and KORLYM’s label does not map to a broad screening-driven population, large penetration-driven growth is unlikely. Revenue growth is most likely to come from incremental shifts in medical management selection.

How do clinical and regulatory considerations affect KORLYM adoption?

Adoption is influenced by:

• label specificity and alignment with typical clinical practice,
• monitoring feasibility (glucocorticoid receptor antagonism requires structured oversight),
• and physician experience with long-term management.

In this setting, even without large pipeline expansion, the product can hold or gain share if:

• clinical pathways steer medically managed Cushing’s patients toward glucocorticoid receptor antagonism,
• and payers maintain coverage for the intended clinical phenotype.

What are the current risks to KORLYM revenue?

Revenue risk is concentrated in four buckets:

• Competitive substitution by steroidogenesis inhibitors and other endocrine medical options.
• Payer coverage friction that restricts new starts or delays therapy initiation.
• Discontinuation driven by tolerability and monitoring burden.
• Treatment paradigm shifts if guidelines or payer policies prioritize other medical routes.

What are the most decision-relevant “watch items” for the next 24-36 months?

The business-relevant watchlist for KORLYM is less about new indications and more about evidence and practice signals tied to Cushing’s syndrome care:

• updates from ongoing or newly published Cushing’s syndrome studies with mifepristone,
• real-world persistence and discontinuation patterns,
• payer coverage changes in the US and EU,
• label or dosing guidance updates that shift initiation thresholds.

Key Takeaways

• KORLYM is positioned for a high-specificity, medically managed endogenous Cushing’s syndrome population where surgery is delayed, incomplete, or not feasible.
• The commercial horizon is dominated by adoption, persistence, and payer coverage, not by broad label expansion.
• Forecasting KORLYM requires a patient-start and persistence model due to the rarity of the indication and the small absolute patient base.
• The main revenue upside comes from incremental share shifts in medical management selection, while the main downside comes from competitive substitution and coverage friction.

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FAQs

1) Is KORLYM’s market growth driven by new indications?

No. Market expansion depends primarily on uptake in the existing endogenous Cushing’s syndrome population aligned to clinical practice and payer criteria.

2) What is KORLYM’s competitive set?

It competes against steroidogenesis inhibitors and other endocrine medical management options used in endogenous Cushing’s syndrome when surgery is not definitive or not immediately available.

3) What determines KORLYM persistence?

Persistence depends on tolerability, structured monitoring requirements, and clinician comfort managing long-term glucocorticoid receptor antagonism.

4) Where does payer policy matter most?

Payer policy matters most for authorization for medically managed patients and for maintaining coverage over chronic treatment.

5) What is the best forecasting approach for KORLYM?

A patient flow model built on annual starts, average duration on therapy, and net price is the most decision-relevant method for rare endocrine indications.

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References

[1] US Food and Drug Administration. KORLYM (mifepristone) Prescribing Information. (Accessed via FDA label documentation).
[2] EMEA/European Medicines Agency. Assessment documents and EPAR for mifepristone (KORLYM).
[3] Published clinical trials and reviews on mifepristone in endogenous Cushing’s syndrome, including pivotal study publications referenced in regulatory reviews.