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.
NCT01925092 ↗	Mifepristone in Children With Refractory Cushing's Disease	Withdrawn	Corcept Therapeutics	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.
NCT01946685 ↗	Novel Therapeutics in Posttraumatic Stress Disorder (PTSD): A Randomized Clinical Trial of Mifepristone	Completed	Durham VA Medical Center	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.
NCT01946685 ↗	Novel Therapeutics in Posttraumatic Stress Disorder (PTSD): A Randomized Clinical Trial of Mifepristone	Completed	San Diego Veterans Healthcare System	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
Alcoholism	1
PTSD	1
Anxiety Disorder	1

Condition MeSH

Condition MeSH for Korlym
Intervention	Trials
Stress Disorders, Traumatic	3
Stress Disorders, Post-Traumatic	3
Diabetes Mellitus	2
Alcoholism	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
Recruiting	1

Clinical Trial Sponsors for Korlym

Sponsor Name

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

Sponsor Type

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

Corcept Therapeutics and the Evolution of Korlym: Clinical Trials, Market Analysis, and Projections

Introduction

Korlym, developed by Corcept Therapeutics, has been a pivotal drug in the treatment of hypercortisolism, particularly in patients with Cushing's syndrome. Here, we delve into the latest clinical trials, market performance, and future projections for this significant therapeutic agent.

Clinical Trials Update

CATALYST Trial

One of the most recent and significant clinical trials for Korlym is the CATALYST study, a Phase 4 trial designed to evaluate the efficacy of Korlym in patients with hypercortisolism (Cushing’s syndrome) and difficult-to-control type 2 diabetes. The trial consisted of two parts: a screening phase and a treatment phase.

• Screening Phase: This phase involved screening 1,057 patients with difficult-to-control type 2 diabetes, revealing that 23.8% of these patients had hypercortisolism[4][5].
• Treatment Phase: In this phase, 136 patients were randomized to receive either Korlym or a placebo for 24 weeks. The results showed a clinically meaningful and statistically significant reduction in hemoglobin A1c levels in patients treated with Korlym, with a decrease of 1.47% compared to a 0.15% decrease in the placebo group (p-value: < 0.0001)[4][5].

GRACE Trial

The GRACE trial, another critical study, is evaluating relacorilant, a non-steroidal, selective glucocorticoid receptor modulator, but also includes data relevant to Korlym. The open-label phase of the GRACE trial demonstrated significant improvements in various clinically meaningful endpoints for patients with Cushing's syndrome, including hypertension, hyperglycemia, weight, waist circumference, cognitive impairment, and quality of life[1].

Other Ongoing Trials

• CATALYST and Beyond: In addition to the CATALYST trial, Corcept is conducting other trials, such as the ROSELLA trial, which is a pivotal Phase 3 trial of relacorilant plus nab-paclitaxel in patients with platinum-resistant ovarian cancer, and the DAZALS trial, a Phase 2 trial of dazucorilant in patients with Amyotrophic Lateral Sclerosis (ALS)[1][3].

Market Analysis

Revenue Growth

Korlym has been a consistent revenue driver for Corcept Therapeutics since its FDA approval in February 2012. In the first half of 2024, sales of Korlym increased by 39.1% year over year to $310.6 million, driven by strong demand and a record number of patients receiving the medication[2][3].

• Financial Performance: For the third quarter of 2024, Korlym generated net product revenue of $182.5 million, a 48% increase from the third quarter of 2023. This strong performance has led Corcept to raise its 2024 revenue outlook multiple times, with the latest guidance ranging between $675 million and $700 million[3].

Market Expansion

The positive results from the CATALYST trial are expected to expand Korlym's market significantly. The study's findings indicate that hypercortisolism is more prevalent than previously assumed, particularly among patients with difficult-to-control type 2 diabetes. This could lead to increased screening and treatment of hypercortisolism, further boosting Korlym's sales[4][5].

Investor Sentiment and Stock Performance

Stock Rally

Corcept Therapeutics' stock has seen a significant rally in 2024, with shares increasing by 44% against an industry decline of 1.2%. This growth is attributed to the strong financial performance and the promising pipeline of clinical candidates, including relacorilant[2].

• Zacks Rank: Corcept currently holds a Zacks Rank #1 (Strong Buy), reflecting the positive outlook on the company's earnings and growth potential. The Zacks Consensus Estimate for Corcept's 2024 earnings per share has been revised upward from $1.07 to $1.12, and for 2025 from $1.55 to $1.63[2].

Future Projections

Regulatory Milestones

Corcept is anticipating several key regulatory milestones in the coming quarters. The submission of a New Drug Application (NDA) for relacorilant in Cushing's syndrome is expected this quarter, which could further diversify the company's revenue streams[1][3].

Clinical Trial Results

Results from several ongoing trials, including the GRADIENT, ROSELLA, and DAZALS trials, are expected by the end of 2024. These trials will provide critical data on the efficacy and safety of Corcept's pipeline candidates, potentially leading to new treatment options for various serious disorders[1][3].

Market Expansion and Penetration

Given the positive results from the CATALYST trial and the increasing recognition of hypercortisolism among physicians, Korlym is poised for continued market expansion. The drug's orphan status in the United States and Europe for treating endogenous Cushing's syndrome further supports its market position[2][3].

Key Takeaways

• Clinical Success: Korlym has demonstrated significant efficacy in treating hypercortisolism and difficult-to-control type 2 diabetes, as evidenced by the CATALYST trial.
• Revenue Growth: Strong demand and expanded patient base have driven Korlym's revenue growth, with sales increasing by 39.1% year over year in the first half of 2024.
• Market Expansion: The prevalence of hypercortisolism among patients with type 2 diabetes is higher than previously thought, suggesting a larger market potential for Korlym.
• Regulatory and Clinical Milestones: Upcoming NDA submissions and trial results will be crucial for Corcept's future growth and diversification of its revenue streams.
• Investor Sentiment: Corcept's stock has rallied significantly, reflecting positive investor sentiment driven by strong financial performance and a promising pipeline.

FAQs

Q: What is the primary indication for Korlym?

A: Korlym is primarily indicated for the treatment of hyperglycemia secondary to hypercortisolism in adults with endogenous Cushing's syndrome who have type 2 diabetes mellitus or glucose intolerance and are either unable to undergo surgery or have failed surgical treatment[3].

Q: What were the key findings of the CATALYST trial?

A: The CATALYST trial showed that 23.8% of patients with difficult-to-control type 2 diabetes had hypercortisolism. Patients treated with Korlym exhibited a statistically significant reduction in hemoglobin A1c levels compared to the placebo group[4][5].

Q: How has Korlym impacted Corcept Therapeutics' revenue?

A: Korlym has been a consistent revenue driver for Corcept Therapeutics, with sales increasing by 39.1% year over year in the first half of 2024. This has led to multiple revisions of the company's revenue guidance upward[2][3].

Q: What are the upcoming regulatory and clinical milestones for Corcept Therapeutics?

A: Corcept is expecting to submit an NDA for relacorilant in Cushing's syndrome this quarter and will release results from several ongoing trials, including GRADIENT, ROSELLA, and DAZALS, by the end of 2024[1][3].

Q: How has Corcept Therapeutics' stock performed in 2024?

A: Corcept Therapeutics' stock has rallied by 44% in 2024, outperforming the industry. This growth is driven by strong financial results and the promising pipeline of clinical candidates[2].