Cushing Syndrome Pipeline Insight

DelveInsight’s, “Cushing Syndrome - Pipeline Insight, 2025” report provides comprehensive insights about 4+ companies and 4+ pipeline drugs in Cushing Syndrome pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

 

Geography Covered

• Global coverage

 

Cushing Syndrome: Understanding

Cushing Syndrome: Overview

Cushing’s syndrome arises from prolonged exposure to elevated levels of circulating glucocorticoids, with Cushing’s disease, pituitary-dependent hypercortisolism, being the most common cause of endogenous cases.

It is characterized by a wide range of symptoms, with common manifestations including decreased libido, obesity or weight gain, plethora (reddish complexion), rounded face, menstrual changes, hirsutism, hypertension, easy bruising (ecchymoses), lethargy, depression, proximal muscle weakness, and skin changes such as striae. Less common symptoms may involve EKG abnormalities or atherosclerosis, dorsal fat pad, edema, abnormal glucose tolerance, osteopenia or fractures, headaches, backaches, recurrent infections, abdominal pain, acne, and female-pattern baldness.

The etiology of Cushing’s syndrome is broadly classified into ACTH-dependent and ACTH-independent forms. ACTH-dependent cases result from excessive adrenocorticotropic hormone (ACTH) secretion, stimulating all three layers of the adrenal cortex and causing bilateral adrenocortical hyperplasia and hypertrophy, which increases adrenal weight and often produces micronodular or occasionally macronodular changes. This leads to elevated glucocorticoid levels, often accompanied by a mild rise in serum androgens. In contrast, ACTH-independent forms are characterized by low plasma ACTH levels, typically due to autonomous adrenal glucocorticoid overproduction or prolonged exogenous glucocorticoid use. While adrenal adenomas generally secrete only glucocorticoids, other endogenous adrenal disorders may also increase androgen and steroid precursor production. The microscopic and macroscopic features of unaffected adrenal tissue vary depending on the underlying cause.

Cushing’s disease, or pituitary-dependent Cushing’s syndrome, is the leading cause of endogenous Cushing’s syndrome, responsible for approximately 60–80% of cases.

In Cushing disease, the secretion rate of ACTH remains constant, but the normal circadian rhythm is disrupted. Elevated plasma ACTH levels stimulate bilateral adrenal hyperplasia, resulting in excessive cortisol production and loss of cortisol’s normal diurnal pattern. While cortisol primarily acts as a glucocorticoid, at elevated concentrations it also exhibits mineralocorticoid effects, contributing to hypertension and hypokalemia via activation of the renin-angiotensin-aldosterone system. This hormonal system regulates plasma sodium levels and arterial blood pressure, which can indirectly cause potassium depletion.

Treatment of Cushing disease focuses on removing or controlling the source of excess ACTH and cortisol. First-line therapy is transsphenoidal surgery (TSS) to resect the pituitary adenoma, with higher success rates for microadenomas (65–90%) than macroadenomas (<65%). If surgery fails, options include repeat surgery, pituitary radiotherapy (especially effective in children), or bilateral adrenalectomy for immediate cortisol reduction—though this requires lifelong steroid replacement and carries the risk of Nelson syndrome. Medical therapy is used for recurrent, persistent, or inoperable disease, targeting ACTH suppression (pasireotide, cabergoline), adrenal steroidogenesis inhibition (ketoconazole, metyrapone, mitotane, osilodrostat, etomidate), or glucocorticoid receptor blockade (mifepristone). Each drug has distinct benefits and risks, such as hyperglycemia with pasireotide, hepatotoxicity with ketoconazole, and anti-progesterone effects with mifepristone. Lifelong monitoring is essential, as recurrence occurs in about one-third of patients, with late-night salivary cortisol being the most sensitive predictor.

 

"Cushing Syndrome- Pipeline Insight, 2025" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Cushing Syndrome pipeline landscape is provided which includes the disease overview and Cushing Syndrome treatment guidelines. The assessment part of the report embraces, in depth Cushing Syndrome commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Cushing Syndrome collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

 

Report Highlights

• The companies and academics are working to assess challenges and seek opportunities that could influence Cushing Syndrome R&D. The therapies under development are focused on novel approaches to treat/improve Cushing Syndrome.

 

Cushing Syndrome Emerging Drugs Chapters

This segment of the Cushing Syndrome report encloses its detailed analysis of various drugs in different stages of clinical development, including Phase III, II, I, Preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

 

Cushing Syndrome Emerging Drugs

• ST-002: Stero Therapeutics

ST-002 is specifically designed to address the hyperglycemia and non-alcoholic steatohepatitis (NASH) associated with Cushing’s syndrome, offering a unique combination of glucocorticoid inhibition, anti-inflammatory, and anti-diabetic effects. Unlike other anti-glucocorticoid drugs, ST-002 suppresses glucocorticoid activity without reducing glucocorticoid levels, thereby avoiding adrenal insufficiency. Its safety profile has been established in over 120 patients using prior oral formulations, and as a new chemical entity (NCE), ST-002 demonstrates improved bioavailability and solubility compared to earlier versions. The U.S. FDA has granted it Orphan Disease Designation, and SteroTherapeutics is advancing its development in collaboration with the NIH under a Cooperative Research and Development Agreement (CRADA). Currently, the drug is in Phase II stage of its clinical trial for Cushing Syndrome.

Further product details are provided in the report……..

 

Cushing Syndrome: Therapeutic Assessment

This segment of the report provides insights about the different Cushing Syndrome drugs segregated based on following parameters that define the scope of the report, such as:

 

Major  Players in Cushing Syndrome

• There are approx. 4+ key companies which are developing the therapies for Cushing Syndrome. The companies which have their Cushing Syndrome drug candidates in the most advanced stage, i.e. Phase II include, Stero Therapeutics.

 

Phases

DelveInsight’s report covers around 4+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

 

Route of Administration

Cushing Syndrome pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

 

Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

 

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

 

Cushing Syndrome: Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III, II, I, preclinical and discovery stage. It also analyses Cushing Syndrome therapeutic drugs key players involved in developing key drugs.

 

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Cushing Syndrome drugs.

 

Cushing Syndrome Report Insights

• Cushing Syndrome Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

 

Cushing Syndrome Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

 

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Cushing Syndrome drugs?
• How many Cushing Syndrome drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Cushing Syndrome?
• What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Cushing Syndrome therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Cushing Syndrome and their status?
• What are the key designations that have been granted to the emerging drugs?