CLINICAL TRIALS PROFILE FOR ACTHAR

All Clinical Trials for Acthar

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00805753 ↗	Dose-Finding Pilot Study of ACTH in Patients With Idiopathic Membranous Nephropathy	Completed	Mallinckrodt	Phase 1	2009-01-01	This pilot study is aimed at demonstrating the effectiveness of ACTH (H.P. Acthar Gel) on the lipid profile and proteinuria in participants with MN. ACTH or adrenocorticotrophin is a hormone produced by the pituitary gland (a gland at the base of your brain) that is involved in stimulating your adrenal glands to secrete a number of steroid products (e.g. cortisol, aldosterone, corticosterone, and others) that are important in keeping you alive. The drug used in this study has been approved by the Food and Drug Administration (FDA) for routine clinical use in the treatment of patients with proteinuria and patients with idiopathic nephrotic syndrome such as idiopathic MN. However, the most adequate dose to use has not been adequately assessed. This is the reason for conducting this research study.
NCT00805753 ↗	Dose-Finding Pilot Study of ACTH in Patients With Idiopathic Membranous Nephropathy	Completed	Mayo Clinic	Phase 1	2009-01-01	This pilot study is aimed at demonstrating the effectiveness of ACTH (H.P. Acthar Gel) on the lipid profile and proteinuria in participants with MN. ACTH or adrenocorticotrophin is a hormone produced by the pituitary gland (a gland at the base of your brain) that is involved in stimulating your adrenal glands to secrete a number of steroid products (e.g. cortisol, aldosterone, corticosterone, and others) that are important in keeping you alive. The drug used in this study has been approved by the Food and Drug Administration (FDA) for routine clinical use in the treatment of patients with proteinuria and patients with idiopathic nephrotic syndrome such as idiopathic MN. However, the most adequate dose to use has not been adequately assessed. This is the reason for conducting this research study.
NCT00854750 ↗	Modeling and Treating the Pathophysiology of Demyelination in Multiple Sclerosis	Terminated	Mallinckrodt	Phase 4	2009-05-01	The investigators principal hypothesis is that INO and optic neuritis are objective, quantitative, and reproducible models for corroborating the hypothesis that changes in core body temperature are associated with the reversible and stereotypic decay in axonal conduction and that ACTHAR can serve to prevent such changes. The application of ocular motor and optic nerve measures appears to constitute a useful paradigm to detect and monitor responses to therapeutic strategies that stabilize nerve cell membranes in response to temperature induced decay in axonal conduction mechanisms, with implications on activities of daily life that are dependent upon vision (reading, driving, walking, work performance).
NCT00854750 ↗	Modeling and Treating the Pathophysiology of Demyelination in Multiple Sclerosis	Terminated	Elliot Frohman	Phase 4	2009-05-01	The investigators principal hypothesis is that INO and optic neuritis are objective, quantitative, and reproducible models for corroborating the hypothesis that changes in core body temperature are associated with the reversible and stereotypic decay in axonal conduction and that ACTHAR can serve to prevent such changes. The application of ocular motor and optic nerve measures appears to constitute a useful paradigm to detect and monitor responses to therapeutic strategies that stabilize nerve cell membranes in response to temperature induced decay in axonal conduction mechanisms, with implications on activities of daily life that are dependent upon vision (reading, driving, walking, work performance).
NCT01021540 ↗	Prospective Study Evaluating the Effect of Repository Corticotropin in the Treatment of Various Nephrotic Syndromes	Completed	Arizona Kidney Disease and Hypertension Center	Phase 4	2009-12-01	To determine if H.P. Acthar Gel (repository corticotrophin) has the same anti-proteinuric effects seen with the synthetic ACTH analogue in Europe.

Clinical Trial Conditions for Acthar

Condition Name

Condition Name for Acthar
Intervention	Trials
Multiple Sclerosis	8
Sarcoidosis	5
Rheumatoid Arthritis	4
Proteinuria	4

Condition MeSH

Condition MeSH for Acthar
Intervention	Trials
Sclerosis	14
Multiple Sclerosis	14
Kidney Diseases	10
Arthritis	8

Clinical Trial Locations for Acthar

Trials by Country

Trials by Country for Acthar
Location	Trials
United States	254
Mexico	20
Argentina	10
Chile	7
Canada	5

Trials by US State

Trials by US State for Acthar
Location	Trials
California	24
New York	23
Texas	18
Florida	16
Ohio	13

Clinical Trial Progress for Acthar

Clinical Trial Phase

Clinical Trial Phase for Acthar
Clinical Trial Phase	Trials
Phase 4	44
Phase 3	6
Phase 2/Phase 3	3

Clinical Trial Status

Clinical Trial Status for Acthar
Clinical Trial Phase	Trials
Completed	32
Unknown status	11
Withdrawn	10

Clinical Trial Sponsors for Acthar

Sponsor Name

Sponsor Name for Acthar
Sponsor	Trials
Mallinckrodt	55
The Cleveland Clinic	3
University of Colorado, Denver	3

Sponsor Type

Sponsor Type for Acthar
Sponsor	Trials
Other	86
Industry	58

ACTHAR Gel: Clinical Trials, Market Analysis, and Projections

Introduction to ACTHAR Gel

ACTHAR Gel, also known as repository corticotropin injection, is a medication used to treat various autoimmune and inflammatory conditions. It is a purified form of adrenocorticotropic hormone (ACTH), which stimulates the adrenal glands to produce cortisol and other corticosteroids. Here, we will delve into the latest updates on clinical trials, market analysis, and projections for ACTHAR Gel.

Clinical Evidence and Trials

FDA-Approved Indications

ACTHAR Gel has a robust history of clinical evidence supporting its effectiveness in several FDA-approved indications. For instance, it has been approved for the treatment of infantile spasms, with clinical trials showing that 86.7% of patients responded to ACTHAR Gel compared to 28.6% who responded to prednisone[1].

Ongoing and Planned Trials

Mallinckrodt has initiated several company-sponsored clinical trials to further explore the efficacy of ACTHAR Gel. These include Phase 4 clinical trials for rheumatoid arthritis, systemic lupus erythematosus, and focal segmental glomerulosclerosis, as well as a Phase 2 proof-of-concept trial for amyotrophic lateral sclerosis (ALS)[1].

Rheumatoid Arthritis Trials

In a Phase 4 study on rheumatoid arthritis, ACTHAR Gel demonstrated significant improvements in disease activity. A majority of patients (63%) treated with ACTHAR Gel achieved low disease activity (LDA) at Week 12, as measured by the Disease Activity Score with 28 joint count and erythrocyte sedimentation rate (DAS28-ESR)[4].

Multiple Sclerosis and Other Indications

ACTHAR Gel has also shown efficacy in speeding the resolution of acute exacerbations of multiple sclerosis. Additionally, trials are underway or nearing initiation for other indications, including multiple sclerosis and ophthalmic conditions[1].

Market Analysis

Current Market Trends

The ACTH market, which includes ACTHAR Gel and other ACTH products like Cortrophin Gel, has seen a stabilization and return to growth after years of decline. In 2024, the ACTH market is expected to grow by more than 20% on a dollar basis, driven by the launch of new products and the expansion of existing ones into new therapeutic areas[2].

Sales Projections

Mallinckrodt expects around 10% growth for ACTHAR Gel in 2024, with projected sales of $664-668 million for the ACTH market, including both ACTHAR Gel and Cortrophin Gel[2].

Competitive Landscape

The ACTH market has limited competition, with only a few products available. The re-introduction of Cortrophin Gel in January 2022 has provided a much-needed alternative for patients and physicians, contributing to the market's growth[2].

Health Economics and Cost-Effectiveness

Recent health economics analyses presented by Mallinckrodt highlight the cost-effectiveness of ACTHAR Gel in treating conditions such as advanced symptomatic sarcoidosis and nephrotic syndrome. These studies show significant reductions in corticosteroid use and improvements in clinical outcomes, which can lead to better patient care and reduced healthcare costs[3].

Market Projections

Growth Potential

The ACTH market is projected to continue its growth trajectory, driven by the expansion into new therapeutic areas such as ophthalmology and pulmonology. The potential for growth is further enhanced by the low number of patients currently on ACTH therapy, indicating a substantial untapped market[2].

Future Indications and Expansion

With ongoing and planned trials for various indications, ACTHAR Gel is poised to expand its market presence. The drug's versatility and the company's commitment to providing up-to-date research support its potential for future growth[1].

Impact on Patient Care

Clinical Outcomes

ACTHAR Gel has been associated with significant improvements in clinical outcomes across various conditions. For example, in patients with nephrotic syndrome, treatment with ACTHAR Gel led to a reduction in the proportion of patients taking corticosteroids and a decrease in the average daily dose of corticosteroids[3].

Patient and Physician Choice

The availability of ACTHAR Gel and other ACTH products has reintroduced a necessary choice for patients and physicians, particularly in areas where few treatment options were previously available[2].

Key Takeaways

• Clinical Evidence: ACTHAR Gel has robust clinical evidence supporting its effectiveness in multiple FDA-approved indications.
• Ongoing Trials: Several clinical trials are underway to explore new indications and further establish the drug's efficacy.
• Market Growth: The ACTH market is expected to grow significantly, driven by the launch of new products and expansion into new therapeutic areas.
• Cost-Effectiveness: ACTHAR Gel has been shown to be cost-effective in treating various autoimmune and inflammatory conditions.
• Future Potential: The drug has substantial growth potential due to its versatility and the company's commitment to ongoing research.

FAQs

What is ACTHAR Gel used for?

ACTHAR Gel is used to treat various autoimmune and inflammatory conditions, including infantile spasms, rheumatoid arthritis, systemic lupus erythematosus, and nephrotic syndrome.

What are the current clinical trials for ACTHAR Gel?

Current trials include Phase 4 studies for rheumatoid arthritis, systemic lupus erythematosus, and focal segmental glomerulosclerosis, as well as a Phase 2 trial for amyotrophic lateral sclerosis (ALS).

How is the ACTH market expected to grow?

The ACTH market is expected to grow by more than 20% in 2024, driven by new product launches and expansion into new therapeutic areas.

What is the competitive landscape of the ACTH market?

The ACTH market has limited competition, with only a few products available, including ACTHAR Gel and Cortrophin Gel.

What are the health economics implications of using ACTHAR Gel?

ACTHAR Gel has been shown to reduce corticosteroid use and improve clinical outcomes, leading to better patient care and reduced healthcare costs.

Sources

1. Mallinckrodt Statement on H.P. Acthar Gel (Repository Corticotropin Injection) - Mallinckrodt.
2. Jefferies Healthcare Conference - ANI Pharmaceuticals, Inc.
3. Mallinckrodt Presents Latest Health Economics Data on Acthar Gel - PR Newswire.
4. Rheumatoid Arthritis Phase 4 Study - Acthar Gel - Acthar HCP.
5. Pituitary ACTH Hypersecretion Drug Market Analysis 2025-2032 - Wicz.