CLINICAL TRIALS PROFILE FOR ACTHAR GEL

All Clinical Trials for Acthar Gel

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).

Clinical Trial Conditions for Acthar Gel

Condition Name

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

Condition MeSH

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

Clinical Trial Locations for Acthar Gel

Trials by Country

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

Trials by US State

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

Clinical Trial Progress for Acthar Gel

Clinical Trial Phase

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

Clinical Trial Status

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Withdrawn	10

Clinical Trial Sponsors for Acthar Gel

Sponsor Name

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

Sponsor Type

Sponsor Type for Acthar Gel
Sponsor	Trials
Other	86
Industry	58

ACTHAR GEL: Clinical Trials, Market Analysis, and Projections

Introduction

Acthar Gel, a repository corticotropin injection, is a versatile treatment option for various autoimmune and inflammatory conditions. This article provides an update on the clinical trials, market analysis, and future projections for Acthar Gel.

Clinical Trials Update

Diabetic Nephropathy Study

A notable clinical trial update involves the Phase 2A pilot study evaluating Acthar Gel for the treatment of diabetic nephropathy (DN). This study, initiated under previous ownership, faced significant challenges, including under-enrollment and design issues. As a result, the study failed to generate sufficient data to support clinically relevant conclusions. Mallinckrodt, the current owner, has decided not to pursue this indication further[1].

Rheumatoid Arthritis and Other Indications

Acthar Gel has shown promising results in other clinical trials. For instance, in a Phase 4 study on rheumatoid arthritis, Acthar Gel demonstrated efficacy in achieving low disease activity (LDA) in patients with persistently active disease despite treatment. A significant proportion of patients (63%) achieved LDA at Week 12, and the treatment was associated with improvements in swollen and tender joint counts, fatigue, and physical function[4].

Systemic Lupus Erythematosus (SLE)

In the context of SLE, Acthar Gel has been evaluated for its cost-effectiveness compared to the standard of care. The study indicated that Acthar Gel is a cost-effective treatment option for patients with moderate-to-severe SLE, resulting in reduced direct medical and indirect costs. The incremental cost-effectiveness ratio (ICER) was favorable, suggesting that Acthar Gel provides value at a willingness-to-pay threshold of $150,000 over 2-3 years[3].

Market Analysis

Current Market Landscape

The ACTH market, which includes Acthar Gel and other corticotropin products like Cortrophin Gel, has experienced significant fluctuations. However, with the launch of Cortrophin Gel in January 2022, the market has stabilized and returned to double-digit growth. The ACTH market is projected to grow by more than 20% in 2024 on a dollar basis, driven by new and returning prescribers serving appropriate patients[2].

Market Sales Projections

For 2024, the estimated sales for the ACTH market are between $664 million and $668 million, combining the guidance for both Acthar Gel and Cortrophin Gel. Acthar Gel itself is expected to see around 10% growth in 2024, according to Mallinckrodt's third-quarter earnings release[2].

Competition and Market Dynamics

The ACTH market is characterized by limited competition, with only a few products available. Acthar Gel and Cortrophin Gel are the primary players, with Cortrophin Gel recently expanding into therapeutic areas such as ophthalmology and pulmonology. This limited competition and the high barriers to entry contribute to the long-term sustainability of these products[2].

Pricing and Access

Pricing Strategy

Mallinckrodt has announced a 3% price increase for Acthar Gel effective January 1, 2025, which is below the rate of Consumer Price Index inflation. The company emphasizes its commitment to ensuring patient access to therapy through investments in research and development, patient assistance programs, and the introduction of new delivery systems like the Single-Dose Pre-filled SelfJect™ Injector[5].

Discounts and Patient Assistance

While the list price of Acthar Gel is substantial, it is important to note that significant discounts are frequently available to customers. Mallinckrodt continues to invest in patient assistance programs to ensure that appropriate patients can access the therapy[5].

Future Projections

Growth Potential

The ACTH market, driven by products like Acthar Gel and Cortrophin Gel, is expected to continue its growth trajectory. The potential for significant growth is further enhanced by the current lower number of patients on ACTH therapy compared to previous years, offering a substantial opportunity for expansion[2].

Therapeutic Expansion

Acthar Gel has been approved for multiple indications, including neurology, nephrology, rheumatology, ophthalmology, and pulmonology. The recent expansion into new therapeutic areas and the potential for treating conditions like acute gouty arthritis flares not included in previous peak sales estimates suggest a strong multi-year growth trajectory[2].

Cost-Effectiveness and Value

The cost-effectiveness studies, particularly in the context of SLE, highlight Acthar Gel as a value-based treatment option. This positioning is likely to support its continued use and adoption in various clinical settings, contributing to its market growth[3].

Key Takeaways

• Clinical Trials: Acthar Gel has faced challenges in certain trials, such as the diabetic nephropathy study, but has shown efficacy in other indications like rheumatoid arthritis and SLE.
• Market Analysis: The ACTH market is growing, driven by the stabilization and expansion of products like Acthar Gel and Cortrophin Gel.
• Pricing and Access: Mallinckrodt is committed to patient access through pricing strategies and patient assistance programs.
• Future Projections: The market is expected to grow significantly, driven by therapeutic expansions and the potential for treating more patients.

FAQs

Q: What is the current status of Acthar Gel in treating diabetic nephropathy?

A: The Phase 2A pilot study for diabetic nephropathy was under-powered and failed to generate sufficient data, leading Mallinckrodt to decide not to pursue this indication further[1].

Q: How effective is Acthar Gel in treating rheumatoid arthritis?

A: Acthar Gel has shown significant efficacy in achieving low disease activity in patients with rheumatoid arthritis, with 63% of patients achieving LDA at Week 12[4].

Q: Is Acthar Gel cost-effective for treating systemic lupus erythematosus (SLE)?

A: Yes, Acthar Gel is considered a cost-effective treatment option for patients with moderate-to-severe SLE, resulting in reduced direct medical and indirect costs[3].

Q: What are the projected sales for the ACTH market in 2024?

A: The estimated sales for the ACTH market in 2024 are between $664 million and $668 million, combining the guidance for both Acthar Gel and Cortrophin Gel[2].

Q: How is Mallinckrodt ensuring patient access to Acthar Gel?

A: Mallinckrodt is committed to ensuring patient access through investments in research and development, patient assistance programs, and the introduction of new delivery systems like the Single-Dose Pre-filled SelfJect™ Injector[5].

Sources

1. Mallinckrodt plc. "Mallinckrodt Posts Data From Legacy H.P. Acthar® Gel (Repository Corticotropin Injection) Phase 2A Pilot Study in Diabetic Nephropathy Patients." July 25, 2017.
2. ANI Pharmaceuticals, Inc. "Jefferies Healthcare Conference." November 20, 2024.
3. PubMed. "Cost-Effectiveness of Acthar Gel Versus Standard of Care for the Treatment of Active Systemic Lupus Erythematosus." October 20, 2022.
4. Acthar HCP. "Rheumatoid Arthritis Phase 4 Study - Acthar Gel."
5. Mallinckrodt Pharmaceuticals. "Our Product Pricing - Acthar® Gel."