H.P. Acthar Gel (repository corticotropin injection) / Mallinckrodt - LARVOL DELTA

Cost per Response of Acthar® Gel vs Standard of Care for the Treatment of Proteinuria in Nephrotic Syndrome Due to Idiopathic Membranous Nephropathy Among Adults from the US Healthcare Perspective. (PubMed, J Health Econ Outcomes Res) - "Objective: The cost per response of Acthar® Gel vs standard of care (SoC; cyclophosphamide or rituximab) for treatment of proteinuria in NS due to iMN was evaluated among adults who had failed multiple treatments from a US payer perspective over a 1- to 3-year horizon... Based on current model assumptions and clinical inputs, Acthar® Gel may potentially be a cost-effective and value-based treatment strategy vs unapproved SoCs for adults with refractory proteinuria in NS due to iMN, particularly for those who have not responded to conventional therapies over a 1- to 3-year period within a US payer context. These results may inform clinical and payer decision-making in cases when other standard therapies fail to achieve desired outcomes for a specific population."

Journal • Glomerulonephritis • Nephrology • Renal Disease

Treatment Patterns and Outcomes of Acthar Gel in Symptomatic Sarcoidosis: A Physician-Reported Chart Review (WASOG-AASOG 2025) - "This study demonstrates that Acthar Gel is an effective treatment option for sarcoidosis. Patients experienced improvements in multiple areas such as symptoms, physical function, pain relief, fatigue, reduced corticosteroid use and strength. These findings underscore the utility of Acthar Gel in enhancing outcomes for patients with sarcoidosis."

Review • Cardiovascular • Fatigue • Hypertension • Immunology • Inflammation • Musculoskeletal Diseases • Musculoskeletal Pain • Orthopedics • Osteoarthritis • Rheumatology • Sarcoidosis

Treatment Patterns and Outcomes of Acthar Gel in Symptomatic Sarcoidosis: A Physician-Reported Chart Review (WASOG-AASOG 2025) - "This study demonstrates that Acthar Gel is an effective treatment option for sarcoidosis. Patients experienced improvements in multiple areas such as symptoms, physical function, pain relief, fatigue, reduced corticosteroid use and strength. These findings underscore the utility of Acthar Gel in enhancing outcomes for patients with sarcoidosis."

Review • Cardiovascular • Fatigue • Hypertension • Immunology • Inflammation • Musculoskeletal Diseases • Musculoskeletal Pain • Orthopedics • Osteoarthritis • Rheumatology • Sarcoidosis

Oral Abstract Session 3 (Clinical Science) (WASOG-AASOG 2025) - "During this session, top scoring abstracts will be highlighted through brief oral presentations, followed by Q&ADuring this session, top scoring abstracts will be highlighted through brief oral presentations, followed by Q&A. sarcoidosis trialsVivienne Kahlmann 15:1515:30Treatment Patterns and Outcomes of Acthar Gel in Symptomatic Sarcoidosis: A Physician-Reported Chart ReviewJohanna Purcell 15:3015:45Outcomes of Lung Transplantation for Pulmonary Sarcoidosis in the United States in the Lung Allocation Score EraPeter Sporn 15:4516:00Clinical Disease Characteristics and Cognitive Dysfunction in Patients with SarcoidosisElizabeth Wittrock"

Clinical • Cognitive Disorders • Immunology • Sarcoidosis

Melanocortin Receptor-Mediated Anti-Inflammatory Effect of Acthar® Gel in Human Myeloid Cells. (PubMed, Neuroimmunomodulation) - "As shown by microarray gene expression analysis, polarization of MDMs to a proinflammatory phenotype increased the expression and secretion of interleukin-6, tumor necrosis factor α, and CXC motif chemokine ligand 10, which were inhibited in a dose-dependent manner with Acthar Gel treatment. Conclusions These results are consistent with previous insights that Acthar Gel has an immunomodulatory mechanism distinct from glucocorticoids alone and suggest that Acthar Gel can improve clinical outcomes in MS and other inflammation-mediated central nervous system disorders by inhibiting multiple proinflammatory cytokine signaling pathways."

Journal • CNS Disorders • Multiple Sclerosis • Oncology • CXCL10 • IL6 • TNFA

Intercepting the complement amplification loop through podocyte MC5R signaling ameliorates membranous nephropathy. (PubMed, Mol Ther) - "Here, in a heterologous mouse model of THSD7A-associated membranous nephropathy, various melanocortin agents, including repository corticotropin injection, the non-steroidogenic pan-melanocortin receptor agonist NDP-MSH, and the selective melanocortin 5 receptor (MC5R) agonist PG-901, attenuated proteinuria and ameliorated glomerulopathy...Furthermore, glomerular fixation of C5b-9 and C3 was diminished by MC5R agonism but enhanced by MC5R knockout, despite comparable glomerular deposition of the heterologous nephritogenic antibody and activation of C4. Comprehensive complement cascade analysis revealed that this anti-complement effect of MC5R signaling stems from an inhibition of podocyte expression of complement factor B and D, critical regulators of the complement amplification loop, involving a PPARγ-dependent mechanism. Ergo, MC5R-mediated interception of the complement amplification loop in podocytes may serve as a novel therapeutic target for membranous nephropathy."

Journal • Glomerulonephritis • Renal Disease • CFB • PPARG

Cost-per-response of Acthar Gel versus standard of care for the treatment of noninfectious keratitis: a United States healthcare perspective. (PubMed, J Comp Eff Res) - "This study evaluated the cost-per-response of Acthar® Gel compared with standard of care ([SoC]; adalimumab or infliximab) for noninfectious keratitis from a US healthcare payer perspective over 1-3 years...Acthar Gel was a dominant treatment option versus SoC at 2 and 3 years. From a US healthcare payer perspective, Acthar Gel may be a cost-effective, value-based treatment option for appropriate patients with noninfectious keratitis."

Journal • Corneal Abrasion • Keratitis • Ocular Infections • Ocular Inflammation • Ophthalmology

The Therapeutic Effects of Purified Cortrophin Gel on Experimental Autoimmune Uveitis. (PubMed, Ocul Immunol Inflamm) - "A repository corticotropin injection (RCI) from ANI Pharmaceuticals that activates multiple melanocortin pathways was studied for its effects on experimental autoimmune uveitis (EAU), a murine model of human endogenous uveitis...RCI treatment effectively suppressed uveitis and protected the retina from inflammatory damage. These findings further illustrate the potential of melanocortin-based therapies for uveitis."

Journal • Immunology • Ocular Inflammation • Ophthalmology • Uveitis • IFNG • IL10 • IL17A • IL1B • TNFA

Repository corticotropin injection (RCI) attenuates inflammatory response in a pre-clinical murine collagen induced arthritis model (EULAR 2025) - "As monotherapy, RCI significantly attenuated CIA-induced joint swelling in mice. In addition, RCI treatment resulted in a reduction in inflammatory cytokine response, but no disruption of IgG formation and no additive bone loss. These findings demonstrate significant reductions in systemic inflammation in the CIA arthritis model."

Preclinical • Immunology • Inflammation • Osteoporosis • Rheumatology • CXCL1

ANI Pharmaceuticals Announces Presentation of New Preclinical Data (GlobeNewswire) - "ANI Pharmaceuticals, Inc...announced new preclinical data on the use of Purified Cortrophin Gel (repository corticotropin injection USP) (Cortrophin Gel) in a mouse model of murine collagen-induced inflammatory arthritis....The effect of repository corticotropin injection (RCI, an equivalent to Cortrophin Gel in this model), when used as monotherapy, was evaluated using a mouse collagen-induced arthritis (CIA) model to assess the inflammatory response and bone damage. Following CIA induction, mice received subcutaneous RCI (40 or 400 U/kg twice daily), or vehicle control. RCI treatment had a significant dose dependent effect on attenuating CIA-induced joint swelling and clinical scores at both doses when compared to vehicle with the greatest effect at the 400 U/kg dose."

Preclinical • Immunology

Real-World Insights on Satisfaction and Experience with Acthar Gel via SelfJect (RISE™): A Cross-Sectional Patient Survey. (PubMed, Adv Ther) - "Survey participants reported a favorable experience with Acthar Gel via SelfJect, with a high level of satisfaction, confidence, convenience, and ease of use. Participants reported perceptions of anticipated positive persistence and compliance with this device, which may correlate with the possibility of improved continuity of care."

Journal • Real-world evidence • Immunology • Inflammation • Inflammatory Arthritis • Ocular Inflammation • Rheumatoid Arthritis • Rheumatology

Mallinckrodt Presents Data on Real-World Outcomes with Acthar Gel (repository corticotropin injection) at the International Congress on Systemic Lupus Erythematosus (LUPUS 2025) (PRNewswire) - P=NA | N=NA | "Mallinckrodt plc...announced a late-breaking poster abstract on Acthar Gel (repository corticotropin injection) in patients with systemic lupus erythematosus (SLE) at the International Congress on Systemic Lupus Erythematosus (LUPUS 2025), taking place in Toronto, Canada from May 21-24, 2025....84% (47/56) of patients were female and 50% (28/56) were African-American; Common comorbidities included chronic joint disease (34%, 19/56), hyperlipidemia (34%, 19/56), hypertension (34%, 19/56), and arthritis/osteoarthritis (30%, 17/56)....Physicians reported that 89% (50/56) of patients experienced improved health status after Acthar Gel treatment; This included physician-reported improvements in overall symptoms (82%, 41/50), pain (54%, 27/50), fatigue (44%, 22/50), physical function (38%, 19/50), and strength (28%, 14/50).2 A reduction in corticosteroid use (44%, 22/50) was also reported."

Clinical data • Systemic Lupus Erythematosus

MNK: Use of Acthar Gel Single-Dose Pre-Filled SelfJectTM Injector in Patients With Moderate-Severe Keratitis and Autoimmune Disease (clinicaltrials.gov) - P4 | N=20 | Recruiting | Sponsor: Toyos Clinic

New P4 trial • Dry Eye Disease • Immunology • Keratitis • Ocular Inflammation • Ophthalmology • Pain

Microarray analysis of the effects of Acthar Gel versus methylprednisolone in a model of focal segmental glomerulosclerosis in female rats. (PubMed, Physiol Rep) - "Acthar® Gel (repository corticotropin injection) is an alternative treatment for patients with focal segmental glomerulosclerosis (FSGS) who cannot tolerate or do not adequately respond to glucocorticoids or calcineurin inhibitors...These results suggest that Acthar acts as an immunomodulator with a distinct mechanism of action from that of MP. Their differential alteration of gene expression in the kidney cortex suggests that Acthar may be more effective than MP in reducing inflammation and fibrosis in FSGS, which could slow disease progression."

Clinical • Journal • Preclinical • Fibrosis • Focal Segmental Glomerulosclerosis • Glomerulonephritis • Immunology • Inflammation • C1QB • CCR2 • TRB

Use of Acthar in Rheumatoid Arthritis (RA) Related Flares (clinicaltrials.gov) - P4 | N=40 | Recruiting | Sponsor: Veena Ranganath, MD, MS | Trial primary completion date: Dec 2024 ➔ Dec 2025

Trial primary completion date • Immunology • Inflammatory Arthritis • Rheumatoid Arthritis • Rheumatology

Long-term efficacy and tolerability of subcutaneous repository corticotropin injection in patients with ocular inflammatory diseases (ARVO 2025) - "Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

Clinical • Ocular Inflammation • Ophthalmology • Scleritis • Uveitis

Real-world 6-month outcomes of repository corticotropin injection in noninfectious posterior uveitis unresponsive to immunomodulatory therapy (ARVO 2025) - "Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

Clinical • Immunomodulating • Real-world • Real-world evidence • Ocular Inflammation • Ophthalmology • Uveitis

New insights into scleritis: comprehensive analysis of demographics, clinical features, treatment approaches, and complications in a Northern California tertiary referral center (ARVO 2025) - "Biologics (19.09%) included adalimumab (7.47%), rituximab (5.67%), and infliximab (2.58%). Other treatments included repository corticotropin injection (2.93%) and local steroids (e.g., dexamethasone or triamcinolone) (1.71%)...Eye specialists were pivotal in managing these cases, often prescribing complex treatments typically associated with rheumatology. Despite potential complications like cataracts and glaucoma, many patients experienced improved outcomes with modern therapies.This research underscores the importance of specialized care and advanced treatments in managing scleritis, highlighting promising strategies to reduce vision loss and improve quality of life for those with this challenging condition."

Clinical • Cataract • Glaucoma • Ocular Inflammation • Ophthalmology • Scleritis • Uveitis

An Open Label, Multicentered, Randomized Phase 2 Study to Evaluate Safety, Tolerability and Bioactivity of Subcutaneous Repository Corticotropin Injection in Patients With Scleritis: The ATLAS Study (ARVO 2025) - "Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

Clinical • P2 data • Ocular Inflammation • Ophthalmology • Scleritis

The Therapeutic Effects of Purified Cortrophin Gel on Experimental Autoimmune Uveitis (ARVO 2025) - "Purpose The pathways mediated by the melanocortins, a family of conserved receptors and ligands that includes adrenocorticotropic hormone (ACTH) and α-melanocyte stimulating hormone (α-MSH), are central in mediating ocular immune privilege in the normal eye...By studying how melanocortins and related drugs suppress inflammation, we can develop new, well-tolerated therapies. These therapies aim to restore anti-inflammatory activity in cases of uveitis (an autoimmune disease affecting the eye) and help prevent its recurrence."

Ocular Inflammation • Ophthalmology • Uveitis • IFNG • IL10 • IL17A • IL1B • TNFA

Mallinckrodt and Endo to Combine to Create a Global, Scaled, Diversified Pharmaceuticals Leader (PRNewswire) - "Mallinckrodt plc...and Endo, Inc...today announced that they have entered into a definitive agreement to combine in a stock and cash transaction to create a global, scaled, diversified pharmaceuticals leader...Mallinckrodt and Endo plan to combine their generic pharmaceuticals businesses and Endo's sterile injectables business after the close of the transaction and intend to separate that business from the combined company at a later date. Such a separation would be subject to approval by the combined company's Board of Directors and other conditions...The combined company's brands portfolio will comprise leading pharmaceutical brands across a range of therapeutic areas, including XIAFLEX (collagenase clostridium histolyticum), Acthar Gel (repository corticotropin injection), Terlivaz (terlipressin), SUPPRELIN LA (histrelin acetate) and AVEED (testosterone undecanoate)."

M&A • Ankylosing Spondylitis • Diabetic Nephropathy • Fibrosis • Immunology • Lupus • Multiple Sclerosis • Ocular Inflammation • Prostate Cancer • Psoriatic Arthritis • Renal Disease • Rheumatoid Arthritis • Sarcoidosis • Systemic Lupus Erythematosus • Uterine Leiomyoma

Mallinckrodt plc Reports Fourth Quarter and Fiscal Year 2024 Financial Results and Provides 2025 Guidance (PRNewswire) - "Acthar Gel net sales were $138.8 million in the fourth quarter, an increase of 33.0% versus the prior year period, and $485.7 million for the full year, an increase of 14.2%...Terlivaz net sales were $6.1 million in the fourth quarter, up from $5.6 million in the same period last year, and $24.7 million for the full year, up from $15.6 million...INOmax (nitric oxide) net sales were $60.8 million in the fourth quarter, down 14.0% compared to the prior year period, and full-year net sales were $261.4 million, down 13.8%, primarily due to competitive pressures in the U.S."

Sales • Ankylosing Spondylitis • Diabetic Nephropathy • Lupus • Multiple Sclerosis • Myositis • Ocular Inflammation • Psoriatic Arthritis • Pulmonary Arterial Hypertension • Renal Disease • Rheumatoid Arthritis • Sarcoidosis • Steven-Johnson Syndrome • Systemic Lupus Erythematosus

ACTHar in the Treatment of Lupus Nephritis (clinicaltrials.gov) - P4 | N=8 | Terminated | Sponsor: Columbia University | Completed ➔ Terminated; Lack of funding

Trial termination • Glomerulonephritis • Immunology • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology

A novel injection device to administer repository corticotropin injection for inflammatory disease treatment: findings from a market research study. (PubMed, J Comp Eff Res) - " All participants expressed positive perceptions for SelfJect including easy use, few preparation steps and potential reduced anxiety due to needle phobia. These attributes may help treatment adherence for patients and caregivers."

Journal • Inflammation • Mood Disorders • Psychiatry

Efficacy and tolerability of subcutaneous repository corticotropin injection in refractory ocular inflammatory diseases. (PubMed, J Ophthalmic Inflamm Infect) - "RCI may be considered as a potential therapy with acceptable tolerability for patients with non-infectious scleritis or uveitis."

Journal • Cardiovascular • CNS Disorders • Diabetes • Hypertension • Infectious Disease • Inflammation • Insomnia • Ocular Inflammation • Ophthalmology • Scleritis • Sleep Disorder • Uveitis • Vasculitis