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

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

Activation of Podocyte-Specific MC5R Signaling by Melanocortin Therapy Protects against THSD7A-Associated Membranous Nephropathy (KIDNEY WEEK 2024) - "Background: Melanocortins, exemplified by adrenocorticotropic hormone (ACTH), have demonstrated a unique beneficial effect in membranous nephropathy (MN)... Our findings suggest that podocyte-specific MC5R signaling protects against glomerular injury and proteinuria in MN, and may serve as a novel therapeutic target for treating MN."

Glomerulonephritis • Renal Disease • PODXL

Successful Management of Membranoproliferative Glomerulonephritis with Repository Corticotropin Injection (KIDNEY WEEK 2024) - "Before the presentation she was treated with prednisone 20 mg daily for many years and any attempt to taper prednisone resulted in MPGN flares...Despite induction therapy with cyclophosphamide and rituximab her UPCR remained at 8.5 g/g after three months...Repository corticotropin injection, which stimulates endogenous steroid production, offered lower steroid exposure and fewer side effects. This may be a good option to avoid systemic side effects of steroids in other hard to treat cases."

CNS Disorders • Depression • Gastrointestinal Disorder • Glomerulonephritis • Lupus Nephritis • Nephrology • Psychiatry • Suicidal Ideation

THSD7A-Associated Membranous Nephropathy Involves Both Complement-Mediated and Autonomous Podocyte Injury (KIDNEY WEEK 2024) - "Potential pathomechanisms and therapeutic efficacy of repository corticotropin injection (RCI; Purified Cortrophin® Gel, ANI Pharmaceuticals, Inc.) were tested in this model. Primary mouse podocytes were cultured in complete medium supplemented with fetal bovine serum containing complements or preheated to inactivate heat-labile complements, followed by exposure to a commercial rabbit anti-THSD7A antibody... Both complement-dependent and autonomous podocytopathy are involved in the mouse model of THSD7A-associated MN, which could be attenuated by RCI. This model, based on the use of a commercial anti-THSD7A antibody, could be an important tool for MN research."

Glomerulonephritis • Renal Disease • THSD7A

Clinical assessment of the potential use of a novel single-dose prefilled injection device for the administration of Acthar Gel in children: a narrative review. (PubMed, J Comp Eff Res) - "Acthar® Gel (repository corticotropin injection; Mallinckrodt Pharmaceuticals, NJ, USA) is indicated for the treatment of myriad inflammatory disorders and is currently administered manually via a vial and syringe...The risk of needlestick injury from SelfJect is mitigated by a needle guard. In summary, this review of injection device considerations demonstrates that SelfJect is appropriate for use in the pediatric population."

Journal • Review • CNS Disorders • Inflammation • Insomnia • Pain • Pediatrics • Sleep Disorder

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 (AAO 2024) - "Four in Group 1 (57.2%) and three in Group 2 (50%) showed improvement in NRS at Week 16, and change in best corrected visual acuity was +3.9 and +6 letters, respectively. Conclusion Subcutaneous RCI is tolerated by patients and can be effective in eyes with active anterior scleritis."

Clinical • P2 data • Ocular Inflammation • Ophthalmology • Scleritis

Formative and validation human factors studies of a new disposable prefilled injection device for subcutaneous delivery of acthar gel (repository corticotropin injection). (PubMed, Expert Opin Drug Deliv) - "SelfJect was able to be used in a safe and effective manner by the intended users. Iterative HF studies informed the mitigation of use-related risks to reduce the occurrence of use deviations during simulated use."

Journal

THSD7A-associated membranous nephropathy involves both complement-mediated and autonomous podocyte injury. (PubMed, Front Pharmacol) - "Furthermore, as the first FDA-approved treatment for primary MN, adrenocorticotropic hormone therapy with repository corticotropin injection (Purified Cortrophin Gel®) appeared to be beneficial and significantly attenuated proteinuria and glomerular injury, suggesting that this model may be useful for developing novel treatments or understanding the pathogenesis of MN. Collectively, our model, based on the use of a commercially available anti-THSD7A antibody, will be an important tool for MN research."

Journal • Glomerulonephritis • Nephrology • Renal Disease • THSD7A

ACTHar in the Treatment of Lupus Nephritis (clinicaltrials.gov) - P4 | N=8 | Completed | Sponsor: Columbia University | Recruiting ➔ Completed | N=20 ➔ 8 | Trial completion date: Jul 2024 ➔ Jan 2024

Enrollment change • Trial completion • Trial completion date • Glomerulonephritis • Immunology • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology