Uplizna (inebilizumab-cdon) / Mitsubishi Tanabe, Jiangsu Hansoh Pharma, Amgen - LARVOL DELTA

Real World Effectiveness and Tolerability of Novel Monoclonal Antibodies and Rituximab for NMOSD. (PubMed, Ann Clin Transl Neurol) - "In this multicenter retrospective cohort study of 135 people with aquaporin-4 IgG+ neuromyelitis optica spectrum disorder (NMOSD), some of whom were exposed to multiple therapies, we evaluated the effectiveness and tolerability of rituximab (n = 111) and novel monoclonal antibodies (nMAbs): eculizumab (n = 9), inebilizumab (n = 23), and satralizumab (n = 14). Among those with available MRI data, 10/73 rituximab and 1/14 inebilizumab developed new lesions. There were no serious adverse events. These findings support nMAbs as effective and well-tolerated first-line therapies for NMOSD."

Journal • Real-world evidence • CNS Disorders • Neuromyelitis Optica Spectrum Disorder • Rare Diseases

N-MOmentum LT: Long-term, Open-label, Safety Study of Inebilizumab in Neuromyelitis Optica Spectrum Disorder (NMOSD) (clinicaltrials.gov) - P4 | N=32 | Active, not recruiting | Sponsor: Amgen | Recruiting ➔ Active, not recruiting

Enrollment closed • CNS Disorders • Hematological Disorders • Neuromyelitis Optica Spectrum Disorder • Rare Diseases

Disseminated Gonococcal Infection Developing Two Days After Initial Eculizumab Administration in a Patient with Neuromyelitis Optica Spectrum Disorder: A Case Report and Literature Review. (PubMed, J Infect Chemother) - "Antimicrobial susceptibility testing revealed elevated minimum inhibitory concentrations (MICs) for penicillin G, ciprofloxacin, azithromycin, and minocycline, whereas the MIC for ceftriaxone remained low. Following the patient's refusal to continue eculizumab therapy, the patient has chosen inebilizumab, resulting in stable management of NMOSD symptoms...This is a case of DGI in a patient with NMOSD, and the earliest reported onset of DGI following eculizumab initiation. Clinicians should remain vigilant for invasive gonococcal infections, even shortly after initiating complement-inhibiting therapy."

Journal • CNS Disorders • Complement-mediated Rare Disorders • Hematological Disorders • Infectious Disease • Meningococcal Infections • Neuromyelitis Optica Spectrum Disorder • Pain • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • CRP

Effective Control of Neuropathic Pain With Amitriptyline in Neuromyelitis Optica Spectrum Disorder: A Case Report. (PubMed, Cureus) - "The patient was treated with methylprednisolone pulse therapy, followed by maintenance immunosuppressive therapy with inebilizumab and prednisolone, resulting in neurological improvement. This case highlights the potential of amitriptyline as an effective treatment for NP in NMOSD, a common and often resistant symptom in this condition. Given the complex mechanisms underlying NMOSD-related pain, individualized management strategies that include pharmacological agents like amitriptyline are crucial for improving patient quality of life (QOL), and further research is needed to optimize these approaches."

Journal • CNS Disorders • Musculoskeletal Pain • Neuralgia • Neuromyelitis Optica Spectrum Disorder • Pain • Rare Diseases

IgG4-Related Disease: Emerging Roles of Novel Genetic Variants, Immune Cell Subsets and Therapeutic Targets. (PubMed, Allergy) - "We also discuss the recent advances in understanding the genetic underpinnings of IgG4-RD, highlighting the significance of germline gene variants and implicated immune cell types. Finally, we explore future research directions, emphasising the need for deeper insights into pathogenesis, specific biomarkers and optimised treatment strategies."

Journal • Review • Fibrosis • Immunology • Inflammation

A Phase 2a Master Protocol Assessing Inebilizumab and Blinatumomab in Autoimmune Diseases (clinicaltrials.gov) - P2 | N=140 | Recruiting | Sponsor: Amgen | Trial completion date: Aug 2028 ➔ Apr 2028 | Trial primary completion date: Nov 2027 ➔ Jul 2027

Trial completion date • Trial primary completion date • Immunology • Inflammatory Arthritis • Lupus • Rheumatoid Arthritis • Rheumatology • Systemic Lupus Erythematosus

STARGlu-NMO: Slow vs. Rapid Glucocorticoids Tapering With Inebilizumab in NMOSD (clinicaltrials.gov) - P3 | N=170 | Not yet recruiting | Sponsor: Tianjin Medical University General Hospital

New P3 trial • CNS Disorders • Neuromyelitis Optica Spectrum Disorder • Rare Diseases

Abrupt Breathlessness: Pneumocystis jirovecii Pneumonia Associated with Inebilizumab in Neuromyelitis Optica Spectrum Disorder: A Case Report and Literature Review. (PubMed, Infect Drug Resist) - "Here, we present the first known NMOSD case who developed Pneumocystis jirovecii pneumonia following maintenance therapy with inebilizumab, showing a probable association (Naranjo score=6). We explored the underlying mechanisms linking B-cell depletion to Pneumocystis jirovecii, including antibody-dependent and immunomodulatory pathways. Furthermore, we provided an overview of Pneumocystis jirovecii pneumonia in autoimmune central nervous system disorders and summarized its clinical features, previous medication use, and prognostic factors. We advocate for risk-adapted prophylaxis to reduce morbidity and mortality associated with opportunistic infections in NMOSD."

Journal • CNS Disorders • Immunology • Infectious Disease • Neuromyelitis Optica Spectrum Disorder • Pneumonia • Rare Diseases • Respiratory Diseases

Immunotherapies in neuromyelitis optica: Bayesian network meta-analysis. (PubMed, J Neurol) - "These findings suggest that ravulizumab had the highest probability of being the most superior treatment in decreasing relapse risk in NMO-SD."

Clinical • Journal • Retrospective data • Review • CNS Disorders • Neuromyelitis Optica Spectrum Disorder • Rare Diseases

Clinical study on the treatment of neuromyelitis optica spectrum disorders with plasma exchange combined with inebilizumab (ChiCTR) - P=N/A | N=20 | Not yet recruiting | Sponsor: Punan hospital in Pudong new district, Shanghai; Punan hospital in Pudong new district, Shanghai

New trial • CNS Disorders • Neuromyelitis Optica Spectrum Disorder • Rare Diseases

AMGEN REPORTS SECOND QUARTER 2025 FINANCIAL RESULTS (Amgen Press Release) - "TEPEZZA (teprotumumab-trbw) sales increased 5% year-over-year to $505 million in the second quarter, primarily driven by higher inventory levels....UPLIZNA (inebilizumab-cdon) sales increased 91% year-over-year to $176 million in the second quarter, driven by 79% volume growth, with 16% derived from higher inventory levels. Year-over-year sales benefited from the timing of shipments to our ex-U.S. partner that occurred in the third quarter of 2024. Excluding these shipments, sales grew by 56% year-over-year in the second quarter."

Sales • Neuromyelitis Optica Spectrum Disorder • Ophthalmology

Comment on "Viscoelastic mechanics of living cells" by Hui Zhou, Ruye Liu, Yizhou Xu, Jierui Fan, Xinyue Liu, Longquan Chen, Qiang Wei. (PubMed, Phys Life Rev) - No abstract available

Journal

Anti-CD19 treatment: the new gold standard for IgG4-related diseases (PubMed, Z Rheumatol) - "Immunosuppressants, such as methotrexate, azathioprine, mycophenolate mofetil or rituximab (anti-CD20) are used but larger studies on proof of benefits are lacking so that no approved treatment options for the disease currently exist. The anti-CD19 targeted treatment with inebilizumab could therefore become the new approved gold standard; however, long-term data on remission maintenance and health economic issues, especially in comparison to off-label treatment such as rituximab remain the subject of further research. New approaches, such as CD19 chimeric antigen receptor (CAR) T cell treatment or bispecific T cell engagers could furthermore open up future treatment options."

Gold standard • Journal • Fibrosis • Infectious Disease • Inflammation

Xinyue capsule treatment on long-term outcomes in patients with stable coronary artery disease after PCI: A 10-year observational follow-up. (PubMed, Phytomedicine) - "Early Xinyue capsule intervention in addition to the conventional treatment was associated with a significant reduction in composite cardiovascular endpoint over the subsequent 10 years in patients with stable CAD post-PCI."

Journal • Acute Coronary Syndrome • Cardiovascular • Coronary Artery Disease • Myocardial Infarction

A Phase-2B Double-Blind Randomized International Prospective Trial of Inebilizumab in NMDAR Encephalitis: The ExTINGUISH Trial. (PubMed, Neurol Open Access) - "In addition, cyclophosphamide rescue therapy will be offered to participants with persistent moderate-to-severe disease 6 weeks after randomization. These innovations will inform development of future treatments and trials for patients with NMDAR encephalitis and other types of autoimmune encephalitis. The ExTINGUISH trial opened to enrollment of adult patients (older than 18 years) in 2022 and pediatric patients (older than 12 years) in 2024; enrollment is ongoing."

Clinical • IO biomarker • Journal • P2b data • Cardiovascular • CNS Disorders • Immunology • Pediatrics

ExTINGUSH: The ExTINGUISH Trial of Inebilizumab in NMDAR Encephalitis (clinicaltrials.gov) - P2 | N=116 | Recruiting | Sponsor: University of Utah | Trial completion date: Aug 2026 ➔ Sep 2028 | Trial primary completion date: Oct 2025 ➔ Sep 2028

Trial completion date • Trial primary completion date • CNS Disorders • Immunology

Alterations in peripheral blood immune cell profiles of neuromyelitis optica spectrum disorder across different phases and after B cell depletion therapy. (PubMed, Front Immunol) - "Forty-two of 76 patients with acute NMOSD were followed in remission, of whom 13, 15, and 11 patients received rituximab treatment, inebilizumab treatment, or conventional immunosuppressive therapies alone, respectively. Peripheral blood immune cell profiles of acute NMOSD patients showed widespread distinctions compared to those of remission NMOSD patients, acute MS patients, acute MOGAD patients, and controls, as well as after differential therapies. Our findings provide clues to comprehensively understand the abnormality of the dynamic and integrated immune network in NMOSD, distinguish NMOSD from MS and MOGAD, and search for more effective and safe therapeutic targets."

Journal • CNS Disorders • Inflammation • Multiple Sclerosis • Neuromyelitis Optica Spectrum Disorder • Rare Diseases • Solid Tumor • CD4 • CD8

Ravulizumab is Effective and Safe for Neuromyelitis Optica Spectrum Disorder Patients in Various Clinical Settings: A Single-Center Case Series with Concomitant Use of Rituximab. (PubMed, Cureus) - "Neuromyelitis optica spectrum disorder (NMOSD) is an autoimmune disease characterized by inflammation of the optic nerves and spinal cord, often associated with anti-aquaporin-4 antibodies. Further studies are warranted to evaluate whether the relapse rate in patients who switched to RTX using this method is comparable to that in patients who initiated RTX in combination with conventional steroids. Additionally, research is needed to establish safe switching strategies from RVZ to other biologic agents such as satralizumab or inebilizumab."

Journal • CNS Disorders • Immunology • Infectious Disease • Inflammation • Neuromyelitis Optica Spectrum Disorder • Novel Coronavirus Disease • Optic Neuritis • Pain • Rare Diseases

Neutropenia Associated With B Cell-Depleting Therapies in Multiple Sclerosis and Neuromyelitis Optica Spectrum Disorder. (PubMed, Neurol Neuroimmunol Neuroinflamm) - "This study is comprehensive and reflects a large cohort, examining incidence rates of neutropenia with BCDT in MS and NMOSD. Overall, neutropenia was still rare, occurring at a rate of 0.66 per 100 person-years, and infections were associated triggers in some patients. Yet, of relevance to clinicians, neutropenia was not benign: half required hospitalization and 35% experienced recurrence, which is higher than what was reported in clinical trials."

Journal • Retrospective data • CNS Disorders • Hematological Disorders • Infectious Disease • Inflammation • Multiple Sclerosis • Neuromyelitis Optica Spectrum Disorder • Neutropenia • Rare Diseases

A Phase 2a Master Protocol Assessing Inebilizumab and Blinatumomab in Autoimmune Diseases (clinicaltrials.gov) - P2 | N=140 | Recruiting | Sponsor: Amgen | Trial completion date: Mar 2027 ➔ Aug 2028 | Trial primary completion date: Jul 2026 ➔ Nov 2027

Trial completion date • Trial primary completion date • Immunology • Inflammatory Arthritis • Lupus • Rheumatoid Arthritis • Rheumatology • Systemic Lupus Erythematosus

BEST-NMOSD: Rituximab Versus Ravulizumab, Inebilizumab, Satralizumab, and Eculizumab in NMOSD (clinicaltrials.gov) - P4 | N=160 | Not yet recruiting | Sponsor: Massachusetts General Hospital

New P4 trial • CNS Disorders • Neuromyelitis Optica Spectrum Disorder • Rare Diseases

Emerging Role of Targeted Monoclonal Antibodies in Neuromyelitis Optica Spectrum Disorders. (PubMed, BioDrugs) - "Recently, four monoclonal antibodies (mAbs) were granted Food and Drug Administration approval for aquaporin-4 antibody (AQP4-IgG)-seropositive NMOSD treatment: eculizumab, inebilizumab, satralizumab, and ravulizumab. We explore the role of these preventative mAbs for NMOSD management by reviewing the efficacy, safety, mechanisms of action, and administration of these agents, and compare them to rituximab and traditional immunosuppressants. We discuss therapy selection and the clinical challenges of therapeutic management, including medication adherence, therapeutic monitoring strategies, economic considerations, and medication accessibility, while managing therapy failure and indications for transitioning to alternative therapies."

Journal • Review • CNS Disorders • Immunology • Neuromyelitis Optica Spectrum Disorder • Rare Diseases

Phase 2B ExTINGUISH Trial Aims to Assess Efficacy of Inebilizumab for NMDAR Encephalitis (NeurologyLive) - "In the study, participants (target enrollment of 116) from across the United States and Europe will receive first-line immunotherapies before randomization to either inebilizumab or placebo. Outcomes will be measured at 6, 16, 24, 32, and 96 weeks, with comprehensive neurophysological tests and cognitive screening tools being used at the final 96-week time point...The trial is enrolling participants in that age group and plans to continue accepting patients who have experienced new onset of NMDAR encephalitis within the past 3 months."

Clinical protocol • Immunology

Observational Safety Study in Women With Neuromyelitis Optica Spectrum Disorder (NMOSD) Exposed to UPLIZNA® During Pregnancy (clinicaltrials.gov) - P=N/A | N=60 | Recruiting | Sponsor: Amgen | Trial completion date: Oct 2032 ➔ Jul 2033

Trial completion date • CNS Disorders • Neuromyelitis Optica Spectrum Disorder • Rare Diseases

Safety and Efficacy of Inebilizumab in IgG4-Related Disease Across Various Participant Demographics: Subgroup Analysis from the MITIGATE Study (EULAR 2025) - P3 | "*Flare-free, treatment-free complete remission is defined as the absence of evident disease activity at week 52; defined as an IgG4-RD Responder Index score of 0 or determination by the investigator that no disease activity is present on the basis of physical, laboratory, pathology, or other evidence), no AC-determined flare during the RCP of the trial, and no treatment for flare or disease control beyond the protocol required prednisone taper. The MITIGATE trial demonstrated that participants across various demographic subgroups derived similar clinical benefits with INEB treatment, making INEB a potentially effective and broadly applicable treatment choice for patients with IgG4-RD."

Clinical • Inflammation