Enspryng (satralizumab) / Roche - LARVOL DELTA

SHIELD DMD: A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - P2 | N=50 | Recruiting | Sponsor: Hoffmann-La Roche | Trial completion date: Jun 2027 ➔ Nov 2027 | Trial primary completion date: Jun 2026 ➔ Nov 2026

Trial completion date • Trial primary completion date • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Pediatrics

Subjective sleep quality evaluation in patients with neuromyelitis optica spectrum disorders (PubMed, Zh Nevrol Psikhiatr Im S S Korsakova) - "In a cohort of patients with NMOSD, a high incidence of sleep disorders, anxiety and depression has been established. These disorders significantly affect the quality of life of patients and require constant assessment and correction."

Journal • CNS Disorders • Depression • Gene Therapies • Insomnia • Mood Disorders • Musculoskeletal Pain • Neuromyelitis Optica Spectrum Disorder • Ocular Inflammation • Ophthalmology • Optic Neuritis • Pain • Psychiatry • Rare Diseases • Sleep Disorder

Case report: Satralizumab as an adjunctive therapy for AQP-4 antibody and MOG antibody dual-negative optic neuritis in a third-trimester pregnancy case. (PubMed, Front Med (Lausanne)) - "No systemic or ocular side effects related to satralizumab therapy were observed in the patient or her fetus during the 9-month follow-up. Our findings in this case suggest that satralizumab may be a safe and efficient adjunctive therapy for pregnant patients with DON who poorly respond to IVMP and IVIG therapy, even in cases of dual-negative AQP-4 Ab and MOG-Ab."

Journal • Ocular Inflammation • Ophthalmology • Optic Neuritis • Pain • Solid Tumor

Evaluating effectiveness and safety of Satralizumab in Real-World Chinese patients with Neuromyelitis Optica Spectrum Disorder (NMOSD): An observational, multicenter, cohort study (ChiCTR) - P=N/A | N=300 | Recruiting | Sponsor: Beijing Tiantan Hospital, Capital Medical University; Beijing Tiantan Hospital, Capital Medical University | Not yet recruiting ➔ Recruiting

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

An evaluation of ravulizumab for the treatment of neuromyelitis optica spectrum disorder. (PubMed, Expert Opin Biol Ther) - "We predict ravulizumab will eventually replace eculizumab, but may not have a similar impact on inebelizumab or satralizumab. Patients taking C5 complement inhibitors have an increased risk of serious meningococcal infections, such as invasive meningococcal disease (IMD), and have incomplete protection against IMD despite immunization. Thus, we recommend that in addition to standard pre-immunizations against Neisseria meningitidis, patients should also be assessed for starting on appropriate antibiotic prophylaxis against IMD, based on local resistance patterns."

Journal • Review • CNS Disorders • Infectious Disease • Meningococcal Infections • Neuromyelitis Optica Spectrum Disorder • Rare Diseases

Usage status of biologics for the chronic treatment of optic neuritis in neuromyelitis optica spectrum disorders in Japan. (PubMed, Jpn J Ophthalmol) - "Satralizumab was the most commonly used biologic for AQP4-Ab positive optic neuritis in Japan. This study demonstrates the efficacy and safety of satralizumab in preventing the relapse of optic neuritis."

Journal • CNS Disorders • Infectious Disease • Neuromyelitis Optica Spectrum Disorder • Ocular Inflammation • Ophthalmology • Optic Neuritis • Rare Diseases

A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Satralizumab in Participants With Thyroid Eye Disease (clinicaltrials.gov) - P3 | N=120 | Recruiting | Sponsor: Hoffmann-La Roche | Trial primary completion date: Mar 2025 ➔ Sep 2025

Trial primary completion date • Ophthalmology • Thyroid Eye Disease

SHIELD DMD: An Open-label Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (clinicaltrials.gov) - P2 | N=50 | Recruiting | Sponsor: Hoffmann-La Roche | Not yet recruiting ➔ Recruiting

Enrollment open • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Pediatrics

Immunogenicity dynamics and covariate effects after satralizumab administration predicted with a hidden Markov model. (PubMed, CPT Pharmacometrics Syst Pharmacol) - P3 | "Satralizumab exposure and body mass index impacted transition probabilities and, therefore, the likelihood of developing ADAs. In conclusion, the mHMM model was able to describe the time course of ADA development and identify patterns of ADA development in NMOSD patients following treatment with satralizumab, which may allow for the formulation of strategies to reduce the emergence or limit the impact of ADA in the clinical setting."

Journal • CNS Disorders • Neuromyelitis Optica Spectrum Disorder • Rare Diseases • IL6

A Study To Evaluate Efficacy, Safety, Pharmacokinetics, And Pharmacodynamics Of Satralizumab In Patients With Generalized Myasthenia Gravis (clinicaltrials.gov) - P3 | N=188 | Completed | Sponsor: Hoffmann-La Roche | Active, not recruiting ➔ Completed

Trial completion • CNS Disorders • Myasthenia Gravis

Real World-Evidence: Efficacy data of Rituximab in patients with Neuromyelitis Optica Spectrum AQP4-IgG serum-positive patients (ECTRIMS 2024) - "Introduction: Neuromyelitis optica (NMO) is a group of severe inflammatory demyelinating conditions that produce recurrent episodes of transverse myelitis optic neuritis, and other clinical characteristics according to Wingerchuk criteria. RTX had better efficacy in this real-world data study than clinical trials of Satralizumab, both SakuraStar and SakuraSky. We had a better free relapse rate of 74.68% vs 71-73%, with greater follow-up after administrating the treatment at 1621 days vs 768 days. RTX remains a cost-effective treatment in the Mexican population."

Clinical • HEOR • Real-world • Real-world evidence • CNS Disorders • Neuromyelitis Optica Spectrum Disorder • Ocular Inflammation • Ophthalmology • Optic Neuritis • Rare Diseases

IL-6 Blockade in NMOSD: Unveiling B Cell Dynamics and Regulatory Plasmablast Induction (ECTRIMS 2024) - "Introduction: IL-6 signaling blockade therapies, including satralizumab, are increasingly used in the prevention of relapses in aquaporin 4 (AQP4) antibody-seropositive neuromyelitis optica spectrum disorder (NMOSD)... The treatment of IL-6 signaling blockade elicits a regulatory phenotype in B cells and PBs. CD200+ PBs might be a marker for treatment responsiveness in the context of NMOSD pathophysiology."

IO biomarker • CNS Disorders • Neuromyelitis Optica Spectrum Disorder • CD200 • CD27 • IL10 • IL6

Management practices and outcomes in the evolving landscape of neuromyelitis optic spectrum disorder treatment: real-world data from a multicenter academic cohort in the United States (ECTRIMS 2024) - "At most recent visit, inebilizumab was most used (32.6%), followed by rituximab (27.9%) and eculizumab (25.6%); satralizumab was not widely adopted (8.9%). There were no patients on ravulizumab... Use of FDA-approved DMT in this multicenter academic cohort was influenced by perceived drug efficacy and therapy-specific features and associated with race/ethnicity. Future research in our larger multicenter cohort will focus on how race, income and other sociodemographic factors may impact access to care and disease outcomes in the U.S."

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

Anti-IL-6 receptor antibody attenuates myelitis severity of AQP4 peptide immunized mice (ECTRIMS 2024) - "Satralizumab, a novel humanized anti-IL-6 receptor (IL-6R) monoclonal recycling antibody, is used as a therapeutic for suppressing the relapse...Anti-IL-6R antibody (MR16-1) or vehicle was intraperitoneally administered on day 9... These results may suggest that IL-6 signaling promotes microglial proliferation in the spinal cord, possibly promotes severity of the spinal cord damage. Further analysis, RNA sequencing of the spinal cord, is now carrying out in order to dig the mechanism in detail."

Preclinical • CNS Disorders • Immunology • Infectious Disease • Inflammation • Neuromyelitis Optica Spectrum Disorder • Rare Diseases • Respiratory Diseases

Comparative Effectiveness and Safety of Disease-modifying Treatments (DMTs) in a Real-World Neuromyelitis Optica Spectrum Disorder Cohort (NMOSD) (ECTRIMS 2024) - "Introduction: In addition to the 4 FDA-approved treatments for AQP4-positive NMOSD (satralizumab, eculizumab, ravulizumab, and inebilizumab), rituximab (RTX), mycophenolate mofetil (MMF) and azathioprine (AZA) are commonly used to treat NMOSD. A significant proportion of relapses occurred on rituximab despite suppressed B-cells and on MMF despite lymphopenia. Most non-relapse hospitalizations occurred on rituximab, while very few occurred on the new therapies. While the number of relapses and non-relapse hospitalizations on rituximab can be partly explained by the longer treatment duration and number of patients treated, the treatment failure rate and toxicity of rituximab and mycophenolate are higher in this real-world study than previously reported."

Clinical • HEOR • Real-world • Real-world evidence • CNS Disorders • Infectious Disease • Neuromyelitis Optica Spectrum Disorder • Rare Diseases • CD20

Real-world Safety Data of Rituximab among Mexican Patients with Neuromyelitis Optica AQP4-IgG serum positive from 2007 to 2024 (ECTRIMS 2024) - "Accepted DMTs, such as satralizumab, have reported acceptable tolerance in clinical trials such as SakuraSky and SakuraStar. RTX had adequate tolerance in our patients. The patients that had severe ADEs were hospitalized due to UTI septicemia. Nevertheless, it is important to acknowledge that even though according to the CTCAE is classified as severe, all of them had sphincter dysfunctions, which might also lead to complicated UTIs and septicemia."

Clinical • Real-world • Real-world evidence • CNS Disorders • Infectious Disease • Inflammation • Nephrology • Neuromyelitis Optica Spectrum Disorder • Ocular Inflammation • Ophthalmology • Optic Neuritis • Rare Diseases

Clinical Practice Expert Guidance for the use of Inebilizumab in NMOSD (ECTRIMS 2024) - "In China, Inebilizumab, Satralizumab, and Eculizumab have been approved for NMOSD treatment, with Inebilizumab being the most widely used monoclonal antibody. Based on evidence and clinical practice, expert guidance for inebilizumab use in NMOSD were established through an improved Delphi method. These guidelines will be valuable for standardizing clinical medication practices and optimizing patient outcomes."

Clinical • CNS Disorders • Immunology • Neuromyelitis Optica Spectrum Disorder

Effectiveness and Safety of Satralizumab in a Real-world Clinical Setting in Japan: Interleukin-6 Receptor Inhibition in Neuromyelitis Optica Spectrum Disorders: A Six-month Interim Analysis of Multicenter Medical Chart Review (ECTRIMS 2024) - "Secondary endpoints included changes in oral glucocorticoid (GC) and immunosuppressant (azathioprine [AZA], tacrolimus [TAC]) doses among relapse-free patients. The relapse-free rate at 26 weeks after satralizumab treatment in patients with AQP4[+] NMOSD in a real-world clinical setting was 96.6%, and the relapse symptoms were not severe. These results support the relapse-preventive effect of satralizumab shown in the Phase 3 study."

Clinical • Real-world • Real-world evidence • Review • CNS Disorders • Neuromyelitis Optica Spectrum Disorder • Ocular Inflammation • Ophthalmology • Optic Neuritis • Rare Diseases • IL6R

Long-term effectiveness of ravulizumab compared with other approved interventions in neuromyelitis optica spectrum disorder: a modelling study using network meta-analysis data (ECTRIMS 2024) - "Using this model, proportions of patients remaining relapse free at 15 years were greater with ravu (81%) and eculizumab (ecu) (81%) than inebilizumab (ineb) (9%), satralizumab (satra) (7%), or placebo (pbo) (0%) monotherapy. The modelled long-term comparative effectiveness led to a difference in clinical benefit as measured by relapse-free years. The difference was driven by greater relapse risk reduction observed in clinical trials. When modelled over 15 years, ravu and ecu monotherapy offered considerably greater numbers of relapse-free years and, to a lesser degree, more QALYs than other approved treatment options."

Retrospective data • CNS Disorders • Neuromyelitis Optica Spectrum Disorder • Rare Diseases

Experience with satralizumab in neuromyelitis optica spectrum patients in southeast Spain (ECTRIMS 2024) - "Satralizumab was used in monotherapy as the first drug in a pediatric patient; with the other patients was used as add-on to mycophenolate mofetil for inefficacy or after switch from tocilizumab due to safety reasons. The effectiveness and safety profile of Satralizumab in our patients appears to be similar to overall population in clinical trials. Long-term data in clinical practice have not yet been determined."

Clinical • CNS Disorders • Infectious Disease • Neuromyelitis Optica Spectrum Disorder • Pediatrics • Rare Diseases • IL6

Real-world clinico-laboratory and therapeutic characteristics of aquaporin-4 IgG seropositive Neuromyelitis Optica Spectrum Disorder patients: the first multicenter study in Greece. (ECTRIMS 2024) - "During an attack, all patients received high-dose intravenous methylprednisolone and 28%(n=25/88) underwent plasmapheresis too...At data collection, patients were treated with azathioprine/mycophenolate mofetil(n=23), cyclophosphamide(n=1), eculizumab(n=1), ravulizumab(n=5), rituximab(n=37), satralizumab(n=10), and no treatment(n=11); 40% of patients received ⩾2 preventive treatments... This is the first multicenter study in Greece providing real-world data of a large cohort of seropositive NMOSD patients, concluding also certain risk factors for unfavorable outcomes on disability. Rituximab demonstrated superiority over the other off-label immunotherapies. Additionally, the recently approved drugs for NMOSD showed high efficacy with zero relapses."

Clinical • Real-world • Real-world evidence • CNS Disorders • Immunology • Neuromyelitis Optica Spectrum Disorder • Ocular Inflammation • Ophthalmology • Optic Neuritis • Rare Diseases

Organizing Pneumonia related to anti-CD20 monoclonal antibodies in patients with inflammatory CNS diseases: a Case Series. (ECTRIMS 2024) - "The first patient was shifted to teriflunomide, the second to ozanimod...Azathioprine was maintained as alternative to Ocrelizumab...Since satralizumab was not available, Rituximab was rechallenged without relapse of the pulmonary symptoms. Anti-CD20 mAbs are highly effective therapies for MS and NMOSD. Development of pneumonia not responsive to antibiotics, in patients treated with anti-CD20, must warrant consideration of drug induced pneumotoxicity such as OP."

Clinical • CNS Disorders • Cough • Hematological Disorders • Infectious Disease • Inflammation • Interstitial Lung Disease • Multiple Sclerosis • Neuromyelitis Optica Spectrum Disorder • Novel Coronavirus Disease • Pneumonia • Rare Diseases • Respiratory Diseases • CD8

Long term safety of Satralizumab and Japanese PMS data (ECTRIMS 2024) - "supported by Chugai Pharmaceutical Co., Ltd."

Clinical • Neuromyelitis Optica Spectrum Disorder

Long term efficacy of Satralizumab (ECTRIMS 2024) - "supported by Chugai Pharmaceutical Co., Ltd."

Clinical • Neuromyelitis Optica Spectrum Disorder

Real-world efficacy and Tolerability of Novel Monoclonal Antibodies and Rituximab for Neuromyelitis Optica Spectrum Disorder (ECTRIMS 2024) - "Introduction: There is limited real world data available for novel monoclonal antibodies (nMAb); eculizumab (ECU), inebilizumab (INEB) and satralizumab (SAT) use in AQP4-IgG+ neuromyelitis optica spectrum disorder (NMOSD). This real-world data shows that nMAb reduce the risk of relapse in AQP4-IgG+ NMOSD and are well tolerated. Longer real-world follow up is required for comparison between nMAb and RTX."

Clinical • Late-breaking abstract • Real-world • Real-world effectiveness • Real-world evidence • CNS Disorders • Gastrointestinal Disorder • Hematological Disorders • Infectious Disease • Leukopenia • Neuromyelitis Optica Spectrum Disorder • Pain • Rare Diseases • Respiratory Diseases