As reported by drugs.com, at the American College of Rheumatology (ACR) Convergence congress, Novartis presented encouraging results from its Phase III NEPTUNUS-1 and NEPTUNUS-2 trials, highlighting the potential of ianalumab as a novel treatment for Sjögren’s disease—an autoimmune condition second only to rheumatoid arthritis in prevalence among rheumatic diseases.
A New Hope for a Complex Autoimmune Disorder
Sjögren’s disease is a chronic, systemic autoimmune disorder that affects millions worldwide. It is characterized by symptoms such as dry eyes and mouth, fatigue, joint pain, and in some cases, serious complications like lymphoma. Despite its impact, there are currently no approved targeted therapies for the condition.
Ianalumab, a fully human monoclonal antibody, is designed to address this unmet need. It works through a dual mechanism: depleting B cells and inhibiting their survival and activation by blocking the BAFF receptor. B-cell dysfunction is a key driver of the autoimmune response in Sjögren’s, making this approach particularly promising.
NEPTUNUS Trials: Efficacy and Safety Highlights
The NEPTUNUS-1 and NEPTUNUS-2 trials enrolled patients across 35 countries and over 200 sites, evaluating the efficacy and safety of ianalumab 300 mg administered monthly via subcutaneous injection. Both studies demonstrated that ianalumab led to a statistically significant improvement in disease activity, as measured by the EULAR Sjögren’s Syndrome Disease Activity Index (ESSDAI), by Week 48. Notably, improvements were already evident by Week 16 and sustained through the study duration.
Additional findings included:
- A greater proportion of patients achieving low disease activity.
- Improvements in physician and patient global assessments.
- Reductions in symptoms such as dryness, fatigue, and pain, as captured by patient-reported outcome tools.
- Enhanced salivary flow and reduced oral dryness in a subset of patients.
While some secondary endpoints did not reach statistical significance, the overall trend favored ianalumab, with consistent numerical improvements across multiple measures.
Safety Profile and Future Outlook
The safety data from both trials were reassuring, with adverse events and serious adverse events occurring at rates comparable to placebo. Patients completing the trials have the option to continue in long-term extension studies to further evaluate the durability of response and long-term safety.
Dr. Shreeram Aradhye, President of Development and Chief Medical Officer at Novartis, emphasized the significance of these findings, stating that ianalumab could represent a transformative option for patients with Sjögren’s disease.
Expanding the Pipeline
Beyond Sjögren’s, ianalumab is also under investigation for several other B-cell mediated autoimmune conditions, including systemic lupus erythematosus, lupus nephritis, immune thrombocytopenia, and systemic sclerosis.
As Novartis prepares to engage with regulatory authorities worldwide, the NEPTUNUS data mark a pivotal step toward potentially bringing the first targeted therapy to patients living with Sjögren’s disease.
