- Design
- Two phase 3, 52-week, double-blind, placebo-controlled randomised trials
- Population
- 779 patients with active Sjögren's disease (275 and 504)
- Primary outcome
- Change in ESSDAI at week 48
- Effect
- −1.3 (−2.6 to 0.0) and −1.0 (−2.0 to 0.0) vs placebo; no difference in symptom scores
NEPTUNUS-1 (275 patients) and NEPTUNUS-2 (504 patients) were 52-week, double-blind, placebo-controlled trials of ianalumab, a monoclonal antibody that both depletes B cells and blocks the BAFF receptor, in active Sjögren's disease.
Monthly 300 mg ianalumab reduced the ESSDAI disease activity score at week 48 more than placebo, by 1.3 points (95% CI −2.6 to 0.0) in NEPTUNUS-1 and 1.0 point (−2.0 to 0.0) in NEPTUNUS-2 — both just reaching significance. ESSDAI response rates and patient-reported symptom scores, including dryness and fatigue measures, did not differ. Serious adverse events were similar; injection reactions were more common with the drug.
On a scale running to 123, a 1-point difference is small, and patients did not feel better. Sjögren's disease has no approved targeted therapy, and this result is not yet a reason to change practice.
- Continue symptomatic and organ-directed treatment for Sjögren's disease as now.
- Explain to patients asking about ianalumab that trial benefit was small and did not improve dryness or fatigue scores.
- Score systemic activity with ESSDAI to identify patients who might be candidates for future targeted therapy.
- Watch for regulatory decisions before considering access.
Why it matters
A targeted drug met its phase 3 endpoint in a disease with none approved, yet patients did not feel better.
Don't overread it
The primary endpoint was met by a small margin with intervals touching zero, and key secondary endpoints were not.
The statistics, in plain English
A difference of about 1 point on a 0–123 scale, with an interval ending at 0, means the true effect could be close to nothing. Statistical significance here does not mean a difference patients would notice.
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