Novartis AG (NYSE:NVS) released topline results on Tuesday from the VAYHIT2 Phase 3 trial evaluating ianalumab plus eltrombopag in patients with primary immune thrombocytopenia (ITP) previously treated with corticosteroids.
Primary ITP is a rare, autoimmune disorder in which the immune system mistakenly targets and destroys platelets, the cells essential for blood clotting, leading to symptoms such as prolonged bleeding, easy bruising, and chronic fatigue.
Ianalumab plus eltrombopag, compared to placebo plus eltrombopag, significantly prolonged the time to treatment failure (TTF), the primary endpoint that assesses how long patients maintain safe platelet levels during and after treatment.
Ianalumab is being investigated in other B-cell-driven autoimmune diseases, including ongoing Phase 3 trials in first-line ITP and second and later lines of warm autoimmune hemolytic anemia, with readouts expected in 2026.
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In VAYHIT2, patients treated with ianalumab plus eltrombopag experienced a significantly higher rate of sustained improvements in platelet count at six months, the key secondary endpoint of the study.
The safety profile of ianalumab was consistent with what was previously observed in clinical studies, with no new safety signals.
"While current treatments for ITP are generally effective in raising platelet counts, many patients require lifelong treatment to maintain safe levels, which can create a lasting treatment burden," said Adam Cuker, Professor of Medicine and Chief, Section of Hematology, University of Pennsylvania. "The results from VAYHIT2 are encouraging, as they suggest that ianalumab may support longer periods of disease control and reduce the need for continuous treatment."
Data will be presented at an upcoming medical meeting and included in future regulatory submissions in 2027, along with results from the ongoing first-line ITP trial, VAYHIT1.
Ianalumab received the U.S. Food and Drug Administration and European Medicines Agency Orphan Drug Designations.
Novartis on Monday shared topline data from two Phase 3 trials, NEPTUNUS-1 and NEPTUNUS-2, evaluating ianalumab (VAY736) in adults with active Sjögren's disease.
Both trials met the primary endpoint of demonstrating statistically significant improvements in disease activity.
These results support the potential for ianalumab, a drug with a dual mechanism of action —B-cell depletion and BAFF-R inhibition —to become the first targeted treatment for patients with Sjögren's disease.
In June, Novartis released results from APPULSE-PNH, a Phase 3B study evaluating the efficacy and safety of twice-daily oral monotherapy with Fabhalta (iptacopan) in adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who had Hb levels ≥10g/dL and had switched from anti-C5 therapies (eculizumab or ravulizumab).
After 24 weeks of treatment with Fabhalta, the Hb level improved on average by 2.01 g/dL, with most patients achieving normal or near-normal levels.
Price Action: NVS stock is up 0.67% at $119.34 during the premarket session at the last check on Tuesday.
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