Novartis scores phase III wins for two ianalumab indications

Novartis building in Stein, Switzerland [Credit: Taljat David shutterstock.com]

Novartis’ ianalumab has met its primary endpoints in two sets of late-stage clinical trials, including for the hard-to-treat chronic autoimmune disorder Sjögren’s disease.

The results are a substantial mark of confidence in the treatment, which Novartis added to its portfolio through its €2.7 billion acquisition of Morphosys in 2024.

A novel fully human monoclonal antibody, ianalumab has a dual mechanism of action that combines B-cell depletion and BAFF-R inhibition.

In Sjögren’s disease it showed a significant reduction in disease activity, as measured by a reduction in EULAR Sjögren’s syndrome disease activity index (ESSDAI), in the NEPTUNUS-1 and NEPTUNUS-2 studies, making them the first phase III trials to reach that milestone.

The progressive, systemic autoimmune condition Sjögren’s disease is often unrecognised or misdiagnosed and currently has very limited options, with Novartis hoping to be able to offer the first targeted treatment.

The pair of studies saw ianalumab well tolerated, with a favourable safety profile, and Novartis plans to present the data at an upcoming medical congress. The results will also be used in the product’s regulatory submissions, including in the US where it has Fast Track Designation from the Food and Drug Administration (FDA).

In addition to Sjögren’s disease, the candidate is also being investigated as a treatment for a number of B cell-driven autoimmune diseases, including immune thrombocytopenia (ITP), systemic lupus erythematosus and lupus nephritis.

Novartis’ ianalumab in immune thrombocytopenia (ITP)

Novartis also released phase III results for ianalumab in ITP, an indication for which is has been granted orphan drug status by US and European regulators.

The rare autoimmune condition causes a low platelet count, making sufferers susceptible to bruising, bleeding and chronic fatigue.

The top-line trial results saw ianalumab, given as four once-monthly doses, meet its primary endpoint by demonstrating a statistically significant improvement in time to treatment failure, by prolonging the duration of safe platelet levels during, and after, treatment in patients with primary ITP who had previously received corticosteroids.

The treatment also met its secondary endpoint, with patients experiencing a significantly higher rate of sustained improvements in platelet count.

Shreeram Aradhye, MD, President, Development and Chief Medical Officer at Novartis, said: “For many people living with ITP, chronic treatment can disrupt their daily life due to the burden of regular dosing, dose adjustments and side effects.

“These positive top-line results from the phase III study highlight the potential of ianalumab, if approved, to deliver long-term disease control with four once-monthly doses and enable extended time off treatment.”

The trial data will be presented at an upcoming medical meeting and be included, alongside results from the ongoing first-line ITP trial VAYHIT1, in regulatory submissions that are expected to be made in 2027.