For Novartis, this week is all about ianalumab. A day after posting a pair of phase 3 wins for the tricky indication of Sjögren's syndrome, the Swiss pharma has unveiled a separate late-stage victory in another B-cell-driven autoimmune disease.
The latest clinical success comes from a phase 3 study of two different doses of ianalumab, a dual-mechanism, B-cell-depleting antibody that targets the protein BAFF-R, in patients with a low platelet count disorder called primary immune thrombocytopenia (ITP). Participants also received Novartis' thrombocytopenia drug Promacta as part of their treatment and had found that corticosteroids hadn’t resolved their condition.
While Novartis didn’t dig into the data, the company confirmed that treatment with ianalumab plus eltrombopag “significantly prolonged” the time to treatment failure (TTF) when compared to patients who received Promacta alone, hitting the trial’s primary endpoint. TTF measures how long patients maintain safe platelet levels during and after treatment.
When it came to a key secondary endpoint, the ianalumab-eltrombopag treatment also showed a “significantly higher” rate of sustained improvements in platelet count at six months, Novartis explained in an Aug. 12 release.
Ianalumab is administered as four once-monthly doses to treat ITP. Novartis is touting the therapy as potentially offering “long-term disease control through a short course of treatment” that could “allow patients extended time off treatment.”
This would help it stand out from the current crop of ITP therapies, which often require lifelong treatment.
“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,” Novartis Chief Medical Officer Shreeram Aradhye, M.D., said in the release. “These positive topline results from the phase 3 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.”
Ianalumab's story begins with Novartis’ collaboration with MorphoSys, before the Big Pharma went on to buy the biotech last year. The pharma is currently eyeing 2027 for an approval application for ianalumab in ITP, for which it hopes to combine data from this morning’s study with an ongoing trial of the drug as a first-line treatment for the disorder.
In the meantime, the company is expecting phase 3 readouts for ianalumab in other B-cell-driven autoimmune diseases next year, such as warm autoimmune hemolytic anemia.
This morning’s late-stage win in ITP comes just a day after Novartis reported a pair of phase 3 successes in Sjögren's syndrome, an autoimmune disorder that has defeated some of the company’s peers. Novartis hailed the triumph yesterday as the “first ever global phase 3 trials to demonstrate statistically significant reduction in disease activity for Sjögren's disease.”
Ianalumab hasn’t always been so lucky in the clinic. Only last month, the company reported that the B-cell-depleting antibody had failed to hit the efficacy threshold in a phase 2 study for a painful skin condition called hidradenitis suppurativa.