Things are going very well for Novartis when it comes to its drug Ianalumab, whereby it reported that the ongoing phase 3 VAYHIT2 trial, which was targeting patients with primary immune thrombocytopenia who were previously given corticosteroids for their disease, had met the primary endpoint. The scope of this is that this drug is really starting to shine in many B-cell-driven autoimmune diseases. Just the other day, the company released positive results from a pair of two phase 3 trials using this drug to treat patients with Sjögren’s disease (program under NEPTUNUS-1 and NEPTUNUS-2 trials). It was noted that the company was in the process of evaluating the use of this drug against immune thrombocytopenia, systemic lupus erythematosus (SLE), and other B cell-mediated autoimmune disorders.
This latest win for this big pharma in the late-stage VAYHIT2 trial reinforces the dual mode of action of ianalumab, which is to provide the depletion of B-cells that drive disease via deploying antibody-dependent cellular cytotoxicity (ADCC) and then take away cancer cell survival by blocking the BAFF-R pathway. With immune thrombocytopenia, the immune system attacks a person’s platelets in their body, and this is bad because they are required for proper blood clotting. Furthermore, it is important to note that there are two types of ITP. There is primary ITP, which consists of disease occurring with no known main cause, and then secondary ITP, which stems from a specific cause like a drug or another autoimmune disorder like SLE.
The differentiation is quite important, because the top-line results it just released from the phase 3 VAYHIT2 study were those of patients with primary ITP. This randomized, double-blind study tested out two doses of ianalumab versus placebo and then evaluated these patients based on a primary and secondary endpoint. The primary endpoint of time to treatment failure (TTF) was met in a statistically significant manner. This significance was not just achieved with ianalumab alone; instead, this BAFF-R pathway inhibitor was added to eltrombopag to target these primary ITP patients. Eltrombopag itself has a different mode of action entirely in that it is responsible for causing the bone marrow to produce more platelets.
Ianalumab plus eltrombopag was able to achieve statistical significance of the primary endpoint TTF versus placebo plus eltrombopag. This specific late-stage study had another important target, and that is that this specifically targeted 2nd-line primary ITP patients. What this means is that the patients who were actually brought into the study had to have been previously treated with corticosteroids. This is important to note, because Novartis is already in the process of evaluating frontline (1st-line) ITP patients in another phase 3 trial, VAYHIT1, using ianalumab. This specific trial itself, along with later lines of warm autoimmune hemolytic anemia study data, is expected to be released in 2026.
These other milestones further demonstrate ianalumab’s versatility to be applied to multiple B cell-mediated autoimmune disorders, beyond what has been reported by Novartis thus far. Other catalysts to consider going forward would be the company presenting this and other ianalumab data at an upcoming medical meeting, along with regulatory submissions across the globe in targeting these primary ITP patients. The company scoring regulatory approval of this drug doesn’t only serve to allow it to generate revenues from it. It could possibly change the scope or way these patients are treated. They just have to deal with continuous treatment, which in and of itself is annoying to begin with.
Beyond this, relapse generates a problem where they don’t remain on therapy, and chronic dosing could also cause potential side effects. This is where ianalumab can come into play to help these ITP patients, mainly because it is offered as four once-monthly doses. The positive of this is that it would offer a period of time whereby a patient wouldn’t need to be treated immediately. The bottom line of this is that this drug could offer long-term disease control and have patients avoid the need for continuous treatment with currently available therapies.
While ianalumab had its share of good fortune with these disorders, believe it or not, the drug didn’t perform up to par to treat patients with hidradenitis suppurativa (HS). This autoinflammatory condition attacks the hair follicles, leading to the formation of painful lumps in sweaty parts of a person’s body. It was noted by the company in a Q2 of 2025 earnings release that despite the drug performing well versus placebo, its target criteria was not met for further advancement of it. This is not a huge problem, as the company has now shown this monoclonal antibody to do well in targeting multiple B cell-mediated disorders. The hope now is that when the company does release treatment data in 1st-line primary ITP and warm autoimmune hemolytic anemia in 2026, it further enhances its win streak with this drug. Along with reinforcing the dual mechanism of action of it in showing its true potential as a BAFF-R inhibitor.