Novartis antibody ianalumab helps prolong control of rare blood disorder ITP
The news refers to ianalumab, a B‑cell depleting monoclonal antibody from Novartis being studied for primary immune thrombocytopenia (ITP), a rare autoimmune blood disorder in which the immune system destroys platelets.136
In the Phase 3 VAYHIT2 trial, adding ianalumab to the standard ITP therapy eltrombopag (Promacta) significantly prolonged time to treatment failure, effectively extending how long patients’ disease remained under control compared with eltrombopag plus placebo.156
Patients receiving ianalumab plus eltrombopag had platelet counts maintained at safe levels for a median 13 months, versus 4.7 months for eltrombopag plus placebo, corresponding to about a 45% extension of ITP disease control at the higher ianalumab dose.1
A lower ianalumab dose (3 mg/kg) also achieved a statistically significant 42% improvement in time to treatment failure compared with control, although the exact median time could not yet be calculated because many patients on the antibody had not failed treatment by the time of analysis.1
The trial enrolled adults with recently diagnosed ITP (about 3–4 months from diagnosis) who had not responded adequately to first‑line corticosteroids, aiming to intervene earlier than most prior ITP studies that focus on long‑standing, treatment‑refractory disease.1
Patients on ianalumab reported greater improvements in fatigue, a frequent and burdensome symptom of ITP, supporting clinical benefit beyond platelet counts alone.1
Safety findings showed slightly more infusion reactions with ianalumab, generally mild to moderate, and no increase in infections versus placebo, despite the drug’s mechanism of B‑cell depletion.1
Investigators and Novartis suggest that by resetting B‑cells early, ianalumab plus eltrombopag may help prevent progression to more severe, chronic ITP, potentially changing the treatment paradigm if ongoing trials confirm long‑term benefit.16
Novartis plans to submit ianalumab for FDA approval in ITP in 2027, using data from VAYHIT2 and an ongoing first‑line trial, VAYHIT1.15
Ianalumab, originally developed with MorphoSys and later acquired by Novartis, is also in late‑stage or ongoing studies for other B‑cell–driven autoimmune diseases, including Sjögren’s syndrome, systemic lupus erythematosus, and lupus nephritis.13
Sources:
1. https://www.fiercebiotech.com/biotech/ash-novartis-details-ianalumabs-phase-3-win-rare-blood-disease
3. https://pharmatimes.com/news/novartis-blood-disorder-drug-shows-promise-in-late-stage-trial/
5. https://www.novartis.com/news/media-releases/novartis-ianalumab-phase-iii-trial-meets-primary-endpoint-itp-demonstrating-statistically-significant-improvement-time-treatment-failure