Novartis Reports Positive Phase III Results for Ianalumab in Immune Thrombocytopenia (ITP)

Novartis Reports Positive Phase III Results for Ianalumab in Immune Thrombocytopenia (ITP)

By, Admin 14 August 2025

A potential step toward reducing long-term treatment burden for ITP patients.
Novartis has announced positive top-line results from VAYHIT2, a Phase III clinical trial evaluating ianalumab plus eltrombopag in patients with primary immune thrombocytopenia (ITP) who had previously been treated with corticosteroids.

The combination therapy significantly prolonged time to treatment failure (TTF) compared to placebo plus eltrombopag — meaning patients were able to maintain safe platelet counts for longer.

Key Findings from VAYHIT2

  • Primary endpoint: Ianalumab + eltrombopag significantly extended time to treatment failure versus placebo + eltrombopag.
  • Key secondary endpoint: Higher proportion of patients achieved sustained platelet count improvements at 6 months.
  • Safety profile: Consistent with previous clinical studies of ianalumab; no new safety signals detected.

Expert Perspectives

“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.”

  • Adam Cuker, MD, University of Pennsylvania

“If approved, ianalumab could deliver long-term disease control with just four once-monthly doses — enabling extended time off treatment.”

  • Shreeram Aradhye, President, Development and CMO, Novartis

Why It Matters for ITP Patients

ITP is a rare autoimmune disorder where the immune system destroys platelets, leading to:

  • Increased bleeding risk
  • Easy bruising
  • Chronic fatigue

Many patients require lifelong therapy to maintain platelet counts, often facing regular dosing, side effects, and dose adjustments. A therapy that extends remission could reduce treatment burden and improve quality of life.

Next Steps

  • Full data will be presented at an upcoming medical meeting.
  • Results will support future regulatory submissions in 2027, alongside outcomes from the first-line ITP trial (VAYHIT1).
  • Ianalumab has Orphan Drug Designation from both the USFDA and EMA.

About Ianalumab

  • Type: Fully human monoclonal antibody
  • Targets: B cells through dual mechanism:
    - B cell depletion via ADCC
    - BAFF-R signal interruption to reduce B cell function and survival
  • Development scope: Being studied for Sjögren’s disease, ITP, SLE, lupus nephritis, wAIHA, and systemic sclerosis.
  • Origin: Developed in collaboration with MorphoSys AG, later acquired by Novartis in 2024.

Bottom line:

With VAYHIT2 showing significant efficacy and a consistent safety profile, ianalumab could become a game-changer in ITP treatment, offering longer disease control and fewer treatment days.

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