1. Ianalumab helps extend ITP disease control by 45% compared to placebo. 2. 62% achieved sustained platelet response versus 39% with placebo. 3. Data will be submitted for regulatory review in 2027. 4. Novel treatment reduces chronic treatment needs for ITP. 5. Ianalumab exhibits a promising safety profile in patients.
FAQ
Why Bullish?
Positive Phase III trial results typically drive investor confidence. Historical strength from similar announcements confirms potential share price increases.
How important is it?
The article highlights significant efficacy of ianalumab, a key product for Novartis, enhancing strategic market positioning.
Why Long Term?
Approval processes and market entry are usually protracted. Historical patterns, such as with other drug launches, confirm long timelines.
Novartis Reports Encouraging Results for Ianalumab in Immune Thrombocytopenia
On December 9, 2025, Novartis announced promising findings from the Phase III VAYHIT2 trial, which evaluated the efficacy of ianalumab (9 mg/kg) combined with eltrombopag in patients suffering from primary immune thrombocytopenia (ITP) who had previously been treated with corticosteroids. The results indicate that this new treatment regimen can significantly prolong disease control, suggesting a potential shift in therapeutic strategies for managing ITP.
Key Findings from the VAYHIT2 Trial
- 45% increase in disease control duration for patients treated with ianalumab plus eltrombopag compared to those receiving placebo.
- Median time to treatment failure was 2.8 times longer for the ianalumab group (13.0 months) than for placebo (4.7 months).
- 62% of patients treated with ianalumab achieved a sustained platelet response at six months, compared to 39% in the placebo group.
- A notable 7.7 point reduction in fatigue symptoms was reported with ianalumab versus 3.6 points for placebo.
Implications for Treatment and Patient Quality of Life
The VAYHIT2 trial results indicate that ianalumab has the potential to not only offer effective disease control but also to alleviate the chronic treatment burden that many ITP patients face. Dr. Hanny Al-Samkari of Mass General Brigham commented, “Treatments for ITP have historically focused on raising platelet counts, often requiring chronic therapy to control ITP... The VAYHIT2 trial results are encouraging, as they demonstrated improved disease control even while patients spend time off treatment.”
Trial Background and Future Steps
The VAYHIT2 trial involved a randomized, double-blind study assessing the efficacy and safety of two doses of ianalumab added to eltrombopag for adults with treatment-resistant ITP. Novartis plans to submit data from this trial, along with results from the ongoing first-line ITP trial, VAYHIT1, to health authorities in 2027, highlighting its commitment to advancing treatment options for ITP patients.
Safety Profile and Tolerability of Ianalumab
Ianalumab was well-tolerated throughout the trial, with an adverse event profile similar to prior studies. The most frequent adverse events included headaches and infusion-related reactions, with neutropenia occurring more commonly in the ianalumab treatment groups compared to placebo. Importantly, no serious adverse events led to treatment discontinuation.
About Ianalumab and Primary Immune Thrombocytopenia
Ianalumab, originally developed through a collaboration with MorphoSys AG (acquired by Novartis), is a fully human monoclonal antibody aimed at treating autoimmune diseases like ITP. ITP itself is a rare disorder characterized by low platelet counts, which can lead to significant health complications such as prolonged bleeding and fatigue.