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Novartis Drug Combination Nearly Triples Disease Control Time in Rare Blood Disorder Trial

MarketDash Editorial Team
11 hours ago
Novartis' Phase 3 trial shows ianalumab combined with eltrombopag extends disease control by 45% in immune thrombocytopenia patients, with median time to treatment failure reaching 13 months versus 4.7 months for placebo.

Novartis AG (NVS) delivered some genuinely promising news Tuesday for patients battling primary immune thrombocytopenia, a rare blood disorder where your immune system essentially attacks your platelets. The company's VAYHIT2 Phase 3 trial showed that combining its investigational drug ianalumab with an existing therapy called eltrombopag significantly extends how long patients can keep their disease under control.

The headline number is striking: patients receiving ianalumab (at the 9 mg/kg dose) plus eltrombopag maintained disease control 2.8 times longer than those getting placebo plus eltrombopag. We're talking 13 months versus 4.7 months before treatment failure, which represents a 45% extension in disease control based on the trial's primary endpoint.

Here's why that matters. Time to treatment failure measures how long patients can maintain safe platelet levels during and after treatment. For people with immune thrombocytopenia, who previously needed corticosteroid treatment, keeping platelets at healthy levels without constant intervention is a big deal.

The detailed results were presented at the American Society of Hematology Annual Meeting and simultaneously published in The New England Journal of Medicine. Novartis had already shared topline results from VAYHIT2 back in August, but now we're getting the full picture.

The trial also hit its key secondary endpoint. Patients on the ianalumab combination achieved sustained platelet count improvement at six months at significantly higher rates than the control group: 62% versus 39%. That's not a marginal difference.

There were other encouraging signals too. Fatigue improvement showed a mean reduction of 7.7 points with the drug combination versus 3.6 points with placebo. And looking at the 12-month mark, the estimated probability of being free from treatment failure was 54% in the 9mg group, 51% in the 3mg group, and just 30% in the placebo group.

Novartis isn't stopping here. The company is investigating ianalumab in other B-cell-driven autoimmune diseases, including ongoing Phase 3 trials for first-line ITP treatment and for second and later lines of warm autoimmune hemolytic anemia. Readouts from those studies are expected in 2026.

Novartis stock closed up 1.46% at $132.07 on Tuesday following the announcement.

Back to News

Novartis Drug Combination Nearly Triples Disease Control Time in Rare Blood Disorder Trial

MarketDash Editorial Team
11 hours ago
Novartis' Phase 3 trial shows ianalumab combined with eltrombopag extends disease control by 45% in immune thrombocytopenia patients, with median time to treatment failure reaching 13 months versus 4.7 months for placebo.

Novartis AG (NVS) delivered some genuinely promising news Tuesday for patients battling primary immune thrombocytopenia, a rare blood disorder where your immune system essentially attacks your platelets. The company's VAYHIT2 Phase 3 trial showed that combining its investigational drug ianalumab with an existing therapy called eltrombopag significantly extends how long patients can keep their disease under control.

The headline number is striking: patients receiving ianalumab (at the 9 mg/kg dose) plus eltrombopag maintained disease control 2.8 times longer than those getting placebo plus eltrombopag. We're talking 13 months versus 4.7 months before treatment failure, which represents a 45% extension in disease control based on the trial's primary endpoint.

Here's why that matters. Time to treatment failure measures how long patients can maintain safe platelet levels during and after treatment. For people with immune thrombocytopenia, who previously needed corticosteroid treatment, keeping platelets at healthy levels without constant intervention is a big deal.

The detailed results were presented at the American Society of Hematology Annual Meeting and simultaneously published in The New England Journal of Medicine. Novartis had already shared topline results from VAYHIT2 back in August, but now we're getting the full picture.

The trial also hit its key secondary endpoint. Patients on the ianalumab combination achieved sustained platelet count improvement at six months at significantly higher rates than the control group: 62% versus 39%. That's not a marginal difference.

There were other encouraging signals too. Fatigue improvement showed a mean reduction of 7.7 points with the drug combination versus 3.6 points with placebo. And looking at the 12-month mark, the estimated probability of being free from treatment failure was 54% in the 9mg group, 51% in the 3mg group, and just 30% in the placebo group.

Novartis isn't stopping here. The company is investigating ianalumab in other B-cell-driven autoimmune diseases, including ongoing Phase 3 trials for first-line ITP treatment and for second and later lines of warm autoimmune hemolytic anemia. Readouts from those studies are expected in 2026.

Novartis stock closed up 1.46% at $132.07 on Tuesday following the announcement.