Swiss drugmaker Novartis said its oral multiple sclerosis treatment Rhapsido (ingredient name remibrutinib), now in development, met the primary endpoints in a phase 3 clinical trial.
The company said it not only confirmed a greater effect in reducing relapses than existing treatments, but also saw no liver toxicity signals that had posed problems during development of drugs in the same class. On the news, Novartis shares on the New York stock market finished the day at $161.25, up 6% from the previous day. Intraday, they rose as high as $163.41.
Novartis said on the 1st (local time) that two phase 3 trials (REMODEL-1 and REMODEL-2) of the oral BTK inhibitor Rhapsido (ingredient name remibrutinib) for relapsing multiple sclerosis met the primary endpoints.
Multiple sclerosis is an autoimmune disease in which the immune system attacks the myelin that surrounds nerves in the central nervous system, including the brain and spinal cord. It can cause visual disturbances, reduced sensation and muscle weakness, and relapses may recur or disability may progress. The core of treatment is to reduce relapses and slow disability progression.
About 2,000 patients with relapsing multiple sclerosis took part in the two trials. Patients were randomly assigned 1-to-1 to Rhapsido or Sanofi's existing treatment Aubagio (ingredient name teriflunomide).
In both trials, Rhapsido significantly lowered the annualized relapse rate compared with Aubagio. Inflammatory brain lesions confirmed by MRI also improved. The company said clinically meaningful improvements were seen in key secondary endpoints related to disability progression.
Liver safety is particularly noteworthy. BTK inhibitors block signal transmission in immune cells to suppress excessive immune responses. They drew attention as they were developed as treatments for multiple sclerosis, but some candidates showed severe liver injury.
Sanofi's BTK inhibitor tolebrutinib showed severe drug-induced liver injury in clinical trials, and the U.S. Food and Drug Administration (FDA) rejected approval for the nonrelapsing secondary progressive multiple sclerosis indication. Cases of liver injury were also reported with Roche's fenebrutinib.
By contrast, Novartis said there were no cases of liver injury meeting Hy's Law criteria in the Rhapsido trials. Hy's Law is a criterion used to assess the risk of severe drug-induced liver injury.
However, this announcement is the topline result of phase 3. How competitive it will be compared with existing treatments needs to be confirmed through data to be released later. Detailed clinical data analyses are scheduled to be presented at the international multiple sclerosis conference MS Toronto 2026, to be held in Toronto, Canada, in Oct.
Rhapsido won FDA approval last Sept. as a treatment for chronic spontaneous urticaria. Novartis is seeking to expand indications beyond multiple sclerosis to food allergies and hidradenitis suppurativa, among others. Based on these results, the company plans to file for approval of Rhapsido as a treatment for relapsing multiple sclerosis.
In today's multiple sclerosis treatment market, B cell–targeting therapies such as Roche's Ocrevus and Novartis' Kesimpta have become mainstays. Novartis' strategy is to add the oral Rhapsido to broaden treatment options and strengthen its market position.