Novartis today presented data from the RemIND trial at the European Academy of Allergy and Clinical Immunology (EAACI) Congress demonstrating that Rhapsido (remibrutinib) met its primary endpoints across the three most common chronic inducible urticaria (CIndU) subtypes. The data positions Rhapsido as a potential first targeted therapy for a condition affecting an estimated 29 million people globally, where no approved targeted treatments currently exist. The trial results showed statistically significant and clinically meaningful symptom control in twice as many patients compared to placebo, with a favorable safety profile and no observed liver safety concerns.

The RemIND trial is a global Phase III, multicenter, randomized, double-blind, placebo-controlled, parallel-group study evaluating the efficacy, safety, and tolerability of Rhapsido in adults with CIndU inadequately controlled by H1-antihistamines. The primary endpoint was the proportion of complete responders at Week 12, assessed through provocation tests specific to three CIndU subtypes: symptomatic dermographism (SD), cold urticaria, and cholinergic urticaria. Higher rates of complete responses were observed at week 12, with responses seen as early as week 2 in two subtypes.

Primary endpoint results at Week 12

Rhapsido is a highly selective, oral BTK inhibitor that blocks the BTK pathway involved in the release of histamine, a key driver of hives and swelling. It is currently approved in the U.S., European Union, China, and several other countries for the treatment of chronic spontaneous urticaria (CSU) in adult patients with inadequate response to H1-antihistamines. Novartis has submitted a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) seeking approval of Rhapsido for the treatment of the SD subtype and will continue additional filings to health authorities globally throughout 2026.

"Rhapsido significantly improves symptom control for patients living with the three most common subtypes of chronic inducible urticaria, and it has the potential to become the first approved targeted therapy," said Angelika Jahreis, Global Head, Immunology Development, Novartis. "The CIndU data presented today are consistent with Rhapsido's proven efficacy and favorable safety profile in chronic spontaneous urticaria and demonstrate Novartis' commitment to developing truly meaningful innovation for patients with complex immune-mediated diseases."

Novartis announced that the biomarker cohort of the FORTITUDE Phase I/II study of del-brax met its primary and key secondary endpoints, demonstrating reductions in KHDC1L and creatine kinase biomarker levels. These reductions indicate strong target engagement and a reduction in muscle damage in patients with facioscapulohumeral muscular dystrophy (FSHD). The therapy shows potential to become the first disease-modifying treatment for FSHD, a rare, irreversible neuromuscular disease affecting approximately 45,000 to 87,000 people in the US and EU.

The FORTITUDE Phase I/II study is a randomized, double-blind, placebo-controlled clinical trial evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of del-brax. The trial includes three dose cohorts. Cohorts A and B assessed del-brax 2 mg/kg or 4 mg/kg versus placebo, while Cohort C, the biomarker cohort, evaluated del-brax 2 mg/kg every 6 weeks versus placebo for 12 months in 51 FSHD patients aged 16-70. The primary endpoint was the change in plasma concentration of KHDC1L, a DUX4-regulated circulating biomarker, and the key secondary endpoint was the change in creatine kinase levels.

Clinical Development Status

Based on the results from the initial cohorts, the del-brax 2 mg/kg dose every 6 weeks was selected for Cohort C and the ongoing Phase III study. Novartis is currently enrolling patients for FORTITUDE-3, a randomized, double-blind, placebo-controlled Phase III study evaluating the efficacy and safety of del-brax in 200 patients with FSHD aged 16-70 years. The primary endpoint for the Phase III study is quantitative muscle testing (QMT) in the US and the 10-meter walk/run test (10MWRT) in Europe.

Del-brax is an investigational antibody oligonucleotide conjugate (AOC) designed to address the root cause of FSHD by suppressing the aberrant expression of DUX4. It combines the tissue specificity of monoclonal antibodies with the precision of oligonucleotides to deliver siRNA to muscle cells. The therapy has received FDA Orphan Drug and Fast Track designations, as well as EMA Orphan Drug designation.

Del-brax is one of three potential first-in-class, late-stage, disease-modifying AOC therapies added to the Novartis neuroscience pipeline through the acquisition of Avidity Biosciences completed in February 2026. The other therapies include delpacibart-etedesiran (del-desiran) in Phase III development for myotonic dystrophy type 1 (DM1) and delpacibart-zotadirsen (del-zota) in Phase II development for Duchenne muscular dystrophy (DMD).