top of page

American Thoracic Society (ATS) International Conference | 2024 | News | Updates | iPharmaCenter

Promising Phase 2 Results for Rilzabrutinib as a Potential Oral Treatment for Moderate-to-Severe Asthma

New findings from a phase 2 study of rilzabrutinib suggest it could become the first advanced oral therapy for moderate-to-severe asthma. The latest data, presented at the American Thoracic Society (ATS) International Conference, indicated that rilzabrutinib reduced the frequency of asthma control loss events, improved symptoms, and was well-tolerated with no new safety concerns identified. These results pave the way for further development and a phase 3 clinical trial.

Rilzabrutinib is a significant candidate in Sanofi's expansive immunology portfolio, which aims to deliver next-generation treatments for immune diseases. The drug is currently being tested for multiple conditions, including immune thrombocytopenia (ITP), where it has shown positive phase 3 results, and chronic spontaneous urticaria (CSU), also demonstrating promising outcomes in phase 2 trials.

In the phase 2 study, rilzabrutinib was evaluated for its efficacy in adults with uncontrolled moderate-to-severe asthma. Participants received either a high or low dose of the oral treatment. The study showed a numerical reduction in loss of asthma control (LOAC) events, the primary endpoint, and improvements in asthma symptoms. These findings were presented as a late-breaking poster at the ATS conference and will support the phase 3 program, which will investigate a twice-daily dose of rilzabrutinib for moderate-to-severe asthma.

Asthma remains a prevalent chronic respiratory condition affecting millions globally. Despite current treatments, approximately 50% of asthma patients experience poor control over their symptoms, significantly affecting their quality of life. In this proof-of-concept study, rilzabrutinib at high and low doses resulted in a 36% and 25% relative risk reduction in LOAC events, respectively, at 12 weeks. Additionally, significant and meaningful symptom improvements were observed, with LS mean differences of -0.54 and -0.59 in the asthma control questionnaire (ACQ-5) scores, noticeable as early as two weeks into treatment.

The study found that rilzabrutinib was well-tolerated over the 12-week period, with no reports of cytopenia, hemorrhagic events, or atrial fibrillation, and no significant liver function test imbalances. Common adverse events included diarrhea, reported in 10.9% and 9.4% of patients on high and low doses of rilzabrutinib, compared to 0% and 3.1% on placebo, respectively.

This phase 2 trial was a randomized, double-blind, placebo-controlled, parallel-group study lasting 12 weeks. It assessed the efficacy, safety, and tolerability of rilzabrutinib in patients with moderate-to-severe asthma uncontrolled by inhaled corticosteroid (ICS) and long-acting β2 adrenergic agonist (LABA) therapy. The participants were randomized to receive either rilzabrutinib (800 mg or 1200 mg daily) or placebo, in addition to their ICS/LABA therapy, which was withdrawn during the treatment period.

The primary endpoint was the reduction in LOAC events, while secondary endpoints included asthma control (measured by ACQ-5), quality of life (measured by the asthma quality of life questionnaire, AQLQ), and lung function (measured by FEV1).

Rilzabrutinib, an oral reversible covalent BTK inhibitor, shows potential as a first- or best-in-class treatment for various immune-mediated diseases. By targeting BTK, which is expressed in B cells and mast cells, rilzabrutinib plays a crucial role in many immune processes. Utilizing Sanofi’s TAILORED COVALENCY technology, rilzabrutinib can selectively inhibit BTK while minimizing off-target effects.

Currently, rilzabrutinib is being explored for multiple immune-mediated conditions, including ITP, asthma, chronic spontaneous urticaria, prurigo nodularis, IgG4-related disease, and warm autoimmune hemolytic anemia.


Recent Posts

See All


bottom of page