Principia Biopharma Inc., a Sanofi company focused on developing treatments for immune-mediated diseases, announced the early completion of patient enrollment for the global Phase 3 PEGASUS trial of rilzabrutinib. This oral investigational Bruton tyrosine kinase (BTK) inhibitor is being tested for its efficacy and safety in treating pemphigus, a group of potentially life-threatening disorders characterized by blisters in mucous membranes and skin.
About the PEGASUS Trial
The PEGASUS trial is a global, randomized, double-blind, placebo-controlled, pivotal Phase 3 clinical trial that has enrolled approximately 120 participants. These participants, who have moderate to severe pemphigus and are either newly diagnosed or relapsing with chronic disease, represent about three quarters of the pemphigus patient population. The trial evaluates rilzabrutinib versus placebo, with a background treatment of tapering doses of corticosteroids (CS). The primary efficacy endpoint is the ability of rilzabrutinib to achieve durable complete remission (CR) at 37 weeks of treatment, defined as all lesions healing on very low dose CS (5mg/day) and no new lesions appearing for at least eight weeks. Key secondary endpoints include cumulative CS use and time to CR. After 37 weeks, all patients have the option to continue treatment with active rilzabrutinib therapy in an open-label extension period of 24 weeks.
About Rilzabrutinib
Rilzabrutinib is an oral, reversible covalent BTK inhibitor under investigation for treating immune-mediated diseases. It blocks inflammatory immune cells, eliminates autoantibody destructive signaling, and prevents new autoantibody production without depleting B cells. Its safety and efficacy have not yet been reviewed by any regulatory authority.
About Pemphigus
Pemphigus is a group of disorders characterized by blisters in mucous membranes and skin due to an immune response that attacks the 'glue' holding these cells together. The condition includes several types, with pemphigus vulgaris being the most common and severe form. The disease significantly impacts patients' quality of life, highlighting the need for effective treatments.
Principia Biopharma, through its proprietary Tailored Covalency® platform, is dedicated to developing transformative therapies for patients with significant unmet medical needs in immune-mediated diseases. Rilzabrutinib is one of three drug candidates currently under clinical development by the company.
