Press Release

Phase 2 results demonstrate rilzabrutinib rapidly reduced itch severity and significantly improved disease activity in adults with chronic spontaneous urticaria - Seite 2

Key Results 
In this dose-ranging study, different doses of rilzabrutinib were evaluated: 400 mg once every evening (QPM), 400 mg twice a day (BID), 400 mg three times a day (TID).

In the intent-to-treat (ITT) population which included either patients who were previously naïve or incomplete responders to omalizumab, Rilzabrutinib 400 mg TID demonstrated:

• Significant reduction from baseline in weekly itch severity score (ISS7) at Week 12, a key symptom of the disease, [least squares mean (LSM) -9.58 vs -6.31, respectively; p=0.0181]. Significant changes in ISS7 were seen as early as Week 1.
• Significant reduction from baseline to week 12 in weekly urticaria activity score (UAS7) [LSM -17.95 vs -11.20, respectively; p=0.0116].
• Significant reduction from baseline to week 12 in weekly hives severity score (HSS7) [LSM -8.31 vs -4.89; p<0.0100].
  
  

Rilzabrutinib was generally well-tolerated with no events of cytopenia, bleeding or atrial fibrillation seen with other BTK inhibitor. Treatment-emergent AEs occurring at a higher frequency with rilzabrutinib vs placebo were diarrhea (29.3% TID and BID, 7.9% QPM, 15% placebo), nausea (19.5% TID, 17.1% BID, 13.2% QPM, 5.0% placebo), headache (9.8% TID, 14.6% BID, 5.3% QPM, 0.0% placebo) and abdominal pain (0.0% TID, 12.2% BID, 2.6% QPM, 5.0% placebo).

Rilzabrutinib is currently under clinical investigation, and its safety and efficacy have not been evaluated by any regulatory authority.

About CSU
CSU is an inflammatory skin condition driven mainly by the activation of cutaneous mast cells, which causes itchy recurrent hives, swelling, or both. CSU is typically treated with H1 antihistamines and biologics; however, the disease remains uncontrolled in up to 50% of patients, who are left with limited alternative treatment options. These individuals continue to experience debilitating symptoms that can significantly impact quality of life.

About the RILECSU study
RILECSU is a 52-week Phase 2 study, comprising a 12-week randomized, double-blind, placebo-controlled, dose-ranging, efficacy and safety period, followed by a 40-week open-label extension period.

RILECSU is evaluating rilzabrutinib in adult patients with moderate-to-severe CSU who remain symptomatic despite use of H1 antihistamine treatment and are either naïve to or incomplete responders to omalizumab. The primary endpoint was change from baseline in weekly itch severity score ISS7 at 12 weeks. Secondary endpoints include change from baseline in weekly UAS7 at 12 weeks and change from baseline weekly HSS7 at 12 weeks.