Dedee F Murrell, Frédéric Caux, Aikaterini Patsatsi, Owen Hagino, Lidia Rudnicka, Snejina Vassileva, Soner Uzun, Jenny Ye, Karl Yen, Puneet Arora, Steven G Gourlay, Pascal Joly, Victoria P Werth
TRIAL DESIGN: Pemphigus is a rare, but life-threatening autoimmune disease requiring long-term treatment that minimizes corticosteroid (CS) exposure while providing consistent disease control. The phase 2 pemphigus study of oral, reversible, covalent Bruton tyrosine kinase (BTK) inhibitor rilzabrutinib demonstrated rapid and sustained efficacy with well-tolerated safety. METHODS: Adults (18-80 y) were randomized 1:1 to rilzabrutinib 400 mg (n=65) or placebo (n=66) twice daily (with CS ≤0...
March 15, 2024: Journal of Investigative Dermatology