Evaluating rilzabrutinib in the treatment of immune thrombocytopenia

There is an unmet need for newer treatment options in immune thrombocytopenia (ITP) that address its underlying complex immune dysregulation, induce durable platelet responses, are well tolerated, and improve fatigue and overall quality of life. Rilzabrutinib, an oral, reversible covalent Bruton tyrosine kinase (BTK) inhibitor, is effective through its multi-immune modulation mechanisms by inhibiting B-cell activation, possibly decreasing autoantibody production, preventing FcγR-mediated phagocytosis in the spleen and liver, and reducing chronic inflammation. Preclinical studies of rilzabrutinib in immune-mediated disease settings demonstrated high selectivity, full reversibility, and durable BTK occupancy. Oral rilzabrutinib 400 mg BID demonstrated rapid, durable platelet count increases in patients with persistent/chronic ITP in the phase 2 LUNA2 study and significantly improved durable platelet response vs placebo in the pivotal phase 3 LUNA3 trial. Additionally, rilzabrutinib improved multiple disease aspects including fatigue and bleeding with a well-tolerated safety profile. Through multi-immune modulation, rilzabrutinib achieves rapid and durable platelet response, improves fatigue, and decreases bleeding in ITP patients. It is well tolerated with an acceptable safety profile. Efficacy may be increased if administered earlier in the course of disease. Longer-term studies, and investigations in pediatric patients and other immune-mediated diseases are underway. Immune thrombocytopenia (ITP) is a disease where people have abnormally low platelets, a type of blood cell mainly responsible for stopping bleeding. This occurs because the immune system mistakenly attacks and destroys platelets. There is a great need for newer ITP treatment options for people whose disease is not responsive or becomes intolerant to available therapies. Rilzabrutinib is an oral investigational medicine that acts on many mechanisms of the immune system that cause ITP. This summary article reviews the currently available preclinical and clinical data on the drug. Data from clinical studies showed that rilzabrutinib, when taken twice a day, increased platelet count levels rapidly and for long periods. Additionally, rilzabrutinib improved other disease aspects including fatigue and bleeding. Rilzabrutinib was well tolerated and showed a favorable safety profile. The quick and durable increase in platelet count levels, improvement in fatigue, decreased bleeding and need for rescue medication, and safety profile make rilzabrutinib a potentially promising treatment for ITP. Better effects are achieved if people receive the medicine earlier in their disease course. Studies of rilzabrutinib over longer periods of time, in children, and in other diseases are ongoing. Rilzabrutinib, an oral BTK inhibitor, offers rapid, durable platelet responses in ITP via multi-immune modulation. Rilzabrutinib is potentially promising for ITP & other immune-mediated diseases. #Immunotherapy #ITP