PRN1008: BTK Inhibitor Autoimmune / Inflammatory Diseases

  • BTK is present in the signaling pathways of most types of white blood cells except for T cells and plasma cells
  • Inhibition of BTK results in rapid anti-inflammatory effects, neutralization of pathogenic autoantibodies, and blocks the production of new autoantibodies
  • First-generation irreversible covalent BTK inhibitors have made significant contributions to the treatment of hematological malignancies; however, their adverse effects provided the inspiration for our development of next generation molecules more suitable for the chronic autoimmune diseases we seek to address
  • Inflammation and autoantibodies are the key drivers in autoimmune diseases like pemphigus (Pemphigus Vulgaris, PV, and Pemphigus Foliaceus, PF) and Immune Thrombocytopenia (ITP)
  • The action of BTK inhibition offers broad activity compared to other agents in the treatment of multiple autoimmune diseases without long-term impact of B cell depletion or the inconvenience of injectable therapies

Pemphigus (PV and PF)

Phase 3 (the PEGASUS study) now enrolling

  • Pemphigus is a group of rare, potentially life-threatening, chronic diseases characterized by acantholysis, or the loss of intercellular adhesion among keratinocytes, which results in erosions and blisters of the skin and mucous membrane

  • We have achieved proof of concept based on the interim results from 27 patients in our Phase 2 trial and expect the final results from this trial to be available soon

Immune Thrombocytopenia (ITP)

Phase 2 ongoing

  • ITP is a rare and often chronic autoimmune disease where blood levels of platelets, a key component in normal blood clotting, drop to very low levels

  • In contrast to other BTK inhibitors, PRN1008 did not impact platelet aggregation in blood samples from either normal healthy volunteers or ITP patients

  • We are conducting an open-label adaptive Phase 2 trial in up to 24 patients with relapsed primary or secondary ITP