Kezar Life Sciences Announces Orphan Drug Designations for KZR-616 for the Treatment of Polymyositis and Dermatomyositis
Kezar Life Sciences, Inc. (Nasdaq: KZR), a clinical-stage biotechnology company discovering and developing breakthrough treatments for immune-mediated and oncologic disorders, today announced that the U.S. Food and Drug Administration has granted Orphan Drug Designations (ODD) for KZR-616 for the treatment of polymyositis (PM) and dermatomyositis (DM). Both orphan diseases are autoimmune inflammatory myopathies that are chronic and debilitating diseases characterized by marked morbidity and mortality. The estimated prevalence of PM and DM in the United States is up to 51,000 and 71,000, respectively.
KZR-616 is a first-in-class selective immunoproteasome inhibitor with the potential to impact multiple drivers of immune-mediated diseases and inflammation. PRESIDIO, a Phase 2 placebo-controlled cross-over clinical trial to evaluate KZR-616 for the treatment of PM and DM, is currently enrolling subjects.
“We are very pleased to have received orphan drug designations for both polymyositis and dermatomyositis. This recognition spotlights the significant unmet need for patients living with these autoimmune myopathies. KZR-616 has potential to truly modify the underlying pathophysiology of these two diseases,” said Noreen R. Henig, MD, Kezar’s Chief Medical Officer. “We will share more detail on the preclinical scientific rationale for the use of selective immunoproteasome inhibition to treat these rare and debilitating diseases during the 2020 American College of Rheumatology Conference next month.”
Orphan Drug Designation is intended to advance drug development for rare diseases. The FDA defines rare diseases as those affecting fewer than 200,000 people in the United States. Orphan Drug Designation can provide certain benefits and incentives for KZR-616, including a period of marketing exclusivity for the first marketing application, if regulatory approval is received for the designated indication, potential tax credits for qualified clinical testing and waiver of certain administrative fees.
KZR-616 is a novel, first-in-class, selective immunoproteasome inhibitor with broad therapeutic potential across multiple autoimmune diseases. Preclinical research demonstrates that selective immunoproteasome inhibition results in a broad anti-inflammatory response in animal models of several autoimmune diseases, while avoiding immunosuppression. Data generated from Phase 1a and 1b trials provide evidence that KZR-616 exhibits a favorable safety and tolerability profile for development in chronic autoimmune diseases. Phase 2 trials are underway in severe autoimmune diseases.