A Phase 3, Randomized, Double-Blind, Placebo-Controlled, 3-Arm, 3-Period Study to assess the Efficacy and Safety of a New Formulation of Oral Cladribine compared with Placebo in Participants with Generalized Myasthenia Gravis (gMG) (MyClad)

Generalized Myasthenia Gravis (gMG) is a chronic neuromuscular disease that causes weakness in the voluntary muscles. Voluntary muscles include muscles that connect to a person’s bones, muscles in the face, throat, and diaphragm. They contract to move the arms and legs and are essential for breathing, swallowing and facial movements. Myasthenia gravis is an autoimmune disease, which means that the body’s defence system mistakenly attacks healthy cells or proteins needed for normal functioning. The onset of the disorder may be sudden. Symptoms may not be immediately recognized as myasthenia gravis. The degree of muscle weakness involved varies greatly among individuals. Currently, there is no known cure for myasthenia gravis. Most people with myasthenia gravis live to an average life expectancy. Cladribine is a short-course oral therapy for generalized myasthenia gravis (gMG). It is designed to lower the levels of the immune cells that drive inflammatory attacks in gMG. gMG is recognized as a T cell dependent, B cell mediated autoimmune disease, and the role of different B cell subtypes and regulatory T cells dysfunction has been described. Given the mechanism of action of cladribine on cell types involved in both MS and MG pathologies, it is hypothesized that cladribine will act on similar immune pathways of MG pathology. The current Phase 3 study is designed to demonstrate the efficacy and safety of oral cladribine compared to placebo in participants with gMG, as well as to collect additional long-term safety and efficacy data