GRM-01

GRM-01 is a novel, orally available, non-steroidal selective glucocorticoid receptor agonist and modulator (SEGRAM) being developed for the treatment of Duchenne muscular dystrophy (DMD). It is designed to optimize the benefit-risk profile of glucocorticoid therapy by dissociating the anti-inflammatory effects (mediated by transrepression) from the metabolic and systemic side effects (primarily driven by transactivation). In preclinical studies using the B10.mdx mouse model, GRM-01 significantly improved skeletal limb muscle function and reduced diaphragm inflammation without the significant increases in fasting blood glucose typically associated with conventional glucocorticoids like prednisolone. Phase I clinical studies in healthy volunteers have demonstrated dose-dependent suppression of cortisol, indicating successful target engagement, while maintaining normal glucose-insulin homeostasis. A Phase II clinical trial in patients with DMD is planned for 2026.