Next-Gen Therapies for RASopathies and MAPK Pathway-Driven Tumors and Other Diseases
A next-generation oral macrocyclic MEK1/2 inhibitor designed to address certain limitations associated with currently approved MEK inhibitors. To date, interim clinical data for PAS-004 have demonstrated favorable pharmacokinetic (PK) properties, including a long half-life, dose-proportional exposure, and sustained target inhibition, which may support improved tolerability and more consistent pathway suppression compared to earlier-generation MEK inhibitors.
PAS-004 is currently being evaluated in ongoing Phase 1 clinical studies in both advanced cancer patients and adults with NF1-associated plexiform neurofibromas.
Neurofibromatosis Type 1 (NF1) is a genetic disorder from NF1 gene mutations, resulting in neurofibromin deficiency, RAS/MAPK pathway hyperactivation, and uncontrolled tumor growth. It frequently causes disfiguring and morbid plexiform neurofibromas (PNs) that are often inoperable, with current treatments limited by toxicity, prompting our efforts to target the pathway with our macrocyclic, extended-half-life drug candidate, PAS-004, currently in international clinical trials.