It has been previously demonstrated that therapeutic inhibition of the prostaglandin E2 (PGE2)-degrading enzyme, 15-prostaglandin dehydrogenase (15-PGDH), was able to improve muscle strength in aged mice. Researchers from Epirium Bio Inc. have now reported the discovery and preclinical characterization an orally bioavailable small molecule inhibitor of 15-PGDH – MF-300 – being developed for the treatment of neuromuscular dysfunction.
While glucocorticoids are used as first-line therapy for Duchenne muscular dystrophy (DMD), these agents are associated with several adverse events, including accelerated bone loss and muscle atrophy. As a result, prolonged glucocorticoid treatment is one of the primary contributors to the high fracture rate in patients with DMD.