MARKETERJAZZ PHARMACEUTICALS

Modeyso is the first and only FDA-approved treatment for adult and pediatric patients aged 1 year and older with H3 K27M-mutant diffuse midline glioma (DMG) following progression after prior therapy. Dordaviprone is an orally administered, blood-brain barrier-penetrant small molecule imipridone that selectively targets dopamine receptor D2 (DRD2) and the mitochondrial protease ClpP, activating the integrated stress response, inducing apoptosis, and altering mitochondrial metabolism in tumor cells. Through these mechanisms, the therapy is designed to restore histone H3 K27 trimethylation and address the underlying biology of H3 K27M-mutant gliomas.

H3 K27M-mutant diffuse midline glioma is a rare and highly aggressive brain tumor that primarily affects the midline structures of the brain and spinal cord, most commonly in children and young adults. Patients face an extremely poor prognosis, with limited therapeutic options and very low survival rates following recurrence. Median survival is approximately one year from diagnosis and less than six months following progression after frontline therapy, underscoring the significant unmet need in this patient population.

Background

HCRx purchased a portion of royalties on sales of Modeyso from the former shareholders of Oncoceutics to unlock and accelerate proceeds owed to them.