SpringWorks starts trial re neurofibromas

SpringWorks reports that the first patient has been dosed in the Phase 2b ReNeu clinical trial evaluating mirdametinib (formerly PD-0325901), an oral, small molecule designed to inhibit MEK1 and MEK2, in children and adult patients with neurofibromatosis type 1 (NF1)-associated plexiform neurofibromas (NF1-PN).

“Unfortunately, up to half of all NF1 patients are at risk of developing plexiform neurofibromas, a more severe form of NF1 that causes painful tumors to grow on nerves throughout the body,” said Saqib Islam, Chief Executive Officer of SpringWorks. “While there are no therapies currently approved for these patients, several prior studies, including a Phase 2 study evaluating mirdametinib, have provided support for the mechanism of MEK inhibition in plexiform neurofibromas. We look forward to continuing to enroll patients in the ReNeu trial to further evaluate the clinical benefits of mirdametinib for children and adults with these devastating tumors.” more