SpringWorks Therapeutics (NASDAQ:SWTX) dosed the first patient in its Phase 2b trial evaluating mirdametinib in children and adults with neurofibromatosis type 1 (NF1)-associated plexiform neurofibromas (PN).
NF1 is a rare genetic disorder that affects the MAPK pathway, a signalling network that regulates cell growth and survival. Mirdametinib is an oral, small molecule inhibitor of MEK1 and MEK2, which play key roles in the MAPK pathway.
“Unfortunately, up to half of all NF1 patients are at risk of developing PNs, a more severe form of NF1 that causes painful tumors to grow on nerves throughout the body,” CEO Saqib Islam said in a statement.
“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 PN,” he added.
The study will enroll some 100 patients with a primary endpoint of objective response rate.
