SpringWorks Therapeutics is a clinical-stage biopharmaceutical company that takes a precision medicine approach in acquiring, developing and commercializing medicines for patient populations that are suffering from rare diseases and cancer.
Its lead pipeline programs are in potentially registrational clinical trials for rare oncology indications. Nirogacestat, its gamma secretase inhibitor, is in a Phase 3 clinical trial for treating desmoid tumors, which are rare and often debilitating and disfiguring soft-tissue tumors.
Mirdametinib, its MEK inhibitor, is in a Phase 2b clinical trial for treating adults and children with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN), which are peripheral nerve sheath tumors that cause severe pain, disfigurement and shortened life span.
SpringWorks is advancing, in partnership with industry leaders, several other programs addressing highly prevalent, genetically defined cancers, including evaluating nirogacestat as a cornerstone of BCMA combination therapy for patients with several myeloma and several efforts in RAS and RAF-mutated solid tumors with advanced precision medicine approaches for these types of cancer.