Let’s get novel for patients living with rare tumors
Our lead program, nirogacestat, is an oral, small molecule gamma secretase inhibitor (GSI) in Phase 3 development for adult patients with desmoid tumors. These are rare, aggressive soft-tissue tumors characterized by locally invasive growth, significant morbidity, and a high rate of recurrence. We are also developing mirdametinib, our MEK inhibitor, for patients with neurofibromatosis type 1 associated plexiform neurofibromas (NF1-PN), which are peripheral nerve sheath tumors that cause severe disfigurement, pain and functional impairment. NF1-PN can also become malignant over time.
Let’s dig into BCMA combinations for multiple myeloma patients
Several BCMA-targeted therapies have demonstrated promising clinical activity in patients with relapsed or refractory multiple myeloma. We believe that by inhibiting gamma secretase with nirogacestat, membrane-bound BCMA can be preserved, thereby increasing target density while simultaneously reducing levels of soluble BCMA, which may interfere with BCMA-directed therapies. In preclinical models of human multiple myeloma, nirogacestat has shown the ability to meaningfully enhance the activity of BCMA-targeted therapies, and we are currently studying nirogacestat in multiple myeloma patients through clinical collaborations with seven industry–leading developers of BCMA therapies.
Let’s take on biomarker-defined metastatic solid tumors
We are developing targeted therapies for highly prevalent, biomarker-defined metastatic cancers. Our portfolio currently includes investigational treatments for cancers driven by mutations in the MAPK and Hippo pathways, which in aggregate could represent more than one-third of all solid tumor patients.
Let’s unlock the future of targeted oncology
We are meticulous about the science and passionate about its possibilities. This leads us to dig deeper – to explore the full potential of our molecules. You can see that approach in our pipeline. We have a diversified targeted oncology portfolio that includes standalone and combination therapies across various stages of development, including late-stage clinical trials in rare tumor types and multiple programs addressing highly prevalent, biomarker-defined cancers.