Focus Areas

Too many loved ones live with uncertainty.
Let’s find answers.

We focus on patients with rare tumors, hematological cancers and biomarker-defined metastatic solid tumors. Because the need is great, we are digging deep into the science of our lead product candidates – nirogacestat and mirdametinib – to unlock their full potential while simultaneously developing other promising molecules. As part of our approach, we are actively pursuing new licensing and partnership opportunities with other innovators to help advance promising science as efficiently as possible so people living with devastating cancers can live better.

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.