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

OGSIVEO (nirogacestat), is an oral, small molecule gamma secretase inhibitor (GSI) that is approved in the U.S. for the treatment of adult patients with progressing desmoid tumors who require systemic treatment. We are also evaluating nirogacestat as a monotherapy in children and adults with progressive, surgically unresectable desmoid tumors. Additionally, we are evaluating nirogacestat as a potential treatment for patients with ovarian granulosa cell tumors and for patients with multiple myeloma as part of several B-cell maturation agent (BCMA) combination therapy regimens in collaboration with industry and academia.

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 can cause severe disfigurement, pain and functional impairment. NF1-PN can also become malignant over time.

Please see important safety information including link to full Prescribing Information for OGSIVEO at the bottom of this page.

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 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.



  • Diarrhea: Diarrhea, sometimes severe, can occur in patients treated with OGSIVEO. Diarrhea occurred in 84% of patients treated with OGSIVEO, and included Grade 3 events in 16% of patients. Median time to first diarrhea event was 9 days (range: 2 to 434 days). Monitor patients and manage using antidiarrheal medications. Modify dose as recommended.
  • Ovarian Toxicity: Female reproductive function and fertility may be impaired in patients treated with OGSIVEO. Impact on fertility may depend on factors like duration of therapy and state of gonadal function at time of treatment. Long-term effects of OGSIVEO on fertility have not been established. Advise patients on the potential risks for ovarian toxicity before initiating treatment. Monitor patients for changes in menstrual cycle regularity or the development of symptoms of estrogen deficiency, including hot flashes, night sweats, and vaginal dryness.
  • Hepatotoxicity: ALT or AST elevations occurred in 30% and 33% of patients, respectively. Grade 3 ALT or AST elevations (>5 × ULN) occurred in 6% and 2.9% of patients. Monitor liver function tests regularly and modify dose as recommended.
  • Non-Melanoma Skin Cancers: New cutaneous squamous cell carcinoma and basal cell carcinoma occurred in 2.9% and 1.4% of patients, respectively. Perform dermatologic evaluations prior to initiation of OGSIVEO and routinely during treatment.
  • Electrolyte Abnormalities: Decreased phosphate (65%) and potassium (22%) occurred in OGSIVEO-treated patients. Phosphate <2 mg/dL occurred in 20% of patients. Grade 3 decreased potassium occurred in 1.4% of patients. Monitor phosphate and potassium levels regularly and supplement as necessary. Modify dose as recommended.
  • Embryo-Fetal Toxicity: OGSIVEO can cause fetal harm when administered to pregnant women. Oral administration of nirogacestat to pregnant rats during the period of organogenesis resulted in embryo-fetal toxicity and death at maternal exposures below human exposure at the recommended dose of 150 mg twice daily. Advise pregnant women of the potential risk to a fetus. Advise females and males of reproductive potential to use effective contraception during treatment with OGSIVEO and for 1 week after the last dose.


  • The most common (≥15%) adverse reactions were diarrhea (84%), ovarian toxicity (75% in the 36 females of reproductive potential), rash (68%), nausea (54%), fatigue (54%), stomatitis (39%), headache (30%), abdominal pain (22%), cough (20%), alopecia (19%), upper respiratory tract infection (17%), and dyspnea (16%).
  • Serious adverse reactions occurred in 20% of patients who received OGSIVEO. Serious adverse reactions occurring in ≥2% of patients were ovarian toxicity (4%).
  • The most common laboratory abnormalities (≥15%) were decreased phosphate, increased urine glucose, increased urine protein, increased AST, increased ALT, and decreased potassium.


  • CYP3A Inhibitors and Inducers: Avoid concomitant use with strong or moderate CYP3A inhibitors (including grapefruit products, Seville oranges, and starfruit) and strong or moderate CYP3A inducers.
  • Gastric Acid Reducing Agents: Avoid concomitant use with proton pump inhibitors and H2 blockers. If concomitant use cannot be avoided, OGSIVEO can be staggered with antacids (e.g., administer OGSIVEO 2 hours before or 2 hours after antacid use).
  • Consult the full Prescribing Information prior to and during treatment for important drug interactions.


  • Because of the potential for serious adverse reactions in breastfed children, advise women not to breastfeed during treatment with OGSIVEO and for 1 week after the last dose.

Please see accompanying full Prescribing Information.