The four compounds licensed to SpringWorks are:

The gamma-secretase inhibitor nirogacestat (PF-03084014) for desmoid tumors, which affect about 900-1,200 people annually in the US. The rare, non-metastatic tumors of connective tissue cells may lead to severe pain and loss of function and early death for children and adults. SpringWorks will work collaboratively with the Desmoid Tumor Research Foundation to test nirogacestat in Phase III.
SpringWorks also plans to initiate a Phase III program for the MEK 1/2 inhibitor PD-0325901 in neurofibromatosis (NF), which refers to three genetic disorders – NF1, NF2 and schwannomatosis – that cause tumors to grow on nerves throughout the body, leading to blindness, deafness, disfigurement, cancer, bone abnormalities, learning disabilities and severe pain. The Phase III program will focus on NF1, which affects one in 3,000 people who usually are diagnosed as children. The company will work with the Children's Tumor Foundation on this program.
Senicapoc (PF-05416266), which already has been evaluated in Phase I, II and III studies, will be assessed for the treatment of hereditary xerocytosis (HX), a genetic disorder in which red blood cells become dehydrated due to loss of potassium and cell water, causing anemia, jaundice, fatigue, splenomegaly and gallstones. SpringWorks will determine a path forward for a Phase II program in HX, which affects one in 10,000 children.
About 8.6m people in the US suffer from some form of PTSD, so SpringWorks will work with Cohen Veterans Bioscience to evaluate the path forward in Phase II for the FAAH inhibitor PF-0445784, which appeared to be safe and well-tolerated in Phase I studies. PTSD generally is treated with antidepressants and psychotherapy, but suicide is common among patients even with treatment.

SpringWorks Launches With $103m, Four Pfizer Drugs And A Focus On Underserved Patients

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