Lara Sullivan, MD, President and Founder at SpringWorks Therapeutics discusses her company, which was originally conceived by Pfizer as a way to advance investigational therapies that may hold promise for underserved patients. SpringWorks Therapeutics’ collaborative business model is designed to deliver both social and financial returns via partnerships with a variety of stakeholders, including scientists, biopharmaceutical partners, patient groups, funders and philanthropists. Pfizer’s contribution consists of both equity capital and royalty- and milestone-bearing licenses to experimental therapies.

SpringWorks is focused on underserved patient populations where there is great medical need. The company plans to move forward potential programs for four diseases, all of which currently have no cure. SpringWorks Therapeutics plans to expand its pipeline by partnering with other life science companies and academic institutions who share in the company’s mission.

Desmoid Tumor
A desmoid tumor is a rare, non-metastatic tumor of connective tissue cells, which can cause severe morbidity, pain and loss of function in children and adults. Desmoid tumors can show up in almost any part of the body, and desmoids that are faster growing or located near vital organs can cause life-threatening problems. Approximately 900-1,200 people are diagnosed with desmoid tumors each year in the U.S.1 Currently available treatments include unapproved medical therapy, radiation therapy, thermal ablation and surgery, which can be dangerous, costly and offer limited effectiveness. SpringWorks Therapeutics is planning to initiate a Phase 3 program to establish safety and efficacy of nirogacestat (PF-03084014), its gamma-secretase inhibitor, and will work collaboratively with the Desmoid Tumor Research Foundation to enable the needs of the patient community to be addressed.

Neurofibromatosis
Neurofibromatosis (NF) refers to three genetic disorders—NF1, NF2 and schwannomatosis—which cause tumors to grow on nerves throughout the body and can lead to blindness, deafness, disfigurement, cancer, bone abnormalities, learning disabilities and severe pain. NF1 affects one in 3,000 individuals and usually is diagnosed in childhood when symptoms begin to appear.2 MEK inhibitors have shown encouraging activity in reducing tumor size in clinical Phase 1-2 studies in patients with plexiform neurofibromatosis, one of the many manifestations of NF1. SpringWorks Therapeutics is planning to initiate a Phase 3 program to establish safety and efficacy of its MEK 1/2 inhibitor (PD-0325901) in the NF1 population and will work collaboratively with the Children’s Tumor Foundation to enable the needs of the patient community to be addressed.

Hereditary Xerocytosis
Hereditary xerocytosis (HX) is a genetic disorder in which red blood cells become dehydrated due to loss of potassium and cell water. The fragility of the dehydrated red cells can lead to a ranging severity of anemia and can cause complications including jaundice, fatigue, splenomegaly and gallstones. In some cases, it will lead to severe anemia that requires frequent blood transfusions. HX affects an estimated one in 10,000 people, and symptoms begin shortly after birth.3 There is no approved therapy for this disease. Senicapoc (PF-05416266) has demonstrated a good safety/tolerability profile in previous Phase 1-3 studies in other indications. SpringWorks Therapeutics plans to assess the potential activity of senicapoc in hereditary xerocytosis.