The late-stage studies funded by the financing round could set the stage for two potential regulatory approvals if the data remains strong.
SpringWorks Therapeutics snagged $125 million in a Series B funding round that will help it accelerate its two late-stage cancer treatments into late-stage trials and toward potential regulatory approval, as well as support the continued expansion of the company’s emerging targeted oncology programs.
The Series B round was backed by stalwart investors in the pharma arena, including ArrowMark Partners, Samsara BioCapital, GlaxoSmithKline and Laurion Capital Management. SpringWorks also saw continued financial support from long-time investors that includes Pfizer, OrbiMed, Bain Capital and LifeArc.
SpringWorks is driving toward potential approval with nirogacestat, a gamma-secretase inhibitor for the treatment of desmoid tumors and PD-0325901, a MEK inhibitor for the treatment of neurofibromatosis type 1-associated plexiform neurofibromas. Both of these late-stage drugs were part of Pfizer’s pipeline when SpringWorks was spun out of that company in 2017. When SpringWorks was launched, the company secured $103 million in a Series A. The company was launched in order to provide some hope for the development of medicines across therapeutic areas where there is an urgent need.
Saqib Islam, SpringWorks chief executive officer, said the latest financing round “underscores the progress” the company has made in advancing its late-stage programs toward pivotal studies.
We are well positioned to continue to execute on our strategy to build a leading rare disease and targeted oncology company that brings promising science to underserved patient communities,” he said in a statement.
In November, SpringWorks received Orphan Drug designation from the U.S. Food and Drug Administration (FDA) for PD-0325901. PD-0325901 inhibits MEK, a key signaling protein for cellular survival and proliferation. The investigational therapeutics has been shown to block MEK phosphorylation in clinical biopsies. That blocking causes the cells to die. Neurofibromatosis type 1, or NF1, is a rare genetic disorder that is caused by mutations in the NF1 gene, and that affects both children and adults. The company is currently evaluating PD-0325901 as a monotherapy for NF1. But, the company said that given the critical role that the MAPK pathway plays in the growth and proliferation of a large number of tumor types, SpringWorks is also pursuing PD-0325901 in combination with other rational anti-cancer agents across a range of solid tumors. The company anticipates initiating Phase III trial this year. In September, just ahead of the Orphan Drug Designation approval, SpringWorks and Beigene teamed up to develop treatments with PD-0325901. The two companies will pair PD-0325901 with BeiGene’s investigational RAF dimer inhibitor, lifirafenib (BGB-283) in patients with advanced solid tumors.
Also in November, SpringWorks received Fast Track designation from the FDA for nirogacestat, for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis. Desmoid tumors are rare and debilitating soft-tissue tumors that can occur in both children and adults. Depending on their size and location, desmoid tumors can cause severe morbidities such as pain, internal bleeding, disfigurement, and limited range-of-motion. In June 2018, the U.S. FDA granted Orphan Drug designation for nirogacestat for the treatment of desmoid tumors. SpringWorks will launch a Phase III trial for nirogacestat this year.
