Pfizer Spin-off SpringWorks Therapeutics Files for $115 Million IPO to Advance Rare Disease and Oncology Therapies

On August 19, 2019, VCBeat (WeChat ID: vcbeat) learned from foreign media reports that biopharmaceutical company SpringWorks Therapeutics (“SpringWorks”) announced its plan to conduct an initial public offering (IPO), aiming to raise $115 million. The company has filed a preliminary prospectus.

It is reported that SpringWorks has completed two rounds of financing. The company closed a $125 million Series B round in April 2019 and a $103 million Series A round in September 2017.

SpringWorks Therapeutics, a clinical-stage biopharmaceutical company headquartered in Connecticut, USA, was spun off from Pfizer in 2017. The company primarily focuses on developing and commercializing therapeutic drugs for severe rare diseases and cancer, providing patients with new treatment options. Since its inception, SpringWorks has collaborated with multiple pharmaceutical companies and research institutions and has secured funding from various investment partners.

One of SpringWorks’ lead drug candidates, nirogacestat, is a small-molecule γ-secretase inhibitor primarily indicated for the treatment of patients with progressive, unresectable, and recurrent desmoid tumors. SpringWorks has completed Phase I and Phase II clinical trials of this drug. In June 2018, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to nirogacestat. In November of the same year, the drug also received FDA Fast Track designation.

Desmoid tumors, also known as aggressive fibromatosis, are rare soft tissue tumors that predominantly affect young adults. These tumors can invade surrounding healthy tissues, including joints, muscles, blood vessels, nerves, and internal organs, and may occur in any part of the body. The severity of desmoid tumors and their associated symptoms often varies depending on the tumor’s size, location, and growth rate. It is estimated that 2 to 5 individuals per million worldwide are affected by desmoid tumors, with 1,000 to 1,500 new cases diagnosed annually in the United States.

Traditional treatment for desmoid tumors involves surgical resection, with amputation even required in more severe cases. However, despite such interventions, the tumor recurrence rate remains high. Currently, there are no approved medications for desmoid tumors.

SpringWorks Therapeutics has also developed mirdametinib for the treatment of neurofibromatosis type 1 (NF1). Mirdametinib is a small-molecule oral inhibitor that blocks the mitogen-activated protein kinase 1 and 2 (MEK1/2) signaling pathways involved in tumorigenesis and tumor progression. The U.S. Food and Drug Administration (FDA) and the European Commission have granted orphan drug designation to mirdametinib for the treatment of NF1. The FDA has also granted fast track designation to mirdametinib for the treatment of patients aged two years and older with NF1 who are not amenable to complete surgical resection.

Neurofibromatosis type 1 (NF1) is a rare genetic disorder caused by mutations in the NF1 gene, which impair or completely abolish the tumor-suppressor function of neurofibromin. Approximately 30%–50% of patients with NF1 develop severe manifestations such as plexiform neurofibromas (PNF), which can lead to pain, disfigurement, and loss of motor function, resulting in reduced survival rates. It is estimated that there are about 100,000 individuals with NF1 in the United States, with symptoms typically emerging in early childhood.

(Compiled by Xu Xiaoxue)