Pfizer Rare Disease Spin Out SpringWorks Therapeutics Raises $103 Million

Rare Daily Staff

SpringWorks Therapeutics, which Pfizer spun out as an effort to advance certain stalled drugs in  development that have promise for benefitting underserved patient populations, said it completed a $103 million Series A round with investment from Bain Capital Life Sciences, Bain Capital Double Impact, OrbiMed, Pfizer, and LifeArc.

The New York-based company has rights to four clinical-stage experimental therapies from Pfizer. Lara Sullivan, founder and president of SpringWorks and a former vice president at Pfizer, said the company also plans to expand its pipeline by partnering with other life science companies and academic institutions. Daniel Lynch, a biotechnology industry veteran with more than 25 years of experience in management within the industry, serves as executive chairman of the newly formed company.

Freda Lewis-Hall, executive vice president and CMO of Pfizer, said SpringWorks represents a new way to get things done with and for patients. “Pfizer sees SpringWorks Therapeutics as a groundbreaking new model for collaboration to deliver on the promise of medical research and development, so that more people have the potential to overcome disease,” said Lewis-Hall. “We hope that our investment in SpringWorks Therapeutics will, over time, enable us to realize even more value for patients and society.”  

SpringWorks said it is pursuing what it has termed a “collaborative business model” that is designed to deliver both social and financial returns. These partnerships include a range of entities including scientists, biopharmaceutical partners, patient groups, funders, and philanthropists.

The company is initially focusing its efforts on four development programs. This includes a phase 3-ready program for nirogacestat, a gamma-secretase inhibitor to treat desmoid tumors, a rare, non-metastatic tumor of connective tissue cells. Desmoid tumors can cause severe morbidity, pain, and loss of function in children and adults.

It is also working to initiate a phase 3 program of its MEK 1/2 inhibitor to treat neurofibromatosis, a set of three genetic disorders that cause tumors to grow on nerves throughout the body and can lead to blindness, deafness, disfigurement, cancer, bone abnormalities, learning disabilities, and severe pain.

A third program is working to advance semipro, an experimental therapy for hereditary xerocytosis, a genetic disorder in which red blood cells become dehydrated due to loss of potassium and cell water. The condition can cause anemia and complications including jaundice, fatigue, splenomegaly, and gallstones.

In the case of each of these programs, SpringWorks identified patient groups it is working with to work to see that the needs of the patient community will be addressed.

The final program involves the development of an experimental FAAH inhibitor to treat post-traumatic stress disorder, a chronic condition that some people develop after experiencing traumatic or life-threatening events, serious injury, or sexual violence. The therapy has demonstrated a good safety profile in previous early-stage studies. SpringWorks is working with the nonprofit Cohen Veterans Bioscience to assess patient populations that could benefit from this mechanism.

September 25, 2017