Pfizer spins out company to advance promising rare-disease drugs

 

However, in what is a first-of-its-kind move for Pfizer, the company is spinning out a new company with the help of other investors to advance promising investigational therapies in areas of urgent patient need.

 

As noted in a Sept. 25 news release, SpringWorks Therapeutics, a mission-driven medicines company dedicated to developing innovative potential new treatments for underserved patient communities, announced its launch with a completed $103-million Series A financing funded by Bain Capital Life Sciences, Bain Capital Double Impact, OrbiMed, Pfizer and LifeArc (formerly known as MRC Technology). SpringWorks Therapeutics also has rights to four clinical-stage experimental therapies from Pfizer.

 

“SpringWorks Therapeutics will pursue the development of medicines across therapeutic areas, focused on diseases where there is an urgent need and the potential for the greatest impact for patients,” said Dr. Lara S. Sullivan, founder and president of SpringWorks Therapeutics and a former vice president at Pfizer. “We initially have rights to four very promising experimental therapies and, over time, plan to expand our pipeline by partnering with other life-science companies and academic institutions who share in our mission.”

 

As noted in the announcement, Pfizer conceived the spinout in large part as a “collaborative business model [that] 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.”

 

“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. We hope that our investment in SpringWorks Therapeutics will, over time, enable us to realize even more value for patients and society,” said Dr. Freda Lewis-Hall, an executive vice president and the chief medical officer for Pfizer. “SpringWorks Therapeutics started as an idea about a new way to get things done with—and for—patients, it’s been a tremendous team effort, and we and our partners are excited to see it become a reality.”

 

As Forbes noted of the new company, many Pfizer drugs have found success outside the company over the past decade—for example, Puma Biotechnology has run with breast cancer drug Nerlynx, originally a Pfizer compound, and boasts a $3.89-billion market capitalization; cardiovascular drugmaker Esperion was bought by Pfizer and spun out again, and it has a $1.27-billion market capitalization: and Durata Therapeutics, bought by Actavis in 2014 for $675 million, was also based upon Pfizer drugs. As Forbes quoted John LaMattina, a senior partner at PureTech Ventures and former head of research and development at Pfizer: “Pfizer perhaps has learned from the fact that it’s had a lot of compounds on its shelf that others capitalized on.”

 

Xconomy.com noted, for its part, that SpringWorks plans not only to work on the four Pfizer drugs but also potential therapeutics abandoned or neglected by other pharma companies. The criteria, said Xconomy, is that “SpringWorks favors drugs ready for Phase 2 that it can develop for diseases with either no approved therapies, or ‘minimal to no competition from others,’” as Sullivan puts it.

 

Of the four compounds, three are for rare diseases and one is for post-traumatic stress disorder, as follows, according to the SpringWorks announcement:

 

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 to 1,200 people are diagnosed with desmoid tumors each year in the United States. 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 (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. 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 (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. 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.

 

Post-traumatic stress disorder (PTSD) is a chronic condition that some people develop after experiencing traumatic or life-threatening events, serious injury or sexual violence. PTSD involves the persistent re-experiencing of the traumatic event, which results in avoidance of trauma-related stimuli, as well as negative feelings and heightened anxiety-like symptoms. PTSD is often seen in military veterans, first responders, rape and battery victims and abused children. Around 8.6 million people in the U.S. between the ages of 18-64 have been diagnosed with the disease. Over 200,000 veterans in the U.S. live with PTSD, which is currently treated with antidepressants such as SSRIs and trauma-focused psychotherapy; however, the disease can result in suicide even with treatment. SpringWorks Therapeutics’ FAAH inhibitor (PF-0445784) has demonstrated a good safety/tolerability profile in previous Phase 1 studies, though its effectiveness in PTSD is to be determined. Cohen Veterans Bioscience and SpringWorks Therapeutics plan to assess patient populations that could benefit from this mechanism.