Pfizer spin-off SpringWorks Therapeutics is carving out a niche repurposing useful molecules from moribund projects. This week, the New York City-based startup brought in $103 million in Series A funding to make that happen.
“SpringsWorks was formed to advance exciting science for unserved and underserved patient populations,” said president and founder Lara Sullivan in a phone interview. “There are certain populations that may not fit the strategic or budget parameters of innovator companies. We felt we could provide a development pathway where these programs could find a home.”
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Sometimes science is an uncooperative friend that doesn’t play nice with corporate strategies. Promising molecules can bottom out during development – potential oncology or cardiovascular disease drugs may move the needle in those indications. Some can find new life treating less profitable conditions, provided a company has the interest. SpringWorks wants to be that company.
“It’s not that they’re poor quality programs,” noted Sullivan, “it’s that the science took you in a different direction than maybe you anticipated.”
Their first drug candidate is a good example. Nirogacestat is gamma secretase inhibitor that was not effective enough against breast cancer. However, researchers at the National Institutes of Health thought the compound might be useful against desmoids — nonmalignant tumors that often recur. The market is small, around 1,200 patients a year, but there is a need.
“There’s a patient population that we have to find a different way to serve,” said Sullivan. “But we wondered: ‘Is it just the desmoids or is there something deeper?’ As we dug into the portfolio at Pfizer, we found it wasn’t just a one-off.”
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
Their second compound, an MEK inhibitor, was also a failed oncology therapy. After positive results from a trial at Cincinnati Children’s Hospital, it’s showing potential against neurofibromatosis, which causes nonmalignant tumors on nerve tissue. The company is also pursuing therapies for hereditary xerocytosis and post-traumatic stress disorder.
SpringWorks is the latest in a series of spinoffs. Johnson & Johnson recently created Provention Bio to target immune conditions. Cerevance is a neuroscience-focused company launched by Takeda.
In addition to rescuing these and other shelved molecules, SpringWorks is forging relationships with capital sources, government agencies and patient groups. They recognize that patients have become increasingly sophisticated about pharma R&D and can be a lever to pry away languishing compounds to be reborn against smaller indications.
“In a lot of ways, it starts with patient groups, who help us look at the landscape and understand where the science is that may not be organically happening in the industry right now,” said Sullivan.
The Series A was funded by Pfizer, Bain Capital Life Sciences, Bain Capital Double Impact, OrbiMed and LifeArc. Though Pfizer maintains a stake in the company, SpringWorks will work independently, giving it the flexibility to seek out and repurpose compounds from other industry leaders.
“We debated what the optimal business model would be: a captive subsidiary or an external independent company,” said Sullivan. “The optimal thing for the science was to make it a standalone independent company.”
Photo: theasis, Getty Images