New biotech firm will focus on rare diseases

Specialized Spinoff

New biotech firm will focus on rare diseases

The major pharma company, Pfizer, has created a new independent company, SpringWorks Therapeutics, which will focus on the production of drugs aimed at rare diseases, which is not profitable for Pfizer itself. Some of these drugs, which the drug giant did not choose to move forward internally, are being repurposed by the new spinoff biotech company.

Ellie Kincaid, writing in Forbes, reports that the new venture is being financed by $103 million from several investors. These companies include Pfizer, Bain Capital Life Sciences, Bain Capital Double Impact, Orbimed and LifeArc.

Pfizer follows Johnson & Johnson as a large drug company creating a new smaller one with the help of outside investment. J&J gave rise to Provention Bio earlier this year on an investment of $28.4 million. The Pfizer deal “is the biggest one of these that we’ve seen,” says David Grainger, partner at Medicxi Ventures.

Lara Sullivan, a former vice president of Pfizer and now president of SpringWorks, joined with Freda Lewis-Hall to develop the new company over a period of 2½ years. They were aware of substances in Pfizer’s inventory that might treat rare diseases and benefit desperate patients. “A more partnered development paradigm and channel would be optimal to help those compounds go forward more quickly,” Sullivan said.

Freda Lewis-Hall, Pfizer’s chief medical officer, said, “We’re left with some compounds that we think have great promise for patients but are not necessarily core. We originally conceived of this as kind of a new and innovative way for us to advance the investigational therapies that we thought hold significant promise for underserved populations.”

Other drugs developed by Pfizer have wound up being profitable for other companies. Puma Biotechnology has had success with Nerlynx, a drug that treats breast cancer and was developed by Pfizer. Esperion has benefitted from the sale of cardiac medication that originated with Pfizer, and Durata Therapeutics was based on Pfizer drugs. John LaMattina, senior partner at PureTech Ventures and former head of R&D at Pfizer, explained, “Pfizer perhaps has learned from the fact that it’s had a lot of compounds on its shelf that others capitalized on.”

SpringWorks has four drugs licensed from Pfizer that it hopes to bring to market. Three of them are ready for phase 3 clinical trials. Senicapoc is intended for hereditary xerocytosis, a rare genetic form of anemia in which red blood cells become dehydrated, causing symptoms such as jaundice, tiredness, an enlarged spleen and gallstones. Nirogacestat is a gamma-secretase inhibitor for patients with noncancerous connective tissue growths called desmoid tumors. An MEK inhibitor for neurofibromatosis type 1, a genetic disorder in which noncancerous tumors grow next to nerves, is another candidate. The final compound SpringWorks is developing is a FAAH inhibitor for treating post-traumatic stress disorder.

“The common denominator is that these four programs all serve patient groups that have particular needs that have not been satisfied by current therapies,” Sullivan concluded.