What does a big pharma do if it has a few promising but surplus drugs lying around? If you’re Pfizer, you bundle them together into a new biotech along with a block of start-up cash.
The new company – called SpringWorks Therapeutics – made its debut yesterday with four Pfizer candidates targeting an eclectic mix of indications outside the big pharma’s target therapeutic categories, plus $103m in funding from the drugmaker plus venture capitalists Bain Capital and Orbimed and medical charity LifeArc.
The four drugs are nirogacestat for use in patients with desmoid tumours, the potential neurofibromatosis treatment PD-0325901, senicapoc for hereditary xerocytosis and PF-0445784 to treat post-traumatic stress disorder (PTSD) – all of which are ready for phase II or III testing.
What they have in common is that they “may hold significant promise for underserved patients” according to Pfizer’s chief medical officer Freda Lewis-Hall, who said that the formation of SpringWorks is “a groundbreaking new model for collaboration to deliver on the promise of medical research and development”.
The new company is headed by former Pfizer vice president Lara Sullivan, who said the intention is to add to the first block of four drugs with additional pipeline programmes by partnering with other companies and academic institutions. Pfizer is contributing both equity capital and royalty- and milestone-bearing licenses to the experimental therapies.
Other senior figures at the new company include executive chairman Daniel Lynch, acting head of R&D Stephen Squinto, chief business officer Saqib Islam and Mary Smith vice president of clinical R&D.
Among the drug candidate, nirogacestat is a gamma-secretase inhibitor that failed to hit the mark in a breast cancer trial but could see a new lease of life in desmoid tumours, a rare cancer affecting connective tissue that can cause severe pain and loss of function and affects 900-1,200 patients in the US every year. SpringWorks is planning a phase III trial in collaboration with the Desmoid Tumor Research Foundation.
Neurofibromatosis candidate PD-0325901 is a MEK 1/2 inhibitor that it is hoped could reduce the tumours that grow on nerves throughout the body in the disease, leading to disfigurement and a host of other complications including blindness, deafness, learning disabilities and pain. It’s already shown promise in a phase I/II study and a phase III trial is planned in partnership with the Children’s Tumor Foundation.
Hereditary xerocytosis (HX) is a genetic disorder affecting 1 in 10,000 people in which red blood cells become dehydrated due to loss of potassium and cell water, causing anaemia and other symptoms. SpringWorks’ senicapoc is a potassium channel blocker that previously failed a trial in sickle cell anaemia but has shown a good safety profile in earlier studies.
Finally, FAAH inhibitor PF-0445784 didn’t pass muster in pain trials but according to SpringWorks has potential in PTSD and has cleared phase I studies.