A new way to trial more drugs?

Pfizer announced this week that it has backed a new drug development company with the task of shepherding four of Pfizer’s experimental medicines through clinical trials, in an unusual deal designed to prevent promising products from languishing on its laboratory shelves. Whilst it is not unusual for emerging drug development companies to license in candidate drugs from big pharma, the novel twist here appears to be that Pfizer is pro-actively taking drugs from its shelves and licensing them to, while investing in, its SpringWorks Therapeutics start-up.

SpringWorks starts life with a $103 million series A investment from Bain Capital Life Sciences, Bain Capital Double Impact, Orbimed and LifeArc. Pfizer’s input consists of both equity capital and royalty- and milestone-bearing licenses to a series of experimental medicines. These comprise four compounds, two of which are already set for Phase 3 trials, including a gamma-secretase inhibitor targeted against desmoid tumour (a slow-growing, benign tumour that grows in connective tissue and affects 900 to 1,200 people each year). SpringWorks is also targeting neurofibromatosis (a rare condition that consists of benign tumours surrounding neurons) in late-stage tests with its MEK 1/2 inhibitor. The other two earlier-stage compounds target a rare blood disorder, hereditary xerocytosis, and post-traumatic stress disorder.

Freda Lewis-Hall, Pfizer’s chief medical officer, said the pharmaceutical group conceived of the new company as a way of ensuring medicines do not end up being shelved, a common problem at large drug makers that often unearth more molecules than they want to develop. “If you think about today’s rapid pace of scientific breakthroughs, we simply have more leads than we can follow,” she said. “These compounds are exciting, but no one can do it all by themselves.”

The phenomenon whereby big pharma abandons encouraging drugs has long bedevilled the industry. In recent weeks GlaxoSmithKline, Lilly, AstraZeneca and Teva Pharmaceuticals have announced they are abandoning some programmes to sharpen their focus on core areas. Unusually, Pfizer is licensing the four drugs to SpringWorks without receiving an upfront payment, although it will earn undisclosed fees if the medicines end up succeeding in clinical trials, as well as royalties if they go on sale.

If the model is successful then Pfizer could hand over more of its drugs to SpringWorks, Dr Lewis-Hall said, while the new company will also offer to develop medicines discovered by other pharma groups and academic institutions.

Pfizer has backed biotechs in the past, including more recently taking a stake in Ignite Immunotherapy and its cancer vaccine work, as well as Metabomed, a biotech that is using computational biology to discover cancer metabolism drugs. It has also helped shape Cydan, an orphan disease incubator created by Pfizer’s venture arm with New Enterprise Associates, but the SpringWorks model is a first for the company.

SpringWorks will be led by executive chairman Dan Lynch, who has held various executive jobs at a string of biotechs, and Lara Sullivan, who is leaving her role at Pfizer, where she has been VP of strategy and portfolio solutions, worldwide R&D for the past six years, to become president of the new group. “Sometimes great science, through no fault of its own, didn’t have the ability to go forward inside the walls of Pfizer,” said Ms Sullivan. “Every year we saw great programmes that didn’t make it for strategic or business reasons.” SpringWorks is the latest in a spate of companies that have been set up to act as repositories for drugs that have been discarded or are at risk, either because they have fallen victim to cost cutting or because they do not fit with increasingly narrow research and development priorities.

Large drug makers are also coming under increasing pressure from well-organised patient advocacy groups, which have demanded that they do not jettison medicines that could make a difference to sick people. SoftBank recently led a $1.1bn fundraising in Roivant Sciences to help fund the company’s strategy of “developing promising drugs that get stuck in an industry log jam that has nothing to do with science”. Axovant, a publicly listed offshoot of Roivant, announced this week results from a late-stage clinical trial of an Alzheimer’s medicine that it licensed from GSK, which had discarded the drug after a series of lacklustre studies. Unfortunately, the results were negative, an illustration not only of the challenges involved in developing medicines for Alzheimer’s but also the general risks inherent in drug development.

It remains to be seen if the SpringWorks model will work as a means of trialling drugs that have been shelved by big pharma. It is noteworthy that of the four experimental drugs licensed to SpringWorks, three are for rare diseases. Whilst the potential benefits for Pfizer are clear, it will be interesting to see if other big pharma companies will be willing to license their experimental medicines to a start-up in which Pfizer has a minority stake.

Dr Paul Rodgers