Buoyed by blockbuster Vertex payments, the CF Foundation seeds new advances with $500M fund
What does a large foundation do when they receive a check for 10 times their annual revenue?
For five years now, the Cystic Fibrosis Foundation has been a living experiment to that question. In 2014, the group saw its foray into venture philanthropy pay off with a $3.3 billion check from Royalty Pharma for royalties to Vertex’s CF treatments. (The Eli Lilly heiress-endowed Poetry Foundation has been a smaller long-running experiment in the same question, for the literary pharma nerds out there.)
Today, CFF unveiled their biggest project since Vertex, launching a $500 million fund called Path to a Cure dedicated to supporting gene-based therapies to help the 10% of patients who have been passed over by the new round of treatments that have remade the disease for many patients since they first debuted in 2012. The fund is intended to last through 2025.
“This is a way of getting treatment for everyone,” Mike Boyle, who will be promoted to CEO of the foundation on January 1, told Endpoints News. “It’s a whole new technology.”
The new wave of CF treatments that earned the foundation its billions – including the three-drug combo Trikafta that the FDA approved last week – work by modulating the defective CFTR protein that causes the disease, helping it get to the right location and function properly there. But 10% of patients either have proteins folded in such a way they don’t respond to the modulators or they lack the protein altogether.
For those patients, the foundation is looking to fund potential gene editing or gene transfer solutions that would fix cystic fibrosis’s underlying genetic cause. CF is caused by mutations in both copies of the CTFR gene. An editing solution would fix the specific misspelled nucleotides, while a transfer would insert a complete functioning copy of the gene.
CFF will find themselves with far more funds than they used to fuel the rise of the protein modulators. They gave Vertex $150 million over 12 years. This new fund will give out more than 3 times that in half the time, and the group said they’re ready to invest far more if a particularly promising solution gains traction.
Boyle said they would and have funded proposals from all types of drug developers. But he’s also hoping the large dollar figure, along with the promise of the foundation’s experts and clinical network, would attract biotechs who have had success in other areas to take a chance on CF. CF is a more difficult target than muscles or other tissues, he said, because the immune system is stronger (to guard against the constant flow of airborne viruses) and cellular turnover is higher.
“We’ve been doing this [funding] all along but this was an effort to grab attention,” Boyle said. “I want the world’s best research focused on CF.”
In addition to the Path fund, the foundation has recently poured millions into research on anti-inflammatories and anti-infectives to curb some of the side effects and risks CF patients face, along with research for co-morbidities now emerging as patients live longer.
But if the new wave of research is effective – and far more dollars than this have been pushed into therapies that have dead ended – it could have a greater impact than any of the foundation’s other ventures. Although the gene therapies are first targeted at the untreatable forms of the disease, if successful they hold the potential to fix the disease entirely.
“Over time everyone would benefit — which is an important thing for companies as they think about their market,” Boyle said. “It would have to be effective, though, and that will take time.”