Last fall, all eyes darted to the concept of venture philanthropy – using venture dollars to empower patient advocacy efforts – when the Cystic Fibrosis Foundation sold its royalty rights to the Vertex drugs it funded for $3.3 billion.
A sort of forced collaboration between academia and industry helped the CFF usher in the development of the CF drug Kalydeco – and the concept of venture philanthropy, said Preston W. Campbell, the executive vice president for medical affairs at the CFF. It wasn’t easy, however – academics didn’t respect industry, and vice versa.
“We insisted that they collaborate,” Campbell said. “If the money got them into the kitchen, the collaboration was the secret sauce that ultimately led to success.”
When Investment Rhymes with Canada
Canada has a proud history of achievement in the areas of science and technology, and the field of biomanufacturing and life sciences is no exception.
Campbell spoke about the disruptive nature of venture philanthropy and patient advocacy on a panel at this week’s CED Life Sciences conference in Raleigh. He was joined by two others in this space – Ilan Ganot of Solid Ventures and Josh Sommer of the Chordoma Foundation.
Ganot and Sommer have personal stakes in their respective ventures. Ganot, a former investment banker, finetuned his focus to raising funds for Duchenne muscular dystrophy when his young son was diagnosed with the degenerative condition. And Sommer was diagnosed with chordoma, a rare cancer, as an undergrad at Duke – and has since spun his personal stake in research and drug development into a foundation that’s following the path of CFF.
“Importantly, we didn’t want to be a grant-making organization,” Sommer said. “We want to be outcome oriented.”
This, he said, is why a venture philanthropy approach to funding patient-generated research ideas makes sense. The foundation helps patients connect with clinical trials that it’s helped build – another example of being a philanthropic conduit to partnering academia and industry.
“One of the most surprising things is that there’s a great deal of interest among companies to find orphan indications for drugs that are in their oncology portfolios or pipelines,” Sommer said.
And companies have a real incentive to go down this orphan route, of course – thanks to the 21st Century Cures act.
Ganot transferred his banking expertise into building a venture philanthropy model of his own in Solid Ventures.
“My whole career, I kind of figured out what was a good idea, then got people to invest, did some good business, made some money,” Ganot said.
So when his son got sick, he put together a business plan with a couple of partners, and built a company – Solid Ventures – to focus singularly on Duchenne muscular dystrophy.
“It’s a disease we know very, very well, and have a great motivation to address it because my son is sick,” Ganot said. “The idea is that maybe we can just make enough of a difference to justify the risk to our investors.”
Melding this personal drive to find a better treatment for the disease with an aligned business mission from industry and the right research from academics is the basic formula for success in venture philanthropy, the panelists said.
Campbell of the CFF said the concept of venture philanthropy came, after all, out of unmet need – back in 1998, there was a glaring gap in CF research and drug development, and the foundation set out to bridge it. That CFTR gene had been identified, “but no one was translating that information into new therapies that address the basic defect,” he said. “We were very, very frustrated.”
The CFF then built its therapeutics development program, which has since been the industry’s prime model for venture philanthropy. Campbell said of the CFF’s massive influx of new cash: “We’re going to plow it all back into patient care” – fund new therapies, help fund care centers and so forth.
There are some noticeable gaps – Kalydeco is criticized for being hugely expensive and the CFF played a passive role in that. Campbell said, when initially setting up the venture philanthropy infrastructure:
“We tried to have contractually a place at the table for pricing,” he said. “It was very clear that was a nonstarter – we would either have a program without a say in the price, or we would have to walk away.”
Now that it’s flush with cash, he said the CFF’s trying to spark a national debate on the balance in innovation and pricing and sustainable access for patients. And when it comes to butting heads with insurers – “we’re trying to sort out what value-based care would look like.”
At the end of the day, the model for success in venture philanthropy’s based on common goals and collaboration, Campbell said.
“When you have people who collaborate with fundraising or science, with passion that’s infectious, then your chances of success go up astronomically,” he said.
