Focal Medical, a startup with roots at UNC-Chapel Hill, is finalizing plans with the FDA to enroll pancreatic cancer patients in its first clinical trial. The company’s implantable technology uses an electrical field to deliver drugs in higher concentrations to hard-to-reach tumor cells.
When Tony Voiers walked into a conference room at the Food and Drug Administration (FDA) in early November 2016, he saw an imbalance. Voiers, an engineering-minded entrepreneur who had just begun consulting with UNC-affiliated startup Focal Medical months earlier, was in Washington, DC to meet with FDA officials about the company’s promising but nascent technology. Initial research indicated the technology could help shrink pancreatic cancer tumors at a scale never seen before. But for Voiers, the data point on his mind in that moment involved the number of seats, not cells. “We walked into the room, and the FDA had brought 21 people to the meeting, and there were just three of us,” he said.
Voiers wasn’t sure what kind of reaction the FDA would have, but he hoped the large contingent was a signal of the regulatory agency’s interest. “I was personally very interested in the data that Focal Medical had, but the technology was so new and innovative that I really didn’t know what the FDA would think of it—what clinical trials would look like for approval,” he said.
Voiers’ intuition was right. He said the meeting was “overwhelmingly positive,” recalling that the FDA “gave us very positive, tangible feedback on how we could move the technology forward.”
The technology is an implantable medical device that uses an electric field to precisely deliver drugs in higher concentrations to targeted hard-to-reach cells than is possible by pill or injection. It was developed via a collaboration between former UNC-Chapel Hill chemistry professor and serial entrepreneur Joseph DeSimone, PhD, and Jen Jen Yeh, MD, a distinguished cancer surgeon, who is a professor in the departments of surgery and pharmacology at the UNC School of Medicine.
The FDA meeting was a pivotal point for Focal Medical’s go-to-market path and Voiers’ confidence. “That was the point when I personally said to myself, ‘Yeah, I’m in this 100 percent. We’re going to do this.’” At the time, Voiers was winding down his leadership role with another startup and was looking for his next venture. A serendipitous lunch meeting months earlier in Chapel Hill—during which he was introduced to Yeh for the first time—initially captured his attention like no other opportunity had. Yeh, who is co-founder of Focal Medical and works as the director of the Pancreatic Cancer Center of Excellence at the UNC Lineberger Comprehensive Cancer Center, told Voiers about the impressive research results her lab received using the technology to target human pancreatic tumors implanted in mice.
“I had talked to seven university tech transfer offices, looking for a technology or venture that I thought was worth putting energy into. And when I saw the data that Jen Jen shared, I thought, ‘This is it,’” said Voiers, now Focal Medical’s chief operating officer. “That was the best data I’d ever seen from a lab in a research university because, most of the time, you stop research at a certain point. But Jen Jen and the UNC team had taken it so much further. The data was super compelling.”
As researchers, Yeh and DeSimone recognized the need for a technology that could deliver drugs to hard-to-reach places in the body. For example, Yeh says that when people swallow a pill or receive an injection, drugs enter the bloodstream, but some regions of the body have too few blood vessels to effectively deliver the drug. Physical barriers also create issues, as with pancreatic cancer. “For hard-to-treat diseases, you often can’t get your treatment of choice to the specific region because there are physical or other types of barriers,” said Yeh. “With pancreatic cancer, there is almost like a fortress around the tumor cells, so the vision for this device was to essentially collapse the fortress so that the drug can get to the tumor.”
The device was invented by Yeh, DeSimone and James Byrne, a former Carolina MD-PhD student whom Yeh and DeSimone co-mentored. Yeh said the technology, which is surgically implanted, uses an electrical field and works similarly to a magnet, in which the positive pole attracts the negative pole—a force of energy that is useful for directing drugs into tumors that are otherwise challenging to reach. “If we give the right drug the right charge, the device can essentially be used as a magnet to suck the drug into the right place,” said Yeh. She said targeted treatment is crucial because, even though the survival rate for pancreatic cancer doubled over the past decade, the odds still stand at only 12 percent.
After seeing drugs produce little-to-no shrinkage in pancreatic tumors via traditional methods—such as cultured tumor cell lines in the lab—Yeh, DeSimone and Byrne devised another way. They implanted human tumors, which had been donated to research by pancreatic cancer patients, into mice. Still, even when injected into the mice, the drugs weren’t effective—not until the team used its new technology.
“At that point, none of the tumors were shrinking—even in the mice–and we had not seen any response from the eight or nine drugs we tested,” said Yeh. “So, we decided to use the device on the mice, and there was near-complete shrinkage of tumors that we treated with the device compared to just a little bit of shrinkage without the device.”
Yeh said that the “shockingly good results” demonstrated about 150-percent shrinkage when using the device compared to no shrinkage with traditional injection. “None of us had ever consistently seen tumor shrinkage, much less to that extent in this model, and it was incredible,” she said.
Focal Medical is finalizing plans with the FDA and, by summer 2024, expects to enroll patients in its first clinical trial for treating pancreatic cancer. Yeh said the trials should offer hope.
“We know for many cancers, including pancreatic cancer, that one shot on goal isn’t enough: surgery isn’t enough, and our current chemotherapy isn’t enough. You have to tackle the tumor from many different directions,” said Yeh. “This technology is huge for patients, because it gives us ways to help weaken the entire tumor system so that we can eventually cure the cancer.”
Photo courtesy of UNC Lineberger Comprehensive Cancer Center
Pancreatic cancer is the first in a line of serious diseases that Yeh and Voiers believe the device can help treat, including genetic diseases and other forms of cancer. “If you have a genetic defect in an organ, like a heart defect or kidney defect, how do you get the drug to those places? If you have cancer in a hard-to-reach place and want to localize drug treatment to that area because systemic therapy would be toxic to the entire body, what can you do? Those are the kinds of instances in which we think this technology can help because it doesn’t use the bloodstream, but rather an electrical current to directly target the area.”
The Focal Medical team continues to explore future applications. “Because the device is drug agnostic, it broadens the ability to treat different diseases, whether those are tumors or other hard-to-treat diseases,” said Yeh. “There are many diseases where more hope is needed—we believe that this device can make an impact.”
Initial funding for Focal Medical came from angel investors who previously invested in other Carolina ventures. After Voiers joined, the company also applied for and received a federal Small Business Innovaiton Research (SBIR) Fast-Track Grant via the National Institutes for Health for approximately $2 million.
Voiers points to the SBIR Fast-Track Grant as a particularly important source of funding that set the stage for future success. “Early on, the award for the first SBIR grant got us off the ground because, immediately afterwards, we closed on angel money,” he said. “It wasn’t just that SBIR grant money itself that was so important. It had a multiplicative effect.”
Using its SBIR grant and angel funding as springboards, Focal Medical pivoted to secure more substantial funding via venture firms. In late 2020, the company closed an $11 million Series A round. Between grants and investments, it has raised approximately $20 million in funding.
Voiers encourages early-stage ventures to consider the angel investor route as a first step. “Unless you have an excellent track record, successful exits, and existing relationships with venture firms, most companies are too early for VC funding,” he said. “Our early funding approach was a little more than a friends-and-family round, and it targeted high-net-worth individuals who could write substantial checks. Targeting high-net-worth angel investors is a good strategy because these individuals are often associated with universities and can usually meet with you and make decisions quickly.”
From Voiers’ perspective, when it comes to finding angel investors, networking is paramount. “You have to talk to people who know the investors—your business attorney and accountant should be able to put you in touch,” he said. “If you’ve licensed technology out of a university, your tech transfer office knows these investors, and they have a vested interest in getting the company funded because it’s their intellectual property you’re working on. You have to leverage those networks to find investors.”
With only Voiers, Yeh and chief technology officer Bill Daunch involved on a routine basis in the early days, Focal Medical rented a small office in Raleigh with converted lab space. “The University was nearby, so we wanted to be in the area,” said Voiers. “But we didn’t have much money to put into one of the nicer spots, so we simply found whatever we could.”
Today, the company rents a larger 6,000 square-foot office space with upfitted labs in Cary. “We transitioned space because we raised a Series A, so we could hire people who needed research space,” said Voiers. “We succeeded in our fundraising, so we could hire people to execute on our vision.”
Like most startups, Focal Medical began with a streamlined staff—only Voiers and Daunch full-time—and found resourceful ways to maximize talent.
“It’s critical to have clinicians involved, so having Jen Jen as a founder was extremely important,” said Voiers. “And because we didn’t initially have money to hire employees, we leveraged our network of contacts as contractors or for favors.”
As the company raised money and hired staff, it focused on finding scientists with experience in cancer, genetic medicine or engineering—and particularly those with backgrounds in device development. “There is a really high skillset in those scientific disciplines in the RTP region, so we haven’t had to go outside the area to hire people,” said Voiers.
With limited ability to pay everyone working on behalf of Focal Medical, Voiers turned to an approach from prior ventures. “In all the smaller companies where I’ve worked, I was trained that, for something to be successful, everybody needs to have aligned incentives. So, even early on, if we couldn’t pay people, we would offer stock options in lieu of payment,” he said. “If everyone works toward the same company goal, that’s a powerful motivating force.”
Yeh says that while methodical information gathering can be ideal when learning to build a company, in the case of Focal Medical, “necessity was the mother of invention.” She and DeSimone founded the company a year before she met Voiers and the team met with the FDA. “It was just something we had to do, and so we did it,” said Yeh. “We founded the company with nobody in it, jumped in feet first and then figured out the rest on the backend. It was serendipity.”
Despite the learn-as-you-go effort, Yeh didn’t proceed in a vacuum. “As I learned the ropes, I reached out to KickStart Venture Services for advice, and they connected us to a network that was valuable in terms of learning,” she said. “I never thought I’d be an entrepreneur, so they were incredibly helpful.”
KickStart, which provides entrepreneurial education, early-stage funding, and on-campus accelerator space, is part of Innovate Carolina, UNC-Chapel Hill’s central team for innovation, entrepreneurship and economic development. Focal Medical also worked with Innovate Carolina’s Office of Technology Commercialization to protect the intellectual property that arose the technology’s underlying research. The company currently holds multiple patents issued on a global basis.
For Voiers, the entrepreneurial learning path involves investigating markets where Focal Medical may be poised to make an impact. He uses the library services at the North Carolina Biotechnology Center for market research, such as understanding the market forecast for drugs and identifying related companies. He also works with Innovate Carolina’s patent landscaping and market research team for in-depth market insights.
“Because we’re considering new markets, I pulled a research article on the different modes of energy-based drug delivery. I wanted to understand the strengths and weaknesses of each delivery mode, the patent landscape, and the different players.” he said. “So I contacted the patent landscaping and market research team, and they came back with a nice report. It’s a great resource.”
| UNC-CH