Investing in Cures Summit Highlights the Intersection of Science and Business in Advancement of Retinal Disease Therapies
The online event hosted by the Foundation Fighting Blindness and RD Fund included lab tours, researcher and executive interviews, and business and science stakeholder panels.
The most compelling news coming out of the Investing in Cures Summit (April 12–17, 2021) was the sheer number of therapies for retinal degenerative diseases that are now in, or poised for, clinical trials and have gained commercial support. More than 40 emerging treatments are in human studies with dozens more at the clinical trial doorstep.
As Adrienne Graves, PhD, said during the closing session, “We are truly in a retinal renaissance.” Graves is a former chief executive officer in the pharmaceutical industry and board director of the Foundation Fighting Blindness and RD Fund. With Warren Thaler as its chair, the RD Fund is a venture philanthropy fund with more than $70 million in investments for 10 projects.
Graves remarked that hope for retinal disease patients was hard to come by for many decades. She recalled, early in her career at Boston Children’s Hospital, the dread she felt when administering electroretinogram (ERG) tests to measure retinal sensitivity in young patients. “What I remember most vividly was praying. When I would flash a light to record their ERGs, I would pray for normal responses because that’s all I could do. I knew that an abnormal ERG would lead to an ever-darkening world for that child,” she said. “But now, the scientific advances have been transformative.”
Iveric Bio is a prime example of this transformation. The company is actively engaged in moving treatment advances out of academic labs into clinical trials. At the Summit, Glenn Sblendorio, Iveric’s chief executive officer, reviewed the company’s portfolio which includes Zimura, a promising C5 complement inhibitor in clinical trials for Stargardt disease and dry age-related macular degeneration (AMD), and five preclinical gene therapy projects underway for: retinitis pigmentosa (RP-RHO), Best disease (BEST1), Stargardt disease (ABCA4), Leber congenital amaurosis (LCA-CEP290), and RP and Usher syndrome type 2A (USH2A).
Some of the other Summit presenters involved in clinical therapy development included: Steve Pakola, MD, chief medical officer at REGENXBIO, which is planning a Phase 3 trial for its wet AMD gene therapy; Stéphane Boissel, chief executive officer at SparingVision, which is planning clinical trials of its gene-agnostic gene therapy for RP in late 2021; and Kapil Bharti, PhD, at the National Eye Institute, which is conducting a Phase 1/2 clinical trial of patient-derived retinal pigment epithelial cells for treating dry AMD.
But as attendees learned from The Ecosystem panel on the final day, therapy developers have many hurdles to clear to get their product candidate to a clinical trial, and some of those hurdles may not be as obvious or as well appreciated as, for example, confirming safety and efficacy in animal models. For example, Tarran Pierfelice, PhD, MBA, business head at the biopharmaceutical manufacturer Resilience, said that many companies need to spend more time and investment in therapy manufacturing to increase yields, improve quality, and reduce the production ramp-up.
Of course, fundraising for new therapies is often a significant challenge, but Kevin Bitterman, PhD, MBA, partner at the venture capital firm, Atlas Venture, explained that he and his partners are making investments in high risk ventures that are backed by good science, a good management team, and show potential for high return. He cited his firm’s and the RD Fund’s investment in the company Vedere, which is developing an optogenetic therapy to potentially restore vision for people who are completely blind, as an example. Vedere, which means “to see” in Italian, was acquired by Novartis in October 2020 for approximately $280 million, and the team continues to work on next generation products.
“The Ecosystem panel underscored well the need to bring many resources and stakeholders together to get a therapy into and through a clinical trial. We have to be running on all cylinders. Yes, time is always of the essence, but we also have to do it right,” said Ben Yerxa, PhD, chief executive officer at the Foundation, who hosted The Ecosystem panel along with Eugene De Juan, Jr., MD, vice chairman at Forsight Labs and board director of RD Fund.
Ecosystem panelists also included: Richard Awdeh, MD, chief executive officer at CheckedUp; Stéphane Boissel, chief executive officer at SparingVision; Wiley Chambers, MD, director, Division of Ophthalmology, Office of Specialty Medicine, Office of New Drugs, at the FDA; Laura Manfre, co-founder at Sofia Sees Hope; and Steve Pakola, MD, chief medical officer at REGENXBIO.
Saturday’s Art of Translation panel, moderated by Chad Jackson, PhD, director of the Foundation’s preclinical translational research program, also provided insightful perspectives on the many challenges of moving emerging therapies out of labs and into clinical trials. Panelists for the session included: Ilyas Washington, PhD, co-founder at Alkeus Pharmaceuticals; Christine Monville, PhD, professor, Evry’s University, Mark Vézina, director of the Ocular and Neuroscience Department, Charles River; and Rob Collin, PhD, professor, Radboud University Medical Center.
The Investing in Cures Summit opened with compelling patient perspectives on blindness and the hope that research is bringing. Evan Mittman, a Foundation board director who started a fashion business that grew into a $150 million company, recalled the many challenges he had as a young man, including those on the dating scene. “Dating was interesting. There I am, a single guy, trying to date, go to a restaurant, and I can’t read the menu. So, what did I do? I asked the waiter about the specials. And I ate them, whether I liked them or not. And it worked out fine. You got to do what you got to do in this world.”
On a more serious and reflective note, Mittman said that he has been proud to be a part of the Foundation since 1972. “The Foundation started as a small company to become the center of the world for curing blindness,” he said. “This week is one of the most exciting weeks there is, because we’ve gone through the desert for 50 years, but we’ve come to a point where we actually see water. The whole team – the FDA, patients, donors, researchers, the clinicians, pharma, the insurers – needs to come together now to make it happen.”
