People lose their vision most often because, simply speaking, they grow old. But in rare cases, a genetic defect leads to a blindness in children and teenagers called Stargardt disease.
As with many rare diseases, there are no effective treatments for Stargardt. The Boston startup Vision Medicines hopes to change that.
There’s a long way to go for Vision to successfully treat Stargardt. Its experimental drug for the condition, which affects about 100,000 folks in the U.S., Europe and Japan, hasn’t been tested yet in humans. But today Vision announced it has raised $7.5 million from the non-profit Foundation Fighting Blindness, enough cash to reach a first trial. It’s the largest funding in the foundation’s history, and Vision is matching that sum to co-develop VM200, its proposed drug for Stargardt.
Vision was formed two years ago by Chris Varma (pictured above), a veteran of the Boston biotech startup scene. Varma had stints at Flagship Ventures (as a partner) and Third Rock Ventures (entrepreneur-in-residence), and has helped form companies like Warp Drive Bio, Blueprint Medicines (NASDAQ: BPMC), and Eleven Biotherapeutics (NASDAQ: EBIO). He was most recently Blueprint’s founding CEO, and led the company from its 2010 inception through May 2013. Blueprint went public earlier this year, well after Varma’s departure.
These companies all started up with the backing of well-known Boston biotech venture groups. But Varma says he is trying something different with Vision.
He’s not trying to raise big rounds from VC firms and eventually take Vision public. Rather, he says, the goal is to “grow through mergers.” That is, to acquire assets for eye diseases with few, if any effective treatments, take them forward, and hopefully at some point merge with another eye drug company.
Varma is vetting these assets with the help of a number of experts in the eye drug field, including Vision co-founder Reza Dana, a Harvard Medical School ophthalmology professor who chairs the company’s scientific advisory board.
So far, for instance, Vision has picked up two drugs. One is VM200 for Stargardt, which Vision has licensed from Cleveland’s Case Western Reserve University.
The second, VM100, was being developed by an unnamed large pharma company for the “dry” form of age-related macular degeneration. Varma won’t say much else about the drug, other than it was being discarded for “strategic reasons” and Vision now wants to carry it forward.
“We certainly could have just licensed one asset and said, ‘OK, let’s do a venture round and just focus on that,’ but that wasn’t really something that got our juices flowing,” Varma says. “[We wanted to] build something more substantial more quickly.”
Varma helped seed Vision with Dana and the other members of the company’s team, and gravitated towards AMD and Stargardt for a personal reason—his mother was diagnosed with dry AMD in 2013. He won’t say how much has gone into the company so far, just that it’s nowhere near the type of big Series A rounds that his former employers Third Rock and Flagship typically deploy to start a company. (Flagship, for instance, started Evelo Therapeutics last week with a $35 million round.) “It’s a pretty tight group,” he says of Vision’s founding team.
For this all to work, however, Vision has to make headway in clinical trials, and that starts with Stargardt. The disease is caused by a mutation to the gene called ABCA4, which helps transport food and waste to and from the eye’s photoreceptor cells. When ABCA4 doesn’t function properly, tiny pieces of waste known as lipofuscin build up in the retina, blocking vision and other cellular functions, like clumps of dirt on your windshield blocking the view and fouling the wipers.
Diagnosed in children and teenagers, Stargardt virtually always causes patients to become legally blind. There are no approved treatments, though there are others in development, such as an experimental gene therapy being developed by Sanofi and its U.K.-based partner, Oxford Biomedica, and a stem cell approach from Ocata Therapeutics (formerly Advanced Cell Technology).
A gene therapy and a stem cell therapy, if effective, would represent a long-lasting fix. Vision is offering a different solution: a pill meant to be taken chronically. Varma says VM200 is a small molecule that is supposed to bind to and neutralize the toxic proteins that build up in the retina, with the goal of slowing or halting the march to blindness. Though he won’t say what type of compound the drug is, Varma likens its mechanism to how antacids counteract acids in the stomach. “It’s a very elegant and simple mechanism,” he says.
The first study will be a Phase 1/2 trial that is expected to begin next year.