Foundation Fighting Blindness Partners with Vision Medicines to Develop Stargardt Disease Therapy

November 10, 2015

The Foundation Fighting Blindness and the biopharmaceutical company Vision Medicines are partnering to develop a drug known as VM200 for preserving vision in people with Stargardt disease, the leading cause of inherited juvenile macular degeneration. The Foundation is committing $7.5 million to co-fund VM200 development. Vision Medicines plans to launch a human study of the emerging therapy in 2016.

“VM200 neutralizes toxic chemicals in the retina that are responsible for progressive retinal damage in Stargardt disease,” explains Robert Kim, M.D., chief medical officer and head of research and development at Vision Medicines. “It is delivered orally and has the potential to treat both eyes simultaneously in a convenient, non-invasive manner. We believe VM200 has the potential to help prevent Stargardt patients from becoming legally blind.”

“We are delighted to join with Vision Medicines to advance this promising therapy into a clinical trial,” says Patricia Zilliox, Ph.D., chief drug development officer at the Foundation’s Clinical Research Institute. “The collaboration brings significant clinical and drug development expertise to the project, which gives us the best opportunity to move VM200 out to the people who need it.”

There are currently no therapies for Stargardt disease, which affects approximately 100,000 people in the United States, Europe and Japan. The condition is usually diagnosed in childhood or adolescence. The hallmark of Stargardt disease is the buildup of toxic waste products in a layer of supportive cells known as the retinal pigment epithelium which usually leads to severe, progressive central vision loss.

ProgSTAR, the $5 million, 250-participant, natural-history study for Stargardt disease funded by the Foundation, could bring two important benefits to the launch and design of the VM200 clinical trial. First, it could identify participants for the human study. Second, the study could lead to development of an outcome measure to quickly and effectively help researchers determine if the treatment is saving vision.

“The Foundation’s collaboration with Vision Medicines is a major step forward in finding a vision-saving treatment for Stargardt disease,” says William T. Schmidt, chief executive officer, Foundation Fighting Blindness. “Along with our ProgSTAR natural history, we are in a good position to get a clinical trial for VM200 launched soon.”