Vision Medicines is working on developing two agents for the treatment of ocular diseases: VM100 for treatment of geographic atrophy and intermediate age-related macular degeneration (AMD), and VM200 for treatment of Stargardt disease.
VM100 is a monoclonal antibody whose mechanism of action is to clear retinal amyloid (A beta) and its complement. VM100 can be dosed systemically for treatment in both eyes simultaneously. Vision Medicines expects to initiate Phase II/III trials in 2016 and report out its concept data in geographic atrophy in 2017, President and CEO Chris Varma, PhD, told OIS@AAO.
A100 prevented deposition of A beta and its complement in a mouse model of AMD, preserving retinal structure and function, Dr. Varma said. He noted that Biogen has already validated A beta as a driver of Alzheimer’s disease and drusen, the latter of which has been shown to contain both toxic A beta and its complement and has been identified as a biomarker for progression to advanced AMD.
VM200 has the potential to be the first treatment for Stargardt disease, an orphan disease that causes blindness in about 100,000 people a year in the US,, Europe, and Japan. VM200 is an oral, small-molecule agent that works through an alkaline trap mechanism to neutralize toxins during the visual cycle and thereby prevent subsequent retinal damage and cell death, Dr. Varma said.
In a preclinical Stargardt disease mouse model, VM200 preserved both retinal structure and function in a dose-dependent manner without impairing the vitamin A visual cycle. VM200 has the potential to be used in all stages of Stargardt disease. Dr. Varma noted the Foundation Fighting Blindness committed up to $7.5 million to co-fund VM200 development for Stargardt disease, the organization’s largest co-funding agreement to date. Vision Medicines anticipates initiating Phase I/II trials of VM200 in 2016 with readout in 2018, he said.
Chris Varma, PhD
Chris co-founded Vision Medicines in May 2013. In September 2010, Chris co-founded Blueprint Medicines, a leading oncology therapeutics company located in Cambridge, MA. Chris served as Blueprint’s President, CE0, and Director from its founding in May 2013.