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Phase I/II trial planned for Stargardt disease through collaboration between Foundation Fighting Blindness and Vision Medicine

Research into the development of therapies for Stargardt disease will receive a significant boost following announcement of the largest co-funding investment to date by the US Foundation Fighting Blindness (FFB). The Foundation will commit $7.5 million to co-fund the clinical development of a small molecule drug candidate (“VM200”), designed to neutralize the toxic build-up of vitamin A aldehyde, characteristic of early onset macular degeneration. The development programme will be led and co-funded by Boston-based “Vision Medicine”, a clinical-stage biopharmaceutical company with a focus on retinal degenerative disorders and orphan disease markets.


Vision Medicine separately announced the exclusive worldwide licensing of the VM200 opportunity from Case Western Reserve University, Ohio. The company’s scientific co-founder and Chair of the Scientific Advisory Board of Vision Medicine, Prof. Reza Dana, stated that, “dry age-related macular degeneration (AMD) and inherited retinal diseases, such as Stargardt disease, are areas of great unmet medical need. Through a very stringent diligence process that included world-class experts and involved vetting nearly 50 programs and technologies, we identified and in-licensed drug candidates that we believe have a very high probability of technical success and could be important new treatment options. Dry AMD and Stargardt disease can both lead to severe vision loss, and our goal is to advance innovative therapies for these patients and to help change their lives, and we feel confident that our programs provide such a path.”


The collaborative and financial support from Foundation Fighting Blindness follows an equally critical investment made by the society in charting the natural history of Stargardt disease, known as “ProgSTAR”. Securing a deeper understanding of the pathology and clinical progression of Stargardt is a significantly important part of the clinical development program as it is will ultimately provide key benchmarks of disease pathology that may be used to construct clinical trial outcome measures to be used in studies such as Vision Medicine’s VM200 trial. Stargardt disease is estimated to afflict approximately 100,000 individuals in the US, Europe and Japan and at present no treatments are available to tackle the disease. ProgSTAR has already succeeded in the the rapid enrollment of 250 patients, a number of which may be subsequently seek to participate in clinical trials of new therapeutic opportunities. Commenting on the licensing deal with Case Western, Dr. Chris Varma, PhD, Chairman and CEO of Vision Medicines commented that, “This is a fantastic opportunity to combine the world-class visual research of Krzysztof Palczewski (the scientific innovator who developed VM200) with the experienced team and ophthalmology expertise at Vision Medicines. We are enthusiastic about the potential for VM200 and have initiated investigational new drug application-enabling studies to advance VM200 into a Phase 1/2 program for Stargardt disease in 2016.”