Foundation Fighting Blindness and Vitrisa Therapeutics Collaborate in Developing Innovative Clinical Trial Designs for Stargardt Disease Treatments

Under a newly formed collaboration, the Foundation Fighting Blindness Clinical Research Institute (FFB-CRI) will provide functional and structural data from its ProgStar natural history study for people with Stargardt disease to Vitrisa Therapeutics. The collaboration will enable Vitrisa to use the anonymized data to evaluate and simulate innovative clinical trial designs for the treatment of Stargardt disease caused by mutations in the ABCA4 gene.

Data from ProgStar, the largest natural history study for Stargardt disease, is publicly available. Using ProgStar data, FFB-CRI and Vitrisa plan to develop and rigorously test innovative new study strategies using "virtual clinical trials." The results of this virtual approach may make it possible for companies developing therapies to implement clinical trials that could lead to the approval of therapies much more quickly than traditional approaches. As with all ProgStar-related collaborations, relevant findings will be publicly available.

Stargardt disease is the leading cause of inherited macular degeneration, affecting 30,000 people in the U.S. and tens of thousands more around the world. The condition is usually diagnosed in children or young adults and leads to devastating loss of central vision. A majority of cases are caused by mutations in ABCA4.

"FFB-CRI is excited to help Vitrisa in the innovative design of human studies for promising Stargardt disease treatments," said Benjamin Yerxa, PhD, FFB's chief executive officer. "We launched ProgStar with the goal of sharing robust patient data broadly with companies and researchers to advance the clinical development of emerging vision-saving therapies, and a number of companies have already engaged with us for collaborative support."

ProgStar assessed disease progression in more than 300 people with Stargardt disease for more than two years at nine clinical trial sites around the world. The study included prospective and retrospective analyses. The study's primary focus is to recommend efficacy measures -- for example, changes in vision and/or retinal structure -- that will be most efficient and reliable for evaluating potential therapies in clinical trials. The overall goal of the study is to advance therapy development by making disease information available to researchers and companies around the world. Thus far, 11 research papers on ProgStar have been published in peer-reviewed journals.

"We are delighted to be able to access the rigorous and comprehensive ProgStar study to better understand the progression of Stargardt disease, so that we can more effectively design and conduct clinical trials to bring therapeutics to those affected by this disease," said Carl Erickson, Vitrisa's chief executive officer. "FFB-CRI's visionary support for establishing a robust clinical data set around Stargardt disease, along with its strong clinical development expertise, make it possible for us to rigorously evaluate clinical trial design strategies that could significantly expedite the time to the first approved therapy for Stargardt disease. We look forward to disseminating the results of our analyses, in collaboration with FFB, and to working together to address the significant unmet medical needs of those affected by Stargardt disease and other inherited retinal disorders."

The Foundation Fighting Blindness was established in 1971. It has since raised more than $725 million for research aimed at preventing, treating and curing blindness caused by retinal degenerative diseases. More than 10 million Americans, and millions more worldwide, have vision loss due to retinal degeneration. Through its support of focused and innovative science, the Foundation drives the research that has and will continue to provide treatments and cures for people affected by retinitis pigmentosa, macular degeneration, Usher syndrome and other inherited retinal diseases. FFB-CRI was established in 2004 to advance translational research and clinical development of emerging therapies for inherited retinal diseases.

Vitrisa Therapeutics, Inc. is a privately held and venture-backed biotechnology company based in the San Francisco Bay Area. Vitrisa is focused on developing a suite of platform technologies optimized for ocular therapeutics and an associated pipeline of therapeutics to address significant unmet medical needs in sight-threatening ocular diseases.

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