Foundation Fighting Blindness will provide up to $7.5 million in funding to develop ProQR’s candidate QR-421a for Usher syndrome type 2A 12
Foundation Fighting Blindness and ProQR have entered into a partnership to develop QR-421a for Usher syndrome type 2A, targeting mutations in exon 13 of the causative USH2A gene. Foundation Fighting Blindness will provide milestone-based co-funding of up to $7.5 million to ProQR to advance the program into the clinic. QR-421a received orphan drug designation from the FDA.
There are currently no therapies commercially available or in clinical development for the vision loss associated with Usher syndrome type 2A. QR-421a is part of ProQR’s growing ophthalmology pipeline which also includes lead candidate, QR-110 for Leber’s congenital amaurosis 10 currently in clinical trials, and three additional pipeline programs, QR-411 that addresses another genetic mutation resulting in Usher syndrome type 2A, QRX-1011 for Stargardt’s disease and QRX-504 for Fuchs endothelial corneal dystrophy. QR-421a is expected to advance towards the clinic in 2018, with clinical data expected in 2019.
Foundation Fighting Blindness’ Clinical Research Institute (FFB-CRI) has also launched a natural history study in 120 people with USH2A mutations. The study — known as RUSH2A (“R” stands for “rate of progression”) — was launched in 2017 and is being conducted at about 20 clinical sites around the world.
RUSH2A investigators will use a variety of technologies to monitor changes in vision and retinal structure to document and analyze disease progression. Knowledge and data obtained from this trial are intended to provide a better understanding of how USH2A mutations affect the severity and progression of vision loss and help to inform the development of QR-421a.
“Teaming with corporate partners to help promising therapies move through preclinical and clinical development is central to FFB’s strategy so we are very pleased to enter into this partnership with ProQR,” said Benjamin R. Yerxa, PhD, CEO at Foundation Fighting Blindness. “The fact that there are currently no available treatments for Usher syndrome type 2A makes this work that much more exciting and critical.” QR-421a for Usher syndrome is the second program in ProQR’s growing ophthalmology pipeline scheduled to enter clinical trials.
The lead program in the ophthalmology pipeline, QR-110, is currently in a Phase 1/2 safety and efficacy trial in adult and paediatric patients with Leber’s congenital amaurosis 10, due to the p.Cys998X mutation in the CEP290 gene. This pipeline also contains several other molecules for genetic eye diseases, including QR-411 for Usher syndrome type 2A due to the PE40 mutation, QRX-1011 for Stargardt’ disease and QRX-504 for Fuchs endothelial