ProQR Therapeutics N.V. (NASDAQ: PRQR) and the US charity Foundation Fighting Blindness (FFB) have announced a new partnership to accelerate the development of a novel therapeutic, “QR-421a”, for the treatment of Usher syndrome 2A. The sensorineural disease, which affects approximately 16,000 patients worldwide, is caused by a mutation in exon 13 of the causative USH2A gene, leading to combined deafness and blindness. The Dutch company’s therapeutic, QR-421a, is a first-in-class RNA-based oligonucleotide designed to repair the genetic defect in the USH2A gene by excluding exon 13, leading to translation of a shortened but functional usherin protein. The novel intervention, invented by Dutch researcher, Dr. Erwin van Wyck, is understood to have been in-licensed by ProQR from Radboud University Medical Center in the Netherlands. QR-421a has received orphan drug designation from the U.S. Food and Drug Administration.
The agreement between ProQR and FFB sees the US charity providing up to $7.5 million in research funding to support both preclinical and clinical studies of the promosing RNA based therapeutic. According to FFB, the experimental treatment is expected to advance towards clinical trial studies in FY2018 with preliminary saftety and efficacy results expected from a Phase I/II study in FY2019. In addition to the direct support to progress the R&D of QR-421a, the FFB’s Clinical Research Institute (“FFB-CRI”) has also launched a natural history study of patients with USH2A mutations. The study of approximately 120 participants, entitled RUSH2A (where “R” stands for “rate of progression”), was formally initiated in 2017 across an estimated 20 international clinical sites. Aside from collating fundamental pathophysiological information on the disease, the natural history data will assist in determining the baseline against which efficacy of treatments such as QR-421a may be measured.
According to ProQR, QR-421a is part of an expanding ophthalmology pipeline which also includes a potential treatment for Leber’s congenital amaurosis 10 (QR-110), currently in clinical trials, and three further experimental treatments, QR-411 for a different mutation causing Usher syndrome type 2A, QRX-1011 for Stargardt’s disease and QRX-504 for Fuchs endothelial corneal dystrophy. Commenting on the partnership, Benjamin R. Yerxa, PhD, CEO at Foundation Fighting Blindness stated, “[t]eaming 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. The fact that there are currently no available treatments for Usher syndrome type 2A makes this work that much more exciting and critical.” In addition, Daniel A. de Boer, CEO of ProQR was equally enthusiastic for the potential work ahead stating, “[w]e are excited to team up with the Foundation Fighting Blindness to develop QR-421a for patients that suffer from Usher syndrome due to exon 13 mutations. They are the leading private funder of retinal disease research with a very patient centric approach which is a core pillar of our strategy. Through this partnership with the Foundation we plan to gain access to important know-how to develop programs in retinal diseases. We expect that the additional funding will allow us to rapidly advance this novel therapy for this orphan disease with a severe unmet need.”