- ProQR’s drug candidate QRX-421 for Usher syndrome receives orphan drug designation from the
FDAand EMA, representing the third candidate in the company’s ophthalmology pipeline and the fourth in the broader pipeline to receive ODD in the U.S. and EU.
- There are currently no therapies commercially available or in clinical development for the vision loss associated with Usher syndrome type 2.
- QR-421 is part of the company’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, QRX-411 that addresses another genetic mutation resulting in Usher syndrome, QRX-1011 for Stargardt’s disease, and QRX-504 for Fuchs endothelial corneal dystrophy.
- Promising QRX-421 pre-clinical data in both patient fibroblasts and the optic cup model for mRNA restoration were presented at the
May 2017Annual Meeting of the Association for Research in Vision and Ophthalmology(ARVO) in Baltimore, MD.
July 2017, QRX-411, ProQR’s second candidate for Usher syndrome received ODD from the FDAand EMA.
ODD in the U.S. and European Union provides a special status for investigational drugs being developed for rare diseases. The ODD programs offer development program tax benefits and a waiver of the NDA application user fee, as well as market exclusivity for up to seven years in the U.S. and ten years in the European Union following market approval.
“We are pleased to have ODD designation for both our programs targeting Usher syndrome in the U.S. and EU, representing yet another important milestone for our company and highlighting the unmet need for patients in this disease,” said
ProQR’s ophthalmology pipeline includes the following:
- QR-110 for Leber’s congenital amaurosis 10 (LCA 10) due to the p.Cys998X mutation, which received IND and CTA clearance and is in clinical development (PQ-110-001 Phase 1/2 safety and efficacy study). QR-110 was also granted Fast Track designation by the
FDAand ODD designation by the FDAand EMA.
- QRX-421 for Usher syndrome type 2 due to exon 13 mutations in the USH2A gene, for which a clinical candidate has been selected and is ready for IND enabling development studies.
- QRX-411 for Usher syndrome type 2 due to the PE-40 mutation in the USH2A gene, for which a clinical candidate has been selected and is ready for IND enabling development studies. QRX-411 also received ODD designation by the
- QRX-1011 for Stargardt’s disease due to c.5461-10T>C mutations in the ABCA4 gene, which is in optimization phase.
- QRX-504 for Fuchs endothelial corneal dystrophy (FECD), for which a clinical candidate has been selected and is ready for IND enabling development studies.
QRX-421 is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of Usher syndrome due to mutations in exon 13 of the USH2A gene. Mutations in this exon can cause loss of functional USH2A protein that causes the disease. QRX-421 is designed to exclude exon 13 from the mRNA (exon skipping) and produce truncated but functional USH2A protein, thereby modifying the underlying disease.
About Usher Syndrome
Usher syndrome is the leading cause of combined deafness and blindness. Patients with this syndrome generally progress to a stage in which they have very limited central vision and moderate to severe deafness. To date, there are no treatments approved or products in clinical development that treat the vision loss associated with Usher syndrome type 2. Usher syndrome type 2 is one of the most common forms of Usher syndrome and is caused by mutations in the USH2A gene.
This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “anticipate,” “believe,” “could,” “estimate,” “expect,” “goal,” “intend,” “look forward to”, “may,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” and similar expressions. Forward-looking statements are based on management’s beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, statements regarding QRX-421, QRX-411, QRX-504, QRX-1011 and QR-110 and the clinical development and therapeutic potential thereof, statements regarding orphan drug designation, including the intended benefits of such status, statements regarding our ongoing and planned discovery and development of product candidates and the timing thereof, including those in our opthalmology portfolio, and statements regarding our oligonucleotide drug discovery platform. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our clinical development activities, manufacturing processes and facilities, regulatory oversight, product commercialization, intellectual property claims, and the risks, uncertainties and other factors in our filings made with the
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