- ProQR’s QRX-411 receives Orphan Drug Designation by the
FDAand EMA for the treatment of retinitis pigmentosa, including Usher syndrome, the subtype targeted by QRX-411. Usher syndrome is an inherited condition that is characterized by combined deafness and blindness.
- QRX-411 targets the pseudo-exon 40 (PE-40) mutation in the USH2A gene and currently there are no therapies commercially available or in clinical development for the vision loss associated with this disease.
- QRX-411 has shown promising preclinical data in both patient fibroblasts and the optic cup model for mRNA restoration, which was presented at the Annual Meeting of the
Association for Research in Vision and Ophthalmology(ARVO) in May 2017.
- A lead candidate has been selected for this program and is currently ready for IND-enabling studies.
- QRX-411 is part of ProQR’s ophthalmology pipeline that currently also includes one clinical compound, QR-110 for Leber’s Congenital Amaurosis Type 10, and three preclinical programs, QRX-421 for Usher syndrome, QRX-1011 for Stargardt’s disease and QRX-504 for Fuchs endothelial corneal dystrophy.
ODD in the
“We are pleased with the progress we have made to date with our novel RNA based therapeutic ophthalmology pipeline for patients suffering from genetic eye diseases. Securing orphan drug designations from the
Chief Development Strategy Officer,
ProQR’s growing ophthalmology portfolio includes:
- QR-110 for Leber’s congenital amaurosis Type 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 Orphan Drug designation by the FDAand EMA.
- QRX-411 for Usher syndrome type II 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-421 for Usher syndrome type II 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-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.
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 the disease. Usher syndrome Type II is one of the most common forms of Usher syndrome and is caused by mutations in the USH2A gene.
QRX-411 is a first-in-class RNA-based oligonucleotide designed to address the underlying cause of Usher syndrome due to the c.7595-2144A>G mutation in the USH2A gene. The mutation is a substitution of one nucleotide in the pre-mRNA that leads to aberrant splicing of the mRNA and non-functional or absence of USH2A protein. QRX-411 is designed to restore wild-type USH2A mRNA leading to the production of wild-type USH2A protein by binding the mutated pre-mRNA causing normal splicing of the pre-mRNA.
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-411 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 ophthalmology 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
Director, Investor Relations
T: +1 858 245 3983