ProQR receives exclusive worldwide license for IONIS-RHO-2.5Rx, now QR-1123, for autosomal dominant retinitis pigmentosa (adRP), a rare inherited form of blindness with no approved therapy
A first in human Phase 1/2 clinical trial in adRP patients is expected to start in 2019
ProQR Therapeutics is a company dedicated to the creation of transformative RNA medicines for the treatment of severe genetic rare diseases and have announced the signing of an agreement with Ionis Pharmaceuticals to license QR-1123 (formerly “IONIS-RHO-2.5Rx”), an RNA medicine for autosomal dominant retinitis pigmentosa (adRP) caused by the P23H mutation in the rhodopsin (RHO) gene.
“Building on the recent proof of concept with QR-110 in LCA10 patients, we have confidence in the potential of single stranded RNA-targeted therapies to treat patients with genetic eye diseases,” said Daniel A. de Boer, chief executive officer of ProQR. “By acquiring the rights to QR-1123, we have strategically expanded our pipeline in retinal diseases. Ionis has a wealth of experience in discovery and development of antisense drugs and we look forward to working with them on this important program. Unlike other molecules we are developing, QR-1123 is a gapmer with a mutant allele-specific knockdown mechanism of action. If validated, it would further broaden the potential of RNA-targeted therapies in retinal diseases. We are excited to start clinical development to explore QR-1123’s potential in helping patients with P23H adRP.”
Preclinical activities have been completed by Ionis and were presented at the 2015 annual meeting of the Association for Research in Vision and Ophthalmology (ARVO) and published in a peer-reviewed article, Murray et al 2015, IOVS. A natural history study in patients with P23H adRP has been conducted. The results of this study provide valuable information on disease progression in these patients, which is important data for designing the clinical development plan. ProQR expects to start a Phase 1/2 clinical trial in patients with adRP in 2019, pending submission and clearance of the IND application by the U.S. Food and Drug Administration (FDA).
Brett P. Monia, Ph.D., chief operating officer and senior vice president of translational medicine at Ionis, added, “IONIS-RHO-2.5Rx, now QR-1123, is an antisense oligonucleotide we designed to specifically target only the mRNA from the disease-causing rhodopsin gene, which has a single nucleotide P23H mutation, while preserving expression of the mRNA from the normal rhodopsin gene, which is important for the eye to function properly. IONIS-RHO-2.5Rx was developed by Ionis scientists dedicated to discovering innovative therapeutics to fight diseases where no other treatments have proven effective or even existed. We believe that ProQR has the ophthalmology expertise and experience in developing oligonucleotide drugs for the eye to rapidly bring this program through clinical development and ultimately to patients.”