ProQR Therapeutics announced that the FDA has cleared the investigational new drug (IND) application for QR-1123. ProQR plans to start enrolling patients in a phase 1/2 trial for QR-1123 in 2019.
QR-1123 is a first-in-class investigational oligonucleotide designed to address the underlying cause of the vision loss associated with autosomal dominant retinitis pigmentosa (adRP) due to the P23H mutation in the rhodopsin (RHO) gene.
P23H is the most prevalent mutation associated with adRP in the U.S. This disease causes progressive vision loss in approximately 2,500 patients in the United States, leading to blindness in mid-adulthood. There are no approved therapies for adRP and QR-1123 is the first investigational medicine to be developed for patients that suffer from this disease.
“We are pleased to have an open IND for QR-1123, based on which we will be advancing our next inherited retinal disease program into the clinic this year,” Daniel A. de Boer, Chief Executive Officer of ProQR, said in a company news release. “This represents our fifth IND in less than 5 years and our third clinical program for severe genetic eye diseases. With a strong in vitro and in vivo proof-of-concept, we are excited about the potential of this medicine to make a positive impact on the lives of patients with adRP.”
QR-1123 is a first-in-class investigational antisense oligonucleotide that was discovered and developed by Ionis Pharmaceuticals using Ionis’ proprietary antisense technology for the treatment of adRP due to the P23H mutation in the RHO gene. The therapy aims to inhibit the formation of the mutated toxic version of the rhodopsin protein by specifically binding the mutated RHO mRNA. Binding of QR-1123 causes allele specific knockdown of the mutant mRNA by a mechanism called RNase H mediated cleavage without affecting the normal RHO mRNA. QR-1123 is intended to be administered through intravitreal injections in the eye. QR-1123 was in-licensed from Ionis Pharmaceuticals in 2018.