ProQR Therapeutics has announced plans to conduct a Phase I/II clinical trial to evaluate QR-1123 for the treatment of patients with autosomal dominant retinitis pigmentosa (adRP).
The announcement comes after the company received US Food and Drug Administration (FDA) clearance for its investigational new drug (IND) application.
adRP is a rare genetic disorder characterised by a progressive decrease in night and peripheral vision during childhood. The disease usually results in blindness in mid-adulthood.
QR-1123 is an investigational oligonucleotide intended to target the underlying cause of the vision loss caused because of the P23H mutation in the rhodopsin (RHO) gene.
P23H is known to be the most prevalent mutation in adRP patients in the US.
Ionis Pharmaceuticals discovered and developed QR-1123 to block the formation of the mutated rhodopsin protein by selectively binding the mutated RHO mRNA.
ProQR Therapeutics licensed the drug candidate last year.
Set to be initiated by the end of this year, the Phase I/II study will assess the safety, tolerability, pharmacokinetics, and efficacy of QR-1123 in up to 12 adult patients at sites in North America.
The trial will involve an open-label, single-dose escalation arm and a double-masked, multiple-dose arm. Participants will receive a single intravitreal injection of QR-1123 or sham procedure in a single eye.
During the study, restoration or improvement of visual function and retinal structure will be measured by ophthalmic endpoints such as visual acuity (BCVA), along with changes in quality of life.
ProQR Therapeutics CEO Daniel de Boer said: “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.
“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.”
Subjects who complete the Phase I/II study may be able to enrol into an extension study.