ProQR is licensing an RNA medicine from Ionis Pharmaceuticals for the treatment of retinitis pigmentosa, a form of rare, inherited blindness. The plan is to start a phase 1/2 trial of the drug in 2019.
Leiden, the Netherlands-based ProQR is handing over an upfront payment of $2.5 million in ordinary shares in exchange for the worldwide rights to IONIS-RHO-2.5Rx, now called QR-1123. The candidate is being developed specifically for autosomal dominant retinitis pigmentosa (adRP) caused by a P23H mutation in the rhodopsin gene. This gene codes for a protein of the same name that is found in the eye’s rod cells and are important for vision, especially in low light.
QR-1123 is an antisense oligonucleotide designed to target mRNA from faulty rhodopsin genes while leaving mRNA from healthy rhodopsin genes unharmed, said Brett Monia, Ph.D., chief operating officer and senior vice president of translational medicine at Ionis, in a statement.
“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,” Monia said.
ProQR has programs in multiple therapeutic areas, such as cystic fibrosis and the rare skin condition Dystrophic epidermolysis bullosa, but ophthalmology assets make up the bulk of its pipeline. Its lead asset is heading toward a registrational phase 2/3 trial in another inherited form of blindness.
LCA10 is a form of the eye disorder Leber congenital amaurosis that is caused by a p.Cys998X mutation in the CEP290 gene. There is no treatment for LCA10, though Spark Therapeutics markets Luxturna, a gene therapy for LCA2, which is caused by a different mutation. Editas Medicine is working on a CRISPR-based treatment for LCA10, but has had to delay its clinical trials due to manufacturing issues.
Under the deal, Ionis stands to collect an undisclosed sum in milestone payments, to be made in cash or equity. The company is also in line to receive 20% royalties on net sales.