ProQR in licence deal with Ionis Pharmaceuticals

Currently, there is no approved therapy for the rare heritable defect in the rhodopsin (RHO) gene that causes blindness in-mid adulthood and affects 2,500 people in the US. ProQR announced it expects to start human testing with the licenced antisense RNA molecule, which knocks down the mutant allele with the P23H point mutation, in patients with adRP, next year. The gain of function mutation point mutation, which causes misfolding of the rhodopsin protein and subsequent death of photoreceptor cells in the retina, accounts for approximately 25 percent of all adRP cases.

ProQR paid $2.5m upfront in shares to acquire the exclusive worldwide license to QR-1123 and associated intellectual property. Under the terms of the agreement, Ionis Pharma will also receive milestone payments, either in equity or in cash, and royalties on net sales of 20%.

The drug candidate, which was originally developed in a collaboration of Isis Pharmaceuticals and GlaxoSmithKline was well-tolerated in a 10-week monkey study. Preclinical activities have been completed. ProQR will submit an IND to conduct Phase 1/2 clinical trial to the FDA..