Type of information: Licensing agreement
Compound: QR-1123 (formerly “IONIS-RHO-2.5Rx”)
Company: Ionis Pharmaceuticals (USA - CA) ProQR Therapeutics (The Netherlands)
Therapeutic area: Rare diseases - Genetic diseases - Ophtalmological diseases
Type agreement: licensing
- antisense oligonucleotide/gene therapy. QR-1123 is a first-in-class investigational oligonucleotide (gapmer) that was 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 mutated 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.
Disease: autosomal dominant retinitis pigmentosa caused by the P23H mutation in the rhodopsin (RHO) gene (P23H adRP)
- • On October 29, 2018, ProQR Therapeutics 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.
- Under the terms of the agreement, ProQR was granted an exclusive worldwide license to QR-1123 and relevant patents.
- 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 FDA.
- ProQR made an upfront payment in ordinary shares in the aggregate amount of $2.5 million, at $22.23 per share, which represents a 20% premium (based on the volume weighted average price of the previous 20 trading days) to its common stock, to Ionis upon signing the agreement. ProQR will also make future milestone payments, certain of which will be made in equity and others in cash or equity at ProQR’s discretion, and royalties on net sales of 20% through the royalty term.