ProQR In-licenses Worldwide Rights to Ophthalmology Drug Candidate from Ionis Pharmaceuticals

ProQR Therapeutics (Nasdaq:PRQR) a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, today announced the signing of an agreement with Ionis Pharmaceuticals to license QR-1123 (formerly “IONIS-RHO-2.5_Rx”), an RNA medicine for autosomal dominant retinitis pigmentosa (adRP) caused by the P23H mutation in the rhodopsin (RHO) gene.

As quoted in the press release:

“Building on the recent proof of concept with QR-110 in LCA10 patients, we have confidence in the potential of single stranded RNA-targeted therapies to treat patients with genetic eye diseases,” said Daniel A. de Boer, chief executive officer of ProQR. “By acquiring the rights to QR-1123, we have strategically expanded our pipeline in retinal diseases. Ionis has a wealth of experience in discovery and development of antisense drugs and we look forward to working with them on this important program. Unlike other molecules we are developing, QR-1123 is a gapmer with a mutant allele-specific knockdown mechanism of action. If validated, it would further broaden the potential of RNA-targeted therapies in retinal diseases. We are excited to start clinical development to explore QR-1123’s potential in helping patients with P23H adRP.”

Under the terms of the agreement, ProQR was granted an exclusive worldwide license to QR-1123 and relevant patents. 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.

Click here to read the full press release.