Laboratoires Théa Acquires ProQR Therapeutics' Sepofarsen and Ultevursen

ProQR Therapeutics and Laboratoires Théa announced an agreement in which ProQR will divest its late stage ophthalmic assets, sepofarsen and ultevursen, to Théa.

Under the terms of the agreement, ProQR will receive an initial payment of €12.5 million ($13.7 million) and will also be eligible for up to €135 million ($148 million) in further development, regulatory, and commercial payments, as well as additional earn outs up to high teens percentage based on commercial sales in the US and EU.

“Théa’s proven expertise in the research, development, and commercialization of eye care products makes them the ideal company to continue the development of sepofarsen and ultevursen for patients with rare genetic eye diseases,” Daniel A. de Boer, Founder and Chief Executive Officer of ProQR, said in a company news release. “We look forward to continuing to advance our Axiomer RNA editing platform, with an initial focus on targets for cholestatic and cardiovascular diseases, as we seek to develop a new class of therapies for patients with high unmet need.”

Sepofarsen (QR-110) is an investigational RNA therapy designed to restore vision in Leber congenital amaurosis 10 due to the c.2991+1655A>G mutation (p.Cys998X) in the CEP290 gene. It is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the US and the EU and received fast-track designation and rare pediatric disease designation from the FDA as well as access to the PRIME scheme by the EMA.

Ultevursen (formerly QR-421a) is a first-in-class investigational RNA therapy designed to address the underlying cause of vision loss in Usher syndrome type 2a and non-syndromic retinitis pigmentosa due to mutations in exon 13 of the USH2A gene. It is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the US and the European Union and received fast-track and rare pediatric disease designations from the FDA.

Within Théa, a team specializing in inherited retinal disorders and a new organization are currently being set up to manage these two projects, according to Théa. More information on the next steps for these programs will be available in the coming weeks.

The transaction is expected to close in the third quarter of 2023, subject to the satisfaction of certain closing conditions.