ProQR Sells Two Ophthalmic Assets to Laboratoires Théa for Up to $162 Million

Rare Daily Staff

ProQR Therapeutics and the leading independent eye care group in Europe, Laboratoires Théa, signed 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 $13.7 million (€12.5 million) and will also be eligible for up to $147.5 million (€135 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 U.S. 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,” said Daniel de Boer, founder and CEO of ProQR.

ProQR’s next-generation RNA technology called Axiomer uses a cell’s own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need.

Within Théa, a fully dedicated team specializing in inherited retinal disorders and a new organization are currently being set up to manage these two projects.

Sepofarsen 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. The mutation leads to aberrant splicing of the mRNA and non-functional CEP290 protein. Sepofarsen is designed to enable normal splicing, resulting in restoration of normal (wild type) CEP290 mRNA and subsequent production of functional CEP290 protein. Sepofarsen is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the United States and the European Union 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 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. QR-421a is designed to restore functional usherin protein by using an exon skipping approach with the aim to stop or reverse vision loss in patients. Ultevursen 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.

“These two programs can deliver hope for patients suffering from retinal diseases that lead to blindness,” said Jean-Frédéric Chibret, president of the Théa group.

Photo: Daniel de Boer, founder and CEO of ProQR