ProQR draws line under its eye disease era, offloading 2 assets to Théa for $14M upfront

ProQR Therapeutics has found a buyer for its ophthalmology assets. One year after putting the programs up for sale, the Dutch biotech has offloaded sepofarsen and ultevursen to Laboratoires Théa for 12.5 million euros ($13.7 million) upfront.

Last year, ProQR underwent a two-phase reorganization, first laying off 30% of its staff to focus on two areas and later pulling back from genetic eye diseases to go all in on its RNA-editing technology. The biotech began seeking partners for its genetic eye disease candidates but, with the retreat being sparked by a delay to a key program, it was unclear whether anyone would be willing to bankroll further development.

Théa has decided the programs are worth a punt. As well as the upfront fee, the French drugmaker is committing up to 135 million euros ($148 million) in development, regulatory and commercial payments for the rights to sepofarsen and ultevursen.

Sepofarsen, an RNA therapy, is designed to restore vision in people with the severe inherited retinal disease Leber congenital amaurosis 10. The candidate failed to improve vision in a phase 2/3 trial, and, while ProQR found an “encouraging efficacy signal” in a post hoc analysis, European regulators asked the biotech to run an additional pivotal study before seeking approval.

The regulatory feedback prompted ProQR to pivot to its RNA platform and stop trials of sepofarsen and ultevursen, an RNA therapy designed to treat vision loss in Usher syndrome type 2a and non-syndromic retinitis pigmentosa. ProQR began trials to study ultevursen in retinitis pigmentosa in 2021, but its pivot brought the program to a premature end.

Théa will need to rebuild momentum behind the stalled programs. Work is underway to establish a new organization to manage the two programs and dedicate a team specializing in inherited retinal disorders to their development.