Usher Syndrome type 2

ProQR and Laboratoires Théa have finally reached a pact that fits both companies' needs. After an earlier version of the deal crumbled, ProQR has finally managed to sell off its ophthalmology assets to Laboratoires Théa, this time for €8 million ($8.6 million) with the potential to make up to €165 million ($177 million) in biobucks. Dutch biotech ProQR has been trying to offload RNA therapies sepofarsen and ultevursen for more than a year now, with hopes of turning its focus to targets for cholestatic and cardiovascular diseases. In August, the company seemed to have found a taker with eye care-focsed Théa. The two biotechs announced a deal in which Théa would pay €12.5 million ($13.7 million) upfront for the two assets, while also giving ProQR the chance to make up to €135 million ($148 million) in biobucks. The offer was short-lived though, with Théa canceling the pact at the end of September after ProQR failed to meet the conditions. The sale depended on Théa hiring several key ophthalmology personnel from ProQR to help build out a team specializing in inherited retinal disorders. However, some of the ProQR employees decided not to work at Théa, so the conditions weren’t met. Now, though, the two have closed on a revised agreement. ProQR plans to ask for confidential treatment from the Securities and Exchange Commission for certain parts of the amended deal, according to SEC documents. Théa now owns the rights to sepofarsen, which is designed to restore vision in people with the severe inherited retinal disease Leber congenital amaurosis 10, and ultevursen, a therapy to treat vision loss in Usher syndrome type 2a and non-syndromic retinitis pigmentosa. Sepofarsen previously failed to improve vision in a phase 2/3 trial and European regulators had asked for an additional pivotal study before ProQR could seek approval. The regulatory feedback prompted ProQR to halt trials of both sepofarsen and ultevursen, followed by a restructuring that included a 30% workforce reduction and eventually, a shift away from genetic eye diseases.

It’s unclear if ProQR will continue to try to sell off its eye disease candidates. In a reversal of fortunes, ProQR Therapeutics’ ophthalmology asset sale to Laboratoires Théa has been cancelled after ProQR failed to meet the conditions of the deal.  In August, Dutch biotech ProQR announced its plan to offload RNA therapies sepofarsen and ultevursen to eye care company Théa for 12.5 million euros ($13.7 million) upfront and up to 135 million euros ($141.9 million) in biobucks. ProQR had initially put the two eye disease candidates up for sale about a year ago. Sepofarsen 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 European regulators asked the biotech to run an additional pivotal study before seeking approval. The regulatory feedback prompted ProQR to halt trials of sepofarsen and ultevursen, a therapy designed to treat vision loss in Usher syndrome type 2a and non-syndromic retinitis pigmentosa. A two-phase reorganization saw ProQR lay off 30% of staff and later pull back from genetic eye diseases to go all in on its RNA-editing tech. However, Théa has now terminated its purchase of the eye disease candidates, according to a Sept. 27 release from ProQR. The deal had been expected to close this quarter. The sale depended on Théa hiring several key ophthalmology personnel from ProQR to help build out a team specializing in inherited retinal disorders. However, some of the ProQR employees decided not to work at Théa, so the conditions weren’t met. It’s unclear if ProQR will continue to try to sell the eye disease candidates, though the company’s focus is currently on targets for cholestatic and cardiovascular diseases, alongside its genetic medicine partnership with Eli Lilly, ProQR founder and CEO Daniel de Boer said in the release.

Théa will need to rebuild momentum behind the stalled programs 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.