ProQR Therapeutics N.V. and Laboratoires Théa have agreed to a deal in which Théa will receive two late-stage ophthalmic assets from ProQR: sepofarsen and ultevursen.
Refresh me on the companies.
Théa is a European pharmaceutical company specializing in the research, development, and commercialization of ophthalmic products available in 75 countries.
Based in the Netherlands, ProQR is a Dutch biotechnology company focused on developing RNA-based therapies for the treatment of rare genetic diseases—including inherited eye diseases (IRDs).
Now these products .. starting with sepofarsen.
Sepofarsen (QR-110) is an investigational RNA therapy that is designed to potentially restore vision in Leber congenital amaurosis 10 (LCA10) due to mutations in the CEP290 gene (which results in mRNA and non-functional CEP290 protein abnormal splicing).
How does it work?
The intravitreally-injected therapy is designed to allow for normal splicing, leading to the possible restoration of normal CEP290 mRNA and production of functional CEP290 protein, according to ProQR.
To note, sepofarsen has received orphan drug designation (ODD) in the European Union and United States, as well as fast-track designation and rare pediatric disease designation from the FDA.
Now ultevursen.
Formerly QR-421a, ulevursen is a first-in-class investigational RNA therapy that targets the underlying cause for vision loss in Usher syndrome type2a (USH2A) and non-syndromic retinitis pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.
And it works by … ?
Also administered via intravitreal injection, the therapy works to restore functional usherin protein (using the exon skipping approach) and, as a result, might stop or reverse vision loss.
Similar to sepofarsen, ulevursen has been granted ODD in the E.U. and U.S., as well as fast track and rare pediatric disease designations from the FDA.
Gotcha. Talk about this agreement.
Per the deal, ProQR will receive an initial payment of €12.5M (estimated US $13.7M), up to €135M (estimated US $148.47M) in further payments, and potential earn outs based on US and EU commercial sales.
In exchange, Théa will gain development, regulatory, and commercial rights for both products.
How is Théa preparing for these products?
According to the company, a team specializing in IRDs will be formed and a new organization will manage the two projects (details coming soon!).
When will the deal close?
Per ProQR, the transaction is slated to close in Q3 2023 (pending certain closing conditions).
And the bigger picture?
This acquisition comes a year after ProQR originally put the ophthalmology assets up for sale, officially freeing the company to focus more on its proprietary Axiomer RNA-editing technology platform and advancement of pipeline programs for genetic diseases originating in the liver.