RARE Daily

ProQR and EB Research Form Drug Company

March 27, 2019

Dutch biotech ProQR and EB Research Partnership, the largest global non-profit focused on treating and curing dystrophic epidermolysis bullosa, have formed a new company dubbed Wings Therapeutics to develop treatment for the rare skin disorder.

Dystophic epidermolysis bullosa (DEB) is one of the most severe forms of epidermolysis bullosa, a group of rare genetic skin diseases. DEB is caused by a mutation in the COL7A1 gene which is responsible for the formation of the collagen type C7 protein that anchors fibrils that bind the inner and outer skin layers together. This mutation causes a loss of the anchoring fibrils resulting in fragile skin. People with DEB live with constant pain and have a high risk of malnutrition and infections. Symptoms include poorly healing wounds, skin infections, fusion of fingers and toes, anemia, gastrointestinal tract problems and with adulthood some develop very aggressive forms of skin cancer. There are no approved treatment options available that target the underlying cause of DEB.

ProQR contributed its DEB activities while EB Research formed and financed the company, which will be led by interim CEO Mark de Souza, former CEO of Lotus Tissue Repair, and Hal Landy, former medical advisor to Lotus Tissue Repair and CMO of Enobia.

ProQR has a minority stake in Wings Therapeutics and will be eligible for milestone and royalty rights to commercial products. Financial details were not disclosed.

“We are very pleased with this strategic transaction. In partnership with Mark and Hal, we are able to focus Wings Therapeutics solely on exon skipping therapy for treating EB,” said Alex Silver, chairman at EBRP. “We are grateful for ProQR’s work to develop exon skipping drugs for DEB, their continued partnership to advance QR-313 through clinical trials, and we look forward to continuing on this path.”

Based in Berkeley, California, Wings Therapeutics will focus on developing therapies for dystrophic epidermolysis bullosa and continue to conduct clinical trials with QR-313 in exon 73 as well as progress other RNA molecules that are designed for other mutations that cause DEB.

ProQR’s QR-313 is a potential first-in-class RNA-based oligonucleotide designed to address the underlying cause of dystrophic epidermolysis bullosa (DEB) due to mutations in exon 73 of the COL7A1 gene. QR-313 is designed to exclude exon 73 from the mRNA (exon skipping) and produce a functional C7 protein, thereby restoring functionality of the anchoring fibrils.

After an interim analysis and strategic review, PRoQR management decided to focus on its ophthalmology programs and to transfer the conduct and completion of the ongoing phase 1/2 study of QR-313 to Wings Therapeutics. The study will remain blinded and continues to enroll patients. ProQR will work closely with Wings Therapeutics and EB Research Partnership to support its efforts to advance QR-313 for patients with DEB.

Photo: Alex Silver, Chairman of the EB Research Partnership

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