ProQR Therapeutics has spun out its early-phase oligonucleotide treatment for a rare inherited skin disorder to form a new company. The spinout, Wings Therapeutics, starts life with a phase 1/2 asset and funding from a disease-focused nonprofit.
Dutch RNA specialist ProQR took its topical oligonucleotide treatment for dystrophic epidermolysis bullosa, QR-313, into an eight-patient phase 1/2 recessive dystrophic epidermolysis bullosa trial in June. Back then, ProQR talked up the prospect of improving outcomes by taking an exon skipping approach to drive production of a protein that is lacking in patients with the disease.
Since then, ProQR has spoken more about its ophthalmology programs than QR-313. With hindsight, the shift in focus appears to have been evident at ProQR’s R&D in January, which featured one quick reference to QR-313 in a 143-page slide deck. ProQR spent far more time discussing ophthalmology assets that are yet to start human testing.
Now, ProQR has confirmed QR-313 is no longer part of its plans. ProQR has taken a minority stake in the spinout and is in line to receive milestones and royalties if the drug advances. But the company has otherwise severed its ties to the program.
The decision follows an interim analysis and strategic review. The phase 1/2 trial remains blinded and is continuing to enroll patients. ProQR is handing over responsibility for continuing and finishing the trial to Wings, raising questions about what it learned during the interim analysis.
EB Research Partnership (EBRP), a nonprofit focused on treating epidermolysis bullosa, has seen enough potential in QR-313 to finance Wings. EBRP and EB Medical Research Foundation (EBMRF) committed up to $5 million in June to co-fund the phase 1/2. It is unclear how Wings will finance its activities in the longer term, assuming the phase 1/2 readout justifies further investment.
Mark de Souza and Hal Landy, both of whom were involved with dystrophic epidermolysis bullosa startup Lotus Tissue Repair in the run-up to its takeover by Shire, will lead Wings as it heads toward the readout.