ProQR Therapeutics Is Trading Higher On Positive QR-421a Data In Usher Syndrome

ProQR Therapeutics NV PRQR has announced results from a planned analysis of its Phase 1/2 Stellar trial evaluating QR-421a in adults with Usher syndrome and non-syndromic retinitis pigmentosa due to USH2A exon 13 mutations.

• In the trial, single-dose QR-421a demonstrated benefit on multiple vision measures that moved in concordance, including visual acuity, visual fields, and optical coherence tomography retinal imaging.
• QR-421a was well tolerated, with no serious adverse events reported.
• A mean benefit of 6.0 letters was observed at week 48 in the treated eyes compared to the untreated (contralateral) eyes after a single injection.
• Among advanced disease patients (n=6), a mean benefit of 9.3 letters was observed at week 48 in the treated eyes compared to the untreated eyes. The benefit was maintained for around 12 months.
• The mean total retinal sensitivity improvement was up to 40dB higher in the treated eyes compared to the untreated eyes, and the benefit was maintained for around six months.
• Now the company plans to advance QR-421a to two parallel pivotal Phase 2/3 trials by year-end 2021 – one in early-moderate patients, one in advanced patients.
• Management will conduct a conference call today at 8:15 am E.T.
• Price Action: PRQR shares are trading 9.26% higher at $5.90 in the premarket on the last check Wednesday.