PRQR - ProQR says EU regulator seeks additional trial for lead candidate

August, 11 2022 07:26 AM ProQR Therapeutics N.V.

Dutch biotech ProQR Therapeutics N.V. ( NASDAQ: PRQR ) announced on Thursday that the European Medicines Agency (EMA) recommended an additional pivotal clinical trial before submitting a marketing application for the company’s lead candidate sepofarsen.
An investigational RNA therapy, sepofarsen, is designed to target Leber congenital amaurosis 10, a common cause of blindness due to genetic factors in children.
Following the EMA feedback, ProQR ( PRQR ) has decided to look for a strategic partner to further advance its ophthalmology portfolio, which also includes RNA candidate ultevursen (QR-421a) targeted at Usher syndrome and retinitis pigmentosa.
Until a partnership is formed and to preserve capital, the company will terminate the ongoing trials for sepofarsen and ultevursen, including Illuminate, Insight, and Brighten studies for sepofarsen, along with Sirius and Helia trials for ultevursen.
In addition, ProQR ( PRQR ) announced a workforce reduction which it said will help extend the cash runway into 2026, along with the decision to wind down trials for sepofarsen and ultevursen.
Early this year, the company announced that its pivotal Phase 2/3 study for sepofarsen did not meet the main goal in LCA10.

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