ProQR to re-focus following sepofarsen setback

11 August 2022

Dutch rare diseases biotech firm ProQR Therapeutics (Nasdaq: PRQR) today provided an update on its ophthalmology programs following feedback from the European Medicines Agency (EMA) related to sepofarsen and will now focus exclusively on its Axiomer RNA-editing technology platform.

ProQR’s New York traded shares were down 2.4% at $0.82 in pre-market activity following the announcement.

Following the results from the sepofarsen Illuminate trial, the EMA has recommended an additional clinical trial be conducted for sepofarsen prior to submitting a Marketing Authorization Application (MAA). In light of this feedback and in order to continue advancement of the portfolio of ophthalmic product candidates, including sepofarsen for LCA10 and ultevursen (QR-421a) for USH2A-mediated Usher syndrome and retinitis pigmentosa, the company will seek to identify a strategic partner to take the ophthalmology portfolio forward.

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