ProQR Therapeutics Pulls Plug On Inherited Retinal Disease Programs, Cuts 30% Workforce

ProQR Therapeutics N.V. PRQR has decided to prioritize the development of genetic eye disease candidates and the Axiomer RNA base-editing technology platform.
The Company will suspend the development of QR-1123 for autosomal dominant retinitis pigmentosa, QR-504a for Fuchs endothelial corneal dystrophy, and all other Inherited Retinal diseases (IRD)-related research activities.
ProQR will implement restructuring initiatives, including a 30% workforce reduction and the departure of Chief Scientific Officer Naveed Shams, expected to be effective in Q2.
ProQR will prioritize the development path for selected ophthalmology programs by comparing active treatment and sham eyes to their corresponding contralateral eyes.
It will accelerate the development of the Axiomer RNA base-editing technology platform, including an initial focus on the liver, central nervous system (CNS), and the eye.
The Company ended 2021 with €187.5 million of cash and cash equivalents. The anticipated expense reductions will fund currently planned operations into 2025.
After disappointing data, the Company undertook a post hoc analysis of the Phase 2/3 Illuminate trial.
Overall, the analyses showed that the efficacy signal is seen with sepofarsen when comparing active treatment and sham eyes to their corresponding contralateral eyes.
In Q3, the Company plans to meet with the EMA and FDA to discuss these data from the Illuminate trial.
The Company will focus on the ultevursen (QR-421a) program for USH2A-mediated Usher syndrome and retinitis pigmentosa on a single Phase 2/3 Sirius trial with the potential addition of an interim/futility analysis in 2023.
Price Action: PRQR shares are down 0.48% at $0.90 during the market session on the last check Wednesday.

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