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Vandana Singh

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

  • ProQR Therapeutics N.V. (NASDAQ: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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