ProQR Therapeutics (NASDAQ: PRQR) has announced that it is ceasing further R & D on its antisense inherited retinal eye disease program.
ProQR has been struggling since announcing in February that the Phase 2/3 pivotal trial in its lead program of sepofarsen in LCA failed to meet both primary and secondary endpoints. After teasing out some positive data from additional analysis of the data, the company consulted with both the EMA and FDA, but it became clear that a further pivotal trial would be required. Instead, ProQR has opted to seek a strategic partner for its ophthalmology portfolio.
The company will now focus on its Axiomer RNA-editing technology platform in the therapeutic areas of liver and CNS, which are already focus areas of its research partnership with Eli Lilly.
PRQR closed yesterday with a market cap of US$60m. Cash reserves at 30/06 were US$160m.
https://www.proqr.com/press-release...technology-and-partner-ophthalmology-programs
https://medcitynews.com/2022/08/aft...e-eye-proqr-therapeutics-pivots-to-liver-cns/
https://hotcopper.com.au/threads/sepofarsen-fails-in-pivotal-trial.6580268/?post_id=59530985
https://hotcopper.com.au/threads/the-attraction-of-reversible-editing.6283772/?post_id=56037509
https://hotcopper.com.au/threads/progress-updates.6076458/page-10?post_id=53480861
Ann: PYC Therapeutics Investor Call - 18 August 2022, page-10
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