ProQR Remains Confident In Pipeline After Sepofarsen Flop, Analysts Not So Much

The company is pinning hopes on its Usher syndrome and retinitis pigmentosa programs, but the disconnect between Phase I/II success and Phase II/III failure dented analysts’ enthusiasm.

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ProQR said its Phase II/III trial of sepofarsen in LBA10 failed • Source: Shutterstock

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