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

 
• By Alaric DeArment

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.

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

The failure of ProQR Therapeutics N.V.’s sepofarsen in CEP290-mediated Leber congenital amaurosis 10 (LCA10), an inherited retinal disease (IRD), in a pivotal Phase II/III study was a surprise, but the company has not lost confidence in its overall RNA drug development platform. However, the LCA10 failure has led to skepticism from analysts about the company’s pipeline overall and the entire IRD field.

ProQR said 11 February that the ILLUMINATE trial met neither its primary endpoint of best corrected visual acuity (BCVA) at month 12 nor key secondary endpoints.

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