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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...

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In Brief: Nanoscope Ploughs Forward With FDA Application For Retinitis Pigmentosa Gene Therapy

 
• By Manas Mishra

Nanoscope’s experimental gene therapy, MCO-010, is designed to target the broader retinitis pigmentosa population, regardless of the underlying gene mutation, with a completed US regulatory submission expected in early 2026.

In Brief: 4DMT Fast-Tracks Long-Acting Wet AMD Therapy Trials, Cuts 25% Of Workforce

 
• By Sushmita Panda

4D Molecular Therapeutics has fast-tracked its Phase III trials for 4D-150 in wet AMD, with top-line data from one now expected in H1 2027 and another underway ahead of schedule. The company is also cutting a quarter of its staff as it focuses on its late-stage pipeline and to help fund the trials.

In Brief: Opus Gears Up For FDA Filing In Presbyopia

 
• By Sushmita Panda

Data from a second pivotal trial support a filing in the US for Opus Genetics’ phentolamine ophthalmic solution, to treat presbyopia.

Bayer Buoyant After EU Nod For Twice-Yearly Eylea

 
• By Kevin Grogan

Green light follows a recent rejection by the US FDA to partner Regeneron's request for an extension to the dosing interval.

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GSK’s Blenrep US Setback Could Go From Bad To Worse

 
• By Sushmita Panda

With a US approval any time soon now looking unlikely, analysts are revising Blenrep’s peak sales guidance downwards.

Boehringer Ingelheim Expects Two Key Approvals In Second Half Of 2025

 
• By Andrew McConaghie

The privately held German pharma firm is hoping to bring a new generation of pulmonary fibrosis and cancer drugs to market but their commercial success is not yet a certainty.

More Pressure on Elevidys As The EU’s CHMP Says ‘No’

 
• By Alexandra Shimmings

Just days after Roche stopped shipments of the DMD gene therapy following safety concerns raised by two patient deaths, its EU approval application has hit a block.