Shifting focus, ProQR to seek partners for stalled eye drugs

Dive Brief:

  • ProQR Therapeutics will seek a “strategic partner” to take forward its experimental eye drugs, announcing Thursday a change in strategy that will prioritize other research.
  • Moving forward, ProQR will focus on its RNA editing technology platform, dubbed “Axiomer,” which has drawn the interest of Eli Lilly. The companies’ partnership could involve up to five potential treatments targeting the liver or nervous system.
  • As a result of the shift, ProQR will cut staff and reduce costs as it winds down five trials testing its two eye drug candidates sepofarsen and ultevursen.

Dive Insight:

ProQR had expected positive results for sepofarsen, and planned to seek approval if a Phase 2/3 trial testing the drug reached its goal. However, data released in February showed the drug failed to significantly improve patient’s vision, forcing the company to rethink its potential.

Sepofarsen is what’s known as an antisense oligonucleotide, a type of RNA therapy that ProQR designed to treat a genetic disorder that causes blindness. Ultevursen, which works similarly, was in testing for the retinal disorders Usher syndrome and retinitis pigmentosa. 

Following the setback with sepofarsen, the company in April announced it would reduce its workforce by 30% and no longer focus on its two eye disease programs, as well as move away from other types of research to save cash. The additional layoffs come on top of the 30%, leaving 100 employees remaining. 

ProQR expects that cost savings related to the new layoffs and winding down trials will extend its cash runway into 2026. ProQR is one of many other biotech companies that have had to reduce its workforce and rethink its research plans to save cash during the recent market downturn. 

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