Ophthalmology-focused ProQR is axing 30% of its workforce, including its chief scientific officer, and re-strategizing, more than two months after its most developed asset, sepofarsen, failed to improve vision in patients with congenital eye disease in a phase 2/3 trial.
The realignment was announced Wednesday as part of an updated corporate strategy that had been in the works since the med’s failure. As part of the workforce cuts, Chief Scientific Officer, Naveed Shams, M.D., Ph.D., is headed to the exits effective in the second quarter.
In a statement, de Boer acknowledged the tough slate of bad news.
“These have been extremely difficult decisions to make as we position the business to drive long-term growth and value,” he said. “We believe deeply in the promise of RNA therapies, the company’s pioneering efforts in this field, and ProQR employees.”
ProQR is also narrowing its research activities, halting all non-sepofarsen-related work for inherited retinal diseases. More specifically, the company is suspending the development of its Fuchs endothelial corneal dystrophy program and QR-1123 for autosomal dominant retinitis pigmentosa, which was licensed from Ionis Pharmaceuticals. Both of those programs have been removed from the company’s pipeline. The company will now shift focus to genetic eye diseases and its RNA editing platform.
The decision is reflective of how many chips were pushed towards sepofarsen. Topline results from a phase 2/3 trial found that sepofarsen did not improve vision or modality among patients with LCA10, a rare inherited eye disease. At the time of the February announcement, ProQR CEO Daniel de Boer called the news “unexpected and disappointing”.
According to ProQR's latest earnings report, the company had more than €100 million ($108 million) in additional funds at the end of 2021 compared to 2020, thanks in large part to a biobucks deal with Eli Lilly. With the cost reductions, the company now has enough cash to last until 2025.
ProQR is not calling it quits on sepofarsen yet, even as costs are cut elsewhere. Additional analysis of the trial found an improvement in vision when comparing patients’ treated eye with their untreated eye. The same was not found among patients that received placebo. That’s evidently enough for the company to keep chugging along, with plans to talk to U.S. and European regulators in Q3.
Additionally, at the recommendation of its data safety monitoring board, ProQR is going to continue three ongoing studies of the drug while awaiting regulatory guidance. But they noted no new trials are planned at this time.
Now, the company’s attention turns toward another RNA therapy, ultevursen, which is indicated for retinitis pigmentosa and Usher syndrome type 2 due to mutations in the USH2A gene. ProQR expects to be able to fund through topline results of a phase 2/3 trial but noted that the study design may tweak based on the additional data from the sopfarsen trial. But those changes would come only after “alignment with regulatory authorities," the company said.