ProQR Therapeutics, which is developing RNA therapies for genetic eye diseases, announced Wednesday that it will be restructuring and laying off 30% of its staff.
Netherlands-based ProQR Therapeutics, which is developing RNA therapies for genetic eye diseases, announced Wednesday that it will be restructuring and laying off 30% of its staff. This news is the outcome of several recent tough events for ProQR.
The impacts will be felt at a high level as well. Among those being let go are the company’s chief scientific officer, Naveed Shams, M.D., Ph.D. His departure will be effective in Q2 of this year.
The company is also suspending the development of two assets: QR-1123, which was being developed for autosomal dominant retinitis pigmentosa and QR-504a, which was intended for the treatment of Fuchs endothelial corneal dystrophy. The suspension of these programs, as well as the attrition of 30% of the workforce, will help the company fund its essential programs into 2025.
“These have been extremely difficult decisions to make as we position the business to drive long-term growth and value,” said Daniel A. de Boer, CEO of ProQR. “These are the right steps to take to offer the best opportunity to create long-term value for all of our stakeholders, including our shareholders.”
These layoffs and program cuts are the result of several events that have taken place over the last few months. In February, ProQR reported that Phase II/III clinical trials of sepofarsen failed to meet both primary and secondary endpoints. Sepofarsen is a drug that treats CEP290-mediated Leber congenital amaurosis 10, a genetic eye disorder that affects the retina and can cause blindness.
In 2018, a Phase I/II trial of sepofarsen had gone well. The results of the study showed that patients saw vision improvements and that the benefits were stable over time. However, when the Phase II/III trial data came in, no significant improvement in patient vision was detected. The data spooked investors and dropped company stock by more than 75%. de Boer called the results “unexpected and disappointing.”
The harsh news hit just before ProQR released its Q4/full-year 2021 results. The report revealed that the company’s end-of-year losses totaled €61.7 million. In December, ProQR entered into a debt financing agreement with Kreos and Pontifax in an attempt to manage finances.
If ProQR can navigate this turbulent time, though, the company still has the potential to be successful. ProQR will focus on developing its Axiomer RNA base-editing technology platform, with initial efforts going toward liver disease and central nervous system diseases. More internal development targets produced based on Axiomer will be announced later this year.
In addition to its own Axiomer work, ProQR struck a $1.5 billion deal with Eli Lilly in September 2021 to develop RNA-edited genetic drugs using the Axiomer platform.
The company is also anticipating a readout of the Phase II/III Sirius clinical trial of ultevursen, an experimental treatment for Usher syndrome and retinitis pigmentosa. The company hopes to provide data in 2023.
“We are focusing our strategy on accelerating our Axiomer® RNA-base editing platform technology, and a select pipeline of RNA therapies for inherited retinal diseases as we remain committed to developing RNA therapies for patients with high unmet need,” de Boer affirmed.
