Dive Brief:
- ProQR Therapeutics is overhauling its research, cutting staff and trying to find a new way forward for its lead drug after a surprising study failure in February.
- The Dutch genetic medicines company said Wednesday that Chief Scientific Officer Naveed Shams will leave amid layoffs that will shrink ProQR’s workforce by about 30%. The biotech is also giving up on two experimental eye disease programs and suspending certain other research activities in an effort to free up enough cash to continue operations into 2025.
- At the same time, ProQR said it’s found encouraging data in a new analysis of the Phase 2/3 study that failed to meet its primary goal two months ago. Company executives plan to meet with regulators to see if they can revive prospects for the eye disease treatment, known as sepofarsen.
Dive Insight:
ProQR is regrouping after the study failure in February wiped out more than three-quarters of the company’s value. In January, the share price rose above $8; early Wednesday, the stock traded around 90 cents.
The biotech is now pinning its hopes on a different way of evaluating sepofarsen in patients with a form of the eye disease Leber congenital amaurosis. The failed study, called Illuminate, compared vision changes between patients treated with sepofarsen and a control group that underwent a procedure mimicking an eye injection. After 12 months, there was no significant difference between the groups.
But ProQR says there was a difference when comparing the treated eye to the untreated eye. In patients who received sepofarsen, the treated eye showed a benefit in vision that wasn’t present in comparisons of the two eyes of patients who underwent the sham procedure.
That finding matched up with patients’ own reporting of better vision in the treatment group, ProQR said. And it fit with results from a smaller clinical trial announced in 2019 in which several patients reported substantial improvements. That study also used the untreated eye for comparison.
ProQR will now have to persuade regulators the drug shows enough promise to move forward, although it’s still unclear what its next steps might include. The company said European regulators had preferred the use of a patient’s own untreated eye as the control in research, but it designed the Phase 2/3 trial with a parallel group undergoing a sham procedure to meet U.S. requirements.
While it awaits those discussions, ProQR said it won’t invest in new trials of sepofarsen or in preparations for a commercial launch.
Leber congenital amaurosis is the most common genetic cause of blindness in children. ProQR’s drug is designed to treat a form caused by mutations in the CEP290 gene, responsible for about 2,000 cases in the Western world, according to the company.
ProQR isn’t alone in retrenching amid a broader biotech market downturn and its announcement comes days after Akebia Therapeuticssaid it would lay off 42% of its employees. In the field of genetic medicine, at least eleven biotechs have reported layoffs, costs cuts or research restructurings since December.
