ProQR to Focus Exclusively on Axiomer RNA-editing Technology

ProQR to Focus Exclusively on Axiomer RNA-editing Technology

August 12, 2022

Following comments from the European Medicines Agency (EMA) about sepofarsen, ProQR Therapeutics released an update on its ophthalmology activities and will now exclusively concentrate on its Axiomer RNA-editing technology platform.

The EMA has advised that sepofarsen undergo another clinical trial before filing a Marketing Authorization Application (MMA) as a result of the Illuminate trial's findings.

The company will look to find a strategic partner to advance the ophthalmology portfolio in light of this feedback and in order to continue advancement of the portfolio of ophthalmic product candidates, including sepofarsen for LCA10 and ultevursen (QR-421a) for USH2A-mediated Usher syndrome and retinitis pigmentosa.

The current active studies of sepofarsen and ultevursen—including Illuminate, Insight, and Brighten for sepofarsen, as well as Sirius and Helia for ultevursen—will be wound down in order to preserve operational funds and until a partner is found that can pay the clinical programs moving forward. ProQR will continue to make sepofarsen or ultevursen accessible to those who are currently taking part in these trials.

Strategic Focus and Operational Update

As a follow-up to the company's strategic announcement in April 2022, ProQR will now exclusively concentrate on speeding the development of its Axiomer RNA-editing platform technology.

The expansion of ProQR's Axiomer RNA editing platform is one of the company's strategic growth priorities. This includes both internal development of a few pipeline items employing the technology and a partnering strategy for the platform.

The business continues to carry out its global research and licensing agreement with Eli Lilly, which is centered on the development and commercialization of potential new treatments for genetic diseases of the liver and nervous system. The business may also carefully enter into other partnerships in order to advance and realize the full potential of the platform.

Due to the broad application of the platform, which has a considerable potential to treat a number of diseases, ProQR has created a leading IP estate in the ADAR-editing area. In late 2022 or early 2023, the corporation will announce its internal development goals as part of subsequent updates on the Axiomer plan.

ProQR will experience cost reductions in staffing, program, and related support activities as a result of the winding down of the sepofarsen and ultevursen clinical trials and the resulting workforce reduction, which are anticipated to extend ProQR's financial runway until 2026. Any earnings from the business's current partnerships and any possible new partnerships are not included in the cash runway into 2026.