ProQR to Focus Exclusively on Axiomer RNA-Editing Technology and Partner Ophthalmology Programs
ProQR Therapeutics provided an update on its ophthalmology programs following feedback from the European Medicines Agency (EMA) related to sepofarsen and will now focus exclusively on its Axiomer RNA-editing technology platform.
Following the results from the sepofarsen Illuminate trial, the EMA has recommended an additional clinical trial be conductedfor sepofarsen prior to submitting a Marketing Authorisation Application (MAA). 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 company will seek to identify a strategic partner to take the ophthalmology portfolio forward.
To preserve operating capital, and until a partner is found that can fund the clinical programs moving forward, the current ongoing trials of sepofarsen and ultevursen—including Illuminate, Insight, and Brighten for sepofarsen, along with Sirius and Helia for ultevursen—will be wound down. For people currently participating in these trials, ProQR will offer continued access to currently available sepofarsen or ultevursen.
Strategic Focus and Operational Update
Further to the announcement in April 2022 on the company’s strategy, going forward, ProQR will focus exclusively on accelerating the development of its Axiomer RNA-editing platform technology. Initial therapeutic areas include liver and CNS, which we believe have strong alignment with ProQR’s oligonucleotide delivery approaches.
ProQR’s strategic growth initiatives related to the advancement of its Axiomer RNA editing platform include both in-house development of select pipeline programs using the technology and a partnering strategy for the platform. The company continues to execute on its global licensing and research partnership with Eli Lilly, focused on the discovery, development, and commercialization of potential new medicines for genetic disorders in the liver and nervous system, and may selectively enter additional partnerships designed to advance and capture the full potential value of the platform. ProQR has established a leading IP estate in the ADAR-editing space related to its RNA-editing technology and the platform’s broad applicability has significant potential to treat a number of diseases. The company will provide further updates on the Axiomer strategy, including announcing its internal development targets in late 2022/early 2023.
Based on the wind down of sepofarsen and ultevursen clinical trials and associated workforce reduction, ProQR will realize cost savings in headcount, program, and related support activities, which are expected to extend ProQR’s cash runway into 2026. The cash runway into 2026 excludes any revenue generated from the company’s existing partnership and any potential new partnering deals.
“As we prioritize the development of our Axiomer RNA editing platform technology, we believe partnering our ophthalmology assets is the best strategy to drive these programs forward to patients,” Daniel A. de Boer, Chief Executive Officer of ProQR Therapeutics, said in a company news release. “The feedback we received from the EMA is helpful in designing an additional registration trial for sepofarsen based on the learnings from the Illuminate trial and we will seek a strategic partner for the further development of our ophthalmology programs, including sepofarsen and ultevursen. As a company dedicated to developing therapies to improve the lives of patients, we will offer clinical trial participants continued access to currently available sepofarsen or ultevursen. I want to thank the employees who will be separating from ProQR for their significant contributions in advancing sepofarsen and ultevursen to this stage, as well as the patients, providers, and supporters of these programs.”
De Boer continued, “Going forward, we will focus our strategy and resources exclusively on advancing our Axiomer RNA-editing platform technology and the changes announced today will also enable us to extend our cash runway in 2026. We look forward to continued progress with the business, including sharing details of our development plans for Axiomer.”