News update ProQR Update August 2022

Written by Andrew Bolan

Announced today (August 11, 2022), ProQR will focus exclusively on the development of the Axiomer® RNA editing technology platform across multiple therapeutic areas. This comes following feedback from the European Medicines Agency (EMA) related to ProQR’s sepofarsen for Leber Congenital Amaurosis type 10 (LCA10).

RNA illustration

The information within this community update is now out of date but provides information that was correct at the time of publication. Please read our latest community news update: ProQR agreement with Laboratoires Théa for sepofarsen and ultevursen inherited retinal disease programs.

Following the results from the sepofarsen Illuminate trial in February, ProQR sought regulatory guidance on the next phase for the program. The EMA has recommended an additional clinical trial be conducted for sepofarsen prior to submitting a Marketing Authorization Application (MAA). Considering this feedback and 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, ProQR has engaged Lazard Ltd. to run a competitive process to identify a strategic partner to take the ophthalmology portfolio forward.

“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

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.

Further to the announcement in April 2022 on ProQR’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 central-nervous system, which have strong alignment with ProQR’s oligonucleotide delivery approaches. ProQR’s strategy for its Axiomer RNA editing platform includes both in-house development of select pipeline programs using this technology, and a partnering strategy for the platform. As an example, ProQR entered into a partnership with Eli Lilly in 2021 under which we licensed five targets to them in the liver and nervous system areas.

Commenting on the announcement, Daniel A. de Boer, Chief Executive Officer of ProQR Therapeutics, said:

“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 have engaged Lazard to identify 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. 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”

For further information or enquiries about the announcement today, please email: