ProQR Pivots From Ophthalmic to CNS, Liver RNA Therapies


The move comes after the EMA recommended an additional clinical trial of sepofarsen before applying for marketing authorization.

ProQR has announced their intention to focus exclusively on their Axiomer RNA-editing technology platform after negative results and feedback on their phase 2/3 ILLUMINATE trial (NCT03913143) of sepofarsen for treating Leber congenital amaurosis 10 (LCA10).1

ProQR made the announcement after the EMA recommended an additional clinical trial for sepofarsen before submitting a marketing authorization application. The company is deprioritizing their current ophthalmic candidate portfolio, including sepofarsen and ultevursen (QR-421a) for USH2A-mediated Usher syndrome and retinitis pigmentosa, and seeking strategic partnerships to continue their development.

“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, ProQR Therapeutics, said in a statement.1 “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.”

READ MORE: First Patients Dosed in Gene Therapy Trial for Usher Syndrome, Retinitis Pigmentosa

Current clinical trials includingsepofarsen’s ILLUMINATE, INSIGHT (NCT03913130), and BRIGHTEN (NCT04855045) and ultevursen’s Sirius (NCT05158296) and Helia (NCT05085964) will be wound down, with continued access to patients currently receiving these therapies. These measures were taken to preserve capital under partnerships are found.

ProQR will instead be focusing on their Axiomer RNA-editing platform for initial targets including central nervous system and liver indications. The company believes that their oligonucleotide delivery techniques have potential in these indications. The platform uses editing oligonucleotides (EONs) to mediate single nucleotide changes in RNA with high specificity. The EONs recruit the endogenous RNA editing system ADAR to edit RNA.

“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,” Boer added.1

ProQR had announced topline data from ILLUMINATE in February 2022 that showed that the study did not meet its primary endpoint of Best Corrected Visual Acuity (BCVA) at month 12 or notable secondary endpoints assessing full-field stimulus test (FST) and mobility.2

“This was not the outcome we had hoped for and we share in the disappointment many are feeling in the community,” Benjamin R. Yerxa, chief executive officer, Foundation Fighting Blindness, said in a statement at that time.2 “We will continue to work alongside ProQR to learn more from the ongoing analyses and as they work to advance RNA therapies to potentially help children, adults, and families who are affected by rare genetic eye diseases.”

1. ProQR to focus exclusively on axiomer RNA-editing technology and partner ophthalmology programs. News release. ProQR. August 11, 2022.
2. ProQRannounces top-line results from phase 2/3 Illuminate trial of sepofarsen in CEP290-mediated LCA10. News release. ProQR. February 11, 2022.
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