ProQR Seeking to Partner Ophthalmic Programs
Eye On the Cure Research News
The company is halting its clinical programs for LCA10 and USH2A as it seeks a new partner
ProQR Therapeutics, a developer of RNA therapies, has announced that it will wind down its clinical development of sepofarsen for Leber congenital amaurosis 10 (LCA10) caused by the mutation p.Cys998X in the gene CEP290 and ultevursen for Usher syndrome type 2A (USH2A) caused by mutations in exon 13.
Previously, ProQR reported that sepofarsen did not meet its endpoints for efficacy in a Phase 2/3 clinical trial. The European Medicines Agency subsequently recommended that ProQR conduct another clinical trial for sepofarsen before seeking marketing approval in Europe.
ProQR has not yet reported any results for its Phase 2/3 clinical trial of ultevursen for USH2A initiated in December of 2021.
The company is seeking a partner to take on its programs for sepofarsen, ultevursen, and an additional program for retinitis pigmentosa. ProQR will continue to provide access to sepofarsen and ultevursen for patients in the Phase 2/3 clinical trials.
ProQR’s decision to wind down the sepofarsen and ultevursen programs was based on conserving cash for the development of its Axiomer® RNA-editing platform technology for targeting other therapeutic areas, including the liver and central nervous system.
“ProQR’s decision to end development of its RNA therapies for retinal diseases is difficult news, but the Foundation recognizes the progress the company has made in advancing the field to date," said Jason Menzo, CEO of the Foundation Fighting Blindness. “We are committed to help the company find a new partner and optimistic about the future prospects of these programs to address the many affected by these conditions.”
ProQR’s RNA therapies are known as antisense oligonucleotides (AONs) — small pieces of genetic material that can bind complementary molecules of messenger RNA (mRNA) — designed to mask mutations in the mRNA of the affected gene. RNA are genetic messages that cells read to make proteins critical to their health and function.
The Foundation Fighting Blindness has a partnership agreement for the development of ultevursen through its venture arm, the RD Fund, for advancing emerging therapies in or approaching clinical trials.