ProQR, a biotech in the Netherlands developing therapies for rare diseases, has received authorization from the US Food and Drug Administration to launch a Phase 1/2 clinical trial for QR-421a, its treatment targeting mutations in exon 13 of the USH2A gene. The mutations cause Usher syndrome type 2A (combined vision and hearing loss) and non-syndromic retinitis pigmentosa (vision loss only) in approximately 16,000 people in the Western World. ProQR plans to begin enrolling patients in the QR-421a Phase 1/2 trial named STELLAR in the coming months with preliminary data expected in mid-2019.
In February 2018, the Foundation Fighting Blindness announced funding of up to $7.5 million for development of QR-421a. The project is part of the Foundation’s RD Fund, its new venture philanthropy fund for advancing promising therapies into and through clinical trials. The RD Fund currently has $70 million in research investments.
“We are very pleased that ProQR has received the go-ahead to evaluate its promising USH2A therapy in a clinical trial,” says Benjamin Yerxa, PhD, chief executive officer at the Foundation. “This is great news for people affected by mutations in USH2A, and it affirms the Foundation’s goal of advancing emerging treatments into human studies as quickly as possible.”
QR-421a is an antisense oligonucleotide, or AON, which is designed to mask mutations in RNA, the genetic messages that lead to production of proteins critical for the health and function of retinal cells.
In September 2018, ProQR announced vision improvements in a Phase 1/2 clinical trial for QR-110, its emerging AON for people with Leber congenital amaurosis 10 (LCA10) caused by a mutation in the gene CEP290. The company is planning a Phase 2/3 clinical trial for QR-110 in the first half of 2019.
“AONs are potentially a very powerful technology for fighting blindness caused by inherited retinal diseases,” says Dr. Yerxa. “We are excited to see these early, promising results for LCA.”