ProQR announces first results from STELLAR

Summary of Phase 1/2 Interim Analysis of QR-421a for Usher Syndrome Program

A first-in-human clinical trial of QR-421a is ongoing. The Phase 1/2 study, named Stellar, includes adults that experience vision loss due to mutation(s) in exon 13 of the USH2A gene. This Phase 1/2 clinical trial is designed to evaluate the safety and tolerability of QR-421a. Patient benefit will be assessed as well.

Three-months interim findings from Stellar, reported in March 2020, suggested QR-421a given as a single intravitreal injection was safe and well tolerated. QR-421a showed early and encouraging evidence of activity, with 25% of patients showing a benefit across multiple concordant outcome measures and was well tolerated with no serious adverse events. Two of eight participants in the treated groups responded to QR-421a treatment thus far showing benefit in multiple outcome measures.

Across both cohorts thus far, QR-421a was observed to be generally well tolerated with no serious adverse events noted. Based on these early positive findings we will continue the trial as designed at expert centers in North America and select European countries.

Read here the PDF:  Interim Findings of QR-421a Phase 1/2 Clinical Trial for Usher Syndrome and nsRP 

Read press release: ProQR Announces Positive Findings From an Interim Analysis in the Phase 1/2 trial of QR-421a for Usher Syndrome and Provides Business Update