Phase 2/3 pivotal Illuminate trial of sepofarsen for LCA10 enrollment complete with top-line data anticipated in H1 2022
Positive data reported from the Phase 1/2 clinical trial of QR-421a for the treatment of Usher syndrome and non-syndromic retini
Illuminate Phase 2/3 trial of sepofarsen expected to complete enrollment in Q1 2021; additional data from Phase 1/2 InSight extension study to be reported in H2 2021
Enrollment completed for QR-421a dosing cohorts and data from next Phase 1/2 interim
… on key steps required to advance AX-0810 and AX-1412 to clinical trials with translational data updates ahead of … the winding down of ophthalmology programs, including the clinical trials. General and administrative costs for the …
AX-0810 targeting NTCP for cholestatic diseases to be first ProQR Axiomer™ RNA editing program to enter the clinic Company to host virtual Investor and Analyst Event in December to highlight translational data and clinical plans for AX-0810, as well as AX-1412, targeting
… and efficacy of sepofarsen in a Ph1b/2 INSIGHT extension trial in CEP290-associated Leber congenital amaurosis (LCA10) … missing the primary endpoint in the Illuminate Phase 2/3 clinicaltrial, sepofarsen showed an encouraging efficacy …
… studies closely resemble the design of the first in human trial that will focus on target engagement and biomarkers. We … data for our AX-0810 program and to announcing our clinical program candidate later this year, as we progress to … reported in H2 2024, with further detail on design for the clinicaltrial. AX-1412 preclinical proof of concept data and …
… Additional analyses ongoing from Phase 2/3 Illuminate trial of sepofarsen in LCA10 with updates on any potential … anticipated in Q2 2022 Phase 2/3 Sirius and Celeste clinical trials of ultevursen (QR-421a) in Usher syndrome and …