Positive preliminary data from InSight extension study of sepofarsen for LCA10 reported – consistent with benefit seen in Phase 1/2 and building confidence in Phase 2/3 Illuminate trial;
Strategic convertible debt financing extends runway into 2023 if fully draw
Senior leader with extensive global rare disease commercialization experience
Top-line data from Phase 2/3 pivotal Illuminate trial of sepofarsen for CEP290-mediated LCA10 on track for H1 2022
… Cardiovascular Disease targeting B4GALT1; initiation of clinical trials anticipated in late 2024/early 2025 Axiomer … winding down of our ophthalmology programs, including the clinical trials. General and administrative costs for the …
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
Preclinical and translational pipeline program data for AX-0810 for Cholestatic Diseases targeting NTCP and AX-1412 for Cardiovascular Disease targeting B4GALT1 anticipated in 2024, building further momentum toward the clinicAccelerated development of Axiomer™ for CNS ap
… Event in December to highlight translational data and clinical plans for AX-0810, as well as AX-1412, targeting … updates on this program, including preclinical data, clinicaltrial design, and biomarkers that will be assessed, along …
