AVROBIO has two ongoing clinical studies of AVR-RD-01. AVR-RD-01 is an investigational gene therapy for Fabry disease.
Eight patients have been dosed across these two studies. There are five patients in the investigator-sponsored Phase 1 trial, which is fully enrolled. There are three patients enrolled so far in the Phase 2 trial. We anticipate enrolling 8 to 12 patients in Phase 2.
The primary endpoint of the Phase 2 trial is the average number of Gb3 inclusions per kidney peritubular capillary (PTC), which is measured through a kidney biopsy. This is an FDA-recognized endpoint in Fabry disease.
Gb3 is a substrate, which is a substance that builds up in tissue cells due to the lack of AGA enzyme activity in people with Fabry.
New Data Release
At the one year follow up mark, the first patient dosed in the Phase 2 trial showed an 87% reduction in the average number of Gb3 inclusions in the first kidney biopsy.
In the first four patients enrolled in the Phase 1 study, plasma (blood) lyso-Gb3 was reduced between 33% and 41% below the patients’ enzyme replacement therapy (ERT) levels before receiving AVR-RD-01.
There are six patients across the Phase 1 and Phase 2 trials for whom data are reported at six months or longer post-treatment with AVR-RD-01. These data show sustained AGA enzyme activity and consistent vector copy number (VCN) trends, with VCN levels for the first Phase 1 patient stable at more than two years post-treatment.
In both the Phase 1 and Phase 2 trials, no serious adverse events (SAEs) related to AVR-RD-01 have been reported. The SAEs and AEs reported have been consistent with the conditioning regimen, the underlying disease, or pre-existing conditions.
As you may have also seen, The U.S. Food and Drug Administration (FDA) recently cleared AVROBIO’s Investigational New Drug (IND) application for AVR-RD-01. This means the company can begin enrolling Fabry patients at clinical sites in the U.S. and plans to start enrolling in the second half of 2019.
The Canadian Fabry Association (CFA) is committed to improving the lives of those who live with and are affected by Fabry disease in Canada.