AVROBIO is switching priorities in 2022 away from its Fabry disease program and into its other more lucrative clinical-stage projects.
AVROBIO is switching priorities in 2022 away from its Fabry disease program and into its other more lucrative clinical-stage projects.
In a statement, the gene therapy company said that it is no longer prioritizing Fabry disease-related activities due mostly to the challenging regulatory environment and market for the disease. AVROBIO also recently found that its Phase II clinical study into FAB-GT patients would need to be extended for a much longer period.
Data from 13 patients demonstrated durable engraftment out of nine to 54 months. However, findings from the latest five patients dosed showed different results, including a reduction to near baseline levels in alpha-galactosidase A (AGA) enzyme activity in plasma and leukocytes, plus a drop in the vector copy number (VCN) in whole blood. Due to the huge variations in results, AVROBIO will be forced to reassess its processes and other parameters, which could be costly.
“We’re fully aware of the impact this difficult decision has on the patients and families whom we have had the privilege to get to know over the years, but we believe deprioritizing and halting enrollment in our Fabry disease program is the right step forward for AVROBIO and preserves our ability to continue developing therapies with the potential to address urgent unmet needs in the lysosomal disorder community,” commented Geoff MacKay, the president and chief executive of AVROBIO, in a statement.
AVROBIO will halt enrollment activities for the FAB-GT clinical trial but will continue monitoring patients who have already been dosed for up to 15 years, as required by regulatory authorities.
With that said, the company will focus on other items in its pipeline for 2022, including AVR-RD-04 for cystinosis, AVR-RD-02 for Gaucher disease type 1, AVR-RD-06 for Gaucher disease type 3, AVR-RD-05 for Hunter syndrome and AVR-RD-03 for Pompe disease. It will also focus on its plato platform, which studies the potential of using monoclonal antibody conditioning agents in Gaucher disease type 1.
“Following steady progress in 2021, we have reset our corporate priorities and will extend our cash runway to strengthen our ability to deliver on the promise of our gene therapy programs. Powered by our proprietary plato® gene therapy platform, we will focus our efforts on moving value driving clinical-stage programs forward in 2022, with data updates expected for our cystinosis and Gaucher disease type 1 programs, as well as regulatory interactions anticipated across multiple programs in our pipeline,” noted MacKay.
With the shift in priorities, the company said it will be able to extend its cash reserves of $201 million (as of September 20, 2021) well into the first quarter of 2024.
