AVROBIO Inc (NASDAQ: AVRO) dropped by 30% after announcing it was changing its portfolio priorities to concentrate on other clinical-stage pipeline programs. The company plans to deprioritize the Fabry program because of several reasons, including new clinical findings from the most recent patients given the Phase II FAB-GT drug candidate.
These findings would significantly extend the Fabry disease program’s developmental timeline and increasingly challenging regulatory and market environments regarding the Fabry disease.
What does top management have to say?
AVROBIO’s President and Chief Executive Officer said:
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.
The CEO claimed that they have reset their corporate policies and will extend its cash runway to strengthen its ability to deliver on the promise of its gene-based therapy programs. The CEO continued and said:
Previously reported data from 13 patients treated across our three clinical-stage programs have shown durable engraftment out 9 to 54 months. It is the new data from the five most recently dosed Phase 2 FAB-GT patients that are discordant with these other data and show variable engraftment.
New information from Phase II FAB-GT trials
The clinical data from the five patients who most recently got the FAB-GT dose shows variable engraftment patterns. Data from three of these patients showed reduced baseline levels in AGA (alpha-galactosidase A) enzyme activity in plasma and leukocytes. They also showed reduced VCN (vector copy number) in whole blood.
According to the findings it has received, the company believes that in some scenarios, there might be some intrinsic resistance to persistent engraftment linked to the underlying pathophysiology of Fabry disease that has been left untreated.
AVROBIO has also examined conditioning parameters and procedure-related factors in the context of Fabry disease that’s been left untreated. In addition, safety data derived from the trials showed no adverse events. Therefore, the company will halt the enrollment for these clinical trials and continue analyzing the patients that were previously dosed with FAB-GT.
As of Q3 2021, the company had about $201 million in cash, and it plans to expand its cash runway through to Q1 of 2024.
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