DJ Axovant Gene Therapies: FDA Lifts Clinical Hold on AXO-AAV-GM2

By Chris Wack

Axovant Gene Therapies Ltd. said Monday the U.S. Food and Drug Administration has lifted its clinical hold and cleared the investigational new drug application to initiate a registrational study of AXO-AAV-GM2 gene therapy to treat patients with Tay-Sachs disease and Sandhoff disease.

The company said it has received a letter from the FDA indicating that it has satisfactorily addressed all issues related to the clinical hold. In October, Axovant received the FSA's rare-pediatric-disease designation for AXO-AAV-GM2.

Axovant said it is aiming to advance the program through strategic partnerships with leading research organizations. The company recently announced a partnership with Viralgen to support AAV-based vector manufacturing of clinical trial material for the registrational study. Axovant expects to begin patient identification and site startup activities in preparation for dosing children in the planned clinical study.

AXO-AAV-GM2 is an investigational gene therapy for Tay-Sachs and Sandhoff diseases, which are rare, monogenic neurodegenerative lysosomal storage disorders caused by mutations in the genes.

In 2019, clinical evidence from two patients under an investigator-initiated study found that treatment with AXO-AAV-GM2 was generally well-tolerated and associated with improved bioactivity outcomes.

The study will enroll both infantile and juvenile subjects with GM2 gangliosidosis in the U.S. The two-part trial, sponsored by Axovant, will consist of a dose ranging cohort evaluating the safe and efficacious dose of the gene therapy, followed by an efficacy cohort, both of which form the basis of the registrational program.

Axovant Gene shares were up 8% to $2.06 in premarket trading.

Write to Chris Wack at chris.wack@wsj.com

(END) Dow Jones Newswires

November 09, 2020 09:05 ET (14:05 GMT)

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