Axovant in-licenses investigational gene therapies

Swiss cell and gene therapy specialist Axovant Sciences (Nasdaq: AXON) has licensed exclusive worldwide rights for the development and commercialization of two novel gene therapy programs to address GM1 gangliosidosis and GM2 gangliosidosis (also known as Tay-Sachs and Sandhoff diseases) from the University of Massachusetts (UMass) Medical School.

GM1 gangliosidosis, Tay-Sachs and Sandhoff diseases are rapidly progressive and fatal pediatric lysosomal storage disorders that reduce life expectancy to less than two to four years of age in the severe forms of the diseases. GM1 gangliosidosis has an incidence of approximately one out of 100,000 live births worldwide, and Tay-Sachs and Sandhoff diseases have an incidence of around one out of 180,000 live births worldwide. GM1 gangliosidosis is caused by defects in the GLB1 gene and GM2 gangliosidosis is caused by defects in the HEXA (leading to Tay-Sachs disease) and HEXB (leading to Sandhoff disease) genes, resulting in impaired enzyme function and the accumulation of toxic gangliosides primarily in the central nervous system.

In exchange for these exclusive worldwide licenses for the gene therapy programs for GM1 and GM2 gangliosidoses, Axovant, a Roivant Sciences company, will be making payments of undisclosed amounts to UMass Medical School tied to development, regulatory and commercial milestones.

AXO-AAV-GM1 will be evaluated in an investigator-initiated clinical program conducted at the National Institutes of Health (NIH), with the first patient expected to be dosed in the first half of 2019. The NIH has assembled one of the largest natural history databases of patients with GM1 gangliosidosis, Tay-Sachs and Sandhoff diseases that documents the clinical progression of the disease in affected patients, which could enable a historical control group for registrational studies. We expect initial data from this clinical program in the second half of 2019 and expect continued enrollment of patients in this program throughout 2019.

The AXO-AAV-GM2 clinical program is ongoing with the first subject having been dosed with the therapy. Initial data from this program are expected in first quarter 2019 and we expect patients to be enrolled in a multi-subject clinical trial in 2019.