NEW YORK – UniQure has treated the first patient with Fabry disease within its Phase I/IIa clinical trial testing AMT-191, the company said Thursday.
Amsterdam-based UniQure is evaluating the safety, tolerability, and early signs of efficacy of AMT-191 in patients with Fabry disease, a rare X-linked inherited lysosomal storage disorder caused by mutations in the GLA gene. These mutations result in toxic accumulation of fatty acids in multiple organs through deficient alpha-galactosidase (GLA) enzyme activity.
AMT-191, which is administered to patients intravenously, uses an adeno-associated virus 5 vector to deliver a GLA transgene to the liver.
The open-label clinical trial will be conducted in the US, and investigators will recruit up to six adult male patients into each of two cohorts, who will receive one of two dose levels of the gene therapy. Patients will continue to receive routine enzyme replacement therapy until they meet specific criteria for its withdrawal and will be tracked for two years.
Investigators will monitor expression of the lysosomal enzyme aGLA-A, among other outcomes.
"Our trial is designed to capture well-established endpoints in Fabry disease and to rapidly generate clinical proof-of-concept data for AMT-191 with a differentiated product profile relative to other Fabry programs in clinical development," UniQure Chief Medical Officer Walid Abi-Saab said in a statement.
Other biopharmaceutical companies are developing gene therapy candidates for Fabry disease, although they have faced challenges.
Last week, 4D Molecular Therapeutics said that the US Food and Drug Administration removed a clinical hold on its Phase I/II trial of its investigational gene therapy candidate for Fabry disease cardiomyopathy, 4D-310, for which it expects to resume enrollment later this year. Meanwhile, Sangamo Therapeutics is evaluating ST-920 as a gene therapy for Fabry disease in a Phase I/II trial, but said it will not invest in a registrational trial until it secures a commercialization partner.