UniQure: AAV5-Based Therapy Shows Positive Topline Results In Phase I/II Trial

(RTTNews) - uniQure N.V. (QURE) announced the topline results of one-year follow-up data from a Phase I/II clinical trial conducted by Institut Pasteur in four Sanfilippo B syndrome patients treated with a gene therapy, AMT-110. The therapy consisted of uniQure's AAV5 viral vector and a gene cassette including the N-acetylglucosaminidase, alpha (NAGLU) gene, manufactured with uniQure's insect cell based-technology. The company said, in all four patients, researchers verified the restoration of catalytical activity of the NaGlu protein in the cerebrospinal fluid from 0% at baseline up to 14-17% of normal at 3 months with persistent effect at 12 months.

uniQure said the results validate the effective transmission of the NAGLU gene with the AAV5 vector. The trial demonstrated that incremental cognitive development was maintained in all four patients, aged 20 to 53 months at study onset.

The clinical trial was conducted by Biotherapies for Neurodegenerative Diseases Unit, Institut Pasteur/INSERM, in partnership with the French Muscular Dystrophy Association and Vaincre les Maladies Lysosomales, or collectively, the consortium.

"The data validates AAV5 from our insect cell-based manufacturing platform as a safe and effective vector choice for CNS administration. Based on these results, we are negotiating an agreement with the consortium to take over the sponsorship of the program and we intend to advance the program into the pivotal stage of development," said Joern Aldag, CEO of uniQure.