Clinical Trials

* On September 19, 2015, uniQure announced the topline results of one-year follow-up data from a Phase I/II clinical trial conducted by Institut Pasteur (Biotherapies for Neurodegenerative Diseases Unit, Institut Pasteur/INSERM) in partnership with the French Muscular Dystrophy Association and Vaincre les Maladies Lysosomales in four Sanfilippo B syndrome (MPSIIIB) patients treated with a novel gene therapy, AMT-110. In all four patients, researchers verified the restoration of catalytical activity of the NaGlu protein in the cerebrospinal fluid (CSF) from 0% at baseline up to 14-17% of normal at 3 months with persistent effect at 12 months. These 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 therapy consists of uniQure’s proprietary AAV5 viral vector and a gene cassette including the N-acetylglucosaminidase, alpha (NAGLU) gene, manufactured with uniQure’s proprietary insect cell based-technology. The results of the trial were presented on September 19th at the European Society of Gene and Cell Therapy (ESGCT) and Finnish Society of Gene Therapy (FSGT) Collaborative Congress held in Helsinki, Finland, by Dr. Marc Tardieu, Professor at the Université and Hôpitaux Universitaires Paris Sud and primary investigator of the trial with co-investigators Professors Jean-Michel Heard and Michel Zérah.

In the trial, four patients received a one-time administration of AAV5 gene therapy dosed over two hours directly into the brain. All patients were maintained under coverage of a continuous immune suppression regimen. No local inflammation or other safety concerns related to the therapy or the procedure have been identified. In addition to establishing the safety of the procedure and the AAV5 viral vector, the most important result was the presence of catalytically active NaGlu protein in the CSF measured at 1, 3, and 12 months after treatment. The fact that all treated Sanfilippo B subjects continued to gain skills throughout the study is extremely encouraging. “We are gratified that our pursuit of a gene therapy targeting CNS diseases has found this early and promising success. 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. 

* On April 6, 2015, uniQure provided an update on multiple gene therapy programs. The Company expects clinical data from its collaborator-sponsored Sanfilippo B program with Institut Pasteur will be available in the second half of 2015 and plans to present those results at a relevant scientific meeting.

* On April 6, 2015, uniQure provided an update on multiple gene therapy programs. The Company expects clinical data from its collaborator-sponsored Sanfilippo B program with Institut Pasteur will be available in the second half of 2015 and plans to present those results at a relevant scientific meeting.

* On November 28, 2013, Uniqure has announced that a phase I/II gene therapy clinical trial for children suffering from Sanfilippo B syndrome enrolled a first patient in October of this year. The trial is being carried out and coordinated by the Institut Pasteur (the trial’s sponsor), Inserm, AFM-Téléthon and Vaincre les Maladies Lysosomales (VML). It is being conducted at Bicêtre Hospital (AP-HP) in Paris. This clinical trial is the result of 10 years of collaborative research carried out by Professor Jean-Michel Heard and his team at the Institut Pasteur (Biotherapies for Neurodegenerative Diseases Unit, Institut Pasteur/Inserm U1115) in partnership with AFM-Téléthon and Vaincre les Maladies Lysosomales (VML). This trial focuses on the B form of the disease. Cells incorporate the missing gene, provided by the viral vector, into their DNA thus enabling them to produce the missing enzyme. Three other children will be enrolled into the trial over the coming months.