uniQure Announces Results for Fourth Quarter and Financial Year 2015
AMSTERDAM, the Netherlands, April 4, 2016 uniQure N.V. (QURE), a leader in human gene therapy, today announced audited results for the fourth quarter and year ending December 31, 2015, and provided a corporate overview on its pipeline programs and operations.
"Over the past several months, we announced proof-of-concept results from our Phase I/II clinical trials in Sanfilippo B and hemophilia B. Both studies demonstrated not only encouraging safety and efficacy data, but also validated our proprietary AAV5 technology and insect cell, baculovirus production system in both the brain and the liver," said Dan Soland, CEO of uniQure. "In 2016, we will move closer to advancing these lead programs into pivotal studies and initiate Investigational New Drug (IND)-enabling studies on additional product candidates that are expected to enter human clinical trials in 2017. We are particularly looking forward to sharing longer follow-up data from our ongoing Phase I/II clinical study of AMT-060 in hemophilia B and from the Sanfilippo B patients. Since joining uniQure in December, I am increasingly convinced that our validated technology platform, commercial-scale manufacturing capabilities and broad clinical and preclinical programs will continue to drive our leadership in gene therapy."
Corporate Highlights
Liver/Metabolism Therapeutic Focus Area
Reported Encouraging Top-line Data from Low-Dose Cohort in Phase I/II Study of AMT-060 -- On January 7, 2016, uniQure announced preliminary topline results from the first, low-dose cohort in the ongoing AMT-060-01 Phase I/II Hemophilia B study. Initial results showed that AMT-060 was generally well tolerated and the first two patients that completed at least 12 weeks of follow-up showed promising Factor IX expression levels of 5.5% and 4.5% of normal. Additionally, four out of five patients discontinued recombinant FIX prophylaxis as of January 6, 2016. These early data validate successful transduction of the liver using uniQure's proprietary AAV5 vector. uniQure expects to report results on all five patients from the low-dose cohort at a scientific conference in the second quarter of 2016.
Initiated Dosing of AMT-060 in High-Dose Cohort -- On March 14, 2016, the Company announced that the first patient in the high-dose cohort of the AMT-060-01 trial had been treated. As of today, 2 patients in the high-dose cohort have been treated. All 8 patients screened so far in the Phase I/II trial have tested negative for anti-AAV5 antibodies.
CNS Therapeutic Focus Area
Positive Results from Phase I/II Study of AMT-110 in Sanfilippo B -- On September 19, 2015, encouraging results from an academic-sponsored Phase I/II trial in Sanfilippo B using uniQure's novel AAV5-based gene therapy were presented at the European Society of Gene and Cell Therapy (ESGCT) in Helsinki, Finland. 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 three months and maintained further at 12 months. The trial also demonstrated that incremental cognitive development was maintained in all four patients with no progression of brain atrophy detected via MRI scans. These data validate the effective transmission of the NaGlu gene into the brain with uniQure's proprietary AAV5 viral vector. uniQure expects to present 30-month follow-up data from the four patients in early 2017.
Sponsorship of Extension Protocol for Phase I/II Study of AMT-110 to Transition to uniQure -- In January 2016, uniQure and the academic consortium comprised of Institut Pasteur, the French Muscular Dystrophy Association, Vaincre les Maladies Lysosomales and Institut National de la Sante et de la Recherche Medicale (INSERM), executed a term sheet reflecting the license of certain data and intellectual property from the consortium-sponsored Phase I/II study of AMT-110 in Sanfilippo B. uniQure and the consortium are currently negotiating the terms of a definitive agreement. Additionally, uniQure has assumed the sponsorship of the Phase I/II extension study, enabling the Company to continue the follow-up of the four patients treated to date.
Phase I Study in Parkinson's Disease to Complete Enrolling Second Cohort -- A Phase I clinical study in Parkinson's disease led by Krystof Bankiewicz, MD, PhD of the University of California at San Francisco together with John D. Heiss, MD, and colleagues from the National Institutes of Heath, using an AAV-glial cell line-derived neurotrophic factor (GDNF) product licensed from AMGEN for gene therapy applications, has completed enrollment of its first of four six-patient cohorts and is expected to complete the treatment of the second cohort later in the year.
Preclinical Proof-of-Concept Achieved in Huntington's Disease -- On March 22, 2016, uniQure announced the publication of preclinical data in the March 2016 edition of Molecular Therapy-Nucleic Acids which showed that AMT-130, a novel AAV5-based gene therapy candidate for Huntington's disease, achieved preclinical proof-of-concept. The studies, which were conducted in vitro and in a humanized mouse model, demonstrated silencing of the mutated Huntingtin gene with therapeutic microRNAs delivered via an AAV5 vector. uniQure has selected its lead candidate and has initiated IND-enabling studies.
Cardiovascular Therapeutic Focus Area
Four Targets Designated by BMS; Advancing S100A1 Gene Therapy Towards IND Filing -- On April 6, 2015, uniQure announced a collaboration with Bristol-Myers Squibb in which the two companies would develop gene therapies for cardiovascular diseases. In total, the parties may collaborate on up to ten targets, of which four have been designated. uniQure and BMS are currently conducting safety and toxicology studies for the S100A1 gene therapy using uniQure's proprietary insect-cell, baculovirus expression system and are advancing towards an IND filing.
Technology and Manufacturing