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AMSTERDAM, the Netherlands, Sept. 2, 2014 (GLOBE NEWSWIRE) -- uniQure N.V. (Nasdaq:QURE), a leader in human gene therapy, today announced results for the second quarter of 2014 and an update on multiple gene therapy programs.
Corporate Highlights
• In July, uniQure announced the acquisition of InoCard GmbH, an innovative, early-stage biotechnology company focused on the development of gene therapy approaches for cardiac disease. InoCard has developed a novel gene therapy to preclinical proof of concept, for the one-time treatment of congestive heart failure (CHF), a rapidly progressing disease affecting 26 million people worldwide. InoCard founders Prof. Patrick Most and Prof. Hugo Katus will join uniQure as Managing Director of uniQure in Germany and Chairman of the Scientific Advisory Board for Cardiovascular Diseases, respectively.
• uniQure closed an additional $10 million venture debt loan with Hercules Technology Growth Capital, Inc., increasing the total loan amount to $20 million and providing the Company with greater balance sheet strength and flexibility. The additional capital will be devoted both to rapidly advancing uniQure's pipeline and to accessing early-stage opportunities that will enable the Company to leverage its gene therapy platform and manufacturing expertise. As of June 30, 2014, the Company held cash and cash equivalents of €72.1 million.
• Construction has been completed on the Lexington, Massachusetts facility and employees have moved in after an occupancy certificate was received for the GMP rooms in June 2014. The facility is expected to be fully operational in the first half of 2015.
• Will Lewis, MBA/JD, joined uniQure's Supervisory Board. Mr. Lewis is President and Chief Executive Officer of NASDAQ listed Insmed Inc. and brings to uniQure more than 20 years of executive experience in the pharmaceutical and finance industries as well as in the field of orphan diseases.
Pipeline Program Updates
• Hemophilia B Program - The Company remains on target for the initiation of a Phase 1/2 clinical trial for the hemophilia B AAV5 candidate late in the second half of 2014. Manufacturing of clinical material is currently being completed to enable release as planned in Q4 2014. uniQure has submitted the necessary documentation for the clinical trial application to the authorities in Germany.
• Collaborator-sponsored Pipeline Programs - The current clinical trials being conducted in the Sanfilippo B program with Institut Pasteur and the Acute Intermittent Porphyria program with University of Navarra are fully enrolled and on track for 2014 (AIP) and 2015 (Sanfilippo B) data release. In the partnership with UCSF and NIH for Parkinson's disease five of the six patients in the first dose cohort have been dosed.
• AAV5 Vector Validation - In May, uniQure successfully documented the potential clinical utility of its proprietary AAV5 vector for liver-directed gene therapy. Results obtained from an ongoing Acute Intermittent Porphyria (AIP) dose-escalation Phase 1 trial provided safety evidence for the AAV5 vector from the baculovirus production platform by successfully delivering DNA to the liver in AIP patients without liver enzyme perturbations.
• 4D Molecular Therapeutics Collaboration - In the research program with 4D Molecular Therapeutics, under which uniQure gained exclusive access to 4D's AAV vector discovery and optimization technology for gene delivery to the central nervous system and liver, the Company expects to make a preliminary selection of new synthetic vectors in the first half of 2015.
Glybera Updates
• European Launch - In early August uniQure and its commercialization partner Chiesi provided an update on preparations relating to the launch of Glybera® (alipogen tiparvovec) in the European Union for the treatment of the orphan disease lipoprotein lipase deficiency (LPLD). Chiesi has exclusive rights to commercialize Glybera in the EU and selected additional territories. Chiesi and uniQure decided to include the six-year follow-up pancreatitis data from the study AMT 011-05, in the pricing and reimbursement applications as announced on June 3, 2014. Chiesi now expects to launch Glybera in the fourth quarter of 2014/first quarter of 2015.
• Six-year Follow-up Data - uniQure announced the analysis of six-year follow-up data from the study AMT 011-05 for Glybera for the treatment of LPLD, which validate long-term clinical benefits. Following Glybera treatment LPLD patients no longer experienced severe pancreatitis, and the occurrence of less severe events was reduced by approximately 50%.
• U.S. Regulatory Progress - To optimize the time for patients in the US to access Glybera and make the overall process more efficient, uniQure intends to combine both EMA and FDA requirements into one clinical protocol for the Phase 4 study. As a result, the Company expects a delay in generating the clinical data to support the filing in the US. This delay is in part the result of longer than expected EMA approval timelines due to required changes in the protocol in support of the US filing strategy, resulting in a planned Phase 4 trial start mid 2015, and in part the result of limited product supply due to a Glybera-specific batch release assay being out of specification.
• Manufacturing Ramp-up - uniQure continues to optimize its original Glybera-specific manufacturing process to match the higher manufacturing standards already achieved with later pipeline products (e.g. Acute Intermittent Porphyria, Sanfilippo, Hemophilia) and to increase Glybera-specific batch release success rates. The Company expects to be able to meet 2015 EU commercial demand for Chiesi with current stock and from future production runs commencing later in 2014, assuming higher manufacturing standards are successfully implemented.
• New Distribution Alliance - uniQure signed an exclusive distribution agreement with Medison Pharma Ltd., Israel's leading pharmaceutical marketing group. Under the terms of the agreement, Medison will market Glybera in Israel and the Palestinian Authority territories. uniQure continues to negotiate similar marketing agreements for Glybera in those regions where the Company plans to market the product with a commercialization partner.
Jörn Aldag, uniQure Chief Executive Officer, commented: "uniQure is steadily executing its strategy of building a valuable clinical and pre-clinical pipeline from its gene therapy platform. We made major progress toward the achievement of preclinical, clinical, and corporate development goals, which we view to be essential to maintain our position at the forefront of gene therapy. In the second half of the year, we are focused on the start of the hemophilia B clinical trial."