Press Release.
Amsterdam, The Netherlands – November 27, 2007 – Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today announced it has signed an agreement with Digna Biotech/CIMA for the development of AAV-mediated insulin-like growth factor I (IGF-I) to treat late stage liver cirrhosis. At 10:30 a.m. CET today, the company’s management will hold a conference call to discuss today’s announcement and to provide a general business update. Access details can be found at the end of this release.
Liver cirrhosis:
Liver cirrhosis is the seventh-leading cause of death in the world and represents a late stage of progressive liver fibrosis. Today, no available treatment can stop or reverse the disease. The only option may be liver transplantation, which still carries a high-risk and, due to the lack of sufficient donors, is not available to many of these patients. More than 27,000 patients die of cirrhosis and chronic liver disease each year in the U.S. alone.
CIMA IGF-I program:
The agreement stems from the exclusive license to AMT from Digna Biotech to commercialize all gene therapy products resulting from the R&D activities performed at the Center for the Study of Applied Medicine (CIMA) at the University of Navarra, Spain. Employing more than 400 researchers, CIMA is one of the leading gene therapy research centers in Europe. The IGF-1 program is the first initiated under that agreement.
The group of Professor Prieto at CIMA has established in relevant animal models an extensive Proof of Concept demonstrating that expression of low levels of IGF-I in fibrotic and cirrhotic liver is associated with a favorable outcome of the disease and that gene-therapy-mediated IGF-I expression has promising effects on the progression of the disease as well as its systemic complications. Prieto and his collaborators have demonstrated that even low doses of AAV engineered to carry IGF-1 were sufficient to interfere with, or even reverse fibrosis and achieve a long term effect. AAV vectors constitute the gene therapy platform of choice of Amsterdam Molecular Therapeutics.
A pilot clinical trial conducted by investigators in Pamplona, Spain and Groningen, The Netherlands in a small number of cirrhotic patients supports the importance of IGF-1 in treating cirrhosis – both an increased serum albumin and improved energy metabolism were achieved as a result of (subcutaneous) IGF-I protein administration. Because of the short half-life of IGF-1, a treatment based on the subcutaneous administration of recombinant IGF-I would require almost constant infusion and is not considered practical. The gene-therapy-mediated induction of IGF-I expression bypasses this disadvantage and shows long-term effect, as the animal studies at CIMA have shown. Clinical studies will need to confirm the long-term safety and efficacy in men.
Ronald Lorijn, CEO of AMT, said, “We are very pleased with our agreement with CIMA, which gives us access to programs that are already well-advanced and that have tremendous potential. Our technology platform seems ideally suited to develop IGF-I for the treatment of liver cirrhosis, a very serious disorder, which not only causes great human suffering, but also comes at a very high cost for society. AMT is fully dedicated and equipped to add this new program to its product pipeline and plans to start the necessary pre-clinical studies including a full toxicology program next year. We are confident to continue to leverage our close relationship with Digna and CIMA to fill our pipeline with promising products that address unmet medical needs.”
Clinical Program AMT-011:
The clinical development of AMT’s lead product AMT-011 for the treatment of Lipoprotein Lipase Deficiency is proceeding according to plan. All patients have been recruited in the Canadian study. A total of 6 patients have been injected with AMT-011, completing the first 2 dose cohorts. The remaining 8 patients will be injected before the end of February, allowing the Company to file for registration with the EMEA, FDA and Health Canada before the end of the first half of 2008.
Conference call and webcast presentation:
AMT will conduct a conference call open to the public today at 10.30 CET, which will also be webcast. Netherlands dial in: +31 (0)800 949 4517 (toll free); US dial in: +1 866 291 4166 (toll free); UK dial in: +44 207 107 0611. The webcast can be accessed on the investors portion of AMT’s website at www.amtpharma.com. Please go to the website 15 minutes prior to the call to register, download and install the necessary audio software. Playback of the call will be availably for 24 hours after the call. Dial in: +41 91 612 4330; +44 20 7108 6233; or +1 866 416 2558. ID 428#. The archived webcast also will be available for replay shortly after the close of the call.
About Amsterdam Molecular Therapeutics:
AMT has a unique gene therapy platform that to date appears to circumvent many if not all of the obstacles that have prevented gene therapy from becoming a mainstay of clinical medicine. Using adeno-associated viral (AAV) vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate what is probably the first stable and scalable AAV production platform. As such, AMT’s proprietary platform holds tremendous promise for thousands of rare (orphan) diseases that are caused by one faulty gene. AMT currently has a product pipeline with six products at different stages of development.
For information:
André Verwei, CFO
+31 20 566 5686
a.verwei@amtbv.com
Hans Herklots
+31 20 566 8125
h.herklots@amtbv.com
Certain statements in this press release are “forward-looking statements” including those that refer to management’s plans and expectations for future operations, prospects and financial condition. Words such as “strategy,” “expects,” “plans,” “anticipates,” “believes,” “will,” “continues,” “estimates,” “intends,” “projects,” “goals,” “targets” and other words of similar meaning are intended to identify such forward-looking statements. Such statements are based on the current expectations of the management of Amsterdam Molecular Therapeutics only. Undue reliance should not be placed on these statements because, by their nature, they are subject to known and unknown risks and can be affected by factors that are beyond the control of AMT. Actual results could differ materially from current expectations due to a number of factors and uncertainties affecting AMT’s business, including, but not limited to, the timely commencement and success of AMT’s clinical trials and research endeavors, delays in receiving U.S. Food and Drug Administration or other regulatory approvals (i.e. EMEA, Health Canada), market acceptance of AMT’s products, effectiveness of AMT’s marketing and sales efforts, development of competing therapies and/or technologies, the terms of any future strategic alliances, the need for additional capital, the inability to obtain, or meet, conditions imposed for required governmental and regulatory approvals and consents. AMT expressly disclaims any intent or obligation to update these forward-looking statements except as required by law. For a more detailed description of the risk factors and uncertainties affecting AMT, refer to the prospectus of AMT’s initial public offering on June 20, 2007, and AMT’s reports filed from time to time with NYSE Euronext (Holding) N.V.
