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Liver cirrhosis
Liver cirrhosis is the seventh-leading cause of death in the world andrepresents a late stage of progressive liver fibrosis. Today, no availabletreatment can stop or reverse the disease. The only option may be livertransplantation, which still carries a high-risk and, due to the lack ofsufficient donors, is not available to many of these patients. More than27,000 patients die of cirrhosis and chronic liver disease each year in theU.S. alone.
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CIMA IGF-I program
The agreement stems from the exclusive license to AMT from Digna Biotechto commercialize all gene therapy products resulting from the R&D activitiesperformed at the Center for the Study of Applied Medicine (CIMA) at theUniversity of Navarra, Spain. Employing more than 400 researchers, CIMA isone of the leading gene therapy research centers in Europe. The IGF-1 programis the first initiated under that agreement.
The group of Professor Prieto at CIMA has established in relevant animalmodels an extensive Proof of Concept demonstrating that expression of lowlevels of IGF-I in fibrotic and cirrhotic liver is associated with afavorable outcome of the disease and that gene-therapy-mediated IGF-Iexpression has promising effects on the progression of the disease as well asits systemic complications. Prieto and his collaborators have demonstratedthat even low doses of AAV engineered to carry IGF-1 were sufficient tointerfere with, or even reverse fibrosis and achieve a long term effect. AAVvectors constitute the gene therapy platform of choice of Amsterdam MolecularTherapeutics.
A pilot clinical trial conducted by investigators in Pamplona, Spain andGroningen, The Netherlands in a small number of cirrhotic patients supportsthe importance of IGF-1 in treating cirrhosis - both an increased serumalbumin and improved energy metabolism were achieved as a result of(subcutaneous) IGF-I protein administration. Because of the short half-lifeof IGF-1, a treatment based on the subcutaneous administration of recombinantIGF-I would require almost constant infusion and is not considered practical.The gene-therapy-mediated induction of IGF-I expression bypasses thisdisadvantage and shows long-term effect, as the animal studies at CIMA haveshown. Clinical studies will need to confirm the long-term safety andefficacy in men.
Ronald Lorijn, CEO of AMT, said, "We are very pleased with our agreementwith CIMA, which gives us access to programs that are already well-advancedand that have tremendous potential. Our technology platform seems ideallysuited to develop IGF-I for the treatment of liver cirrhosis, a very seriousdisorder, which not only causes great human suffering, but also comes at avery high cost for society. AMT is fully dedicated and equipped to add thisnew program to its product pipeline and plans to start the necessarypre-clinical studies including a full toxicology program next year. We areconfident to continue to leverage our close relationship with Digna and CIMAto fill our pipeline with promising products that address unmet medicalneeds."
Clinical Program AMT-011
The clinical development of AMT's lead product AMT-011 for the treatmentof Lipoprotein Lipase Deficiency is proceeding according to plan. Allpatients have been recruited in the Canadian study. A total of 6 patientshave been injected with AMT-011, completing the first 2 dose cohorts. T
