BioWorld International Correspondent

Amsterdam Molecular Therapeutics (AMT) Holding NV in-licensed a second gene therapy product from the Center for the Study of Applied Medicine (CIMA) at the University of Navarra, in Pamplona, Spain, and the university's spin-out company Digna Biotech, SL, of Madrid, Spain.

The latest addition to its pipeline is an insulin-like growth factor I (IGF-I) therapy based on an adeno-associated virus (AAV) vector, which is in development for treating late-stage liver cirrhosis. The Amsterdam, the Netherlands-based company previously in-licensed from CIMA a treatment for acute intermittent porphyria, based on AAV-mediated delivery of the gene encoding porphobilinogen deaminase.

"The exclusive agreement we have signed with CIMA last July gives us privileged access to the output of over 400 scientists who make up one of the pre-eminent R&D groups working in gene therapy today," AMT CEO Ronald Lorijn said on a conference call Tuesday.

The IGF-1 treatment, now called AMT 070, delivers to the liver the gene-encoding insulin-like growth factor I, a peptide hormone similar in structure to insulin. Normally secreted by the liver, it plays a wide range of physiological roles.

Clinical evidence in support of its potential role in treating cirrhosis has been obtained in a pilot study conducted in the Netherlands and in Spain. It demonstrated increased serum albumin and improved energy metabolism, Sander van Deventer, AMT chief scientific officer, said during the conference call. However, the molecule was delivered subcutaneously in that study, an administration route that generally is not feasible owing to the very short half-life of the peptide. Animal studies have demonstrated that delivery of even low levels of IGF-1 to the liver can halt and even reverse liver fibrosis.

AMT 070, which has been in development for the past six years, will not place a major additional cost burden on AMT, van Deventer said. "The program that we're taking on now is a fairly mature program," he said. "There's a whole lot of things that we do not need to do." Moreover, it will plug directly into the company's existing manufacturing platform, thereby reducing the amount of validation necessary to take it into clinical development, he said. AMT 070 is slated to enter the clinic in 2008 and will initially be aimed at severely ill patients.

The company also reported progress from its lead development program, AMT 011, in development for treating lipoprotein lipase deficiency. Six patients have completed dosing in a registration trial under way in Canada, and another eight are due to receive treatment before the end of February next year.

"This should give us ample time to file for a marketing authorization application first with the EMEA and subsequently with Health Canada and the U.S. FDA during the first half of the year in 2008," AMT Chief Operating Officer Tony Gringeri said on the call. "We are happy with what we're seeing in the trial so far, and we believe we are fully on track for the commercial rollout of AMT011 in 2009."

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