AMT-021, a candidate for acute intermittent porphyria, is expected to start in clinic during 2012.
Amsterdam Molecular Therapeutics (AMT) will receive €1.1 million (about $1.5 million) of a €3.3 million (approximately $4.5 million) grant being awarded by the European Union 7th Framework Program (FP7) to the AIPGENE consortium, which is working to develop a gene therapy product for treating acute intermittent porphyria (AIP).
As part of the consortium AMT has commercialization rights to the gene therapy product, AMT-021, and projects patient enrolment into a clinical trial could start in 2012. It says the grant will cover development costs of the product to completion of Phase I/II trials.
AMT-021 is designed to deliver a normal version of the porphobilinogen deaminase (PBDG) liver protein gene that is mutated in AIP. AMT says its studies have already shown treatment using AMT-021 results in normalization of the PBGD protein in an animal model of AIP as well as prevents the occurrence of attacks and significantly ameliorates the neuropathy that otherwise occurs in untreated animals. The firm in addition notes that work by FIMA (Fundación para la Investigatión Médica Aplicada), which is leading the multinational AIPGENE consortium, has demonstrated that gene expression in the liver will persist for over a year using AAV-mediated delivery methods similar to that used for AMT-021.