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news.Amsterdam Molecular Therapeutics has announced positive interim data from its pivotal clinical trial with the company's lead product Glybera, a gene therapy product that corrects the genetic defect in lipoprotein-lipase deficient patients.
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In the pivotal study conducted in Quebec, Canada, patients with LPL-deficiency were first enrolled in an observation study and subsequently treated in a study in which there are two different dose groups. All patients showed very high fat levels in the observation study and had experienced (recurrent) pancreatitis episodes prior to treatment. To date six patients have been treated with the lower dose and seven with the higher dose.
The company presented data on the first 10 patients, of which six were in the lower dose group and four were in the higher dose group. The data showed that treatment was well tolerated. Fat concentrations were reduced after treatment in all patients, with one exception. In all patients with fat accumulations in skin or retina, these accumulations disappeared or were reduced. All patients reported gain of energy.
Upon completion of the study the results will be part of the filing dossier for the marketing approval of Glybera.