Genzyme Announces Positive Initial Observations in Trial Evaluating Novel Oral Treatment for Gaucher Disease
Genzyme Corp. announced that it has completed enrollment in the ongoing Phase 2 trial of Genz-112638, an oral therapy being developed for the treatment of Gaucher disease. Based on positive results seen in the trial to date, Genzyme intends to meet with regulatory agencies in the coming weeks to discuss an expedited development strategy.
Initial observations from the first five patients suggest that Genz-112638 may produce a rapid and meaningful impact on important clinical endpoints including reductions in spleen and liver volume, and an increase in platelet counts and hemoglobin concentration. Safety observations from all patients enrolled to date suggest that the only drug-related adverse events seen in the trial have been mild and transient in nature, including one possibly related serious adverse event that is currently being investigated.
If these early improvements continue and are observed in other patients enrolled in the trial, Genz-112638 may represent a promising novel approach to treating patients with Gaucher disease.
Genz-112638, a novel ceramide analog given orally, is designed to inhibit the enzyme glucosylceramide synthase, which results in reduced production of glucocerebroside. This is the substance that builds up in the cells and tissues of people with Gaucher disease. In preclinical studies, the molecule has shown high potency and specificity.
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