BioMarin Pharmaceutical Inc announced positive results from its phase 3 clinical trial of Aryplase, an investigational enzyme replacement therapy for the treatment of mucopolysaccharidosis VI (MPS VI).

The clinical trial demonstrated a statistically significant improvement in endurance (p=0.025) in patients receiving Aryplase compared to patients receiving placebo as measured by the distance walked in 12 minutes, the primary endpoint in the trial. The data from the trial demonstrated a statistically significant reduction in glycosaminoglycans (GAGs) excreted in the urine (p<0.001) in patients receiving Aryplase compared to patients receiving placebo.

The 3-minute stair climb, another measure of endurance and also a secondary endpoint, demonstrated a positive trend (p=0.053) in patients receiving Aryplase compared to patients receiving placebo.

According to the company officials, the results of the clinical trial indicate that treatment with Aryplase was generally well-tolerated. Adverse events during infusions were more common in patients receiving Aryplase but were generally mild to moderate in nature. The frequency of serious adverse events (SAEs) was more common in the placebo group.

"We are pleased that this trial has confirmed the favorable safety and efficacy profile of Aryplase that we have seen in the earlier Phase 1 and Phase 2 trials," stated Stuart J. Swiedler, vice president of Clinical Affairs at BioMarin. "We hope to provide MPS VI patients with the first specific treatment option for this progressive and debilitating disease in the not-too-distant future," he added.

"Through our three clinical studies, one disease survey study, and efforts led by our commercial planning group, we have already identified more than 200 patients with MPS VI who could potentially benefit from treatment with Aryplase, if approved," he said.

Paul Harmatz, associate director of the Pediatric Clinical Research Center at Children's Hospital & Research Center at Oakland, California, who served as principal investigator of the phase 3 trial stated, "It is encouraging to see the functional benefits MPS VI patients experience with Aryplase - benefits that can greatly contribute to an improved quality of life."

MPS VI (also known as Maroteaux-Lamy Syndrome) is a debilitating, life-threatening genetic disease for which no drug therapies are currently available. MPS VI is caused by a deficiency of the enzyme human N-acetylgalactosamine 4-sulfatase, also known as arylsulfatase B. The deficiency leads to the accumulation of GAG in the lysosomes, the digestive organelles of the cell, giving rise to progressive cellular, tissue and organ system dysfunction.