Breaking Barriers Clinical Trial Program – Exceptional Parent Magazine
A clinical trial program is now enrolling patients for its second cohort in a Phase 1/2a study in Hunter syndrome, a rare, hereditary, lysosomal storage disease. The clinical trial is called Breaking Barriers, and explores AGT-182, an investigational enzyme replacement therapy (ERT) that utilizes the body’s natural system for transporting products across the blood-brain barrier (BBB) by targeting the receptor that delivers insulin to all cells of the body, including the brain. Its ability to cross the BBB makes AGT-182 unique among potential treatments for Hunter syndrome.
In people with Hunter syndrome, also known as mucopolysaccharidosis type II, or MPS II, an enzyme called iduronate-2-sulfatase (IDS) is missing or does not work properly, leading to an abnormal buildup of complex sugars in tissues throughout the body, including the skeleton, joints, brain, spinal cord, heart, spleen and liver.
Hunter syndrome primarily affects males and is often severe and always progressive in the approximately 7,600 people it impacts worldwide, of which 25% reside in the United States.
Symptoms of Hunter syndrome include growth delay, joint stiffness and coarsening of facial features. In severe cases, patients experience respiratory and cardiac problems, enlargement of the liver and spleen, and neurological deficits that can lead to premature death. Hunter syndrome is almost always severe, progressive and life-limiting. Available treatments for Hunter syndrome are not expected to cross the BBB in clinically relevant amounts and therefore do not address the progressive neurological complications of the disease.
Breaking Barriers is led by ArmaGen, Inc., a privately held biotechnology company located in Calabasas, California, focused on developing revolutionary therapies to treat severe neurological disorders. Breaking Barriers is an open-label, sequential, multidose study designed to determine a safe and well-tolerated dose of AGT-182.
Data from the first cohort of the Breaking Barriers trial has proven AGT-182 to be generally well-tolerated in a small cohort, and based on a review of the available safety, clinical, and bio-analytical data, an independent Data Monitoring Committee (DMC) has recommended proceeding to the study’s second cohort.
“We are very excited about AGT-182’s potential to address the developmental delays and other neurological symptoms associated with Hunter syndrome,” said Patrice Rioux, M.D., Ph.D., and senior vice president of Global Clinical Development at ArmaGen. “Breaking Barriers is a promising clinical trial program for the patient community and ArmaGen is committed to continuing its development.”
Further information and details on the Breaking Barriers trial is available at https://clinicaltrials.gov/ct2/home (use the identifier # NCT02262338) and http://breakingbarriershuntertrial.com where visitors can find more information on trial sites, signing up for the trial, support groups and frequently asked questions.
This article was originally published on Exceptional Parent Magazine. Here is a link to the article.