ArmaGen has presented data from the first cohort of adult patients (n=4) enrolled in the Phase 1/2a Breaking Barriers clinical trial of AGT-182, which is an investigational enzyme replacement therapy (ERT) for the treatment of Hunter syndrome.
Hunter syndrome, also known MPS II, is a rare, severe, progressive, and life-limiting lysosomal storage disorder.
This clinical trial is an open-label, sequential, multi-dose study to determine a safe and well-tolerated dose of AGT-182, a compound that makes use of 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.
Due to its ability to cross the BBB, AGT-182 is unique among potential treatments for Hunter syndrome, noted the privately-held biotechnology company.
The data indicates that in a small (n=4) cohort, a weekly 1.0-mg / kg dose of AGT-182 administered as an intravenous (IV) infusion to patients, was found to be generally well-tolerated.
After a review of the available safety, clinical, and bioanalytical data, an independent data monitoring committee (DMC) has recommended proceeding to the study's second cohort, in which adult patients are to receive a weekly IV infusion beginning at a dose of 3mg / kg.
As with the first cohort, patients enrolled in the second cohort will be aged 18 and above.
ArmaGen global clinical development senior vice-president Patrice Rioux said: "We are very encouraged by the safety and tolerability data from cohort 1, as well as by the somatic effect of AGT-182, since these findings suggest that participating in a six-week washout period followed by eight weeks of AGT-182 does not have any negative impact on the symptoms of the disease, which allows us to advance to the next dose level (3mg / kg).
"We look forward to initiating the second cohort of this study as we continue to advance the clinical development of this promising compound, which may address the developmental delays and other neurological symptoms associated with Hunter syndrome."