Hunterase Phase 3 Trial Shows Promising Results for Hunter Syndrome Treatment
Yongin, Friday, 30 May 2025.
GC Biopharma’s Phase 3 trial at Samsung Medical Center highlights significant improvements in mobility and organ function for Hunter Syndrome patients, marking a key advancement in rare disease therapeutics.
Significant Mobility and Metabolic Improvements
The Phase 3 clinical trial for Hunterase (idursulfase beta), conducted by GC Biopharma at Samsung Medical Center, revealed substantial improvements in patient mobility and metabolic markers. Participants in the trial, 24 in total, managed to walk an average of 62.2 meters more in the 6-Minute Walk Test (6-MWT), a stark contrast to the 7.3 meter increase in the placebo group [2]. Secondary outcomes included significant reductions in urinary glycosaminoglycan levels, with decreases in heparan sulfate and dermatan sulfate by 89% and 88%, respectively, and a 71% overall decrease in total GAG levels [2][7].
Reductions in Organ Volumes, Safety Profile
Improvements were also observed in organ volumes, with liver and spleen sizes shrinking by 27% and 26%, respectively [7]. These findings underscore Hunterase’s potential in mitigating some of the critical physical symptoms associated with Hunter Syndrome. Safety evaluations showed that adverse events were generally mild or moderate, with a notably lower incidence of neutralizing antibody production in the treatment cohort (19%) compared to 62.5% in current mainstream therapies [2][7].
Pioneering Research in Asia
The trial, seen as especially significant for being the first Phase 3 study of its kind involving Asian patients, validates Hunterase’s clinical efficacy within this demographic. Professor Young Bae Sohn from the Ajou University School of Medicine remarked that this study is a milestone in validating the clinical performance of Hunterase, which was developed using domestic technology [7].
Global Implications for Rare Disease Treatment
Hunter Syndrome, affecting approximately 1 in 100,000 male births, can lead to severe health decline without early intervention, sometimes resulting in death before adulthood [7]. With only two major treatments currently available—GC Biopharma’s Hunterase and Takeda’s Elaprase—this study positions Hunterase as a potentially superior therapeutic option due to its enhanced performance metrics and improved safety profile [7]. These advancements represent a pivotal moment not just for GC Biopharma but also for the broader field of rare diseases treatment, potentially influencing future global therapeutic strategies.