First-in-human study with new recombinant agalsidase beta (ISU303) in healthy subjects

摘要: ISU303 is a new recombinant agalsidase beta (Agal) enzyme replacement therapy under investigation for Fabry disease, caused by deficiency in α-galactosidase A activity that leads to fatty deposits tissues. We evaluated the pharmacokinetic (PK) parameters, safety and tolerability of healthy adult volunteers. The study was dose block-randomized, double-blinded, placebo-controlled, single-dosing, escalation phase 1 clinical trial. total 18 subjects were enrolled (0.3 mg/kg, n = 6; 1.0 mg/kg, placebo, n = 6). Blood samples PK analysis collected according planned time. parameters each 0.3 1.0 mg/kg Agal group as follows: Cmax (mU/mL) 43.19 ± 5.9 195.86 ± 32.3; AUClast (h·mU/mL) 207.91 ± 25.1 939.96 ± 158.3; t1/2 (hours) 1.13 ± 0.3 1.46 ± 0.2; Cl (mL/min/kg) 1.79 ± 0.2 1.34 ± 0.2, respectively. There seven adverse events (AE) overall. All AEs resolved without any complications. None related drug. no immunogenicity or significant infusion-related reactions. product exhibited dose-dependent well tolerated with