Abstract Details

Cipaglucosidase alfa/miglustat is an investigational, two-component therapy for Pompe disease. We report data up to 48 months for 6-minute walk distance (6MWD) and % predicted sitting forced vital capacity (FVC) for ATB200-02.

The ongoing ATB200-02 (NCT02675465), open-label, Phase I/II clinical trial aims to evaluate the long-term efficacy and safety of cipaglucosidase alfa/miglustat in adults with Pompe disease.

Three adult ambulatory cohorts - two enzyme replacement therapy (ERT)-experienced (2–6 years [n=11] and ≥7 years [n=6]) and one ERT-naïve (n=6) - received 20 mg/kg intravenous-infused cipaglucosidase alfa plus 260 mg miglustat orally biweekly in an ongoing study. Changes from baseline (CFBL) in multiple endpoints were assessed at intervals. We report data from 6, 12, 24, 36 and 48 months.

Baseline characteristics represented the Pompe disease population. Durable improvements occurred at 6, 12, 24, 36 and 48 months in 6MWD (m): pooled analyses of ERT-experienced cohorts, mean(±SD) CFBL: 23.1(±44.75), n=16; 33.5(±49.62), n=16; 25.2(±63.30), n=13; 9.8(±85.98), n=12; 20.7(±101.84), n=9, respectively; ERT-naïve cohort: 36.7(±29.08), n=6; 57.0(±29.96), n=6; 54.4(±36.18), n=6; 43.5(±45.19), n=5; 52.2(±46.59), n=4, respectively. FVC (%) was stable or improved in ERT-experienced cohorts, mean(±SD) CFBL: −0.9(±8.59), n=16; −1.2(±5.95), n=16; 1.0(±7.96), n=13; −0.3(±6.68), n=10; 1.0(±6.42), n=6, respectively, and improved in the ERT-naïve cohort: 4.2(±5.04), n=6; 3.2(±8.42), n=6; 4.7(±5.09), n=6; 6.2(±3.35), n=5; 8.3(±4.50), n=4, respectively. Over 48 months, serum CK and urine Hex4 biomarkers improved in ERT-experienced and ERT-naïve cohorts.