Study 011: Stage 1 (Baseline to Month 6) Updated Data Highlights at LDN WORLD Symposium:
- Post-Hoc Subgroup Analysis of Primary Endpoint (modified intent-to-treat (mITT), n=60): in a post-hoc sub-group analysis, patients with a higher baseline disease burden (0.3 inclusions or more per interstitial capillary, n=25) were compared to those with a lower baseline disease burden (0.3 or fewer inclusions per interstitial capillary, n=35). In the 25 patients (14 males and 11 females) with higher baseline disease burden, 7/11 (64%) on migalastat HCl and 2/14 (14%) on placebo were classified as responders. Among the 35 patients (8 males and 27 females) with lower baseline disease burden, 6/19 (32%) on migalastat HCl and 7/16 (44%) on placebo were classified as responders.
- Urine GL-3: The observed median reduction in urine GL-3 from baseline was 17% for placebo and 12% for migalastat. However due to unexpected variability in the pre-treatment urine GL-3 data (>1.6 fold difference between values at screening and baseline), any potential treatment effects on urine GL-3 cannot be determined.
- Renal Function from Baseline to Month 6: Renal function remained stable and changes from baseline were similar in both treatment groups during stage 1. Themean (SD) increase in estimated glomerular filtration rate (eGFR) was 2.7 (15.1) mL/min/1.73m 2 in the migalastat HCl group compared to a mean decrease of 2.4 (10.8) mL/min/1.73m 2 in the placebo group. No clinically meaningful changes in proteinuria were observed, and iohexol GFR data are currently being analyzed.
- Safety: During the first 6 months, no drug-related serious adverse events have been observed. No subjects discontinued migalastat HCl therapy due to a treatment emergent adverse event and the majority of adverse events in both treatment groups were mild in nature.