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Table 2 Serum TTR knockdown by dose group

From: Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study

Dose group (mg/kg) Dose 1 Dose 2
Maximum TTR KD (%) TTR KD at Nadir (Mean % ± SD) Maximum TTR KD (%) TTR KD at Nadir (Mean % ± SD)
0.01 Q4W (n = 4a) 37.8 22.1 ± 12.5 34.4 32.9 ± 2.3
0.05 Q4W (n = 3) 58.0 48.4 ± 16.2 58.5 46.9 ± 15.0
0.15 Q4W (n = 3) 81.7 74.5 ± 6.8*** 86.0 77.0 ± 7.8
0.3 Q4W (n = 7b) 87.5 82.9 ± 5.4*** 90.8 85.7 ± 9.6***
0.3 Q3W (n = 12c) 94.2 83.8 ± 5.1*** 96.0 86.7 ± 7.0***
  1. p values from ANCOVA models including baseline TTR as covariate and dose groups as factor; models significant at p < 0.001 for Dose 1, p < 0.001 for Dose 2
  2. ***p < 0.001 vs. 0.01 mg/kg group
  3. aIncludes first-dose data from additional patient prior to protocol amendment
  4. bExcludes post-day 28 data from patient who experienced drug extravasation during second infusion
  5. cOne patient discontinued the study before second dose of patisiran
  6. ANCOVA analysis of variance; KD knockdown; Q3W every 3 weeks; Q4W every 4 weeks; SD standard deviation; TTR transthyretin