Table 3 Response to PXT3003 on efficacy outcomes in treatment groups, with comparisons of active doses versus Placebo (Full Analysis Set, n = 80)

CMTNS

−0.25 (17.3)

−3.8 (20.4)

−5.8 (17.7)

5.2 (12.5)

−2.6 (−11.9;7.6)

0.67

−3.1 (−11.0;5.4)

0.74

5.5 (−3.4;15.2)

0.16

0.54

0.30

ONLS

−11.8 (33.7)

−12.7 (31.7)

1.2 (16.7)

6.8 (18.2)

−3.9 (−14.2;7.6)

0.72

6.9 (−3.8;18.8)

0.15

14.4 (0.55;30.2)

0.043*

0.28

0.006*

6MWT (m)

9.0 (8.3)

6.2 (8.3)

6.4 (9.4)

9.9 (6.9)

−2.4 (−6.2;1.5)

0.85

−2.4 (−6.6;2.0)

0.82

0.7 (−3.2;4.7)

0.38

0.11

0.16

9HPT (s)

3.6 (10.9)

−2.5 (12)

4.4 (9.5)

6.1 (10.6)

−4.6 (−10.3;1.5)

0.89

−0.2 (−5.3;5.2)

0.52

0.3 (−5.7;6.6)

0.47

0.15

0.092

Ankle Dorsiflexion (Nm)

20.2 (88.4)

−3.6 (43.0)

81.5 (369.6)

20.4 (64.1)

−4.0 (−21.7;17.8)

0.63

11.4 (−15.4;46.8)

0.26

8.2 (−13.8;35.9)

0.28

0.11

0.16

Grip (kg)

9.9 (24.2)

1.3 (15.6)

4.7 (12.5)

11.7 (18.1)

−7.1 (−15.6;2.1)

0.90

−3.6 (−11.8;5.4)

0.75

1.6 (−7.7;11.9)

0.39

0.12

0.15

CMAP (milliV)

34.4 (62.0)

1.4 (38.7)

22.9 (62.6)

64.2 (208.5)

−25.1 (−44.8;1.5)

0.94

−9.2 (−27.3;13.5)

0.77

−5.1 (−27.1;23.6)

0.63

−0.001

0.50

MCV (m/s)

3.7 (8.5)

3.0 (11.5)

5.7 (12.3)

9.0 (17.6)

−1.0 (−6.5;4.9)

0.61

0.5 (−4.8;6.2)

0.44

2.8 (−3.4;9.4)

0.23

0.11

0.18

DML (ms)

−0.33 (8.7)

0.33 (16.1)

8.3 (18.1)

5 (15.2)

3.4 (−4.3;11.7)

0.24

13.8 (4.2;24.3)

0.009*

8.0 (0.59;16.0)

0.038*

0.21

0.035*

SNAP (microV)

12.4 (121.7)

11.5 (88.2)

23.3 (128.4)

5.2 (69.0)

−1.2 (−42.9;71.0)

0.52

8.7 (−31.2;71.6)

0.38

13.9 (−24.1;71.0)

0.29

0.09

0.30

SCV (m/s)

3.4 (11.0)

5.3 (11.2)

29.5 (63.4)

30.5 (10.0)

1.5 (−5.8;9.4)

0.36

17.5 (−5.5;46.2)

0.11†

26.6 (15.5;38.8)

0.00037*

0.42

0.01*