|
Parameter
|
0.01 mg/kg
|
0.05 mg/kg
|
0.15 mg/kg
|
0.3 mg/kg
|
0.3 mg/kg
|
All patients
|
|---|
|
Q4W
|
Q4W
|
Q4W
|
Q4W
|
Q3W
|
(n = 29)
|
|---|
|
(n = 4)
|
(n = 3)
|
(n = 3)
|
(n = 7)
|
(n = 12)
|
|---|
|
Sex, n (%)
| | | | | | |
|
Male
|
3 (75.0)
|
3 (100.0)
|
2 (66.7)
|
3 (42.9)
|
9 (75.0)
|
20 (69.0)
|
|
Female
|
1 (25.0)
|
0
|
1 (33.3)
|
4 (57.1)
|
3 (25.0)
|
9 (31.0)
|
|
Age, years
| | | | | | |
|
Mean (SD)
|
65.8 (8.96)
|
55.7 (24.83)
|
41.7 (2.52)
|
58.7 (16.07)
|
53.8 (15.6)
|
55.6 (15.61)
|
|
Race, n (%)
| | | | | | |
|
White/Caucasian
|
4 (100.0)
|
3 (100.0)
|
3 (100.0)
|
7 (100.0)
|
12 (100.0)
|
29 (100.0)
|
|
Country
| | | | | | |
|
Portugal
|
0
|
1 (33.3)
|
3 (100.0)
|
2 (28.6)
|
3 (25.0)
|
9 (31.0)
|
|
Sweden
|
2 (50.0)
|
1 (33.3)
|
0
|
2 (28.6)
|
1 (8.33)
|
6 (20.7)
|
|
France
|
2 (50.0)
|
1 (33.3)
|
0
|
2 (28.6)
|
3 (25.0)
|
8 (27.6)
|
|
Brazil
|
0
|
0
|
0
|
0
|
1 (8.33)
|
1 (3.4)
|
|
Germany
|
0
|
0
|
0
|
0
|
1 (8.33)
|
1 (3.4)
|
|
Spain
|
0
|
0
|
0
|
0
|
3 (25.0)
|
3 (10.3)
|
|
United States
|
0
|
0
|
0
|
1 (14.3)
|
0
|
1 (3.4)
|
|
TTR genotype, n (%)
| | | | | | |
|
Val30Met
|
2 (50.0)
|
2 (66.7)
|
3 (100.0)
|
6 (85.7)
|
9 (75.0)
|
22 (75.9)
|
|
Othera
|
2 (50.0)
|
1 (33.3)
|
0
|
1 (14.3)
|
3 (25.0)
|
7 (24.1)
|
|
Mean (SD) serum TTR at baseline, μg/mL
|
272.9 (98.96)
|
226.5 (12.67)
|
276.1 (7.65)
|
242.6 (38.30)
|
235.5 (44.45)
|
245.64 (49.44)
|
|
FAP stageb
| | | | | | |
|
1
|
–
|
–
|
–
|
–
|
–
|
25 (86.2)
|
|
2
|
–
|
–
|
–
|
–
|
—
|
4 (13.8)
|
|
Prior exposure to ALN-TTR01c
| | | | | | |
|
Yes
|
2 (50.0)
|
0
|
2 (66.7)
|
3 (42.9)
|
2 (16.67)
|
9 (31.0)
|
|
No
|
2 (50.0)
|
3 (100.0)
|
1 (33.3)
|
4 (57.1)
|
10 (83.33)
|
20 (69.0)
|
|
Concurrent TTR stabilizer use
| | | | | | |
|
Diflunisal
|
2 (50.0)
|
1 (33.3)
|
0
|
3 (42.9)
|
1 (33.3)
|
7 (24.1)
|
|
Tafamidis
|
0
|
1 (33.3)
|
2 (66.7)
|
4 (57.1)
|
7 (66.7)
|
14 (48.3)
|
-
aNon-Val30Met mutations: Arg45Thr (n = 1), Phe46Leu (n = 1), Ser77Tyr (n = 2), Ser77Phe (n = 2), Tyr116Ser (n = 1)
-
bFAP stage: 1 = unimpaired ambulation, mostly mild neuropathy in lower limbs; 2 = assistance with ambulation required, mostly moderate neuropathy with progression to lower limbs, upper limbs, and trunk; 3 = wheelchair-bound or bedridden, severe neuropathy of all limbs
-
cALN-TTR01 was the first-generation siRNA-LNP used in phase I studies in patients with ATTR
-
ATTR TTR amyloid fibrils, FAP familial amyloid polyneuropathy; Q3W every 3 weeks; Q4W every 4 weeks; SD standard deviation; siRNA-LNP small interfering RNA-lipid nanoparticles; TTR transthyretin
