Table 2.

Mean (SD) PTC299 Pharmacokinetic Parameters: Study 1—Stage 1 and Stage 2

	PTC299 Dose, mg/kg
	Stage 1					Stage 2
	0.03 Fasted	0.10 Fasted	0.30 Fasted	1.00 Fasted	3.00 Fasted	1.0 Fasted	1.0 Fed
Parameter, Units	n = 6	n = 6	n = 6	n = 6	n = 6	n = 12	n = 12
Tmax,a h	4.34	3.84	5.17	5.33	3.50	4.34	3.58
	(2.17)	(2.13)	(1.33)	(1.03)	(0.55)	(1.57)	(1.38)
Cmax,a μg/mL	0.0195	0.0632	0.214	0.682	1.87	0.663	0.933
	(0.004)	(0.016)	(0.078)	(0.166)	(0.64)	(0.223)	(0.348)
C24 h,a μg/mL	0.0	0.002	0.03	0.12	0.27	0.10	0.11
	(0.0)	(0.004)	(0.01)	(0.03)	(0.06)	(0.03)	(0.04)
AUC0–24,a μg·h/mL	0.132	0.574	2.33	8.39	19.7	7.39	8.25
	(0.044)	(0.131)	(0.814)	(2.11)	(6.16)	(2.39)	(3.56)
AUC0–t,a μg·h/mL	0.116	0.569	2.75	12.9	31.4	11.4	12.3
	(0.045)	(0.135)	(1.11)	(3.29)	(7.17)	(3.66)	(4.86)
Dose‐normalized Cmax,a μg/mL/mg/kg	0.65	0.63	0.71	0.68	0.62	0.66	0.93
	(0.13)	(0.16)	(0.26)	(0.17)	(0.21)	(0.22)	(0.35)
Dose‐normalized AUC0–24,a μg·h/mL/mg/kg	4.4	5.7	7.8	8.4	6.6	7.4	8.2
	(1.5)	(1.3)	(2.7)	(2.1)	(2.1)	(2.4)	(3.6)
t1/2, α,b h	NE	NE	2.96	3.33	3.50	3.37	3.18
	(NE)	(NE)	(0.72)	(0.67)	(1.27)	(1.13)	(1.13)
t1/2, β,b h	NE	NE	28.3	42.2	55.8	53.4	43.4
	(NE)	(NE)	(15.1)	(17.6)	(16.9)	(27.0)	(25.8)

AUC, area under the concentration–time curve; C_max, maximum compound concentration; t_1/2, half‐life; T_max, time of maximum compound concentration; NE, not estimatable.

Values represent male and female subjects combined.

^aNoncompartmental.

^bBased on 2‐compartment model fitted with time lag and weighted with 1/concentration.