Trial Outcomes & Findings for Safety and Pharmacokinetics of AT-007 in Healthy Subjects and in Adult Subjects With Classic Galactosemia (NCT NCT04117711)

Last Updated: 2024-05-30

Results Overview

To evaluate the safety and tolerability of AT-007 after administration to healthy subjects, including clinically-significant changes in clinical laboratory test results, physical examination findings, vital sign evaluations, and electrocardiogram results.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

114 participants

Primary outcome timeframe

Events after 1 day of administration.

Results posted on

2024-05-30

Participant Flow

Participant milestones

Participant milestones
Measure	Part A Cohort 1: AT-007 0.5mg/kg AT-007 administered once daily before breakfast as a single dose.	Part A Cohort 2: AT-007 5mg/kg AT-007 administered once daily before breakfast as a single dose.	Part A Cohort 3: AT-007 10mg/kg AT-007 administered once daily before breakfast as a single dose.	Part A Cohort 4: AT-007 20mg/kg AT-007 administered once daily before breakfast as a single dose.	Part A Cohort 5: AT-007 40mg/kg AT-007 administered once daily before breakfast as a single dose.	Part A: Placebo Comparator Placebo: Matching placebo will be administered once in the morning before breakfast as a single dose	Part B & C: AT-007 5mg/kg Part B and Part C are combined with results pooled for each dose level. Healthy adult volunteers received multiple doses of AT-007 once daily for 7 days.	Part B & C: AT-007 10mg/kg Part B and Part C are combined with results pooled for each dose level. Healthy adult volunteers received multiple doses of AT-007 once daily for 7 days.	Part B & C: AT-007 20mg/kg Part B and Part C are combined with results pooled for each dose level. Healthy adult volunteers received multiple doses of AT-007 once daily for 7 days.	Part B & C: AT-007 40mg/kg Part B and Part C are combined with results pooled for each dose level. Healthy adult volunteers received multiple doses of AT-007 once daily for 7 days.	Part B &C: Placebo Comparator Part B and Part C are combined with results pooled for each dose level. Healthy adult volunteers received multiple doses of AT-007 once daily for 7 days.	Part D Cohort 1: AT-007 5mg/kg AT-007 administered as a single dose once followed by a 5-day wash followed by 28 days of dosing.	Part D Cohort 1 (Placebo) Then Cohort 2 (AT-007 20mg/kg Placebo administered as a single dose once followed by a 5-day wash followed by 28 days of dosing in Cohort 1. Then, after washout, AT-007 administered as a single dose once followed by a 5-day wash followed by 28 days of dosing in Cohort 2.	Part D Cohort 1 (AT-007 5mg/kg) Then Cohort 3 (AT-007 40mg/kg) AT-007 administered as a single dose once followed by a 5-day wash followed by 28 days of dosing for each dosing cohort after a washout between cohort dosing.	Part D Cohort 1 (Placebo) Then Cohort 3 (AT-007 40mg/kg) Placebo administered as a single dose once followed by a 5-day wash followed by 28 days of dosing in Cohort 1. After washout, AT-007 administered as a single dose once followed by a 5-day wash followed by 28 days of dosing in Cohort 3.	Part D Cohort 2: AT-007 20mg/kg AT-007 administered as a single dose once followed by a 5-day wash followed by 28 days of dosing.	Part D Cohort 2 (AT-007 20mg/kg) Then Cohort 3 (AT-007 40mg/kg) AT-007 administered as a single dose once followed by a 5-day wash followed by 28 days of dosing for each cohort with washout between cohorts.	Part D Cohort 3: AT-007 40mg/kg AT-007 administered as a single dose once followed by a 5-day wash followed by 28 days of dosing.	Part D: Placebo Comparator Placebo administered as a single dose once followed by a 5-day wash followed by 28 days of dosing.	Part D Extension Cohort 1: AT-007 20mg/kg Adults with Classic Galactosemia, daily doses of AT-007 for 90 days	Part D Extension Cohort 2: 40mg/kg Adults with Classic Galactosemia, daily doses of AT-007 for 90 days	Part D Extension: Placebo Compartor Adults with Classic Galactosemia, daily doses of placebo for 90 days
Part A Cohort 1 STARTED	6	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part A Cohort 1 COMPLETED	6	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part A Cohort 1 NOT COMPLETED	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part A Cohort 2 STARTED	0	6	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part A Cohort 2 COMPLETED	0	6	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part A Cohort 2 NOT COMPLETED	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part A Cohort 3 STARTED	0	0	6	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part A Cohort 3 COMPLETED	0	0	6	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part A Cohort 3 NOT COMPLETED	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part A Cohort 4 STARTED	0	0	0	6	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part A Cohort 4 COMPLETED	0	0	0	6	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part A Cohort 4 NOT COMPLETED	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part A Cohort 5 STARTED	0	0	0	0	6	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part A Cohort 5 COMPLETED	0	0	0	0	6	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part A Cohort 5 NOT COMPLETED	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part A Placebo Comparator STARTED	0	0	0	0	0	10	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part A Placebo Comparator COMPLETED	0	0	0	0	0	10	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part A Placebo Comparator NOT COMPLETED	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part B & C AT-007 5mg/kg Cohort STARTED	0	0	0	0	0	0	6	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part B & C AT-007 5mg/kg Cohort COMPLETED	0	0	0	0	0	0	6	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part B & C AT-007 5mg/kg Cohort NOT COMPLETED	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part B & C AT-007 10mg/kg Cohort STARTED	0	0	0	0	0	0	0	8	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part B & C AT-007 10mg/kg Cohort COMPLETED	0	0	0	0	0	0	0	8	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part B & C AT-007 10mg/kg Cohort NOT COMPLETED	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part B & C AT-007 20mg/kg Cohort STARTED	0	0	0	0	0	0	0	0	10	0	0	0	0	0	0	0	0	0	0	0	0	0
Part B & C AT-007 20mg/kg Cohort COMPLETED	0	0	0	0	0	0	0	0	9	0	0	0	0	0	0	0	0	0	0	0	0	0
Part B & C AT-007 20mg/kg Cohort NOT COMPLETED	0	0	0	0	0	0	0	0	1	0	0	0	0	0	0	0	0	0	0	0	0	0
Part B & C AT-007 40mg/kg STARTED	0	0	0	0	0	0	0	0	0	9	0	0	0	0	0	0	0	0	0	0	0	0
Part B & C AT-007 40mg/kg COMPLETED	0	0	0	0	0	0	0	0	0	7	0	0	0	0	0	0	0	0	0	0	0	0
Part B & C AT-007 40mg/kg NOT COMPLETED	0	0	0	0	0	0	0	0	0	2	0	0	0	0	0	0	0	0	0	0	0	0
Part B & C Placebo Comparator STARTED	0	0	0	0	0	0	0	0	0	0	8	0	0	0	0	0	0	0	0	0	0	0
Part B & C Placebo Comparator COMPLETED	0	0	0	0	0	0	0	0	0	0	8	0	0	0	0	0	0	0	0	0	0	0
Part B & C Placebo Comparator NOT COMPLETED	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part D Cohort 1 STARTED	0	0	0	0	0	0	0	0	0	0	0	3	1	1	1	0	0	0	0	0	0	0
Part D Cohort 1 COMPLETED	0	0	0	0	0	0	0	0	0	0	0	3	1	1	1	0	0	0	0	0	0	0
Part D Cohort 1 NOT COMPLETED	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part D Cohort 2 STARTED	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	3	1	0	0	0	0	0
Part D Cohort 2 COMPLETED	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	3	1	0	0	0	0	0
Part D Cohort 2 NOT COMPLETED	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part D Cohort 3 STARTED	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	1	0	0	0	0
Part D Cohort 3 COMPLETED	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	1	0	0	0	0
Part D Cohort 3 NOT COMPLETED	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part D Placebo Comparator STARTED	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	4	0	0	0
Part D Placebo Comparator COMPLETED	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	4	0	0	0
Part D Placebo Comparator NOT COMPLETED	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part D Extension Cohort 1 STARTED	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	9	0	0
Part D Extension Cohort 1 COMPLETED	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	7	0	0
Part D Extension Cohort 1 NOT COMPLETED	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	2	0	0
Part D Extension Cohort 2 STARTED	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	2	0
Part D Extension Cohort 2 COMPLETED	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part D Extension Cohort 2 NOT COMPLETED	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	2	0
Part D Extension Placebo Comparator STARTED	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	4
Part D Extension Placebo Comparator COMPLETED	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	3
Part D Extension Placebo Comparator NOT COMPLETED	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Galactitol is NOT measurable in healthy people because they do not produce it; as such and as per protocol it was not collected and not measured in healthy adults. This means that no healthy adults had galactitol measured so it is not present for Part A, Part B, or Part C.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Part D Extension Placebo Comparator n=4 Participants Baseline characteristics of the adult subjects with Classic Galactosemia enrolled in Part D Extension were well-balanced between active and placebo across all dosing cohorts.	Part D Extension Cohort 1: AT-007 20mg/kg n=9 Participants Baseline characteristics of the adult subjects with Classic Galactosemia enrolled in Part D Extension were well-balanced between active and placebo across all dosing cohorts.	Part D Extension Cohort 2: AT-007 40mg/kg n=2 Participants Baseline characteristics of the adult subjects with Classic Galactosemia enrolled in Part D Extension were well-balanced between active and placebo across all dosing cohorts.	Total n=110 Participants Total of all reporting groups	Part A Cohort 1: AT-007 0.5mg/kg n=6 Participants Baseline characteristics of the healthy adult subjects enrolled in Part A were well-balanced between active and placebo across all dosing cohorts.	Part A Cohort 2: AT-007 5mg/kg n=6 Participants Baseline characteristics of the healthy adult subjects enrolled in Part A were well-balanced between active and placebo across all dosing cohorts.	Part A Cohort 3: AT-007 10mg/kg n=6 Participants Baseline characteristics of the healthy adult subjects enrolled in Part A were well-balanced between active and placebo across all dosing cohorts.	Part A Cohort 4: AT-007 20mg/kg n=6 Participants Baseline characteristics of the healthy adult subjects enrolled in Part A were well-balanced between active and placebo across all dosing cohorts.	Part A Cohort 5: AT-007 40mg/kg n=6 Participants Baseline characteristics of the healthy adult subjects enrolled in Part A were well-balanced between active and placebo across all dosing cohorts.	Part A Placebo Comparator n=10 Participants Baseline characteristics of the healthy adult subjects enrolled in Part A were well-balanced between active and placebo across all dosing cohorts.	Part B & C AT-007 5mg/kg Cohort n=6 Participants Baseline characteristics of the healthy adult subjects enrolled in Part B \& C were well-balanced between active and placebo across all dosing cohorts.	Part B & C AT-007 10mg/kg Cohort n=8 Participants Baseline characteristics of the healthy adult subjects enrolled in Part B \& C were well-balanced between active and placebo across all dosing cohorts.	Part B & C AT-007 20mg/kg Cohort n=10 Participants Baseline characteristics of the healthy adult subjects enrolled in Part B \& C were well-balanced between active and placebo across all dosing cohorts.	Part B & C AT-007 40mg/kg Cohort n=9 Participants Baseline characteristics of the healthy adult subjects enrolled in Part B \& C were well-balanced between active and placebo across all dosing cohorts.	Part B & C Placebo Comparator n=8 Participants Baseline characteristics of the healthy adult subjects enrolled in Part B \& C were well-balanced between active and placebo across all dosing cohorts.	Part D Cohort 1: AT-007 5mg/kg n=3 Participants Baseline characteristics of the adult subjects with Classic Galactosemia enrolled in Part D were well-balanced between active and placebo across all dosing cohorts.	Part D Cohort 1 (Placebo) Then Cohort 2 (AT-007 20mg/kg) n=1 Participants Baseline characteristics of the adult subjects with Classic Galactosemia enrolled in Part D were well-balanced between active and placebo across all dosing cohorts.	Part D Cohort 1 (AT-007 5mg/kg) Then Cohort 3 (AT-007 40mg/kg) n=1 Participants Baseline characteristics of the adult subjects with Classic Galactosemia enrolled in Part D were well-balanced between active and placebo across all dosing cohorts.	Part D Cohort 1 (Placebo) Then Cohort 3 (AT-007 40mg/kg) n=1 Participants Baseline characteristics of the adult subjects with Classic Galactosemia enrolled in Part D were well-balanced between active and placebo across all dosing cohorts.	Part D Cohort 2: AT-007 20mg/kg n=2 Participants Baseline characteristics of the adult subjects with Classic Galactosemia enrolled in Part D were well-balanced between active and placebo across all dosing cohorts.	Part D Cohort 2 (AT-007 20mg/kg) Then Chort 3 (AT-007 40mg/kg) n=1 Participants Baseline characteristics of the adult subjects with Classic Galactosemia enrolled in Part D were well-balanced between active and placebo across all dosing cohorts.	Part D Cohort 3: AT-007 40mg/kg n=1 Participants Baseline characteristics of the adult subjects with Classic Galactosemia enrolled in Part D were well-balanced between active and placebo across all dosing cohorts.	Part D Placebo Comparator n=4 Participants Baseline characteristics of the adult subjects with Classic Galactosemia enrolled in Part D were well-balanced between active and placebo across all dosing cohorts.
Age, Continuous	29.5 years n=4 Participants	22.0 years n=9 Participants	36.0 years n=2 Participants	NA years n=110 Participants	42.5 years n=6 Participants	54.0 years n=6 Participants	35.0 years n=6 Participants	44.0 years n=6 Participants	31.5 years n=6 Participants	54.5 years n=10 Participants	32.5 years n=6 Participants	35.5 years n=8 Participants	35.0 years n=10 Participants	27.0 years n=9 Participants	37 years n=8 Participants	19.5 years n=3 Participants	37 years n=1 Participants	50 years n=1 Participants	19 years n=1 Participants	20.5 years n=2 Participants	28 years n=1 Participants	43 years n=1 Participants	32 years n=4 Participants
Sex: Female, Male Female	3 Participants n=4 Participants	3 Participants n=9 Participants	0 Participants n=2 Participants	56 Participants n=110 Participants	2 Participants n=6 Participants	5 Participants n=6 Participants	2 Participants n=6 Participants	4 Participants n=6 Participants	4 Participants n=6 Participants	8 Participants n=10 Participants	5 Participants n=6 Participants	3 Participants n=8 Participants	4 Participants n=10 Participants	5 Participants n=9 Participants	3 Participants n=8 Participants	1 Participants n=3 Participants	0 Participants n=1 Participants	0 Participants n=1 Participants	1 Participants n=1 Participants	1 Participants n=2 Participants	0 Participants n=1 Participants	0 Participants n=1 Participants	2 Participants n=4 Participants
Sex: Female, Male Male	1 Participants n=4 Participants	6 Participants n=9 Participants	2 Participants n=2 Participants	54 Participants n=110 Participants	4 Participants n=6 Participants	1 Participants n=6 Participants	4 Participants n=6 Participants	2 Participants n=6 Participants	2 Participants n=6 Participants	2 Participants n=10 Participants	1 Participants n=6 Participants	5 Participants n=8 Participants	6 Participants n=10 Participants	4 Participants n=9 Participants	5 Participants n=8 Participants	2 Participants n=3 Participants	1 Participants n=1 Participants	1 Participants n=1 Participants	0 Participants n=1 Participants	1 Participants n=2 Participants	1 Participants n=1 Participants	1 Participants n=1 Participants	2 Participants n=4 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=4 Participants	0 Participants n=9 Participants	0 Participants n=2 Participants	43 Participants n=110 Participants	1 Participants n=6 Participants	5 Participants n=6 Participants	2 Participants n=6 Participants	2 Participants n=6 Participants	3 Participants n=6 Participants	9 Participants n=10 Participants	4 Participants n=6 Participants	3 Participants n=8 Participants	6 Participants n=10 Participants	5 Participants n=9 Participants	3 Participants n=8 Participants	0 Participants n=3 Participants	0 Participants n=1 Participants	0 Participants n=1 Participants	0 Participants n=1 Participants	0 Participants n=2 Participants	0 Participants n=1 Participants	0 Participants n=1 Participants	0 Participants n=4 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	4 Participants n=4 Participants	9 Participants n=9 Participants	2 Participants n=2 Participants	67 Participants n=110 Participants	5 Participants n=6 Participants	1 Participants n=6 Participants	4 Participants n=6 Participants	4 Participants n=6 Participants	3 Participants n=6 Participants	1 Participants n=10 Participants	2 Participants n=6 Participants	5 Participants n=8 Participants	4 Participants n=10 Participants	4 Participants n=9 Participants	5 Participants n=8 Participants	3 Participants n=3 Participants	1 Participants n=1 Participants	1 Participants n=1 Participants	1 Participants n=1 Participants	2 Participants n=2 Participants	1 Participants n=1 Participants	1 Participants n=1 Participants	4 Participants n=4 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=4 Participants	0 Participants n=9 Participants	0 Participants n=2 Participants	0 Participants n=110 Participants	0 Participants n=6 Participants	0 Participants n=6 Participants	0 Participants n=6 Participants	0 Participants n=6 Participants	0 Participants n=6 Participants	0 Participants n=10 Participants	0 Participants n=6 Participants	0 Participants n=8 Participants	0 Participants n=10 Participants	0 Participants n=9 Participants	0 Participants n=8 Participants	0 Participants n=3 Participants	0 Participants n=1 Participants	0 Participants n=1 Participants	0 Participants n=1 Participants	0 Participants n=2 Participants	0 Participants n=1 Participants	0 Participants n=1 Participants	0 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=4 Participants	0 Participants n=9 Participants	0 Participants n=2 Participants	2 Participants n=110 Participants	1 Participants n=6 Participants	0 Participants n=6 Participants	0 Participants n=6 Participants	0 Participants n=6 Participants	0 Participants n=6 Participants	0 Participants n=10 Participants	0 Participants n=6 Participants	0 Participants n=8 Participants	1 Participants n=10 Participants	0 Participants n=9 Participants	0 Participants n=8 Participants	0 Participants n=3 Participants	0 Participants n=1 Participants	0 Participants n=1 Participants	0 Participants n=1 Participants	0 Participants n=2 Participants	0 Participants n=1 Participants	0 Participants n=1 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Asian	0 Participants n=4 Participants	0 Participants n=9 Participants	0 Participants n=2 Participants	1 Participants n=110 Participants	0 Participants n=6 Participants	0 Participants n=6 Participants	1 Participants n=6 Participants	0 Participants n=6 Participants	0 Participants n=6 Participants	0 Participants n=10 Participants	0 Participants n=6 Participants	0 Participants n=8 Participants	0 Participants n=10 Participants	0 Participants n=9 Participants	0 Participants n=8 Participants	0 Participants n=3 Participants	0 Participants n=1 Participants	0 Participants n=1 Participants	0 Participants n=1 Participants	0 Participants n=2 Participants	0 Participants n=1 Participants	0 Participants n=1 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=4 Participants	0 Participants n=9 Participants	0 Participants n=2 Participants	0 Participants n=110 Participants	0 Participants n=6 Participants	0 Participants n=6 Participants	0 Participants n=6 Participants	0 Participants n=6 Participants	0 Participants n=6 Participants	0 Participants n=10 Participants	0 Participants n=6 Participants	0 Participants n=8 Participants	0 Participants n=10 Participants	0 Participants n=9 Participants	0 Participants n=8 Participants	0 Participants n=3 Participants	0 Participants n=1 Participants	0 Participants n=1 Participants	0 Participants n=1 Participants	0 Participants n=2 Participants	0 Participants n=1 Participants	0 Participants n=1 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Black or African American	0 Participants n=4 Participants	0 Participants n=9 Participants	0 Participants n=2 Participants	24 Participants n=110 Participants	1 Participants n=6 Participants	0 Participants n=6 Participants	2 Participants n=6 Participants	3 Participants n=6 Participants	2 Participants n=6 Participants	1 Participants n=10 Participants	1 Participants n=6 Participants	4 Participants n=8 Participants	3 Participants n=10 Participants	2 Participants n=9 Participants	5 Participants n=8 Participants	0 Participants n=3 Participants	0 Participants n=1 Participants	0 Participants n=1 Participants	0 Participants n=1 Participants	0 Participants n=2 Participants	0 Participants n=1 Participants	0 Participants n=1 Participants	0 Participants n=4 Participants
Race (NIH/OMB) White	4 Participants n=4 Participants	9 Participants n=9 Participants	2 Participants n=2 Participants	82 Participants n=110 Participants	4 Participants n=6 Participants	6 Participants n=6 Participants	3 Participants n=6 Participants	3 Participants n=6 Participants	3 Participants n=6 Participants	9 Participants n=10 Participants	5 Participants n=6 Participants	4 Participants n=8 Participants	6 Participants n=10 Participants	7 Participants n=9 Participants	3 Participants n=8 Participants	3 Participants n=3 Participants	1 Participants n=1 Participants	1 Participants n=1 Participants	1 Participants n=1 Participants	2 Participants n=2 Participants	1 Participants n=1 Participants	1 Participants n=1 Participants	4 Participants n=4 Participants
Race (NIH/OMB) More than one race	0 Participants n=4 Participants	0 Participants n=9 Participants	0 Participants n=2 Participants	1 Participants n=110 Participants	0 Participants n=6 Participants	0 Participants n=6 Participants	0 Participants n=6 Participants	0 Participants n=6 Participants	1 Participants n=6 Participants	0 Participants n=10 Participants	0 Participants n=6 Participants	0 Participants n=8 Participants	0 Participants n=10 Participants	0 Participants n=9 Participants	0 Participants n=8 Participants	0 Participants n=3 Participants	0 Participants n=1 Participants	0 Participants n=1 Participants	0 Participants n=1 Participants	0 Participants n=2 Participants	0 Participants n=1 Participants	0 Participants n=1 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=4 Participants	0 Participants n=9 Participants	0 Participants n=2 Participants	0 Participants n=110 Participants	0 Participants n=6 Participants	0 Participants n=6 Participants	0 Participants n=6 Participants	0 Participants n=6 Participants	0 Participants n=6 Participants	0 Participants n=10 Participants	0 Participants n=6 Participants	0 Participants n=8 Participants	0 Participants n=10 Participants	0 Participants n=9 Participants	0 Participants n=8 Participants	0 Participants n=3 Participants	0 Participants n=1 Participants	0 Participants n=1 Participants	0 Participants n=1 Participants	0 Participants n=2 Participants	0 Participants n=1 Participants	0 Participants n=1 Participants	0 Participants n=4 Participants
Region of Enrollment United States	4 participants n=4 Participants	9 participants n=9 Participants	2 participants n=2 Participants	110 participants n=110 Participants	6 participants n=6 Participants	6 participants n=6 Participants	6 participants n=6 Participants	6 participants n=6 Participants	6 participants n=6 Participants	10 participants n=10 Participants	6 participants n=6 Participants	8 participants n=8 Participants	10 participants n=10 Participants	9 participants n=9 Participants	8 participants n=8 Participants	3 participants n=3 Participants	1 participants n=1 Participants	1 participants n=1 Participants	1 participants n=1 Participants	2 participants n=2 Participants	1 participants n=1 Participants	1 participants n=1 Participants	4 participants n=4 Participants
Galactitol	2612.5 ng/mL n=4 Participants • Galactitol is NOT measurable in healthy people because they do not produce it; as such and as per protocol it was not collected and not measured in healthy adults. This means that no healthy adults had galactitol measured so it is not present for Part A, Part B, or Part C.	2300 ng/mL n=9 Participants • Galactitol is NOT measurable in healthy people because they do not produce it; as such and as per protocol it was not collected and not measured in healthy adults. This means that no healthy adults had galactitol measured so it is not present for Part A, Part B, or Part C.	NA ng/mL n=2 Participants • Galactitol is NOT measurable in healthy people because they do not produce it; as such and as per protocol it was not collected and not measured in healthy adults. This means that no healthy adults had galactitol measured so it is not present for Part A, Part B, or Part C.	2434.4 ng/mL n=29 Participants • Galactitol is NOT measurable in healthy people because they do not produce it; as such and as per protocol it was not collected and not measured in healthy adults. This means that no healthy adults had galactitol measured so it is not present for Part A, Part B, or Part C.	—	—	—	—	—	—	—	—	—	—	—	2490 ng/mL n=3 Participants • Galactitol is NOT measurable in healthy people because they do not produce it; as such and as per protocol it was not collected and not measured in healthy adults. This means that no healthy adults had galactitol measured so it is not present for Part A, Part B, or Part C.	2380 ng/mL n=1 Participants • Galactitol is NOT measurable in healthy people because they do not produce it; as such and as per protocol it was not collected and not measured in healthy adults. This means that no healthy adults had galactitol measured so it is not present for Part A, Part B, or Part C.	2140 ng/mL n=1 Participants • Galactitol is NOT measurable in healthy people because they do not produce it; as such and as per protocol it was not collected and not measured in healthy adults. This means that no healthy adults had galactitol measured so it is not present for Part A, Part B, or Part C.	2360 ng/mL n=1 Participants • Galactitol is NOT measurable in healthy people because they do not produce it; as such and as per protocol it was not collected and not measured in healthy adults. This means that no healthy adults had galactitol measured so it is not present for Part A, Part B, or Part C.	2170 ng/mL n=2 Participants • Galactitol is NOT measurable in healthy people because they do not produce it; as such and as per protocol it was not collected and not measured in healthy adults. This means that no healthy adults had galactitol measured so it is not present for Part A, Part B, or Part C.	2340 ng/mL n=1 Participants • Galactitol is NOT measurable in healthy people because they do not produce it; as such and as per protocol it was not collected and not measured in healthy adults. This means that no healthy adults had galactitol measured so it is not present for Part A, Part B, or Part C.	2810 ng/mL n=1 Participants • Galactitol is NOT measurable in healthy people because they do not produce it; as such and as per protocol it was not collected and not measured in healthy adults. This means that no healthy adults had galactitol measured so it is not present for Part A, Part B, or Part C.	2480 ng/mL n=4 Participants • Galactitol is NOT measurable in healthy people because they do not produce it; as such and as per protocol it was not collected and not measured in healthy adults. This means that no healthy adults had galactitol measured so it is not present for Part A, Part B, or Part C.

PRIMARY outcome

Timeframe: Events after 1 day of administration.

Population: All subjects dosed were analyzed.

Outcome measures

Outcome measures
Measure	Part B & C Cohort 4: AT-007 40mg/kg Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part D Cohort 1: AT-007 5mg/kg Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Cohort 2: AT-007 20mg/kg Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Cohort 3: AT-007 40mg/kg Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 30 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 60 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part A Cohort 1: AT-007 0.5mg/kg n=6 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 2: AT-007 5mg/kg n=6 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 3: AT-007 10mg/kg n=6 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 4: AT-007 20mg/kg n=6 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 5: AT-007 40mg/kg n=6 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A: Placebo Comparator n=10 Participants Single doses of orally administered matched-placebo were administered to Healthy Adult Subjects and all TEAEs recorded.	Part B & C Cohort 2: AT-007 10mg/kg Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part B & C Cohort 3: AT-007 20mg/kg Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 90 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part D Extension Cohort 2: AT-007 40mg/kg DAY 30 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia
Number of Participants With Treatment-emergent Adverse Events	—	—	—	—	—	—	1 Participants	0 Participants	0 Participants	2 Participants	1 Participants	2 Participants	—	—	—	—

PRIMARY outcome

Timeframe: 7 Days after Dosing

Population: All subjects dosed were analyzed

To evaluate the safety and tolerability of AT-007 after multiple ascending doses are administered to healthy subjects, including clinically-significant changes in clinical laboratory test results, physical examination findings, vital sign evaluations, and electrocardiogram results. Part B and Part C are combined.

Outcome measures

Outcome measures
Measure	Part B & C Cohort 4: AT-007 40mg/kg Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part D Cohort 1: AT-007 5mg/kg Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Cohort 2: AT-007 20mg/kg Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Cohort 3: AT-007 40mg/kg Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 30 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 60 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part A Cohort 1: AT-007 0.5mg/kg n=6 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 2: AT-007 5mg/kg n=8 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 3: AT-007 10mg/kg n=10 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 4: AT-007 20mg/kg n=9 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 5: AT-007 40mg/kg n=8 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A: Placebo Comparator Single doses of orally administered matched-placebo were administered to Healthy Adult Subjects and all TEAEs recorded.	Part B & C Cohort 2: AT-007 10mg/kg Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part B & C Cohort 3: AT-007 20mg/kg Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 90 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part D Extension Cohort 2: AT-007 40mg/kg DAY 30 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia
Number of Participants With Treatment-emergent Adverse Events	—	—	—	—	—	—	1 Participants	2 Participants	3 Participants	1 Participants	2 Participants	—	—	—	—	—

PRIMARY outcome

Timeframe: 28 Days of Dosing

Population: All subjects dosed were analyzed.

To evaluate the safety and tolerability of AT-007 after multiple doses administered to Classic Galactosemia Patients, including clinically-significant changes in clinical laboratory test results, physical examination findings, vital sign evaluations, and electrocardiogram results.

Outcome measures

Outcome measures
Measure	Part B & C Cohort 4: AT-007 40mg/kg Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part D Cohort 1: AT-007 5mg/kg Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Cohort 2: AT-007 20mg/kg Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Cohort 3: AT-007 40mg/kg Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 30 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 60 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part A Cohort 1: AT-007 0.5mg/kg n=4 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 2: AT-007 5mg/kg n=4 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 3: AT-007 10mg/kg n=4 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 4: AT-007 20mg/kg n=6 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 5: AT-007 40mg/kg Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A: Placebo Comparator Single doses of orally administered matched-placebo were administered to Healthy Adult Subjects and all TEAEs recorded.	Part B & C Cohort 2: AT-007 10mg/kg Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part B & C Cohort 3: AT-007 20mg/kg Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 90 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part D Extension Cohort 2: AT-007 40mg/kg DAY 30 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia
Number of Participants With Treatment-emergent Adverse Events	—	—	—	—	—	—	3 Participants	1 Participants	2 Participants	2 Participants	—	—	—	—	—	—

PRIMARY outcome

Timeframe: 90 Days after Dosing

Population: All subjects dosed were analyzed.

To evaluate the safety and tolerability of AT-007 administered to Classic Galactosemia Patients, including clinically-significant changes in clinical laboratory test results, physical examination findings, vital sign evaluations, and electrocardiogram results.

Outcome measures

Outcome measures
Measure	Part B & C Cohort 4: AT-007 40mg/kg Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part D Cohort 1: AT-007 5mg/kg Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Cohort 2: AT-007 20mg/kg Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Cohort 3: AT-007 40mg/kg Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 30 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 60 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part A Cohort 1: AT-007 0.5mg/kg n=9 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 2: AT-007 5mg/kg n=2 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 3: AT-007 10mg/kg n=4 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 4: AT-007 20mg/kg Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 5: AT-007 40mg/kg Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A: Placebo Comparator Single doses of orally administered matched-placebo were administered to Healthy Adult Subjects and all TEAEs recorded.	Part B & C Cohort 2: AT-007 10mg/kg Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part B & C Cohort 3: AT-007 20mg/kg Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 90 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part D Extension Cohort 2: AT-007 40mg/kg DAY 30 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia
Number of Participants With Treatment-emergent Adverse Events	—	—	—	—	—	—	7 Participants	1 Participants	2 Participants	—	—	—	—	—	—	—

SECONDARY outcome

Timeframe: Part A: Sampling- predose, 0.5, 1, 1.5, 2, 4, 6, 8, 12, 24, 36, 48 hrs Parts B, C, D: Day of Last Dose for each Part- predose, 0.5, 1, 1.5, 2, 4, 6, 8, 12, 24, 36, 48 hrs D Extension: Day 30, 60, 90- predose, 0.5, 1, 1.5, 2, 4, 6, 8, 12, 24, 36, 48 hrs

Population: Part D Extension Cohort 2 40mg/kg ended early at Day 30 as recommended by Monitoring Committee. As such, only 1 subject has D30 data available; the other subject did not have enough blood samples to calculate.

Maximum (peak) plasma drug concentration

Outcome measures

Outcome measures
Measure	Part B & C Cohort 4: AT-007 40mg/kg n=7 Participants Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part D Cohort 1: AT-007 5mg/kg n=4 Participants Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Cohort 2: AT-007 20mg/kg n=4 Participants Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Cohort 3: AT-007 40mg/kg n=4 Participants Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 30 n=9 Participants Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 60 n=8 Participants Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part A Cohort 1: AT-007 0.5mg/kg n=6 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 2: AT-007 5mg/kg n=6 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 3: AT-007 10mg/kg n=6 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 4: AT-007 20mg/kg n=6 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 5: AT-007 40mg/kg n=6 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A: Placebo Comparator n=6 Participants Single doses of orally administered matched-placebo were administered to Healthy Adult Subjects and all TEAEs recorded.	Part B & C Cohort 2: AT-007 10mg/kg n=8 Participants Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part B & C Cohort 3: AT-007 20mg/kg n=9 Participants Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 90 n=6 Participants Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part D Extension Cohort 2: AT-007 40mg/kg DAY 30 n=1 Participants Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia
Cmax of AT-007	72.8 microgram/mL Geometric Coefficient of Variation 44.9	13.7 microgram/mL Geometric Coefficient of Variation 34.0	39.9 microgram/mL Geometric Coefficient of Variation 70.8	67.9 microgram/mL Geometric Coefficient of Variation 68.0	48.6 microgram/mL Geometric Coefficient of Variation 21.8	48.0 microgram/mL Geometric Coefficient of Variation 24.4	1.53 microgram/mL Geometric Coefficient of Variation 55.2	8.92 microgram/mL Geometric Coefficient of Variation 31.4	15.2 microgram/mL Geometric Coefficient of Variation 49.5	27 microgram/mL Geometric Coefficient of Variation 103	57.4 microgram/mL Geometric Coefficient of Variation 23.2	15.2 microgram/mL Geometric Coefficient of Variation 45.0	23.0 microgram/mL Geometric Coefficient of Variation 39.0	47.8 microgram/mL Geometric Coefficient of Variation 35.4	44.1 microgram/mL Geometric Coefficient of Variation 36.5	70.8 microgram/mL Geometric Coefficient of Variation NA Only 1 subject so value cannot be calculated.

SECONDARY outcome

Time to reach maximum (peak) plasma drug concentration

Outcome measures

Outcome measures
Measure	Part B & C Cohort 4: AT-007 40mg/kg n=7 Participants Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part D Cohort 1: AT-007 5mg/kg n=4 Participants Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Cohort 2: AT-007 20mg/kg n=4 Participants Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Cohort 3: AT-007 40mg/kg n=4 Participants Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 30 n=9 Participants Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 60 n=8 Participants Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part A Cohort 1: AT-007 0.5mg/kg n=6 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 2: AT-007 5mg/kg n=6 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 3: AT-007 10mg/kg n=6 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 4: AT-007 20mg/kg n=6 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 5: AT-007 40mg/kg n=6 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A: Placebo Comparator n=6 Participants Single doses of orally administered matched-placebo were administered to Healthy Adult Subjects and all TEAEs recorded.	Part B & C Cohort 2: AT-007 10mg/kg n=8 Participants Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part B & C Cohort 3: AT-007 20mg/kg n=9 Participants Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 90 n=6 Participants Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part D Extension Cohort 2: AT-007 40mg/kg DAY 30 n=1 Participants Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia
Tmax of AT-007	4.00 hours Interval 4.0 to 6.07	4.00 hours Interval 2.0 to 6.0	4.96 hours Interval 4.0 to 8.0	4.00 hours Interval 1.92 to 8.0	4.03 hours Interval 1.97 to 8.02	4.01 hours Interval 2.0 to 7.97	4.00 hours Interval 4.0 to 6.02	4.00 hours Interval 2.0 to 4.0	4.00 hours Interval 4.0 to 4.0	4.00 hours Interval 4.0 to 24.1	4.00 hours Interval 4.0 to 8.0	4.00 hours Interval 4.0 to 8.0	3.96 hours Interval 2.0 to 8.0	4.00 hours Interval 4.0 to 8.0	4.00 hours Interval 2.0 to 4.33	4.17 hours Interval 4.17 to 4.17

SECONDARY outcome

Timeframe: Part A: Sequential sampling at predose, 0.5, 1, 1.5, 2, 4, 6, 8, 12, 24, 36, and 48 hours. Parts B, C, D: Determined using Day of Last Dose for the respective Part; predose, 0.5, 1, 1.5, 2, 4, 6, 8, 12, 24, 36, and 48 hours.

Population: For Part D Extension, t1/2 was not performed so these cohorts are not presented.

Terminal elimination half life

Outcome measures

Outcome measures
Measure	Part B & C Cohort 4: AT-007 40mg/kg n=7 Participants Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part D Cohort 1: AT-007 5mg/kg n=3 Participants Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Cohort 2: AT-007 20mg/kg n=3 Participants Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Cohort 3: AT-007 40mg/kg n=4 Participants Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 30 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 60 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part A Cohort 1: AT-007 0.5mg/kg n=6 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 2: AT-007 5mg/kg n=4 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 3: AT-007 10mg/kg n=6 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 4: AT-007 20mg/kg n=1 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 5: AT-007 40mg/kg n=2 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A: Placebo Comparator n=6 Participants Single doses of orally administered matched-placebo were administered to Healthy Adult Subjects and all TEAEs recorded.	Part B & C Cohort 2: AT-007 10mg/kg n=8 Participants Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part B & C Cohort 3: AT-007 20mg/kg n=9 Participants Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 90 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part D Extension Cohort 2: AT-007 40mg/kg DAY 30 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia
t1/2 of AT-007	9.96 hours Geometric Coefficient of Variation 27.4	13.9 hours Geometric Coefficient of Variation 11.0	16.8 hours Geometric Coefficient of Variation 56.6	16.1 hours Geometric Coefficient of Variation 21.1	—	—	13.7 hours Geometric Coefficient of Variation 45.3	22.0 hours Geometric Coefficient of Variation 92.6	10.3 hours Geometric Coefficient of Variation 31.7	8.42 hours Geometric Coefficient of Variation NA Only 1 subject data available so cannot calculate.	8.2 hours Geometric Coefficient of Variation 7.55	13.1 hours Geometric Coefficient of Variation 39.5	15.7 hours Geometric Coefficient of Variation 79.9	9.76 hours Geometric Coefficient of Variation 22.4	—	—

SECONDARY outcome

Timeframe: Part A: Sequential sampling at predose, 0.5, 1, 1.5, 2, 4, 6, 8, 12, 24, 36, and 48 hours. Parts B, C, D: Determined using Day of Last Dose for the respective Part at predose, 0.5, 1, 1.5, 2, 4, 6, 8, 12, 24, 36, and 48 hours.

Population: For Part D Extension, AUClast was not performed so these cohorts are not presented.

Area under the plasma concentration-time curve from time zero to time of last quantifiable concentration

Outcome measures

Outcome measures
Measure	Part B & C Cohort 4: AT-007 40mg/kg n=7 Participants Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part D Cohort 1: AT-007 5mg/kg n=4 Participants Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Cohort 2: AT-007 20mg/kg n=4 Participants Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Cohort 3: AT-007 40mg/kg n=4 Participants Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 30 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 60 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part A Cohort 1: AT-007 0.5mg/kg n=6 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 2: AT-007 5mg/kg n=6 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 3: AT-007 10mg/kg n=6 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 4: AT-007 20mg/kg n=6 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 5: AT-007 40mg/kg n=6 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A: Placebo Comparator n=6 Participants Single doses of orally administered matched-placebo were administered to Healthy Adult Subjects and all TEAEs recorded.	Part B & C Cohort 2: AT-007 10mg/kg n=8 Participants Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part B & C Cohort 3: AT-007 20mg/kg n=9 Participants Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 90 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part D Extension Cohort 2: AT-007 40mg/kg DAY 30 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia
AUClast of AT-007	1110 microgram*hour/mL Geometric Coefficient of Variation 50.2	172 microgram*hour/mL Geometric Coefficient of Variation 25.3	698 microgram*hour/mL Geometric Coefficient of Variation 21.8	1006 microgram*hour/mL Geometric Coefficient of Variation 62.3	—	—	12.6 microgram*hour/mL Geometric Coefficient of Variation 44.7	89.0 microgram*hour/mL Geometric Coefficient of Variation 18.5	158 microgram*hour/mL Geometric Coefficient of Variation 35.0	352 microgram*hour/mL Geometric Coefficient of Variation 75.0	624 microgram*hour/mL Geometric Coefficient of Variation 28.0	181 microgram*hour/mL Geometric Coefficient of Variation 44.3	311 microgram*hour/mL Geometric Coefficient of Variation 47.4	592 microgram*hour/mL Geometric Coefficient of Variation 26.9	—	—

SECONDARY outcome

Timeframe: Part A: Sequential sampling at predose, 0.5, 1, 1.5, 2, 4, 6, 8, 12, 24, 36, and 48 hours. Part D: Determined using Day 1 with sampling at predose, 0.5, 1, 1.5, 2, 4, 6, 8, 12, 24, 36, and 48 hours.

Population: AUCinf was only calculated for all cohorts of Part A and on Day 1 of Part D. It was not calculated for Parts B, C, or D Extension so those cohorts are not presented.

Area under the plasma concentration-time curve from time zero extrapolated to infinity

Outcome measures

Outcome measures
Measure	Part B & C Cohort 4: AT-007 40mg/kg Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part D Cohort 1: AT-007 5mg/kg Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Cohort 2: AT-007 20mg/kg Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Cohort 3: AT-007 40mg/kg Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 30 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 60 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part A Cohort 1: AT-007 0.5mg/kg n=5 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 2: AT-007 5mg/kg n=2 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 3: AT-007 10mg/kg n=6 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 4: AT-007 20mg/kg n=1 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 5: AT-007 40mg/kg n=2 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A: Placebo Comparator n=4 Participants Single doses of orally administered matched-placebo were administered to Healthy Adult Subjects and all TEAEs recorded.	Part B & C Cohort 2: AT-007 10mg/kg n=3 Participants Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part B & C Cohort 3: AT-007 20mg/kg n=3 Participants Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 90 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part D Extension Cohort 2: AT-007 40mg/kg DAY 30 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia
AUCinf of AT-007	—	—	—	—	—	—	14.3 microgram*hour/mL Geometric Coefficient of Variation 43.7	81.8 microgram*hour/mL Geometric Coefficient of Variation 15.1	169 microgram*hour/mL Geometric Coefficient of Variation 35.3	163 microgram*hour/mL Geometric Coefficient of Variation NA Only 1 subject had data available so cannot calculate this.	655 microgram*hour/mL Geometric Coefficient of Variation 0.45	109 microgram*hour/mL Geometric Coefficient of Variation 28.2	518 microgram*hour/mL Geometric Coefficient of Variation 65.2	1327 microgram*hour/mL Geometric Coefficient of Variation 17.2	—	—

SECONDARY outcome

Timeframe: Part D: Samples at predose, 0.5, 1, 1.5, 2, 4, 6, 8, 12, 24, 36, and 48 hours on days 1, 12, 32. Part D Extension: Samples at predose, 0.5, 1, 1.5, 2, 4, 6, 8, 12, 24, 36, and 48 hours on days 1, 30, 60, & 90.

Population: Part D Extension Cohort 2 40mg/kg ended early at Day 30 as recommended by Monitoring Committee. As such, there are no results for this outcome measure for the Part D Extension Cohort 2 40mg/kg.

Disease-Specific Biomarker in Classic Galactosemia Patients

Outcome measures

Outcome measures
Measure	Part B & C Cohort 4: AT-007 40mg/kg Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part D Cohort 1: AT-007 5mg/kg Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Cohort 2: AT-007 20mg/kg Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Cohort 3: AT-007 40mg/kg Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 30 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 60 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part A Cohort 1: AT-007 0.5mg/kg n=4 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 2: AT-007 5mg/kg n=4 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 3: AT-007 10mg/kg n=4 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 4: AT-007 20mg/kg n=5 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 5: AT-007 40mg/kg n=9 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A: Placebo Comparator n=4 Participants Single doses of orally administered matched-placebo were administered to Healthy Adult Subjects and all TEAEs recorded.	Part B & C Cohort 2: AT-007 10mg/kg Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part B & C Cohort 3: AT-007 20mg/kg Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 90 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part D Extension Cohort 2: AT-007 40mg/kg DAY 30 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia
Maximal Galactitol Reduction in Plasma	—	—	—	—	—	—	-475 maximal change from baseline in ng/mL Standard Deviation 305	-1175 maximal change from baseline in ng/mL Standard Deviation 239	-1259 maximal change from baseline in ng/mL Standard Deviation 220	-393 maximal change from baseline in ng/mL Standard Deviation 239	-974.4 maximal change from baseline in ng/mL Standard Deviation 322.42	-344.0 maximal change from baseline in ng/mL Standard Deviation 449.67	—	—	—	—

SECONDARY outcome

Timeframe: Part D: Day 32. Part D Extension: Days 30, 60, & 90.

Disease-Specific Biomarker in Classic Galactosemia Patients

Outcome measures

Outcome measures
Measure	Part B & C Cohort 4: AT-007 40mg/kg n=3 Participants Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part D Cohort 1: AT-007 5mg/kg n=3 Participants Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Cohort 2: AT-007 20mg/kg Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Cohort 3: AT-007 40mg/kg Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 30 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 60 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part A Cohort 1: AT-007 0.5mg/kg n=4 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 2: AT-007 5mg/kg n=4 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 3: AT-007 10mg/kg n=4 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 4: AT-007 20mg/kg n=5 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 5: AT-007 40mg/kg n=9 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A: Placebo Comparator n=7 Participants Single doses of orally administered matched-placebo were administered to Healthy Adult Subjects and all TEAEs recorded.	Part B & C Cohort 2: AT-007 10mg/kg n=6 Participants Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part B & C Cohort 3: AT-007 20mg/kg n=4 Participants Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 90 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part D Extension Cohort 2: AT-007 40mg/kg DAY 30 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia
Galactose Concentration in Plasma	98.67 percent change from baseline Standard Deviation 132.115	130.00 percent change from baseline Standard Deviation 101.059	—	—	—	—	8.94 percent change from baseline Standard Deviation 8.71	-27.8 percent change from baseline Standard Deviation 29.3	25.4 percent change from baseline Standard Deviation 69.5	39.8 percent change from baseline Standard Deviation 62.7	-153.78 percent change from baseline Standard Deviation 381.475	52.29 percent change from baseline Standard Deviation 170.753	30.42 percent change from baseline Standard Deviation 102.15	139.75 percent change from baseline Standard Deviation 273.418	—	—

SECONDARY outcome

Timeframe: Part D: Day 32. Part D Extension: Days 30, 60, & 90.

Disease-Specific Biomarker in Classic Galactosemia Patients

Outcome measures

Outcome measures
Measure	Part B & C Cohort 4: AT-007 40mg/kg n=3 Participants Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part D Cohort 1: AT-007 5mg/kg n=3 Participants Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Cohort 2: AT-007 20mg/kg Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Cohort 3: AT-007 40mg/kg Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Day 32 is presented as it reflects steady state.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 30 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 60 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part A Cohort 1: AT-007 0.5mg/kg n=4 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 2: AT-007 5mg/kg n=4 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 3: AT-007 10mg/kg n=4 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 4: AT-007 20mg/kg n=5 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A Cohort 5: AT-007 40mg/kg n=9 Participants Single doses of orally administered AT-007 were administered to Healthy Adult Subjects and all TEAEs recorded.	Part A: Placebo Comparator n=7 Participants Single doses of orally administered matched-placebo were administered to Healthy Adult Subjects and all TEAEs recorded.	Part B & C Cohort 2: AT-007 10mg/kg n=6 Participants Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part B & C Cohort 3: AT-007 20mg/kg n=4 Participants Following multiple QD dose administration of AT-007 for 7 days in Healthy Adult Subjects, Cmax was calculated. Day 7 is presented as it reflects steady state.	Part D Extension Cohort 1: AT-007 20mg/kg DAY 90 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia, Cmax was calculated. Days 30, 60, and 90 are presented to reflect values over time.	Part D Extension Cohort 2: AT-007 40mg/kg DAY 30 Following multiple QD dose administration of AT-007 in Adults with Classic Galactosemia
Galactose-1-Phosphate (Gal-1p) Concentration in Plasma	14.03 percent change from baseline Standard Deviation 13.181	11.97 percent change from baseline Standard Deviation 21.511	—	—	—	—	5.71 percent change from baseline Standard Deviation 15.7	31.5 percent change from baseline Standard Deviation 6.78	7.86 percent change from baseline Standard Deviation 21.0	25.2 percent change from baseline Standard Deviation 8.74	33.1 percent change from baseline Standard Deviation 51.123	16.04 percent change from baseline Standard Deviation 43.218	4.87 percent change from baseline Standard Deviation 24.917	8.63 percent change from baseline Standard Deviation 24.473	—	—

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Part A Cohort 1: AT-007 0.5mg/kg n=6 participants at risk Single ascending doses of orally administered AT-007 were administered to healthy adult subjects.	Part A Cohort 2: AT-007 5mg/kg n=6 participants at risk Single ascending doses of orally administered AT-007 were administered to healthy adult subjects.	Part A Cohort 3: AT-007 10mg/kg n=6 participants at risk Single ascending doses of orally administered AT-007 were administered to healthy adult subjects.	Part A Cohort 4: AT-007 20mg/kg n=6 participants at risk Single ascending doses of orally administered AT-007 were administered to healthy adult subjects.	Part A Cohort 5: 40mg/kg n=6 participants at risk Single ascending doses of orally administered AT-007 were administered to healthy adult subjects.	Part A Placebo Comparator n=10 participants at risk Single ascending doses of orally administered matched placebo were administered to healthy adult subjects.	Part B & C Cohort 1: AT-007 5mg/kg n=6 participants at risk Multiple doses of orally administered AT-007 were administered to healthy adult subjects.	Part B & C Cohort 2: AT-007 10mg/kg n=8 participants at risk Multiple doses of orally administered AT-007 were administered to healthy adult subjects.	Part B & C Cohort 3: AT-007 20mg/kg n=10 participants at risk Multiple doses of orally administered AT-007 were administered to healthy adult subjects.	Part B & C Cohort 4: AT-007 40mg/kg n=9 participants at risk Multiple doses of orally administered AT-007 were administered to healthy adult subjects.	Part B & C Placebo Comparator n=8 participants at risk Multiple doses of orally administered matched placebo were administered to healthy adult subjects.	Part D Cohort 1: AT-007 5mg/kg n=4 participants at risk Multiple doses of orally administered AT-007 were administered to adult subjects with Classic Galactosemia.	Part D Cohort 2: AT-007 20mg/kg n=4 participants at risk Multiple doses of orally administered AT-007 were administered to adult subjects with Classic Galactosemia.	Part D Cohort 3: AT-007 40mg/kg n=4 participants at risk Multiple doses of orally administered AT-007 were administered to adult subjects with Classic Galactosemia.	Part D Placebo Comparator n=6 participants at risk Multiple doses of orally administered matched placebo were administered to adult subjects with Classic Galactosemia.	Part D Extension Cohort 1: 20mg/kg n=9 participants at risk Multiple doses of orally administered AT-007 were administered to adult subjects with Classic Galactosemia.	Part D Extension Cohort 2: 40mg/kg n=2 participants at risk Multiple doses of orally administered AT-007 were administered to adult subjects with Classic Galactosemia.	Part D Extension Placebo Comparator n=4 participants at risk Multiple doses of orally administered matched placebo were administered to adult subjects with Classic Galactosemia.
Hepatobiliary disorders Liver Injury	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/10 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/8 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/10 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/9 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/8 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	11.1% 1/9 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/2 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days

Other adverse events

Other adverse events
Measure	Part A Cohort 1: AT-007 0.5mg/kg n=6 participants at risk Single ascending doses of orally administered AT-007 were administered to healthy adult subjects.	Part A Cohort 2: AT-007 5mg/kg n=6 participants at risk Single ascending doses of orally administered AT-007 were administered to healthy adult subjects.	Part A Cohort 3: AT-007 10mg/kg n=6 participants at risk Single ascending doses of orally administered AT-007 were administered to healthy adult subjects.	Part A Cohort 4: AT-007 20mg/kg n=6 participants at risk Single ascending doses of orally administered AT-007 were administered to healthy adult subjects.	Part A Cohort 5: 40mg/kg n=6 participants at risk Single ascending doses of orally administered AT-007 were administered to healthy adult subjects.	Part A Placebo Comparator n=10 participants at risk Single ascending doses of orally administered matched placebo were administered to healthy adult subjects.	Part B & C Cohort 1: AT-007 5mg/kg n=6 participants at risk Multiple doses of orally administered AT-007 were administered to healthy adult subjects.	Part B & C Cohort 2: AT-007 10mg/kg n=8 participants at risk Multiple doses of orally administered AT-007 were administered to healthy adult subjects.	Part B & C Cohort 3: AT-007 20mg/kg n=10 participants at risk Multiple doses of orally administered AT-007 were administered to healthy adult subjects.	Part B & C Cohort 4: AT-007 40mg/kg n=9 participants at risk Multiple doses of orally administered AT-007 were administered to healthy adult subjects.	Part B & C Placebo Comparator n=8 participants at risk Multiple doses of orally administered matched placebo were administered to healthy adult subjects.	Part D Cohort 1: AT-007 5mg/kg n=4 participants at risk Multiple doses of orally administered AT-007 were administered to adult subjects with Classic Galactosemia.	Part D Cohort 2: AT-007 20mg/kg n=4 participants at risk Multiple doses of orally administered AT-007 were administered to adult subjects with Classic Galactosemia.	Part D Cohort 3: AT-007 40mg/kg n=4 participants at risk Multiple doses of orally administered AT-007 were administered to adult subjects with Classic Galactosemia.	Part D Placebo Comparator n=6 participants at risk Multiple doses of orally administered matched placebo were administered to adult subjects with Classic Galactosemia.	Part D Extension Cohort 1: 20mg/kg n=9 participants at risk Multiple doses of orally administered AT-007 were administered to adult subjects with Classic Galactosemia.	Part D Extension Cohort 2: 40mg/kg n=2 participants at risk Multiple doses of orally administered AT-007 were administered to adult subjects with Classic Galactosemia.	Part D Extension Placebo Comparator n=4 participants at risk Multiple doses of orally administered matched placebo were administered to adult subjects with Classic Galactosemia.
Gastrointestinal disorders Gastrointestinal Disorders	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	16.7% 1/6 • Number of events 2 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	10.0% 1/10 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/8 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	10.0% 1/10 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/9 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/8 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	25.0% 1/4 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	16.7% 1/6 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	22.2% 2/9 • Number of events 2 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/2 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	25.0% 1/4 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days
Hepatobiliary disorders ALT Increased	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	16.7% 1/6 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/10 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	12.5% 1/8 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/10 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/9 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	12.5% 1/8 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	11.1% 1/9 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	50.0% 1/2 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days
Infections and infestations Infections/Infestations	16.7% 1/6 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/10 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	16.7% 1/6 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/8 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/10 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/9 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/8 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	50.0% 2/4 • Number of events 2 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	25.0% 1/4 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/9 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/2 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	25.0% 1/4 • Number of events 2 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days
Musculoskeletal and connective tissue disorders Musculoskelatal & Connective Tissue Disorders	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	16.7% 1/6 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	10.0% 1/10 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/8 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/10 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	11.1% 1/9 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/8 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	25.0% 1/4 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	16.7% 1/6 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/9 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/2 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	25.0% 1/4 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days
Nervous system disorders Nervous System	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	16.7% 1/6 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	20.0% 2/10 • Number of events 2 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/8 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	10.0% 1/10 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/9 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	12.5% 1/8 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	25.0% 1/4 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	16.7% 1/6 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	11.1% 1/9 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/2 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days
Injury, poisoning and procedural complications Injury, poisoning and procedural complications	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/10 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/8 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	20.0% 2/10 • Number of events 2 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/9 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/8 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	25.0% 1/4 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/9 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/2 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days
Investigations Investigations	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	16.7% 1/6 • Number of events 2 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/10 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	12.5% 1/8 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/10 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/9 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	12.5% 1/8 • Number of events 2 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	25.0% 1/4 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	11.1% 1/9 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/2 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days
General disorders General Disorders	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/10 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/8 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/10 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/9 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/8 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	16.7% 1/6 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/9 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/2 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	25.0% 1/4 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days
Immune system disorders Seasonal Allergies	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/10 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/8 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/10 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/9 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/8 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	11.1% 1/9 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/2 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days
Reproductive system and breast disorders Reproductive System Disorder	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/10 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/8 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/10 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/9 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/8 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/6 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	11.1% 1/9 • Number of events 1 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/2 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days	0.00% 0/4 • Part A: 1 day; Part B&C: 7 days; Part D: 32 days; Part D Extension: 90 days

Additional Information

Riccardo Perfetti, MD

Applied Therapeutics, Inc

Phone: 2122209226

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place