Trial Outcomes & Findings for SAD, MAD and Food Effect Evaluation of Safety, Tolerability, and PK of AQ280 in Healthy Subjects (NCT NCT05485779)

Results Overview

The number of participants who experienced a treatment-emergent event (TEAE) are presented.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

66 participants

Primary outcome timeframe

Part A (SAD): Screening up to Day 8 for fasted cohorts and Day 18(±2) for fed/fasted cohorts

Results posted on

2024-12-24

Participant Flow

Participant milestones

Participant milestones
Measure	Part A (SAD): Placebo (Fasted) Single dose of placebo, fasted Dose form: capsule, hard Method of administration: oral	Part A (SAD): 3 mg (Fasted) Single dose of 3 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 9 mg (Fasted) Single dose of 9 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): Placebo (Fasted/Fed) Single dose of placebo fasted on Day 1 of Treatment Period 1 and fed on Day 1 of Treatment Period 2. Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fasted/Fed) Single dose of 16 mg AQ280 fasted on Day 1 of Treatment Period 1 and fed on Day 1 of Treatment Period 2. AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 48 mg (Fasted) Single dose of 48 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 60 mg (Fasted) Single dose of 60 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part B (MAD): Placebo Placebo, once daily (QD) for seven days Dose form: capsule, hard Method of administration: oral	Part B (MAD): 9 mg AQ280 9 mg once daily (QD) for seven days AQ280: Dose form: capsule, hard Method of administration: oral	Part B (MAD): 27 mg AQ280 27 mg once daily (QD) for seven days AQ280: Dose form: capsule, hard Method of administration: oral	Part B (MAD): 60 mg AQ280 60 mg once daily (QD) for seven days AQ280: Dose form: capsule, hard Method of administration: oral
Treatment Period 1 (Fasted) STARTED	8	6	6	2	6	6	6	7	7	6	6
Treatment Period 1 (Fasted) COMPLETED	8	6	6	2	6	6	6	6	6	6	5
Treatment Period 1 (Fasted) NOT COMPLETED	0	0	0	0	0	0	0	1	1	0	1
Treatment Period 2 (Fed Groups Only) STARTED	0	0	0	2	6	0	0	0	0	0	0
Treatment Period 2 (Fed Groups Only) COMPLETED	0	0	0	1	6	0	0	0	0	0	0
Treatment Period 2 (Fed Groups Only) NOT COMPLETED	0	0	0	1	0	0	0	0	0	0	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Part A (SAD): Placebo (Fasted/Fed) Single dose of placebo fasted on Day 1 of Treatment Period 1 and fed on Day 1 of Treatment Period 2. Dose form: capsule, hard Method of administration: oral	Part B (MAD): Placebo Placebo, once daily (QD) for seven days Dose form: capsule, hard Method of administration: oral	Part B (MAD): 9 mg AQ280 9 mg once daily (QD) for seven days AQ280: Dose form: capsule, hard Method of administration: oral	Part B (MAD): 60 mg AQ280 60 mg once daily (QD) for seven days AQ280: Dose form: capsule, hard Method of administration: oral
Treatment Period 1 (Fasted) Adverse Event	0	1	1	0
Treatment Period 1 (Fasted) Physician Decision	0	0	0	1
Treatment Period 2 (Fed Groups Only) Discontinued prior to dosing in fed period due to a failed drug screen at check-in	1	0	0	0

Baseline Characteristics

SAD, MAD and Food Effect Evaluation of Safety, Tolerability, and PK of AQ280 in Healthy Subjects

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Part A (SAD): Placebo (Fasted) n=8 Participants Single dose of placebo, fasted Dose form: capsule, hard Method of administration: oral	Part A (SAD): 3 mg (Fasted) n=6 Participants Single dose of 3 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 9 mg (Fasted) n=6 Participants Single dose of 9 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): Placebo (Fasted/Fed) n=2 Participants Single dose of placebo fasted on Day 1 of Treatment Period 1 and fed on Day 1 of Treatment Period 2. Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fasted/Fed) n=6 Participants Single dose of 16 mg AQ280 fasted on Day 1 of Treatment Period 1 and fed on Day 1 of Treatment Period 2. AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 48 mg (Fasted) n=6 Participants Single dose of 48 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 60 mg (Fasted) n=6 Participants Single dose of 60 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part B (MAD): Placebo n=7 Participants Placebo, once daily (QD) for seven days Dose form: capsule, hard Method of administration: oral	Part B (MAD): 9 mg n=7 Participants AQ280 9 mg once daily (QD) for seven days AQ280: Dose form: capsule, hard Method of administration: oral	Part B (MAD): 27 mg n=6 Participants AQ280 27 mg once daily (QD) for seven days AQ280: Dose form: capsule, hard Method of administration: oral	Part B (MAD): 60 mg n=6 Participants AQ280 60 mg once daily (QD) for seven days AQ280: Dose form: capsule, hard Method of administration: oral	Total n=66 Participants Total of all reporting groups
Age, Continuous	50.8 years STANDARD_DEVIATION 11.65 • n=5 Participants	38.8 years STANDARD_DEVIATION 15.46 • n=7 Participants	40.0 years STANDARD_DEVIATION 14.70 • n=5 Participants	32.0 years STANDARD_DEVIATION 7.07 • n=4 Participants	53.5 years STANDARD_DEVIATION 13.63 • n=21 Participants	39.8 years STANDARD_DEVIATION 21.78 • n=8 Participants	43.0 years STANDARD_DEVIATION 18.21 • n=8 Participants	38.0 years STANDARD_DEVIATION 13.96 • n=24 Participants	43.6 years STANDARD_DEVIATION 12.75 • n=42 Participants	36.3 years STANDARD_DEVIATION 14.64 • n=42 Participants	47.8 years STANDARD_DEVIATION 13.67 • n=42 Participants	43.0 years STANDARD_DEVIATION 14.97 • n=42 Participants
Sex: Female, Male Female	3 Participants n=5 Participants	3 Participants n=7 Participants	2 Participants n=5 Participants	0 Participants n=4 Participants	1 Participants n=21 Participants	4 Participants n=8 Participants	3 Participants n=8 Participants	1 Participants n=24 Participants	5 Participants n=42 Participants	1 Participants n=42 Participants	3 Participants n=42 Participants	26 Participants n=42 Participants
Sex: Female, Male Male	5 Participants n=5 Participants	3 Participants n=7 Participants	4 Participants n=5 Participants	2 Participants n=4 Participants	5 Participants n=21 Participants	2 Participants n=8 Participants	3 Participants n=8 Participants	6 Participants n=24 Participants	2 Participants n=42 Participants	5 Participants n=42 Participants	3 Participants n=42 Participants	40 Participants n=42 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=8 Participants	0 Participants n=8 Participants	0 Participants n=24 Participants	0 Participants n=42 Participants	0 Participants n=42 Participants	0 Participants n=42 Participants	0 Participants n=42 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	8 Participants n=5 Participants	6 Participants n=7 Participants	6 Participants n=5 Participants	2 Participants n=4 Participants	6 Participants n=21 Participants	6 Participants n=8 Participants	6 Participants n=8 Participants	7 Participants n=24 Participants	7 Participants n=42 Participants	6 Participants n=42 Participants	6 Participants n=42 Participants	66 Participants n=42 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=8 Participants	0 Participants n=8 Participants	0 Participants n=24 Participants	0 Participants n=42 Participants	0 Participants n=42 Participants	0 Participants n=42 Participants	0 Participants n=42 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=8 Participants	0 Participants n=8 Participants	0 Participants n=24 Participants	0 Participants n=42 Participants	0 Participants n=42 Participants	0 Participants n=42 Participants	0 Participants n=42 Participants
Race (NIH/OMB) Asian	2 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	1 Participants n=8 Participants	0 Participants n=8 Participants	0 Participants n=24 Participants	0 Participants n=42 Participants	1 Participants n=42 Participants	0 Participants n=42 Participants	4 Participants n=42 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=8 Participants	0 Participants n=8 Participants	0 Participants n=24 Participants	0 Participants n=42 Participants	0 Participants n=42 Participants	0 Participants n=42 Participants	0 Participants n=42 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	1 Participants n=8 Participants	0 Participants n=8 Participants	0 Participants n=24 Participants	1 Participants n=42 Participants	0 Participants n=42 Participants	1 Participants n=42 Participants	3 Participants n=42 Participants
Race (NIH/OMB) White	6 Participants n=5 Participants	6 Participants n=7 Participants	6 Participants n=5 Participants	2 Participants n=4 Participants	6 Participants n=21 Participants	4 Participants n=8 Participants	6 Participants n=8 Participants	7 Participants n=24 Participants	5 Participants n=42 Participants	5 Participants n=42 Participants	5 Participants n=42 Participants	58 Participants n=42 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=8 Participants	0 Participants n=8 Participants	0 Participants n=24 Participants	1 Participants n=42 Participants	0 Participants n=42 Participants	0 Participants n=42 Participants	1 Participants n=42 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=8 Participants	0 Participants n=8 Participants	0 Participants n=24 Participants	0 Participants n=42 Participants	0 Participants n=42 Participants	0 Participants n=42 Participants	0 Participants n=42 Participants
Height	172.58 centimeters STANDARD_DEVIATION 9.031 • n=5 Participants	175.32 centimeters STANDARD_DEVIATION 7.802 • n=7 Participants	173.48 centimeters STANDARD_DEVIATION 11.127 • n=5 Participants	180.05 centimeters STANDARD_DEVIATION 6.293 • n=4 Participants	179.65 centimeters STANDARD_DEVIATION 4.281 • n=21 Participants	165.20 centimeters STANDARD_DEVIATION 10.623 • n=8 Participants	170.87 centimeters STANDARD_DEVIATION 12.692 • n=8 Participants	174.90 centimeters STANDARD_DEVIATION 17.240 • n=24 Participants	168.37 centimeters STANDARD_DEVIATION 6.033 • n=42 Participants	172.35 centimeters STANDARD_DEVIATION 9.957 • n=42 Participants	170.47 centimeters STANDARD_DEVIATION 7.277 • n=42 Participants	172.54 centimeters STANDARD_DEVIATION 10.192 • n=42 Participants
Body weight	80.15 kilograms STANDARD_DEVIATION 12.279 • n=5 Participants	73.88 kilograms STANDARD_DEVIATION 12.495 • n=7 Participants	83.48 kilograms STANDARD_DEVIATION 12.015 • n=5 Participants	81.25 kilograms STANDARD_DEVIATION 15.486 • n=4 Participants	85.20 kilograms STANDARD_DEVIATION 8.323 • n=21 Participants	62.17 kilograms STANDARD_DEVIATION 14.016 • n=8 Participants	75.12 kilograms STANDARD_DEVIATION 17.688 • n=8 Participants	77.77 kilograms STANDARD_DEVIATION 15.225 • n=24 Participants	65.91 kilograms STANDARD_DEVIATION 7.416 • n=42 Participants	72.6 kilograms STANDARD_DEVIATION 18.304 • n=42 Participants	66.73 kilograms STANDARD_DEVIATION 11.898 • n=42 Participants	74.62 kilograms STANDARD_DEVIATION 14.251 • n=42 Participants
Body mass index	26.90 kilograms/meter^2 STANDARD_DEVIATION 3.601 • n=5 Participants	23.97 kilograms/meter^2 STANDARD_DEVIATION 3.315 • n=7 Participants	27.68 kilograms/meter^2 STANDARD_DEVIATION 2.218 • n=5 Participants	24.95 kilograms/meter^2 STANDARD_DEVIATION 3.041 • n=4 Participants	26.37 kilograms/meter^2 STANDARD_DEVIATION 2.069 • n=21 Participants	22.67 kilograms/meter^2 STANDARD_DEVIATION 3.647 • n=8 Participants	25.55 kilograms/meter^2 STANDARD_DEVIATION 4.157 • n=8 Participants	25.37 kilograms/meter^2 STANDARD_DEVIATION 2.914 • n=24 Participants	23.23 kilograms/meter^2 STANDARD_DEVIATION 2.319 • n=42 Participants	24.15 kilograms/meter^2 STANDARD_DEVIATION 4.097 • n=42 Participants	22.88 kilograms/meter^2 STANDARD_DEVIATION 2.785 • n=42 Participants	24.92 kilograms/meter^2 STANDARD_DEVIATION 3.369 • n=42 Participants

PRIMARY outcome

Timeframe: Part A (SAD): Screening up to Day 8 for fasted cohorts and Day 18(±2) for fed/fasted cohorts

Population: Safety population. The 'Part A (SAD): Placebo (Fasted)' cohort includes the 8 participants in the fasted only cohort as well as any AEs experienced by the 2 participants in the 'Part A (SAD): Placebo (Fasted/Fed)' cohort whilst in the fasting during Treatment Period 1. Any AEs experienced by these 2 participants during Treatment Period 2 (ie, the fed stage) only are listed separately under the cohort 'Part A (SAD): Placebo (Fed).

The number of participants who experienced a treatment-emergent event (TEAE) are presented.

Outcome measures

Outcome measures
Measure	Part A (SAD): Placebo (Fasted) n=10 Participants Single dose of placebo, fasted Dose form: capsule, hard Method of administration: oral	Part A (SAD): Placebo (Fed) n=2 Participants Single dose of placebo, fed Dose form: capsule, hard Method of administration: oral	Part A (SAD): 3 mg (Fasted) n=6 Participants Single dose of 3 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 9 mg (Fasted) n=6 Participants Single dose of 9 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fasted) n=6 Participants Single dose of 16 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fed) n=6 Participants Single dose of 16 mg AQ280, fed AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 48 mg (Fasted) n=6 Participants Single dose of 48 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 60 mg (Fasted) n=6 Participants Single dose of 60 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral
Part A (SAD): Number of Treatment Emergent Adverse Events (TEAEs) by Participant	1 Participants	0 Participants	1 Participants	2 Participants	1 Participants	2 Participants	0 Participants	1 Participants

PRIMARY outcome

Timeframe: Part A (SAD): Screening up to Day 8 for fasted cohorts and Day 18(±2) for fed/fasted cohorts

Population: Safety population. The 'Part A (SAD): Placebo (Fasted)' cohort includes the 8 participants in the fasted only cohort as well as any AEs experienced by the 2 participants in the 'Part A (SAD): Placebo (Fasted/Fed)' cohort whilst in the fasting during Treatment Period 1. Any AEs experienced by these 2 participants during Treatment Period 2 (ie, the fed stage) only are listed separately under the cohort 'Part A (SAD): Placebo (Fed).

The number of total events experienced by participants are presented.

Outcome measures

Outcome measures
Measure	Part A (SAD): Placebo (Fasted) n=10 Participants Single dose of placebo, fasted Dose form: capsule, hard Method of administration: oral	Part A (SAD): Placebo (Fed) n=2 Participants Single dose of placebo, fed Dose form: capsule, hard Method of administration: oral	Part A (SAD): 3 mg (Fasted) n=6 Participants Single dose of 3 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 9 mg (Fasted) n=6 Participants Single dose of 9 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fasted) n=6 Participants Single dose of 16 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fed) n=6 Participants Single dose of 16 mg AQ280, fed AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 48 mg (Fasted) n=6 Participants Single dose of 48 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 60 mg (Fasted) n=6 Participants Single dose of 60 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral
Part A (SAD): Number of Treatment Emergent Adverse Events (TEAEs) Experienced	1 events	0 events	1 events	2 events	2 events	3 events	0 events	1 events

PRIMARY outcome

Timeframe: Part B (MAD): Screening up to Day 14(±3)

Population: Safety Population

The number of participants who experienced a treatment-emergent event (TEAE) are presented.

Outcome measures

Outcome measures
Measure	Part A (SAD): Placebo (Fasted) n=7 Participants Single dose of placebo, fasted Dose form: capsule, hard Method of administration: oral	Part A (SAD): Placebo (Fed) n=7 Participants Single dose of placebo, fed Dose form: capsule, hard Method of administration: oral	Part A (SAD): 3 mg (Fasted) n=6 Participants Single dose of 3 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 9 mg (Fasted) n=6 Participants Single dose of 9 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fasted) Single dose of 16 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fed) Single dose of 16 mg AQ280, fed AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 48 mg (Fasted) Single dose of 48 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 60 mg (Fasted) Single dose of 60 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral
Part B (MAD): Number of Treatment Emergent Adverse Events (TEAEs) by Participant	3 Participants	3 Participants	3 Participants	3 Participants	—	—	—	—

PRIMARY outcome

Timeframe: Part B (MAD): Screening up to Day 14(±3)

Population: Safety Population

The number of total TEAE events experienced by participants are presented.

Outcome measures

Outcome measures
Measure	Part A (SAD): Placebo (Fasted) n=7 Participants Single dose of placebo, fasted Dose form: capsule, hard Method of administration: oral	Part A (SAD): Placebo (Fed) n=7 Participants Single dose of placebo, fed Dose form: capsule, hard Method of administration: oral	Part A (SAD): 3 mg (Fasted) n=6 Participants Single dose of 3 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 9 mg (Fasted) n=6 Participants Single dose of 9 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fasted) Single dose of 16 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fed) Single dose of 16 mg AQ280, fed AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 48 mg (Fasted) Single dose of 48 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 60 mg (Fasted) Single dose of 60 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral
Part B (MAD): Number of Treatment Emergent Adverse Events (TEAEs) Experienced	7 events	6 events	8 events	4 events	—	—	—	—

PRIMARY outcome

Timeframe: Part A (SAD): Screening up to Day 3

Population: Safety population. The 'Part A (SAD): Placebo (Fasted)' cohort includes the 8 participants in the fasted only cohort as well as any results for the 2 participants in the 'Part A (SAD): Placebo (Fasted/Fed)' cohort whilst in the fasting during Treatment Period 1. Results for the 2 participants during Treatment Period 2 (ie, the fed stage) only are listed separately under the cohort 'Part A (SAD): Placebo (Fed)'.

The number of participants with clinically significant abnormalities in vital signs is presented. Vital signs: systolic and diastolic blood pressure, pulse rate, and oral body temperature

Outcome measures

Outcome measures
Measure	Part A (SAD): Placebo (Fasted) n=10 Participants Single dose of placebo, fasted Dose form: capsule, hard Method of administration: oral	Part A (SAD): Placebo (Fed) n=2 Participants Single dose of placebo, fed Dose form: capsule, hard Method of administration: oral	Part A (SAD): 3 mg (Fasted) n=6 Participants Single dose of 3 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 9 mg (Fasted) n=6 Participants Single dose of 9 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fasted) n=6 Participants Single dose of 16 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fed) n=6 Participants Single dose of 16 mg AQ280, fed AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 48 mg (Fasted) n=6 Participants Single dose of 48 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 60 mg (Fasted) n=6 Participants Single dose of 60 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral
Part A (SAD): Number of Participants With Clinically Significant Abnormalities in Vital Signs	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants

PRIMARY outcome

Timeframe: Part A (SAD): Screening up to Day 3

Population: Safety population. The 'Part A (SAD): Placebo (Fasted)' cohort includes the 8 participants in the fasted only cohort as well as any results for the 2 participants in the 'Part A (SAD): Placebo (Fasted/Fed)' cohort whilst in the fasting during Treatment Period 1. Results for the 2 participants during Treatment Period 2 (ie, the fed stage) only are listed separately under the cohort 'Part A (SAD): Placebo (Fed)'.

The number of participants with abnormal electrocardiogram (ECG) results is presented. Abnormal ECGs were considered to be those with a QTcF interval of greater than 450 msec for males and greater than 470 msec for females, or change from baseline of greater than 30 msec.

Outcome measures

Outcome measures
Measure	Part A (SAD): Placebo (Fasted) n=10 Participants Single dose of placebo, fasted Dose form: capsule, hard Method of administration: oral	Part A (SAD): Placebo (Fed) n=2 Participants Single dose of placebo, fed Dose form: capsule, hard Method of administration: oral	Part A (SAD): 3 mg (Fasted) n=6 Participants Single dose of 3 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 9 mg (Fasted) n=6 Participants Single dose of 9 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fasted) n=6 Participants Single dose of 16 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fed) n=6 Participants Single dose of 16 mg AQ280, fed AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 48 mg (Fasted) n=6 Participants Single dose of 48 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 60 mg (Fasted) n=6 Participants Single dose of 60 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral
Part A (SAD): Number of Participants With Abnormal ECG	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants

PRIMARY outcome

Timeframe: Part A (SAD): Screening up to Day 3

Population: Safety population. The 'Part A (SAD): Placebo (Fasted)' cohort includes the 8 participants in the fasted only cohort as well as any results for the 2 participants in the 'Part A (SAD): Placebo (Fasted/Fed)' cohort whilst in the fasting during Treatment Period 1. Results for the 2 participants during Treatment Period 2 (ie, the fed stage) only are listed separately under the cohort 'Part A (SAD): Placebo (Fed)'.

The number of participants with clinically significant changes in any laboratory evaluations (clinical chemistry, haematology, coagulation, or urinalysis) is presented.

Outcome measures

Outcome measures
Measure	Part A (SAD): Placebo (Fasted) n=10 Participants Single dose of placebo, fasted Dose form: capsule, hard Method of administration: oral	Part A (SAD): Placebo (Fed) n=2 Participants Single dose of placebo, fed Dose form: capsule, hard Method of administration: oral	Part A (SAD): 3 mg (Fasted) n=6 Participants Single dose of 3 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 9 mg (Fasted) n=6 Participants Single dose of 9 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fasted) n=6 Participants Single dose of 16 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fed) n=6 Participants Single dose of 16 mg AQ280, fed AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 48 mg (Fasted) n=6 Participants Single dose of 48 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 60 mg (Fasted) n=6 Participants Single dose of 60 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral
Part A (SAD): Number of Subjects With Clinically Significant Changes in Laboratory Evaluations	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants

PRIMARY outcome

Timeframe: Part B (MAD): Screening up to Day 14(±3)

Population: Safety population

The number of participants with clinically significant abnormalities in vital signs is presented. Vital signs: systolic and diastolic blood pressure, pulse rate, and oral body temperature

Outcome measures

Outcome measures
Measure	Part A (SAD): Placebo (Fasted) n=7 Participants Single dose of placebo, fasted Dose form: capsule, hard Method of administration: oral	Part A (SAD): Placebo (Fed) n=7 Participants Single dose of placebo, fed Dose form: capsule, hard Method of administration: oral	Part A (SAD): 3 mg (Fasted) n=6 Participants Single dose of 3 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 9 mg (Fasted) n=6 Participants Single dose of 9 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fasted) Single dose of 16 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fed) Single dose of 16 mg AQ280, fed AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 48 mg (Fasted) Single dose of 48 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 60 mg (Fasted) Single dose of 60 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral
Part B (MAD): Number of Participants With Clinically Significant Abnormalities in Vital Signs	0 Participants	0 Participants	0 Participants	0 Participants	—	—	—	—

PRIMARY outcome

Timeframe: Part B (MAD): Screening up to Day 14(±3)

Population: Safety population

The number of participants with abnormal electrocardiogram (ECG) results is presented. Abnormal ECGs were considered to be those with a QTcF interval of greater than 450 msec for males and greater than 470 msec for females, or change from baseline of greater than 30 msec.

Outcome measures

Outcome measures
Measure	Part A (SAD): Placebo (Fasted) n=7 Participants Single dose of placebo, fasted Dose form: capsule, hard Method of administration: oral	Part A (SAD): Placebo (Fed) n=7 Participants Single dose of placebo, fed Dose form: capsule, hard Method of administration: oral	Part A (SAD): 3 mg (Fasted) n=6 Participants Single dose of 3 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 9 mg (Fasted) n=6 Participants Single dose of 9 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fasted) Single dose of 16 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fed) Single dose of 16 mg AQ280, fed AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 48 mg (Fasted) Single dose of 48 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 60 mg (Fasted) Single dose of 60 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral
Part B (MAD): Number of Participants With Abnormal ECG	0 Participants	0 Participants	0 Participants	0 Participants	—	—	—	—

PRIMARY outcome

Timeframe: Part B (MAD): Screening up to Day 14(±3)

Population: Safety population

The number of participants with clinically significant changes in any laboratory evaluations (clinical chemistry, haematology, coagulation, or urinalysis) is presented.

Outcome measures

Outcome measures
Measure	Part A (SAD): Placebo (Fasted) n=7 Participants Single dose of placebo, fasted Dose form: capsule, hard Method of administration: oral	Part A (SAD): Placebo (Fed) n=7 Participants Single dose of placebo, fed Dose form: capsule, hard Method of administration: oral	Part A (SAD): 3 mg (Fasted) n=6 Participants Single dose of 3 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 9 mg (Fasted) n=6 Participants Single dose of 9 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fasted) Single dose of 16 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fed) Single dose of 16 mg AQ280, fed AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 48 mg (Fasted) Single dose of 48 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 60 mg (Fasted) Single dose of 60 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral
Part B (MAD): Number of Participants With Clinically Significant Changes in Laboratory Evaluations Change in leukocyte count (to above the reference range)	0 Participants	0 Participants	0 Participants	1 Participants	—	—	—	—
Part B (MAD): Number of Participants With Clinically Significant Changes in Laboratory Evaluations Change in any other evaluation	0 Participants	0 Participants	0 Participants	0 Participants	—	—	—	—

SECONDARY outcome

Timeframe: Days 1, 2 and 3

Population: Pharmacokinetic population

Primary PK parameters derived from plasma concentration-time profile of AQ280: area under the concentration time curve from time 0 extrapolated to infinity following Single Oral Dose Administration of 3, 9, 16, 48, and 60 mg AQ280 in a Fasted State and 16 mg AQ280 in a Fed State

Outcome measures

Outcome measures
Measure	Part A (SAD): Placebo (Fasted) n=6 Participants Single dose of placebo, fasted Dose form: capsule, hard Method of administration: oral	Part A (SAD): Placebo (Fed) n=6 Participants Single dose of placebo, fed Dose form: capsule, hard Method of administration: oral	Part A (SAD): 3 mg (Fasted) n=6 Participants Single dose of 3 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 9 mg (Fasted) n=6 Participants Single dose of 9 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fasted) n=6 Participants Single dose of 16 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fed) n=6 Participants Single dose of 16 mg AQ280, fed AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 48 mg (Fasted) Single dose of 48 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 60 mg (Fasted) Single dose of 60 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral
Part A (SAD) - Primary PK Parameters Derived From Plasma Concentration-time Profile of AQ280: Area Under the Concentration Time Curve From Time 0 Extrapolated to Infinity	77.0 h*ng/mL Geometric Coefficient of Variation 17.2	318 h*ng/mL Geometric Coefficient of Variation 23.8	539 h*ng/mL Geometric Coefficient of Variation 35.4	527 h*ng/mL Geometric Coefficient of Variation 33.7	1860 h*ng/mL Geometric Coefficient of Variation 43.2	2820 h*ng/mL Geometric Coefficient of Variation 26.5	—	—

SECONDARY outcome

Timeframe: Days 1, 2 and 3

Population: Pharmacokinetic population

Part A (SAD) - Primary PK parameter derived from plasma concentration-time profile of AQ280: maximum observed concentration (Cmax) following Single Oral Dose Administration of 3, 9, 16, 48, and 60 mg AQ280 in a Fasted State and 16 mg AQ280 in a Fed State

Outcome measures

Outcome measures
Measure	Part A (SAD): Placebo (Fasted) n=6 Participants Single dose of placebo, fasted Dose form: capsule, hard Method of administration: oral	Part A (SAD): Placebo (Fed) n=6 Participants Single dose of placebo, fed Dose form: capsule, hard Method of administration: oral	Part A (SAD): 3 mg (Fasted) n=6 Participants Single dose of 3 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 9 mg (Fasted) n=6 Participants Single dose of 9 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fasted) n=6 Participants Single dose of 16 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fed) n=6 Participants Single dose of 16 mg AQ280, fed AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 48 mg (Fasted) Single dose of 48 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 60 mg (Fasted) Single dose of 60 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral
Part A (SAD) - Primary PK Parameter Derived From Plasma Concentration-time Profile of AQ280: Maximum Observed Concentration (Cmax)	20.0 ng/mL Geometric Coefficient of Variation 16.1	70.1 ng/mL Geometric Coefficient of Variation 25.1	113 ng/mL Geometric Coefficient of Variation 16.5	90.8 ng/mL Geometric Coefficient of Variation 19.6	418 ng/mL Geometric Coefficient of Variation 40.8	638 ng/mL Geometric Coefficient of Variation 23.3	—	—

SECONDARY outcome

Timeframe: Days 1, 2 and 3

Population: Pharmacokinetic population

Area under the concentration time curve from time 0 extrapolated to infinity of metabolite AQ282 following single oral dose administration of 3, 9, 16, 48, and 60 mg AQ280 in a fasted state and 16 mg AQ280 in a fed state

Outcome measures

Outcome measures
Measure	Part A (SAD): Placebo (Fasted) n=2 Participants Single dose of placebo, fasted Dose form: capsule, hard Method of administration: oral	Part A (SAD): Placebo (Fed) n=6 Participants Single dose of placebo, fed Dose form: capsule, hard Method of administration: oral	Part A (SAD): 3 mg (Fasted) n=6 Participants Single dose of 3 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 9 mg (Fasted) n=6 Participants Single dose of 9 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fasted) n=6 Participants Single dose of 16 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fed) n=6 Participants Single dose of 16 mg AQ280, fed AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 48 mg (Fasted) Single dose of 48 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 60 mg (Fasted) Single dose of 60 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral
Part A (SAD) - Primary PK Parameter Derived From Plasma Concentration-time Profile of the AQ280 Main Metabolite, AQ282: Area Under the Concentration Time Curve From Time 0 Extrapolated to Infinity	NA h*ng/mL Geometric Coefficient of Variation NA Insufficient number of participants with events	32.7 h*ng/mL Geometric Coefficient of Variation 28.2	43.7 h*ng/mL Geometric Coefficient of Variation 68.9	45.1 h*ng/mL Geometric Coefficient of Variation 66.4	239 h*ng/mL Geometric Coefficient of Variation 75.8	210 h*ng/mL Geometric Coefficient of Variation 82.0	—	—

SECONDARY outcome

Timeframe: Days 1, 2 and 3

Population: Pharmacokinetic population

Maximum observed concentration (Cmax) of metabolite AQ282 following single oral dose administration of 3, 9, 16, 48, and 60 mg AQ280 in a fasted state and 16 mg AQ280 in a fed state

Outcome measures

Outcome measures
Measure	Part A (SAD): Placebo (Fasted) n=6 Participants Single dose of placebo, fasted Dose form: capsule, hard Method of administration: oral	Part A (SAD): Placebo (Fed) n=6 Participants Single dose of placebo, fed Dose form: capsule, hard Method of administration: oral	Part A (SAD): 3 mg (Fasted) n=6 Participants Single dose of 3 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 9 mg (Fasted) n=6 Participants Single dose of 9 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fasted) n=6 Participants Single dose of 16 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fed) n=6 Participants Single dose of 16 mg AQ280, fed AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 48 mg (Fasted) Single dose of 48 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 60 mg (Fasted) Single dose of 60 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral
Part A (SAD) - Primary PK Parameter Derived From Plasma Concentration-time Profile of the AQ280 Main Metabolite, AQ282: Maximum Observed Concentration (Cmax)	0.722 ng/mL Geometric Coefficient of Variation 34.4	3.38 ng/mL Geometric Coefficient of Variation 22.4	3.91 ng/mL Geometric Coefficient of Variation 55.9	3.84 ng/mL Geometric Coefficient of Variation 62.4	24.4 ng/mL Geometric Coefficient of Variation 81.2	24.4 ng/mL Geometric Coefficient of Variation 64.8	—	—

SECONDARY outcome

Timeframe: Days 1, 2 and 3

Population: Pharmacokinetic population of the 16mg fed and fasted groups in Part A. No other dose groups were assessed for food effect.

Difference in area under the concentration time curve from time 0 extrapolated to infinity derived from plasma concentration-time profile of AQ280 in fasted state and in fed state to assess the effect of food on single oral doses of 16 mg AQ280. The within-subject coefficient of variation is presented under the 16 mg fed results.

Outcome measures

Outcome measures
Measure	Part A (SAD): Placebo (Fasted) n=6 Participants Single dose of placebo, fasted Dose form: capsule, hard Method of administration: oral	Part A (SAD): Placebo (Fed) n=6 Participants Single dose of placebo, fed Dose form: capsule, hard Method of administration: oral	Part A (SAD): 3 mg (Fasted) Single dose of 3 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 9 mg (Fasted) Single dose of 9 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fasted) Single dose of 16 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fed) Single dose of 16 mg AQ280, fed AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 48 mg (Fasted) Single dose of 48 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 60 mg (Fasted) Single dose of 60 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral
Part A (SAD) - Difference in Area Under the Concentration Time Curve From Time 0 Extrapolated to Infinity in Fasted State and in Fed State	539 h*ng/mL Geometric Coefficient of Variation NA Within-subject CV of fed versus fasted is presented in the fed group results	527 h*ng/mL Geometric Coefficient of Variation 5.66	—	—	—	—	—	—

SECONDARY outcome

Timeframe: Days 1, 2 and 3

Population: Pharmacokinetic population of the 16mg fed and fasted groups in Part A. No other dose groups were assessed for food effect.

Difference in maximum observed concentration (Cmax) derived from plasma concentration-time profile of AQ280 in fasted state and in fed state to assess the effect of food on single oral doses of 16 mg AQ280. The within-subject coefficient of variation is presented under the 16 mg fed results.

Outcome measures

Outcome measures
Measure	Part A (SAD): Placebo (Fasted) n=6 Participants Single dose of placebo, fasted Dose form: capsule, hard Method of administration: oral	Part A (SAD): Placebo (Fed) n=6 Participants Single dose of placebo, fed Dose form: capsule, hard Method of administration: oral	Part A (SAD): 3 mg (Fasted) Single dose of 3 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 9 mg (Fasted) Single dose of 9 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fasted) Single dose of 16 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fed) Single dose of 16 mg AQ280, fed AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 48 mg (Fasted) Single dose of 48 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 60 mg (Fasted) Single dose of 60 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral
Part A (SAD) - Difference in Maximum Observed Concentration (Cmax) in Fasted State and in Fed State	113 ng/mL Geometric Coefficient of Variation NA Within-subject CV of fed versus fasted is presented in the fed group results	90.8 ng/mL Geometric Coefficient of Variation 20.5	—	—	—	—	—	—

SECONDARY outcome

Timeframe: Day 1 to Day 7

Population: Pharmacokinetic population

Accumulation ratio (AR) AQ280 following multiple oral dose administration of 9, 27, and 60 mg AQ280 once daily for 7 consecutive days in a fasted state on Day 7. ARAUC = accumulation ratio based on area under the concentration-time curve over a dosing interval ARCmax = accumulation ratio based on maximum observed concentration

Outcome measures

Outcome measures
Measure	Part A (SAD): Placebo (Fasted) n=6 Participants Single dose of placebo, fasted Dose form: capsule, hard Method of administration: oral	Part A (SAD): Placebo (Fed) n=6 Participants Single dose of placebo, fed Dose form: capsule, hard Method of administration: oral	Part A (SAD): 3 mg (Fasted) n=5 Participants Single dose of 3 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 9 mg (Fasted) Single dose of 9 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fasted) Single dose of 16 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fed) Single dose of 16 mg AQ280, fed AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 48 mg (Fasted) Single dose of 48 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 60 mg (Fasted) Single dose of 60 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral
Part B (MAD) - Primary PK Parameters Derived From Plasma Concentration-time Profile of AQ280: Accumulation Ratio (AR) ARAUC	1.13 ratio Geometric Coefficient of Variation 12.0	1.03 ratio Geometric Coefficient of Variation 11.4	1.12 ratio Geometric Coefficient of Variation 5.5	—	—	—	—	—
Part B (MAD) - Primary PK Parameters Derived From Plasma Concentration-time Profile of AQ280: Accumulation Ratio (AR) ARCmax	1.11 ratio Geometric Coefficient of Variation 22.7	0.910 ratio Geometric Coefficient of Variation 20.0	1.12 ratio Geometric Coefficient of Variation 13.1	—	—	—	—	—

SECONDARY outcome

Timeframe: Day 1 and Day 7

Population: Pharmacokinetic population

Area under the concentration time curve over a dosing interval (AUCτ) following single oral administration of 9, 27, and 60 mg AQ280 in a fasted state (Day 1) and after multiple oral dose administration of 9, 27, and 60 mg AQ280 once daily for 7 consecutive days in a fasted state (Day 7).

Outcome measures

Outcome measures
Measure	Part A (SAD): Placebo (Fasted) n=7 Participants Single dose of placebo, fasted Dose form: capsule, hard Method of administration: oral	Part A (SAD): Placebo (Fed) n=6 Participants Single dose of placebo, fed Dose form: capsule, hard Method of administration: oral	Part A (SAD): 3 mg (Fasted) n=6 Participants Single dose of 3 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 9 mg (Fasted) Single dose of 9 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fasted) Single dose of 16 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fed) Single dose of 16 mg AQ280, fed AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 48 mg (Fasted) Single dose of 48 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 60 mg (Fasted) Single dose of 60 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral
Part B (MAD) - Primary PK Parameters Derived From Plasma Concentration-time Profile of AQ280: Area Under the Concentration Time Curve Over a Dosing Interval Day 1	300 h*ng/mL Geometric Coefficient of Variation 28.9	978 h*ng/mL Geometric Coefficient of Variation 29.8	2910 h*ng/mL Geometric Coefficient of Variation 36.8	—	—	—	—	—
Part B (MAD) - Primary PK Parameters Derived From Plasma Concentration-time Profile of AQ280: Area Under the Concentration Time Curve Over a Dosing Interval Day 7	345 h*ng/mL Geometric Coefficient of Variation 26.1	1000 h*ng/mL Geometric Coefficient of Variation 21.7	3150 h*ng/mL Geometric Coefficient of Variation 36.4	—	—	—	—	—

SECONDARY outcome

Timeframe: Day 1 and Day 7

Population: Pharmacokinetic population

Maximum observed concentration (Cmax) following single oral administration of 9, 27, and 60 mg AQ280 in a fasted state (Day 1) and after multiple oral dose administration of 9, 27, and 60 mg AQ280 once daily for 7 consecutive days in a fasted state (Day 7).

Outcome measures

Outcome measures
Measure	Part A (SAD): Placebo (Fasted) n=7 Participants Single dose of placebo, fasted Dose form: capsule, hard Method of administration: oral	Part A (SAD): Placebo (Fed) n=6 Participants Single dose of placebo, fed Dose form: capsule, hard Method of administration: oral	Part A (SAD): 3 mg (Fasted) n=6 Participants Single dose of 3 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 9 mg (Fasted) Single dose of 9 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fasted) Single dose of 16 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fed) Single dose of 16 mg AQ280, fed AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 48 mg (Fasted) Single dose of 48 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 60 mg (Fasted) Single dose of 60 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral
Part B (MAD) - Primary PK Parameters Derived From Plasma Concentration-time Profile of AQ280: Maximum Observed Concentration (Cmax) Day 1	64.5 ng/mL Geometric Coefficient of Variation 30.1	248 ng/mL Geometric Coefficient of Variation 24.3	560 ng/mL Geometric Coefficient of Variation 32.1	—	—	—	—	—
Part B (MAD) - Primary PK Parameters Derived From Plasma Concentration-time Profile of AQ280: Maximum Observed Concentration (Cmax) Day 7	72.0 ng/mL Geometric Coefficient of Variation 23.0	226 ng/mL Geometric Coefficient of Variation 20.5	590 ng/mL Geometric Coefficient of Variation 28.8	—	—	—	—	—

SECONDARY outcome

Timeframe: Day 1 and Day 7

Population: Pharmacokinetic population

Area under the concentration time curve over a dosing interval (AUCτ) of metabolite AQ282 following single oral dose administration of 9, 27, and 60 mg AQ280 in a fasted state (Day 1) and multiple oral dose administration of 9, 27, and 60 mg AQ280 once daily for 7 consecutive days in a fasted state (Day 7).

Outcome measures

Outcome measures
Measure	Part A (SAD): Placebo (Fasted) n=7 Participants Single dose of placebo, fasted Dose form: capsule, hard Method of administration: oral	Part A (SAD): Placebo (Fed) n=6 Participants Single dose of placebo, fed Dose form: capsule, hard Method of administration: oral	Part A (SAD): 3 mg (Fasted) n=6 Participants Single dose of 3 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 9 mg (Fasted) Single dose of 9 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fasted) Single dose of 16 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 16 mg (Fed) Single dose of 16 mg AQ280, fed AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 48 mg (Fasted) Single dose of 48 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral	Part A (SAD): 60 mg (Fasted) Single dose of 60 mg AQ280, fasted AQ280: Dose form: capsule, hard Method of administration: oral
Part B (MAD) - Primary PK Parameter Derived From Plasma Concentration-time Profile of the AQ280 Main Metabolite, AQ282: Area Under the Concentration Time Curve Over a Dosing Interval (AUCτ) Day 1	31.3 h*ng/mL Geometric Coefficient of Variation 33.7	121 h*ng/mL Geometric Coefficient of Variation 43.1	217 h*ng/mL Geometric Coefficient of Variation 44.4	—	—	—	—	—
Part B (MAD) - Primary PK Parameter Derived From Plasma Concentration-time Profile of the AQ280 Main Metabolite, AQ282: Area Under the Concentration Time Curve Over a Dosing Interval (AUCτ) Day 7	37.0 h*ng/mL Geometric Coefficient of Variation 29.7	123 h*ng/mL Geometric Coefficient of Variation 61.0	214 h*ng/mL Geometric Coefficient of Variation 71.6	—	—	—	—	—

SECONDARY outcome

Timeframe: Day 1 and Day 7

Population: Pharmacokinetic population

Maximum observed concentration (Cmax) of metabolite AQ282 following single oral dose administration of 9, 27, and 60 mg AQ280 in a fasted state (Day 1) and multiple oral dose administration of 9, 27, and 60 mg AQ280 once daily for 7 consecutive days in a fasted state (Day 7).