Trial Outcomes & Findings for AGN-151597 (Formerly RST-001) Phase I/II Trial for Advanced Retinitis Pigmentosa (NCT NCT02556736)

Results Overview

An adverse event is any untoward medical occurrence in a subject or clinical investigation patient administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

14 participants

Primary outcome timeframe

Baseline (Day 1) to 6 Months

Results posted on

2026-01-12

Participant Flow

A total of 14 participants were enrolled in the study in the United States.

The Safety Population included all enrolled participants who received at least 1 dose of study treatment and was used for all analyses.

Participant milestones

Participant milestones
Measure	Phase 1: Low Dose Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose Four participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 2: High Dose Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Open Label Treatment Period STARTED	3	4	3	4
Open Label Treatment Period COMPLETED	3	4	2	2
Open Label Treatment Period NOT COMPLETED	0	0	1	2
Long-Term Follow-up STARTED	1	4	2	2
Long-Term Follow-up COMPLETED	0	4	2	1
Long-Term Follow-up NOT COMPLETED	1	0	0	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Phase 1: Low Dose Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 2: High Dose Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Open Label Treatment Period Withdrawal by Subject	0	1	1
Open Label Treatment Period Adverse Event	0	0	1
Long-Term Follow-up Lost to Follow-up	1	0	1

Baseline Characteristics

AGN-151597 (Formerly RST-001) Phase I/II Trial for Advanced Retinitis Pigmentosa

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Phase 1: Low Dose n=3 Participants Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose n=4 Participants Four participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose n=3 Participants Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 2: High Dose n=4 Participants Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Total n=14 Participants Total of all reporting groups
Age, Continuous	66.0 Years STANDARD_DEVIATION 17.09 • n=210 Participants	58.8 Years STANDARD_DEVIATION 4.99 • n=19 Participants	64.7 Years STANDARD_DEVIATION 8.33 • n=8 Participants	50.0 Years STANDARD_DEVIATION 24.12 • n=24 Participants	59.1 Years STANDARD_DEVIATION 15.47 • n=5716 Participants
Sex: Female, Male Female	1 Participants n=210 Participants	2 Participants n=19 Participants	0 Participants n=8 Participants	1 Participants n=24 Participants	4 Participants n=5716 Participants
Sex: Female, Male Male	2 Participants n=210 Participants	2 Participants n=19 Participants	3 Participants n=8 Participants	3 Participants n=24 Participants	10 Participants n=5716 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=210 Participants	0 Participants n=19 Participants	0 Participants n=8 Participants	0 Participants n=24 Participants	0 Participants n=5716 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	3 Participants n=210 Participants	4 Participants n=19 Participants	3 Participants n=8 Participants	4 Participants n=24 Participants	14 Participants n=5716 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=210 Participants	0 Participants n=19 Participants	0 Participants n=8 Participants	0 Participants n=24 Participants	0 Participants n=5716 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=210 Participants	0 Participants n=19 Participants	0 Participants n=8 Participants	0 Participants n=24 Participants	0 Participants n=5716 Participants
Race (NIH/OMB) Asian	1 Participants n=210 Participants	0 Participants n=19 Participants	0 Participants n=8 Participants	0 Participants n=24 Participants	1 Participants n=5716 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=210 Participants	0 Participants n=19 Participants	0 Participants n=8 Participants	0 Participants n=24 Participants	0 Participants n=5716 Participants
Race (NIH/OMB) Black or African American	1 Participants n=210 Participants	0 Participants n=19 Participants	0 Participants n=8 Participants	1 Participants n=24 Participants	2 Participants n=5716 Participants
Race (NIH/OMB) White	1 Participants n=210 Participants	4 Participants n=19 Participants	3 Participants n=8 Participants	3 Participants n=24 Participants	11 Participants n=5716 Participants
Race (NIH/OMB) More than one race	0 Participants n=210 Participants	0 Participants n=19 Participants	0 Participants n=8 Participants	0 Participants n=24 Participants	0 Participants n=5716 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=210 Participants	0 Participants n=19 Participants	0 Participants n=8 Participants	0 Participants n=24 Participants	0 Participants n=5716 Participants

PRIMARY outcome

Timeframe: Baseline (Day 1) to 6 Months

Population: Safety Population

An adverse event is any untoward medical occurrence in a subject or clinical investigation patient administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.

Outcome measures

Outcome measures
Measure	Phase 2: High Dose n=4 Participants Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose n=3 Participants Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose n=4 Participants Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose n=3 Participants Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Number of Participants With Any Grade 3 or Greater Adverse Event (AE) Considered Related to AGN-151597	0 participants	0 participants	0 participants	0 participants

PRIMARY outcome

Timeframe: Baseline (Day 1), 6 Months

Population: Safety Population. Number Analyzed is the number of participants who had the assessment for the parameter at the specified visit. Although subjects who passed the Count Fingers assessment did not need to be evaluated for the Hand Motion assessment, and subjects who passed the Hand Motion assessment did not need to be evaluated further for the Light Perception Assessment, some participants who passed the previous assessments were still evaluated in error for subsequent assessments.

Visual acuity of the study eye (with fellow eye covered) was measured using low vision assessment of count fingers, hand motion, and light perception. For count fingers testing, the examiner's hand presenting 1, 2, or 5 fingers is held 2 feet in front of the eye being examined. If the participant correctly identifies three of five presentations, then count fingers vision is noted. If not, then the participant must be tested for hand motion vision. For hand motion testing, the examiner's hand is extended 2 feet in front of the eye and moved horizontally or vertically. If the participant correctly identifies hand movement four out of five times, then hand motion vision is noted. If not, then the participant is tested for light perception. For light perception testing, a beam of light is directed in and out of the eye at least four times from a distance of 3 feet. If the participant correctly perceives the light, vision should be recorded as yes to light perception.

Outcome measures

Outcome measures
Measure	Phase 2: High Dose n=4 Participants Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose n=3 Participants Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose n=4 Participants Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose n=3 Participants Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Visual Acuity in the Study Eye at Baseline and Month 6 Count Fingers (Baseline)	0 Participants	0 Participants	0 Participants	0 Participants
Visual Acuity in the Study Eye at Baseline and Month 6 Count Fingers (6 months)	0 Participants	0 Participants	0 Participants	0 Participants
Visual Acuity in the Study Eye at Baseline and Month 6 Hand Motion (Baseline)	1 Participants	0 Participants	0 Participants	1 Participants
Visual Acuity in the Study Eye at Baseline and Month 6 Hand Motion (6 months)	1 Participants	0 Participants	1 Participants	1 Participants
Visual Acuity in the Study Eye at Baseline and Month 6 Light Perception (Baseline)	4 Participants	3 Participants	4 Participants	1 Participants
Visual Acuity in the Study Eye at Baseline and Month 6 Light Perception (6 months)	2 Participants	3 Participants	3 Participants	1 Participants

PRIMARY outcome

Timeframe: Baseline (Day 1) to Month 6

Population: Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and specified postbaseline visit (whose vision abilities allowed them to participate in the testing).

The light sensitivity of the visual field was measured by recording the threshold at which a participant reported seeing the dimmest flash. A negative value indicates an increase in sensitivity from baseline.

Outcome measures

Outcome measures
Measure	Phase 2: High Dose n=2 Participants Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose n=1 Participants Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose n=1 Participants Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Change From Baseline at Month 6 in Full Field Sensitivity in the Study Eye - Blue Light Threshold	1.235 Decibels Standard Deviation 5.1760	—	0.404 Decibels Standard Deviation NA Not calculable for n=1	-6.600 Decibels Standard Deviation NA Not calculable for n=1

PRIMARY outcome

Timeframe: Baseline (Day 1) to Month 6

Population: Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and at the 6-month visit (whose vision abilities allowed them to participate in the testing).

The light sensitivity of the visual field was measured by recording the threshold at which a participant reported seeing the dimmest flash. A negative value indicates an increase in sensitivity from baseline.

Outcome measures

Outcome measures
Measure	Phase 2: High Dose n=2 Participants Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose n=1 Participants Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose n=1 Participants Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Change From Baseline at Month 6 in Full Field Sensitivity in the Study Eye - Red Light Threshold	-0.232 Decibels Standard Deviation 1.2601	—	0.903 Decibels Standard Deviation NA Not calculable for n=1	-10.900 Decibels Standard Deviation NA Not calculable for n=1

PRIMARY outcome

Timeframe: Baseline (Day 1) to Month 6

Population: Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and at the 6-month visit (whose vision abilities allowed them to participate in the testing).

The light sensitivity of the visual field was measured by recording the threshold at which a participant reported seeing the dimmest flash. A negative value indicates an increase in sensitivity from baseline.

Outcome measures

Outcome measures
Measure	Phase 2: High Dose n=2 Participants Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose n=2 Participants Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose n=2 Participants Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose n=1 Participants Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Change From Baseline at Month 6 in Full Field Sensitivity in the Study Eye - White Light Threshold	-1.887 Decibels Standard Deviation 2.0520	0.109 Decibels Standard Deviation 1.2459	-6.572 Decibels Standard Deviation 9.3734	-4.700 Decibels Standard Deviation NA Not calculable for n=1

PRIMARY outcome

Timeframe: Baseline (Day 1) to Month 6

Population: Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data (whose vision abilities allowed them to participate in the testing).

Evaluated a participant's ability to navigate within a dark room. The time from the start to stop and the distance from the center of the lit panel to where the participant stopped or touched the target (panel) were recorded. The test was first performed binocularly, then on the study eye (with non-study eye patched). A negative value indicates a decrease from baseline in time or distance from start to stop (improvement).

Outcome measures

Outcome measures
Measure	Phase 2: High Dose n=2 Participants Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose n=2 Participants Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose n=4 Participants Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose n=2 Participants Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Change From Baseline at Month 6 in Ambulation in the Study Eye (Time)	-4.50 seconds Standard Deviation 6.364	-15.50 seconds Standard Deviation 20.506	-7.25 seconds Standard Deviation 10.500	-0.50 seconds Standard Deviation 0.707

PRIMARY outcome

Timeframe: Baseline (Day 1) to Month 6

Population: Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data (whose vision abilities allowed them to participate in the testing).

Evaluated a participant's ability to navigate within a dark room. The time from the start to stop and the distance from the center of the lit panel to where the participant stopped or touched the target (panel) were recorded. The test was first performed binocularly, then on the study eye (with non-study eye patched). A negative value indicates a decrease from baseline in time or distance from start to stop (improvement).

Outcome measures

Outcome measures
Measure	Phase 2: High Dose n=2 Participants Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose n=2 Participants Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose n=4 Participants Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose n=2 Participants Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Change From Baseline at Month 6 in Ambulation in the Study Eye (Distance)	0.02 meters Standard Deviation 0.028	-0.45 meters Standard Deviation 1.025	0.16 meters Standard Deviation 0.649	-0.27 meters Standard Deviation 0.410

PRIMARY outcome

Timeframe: Baseline (Day 1), 6 Months

Population: Safety Population. Number Analyzed is the number of participants who had the assessment for the parameter at the specified visit.

Intraocular pressure was measured using the Goldmann applanation tonometer or a hand-held tonometer (same instrument used for each participant throughout the study, when possible). Measurements were taken at baseline (pre-injection) and at 6 months.

Outcome measures

Outcome measures
Measure	Phase 2: High Dose n=4 Participants Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose n=3 Participants Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose n=4 Participants Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose n=3 Participants Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Intraocular Pressure (IOP) Measurements in the Study Eye Baseline (pre-injection)	14.0 mmHg Standard Deviation 2.94	13.3 mmHg Standard Deviation 0.58	13.5 mmHg Standard Deviation 1.91	18.7 mmHg Standard Deviation 3.51
Intraocular Pressure (IOP) Measurements in the Study Eye Month 6	15.0 mmHg Standard Deviation 4.24	13.3 mmHg Standard Deviation 0.58	15.3 mmHg Standard Deviation 5.03	17.0 mmHg Standard Deviation 3.00

PRIMARY outcome

Timeframe: Baseline (Day 1), 6 Months

Population: Safety Population. The Number Analyzed is the number of participants who completed the assessment with evaluable data the specified visit (whose vision abilities allowed them to participate in the testing).

A standardized procedure for the collection of single, non-stereo images of the fundus of both eyes was obtained using the same equipment for each participant throughout the study. Evidence of increased inflammation, hemorrhage, retinal detachment, RPE disturbance or atrophy in the fovea, and any changes from baseline visit were documented. Any changes from baseline in fundus autofluorescence were also documented.

Outcome measures

Outcome measures
Measure	Phase 2: High Dose n=4 Participants Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose n=3 Participants Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose n=4 Participants Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose n=3 Participants Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence Any Evidence of Retinal Pigment Epithelium (RPE) Disturbance (Month 6)	2 Participants	1 Participants	2 Participants	1 Participants
Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence Any Evidence of Atrophy in the Fovea (Month 6)	0 Participants	1 Participants	2 Participants	1 Participants
Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence Any Evidence of Other findings (Month 6)	1 Participants	0 Participants	0 Participants	1 Participants
Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence Any Evidence of Inflammation (Baseline)	0 Participants	0 Participants	0 Participants	0 Participants
Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence Any Evidence of Retinal Hemorrhage (Baseline)	0 Participants	0 Participants	0 Participants	0 Participants
Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence Any Evidence of Retinal Detachment (Baseline)	0 Participants	0 Participants	0 Participants	0 Participants
Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence Any Evidence of Retinal Pigment Epithelium (RPE) Disturbance (Baseline)	3 Participants	1 Participants	2 Participants	2 Participants
Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence Any Evidence of Atrophy in the Fovea (Baseline)	1 Participants	1 Participants	2 Participants	1 Participants
Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence Any Evidence of Other findings (Baseline)	1 Participants	0 Participants	0 Participants	1 Participants
Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence Any findings of Autofluorescence (Baseline)	2 Participants	1 Participants	2 Participants	2 Participants
Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence Any Evidence of Inflammation (Month 6)	1 Participants	0 Participants	0 Participants	0 Participants
Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence Any Evidence of Retinal Hemorrhage (Month 6)	0 Participants	0 Participants	0 Participants	0 Participants
Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence Any Evidence of Retinal Detachment (Month 6)	0 Participants	0 Participants	0 Participants	0 Participants

PRIMARY outcome

Timeframe: Baseline (Day 1) to Month 6

Population: Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and the 6-month visit (whose vision abilities allowed them to participate in the testing).

Qualitative assessment of the change in retinal cross-sectional appearance in the study eye from Baseline at month 6 as Measured by SD-OCT.

Outcome measures

Outcome measures
Measure	Phase 2: High Dose Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose n=1 Participants Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose n=2 Participants Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Change From Baseline at Month 6 in the Anatomical Parameters in the Study Eye as Measured by Spectral Domain-Optical Coherence Tomography (SD-OCT) - Total Retinal Nerve Fibre Layer Volume	—	—	1.760 mm3 Standard Deviation NA Not calculable for n=1	0.005 mm3 Standard Deviation 0.0212

PRIMARY outcome

Timeframe: Baseline (Day 1) to Month 6

Population: Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and specified postbaseline visit (whose vision abilities allowed them to participate in the testing).

Qualitative assessment of the change in retinal cross-sectional appearance in the study eye from Baseline at month 6 as Measured by SD-OCT.

Outcome measures

Outcome measures
Measure	Phase 2: High Dose Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose n=1 Participants Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose n=2 Participants Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Change From Baseline at Month 6 in the Anatomical Parameters in the Study Eye as Measured by Spectral Domain-Optical Coherence Tomography (SD-OCT) - Total Ganglion Cell and Inner Plexiform Layer Volume	—	—	0.180 mm3 Standard Deviation NA Not calculable for n=1	-0.055 mm3 Standard Deviation 0.0636

PRIMARY outcome

Timeframe: Baseline (Day 1) to Month 6

Population: Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and specified postbaseline visit (whose vision abilities allowed them to participate in the testing).

Qualitative assessment of the change in retinal cross-sectional appearance in the study eye from Baseline at month 6 as Measured by SD-OCT.

Outcome measures

Outcome measures
Measure	Phase 2: High Dose Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose n=2 Participants Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Change From Baseline at Month 6 in the Anatomical Parameters in the Study Eye as Measured by Spectral Domain-Optical Coherence Tomography (SD-OCT) - Total Retinal Volume	—	—	—	0.570 mm3 Standard Deviation 0.7495

PRIMARY outcome

Timeframe: Baseline (Day 1) to Month 6

Population: Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and specified postbaseline visit (whose vision abilities allowed them to participate in the testing).

Qualitative assessment of the change in retinal cross-sectional appearance in the study eye from Baseline at month 6 as Measured by SD-OCT.

Outcome measures

Outcome measures
Measure	Phase 2: High Dose Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose n=1 Participants Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose n=2 Participants Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Change From Baseline at Month 6 in the Anatomical Parameters in the Study Eye as Measured by Spectral Domain-Optical Coherence Tomography (SD-OCT) - Total Retinal Thickness	—	—	14.0 um Standard Deviation NA Not calculable for n=1	60.0 um Standard Deviation 86.27

SECONDARY outcome

Timeframe: 3, 12, and 24 Months

Population: Safety Population. Number Analyzed is the number of participants who had the assessment for the parameter at the specified visit. Although subjects who passed the Count Fingers assessment did not need to be evaluated for the Hand Motion assessment, and subjects who passed the Hand Motion assessment did not need to be evaluated further for the Light Perception Assessment, some participants who passed the previous assessments were still evaluated in error for subsequent assessments.

Visual acuity of the study eye (with fellow eye covered) was measured using low vision assessment of count fingers, hand motion, and light perception. For count fingers testing, the examiner's hand presenting 1, 2, or 5 fingers is held 2 feet in front of the eye being examined. If the participant correctly identifies three of five presentations, then count fingers vision is noted. If not, then the participant must be tested for hand motion vision. For hand motion testing, the examiner's hand is extended 2 feet in front of the eye and moved horizontally or vertically. If the participant correctly identifies hand movement four out of five times, then hand motion vision is noted. If not, then the participant is tested for light perception. For light perception testing, a beam of light is directed in and out of the eye at least four times from a distance of 3 feet. If the participant correctly perceives the light, vision should be recorded as yes to light perception.

Outcome measures

Outcome measures
Measure	Phase 2: High Dose n=4 Participants Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose n=3 Participants Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose n=4 Participants Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose n=3 Participants Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Visual Acuity in the Study Eye at Months 3, 12, and 24 Count Fingers (3 months)	0 Participants	0 Participants	0 Participants	0 Participants
Visual Acuity in the Study Eye at Months 3, 12, and 24 Count Fingers (12 months)	0 Participants	0 Participants	0 Participants	0 Participants
Visual Acuity in the Study Eye at Months 3, 12, and 24 Count Fingers (24 months)	0 Participants	0 Participants	0 Participants	0 Participants
Visual Acuity in the Study Eye at Months 3, 12, and 24 Hand Motion (3 months)	1 Participants	0 Participants	2 Participants	1 Participants
Visual Acuity in the Study Eye at Months 3, 12, and 24 Hand Motion (12 months)	1 Participants	0 Participants	1 Participants	1 Participants
Visual Acuity in the Study Eye at Months 3, 12, and 24 Light Perception (12 months)	3 Participants	2 Participants	3 Participants	1 Participants
Visual Acuity in the Study Eye at Months 3, 12, and 24 Hand Motion (24 months)	0 Participants	0 Participants	1 Participants	2 Participants
Visual Acuity in the Study Eye at Months 3, 12, and 24 Light Perception (3 months)	4 Participants	3 Participants	2 Participants	1 Participants
Visual Acuity in the Study Eye at Months 3, 12, and 24 Light Perception (24 months)	1 Participants	3 Participants	3 Participants	—

SECONDARY outcome

Timeframe: Baseline (Day 1) to Months 3, 12, and 24

Population: Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and at least one of the postbaseline visits (whose vision abilities allowed them to participate in the testing).

The light sensitivity of the visual field was measured by recording the threshold at which a participant reported seeing the dimmest flash. A negative value indicates an increase in sensitivity from baseline.

Outcome measures

Outcome measures
Measure	Phase 2: High Dose n=4 Participants Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose n=1 Participants Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose n=2 Participants Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Change From Baseline at Months 3, 12, and 24 in Full Field Sensitivity in the Study Eye - Blue Light Threshold Blue Light Threshold (12 months)	-0.905 Decibels Standard Deviation 3.0047	—	0.008 Decibels Standard Deviation NA Not calculable for n=1	-7.300 Decibels Standard Deviation 7.2125
Change From Baseline at Months 3, 12, and 24 in Full Field Sensitivity in the Study Eye - Blue Light Threshold Blue Light Threshold (24 months)	-1.121 Decibels Standard Deviation NA Not calculable for n=1	—	3.332 Decibels Standard Deviation NA Not calculable for n=1	-8.100 Decibels Standard Deviation NA Not calculable for n=1
Change From Baseline at Months 3, 12, and 24 in Full Field Sensitivity in the Study Eye - Blue Light Threshold Blue Light Threshold (3 months)	-0.475 Decibels Standard Deviation 2.7628	—	-1.097 Decibels Standard Deviation NA Not calculable for n=1	-10.050 Decibels Standard Deviation 12.7986

SECONDARY outcome

Timeframe: Baseline (Day 1) to Months 3, 12, and 24

Population: Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and at least one of the postbaseline visits (whose vision abilities allowed them to participate in the testing).

The light sensitivity of the visual field was measured by recording the threshold at which a participant reported seeing the dimmest flash. A negative value indicates an increase in sensitivity from baseline.

Outcome measures

Outcome measures
Measure	Phase 2: High Dose n=4 Participants Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose n=2 Participants Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose n=2 Participants Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Change From Baseline at Months 3, 12, and 24 in Full Field Sensitivity in the Study Eye - Red Light Threshold Red Light Threshold (3 months)	-1.046 Decibels Standard Deviation 1.9526	—	1.259 Decibels Standard Deviation 1.9389	-5.700 Decibels Standard Deviation 8.7681
Change From Baseline at Months 3, 12, and 24 in Full Field Sensitivity in the Study Eye - Red Light Threshold Red Light Threshold (12 months)	1.537 Decibels Standard Deviation 4.5122	—	-0.301 Decibels Standard Deviation NA Not calculable for n=1	-5.100 Decibels Standard Deviation 8.3439
Change From Baseline at Months 3, 12, and 24 in Full Field Sensitivity in the Study Eye - Red Light Threshold Red Light Threshold (24 months)	-1.876 Decibels Standard Deviation NA Not calculable for n=1	—	2.901 Decibels Standard Deviation 6.2119	-10.000 Decibels Standard Deviation NA Not calculable for n=1

SECONDARY outcome

Timeframe: Baseline (Day 1) to Months 3, 12, and 24

Population: Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and at least one of the postbaseline visits (whose vision abilities allowed them to participate in the testing).

The light sensitivity of the visual field was measured by recording the threshold at which a participant reported seeing the dimmest flash. A negative value indicates an increase in sensitivity from baseline.

Outcome measures

Outcome measures
Measure	Phase 2: High Dose n=4 Participants Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose n=2 Participants Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose n=2 Participants Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose n=2 Participants Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Change From Baseline at Months 3, 12, and 24 in Full Field Sensitivity in the Study Eye - White Light Threshold White Light Threshold (24 months)	-3.887 Decibels Standard Deviation NA Not calculable for n=1	3.882 Decibels Standard Deviation 1.5373	-5.734 Decibels Standard Deviation 12.5384	-3.000 Decibels Standard Deviation NA Not calculable for n=1
Change From Baseline at Months 3, 12, and 24 in Full Field Sensitivity in the Study Eye - White Light Threshold White Light Threshold (3 months)	-0.066 Decibels Standard Deviation 2.4312	-0.666 Decibels Standard Deviation 6.6383	-1.988 Decibels Standard Deviation 2.1383	-7.500 Decibels Standard Deviation 11.7380
Change From Baseline at Months 3, 12, and 24 in Full Field Sensitivity in the Study Eye - White Light Threshold White Light Threshold (12 months)	-1.699 Decibels Standard Deviation 1.2484	-0.351 Decibels Standard Deviation 2.2642	-4.414 Decibels Standard Deviation 7.3348	-9.500 Decibels Standard Deviation 3.8184

SECONDARY outcome

Timeframe: Baseline (Day 1) to Months 3, 12, and 24

Population: Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and at least one of the postbaseline visits (whose vision abilities allowed them to participate in the testing).

Evaluated a participant's ability to navigate within a dark room. The time from the start to stop and the distance from the center of the lit panel to where the participant stopped or touched the target (panel) were recorded. The test was first performed binocularly, then on the study eye (with non-study eye patched). A negative value indicates a decrease from baseline in time or distance from start to stop (improvement).

Outcome measures

Outcome measures
Measure	Phase 2: High Dose n=4 Participants Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose n=2 Participants Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose n=4 Participants Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose n=2 Participants Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Change From Baseline at Months 3, 12, and 24 in Ambulation Light-Guided Walking Test in the Study Eye (Time) Mean Time (seconds) (3 months)	1.50 seconds Standard Deviation 2.082	-19.00 seconds Standard Deviation 14.142	-6.00 seconds Standard Deviation 13.441	0.00 seconds Standard Deviation 0.000
Change From Baseline at Months 3, 12, and 24 in Ambulation Light-Guided Walking Test in the Study Eye (Time) Mean Time (seconds) (12 months)	0.00 seconds Standard Deviation 4.000	-15.50 seconds Standard Deviation 21.920	-12.00 seconds Standard Deviation 16.573	-3.00 seconds Standard Deviation 4.243
Change From Baseline at Months 3, 12, and 24 in Ambulation Light-Guided Walking Test in the Study Eye (Time) Mean Time (seconds) (24 months)	-6.00 seconds Standard Deviation NA Not calculable for n=1	-16.00 seconds Standard Deviation 25.456	-11.50 seconds Standard Deviation 17.253	-2.00 seconds Standard Deviation 1.414

SECONDARY outcome

Timeframe: Baseline (Day 1) to Months 3, 12, and 24

Population: Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and at least one of the postbaseline visits (whose vision abilities allowed them to participate in the testing).

Evaluated a participant's ability to navigate within a dark room. The time from the start to stop and the distance from the center of the lit panel to where the participant stopped or touched the target (panel) were recorded. The test was first performed binocularly, then on the study eye (with non-study eye patched). A negative value indicates a decrease from baseline in time or distance from start to stop (improvement).

Outcome measures

Outcome measures
Measure	Phase 2: High Dose n=3 Participants Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose n=2 Participants Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose n=4 Participants Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose n=2 Participants Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Change From Baseline at Months 3, 12, and 24 in Ambulation Light-Guided Walking Test in the Study Eye (Distance) Mean Distance (meters) (3 months)	-0.01 meters Standard Deviation 0.012	-0.57 meters Standard Deviation 1.011	0.07 meters Standard Deviation 0.369	-0.16 meters Standard Deviation 0.247
Change From Baseline at Months 3, 12, and 24 in Ambulation Light-Guided Walking Test in the Study Eye (Distance) Mean Distance (meters) (12 months)	0.02 meters Standard Deviation 0.000	-0.26 meters Standard Deviation 1.344	0.20 meters Standard Deviation 0.529	0.38 meters Standard Deviation 0.467
Change From Baseline at Months 3, 12, and 24 in Ambulation Light-Guided Walking Test in the Study Eye (Distance) Mean Distance (meters) (24 months)	-0.02 meters Standard Deviation NA Not calculable for n=1	0.13 meters Standard Deviation 0.884	0.14 meters Standard Deviation 0.638	-0.19 meters Standard Deviation 0.318

SECONDARY outcome

Timeframe: Baseline (Day 1) to Months 3, 6, and 24

Population: Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and at least one of the postbaseline visits (whose vision abilities allowed them to participate in the testing).

Participants were to perform two tests concurrently, first identifying if a light displayed on an LED screen can be seen and then if a series of standard images (square, circle, triangle, or star) presented on the screen can be identified. The shapes were presented in blue or red at varying intensities. Data on light color and threshold intensity of the light was collected; shape detection data was not collected. The change from baseline in threshold intensity at 3, 6, and 24 months is reported. A negative change from baseline suggests an improvement.

Outcome measures

Outcome measures
Measure	Phase 2: High Dose n=2 Participants Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose n=2 Participants Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose n=1 Participants Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Change From Baseline at Months 3, 6, and 24 in Object Detection and Discrimination Scores Blue Threshold Intensity (3 months)	-1.865 cd/m2 Standard Deviation 11.8409	-68.939 cd/m2 Standard Deviation 146.2578	—	5.998 cd/m2 Standard Deviation NA Not calculable for n=1.
Change From Baseline at Months 3, 6, and 24 in Object Detection and Discrimination Scores Red Threshold Intensity (3 months)	10323.422 cd/m2 Standard Deviation 14244.9927	-18925.624 cd/m2 Standard Deviation NA Not calculable for n=1.	—	—
Change From Baseline at Months 3, 6, and 24 in Object Detection and Discrimination Scores Blue Threshold Intensity (6 months)	0.515 cd/m2 Standard Deviation 2.3009	-124.924 cd/m2 Standard Deviation 184.2505	—	0.000 cd/m2 Standard Deviation NA Not calculable for n=1.
Change From Baseline at Months 3, 6, and 24 in Object Detection and Discrimination Scores Red Threshold Intensity (6 months)	3.195 cd/m2 Standard Deviation 8.0718	-18952.831 cd/m2 Standard Deviation NA Not calculable for n=1.	—	-184.612 cd/m2 Standard Deviation NA Not calculable for n=1.
Change From Baseline at Months 3, 6, and 24 in Object Detection and Discrimination Scores Blue Threshold Intensity (24 months)	199.747 cd/m2 Standard Deviation NA Not calculable for n=1.	334.603 cd/m2 Standard Deviation NA Not calculable for n=1.	—	—
Change From Baseline at Months 3, 6, and 24 in Object Detection and Discrimination Scores Red Threshold Intensity (24 months)	-1.266 cd/m2 Standard Deviation NA Not calculable for n=1.	—	—	—

SECONDARY outcome

Timeframe: Baseline (Day 1) to Months 3, 6, and 24

Population: Safety Population. The Overall Number of Participants Analyzed is the number of participants who completed assessment with evaluable data at baseline and at least one postbaseline visit at Month 3, Month 6, or Month 24. The Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and specified postbaseline visit (whose vision abilities allowed them to participate in the testing)

Visual Evoked Potential (VEP) is an eye assessment that measures how the brain responds to visual stimuli. The pattern of visual evoked potentials was obtained from each eye using a VEP stimulator according to standard protocols. Three different visual stimuli were assessed. Amplitude measures how strong the signal is when your brain responds to visual stimuli. A higher amplitude indicates an improvement and a lower amplitude indicates a worsening outcome. Latency is the time it takes for the signal to reach the brain after the visual stimulus is presented. A shorter latency indicates an improvement from baseline; a longer latency indicates a worsening outcome.

Outcome measures

Outcome measures
Measure	Phase 2: High Dose n=1 Participants Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose n=2 Participants Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.974 Amplitude) SE/0.974 Amplitude (mV) - 3 months	1.0 mV Standard Deviation NA Not calculable for n=1	0.0 mV Standard Deviation NA Not calculable for n=1	—	—
Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.974 Amplitude) SE/0.974 Amplitude (mV) - 24 months	—	47.0 mV Standard Deviation NA Not calculable for n=1	—	—

SECONDARY outcome

Timeframe: Baseline (Day 1) to Months 3, 6, and 24

Population: Safety Population. The Overall Number of Participants Analyzed is the number of participants assessed at baseline. The Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and specified postbaseline visit (whose vision abilities allowed them to participate in the testing)

Visual Evoked Potential (VEP) is an eye assessment that measures how the brain responds to visual stimuli. The pattern of visual evoked potentials was obtained from each eye using a VEP stimulator according to standard protocols. Three different visual stimuli were assessed. Amplitude measures how strong the signal is when your brain responds to visual stimuli. A higher amplitude indicates an improvement and a lower amplitude indicates a worsening outcome. Latency is the time it takes for the signal to reach the brain after the visual stimulus is presented. A shorter latency indicates an improvement from baseline; a longer latency indicates a worsening outcome.

Outcome measures

Outcome measures
Measure	Phase 2: High Dose n=1 Participants Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose n=3 Participants Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.649 Amplitude) SE/0.649 Amplitude (mV) - 24 months	—	10.3 mV Standard Deviation 19.66	—	—
Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.649 Amplitude) SE/0.649 Amplitude (mV) - 3 months	0.0 mV Standard Deviation NA Not calculable for n=1	0.0 mV Standard Deviation NA Not calculable for n=1	—	—

SECONDARY outcome

Timeframe: Baseline (Day 1) to Months 3, 6, and 24

Population: Safety Population. The Overall Number of Participants Analyzed is the number of participants assessed at baseline. The Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and specified postbaseline visit (whose vision abilities allowed them to participate in the testing).

Visual Evoked Potential (VEP) is an eye assessment that measures how the brain responds to visual stimuli. The pattern of visual evoked potentials was obtained from each eye using a VEP stimulator according to standard protocols. Three different visual stimuli were assessed. Amplitude measures how strong the signal is when your brain responds to visual stimuli. A higher amplitude indicates an improvement and a lower amplitude indicates a worsening outcome. Latency is the time it takes for the signal to reach the brain after the visual stimulus is presented. A shorter latency indicates an improvement from baseline; a longer latency indicates a worsening outcome.

Outcome measures

Outcome measures
Measure	Phase 2: High Dose n=1 Participants Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose n=3 Participants Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.216 Amplitude) SE/0.216 Amplitude (mV) - 3 months	0.0 mV Standard Deviation NA Not calculable for n=1	-4.0 mV Standard Deviation NA Not calculable for n=1	—	—
Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.216 Amplitude) SE/0.216 Amplitude (mV) - 24 months	—	40.5 mV Standard Deviation 55.86	—	—

SECONDARY outcome

Timeframe: Baseline (Day 1) to Months 3, 6, and 24

Population: Safety Population. The Overall Number of Participants Analyzed is the number of participants assessed at baseline. The Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and specified postbaseline visit (whose vision abilities allowed them to participate in the testing).

Visual Evoked Potential (VEP) is an eye assessment that measures how the brain responds to visual stimuli. The pattern of visual evoked potentials was obtained from each eye using a VEP stimulator according to standard protocols. Three different visual stimuli were assessed. Amplitude measures how strong the signal is when your brain responds to visual stimuli. A higher amplitude indicates an improvement and a lower amplitude indicates a worsening outcome. Latency is the time it takes for the signal to reach the brain after the visual stimulus is presented. A shorter latency indicates an improvement from baseline; a longer latency indicates a worsening outcome.

Outcome measures

Outcome measures
Measure	Phase 2: High Dose n=1 Participants Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose n=2 Participants Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose n=1 Participants Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.974 Latency) SE/0.974 Latency (msec) - 3 months	25.0 msec Standard Deviation NA Not calculable for n=1	-1.0 msec Standard Deviation NA Not calculable for n=1	—	—
Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.974 Latency) SE/0.974 Latency (msec) - 24 months	—	40.0 msec Standard Deviation NA Not calculable for n=1	—	—

SECONDARY outcome

Timeframe: Baseline (Day 1) to Months 3, 6, and 24

Population: Safety Population. The Overall Number of Participants Analyzed is the number of participants assessed at baseline. The Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and specified postbaseline visit (whose vision abilities allowed them to participate in the testing).

Visual Evoked Potential (VEP) is an eye assessment that measures how the brain responds to visual stimuli. The pattern of visual evoked potentials was obtained from each eye using a VEP stimulator according to standard protocols. Three different visual stimuli were assessed. Amplitude measures how strong the signal is when your brain responds to visual stimuli. A higher amplitude indicates an improvement and a lower amplitude indicates a worsening outcome. Latency is the time it takes for the signal to reach the brain after the visual stimulus is presented. A shorter latency indicates an improvement from baseline; a longer latency indicates a worsening outcome.

Outcome measures

Outcome measures
Measure	Phase 2: High Dose n=1 Participants Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose n=3 Participants Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.649 Latency) SE/0.649 Latency (msec) - 3 months	15.0 msec Standard Deviation NA Not calculable for n=1	13.0 msec Standard Deviation NA Not calculable for n=1	—	—
Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.649 Latency) SE/0.649 Latency (msec) - 24 months	—	-6.3 msec Standard Deviation 10.02	—	—

SECONDARY outcome

Timeframe: Baseline (Day 1) to Months 3, 6, and 24

Population: Safety Population. The Overall Number of Participants Analyzed is the number of participants assessed at baseline. The Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and specified postbaseline visit (whose vision abilities allowed them to participate in the testing).

Visual Evoked Potential (VEP) is an eye assessment that measures how the brain responds to visual stimuli. The pattern of visual evoked potentials was obtained from each eye using a VEP stimulator according to standard protocols. Three different visual stimuli were assessed. Amplitude measures how strong the signal is when your brain responds to visual stimuli. A higher amplitude indicates an improvement and a lower amplitude indicates a worsening outcome. Latency is the time it takes for the signal to reach the brain after the visual stimulus is presented. A shorter latency indicates an improvement from baseline; a longer latency indicates a worsening outcome.

Outcome measures

Outcome measures
Measure	Phase 2: High Dose n=1 Participants Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose n=3 Participants Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.216 Latency) SE/0.216 Latency (msec) - 3 months	7.0 msec Standard Deviation NA Not calculable for n=1	-21.0 msec Standard Deviation NA Not calculable for n=1	—	—
Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.216 Latency) SE/0.216 Latency (msec) - 24 months	—	22.0 msec Standard Deviation 5.66	—	—

SECONDARY outcome

Timeframe: Baseline (Day 1) to Months 6 and 24

Population: Safety Population. The Overall Number of Participants Analyzed is the number of participants assessed at baseline. The Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and specified postbaseline visit (whose vision abilities allowed them to participate in the testing).

Full field electroretinography based on standards set by the ERG Standardization Committee of the International Society for Clinical Electrophysiology of Vision (ISCEV) was performed at the Screening visit, 6 months, and at 24 months. ERG tests how well the light sensitive part of the eye (retina) is working. Several assessments were performed to evaluate how well different parts of the retina respond to light in different settings. Amplitude is the strength of the electrical signal that the retina produces in response to light during an ERG test. Higher amplitude in A-wave or b-wave indicates improvement; lower amplitude indicates worsening.

Outcome measures

Outcome measures
Measure	Phase 2: High Dose n=3 Participants Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose n=3 Participants Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose n=4 Participants Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose n=3 Participants Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Change From Baseline at Months 6 and 24 in Electroretinogram (ERG) Scores in the Study Eye (Amplitude) Dark Adapted 0.01 ERG b-wave (uV) - 6 months	0.0 uV Standard Deviation NA Not calculable for n=1	0.0 uV Standard Deviation 0.0	0.0 uV Standard Deviation 0.0	0.0 uV Standard Deviation 0.0
Change From Baseline at Months 6 and 24 in Electroretinogram (ERG) Scores in the Study Eye (Amplitude) Dark Adapted 3 ERG a-wave (uV) - 6 months	0.0 uV Standard Deviation NA Not calculable for n=1	0.0 uV Standard Deviation 0.0	0.0 uV Standard Deviation 0.0	0.0 uV Standard Deviation 0.0
Change From Baseline at Months 6 and 24 in Electroretinogram (ERG) Scores in the Study Eye (Amplitude) Dark Adapted 3 ERG b-wave (uV) - 6 months	0.0 uV Standard Deviation NA Not calculable for n=1	0.0 uV Standard Deviation 0.0	0.0 uV Standard Deviation 0.0	0.0 uV Standard Deviation 0.0
Change From Baseline at Months 6 and 24 in Electroretinogram (ERG) Scores in the Study Eye (Amplitude) Light Adapted 3 ERG a-wave (uV) - 6 months	0.0 uV Standard Deviation NA Not calculable for n=1	0.0 uV Standard Deviation 0.0	0.0 uV Standard Deviation 0.0	0.0 uV Standard Deviation 0.0
Change From Baseline at Months 6 and 24 in Electroretinogram (ERG) Scores in the Study Eye (Amplitude) Light Adapted 3 ERG b-wave (uV) - 6 months	0.0 uV Standard Deviation NA Not calculable for n=1	0.0 uV Standard Deviation 0.0	0.0 uV Standard Deviation 0.0	0.0 uV Standard Deviation 0.0
Change From Baseline at Months 6 and 24 in Electroretinogram (ERG) Scores in the Study Eye (Amplitude) Light Adapted 30 Flicker Amplitude (uV) - 6 months	0.0000 uV Standard Deviation NA Not calculable for n=1	-0.0423 uV Standard Deviation 0.14301	-0.2318 uV Standard Deviation 0.28927	0.0000 uV Standard Deviation 0.0000
Change From Baseline at Months 6 and 24 in Electroretinogram (ERG) Scores in the Study Eye (Amplitude) Dark Adapted 0.01 ERG b-wave (uV) - 24 months	0.0 uV Standard Deviation NA Not calculable for n=1	0.0 uV Standard Deviation 0.0	0.0 uV Standard Deviation 0.0	0.0 uV Standard Deviation 0.0
Change From Baseline at Months 6 and 24 in Electroretinogram (ERG) Scores in the Study Eye (Amplitude) Dark Adapted 3 ERG a-wave (uV) - 24 months	0.0 uV Standard Deviation NA Not calculable for n=1	0.0 uV Standard Deviation 0.0	0.0 uV Standard Deviation 0.0	0.0 uV Standard Deviation 0.0
Change From Baseline at Months 6 and 24 in Electroretinogram (ERG) Scores in the Study Eye (Amplitude) Dark Adapted 3 ERG b-wave (uV) - 24 months	0.0 uV Standard Deviation NA Not calculable for n=1	0.0 uV Standard Deviation 0.0	0.0 uV Standard Deviation 0.0	0.0 uV Standard Deviation 0.0
Change From Baseline at Months 6 and 24 in Electroretinogram (ERG) Scores in the Study Eye (Amplitude) Light Adapted 3 ERG a-wave (uV) - 24 months	0.0 uV Standard Deviation NA Not calculable for n=1	0.0 uV Standard Deviation 0.0	0.0 uV Standard Deviation 0.0	0.0 uV Standard Deviation 0.0
Change From Baseline at Months 6 and 24 in Electroretinogram (ERG) Scores in the Study Eye (Amplitude) Light Adapted 3 ERG b-wave (uV) - 24 months	0.0 uV Standard Deviation NA Not calculable for n=1	0.0 uV Standard Deviation 0.0	0.0 uV Standard Deviation 0.0	0.0 uV Standard Deviation 0.0
Change From Baseline at Months 6 and 24 in Electroretinogram (ERG) Scores in the Study Eye (Amplitude) Light Adapted 30 Flicker Amplitude (uV) - 24 months	0.0000 uV Standard Deviation NA Not calculable for n=1	0.7897 uV Standard Deviation 0.95459	-0.2630 uV Standard Deviation 0.26256	0.0000 uV Standard Deviation 0.0000

SECONDARY outcome

Timeframe: Baseline (Day 1) to Months 6 and 24

Population: Safety Population. The Overall Number of Participants Analyzed is the number of participants who completed assessment with evaluable data at baseline and at least one postbaseline visit at Month 6 or Month 24. The Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and specified postbaseline visit (whose vision abilities allowed them to participate in the testing).

Full field electroretinography based on standards set by the ERG Standardization Committee of the International Society for Clinical Electrophysiology of Vision (ISCEV) was performed at the Screening visit, 6 months, and at 24 months. ERG tests how well the light sensitive part of the eye (retina) is working. Several assessments were performed to evaluate how well different parts of the retina respond to light in different settings. Amplitude is the strength of the electrical signal that the retina produces in response to light during an ERG test. Latency is the time it takes for the retina to respond after a light is flashed in the eye during an ERG test. Shorter latency indicates an improvement; longer latency indicates a worsening.

Outcome measures

Outcome measures
Measure	Phase 2: High Dose n=3 Participants Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose n=3 Participants Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose n=3 Participants Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Change From Baseline at Months 6 and 24 in Electroretinogram (ERG) Scores in the Study Eye (Latency) Dark Adapted 0.01 ERG Latency (msec) - 6 months	0.0 msec Standard Deviation NA Not calculable for n=1	0.0 msec Standard Deviation 0.0	0.0 msec Standard Deviation 0.0	—
Change From Baseline at Months 6 and 24 in Electroretinogram (ERG) Scores in the Study Eye (Latency) Dark Adapted 3 ERG Latency (msec) - 6 months	0.0 msec Standard Deviation NA Not calculable for n=1	0.0 msec Standard Deviation 0.0	0.0 msec Standard Deviation 0.0	—
Change From Baseline at Months 6 and 24 in Electroretinogram (ERG) Scores in the Study Eye (Latency) Light Adapted 3 ERG Latency (msec) - 6 months	0.0 msec Standard Deviation NA Not calculable for n=1	0.0 msec Standard Deviation 0.0	0.0 msec Standard Deviation 0.0	—
Change From Baseline at Months 6 and 24 in Electroretinogram (ERG) Scores in the Study Eye (Latency) Light Adapted 3 Flicker Latency (msec) - 6 months	0.0 msec Standard Deviation NA Not calculable for n=1	-20.0 msec Standard Deviation 22.54	-24.3 msec Standard Deviation 37.07	—
Change From Baseline at Months 6 and 24 in Electroretinogram (ERG) Scores in the Study Eye (Latency) Dark Adapted 0.01 ERG Latency (msec) - 24 months	0.0 msec Standard Deviation NA Not calculable for n=1	0.0 msec Standard Deviation 0.0	0.0 msec Standard Deviation 0.0	—
Change From Baseline at Months 6 and 24 in Electroretinogram (ERG) Scores in the Study Eye (Latency) Dark Adapted 3 ERG Latency (msec) - 24 months	0.0 msec Standard Deviation NA Not calculable for n=1	0.0 msec Standard Deviation 0.0	0.0 msec Standard Deviation 0.0	—
Change From Baseline at Months 6 and 24 in Electroretinogram (ERG) Scores in the Study Eye (Latency) Light Adapted 3 ERG Latency (msec) - 24 months	0.0 msec Standard Deviation NA Not calculable for n=1	0.0 msec Standard Deviation 0.0	0.0 msec Standard Deviation 0.0	—
Change From Baseline at Months 6 and 24 in Electroretinogram (ERG) Scores in the Study Eye (Latency) Light Adapted 3 Flicker Latency (msec) - 24 months	0.0 msec Standard Deviation NA Not calculable for n=1	-4.7 msec Standard Deviation 23.16	-40.3 msec Standard Deviation 17.39	—

SECONDARY outcome

Timeframe: Baseline (Day 1) to Months 3, 6, and 24

Population: Safety Population. Number Analyzed is the number of participants who had the assessment for the parameter at the specified visit.

Change in quality of life, based on composite scores of the NEI VFQ-25 from Baseline at 3, 6, and 24 months. The NEI VFQ-25 consists of 25 vision-targeted questions that represent 11 vision-related quality of life subscales and one general health item. The 11 subscales are general vision, difficulty with near vision activities, difficulty with distance vision activities, limitation in social functioning due to vision, role limitation due to vision, dependency on others due to vision, mental health symptoms due to vision, driving difficulties, limitation with peripheral vision, limitation with color vision, and ocular pain. NEI VFQ-25 scores range from 0 to 100, with a higher score representing better functioning. A positive value change from baseline indicates an improvement in vision-related quality of life.

Outcome measures

Outcome measures
Measure	Phase 2: High Dose n=4 Participants Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose n=3 Participants Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose n=4 Participants Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose n=3 Participants Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Change From Baseline at Months 3, 6, and 24 in Composite Score of National Eye Institute (NEI) Visual Functioning Questionnaire (VFQ-25) Scores Month 3	0.9 score on a scale Standard Deviation 14.76	4.3 score on a scale Standard Deviation 9.65	5.5 score on a scale Standard Deviation 6.18	-5.3 score on a scale Standard Deviation 0.84
Change From Baseline at Months 3, 6, and 24 in Composite Score of National Eye Institute (NEI) Visual Functioning Questionnaire (VFQ-25) Scores Month 6	9.4 score on a scale Standard Deviation 18.27	4.8 score on a scale Standard Deviation 14.37	6.2 score on a scale Standard Deviation 7.45	-6.6 score on a scale Standard Deviation 3.37
Change From Baseline at Months 3, 6, and 24 in Composite Score of National Eye Institute (NEI) Visual Functioning Questionnaire (VFQ-25) Scores Month 24	3.1 score on a scale Standard Deviation NA Not calculable for n=1.	8.3 score on a scale Standard Deviation 9.48	3.0 score on a scale Standard Deviation 6.86	-7.4 score on a scale Standard Deviation 3.68

SECONDARY outcome

Timeframe: Months 3 and 24

Population: Safety Population. The Number Analyzed is the number of participants who completed the assessment with evaluable data the specified visit (whose vision abilities allowed them to participate in the testing).

A standardized procedure was used for the collection of single, non-stereo images of the fundus of both eyes using the same equipment for each participant throughout the study. Evidence of increased inflammation, hemorrhage, retinal detachment, RPE disturbance or atrophy in the fovea, and any changes from baseline visit were documented. Presence or absence of changes from baseline in fundus autofluorescence were also documented.

Outcome measures

Outcome measures
Measure	Phase 2: High Dose n=4 Participants Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose n=3 Participants Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose n=4 Participants Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose n=3 Participants Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence Any Evidence of Retinal Hemorrhage (Month 3)	0 Participants	0 Participants	0 Participants	0 Participants
Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence Any Evidence of Retinal Detachment (Month 3)	0 Participants	0 Participants	0 Participants	0 Participants
Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence Any Evidence of Inflammation (Month 3)	1 Participants	0 Participants	0 Participants	0 Participants
Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence Any Evidence of Atrophy in the Fovea (Month 3)	1 Participants	2 Participants	1 Participants	1 Participants
Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence Any Evidence of Retinal Pigment Epithelium (RPE) Disturbance (Month 3)	2 Participants	2 Participants	1 Participants	1 Participants
Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence Any Findings of Autofluorescence (Month 3)	0 Participants	0 Participants	0 Participants	0 Participants
Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence Any Evidence of Other findings (Month 3)	2 Participants	0 Participants	0 Participants	1 Participants
Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence Any Evidence of Inflammation (Month 24)	0 Participants	0 Participants	0 Participants	0 Participants
Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence Any Evidence of Retinal Hemorrhage (Month 24)	0 Participants	0 Participants	0 Participants	0 Participants
Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence Any Evidence of Retinal Detachment (Month 24)	0 Participants	0 Participants	0 Participants	0 Participants
Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence Any Evidence of Retinal Pigment Epithelium (RPE) Disturbance (Month 24)	1 Participants	3 Participants	2 Participants	1 Participants
Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence Any Evidence of Atrophy in the Fovea (Month 24)	0 Participants	3 Participants	2 Participants	0 Participants
Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence Any Evidence of Other findings (Month 24)	0 Participants	0 Participants	0 Participants	0 Participants
Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence Any Findings of Autofluorescence (Month 24)	0 Participants	0 Participants	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Baseline to Months 3, 12, and 24

Population: Safety Population. The Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and specified postbaseline visit (whose vision abilities allowed them to participate in the testing).

Qualitative assessment of the change in retinal cross-sectional appearance in the study eye from Baseline at Months 3, 12, and 24 as Measured by Spectral Domain-OCT (SD-OCT).

Outcome measures

Outcome measures
Measure	Phase 2: High Dose Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose n=1 Participants Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose n=2 Participants Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Change From Baseline at Months 3, 12, and 24 in the Anatomical Parameters in the Study Eye as Measured by Spectral Domain-OCT (SD-OCT) - Volume Total Retinal Volume (mm3) (3 months)	—	—	—	0.945 mm3 Standard Deviation 1.1809
Change From Baseline at Months 3, 12, and 24 in the Anatomical Parameters in the Study Eye as Measured by Spectral Domain-OCT (SD-OCT) - Volume Total Ganglion Cell and Inner Plexiform Layer Volume (mm3) (3 months)	—	—	0.210 mm3 Standard Deviation NA Not calculable for n=1	0.070 mm3 Standard Deviation 0.0990
Change From Baseline at Months 3, 12, and 24 in the Anatomical Parameters in the Study Eye as Measured by Spectral Domain-OCT (SD-OCT) - Volume Total Retinal Nerve Fibre Layer Volume (mm3) (3 months)	—	—	0.350 mm3 Standard Deviation NA Not calculable for n=1	0.060 mm3 Standard Deviation 0.1131
Change From Baseline at Months 3, 12, and 24 in the Anatomical Parameters in the Study Eye as Measured by Spectral Domain-OCT (SD-OCT) - Volume Total Retinal Nerve Fibre Layer Volume (mm3) (12 months)	—	—	-0.040 mm3 Standard Deviation NA Not calculable for n=1	0.080 mm3 Standard Deviation 0.0212
Change From Baseline at Months 3, 12, and 24 in the Anatomical Parameters in the Study Eye as Measured by Spectral Domain-OCT (SD-OCT) - Volume Total Ganglion Cell and Inner Plexiform Layer Volume (mm3) (12 months)	—	—	0.050 mm3 Standard Deviation NA Not calculable for n=1	0.005 mm3 Standard Deviation 0.2051
Change From Baseline at Months 3, 12, and 24 in the Anatomical Parameters in the Study Eye as Measured by Spectral Domain-OCT (SD-OCT) - Volume Total Retinal Volume (mm3) (12 months)	—	—	—	0.085 mm3 Standard Deviation 0.0919
Change From Baseline at Months 3, 12, and 24 in the Anatomical Parameters in the Study Eye as Measured by Spectral Domain-OCT (SD-OCT) - Volume Total Retinal Nerve Fibre Layer Volume (mm3) (24 months)	—	—	-0.060 mm3 Standard Deviation NA Not calculable for n=1	0.060 mm3 Standard Deviation 0.1131
Change From Baseline at Months 3, 12, and 24 in the Anatomical Parameters in the Study Eye as Measured by Spectral Domain-OCT (SD-OCT) - Volume Total Ganglion Cell and Inner Plexiform Layer Volume (mm3) (24 months)	—	—	0.210 mm3 Standard Deviation NA Not calculable for n=1	-0.065 mm3 Standard Deviation 0.1202
Change From Baseline at Months 3, 12, and 24 in the Anatomical Parameters in the Study Eye as Measured by Spectral Domain-OCT (SD-OCT) - Volume Total Retinal Volume (mm3) (24 months)	—	—	—	0.135 mm3 Standard Deviation 0.6152

SECONDARY outcome

Timeframe: Baseline to Months 3, 12, and 24

Population: Safety Population. The Overall Number Analyzed is the number of participants who completed the assessment with evaluable data at both baseline and the specified postbaseline visit (whose vision abilities allowed them to participate in the testing).

Qualitative assessment of the change in retinal cross-sectional appearance in the study eye from Baseline at Months 3, 12, and 24 as Measured by Spectral Domain-OCT (SD-OCT).

Outcome measures

Outcome measures
Measure	Phase 2: High Dose Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Low Dose Three participants were enrolled and received the lowest dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose n=1 Participants Three participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose n=2 Participants Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Change From Baseline at Months 3, 12, and 24 in the Anatomical Parameters in the Study Eye as Measured by Spectral Domain-OCT (SD-OCT) - Total Retinal Thickness Total Retinal Thickness (um) (3 months)	—	—	29.0 um Standard Deviation NA Not calculable for n=1	63.0 um Standard Deviation 83.44
Change From Baseline at Months 3, 12, and 24 in the Anatomical Parameters in the Study Eye as Measured by Spectral Domain-OCT (SD-OCT) - Total Retinal Thickness Total Retinal Thickness (um) (12 months)	—	—	12.0 um Standard Deviation NA Not calculable for n=1	9.5 um Standard Deviation 12.02
Change From Baseline at Months 3, 12, and 24 in the Anatomical Parameters in the Study Eye as Measured by Spectral Domain-OCT (SD-OCT) - Total Retinal Thickness Total Retinal Thickness (um) (24 months)	—	—	17.0 um Standard Deviation NA Not calculable for n=1	-1.0 um Standard Deviation 8.49

Adverse Events

Phase 1: Low Dose

Serious events: 1 serious events

Other events: 2 other events

Deaths: 0 deaths

Phase 1: Mid Dose

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Phase 1: High Dose

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Phase 2: High Dose

Serious events: 1 serious events

Other events: 3 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	Phase 1: Low Dose n=3 participants at risk Three participants were enrolled and received a low dose of AGN-151597 (Low). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: Mid Dose n=4 participants at risk Four participants were enrolled and received a higher dose of AGN-151597 (Mid). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 1: High Dose n=3 participants at risk Three participants were enrolled and received the highest dose of AGN-151597 (High). After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.	Phase 2: High Dose n=4 participants at risk Four participants were enrolled in Phase 2a and received the highest dose of AGN-151597 (High) from Phase 1. After completion of the 2-year core study visits, participants could have enrolled in a long-term follow-up for an additional 3 years to monitor the long term safety of AGN-15197.
Cardiac disorders ARRHYTHMIA	0.00% 0/3 • All-cause mortality and adverse event tables include events reported from time of informed consent to the end of the study. The median time participants were followed was 885.0 days for Phase 1 (low dose); 1991.5 days for Phase 1 (mid dose); 1814.0 days for Phase 1 (high dose); and 800.5 days for Phase 2 (high dose).	0.00% 0/4 • All-cause mortality and adverse event tables include events reported from time of informed consent to the end of the study. The median time participants were followed was 885.0 days for Phase 1 (low dose); 1991.5 days for Phase 1 (mid dose); 1814.0 days for Phase 1 (high dose); and 800.5 days for Phase 2 (high dose).	0.00% 0/3 • All-cause mortality and adverse event tables include events reported from time of informed consent to the end of the study. The median time participants were followed was 885.0 days for Phase 1 (low dose); 1991.5 days for Phase 1 (mid dose); 1814.0 days for Phase 1 (high dose); and 800.5 days for Phase 2 (high dose).	25.0% 1/4 • Number of events 1 • All-cause mortality and adverse event tables include events reported from time of informed consent to the end of the study. The median time participants were followed was 885.0 days for Phase 1 (low dose); 1991.5 days for Phase 1 (mid dose); 1814.0 days for Phase 1 (high dose); and 800.5 days for Phase 2 (high dose).
Gastrointestinal disorders INTESTINAL OBSTRUCTION	33.3% 1/3 • Number of events 1 • All-cause mortality and adverse event tables include events reported from time of informed consent to the end of the study. The median time participants were followed was 885.0 days for Phase 1 (low dose); 1991.5 days for Phase 1 (mid dose); 1814.0 days for Phase 1 (high dose); and 800.5 days for Phase 2 (high dose).	0.00% 0/4 • All-cause mortality and adverse event tables include events reported from time of informed consent to the end of the study. The median time participants were followed was 885.0 days for Phase 1 (low dose); 1991.5 days for Phase 1 (mid dose); 1814.0 days for Phase 1 (high dose); and 800.5 days for Phase 2 (high dose).	0.00% 0/3 • All-cause mortality and adverse event tables include events reported from time of informed consent to the end of the study. The median time participants were followed was 885.0 days for Phase 1 (low dose); 1991.5 days for Phase 1 (mid dose); 1814.0 days for Phase 1 (high dose); and 800.5 days for Phase 2 (high dose).	0.00% 0/4 • All-cause mortality and adverse event tables include events reported from time of informed consent to the end of the study. The median time participants were followed was 885.0 days for Phase 1 (low dose); 1991.5 days for Phase 1 (mid dose); 1814.0 days for Phase 1 (high dose); and 800.5 days for Phase 2 (high dose).
Injury, poisoning and procedural complications POST PROCEDURAL HAEMORRHAGE	33.3% 1/3 • Number of events 1 • All-cause mortality and adverse event tables include events reported from time of informed consent to the end of the study. The median time participants were followed was 885.0 days for Phase 1 (low dose); 1991.5 days for Phase 1 (mid dose); 1814.0 days for Phase 1 (high dose); and 800.5 days for Phase 2 (high dose).	0.00% 0/4 • All-cause mortality and adverse event tables include events reported from time of informed consent to the end of the study. The median time participants were followed was 885.0 days for Phase 1 (low dose); 1991.5 days for Phase 1 (mid dose); 1814.0 days for Phase 1 (high dose); and 800.5 days for Phase 2 (high dose).	0.00% 0/3 • All-cause mortality and adverse event tables include events reported from time of informed consent to the end of the study. The median time participants were followed was 885.0 days for Phase 1 (low dose); 1991.5 days for Phase 1 (mid dose); 1814.0 days for Phase 1 (high dose); and 800.5 days for Phase 2 (high dose).	0.00% 0/4 • All-cause mortality and adverse event tables include events reported from time of informed consent to the end of the study. The median time participants were followed was 885.0 days for Phase 1 (low dose); 1991.5 days for Phase 1 (mid dose); 1814.0 days for Phase 1 (high dose); and 800.5 days for Phase 2 (high dose).
Musculoskeletal and connective tissue disorders MUSCULOSKELETAL CHEST PAIN	33.3% 1/3 • Number of events 1 • All-cause mortality and adverse event tables include events reported from time of informed consent to the end of the study. The median time participants were followed was 885.0 days for Phase 1 (low dose); 1991.5 days for Phase 1 (mid dose); 1814.0 days for Phase 1 (high dose); and 800.5 days for Phase 2 (high dose).	0.00% 0/4 • All-cause mortality and adverse event tables include events reported from time of informed consent to the end of the study. The median time participants were followed was 885.0 days for Phase 1 (low dose); 1991.5 days for Phase 1 (mid dose); 1814.0 days for Phase 1 (high dose); and 800.5 days for Phase 2 (high dose).	0.00% 0/3 • All-cause mortality and adverse event tables include events reported from time of informed consent to the end of the study. The median time participants were followed was 885.0 days for Phase 1 (low dose); 1991.5 days for Phase 1 (mid dose); 1814.0 days for Phase 1 (high dose); and 800.5 days for Phase 2 (high dose).	0.00% 0/4 • All-cause mortality and adverse event tables include events reported from time of informed consent to the end of the study. The median time participants were followed was 885.0 days for Phase 1 (low dose); 1991.5 days for Phase 1 (mid dose); 1814.0 days for Phase 1 (high dose); and 800.5 days for Phase 2 (high dose).
Nervous system disorders CEREBROSPINAL FLUID LEAKAGE	33.3% 1/3 • Number of events 1 • All-cause mortality and adverse event tables include events reported from time of informed consent to the end of the study. The median time participants were followed was 885.0 days for Phase 1 (low dose); 1991.5 days for Phase 1 (mid dose); 1814.0 days for Phase 1 (high dose); and 800.5 days for Phase 2 (high dose).	0.00% 0/4 • All-cause mortality and adverse event tables include events reported from time of informed consent to the end of the study. The median time participants were followed was 885.0 days for Phase 1 (low dose); 1991.5 days for Phase 1 (mid dose); 1814.0 days for Phase 1 (high dose); and 800.5 days for Phase 2 (high dose).	0.00% 0/3 • All-cause mortality and adverse event tables include events reported from time of informed consent to the end of the study. The median time participants were followed was 885.0 days for Phase 1 (low dose); 1991.5 days for Phase 1 (mid dose); 1814.0 days for Phase 1 (high dose); and 800.5 days for Phase 2 (high dose).	0.00% 0/4 • All-cause mortality and adverse event tables include events reported from time of informed consent to the end of the study. The median time participants were followed was 885.0 days for Phase 1 (low dose); 1991.5 days for Phase 1 (mid dose); 1814.0 days for Phase 1 (high dose); and 800.5 days for Phase 2 (high dose).

Other adverse events

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