Trial Outcomes & Findings for A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for the Treatment of Fuchs' Corneal Endothelial Dystrophy (FCED) (NCT NCT02653391)

NCT ID: NCT02653391

Last Updated: 2021-09-17

Results Overview

The incidence and severity of ocular treatment emergent adverse events (TEAEs)

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

22 participants

Primary outcome timeframe

Screening Visit, Baseline (Day 1), Week 1, Week 4, Week 8, Week 12, and Week 16

Results posted on

2021-09-17

Participant Flow

Part A 18 participants were randomized. The treatment eye was chosen at random and the fellow eye was the control/placebo eye. Part B The participants received either treatment in both eyes or placebo in both eyes.

Unit of analysis: eyes

Participant milestones

Participant milestones
Measure	Elamipretide 1.0% Ophthalmic Solution and Placebo Part A (Cohort 1) Part A: Each subject will receive one drop of elamipretide 1.0% ophthalmic solution twice daily (BID) in the randomly selected study eye, and Placebo twice daily (BID) in the paired eye.	Elamipretide 3.0% Ophthalmic Solution Part B (Cohort 2) Part B Each subject will receive one drop of elamipretide 3.0% ophthalmic solution twice daily (BID) in both the right and left study eyes (Cohort 2). Part B Elamipretide 3.0% Ophthalmic Solution	Placebo Part B (Cohort 2) Part B Each subject will receive one drop of placebo comparator solution twice daily (BID) in both the right and left study eyes (Cohort 2). Placebo Part B
Overall Study STARTED	18 36	3 6	1 2
Overall Study COMPLETED	16 32	1 2	1 2
Overall Study NOT COMPLETED	2 4	2 4	0 0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for the Treatment of Fuchs' Corneal Endothelial Dystrophy (FCED)

Baseline characteristics by cohort

PRIMARY outcome

Timeframe: Screening Visit, Baseline (Day 1), Week 1, Week 4, Week 8, Week 12, and Week 16

Population: All participants for whom ocular TEAEs were measured.

The incidence and severity of ocular treatment emergent adverse events (TEAEs)

Outcome measures

Outcome measures
Measure	Part A Placebo n=18 Participants Part A participants includes those receiving vehicle in eye paired with treatment eye.	Part A Elamipretide 1.0% Ophthalmic Solution n=18 Participants Part A participants includes those receiving 1% Elamipretide	Part B Placebo n=1 Participants Part B participants includes those receiving vehicle in both eyes.	Part B Elamipretide 3.0% Ophthalmic Solution n=3 Participants Part B Participants includes those receiving 3% Elamipretide
Incidence and Severity of Ocular TEAEs. Ocular moderate TEAEs	0 Participants	0 Participants	0 Participants	2 Participants
Incidence and Severity of Ocular TEAEs. Ocular severe TEAEs	0 Participants	0 Participants	0 Participants	0 Participants
Incidence and Severity of Ocular TEAEs. Ocular mild TEAEs	3 Participants	6 Participants	1 Participants	1 Participants

PRIMARY outcome

Timeframe: Screening Visit, Baseline (Day 1), Week 1, Week 4, Week 8, Week 12, and Week 16

Population: All participants for whom systemic TEAEs were measured. For Part A systemic events were not necessarily attributed to either intervention or placebo.

The incidence and severity of systemic treatment emergent adverse events (TEAEs)

Outcome measures

Outcome measures
Measure	Part A Placebo n=18 Participants Part A participants includes those receiving vehicle in eye paired with treatment eye.	Part A Elamipretide 1.0% Ophthalmic Solution n=1 Participants Part A participants includes those receiving 1% Elamipretide	Part B Placebo n=3 Participants Part B participants includes those receiving vehicle in both eyes.	Part B Elamipretide 3.0% Ophthalmic Solution Part B Participants includes those receiving 3% Elamipretide
The Incidence and Severity of Systemic Adverse Events Systemic mild TEAEs	3 Participants	1 Participants	2 Participants	—
The Incidence and Severity of Systemic Adverse Events Systemic moderate TEAEs	2 Participants	0 Participants	0 Participants	—
The Incidence and Severity of Systemic Adverse Events Systemic severe TEAEs	2 Participants	0 Participants	0 Participants	—

PRIMARY outcome

Timeframe: Baseline, Week 1, Week 4, Week 8, Week 12, and Week 16

Population: All participants for whom slit lamp outcomes were measured.

Number of participants who had a change from baseline from normal or abnormal not clinically significant, to abnormal clinically significant (CS) findings for slit lamp examinations (SLE) for Part A. Part B is reported as separate outcome since unit of measure is number of eyes.

Outcome measures

Outcome measures
Measure	Part A Placebo n=18 Participants Part A participants includes those receiving vehicle in eye paired with treatment eye.	Part A Elamipretide 1.0% Ophthalmic Solution n=18 Participants Part A participants includes those receiving 1% Elamipretide	Part B Placebo Part B participants includes those receiving vehicle in both eyes.	Part B Elamipretide 3.0% Ophthalmic Solution Part B Participants includes those receiving 3% Elamipretide
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Lids Week 12	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Lids Week 16	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Conjunctiva Week 1	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Conjunctiva Week 4	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Conjunctiva Week 8	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Cornea Week 1	1 participants	1 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Cornea Week 4	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Cornea Week 8	1 participants	1 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Cornea Week 12	1 participants	1 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Cornea Week 16	1 participants	1 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Anterior Chamber Week 1	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Anterior Chamber Week 4	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Anterior Chamber Week 8	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Anterior Chamber Week 12	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Anterior Chamber Week 16	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Posterior Chamber Week 1	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Posterior Chamber Week 4	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Posterior Chamber Week 8	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Posterior Chamber Week 12	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Posterior Chamber Week 16	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Iris Week 1	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Iris Week 4	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Iris Week 8	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Iris Week 12	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Iris Week 16	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Lens Week 1	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Lens Week 4	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Lens Week 8	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Lens Week 12	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Lens Week 16	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Lids Week 1	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Lids Week 4	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Lids Week 8	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Conjunctiva Week 12	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A Conjunctiva Week 16	0 participants	0 participants	—	—

PRIMARY outcome

Timeframe: Baseline, Week 1, Week 4, Week 8, Week 12, and Week 16

Population: All participants for whom slit lamp outcomes were measured with both baseline and post-baseline measurements.

Number of eyes with a change from baseline from normal or abnormal not clinically significant, to abnormal clinically significant (CS) findings for slit lamp examinations (SLE) for Part B. Part A is reported as separate outcome.

Outcome measures

Outcome measures
Measure	Part A Placebo n=2 eyes Part A participants includes those receiving vehicle in eye paired with treatment eye.	Part A Elamipretide 1.0% Ophthalmic Solution n=6 eyes Part A participants includes those receiving 1% Elamipretide	Part B Placebo Part B participants includes those receiving vehicle in both eyes.	Part B Elamipretide 3.0% Ophthalmic Solution Part B Participants includes those receiving 3% Elamipretide
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Lids Week 1	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Lids Week 4	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Lids Week 8	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Lids Week 12	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Lids Week 16	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Conjunctiva Week 1	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Conjunctiva Week 4	0 participants	2 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Conjunctiva Week 8	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Conjunctiva Week 12	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Conjunctiva Week 16	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Cornea Week 1	0 participants	6 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Cornea Week 4	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Cornea Week 8	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Cornea Week 12	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Cornea Week 16	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Anterior Chamber Week 1	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Anterior Chamber Week 4	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Anterior Chamber Week 8	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Anterior Chamber Week 12	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Anterior Chamber Week 16	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Posterior Chamber Week 1	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Posterior Chamber Week 4	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Posterior Chamber Week 8	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Posterior Chamber Week 12	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Posterior Chamber Week 16	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Iris Week 1	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Iris Week 4	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Iris Week 8	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Iris Week 12	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Iris Week 16	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Lens Week 1	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Lens Week 4	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Lens Week 8	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Lens Week 12	0 participants	0 participants	—	—
Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part B Lens Week 16	0 participants	0 participants	—	—

PRIMARY outcome

Timeframe: Baseline, Week 1, Week 4, Week 8, Week 12

Population: All participants for whom intraocular pressure was measured. Part B is reported as a separate Outcome Measure.

Change from Baseline in intraocular pressure (IOP) using Goldmann applanation tonometry for Part A

Outcome measures

Outcome measures
Measure	Part A Placebo n=18 Participants Part A participants includes those receiving vehicle in eye paired with treatment eye.	Part A Elamipretide 1.0% Ophthalmic Solution n=18 Participants Part A participants includes those receiving 1% Elamipretide	Part B Placebo Part B participants includes those receiving vehicle in both eyes.	Part B Elamipretide 3.0% Ophthalmic Solution Part B Participants includes those receiving 3% Elamipretide
Change From Baseline in Intraocular Pressure (IOP) for Part A Week 1	-0.6 mmHg Standard Error 0.64	-0.4 mmHg Standard Error 0.49	—	—
Change From Baseline in Intraocular Pressure (IOP) for Part A Week 4	-0.3 mmHg Standard Error 0.47	-0.2 mmHg Standard Error 0.31	—	—
Change From Baseline in Intraocular Pressure (IOP) for Part A Week 8	-1.1 mmHg Standard Error 0.47	-0.2 mmHg Standard Error 0.64	—	—
Change From Baseline in Intraocular Pressure (IOP) for Part A Week 12	-0.4 mmHg Standard Error 0.45	-0.4 mmHg Standard Error 0.43	—	—
Change From Baseline in Intraocular Pressure (IOP) for Part A Over All 12 Weeks	-0.6 mmHg Standard Error 0.22	-0.3 mmHg Standard Error 0.21	—	—

PRIMARY outcome

Timeframe: Baseline, Week 1, Week 4, Week 8, Week 12 , and Week 16 or early discontinuation visit

Population: All participants for whom intraocular pressures were measured.

Change from Baseline in intraocular pressure (IOP) using Goldmann applanation tonometry for Part B. Part A is reported separately.

Outcome measures

Outcome measures
Measure	Part A Placebo n=2 Eyes Part A participants includes those receiving vehicle in eye paired with treatment eye.	Part A Elamipretide 1.0% Ophthalmic Solution n=6 Eyes Part A participants includes those receiving 1% Elamipretide	Part B Placebo Part B participants includes those receiving vehicle in both eyes.	Part B Elamipretide 3.0% Ophthalmic Solution Part B Participants includes those receiving 3% Elamipretide
Change From Baseline in Intraocular Pressure (IOP) for Part B Week 1	3.0 mmHg Standard Deviation 0.00	0.2 mmHg Standard Deviation 3.19	—	—
Change From Baseline in Intraocular Pressure (IOP) for Part B Week 4	2.0 mmHg Standard Deviation 0.00	-0.8 mmHg Standard Deviation 1.33	—	—
Change From Baseline in Intraocular Pressure (IOP) for Part B Week 8	1.5 mmHg Standard Deviation 2.12	0.8 mmHg Standard Deviation 1.89	—	—
Change From Baseline in Intraocular Pressure (IOP) for Part B Week 12	0.0 mmHg Standard Deviation 0.00	3.5 mmHg Standard Deviation 2.12	—	—
Change From Baseline in Intraocular Pressure (IOP) for Part B Week 16	3.5 mmHg Standard Deviation 2.12	1.0 mmHg Standard Deviation 1.41	—	—

SECONDARY outcome

Timeframe: Baseline, Week 1, Week 4, Week 8, Week 12, and Week 16

Population: All participants for whom central corneal thickness was measured. Part B is reported as a separate Outcome Measure.

Change from Baseline in Central Corneal Thickness by Visit as measured by Pachymetry for Part A. Part B is reported as a separate outcome measure.

Outcome measures

Outcome measures
Measure	Part A Placebo n=18 Participants Part A participants includes those receiving vehicle in eye paired with treatment eye.	Part A Elamipretide 1.0% Ophthalmic Solution n=18 Participants Part A participants includes those receiving 1% Elamipretide	Part B Placebo n=18 Participants Part B participants includes those receiving vehicle in both eyes.	Part B Elamipretide 3.0% Ophthalmic Solution n=18 Participants Part B Participants includes those receiving 3% Elamipretide
Change From Baseline in Central Corneal Thickness by Visit Part A as Measured by Pachymetry for Part A Week 1	15.4 µm Standard Deviation 47.95	7.2 µm Standard Deviation 19.99	1.8 µm Standard Deviation 19.72	2.0 µm Standard Deviation 25.51
Change From Baseline in Central Corneal Thickness by Visit Part A as Measured by Pachymetry for Part A Week 4	9.4 µm Standard Deviation 19.61	6.1 µm Standard Deviation 14.32	4.7 µm Standard Deviation 21.20	4.8 µm Standard Deviation 26.52
Change From Baseline in Central Corneal Thickness by Visit Part A as Measured by Pachymetry for Part A Week 8	15.4 µm Standard Deviation 16.04	13.1 µm Standard Deviation 22.35	6.1 µm Standard Deviation 15.23	7.6 µm Standard Deviation 9.61
Change From Baseline in Central Corneal Thickness by Visit Part A as Measured by Pachymetry for Part A Week 12	12.3 µm Standard Deviation 30.31	2.1 µm Standard Deviation 31.80	-1.8 µm Standard Deviation 28.25	0.0 µm Standard Deviation 25.87
Change From Baseline in Central Corneal Thickness by Visit Part A as Measured by Pachymetry for Part A Week 16	13.7 µm Standard Deviation 19.50	8.4 µm Standard Deviation 17.09	6.3 µm Standard Deviation 21.18	8.7 µm Standard Deviation 27.75

SECONDARY outcome

Timeframe: Baseline (Day 1), Week 1, Week 4, Week 8, Week 12, and Week 16, or Early discontinuation visit

Population: All participants for whom Central Corneal Thickness was measured. Part A is reported as a separate Outcome Measure.

Central Corneal Thickness: by-subject data as measured by Pachymetry and Pentacam. Part A is reported as a separate outcome measure.

Outcome measures

Outcome measures
Measure	Part A Placebo n=4 Participants Part A participants includes those receiving vehicle in eye paired with treatment eye.	Part A Elamipretide 1.0% Ophthalmic Solution n=4 Participants Part A participants includes those receiving 1% Elamipretide	Part B Placebo n=4 Participants Part B participants includes those receiving vehicle in both eyes.	Part B Elamipretide 3.0% Ophthalmic Solution n=4 Participants Part B Participants includes those receiving 3% Elamipretide
Central Corneal Thickness Part B Participant 205 (Elamipretide) Week 4	650 µm	687.6 µm	656.3 µm	656.3 µm
Central Corneal Thickness Part B Participant 205 (Elamipretide) Early Discontinuation	663 µm	660.6 µm	645 µm	642 µm
Central Corneal Thickness Part B Participant 201 (Elamipretide) Baseline (Day 1)	626 µm	593 µm	604 µm	591.3 µm
Central Corneal Thickness Part B Participant 201 (Elamipretide) Week 1	619 µm	613 µm	633 µm	602.3 µm
Central Corneal Thickness Part B Participant 201(Elamipretide) Week 4	623 µm	599 µm	646 µm	600.3 µm
Central Corneal Thickness Part B Participant 201 (Elamipretide) Week 8	608 µm	624.3 µm	607 µm	608.6 µm
Central Corneal Thickness Part B Participant 201 (Elamipretide) Week 12	617 µm	608.6 µm	623 µm	593.6 µm
Central Corneal Thickness Part B Participant 201 (Elamipretide) Week 16	604 µm	598.6 µm	620 µm	600.6 µm
Central Corneal Thickness Part B Participant 203 (Elamipretide) Baseline (Day 1)	620 µm	627 µm	635 µm	622.6 µm
Central Corneal Thickness Part B Participant 203 (Elamipretide) Week 1	607 µm	618.3 µm	623 µm	617.3 µm
Central Corneal Thickness Part B Participant 203 (Elamipretide) Early discontinuation visit	639 µm	621 µm	630 µm	622 µm
Central Corneal Thickness Part B Participant 204 (Vehicle) Baseline (Day 1)	656 µm	618.3 µm	624 µm	604.6 µm
Central Corneal Thickness Part B Participant 204 (Vehicle) Week 1	629 µm	616.6 µm	623 µm	608.3 µm
Central Corneal Thickness Part B Participant 204 (Vehicle) Week 4	645 µm	631 µm	619 µm	626 µm
Central Corneal Thickness Part B Participant 204 (Vehicle) Week 8	640 µm	646.3 µm	631 µm	645 µm
Central Corneal Thickness Part B Participant 204 (Vehicle) Week 12	634 µm	645.3 µm	621 µm	637.6 µm
Central Corneal Thickness Part B Participant 204 (Vehicle) Week 16	646 µm	638.3 µm	627 µm	630 µm
Central Corneal Thickness Part B Participant 205 (Elamipretide) Baseline (Day 1)	655 µm	652.6 µm	632 µm	632 µm
Central Corneal Thickness Part B Participant 205 (Elamipretide) Week 1	664 µm	655.6 µm	643 µm	626.3 µm

SECONDARY outcome

Timeframe: Baseline, Week 1, 4, 8, and 12

Population: All participants had images taken; data was analyzed for those whose images were of sufficient quality.

Change From Baseline in Endothelial Cell Hexagonality in Percentage Over All 12 Weeks for Part A. Specular microscopy is a noninvasive photographic technique that allows visualization and analyzation the corneal endothelium. Using computer-assisted morphometry, specular microscopes analyzes the size, shape and population of the endothelial cells. Histologically, healthy corneal cells initially have a hexagonal shape. As endothelial cells die, neighboring cells enlarge to cover the empty space once occupied by the cell. This, in turn, causes the remaining cells to lose their hexagonal shape. Assessments were performed using the flex center and full auto methods for Part A and data from the flex center method was summarized. The flex center method was used for Part B. The percent of Part B is entered as a separate outcome measure. A decrease from baseline in % cell hexagonality means worse outcome, a increase from baseline means better outcome.

Outcome measures

Outcome measures
Measure	Part A Placebo n=18 Participants Part A participants includes those receiving vehicle in eye paired with treatment eye.	Part A Elamipretide 1.0% Ophthalmic Solution n=18 Participants Part A participants includes those receiving 1% Elamipretide	Part B Placebo Part B participants includes those receiving vehicle in both eyes.	Part B Elamipretide 3.0% Ophthalmic Solution Part B Participants includes those receiving 3% Elamipretide
Change From Baseline Endothelial Cell Hexagonality in Percentage Over All 12 Weeks for Part A Nasal Cornea	-6.812 percentage of hexagonality Standard Deviation 4.3912	-2.952 percentage of hexagonality Standard Deviation 4.0223	—	—
Change From Baseline Endothelial Cell Hexagonality in Percentage Over All 12 Weeks for Part A Central Cornea	1.518 percentage of hexagonality Standard Deviation 7.1703	-3.155 percentage of hexagonality Standard Deviation 7.2515	—	—
Change From Baseline Endothelial Cell Hexagonality in Percentage Over All 12 Weeks for Part A Temporal Cornea	0.738 percentage of hexagonality Standard Deviation 5.0322	-0.764 percentage of hexagonality Standard Deviation 5.1291	—	—

SECONDARY outcome

Timeframe: Baseline, Week 1, Week 4, Week 8, Week 12, and Week 16 or early discontinuation visit

Population: All participants for whom Corneal Endothelial Cell Hexagonality was measured. Part A is reported as a separate Outcome Measure. For images with poor quality or too few cells to register, there was no data available.

Corneal Endothelial Cell Hexagonality in Percentage by-subject data: Part B. Data was only listed in weeks where images were good enough quality to quantify. Specular microscopy is a noninvasive photographic technique that allows visualization and analyzation the corneal endothelium. Using computer-assisted morphometry, specular microscopes analyzes the size, shape and population of the endothelial cells. Histologically, healthy corneal cells initially have a hexagonal shape. As endothelial cells die, neighboring cells enlarge to cover the empty space once occupied by the cell. This, in turn, causes the remaining cells to lose their hexagonal shape. The flex center method was used for Part B. A decrease in percent of cell hexagonality from baseline means worse outcome, an increase from baseline means better outcome.

Outcome measures

Outcome measures
Measure	Part A Placebo n=4 eyes Part A participants includes those receiving vehicle in eye paired with treatment eye.	Part A Elamipretide 1.0% Ophthalmic Solution n=4 eyes Part A participants includes those receiving 1% Elamipretide	Part B Placebo Part B participants includes those receiving vehicle in both eyes.	Part B Elamipretide 3.0% Ophthalmic Solution Part B Participants includes those receiving 3% Elamipretide
Corneal Endothelial Cell Hexagonality Part B Participant 204 (Vehicle) Temporal Week 16	NA µm "Poor Quality Images" or "too few cells to register"	45.00 µm	—	—
Corneal Endothelial Cell Hexagonality Part B Participant 201 (Elamipretide) Central Week 16	27.75 µm	NA µm "Poor Quality Images" or "too few cells to register"	—	—
Corneal Endothelial Cell Hexagonality Part B Participant 201 (Elamipretide) Temporal Baseline (Day 1)	39.50 µm	NA µm "Poor Quality Images" or "too few cells to register"	—	—
Corneal Endothelial Cell Hexagonality Part B Participant 204 (Vehicle) Central Baseline (Day 1)	NA µm "Poor Quality Images" or "too few cells to register"	42.00 µm	—	—
Corneal Endothelial Cell Hexagonality Part B Participant 204 (Vehicle) Central Week 1	NA µm "Poor Quality Images" or "too few cells to register"	31.67 µm	—	—
Corneal Endothelial Cell Hexagonality Part B Participant 204 (Vehicle) Central Week 4	NA µm "Poor Quality Images" or "too few cells to register"	30.00 µm	—	—
Corneal Endothelial Cell Hexagonality Part B Participant 204 (Vehicle) Central Week 8	NA µm "Poor Quality Images" or "too few cells to register"	46.17 µm	—	—
Corneal Endothelial Cell Hexagonality Part B Participant 204 (Vehicle) Central Week 12	NA µm "Poor Quality Images" or "too few cells to register"	33.00 µm	—	—
Corneal Endothelial Cell Hexagonality Part B Participant 204 (Vehicle) Central Week 16	NA µm "Poor Quality Images" or "too few cells to register"	49.50 µm	—	—
Corneal Endothelial Cell Hexagonality Part B Participant 204 (Vehicle) Nasal Baseline (Day 1)	NA µm "Poor Quality Images" or "too few cells to register"	41.50 µm	—	—
Corneal Endothelial Cell Hexagonality Part B Participant 204 (Vehicle) Nasal Week 1	NA µm "Poor Quality Images" or "too few cells to register"	48.34 µm	—	—
Corneal Endothelial Cell Hexagonality Part B Participant 204 (Vehicle) Nasal Week 4	NA µm "Poor Quality Images" or "too few cells to register"	41.34 µm	—	—
Corneal Endothelial Cell Hexagonality Part B Participant 204 (Vehicle) Nasal Week 8	NA µm "Poor Quality Images" or "too few cells to register"	35.84 µm	—	—
Corneal Endothelial Cell Hexagonality Part B Participant 204 (Vehicle) Nasal Week 12	NA µm "Poor Quality Images" or "too few cells to register"	14.50 µm	—	—
Corneal Endothelial Cell Hexagonality Part B Participant 204 (Vehicle) Nasal Week 16	NA µm "Poor Quality Images" or "too few cells to register"	35.43 µm	—	—
Corneal Endothelial Cell Hexagonality Part B Participant 204 (Vehicle) Temporal Baseline (Day 1)	NA µm "Poor Quality Images" or "too few cells to register"	54.50 µm	—	—
Corneal Endothelial Cell Hexagonality Part B Participant 204 (Vehicle) Temporal Week 1	NA µm "Poor Quality Images" or "too few cells to register"	30.50 µm	—	—
Corneal Endothelial Cell Hexagonality Part B Participant 204 (Vehicle) Temporal Week 8	NA µm "Poor Quality Images" or "too few cells to register"	34.00 µm	—	—
Corneal Endothelial Cell Hexagonality Part B Participant 204 (Vehicle) Temporal Week 12	NA µm "Poor Quality Images" or "too few cells to register"	50.00 µm	—	—

SECONDARY outcome

Timeframe: Baseline, Weeks 1, 4, 8, 12, and 16

Population: All participants for whom BCVA score was measured.

Best corrected visual acuity (BCVA) using the using the Early Treatment Diabetic Retinopathy Study (ETDRS) scale by visit. ETDRS charts present a series of five letters of equal difficulty on each row, with standardized spacing between letters and rows; there is a total of 14 lines (70 letters), with letter size increasing further geometrically and equivalently in every line by a factor of 1.2589 (or 0.1 log unit), moving up the chart. Minimum score of zero, maximum score of 100. Change from baseline: a more negative score is worse outcome, a more positive score is better outcome. A lower score means less letters were read correctly (worse outcome) and a higher score means more letters were read correctly (better outcome). Part B was listed separately.

Outcome measures

Outcome measures
Measure	Part A Placebo n=18 Participants Part A participants includes those receiving vehicle in eye paired with treatment eye.	Part A Elamipretide 1.0% Ophthalmic Solution n=18 Participants Part A participants includes those receiving 1% Elamipretide	Part B Placebo Part B participants includes those receiving vehicle in both eyes.	Part B Elamipretide 3.0% Ophthalmic Solution Part B Participants includes those receiving 3% Elamipretide
Change From Baseline in Best Corrected Visual Acuity (BCVA) Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale for Part A. Week 1	1.8 letters Standard Deviation 3.51	2.3 letters Standard Deviation 4.69	—	—
Change From Baseline in Best Corrected Visual Acuity (BCVA) Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale for Part A. Week 4	0.8 letters Standard Deviation 5.67	-0.1 letters Standard Deviation 8.96	—	—
Change From Baseline in Best Corrected Visual Acuity (BCVA) Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale for Part A. Week 8	2.5 letters Standard Deviation 4.87	1.3 letters Standard Deviation 8.11	—	—
Change From Baseline in Best Corrected Visual Acuity (BCVA) Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale for Part A. Week 12	2.6 letters Standard Deviation 4.59	0.8 letters Standard Deviation 6.72	—	—
Change From Baseline in Best Corrected Visual Acuity (BCVA) Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale for Part A. Week 16	3.8 letters Standard Deviation 5.33	2.6 letters Standard Deviation 5.94	—	—

SECONDARY outcome

Timeframe: Baseline, Weeks 1, 4, 8, 12, and 16

Population: All participants for whom BCVA score was measured.

Outcome measures

Outcome measures
Measure	Part A Placebo n=4 Participants Part A participants includes those receiving vehicle in eye paired with treatment eye.	Part A Elamipretide 1.0% Ophthalmic Solution n=4 Participants Part A participants includes those receiving 1% Elamipretide	Part B Placebo Part B participants includes those receiving vehicle in both eyes.	Part B Elamipretide 3.0% Ophthalmic Solution Part B Participants includes those receiving 3% Elamipretide
Best Corrected Visual Acuity (BCVA) Score Using ETDRS Scale for Part B Participant 204 (Vehicle) Week 12	80 letters	69 letters	—	—
Best Corrected Visual Acuity (BCVA) Score Using ETDRS Scale for Part B Participant 205 (Elamipretide) Week 4	63 letters	36 letters	—	—
Best Corrected Visual Acuity (BCVA) Score Using ETDRS Scale for Part B Participant 205 (Elamipretide) Unscheduled	NA letters Not measured	NA letters Not measured	—	—
Best Corrected Visual Acuity (BCVA) Score Using ETDRS Scale for Part B Participant 201 (Elamipretide) Baseline	66 letters	69 letters	—	—
Best Corrected Visual Acuity (BCVA) Score Using ETDRS Scale for Part B Participant 201 (Elamipretide) Week 1	62 letters	68 letters	—	—
Best Corrected Visual Acuity (BCVA) Score Using ETDRS Scale for Part B Participant 201 (Elamipretide) Week 4	64 letters	62 letters	—	—
Best Corrected Visual Acuity (BCVA) Score Using ETDRS Scale for Part B Participant 201 (Elamipretide) Week 8	67 letters	70 letters	—	—
Best Corrected Visual Acuity (BCVA) Score Using ETDRS Scale for Part B Participant 201 (Elamipretide) Week 12	68 letters	73 letters	—	—
Best Corrected Visual Acuity (BCVA) Score Using ETDRS Scale for Part B Participant 201 (Elamipretide) Week 16	62 letters	63 letters	—	—
Best Corrected Visual Acuity (BCVA) Score Using ETDRS Scale for Part B Participant 203 (Elamipretide) Baseline	63 letters	59 letters	—	—
Best Corrected Visual Acuity (BCVA) Score Using ETDRS Scale for Part B Participant 203 (Elamipretide) Week 1	60 letters	60 letters	—	—
Best Corrected Visual Acuity (BCVA) Score Using ETDRS Scale for Part B Participant 203 (Elamipretide) Unscheduled Visit	0 letters	0 letters	—	—
Best Corrected Visual Acuity (BCVA) Score Using ETDRS Scale for Part B Participant 203 (Elamipretide) Early Discontinuation	60 letters	57 letters	—	—
Best Corrected Visual Acuity (BCVA) Score Using ETDRS Scale for Part B Participant 204 (Vehicle) Baseline	79 letters	71 letters	—	—
Best Corrected Visual Acuity (BCVA) Score Using ETDRS Scale for Part B Participant 204 (Vehicle) Week 1	78 letters	71 letters	—	—
Best Corrected Visual Acuity (BCVA) Score Using ETDRS Scale for Part B Participant 204 (Vehicle) Week 4	79 letters	72 letters	—	—
Best Corrected Visual Acuity (BCVA) Score Using ETDRS Scale for Part B Participant 204 (Vehicle) Week 8	76 letters	70 letters	—	—
Best Corrected Visual Acuity (BCVA) Score Using ETDRS Scale for Part B Participant 204 (Vehicle) Week 16	81 letters	68 letters	—	—
Best Corrected Visual Acuity (BCVA) Score Using ETDRS Scale for Part B Participant 205 (Elamipretide) Baseline	68 letters	31 letters	—	—
Best Corrected Visual Acuity (BCVA) Score Using ETDRS Scale for Part B Participant 205 (Elamipretide) Week 1	69 letters	30 letters	—	—
Best Corrected Visual Acuity (BCVA) Score Using ETDRS Scale for Part B Participant 205 (Elamipretide) Early Discontinuation	70 letters	37 letters	—	—

SECONDARY outcome

Timeframe: Baseline, Weeks 1, 4, 8, and 12

Population: All participants for whom endothelial cell counts were measured. All participants had images taken; data was analyzed for those whose images were of sufficient quality.

Change From Baseline in Endothelial Cell Counts, or density (Counts/mm\^2) over all 12 weeks for Part A. Part B is entered as a separate outcome measure.

Outcome measures

Outcome measures
Measure	Part A Placebo n=18 Participants Part A participants includes those receiving vehicle in eye paired with treatment eye.	Part A Elamipretide 1.0% Ophthalmic Solution n=18 Participants Part A participants includes those receiving 1% Elamipretide	Part B Placebo Part B participants includes those receiving vehicle in both eyes.	Part B Elamipretide 3.0% Ophthalmic Solution Part B Participants includes those receiving 3% Elamipretide
Change From Baseline in Endothelial Cell Density Over All 12 Weeks for Part A Central Cornea	11.0 counts/mm^2 Standard Error 224.89	-166.9 counts/mm^2 Standard Error 231.84	—	—
Change From Baseline in Endothelial Cell Density Over All 12 Weeks for Part A Nasal Cornea	-68.9 counts/mm^2 Standard Error 114.22	-33.0 counts/mm^2 Standard Error 105.18	—	—
Change From Baseline in Endothelial Cell Density Over All 12 Weeks for Part A Temporal Cornea	-38.0 counts/mm^2 Standard Error 162.07	-24.0 counts/mm^2 Standard Error 178.93	—	—

SECONDARY outcome

Timeframe: Baseline, Weeks 1, 4, 8, 12, and 16.

Population: All participants for whom Corneal Endothelial Cell Counts/Density was measured. Part A is reported as a separate Outcome Measure. All images from Participant 205 were "poor quality images" and hence, no data was available.

Corneal Endothelial Cell Density: Part B, By-subject data for all time points where images were readable. For all time points where there were "Poor Quality Images" or "too few cells to register", there were no data available, and these were not listed below.

Outcome measures

Outcome measures
Measure	Part A Placebo n=4 Participants Part A participants includes those receiving vehicle in eye paired with treatment eye.	Part A Elamipretide 1.0% Ophthalmic Solution n=4 Participants Part A participants includes those receiving 1% Elamipretide	Part B Placebo Part B participants includes those receiving vehicle in both eyes.	Part B Elamipretide 3.0% Ophthalmic Solution Part B Participants includes those receiving 3% Elamipretide
Corneal Endothelial Cell Density Part B Participant 204 (Vehicle) Central Week 1	NA counts/mm^2 Poor Quality Images" or "too few cells to register"	1069.2 counts/mm^2	—	—
Corneal Endothelial Cell Density Part B Participant 201 (Elamipretide) Central Week 16	934.0 counts/mm^2	NA counts/mm^2 "Poor Quality Images" or "too few cells to register"	—	—
Corneal Endothelial Cell Density Part B Participant 201 (Elamipretide) Temporal Baseline (Day 1)	1449.0 counts/mm^2	NA counts/mm^2 "Poor Quality Images" or "too few cells to register"	—	—
Corneal Endothelial Cell Density Part B Participant 204 (Vehicle) Central Baseline (Day 1)	NA counts/mm^2 Poor Quality Images" or "too few cells to register"	1241.7 counts/mm^2	—	—
Corneal Endothelial Cell Density Part B Participant 204 (Vehicle) Central Week 4	NA counts/mm^2 Poor Quality Images" or "too few cells to register"	1087.7 counts/mm^2	—	—
Corneal Endothelial Cell Density Part B Participant 204 (Vehicle) Central Week 8	NA counts/mm^2 Poor Quality Images" or "too few cells to register"	1149.5 counts/mm^2	—	—
Corneal Endothelial Cell Density Part B Participant 204 (Vehicle) Central Week 12	NA counts/mm^2 Poor Quality Images" or "too few cells to register"	773.2 counts/mm^2	—	—
Corneal Endothelial Cell Density Part B Participant 204 (Vehicle) Central Week 16	NA counts/mm^2 Poor Quality Images" or "too few cells to register"	1094.5 counts/mm^2	—	—
Corneal Endothelial Cell Density Part B Participant 204 (Vehicle) Nasal Baseline (Day 1)	NA counts/mm^2 Poor Quality Images" or "too few cells to register"	897.3 counts/mm^2	—	—
Corneal Endothelial Cell Density Part B Participant 204 (Vehicle) Nasal Week 1	NA counts/mm^2 Poor Quality Images" or "too few cells to register"	1361.2 counts/mm^2	—	—
Corneal Endothelial Cell Density Part B Participant 204 (Vehicle) Nasal Week 4	NA counts/mm^2 Poor Quality Images" or "too few cells to register"	971.5 counts/mm^2	—	—
Corneal Endothelial Cell Density Part B Participant 204 (Vehicle) Nasal Week 8	NA counts/mm^2 Poor Quality Images" or "too few cells to register"	995.2 counts/mm^2	—	—
Corneal Endothelial Cell Density Part B Participant 204 (Vehicle) Nasal Week 12	NA counts/mm^2 Poor Quality Images" or "too few cells to register"	961.3 counts/mm^2	—	—
Corneal Endothelial Cell Density Part B Participant 204 (Vehicle) Nasal Week 16	NA counts/mm^2 Poor Quality Images" or "too few cells to register"	1046.7 counts/mm^2	—	—
Corneal Endothelial Cell Density Part B Participant 204 (Vehicle) Temporal Baseline (Day 1)	NA counts/mm^2 Poor Quality Images" or "too few cells to register"	826.0 counts/mm^2	—	—
Corneal Endothelial Cell Density Part B Participant 204 (Vehicle) Temporal Week 1	NA counts/mm^2 Poor Quality Images" or "too few cells to register"	1195.2 counts/mm^2	—	—
Corneal Endothelial Cell Density Part B Participant 204 (Vehicle) Temporal Week 8	NA counts/mm^2 Poor Quality Images" or "too few cells to register"	1068.3 counts/mm^2	—	—
Corneal Endothelial Cell Density Part B Participant 204 (Vehicle) Temporal Week 12	NA counts/mm^2 Poor Quality Images" or "too few cells to register"	1213.3 counts/mm^2	—	—
Corneal Endothelial Cell Density Part B Participant 204 (Vehicle) Temporal Week 16	NA counts/mm^2 Poor Quality Images" or "too few cells to register"	1032.5 counts/mm^2	—	—

SECONDARY outcome

Timeframe: Baseline, Week 12

Population: All participants had images taken; data was analyzed for those whose images were of sufficient quality.

Change From Baseline in Endothelial Cell Coefficient of Variation Over All 12 Weeks. Coefficient of variation (CV) is Standard deviation (SD) of cell area divided by the mean cell area of endothelial cell analyzed. CV represents the coefficient, or degree, of variation in the sizes of the endothelial cells (polymegethism). By measuring the variation in size between endothelial cells, the system can measure how much cell loss is occurring. The more variation, the worse the outcome. Part B is is entered as a separate outcome measure.

Outcome measures

Outcome measures
Measure	Part A Placebo n=18 Participants Part A participants includes those receiving vehicle in eye paired with treatment eye.	Part A Elamipretide 1.0% Ophthalmic Solution n=18 Participants Part A participants includes those receiving 1% Elamipretide	Part B Placebo Part B participants includes those receiving vehicle in both eyes.	Part B Elamipretide 3.0% Ophthalmic Solution Part B Participants includes those receiving 3% Elamipretide
Change From Baseline in Endothelial Cell Coefficient of Variation Over All 12 Weeks for Part A Central Cornea	-7.644 SD of cell area/Mean cell area, (μm^2) Standard Deviation 7.7617	-7.941 SD of cell area/Mean cell area, (μm^2) Standard Deviation 8.0334	—	—
Change From Baseline in Endothelial Cell Coefficient of Variation Over All 12 Weeks for Part A Nasal Cornea	-3.809 SD of cell area/Mean cell area, (μm^2) Standard Deviation 3.9095	-4.293 SD of cell area/Mean cell area, (μm^2) Standard Deviation 3.6388	—	—
Change From Baseline in Endothelial Cell Coefficient of Variation Over All 12 Weeks for Part A Temporal Cornea	7.823 SD of cell area/Mean cell area, (μm^2) Standard Deviation 5.0620	0.737 SD of cell area/Mean cell area, (μm^2) Standard Deviation 5.0326	—	—

SECONDARY outcome

Timeframe: Baseline, Weeks 1, 4, 8,12, and 16

Part B, By-subject data for all time points where images were readable. For all time points where there were "Poor Quality Images" or "too few cells to register" there were no data available. CoV represents the coefficient, or degree, of variation in the sizes of the endothelial cells. By measuring the variation in size between endothelial cells, the system can measure how much cell loss is occurring. A CoV less than 40 is normal.

Outcome measures

Outcome measures
Measure	Part A Placebo n=4 Participants Part A participants includes those receiving vehicle in eye paired with treatment eye.	Part A Elamipretide 1.0% Ophthalmic Solution n=4 Participants Part A participants includes those receiving 1% Elamipretide	Part B Placebo Part B participants includes those receiving vehicle in both eyes.	Part B Elamipretide 3.0% Ophthalmic Solution Part B Participants includes those receiving 3% Elamipretide
Coefficient of Variation (CoV) Part B Participant 201 (Elamipretide) Central Week 16	64.0 SD of cell area/Mean cell area, (μm^2)	NA SD of cell area/Mean cell area, (μm^2) "Poor Quality Images" or "too few cells to register"	—	—
Coefficient of Variation (CoV) Part B Participant 201 (Elamipretide) Temporal Baseline (Day 1)	51.0 SD of cell area/Mean cell area, (μm^2)	NA SD of cell area/Mean cell area, (μm^2) "Poor Quality Images" or "too few cells to register"	—	—
Coefficient of Variation (CoV) Part B Participant 204 (Vehicle) Central Baseline (Day 1)	NA SD of cell area/Mean cell area, (μm^2) "Poor Quality Images" or "too few cells to register"	61.0 SD of cell area/Mean cell area, (μm^2)	—	—
Coefficient of Variation (CoV) Part B Participant 204 (Vehicle) Central Week 1	NA SD of cell area/Mean cell area, (μm^2) "Poor Quality Images" or "too few cells to register"	69.0 SD of cell area/Mean cell area, (μm^2)	—	—
Coefficient of Variation (CoV) Part B Participant 204 (Vehicle) Central Week 4	NA SD of cell area/Mean cell area, (μm^2) "Poor Quality Images" or "too few cells to register"	47.7 SD of cell area/Mean cell area, (μm^2)	—	—
Coefficient of Variation (CoV) Part B Participant 204 (Vehicle) Central Week 8	NA SD of cell area/Mean cell area, (μm^2) "Poor Quality Images" or "too few cells to register"	58.7 SD of cell area/Mean cell area, (μm^2)	—	—
Coefficient of Variation (CoV) Part B Participant 204 (Vehicle) Central Week 12	NA SD of cell area/Mean cell area, (μm^2) "Poor Quality Images" or "too few cells to register"	60.2 SD of cell area/Mean cell area, (μm^2)	—	—
Coefficient of Variation (CoV) Part B Participant 204 (Vehicle) Central Week 16	NA SD of cell area/Mean cell area, (μm^2) "Poor Quality Images" or "too few cells to register"	56.8 SD of cell area/Mean cell area, (μm^2)	—	—
Coefficient of Variation (CoV) Part B Participant 204 (Vehicle) Nasal Baseline (Day 1)	NA SD of cell area/Mean cell area, (μm^2) "Poor Quality Images" or "too few cells to register"	73.0 SD of cell area/Mean cell area, (μm^2)	—	—
Coefficient of Variation (CoV) Part B Participant 204 (Vehicle) Nasal Week 1	NA SD of cell area/Mean cell area, (μm^2) "Poor Quality Images" or "too few cells to register"	58.7 SD of cell area/Mean cell area, (μm^2)	—	—
Coefficient of Variation (CoV) Part B Participant 204 (Vehicle) Nasal Week 4	NA SD of cell area/Mean cell area, (μm^2) "Poor Quality Images" or "too few cells to register"	59.5 SD of cell area/Mean cell area, (μm^2)	—	—
Coefficient of Variation (CoV) Part B Participant 204 (Vehicle) Nasal Week 8	NA SD of cell area/Mean cell area, (μm^2) "Poor Quality Images" or "too few cells to register"	51.5 SD of cell area/Mean cell area, (μm^2)	—	—
Coefficient of Variation (CoV) Part B Participant 204 (Vehicle) Nasal Week 12	NA SD of cell area/Mean cell area, (μm^2) "Poor Quality Images" or "too few cells to register"	65.0 SD of cell area/Mean cell area, (μm^2)	—	—
Coefficient of Variation (CoV) Part B Participant 204 (Vehicle) Nasal Week 16	NA SD of cell area/Mean cell area, (μm^2) "Poor Quality Images" or "too few cells to register"	50.0 SD of cell area/Mean cell area, (μm^2)	—	—
Coefficient of Variation (CoV) Part B Participant 204 (Vehicle) Temporal Baseline (Day 1)	NA SD of cell area/Mean cell area, (μm^2) "Poor Quality Images" or "too few cells to register"	62.3 SD of cell area/Mean cell area, (μm^2)	—	—
Coefficient of Variation (CoV) Part B Participant 204 (Vehicle) Temporal Week 1	NA SD of cell area/Mean cell area, (μm^2) "Poor Quality Images" or "too few cells to register"	58.2 SD of cell area/Mean cell area, (μm^2)	—	—
Coefficient of Variation (CoV) Part B Participant 204 (Vehicle) Temporal Week 8	NA SD of cell area/Mean cell area, (μm^2) "Poor Quality Images" or "too few cells to register"	63.0 SD of cell area/Mean cell area, (μm^2)	—	—
Coefficient of Variation (CoV) Part B Participant 204 (Vehicle) Temporal Week 12	NA SD of cell area/Mean cell area, (μm^2) "Poor Quality Images" or "too few cells to register"	32.3 SD of cell area/Mean cell area, (μm^2)	—	—
Coefficient of Variation (CoV) Part B Participant 204 (Vehicle) Temporal Week 16	NA SD of cell area/Mean cell area, (μm^2) "Poor Quality Images" or "too few cells to register"	63.0 SD of cell area/Mean cell area, (μm^2)	—	—

SECONDARY outcome

Timeframe: Baseline, Weeks 1, 4, 8, 12, and 16

Population: All participants for whom corneal area affected by microcysts was measured.

Change from Baseline in corneal area affected by microcysts by visit for Part A.

Outcome measures

Outcome measures
Measure	Part A Placebo n=18 Participants Part A participants includes those receiving vehicle in eye paired with treatment eye.	Part A Elamipretide 1.0% Ophthalmic Solution n=18 Participants Part A participants includes those receiving 1% Elamipretide	Part B Placebo Part B participants includes those receiving vehicle in both eyes.	Part B Elamipretide 3.0% Ophthalmic Solution Part B Participants includes those receiving 3% Elamipretide
Change From Baseline in Corneal Area Affected by Microcysts for Part A Week 1 Worsened	0 Participants	1 Participants	—	—
Change From Baseline in Corneal Area Affected by Microcysts for Part A Week 1 No Change	18 Participants	17 Participants	—	—
Change From Baseline in Corneal Area Affected by Microcysts for Part A Week 1 Improved	0 Participants	0 Participants	—	—
Change From Baseline in Corneal Area Affected by Microcysts for Part A Week 4 Worsened	0 Participants	0 Participants	—	—
Change From Baseline in Corneal Area Affected by Microcysts for Part A Week 4 No Change	17 Participants	17 Participants	—	—
Change From Baseline in Corneal Area Affected by Microcysts for Part A Week 4 Improved	0 Participants	0 Participants	—	—
Change From Baseline in Corneal Area Affected by Microcysts for Part A Week 8 Worsened	1 Participants	1 Participants	—	—
Change From Baseline in Corneal Area Affected by Microcysts for Part A Week 8 No Change	16 Participants	16 Participants	—	—
Change From Baseline in Corneal Area Affected by Microcysts for Part A Week 8 Improved	0 Participants	0 Participants	—	—
Change From Baseline in Corneal Area Affected by Microcysts for Part A Week 12 Worsened	1 Participants	2 Participants	—	—
Change From Baseline in Corneal Area Affected by Microcysts for Part A Week 12 No Change	15 Participants	14 Participants	—	—
Change From Baseline in Corneal Area Affected by Microcysts for Part A Week 12 Improved	0 Participants	0 Participants	—	—
Change From Baseline in Corneal Area Affected by Microcysts for Part A Week 16 Worsened	0 Participants	1 Participants	—	—
Change From Baseline in Corneal Area Affected by Microcysts for Part A Week 16 No Change	16 Participants	15 Participants	—	—
Change From Baseline in Corneal Area Affected by Microcysts for Part A Week 16 Improved	0 Participants	0 Participants	—	—

SECONDARY outcome

Timeframe: Baseline, Weeks 1, 4, 8, 12, and 16, or early discontinuation visit

Population: All participants for whom corneal area affected by microcysts was measured.

Corneal area affected by microcysts: by-subject data for Part B. No microcysts were present for any timepoints.

Outcome measures

Outcome measures
Measure	Part B Placebo Part B participants includes those receiving vehicle in both eyes.	Part B Elamipretide 3.0% Ophthalmic Solution Part B Participants includes those receiving 3% Elamipretide
Corneal Area Affected by Microcysts: Part B Participant 201 (Elamipretide) All visits	—	—
Corneal Area Affected by Microcysts: Part B Participant 203 (Elamipretide) All visits	—	—
Corneal Area Affected by Microcysts: Part B Participant 204 (Placebo) All visits	—	—
Corneal Area Affected by Microcysts: Part B Participant 205 (Elamipretide) All visits	—	—

SECONDARY outcome

Timeframe: Baseline, Week 1, Week 4, Week 8, Week 12, Week 16

Population: All participants for whom corneal bullae were measured.

Count of participants in number, size and location of bullae by treatment. Part B is listed separately.

Outcome measures

Outcome measures
Measure	Part A Placebo n=18 Participants Part A participants includes those receiving vehicle in eye paired with treatment eye.	Part A Elamipretide 1.0% Ophthalmic Solution n=18 Participants Part A participants includes those receiving 1% Elamipretide	Part B Placebo Part B participants includes those receiving vehicle in both eyes.	Part B Elamipretide 3.0% Ophthalmic Solution Part B Participants includes those receiving 3% Elamipretide
Change From Baseline in Corneal Bullae for Part A. Central Inferior 3 Bullae >1mm, 8 Bullae <0.5mm	0 Participants	1 Participants	—	—
Change From Baseline in Corneal Bullae for Part A. Central Nasal 3 bullae >1mm, 8 bullae <0.5mm	0 Participants	1 Participants	—	—
Change From Baseline in Corneal Bullae for Part A. Central (Superior) 3 Bullae >1mm, 8 Bullae <0.5mm	0 Participants	1 Participants	—	—
Change From Baseline in Corneal Bullae for Part A. Central (Temporal) 3 Bullae >1mm, 8 Bullae <0.5mm	0 Participants	1 Participants	—	—

SECONDARY outcome

Timeframe: Baseline, Week 1, Week 4, Week 8, Week 12, Week 16, or early discontinuation visit

Population: All participants for whom corneal bullae were measured.

Number, size and location of Corneal bullae: By-subject data: Part B.

Outcome measures

Outcome measures
Measure	Part A Placebo n=4 eye Part A participants includes those receiving vehicle in eye paired with treatment eye.	Part A Elamipretide 1.0% Ophthalmic Solution n=4 eye Part A participants includes those receiving 1% Elamipretide	Part B Placebo Part B participants includes those receiving vehicle in both eyes.	Part B Elamipretide 3.0% Ophthalmic Solution Part B Participants includes those receiving 3% Elamipretide
Corneal Bullae: Part B Participant 201 (Elamipretide ) All weeks	0 number of corneal bullae	0 number of corneal bullae	—	—
Corneal Bullae: Part B Participant 203 (Elamipretide) All weeks	0 number of corneal bullae	0 number of corneal bullae	—	—
Corneal Bullae: Part B Participant 204 (Vehicle) All weeks	0 number of corneal bullae	0 number of corneal bullae	—	—
Corneal Bullae: Part B Participant 205 (Elamipretide) All weeks	0 number of corneal bullae	0 number of corneal bullae	—	—

SECONDARY outcome

Timeframe: Baseline, Weeks 1, 4, 8, 12, and 16

Population: All participants for whom corneal stromal folds was measured.

Change from baseline in severity of corneal stromal folds by visit. Descriptive assessment made by Investigator; severity is not assessed using a scale.

Outcome measures

Outcome measures
Measure	Part A Placebo n=18 Participants Part A participants includes those receiving vehicle in eye paired with treatment eye.	Part A Elamipretide 1.0% Ophthalmic Solution n=18 Participants Part A participants includes those receiving 1% Elamipretide	Part B Placebo Part B participants includes those receiving vehicle in both eyes.	Part B Elamipretide 3.0% Ophthalmic Solution Part B Participants includes those receiving 3% Elamipretide
Change From Baseline in Severity of Corneal Stromal Folds for Part A Week 1 Worsened	1 Participants	1 Participants	—	—
Change From Baseline in Severity of Corneal Stromal Folds for Part A Week 1 No Change	16 Participants	15 Participants	—	—
Change From Baseline in Severity of Corneal Stromal Folds for Part A Week 1 Improved	1 Participants	2 Participants	—	—
Change From Baseline in Severity of Corneal Stromal Folds for Part A Week 4 Worsened	1 Participants	0 Participants	—	—
Change From Baseline in Severity of Corneal Stromal Folds for Part A Week 4 No Change	14 Participants	14 Participants	—	—
Change From Baseline in Severity of Corneal Stromal Folds for Part A Week 4 Improved	2 Participants	3 Participants	—	—
Change From Baseline in Severity of Corneal Stromal Folds for Part A Week 8 Worsened	2 Participants	2 Participants	—	—
Change From Baseline in Severity of Corneal Stromal Folds for Part A Week 8 No Change	11 Participants	11 Participants	—	—
Change From Baseline in Severity of Corneal Stromal Folds for Part A Week 8 Improved	4 Participants	4 Participants	—	—
Change From Baseline in Severity of Corneal Stromal Folds for Part A Week 12 Worsened	2 Participants	2 Participants	—	—
Change From Baseline in Severity of Corneal Stromal Folds for Part A Week 12 No Change	10 Participants	12 Participants	—	—
Change From Baseline in Severity of Corneal Stromal Folds for Part A Week 12 Improved	4 Participants	2 Participants	—	—
Change From Baseline in Severity of Corneal Stromal Folds for Part A Week 16 Worsened	2 Participants	2 Participants	—	—
Change From Baseline in Severity of Corneal Stromal Folds for Part A Week 16 No Change	11 Participants	12 Participants	—	—
Change From Baseline in Severity of Corneal Stromal Folds for Part A Week 16 Improved	3 Participants	2 Participants	—	—

SECONDARY outcome

Timeframe: Baseline, Week 1, 4, 8, 12, and 16, or early discontinuation visit

Population: All participants for whom corneal stromal folds was measured.

Severity of corneal stromal folds by-subject data by visit. Descriptive assessment made by Investigator in the following categories: Not present, trace, mild.

Outcome measures

Outcome measures
Measure	Part A Placebo n=4 eyes Part A participants includes those receiving vehicle in eye paired with treatment eye.	Part A Elamipretide 1.0% Ophthalmic Solution n=4 eyes Part A participants includes those receiving 1% Elamipretide	Part B Placebo Part B participants includes those receiving vehicle in both eyes.	Part B Elamipretide 3.0% Ophthalmic Solution Part B Participants includes those receiving 3% Elamipretide
Severity of Corneal Stromal Folds:Part B Participant 205 (Elamipretide) Early discontinuation: trace	0 eyes	1 eyes	—	—
Severity of Corneal Stromal Folds:Part B Participant 201 (Elamipretide) Baseline-Mild	1 eyes	1 eyes	—	—
Severity of Corneal Stromal Folds:Part B Participant 201 (Elamipretide) Week 1 - Trace	1 eyes	0 eyes	—	—
Severity of Corneal Stromal Folds:Part B Participant 201 (Elamipretide) Week 1 -Mild	0 eyes	1 eyes	—	—
Severity of Corneal Stromal Folds:Part B Participant 201 (Elamipretide) Week 4 -Trace	1 eyes	1 eyes	—	—
Severity of Corneal Stromal Folds:Part B Participant 201 (Elamipretide) Week 8-Mild	1 eyes	0 eyes	—	—
Severity of Corneal Stromal Folds:Part B Participant 201 (Elamipretide) Week 8- no folds	0 eyes	1 eyes	—	—
Severity of Corneal Stromal Folds:Part B Participant 201 (Elamipretide) Week 12-Trace	1 eyes	0 eyes	—	—
Severity of Corneal Stromal Folds:Part B Participant 201 (Elamipretide) Week 12- no folds	0 eyes	1 eyes	—	—
Severity of Corneal Stromal Folds:Part B Participant 201 (Elamipretide) Week 16 -Trace	1 eyes	0 eyes	—	—
Severity of Corneal Stromal Folds:Part B Participant 201 (Elamipretide) Week 16 - no folds present	0 eyes	1 eyes	—	—
Severity of Corneal Stromal Folds:Part B Participant 203 (Elamipretide) Baseline-no folds present	1 eyes	1 eyes	—	—
Severity of Corneal Stromal Folds:Part B Participant 203 (Elamipretide) Week 1: no folds present	1 eyes	1 eyes	—	—
Severity of Corneal Stromal Folds:Part B Participant 203 (Elamipretide) Unscheduled visit: no folds present	1 eyes	1 eyes	—	—
Severity of Corneal Stromal Folds:Part B Participant 203 (Elamipretide) Early discontinuation: no folds present	1 eyes	1 eyes	—	—
Severity of Corneal Stromal Folds:Part B Participant 204 (Vehicle) Baseline- Trace	1 eyes	1 eyes	—	—
Severity of Corneal Stromal Folds:Part B Participant 204 (Vehicle) Week 1: no folds present	1 eyes	1 eyes	—	—
Severity of Corneal Stromal Folds:Part B Participant 204 (Vehicle) Week 4: no folds present	1 eyes	1 eyes	—	—
Severity of Corneal Stromal Folds:Part B Participant 204 (Vehicle) Week 8: no folds present	1 eyes	1 eyes	—	—
Severity of Corneal Stromal Folds:Part B Participant 204 (Vehicle) Week 12: no folds present	1 eyes	1 eyes	—	—
Severity of Corneal Stromal Folds:Part B Participant 204 (Vehicle) Week 16: no folds present	1 eyes	1 eyes	—	—
Severity of Corneal Stromal Folds:Part B Participant 205 (Elamipretide) Baseline: Mild	1 eyes	1 eyes	—	—
Severity of Corneal Stromal Folds:Part B Participant 205 (Elamipretide) Week 1: Mild	1 eyes	1 eyes	—	—
Severity of Corneal Stromal Folds:Part B Participant 205 (Elamipretide) Week 4: no folds present	1 eyes	0 eyes	—	—
Severity of Corneal Stromal Folds:Part B Participant 205 (Elamipretide) Week 4: trace	0 eyes	1 eyes	—	—
Severity of Corneal Stromal Folds:Part B Participant 205 (Elamipretide) Unscheduled visit: No folds present	1 eyes	0 eyes	—	—
Severity of Corneal Stromal Folds:Part B Participant 205 (Elamipretide) Unscheduled visit: trace	0 eyes	1 eyes	—	—
Severity of Corneal Stromal Folds:Part B Participant 205 (Elamipretide) Early discontinuation visit: no folds present	1 eyes	0 eyes	—	—

SECONDARY outcome

Timeframe: Baseline, Week 1, 4, 8, 12 weeks

Population: All participants for whom contrast sensitivity was measured

Change from Baseline in contrast sensitivity over all 12 weeks log score at 3, 6, 12, 18 cycles per degree (cpd) using Vector Vision's CSV-1000E. Standard tables for the VectorVision's CSV-1000E model were used to convert linear results to the log values. Lower log scores equals lower contrast sensitivity and worse outcome. Higher log scores mean higher contrast sensitivity and better outcome. For 3cpd, range is 0.7-2.08; 6 cpd: 0.91-2.29; 12 cpd: 0.61-1.99; 18cpd: 0.17-1.55, unless no gratings were visible. If no gratings were visible, .3 log was subtracted from the lowest score for 3, 6, and 12cpd. For 18cpd .01 log was used, or essentially 100% contrast.

Outcome measures

Outcome measures
Measure	Part A Placebo n=18 Participants Part A participants includes those receiving vehicle in eye paired with treatment eye.	Part A Elamipretide 1.0% Ophthalmic Solution n=18 Participants Part A participants includes those receiving 1% Elamipretide	Part B Placebo Part B participants includes those receiving vehicle in both eyes.	Part B Elamipretide 3.0% Ophthalmic Solution Part B Participants includes those receiving 3% Elamipretide
Change From Baseline in Contrast Sensitivity (Log Score) for Part A 3 cycles per degree	0.084 log score Standard Deviation 0.0554	-0.013 log score Standard Deviation 0.0499	—	—
Change From Baseline in Contrast Sensitivity (Log Score) for Part A 6 cycles per degree	0.193 log score Standard Deviation 0.0745	0.111 log score Standard Deviation 0.0374	—	—
Change From Baseline in Contrast Sensitivity (Log Score) for Part A 12 cycles per degree	0.050 log score Standard Deviation 0.0787	0.013 log score Standard Deviation 0.0503	—	—
Change From Baseline in Contrast Sensitivity (Log Score) for Part A 18 cycles per degree	0.122 log score Standard Deviation 0.0637	0.080 log score Standard Deviation 0.0564	—	—

SECONDARY outcome

Timeframe: Baseline, Week 1, Week 4, Week 8, Week 12, Week 16, or early discontinuation visit

Population: All participants for whom contrast sensitivity was measured.

Contrast Sensitivity log score at 3, 6, 12, 18 cycles per degree (cpd) using Vector Vision's CSV-1000E by-subject data, Part B. Part A is listed separately. Standard tables for the VectorVision's CSV-1000E model were used to convert linear results to the log values. Lower log scores equals lower contrast sensitivity and worse outcome. Higher log scores mean higher contrast sensitivity and better outcome. For 3cpd, range is 0.7-2.08; 6 cpd: 0.91-2.29; 12 cpd: 0.61-1.99; 18cpd: 0.17-1.55, unless no gratings were visible. If no gratings were visible, .3 log was subtracted from the lowest score for 3, 6, and 12cpd. For 18cpd .01 log was used, or essentially 100% contrast.

Outcome measures

Outcome measures
Measure	Part A Placebo n=4 Participants Part A participants includes those receiving vehicle in eye paired with treatment eye.	Part A Elamipretide 1.0% Ophthalmic Solution n=4 Participants Part A participants includes those receiving 1% Elamipretide	Part B Placebo Part B participants includes those receiving vehicle in both eyes.	Part B Elamipretide 3.0% Ophthalmic Solution Part B Participants includes those receiving 3% Elamipretide
Contrast Sensitivity for Part B; By-subject Data Participant 205 (Elamipretide) 18cpd Week 4	0.01 log score	0.01 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 203 (Elamipretide) 3 cpd Screening	0.70 log score	0.70 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 203 (Elamipretide) 3 cpd Week 1	1.00 log score	1.00 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 203 (Elamipretide) 3 cpd Early Discontinuation	0.70 log score	0.70 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 203 (Elamipretide) 6 cpd Screening	0.61 log score	0.90 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 203 (Elamipretide) 6 cpd Week 1	0.90 log score	1.20 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 203 (Elamipretide) 6 cpd Early Discontinuation	0.61 log score	0.61 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 203 (Elamipretide) 12cpd Baseline	0.60 log score	0.31 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 203 (Elamipretide) 12cpd Week 1	0.31 log score	0.60 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 203 (Elamipretide) 12cpd Early Discontinuation	0.60 log score	0.60 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 203 (Elamipretide) 18cpd Baseline	0.01 log score	0.18 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 203 (Elamipretide) 18cpd Week 1	0.01 log score	0.18 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 203 (Elamipretide) 18 cpd Early Discontinuation	0.01 log score	0.18 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 204 (Vehicle) 3cpd Baseline	1.18 log score	1.18 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 204 (Vehicle) 3cpd Week 1	1.63 log score	1.00 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 204 (Vehicle) 3cpd Week 4	1.49 log score	1.34 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 204 (Vehicle) 3cpd Week 8	1.49 log score	1.34 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 204 (Vehicle) 3cpd Week 12	1.79 log score	1.63 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 204 (Vehicle) 3cpd Week 16	1.79 log score	1.34 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 204 (Vehicle) 6cpd Baseline	1.38 log score	1.38 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 204 (Vehicle) 6cpd Week 1	1.85 log score	1.20 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 204 (Vehicle) 6cpd Week 4	1.38 log score	1.56 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 204 (Vehicle) 6cpd Week 8	1.70 log score	1.20 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 204 (Vehicle) 6cpd Week 12	1.70 log score	1.56 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 204 (Vehicle) 6cpd Week 16	2.00 log score	0.90 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 204 (Vehicle) 12cpd Baseline	0.60 log score	0.60 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 204 (Vehicle) 12cpd Week 1	1.08 log score	0.90 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 204 (Vehicle) 12cpd Week 4	1.26 log score	1.08 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 204 (Vehicle) 12cpd Week 8	0.60 log score	0.90 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 204 (Vehicle) 12cpd Week 12	1.08 log score	0.60 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 204 (Vehicle) 12cpd Week 16	1.26 log score	0.60 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 204 (Vehicle) 18cpd Baseline	0.65 log score	0.01 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 204 (Vehicle) 18cpd Week 1	0.65 log score	0.18 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 204 (Vehicle) 18cpd Week 4	0.65 log score	0.65 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 204 (Vehicle) 18cpd Week 8	0.18 log score	0.48 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 204 (Vehicle) 18cpd Week 12	0.65 log score	0.01 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 204 (Vehicle) 18cpd Week 16	0.65 log score	0.65 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 205 (Elamipretide) 3cpd Baseline	1.34 log score	0.40 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 205 (Elamipretide) 3cpd Week 1	1.18 log score	0.40 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 205 (Elamipretide) 3cpd Week 4	0.70 log score	0.40 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 205 (Elamipretide) 3cpd Early Discontinuation	1.00 log score	0.40 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 205 (Elamipretide) 6cpd Baseline	0.90 log score	0.61 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 205 (Elamipretide) 6cpd Week 1	0.90 log score	0.61 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 205 (Elamipretide) 6cpd Week 4	0.61 log score	0.61 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 205 (Elamipretide) 6cpd Early Discontinuation	0.61 log score	0.61 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 205 (Elamipretide) 12cpd Baseline	0.31 log score	0.31 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 205 (Elamipretide) 12cpd Week 1	1.26 log score	0.31 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 205 (Elamipretide) 12cpd Week 4	0.31 log score	0.31 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 205 (Elamipretide) 12cpd Early Discontinuation	0.31 log score	0.31 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 205 (Elamipretide) 18cpd Baseline	0.01 log score	0.01 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 205 (Elamipretide) 18cpd Week 1	0.65 log score	0.01 log score	—	—
Contrast Sensitivity for Part B; By-subject Data Participant 205 (Elamipretide) 18cpd Early Discontinuation	0.01 log score	0.01 log score	—	—

Adverse Events

Part A Elamipretide 1.0% Ophthalmic Solution and Placebo

Serious events: 1 serious events

Other events: 7 other events

Deaths: 0 deaths

Part A Placebo Only

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Part A Elamipretide 1.0% Ophthalmic Solution Only

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Part B Placebo

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Part B Elamipretide 3.0% Ophthalmic Solution

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Part A Elamipretide 1.0% Ophthalmic Solution and Placebo n=18 participants at risk Part A participants includes those receiving 1% Elamipretide in one eye and placebo in other eye. This arm will report systemic events that cannot be attributed to either intervention.	Part A Placebo Only n=18 participants at risk These will be reporting ocular AEs for placebo eye only. Part A participants includes those receiving Placebo in one eye only.	Part A Elamipretide 1.0% Ophthalmic Solution Only n=18 participants at risk These will be reporting ocular AEs for elamipretide eye only. Part A participants includes those receiving 1% Elamipretide in one eye only.	Part B Placebo n=1 participants at risk Part B participants includes those receiving Placebo in both eyes. This arm will be reporting ocular and systemic events.	Part B Elamipretide 3.0% Ophthalmic Solution n=3 participants at risk Part B participants includes those receiving 3% Elamipretide in both eyes. This arm will be reporting ocular and systemic events.
Gastrointestinal disorders Small Intestinal Obstruction	5.6% 1/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Metabolism and nutrition disorders Dehydration	5.6% 1/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.

Other adverse events

Other adverse events
Measure	Part A Elamipretide 1.0% Ophthalmic Solution and Placebo n=18 participants at risk Part A participants includes those receiving 1% Elamipretide in one eye and placebo in other eye. This arm will report systemic events that cannot be attributed to either intervention.	Part A Placebo Only n=18 participants at risk These will be reporting ocular AEs for placebo eye only. Part A participants includes those receiving Placebo in one eye only.	Part A Elamipretide 1.0% Ophthalmic Solution Only n=18 participants at risk These will be reporting ocular AEs for elamipretide eye only. Part A participants includes those receiving 1% Elamipretide in one eye only.	Part B Placebo n=1 participants at risk Part B participants includes those receiving Placebo in both eyes. This arm will be reporting ocular and systemic events.	Part B Elamipretide 3.0% Ophthalmic Solution n=3 participants at risk Part B participants includes those receiving 3% Elamipretide in both eyes. This arm will be reporting ocular and systemic events.
Eye disorders Vision blurred	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	5.6% 1/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	100.0% 1/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Eye disorders Dry Eye	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	5.6% 1/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	5.6% 1/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	100.0% 1/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Eye disorders Conjunctival Haemorrhage	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	100.0% 1/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Eye disorders Lacrimation increased	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	33.3% 1/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Eye disorders Eye pruritis	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	5.6% 1/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	33.3% 1/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Eye disorders Eyelid Oedema	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	33.3% 1/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Eye disorders Conjunctivitis allergic	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	66.7% 2/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Nervous system disorders Burning sensation	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	66.7% 2/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Infections and infestations Influenza	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	33.3% 1/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Nervous system disorders Presyncope	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	33.3% 1/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Psychiatric disorders Depression	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	100.0% 1/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Eye disorders Eye Pain	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	5.6% 1/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Investigations Vital dye staining cornea present	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	11.1% 2/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	5.6% 1/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Cardiac disorders Atrial Flutter	5.6% 1/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Infections and infestations Clostridium difficile colitis	5.6% 1/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Investigations Hepatic enzyme increase	5.6% 1/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Investigations Vitamin B12 deficiency	5.6% 1/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Musculoskeletal and connective tissue disorders Arthralgia	5.6% 1/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Nervous system disorders Dysgeusia	11.1% 2/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	33.3% 1/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Nervous system disorders Headache	5.6% 1/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	33.3% 1/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Skin and subcutaneous tissue disorders Onychoclasis	5.6% 1/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Surgical and medical procedures Cardioversion	5.6% 1/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Surgical and medical procedures Shoulder arthroplasty	5.6% 1/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Vascular disorders Hypotension	5.6% 1/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Vascular disorders Thrombophlebitis superficial	5.6% 1/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Eye disorders Eyelid Disorder	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	5.6% 1/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Eye disorders Cataract Cortical	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	5.6% 1/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Eye disorders Diplopia	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	5.6% 1/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Eye disorders Ocular hyperaemia	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	5.6% 1/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Eye disorders Visual impairment	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	5.6% 1/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
Immune system disorders Hypersensitivity	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	5.6% 1/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.
General disorders Pain	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	0.00% 0/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	11.1% 2/18 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	100.0% 1/1 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.	66.7% 2/3 • From the screening period (Day-28 to Day 1) to Week 16 follow up visit, approximately 5 months in total.

Additional Information

Jim Carr, Pharm.D. Chief Clinical Development Officer

Stealth BioTherapeutics, Inc

Phone: 1-617-600-6888

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: GT60

Measure	Elamipretide 1.0% Ophthalmic Solution and Placebo n=18 Participants Part A participants includes those receiving Elamipretide and placebo	Part B Elamipretide 3.0% Ophthalmic Solution n=3 Participants Part B Participants includes those receiving Elamipretide.	Part B Placebo n=1 Participants Part B Participants include those receiving Placebo.	Total n=22 Participants Total of all reporting groups
Age, Continuous	67.9 years STANDARD_DEVIATION 8.64 • n=5 Participants	NA years STANDARD_DEVIATION NA • n=7 Participants	NA years STANDARD_DEVIATION NA • n=5 Participants	67.9 years STANDARD_DEVIATION 8.64 • n=4 Participants
Sex: Female, Male Female	8 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants	10 Participants n=4 Participants
Sex: Female, Male Male	10 Participants n=5 Participants	2 Participants n=7 Participants	0 Participants n=5 Participants	12 Participants n=4 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	18 Participants n=5 Participants	3 Participants n=7 Participants	1 Participants n=5 Participants	22 Participants n=4 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Black or African American	2 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	2 Participants n=4 Participants
Race (NIH/OMB) White	16 Participants n=5 Participants	3 Participants n=7 Participants	1 Participants n=5 Participants	20 Participants n=4 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Smoking Former Smoker	10 Participants n=5 Participants	3 Participants n=7 Participants	0 Participants n=5 Participants	13 Participants n=4 Participants
Smoking Current Smoker	1 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants
Smoking Never Smoker	7 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants	8 Participants n=4 Participants