An Open-Label, Phase 1 Clinical Study to Evaluate the Safety and Tolerability of Subcutaneous Elamipretide in Subjects With Intermediate Age-Related Macular Degeneration

Inclusion Criteria

• •For this study, only 1 eye of an eligible subject will be included and designated as the study eye. However, all specified ophthalmic testing will be performed on both eyes at each time point. A potential subject must meet the following criteria to be eligible for inclusion in the study:
• •Intermediate AMD - noncentral GA disease group:
• •1. Adults ≥ 55 years of age with 1 eye with intermediate AMD - noncentral GA.
• •2. No evidence of choroidal neovascularization (active or prior history) in the study eye.
• •3. Geographic atrophy may be multifocal, but the cumulative GA lesion size must be:
• •1. ≥ 1.27 mm2 (approximately ≥ 0.5 DA) and ≤ 10.16 mm2 (approximately ≤ 4 DA).
• •2. Must reside completely within the FAF imaging field (field 2 to 30-degree image centered on the fovea).
• •4. Presence of measurable hyperautofluorescence adjacent to the discrete foci of GA.
• •OR
• •Intermediate AMD - high-risk drusen without GA disease group:
• •5. ≥ 55 years of age with one eye with intermediate AMD - high-risk drusen without GA.
• •6. High-risk drusen is defined as presence of either at least 1 large (≥ 125 µm) druse or multiple medium-size (between 63 and 124 µm) drusen.
• •General (both disease groups):
• •7. Able to provide informed consent and willing to comply with all study visits and examinations.
• •8. Women of childbearing potential who are not pregnant or nursing and have a negative serum pregnancy test at screening.
• •9. Best-corrected visual acuity assessed by ETDRS letters ≥ 55 letters (Snellen equivalent ≥ 20/70).
• •10. Low-luminance visual acuity deficit (defined as difference between BCVA and LL visual acuity) \> 5 letters.
• •11. Has at least two Low-Luminance Questionnaire sub scale results, in which one of the abnormal subscales is either general dim light vision or dim light reading.
• •12. The fellow eye may have intermediate AMD without noncentral GA (i.e., high-risk drusen), intermediate AMD with noncentral GA, NV AMD, or central GA. Ongoing treatment with antiangiogenic therapies in the fellow eye is allowable.
• •13. No evidence of visually significant cataract OR pseudophakia without evidence of posterior capsular opacity.
• •14. Sufficiently clear ocular media, adequate pupillary dilation, fixation to permit quality fundus imaging, and able to cooperate sufficiently for adequate ophthalmic visual function testing and anatomic assessment.
• •15. Able to administer SC study drug solution as demonstrated at screening or able to have a care provider or appropriate designee who can administer the study drug (i.e., a capable family member or home health nursing aide).
• •16. If of childbearing potential or in a relationship with a partner of childbearing potential, are able to abstain from sex or use acceptable contraception during the study and for 3 months after dosing.
• •1. For men: Abstinence is only acceptable if it is in line with the preferred and usual lifestyle of the subject. The subject also agrees to use an acceptable method of contraception should they become sexually active. Subjects must use a condom with spermicide from the date of informed consent until at least 3 months after the last dose of study drug. Periodic abstinence (e.g.calendar, ovulation, symptothermal, or postovulation methods) and withdrawal are not acceptable methods of contraception.
• •2. For women: abstinence is only acceptable when it is in line with the preferred and usual lifestyle of the subject. The subject agrees to use an acceptable method of contraception should they become sexually active. Maintenance of a monogamous relationship with a male partner who has been surgically sterilized by vasectomy (the vasectomy procedure must have been conducted at least 60 days before the Screening visit or confirmed via sperm analysis), barrier method (e.g., condom or occlusive cap) with spermicidal foam/gel/film/cream AND either hormonal contraception (oral, implanted, or injectable) or an intrauterine device or system are acceptable methods.
• •17. Ability and willingness to undertake all scheduled visits and assessments.