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Scott W Cousins, Michael John Allingham, Priyatham S Mettu; Elamipretide, a Mitochondria-Targeted Drug, for the Treatment of Vision Loss in Dry AMD with Noncentral Geographic Atrophy: Results of the Phase 1 ReCLAIM Study. Invest. Ophthalmol. Vis. Sci. 2019;60(9):974.
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To report the results from the noncentral geographic atrophy (NCGA) subgroup of the ReCLAIM Study, an open-label, phase 1 clinical trial to evaluate safety, tolerability, and efficacy of elamipretide, a mitochondria-targeted drug.
Open-label, phase 1 clinical trial of daily subcutaneous elamipretide (40 mg) for 24 weeks in individuals with dry AMD. For the pre-defined NCGA subgroup, NCGA was defined as well-demarcated area(s) of hypo-autofluorescence on fundus autofluorescence with cumulative area ≥ 1.27 mm2 (approximately 0.5 disc areas (DA)) and ≤ 10.16 mm2 (approximately 4 DA), sparing the foveola. One eye of each participant was eligible if ETDRS best-corrected visual acuity (BCVA) was ≥ 55 letters with low luminance VA (LLVA) deficit > 5 letters. LLVA was measured as best-corrected ETDRS VA through a log 2 neutral density filter, and LLVA deficit was defined as the difference between BCVA and LLVA. Safety and visual function assessments occurred every four weeks, with outcomes assessed at week 24.
Subcutaneous elamipretide was safe and well tolerated with no treatment-related serious adverse events. Among participants in the NCGA subgroup (n=19, mean age 76.0, 57.9% female), mean BCVA at baseline was 73.7 ± 9.5 letters, with mean increase of 4.6 ± 5.1 letters (p=0.003) at week 24. Mean baseline LLVA was 43.9 ± 19.8 letters, with mean increase of 5.4 ± 7.9 letters (p=0.019). Mean best-corrected reading acuity under standard lighting conditions (BCRA) (tested using MN read acuity chart) at baseline was logMAR 0.15 ± 0.23, with mean change in BCRA of -0.02 ± 0.13 (p=0.55). Mean low luminance reading acuity through log 2 neutral density filter (LLRA) at baseline was logMAR 1.28 ± 1.07, with mean increase in LLRA of -0.52 ± 0.75 (p<0.017), equivalent to approximately 5-line gain. Mean change in NCGA area (SQ RT) was 0.13 mm over the 24-week study period. Patient-reported outcomes on the low luminance and VFQ-39 questionnaires demonstrated statistically significant improvement in daily quality-of-life measures, especially those related to low luminance visual function.
Elamipretide, a mitochondria-targeted drug, is safe and well tolerated in patients with dry AMD and may improve visual acuity in patients with NCGA. A Phase 2b, placebo-controlled clinical trial is being organized.
This abstract was presented at the 2019 ARVO Annual Meeting, held in Vancouver, Canada, April 28 - May 2, 2019.
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