Stealth BioTherapeutics (MITO) Reports Positive Results for Elamipretide in Ophthalmic Conditions

April 30, 2019 5:26 PM EDT

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Stealth BioTherapeutics (NASDAQ: MITO recently presented positive results from its ophthalmic programs in dry age-related macular degeneration (AMD) and Leber's hereditary optic neuropathy (LHON). The data, which were presented at the Association for Research in Vision and Ophthalmology (ARVO) 2019 Annual Meeting, being held April 28 – May 2 in Vancouver, British Columbia, demonstrated significant improvements in visual function following treatment with elamipretide, an investigational drug, in its ReCLAIM study of patients with dry AMD and in the open-label portion of its ReSIGHT study of patients with LHON.

"People with dry AMD typically experience a progressive vision loss that interferes with day-to-day living and limits independence," said Dr. Scott Cousins, principal investigator and Professor of Ophthalmology and Director of the Duke University Center for Macular Diseases, who gave an oral presentation of the non-central geography cohort data at ARVO. "ReCLAIM provides preliminary evidence that elamipretide improved vision in patients with the disease, and we look forward to further evaluation in the ongoing Phase 2 study."

ReCLAIM was a Phase 1 open-label study evaluating daily subcutaneous elamipretide for 24 weeks in patients with dry AMD with non-central geographic atrophy or high-risk drusen. Patients with non-central geographic atrophy (n=15) showed a mean increase in low-luminance visual acuity, or clarity of vision in low light, of 5.4 ± 7.9 letters (baseline of 43.9 ± 19.8 letters; p=0.025) and best-corrected visual acuity, or best distance vision with glasses or contact lenses, of 4.6 ± 5.1 letters (baseline of 73.7 ± 9.5 letters; p=0.003). A significant improvement was also observed in low-luminance smallest line read correctly of -0.52 ± 0.75 (p<0.017), which corresponds to a gain of five lines on an eye chart. Patient-reported outcomes on the low-luminance and visual function questionnaires demonstrated significant improvement in daily quality-of-life measures, especially relating to low-luminance visual function. The patients with high-risk drusen (n=19; fatty deposits under the retina that can lead to future vision loss) also showed significant improvements in best corrected visual acuity, low-luminance visual acuity, reading acuity and patient-reported outcomes. Data were also presented showing similar visual acuity improvements in fellow eyes that had neovascular AMD.

Significant improvements in visual function were also observed in the open-label extension portion of ReSIGHT, a Phase 2, double-masked, placebo-controlled study investigating the safety, tolerability and efficacy of elamipretide topical ophthalmic solution for 52 weeks in 12 patients with established vision loss (greater than one year but less than 10 years) due to genetically confirmed LHON with the G11778A mutation, followed by an ongoing open-label extension in which all 12 patients participated and have completed at least 84 total weeks of treatment. Although no difference in best-corrected visual acuity was observed during the double-masked portion of the trial, trends toward improvement were observed, including significant improvement in patient-reported outcomes. The poster presentation of the week 84 open-label results showed amplification of improvement with introduction of bilateral elamipretide therapy, demonstrating significant improvements compared to baseline in best-corrected visual acuity, visual field mean deviation, contrast sensitivity and color discrimination.

"LHON is a devastating disease that causes profound and sudden vision loss in the prime of life and currently has no FDA-approved treatment," said Dr. Alfredo Sadun, UCLA Doheny Eye Institute, the primary investigator for the study. "It's encouraging that patients reported an impact on their daily activities and vision, both being key goals of a potential treatment for those with LHON."

In both studies, elamipretide was well tolerated and most adverse events were mild to moderate in severity. In the ReCLAIM study, the most commonly reported adverse events included injection site reactions. In the ReSIGHT study, adverse events occurred in a similar frequency between elamipretide and placebo-treated eyes.

"Our visual system, which has the greatest demand and highest mitochondrial density in the body, can be severely compromised by age-related or genetic mitochondrial dysfunction," said Stealth Chief Executive Officer Reenie McCarthy. "Since loss of visual function may have devastating impacts on quality of life, we are highly encouraged by this early evidence that elamipretide may have the potential to improve vision in these severe, progressive ophthalmic diseases for which there are no approved medicines. We look forward to completing enrollment in our ReCLAIM-2 study by early next year, as well as our upcoming regulatory discussion regarding LHON."

The U.S. Food and Drug Administration (FDA) has granted Fast Track designations for elamipretide for the treatment of dry AMD and LHON, as well as Orphan Drug designation for LHON. For additional information on the studies or elamipretide, please refer to Stealth's website and ClinicalTrials.gov.



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