Clinical trials are an important tool within the healthcare realm. These trials assist in developing deeper understandings of diseases, assessing and discovering treatment options, and learning new ways of identification and diagnosis. In some cases, clinical trials show a drug’s benefits while also missing primary endpoints (the results measured to show evidence of a treatment’s efficacy or safety). According to a news release from biotechnology company Stealth BioTherapeutics, this is what recently happened in a Phase 2 clinical trial evaluating elamipretide for people with geographic atrophy secondary to dry age-related macular degeneration (dry AMD). Despite the trial not reaching its primary endpoints, researchers did find potential benefits of elamipretide for patients.
Elamipretide Data: ReCLAIM-2 Trial
So what exactly is elamipretide? According to Stealth BioTherapeutics, elamipretide is:
a peptide compound that readily penetrates cell membranes and targets the inner mitochondrial membrane where it binds reversibly to cardiolipin. Functional benefit is improved through improvement of ATP production and interruption and potential reversal of damaging oxidative stress.
Preclinical studies, as well as a Phase 1 clinical trial, highlighted how elamipretide was able to reduce pathogenic ROS levels, improve ATP production and mitochondrial respiration, and heighten visual function.
Within the Phase 2 ReCLAIM-2 clinical trial, researchers evaluated elamipretide for those with geographic atrophy. In particular, researchers sought to understand how the treatment, administered subcutaneously, improved LLVA and GA. Altogether, 176 patients enrolled. Findings included:
- While elamipretide did improve LLVA when compared to placebo, the changes were not significant enough to meet the trial endpoint. However, the treatment did improve visual function and visual acuity.
- Elamipretide prevented progressive ellipsoid zone loss, helping to preserve vision.
- Altogether, the treatment was safe and relatively well-tolerated. The most common side effect was injection site reactions.
Moving forward, Stealth BioSciences hopes to continue developing elamipretide and address some of the issues observed in this study.
What is Geographic Atrophy?
Geographic atrophy is considered to be an advanced form of age-related macular degeneration, an eye disease which causes vision loss. It occurs when areas of the retina begin to atrophy. According to Prevent Blindness, the retinal atrophy typically begins near the fovea in the center of the macula. As geographic atrophy progresses, it can cause permanent vision loss. Risk factors for geographic atrophy include being older than 60 years old, having a family history of macular degeneration, being Caucasian, having light-colored eyes, high blood pressure or high cholesterol, obesite, or high sun exposure. Typically, if you experience geographic atrophy in one eye, it will spread to the other eye. Symptoms include:
- Dull or washed-out colors
- A loss of vision sharpness or clarity
- Difficulty seeing in dim light or seeing in the center of your vision
- A dim or dark spot in your central vision
