What Does Elamipretide Represent in Dry AMD Patients with GA?


Arshad Khanani, MD points to the viability of the mechanism of action of elamipretide for geographic atrophy in dry AMD, and what it could represent for patient outcomes.

In this HCPLive® Clinical Trial Spotlight, Arshad Khanani, MD breaks down the investigational product candidate, elamipretide, and the Phase 2 ReCLAIM-2 clinical trial, for the treatment of geographic atrophy (GA) in dry age-related macular degeneration (AMD).
Khanani is the director of clinical research at Sierra Eye Associates and a clinical professor of medicine at the University of Nevada, Reno School of Medicine. He serves as an investigator in the Elamipretide clinical trial program.
Each segment features Khanani breaking down the ophthalmic pipeline for GA, elamipretide’s mechanism of action, primary results from the ReCLAIM-2 clinical trial, what role it could play in treatment, and the recently announced Phase 3 clinical trial program.

Khanani: In summary, mitochondrial-targeted therapy, targeting the bioenergetic imbalance that is a major contribution to the progression of dry AMD, is very exciting. Given the mechanism of action of elamipretide and the route of administration, which is subcutaneous, it would be a viable therapy for patients, including the early dry AMD patient population. This therapy can be given at home by the patient or the caregiver.

As we know, dry AMD is bilateral disease in most cases, and a systemic treatment will help these patients benefit in both eyes, while avoiding the treatment burden that is a main concern with the currently approved treatments. We want therapy to be safe, and based on the data that we have seen, elamipretide has no drug-related systemic adverse events, except for injection area irritation that can be addressed with topical steroids or oral antihistamines.

In terms of trial design, learning from Phase 2 informed the selection of a proper endpoint based on the mechanism of action, which is the ellipsoid zone (EZ) to retinal pigment epithelium (RPE) thickness over time, compared to the GA growth endpoint that we have seen. This EZ attenuation endpoint has been validated by the US Food and Drug Administration (FDA) and the Phase 3 programs, ReNEW and ReGAIN studies are ongoing.

The goal is to preserve photoreceptor function and hopefully have some functional benefit for our patients suffering from this disease. Sick mitochondria can hopefully get back to normal or improve in function with this drug, and the Phase 3 trials are looking at patients with early GA.

We can treat these patients and preserve their function. In a nutshell, it's all about structure, functional relationship, and targeting mitochondria structure and architecture, which is important to the function of photoreceptors. It is a druggable target, in my opinion, and I'm looking forward to participating in the Phase 3 studies with elamipretide in patients with dry AMD.

This transcript has been edited for clarity.

Disclosures: Dr. Khanani serves as a consultant to Stealth BioTherapeutics, the sponsor of this content.

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