Advancing Elamipretide into Phase 3 ReNEW and ReGAIN Trials

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Arshad Khanani, MD provides insight into the pivotal Phase 3 clinical trials investigating elamipretide for geographic atrophy in dry AMD.

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: Elamipretide is now in Phase 3 pivotal clinical trials, the ReNEW and ReGAIN studies. When these trials were designed, we took the learnings from Phase 2 and that informed the selection of a proper endpoint for the mechanism of action, which is measuring ellipsoid zone (EZ) to retinal pigment epithelium (RPE) thickness, or that of a typical GA growth endpoint.

These programs, ReNEW and ReGAIN, are global programs looking at photoreceptor preservation and vision function as the endpoint of interest. Inclusion criteria in ReNEW and ReGAIN will enable patients with less severe disease to participate in clinical trials, unlike most dry AMD trials, where we are intervening with large GA lesions.

When you look at the clinical trial design, patients will receive elamipretide 40 mg subcutaneously, or placebo injection subcutaneously. Patients can have very small GA lesion areas to be included in these trials. The key criteria are non-central GA lesions ≥0.50 mm2 or ≤10.16 mm2, and ≥150 µm from the foveal center, a best-corrected visual acuity (BCVA) score of ≥55 ETDRS letters, and low-luminance visual acuity (LLVA) of ≥10 letters, with an LLVA deficit of >5 ETDRS letters that we used in the phase 2 trial.

The primary endpoint will be the slope of change in EZ total attenuation. Secondary and exploratory endpoints will include the change in EZ total attenuation, as well as the categorical change in LLVA. As you recall, in the Phase 2 study, we saw a significant benefit when you're looking at the change in EZ attenuation, as well as the benefit of improving LLVA. This treatment protects against mitochondrial dysfunction. We hope that we will be able to get those sick mitochondria back to functioning again. The program has hit a significant milestone with the first patient recently enrolled in the ReNEW study.

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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