Treatment for Dry Age-Related Macular Degeneration Enters Phase 3 Trials

Introduction to Elamipretide and Dry AMD

Stealth BioTherapeutics has begun testing a new drug called elamipretide in a Phase 3 clinical trial for patients with dry age-related macular degeneration (dry AMD). This trial represents a significant step towards potentially offering patients a home-based treatment for this common eye disease.

About the ReNEW and ReGAIN Trials

The ReNEW and ReGAIN trials are designed to evaluate the efficacy and safety of daily injections of elamipretide. These trials will measure how well the drug can slow the loss of photoreceptors in the retina, which is a key problem in dry AMD. This is done using advanced imaging techniques over a 48-week period.

In the ReNEW trial, 360 participants will be randomly assigned to receive either elamipretide or a placebo for 96 weeks. Participants will also have the option to join the ReTAIN open-label extension trial afterwards.

How Elamipretide Works

Elamipretide is a mitochondrial-targeted therapy. It is designed to boost the function of mitochondria, the energy-producing structures in cells. In dry AMD, mitochondrial dysfunction is thought to contribute to the damage and loss of photoreceptors in the retina.

This treatment can be administered at home by patients themselves, making it a convenient option for managing earlier stages of dry AMD.

Importance of New Treatments for Dry AMD

Currently, there are limited treatment options for dry AMD, which is the leading cause of irreversible blindness in older adults. The disease affects nearly 20 million Americans aged 40 and older, with most cases being the dry form.

New therapies like elamipretide offer hope for slowing the progression of the disease, preserving vision, and improving quality of life for many patients.

What Happens Next?

With the first patient already enrolled and dosed in the ReNEW trial, Stealth BioTherapeutics is set to proceed with further patient enrollments across multiple sites in the U.S. The company is optimistic about the potential benefits of elamipretide, based on the drug's ability to target mitochondrial dysfunction and improve bioenergetic balance in retinal cells.

Further Applications of Elamipretide

In addition to its potential use in treating dry AMD, elamipretide is also being studied in other diseases. It is currently in Phase 3 trials for primary mitochondrial myopathy, a rare muscle disorder, and is under review for treating Barth syndrome, a rare condition affecting the heart and muscles.

Stealth BioTherapeutics is also developing other mitochondrial-targeted drugs, such as bevemipretide, for eye and neurological diseases.

Conclusion

The initiation of the ReNEW and ReGAIN trials marks an important milestone in the quest to find effective treatments for dry AMD. If successful, elamipretide could become a game-changer for patients, offering a new way to manage this debilitating disease from the comfort of their homes.