Stealth BioTherapeutics (NASDAQ:MITO) announced top-line data from its Phase 2 ReCLAIM-2 trial, which was evaluating elamipretide in patients with geographic atrophy (GA) secondary to dry age-related macular degeneration. Stealth management announced results from the ReCLAIM-2 trial that indicated primary endpoints were not met. However, in what we view as an overlooked but important development, the ReCLAIM-2 trial had multiple positive data points which we view as an encouraging outcome.
◦ The trial showed that elamipretide “categorically improved visual function for patients with GA.
◦ Elamipretide also showed proof of mechanism by reducing progressive ellipsoid zone loss, which, according to the company, is a key biomarker of retinal mitochondrial health and has shown to be predictive of long-term GA growth and development.
Additionally, elamipretide was again shown to be well tolerated by the patient group in the Phase 2 trials. We view these results as a net positive and believe the forward progress for elamipretide continues. It’s important to note that there are no current approved treatments for GA and, given the results of the trial, believe the FDA will work with Stealth to get a treatment to market. We believe the results of Phase 2 also increase the chances of some sort of a partnership with a larger drug company. Stealth Biotherapeutics notes that normal mitochondrial play a critical role for ocular function and dysfunctional mitochondria play a part in several rare and common diseases of the eye. The company also notes that ophthalmologic diseases that have not traditionally been considered to have obvious mitochondrial origins are increasingly recognized to result at least in part from impaired mitochondrial function. Oxidative damage that results over time from inherited mtDNA mutations or prolonged oxidative stress instability leads to cumulative mitochondrial damage, which is recognized to be an important factor in disorders such as dry age-related macular degeneration (AMD) and Leber’s hereditary optic neuropathy (LHON).
Stealth is pursuing elamipretide for treatment of geographic atrophy (GA), which is an advanced form of AMD. AMD is estimated to impact more than 10 million people in the United States and there are no known treatments for the disease. AMD results in distorted vision—more specifically, a reduction in low luminance visual acuity, reduced overall visual acuity and blurred vision—and is the leading cause of blindness among older adults in the developed world.
According to the National Institute of Health (NIH), recent evidence suggests that mitochondrial damage and oxidative stress in the retinal pigment epithelium (RPE) may play an important role in AMD. As seem in the graphic below, RPE mitochondria in AMD eyes undergo more pronounced degenerative changes, with lower mitochondrial density, organelle area and cristae number.
The results of the Phase 2 trial, known as the ReCLAIM-2 Study, were announced by the company in early May 2022 and showed that the primary endpoints assessing change in low luminance visual acuity (LLVA) and geographic atrophy (GA) progression. Elamipretide did, however, showed that its use categorically improved visual function for patients with GA. And, what we believe is the most important development from the trial, was the announcement that elamipretide demonstrated proof of mechanism by reducing progressive ellipsoid zone loss, which is a key biomarker of retinal mitochondrial health and has been shown to be predictive of long-term GA growth and development. Further proof that elamipretide positively impact mitochondrial function gives us continued confidence that the drug can and will play an important role in the vital goal of improving mitochondria, the degradation of which is believed to be at the heart of many conditions.
Finally, an investment in Stealth ultimately, in our view, comes down to the belief in the leadership of the company and the processes and vision they have that plays a big part in whether MITO is a stock worthy of consideration. And it is in the talking with management and diving into their research process that makes us believe that Stealth is worth a look for investors that have a higher risk tolerance. Of course, there are risks, some of which are outlined below, but we believe there is upside potential as well. Stealth research is focused on a specific segment of the mitochondria—cardiolipin—which plays a vital role in many human functions, rather than the broader whole mitochondria itself, which we believe leads to a better possibility of success. From discussions with management and digesting statements and presentations they have given in the past, we came away impressed with the dedication and knowledge of the science displayed and believe that the team in place is a solid one to take Stealth to the next level.
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