April 21, 2021
AMD (age related macular degeneration) results from damage to the macula, the part of the eye responsible for central vision and the ability to see clearly. Most cases of macular degeneration occur as part of the aging process. AMD is a progressive retinal disease, usually occurring at age 55 years or older. There are two forms of AMD – dry and wet. All cases begin as the dry form, but 10 to 15 percent progress to the wet form, which can result in sudden and severe central vision loss.
Wet AMD accounts for as much as 90 percent of all AMD-related blindness and is thought to begin when blood vessels form abnormally at the back of the eye through a process called angiogenesis. The blood vessels leak blood or fluid in the macula and form scars that cause central vision to deteriorate and may result in permanent blind spots.
About 200,000 new cases of wet AMD are diagnosed each year in North America due to the aging baby boomer population. There are probably 3 million patients already in North America alone.
There are two widely used FDA approved “treatments” for wet AMD; Lucentis, from Roche/Genentech and Eylea from Regeneron. Eylea is by far the most prescribed. Both are injected directly into the eye(s) of patients on a regular basis to maintain effectiveness. Both drugs basically work by blocking the action of VEGF (vascular endothelial growth factor) whose job is to vascularize (make new blood vessels) in the eye. When that process goes bad, vision is seriously impaired. You can think of these treatments as a sort of temporary patch that fixes leaky vessels and stops new leaks from occurring. On its web site Lucentis describes the possible benefit to patients as “maintaining” their vision. Maintained is defined as losing less than 15 letters or 3 lines on the eye chart after 1 year. Combined, the drugs are multi-billion dollar a year products.
If you are reading this blog then you probably know that Curative has licensed from the NEI (National Eye Institute) what we believe will be a next generation treatment for degenerative eye diseases without the necessity of eye injections. We have licensed a discovery which allows for metformin, one of the most prescribed drugs in the world, to be reformulated into an eye drop for daily use to actually change the conditions in the eye which allow for the angiogenesis which is at the heart of the problems created by wet AMD. We have already started working on our reformulation, and expect to file an IND with the FDA to begin human trials before the end of the year. If you follow us then you also know that Dr. Kapil Bharti, the lead inventor on all of the licensed patents (pending) from NEI at NIH, has joined our Scientific and Clinical Advisory Board to help guide the program. His expertise and insights are invaluable.
Wet AMD is not an orphan or rare disease. And while our initial focus for this product was the rare disease Stargardt’s, we have decided to move forward with wet AMD as our first indication. We still intend to use the drug to treat Stargardt’s patients. A good deal of our non-clinical and clinical work on wet AMD should be applicable for our eventual filing to treat Stargardt’s if the wet AMD drug receives marketing approval from the FDA and/or other national regulatory bodies. But our Advisory Board and Management came to the conclusion that because of the much larger number of patients it will be much easier/quicker/less expensive to recruit the human trials and move towards FDA approval in Wet AMD. Because Metformin is already an approved drug with decades of safety data, we may be eligible for the 505B2 pathway at the FDA, specifically created for new formulations and/or routes of administration of approved drugs. Of course here, in addition to a reformulation and a new route of administration (eye drop instead of pill) we are also proposing use for a new indication. Currently metformin is prescribed to help treat diabetes.
So I thought it was important to explain to our shareholders that while we Are focused on rare or orphan diseases, often our drugs will also be capable of treating disease in larger patient populations. When we do start a program on one of those larger indications, we will always look to leverage all of the work to be able to circle back and treat the orphan disease also. Wet AMD to Stargardt’s is a perfect example and also a good example of when starting big is the right decision.