Age-related Macular Degeneration (AMD): The Primary Disease Target

Macregen’s scientific breakthroughs have led to a new, compelling, and comprehensive understanding of the underlying causes of AMD, allowing the Company to develop innovative therapies to prevent and treat this progressive vision-ravaging disease.



What is the macula?

The retina is a thin sheet of neurons that sends signals about vision to the brain.  The macula is the part of the retina used for detailed vision like reading, recognizing faces, and driving. Behind the retina is the choroid, a bed of blood vessels. The inner surface of the choroid is where AMD pathology is most prominent.

What is AMD?

AMD robs people of central vision and is caused by gradual accumulation of lipid deposits under the macula.

There are two types of AMD:

  • "Dry" form or early and intermediate AMD as determined by the presence of lipid deposits under the macula. The main component of these deposits are lipids more commonly known as drusen.

  • Late AMD which include a “dry” form known as Geographic Atrophy (or “GA”) and a “wet” or neovascular form (nAMD). Without therapeutic intervention, both types of late stage AMD usually results in severe loss of vision.

For many patients with nAMD, there is a well-established treatment involving injections of anti-vascular endothelial growth factor (anti-VEGF) directly into the vitreous of the eye.

There are currently no effective or approved pharmacologic treatments for "dry" form or early and intermediate AMD. However, if there were drugs to slow or prevent this precursor, they could delay or prevent the development of all forms of AMD, including “dry” and “wet”  advanced AMD.


The Unmet Need

AMD is the leading cause of blindness in the developed world.  Its prevalence is increasing dramatically as the population ages. It is estimated that by 2020, there will be about 200 million people worldwide with the condition.

Patients with "dry" AMD comprise more than 90% of the AMD population. There is currently no approved treatment for "dry" AMD.

nAMD affects about 10% of the patient population and causes severe vision loss.  It is currently treatable for most patients with injections of anti-VEGFs directly into the eye.

Intravitreal injections of anti-VEGF drug, while effective for many patients, are burdensome to patients, families, and practitioners, making alternative methods of drug delivery attractive and less costly.

"Dry" AMD Patient Eye
Dry AMD patient after treatment with Mitia(TM) - desired outcome
Normal eye with no age-related changes



Mitia™ – Macregen’s biologically-based, targeted drug candidate for AMD

This first-in-class drug candidate works by removing and/or clearing accumulated lipids under the macula which are the main component of drusen deposits typically observed in patients with "dry" AMD.

Mitia™ is being developed as a disease-modifying drug that will remove the lipids that have accumulated under the macula, thereby treating the underlying cause of AMD.

This therapeutic approach is based on the similarities between the etiologic causes of AMD and another lipid and inflammation-based disease – atherosclerosis. This seismic scientific observation has been validated by compelling evidence obtained by the comparative examination of hundreds of donor eyes from people with AMD and from age-matched controls.

This work was led by Christine Curcio, PhD, Chief Scientific Officer for Macregen, and has resulted in a comprehensive understanding of the anatomical and biochemical changes in AMD, as well as the desired mode of action for a successful drug therapy.

It is envisioned that Mitia™ can be used in combination with anti-VEGF drugs that are designed to treat nAMD. Thus, both the removal of lipid deposits and the inhibition of neovascular complications may be accomplished concurrently or sequentially.

Learn more about Macregen's vision and strategy