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May 27, 2020

Abfero Targeting Excess Iron to Treat AMD, Other Retinal Diseases

An intriguing link between excess body iron and retinal degeneration is being explored by Abfero Pharmaceuticals, a young biotech based in Boston and Oxford, UK.

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An intriguing link between excess body iron and retinal degeneration is being explored by Abfero Pharmaceuticals, a young biotech based in Boston and Oxford, UK.  Abfero Pharmaceuticals (www.abfero.com), a supporter of the Foundation Fighting Blindness, is a US- and UK-based company focused on treating diseases associated with excessive iron in tissues, including the retina.

“Iron is essential for cellular function, but excessive iron can be toxic,” explains Thomas Neenan, Founder and CEO of Abfero. “In particular, so-called free iron or iron not bound to the transport protein transferrin or stored safely as ferritin in cells can catalyse the creation of damaging molecules called reactive oxygen species that can drive damage in multiple organs, including the retina.  We have been working for many years with our inventor, Professor Ray Bergeron of the University of Florida to develop new molecules that can selectively remove free iron from the body and shut down these damaging pathways.  Most of our clinical work to date has been in diseases of iron overload caused by needed blood transfusions, but we have become very interested in the role of iron in retinal diseases and believe that we could bring a fresh set of therapies to this clinical area.”

Abfero has been working with Dr. Joshua Dunaief, Adele Niessen Professor of Ophthalmology at the University of Pennsylvania’s Scheie Eye Institute and a leading expert on retinal diseases and the role of iron in retinal degeneration. His work, and that of others in the retinal community, have shown that iron overload in the retina can cause retinal degeneration and that retinal free iron worsens disease severity in a variety of disease models.

Post mortem age-related macular degeneration (AMD) eyes have increased levels of iron in retina compared to age-matched healthy donors. Iron accumulation in AMD is likely to result, in part, from inflammation, hypoxia, and oxidative stress, all of which can cause iron dysregulation,” says Professor Dunaief. “We and others have shown over the years that iron chelators can be retina protective in a wide variety of mouse models.”   

Abfero’s lead product is SP-420, an orally available small molecule that binds free iron.  The resulting complex is excreted by the body through the liver.  SP-420 has completed three safety trials to date in normal subjects and in patients with b-thalassemia, a genetic condition that requires patients to undergo frequent blood transfusions.  Abfero has held preliminary discussions with FDA and is in discussions with the University of Pennsylvania to test SP-420 as a treatment for AMD.   

“We are obviously attracted to the Scheie Institute because of the involvement of Professor Dunaief and the depth of experienced clinical investigators including Dr. Benjamin Kim, and expert biostatistician and AMD clinical trialist Dr. Maureen Maguire who leads the core imaging center for ophthalmology trials,” says Dr. Neenan.

The role of iron in retinal diseases remains to be proven but Abfero sees great potential in applying their technology to AMD and beyond that into other retinal diseases such as retinitis pigmentosa.    

“Iron chelator research has progressed tremendously over the past decades,” says Dr. Neenan.  “Chemists such as Professor Bergeron have developed molecules with extremely high specificity for finding and removing iron from specific tissues.  We see retinal disease as an area of great potential for our science and look forward to building on our relationship with the Foundation and the dedicated researchers in this field”.

You can find further information by contacting Abfero at tneenan@abfero.com.