Alkeus and Columbia University enter license agreement for potential therapies for dry-AMD, Stargardt disease

Alkeus Pharmaceuticals and Columbia University announced today that they have entered into a license agreement for a set of potential therapies for the treatment of dry age-related macular degeneration (dry-AMD), Stargardt disease, and other degenerative diseases of the eye. Left untreated, these conditions often lead to impaired vision and even blindness.

Dr. Ilyas Washington, inventor of the technology and the Michael Jaharis Assistant Professor in the Department of Ophthalmology at Columbia University Medical Center, has found in preclinical testing that the compounds can reduce accumulation of certain toxic pigments in the eye. These pigments, which accumulate with age, are thought to be partially responsible for the vision loss associated with dry-AMD and Stargardt disease. Other conditions that can lead to impaired vision and are also associated with the accumulation of vitamin A aggregates may also be addressed, including Best's disease and certain forms of retinitis pigmentosa and of cone rod dystrophy.

"We have developed a modified vitamin A with the hope of halting vision loss or even restoring ocular function," Dr. Washington says. "Humans have evolved to make use of natural micronutrients or vitamins that regulate a wide variety of physiological functions. By altering the chemical structure of these vitamins, we can potentially regulate or enhance the biological processes that they control."

Dr. Leonide Saad, Chief Executive Officer of Alkeus, explained, "These vitamin A aggregates accumulate in dry-AMD and, to an even faster extent, in Stargardt disease due to a genetic defect that exacerbates this process. Ilyas has elegantly shown, in a mouse model of Stargardt, that slightly changing vitamin A significantly reduces the rate of formation of these aggregates, eventually preserving visual function.

"While these results are at the preclinical stage, the way vitamin A interacts with the human body is very well understood and gives us confidence of the low risk and the high potential to tackle these serious ocular diseases," said Dr. Saad.

Other therapeutic compositions have been evaluated to reduce the accumulation of these vitamin A aggregates employing more invasive techniques, but the compounds developed by Dr. Washington rely on a novel and innocuous mechanism of action that strengthens the specific chemical bonds that have to be broken in order to form these aggregates.

"The problem with other approaches is that they affect the way vitamin A is processed in the eye, which often results in visual side effects such as slowed dark adaptation, hallucinations and night blindness," commented Dr. Saad.

"We are very pleased to be working with Alkeus for the continued development of these therapies. In Alkeus, we believe we have found the best partner to efficiently and effectively drive commercialization of these therapies," said Donna See, who oversees portfolio strategy and marketing for Columbia Technology Ventures, the technology transfer office of Columbia University. "As yet, there is no therapy to address impaired vision resulting from dry-AMD, a condition which affects millions of people around the world. Perhaps even more urgent is a therapy for the children who are affected by Stargardt. We look forward to hopefully one day being able to offer a way to help these patients and their families."

SOURCE Alkeus Pharmaceuticals and Columbia University

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