Retrotope Starts IND Enabling Studies for its Second Deuterated Polyunsaturated Fatty Acid (D-PUFA) Drug Candidate, Targeting the Treatment of AMD and Related Retinal Pathologies.

LOS ALTOS, CA / ACCESSWIRE / July 21, 2020 / Retrotope announced today that it has entered into preclinical development of its second D-PUFA drug candidate, RT011, a deuterium-stabilized version of docosahexaenoic acid (DHA) to be targeted at degenerative eye diseases, such as age-related macular degeneration (AMD).

“We are excited about this compelling opportunity. Our first D-PUFA drug candidate in development, RT001, has shown robust safety in over 50 patient years of oral dosing, and we expect RT011 to have a similar safety profile. We are delighted to offer an entirely novel mechanism of action (MOA) for this difficult to treat category of diseases.” commented Robert Molinari, Ph.D. CEO of Retrotope.

Karsten Schmidt, Ph.D., head of Retrotope’s retinal drug development efforts, adds: “Photoreceptors contain 15 times more lipids than average cells, and almost half of it consists of DHA, critical for visual function but also the most oxidation-prone fatty acid in human tissues. Toxic byproducts from DHA oxidation are major drivers of AMD and multiple other retinal atrophies. Deuterium stabilization confers oxidation-resistance without changing DHA’s essential functional properties. We are very encouraged by initial animal data that confirm effective RT011 incorporation into all retinal phospholipids leading to the reduction of lipid peroxidation in vivo.”

About AMD:

AMD is a retinal degenerative disease leading to loss of central vision which affects some 200 million people globally, with more than 10 million subjects with AMD in the US alone. The prevalence of AMD is constantly increasing with lengthening lifespan. According to an economic impact study by AMD Alliance International, direct health care system costs are approaching $300 billion globally in 2020. While the advanced wet stage of the disease can be controlled by intravitreal injections of anti-VEGF drugs, there is no approved treatment for prevention or arrest of advanced dry AMD, or its advanced stage known as geographic atrophy (GA). Central GA leads to irreversible vision loss which deteriorates quality of life, and goes along with increased rates of fractures, depression and cognitive decline. Vision impairment starts many years earlier with difficulties to see under dim light or changing light conditions, such as driving at night. Hence, a safe and effective oral drug that could treat it in its earlier phases before progression to advanced stages and vision loss is urgently needed.

About RT011

RT011 is a patented, orally dosed deuterium-stabilized form of the conditionally essential omega 3 fatty acid DHA, that confers resistance to membrane lipid peroxidation. DHA in the eye is extremely susceptible to oxidative attack by reactive oxygen species that are created during chronic exposure to sunlight and indoor blue light. These effect are amplified several fold by cigarette smoking. Toxic DHA degradation products in the eye contribute to formation of lipid deposits known as drusen, which are a hallmark of early AMD. As the disease progresses, DHA-derived oxidation-specific epitopes on cell surfaces lead to chronic complement activation, sterile inflammation, mitochondrial dysfunction, senescence and finally death of photoreceptors and the supporting retinal pigment epithelium. The MOA of RT011 suspends this vicious cycle of collateral damage. It has excellent oral bioavailability since it is absorbed and concentrated in the eye like normal DHA, a challenge that virtually all other drug candidates have failed to surmount.

About Retrotope

Retrotope, a privately held, clinical-stage pharmaceutical company, is creating a new category of drugs to treat degenerative diseases. Composed of proprietary compounds that are chemically stabilized forms of essential nutrients, these compounds are being studied as disease-modifying therapies for many intractable diseases. Retrotope’s first lead candidate RT001, is being tested in clinical trials for i) the treatment of Friedreich’s ataxia, a fatal orphan disease; ii) a fatal, childhood neurodegenerative disease called Infantile Neuroaxonal Dystrophy, and iii) in Progressive SupraNuclear Palsy (PSP) which is also fatal. RT011 will be the second drug candidate entering development using this novel approach.

For more information about Retrotope, please visit

Retrotope, Inc.
4300 El Camino Real, Suite 201
Los Altos, CA 94022


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Robert J Molinari, Ph.D.
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SOURCE: Retrotope, Inc.

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