Synthetic 9-cis ß-carotene for treatment of retinal degeneration

Unmet Need & Background

• Retinal degeneration, including age-related macular degeneration (AMD) and Retinitis pigmentosa (RP), are a leading cause of incurable vision incapacitation and blindness worldwide with no effective treatment. Hence there is an urgent need to develop an efficient treatment for these degenerative diseases.
• Recent clinical and pre-clinical studies support the potential of using retinal pro-drugs for treatment of AMD, RP and Usher syndrome
• We demonstrated in published clinical trials that oral treatment with Dunaliella Bardawil algae extraction, dramatically improved visual function in patients with various indications of retinal degeneration with no safety concerns (3 years follow-up).
• We proved that 9-cis β-carotene (9CBC), a 9-cis retinal pro-drug, is the bioactive API in algae extract.

The Problem

Despite the success of our pre-clinical and clinical trials, the Dunaliella Bardawil treatment is not a viable treatment option, as algae 9CBC content in the last decades has dramatically decreased, and is far below therapeutic levels.

Growing Dunaliella rich in 9CBC and low in all-trans BC is not commercially feasible in terms of quantity and quality, stressing the need for manufacturing pure synthetic 9CBC that will be readily available for treatment, and enable tight control of drug dosage.

Our Solution

• Synthetic 9CBC synthesized through a novel and cost-effective synthetic route.
• Synthetic 9CBC rescues the survival of cone photoreceptors at micro-molar concentrations in mouse retina cultures.

Synthetic 9CBC Advantages

• High drug purity & controlled stability
• Reproducible and consistent synthesis
• Designed for oral treatment for enhanced efficacy and improved patient’s compliance
• Enables controlled dosage
• Supported by extensive preclinical and clinical efficacy and safety data from the alga studies
• No complications of high intracranial pressure (observed when treating with retinal or 9-cis-Retinal analogs).

Patents

• A patent was filed of the proprietary synthetic route of synthetic 9-cis β-carotene and its formulation: "9- cis-β-carotene synthesis and formulations thereof" (May 21, 2017, WO 2017/20351 6).
• The patent application was filed in US and is still pending. US application number 16/303,604

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