Next gen aldehyde traps are at preclinical stage with a novel validated lead and a series of patentably distinct analogs.  The entire drug series has freedom to operate and strong IP with composition of matter and methods of use claims.  

Platform drugs have multiple options for clinical development, by definition.  Depending on the priorities of corporate partners or investors, Oculogics can advance lead compounds to the clinic in oral or topical formulations within two years of M&A, licensing or equity funding.  Each clinical program can have its own financial exit with exclusive rights to one or more proprietary drugs in this series.  

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Diseases associated with chronic inflammation cause lifetime disabilities and over 50% of global deaths (1).  As an inflammation platform drug, aldehyde traps have the potential to become a safe and potent standard of care in any of these diseases.  These include cardiovascular disease, type 2 diabetes and metabolic syndrome, chronic kidney disease, all forms of neurodegenerative disease, and orphan diseases associated with gene defects in reducing enzymes or aldehyde transporters.  

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Abca4 retinopathies such as dry AMD and Stargardt disease are a special case where aldehyde traps have an additional target:  toxic vitamin A dimers called A2E that trigger chronic inflammation (2-4).     As A2E accumulates, it causes recurring injuries in retinal pigment epithelial (RPE) cells that provide essential metabolic support to retinal neurons. Systemic treatment with aldehyde traps have preclinical proof of concept in Abca4 knockout mice, an industry standard mouse model where aldehyde traps reduced A2E formation by over 70% (5).  The same traps also deactivate lipid and oxysterol aldehyde toxins, which injure cells by multiple known mechanisms and trigger complement activation that causes retinal lesions in late stage disease (6).  The high oral availability and CNS exposure of next gen traps supports safe oral dosing instead of repeated ocular injections that trigger multiple injury responses in the eye.

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​Many major diseases are now known to have multiple risk factors (“multifactorial diseases”  [7]).  These risk factors can include the patient's age, heredity, medical history, lifestyle and diet.  Even diseases once thought to be caused by a single gene defect are now understood to involve other genes that alter disease progression by complex gene network interactions.  Clinical studies have shown that in many such diseases, replacing one gene defect, or inhibiting one disease pathway (such as complement activation in late stage AMD), can slow disease progression but not stop it.  Patients with a multifactorial disease can benefit most from combination therapy, where they are treated with two or more drugs that target different disease pathways that are active in their disease.  In every disease associated with chronic inflammation, aldehyde trap therapy is well suited for combination therapy by its high potency in blocking the “collateral damage” of aldehyde toxins to cells in inflamed tissues and organs with high drug safety.   

Stage, FTO and IP