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Abstract Background

Oculogics plans to advance up to three next generation drug products to the clinic within two years of external financing or acquisition.  The critical path for each program runs through FDA regulatory requirements for clinical drug substance and drug product.  Each program may have its own funding or financial exit.

Chronic pain causes suffering and disabilities in patients struggling with cancer, serious injuries, diabetic neuropathy, arthritis and other painful diseases associated with aging and inflammation.  Current pain treatments are often too weak or too addictive.  Oculogics is developing a promising new oral treatment for chronic pain with a validated target and the potential to provide strong pain relief with no safety risks.     

A topical drug product can be advanced to an FDA IND application to treat dry eye disease with greater potency and efficacy than reproxalap.  With its high potency and drug safety, this has the potential to become a standard of care.

Up to Three Programs

1.  Chronic pain

2.  Dry eye

3.  Stargardt Disease

 Abca4 retinopathy 

associated with inflammation 

An oral drug product can be advanced to an FDA IND application to treat Stargardt disease, an orphan retinal degenerative disease of progressive blindness that is associated with the accumulation of toxic vitamin A dimers called A2E.  A2E accumulates abnormally in patients who have disease mutations in their Abca4 gene.  Its gene product is a membrane transporter that returns retinaldehyde, the aldehyde form of vitamin A, to the visual cycle if it escapes after its release from opsin in photoreceptors.  As A2E accumulates, it injures retinal pigment epithelial (RPE) cells next to the retina that provide essential metabolic support and triggers inflammation (1). 

In addition to Abca4, Stargardt disease is clinically associated with two complications of inflammation: 

  • Complement activation consistent with CFH  gene variants 

  • Lipid aldehyde toxicities consistent with elevated levels of lipid aldehyde adducts in donor eyes (2).  Lipid aldehydes amplify inflammation in a positive feedback loop (3).  

Together, RPE cell failure and chronic inflammation lead to regional cell losses in the retina (atrophic lesions) and corresponding losses in visual field (scotomas, blind spots).

Abstract Background

There are no approved treatments for Stargardt disease.  Its etiology is multi-factorial, not monogenic.  Most experimental treatments target CFH with gene therapy or protein inhibitors, or use visual cycle inhibitors to induce a local vitamin A deficiency in the eye, because A2E is a retinoid dimer and with less retinaldehyde, less A2E can form. 


Aldehyde traps use a novel mechanism to block A2E formation in Abca4  knockout mice (4) more effectively than visual cycle inhibitors and with much greater safety:

  • Traps have dual targets in Stargardt disease, by design:  lipid aldehydes that injure cells and amplify inflammation (5), and free retinaldehyde that forms A2E.  Visual cycle inhibitors have one target, proteins that regulate retinaldehyde delivery to the retina.

  • Traps have no adverse effects on vision because of their mechanism of action, as reviewed by the FDA (6).  Visual cycle inhibitors always cause night blindness and other visual losses in a dose dependent manner.  Their side effects do not limit their drug efficacy in mouse studies.  They do limit efficacy in patients because clinical doses are lowered to reduce safety risks and legal liabilities of the side effects.  No side effects, no efficacy, due to their mechanism of action.   

Why will aldehyde traps be better than visual cycle inhibitors?

  • Dual drug targets, by design

  • More effective in mice

  • No vitamin A deficiency

  • No side effects on vision

(1) Sparrow (2012), Prog Ret Eye Res 31:121; (2) Hu (2020), Redox Biol 37:101787 Fig 6B; see also Dhooge (2021), Redox Biol 45:101957; (3) Spite (2009), Br J Pharmacol 158:1062; (4) a validated mouse model of Stargardt disease invented by Dr. Gabriel Travis; (5) Spite (2009), Br J Pharmacol 158:1062; (6) Jordan (2009), FDA IND #104497; (7) Ahmad (2021), Aust Prescr 44:129

Convenient oral dosing

Drug delivery to the back of the eye is challenging because retinal drugs administered by eye drops have never shown clinical efficacy.  Intravitreal injections are unpopular with patients, associated with risks and not ideal  for an early onset genetic disease such as Stargardt disease. 


The higher potency, oral availability and retinal exposure of our next gen traps now make oral dosing feasible for this disease and other retinopathies associated with Abca4 and inflammation.  Many oral drugs are known to reach the retina, as shown functionally by the efficacy of oral steroids and antivirals in certain retinal indications and by the retinal adverse effects of other oral drugs for non-ocular indications (7). 

Which experimental treatment could be best for me?


  • A treatment with only one target may have limited benefits.

  • Combination therapy can be a wise treatment decision for a multi-factorial disease if both treatments are safe and have no adverse effects on vision. 

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