Optic Neuropathies and Retinal Neurodegenerative Diseases
- Optic neuropathies and retinal neurodegeneration diseases share common features because the cellular and molecular responses to neurodegeneration (oxidative stress, apoptosis) are closely similar
- Neuroprotective strategies lead to increased retinal neuronal survival and preserve visual function, and therefore can be used across multiple retinal diseases and optic neuropathies
- Neuroprotective drugs are intended to slow disease progression (disease modifying drugs) and be part of the therapeutics strategy in addition to other therapies targeting a specific cause of retina and optic nerve damage
Neurotrophic Factor Pathways
Neurotrophic factors (NTFs) are a family of neuro specific growth factors endogenously produced to promote cell proliferation, survival or regeneration.
In many retinal diseases and optic neuropathies, the release of these neurotrophic factors declines. The aim of neurotrophic factor-based therapy is to restore NFT levels and/or functions.
OCS-05 is a novel peptidomimetic small molecule with distinct mechanism of action (MoA) activating neurotrophic signaling pathways to protect and prevent damage to the optic nerve and retina. Its unique and differentiated MoA activates the Serum Glucocorticoid Kinase (SGK2), as part of the neurotrophic factor signalling pathways triggering multiple beneficial effects on apoptosis, anti-oxidation and anti inflammation.
What are the potential therapeutic translations?
Oculis’s intent is to demonstrate the neuroprotective and/or restorative benefits of OCS-05 in a first patient population (acute optic neuritis), as a proof-of-concept, and then explore additional ophthalmology indications which could benefit from neuroprotection.
OCS-05’s unique MoA could have applicability in several ophthalmology indications including acute optic neuritis, glaucoma, neurotrophic keratitis, geographic atrophy as well as other optic neuropathies.
Its applicability goes beyond ophthalmology and could potentially help address medical needs in other therapeutic areas such as in neurology.
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- Villoslada P, et al. Axonal and Myelin Neuroprotection by the Peptoid BN201 in Brain Inflammation. Neurotherapeutics. 2019 Jul;16(3):808-827.