Non-arteritic Anterior Ischemic Optic Neuropathy
Privosegtor, a novel peptoid small molecule that penetrates the blood brain and retinal barrier, has the potential to become a neuroprotective therapy for optic neuropathies and other neuro-ophthalmic diseases.
Non-arteritic Anterior Ischemic Optic Neuropathy is an acute optic nerve disorder with no approved therapy and permanent visual impairment in > 60% of patients1.
Non-arteritic Anterior Ischemic Optic Neuropathy (NAION) typically involves one eye, and approximately 20% of patients develop NAION in the second eye.2,3 Vision loss from NAION is usually sudden (rapid onset) and painless. It often looks like blurring or worsened vision across part of a patient’s field view (usually in the lower half) in the affected eye(s). Most people first notice they have vision loss when waking up (either from longer sleep periods or brief naps).4
Unfortunately, there’s no confirmed effective treatment for NAION and it can cause severe permanent visual impairment.
NAION is a condition affecting up to 10.2 per 100,000 people worldwide5,6, with a U.S. incidence estimated to be > 30,000.5,6 It is the most common cause of acute optic nerve injury in individuals over 50 years old.7
Unmet needs remain for therapies that can prevent or reduce vision loss due to NAION, and slow its progression.
Privosegtor
is a novel peptoid small molecule that penetrates the blood brain and retinal barrier, with the potential to be the first neuroprotective therapy for optic neuropathies and other neuro-ophthalmic diseases.
Privosegtor was selected by high-throughput screening (HTS) for neuroprotective properties, confirmed in vivo in glaucoma, multiple sclerosis, and acute optic neuritis models.
The data from in vitro studies also suggest that it modulates the FOXO3 pathway by interacting with the serum–glucocorticoid kinase (SGK) supporting neuronal survival and preservation.
Privosegtor has shown positive results in the prevention of retinal ganglion cell and axonal damage and was associated with improvements in clinical function (disability) in animal models of neuroinflammation and neurodegeneration.8
Most recently, Oculis announced positive results from the Phase 2 ACUITY trial, evaluating the safety, tolerability and efficacy of Privosegtor in patients with acute optic neuritis.
The trial met the primary endpoint for safety and achieved significance on several key efficacy-based secondary endpoints, showing functional vision improvement and neuroprotective anatomical and biological benefits in patients suffering from acute optic neuritis.
Furthermore, the results of the study show that in addition to the standard of care (IV methylprednisolone), Privosegtor was tolerated in participants with acute optic neuritis. The most frequently reported drug-related treatment-emergent adverse events (TEAEs) in the Privosegtor (2 or 3 mg/kg/day) treatment group were headache and acne, each reported in 2 participants (10.5%).
Privosegtor is an investigational drug and its safety or efficacy has not been established and it has not received regulatory approval for commercial use in any country.
Privosegtor was selected by high-throughput screening (HTS) for neuroprotective properties, confirmed in vivo in glaucoma, multiple sclerosis, and acute optic neuritis models.
The data from in vitro studies also suggest that it modulates the FOXO3 pathway by interacting with the serum–glucocorticoid kinase (SGK) supporting neuronal survival and preservation.
Privosegtor has shown positive results in the prevention of retinal ganglion cell and axonal damage and was associated with improvements in clinical function (disability) in animal models of neuroinflammation and neurodegeneration.8
Most recently, Oculis announced positive results from the Phase 2 ACUITY trial, evaluating the safety, tolerability and efficacy of Privosegtor in patients with acute optic neuritis.
The trial met the primary endpoint for safety and achieved significance on several key efficacy-based secondary endpoints, showing functional vision improvement and neuroprotective anatomical and biological benefits in patients suffering from acute optic neuritis.
Furthermore, the results of the study show that in addition to the standard of care (IV methylprednisolone), Privosegtor was tolerated in participants with acute optic neuritis. The most frequently reported drug-related treatment-emergent adverse events (TEAEs) in the Privosegtor (2 or 3 mg/kg/day) treatment group were headache and acne, each reported in 2 participants (10.5%).
Privosegtor is an investigational drug and its safety or efficacy has not been established and it has not received regulatory approval for commercial use in any country.
Visionary Innovation to Deliver Breakthrough Therapies
The positive ACUITY results represent a remarkable step forward in neuroprotection trials. I have worked in this area for more than three decades and what was truly novel in ACUITY was that the main functional and two structural results were so strong, and their independence making it highly unlikely that this occurred by chance. I believe that investigating Privosegtor as a neuroprotective agent in NAION is a logical next step, given the neuroprotective benefits observed in the ACUITY trial. NAION is a disease for which there is no approved therapy, the prognosis is almost always lifelong visual loss, and an effective therapy would have a tremendous impact on the lives of affected people.”
Privosegtor Phase 2 ACUITY Trial
