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LOS ANGELES, Oct. 16, 2025 /PRNewswire/ -- Spinogenix, Inc., a clinical-stage biopharmaceutical company pioneering first-in-class therapeutics that restore synapses to improve the lives of patients worldwide, today announced the publication of a peer-reviewed article in the Experimental Eye Research journal demonstrating neuroprotective effects of the clinical stage synaptic regenerative drug SPG302 in a rodent model of glaucoma. The preclinical results provide initial proof of concept that synaptic regeneration may offer a new approach to treating neurodegenerative diseases of the retina.Glaucoma is an optic neuropathy and the leading cause of irreversible vision loss worldwide, affecting around 80 million people. It is characterized by the loss of retinal ganglion cells (RGCs), the principal output neurons of the retina that give rise to axons of the optic nerve. In addition to optic nerve damage, glaucoma is associated with an early and progressive loss of glutamatergic synapses on RGCs that is thought to disrupt their function and contribute to glaucomatous degeneration. Currently, glaucoma is treated by reducing intraocular pressure (IOP), the only modifiable risk factor. However, many people with glaucoma have normal IOP and glaucoma can progress in some even when IOP is well controlled. Observations that dendritic and synaptic deficits in RGCs are among the earliest indicators of glaucomatous damage during increased IOP and in other contexts raise the prospect that synaptic regeneration may offer a new and broadly relevant therapeutic approach.
"The study results help validate the idea that retinal synapse loss is a critical step in glaucoma pathogenesis and they provide a foundation for positioning SPG302 in glaucoma and potentially other synaptopathies of the retina," said Stella Sarraf, Ph.D., Chief Executive Officer and Founder at Spinogenix. "We are looking forward to using these findings to advance to a clinical program and bring a new treatment modality to the millions suffering from glaucoma."
In the study, three-month-old mice were treated daily with SPG302 for eight weeks following microbead injection to induce elevated IOP. SPG302 administration significantly improved RGC survival and axonal integrity while preserving visual function as assessed by electroretinography, a clinically translatable measure of retinal function. This protective effect was linked to the preservation of retinal synapses as indicated by the normalized expression of synaptic markers.
"Research on glaucoma has been increasingly focused on identifying novel therapeutic strategies for protecting the optic nerve. The results obtained with SPG302 are encouraging as they suggest that protection of retinal ganglion cells can be achieved by reversing synapse loss with a rapid acting, clinical stage compound," said Dr. Robert N. Weinreb, an investigator of the study at University of California, San Diego and member of Spinogenix's scientific advisory board. "I am hopeful that SPG302 will provide a neuroprotective option in glaucoma and potentially other diseases of the retina."
SPG302 is a once-a-day pill being developed as a synaptic regenerative treatment for neurodegenerative and neuropsychiatric diseases. It is being assessed in Phase 2 trials for diseases including Alzheimer's disease, ALS and schizophrenia.
About SPG302
SPG302 is a once-a-day pill being developed as a regenerative treatment for neurodegenerative and neuropsychiatric diseases with the unique ability to restore synapses, the key connections between neurons that allow people to think, plan, remember, and control movement. The synaptic regenerative activity of SPG302 represents a first-in-class approach to treating these diseases and has the potential to reverse declines in cognitive, respiratory, and motor function. SPG302 is being evaluated as an investigational therapeutic in three disease indications: Alzheimer's disease (NCT06427668), ALS (NCT05882695) and Schizophrenia (NCT06442462).
About Spinogenix
Current treatments for neurodegenerative, neuropsychiatric and neurodevelopmental conditions primarily focus on slowing disease progression or minimizing symptoms, leaving many without hope for improvement. Spinogenix is aiming to transform the treatment of these conditions through its pioneering first-in-class and paradigm-shifting synaptic regenerative and synaptic corrective therapeutics designed to restore depleted synapses and reverse synaptic degeneration and dysfunction – offering patients and their families a new reality of hope.
Spinogenix is developing two novel therapeutics: SPG302, which triggers neurons to produce new glutamatergic synapses and restore cognitive, motor, and other functions in ALS, Alzheimer's disease, schizophrenia and other diseases; and SPG601, which works at the synaptic level to correct specific dysfunctions in Fragile X Syndrome (FXS) that underlie many core symptoms. The company has received FDA Orphan Drug and EMA designations for both ALS and FXS as well as FDA Fast Track designation for FXS. More information on Spinogenix can be found at www.spinogenix.com or follow us on LinkedIn.
