Phase 2 Trial Open Enrollment for SPG302, First Synaptic Regenerative Approach to Treat Schizophrenia

Belight/AdobeStock

Spinogenix today announced open enrollment for its phase 2 trial of SPG302 for the treatment of individuals with schizophrenia, following clearance of its Investigational New Drug (IND) application by the US Food and Drug Administration (FDA). The US-based trial evaluating the efficacy, safety, and tolerability of once-daily dosing of SPG302 expands on trials underway in Australia.

“We are thrilled at the possibility of bringing an entirely new approach to treating schizophrenia for people battling this disorder in the United States. Synaptic regeneration may improve the standard of care in schizophrenia and potentially help those who do not benefit from current medications,” said Stella Sarraf, PhD, the CEO and founder of Spinogenix.

SPG302 is a once-a-day pill being developed as the first synaptic regenerative approach to treat schizophrenia with the potential to improve outcomes across all symptom domains. SPG302 represents a new, regenerative medicine approach to schizophrenia treatment that aims to restore glutamatergic synapses in regions of the brain affected by the disease, such as declines in cognitive, respiratory, and motor function. SPG302 has also been granted FDA Orphan Drug Designation for the treatment of amyotrophic lateral sclerosis, and has received preclinical support from the US National Institutes of Health and the Department of Defense.

Dopamine-targeting antipsychotics, the top treatment for schizophrenia for over 50 years, primarily address positive symptoms and do not address negative and cognitive symptoms. The FDA recently approved Cobenfy (KarXT), the first new agent with a novel mechanism of action for schizophrenia more than 50 years.² This marks progress in filling these gaps but does not address a critical aspect of schizophrenia pathogenesis: glutamatergic synapse loss, which may be a major contributor to all symptom areas and is thought to be driven by both genetic and environmental risk factors.

“To date, no treatments are approved to address negative symptoms and cognitive dysfunction associated with schizophrenia,” said Christoph Correll, MD, professor of Psychiatry and Molecular Medicine at the Donald and Barbara Zucker School of Medicine at Hofstra/Northwell and a leading researcher on the cognitive and negative symptoms of schizophrenia. “Since a loss of glutamatergic synapses in the frontal cortex and in other key regions may be crucial to the pathophysiology of schizophrenia, there is a potential for SPG302 to improve positive, negative, as well as cognitive symptoms in people living with schizophrenia. The reparative aspect of neuronal regeneration may lend itself to early illness interventions as well as to interventions for people with treatment-resistant schizophrenia for whom currently only clozapine is approved.”

References

1. Spinogenix announces open enrollment for phase 2 US trial following FDA clearance of IND application for SPG302, the first synaptic regenerative therapy for schizophrenia. News release. March 31, 2025. https://www.prnewswire.com/il/news-releases/spinogenix-announces-open-enrollment-for-phase-2-us-trial-following-fda-clearance-of-ind-application-for-spg302-the-first-synaptic-regenerative-therapy-for-schizophrenia-302415491.html

2. Duerr HA. FDA approves Cobenfy, a first in-class agent for schizophrenia. Psychiatric Times. September 26, 2024. https://www.psychiatrictimes.com/view/fda-approves-cobenfy-for-schizophrenia