Spinogenix Announces Open Enrollment for Phase 2 US Trial Following FDA Clearance of IND Application for SPG302, the First Synaptic Regenerative Therapy for Schizophrenia

LOS ANGELES, March 31, 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 open enrollment for its Phase 2 trial of SPG302 for the treatment of people with schizophrenia, following clearance of its Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA). The U.S.-based trial evaluating the efficacy, safety, and tolerability of once-daily dosing of SPG302 expands on trials underway in Australia.

Spinogenix CEO and Founder Dr. Stella Sarraf stated, "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."

Approximately 24 million people worldwide suffer from schizophrenia, a complex and highly debilitating neuropsychiatric disorder. SPG302 is 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.

Schizophrenia is characterized by a trio of symptom domains: positive (e.g. hallucinations, delusions), negative (e.g. social withdrawal, anhedonia) and cognitive (e.g. memory and language deficits). While dopamine-targeting antipsychotics have been the mainstay treatment for over 50 years, they primarily address positive symptoms, leaving negative and cognitive symptoms largely untreated. The recent FDA approval of COBENFY™, the first of a new class of non-dopaminergic antipsychotics, 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." Correll also serves as Professor of Child and Adolescent Psychiatry, Charité Universitätsmedizin in Berlin. 

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 has been granted U.S. FDA IND clearance for the treatment of schizophrenia, FDA Orphan Drug Designation for the treatment of ALS, and has received preclinical support from the U.S. National Institutes of Health and the Department of Defense.

Spinogenix is conducting a randomized, double-blind, placebo-controlled Phase 2 study of SPG302 (NCT06442462) to assess the efficacy, safety, and tolerability in adults with schizophrenia in the United States and Australia. Additional information on the clinical trials evaluating SPG302 for the treatment of ALS and Alzheimer's disease can be found on ClinicalTrials.gov (NCT05882695 and NCT06427668).

About Schizophrenia
Schizophrenia is a highly prevalent (0.5-1% of the population world-wide) and chronic psychiatric disorder associated with psychosis and a decline in daily functioning. Individuals with schizophrenia present with both positive and negative symptoms, as well as cognitive deficits. Together with co-morbid cognitive deficits, these symptoms have a major impact on the overall quality of life. Despite the availability of many drug therapies, psychosis in many patients remains inadequately controlled and both cognitive and negative symptoms are largely unaddressed.

About Spinogenix

Spinogenix is pioneering first-in-class and paradigm-shifting therapeutics that regenerate brain connections (synapses), moving beyond slowing disease progression to reverse the effects of disease and improve patients' lives worldwide. Spinogenix small molecule-based therapies aim to restore synapses in neurodegenerative, neuropsychiatric and neurodevelopmental conditions including amyotrophic lateral sclerosis (ALS), Alzheimer's disease, schizophrenia, Fragile X syndrome (FXS) and others. Spinogenix has received a U.S. FDA Orphan Drug designation for the treatment of both ALS and FXS, as well as U.S. FDA Fast Track designation for FXS. More information on Spinogenix can be found at www.spinogenix.com or follow us on LinkedIn. 

Contact

Nechama Rosengarten
FINN Partners
[email protected]

SOURCE Spinogenix