Spinogenix Announces Positive First Cohort Results from Phase 2a Trial Evaluating SPG302 for Alzheimer's Disease

SPG302, a Potential First-in-Class Synaptic Regenerative Therapy, Showed Progress in Cognitive Outcome Measures and a Favorable Safety Profile

LOS ANGELES, Calif., Aug. 7, 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 positive results from the first cohort of its Phase 2a trial in Australia, evaluating SPG302 for the treatment of Alzheimer's disease (AD). SPG302 is a once-daily pill that, by promoting synaptic regeneration, has the potential to reverse cognitive decline in people with mild to moderate AD.

The randomized, double-blind, placebo-controlled Phase 2 study of SPG302 (NCT06427668) assessed the safety, tolerability, and clinical efficacy of SPG302. Due to the unique mechanism of action targeting glutamatergic synapses, any adult a with mild-to-moderate AD diagnosis independent of amyloid status was eligible to enroll. Two dose cohorts completed 24 weeks of treatment (inclusive of a 4 week double-blind, placebo-controlled phase and an open label extension period). Clinical cognitive assessments of Mini-Mental State Examination (MMSE) and Clinical Dementia Rating Sum of Boxes (CDR-SB) were measured.

Results from the first cohort, presented at the recent Alzheimer's Association International Conference (AAIC), demonstrated:

• SPG302 was safe and well tolerated with no severe or treatment-related adverse events.
• Participants displayed evidence of rapid cognitive benefits, with a nearly 3-point increase in MMSE score within four weeks.
• Cognitive improvement was sustained throughout six months of open-label treatment.
• Participants in the open label extension also achieved durable improvements in CDR-SB between four-to-six months of treatment.
• SPG302 as a monotherapy or in combination with standard of care therapeutics was well tolerated by participants.

"The encouraging initial results from our Phase 2a trial reinforce SPG302's potential to improve care and bring new hope for patients and their loved ones suffering from Alzheimer's disease," said Dr. Stella Sarraf, Spinogenix CEO and Founder. "We look forward to sharing the complete data set from this trial at CTAD in December, and with our IND cleared in the U.S., continue our development of this first-in-class and potentially transformative treatment, to bring it to the millions of patients in the U.S.."

AD is the most common cause of dementia, accounting for 60-70% of cases worldwide. Loss of synapses occurs very early in the disease's progression and is a major driver of cognitive decline and memory loss. SPG302 offers the first synaptic regenerative approach to treating AD with the potential to improve cognition and quality of life. It represents a potential new class of regenerative therapeutics that can be used in combination with standard of care cholinesterase inhibitors, as well as recently approved antibody therapies targeting amyloid beta.

"There is a clear link between synapse loss and cognitive decline in Alzheimer's disease, yet the pursuit for an effective treatment has remained out of reach until now," said Bruce Brew, MD, DSc, FRACP, FAAN, neurologist and Principal Investigator at St. Vincent's Hospital in Sydney, Australia. "The results from this first cohort phase 2 study demonstrate the considerable potential of SPG302, with early efficacy results showing competitive improvements in both MMSE and CDR-SB. In addition, the cohort of participants who received SPG302 on top of standard-of-care therapies demonstrates the potential for SPG302 to be developed as either a monotherapy or in combination with other treatments, widening the possibilities for patients. Definitive phase 3 studies are being pursued."

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. 

Media Contact

Arielle Bernstein Pinsof
FINN Partners
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Investor Relations

Dan Albosta
Spinogenix, Inc.
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SOURCE Spinogenix