Findings From First Cohort of SPG302 in Phase 2 Alzheimer Disease Trial: Sharron Gargosky, PhD

WATCH TIME: 6 minutes

"I think the implications [of these findings with SPG302] for patients are incredible. This could be a standalone therapy, with its cognitive regenerative properties that allow patients to have regular daily lives and because it's a different mechanism of action, it can be additive to the existing standard of care."

SPG302 (Spinogenix) is a novel synaptogenic small molecule currently being investigated in a phase 2 trial for its safety and potential efficacy among adult patients with mild-to-moderate Alzheimer disease (AD) in Australia. In the first completed cohort of the study, findings showed that once-daily treatment with SPG302 was safe and well tolerated in patients with AD over the course of 28 weeks, revealing early signs of cognitive improvements. Coauthor Sharron Gargosky, PhD, and colleagues, presented these results at the recently concluded 2025 Alzheimer’s Association International Conference, held July 27-30, in Toronto, Canada.¹

The initial cohort of the phase 2 study randomized 12 participants with AD 2:1 to receive 300mg SPG302 or placebo daily as an oral tablet for a treatment period of 4 weeks, followed by open-label extension for an additional 24 weeks. Investigators assessed outcomes on a monthly basis, including standard cognitive assessments, quantitative neurophysiological measures sensitive to synaptic density, quality of life and activities of daily living measures, and other measures. Researchers observed improvements in Mini-Mental State Examination in most patients, with corresponding changes in other cognitive measures.

At AAIC 2025, Gargosky, the chief development officer at Spinogenix, spoke with NeurologyLive^® to provide more details on the company’s phase 2 trial assessing SPG302 in AD. During the interview, she shared that the oral therapy had a favorable safety profile in the first cohort consistent with prior findings observed in healthy volunteers, showing no treatment-emergent adverse events of concern. She also discussed the clinically meaningful cognitive improvements reported, with early effects apparent as soon as 1 week and sustained over a 24-week open-label extension. Overall, she highlighted that these results support further development of SPG302 both as a monotherapy and as an adjunctive therapy to standard of care in AD.

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REFERENCES
1. Priest L, Cayzer A, Brew B, et al. Early positive signals of cognitive outcomes in mild to moderate Alzheimer patients treated with the synaptic regenerative small molecule, SPG302. Presented at: 2025 Alzheimer’s Association International Conference; July 27-31; Toronto, Canada. Abstract 109023.