Tazbentetol, a First-in-Class, Oral Synaptic Regenerative Investigational Therapy, Demonstrated Favorable Safety Profile and Durable Therapeutic Benefits
Clinical Case Study Presented at AAIC Highlights Caregiver Report of Rapid Cognitive Improvement with Supporting Clinical Outcome Measures Consistent with Durable Treatment Benefit Through 18 Months
LOS ANGELES, July 14, 2026 /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 extended data from patients who continued treatment under a compassionate use Special Access Scheme (SAS) following its completed Phase 2a trial evaluating Tazbentetol (SPG302) for the treatment of Alzheimer’s disease (AD). Once-daily treatment with Tazbentetol, a first-in-class synaptic regenerative investigational therapy, initially demonstrated rapid improvement in multiple measures of cognition during placebo-controlled and open label extension periods of the Phase 2a trial and has shown sustained improvement for up to 84 weeks in patients participating in the SAS program.
Participants treated with tazbentetol under the SAS program had previously completed a Phase 2a study (NCT06427668) in Australia that assessed the safety, tolerability, and preliminary clinical efficacy of tazbentetol in 24 patients with mild-to-moderate AD. The trial consisted of a 4-week double-blind placebo-controlled period in which participants received 150mg or 300mg tazbentetol QD (or placebo), followed by an open label extension (OLE) period during which all participants received 300mg tazbentetol through 24 weeks.
Key findings from the Phase 2a trial were presented at CTAD 2025, including: favorable safety and tolerability, both as a monotherapy and in combination with standard-of-care medications (e.g. donepezil); rapid cognitive improvement with a > 2-point average increase in the Standardized Mini-Mental Status Examination (SMMSE) during the 4-week placebo-controlled period (p < 0.05) that was sustained through the 24-week OLE period; a rapid reduction (improvement) in scores on the Clinical Dementia Rating Scale Sum of Boxes (CDR-SB) scores, an FDA-acceptable endpoint to demonstrate efficacy, with a -0.81 point reduction over 24 weeks and continued improvement through longer term follow-up. At the conclusion of the trial, participants had the option to remain on treatment through a SAS for up to two years at 300mg QD and some participants have now completed 84 weeks of treatment.
Extended treatment results presented at AAIC 2026: At this year’s AAIC meeting, the Phase 2a data were presented alongside new observations on the durability of cognitive improvement over the SAS period in a case study highlighting correspondence between clinical outcome measures and caregiver-reported observations of cognitive improvement. Group data of SMMSE scores support continued treatment benefit beyond 1 year, including some patients remaining improved over baseline at 84 weeks (~19 months) of treatment.
“We are excited to share these extended data with the Alzheimer’s community, demonstrating the potential of tazbentetol as an efficacious and well-tolerated treatment,” said Dr. Stella Sarraf, Chief Executive Officer and Founder of Spinogenix. “This new data demonstrates the long-term durability of tazbentetol’s cognitive benefits and its potential.”
An illustrative case study from extended Phase 2a trial: Spinogenix presented a case study of a 72-year-old woman with mild AD presenting with short-term memory loss, anomia, and impaired social skills and etiquette. Within three months of treatment with 300mg tazbentetol, her caregiver reported that she was markedly improved, regaining the ability to read books, follow movies, and recall story lines. These observable improvements were clinically corroborated by rapid improvements in her scores on the SMMSE (a 4 to 6-point increase through 3 months) and CDR-SB (a 2.5 decrease through 3 months, with further reduction at 24 weeks) outcome measures. SMMSE scores showed continued improvement over baseline out to latest visit of 81 weeks.
“I look forward to supporting the company’s efforts in planning their next Alzheimer’s trial to advance a first-in-class synaptic regenerative therapy in the field,” said Steven Arnold, MD, Professor of Neurology at Harvard Medical School and member of the Spinogenix Scientific Advisory Board.
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.
Spinogenix is developing two novel therapeutics: Tazbentetol (SPG302), a synaptic regenerative therapy designed to restore cognitive, motor, and other functions in Alzheimer’s disease, ALS, schizophrenia and other diseases; and SPG601, a synaptic corrective therapy designed to work at the BK channel to normalize brain activity and help with core symptoms in Fragile X Syndrome (FXS). The company has received Orphan Drug designations from FDA and EMA for tazbentetol in ALS as well as FDA Orphan Drug and Fast Track designations for SPG601 in FXS. More information on Spinogenix can be found at www.spinogenix.com or follow us on LinkedIn.
Media Contact:
Daniel Davis
FINN Partners
daniel.davis@finnpartners.com
Investor Relations Contact:
Sasha Damouni Ellis
Spinogenix, Inc.
ir@spinogenix.com
SOURCE Spinogenix