Anavex Schizophrenia Drug Shows Safety Results in Phase 2 Trial

Author: Benzinga Newsdesk | October 02, 2025 06:32am

Anavex Life Sciences Corp. ("Anavex" or the "Company") (NASDAQ:AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative, neurodevelopmental, and neuropsychiatric disorders, today announced positive topline results from its placebo-controlled Phase 2 clinical study evaluating ANAVEX®3-71 for the treatment of schizophrenia in adults on stable antipsychotic medication (ANAVEX3-71-SZ-001, NCT06245213).

The study successfully achieved its primary endpoint, demonstrating that ANAVEX®3-71 was safe and well-tolerated. The safety profile was consistent with previous studies of ANAVEX®3-71 in healthy volunteers, with no serious treatment-emergent adverse events (TEAEs) and no severe TEAEs reported in either Part A or Part B of the study.

 

Overview of Adverse Events (Part A)
ANAVEX3-71 30mg (N=6)			ANAVEX3-71 60mg (N=6)			Placebo (N=4)			Total (N=16)
n (Participants)	%	n (Events)	n (Participants)	%	n (Events)	n (Participants)	%	n (Events)	n (Participants)	%	n (Events)
AE starting before study	1	16.7	1	0	0	0	0	0	0	1	6.3	1
AEs leading to death	0	0	0	0	0	0	0	0	0	0	0	0
Treatment emergent AE (TEAE)	1	16.7	1	0	0	0	0	0	0	1	6.3	1
Related TEAE	1	16.7	1	0	0	0	0	0	0	1	6.3	1
Serious TEAE	0	0	0	0	0	0	0	0	0	0	0	0
Serious related TEAE	0	0	0	0	0	0	0	0	0	0	0	0
Non-serious TEAE	1	16.7	1	0	0	0	0	0	0	1	6.3	1
Severe TEAEs	0	0	0	0	0	0	0	0	0	0	0	0
Related severe TEAEs	0	0	0	0	0	0	0	0	0	0	0	0
TEAE leading to death	0	0	0	0	0	0	0	0	0	0	0	0
TEAE leading to discontinuation of study drug	0	0	0	0	0	0	0	0	0	0	0	0
Overview of Adverse Events (Part B)
ANAVEX3-71 60mg (N=28)			Placebo (N=27)			Total (N=55)
n (Participants)	%	n (Events)	n (Participants)	%	n (Events)	n (Participants)	%	n (Events)
AE starting before study	1	3.6	1	0	0	0	1	1.8	1
AEs leading to death	0	0	0	0	0	0	0	0	0
Treatment emergent AE (TEAE)	11	39.3	16	13	48.1	21	24	43.6	37
Related TEAE	5	17.9	6	4	14.8	6	9	16.4	12
Serious TEAE	0	0	0	0	0	0	0	0	0
Serious related TEAE	0	0	0	0	0	0	0	0	0
Non-serious TEAE	11	39.3	16	13	48.1	21	24	43.6	37
Severe TEAEs	0	0	0	0	0	0	0	0	0
Related severe TEAEs	0	0	0	0	0	0	0	0	0
TEAE leading to death	0	0	0	0	0	0	0	0	0
TEAE leading to discontinuation of study drug	0	0	0	2	7.4	4	2	3.6	4
Key
n (Participants) = number of study participants with at least one event in the corresponding category
n (Events) = number of events in the corresponding category and treatment group
N = total number of study participants in the corresponding treatment group
% = (number of study participants with at least one event in the corresponding category/total number of patients in the corresponding treatment group)*100

In addition to meeting the primary safety endpoint, secondary and exploratory analyses revealed encouraging trends in several outcome measures. The study demonstrated positive trends in objective electroencephalography (EEG) and event-related potential (ERP) biomarkers of schizophrenia.

Furthermore, neuroinflammatory biomarker assessments showed that glial fibrillary acidic protein (GFAP), a marker of neuroinflammation, was reduced in participants receiving ANAVEX®3-71 compared to placebo. This reduction in neuroinflammatory markers suggests a potential disease-modifying effect that may become more pronounced with longer treatment durations. GFAP is a marker of astrocyte reactivity to neuronal injury and disease1 with known relevance to both neuropsychiatric2 and neurodegenerative3,4 disorders.

"We are encouraged that our ANAVEX3-71-SZ-001 study aligns with our expectations for safety and tolerability," said Juan Carlos Lopez-Talavera, MD, PhD, Head of Research and Development of Anavex. "We believe we are well-positioned to advance a competitive candidate into future studies aimed at addressing the ongoing and unmet medical needs of individuals living with schizophrenia and neurodegenerative diseases."

"We believe this study is a manifestation of Anavex's continued platform expansion aiming to provide potential beneficial effect for patients with oral compounds," said Christopher U Missling, PhD, President and Chief Executive Officer of Anavex. "The positive safety profile and encouraging biomarker trends support the continued development of ANAVEX®3-71 as a potential treatment for CNS disorders that could address underlying pathophysiology beyond symptomatic control."

ANAVEX®3-71 (formerly AF710B) is a dual SIGMAR1 receptor agonist and M1 positive allosteric modulator with agonistic effects.5,6 ANAVEX®3-71 has previously been studied in healthy volunteers prior to study ANAVEX3-71-SZ-001.7,8 This novel mechanism of action offers the potential to treat all symptom domains (positive, negative, and cognitive) of schizophrenia without the side effects of standard of care antipsychotics.

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