Drexel University

Findings underpin the scientific rationale for Anavex’s targeted autophagy approach with orally administered blarcamesine Convergence of impaired autophagy and synaptic dysfunction across neurodevelopmental and neurodegenerative conditions aligns with blarcamesine’s mechanism of action These findings support Anavex’s intent to advance blarcamesine into pivotal clinical studies to further evaluate its potential in addressing unexpectedly common CNS disease mechanisms NEW YORK, April 14, 2026 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company focused on developing innovative treatments for Alzheimer’s disease, Parkinson’s disease, schizophrenia, neurodevelopmental, neurodegenerative, and rare diseases, including Rett syndrome, and other central nervous system (CNS) disorders, today announced new findings on the shared biology between autism spectrum disorder (ASD) and Alzheimer’s disease (AD), a core area of Anavex’ development plans with its autophagy enhancing orally administered blarcamesine. Key Highlights Multiple peer-reviewed publications point to biological link between autism spectrum disorder (ASD) and Alzheimer’s disease (AD), including shared disruptions in autophagy. Epidemiological data show that autistic adults may be diagnosed with Alzheimer’s and related dementias at rates up to 8 times higher than the general population, with onset occurring years or decades earlier than typical. Converging human genetic evidence links numerous high-confidence ASD risk genes — including TSC1/TSC2, PTEN, SHANK3, and FMRP — to impaired cellular autophagy, establishing autophagy dysfunction as a shared molecular substrate across genetically diverse forms of ASD. Synaptic dysfunction in ASD is now understood to arise, in substantial part, from a failure of autophagy-dependent synaptic pruning — causing an excess of poorly regulated synaptic connections and disrupted excitatory–inhibitory balance in neural circuits. The brain’s extracellular matrix (ECM) is pathologically altered in ASD and is bidirectionally coupled to autophagy. Restoration of autophagy impairment, now emerging as a central shared pathway in both ASD and AD, is precisely the biological system targeted by blarcamesine through its activation of SIGMAR1. Blarcamesine has demonstrated restoration of autophagy through SIGMAR1 activation in preclinical models and has shown clinical effects in Phase IIb/III trials in early Alzheimer’s disease, Phase II/III in Rett syndrome (a neurodevelopmental disorder caused by MECP2 mutation), and Phase II in Parkinson’s disease dementia. Collectively, these data provide a scientific basis for advancing blarcamesine into pivotal clinical studies, subject to further evaluation and regulatory considerations. Reframing Brain Disorders: Converging Pathways in Neurodevelopment and Neurodegeneration For decades, autism and Alzheimer’s disease were treated as conditions on opposite ends of the lifespan — one affecting brain development in early childhood, the other driving decline in old age. New research is adding a critical new twist. A landmark April 2025 study published in JAMA analyzed Medicare and Medicaid records covering more than 114,000 autistic adults and found that dementia prevalence among this population was dramatically elevated compared to the general population.¹ A separate recent 2026 paper in Frontiers in Neuroscience identified convergent disruptions in two critical systems shared by both conditions: The autophagy network and the synaptic regulation machinery.² Autophagy is the cell’s natural process for clearing misfolded proteins, damaged organelles, lipids, and other cellular waste. In autism, excess synaptic connections form which are not properly pruned during development. In Alzheimer’s disease, impaired autophagy, worsened by ApoE4 lipoproteins, allows toxic protein aggregates — including amyloid-beta and fibrillary tau — to accumulate unchecked. Both conditions, in essence, share a common driver of disease pathogenesis: A failure of the brain’s housekeeping system. Synaptic Dysfunction in ASD: When the Brain’s Pruning Mechanism Fails The human brain is sculpted by a process of exuberant synapse formation followed by selective elimination — synaptic pruning — that removes excess connections and renders neural circuits fully functional. Autophagy is a core cellular mechanism enabling this pruning. A landmark Neuron study³ found excess dendritic spines in postmortem ASD cortical tissue compared to controls — direct evidence of failed pruning correlating with impaired autophagy. Blocking neural autophagy genetically reproduced core ASD features: Excess synapse density, impaired social behavior, and repetitive behaviors; restoring autophagy normalized both synaptic architecture and behavior. Microglia, the brain’s resident immune cells, depend equally on autophagy for synapse elimination.⁴ The downstream consequence of both failures is a disruption of excitatory–inhibitory balance — a core pathobiological signature of ASD.⁵ , ⁶ The Genetic Architecture of ASD Converges on Autophagy ASD is genetically heterogeneous, yet genome analyses repeatedly converge on a common theme: Mutations and copy-number variants in genes whose protein products regulate autophagy. Among the most studied are mutations in TSC1/TSC2 and PTEN genes, whose loss of function suppresses autophagy and is associated with high rates of ASD alongside epilepsy and intellectual disability.⁷ Fragile-X syndrome — the most common inherited cause of intellectual disability and autism — likewise involves reduced autophagic flux in hippocampal neurons, with activation of autophagy rescuing aberrant spine morphology, synaptic plasticity, and cognition in preclinical models.⁵ SHANK3 mutations further alter autophagy-dependent protein homeostasis at the synapse.⁸ Whole-exome sequencing has additionally identified copy-number variants in core autophagy genes in sporadic ASD cases.⁹ This genetic convergence is not coincidental; it is pathobiologically instructive. The Brain’s Extracellular Matrix and Autophagy: A Bidirectional Relationship A third dimension of ASD biology is receiving growing attention: The extracellular matrix (ECM) — the structural scaffold of proteins and proteoglycans surrounding all brain cells. Its most specialized form, the perineuronal net (PNN), enwraps key inhibitory interneurons and governs critical-period synaptic plasticity and circuit stability.¹⁰ PNN architecture is consistently altered in genetic ASD mouse models and in postmortem human ASD brain tissue.¹¹ , ¹² The ECM and autophagy are bidirectionally coupled: The ECM modulates intracellular autophagic activity, while autophagic flux is required for normal ECM remodeling and synaptic structural integrity.¹³ , ¹⁴ , ¹⁵ PNN disruption has also been identified as relevant for Alzheimer’s disease,¹⁶ reinforcing that the brain’s ECM is not a passive structural scaffold but an active participant in the same homeostatic networks that autophagy governs — and that blarcamesine targets through SIGMAR1 activation. Why This Matters for Anavex and Blarcamesine Blarcamesine is an investigational oral therapy that activates SIGMAR1, a key intracellular chaperone protein that sits at a critical junction of cellular homeostasis. Peer-reviewed research has established that SIGMAR1 activation by blarcamesine restores impaired autophagy by stimulating ULK1 phosphorylation — a central signaling node that initiates the formation of autophagosomes, the cellular organelles responsible for engulfing and recycling damaged proteins and organelles. This mechanism was first demonstrated at the molecular level in a 2019 publication in Cells, which showed that blarcamesine enhances autophagic flux in human cells and increases proteostasis capacity in the roundworm C. elegans , ultimately rescuing the organism from paralysis caused by protein aggregation.¹⁷ A 2025 iScience publication further elucidated the molecular protein binding mechanism of blarcamesine to SIGMAR1 and GABARAP, a core autophagy protein.¹⁸ The emerging picture of ASD — as a condition with co-contribution of autophagy failure, synaptic pruning deficits, convergent genetic disruption of protein homeostasis pathways, and ECM dysregulation — maps directly onto the disease biology that blarcamesine is designed to address. The therapeutic rationale is not inferential; it is mechanistically grounded in the same autophagic machinery that blarcamesine has been shown to restore across multiple model systems and in human clinical trials. Clinical Evidence Across the Lifespan Anavex’s clinical development program spans both neurodegenerative and neurodevelopmental conditions, reflecting precisely the kind of cross-lifespan therapeutic strategy that the emerging autism–Alzheimer’s research now suggests is needed: Alzheimer’s Disease: In the Phase IIb/III ANAVEX ® 2-73-AD-004 trial, once-daily oral blarcamesine significantly slowed cognitive and functional decline in patients with early Alzheimer’s disease over 48 weeks, supported by biomarker evidence including improved plasma Aβ42/40 ratio and significant reduction in brain atrophy in key brain areas. A precision medicine approach demonstrated that patients carrying wild-type SIGMAR1 (the ABCLEAR1 population, representing approximately 70% of the global population) showed enhanced clinical responses. This approach was further amplified in the ABCLEAR3 population, including the brain’s extracellular matrix (ECM) wild-type protein COL24A1. Rett Syndrome: The AVATAR trial demonstrated clinical effects in adult patients with Rett syndrome, a severe neurodevelopmental disorder caused by mutations in the MECP2 gene — one of the genes now identified as key for brain development. Treatment was associated with changes in disease-relevant biomarkers, including a significant increase in GABA levels. Open-label extension data indicated disease-modifying effects. A Phase II/III pediatric trial (EXCELLENCE) has also been completed. Parkinson’s Disease Dementia: Blarcamesine has shown proof-of-concept efficacy in a Phase II study in Parkinson’s disease dementia, a condition also linked to impaired autophagy and protein aggregation (alpha-synuclein). Advanced clinical development in Parkinson’s disease is ongoing. “The growing recognition that neurodevelopmental and neurodegenerative conditions share fundamental disease biology — particularly around autophagy — validates the foundational science behind Anavex’s platform,” said Wolfgang Liedtke, MD, PhD, Senior Vice President and Global Head of Neurology at Anavex. “We are looking forward to proceeding with pivotal clinical trials on the principle that restoring cellular homeostasis through SIGMAR1 activation may address disease biology across the CNS, from childhood through old age.” “As independent researchers from institutions including Mount Sinai, Boston University, Drexel University, and the Karolinska Institute continue to map the molecular connections between autism and Alzheimer’s, the therapeutic rationale for a precision medicine approach targeting autophagy with blarcamesine has never been more encouraging,” said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex. “Anavex’s unique precision medicine approach recognizes autophagy dysfunction as a common upstream contributor preceding diverse downstream pathologies across the age spectrum.” This release discusses investigational uses of an agent in development and is not intended to convey conclusions about efficacy or safety. There is no guarantee that any investigational uses of such product will successfully complete clinical development or gain health authority approval. About Anavex Life Sciences Corp. Anavex Life Sciences Corp. (Nasdaq: AVXL) is a publicly traded biopharmaceutical company dedicated to the development of novel therapeutics for the treatment of neurodegenerative, neurodevelopmental, and neuropsychiatric disorders, including Alzheimer's disease, Parkinson's disease, schizophrenia, Rett syndrome, and other central nervous system (CNS) diseases, pain, and various types of cancer. Anavex's lead drug candidate, blarcamesine ( ANAVEX ® 2-73 ), has successfully completed a Phase 2a and a Phase 2b/3 clinical trial for Alzheimer's disease, a Phase 2 proof-of-concept study in Parkinson's disease dementia, and both a Phase 2 and a Phase 3 study in adult patients and one Phase 2/3 study in pediatric patients with Rett syndrome. Blarcamesine is an orally available drug candidate designed to restore cellular homeostasis by targeting SIGMAR1 and muscarinic receptors. Preclinical studies demonstrated its potential to halt and/or reverse the course of Alzheimer's disease. Blarcamesine also exhibited anticonvulsant, anti-amnesic, neuroprotective, and anti-depressant properties in animal models, indicating its potential to treat additional CNS disorders, including epilepsy. The Michael J. Fox Foundation for Parkinson's Research previously awarded Anavex a research grant, which fully funded a preclinical study to develop blarcamesine for the treatment of Parkinson's disease. We believe that ANAVEX ® 3-71, which targets SIGMAR1 and M1 muscarinic receptors, is a promising clinical stage drug candidate demonstrating disease-modifying activity against the major hallmarks of Alzheimer's disease in transgenic (3xTg-AD) mice, including cognitive deficits, amyloid, and tau pathologies. In preclinical trials, ANAVEX ® 3-71 has shown beneficial effects on mitochondrial dysfunction and neuroinflammation. Further information is available at . You can also connect with the Company on Twitter, Facebook , Instagram , and LinkedIn . Forward-Looking Statements Statements in this press release that are not strictly historical in nature are forward-looking statements. These statements are only predictions based on current information and expectations and involve a number of risks and uncertainties. Actual events or results may differ materially from those projected in any of such statements due to various factors, including the risks set forth in the Company’s most recent Annual Report on Form 10-K filed with the SEC. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement and Anavex Life Sciences Corp. undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof. For Further Information: Anavex Life Sciences Corp. Research & Business Development Toll-free: 1-844-689-3939 Email: info@anavex.com Investors: Andrew J. Barwicki Investor Relations Tel: 516-662-9461 Email: andrew@barwicki.com References Vivanti G et al. “Prevalence of Dementia Among US Adults With Autism Spectrum Disorder.” JAMA Network Open. 2025;8(1):e2453691. Phillips WT et al. “Pathophysiologic similarities between autism spectrum disorder and Alzheimer’s disease: therapeutic possibilities.” Frontiers in Neuroscience. 2026;19:1737007. Tang G et al. “Loss of mTOR-dependent macroautophagy causes autistic-like synaptic pruning deficits.” Neuron. 2014;83(5):1131–1143. Kim HJ et al. “Deficient autophagy in microglia impairs synaptic pruning and causes social behavioral defects.” Molecular Psychiatry. 2017;22(11):1576–1584. Yan J et al. “Activation of autophagy rescues synaptic and cognitive deficits in fragile X mice.” PNAS. 2018;115(41):E9707–E9716. Hui KK et al. “GABARAPs dysfunction by autophagy deficiency in adolescent brain impairs GABA₁ receptor trafficking and social behavior.” Sci Adv. 2019;5(4):eaau8237. Kwon CH et al. “Pten regulates neuronal arborization and social interaction in mice.” Neuron. 2006;50(3):377–388. Bourgeron T. “A synaptic trek to autism.” Curr Opin Neurobiol. 2009;19(2):231–234. Dere E et al. “Heterozygous ambra1 deficiency in mice: a genetic trait with autism-like behavior restricted to the female gender.” Front Behav Neurosci. 2014;8:181. Fawcett JW et al. “The roles of perineuronal nets and the perinodal extracellular matrix in neuronal function.” Nat Rev Neurosci. 2019;20(8):451–465. Burket JA et al. “Region-Specific Alterations of Perineuronal Net Expression in Postmortem Autism Brain Tissue.” Front Psychiatry. 2022;13:872615. Adeyelu TT et al. “Behavioral regulation by perineuronal nets in the prefrontal cortex of the CNTNAP2 mouse model of autism spectrum disorder.” Front Behav Neurosci. 2023;17:1114789. Fung C et al. “Induction of Autophagy during Extracellular Matrix Detachment Promotes Cell Survival.” Mol Biol Cell. 2008;19(3):797–806. Bhatt DL et al. “Cooperation of cell adhesion and autophagy in the brain: Functional roles in development and neurodegenerative disease.” Autophagy Reports. 2021;1(1):192–222. Iozzo RV et al. “Instructive Roles of Extracellular Matrix on Autophagy.” Mol Cell Biol. 2014;34(22):4001–4012. Vries LE et al. “Resilience to Alzheimer’s disease associates with alterations in perineuronal nets.” Alzheimer’s & Dementia. 2025. Christ MG et al. “Sigma-1 receptor activation induces autophagy and increases proteostasis capacity in vitro and in vivo.” Cells. 2019;8(3):211. Baeken MW et al. “Conserved LIR-specific interaction of Sigma-1 receptor and GABARAP.” iScience. 2025;28(9):113287.

Landmark US FDA Clinical Trial | First Participant Enrolled in Autism Diagnostic Study Across 10 Elite American Hospitals BlinkLab Ltd (ASX:BB1) With a clear pathway toward FDA submission and strong execution underway, we believe BlinkLab is well positioned to transform how autism is assessed in clinical practice.” — BlinkLab CEO and Managing Director, Dr Henk-Jan Boele PRINCETON, NJ, UNITED STATES, March 23, 2026 / EINPresswire.com / -- A Western Australia-headquartered med tech company developing AI-powered, smartphone-based diagnostic technology for autism spectrum disorder, today announced the commencement of its pivotal validation study to support its planned US Food and Drug Administration (FDA) 510(k) submission, with the first child participant now enrolled and tested using BlinkLab 's flagship diagnostic platform, BlinkLab Dx1. The milestone marks the opening of the most significant clinical programme in the company's history, and represents a major advance in the global effort to make autism diagnosis faster, more objective, and more accessible to the millions of families currently facing multi-year waits for a formal assessment. BlinkLab CEO and Managing Director, Dr Henk-Jan Boele, commented: “We are excited to initiate this next phase of our FDA 510(k) regulatory program. It marks what I believe is the most important milestone for BlinkLab to date, following years of focused development, extensive testing, optimisation of our tech, close collaboration and meetings with the FDA, and securing IRB approvals to support clinical execution. With a clear pathway toward FDA submission and strong execution underway, we believe BlinkLab is well positioned to transform how autism is assessed in clinical practice.” The Autism Diagnosis Problem — and Why It Matters Autism spectrum disorder (ASD) affects approximately 1 in 31 children in the United States, according to data published by the Centers for Disease Control and Prevention in May 2025. Despite this prevalence, access to timely, accurate diagnosis remains critically constrained. Families in the US face an average wait of 18 to 24 months for formal assessment. Approximately 21% of US autism clinics are currently unable to accept new referrals due to workforce shortages and overwhelming demand. The current diagnostic gold standard, the Autism Diagnostic Observation Schedule (ADOS), relies heavily on subjective clinician observation, requires rare specialist expertise, and cannot be readily scaled or standardised across diverse clinical settings. The result is a diagnostic bottleneck that denies children access to early intervention during the critical developmental window in which therapeutic support has the greatest impact. BlinkLab Dx1 is designed to address this gap. Using a standard smartphone camera and proprietary AI-driven computer vision, Dx1 captures objective, reflex-based neurometric biomarkers during a 15-minute session in which a child watches age-appropriate video content. The platform analyses facial reflexes, eye and eyelid movements, startle responses, postural stability, and vocal responses, producing reproducible, clinician-interpretable diagnostic data that supports earlier and more accurate autism identification. Pivotal Study Design The pivotal validation study has been designed in full alignment with FDA expectations for a 510(k) diagnostic aid submission. The study employs a double-blinded, multi-centre, within-subjects comparison design, evaluating Dx1 performance against established gold standard autism assessments. Key endpoints include sensitivity, specificity, negative predictive value (NPV), and positive predictive value (PPV). The study is being led and conducted by IQVIA-MCRA, one of the world's largest and most experienced contract research organisations, ensuring full adherence to FDA study protocols, data quality standards, and regulatory requirements. The programme commences with a dedicated usability phase, consistent with FDA guidance, ensuring clinicians and caregivers can safely and effectively deploy the platform in real-world clinical settings, before transitioning seamlessly into the clinical validation phase. The study is being conducted across a network of ten leading US clinical institutions, representing broad geographic and demographic diversity across the American population: Cincinnati Children's Hospital Seattle Children's Hospital University of Pennsylvania, Perelman School of Medicine Vanderbilt Kennedy Center Rush University Medical Center Drexel University University of Nebraska Medical Center MU Thompson Center for Autism & Neurodevelopmental Disorders Southwest Autism Research & Resource Center (SARRC) University of Arkansas Participant recruitment is expected to be completed within approximately eight months, with the FDA 510(k) submission targeted by end of calendar year 2026. World-First Commercial Deployment in Morocco The commencement of the US pivotal trial is complemented by the recent announcement of a world-first sovereign deployment of Dx1. BlinkLab has entered into a formal agreement with the Foundation Mohammed V for Solidarity to provide the Dx1 platform to Morocco's national health system, targeting approximately 600,000 children born annually, beginning at 18 months of age. The program is government-funded, with all implementation costs covered by the Moroccan government and the Foundation Mohammed V for Solidarity. BlinkLab retains all data rights generated under the program. Initial screening centres are expected to be operational from April 2026, with phased expansion planned across approximately 3,000 public primary healthcare centres. The Morocco framework is explicitly designed as a replicable model for North Africa and the Middle East. This real-world commercial deployment at national scale provides direct complementary evidence to BlinkLab's US FDA programme demonstrating that the platform can be deployed effectively across diverse populations and healthcare infrastructure settings, even prior to formal regulatory clearance. About BlinkLab Limited BlinkLab Limited was founded by neuroscientists at Princeton University and is developing a smartphone-based diagnostic platform for autism. Its most advanced product, BlinkLab Dx1, is an autism diagnostic aid for clinicians that leverages smartphones, artificial intelligence, and machine learning to capture objective, reflex-based measures, supporting earlier and more accurate autism identification. This enables timely intervention during critical periods of brain development. BlinkLab is led by an experienced management team and Board with deep expertise in digital healthcare, computer vision, and AI, supported by a Scientific Advisory Board of leading experts in autism and brain development. Jane Morgan Investor and Media Relations +61 405 555 618 info@janemorganmanagement.com.au Visit us on social media: LinkedIn YouTube X Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

MONDAY, March 16, 2026 -- In a clinical practice guideline issued by the American Academy of Pediatrics, in partnership with the North American Society for Pediatric Gastroenterology, Hepatology and Nutrition, recommendations are presented for the diagnosis and management of faltering weight in young pediatric patients. The guideline is published online March 16 in Pediatrics. Hans B. Kersten, M.D., from the St. Christopher's Hospital for Children at Drexel University College of Medicine in Philadelphia, and colleagues developed a clinical practice guideline to support health care providers caring for children with poor weight gain. The term "failure to thrive" was updated to faltering weight, and z-score cutoffs were used as diagnostic criteria. The authors note that a diagnosis of faltering weight includes any of weight-for-length of body mass index (BMI)-for-age less than −1.65 z-score; weight gain velocity less than −2 z-score for age in children younger than 2 years; or decline in weight, weight-for-length, or BMI greater than or equal to 1 z-score. Eight Key Action Statements were provided and four Good Practice Statements were offered for additional guidance. Only children who have specific conditions that suggest a focal evaluation or persistent faltering weight are recommended to undergo diagnostic testing. Endoscopy with biopsy is suggested for children with persistent faltering weight or who have concerns for conditions that cannot be diagnosed without endoscopy. Use of increased calories of food/energy, oral nutritional supplementation, and therapy for pediatric feeding disorder are recommended. "The guideline takes a whole-child approach to evaluation and treatment and recognizes that safe, stable, nurturing relationships are necessary components for normal growth and development," Kersten said in a statement. "The goals are to help doctors make accurate diagnoses, avoid unnecessary tests, and use health care resources wisely." Clinical Practice Guideline Technical Report 1 Technical Report 2