AVXL Anavex Life Sciences Corporation

Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome, schizophrenia and other central nervous system (CNS) diseases, today reported financial results for its fiscal quarter ended December 31, 2023.

“Exciting progress in the field related to treating neurodegenerative diseases highlights the significant potential for our broad therapeutic portfolio and differentiated precision medicine platform to deliver easy access and scalable treatment options demonstrated by the initiated process of Marketing Authorisation application to the European Medicines Agency (EMA) for blarcamesine related to the treatment of Alzheimer’s disease,” said Christopher U Missling, PhD, President and Chief Executive Officer of Anavex. “We continue to focus on execution and commercial readiness as we advance our therapeutic pipeline to potentially improve patients' lives living with neurodegenerative, neurodevelopmental disorders and schizophrenia.”

Key Pipeline Updates:

• Alzheimer’s disease: Full data from the blarcamesine in Alzheimer’s disease Phase 2b/3 randomized clinical trial will be published in an upcoming peer-reviewed journal. The Company has initiated the process for submitting a Marketing Authorisation application to the European Medicines Agency (EMA) under the Centralised Procedure. The Marketing Authorisation would allow direct market access throughout the European Union for oral blarcamesine for the treatment of Alzheimer’s disease. There are an estimated 7 million people in Europe with Alzheimer’s disease, a number expected to double by 2030, according to the European Brain Council.1
• Alzheimer’s disease: Ongoing ATTENTION-AD open-label extension 96-week trial.
• Rett syndrome: Topline data from ANAVEX®2-73-RS-003 Phase 2/3 EXCELLENCE pediatric clinical trial was announced last month. We intend to further assess the results and discuss with the regulatory authorities next steps.
• Schizophrenia: Initiation of FDA cleared placebo-controlled ANAVEX®3-71 Phase 2 clinical trial expected to begin in Q2 2024.
• Parkinson’s disease: Initiation of ANAVEX®2-73 imaging-focused trial and Phase 2b/3 6 months trial.
• Fragile X: New disease-specific, translatable, and objective biomarker data generated with ANAVEX®2-73, support the initiation of the potentially pivotal ANAVEX®2-73 Phase 2/3 clinical trial.
• New Rare disease: Initiation of potentially pivotal ANAVEX®2-73 Phase 2/3 clinical trial.
• Publications: Continued clinical publications involving ANAVEX®2-73 and ANAVEX®3-71.

1 https://www.braincouncil.eu/projects/rethinking-alzheimers-disease/

Recent Business Highlights:

• On January 24, 2024, the Company announced a peer-reviewed publication in Clinical Pharmacology in Drug Development, findings from the ANAVEX®3-71 first-in-human study which achieved its safety objectives. The publication is entitled, ‘Population-Based Characterization of the Pharmacokinetics and Food Effect of ANAVEX3-71, a Novel Sigma-1 Receptor and Allosteric M1 Muscarinic Receptor Agonist in Development for Treatment of Frontotemporal Dementia, Schizophrenia, and Alzheimer Disease’.2 The publication reports pharmacokinetic (PK) dose proportionality of ANAVEX®3-71 in humans and food had no effect on the PK of ANAVEX®3-71. This data also expands the safety objectives met in this first-in-human study of ANAVEX®3-71, further supporting its drug development program.
• On January 16, 2024, the Company announced the initiation of the U.S. FDA cleared placebo-controlled Phase 2 trial of ANAVEX®3-71 for the treatment of schizophrenia, which is expected to begin in Q2 2024.
• On January 8, 2024, the Company announced that the United States Patent and Trademark Office (USPTO) granted U.S. Patent No. 11,839,600 entitled “NEURODEVELOPMENTAL DISORDER THERAPY” from the United States Patent and Trademark Office (USPTO) for its patent application number 17/890,083. Anavex’s newest patent expands coverage of ANAVEX®2-73 (blarcamesine) therapy to ameliorate various conditions associated with loss-of-function mutations of the gene encoding methyl-CpG binding protein (MeCP2).
• On January 2, 2024, the Company reported topline results from the randomized, double-blind, placebo-controlled, Phase 2/3 EXCELLENCE clinical trial, which evaluated the clinical efficacy, safety, and tolerability of 30 mg ANAVEX®2-73 in 92 pediatric patients with Rett syndrome (RTT) between the ages of 5 through 17 years. After 12 weeks, the study showed improvement on the key co-primary endpoint RSBQ. The other co-primary endpoint, the CGI-I, which represents a less granular assessment by the site investigators, was not met. The trial reinforced the favorable and manageable safety profile observed in ANAVEX®2-73 to date, and a high enrollment rate into the OLE of over 91% as well as the high level of requests for the Compassionate Use Program (93%) provide solid numerical evidence for the reported positive Real World Evidence (RWE) from patients with Rett syndrome under Compassionate Use Authorization.
• On December 20, 2023, the Company announced the first entire clinical gene pathway data from the ANAVEX®2-73-RS-002 AVATAR Rett syndrome trial. We believe that this is the first time a whole genome and exome analysis comparing drug and placebo in patients with Rett syndrome was performed and we believe the results confirm that Rett syndrome is indeed a neurodevelopmental disorder with a key metabolic component, which can be addressed with therapeutic intervention and is likely relevant for other neurodevelopmental disease indications.
• On December 19, 2023, the Company announced that the Committee for Medicinal Products for Human Use (CHMP) within the European Medicines Agency (EMA) agreed that oral blarcamesine for Alzheimer’s disease is eligible for submission of an application for a Union Marketing Authorisation in the EU under the European Medicines Agency’s centralised procedure. Anavex is aiming to submit the Marketing Authorisation Application as early as possible in 2024.

2 Fadiran EO, Hammond E, Tran J, Missling CU, Ette E. Population-Based Characterization of the Pharmacokinetics and Food Effect of ANAVEX3-71, a Novel Sigma-1 Receptor and Allosteric M1 Muscarinic Receptor Agonist in Development for Treatment of Frontotemporal Dementia, Schizophrenia, and Alzheimer Disease. Clin Pharmacol Drug Dev. 2024;13(1):21-31. doi:10.1002/cpdd.1323

Financial Highlights:

• Cash and cash equivalents of $143.8 million at December 31, 2023 compared to $151.0 million at fiscal yearend September 30, 2023.
• General and administrative expenses for the quarter of $2.6 million compared to $3.3 million for the comparable first quarter of fiscal 2023.
• Research and development expenses for the fourth quarter of $8.7 million compared to $12.1 million for the comparable first quarter of fiscal 2023.
• Net loss for the first quarter of $8.6 million, inclusive of $2.3 million in non-cash items, or $0.11 per share, compared to a net loss of $13.0 million, inclusive of $5.3 million in non-cash items, or $0.17 per share for the comparable first quarter of fiscal 2023.

The financial information for the fiscal quarter ended December 31, 2023, should be read in conjunction with the Company’s condensed consolidated interim financial statements, which will appear on EDGAR, www.sec.gov and will be available on the Anavex website at www.anavex.com.

Webcast / Conference Call Information:

The live webcast of the conference call will be available on Anavex’s website at www.anavex.com.

The conference call can be also accessed by dialing 1 929 205 6099 for participants in the U.S. using the Meeting ID# 821 3683 5450 and reference passcode 121725. A replay of the conference call will also be available on Anavex’s website for up to 30 days.

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 and neurodevelopmental disorders, including Alzheimer's disease, Parkinson's disease, Rett syndrome, schizophrenia and other central nervous system (CNS) diseases, pain, and various types of cancer. Anavex's lead drug candidate, ANAVEX®2-73 (blarcamesine), 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 in pediatric patients with Rett syndrome. ANAVEX®2-73 is an orally available drug candidate that restores cellular homeostasis by targeting SIGMAR1 and muscarinic receptors. Preclinical studies demonstrated its potential to halt and/or reverse the course of Alzheimer's disease. ANAVEX®2-73 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 ANAVEX®2-73 for the treatment of Parkinson's disease. 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 www.anavex.com. 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.

Anavex Life Sciences Corp.
Condensed Consolidated Interim Statements of Operations and Comprehensive Loss
(in thousands, except share and per share amounts)
	Three months ended December 31,
		2023			2022
Operating Expenses
General and administrative	$	2,609		$	3,317
Research and development		8,684			12,067
Total operating expenses		11,293			15,384
Operating loss		(11,293	)		(15,384	)
Other income
Grant income		-			25
Research and development incentive income		592			733
Interest income, net		2,008			1,268
Foreign exchange gain, net		156			366
Total other income, net		2,756			2,392
Net loss before provision for income taxes		(8,537	)		(12,992	)
Income tax recovery (expense), current		(85	)		20
Net loss and comprehensive loss	$	(8,622	)	$	(12,972	)
Net loss per share
Basic and diluted	$	(0.11	)	$	(0.17	)
Weighted average number of shares outstanding
Basic and diluted		82,077,815			77,977,112

Anavex Life Sciences Corp.
Condensed Consolidated Interim Balance Sheets
(in thousands, except share and per share amounts)
	December 31,			September 30,
		2023			2023
Assets
Current
Cash and cash equivalents	$	143,765		$	151,024
Incentive and tax receivables		3,549			2,709
Prepaid expenses and other current assets		756			653
Total Assets	$	148,070		$	154,386
Liabilities and stockholders' equity
Current Liabilities
Accounts payable	$	4,292		$	4,322
Accrued liabilities		7,286			7,295
Deferred grant income		917			917
Total Liabilities		12,495			12,534
Capital Stock		82			82
Additional paid-in capital		437,184			434,839
Accumulated deficit		(301,691	)		(293,069	)
Total Stockholders' Equity		135,575			141,852
Total Liabilities and Stockholders' Equity	$	148,070		$	154,386

For Further Information:Anavex Life Sciences Corp.Research & Business DevelopmentToll-free: 1-844-689-3939Email: info@anavex.com

Investors:Andrew J. BarwickiInvestor RelationsTel: 516-662-9461Email: andrew@barwicki.com