Rett Syndrome

First patient screened ahead of schedule Trial to investigate positive, negative, and cognitive domains of schizophrenia ANAVEX®3-71’s differentiated dual novel mechanism of action offers the potential to synergistically activate both SIGMAR1 and M1 muscarinic receptors and treat all symptom domains of schizophrenia without the side effects of standard of care antipsychotics NEW YORK, March 18, 2024 (GLOBE NEWSWIRE) -- 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 psychiatric disorders, including Alzheimer's disease, Parkinson's disease, Rett syndrome, schizophrenia, and other central nervous system (CNS) diseases, today announced that the first patient in its U.S. FDA cleared placebo-controlled Phase 2 clinical study of ANAVEX®3-71 for the treatment of schizophrenia has been screened ahead of schedule. “Initiating our Phase 2 trial of ANAVEX®3-71 in schizophrenia ahead of schedule is a testament to the hard work and dedication of our clinical study team,” stated Christopher U Missling, PhD, President and Chief Executive Officer of Anavex. “Schizophrenia is a challenging disorder that can impair social and occupational functions and overall quality of life for the nearly 24 million people affected worldwide. Following on from our positive initial Phase 1 results in healthy volunteers, our Phase 2 study will apply novel neuroinflammatory, metabolomic, and transcriptomic biomarkers at the intersection of schizophrenia pathophysiology and ANAVEX®3-71’s novel, dual mechanism of action, with the goal of addressing the large unmet need in this patient population. We are excited to build on our diverse Precision Medicine Platform, which advanced blarcamesine (ANAVEX®2-73) onto a regulatory pathway for potential treatment of Alzheimer’s disease.” ANAVEX®3-71’s differentiated mechanism is a dual SIGMAR1 receptor agonist and M1 positive allosteric modulator with agonistic effects.1,2 This novel mechanism of action offers the potential to treat all symptom domains of schizophrenia -- positive symptoms (hallucinations and delusions), negative symptoms (difficulty enjoying life and withdrawal from others), and cognitive impairment (deficits in memory, concentration, and decision-making) -- without the side effects of standard of care antipsychotics, which do not adequately address all symptom domains in schizophrenia. ANAVEX®3-71’s ability to modulate both SIGMAR1 and M1 receptors is expected to potentially address disruptions to neuronal homeostasis observed in individuals with schizophrenia, upstream of the targets leveraged by standard of care medications. Preceding this study, ANAVEX®3-71 demonstrated an adequate safety profile in a Phase 1 trial in healthy volunteers.3,4 ANAVEX®3-71 Phase 2 Program Outline The placebo-controlled Phase 2, two-part, in-patient trial, ANAVEX®3-71-SZ-001 (NCT06245213), will investigate the effects of ANAVEX®3-71 in patients with schizophrenia. Part A is a double-blind, placebo-controlled, multiple ascending dose trial. The results of Part A will be used to select a dose for Part B, a proof-of-concept efficacy, double-blind, placebo-controlled trial.Participants will undergo either 10 or 28 days of dosing (Part A and Part B, respectively).Standard clinical outcome measures, including the Positive and Negative Syndrome Scale (PANSS), and novel fluid and electrophysiological biomarkers will be assessed. Anavex is leveraging several advances in electroencephalography/event-related potential (EEG/ERP) biomarkers in schizophrenia developed in collaboration with the industry-led ERP Biomarker Qualification Consortium.5 This includes a validated, and automated data analysis pipeline for quantitative EEG and ERP biomarkers, as well as normative EEG/ERP data in patients with schizophrenia and matched healthy volunteers. These advances also include recently established relationships between EEG/ERP biomarkers and clinically important measures in schizophrenia. In addition to the electrophysiological biomarkers, Anavex is applying novel neuroinflammatory, metabolomic, and transcriptomic biomarkers at the intersection of schizophrenia pathophysiology and ANAVEX®3-71’s novel, dual mechanism of action. About Schizophrenia Schizophrenia is a persistent and often disabling mental illness impacting how a person thinks, feels, and behaves, and affects nearly 24 million people worldwide, including 2.8 million people in the U.S. It is characterized by three symptom domains: positive symptoms (hallucinations and delusions), negative symptoms (difficulty enjoying life and withdrawal from others), and cognitive impairment (deficits in memory, concentration, and decision-making). In part due to limitations with current treatments, people living with schizophrenia often struggle to maintain employment, live independently, and manage relationships. While current treatments can be effective in managing select symptoms, approximately 34% of people do not respond to therapy, 6 with an additional 50-60% experiencing only a partial improvement in symptoms or unacceptable side effects.7 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. 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 1 Fisher A, Bezprozvanny I, Wu L, et al. AF710B, a Novel M1/σ1 Agonist with Therapeutic Efficacy in Animal Models of Alzheimer’s Disease. Neurodegener Dis. 2016;16(1-2):95-110. doi:10.1159/0004408642 Hall H, Iulita MF, Gubert P, et al. AF710B, an M1/sigma-1 receptor agonist with long-lasting disease-modifying properties in a transgenic rat model of Alzheimer's disease. Alzheimers Dement. 2018;14(6):811-823. doi:10.1016/j.jalz.2017.11.0093 Fadiran EO, Hammond E, Tran J, et al. Concentration-QTc Relationship from a Single Ascending Dose Study of ANAVEX3-71, a Novel Sigma-1 Receptor and Allosteric M1 Muscarinic Receptor Agonist in Development for the Treatment of Frontotemporal Dementia, Schizophrenia, and Alzheimer's Disease. Clin Pharmacol Drug Dev. 2023;12(9):888-901. doi:10.1002/cpdd.13034 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.13235 Cecchi M, Adachi M, Basile A, et al. Validation of a suite of ERP and QEEG biomarkers in a pre-competitive, industry-led study in subjects with schizophrenia and healthy volunteers. Schizophr Res. 2023;254:178-189. doi:10.1016/j.schres.2023.02.0186 Potkin SG, Kane JM, Correll CU, et al. The neurobiology of treatment-resistant schizophrenia: paths to antipsychotic resistance and a roadmap for future research. NPJ Schizophr. 2020;6(1):1. Published 2020 Jan 7. doi:10.1038/s41537-019-0090-z7 Nucifora FC Jr, Woznica E, Lee BJ, Cascella N, Sawa A. Treatment resistant schizophrenia: Clinical, biological, and therapeutic perspectives. Neurobiol Dis. 2019;131:104257. doi:10.1016/j.nbd.2018.08.016

- FDA completed Pre-License Inspection of Abeona’s Cleveland manufacturing facility, as well as inspection of clinical trial sites for pz-cel pivotal Phase 3 VIITAL™ study -CLEVELAND, March 18, 2024 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today reported financial results for the full year of 2023 and provided an update on progress toward achieving key corporate objectives. Abeona also announced today that the U.S. Food and Drug Administration (FDA) has completed a Pre-License Inspection (PLI) of its Cleveland, Ohio manufacturing facility related to the Company’s Biologics License Application (BLA) for pz-cel (prademagene zamikeracel) for recessive dystrophic epidermolysis bullosa (RDEB). During the inspection, the FDA reviewed the facilities, systems, and processes at the Cleveland site. The FDA also observed the manufacturing process for pz-cel, as well as performance of in-process and release assays. The two-week PLI, which was conducted by five FDA inspectors, concluded on March 1, 2024. Upon completion of the inspection, a Form 483 was issued with observations related to process controls. On March 15, 2024, the Company submitted a response to the FDA, outlining already implemented and ongoing steps toward resolution that follow FDA guidance provided during the audit. In addition, the FDA has completed the clinical study site inspections of Stanford University School of Medicine in Palo Alto, CA and University of Massachusets Medical School in Worcester, MA, both of which enrolled subjects in the pivotal Phase 3 VIITAL™ study supporting the pz-cel BLA, with no Form 483 observations noted. The FDA’s review of Abeona’s pz-cel BLA is ongoing, with a target Prescription Drug User Fee Act (PDUFA) date of May 25, 2024, and the FDA does not currently plan to conduct an Advisory Committee. “We appreciate the FDA’s collaborative conduct of the PLI, and look forward to working with them through the remainder of the BLA review.” said Vish Seshadri, Chief Executive Officer of Abeona. Fourth Quarter and Recent Progress Pz-cel for RDEB In November 2023, the FDA accepted and granted Priority Review for the Company’s BLA for pz-cel for RDEB with a target PDUFA date of May 25, 2024. The FDA also advised that it does not plan to convene an Advisory Committee meeting to discuss the pz-cel application.In January 2024, the FDA completed both a Bioresearch Monitoring (BIMO) inspection of Abeona and the Mid-Cycle Meeting regarding the pz-cel BLA. Following completion of the BIMO inspection, the FDA inspector did not issue any observations or FDA Form 483s. At the BLA Mid-Cycle Review meeting, the FDA reaffirmed that it does not currently plan to convene an Advisory Committee for pz-cel. In addition, the FDA advised that Risk Evaluation and Mitigation Strategies (REMS) are not anticipated for the pz-cel BLA at this time, though review is ongoing, and reconfirmed the target PDUFA date of May 25, 2024.In March 2024, Abeona received a written Establishment Inspection Report (EIR), the formal report from the FDA regarding the BIMO inspection. The results of the inspection determined there were no deficiencies with the monitoring or management of study sites, IP accountability, retention of study site documents, or safety oversight. U.S. commercial launch preparations for pz-cel Abeona continues to advance key commercial activities in preparation for a potential U.S. launch for pz-cel, including onboarding discussions with EB treatment sites, payer engagement, hiring key commercial roles, and conducting market research.The Company recently completed payer market research that supports reimbursement coverage of pz-cel at a price commensurate with the value of approved gene therapies that offer years of durable benefit following a one-time application. Corporate highlights In January 2024, Abeona entered a $50 million credit facility with the Avenue Venture Opportunities Fund, L.P. The credit agreement includes a first tranche of $20 million, which was funded in January 2024, a second tranche of $10 million of committed capital, and an option for an additional $20 million upon satisfaction of certain terms and conditions. Proceeds from the facility are intended to support the Company’s ongoing preparations for launch and commercialization in anticipation of a potential FDA approval of pz-cel. Full Year 2023 Financial Results and Cash Runway Guidance Cash, cash equivalents, restricted cash and short-term investments totaled $52.6 million as of December 31, 2023, compared to $52.5 million as of December 31, 2022. Net cash used in operating activities was $37.0 million for the full year of 2023, compared to $43.5 million in the full year of 2022. In January 2024, Abeona received a first tranche of $20 million as part of the credit facility with Avenue Venture Opportunities Fund, L.P. Abeona estimates that its current cash and cash equivalents, restricted cash and short-term investments, as well as the credit facility with Avenue Venture Opportunities Fund, L.P, are sufficient resources to fund operations into the first quarter of 2025 before accounting for any revenue from commercial sales of pz-cel, if approved, or proceeds from the sale of a Priority Review Voucher, if awarded by the FDA. License and other revenues for the year ended December 31, 2023 were $3.5 million, as compared to $1.4 million for the same period of 2022. The revenues in both years primarily represent clinical milestone payments under a licensing agreement with Taysha Gene Therapies for investigational AAV-based gene therapy for Rett syndrome. Research and development expenses for the full year ended December 31, 2023 were $31.1 million, compared to $29.0 million for the full year ended December 31, 2022, primarily a result of increased headcount related to BLA activities. General and administrative expenses were $19.0 million for the full year ended December 31, 2023, compared to $17.3 million for the year ended December 31, 2022, primarily a result of increased headcount for the potential launch of pz-cel. Net loss attributable to common shareholders for the full year ended December 31, 2023 was $54.2 million, or $2.53 loss per common share as compared to $43.5 million, or $5.53 loss per common share, for the full year of 2022. Conference Call Details Abeona Therapeutics will host a conference call and webcast today, March 18, 2024, at 8:30 a.m. ET. To access the call, dial 888-506-0062 (U.S. toll-free) or 973-528-0011 (international) and Entry Code: 428606 five minutes prior to the start of the call. A live, listen-only webcast and archived replay of the call can be accessed at https://investors.abeonatherapeutics.com/events. The archived webcast replay will be available for 30 days following the call. About Abeona Therapeutics Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing cell and gene therapies for serious diseases. The U.S. FDA has accepted and granted Priority Review with a PDUFA target action date of May 25, 2024 for the Biologics License Application for pz-cel (prademagene zamikeracel), Abeona’s investigational autologous, COL7A1 gene-corrected epidermal sheets currently in development for recessive dystrophic epidermolysis bullosa. The Company’s fully integrated cell and gene therapy cGMP manufacturing facility served as the manufacturing site for pz-cel used in its Phase 3 VIITAL™ trial, and is capable of supporting commercial production of pz-cel upon FDA approval. The Company’s development portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical need. Abeona’s novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. For more information, visit www.abeonatherapeutics.com. Forward-Looking Statements This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. We have attempted to identify forward-looking statements by such terminology as “may,” “will,” “believe,” “anticipate,” “expect,” “intend,” “potential,” and similar words and expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to, the timing and outcome of our Biologics License Application submission to the FDA for pz-cel; the FDA’s grant of a Priority Review Voucher; continued interest in our rare disease portfolio; our ability to enroll patients in clinical trials; the outcome of future meetings with and inspections from the FDA or other regulatory agencies, including those relating to preclinical programs; the ability to achieve or obtain necessary regulatory approvals; the impact of any changes in the financial markets and global economic conditions; risks associated with data analysis and reporting; and other risks disclosed in the Company’s most recent Annual Report on Form 10-K and subsequent periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws. ABEONA THERAPEUTICS INC. AND SUBSIDIARIESConsolidated Statements of Operations and Comprehensive Loss(In thousands, except share and per share amounts) For the years ended December 31, 2023 2022 Revenues: License and other revenues $3,500 $1,414 Expenses: Royalties 1,605 450 Research and development 31,091 28,965 General and administrative 19,004 17,256 Impairment of licensed technology — 1,355 Loss/(gain) on operating lease right-of-use assets (1,065) 2,511 Impairment of construction-in-progress — 1,792 Total expenses 50,635 52,329 Loss from operations (47,135) (50,915) Interest income 2,117 431 Interest expense (418) (736)Change in fair value of warrant liabilities (11,695) 11,383 Other income 2,943 141 Net loss $(54,188) $(39,696)Deemed dividends related to Series A and Series B Convertible Redeemable Preferred Stock — (3,782)Net loss attributable to Common Shareholders $(54,188) $(43,478) Basic and diluted loss per common share $(2.53) $(5.53) Weighted average number of common shares outstanding – basic and diluted 21,380,476 7,861,515 Other comprehensive income (loss): Change in unrealized gains (losses) related to available-for-sale debt securities 34 (99)Foreign currency translation adjustments 29 (3)Comprehensive loss $(54,125) $(43,580) ABEONA THERAPEUTICS INC. AND SUBSIDIARIESConsolidated Balance Sheets(In thousands, except share and per share amounts) December 31, 2023 December 31, 2022 ASSETS Current assets: Cash and cash equivalents$14,473 $14,217 Short-term investments 37,753 37,932 Restricted cash 338 338 Other receivables 2,444 188 Prepaid expenses and other current assets 729 424 Total current assets 55,737 53,099 Property and equipment, net 3,533 5,741 Operating lease right-of-use assets 4,455 5,331 Other assets 277 43 Total assets$64,002 $64,214 LIABILITIES AND STOCKHOLDERS' EQUITY Current liabilities: Accounts payable$1,858 $1,811 Accrued expenses 5,985 3,991 Current portion of operating lease liability 998 1,773 Current portion of payable to licensor 4,580 — Other current liabilities 1 204 Total current liabilities 13,422 7,779 Payable to licensor — 4,163 Long-term operating lease liabilities 4,402 5,854 Warrant liabilities 31,352 19,657 Total liabilities 49,176 37,453 Commitments and contingencies Stockholders' equity: Preferred stock - $0.01 par value; authorized 2,000,000 shares; No shares issued and outstanding as of December 31, 2023 and December 31, 2022, respectively — — Common stock - $0.01 par value; authorized 200,000,000 shares; 26,523,878 and 17,719,720 shares issued and outstanding as of December 31, 2023 and December 31, 2022, respectively 265 177 Additional paid-in capital 764,151 722,049 Accumulated deficit (749,524) (695,336)Accumulated other comprehensive loss (66) (129)Total stockholders' equity 14,826 26,761 Total liabilities and stockholders' equity$64,002 $64,214

Expanded Phase 1/2 gene therapy trial for Rett syndrome to inform future registrational study design; Company remains on track to share interim clinical data in 4Q:24 Strong financial position with runway into 2H:26 following reverse merger and private financing in December 2023 NEW YORK--(BUSINESS WIRE)-- Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced fourth quarter and full year 2023 financial results and highlighted recent corporate updates. “We started the year with strong execution in our Phase 1/2 NGN-401 gene therapy trial for female pediatric patients with Rett syndrome, meeting our goal to expand the trial and add a high-dose cohort. We believe this expanded dataset will support future regulatory discussions to align on the design of a potential registrational study,” said Rachel McMinn, Ph.D., Chief Executive Officer and Founder of Neurogene. “NGN-401 has the potential to be a best-in-class treatment option with a targeted route of administration to deliver full-length, functional copies of the MECP2 gene. We are pleased that NGN-401 has been well-tolerated in all three patients dosed, with no signs of overexpression-related toxicity, demonstrating the potential of our EXACT transgene regulation technology to constrain MECP2 levels within a narrow and therapeutically relevant range. As we look ahead, we remain on track for interim clinical data from the NGN-401 trial in the fourth quarter of this year.” Continued Dr. McMinn, “Our decision to execute a reverse merger, along with the concurrent private financing, provides us with cash runway into the second half of 2026, beyond key inflection points in our NGN-401 program and our NGN-101 program for CLN5 Batten disease. We look forward to sharing data from both programs later this year and continuing to advance our pipeline to enable an additional development candidate using EXACT in 2025.” Fourth Quarter 2023 and Recent Highlights, and Anticipated Milestones Phase 1/2 Trial of NGN-401 Gene Therapy for Treatment of Rett Syndrome As previously planned, amended the protocol, expanding the trial to include more patients in low-dose Cohort 1 and added a high-dose Cohort 2; these updates are expected to provide a robust dataset and inform the design of a future registrational study Remains on track to report interim clinical data from Cohort 1 in the fourth quarter of 2024; additional interim data, including from Cohort 2, are expected in the second half of 2025 Dosed the third patient early in the first quarter of 2024; removed the dosing stagger in Cohort 1, enabling the remaining patients in the cohort to be dosed in parallel Plans to complete enrollment in Cohort 1 in the second half of 2024; enrollment in Cohort 2 is expected to begin in the second quarter of 2024 Reported that NGN-401 has been generally well-tolerated and there have been no treatment-emergent or procedure-related serious adverse events or signs of overexpression-related toxicity observed in any patient Received NGN-401 clinical trial application approval from the United Kingdom (“UK”) Medicines and Healthcare products Regulatory Agency and working to on-board clinical sites in the UK Phase 1/2 Trial of NGN-101 Gene Therapy for Treatment of CLN5 Batten Disease Continuing enrollment in high-dose Cohort 3, and expects to provide interim clinical data and regulatory update in the second half of 2024 Additional Corporate Updates Expanded the leadership team with appointment of Julie Jordan, M.D., as Chief Medical Officer; Dr. Jordan brings more than 20 years of experience to Neurogene, including design and execution of clinical trials for central nervous system disorders and gene therapies Completed reverse merger and concurrent private placement of $95 million, and began trading on the Nasdaq Global Market under “NGNE” in mid-December 2023 Continues to expect to advance one additional product candidate, using EXACT technology, from the discovery stage into the clinic in 2025 Fourth Quarter and Full Year 2023 Financial Results Cash and Cash Equivalents: Cash, cash equivalents and investments as of December 31, 2023 were $197.2 million. Research & Development (“R&D”) Expenses: R&D expenses were $12.2 million and $44.4 million for the three and twelve months ended December 31, 2023, respectively, compared to $11.0 million and $47.5 million for the three and twelve months ended December 31, 2022, respectively. The decrease in R&D expenses for the twelve months ended December 31, 2023 was primarily driven by a decrease in manufacturing and development and pre-clinical costs offset by increased clinical trial costs. General & Administrative (“G&A”) Expenses: G&A expenses were $2.5 million and $11.2 million for the three and twelve months ended December 31, 2023, respectively, compared to $2.0 million and $9.0 million for the three and twelve months ended December 31, 2022, respectively. The increase in G&A expenses for the twelve months ended December 31, 2023 was primarily driven by an increase in personnel costs, increased professional and consulting fees related to the reverse merger, and increased insurance and information technology costs. Due to the timing of the closing of the reverse merger in mid-December 2023, additional transaction-related expenses are expected to be recognized in the first quarter of 2024. Net Income and Net Loss: Net income was $2.4 million and net loss was $36.3 million for the three and twelve months ended December 31, 2023, respectively, compared to net loss of $12.3 million and $55.2 million for the three and twelve months ended December 31, 2022, respectively. Net income for the three months ended December 31, 2023 included a one-time $16.4 million bargain purchase gain related to the reverse merger. Upcoming Events Stifel 2024 Virtual CNS Days: Management will participate in a fireside chat on Wednesday, March 20 at 9:30 a.m. ET About Neurogene The mission of Neurogene is to treat devastating neurological diseases to improve the lives of patients and families impacted by these rare diseases. Neurogene is developing novel approaches and treatments to address the limitations of conventional gene therapy in central nervous system disorders. This includes selecting a delivery approach to maximize distribution to target tissues and designing products to maximize potency and purity for an optimized efficacy and safety profile. The Company’s novel and proprietary EXACT transgene regulation platform technology allows for the delivery of therapeutic levels while limiting transgene toxicity associated with conventional gene therapy. Neurogene has constructed a state-of-the-art gene therapy manufacturing facility in Houston, Texas. CGMP production of NGN-401 was conducted in this facility and will support pivotal clinical development activities. For more information, visit . Cautionary Note Regarding Forward-Looking Statements Statements in this press release which are not historical in nature are intended to be, and hereby are identified as, forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current expectations and beliefs of the management of Neurogene, as well as assumptions made by, and information currently available to, management of Neurogene, including, but not limited to, statements regarding: the therapeutic potential and utility, efficacy and clinical benefits of NGN-401; the safety and tolerability pro NGN-401; trial designs, clinical development plans and timing for NGN-401, including anticipated timing of enrollment in and clinical trial results from the Company’s NGN-401 Phase 1/2 trial for Rett syndrome; effects of the removal of staggered dosing in Cohort 1; initiation of new clinical sites for NGN-401 in the UK; expected interactions with the FDA regarding NGN-101; nomination of additional preclinical product candidates; and our expected cash resources and liquidity. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” “on track,” and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Forward-looking statements are based on current beliefs and assumptions that are subject to risks, uncertainties and assumptions that are difficult to predict with regard to timing, extent, likelihood, and degree of occurrence, which could cause actual results to differ materially from anticipated results and many of which are outside of Neurogene’s control. Such risks, uncertainties and assumptions include, among other things: risks related to the timing and success of enrolling patients in the expanded cohort of our Phase 1/2 clinical trial of NGN-401 for the treatment of Rett syndrome; the expected timing and results of dosing of patients in our clinical trials, including NGN-401; the potential that we may not be able to expand our Phase 1/2 clinical trial of NGN-401 for the treatment of Rett syndrome into the UK based on a variety of factors, including but not limited to any decisions of regulatory authorities, costs of expanding the trial in the UK, the availability of suitable clinical test sites, and the ability to enroll patients in the UK or other reasons; the potential for negative impacts to patients resulting from using a higher dose of NGN-401 in Cohort 2 of the Phase 1/2 clinical trial for the treatment of Rett syndrome; the risk that we may not be able to report our data on the predicted timeline; our limited operating history; the risk that we may not be able to raise adequate additional capital to finance our operations, complete our clinical trials and commercialize our products; risks related to our ability to obtain regulatory approval for, and ultimately commercialize, our product candidates, including NGN-401; risks related to the outcome of non-clinical testing and early clinical trials for our product candidates, including the ability of those trials to satisfy relevant governmental or regulatory requirements; risks related to our limited experience in designing clinical trials and lack of experience in conducting clinical trials; and other risks and uncertainties identified under the heading "Risk Factors" included in our Annual Report on Form 10-K for the year ended December 31, 2023, filed with the Securities and Exchange Commission (“SEC”) on March 18, 2024, and other filings that the Company has made and may make with the SEC in the future. Nothing in this communication should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that the contemplated results of any such forward-looking statements will be achieved. Forward-looking statements in this communication speak only as of the day they are made and are qualified in their entirety by reference to the cautionary statements herein. Except as required by applicable law, Neurogene undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise. This communication contains hyperlinks to information that is not deemed to be incorporated by reference into this communication. - Financial Tables Follow - Neurogene Inc. Condensed Consolidated Balance Sheet Data (In thousands of U.S. dollars) December 31, 2023 December 31, 2022 Assets Cash and cash equivalents $ 148,210 $ 82,021 Other current assets 52,138 2,698 Non-current assets 22,225 24,546 Total assets $ 222,573 $ 109,265 Liabilities Current liabilities $ 22,973 $ 6,651 Non-current liabilities 13,576 3,987 Total liabilities 36,549 10,638 Total convertible preferred stock — 244,366 Stockholders' equity (deficit) 186,024 (145,739) Total liabilities, convertible preferred stock and stockholders' equity (deficit) $ 222,573 $ 109,265 Neurogene Inc. Condensed Consolidated Statements of Operations (In thousands of U.S. dollars, except share information) Year Ended December 31, 2023 2022 Operating expenses: Research and development $ 44,394 $ 47,505 General and administrative 11,189 9,012 Total operating expenses 55,583 56,517 Loss from operations (55,583) (56,517) Other income, net 2,911 1,328 Bargain purchase gain 16,355 — Net loss $ (36,317) $ (55,189) Pre-Merger Post- Merger Pre-Merger Per share information (1): (a) (b) (c) (a) (b) Net income (loss) per share, basic $ (117.28) $ — $ 27.76 $ (139.88) $ — Weighted-average shares outstanding used in computing net income (loss) per share, basic 426,097 — 491,867 394,533 — Net income (loss) per share, diluted $ (117.28) $ — $ 2.93 $ (139.88) $ — Weighted-average shares outstanding used in computing net income (loss) per share, diluted 426,097 — 4,656,947 394,533 — (1) On December 18, 2023, the Company completed its reverse merger, which among other things, resulted in Neurogene OpCo merging with and into a wholly owned subsidiary of Neoleukin Therapeutics, Inc. As the earnings per share information for the pre-merger period is not comparable to the earnings per share information for the post-merger period, the earnings per share information is being presented separately for these periods. See Note 3, Net Income (Loss) Per share, for additional information. (a) Presents information for the pre-merger period for Class A common stock. The pre-merger period is January 1, 2023 through December 17, 2023 for the year ended December 31, 2023 and the full fiscal year ended December 31, 2022. (b) Presents information for the pre-merger period for Class B common stock. The pre-merger period is January 1, 2023 through December 17, 2023 for the year ended December 31, 2023 and the full fiscal year ended December 31, 2022. (c) Presents information for the post-merger period for common stock. The post-merger period is December 18, 2023 through December 31, 2023.