Spinal Muscular Atrophy

- Enrollment completed in Phase 1b/2a clinical trial of AJ201 for spinal and bulbar muscular atrophy; topline data expected in second quarter of 2024 - - Presented positive BAER-101 preclinical data at American Epilepsy Society (AES) and American Society for Experimental Neurotherapeutics (ASENT) Annual Meetings and in publication Drug Development Research - - Final agreement reached with FDA on study design and analysis approach for Phase 3 safety study of IV tramadol – MIAMI, March 18, 2024 (GLOBE NEWSWIRE) -- Avenue Therapeutics, Inc. (Nasdaq: ATXI) (“Avenue” or the “Company”), a specialty pharmaceutical company focused on the development and commercialization of therapies for the treatment of neurologic diseases, today reported financial results and recent corporate highlights for the year ended December 31, 2023. “We made considerable progress across our pipeline of differentiated neurologic therapies in 2023," said Alexandra MacLean, M.D., Chief Executive Officer of Avenue. “Avenue demonstrated tremendous execution capabilities with our lead clinical program AJ201, a potential first-in-class treatment for spinal and bulbar muscular atrophy (SBMA), also known as Kennedy’s Disease. Within nine months of acquiring the rights to AJ201 in March 2023, we dosed the first patient in the Phase 1b/2a clinical trial, and we completed trial enrollment of 25 SBMA patients across six sites in the U.S. We remain on track to report topline data for AJ201 in the second quarter of 2024, an incredibly exciting milestone for both the Company and patients suffering from SBMA, a debilitating rare neuromuscular disorder with no approved treatments in the U.S. Additionally, we have progressed BAER-101 for the treatment of epilepsy by presenting preclinical data from a translational animal model in multiple peer-reviewed forums. We have also advanced IV tramadol, reaching final agreement with the U.S. Food and Drug Administration (FDA) on the safety study and statistical analysis approach for the final Phase 3 study. Pending additional financing, we look forward to progressing BAER-101 and IV tramadol for patients facing great unmet need. We look forward to another productive year in 2024 as we continue to make significant strides across our clinical pipeline and advance our mission of providing impactful therapies to patients suffering from neurologic diseases.” Recent Corporate Highlights: AJ201 (Nrf1 and Nrf2 activator, androgen receptor degradation enhancer for SBMA) In January 2024, Avenue completed enrollment in the Phase 1b/2a clinical trial of AJ201 for the treatment of SBMA. The 12-week, multicenter, randomized, double-blind Phase 1b/2a clinical trial of AJ201 enrolled 25 patients randomly assigned to AJ201 (600 mg/day) or placebo. The primary endpoint of the study is to assess safety and tolerability of AJ201 in subjects with clinically and genetically defined SBMA. Topline data for the Phase 1b/2a study are anticipated in the second quarter of 2024. More information about this study can be found at ClinicalTrials.gov (Identifier: NCT05517603). BAER-101 (GABAA α2/3 positive allosteric modulator) Avenue presented preclinical results for BAER-101, a potentially best-in-class selective GABAA α2,3 positive allosteric modulator, in three scientific peer-reviewed settings, including the American Epilepsy Society (AES) Annual Meeting in December 2023, the publication Drug Development Research in February 2024 and the American Society for Experimental Neurotherapeutics (ASENT) Annual Meeting in March 2024. The in vivo data showcase BAER-101’s ability to significantly suppress seizures using the SynapCell’s Genetic Absence Epilepsy Rat from Strasbourg (“GAERS”) model of epilepsy. BAER-101 fully suppressed seizure activity in the GAERS model with a minimal effective dose of 0.3 mg/kg, PO, and the effect was fast in onset and stable throughout the duration of testing. The data also demonstrated BAER-101’s ability to selectively target GABAA α2 and α3 subtypes more than α1 and α5, potentially improving anti-convulsant and anxiolytic activity while minimizing the risk of tolerance and abuse associated with existing treatments in this drug class. Subject to obtaining the necessary financing, which could be provided through a strategic partnership, Avenue plans to initiate a Phase 2a clinical trial of BAER-101 to further study its anti-seizure properties in patients with common or rare epilepsies. IV Tramadol In January 2024, Avenue reached final agreement with the FDA on the safety study protocol and statistical analysis approach for the Phase 3 study for intravenous (“IV”) tramadol, which is in development for the treatment of acute post-operative pain in a medically supervised setting. The final non-inferiority study is designed to assess the theoretical risk of opioid-induced respiratory depression related to opioid stacking on IV tramadol compared to IV morphine. The study will randomize approximately 300 post bunionectomy patients to IV tramadol or IV morphine for pain relief administered during a 48-hour post-operative period. Patients will have access to IV hydromorphone, a Schedule II opioid, for rescue of breakthrough pain. Pending additional financing, Avenue aims to initiate the Phase 3 safety study as soon as feasible. The Company believes that the study can be completed and submitted to the FDA within 12 months of the study’s initiation. General Corporate In March 2024, Avenue announced that the Nasdaq Hearings Panel granted the Company’s request for an extension to evidence compliance with all applicable criteria for continued listing on The Nasdaq Capital Market, including the $1.00 bid price and $2.5 million stockholders’ equity requirements, through May 20, 2024. 2023 Financial Results: Cash Position: As of December 31, 2023, cash and cash equivalents totaled $1.8 million, compared to $6.7 million at December 31, 2022, a decrease of $4.9 million. In January 2024, the Company completed a warrant inducement transaction resulting in $5.0 million in gross proceeds.R&D Expenses: Research and development expenses for the full year 2023 were $6.1 million, compared to $2.7 million in 2022. Additionally, there was $4.2 million in expense for the acquisition of the AJ201 license in 2023.G&A Expenses: General and administrative expenses for the full year 2023 were $4.2 million, compared to $5.3 million in 2022.Net Loss: Net loss for the full year 2023 was $10.5 million, or $0.98 per share, compared to a net loss of $3.6 million, or $1.63 per share, in 2022. About Avenue TherapeuticsAvenue Therapeutics, Inc. (Nasdaq: ATXI) is a specialty pharmaceutical company focused on the development and commercialization of therapies for the treatment of neurologic diseases. It is currently developing three assets including AJ201, a first-in-class asset for spinal and bulbar muscular atrophy, BAER-101, an oral small molecule selective GABAA α2, α3 receptor positive allosteric modulator for CNS diseases, and IV tramadol, which is in Phase 3 clinical development for the management of acute postoperative pain in adults in a medically supervised healthcare setting. Avenue is headquartered in Miami, FL and was founded by Fortress Biotech, Inc. (Nasdaq: FBIO). For more information, visit www.avenuetx.com. Forward-Looking StatementsThis press release contains predictive or “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other than statements of current or historical fact contained in this press release, including statements that express our intentions, plans, objectives, beliefs, expectations, strategies, predictions or any other statements relating to our future activities or other future events or conditions are forward-looking statements. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “will,” “should,” “would” and similar expressions are intended to identify forward-looking statements. These statements are based on current expectations, estimates and projections made by management about our business, our industry and other conditions affecting our financial condition, results of operations or business prospects. These statements are not guarantees of future performance and involve risks, uncertainties and assumptions that are difficult to predict. Therefore, actual outcomes and results may differ materially from what is expressed or forecasted in, or implied by, the forward-looking statements due to numerous risks and uncertainties. Factors that could cause such outcomes and results to differ include, but are not limited to, risks and uncertainties arising from: the fact that we currently have no drug products for sale and that our success is dependent on our product candidates receiving regulatory approval and being successfully commercialized; the possibility that serious adverse or unacceptable side effects are identified during the development of our current or future product candidates, such that we would need to abandon or limit development of some of our product candidates; our ability to successfully develop, partner, or commercialize any of our current or future product candidates including AJ201, IV tramadol, and BAER-101; the substantial doubt raised about our ability to continue as a going concern, which may hinder our ability to obtain future financing; the significant losses we have incurred since inception and our expectation that we will continue to incur losses for the foreseeable future; our need for substantial additional funding, which may not be available to us on acceptable terms, or at all, which unavailability of could force us to delay, reduce or eliminate our product development programs or commercialization efforts; our reliance on third parties for several aspects of our operations; our reliance on clinical data and results obtained by third parties that could ultimately prove to be inaccurate, or unreliable, or unacceptable to regulatory authorities; the possibility that we may not receive regulatory approval for any or all of our product candidates, or that such approval may be significantly delayed due to scientific or regulatory reasons; the fact that even if one or more of our product candidates receives regulatory approval, they will remain subject to substantial regulatory scrutiny; the effects of current and future laws and regulations relating to fraud and abuse, false claims, transparency, health information privacy and security, and other healthcare laws and regulations; the effects of competition for our product candidates and the potential for new products to emerge that provide different or better therapeutic alternatives for our targeted indications; the possibility that the government or third-party payors fail to provide adequate coverage and payment rates for our product candidates or any future products; our ability to establish sales and marketing capabilities or to enter into agreements with third parties to market and sell our product candidates; our exposure to potential product liability claims; related to the protection of our intellectual property and our potential inability to maintain sufficient patent protection for our technology and products; our ability to maintain compliance with the obligations under our intellectual property licenses and funding arrangements with third parties, without which licenses and arrangements we could lose rights that are important to our business; the fact that Fortress Biotech, Inc. controls a majority of the voting power of our outstanding capital stock and has rights to receive significant share grants annually;; and those risks discussed in our filings which we make with the SEC. Any forward-looking statements speak only as of the date on which they are made, and we undertake no obligation to publicly update or revise any forward-looking statements to reflect events or circumstances that may arise after the date of this press release, except as required by applicable law. Investors should evaluate any statements made by us in light of these important factors. Contact: Jaclyn JaffeAvenue Therapeutics, Inc. (781) 652-4500ir@avenuetx.com AVENUE THERAPEUTICS, INC.Consolidated Balance Sheets($ in thousands, except for share and per share amounts) December 31, December 31, 2023 2022 ASSETS Current assets: Cash and cash equivalents$1,783 $6,708 Prepaid expenses and other current assets 67 137 Total assets$1,850 $6,845 LIABILITIES AND STOCKHOLDERS’ EQUITY Current liabilities: Accounts payable and accrued expenses$287 $949 Accounts payable and accrued expenses - related party 323 21 Warrant liability 586 2,609 Total current liabilities 1,196 3,579 Total liabilities 1,196 3,579 Commitments and Contingencies Stockholders’ equity Preferred stock ($0.0001 par value), 2,000,000 shares authorized Class A Preferred stock, 250,000 shares issued and outstanding as of December 31, 2023 and 2022, respectively — — Common stock ($0.0001 par value) Common shares, 75,000,000 shares authorized and 25,597,622 shares issued and outstanding as of December 31, 2023; and 20,000,000 shares authorized and 4,773,841 shares issued and outstanding as of December 31, 2022 3 — Additional paid-in capital 92,507 84,456 Accumulated deficit (90,928) (80,551)Total stockholders’ equity attributed to the Company 1,582 3,905 Non-controlling interests (928) (639)Total stockholders’ equity 654 3,266 Total liabilities and stockholders’ equity$1,850 $6,845 AVENUE THERAPEUTICS, INC.Consolidated Statements of Operations($ in thousands, except for share and per share amounts) For the Years Ended December 31, December 31, 2023 2022 Operating expenses Research and development$6,131 $2,698 Research and development - licenses acquired 4,230 — General and administrative 4,179 5,345 Loss from operations (14,540) (8,043) Interest income (126) (20)Financing costs – warrant liabilities 332 1,160 Change in fair value of warrant liabilities (4,258) (5,580)Net loss$(10,488) $(3,603) Net loss attributable to non-controlling interests (111) (51)Net loss attributable to common stockholders$(10,377) $(3,552) Net loss per common share attributable to common stockholders, basic and diluted$(0.98) $(1.63) Weighted average number of common shares outstanding, basic and diluted 10,591,636 2,185,159

NEW YORK, March 18, 2024 /PRNewswire/ -- Technavio categorizes the global gene therapy market size as estimated to increase by USD 4,613.9 million between 2022 and 2027. The market's growth momentum will be progressing at a CAGR of 19.91% during the forecast period. During the forecast period, North America is anticipated to drive 39% of global market growth, as per Technavio's analysts. The US, particularly, emerges as a promising investment hub within the region, supported by substantial funding from entities like the National Institutes of Health (NIH) and the Biomedical Advanced Research and Development Authority (BARDA) to emerging players in the market. This influx of government and private sector investment is propelling the development of innovative gene therapies, with 18 clinical trials and 17 biotech startups underway. Such financial backing is poised to significantly stimulate market demand in the coming years. Continue Reading Technavio has announced its latest market research report titled Global Gene Therapy Market 2023-2027 Discover some insights on market size historic data (2017 to 2021) and Forecast (2023 to 2027) before buying the full report - Request a sample report Technavio has segmented the market based on therapy areas (oncology, CNS, ophthalmology, rare diseases, and others), and delivery mode (in vivo and ex vivo). The market growth in the oncology segment will be significant during the forecast period. The global oncology drugs market has a huge unmet demand for the treatment of various indications. Also, the increasing prevalence of cancer indications has led pharmaceutical companies and research institutions to conduct extensive research to develop drugs for these indications. The lack of efficacy of small molecules and biomolecules has led companies to move towards effective therapeutic options such as gene therapy. Such factors will increase the segment growth during the forecast period. For insights on global, regional, and country-level parameters with growth opportunities from 2017 to 2027 - Download a Sample Report Key Companies The global gene therapy market is fragmented, with the presence of several global as well as regional vendors. A few prominent vendors that offer gene therapy in the market are Abeona Therapeutics Inc., Adaptimmune Therapeutics plc, Adverum Biotechnologies Inc., Amgen Inc., Astellas Pharma Inc., Biogen Inc., bluebird bio Inc., Editas Medicine Inc., ElevateBio, F. Hoffmann La Roche Ltd., Generation Bio Co., Gilead Sciences Inc., Novartis AG, Orchard Therapeutics Plc, Poseida Therapeutics Inc., Sangamo Therapeutics Inc., Sibiono GeneTech Co. Ltd., Syncona Ltd., uniQure NV, and Voyager Therapeutics Inc. and others. Abeona Therapeutics Inc.: The company offers gene therapy solutions such as Phase 3 VIITAL Clinical Trial of EB 101. Amgen Inc.: The company offers gene therapy solutions such as CAR T cell immunotherapies. Astellas Pharma Inc.: The company offers gene therapy solutions such as AT132, AT845, AT466. Biogen Inc.: The company offers gene therapy solutions such as GTxAU. The growth of the market is driven by the increase in special drug designations. Most gene therapy programs have been granted orphan drug designation, fast track designation, or breakthrough designation by the U.S. FDA, EMA, and other regulatory bodies for the treatment of certain diseases. In addition, orphan drug status will also help the company with tax incentives in its next research. Such advantages of specialty drug designations will drive the growth of the market during the forecast period. Growing research in gene therapy for CVDs and orphan diseases is one of the key market trends that is contributing to the market growth. High treatment cost is one of the factors hindering the gene therapy market growth. Driver, Trend & Challenges are the factor of market dynamics which states about consequences & sustainability of the businesses, find some insights from a sample report! Analyst Review Gene therapy stands at the forefront of medical innovation, offering promising solutions for a myriad of genetic disorders ranging from atrophy to muscular dystrophy. As the world grapples with health challenges like the COVID-19 pandemic, companies like Applied Genetic Technologies Corporation and Benitec Biopharma in countries like Australia and Belgium are actively engaged in pioneering gene therapy research to combat such conditions. Beta thalassemia, sickle cell disease, and spinal muscular atrophy represent just a fraction of the genetic issues that gene therapy aims to address. With the advent of breakthrough treatments like betibeglogene autotemcel and onasemnogene abeparvovec, patients suffering from these debilitating conditions now have newfound hope. The gene therapy market, bolstered by advancements in CRISPR Therapeutics and lentivirus technology, is witnessing exponential growth. As companies like Pfizer, Cellectis, and Novartis Gene Therapies invest in research and development, the market is projected to expand further, especially in regions like California and Massachusetts, known for their biotechnology hubs. However, this growth isn't without challenges. Regulatory bodies like ClinicalTrials.gov, the FDA, and the International Organization for Standardization (ISO 9000) play pivotal roles in ensuring the safety and efficacy of gene therapies. Striking a balance between innovation and compliance with regulations such as the California Consumer Privacy Act and the General Data Protection Regulation remains paramount. One of the critical factors driving the gene therapy market is the increasing prevalence of genetic disorders worldwide. From hereditary illnesses to congenital diseases, the demand for innovative treatments continues to rise. Moreover, with advancements in gene editing and therapeutic gene delivery systems, the potential to target specific cells and protein molecules offers new avenues for treatment. Venture capital financing and partnerships between academia and industry players further fuel research efforts. Companies like Lonza Group and Oxford Biomedica contribute to the manufacturing and logistics aspects of gene therapy, ensuring efficient drug delivery and scalability. In conclusion, the gene therapy market holds immense promise in addressing genetic disorders and improving patient outcomes. As the field continues to evolve, collaboration, adherence to regulations, and a focus on patient-centric approaches will be essential in unlocking the full potential of gene therapy to tackle the most challenging medical conditions of our time. Download a Sample Report Market Overview The Gene Therapy Market is witnessing exponential growth globally, with companies like Applied Genetic Technologies Corporation and Benitec Biopharma leading the charge. This revolutionary approach offers hope for conditions such as atrophy and beta thalassemia, providing targeted solutions to genetic disorders. Recent advancements include Betibeglogene autotemcel, a promising treatment for beta-thalassemia pioneered by CRISPR Therapeutics. Biomedicine is at the forefront of this transformative field, driving innovation and clinical trials. Amidst the COVID-19 pandemic, gene therapy research continues unabated, with countries like Australia and Belgium actively contributing to the global effort. As the market expands, gene therapy emerges as a beacon of hope for patients worldwide, offering personalized treatments and potential cures. Download a Sample Report Related Reports: The cell and gene therapy market size is expected to increase by USD 9.97 billion from 2021 to 2026, and the market's growth momentum will accelerate at a CAGR of 20.15%. The cell therapy market size is expected to increase by USD 21.061 billion from 2021 to 2026, and the market's growth momentum will accelerate at a CAGR of 56.79%. TOC Executive Summary Market Landscape Market Sizing Historic Market Size Five Forces Analysis Market Segmentation by Therapy Areas market Segmentation by Delivery Mode Customer Landscape Geographic Landscape Drivers, Challenges, and Trends Company Landscape Company Analysis Appendix About US Technavio is a leading global technology research and advisory company. Their research and analysis focuses on emerging market trends and provides actionable insights to help businesses identify market opportunities and develop effective strategies to optimize their market positions. With over 500 specialized analysts, Technavio's report library consists of more than 17,000 reports and counting, covering 800 technologies, spanning across 50 countries. Their client base consists of enterprises of all sizes, including more than 100 Fortune 500 companies. This growing client base relies on Technavio's comprehensive coverage, extensive research, and actionable market insights to identify opportunities in existing and potential markets and assess their competitive positions within changing market scenarios. Contact Technavio Research Jesse Maida Media & Marketing Executive US: +1 844 364 1100 UK: +44 203 893 3200 Email: [email protected] Website: SOURCE Technavio

Pictured: Novartis' office in Germany/iStock, TBE Novartis is acquiring IFM Due, a subsidiary of privately held IFM Therapeutics, with the biotech getting $90 million in an upfront payment and remaining eligible for up to $745 million in milestone payments, the companies announced Wednesday. This is not the first deal between the companies. In September 2019, IFM forged an option and collaboration deal with Novartis, which saw the Swiss pharma make fixed payments to finance R&D costs for the program in exchange for the option to acquire the company. Under Wednesday’s acquisition, Novartis now has the full rights to IFM Due’s portfolio of STING antagonists, which can potentially treat inflammation-driven diseases “characterized by excessive interferon” and other pro-inflammatory cytokine signaling, according to the announcement. “The acquisition of IFM Due represents the culmination of a highly productive, four-year preclinical collaboration between Novartis and IFM to develop novel small-molecule STING inhibitors with the potential to treat a spectrum of inflammatory diseases,” Richard Siegel, global head of immunology research at Novartis, said in a statement. “We are excited to advance IFM Due’s STING program and leverage our deep expertise in inflammation science to bring forward transformative medicines that address major unmet patient needs.” Other deals between Novartis and IFM have also been made. In April 2019, the Swiss pharma acquired subsidiary IFM Tre, paying $310 million upfront and another $1.26 billion in various milestones. That deal was focused on developing NLRP3 antagonists for treating inflammatory diseases. Novartis has made other acquisition and licensing moves this year. In February 2024, it bought German biotech MorphoSys for around $2.9 billion, expanding its oncology offerings. In January 2024, Novartis entered a strategic collaboration and licensing deal with Voyager Therapeutics, paying $100 million upfront and $1.2 billion in potential milestones. The deal will see the development of gene therapies for Huntington’s disease and spinal muscular atrophy. Tyler Patchen is a staff writer at BioSpace. You can reach him at tyler.patchen@biospace.com. Follow him on LinkedIn.