Epstein-Barr Virus Infections

VIP236, first-in-class small molecule-drug conjugate (SMDC), preliminary Phase 1 data and update on pipeline progress will be presented by management at a virtual investor event on April 8 at 2:00 PM PT Phase 1 trial ongoing for VIP943, a best-in-class anti-CD123 antibody-drug conjugate (ADC); preliminary data anticipated on or around the 2024 European Hematology Association (EHA)Annual Meeting; in addition, pharmacokinetic data will be shared at the 2024 American Association of Cancer Research (AACR) Annual Meeting Expected cash runway into early Q3 2024 PALO ALTO, Calif., March 29, 2024 (GLOBE NEWSWIRE) -- Vincerx Pharma, Inc. (Nasdaq: VINC), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, today reported financial results for the fourth quarter and full year ended December 31, 2023, and provided a corporate update. “The last calendar year was pivotal for Vincerx,” said Ahmed Hamdy, M.D., Chief Executive Officer. “Having progressed three clinical programs, including VIP236 and VIP943, both developed with our VersAptxTM platform, and enitociclib, we firmly established ourselves as a clinical-stage cancer therapeutics company with novel product candidates that aim to improve safety and efficacy over traditional chemotherapy.” Raquel Izumi, Ph.D., Chief Operations Officer, added, “VIP236, our first-in-class SMDC, is in a Phase 1 dose-escalation trial for advanced and metastatic solid tumors. To date, we have dosed 20 patients across two dosing schedules and results from the once every 3-week dosing schedule continue to show a strong safety pro dose-dependent clinical activity. We are excited to share more details regarding the preliminary Phase 1 data during the 2024 AACR Annual Meeting.” Dr. Izumi continued, “VIP943, our best-in-class anti-CD123 ADC, is in a Phase 1 dose-escalation trial in hematologic malignancies. Enrollment in the third cohort is underway with a total of 9 patients dosed. Preliminary pharmacokinetic data from the first two cohorts show very little payload in circulation, which aligns with the favorable safety pro in nonhuman primates in our preclinical studies. We look forward to sharing pharmacokinetic data for VIP943 during AACR and more details regarding the preliminary Phase 1 data on or around EHA. Enitociclib, our highly selective CDK9 inhibitor, in a Phase 1 dose-escalation study with the National Institutes of Health (NIH), has signaled encouraging efficacy in Phase 1 combination studies in peripheral T-cell lymphoma (PTCL) and double-hit B-cell lymphoma (DLBCL). Earlier this year, we reported partial responses at doses below expected efficacy levels, highlighting the drugs synergistic potential with other treatments.” Dr. Hamdy concluded, “We entered 2024 with strong momentum and remain focused on aggressively advancing our programs and maximizing the value of our next-generation VersAptx platform.” FOURTH QUARTER AND FULL YEAR 2023 CLINICAL PROGRAM HIGHLIGHTS VersAptx™ Platform VersAptx is Vincerx’s versatile and adaptable, next-generation bioconjugation platform. The modular nature of this platform enables the combination of different targeting, linker, and payload technologies to develop bespoke bioconjugates to address different cancer biologies. Recent preclinical data demonstrated the ability of our legumain linker + KSPi payload with CellTrapper effector chemistry to enhance the potency of TRODELVY® and ENHERTU®, two marketed ADCs, by conjugating TRODELVY's TROP2 and ENHERTU's HER2 antibodies with our effector chemistry. The results of this study demonstrated a significant improvement in the potency of both drugs, with TRODELVY potency amplified by a factor of 20 and ENHERTU potency by a factor of 8. Vincerx published a manuscript in Frontiers in Pharmacology titled, “Neutrophil elastase as a versatile cleavage enzyme for activation of αvβ3 integrin-targeted small molecule drug conjugates with different payload classes in the tumor microenvironment.” Vincerx presented a preclinical poster at the 2023 AACR Annual Meeting demonstrating the promise of VIP924, a first-in-class CXCR5-targeted ADC from the VersAptx platform. VIP236 VIP236 is a αVβ3 SMDC conjugated to an optimized camptothecin (CPT) from the VersAptx platform. VIP236 is a first-in-class product candidate designed to deliver its optimized CPT payload directly to tumor tissues to overcome the chemotherapy-related side effects and transporter liabilities of this well-established class of anticancer drugs. Preclinical studies have shown 11 times more optimized CPT is delivered to the cancerous tissues than found circulating in the blood. Additionally, the active component of VIP236 is designed to bypass cancer resistance mechanisms. The favorable characteristics of VIP236, including improved linker attachment, stable solubility, and tumor-specific payload release, were featured in a peer-reviewed publication titled, “Unleashing the Potential of Camptothecin: Exploring Innovative Strategies for Structural Modification and Therapeutic Advancements,” by Zheng Chen in the March 14, 2024 issue of Journal of Medicinal Chemistry. To date, the VIP236 Phase 1 dose-escalation trial (NTC05371054) has enrolled 20 patients with advanced or metastatic cancer unresponsive to standard therapies. Vincerx will present more details around the preliminary Phase 1 data at the upcoming 2024 AACR Annual Meeting, accompanied by a virtual investor event to review the data and provide a pipeline update on April 8 at 2:00 PM PT. The event can be accessed through the Investors Page on the Vincerx website. VIP943 VIP943 is a novel CD123-targeted antibody-drug conjugate (ADC) from the VersAptx platform. In August 2023, Vincerx received IND clearance for a Phase 1 dose-escalation trial of VIP943 in relapsed/refractory acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), and B-cell acute lymphoblastic leukemia (B-ALL) (NCT06034275). In September 2023, Vincerx announced the dosing of its first patient in that study. Enrollment in the third cohort is underway, with a total of 9 patients dosed. Preliminary pharmacokinetic results continue to show very little payload circulating in the blood, validating the favorable safety pro in nonhuman primates in preclinical studies. Vincerx will present more details regarding the preliminary Phase 1 data for VIP943 on or around the 2024 EHA Annual Meeting. Enitociclib Enitociclib is a highly selective CDK9 inhibitor designed to block the activation of RNA polymerase II, leading to the reduction of oncogenes MYC and MCL1. In 2023, Vincerx presented preclinical data supporting the continued advancement of enitociclib at multiple medical congresses. Vincerx also published a manuscript titled, “Enitociclib, a Selective CDK9 Inhibitor, Induces Complete Regression of MYC+ Lymphoma by Downregulation of RNA Polymerase II Mediated Transcription” in Cancer Research Communications, an AACR journal. Enitociclib is currently in a Phase 1 dose-escalation trial (NTC05371054) evaluating the combination of enitociclib, venetoclax, and prednisone in DLBCL and PTCL. This trial is being conducted in collaboration with the NIH. Earlier this year, Vincerx announced that 2 out of 3 patients with PTCL had a partial response (PR). The first patient saw a 91% reduction in their tumor burden, while the second patient saw an 86% reduction in pulmonary lesions and resolution of skin lesions. In addition, 1 out of 2 patients with DH-DLBCL had a PR after one cycle of treatment, seeing an 80% reduction in tumor burden. These responses occurred at doses below expected efficacy levels. VIP924 VIP924 is a first-in-class CXCR5-targeted ADC from the VersAptx platform. Vincerx presented preclinical data at the 2023 AACR Annual Meeting demonstrating significant activity in patient-derived xenograft (PDX) lymphoma mouse models. Vincerx shared preclinical data at the 2023 ASH Annual Meeting showing superior activity and safety compared with commercially available B-cell targeted ADCs. IND application anticipated in late 2025 or early 2026, pending funding. FOURTH QUARTER AND FULL YEAR 2023 FINANCIAL RESULTS Vincerx had $12.8 million in cash and cash equivalents as of December 31, 2023, as compared to $52.5 million in cash, cash equivalents and marketable securities as of December 31, 2022. Based on its current business plans and assumptions, Vincerx believes its available capital will be sufficient to meet its operating requirements into early third quarter of 2024. Research and development (R&D) expenses for the fourth quarter and full year 2023 were $3.7 million and $29.0 million, respectively, as compared to $11.5 million and $49.8 million, respectively, for each of the same periods in 2022. The year over year decrease of approximately $20.9 million is primarily the result of decreases in third party service and supplier expenses, including manufacturing services associated with our ADC program of approximately $9.7 million and clinical services of approximately $2.6 million, as well as decreases in expenses related to headcount, including declines in stock-based compensation expense of approximately $3.3 million and employee salaries of approximately $3.1 million as a result of our headcount reduction in June 2022. General and administrative (G&A) expenses for the fourth quarter and full year 2023 were $1.8 million and $13.6 million, respectively, as compared to $4.0 million and $18.9 million for the same periods in 2022. The year over year decrease of approximately $5.2 million was primarily driven by decreases in stock-based compensation expense of $2.8 million, professional services of $1.4 million, and a decrease in expenses related to headcount of $0.7 million. For the fourth quarter and full year 2023, Vincerx reported a net loss of $4.9 million, or $0.23 per share, and a net loss of $40.2 million, or $1.89 per share, respectively. For the fourth quarter and full year 2022, Vincerx reported a net loss of $13.8 million, or $0.65 per share, and a net loss of $63.0 million, or $3.00 per share, respectively. About Vincerx Pharma, Inc. Vincerx Pharma, Inc. is a clinical-stage biopharmaceutical company committed to developing differentiated and novel therapies to address the unmet medical needs of patients with cancer. Vincerx has assembled a seasoned management team with a proven track record of successful oncology drug development, approvals, and value creation. Vincerx’s diverse pipeline consists of the next-generation antibody-drug conjugate, VIP943, in Phase 1; small molecule-drug conjugate, VIP236, in Phase 1; preclinical antibody-drug conjugate, VIP924; CDK9 inhibitor, enitociclib, in an NIH-sponsored Phase 1; and VersAptx, its versatile and adaptable, next-generation bioconjugation platform. Vincerx is based in Palo Alto, California, and has a research facility in Monheim, Germany. For more information, please visit and follow Vincerx on LinkedIn. Forward-Looking Statement This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended, that are intended to be covered by the “safe harbor” created by those sections. Forward-looking statements, which are based on certain assumptions and describe future plans, strategies, expectations and events, can generally be identified by the use of forward-looking terms such as “believe,” “expect,” “may,” “will,” “should,” “would,” “could,” “suggest,” “seek,” “intend,” “plan,” “goal,” “potential,” “on-target,” “on track,” “project,” “estimate,” “anticipate,” or other comparable terms. All statements other than statements of historical facts included in this press release are forward-looking statements. Forward-looking statements include, but are not limited to, Vincerx’s business model, cash runway, pipeline, strategy, timeline, product candidates and attributes, and preclinical and clinical development, timing, and results. Forward-looking statements are neither historical facts nor assurances of future performance or events. Instead, they are based only on current beliefs, expectations, and assumptions regarding future business developments, future plans and strategies, projections, anticipated events and trends, the economy, and other future conditions. Forward-looking statements are subject to inherent uncertainties, risks, and changes in circumstances that are difficult to predict, many of which are outside Vincerx’s control. Actual results, conditions, and events may differ materially from those indicated in the forward-looking statements. Therefore, you should not rely on any of these forward-looking statements. Important factors that could cause actual results, conditions, and events to differ materially from those indicated in the forward-looking statements include, but are not limited to, general economic, financial, legal, political, and business conditions; risks associated with preclinical or clinical development and trials, including those conducted prior to Vincerx’s in-licensing; failure to realize the benefits of Vincerx’s license agreement with Bayer; risks related to the timing of expected business and product development milestones; changes in the assumptions underlying Vincerx’s expectations regarding its future business or business model; Vincerx’s ability to successfully develop and commercialize product candidates; Vincerx’s capital requirements, availability and uses of capital, and cash runway; and the risks and uncertainties set forth in Form 10-K for the year ended December 31, 2023 and other reports filed with the Securities and Exchange Commission by Vincerx. Forward-looking statements speak only as of the date hereof, and Vincerx disclaims any obligation to update any forward-looking statements. Vincerx, the Vincerx logo, CellTrapper, and VersAptx are our trademarks. This press release also contains trademarks and trade names that are the property of their respective owners. Contacts Gabriela Jairala gabriela.jairala@vincerx.com Vincerx Pharma, Inc. Totyana Simien Inizio Evoke Comms totyana.simien@inizioevoke.com Vincerx Pharma, Inc. Condensed Consolidated Balance Sheets (in thousands) Dec 31, 2023 Dec 31, 2022 (Unaudited) ASSETS Current assets: Cash and cash equivalents $ 12,782 $ 11,663 Restricted cash 72 70 Short-term marketable securities - 40,796 Prepaid expenses 51 134 Grant receivable 1,044 1,372 Other current assets 784 1,929 Total current assets 14,733 55,964 Right-of-use assets 2,201 3,064 Property, plant and equipment, net 125 177 Other assets 1,158 81 Total assets $ 18,217 $ 59,286 LIABILITIES AND STOCKHOLDERS' EQUITY Current liabilities Accounts payable $ 2,497 $ 4,065 Accrued expenses 1,755 3,923 Lease liability 1,162 1,024 Common stock warrant liabilities 191 144 Total current liabilities 5,605 9,156 Lease liability, net of current portion 1,340 2,412 Other noncurrent liabilities 50 50 Total liabilities 6,995 11,618 Total stockholders' equity 11,222 47,668 Total liabilities and stockholders' equity $ 18,217 $ 59,286 Vincerx Pharma, Inc. Condensed Consolidated Statements of Operations (unaudited) (in thousands, except per share amounts) For the three months ended For the year ended December 31, December 31, 2023 2022 2023 2022 Operating expenses: General and administrative $ 1,820 $ 4,015 $ 13,636 $ 18,885 Research and development 3,713 11,536 28,973 49,837 Restructuring - - - 2,469 Total operating expenses 5,533 15,551 42,609 71,191 Loss from operations (5,533 ) (15,551 ) (42,609 ) (71,191 ) Other income (expense) Change in fair value of warrant liabilities (59 ) (31 ) (47 ) 6,303 Interest income 198 460 1,251 664 Other income (expense) 444 1,351 1,248 1,240 Total other income (expense) 583 1,780 2,452 8,207 Net loss $ (4,950 ) $ (13,771 ) $ (40,157 ) $ (62,984 ) Net loss per common share, basic and diluted $ (0.23 ) $ (0.65 ) $ (1.89 ) $ (3.00 ) Weighted average common shares outstanding, basic and diluted 21,370 21,138 21,295 21,029

▎药明康德内容团队编辑今日，Moderna公司召开了疫苗日（vaccine day）活动，公布了该公司在mRNA疫苗领域的研发布局和最新进展。该公司首席执行官Stéphane Bancel先生表示，未来3年多款疫苗产品有望上市，其中预防呼吸道合胞病毒（RSV）的mRNA疫苗有望在今年获得FDA的批准。同时，该公司宣布与Blackstone Life Sciences达成合作，获得7.5亿美元投资支持其流感疫苗的研发项目。今天这篇文章里，药明康德内容团队将与读者分享疫苗日上的精彩内容，点击文末“阅读全文/Read more“，即可下载研发日PPT报告。爱泼斯坦·巴尔病毒、带状疱疹病毒、诺如病毒疫苗获积极临床结果Moderna疫苗开发的重心之一是针对潜伏病毒的疫苗开发，这些病毒在急性感染之后会一直潜伏在体内，在人体免疫系统削弱的时候，潜伏的病毒可能再度爆发。潜伏的病毒也可能提高其它疾病的风险，比如爱泼斯坦·巴尔病毒（Epstein Barr virus，EBV），它不但显著提高感染者患上多发性硬化（MS）的风险，还与多种癌症相关。Moderna公司的EBV候选疫苗mRNA-1189编码4种EBV病毒抗原，在1期临床试验中，接种参与者激发的针对性抗体水平高于受到自然EBV感染后的抗体水平。此外，与安慰剂相比，接种mRNA-1189的参与者唾液中的病毒水平有所下降。Moderna计划进一步开发mRNA-1189，推动这一研发项目进入关键性临床试验阶段。▲Moderna公司的EBV候选疫苗简介（图片来源：Moderna官网）水痘带状疱疹病毒（VZV）是在老年人群中导致带状疱疹的主要原因，这种潜伏病毒在老年人免疫力下降后再度激活，感染神经元，造成严重的瘙痒和疼痛。今日公布的1/2期临床试验最新结果显示，两剂mRNA疫苗mRNA-1468接种可以激发VZV抗原特异性CD4阳性T细胞和CD8阳性T细胞反应。激发的T细胞反应与已经获批的带状疱疹疫苗相似。Moderna计划将这款疫苗推进至关键性3期临床试验。诺如病毒（norovirus）是导致腹泻的首要原因之一，在全球范围内与18%的急性胃肠炎相关。在1期临床试验中，单剂诺如病毒疫苗mRNA-1403的接种激发强力的抗体反应。这款疫苗也将被推进至关键性3期临床试验。▲mRNA-1403疫苗接种激发强力抗体反应（图片来源：Moderna官网）人工智能辅助疫苗研发在疫苗日上，Moderna公司还特别展示了人工智能（AI）辅助疫苗研发的效果。该公司的Dose ID GPT是一种生成式AI系统，可以通过分析已有临床数据推荐最佳接种剂量，更好地平衡激发强力免疫反应和保障疫苗的安全性。▲Moderna公司的生成式人工智能系统可用于在疫苗开发过程中推荐最佳接种剂量（图片来源：Moderna公司官网）该公司计划使用Dose ID GPT系统辅助多个研发项目进入后期临床开发阶段，包括上面提到的VZV、EBV、和诺如病毒疫苗。流感疫苗达到3期临床主要终点，7.5亿美元助力开发和商业化Moderna今日还宣布与Blackstone Life Sciences达成合作，获得7.5亿美元投资帮助流感疫苗的开发和商业化。该公司的流感疫苗mRNA-1010在3期临床试验P303中达到所有免疫原性共同主要终点。并且与已有疫苗相比，针对研究中四种病毒株的抗体滴度和血清转化率更高。Moderna计划在今年向监管机构递交这一疫苗的上市申请。▲mRNA-1010在3期临床试验P303中的免疫原性结果（图片来源：Moderna官网）Moderna公司在疫苗日上还介绍了多项研发项目，限于篇幅本文不做一一介绍，点击文末“阅读全文/Read more“，即可下载研发日PPT报告。大家都在看药明康德为全球生物医药行业提供一体化、端到端的新药研发和生产服务，服务范围涵盖化学药研发和生产、生物学研究、临床前测试和临床试验研发、细胞及基因疗法研发、测试和生产等领域。如您有相关业务需求，欢迎点击下方图片填写具体信息。▲如您有任何业务需求，请长按扫描上方二维码，即可访问业务对接平台，填写业务需求信息▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料：[1] Moderna Advances Multiple Vaccine Programs to Late-Stage Clinical Trials. Retrieved March 27, 2024, from https://investors.modernatx.com/news/news-details/2024/Moderna-Advances-Multiple-Vaccine-Programs-to-Late-Stage-Clinical-Trials/default.aspx[2] Vaccine & Business Updates. Retrieved March 27, 2024, from https://s29.q4cdn.com/435878511/files/doc_events/2024/Mar/27/vaccines-day-2024_final_v5.pdf免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

Announces next-generation COVID-19 vaccine candidate as fourth respiratory vaccine to successfully meet its Phase 3 endpoints Expects two more Phase 3 readouts in 2024, including combination vaccine against flu and COVID-19, and vaccine against CMV Announces positive clinical trial data from three new vaccines against viruses that cause significant burden (Epstein-Barr virus, Varicella-Zoster virus, norovirus) and advances programs toward Phase 3 development Anticipates U.S. launch of vaccine against RSV following FDA approval and ACIP recommendation in 2024 Announces development and commercialization funding agreement with Blackstone Life Sciences for up to $750 million to advance flu program CAMBRIDGE, MA / ACCESSWIRE / March 27, 2024 / Moderna, Inc. (NASDAQ:MRNA) today announced at its fifth Vaccines Day event clinical and program updates demonstrating advancement and acceleration of its mRNA pipeline. The updates include data readouts in the Company's respiratory and latent and other vaccine portfolios, as well as commercial, manufacturing and financial announcements for its vaccines business. "Our mRNA platform continues a remarkable track record across our broad vaccine portfolio. Today, we are excited to share that four vaccines in our pipeline have achieved successful clinical readouts across our respiratory, latent and other virus franchises," said Stéphane Bancel, Chief Executive Officer of Moderna. "With five vaccines in Phase 3, and three more moving toward Phase 3, we have built a very large and diverse portfolio addressing significant unmet medical needs. We are focused on execution to further build momentum across our pipeline and business, and to deliver for patients who are impacted by these infectious diseases." Portfolio Overview The vaccine portfolio seeks to address infectious diseases that cause considerable health burdens and includes 28 vaccines addressing respiratory, latent and other pathogens. Latent and Other Vaccine Portfolio Moderna is advancing five vaccine candidates against viruses that cause latent infections, all of which are in clinical trials. When latent, a virus is present in the body but exists in a resting state, typically without causing any noticeable symptoms. Latent viruses can reactivate and cause clinical symptoms as a person ages, during times of stress or when immunity is compromised. The capacity for latency is a defining feature of members of the Herpesviridae family, including cytomegalovirus (CMV), Epstein-Barr virus (EBV), herpes simplex virus (HSV) and Varicella-Zoster virus (VZV). Cytomegalovirus (CMV) CMV is the most common infectious cause of birth defects in the U.S. and is responsible for several billion dollars in annual healthcare costs. One in 200 babies in the U.S. are born with a congenital CMV infection, and of those affected, one in five will have severe, life-altering health problems. Possible short- and long-term sequelae of CMV infection include microcephaly, chorioretinitis, seizures, sensorineural hearing loss, cognitive impairment and cerebral palsy. There is currently no approved vaccine to prevent congenital CMV. CMVictory is a pivotal Phase 3 trial evaluating mRNA-1647 against primary CMV infection in women 16 to 40 years of age. The trial is a randomized, observer-blind, placebo-controlled study designed to evaluate the efficacy, safety and immunogenicity of mRNA-1647. The trial is fully enrolled with approximately 7,300 participants from 290 clinical sites globally. To date, 50 primary infection cases have accrued and are undergoing confirmation. The first interim analysis for the evaluation of vaccine efficacy, which will be triggered when both 81 confirmed per-protocol cases and 12 median months of safety follow-up have occurred, is expected as early as the end of 2024. Moderna's CMV vaccine candidate mRNA-1647 has advanced to indication expansion studies in adolescents 9 to 15 years of age and adult transplant patients, both of which have begun enrollment. Epstein-Barr virus (EBV) EBV is a major cause of infectious mononucleosis (IM) in the U.S., accounting for more than 90% of IM cases annually. Importantly, EBV and IM are associated with a higher lifetime risk of more serious sequelae including certain cancers such as gastric carcinoma, nasopharyngeal carcinoma and multiple types of lymphoma. The lifetime risk of developing multiple sclerosis (MS) is increased by 32-fold after EBV infection. There is currently no approved vaccine to prevent EBV. Moderna's EBV vaccine candidates are designed to tackle multiple EBV-associated conditions, including prevention of IM (mRNA-1189) and MS and post-transplant lymphoproliferative disorder, a subcategory of lymphoma in solid organ transplant patients (mRNA-1195). The Phase 1 trial for mRNA-1189 was designed to test the safety, reactogenicity and immunogenicity of four different dose levels in participants 12 to 30 years of age in the U.S. The randomized, observer-blind, placebo-controlled study showed mRNA-1189 was immunogenic and generally well tolerated across all dose levels. The Company is advancing mRNA-1189 toward a pivotal Phase 3 trial. The Phase 1 trial for mRNA-1195 was designed to test the safety, reactogenicity and immunogenicity of two drug products at four different dose levels in healthy EBV seropositive participants 18 to 55 years of age in the U.S. The randomized, observer-blind, placebo-controlled study is fully enrolled. Herpes simplex virus (HSV) Herpes simplex virus type 2 (HSV-2) infects approximately 13% of adults globally and is the primary cause of genital herpes. There are an estimated four billion people globally infected with HSV, of which 491 million cases are HSV-2. Recurrent genital herpes causes a reduction in quality of life, which antivirals (current standard of care) only partially restore. Moderna expects that if an HSV vaccine candidate could deliver similar efficacy as a suppressive antiviral treatment, compliance with recommended therapy and associated quality of life would improve. There is currently no approved vaccine to treat HSV-2. The first in human, fully enrolled Phase 1/2 trial of mRNA-1608 is designed to test safety and immunogenicity and to establish a proof-of-concept of clinical benefit in adults 18 to 55 years of age with recurrent HSV-2 genital herpes. The randomized 1:1:1:1, observer-blind, controlled study is fully enrolled with 300 participants in the U.S. Varicella-Zoster virus (VZV) Herpes zoster, also known as shingles, is caused by reactivation of latent VZV, the same virus that causes chickenpox. Declining immunity in older adults decreases immunity against VZV, allowing reactivation of the virus from latently infected neurons, causing painful and itchy lesions. Herpes Zoster occurs in one out of three adults in the U.S. in their lifetime and the incidence increases at 50 years of age. There is potential to reach a growing and underserved patient population. Moderna's VZV vaccine candidate mRNA-1468 has initial data available from a Phase 1/2 trial, which was designed to test safety and immunogenicity in healthy adults 50 years of age and older in the U.S. The randomized 1:1:1:1:1, observer-blind, active-controlled study of mRNA-1468 elicited strong antigen-specific T cell responses at one month after the second dose and was generally well tolerated. Results of the first interim analysis support the further clinical development of mRNA-1468 for the prevention of shingles. Additional results from the ongoing Phase 1/2 study will be available later this year, including persistence data. The Company is planning for a pivotal Phase 3 trial. Norovirus Enteric viruses, including norovirus, are a leading cause of diarrheal diseases, resulting in significant morbidity and mortality worldwide, particularly among young children and older adults. Norovirus is highly contagious and a leading cause of diarrheal disease globally, associated with 18% of all acute gastroenteritis (AGE), resulting in approximately 200,000 deaths per year and substantial healthcare costs. Given the wide diversity of norovirus genotypes, a broadly effective norovirus vaccine will require a multivalent vaccine design. There is currently no approved vaccine to prevent norovirus. The randomized, observer-blind, placebo-controlled Phase 1 trial was designed to evaluate the safety, reactogenicity and immunogenicity of trivalent (mRNA-1403) and pentavalent (mRNA-1405) norovirus vaccine candidates in 664 participants 18 to 49 years of age and 60 to 80 years of age in the U.S. An interim analysis showed that a single dose of mRNA-1403 elicited a robust immune response across all dose levels evaluated with a clinically acceptable reactogenicity and safety profile. The Company is advancing mRNA-1403 toward a pivotal Phase 3 trial. Respiratory Vaccine Portfolio Moderna's approach to ease the global burden of respiratory infections includes vaccine candidates against major causative pathogens, including SARS-CoV-2, respiratory syncytial virus (RSV) and influenza virus. Respiratory infections are a top cause of death in the U.S. and are particularly harmful to the young, immunocompromised, and older adults who experience more severe illness, greater incidence of hospitalization, and greater mortality than younger adults. Moderna's respiratory pipeline includes Phase 3 trials for investigational vaccines including a next-generation COVID-19 vaccine, an RSV vaccine, a flu vaccine, and a flu and COVID-19 combination vaccine. The pipeline includes three additional flu vaccine candidates with expanded antigen coverage as well as combination vaccine programs. COVID-19 Moderna continues to address the needs of the endemic COVID-19 market by focusing on public health efforts to increase vaccination coverage rates to reduce the substantial burden of COVID-19 as well as by advancing next-generation vaccines. The Company's mRNA platform can produce variant-matched vaccines on an accelerated time horizon, consistent with recent U.S. Food and Drug Administration (FDA) comments on the timing of potential strain selection for the fall booster season. A recent announcement of positive interim results from the NEXTCove Phase 3 trial showed that mRNA-1283 elicited a higher immune response against both the Omicron BA.4/BA.5 and original virus strains of SARS-CoV-2 compared to mRNA-1273.222, Moderna's licensed COVID-19 vaccine. mRNA-1283 is designed to be refrigerator-stable and paves the way for a combination vaccine against influenza and COVID-19, mRNA-1083, enhancing the Company's overall respiratory portfolio. This is Moderna's fourth infectious disease vaccine program with Phase 3 data. Respiratory Syncytial Virus (RSV) RSV is the leading cause of respiratory illness in young children, and older adults are at increased risk relative to younger adults for severe outcomes. In addition to acute mortality and morbidity, RSV infection is associated with long-term sequelae such as asthma and impaired lung function in pediatric populations, and exacerbation of chronic obstructive pulmonary disease in older adults. Annually, there are approximately two million medically attended RSV infections and 58,000 to 80,000 hospitalizations in children younger than five years old in the U.S. In the U.S., each year there are up to 160,000 hospitalizations and 10,000 deaths in adults 65 years and older due to RSV. Across high-income countries in 2019, RSV caused an estimated 5.2 million cases, 470,000 hospitalizations and 33,000 in-hospital deaths in adults 60 years and older. mRNA-1345 Moderna's RSV vaccine candidate, mRNA-1345, is in an ongoing Phase 2/3, randomized, observer-blind, placebo-controlled case-driven trial (ConquerRSV) in adults over 60 years of age. In this study, approximately 37,000 participants from 22 countries were randomized 1:1 to receive one dose of mRNA-1345 or placebo. Based on positive data from the ConquerRSV trial, Moderna has filed for regulatory approvals for mRNA-1345 for the prevention of RSV-associated lower respiratory tract disease (RSV-LRTD) and acute respiratory disease (ARD) in adults over 60 years of age. The trial met both its primary efficacy endpoints, with a vaccine efficacy (VE) of 83.7% (95.88% CI: 66.1%, 92.2%; p<0.0001) against RSV-LRTD as defined by two or more symptoms, and a VE of 82.4% (96.36% CI: 34.8%, 95.3%; p=0.0078) against RSV-LRTD defined by three or more symptoms. These data were published in the New England Journal of Medicine in December 2023. A subsequent analysis from the ConquerRSV study with a longer median follow-up duration of 8.6 months (versus 3.7 months in the primary analysis), with a range of 15 days to 530 days, and including subjects from the Northern and Southern Hemispheres was recently presented at the RSVVW'24 conference. In this supplemental analysis, mRNA-1345 maintained durable efficacy, with sustained VE of 63.3% (95.88% CI: 48.7%, 73.7%) against RSV-LRTD including two or more symptoms. VE was 74.6% (95% CI: 50.7%, 86.9%) against RSV-LRTD with ≥2 symptoms, including shortness of breath and 63.0% (95% CI: 37.3%, 78.2%) against RSV-LRTD including three of more symptoms. The stringent statistical criterion of the study, a lower bound on the 95% CI of >20%, continued to be met for both endpoints. mRNA-1345 has been granted Breakthrough Therapy designation by the FDA for the prevention of RSV-LRTD in adults over 60 years of age. The Company is awaiting regulatory approvals and the U.S. ACIP recommendation in 2024. Indication expansion studies for mRNA-1345 mRNA-1345 has the potential to protect all vulnerable populations from RSV. Moderna has initiated multiple Phase 3 expansion studies in adults over 50 years of age to evaluate co-administration and revaccination. Additional trials (Phase 1 - Phase 3) have been initiated for high-risk adults, as well as maternal and pediatric populations. Interim data from these studies could be available as early as 2024. Influenza (Flu) Worldwide, influenza leads to 3-5 million severe cases of flu and 290,000-650,000 flu-related respiratory deaths annually. Two main types of influenza viruses (A and B) cause seasonal flu epidemics, and the influenza A viruses lead to most flu-related hospitalization in older adults. The Company has several seasonal influenza vaccine candidates in clinical development. Moderna's seasonal flu vaccine, mRNA-1010, demonstrated consistently acceptable safety and tolerability across three Phase 3 trials. In the most recent Phase 3 trial (P303), which was designed to test the immunogenicity and safety of an optimized vaccine composition, mRNA-1010 met all immunogenicity primary endpoints, demonstrating higher antibody titers compared to a currently licensed standard-dose flu vaccine. In an older adult extension study of P303, mRNA-1010 is being studied against high dose Fluzone HD®; the trial is fully enrolled. The Company is in ongoing discussions with regulators and intends to 2024. Combination Respiratory Vaccines Moderna's combination vaccine candidates cover respiratory viruses associated with the largest disease burden in the category. The Phase 3 combination study of the Company's investigational combination vaccine against flu and COVID-19 (mRNA-1083) for adults aged 50 years and older is fully enrolled and data are expected in 2024. mRNA-1083 was granted Fast Track designation by the FDA in May 2023. Commercial Updates Respiratory viruses in addition to latent and other viruses represent large unmet or underserved medical needs, and the human and economic costs from these infectious diseases highlight the need for effective vaccines. To help address this need, Moderna expects multiple vaccine product launches in the next few years, each with significant addressable markets. The 2024 global endemic COVID-19 vaccine market alone is estimated by Moderna to be approximately $10 billion. COVID-19 continues to show a high burden of disease, and while COVID-19 hospitalizations remain high relative to RSV and flu, the risks of Long COVID are also becoming better understood. Moderna is focused on improving education and awareness to increase vaccination rates as Long COVID data suggests even traditionally low-risk groups should be vaccinated. Moderna is also working with health authorities to align the timing of COVID-19 and flu vaccine launches to help improve public health. For RSV, Moderna estimates the peak annual market to be approximately $10 billion. The Company expects a strong RSV vaccine launch into a large market in 2024. As the only mRNA investigational vaccine with positive Phase 3 data, Moderna's RSV vaccine candidate has a strong pro consistently strong efficacy across vulnerable and older populations, a well-established safety and tolerability profile, and ease of administration with a ready-to-use, pre-filled syringe formulation, which could relieve some of the burden that falls on pharmacies during the fall vaccination season. An interim analysis from an ongoing time and motion study evaluating differences in preparation time between a pre-filled syringe (PFS) presentation and vaccines that require reconstitution showed that a PFS presentation could relieve some of the burden that falls on pharmacies during the fall vaccination season. Results from this study suggest that pharmacies may be capable of preparing up to four times as many doses of PFS in an hour compared to vaccines requiring reconstitution. Moderna estimates flu vaccines represent an approximately $7 billion market in 2024. The market is expected to grow with the rise of more effective vaccines and there is an opportunity to expand the market with next-generation premium flu vaccines as well as combination respiratory vaccines, adding increased value to the health ecosystem. CMV is expected to be a $2-5 billion annual market. With no vaccine currently on the market and a potential vaccine launch in 2026, Moderna could be the first CMV vaccine in multi-billion-dollar latent vaccine market. In addition, EBV has the potential to address and reduce the burden and cost of EBV infection in multiple populations, while VZV provides the opportunity to enter a large and growing market, which could be $5-6 billion annually. The market for norovirus vaccines is similar to that of rotavirus in pediatrics with opportunity to expand into the adult population, and represents a $3-6 billion annual market. Moderna's vaccine portfolio targets large addressable markets, with an estimated total addressable market (TAM) of $52 billon for Moderna infectious disease vaccines, which includes a respiratory vaccines TAM of more than $27 billion and a latent and other vaccines TAM of more than $25 billion. Manufacturing The Company's manufacturing innovation supports expanding commercialization of a diverse pipeline through efficiency and productivity gains. Its mRNA manufacturing platform enables benefits such as quality, speed, scale and cost efficiency across a footprint that broadly includes the manufacture of plasmid, mRNA, lipid nanoparticles, as well as fill/finish and quality control capabilities. As the Company continues to build its footprint for the future, it is developing an agile global manufacturing network to meet commercial demand and support its growing pipeline. Pre-clinical through commercial manufacturing occurs at the Moderna Technology Center in Norwood, Massachusetts, which remains central to the Company's network. New facilities being constructed in Australia, Canada and the UK are expected to come online in 2025, and drug product capacity is achieved through a flexible contract manufacturing network. Additionally, the Company has purchased and started build-out of a manufacturing site in Marlborough, Massachusetts, to enable commercial scale of its individualized neoantigen therapy program. By continuing to pioneer new technologies, including advanced robotics, applying AI and other digital solutions, and driving network and capital efficiency, Moderna's manufacturing network is expected to also drive more predictable cost of sales. Research and Development Investment Strategy Today's updates provide further evidence that Moderna's mRNA technology platform is working, and with a rate of success higher than industry standard. Looking ahead, research and development will continue to be the Company's top capital allocation priority. As Moderna looks to create value through the research and development strategy for its vaccine portfolio, it is taking three prioritization parameters into consideration: pipeline advancement, revenue diversification and risk reduction. As part of its strategy, the funding options Moderna considers are self-funding, project financing and partnerships. Moderna recently entered into a development and commercialization funding agreement with Blackstone Life Sciences to advance the Company's flu program. As part of the agreement, Blackstone will fund up to $750 million with a return based on cumulative commercial milestones and low-single digit royalties. Moderna expects to recognize the funding as a reduction in research and development expenses and will retain full rights and control of the Company's flu program. This funding does not result in any change to Moderna's 2024 research and development framework of approximately $4.5 billion. About Moderna Moderna is a leader in the creation of the field of mRNA medicine. Through the advancement of mRNA technology, Moderna is reimagining how medicines are made and transforming how we treat and prevent disease for everyone. By working at the intersection of science, technology and health for more than a decade, the company has developed medicines at unprecedented speed and efficiency, including one of the earliest and most effective COVID-19 vaccines. Moderna's mRNA platform has enabled the development of therapeutics and vaccines for infectious diseases, immuno-oncology, rare diseases and autoimmune diseases. With a unique culture and a global team driven by the Moderna values and mindsets to responsibly change the future of human health, Moderna strives to deliver the greatest possible impact to people through mRNA medicines. For more information about Moderna, please visit modernatx.com and connect with us on X (formerly Twitter), Facebook, Instagram, YouTube and LinkedIn. INDICATION (U.S.) SPIKEVAX (COVID-19 Vaccine, mRNA) is a vaccine indicated for active immunization to prevent coronavirus disease 2019 (COVID-19) caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) in individuals 18 years of age and older. IMPORTANT SAFETY INFORMATION Do not administer to individuals with a known history of severe allergic reaction (e.g., anaphylaxis) to any component of the vaccine. Appropriate medical treatment to manage immediate allergic reactions must be immediately available in the event an acute anaphylactic reaction occurs following administration of the vaccine. Postmarketing data demonstrate increased risks of myocarditis and pericarditis, particularly within 7 days following the second dose. The observed risk is higher among males under 40 years of age than among females and older males. The observed risk is highest in males 18 through 24 years of age. Syncope (fainting) may occur in association with administration of injectable vaccines. Procedures should be in place to avoid injury from fainting. Immunocompromised persons, including individuals receiving immunosuppressive therapy, may have a diminished response to the vaccine. The vaccine may not protect all vaccine recipients. Adverse reactions reported in clinical trials following administration of the vaccine include pain at the injection site, fatigue, headache, myalgia, arthralgia, chills, nausea/vomiting, axillary swelling/tenderness, fever, swelling at the injection site, and erythema at the injection site, and rash. The vaccination provider is responsible for mandatory reporting of certain adverse events to the Vaccine Adverse Event Reporting System (VAERS) online at or by calling 1-800-822-7967. Please see the SPIKEVAX Full Prescribing Information. For information regarding authorized emergency uses of the Moderna COVID-19 Vaccine, please see the EUA Fact Sheet. Spikevax® is a registered trademark of Moderna. Fluzone® is a registered trademark of Sanofi Pasteur. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding: the advancement of Moderna's programs under clinical development; the timing for anticipated approvals of vaccine candidates; the efficacy, safety and tolerability of vaccine candidates; the total addressable markets for programs under development; the efficiencies and advantages of Moderna's mRNA platform; future capital allocation and financing efforts; and anticipated spending for R&D in 2024. In some cases, forward-looking statements can be identified by terminology such as "will," "may," "should," "could," "expects," "intends," "plans," "aims," "anticipates," "believes," "estimates," "predicts," "potential," "continue," or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. The forward-looking statements in this press release are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Moderna's control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties, and other factors include, among others, those risks and uncertainties described under the heading "Risk Factors" in Moderna's Annual Report on Form 10-K for the fiscal year ended December 31, 2023, filed with the U.S. Securities and Exchange Commission (SEC), and in subsequent filings made by Moderna with the SEC, which are available on the SEC's website at . Except as required by law, Moderna disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this presentation in the event of new information, future developments or otherwise. These forward-looking statements are based on Moderna's current expectations and speak only as of the date of this press release. ​ ### Moderna Contacts Media: Chris Ridley Head, Global Media Relations +1 617-800-3651 Chris.Ridley@modernatx.com Investors: Lavina Talukdar Senior Vice President & Head of Investor Relations +1 617-209-5834 Lavina.Talukdar@modernatx.com SOURCE: Moderna, Inc. View the original press release on accesswire.com