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Integration of AL102 and IM-1021 completed Topline data for Phase 3 RINGSIDE trial of AL102 expected in second half of 2025 IM-1021 and IM-3050 IND submissions expected in first quarter of 2025 BOTHELL, Wash.--(BUSINESS WIRE)-- Immunome, Inc. (Nasdaq: IMNM), a biotechnology company focused on developing first-in-class and best-in-class targeted cancer therapies, today announced financial results for the first quarter ended March 31, 2024, and provided a business update. “Immunome continues to build momentum. We have completed the integration of AL102 and are executing activities necessary for regulatory submissions,” said Clay B. Siegall, Ph.D., President and Chief Executive Officer. “We have also completed the integration of IM-1021 and are advancing that program and IM-3050 towards clinical trials.” Dr. Siegall continued, “In addition, we are working internally to design and develop additional next-generation ADC technology. Those efforts are complemented by our ongoing business development efforts, which seek to identify and procure high-quality technologies and clinical programs. Both our internal and external efforts are focused on expanding Immunome’s pipeline with additional programs that have first-in-class or best-in-class potential and can improve the lives of cancer patients.” Pipeline Highlights The purchase of AL101 and AL102 from Ayala Pharmaceuticals closed on March 25, 2024. Full enrollment for the Phase 3 RINGSIDE Part B study of AL102 for the treatment of desmoid tumors was completed in February 2024. Immunome expects to report topline data for RINGSIDE Part B in the second half of 2025. In parallel, Immunome continues to perform additional manufacturing and pharmacology work required to support a new drug application filing for AL102. Immunome anticipates submitting INDs for two preclinical candidates in the first quarter of 2025: IM-1021, a ROR1 ADC, and IM-3050, a FAP-targeted RLT. First Quarter 2024 Financial Results As of March 31, 2024, cash, cash equivalents and marketable securities totaled $309.7 million. Immunome’s current cash runway is expected to extend into 2026. Research and development expenses for the quarter ended March 31, 2024 were $15.4 million, including stock-based compensation costs of $0.4 million. In-process research and development expenses for the quarter ended March 31, 2024 were $112.0 million. These expenses were related to Immunome’s acquisition of AL101 and AL102 from Ayala Pharmaceuticals, Inc. and the exclusive license of IM-1021 and related ADC platform technology from Zentalis Pharmaceuticals, Inc., both of which closed in the quarter ended March 31, 2024. General and administrative expenses for the quarter ended March 31, 2024 were $6.0 million, including stock-based compensation expense of $1.8 million. Immunome reported a net loss of $129.5 million, or basic and diluted net loss per share attributable to common stockholders of $2.51, for the quarter ended March 31, 2024. About Immunome, Inc. Immunome is a biotechnology company dedicated to developing first-in-class and best-in-class targeted cancer therapies. Our portfolio pursues each target with a modality appropriate to its biology, including small molecules, ADCs, and RLTs. We believe that pursuing underexplored targets with appropriate drug modalities leads to transformative therapies. Our proprietary memory B cell hybridoma technology allows for the rapid screening and functional characterization of novel antibodies and targets. For more information, visit or follow us on Twitter and LinkedIn. Cautionary Statement Regarding Forward-Looking Statements Certain statements contained in this communication regarding matters that are not historical facts, are forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995 (the “PSLRA”). We use words such as “may,” “could,” “potential,” “will,” “plan,” “believe,” “expect,” and similar expressions to identify these forward-looking statements that are intended to be covered by the safe-harbor provisions of the PSLRA. These forward-looking statements include, but are not limited to, Immunome’s expectation to report topline data for the ongoing Phase 3 RINGSIDE Part B trial of AL102 in the second half of 2025; Immunome’s expected timeline for submitting INDs for IM-3050 and IM-1021 in the first quarter of 2025; Immunome’s expected cash runway; Immunome’s ability to expand its pipeline through both internal and external efforts; and other statements regarding management’s intentions, plans, beliefs, expectations or forecasts for the future. No forward-looking statement can be guaranteed, and actual results may differ materially from those projected. Such forward-looking statements are based on Immunome’s expectations and involve risks and uncertainties; consequently, actual results may differ materially from those expressed or implied in the statements due to a number of factors, including, but not limited to, the risk that Immunome will not be able to realize the benefits of its strategic transactions; Immunome’s ability to grow and successfully execute on its business plan, including the development and commercialization of its pipeline and integration of newly acquired assets; changes in the applicable laws or regulations; the possibility that Immunome may be adversely affected by other economic, business, and/or competitive factors; the risk that regulatory approvals for Immunome’s programs and product candidates are not obtained, are delayed or are subject to unanticipated conditions; the risk that pre-clinical data may not be predictive of clinical data; the risk that interim results of a clinical trial do not necessarily predict final results; potential delays in the commencement, enrollment and completion of clinical trials and the reporting of data therefrom; the risk that Immunome’s product and development candidates fail to achieve their intended endpoints; the complexity of numerous regulatory and legal requirements that Immunome needs to comply with to operate its business; the reliance on Immunome’s management; the prior experience and successes of the Immunome’s management team not being indicative of any future success; uncertainties related to Immunome’s capital requirements and Immunome’s expected cash runway; the failure to obtain, adequately protect, maintain or enforce Immunome’s intellectual property rights; and other risks and uncertainties indicated from time to time described in Immunome’s Annual Report on Form 10-K for the year ended December 31, 2023, filed with the SEC on March 26, 2024, in ARS Pharma’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2024, being filed with the SEC today, and in Immunome’s other filings with the SEC. Immunome cautions that the foregoing list of factors is not exclusive and not to place undue reliance upon any forward-looking statements which speak only as of the date made. Moreover, Immunome operates in a very competitive and rapidly changing environment. New risks emerge from time to time. Except as required by law, Immunome does not undertake any obligation to update publicly any forward-looking statements for any reason after the date of this press release to conform these statements to actual results or to changes in their expectations. Immunome, Inc. Condensed Consolidated Balance Sheets (Unaudited; In thousands) March 31, 2024 December 31, 2023 Assets Current assets: Cash and cash equivalents $ 269,723 $ 98,679 Marketable securities 39,983 39,463 Prepaid expenses and other current assets 3,620 6,561 Total current assets 313,326 144,703 Property and equipment, net 4,302 2,073 Operating right-of-use assets 1,458 1,564 Restricted cash 100 100 Other long-term assets 568 100 Total assets $ 319,754 $ 148,540 Liabilities and stockholders’ equity Current liabilities: Accounts payable $ 7,179 $ 3,311 Accrued expenses and other current liabilities 10,844 8,025 Deferred revenue, current 12,745 10,493 Total current liabilities 30,768 21,829 Deferred revenue, non-current 2,208 5,489 Operating lease liabilities, net of current portion 1,206 1,340 Total liabilities 34,182 28,658 Stockholders’ equity: Preferred stock — — Common stock 6 4 Additional paid-in capital 637,861 342,663 Accumulated other comprehensive income 4 22 Accumulated deficit (352,299 ) (222,807 ) Total stockholders’ equity 285,572 119,882 Total liabilities and stockholders’ equity $ 319,754 $ 148,540 Immunome, Inc. Condensed Consolidated Statements of Operations and Comprehensive Loss (Unaudited; In thousands, except share and per share amounts) Three Months Ended March 31, 2024 2023 Collaboration revenue $ 1,029 $ 2,364 Operating expenses: In-process research and development 111,954 — Research and development(1) 15,369 3,913 General and administrative(1) 6,005 2,922 Total operating expenses 133,328 6,835 Loss from operations (132,299 ) (4,471 ) Interest income 2,807 201 Net loss $ (129,492 ) $ (4,270 ) Net loss per share, basic and diluted $ (2.51 ) $ (0.35 ) Weighted-average shares outstanding, basic and diluted 51,544,383 12,182,478 Comprehensive loss Net loss $ (129,492 ) $ (4,270 ) Unrealized loss on marketable securities (18 ) — Comprehensive loss $ (129,510 ) $ (4,270 ) (1) Amounts include non-cash, stock-based compensation expense as follows (in thousands): Three Months Ended March 31, 2024 2023 Research and development $ 383 $ 430 General and administrative 1,776 794 Total share-based compensation expense $ 2,159 $ 1,224

▎药明康德内容团队编辑随着Leqembi获批为阿尔茨海默病（AD）治疗领域带来的显著进展，神经病学（Neurology）领域近年来受到业界的高度关注。这一趋势不仅促进了大型交易的频繁发生，也推动了临床研究积极进展。近日，行业媒体Evaluate发布了一份行业报告，深入分析了神经病学领域内的交易动态和研发趋势。在今天的文章里，药明康德内容团队将结合公开资料，与读者分享其中的精彩内容。点击文末“阅读全文/Read more”，即可访问报告原文网址。近年神经病学领域的大型收购与交易趋势神经病学是一门专注于神经系统疾病的医学分支，涵盖了从退行性疾病（如阿尔茨海默病和帕金森病）到癫痫、抑郁症、偏头痛、疼痛管理，乃至众多罕见疾病的广泛领域。相较于如癌症这样的高关注度领域，神经病学在过去数十年里获得的投资相对缺乏，这导致了大量未竟医疗需求。这种投资不足的背景，结合持续的精神健康危机和有限的治疗选择，为神经病学领域带来了独特的市场机遇和挑战。图片来源：123RF2022-2023年间，神经病学领域的并购活动活跃，其中较为突出的例子包括百时美施贵宝（Bristol Myers Squibb）以约140亿美元收购Karuna Therapeutics，此举旨在将精神分裂症疗法KarXT（xanomeline-trospium）纳入其产品管线，该药物的新药申请（NDA）已被美国FDA受理，有望在今年获得批准。艾伯维（AbbVie）则在去年12月以约87亿美元收购Cerevel Therapeutics，进一步加强艾伯维在神经病学领域的布局。此外，渤健（Biogen）以约73亿美元收购Reata Pharmaceuticals，获得了其针对弗里德赖希共济失调症的药物Skyclarys（omaveloxolone）。辉瑞（Pfizer）公司也加大了在神经病学领域的投入，于2022年以总计约116亿美元收购Biohaven Pharmaceutical，增强了其偏头痛治疗产品组合。这一系列并购案例不仅展示了大型医药企业在神经病学领域的布局，也凸显了该领域积极的发展前景。尽管过去神经病学曾经面临投资不足的挑战，但如今它正吸引着大型药企的集中关注和大规模投资。▲2014-2024年间，神经病学领域內＞10亿美元的交易活动（药明康德内容团队制表，数据来源：参考资料[1]）回顾近年来神经病学领域的交易活动，显而易见的一种趋势是医药公司对于“低风险、后期研发阶段项目”的偏好。神经病学药物开发的历程充满了挑战，多个项目在研发过程中曾遭遇了不可预见的挫折，凸显了该领域内药物开发的固有不确定性和高风险。鉴于此，相较于那些仍处于探索初期、前景充满不确定性的新药项目，大部分医药公司更倾向于投资于那些已经进入到后期研发阶段、相对风险更低的项目。这种策略反映了企业对于风险管理的审慎态度，以及在资本投入时寻求更高确定性的倾向，有些公司甚至避免涉足处于中期临床研究阶段项目的风险，倾向于在药物研发进展到更加成熟和风险更可控的阶段进行投资或并购。除了频繁的并购活动外，近年来神经病学领域的融资活动也十分活跃。在2024年3月，Engrail Therapeutics完成了1.57亿美元的B轮融资。而就在不久前，Alto Neuroscience也成功完成了初次IPO交易。2023年9月，Neumora Therapeutics也实现了上市，募集资金高达2.5亿美元。Neumora的主要产品navacaprant目前正处于针对抑郁症的3期临床试验阶段，该公司预计这一关键试验结果将为预定于2025年递交的新药申请提供支持。这些融资活动凸显了产业在神经病学领域的高度活跃性，也展示该领域在医药研发和创新方面的巨大潜力。图片来源：123RF神经病学领域的新药研发趋势随着科技的不断进步以及我们对人脑复杂机制的深入理解，神经病学领域正处于一场创新疗法和药物开发的浪潮中。在此过程中，一些关键趋势尤为突出：首先，药物的选择性变得更加精准，能够特异性地调节特定的神经通路或受体，旨在降低副作用并提升治疗效果。其次，致幻药物在精神疾病治疗中的潜在应用正受到重新评估，特别是在治疗抑郁症和创伤后应激障碍（PTSD）方面。此外，人工智能（AI）技术正助力脑部药物的发现和开发，加速了新药候选分子的筛选和优化过程。同时，对新分子和创新作用机制的探索正不断扩大治疗工具箱，为患者提供更多的治疗选择。这些进展共同推动了神经病学领域的创新和发展，为未来治疗带来新的希望。1.新一代药物更具选择性在开发更有效和安全的中枢神经系统药物方面，药物选择性的提高是一个重要因素。以强生公司（Johnson & Johnson）的aticaprant和Neumora公司的navacaprant为例，这两种药物针对Kappa阿片受体（KORs）的选择性上优于对μ或δ等其他阿片受体亚型，展现了对KORs的高度特异性。这种特性使得这两种药物在治疗重度抑郁症的3期临床试验中表现出潜力，并且还有望扩展到治疗焦虑症和药物滥用等领域。这种对特定受体的高选择性不仅提高了治疗的针对性，也有望减少潜在的副作用。2.重新思考致幻药物的药用价值近来，迷幻药物的医疗用途得到了重新评估和认可。今年2月，美国FDA宣布受理Lykos Therapeutics（前MAPS公益公司）为3，4-亚甲二氧基甲基苯丙胺（MDMA）递交的新药申请，用于治疗PTSD。该申请还获得了FDA授予的优先审评资格，预计审评结果将于今年8月11日之前发布。新闻稿指出，若该药物获批，它将成为第一款MDMA辅助疗法，同时也是首个获批的迷幻药辅助疗法。此外，Compass Pathways公司的在研裸盖菇素（psilocybin，致幻蘑菇中的致幻成分）疗法在2期临床试验中取得了积极结果，目前已进入治疗耐药性抑郁症的3期临床试验。大麻也正式进入医疗领域。例如，Jazz Pharmaceuticals的癫痫发作药物Epidiolex（cannabidiol，大麻的一个主要非致幻成分）在2018年获得FDA的批准上市，治疗两类罕见而严重的癫痫。新闻稿指出，这是首款获美国FDA批准上市的含纯化大麻提取物的新药。人们对致幻剂疗法潜力的兴趣与日俱增，促使FDA在2023年年中起草了指南草案，以支持相关临床研究，这标志着迷幻药物作为治疗手段就正在逐步正规化和科学化。3.AI辅助神经药物开发2024年，Alto Neuroscience成功地募集到1.29亿美元上市。Alto公司以“人工智能驱动的大脑精准医疗”为其核心理念，专注于AI辅助精准药物开发。其成功的融资活动背后是基于该公司主打项目展示出的积极研究数据：公司的ALTO-300项目相关研究显示，采用脑电图（EEG）生物标志物辅助筛选的患者相较于未使用EEG筛选患者获得了更大的益处。在神经病学领域，精准医疗仍处于起步阶段。然而，新兴的测序技术、传感器工具以及更为强大的数据分析能力正助力科学家们深入解析大脑的复杂机制。值得关注的是，2024年初，美国FDA批准了一项名为BrainSee的人工智能临床测试，专门用于阿尔茨海默病。该测试结合大脑成像技术和认知评估，旨在预测患者从轻度认知障碍进展到痴呆症的可能性，标志着人工智能在神经病学领域应用的一大进步。图片来源：123RF4.新靶点，新机制随着基因研究在神经病学领域的深入，新的治疗靶点不断涌现。例如，Denali Therapeutics和渤健合作开发的LRRK2靶向抑制剂BIIB122/DNL151，目前处于治疗帕金森病的2b期试验阶段。另外，Alector Therapeutics正在与艾伯维合作进行一项2b期临床研究，评估其TREM2靶向疗法AL002用以治疗阿尔茨海默病的作用。此外，Alector还与GSK达成合作，共同开发两款progranulin靶向单抗AL001和AL101以治疗神经退行性疾病。Lexeo Therapeutics正在研发的基因疗法LX1001，旨在通过递送APOE2基因—一种具有保护作用的基因——来降低携带纯合APOE4基因患者罹患阿尔茨海默病的风险。该候选药物目前正在进行1/2期临床试验，初步研究数据表明，接受低剂量LX1001治疗的患者脑脊液中的APOE2蛋白表达增加，在超过12个月的随访期内，两名患者的脑脊液中tau蛋白和磷酸化tau蛋白水平与基线相比降低，这两种蛋白是反映阿尔茨海默病患者大脑关键病理变化的生物标志物。图片来源：123RF神经病学领域的弄潮儿在神经病学领域的创新浪潮中，众多公司凭借其独特的技术优势和丰富的产品线，促进了该领域的快速发展，并为治疗神经病学相关疾病提供了更广泛的选择。接下来，我们将介绍几家近年来专注于神经病学领域的公司。Neurocrine BiosciencesNeurocrine Biosciences专注于为患有神经性、神经内分泌性及神经精神疾病的患者开发新型治疗方案。该公司的产品组合多样化，包括已经获得FDA批准的Ingrezza，这是一款已获批用于治疗迟发性运动障碍和亨廷顿病相关舞蹈症的药物。除此之外，Neurocrine还拥有一系列正处于中到后期临床开发阶段的候选疗法。▲Neurocrine Biosciences在研管线（图片来源：Neurocrine Biosciences官网）Acadia PharmaceuticalsAcadia公司已有2款药物获得FDA批准，分别是Nuplazid（pimavanserin）和Daybue（trofinetide）。根据Acadia公司官网，pimavanserin是美国FDA批准的首个用于治疗帕金森病精神病相关幻觉和妄想症状的处方药。值得一提的是，该药物针对精神分裂症的阴性症状的适应症目前正处于3期临床研究中，此前公布的2期试验结果表明，当与现有抗精神病药物联合用药治疗精神分裂症患者时，该药物显著改善了患者在NSA-16量表上的表现。Acadia预计在2024年获得pimavanserin的3期研究的顶线结果。Daybue则是Acadia公司的第二款获批产品，它在2023年3月获得FDA批准用于治疗Rett综合征成人和2岁以上儿童患者，成为FDA批准的首款治疗Rett综合征的药物。▲Acadia Pharmaceuticals在研管线（图片来源：Acadia Pharmaceuticals官网）Alector TherapeuticsAlector公司的研发管线包含3个处在临床阶段的潜在“first-in-class”项目，这些项目吸引了包括GSK和艾伯维在内的大型医药公司的合作。其中，AL001和AL101是与GSK合作开发的两种新型单抗。AL001目前正在进行一项关键性3期临床试验，用于治疗因GRN基因突变而存在额颞叶痴呆（FTD）风险、或患有额颞叶痴呆的患者。而AL101目前则处于2期临床试验阶段，旨在治疗阿尔茨海默病。此外，Alector管线中的AL002是一种针对TREM2的在研人源化单抗，TREM2是散发性AD的一个重要遗传风险因素。该产品由Alector和艾伯维合作开发，目前正在一项2期临床试验中接受检验。▲Alector Therapeutics在研管线（图片来源：Alector Therapeutics官网）渤健渤健在神经病学领域拥有深厚的研发历史和广泛的产品组合，包括19款已经上市的产品，涵盖了多个重要的治疗领域。其中包括首款获得FDA完全批准的阿尔茨海默病疗法Leqembi，以及基于生物标志物加速批准的首款基于生物标志物加速批准的肌萎缩侧索硬化症（ALS）疗法Qalsody（tofersen）。渤健的研发管线中也不乏创新药物，如针对tau蛋白的反义寡核苷酸（ASO）疗法BIIB080，在针对轻度阿尔茨海默病患者的1b期临床试验中展现了积极结果，使其成为首款被证明可以减少tau蛋白聚集、并展现对患者临床结局有利的趋势的tau蛋白靶向药物。此外，渤健还正在开发针对LRRK2的小分子抑制剂BIIB122，展现了其在神经病学领域研发的广度与深度。Denali TherapeuticsDenali Therapeutics致力于开发治疗神经退行性疾病和溶酶体贮积症疗法。Denali拥有多样化的产品管线，其中多个项目已经步入中后期临床开发阶段。包含与渤健合作开发用于治疗帕金森病的LRRK2小分子抑制剂DNL151，与赛诺菲（Sanofi）合作研发用于治疗多发性硬化的RIPK1小分子抑制剂SAR443820/DNL788，以及基于Denali的转运载体平台技术构建的融合蛋白药物DNL310。DNL310可被成功递送到大脑从而缓解II型黏多糖贮积症（MPS II）的中枢神经系统症状。目前，DNL310正在2/3期临床试验中接受评估，Denali公司的目标是在今年完成2/3期临床试验的患者注册。大家都在看▲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] EVALUATING: Neurology，Retrieved April 2, 2024 from https://www.evaluate.com/thought-leadership/evaluating-neurology/?utm_source=Eloqua&utm_medium=Email&utm_campaign=202403-CMP-TL-NEUROLOGY&utm_content=EVALPHARMA-EMN-TL-2403GL-NEUROLOGY-DLRT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