POLB-001

早先我们介绍过被高瓴清仓，被罗氏退货的Biotech公司HOOKIPA Pharma，现在这家公司又有了新近况，HOOKIPA Pharma正在以全股票交易方式收购专注于罕见病的Poolbeg Pharma，以创建一家合并后的公司，合并之后Hookipa会起头一个3000万美元的私募来为合并后的公司推荐管线。 2023年以来，套着诺奖明星光环的HOOKIPA Pharma境遇不妙，先是首发抗病毒管线毫无效果，接着被罗氏退货，现在公司股价和现金流均亮起红灯，24年年中时，不得不反向分割股票来维持最低上市要求，现在Q3现金及其等价物只有6000万美元左右，按照现行的现金花费速度，在2026年内前就可能无法维持运营。 如何一步步跌落谷底 从HOOKIPA Pharma的发展历史来看，首发管线失败，被罗氏退货，和临床设计遭到诟病是该公司价值大幅缩水的主要诱因。 HOOKIPA Pharma的核心技术是沙粒病毒，理论上能产生比传统溶瘤单疱病毒更持久，靶向更广泛的效果，该公司首先打算将沙粒病毒用作抗病毒领域。 然而，不幸的是，首发管线巨细胞病毒（CMV）疫苗HB-101 II期临床失败，HB-101并没有展现出超过安慰剂的疗效。 随后该公司开始转向肿瘤学。然而，肿瘤学临床设计时HOOKIPA Pharma却有些不严谨，该公司管线Eseba-vec（HB-200）治疗HPV16+癌症患者的数据建立在有相当部分患者有K药使用背景。 数据看似不错，但是缺乏和K药的对照，后续加增Eseba-vec联合K药vsK药单用的临床为时已晚，无法挽回投资者的信心。 至于与罗氏的合作还未开始就胎死腹中，靶向KRAS的HB-700甚至连IND都没来得及申请就被罗氏退货，同时公司因此裁员30%。 现在公司的收入来自于吉利德I期临床合作带来的里程碑收入，但是杯水车薪，这些收入仍然不足够弥补推进Eseba-vec需要的花费，而关键节点在于2025年下半年的II期临床关键数据。 声称有100亿美元前景，但是账上只有1250万美元…… 而被收购方Poolbeg Pharma的财务状况同样不容乐观，此前我们介绍过英国市场整体环境的不景气，伦敦证交所的AIM（另类投资市场）板块中出现了Biotech的退市潮，英国制药公司根本无法从AIM渠道中融到钱。Poolbeg Pharma反倒是退市潮中坚守的一员。 现在这家公司也撑不住了。 在2024年7月时，这家公司从OTCQB市场退市，OTCQB市场是美国的场外交易市场专为中小企业和初创企业提供融资和交易平台。这意味着不光光英国市场拿不到钱，现在美国市场也拿不到钱了。 糟糕的是，Poolbeg Pharma的财务状况也相当不佳，截止2024年年中时只剩下了1010万英镑，约合1250万美元。 然而，在这种情况下，这家公司仍然对自己的管线充满了信心，Poolbeg委托的独立研究更认为核心管线POLB 001有机会扩张出超过100亿美元的市场。因为POLB 001是一种口服片剂，本身的适应症除了治疗病毒感染之外，这家公司主推的适应症就是癌症免疫治疗导致的CRS，当出现CRS后，可能就需要利妥昔单抗托珠单抗，糖皮质激素等应对手段，POLB 001作为口服片剂有机会比双抗和CAR-T中出现CRS应对手段中的其他手段更具有经济性。 很显然，且不说未来双抗和CAR-T的市场发展，单从实际情况来看，这一说法多少有点夸大的成分。 一方面CAR-T和双抗的CRS本身就具有紧急应对手段，POLB 001想要取而代之，需要临床试验证明使用。但是显然Poolbeg Pharma的资金支撑不到II期临床的第一例患者给药。（2025年下半年）顶线数据则需要等到2026年下半年。这也是这家公司亟需资金的原因。 甚至于一些CAR-T和双抗会朝向减少CRS的开发方向发展。因此如果该市场真有如Poolbeg Pharma所吹嘘的那样有100亿美元的前景，那么POLB 001早就被其他大药企看中了。但显然现在的大药企并不会在数据出来之前，对POLB 001操之过急。 另外该公司除了POLB 001之外，还有一项人工智能药物发现项目和一款GLP-1R激动剂，基本都处于非常早期阶段。 总结 总的来看，HOOKIPA Pharma和Poolbeg Pharma其实都缺钱，这次的收购实际上最核心的项目还是其中的3000万美元私募，这可以让这俩家公司至少能拖延到关键节点出数据。 即便某个节点失败了，也可以转头去推动另外一个可能成功的管线。 参考来源： https://polaris.brighterir.com/public/poolbeg_pharma/news/rns/story/xpvv1qx https://www.globenewswire.com/news-release/2024/11/14/2981605/0/en/HOOKIPA-Pharma-Reports-Third-Quarter-2024-Financial-Results-and-Provides-Recent-Business-Updates.html

NEW YORK, NY, USA and VIENNA, Austria I January 02, 2025 I The boards of directors (each a “ Board ”) of HOOKIPA Pharma Inc. (“ HOOKIPA ”) and Poolbeg Pharma plc (“ Poolbeg ”) are pleased to announce that they have entered into non-binding discussions for an all-share acquisition by HOOKIPA of Poolbeg (the “ Potential Combination ”) to create a strong clinical-stage biopharmaceutical company focused on developing and commercialising innovative medicines for critical unmet medical needs, with a special focus on next-generation immunotherapies for the treatment of cancer and other serious diseases (the “ Combined Group ”). The Potential Combination is intended to be implemented by means of a scheme of arrangement under Part 26 of the Companies Act 2006. The Boards believe that the Potential Combination would create a Nasdaq-listed Combined Group operated by a combined management team experienced in successfully developing and commercializing medicines with a focus on execution and operational excellence. The Boards also believe the Potential Combination would create a diversified clinical pipeline led by multi-KRAS targeting HB-700, a next generation immunotherapy potentially offering additional treatment options for cancers with limited treatment options, and Phase 2-ready small molecule POLB 001, a potentially breakthrough orally delivered preventative therapy for cancer immunotherapy-induced CRS, with potential value inflection points in areas of interest in the pharmaceutical industry. Furthermore, the Boards expect the Potential Combination to bolster near-term clinical data catalysts, with clinical data expected across multiple programmes over the next 24-months in large therapeutic areas with unmet medical needs. The Combined Group would also have two partnered programmes with Gilead Sciences, Inc. (“ Gilead ”), offering the potential of significant development and commercialisation milestones in addition to significant sales royalties (if either product is approved) for the Combined Group’s shareholders. HOOKIPA is listed on the Nasdaq Capital Market under the symbol HOOK and Poolbeg is listed on AIM under the symbol POLB. Expected key terms of the Potential Combination based on discussions to date comprise of: The announcement by HOOKIPA of any firm offer under Rule 2.7 of the Code in respect of the Potential Combination is subject to the satisfaction or waiver of a number of customary pre-conditions, including, amongst other things, the satisfactory completion of customary due diligence, finalisation of the terms of the transaction, Board approvals of binding terms and sufficient prior expressions of interest from participants in respect to the Fundraise. Before the announcement of any firm offer under Rule 2.7 of the Code, both HOOKIPA and Poolbeg intend to engage with potential investors in the Fundraise, further details of which will be included in a separate presentation (the “ Fundraise Presentation ”), which upon first use will be made available on HOOKIPA’s website at ir.hookipapharma.com/potential-combination. The Potential Combination would be subject to the approval of both HOOKIPA and Poolbeg shareholders and other conditions. All discussions to date have been non-binding and on a non-exclusive basis, and there can be no assurance that a firm offer will be made or that any transaction will be completed. Information on HOOKIPA HOOKIPA is a clinical-stage biopharmaceutical company focused on developing next generation immunotherapies based on its proprietary arenavirus platform. HOOKIPA’s product candidates are designed to induce specific, robust and durable CD8+ T cells and antibodies to eliminate cancers and serious infectious diseases. HOOKIPA’s pipeline includes biological therapies for oncology, targeting human papillomavirus type 16-positive (“ HPV16+ ”) cancers, KRAS mutated cancers, and other targets. In addition, HOOKIPA has partnered with Gilead to develop therapies that are intended to provide functional cures for hepatitis B virus (“ HBV ”) and human immunodeficiency virus-1 (“ HIV ”). HOOKIPA’s next-generation vaccine platform is designed to supercharge immunity with its T cell activation platform based on work of Nobel laureate and HOOKIPA co-founder, Rolf Zinkernagel. Further details of HOOKIPA’s platform are set out in Appendix C. Information on Poolbeg Poolbeg is a clinical-stage biopharmaceutical company focused on acquiring, developing and commercialising innovative medicines that will help improve the lives of patients with rare and orphan diseases and where there is a high unmet medical need. Poolbeg’s clinical programmes target large addressable markets including cancer immunotherapy-induced Cytokine Release Syndrome (“ CRS ”), infectious disease, and metabolic conditions such as obesity with the development of an oral GLP-1R agonist. Further details of Poolbeg’s platform are set out in Appendix C. SOURCE: Poolbeg