Mirvetuximab soravtansine

来源：药渡 撰文：天际微步    编辑：哦呼 武田制药（武田药品工业株式会社，Takeda）是一家位于日本东京的老牌药企，从中草药销售起步已经拥有243年历史，如今已成为亚洲最大的制药公司，在全球MNC药企中排名TOP20之列。2023财年武田制药的营收为4.26万亿日元，同比增长5.9%，在全球药企中的排名为第13位。 与强生、辉瑞等其他大MNC有较强自研能力不同，武田制药从药物销售贸易起步，积攒了原始资本，后在仿制美国药上布局，凭借四大me too品种（亮丙瑞林微球、兰索拉唑、坎地沙坦、吡格列酮）崛起。在这过程中虽有布局自主研发管线，但效果不佳，只得不断地并购或者交易来拓展管线，因此武田制药的交易事件相当频繁。 从2005年第一次海外收购美国syrrx inc，武田逐渐形成了以肿瘤、消化、罕见病和神经为主，疫苗和血制品为辅的业务版图。从近两年武田的药物交易事件来看一下武田的重点方向和布局策略变化。 2023年以来武田的药物交易事件盘点 从2023年初开始武田制药就一直在进行内部调整，以应对重磅产品专利到期（替度鲁肽、利右苯丙胺等）带来的业绩冲击。一方面，砍掉尚未见效的早期管线、收缩战线，据公开资料，2023年武田制药共有包括肺癌产品Exkivity、阿尔茨海默病药物DNL-919等18款药物从管线中剔除，还终止了与JCR制药公司的AAV基因治疗，与Noile-Immune的新一代实体瘤CAR-T疗法等的项目合作。在其362个药物管线中，135个已经处于终止状态。 图1.武田制药的管线进展情况（来源：药渡数据） 但另一方面，武田仍继续购进具有上市潜力的管线来形成新增长力。据不完全统计，2023年至2024年6月份，武田制药产生的药物交易有12笔。 表1.2023年以来武田制药的药物交易事件（来源：药渡数据及公开资料整理） 2024年6月14日，武田制药引进亚盛医药的奥雷巴替尼，用于治疗慢性髓性白血病（CML），获得其中国以外的商业化权利。此前，武田已有CML治疗药物泊那替尼，但即将在2026年专利到期，引进奥雷巴替尼可以继续利用此前积累的市场方面优势将新产品大卖，保持在CML治疗领域的领先地位。 2024年5月23日，与上海达歌生物达成独家许可协议，以发现和开发用于肿瘤学、神经科学和炎症领域多个靶点的新型分子胶降解剂。达歌生物将利用其研发平台，针对武田选定的特定疾病靶点，发现、验证和优化分子胶降解剂，在达到一定进展阶段后，这些项目将移交给武田进行进一步开发和商业化。此次主要利用达歌生物的研发创新能力来帮助武田在分子胶降解剂这一类新型药物上进行探索。 2024年5月13日，武田制药引进AC Immune的阿尔茨海默症Aβ疫苗疗法ACI-24.060，阿尔茨海默症也属于武田重点布局的神经性疾病范畴，需要一款重磅新药来塑造竞争力。 2024年4月16日，与Kumquat生物科学达成战略合作和独家全球许可协议，推进新型口服免疫肿瘤药物，武田承担并资助除了由Kumquat主导的1期试验活动之外的所有开发和商业化活动。 2024年1月31日，与Protagonist达成全球许可和合作协议，共同开发罕见血液病药物Rusfertide（其基于多肽的新化学实体），目前正处于关键的 3 期试验，用于治疗真性红细胞增多症。 2023年期间，先后引进和黄医药的用于治疗经治转移性结直肠癌的呋喹替尼， Nimbus Lakshmi的银屑病药物TYK2抑制剂NDI-034858，中国生物制药子公司F-star的肿瘤多特异性抗体免疫疗法，ImmunoGen针对铂耐药卵巢癌的FRα ADC新药ELAHERE，以及AcuraStem的用于治疗肌萎缩性侧索硬化症（ALS）的创新反义寡核苷酸（ASO）疗法AS-202，信念医药治疗B型血友病的AAV基因治疗BBM-H901注射液…… 武田的布局重点和变化？ 从2023年以来的交易情况看，武田的布局焦点，仍然是围绕其四大核心疾病业务领域，包括肿瘤药物（亚盛医药的奥雷巴替尼、Kumquat合作的肿瘤药物、F-star的肿瘤多特异性抗体、ImmunoGen的FRα ADC新药），偏爱和引进了当下热点的ADC和多抗药物；胃肠道药物（和黄的呋喹替尼），通过引进销量不错的呋喹替尼来延续消化疾病领域的优势；罕见病药物（罕见血液病药物Rusfertide、肌萎缩性侧索硬化症的ASO疗法、血友病的AAV基因治疗）。神经疾病药物（AC Immune的阿尔茨海默症Aβ疫苗）、还有自免疾病（银屑病药物TYK2抑制剂）。 从药物类型看，引进新技术、新型疗法如分子胶降解剂、AAV基因疗法、多肽药物分子、ADC等，这些新药物能解决传统药物难以解决的问题，更具明显疗效和价值。 总之，武田近两年的布局仍然是主要基于其多年来在肿瘤、胃肠道、罕见病和神经等疾病领域所积累的市场销售优势，引进相应的新产品管线，来延续和保持公司的领先优势。在布局疾病适应症上肿瘤已成为重量板块，相关产品数量最多，这也是因为当下肿瘤仍是需求空间最大的疾病类型。在药物类型上新兴技术的引进也是围绕前述疾病类型，可见武田深耕的决心。 编者按： 近年来，武田制药通过不断引进新的药物管线和技术，以应对市场挑战，保持公司的领先优势。在疾病领域方面，武田制药始终围绕上述四大核心业务领域进行布局。在技术引进方面，武田制药注重新兴技术和新型疗法的研发与应用。未来，随着科技的不断进步和市场需求的不断变化，武田制药能否继续保持敏锐的市场洞察力和创新能力？药渡将持续关注。 *声明：本文仅是介绍医药疾病领域研究进展或简述研究概况或分享医药相关讯息，并非也不会进行治疗或诊断方案推荐，也不对相关投资构成任何建议。内容如有疏漏，欢迎沟通指出！ 点击下方“药渡“，关注更多精彩内容 100%预防艾滋！吉利德科学创新药物Lenacapavir 3期临床结果喜人 欧洲杯来了！哪些国家最有望夺得“最佳药物管道”奖杯？ 美国Arthrosi公司创新药AR882溶解痛风石，2期临床试验成果亮相2024欧洲风湿病学大会 点击下方关注，聚焦医药创新一手资讯

Multiple patients have now exceeded 30 weeks on treatment Boston (June 25, 2024)—Allarity Therapeutics, Inc. (“Allarity” or the “Company”) (NASDAQ: ALLR), a Phase 2 clinical-stage pharmaceutical company dedicated to developing personalized cancer treatments, today announced that multiple patients in its Phase 2 clinical trial of stenoparib for advanced recurrent ovarian cancer have been on treatment for more than 30 weeks. The continued durability of clinical benefit further bolsters the Company’s announcement in early May 2024 that stenoparib had shown clear clinical benefit, including significant tumor shrinkage and long-term disease stability, in patients who had been heavily pre-treated for their ovarian cancer and otherwise have limited life expectancy. These results provided clinical proof of concept for stenoparib as a treatment in this patient population, prompting the company to halt patient enrollment to focus its resources on developing a follow-on trial designed to accelerate the path for stenoparib toward regulatory approval. Kathleen N. Moore, MD, MS, Deputy Director of the Stephenson Cancer Center, Professor of the Section of Gynecologic Oncology and the Virginia Kerley Cade Chair in Developmental Therapeutics, serves as the Principal Investigator for the current Allarity trial: “Inhibition of poly-ADP-ribose polymerase or PARP has been transformational for the treatment of ovarian cancer with first-generation agents significantly improving progression-free and overall survival especially among patients with biomarkers for their use. However, there are a significant number of patients who either don’t benefit at all from inhibition of PARP or only modestly and for them, continual development of next-generation agents remains a high priority. The promising results observed with stenoparib - particularly with the tolerability demonstrated so far, warrant further development of this drug. I look forward to continuing the discussions with the team at Allarity to refine the future trial design and subsequently continue to investigate the potential of this novel PARP/Tankyrase inhibitor.” "The continued durability of clinical benefit from stenoparib furthers our enthusiasm for stenoparib for patients who traditionally have limited treatment options and often only a few months of continued life expectancy. We continue to see patients maintain their quality of life with minimal side effects for extended periods,” stated Allarity Therapeutics CEO Thomas Jensen. He elaborated, “Not only does the safety pro out when compared to chemotherapies, which is often the alternative for this patient group, but the safety metrics are indeed also favorable when compared to first-generation PARP inhibitors. As we see it, stenoparib may represent the next-generation alternative for advanced ovarian cancer patients. Therefore, we are aggressively working with Dr. Moore and other leading experts to design a trial that will help advance and quicken stenoparib’s clinical progress toward registration with the FDA.” After conducting a detailed review of the stenoparib monotherapy trial data, including the data announced today, the Company has concluded that these data may be of significant interest among key oncologists, potential commercial partners, and other relevant stakeholders and therefore warrant presentation within a high-level scientific conference. Further release of these clinical data is intended to be staged to comply with the common rules of scientific conferences, which do not allow data to be published before the event. The PARP inhibitor market saw a major shift in 2022 as rucaparib, olaparib, and niraparib were withdrawn for heavily pretreated ovarian cancer patients, underscoring the need for new, effective PARP inhibitors with a more favorable safety profile. The PARP inhibitor market is expected to reach $22 billion in revenue by 2028, and has historically seen significant partnerships and acquisitions. Recent interest in the market has focused on the development of PARP inhibitors with improved tolerability and safety pro leading to notable activity in the sector. For example, one significant acquisition included a multi-asset deal potentially totaling $1.5 billion, which featured an advanced PARP inhibitor. Stenoparib is orally available and also shares an advantaged safety pro to 1st generation PARP inhibitors. It also inhibits the Tankyrase 1 and 2 enzymes, which helps block the activity of the WNT/Beta-catenin pathway, a pathway often overactive in ovarian and many other solid cancers. The safety and unique, dual inhibitory activities of stenoparib make it a differentiated and compelling potential therapeutic product. Background Information about the Trial The above-mentioned trial is a Phase 2, prospective open-label, single-arm study with multiple sites in both the US and the UK. Investigators prescreened women with advanced, recurrent ovarian cancer using Allarity’s DRP® companion diagnostic (CDx), which comprises a complex transcriptomic signature of 414 mRNA biomarkers indicative of drug response or resistance. Each participant was assigned a DRP score, and those with scores above 50 -suggesting a higher likelihood of benefiting from treatment - were selected to receive stenoparib. The selected patients were administered stenoparib under a revised protocol implemented in Q1 2023, which involved a twice-daily dosing regimen (200 mg in the morning and 400 mg in the evening) instead of the previous once-daily 600 mg dose. This change was made to optimize daily drug exposure and target inhibition. The patients enrolled have advanced through multiple lines of therapy, including platinum, taxanes, anti-angiogenesis inhibitors, and even the recently approved Antibody Drug Conjugate, Elahere. Importantly, all but two enrolled patients to date have been previously treated with a PARP inhibitor. These patients have few, if any, effective treatment options and typically advance through available therapies after only a few months. About stenoparib Stenoparib is an orally available, small-molecule dual-targeted inhibitor of PARP1/2 and Tankyrase 1 and 2. At present, tankyrases are attracting significant attention as emerging therapeutic targets for cancer, principally due to their role in regulating the Wnt signaling pathway. Aberrant Wnt/β-catenin signaling has been implicated in the development and progression of numerous cancers. By inhibiting PARP and blocking Wnt pathway activation, stenoparib’s unique therapeutic action shows potential as a promising therapeutic. Allarity has exclusive global rights for the development and commercialization of stenoparib, which was originally developed by Eisai Co. Ltd. and was formerly known under the names E7449 and 2X-121. About the Drug Response Predictor – DRP® Companion Diagnostic Allarity uses its drug-specific DRP® to select those patients who, by the gene expression signature of their cancer, are found to have a high likelihood of benefiting from a specific drug. By screening patients before treatment, and only treating those patients with a sufficiently high, drug-specific DRP score, the therapeutic benefit rate may be significantly increased. The DRP method builds on the comparison of sensitive vs. resistant human cancer cell lines, including transcriptomic information from cell lines combined with clinical tumor biology filters and prior clinical trial outcomes. DRP is based on messenger RNA expression profiles from patient biopsies. The DRP® platform has proven its ability to provide a statistically significant prediction of the clinical outcome from drug treatment in cancer patients dozens of clinical studies (both retrospective and prospective). The DRP platform, which can be used in all cancer types and is patented for more than 70 anti-cancer drugs, has been extensively published in the peer-reviewed literature. About Allarity Therapeutics Allarity Therapeutics, Inc. (NASDAQ: ALLR) is a clinical-stage biopharmaceutical company dedicated to developing personalized cancer treatments. The Company is focused on development of stenoparib, a novel PARP/Tankyrase inhibitor for advanced ovarian cancer patients, using its DRP® companion diagnostic for patient selection in the ongoing phase 2 clinical trial, NCT03878849. Allarity is headquartered in the U.S., with a research facility in Denmark, and is committed to addressing significant unmet medical needs in cancer treatment. For more information, visit . Follow Allarity on Social Media LinkedIn: X: Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements provide the Company’s current expectations or forecasts of future events. The words “anticipates,” “believe,” “continue,” “could,” “estimate,” “expect,” “intends,” “may,” “might,” “plan,” “possible,” “potential,” “predicts,” “project,” “should,” “would” and similar expressions may identify forward-looking statements, but the absence of these words does not mean that a statement is not forward-looking. These forward-looking statements include, but are not limited to, the impact of recent clinical and operational achievements on future trial designs, potential commercial partnerships, planning and carrying out registrational intent clinical trials, the anticipated regulatory progress of stenoparib following the early conclusion of our Phase 2 clinical trial, and the possibility that there may not be any presentation at a scientific event. Any forward-looking statements in this press release are based on management’s current expectations of future events and are subject to multiple risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risks associated with maintaining compliance with Nasdaq's continued listing requirements, the trading price of Allarity’s shares of common stock may be volatile and other risks inherent in Allarity’s business, including, the risk that the Company is not able to raise sufficient capital to support its current and anticipated clinical trials, the risk that early results of a clinical study do not necessarily predict final results and that one or more of the clinical outcomes may materially change following more comprehensive reviews of the data, and as more patient data become available, the risk that results of a clinical study are subject to interpretation and additional analyses may be needed and/or may contradict such results, the receipt of regulatory approval for stenoparib or any of our other therapeutic candidates and companion diagnostics or, if approved, the successful commercialization of such products, the risk of cessation or delay of any of the ongoing or planned clinical trials and/or our development of our product candidates, and the risk that the results of previously conducted studies will not be repeated or observed in ongoing or future studies involving our therapeutic candidates. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in our Form S-1 registration statement filed on April 17, 2024, and our Form 10-K annual report on the Securities and Exchange Commission (the “SEC”), available at the SEC’s website at and as well as discussions of potential risks, uncertainties and other important factors in the Company’s subsequent filings with the SEC. All information in this press release is as of the date of the release, and the Company undertakes no duty to update this information unless required by law. ### Company Contact: investorrelations@allarity.com Media Contact: Thomas Pedersen Carrotize PR & Communications +45 6062 9390 tsp@carrotize.com Attachment Allarity Therapeutics Press Release - Stenoparib Continues to Show Extended Clinical Benefit in AOC