Bayer Schering Pharma AG

HOUSTON, Sept. 9, 2024. Moleculin Biotech, Inc., ("Moleculin" or the "Company), a Phase 3 clinical-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat tumors and viruses, today announced the enrollment and treatment of patients in an Investigator-initiated Phase 2 study evaluating WP1066 in combination with radiation therapy for the treatment of adults with glioblastoma (NU 21C06). The study is being conducted under Northwestern University's Investigative New Drug application (IND) which cross references the Company's own IND, which received clearance from the U.S. Food and Drug Administration (FDA) in April 2022. This trial is funded by the National Institutes of Health (NIH) and BrainUp, a non-profit organization dedicated to bringing awareness to brain cancer. The NU 21C06 trial is a Phase 2, open-label, multi-arm trial of radiation therapy in combination with WP1066 in newly diagnosed IDH (isocitrate dehydrogenase) wild-type, MGMT-unmethylated glioblastoma patients. The primary outcome measure for the study is progression-free survival and secondary outcome measures include tumor microenvironment analysis. Dr. Priya Kumthekar, Associate Professor at Northwestern University and the Co-Investigator for the study commented, "The start of patient dosing is a significant step towards addressing the unmet need in the treatment of glioblastoma. We were very encouraged by the preclinical data that demonstrated by WP1066 in combination with radiation therapy increased survival and induced anti-tumor immune responses and are excited to finally be treating patients with this approach." Dr. Amy Heimberger, Co-Investigator and Vice Chair for Research, Department of Neurological Surgery for Northwestern University added, "The significance of the unmet need can be seen in the pace of recruitment for this trial. Within the first few months of opening the trial, we have four subjects active in the study and expect to fill the safety lead-in group of six subjects soon. If treatment is well tolerated in these subjects, we will continue enrollment for the balance of the trial." WP1066 is Moleculin's flagship Immune/Transcription Modulator designed to stimulate the immune response to tumors by inhibiting the errant activity of regulatory T cells while also inhibiting key oncogenic transcription factors, including p-STAT3 (phosphorylated signal transducer and activator of transcription 3 ), c-Myc (a cellular signal transducer named after a homologous avian virus called Myelocytomatosis) and HIF-1α (hypoxia-inducible factor 1α). These transcription factors are widely sought targets because of their role in cancer cell survival and proliferation, angiogenesis (coopting vasculature for blood supply), invasion, metastasis, and inflammation associated with tumors. "The NU 21C06 study is a critical next step in the development of STAT3-targeting technologies. STAT3 has long been a sought-after but elusive cell-signaling target in cancer therapy but the work we are doing in collaboration with Northwestern University supported by the NIH and BrainUp® could change all that. We believe that based on the data demonstrated to date, WP1066 has the potential for significant anti-tumor activity in a wide range of hard-to-treat cancers," added Mr. Walter Klemp, Chairman and Chief Executive Officer of Moleculin. Glioblastoma is a common type of tumor originating in the brain. The average annual age-adjusted incidence rate of glioblastoma is 3.19 per 100,000 persons in the United States. 1 Glioblastoma is the most aggressive malignant primary brain tumor with a median survival of only 15 months 2 . It is the most common malignant primary brain tumor making up 54% of all gliomas and 16% of all primary brain tumors.3 Despite advancements for other cancers, the survival rate for glioblastoma has not changed significantly in the last three decades. 4 Moleculin has received Orphan Drug Designation for WP1066 for the treatment of brain tumors, as well as Rare Pediatric Disease designation for three other pediatric indications. For more information about the NU 21C06 Phase 2 study, visit clinicaltrials.gov and reference identifier NCT05879250. About Moleculin Biotech, Inc. Moleculin Biotech, Inc. is a Phase 3 clinical stage pharmaceutical company advancing a pipeline of therapeutic candidates addressing hard-to-treat tumors and viruses. The Company's lead program, Annamycin, is a next-generation anthracycline designed to avoid multidrug resistance mechanisms and to eliminate the cardiotoxicity common with currently prescribed anthracyclines. Annamycin is currently in development for the treatment of relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases. The Company is initiating the MIRACLE (Moleculin R/R AML AnnAraC Clinical Evaluation) Trial (MB-108), a pivotal, adaptive design Phase 3 trial evaluating Annamycin in combination with cytarabine, together referred to as AnnAraC, for the treatment of relapsed or refractory acute myeloid leukemia. Following a successful Phase 1B/2 study (MB-106), with input from the FDA, the Company believes it has substantially de-risked the development pathway towards a potential approval for Annamycin for the treatment of AML. This study is subject to appropriate future filings with potential additional feedback from the FDA and their foreign equivalents. Additionally, the Company is developing WP1066, an Immune/Transcription Modulator capable of inhibiting p-STAT3 and other oncogenic transcription factors while also stimulating a natural immune response, targeting brain tumors, pancreatic and other cancers. Moleculin is also engaged in the development of a portfolio of antimetabolites, including WP1122 for the potential treatment of viruses, as well as certain cancer indications. Forward-Looking Statements Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995, which involve risks and uncertainties. Although Moleculin believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward-looking statements. Moleculin has attempted to identify forward-looking statements by terminology including 'believes,' 'estimates,' 'anticipates,' 'expects,' 'plans,' 'projects,' 'intends,' 'potential,' 'may,' 'could,' 'might,' 'will,' 'should,' 'approximately' or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. These forward-looking statements, include, but are not limited to, the ability for the study to fill the safety lead-in group of six subjects on a timely basis and whether the preclinical results will be demonstrated in the Phase 2 trial. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including those discussed under Item 1A. "Risk Factors" in our most recently filed Form 10-K filed with the Securities and Exchange Commission (SEC) and updated from time to time in our Form 10-Q filings and in our other public filings with the SEC. Any forward-looking statements contained in this release speak only as of its date. We undertake no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events. References 1 Thakkar J, Dolecek TA, Horbinski C, Ostrom QT, Lightner DD, Barnholtz-Sloan JS, et al. Epidemiologic and molecular prognostic review of glioblastoma. Cancer Epidemiol. Biomarkers Rev. 2014;23(10):1985-96. 2 Koshy M, Villano JL, Dolecek TA, Howard A, Mahmood U, Chmura SJ, et al. Improved survival time trends of glioblastoma using the SEER 17 population-based registries. J Neuro Oncol. 2012;107(1):207-12 3 Ostrom QT, Gittleman H, Farah P, Ondracek A, Chen Y, Wolinsky Y, et al. CBTRUS statistical report: Primary brain and central nervous system tumors diagnosed in the United States in 2006-2010. Neuro Oncol. 2013;15 Suppl:2ii-56. 4 De Vleeschouwer S, editor. Brisbane (AU): Codon Publications; 2017 Sep 27. Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.

近日，Citeline发布了《2024年医药研发年度回顾》, 一起来了解下最新药物研发领域的动态和趋势。（后台回复“2024年医药研发趋势”可获取PDF&PPT最全合集） 1 近几年研发管线总规模 2024年总计有22,825 款在开发药物，相较2023年21,292款增长7.20%，这与五年的平均增长率7.14%的数字较契合。总体管线数量再创新高，医药行业呈现持续增长的迹象。 （注：本报告中“管线”即制药公司在研的各个阶段药物，包括已上市但还有其他在研适应症的药物，上市且完成开发的药物不包括在内，数据采集时间为2024 年1月） 在过去一年，增加的新药总数为5428种，但期内有3,895 款候选药物退出了在研管线，最终净增1533种，相较之前2023年流失数字基本相同，说明这一年管线流失率虽然较高，但流失变化量比较稳定。 从研发阶段来看，2023~2024年期间临床l期增长13.5%，临床ll期增长 7.8%，均比去年有较大增长。临床前增长5.5%，低于总体增幅但高于去年4.3%。而进入III 期的药物数量增长明显放缓，仅增长了1.5%，后期管线开发仍面临强大阻力。 而事实上 III 期试验是药物开发中最费钱的阶段。行业分析机构数据显示，药物开发成本逐年递增，而投资回报率却在不断下降。 2 头部企业 2024 年按管线规模排名的前10家医药公司如下图。其中，罗氏蝉联两年首位，辉瑞、礼来、阿斯利康管线排名有所提高，这与2023年大型并购活动增多有关。 辉瑞去年12月完成了对 Seagen的收购，产品组合比去年同期增长约 20%。 阿斯利康在收购了Neogene Therapeutics 和CinCor Pharma之后跃居第 3 位 礼来在收购Versanis Bio、DiCeTherapeutics 和Sigilon Therapeutics后排名由第10 位骤升至第 4 位，收获颇丰。 据Pharmaprojects 报告，2023年完成了88项并购，较2022年记录的81项略有增加。总体而言，全行业的并购活动几乎较去年谷底略有上升。 诺华由于管线缩减由第二位降至第六位，而江苏恒瑞医药凭借 38.7%的管线增长率成为首个有史以来进入前 10 位的总部位于中国的公司。其自主研发的药物比例近94% ！恒瑞排名的“大跃进”一定程度上彰显了中国医药行业的快速发展。 从近十年的发展趋势来看，大型药企所占的管线份额在逐渐下降 ，而小公司（只有一种或两种药物的公司）份额有所上升。 排名前10的药企仍对多种治疗领域表现出兴趣，前5的药企开发的药物涉及全部14个治疗领域。 3 治疗领域及适应症 在所有在研管线中，主要领域为癌症（+7.8%）、神经病学（+7.5%）、消化/代谢（+13.1%）和抗感染（+0.8%）。 癌症药物占比在逐年增长。 消化/代谢类药物在2024管线增长率最高，高达13.1%！相比之下，在COVID-19期间大幅增长的抗感染类药物研发增长几乎完全停滞，增幅仅为0.8%。总之，热门药物增幅略降，去年制药公司产品组合的各治疗领域候选药物的分布变宽泛。 就细分的适应症来说，主要癌种为乳腺癌（+6.8%）、NSCLC（+9.2%）、结直肠癌（+11.3%）、胰腺癌和卵巢癌。 阿尔茨海默氏症依然是进入前 10 排行榜的唯一的另一种非肿瘤疾病，同时肝癌首次进入前10，大幅增长12.8%。值得注意的是，治疗肥胖的药物研发，增幅达30%！ 4 MOA及靶点 在MOA排名中，免疫肿瘤学在首位（+15.9%），但其进入研发后期（预注册PR、注册R和上市L）的药物百分比仅为2.7%。 在排名前25的靶点中，癌症相关靶点占65.3%，有明显增长趋势的靶点为EGFR（+15.1%）、PD-1和GLP1R。此外，受Wegovy推出和巨大成功以及肥胖和糖尿病领域正在进行的研究推动，GLP1R显著增长34.1%！ CAR-T 疗法广泛采用的CD3e取代另一种基于IO的靶点PD-L1回归榜首。传统抗癌靶点Her2升至第2位，另一种CAR-T采用的IO的靶点CD19也被降至第5位。 2023年新发现的药物靶点数量93种，与2022年差别不大。 5 个别领域发展趋势 罕见病 罕见病药物开发的数量持续上升，2024年初的达到7,191 款，较去年上升了 7.6%，占管线总药物的 31.5%。 按各TA分析，消化/代谢疾病占罕见病的最大份额，为19% 。 CGT 过去十年，细胞与基因治疗呈持续上升趋势。 使用的病毒载体方面，腺相关病毒 (AAV) 使用最为普遍，疗法最成熟，这种病毒载体最常用于体内基因治疗，AAV 似乎已成为首选武器。 比较常用的细胞类型是T细胞、干细胞和NK细胞。 6 研发地区 从研发地区分布来看，药物研发的风继续从西方吹向东方。总部设在美国的药物研发公司数量由去年的43%降至今年的39%，而设在中国的医药公司比例却由13%升至16%。 目前在进行药物开发的地区，美国仍占主导地位，在所有在研药物中，49.1%的药物研发活动在美国，但低于去年的51.1%，并首次跌至 50%标志线以下。而中国的份额由23.6% 增至 26.7%。亚洲另一个国家韩国悄然升至第三位。 注：仅统计在研药物比例为 5% 或更高的国家/地区，取前10展示 7 中国已成为第二大新药开发国家 从2019年开始，中国医药开发在过去五年迎来了鼎盛时期，增幅高达200.9%，部分得益于政府在医药领域的投资剧增。从1997年才出现在医药市场中且仅有一款在研药物到如今占全球在研药物的26.7%，中国经历了惊人的增长，已然成为第二大新药开发国家。 2023~2024年期间，10大中国公司中有9 家公司的在研管线规模出现增长。 中国管线药物排名前 10 的疾病中癌症占9种，前3分别为NSCLC、乳腺癌、胃肠道肿瘤。 8 2024年面临挑战 VS 迎来转折？ 2024年医药行业的预测结果又是怎样的呢？ BioMedtracker在2023年报告了2,218项医药交易，包括1,237项融资交易。这些数字与上一年的2,600项与1,340项相比有所下降，且下降趋势还在继续。 英国生命科学风险投资公司 Start Codon 的投资总监Michael Salako特别指出:"有迹象表明，2024 年的融资环境将普遍面临困难。"预计2024年生物制药初创公司的融资环境普遍面临挑战，"日本初创公司Noile-lmmune Biotech 的CEO Koji Tamada对此表示同意。 而部分行业人士态度比较乐观。“我确信2024 年是生物技术领域的转折点。"Adial Pharmaceuticals 的首席执行官Cary Claiborne说道。“低估值可能引发收购热潮，从而在逆境中变成机会。”安斯泰来制药集团业务开发负责人高级副总裁Issei Tsukamoto 对此表示同意。 End 声明：本公众号所有发文章(包括原创及转载文章)系出于传递更多信息之目的，且注明来源和作者。本公众号欢迎分享朋友圈或大群，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/商务/投稿 | 联系微信 wfj_GM 商务合作 稿件征集 点击了解详情 往期回顾 1 5月| 20家药企裁员（附名单） 2 18亿美元，ADC又出海! 宜联生物宣布新授权 3 2024 ASCO| 默沙东/科伦TROP2 ADC、信达、礼新CLDN18.2 ADC数据公布

嘉宾简介陈兆荣博士深耕医药研发行业三十余载，在新药临床研发、新药审评、医药注册，医学事务和药物安全等领域有着丰厚经验。在加入祐和前曾在Citrine Medicine, JHL等几家Biotech公司担任CMO。在GSK曾担任副总裁兼卓越注册中心负责人、中国/香港区医学总监。在此之前，他历任Bayer-Schering Pharma、Sanofi高管职位。陈兆荣博士毕业于山东医科大学医疗系，并在澳大利亚阿德莱德皇家医院获得临床药理学博士学位。他曾获阿德雷得皇家医院Florey 研究基金及NH&MRC研究基金。也曾在澳大利亚药品管理局（TGA）从事药物审评与审批工作。祐和医药作为百奥赛图的全资子公司，祐和医药承担着为百奥赛图研发管线进行临床开发的使命。依托强大的临床开发团队及其丰富的临床开发经验，祐和医药聚焦于肿瘤和非肿瘤的抗体药物治疗。公司已构建起针对十多个靶点的强大研发管线。现阶段1个产品已启动国际多中心（MRCT）II期临床研究，3个产品在临床I期，1个产品获得IND批件。详情请访问：www.eucure.com。发展历程201611月，祐和医药科技（北京）有限公司成立12月，获得百奥财富和国投创业的A轮投资20177月，获得百奥财富和国投创业的A+轮投资20183月，完成由招银国际资本领投，本草资本跟投的B轮投资20195月，成立波士顿子公司10月，组建国际化临床开发团队10月，YH001项目IND获得FDA批准2020YH001项目：①联合君实生物PD-1澳洲I期临床研究完成首例患者筛选②项目IND获得NMPA批准YH002项目：①项目IND获得FDA批准②澳洲I期临床研究完成首例患者筛选③项目IND获得NMPA批准YH003项目：联合君实生物PD-1澳洲I期临床研究完成首例患者筛选2021YH001项目：①中国I期临床研究完成首例患者筛选②和YH003在美国进入II期临床研究③II期国际多中心临床试验（MRCT）获NMPA批准YH003项目：①联合君实生物PD-1在澳洲顺利进入II期临床研究②项目IND获得NMPA批准③中国I期临床研究完成首例患者用药④II期国际多中心临床试验（MRCT）获NMPA批准⑤联合PD-1抗体的国际多中心（MRCT）II期临床试验完成首例患者给药YH004项目：澳洲I期临床研究完成首例患者给药技术平台基于RenMice®（RenMab®，RenLite®和RenNano®）平台，百奥赛图建立了六大全人抗体技术平台，满足多种靶点、多种成药形式的抗体药物开发。产品管线Innovative Drug Pipeline质量源于设计，又贯穿始终。QbD大会历经四载，在行业同仁的持续关注和支持下，2024第五届QbD生物药质量科学大会（QbD2024）将于7月26-27日在北京悠唐皇冠假日酒店再次召开。将重点围绕创新生物药的质控、分析、法规等内容展开讨论。从案例、实践去解读创新药质量研究管理的进程，持续深化QbD理念。祐和医药首席执行官&首席医学官陈兆荣将在大会会场四：国际药品监管法规专场为大家带来主题为“遵循监管政策，加速新药研发”的报告，诚邀各位同仁与会！药企福利-享免费观摩门票推荐阅读归来花满树，携手再破千层浪！金斯瑞蓬勃生物倾情加盟QbD2024第五届生物药质量科学大会茵冠生物质量总监邹沛华：间充质干细胞质量控制与质量体系的搭建 | QbD2024风云录泽辉生物质量负责人单晓鹏：细胞治疗产品控制策略 | QbD2024风云录原启生物质量副总裁陶铜静：CGT药物CMC质量控制策略 | QbD2024风云录景达生物CEO杨月峰：通用现货型NK细胞的工艺开发及质量控制 | QbD2024风云录贝思奥生物创始人罗光佐：AAV药物关键质量属性及其分析质控难点 | QbD2024风云录