PT-112

▲5月30-31日 第八届广州生物医药创新者峰会 扫码立即报名 注：本文不构成任何投资意见和建议，以官方/公司公告为准；本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及），不代表平台立场。任何文章转载需得到授权。药融云数据（www.pharnexcloud.com）监测显示：赛生药业控股有限公司（6600.HK）（简称：赛生药业）是一家拥有产品开发和商业化集成平台的生物制药公司，战略上专注于在中国规模较大、增长迅速且存在巨大未被满足医疗需求的治疗领域，包括肿瘤科及重症感染。凭借赛生药业的产品开发及商业化实力，公司在重点治疗领域均衡和优质的上市产品及具有较佳潜力的在研产品的开发及商业化组合方面已建立良好的往绩记录。第一次私有化赛生药业前身是 SciClone US，最早成立于1990年，早期创始人为创始人Thomas E. Moore和Nelson M. Schneider；于1992 年在美国纳斯达克上市，是一家老牌上市公司。2013年新的中国管理团队加入公司，后公司在2017年被私有化。(2017年6月7日，由德福资本/GL发起、中银投资、鼎晖投资、上达资本、Boying  Investment组成的买方财团Silver Biotech与SciClone US签订合并协议，以每股11.18美元的现金对价，收购SciClone US所有发行在外的普通股。)港股上市2021上市招股书，所示图片进展2021 年“全新”的赛生药业于中国香港上市，当时募集资金总额约为21.80亿港元。目前赛生药业三大业务：自有特色产品日达仙（胸腺法新的原研产品）(港股上市前2017~2019年间，日达仙分别实现销售额人民币11.26亿元、人民币11.68亿元和人民币13.49亿元)；授权引入候选新药产品（肿瘤与抗感染领域）；销售合作推广分销服务（辉瑞及百特成熟产品）。具体到新药引进/License-in合作开发方面：根据赛生药业2022年8月与美纳里尼亚太公司达成的许可及合作协议，赛生药业可根据美纳里尼与 Melinta Therapeutics, Inc. 此前签订的相关协议，在中国境内独家开发和商业化Vaborem®（注射用美罗培南韦博巴坦）。2023年3月，赛生药业递交的Vaborem®临床试验申请获得了中国国家药品监督管理局（NMPA）批准。Vaborem®在中国开展的III期临床试验于2023年7月完成首例受试者入组给药，并于2024年1月完成全部受试者入组。药融云数据www.pharnexcloud.com；截图来源该研究是在中国开展的一项随机、双盲双模拟、阳性药物对照(辉瑞的注射用哌拉西林钠他唑巴坦钠 )、多中心的III期临床试验，旨在评价Vaborem®在复杂性尿路感染（包括急性肾盂肾炎）患者中的有效性和安全性，由复旦大学附属华山医院王明贵教授和上海交通大学医学院附属仁济医院郑军华教授担任主要研究者，在全国27家研究中心共入组了108例受试者。该研究结果，及同时开展的一项在中国健康志愿者中评估Vaborem®药代动力学特征的研究结果，将用于桥接国外临床试验数据，并支持Vaborem®在中国的上市申请。其他新药布局：ORSERDU®（艾拉司群）ORSERDU®（艾拉司群）是一款口服选择性雌激素受体降解剂，用于治疗既往至少接受过一线内分泌治疗后疾病进展的雌激素受体1基因（ESR1）突变的雌激素受体（ER）阳性、人类表皮生长因子受体2（HER2）阴性晚期或转移性乳腺癌的绝经后女性和成年男性。Vibativ®(telavancin)Vibativ®是新型脂糖肽类抗生素，用于治疗金黄色葡萄球菌所致的医院获得性细菌性肺炎（HABP）与呼吸机相关细菌性肺炎（VABP），以及复杂性皮肤及皮肤软组织感染（cSSSIs）。RRx-001RRx-001是一款具有First-in-Class（FIC）潜质的小分子药物，具有多种作用机制，包括CD47-SIRPα靶向、RONs生成以及表观遗传调节。RRx-001可以对多种肿瘤微环境异常起到调节作用，包括：下调CD47-SIRPα, 使肿瘤相关巨噬细胞（TAM）复极化，由抗炎症M2表型转为促炎症 M1表型；通过表观遗传抑制活性激活抑癌基因，逆转化疗耐药；使肿瘤血管正常化以增加化疗药物渗透，产生代谢产物RONs导致肿瘤细胞坏死。OmburtamabOmburtamab是一种放射性核素碘131标记的靶向B7-H3（CD276）的单克隆抗体，靶向实体肿瘤中表达B7-H3的细胞，并结合到B7-H3分子上的生物功能的关键区域--FG环依赖型构象。药融云数据www.pharnexcloud.com；截图来源PEN-866PEN-866是一款小分子偶联药物，药物的一端为可靶向结合被激活的HSP90蛋白，另一端为拓扑异构酶抑制剂（SN-38）。PEN-866的独特桥接设计及细胞毒素释放机理使得SN-38可以特异性地在肿瘤部位聚集，同时随着连接分子的持续分解，持久地在肿瘤部位释放，并最终造成DNA的破坏及肿瘤的消退。PT-112这是一种创新型铂类小分子药物，通过细胞凋亡、免疫特性及高耐受性来抗肿瘤效果。PT-112的设计初衷是为了减少传统铂类药化疗中的毒性及耐药性。ABTL-0812这是一种具有独特作用机制的潜在候选药物。ABTL-0812通过与核受体PPARα和γ偶联来抑制PI3K/Akt/mTOR (PAM) 通路，从而诱导与Akt结合并阻碍其激活的假激酶TRIB3，并导致mTOR发生抑制，从而导致自噬依赖性癌细胞死亡。HSP90-PI3K偶联物HSP90-PI3K偶联物是一款小分子偶联药物，由磷脂酰肌醇3-激酶（PI3K）抑制剂、连接体与热休克蛋白90（HSP90）结合构成。再次被提出要约私有化2023财年，赛生药业营收为31.56亿元，同比增长约为14.8%；净利润同比增长增31.2%至11.2亿元。此前在2024年3月28日，德福资本及赛生药业联合公告：3月19日，要约人(Silver Pegasus Investment Limited)根据公司法第86条以协议安排方式将赛生药业私有化，要求董事会向计划股份持有人提呈建议，其中涉及注销计划股份，作为代价，就每股计划股份以现金向计划股东支付注销价（以换取每股计划股份现金18.8港元），并撤销股份于联交所的上市地位。注销价18.8港元较不受干扰日期收市价每股14.04港元溢价约33.90%，较最后交易日(2024年3月18日)收市价每股16.04港元溢价约17.21%。药融圈密切关注后续进展。参考：NMPA/CDE；药融云数据，www.pharnexcloud.com；FDA/EMA/PMDA；相关公司公开披露；www.sciclone.com；http://www.sciclone.com/Modules/research.aspx；https://pharma.bcpmdata.com/database/1/detail/39/Wsbrmo4B6rPXm79Mnp50?aliasId=d361ca6640；https://quote.eastmoney.com/hk/06600.html；等等。 本文仅用于向医疗卫生专业人士提供科学信息，不代表平台立场，不作任何用药推荐 活动推荐  5月 • 第八届大湾区生物医药创新者峰会  关键词：  小分子，XDC，多肽及GLP-1热点话题，80+行业专家（点击下方图片查看详情）▼【关于药融圈】药融圈PRHub旨在帮助生物医药科技型企业进行品牌推广及商务拓展服务，针对客户的真实需求制定系统化解决方案，通过“翻译-降维-场景化”将客户的品牌信息以直白易懂的方式被公众知悉，同时在流量渠道覆盖100万+垂直用户基础上实现合作目的，帮助合作伙伴完成从品牌开始到商务为终的闭环营销服务。我们已经完成了数十场线下1000人规模的生物医药研发类会议，涵盖小分子新药，大分子新药，改良型新药，BD跨境交易等多个领域，服务了百余家上市/独角兽/生物技术/制药企业。

Ongoing clinical trial has reached target enrollment of 109 patients across clinical sites in the United States and France Open-label dose optimization and proof of concept study of PT-112 monotherapy includes interim data results, with top-line read-out and conference presentations expected in late 2024 NEW YORK, March 8, 2024 /PRNewswire/ -- Promontory Therapeutics Inc., a clinical stage pharmaceutical company advancing immunogenic small molecule approaches in oncology, has completed enrollment of its Phase 2 clinical trial of lead therapeutic candidate, PT-112, reaching its target enrollment of 109 patients with late-line metastatic castration-resistant prostate cancer (mCRPC) (ClinicalTrials.gov Identifier: NCT02266745). The study is evaluating PT-112 in patients who have been treated with at least three prior life-prolonging therapies for mCRPC, and who exhibited radiographic evidence of disease progression at study entry. Patients with bone-only metastatic disease are also included in the study. These requirements create a representative treatment population following standard of care of androgen receptor signaling inhibitors, taxane chemotherapies, and any other drug approved by FDA on the basis of overall survival. The clinical trial enrolled patients across leading sites in the United States (23) and France (9). "This clinical trial is the largest study to date of PT-112 and will establish the optimal dose in line with the FDA's Project Optimus, as well as proof of concept in our late-line mCRPC patient population," said Promontory Therapeutics Chief Medical Officer Johan Baeck, MD. "Data from our earlier Phase 1/2 studies have shown that PT-112 is clinically safe and active, and promotes immunogenic cancer cell death induced by the inhibition of ribosomal biogenesis, which is a promising mechanism of action for late-stage patients with prostate cancer — an 'immune-cold' disease with no broadly approved and effective immunotherapy." Promontory Therapeutics plans for a Type C meeting with the FDA in the second half of 2024, followed by an End-of-Phase 2 meeting with FDA and further engagement with European regulatory authorities. In addition to safety and efficacy findings among late-stage metastatic patients, the study aims to generate meaningful supportive data via correlative research, including on immune activation by PT-112 monotherapy as evaluated by the propagation of new T cell populations, as well as reductions in circulating tumor cells and ctDNA. "Promontory is grateful to our clinical partners across the U.S. and France, including Gustave Roussy Paris, for completing enrollment of this critical Phase 2 study. We are confident in PT-112's potential as an effective treatment for patients with mCRPC who have progressed on androgen receptor directed therapy, chemotherapy or radioligand therapy and who lack any effective immunotherapy," said Promontory Therapeutics Chief Executive Officer Robert Fallon. "We look forward to presenting topline safety, efficacy, and correlative data at relevant research and medical conferences later this year." About PT-112 PT-112 is the first small-molecule conjugate of pyrophosphate in clinical development in oncology. PT-112 has numerous advantages — including its tolerability and inhibition of ribosomal biogenesis which leads to immunogenic cell death, through the release of damage associated molecular patterns that bind to dendritic cells and lead to downstream immune effector cell recruitment in the tumor microenvironment. PT-112 represents a highly potent inducer of this immunological form of cancer cell death. Further, PT-112 harbors a property known as osteotropism, or the propensity of the drug to reach its highest concentrations in certain areas of the bone, making it a candidate for treatment of patients with cancers that originate in, or metastasize to, the bone. The first in-human study of PT-112 demonstrated an attractive safety profile and evidence of long-lasting responses among heavily pre-treated patients and data were published in eClinicalMedicine, part of The Lancet. The combination Phase 1b dose escalation study of PT-112 with PD-L1 checkpoint inhibitor avelumab in solid tumors was reported in a mini-oral presentation at the ESMO 2020 Virtual Congress, and the Phase 2a dose confirmation cohort in non-small cell lung cancer patients was reported at ESMO I-O 2022. The Phase 1 study in patients with relapsed or refractory multiple myeloma presented at ASH 2020 is the third completed Phase 1 study of PT-112. Monotherapy Phase 2 development is ongoing in mCRPC, and includes the Phase 2 proof of concept study in thymic epithelial tumors under the company's formal CRADA with the NCI. Interim data from the NCI study were published at ASCO 2023. About Promontory Therapeutics Promontory Therapeutics Inc. is a privately held, clinical stage pharmaceutical company focused on small molecule immunotherapy, based in New York City. Clinical data generated across three Phase 1 studies have demonstrated single-agent and combination anti-cancer activity and an attractive tolerability profile, and two Phase 2 studies of PT-112 are underway. The company's research and development work has been conducted in the United States, Europe and Asia. The company sponsored the completed clinical study of PT-112 in combination with the PD-L1 inhibitor avelumab, and has an active Phase 2 trial under formal CRADA collaboration with the National Cancer Institute (NCI), utilizing PT-112 in thymic epithelial tumors, rare cancers with no FDA approved drug, where PT-112 has received Orphan Drug designation. Promontory Therapeutics is also a company member of the Paris-Saclay Cancer Cluster, Europe's emerging bio-cluster for oncology, and was a 2023 Nominee for Best Startup by the Prix Galien USA. To learn more about Promontory Therapeutics, visit the company's website here. CONTACTS: Promontory Therapeutics Brooke Raphael, MS VP, Strategy & Operations Tel: +1 (646) 974-6453 Email: [email protected] Investors: Stan Musial Tel: +1 (646) 222-6932 Email: [email protected] ICR Westwicke Media: Alexis Feinberg Tel: +1 (203) 939-2225 Email: [email protected] SOURCE Promontory Therapeutics Inc.