BAY-1158061

近日，和其瑞医药宣布，公司针对HMI-115在中度至重度子宫内膜异位症相关疼痛女性患者中，开展的一项2 期研究中期分析结果积极。HMI-115是一款靶向泌乳素受体（PRLR）的单克隆抗体，具有全球首创的治疗机制；公司计划快速推进全球 3 期试验，以便患者能够尽快开始从 HMI-115 治疗中受益。 该2期研究是一项国际多中心、随机、双盲、安慰剂对照试验，用于评估HMI-115 在12 周治疗期内对中度至重度子宫内膜异位症相关疼痛女性的安全性和有效性。该研究在142 名包括美国、波兰和中国的女性患者中开展，中期研究显示： 与基线相比，240 mg q2w 组的平均痛经疼痛评分降低了 42%； 平均非月经期盆腔疼痛评分降低了 50%。 大多数患者报告月经正常。 没有报告典型的围绝经期症状。 骨矿物质密度和性激素水平（包括雌二醇、LH、FSH 和孕酮）没有显著变化。 HMI-115以非竞争性方式阻断 PRLR介导的信号传导，获得中国国家药品监督管理局 (NMPA) 的突破性疗法认定，用于治疗子宫内膜异位症相关的中重度疼痛。该最早由拜耳开发，和其瑞医药与其签署了一项全球独家许可协议，在全球范围内针对HMI-115（BAY 1158061）进行多个适应症的开发和产业化。 除子宫内膜异位症适应症外，公司已完成一项Ib 期研究，旨在评估HMI-115 治疗雄激素性脱发患者的安全性、耐受性和疗效，研究显示：2 名男性患者的平均非毫毛靶区毛发数 (TAHC) 与基线相比增加了 14 根/cm2，药物安全且耐受性良好。 PRLR是1型细胞因子/造血功能受体超家族的一员，在免疫系统中广泛表达，包括单核细胞、淋巴细胞、巨噬细胞、NK细胞、粒细胞和胸腺上皮细胞。研究表明，PRL与自身免疫性疾病、乳腺癌、前列腺癌、卵巢癌、胰腺癌和子宫内膜癌等疾病相关。 PRLR结合配体时，发生构象变化，并激活下游多条信号通路，包括Jak2/Stat5和Jak1/Stat3通路，以及通过Src家族激酶和粘着斑激酶（FAK）的激活，有助于在癌细胞系中诱导PI3K/Akt和Raf/MEK/ERK的激活。 据不完全统计，目前在研的PRLR药物不足十种。 内异症是生育年龄女性的多发病、常见病，具有性激素依赖的特点。内异症的临床表现多样，其中最为重要和影响巨大的是疼痛（常与经期相关）和不孕。内异症严重影响患者的生理、心理、性爱、交际、生育，并带来沉重的家庭、社会经济及国家发展负担。 据统计，全球约有 1.9 亿女性患有子宫内膜异位症，市场规模约为 2000 亿美元。 现有的内异症的治疗手段主要包括手术和药物，均不能从根本上为患者提供有效安全且可长期使用的治疗手段。手术治疗可切除肉眼可见的病变，但因疾病本身发病机制不明朗，术后复发率居高不下（术后复发率每年增10%，术后5年累积复发率为 50%），且手术容易引发肠道、泌尿道和血管并发症。当前各种治疗药物（常用药物：非甾体抗炎药类、复方口服避孕药、孕激素类、GnRH激动剂）存在各自缺陷，患者可能因疗效不佳或者不能耐受副作用等情况而停止治疗，使得病情失去监控，再次反复。 和其瑞医药是一家科学驱动的临床阶段创新生物制药公司，基于北京大学未来技术学院院长肖瑞平教授及其团队在转化医学研究领域的深入理解及数十年的研究成果。公司针对威胁人类健康的常见病和重大疾病，致力于研究、开发和商业化首创新药。 参考资料 1、公司官网 内容来源于网络，如有侵权，请联系删除。

Plus, news about Interius BioTherapeutics, Hope Medicine, PMV Pharmaceuticals, Epitopea, Alyssum Therapeutics and a new dementia trial accelerator in the UK: Bayer exercises option to develop heart drug with Dewpoint Therapeutics : The companies, which first teamed up in 2019, now plan to develop a therapy for dilated cardiomyopathy, a heart muscle disease. Bayer is paying $424 million in upfront and milestone payments. — Jaimy Lee Viking Therapeutics is already thinking about an obesity launch: The company plans to meet with the FDA before the end of the year to discuss Phase 3 plans for its subcutaneous GLP-1/GIP receptor agonist, VK2735, according to remarks made by CEO Brian Lian on Wednesday’s earnings call. Viking is already in conversation with manufacturers, and Lian said he’s confident the company will be able to supply “a blockbuster-sized product at the appropriate time.” Viking is also launching a Phase 2 trial this quarter for an oral version of VK2735. Lian described the experimental oral medicine as more of an “add-on” to the portfolio, while the subcutaneous version would be the “anchor piece.” — Nicole DeFeudis Interius BioTherapeutics doses first ever patient with in vivo CAR-T: The biotech gave an in vivo CAR-T therapy to a patient enrolled in a Phase 1 study of INT2104, which it’s developing for B cell cancers. The trial, which is being run in Australia, is the first test of its kind. Umoja Biopharma has said it plans to dose a patient in its US-based study of its own in vivo CAR-T therapy by the end of this year . — Lei Lei Wu Hope Medicine touts interim data for endometriosis treatment: The company’s monoclonal antibody, dubbed HMI-115 , demonstrated statistically significant improvement in endometriosis pain at 12 weeks. The Phase 2 trial enrolled 142 female patients in the US, Poland and China. The mean dysmenorrhea pain score was reduced by 42%, and non-menstrual pelvic pain score was reduced by 50%. Hope CEO Nathan Chen said the company plans to “move quickly” toward global Phase 3 trials. — Katherine Lewin PMV Pharmaceuticals discontinues early-stage cancer trial: Citing toxicities and limited clinical benefit, the company abandoned its Phase 1b study assessing rezatapopt and Merck’s Keytruda. It had been testing the combo in patients with advanced solid tumors harboring a TP53 Y220C mutation. PMV said enrollment for a Phase 2 trial evaluating rezatapopt as a monotherapy for patients with TP53 Y220C and KRAS wild-type advanced solid tumors is still on track. — Katherine Lewin Epitopea closes $31M pre-Series A: With today’s round, the cancer biotech has raised more than $45 million. The funds are set to go toward its preclinical discovery work in solid tumors and clinical development plans for its RNA-based immunotherapies. — Katherine Lewin Alyssum Therapeutics emerges from stealth with $26M Series A: The Cambridge, MA-based biotech’s lead asset is AT-1965, a clinical-stage small molecule CMTR2 inhibitor. This round was led by co-founder Patrick Soon-Shiong and Accel, Famy and Navam Capital. — Katherine Lewin UK government pours £20M into dementia trial accelerator: The new initiative aims to increase the number of participants in late-stage dementia studies to the tens of thousands and “address pharma-identified roadblocks” to running the trials. The Dementia Trials Accelerator’s objectives include creating a biomarker toolbox and extending trial enrollment to community settings. — Ayisha Sharma