Harbour BioMed (Shanghai) Co., Ltd.

1. Keytruda获FDA优先审评资格，针对头颈部鳞癌 2月25日，默沙东（MSD）宣布，美国FDA已接受该公司为重磅免疫疗法Keytruda递交的补充生物制品许可申请（sBLA），寻求批准Keytruda作为手术前的新辅助疗法，用于治疗可切除局部晚期头颈部鳞状细胞癌（LA-HNSCC）患者，随后联合标准放疗（有或无顺铂）作为辅助治疗，然后再作为单药进行辅助治疗。FDA同时授予这一申请优先审评资格，预计在今年6月23日之前完成审评。该sBLA基于3期临床试验KEYNOTE-689的积极数据，预先设定的首次中期分析结果将在即将召开的医学会议上公布。结果显示，在可切除LA-HNSCC患者中，包含Keytruda的围手术期治疗方案与单独采用辅助放疗（有或无顺铂）相比，在无事件生存期（EFS）方面具有统计学显著且临床意义重大的改善。 2. 阿斯利康下一代口服SERD一线乳腺癌III期研究成功 2月26日，阿斯利康宣布III期SERENA-6研究积极的中期分析结果，口服选择性雌激素受体降解剂（SERD）camizestrant联合CDK4/6抑制剂（palbociclib、ribociclib或abemaciclib）在无进展生存期（PFS）的主要终点方面显示出具有统计学意义和临床意义的改善。这意味着，camizestrant成为了首个也是唯一一个证明与已获批的CDK4/6抑制剂联用时具有一线治疗获益的新一代口服SERD和完全ER拮抗剂。SERENA-6研究评估了在一线治疗HR阳性、HER2阴性且具有ESR1突变晚期乳腺癌患者中，改用camizestrant组合疗法与继续使用芳香化酶抑制剂（阿那曲唑或来曲唑）联合CDK4/6抑制剂这一标准护理治疗的疗效与安全性比较。 3. 一周一次创新抗菌药在中国申报上市 2月26日，中国国家药监局药品审评中心（CDE）官网最新公示，由萌蒂制药（Mundipharma）申报的5.1类新药注射用醋酸瑞扎芬净的新药上市申请已获得受理。公开资料显示，瑞扎芬净（rezafungin）是一种环肽药物，是一种创新、一周一次的棘球白素（echinocandin），其已经于2023年3月获得美国FDA批准，用于治疗没有替代治疗选择或选择有限的念珠菌血症与侵袭性念珠菌病患者。瑞扎芬净最初由Cidara Therapeutics开发，萌蒂制药此前通过合作拥有rezafungin在美国和日本以外的商业权利。2024年4月，Cidara Therapeutics宣布将rezafungin的所有权利剥离给萌蒂制药。 4. Alligator更新CD40抗体胰腺癌2期临床数据，股价大涨90% 2月26日，Alligator Bioscience报告了CD40激动型抗体药物MITAZALIMAB（Mitazalimab，米佐利单抗）在胰腺癌的二期临床OPTIMIZE-1研究的最新结果。OPTIMIZE-1是一项开放标签、单臂、多中心的1b/2期临床试验，旨在评估Mitazalimab联合mFOLFIRINOX（改良FOLFIRINOX方案）作为一线治疗方案在转移性胰腺导管腺癌（mPDAC）患者中的疗效和安全性。数据显示，接受MITAZALIMAB单抗联用mFOLFIRINOX治疗的患者的24个月生存率为29.4%，中位总生存期 (mOS) 为14.9个月，显著优于FOLFIRINOX和最近的NALIRIFOX。基于这些结果和已确认的监管途径，Alligator Bioscience 仍在推进MITAZALIMAB单抗单抗进入确认性临床试验。受此消息推动，Alligator股价一度大涨100%以上。 5. 恒瑞ADC新药NSCLC研究结果荣登《柳叶刀·肿瘤学》 2月25日，由上海交通大学附属胸科医院陆舜教授以及李子明教授团队牵头开展的HORIZON-Lung研究II期阶段主要结果发表于国际肿瘤学领域顶级期刊《柳叶刀·肿瘤学》《The Lancet Oncology》（IF:41.6）。该研究结果显示，瑞康曲妥珠单抗（研发代号：SHR-A1811）单药用于既往经治HER2突变晚期非小细胞肺癌患者，表现出了令人鼓舞的抗肿瘤疗效及良好的安全性。HORIZON-Lung研究是一项开放性、多中心、剂量递增/拓展的I/II期临床试验。I期阶段旨在评估瑞康曲妥珠单抗在治疗HER2过表达、扩增或者突变的非小细胞肺癌（NSCLC）受试者中的安全性、耐受性、PK特征及有效性，II期阶段作为关键单臂研究，旨在评估瑞康曲妥珠单抗单药对HER2突变的晚期NSCLC受试者的抗肿瘤疗效和安全性，主要研究结果已于2024年9月递交上市申请且已被纳入优先审评。 6. Arrowhead公布ALK7 siRNA临床前结果，可增强替尔泊肽疗效 2月25日，Arrowhead Pharmaceuticals 公布了ARO-ALK7的临床前结果，这是一种靶向激活素受体样激酶7（ALK7）的在研RNA干扰（RNAi）疗法，有望成为治疗肥胖症的潜在疗法。ARO-ALK7是首个直接靶向脂肪组织中表达基因的RNAi疗法，目前已在新西兰启动ARO-ALK7的1/2a期临床试验，Arrowhead预计将于2025年第二季度开始给药。在啮齿动物和非人灵长类动物的临床前研究中，ARO-ALK7显示出脂肪组织中ALK7 mRNA表达的剂量依赖性和持久性降低。在饮食诱导的肥胖小鼠模型中的药理学研究表明，脂肪组织中ALK7的沉默导致身体成分改善，脂肪量减少约50%，同时保留了瘦体重。此外，GLP-1/GIP受体共激动剂替尔泊肽与ALK7 siRNA的联合治疗增强了治疗效果，与替尔泊肽单药治疗相比，在减轻体重和脂肪量方面具有叠加效应，同时缓解了替尔泊肽单药治疗相关的瘦体重显著减少。 1. 3.95亿美元！和铂医药就一款抗体新药达成授权合作 2月26日，和铂医药宣布，由其孵化的创新型生物技术公司HBM Alpha Therapeutics宣布与一家业务合作伙伴达成战略合作与许可协议，共同推进针对多种疾病的新型促肾上腺皮质激素释放激素（CRH）靶向疗法。根据协议，该合作伙伴获得在大中华区（包括中国大陆、中国台湾、中国香港和中国澳门地区）以外的全球市场开发和商业化HAT001（一款强效且选择性靶向CRH的中和抗体，和铂医药内部代号为HBM9013）的独家权利。HBMAT将获得高达3.95亿美元的总付款，包括首付款和开发、监管及商业里程碑付款，以及基于未来产品净销售额的分级特许权使用费。此外，HBMAT还有权获得认股权证以获取合作伙伴的少数股权。 内容来源于网络，如有侵权，请联系删除。

Plus, news about Idorsia, Enveda, Zymeworks, BerGenBio and Ignota Labs: Harbour BioMed does another deal: The Shanghai-based biotech’s subsidiary, called HBM Alpha Therapeutics, inked a pact with an undisclosed company for up to $395 million in upfront fees and biobucks, plus the potential for a minority stake. The undisclosed partner also gets exclusive ex-China global rights to an antibody known as HAT001/HBM9013 for congenital adrenal hyperplasia (CAH) and other rare endocrine disorders. HBMAT raised a seed round of undisclosed size in January 2023, and Harbour did a NewCo deal for an anti-TSLP antibody with Windward Bio last month. — Kyle LaHucik ORIC ‘refines’ development plans for two drugs: The ORIC-944 program is set to start a Phase 3 trial in the first half of 2026 looking at patients with metastatic castration-resistant prostate cancer. Its ORIC-114 candidate is expected to begin registrational trials sometime in 2026 in the first-line lung cancer setting. Its stock $ORIC was up about 13% on Wednesday morning. — Max Gelman Idorsia’s big updates: The Swiss drugmaker said an undisclosed party opted not to move forward with a deal for the global rights to Idorsia’s experimental hypertension drug, called aprocitentan, but it will keep the $35 million exclusivity fee that it got in December. Meanwhile, Idorsia and Viatris adjusted the terms of their collaboration for selatogrel and cenerimod, with Idorsia contributing $100 million less toward development while losing $250 million in potential milestone payments from Viatris. It is also restructuring its debt and getting new funding . — Kyle LaHucik Sanofi backs Enveda with $20M: The French pharma giant is joining the Series C of the Colorado biotech, which mines plant chemistry to find new drugs. The Series C now totals $150 million, up from the $130 million disclosed in November . — Kyle LaHucik Zymeworks’ $14M milestone: The company received the payment from GSK from a bispecific antibody deal they inked in 2016 and then expanded in 2019. — Jaimy Lee BerGenBio discontinues lung cancer study, to seek strategic alternatives: The company was evaluating whether bemcentinib, an AXL kinase inhibitor, could treat patients with first-line non-squamous non-small cell lung cancer in combination with standard of care. Patients also needed to have a mutation of the STK11 gene. In a statement, CEO Olav Hellebø said the results were not strong enough for BerGenBio to obtain additional financing and is now looking for a merger, sale or other transaction. — Max Gelman Ignota Labs closes £5.5M seed round: The startup’s goal is to “turn around failed drugs” using AI, acquiring assets from bigger companies that stopped being developed and then testing them in new clinical trials. Its first asset is a PDE9A inhibitor, which the company says has potential in cardiovascular, metabolic and neurodegenerative diseases. — Max Gelman

While not disclosing the mysterious business partner for HAT001, Harbour\'s CEO said it had “expertise in drug development” that made it “well-positioned to help bring this therapy to patients worldwide.”\n With Neurocrine Biosciences having finally shaken up treatment for a rare genetic hormone disease, Harbour BioMed’s antibody offshoot has sold the rights for its own preclinical therapy option for $395 million in biobucks.The deal will see HBM Alpha Therapeutics (HBMAT), which was formed around Harbour’s antibody expertise, transfer the global rights outside of China for a corticotropin-releasing hormone (CRH) therapy to an unnamed “business partner.”The therapy—called HAT001 at HBMAT and HBM9013 at Harbour—is an anti-CRH-neutralizing antibody that is being considered for a range of disorders including congenital adrenal hyperplasia (CAH). CAH itself encompasses various autosomal recessive diseases caused by mutations in genes that encode for enzymes used for synthesizing adrenal hormones.There had been decades of silence in the CAH space until December, when Neurocrine scored FDA approval for its oral med Crenessity. That drug is designed to reduces excess adrenocorticotropic hormone and downstream adrenal androgen production, allowing CAH patients to reduce their dose of glucocorticoid treatment.It marked a key breakthrough for CAH patients as long-term use of glucocorticoid replacement therapies at high doses can cause weight gain, heart problems and bone loss.HBMAT is also hoping that HAT001 will allow CAH patients to reduce their use of glucocorticoid replacement therapies. The aim is to “effectively cause reversible pharmacologic adrenalectomy, converting CAH patient treatment into that for primary adrenal insufficiency, which can be easily treated with much lower, physiological doses of glucocorticoid,” according to Harbour’s postmarket release Feb. 25. The therapy has already shown “strong preclinical efficacy in downregulating CRH-mediated induction of adrenocorticotropic hormone,” leading HBMAT to gear up the asset for human studies, Harbour said.In return for the exclusive rights to HAT001 outside of greater China, the unnamed buyer is eligible to hand HBMAT up to $395 million in a combination of upfront, development, regulatory and commercial milestone payments, as well as tiered royalties should the therapy make it to market.As an extra sweetener, HBMAT can receive a warrant for minority interest in the partner, according to the release.HBMAT was founded in 2019 as a joint venture between Harbour and the Boston Children\'s Hospital. The idea was to combine the antibody expertise at China-based Harbour and certain endocrine-disorder-focused tech from the hospital’s research arm.Today’s deal marks Harbour’s “partial exit” from HBMAT, according to Harbour’s CEO Jingsong Wang, M.D., Ph.D., who described HBMAT as “the first global NewCo we incubated.”While not disclosing the mysterious business partner for HAT001, Wang said it had “expertise in drug development” that made it “well-positioned to help bring this therapy to patients worldwide.”