Esocap AG

本期看点 ① 百时美施贵宝免疫疗法治疗结直肠癌3期临床达终点 ② 安必生制药提交美沙拉秦栓仿制药上市申请 ③ Phathom提交VOQUEZNA新适应证NDA ④ EsoCap公布ESO-101治疗EoE 2期临床积极顶线结果 ⑤ 三诺生物血糖监测产品获得FDA 510（k）认证 ⑥ M20 Genomics发布可用于肠道微生物组的高通量单细菌转录组产品 ① 百时美施贵宝免疫疗法治疗结直肠癌3期临床达终点 作者：药明康德 解读：Richard 来源：BMS官网 发布日期：2023-12-08 ■ 内容要点 12月8日，百时美施贵宝对外公布了3期临床试验CheckMate-8HW研究的积极结果。该试验旨在评估其PD-1抑制剂Opdivo与CTLA-4靶向抗体Yervoy联合疗法与研究者选择的化疗，作为转移性结直肠癌（mCRC）患者一线疗法的疗效与安全性。这些患者的肿瘤属于微卫星不稳定性高（MSI-H）或错配修复缺陷（dMMR）。 该试验的双重主要终点是根据盲法独立中央审评（BICR）检视Opdivo加Yervoy联合疗法与研究者选择的一线化疗相比的无进展生存期（PFS），以及根据BICR评估所有治疗线中联合疗法与单独Opdivo相比的PFS。 根据BICR所评估的预定中期分析显示，该试验达成主要终点之一，即与化疗相比，Opdivo联合Yervoy的双重免疫治疗组合使得患者的PFS具有统计学和临床意义的改善。 目前该试验仍在持续进行中，以评估联合疗法与Opdivo单药相比的PFS（另一项主要终点）以及次要终点。 原文链接： https://news.bms.com/news/details/2023/Bristol-Myers-Squibb-Announces-Phase-3-CheckMate--8HW-Trial-Evaluating-Opdivo-nivolumab-Plus-Yervoy-ipilimumab-Compared-to-Chemotherapy-in-Microsatellite-InstabilityHigh-or-Mismatch-Repair-Deficient-Metastatic-Colorectal-Cancer-Meets-Primary/default.aspx ② 安必生制药提交美沙拉秦栓仿制药上市申请 作者：伍玥 解读：Richard 来源： 米内网 发布日期：2023-12-05 ■ 内容要点 12月5日， 江苏安必生制药提交了美沙拉秦栓的4类仿制药上市申请，并有望争取国产第二家获批。 安必生制药于今年2月提交了该品种首款产品美沙拉秦肠溶缓释胶囊的仿制上市申请，后续再报产了美沙拉秦肠溶缓释颗粒、美沙拉秦肠溶片。本次报产的美沙拉秦栓是安必生制药在该品种申报的第四款产品。 美沙拉秦是轻中度溃疡性结肠炎诱导治疗和维持缓解的一线药物，其通过作用于肠道炎症黏膜，抑制引起炎症的前列腺素的合成和炎性介质白三烯的形成，从而对肠道壁炎症起到显著的消炎作用，对发炎的肠壁结缔组织效果尤佳。 原文链接： https://mp.weixin.qq.com/s/ufniXaRgbZib4GslsZAP_A ③ Phathom提交VOQUEZNA新适应证NDA 作者：Phathom Pharmaceuticals 解读：Richard 来源：Phathom Pharmaceuticals官网 发布日期：2023-12-06 ■ 内容要点 12月6日，Phathom Pharmaceuticals公司对外表示， VOQUEZNA治疗非糜烂性胃食管反流病（GERD）引起的烧心适应证新药上市申请（NDA）获得FDA受理。 根据《处方药使用者付费法案》，FDA有10个月的审查时间，行动截止日期为2024年7月19日。 Vonoprazan是一款新型钾离子竞争性酸阻滞剂，可以竞争性阻滞钾离子与氢/钾离子ATP酶的结合，从而快速抑制胃酸的分泌。目前，该药物已经获得FDA批准治疗成人糜烂性GERD、糜烂性GERD治愈后的维持治疗，以及缓解与糜烂性GERD相关的胃灼热症状。 此次递交NDA是基于3期临床试验PHALCON-NERD-301研究的结果。Phathom Pharmaceuticals公司表示，已经着手完成了另外一项3期临床研究计划，来研究Vonoprazan作为按需治疗方案治疗非糜烂性GERD引起的烧心的作用。 原文链接： https://investors.phathompharma.com/news-releases/news-release-details/phathom-pharmaceuticals-announces-fda-acceptance-filing-0 ④ EsoCap公布ESO-101治疗EoE 2期临床积极顶线结果 作者：EsoCap 解读：Richard 来源：GlobeNewswire 发布日期：2023-12-05 ■ 内容要点 12月5日，瑞士生物技术公司EsoCap公布了在研管线ESO-101治疗嗜酸性粒细胞食管炎（EoE）2期临床ACESO研究积极结果。 组织解剖学显示，与安慰剂组相比，ESO-101治疗组患者食管处嗜酸性粒细胞峰值数显著减少，研究达到了主要评价终点。 管线药物ESO-101采用了EsoCap公司专有药物递送技术，外层为黏膜亲和力高的薄膜，内含皮质类固醇和糠酸莫米松抗炎成分，有效增加了药物在食管黏膜处的接触时间。 ACESO研究是一项随机、双盲、安慰剂对照试验，共在欧洲5个国家招募了43名峰值嗜酸性粒细胞数≥15的成年EoE患者。患者按2:1比例接受为期28天的治疗。研究还显示，管线药物ESO-101在患者体内展现出了良好的安全性和耐受性。 原文链接： https://www.globenewswire.com/news-release/2023/12/05/2790628/0/en/EsoCap-reports-positive-topline-results-from-ACESO-Phase-II-trial-investigating-ESO-101-in-eosinophilic-esophagitis.html ⑤ 三诺生物血糖监测产品获得FDA 510（k）认证 作者：三诺生物 解读：Richard 来源：东方财富网 发布日期：2023-12-06 ■ 内容要点 12月6日，三诺生物发布公告称，旗下TRUENESS/TRUENESS AIR血糖监测系统产品获得了FDA的510（k）认证。这一认证标志着该产品具备了进入美国市场的准入资格，有利于进一步增强三诺生物血糖监测产品的综合竞争力和海外市场拓展能力。 TRUENESS血糖监测系统由TRUENESS血糖仪和TRUENESS血糖试条组成；TRUENESS AIR血糖监测系统由TRUENESS AIR血糖仪和TRUENESS血糖试条组成。 产品预期用途为定量测量手指毛细血管全血中的葡萄糖。 糖尿病患者在家中使用该血糖监测系统产品，可监测其糖尿病控制计划的有效性。TRUENESS/TRUENESS AIR血糖监测系统供单人使用，不应与他人共享，仅用于体外诊断，不用于糖尿病的诊断或筛查，不适用于新生儿。 原文链接： https://data.eastmoney.com/notices/detail/300298/AN202312051613388750.html ⑥ M20 Genomics发布可用于肠道微生物组的高通量单细菌转录组产品 解读：617 来源：M20 Genomics 发布日期：2023-11-27 ■ 内容要点 近期，单细胞测序公司M20 Genomics宣布， 正式推出全球首个可用于肠道微生物组的高通量单细菌转录组产品——VITA GutMicrobiome，助力研究人员开启肠道菌群研究的单细菌转录组时代。 该产品由VITApilote GutMicrobiome试剂盒和芯片、VITAcruizer单细胞制备仪及VITAseer分析软件组成，配套使用，为肠道微生物组的单细菌转录组测序提供端到端的解决方案。同时，该产品适配M20 Genomics推出的VITAcruizer仪器，无需购置多重仪器，能够在同一台仪器上解锁从真核FFPE样本到微生物组样本的高通量单细胞转录组实验，以实现“全栈式”全样本高通量单细胞转录组。 VITA GutMicrobiome高通量单细菌转录组产品可直接用于肠道微生物组样本，不需纯培养，可用于分析肠道微生物组的功能及表型异质性、细菌-噬菌体精准关联、细菌间及细菌与宿主间的互作模式等。据报道，目前该产品已应用于500多例微生物组样本。 原文链接： https://mp.weixin.qq.com/s/EocT_HotSmaYlvE4dTHGvA

ESO-101, EsoCap's lead product candidate, consists of a capsule with a rolled-up, thin mucoadhesive film with the anti-inflammatory corticosteroid, mometasone furoate EsoCap's novel targeted delivery platform increases mucosal contact time and drug deposition in the esophagus The ACESO trial met the primary endpoint, demonstrating efficacy by histologic response and a very favorable safety and tolerability pro > BASEL, Switzerland, Dec. 05, 2023 (GLOBE NEWSWIRE) -- EsoCap AG, the Swiss biotech company dedicated to improving the lives of patients with serious diseases of the upper gastrointestinal tract, announced today that the ACESO Phase II study comparing ESO-101 to placebo for the treatment of eosinophilic esophagitis has met its primary endpoint by significantly reducing the peak eosinophil count in the histological assessment of esophageal biopsies. The ACESO study is a randomized, placebo-controlled, double-blind Phase II study to evaluate the efficacy, tolerability and safety of ESO-101 in patients with active eosinophilic esophagitis (EoE). Forty-three adult patients with EoE and a peak eosinophil count of ≥15 eosinophils per high-power field (hpf) were enrolled in five European countries. Patients were randomized in a 2:1 ratio and treated for 28 days. EoE is an increasingly recognized, chronic, local, immune-mediated esophageal disease characterized clinically by symptoms associated with esophageal dysfunction, including dysphagia, food impaction, heartburn and vomiting, and histologically by eosinophil-dominated inflammation. The results of the study showed that the primary endpoint was met with a statistically significant reduction of the peak eosinophil count (p=0.0318) compared to placebo. The study also showed that ESO-101 demonstrated a highly favorable safety and tolerability profile. 7.1 % (n=2) of patients who received ESO-101 treatment and 26.7 % (n=4) in the placebo group reported mild to moderate adverse events related to the investigational product; no drug related serious adverse events were reported. There were no instances of candidiasis, a type of fungal infection commonly associated with corticosteroid-based topical products. The unique EsoCap drug delivery system was shown to be easy for patients to swallow. Upon drinking the capsule through a specially designed drinking cup, the film unrolls and adheres to the patient's esophageal mucosa, where it slowly dissolves and releases mometasone furoate. "Eosinophilic esophagitis is a chronic disease with few therapeutic options. This limitation in treatment is due to the unique anatomical and functional characteristics of the esophagus, which result in an extremely short transit time within the esophageal tract. A higher efficacy of topical corticosteroids has been shown to be directly related to the duration of mucosal contact time, but the methods for topical delivery of drugs to the esophagus have remained a challenge," said Dr. Alfredo J. Lucendo, Department of Gastroenterology, Hospital General de Tomelloso, Spain, and coordinating principal investigator of the ACESO trial. "We are pleased that the results of the ACESO study confirm that ESO-101, which is specifically designed to increase contact time with the mucosa, can achieve a statistically significant histologic response compared to placebo, providing the important benefits of an esophageal-specific drug for patients with EoE." "The ACESO study represents an important milestone in the development of our unique application platform for the treatment of esophageal diseases. With our lead product ESO-101, we have demonstrated that the EsoCap system can provide long-lasting, targeted topical delivery of the glucocorticosteroid mometasone furoate. Following the successful results of this Phase II study, we now plan to initiate a Phase III program in eosinophilic esophagitis,” said Isabelle Racamier, CEO of EsoCap. “Our technology is extremely flexible. Multiple relevant agents, including biologics and other innovative substances, can be incorporated into the film, making our platform suitable for drug delivery in a variety of clinical indications, including reflux disease, Barrett's disease and esophageal cancer - all areas of high unmet medical need." The detailed results from the ACESO study, including secondary endpoint data, will be submitted for presentation at a future scientific conference. About eosinophilic esophagitis Eosinophilic esophagitis (EoE) is an increasingly recognized, chronic, local immune-mediated esophageal disease, characterized clinically by symptoms related to esophageal dysfunction and histologically by eosinophil-predominant inflammation. The symptoms of EoE include swallowing disorders, food impaction, vomiting, and heartburn. EoE is the leading cause of dysphagia and food impaction in children and young adults. The only treatment options for the condition are extremely strict diets, off-label treatment with steroids or proton pump inhibitors, or an orodispersible budesonide tablet available only in limited territories. These treatment options remain suboptimal for the vast majority of affected patients. Recently, a monoclonal antibody targeting Th2 cytokines was approved to treat EoE in a limited treatment-resistant patient population. Currently, about 500,000 patients worldwide suffer from this disease. The prevalence of EoE is over 6 in 10,000. The current incidence is estimated at about 5 cases per 100,000. The incidence of EoE increases with age and peaks at 30-50 years of age. About the ACESO Phase II trial The ACESO trial is a multicenter, randomized, double-blinded, placebo-controlled clinical Phase II trial evaluating the efficacy, tolerability, and safety of ESO-101 in adult patients with active EoE. Patients are treated once daily for 28 days. The trial’s primary objective is to evaluate efficacy based on histological response. Secondary objectives include efficacy based on 1) histological response and clinical symptoms, 2) clinical response assessed by patient-reported outcomes, and 3) endoscopic response; patient-reported treatment satisfaction; as well as evaluation of tolerability and safety. About ESO-101 The EsoCap system is a unique drug delivery system for the upper gastrointestinal tract, consisting of a capsule holder containing a hard gelatin capsule, with a rolled, thin mucoadhesive film, a sinker, and a soluble retainer. The capsule holder is screwed onto the lid of a drinking cup to facilitate swallowing while drinking from the cup. Upon swallowing, the film unrolls and sticks to the esophageal mucosa, where it dissolves, with a contact time of 15 minutes1,2, significantly longer than the mucosal contact time of pharmaceutical dosage forms, such as orodispersible tablets (less than one minute)3. The use of mometasone furoate as a swallowed aerosol formulation has been studied previously in several trials assessing its effect on EoE in adults. In these trials, mometasone furoate was shown to significantly decrease esophageal eosinophilic inflammation and improve clinical symptoms. ESO-101 was designed as a locoregional, esophagus-adjusted drug formulation and novel delivery system to optimize mucosal contact time and maximize esophageal deposition of mometasone furoate. ESO-101 has the potential to provide significant clinical benefits to EoE patients. About EsoCap EsoCap AG is a privately funded company based in Basel, Switzerland. EsoCap’s vision is to improve the lives of patients with serious diseases of the upper gastrointestinal tract through development of a unique and innovative topical drug delivery platform. Effective topical treatment of the esophagus is extremely difficult to achieve with the current standard of care, due to the ultra-short drug contact time of 45 seconds from the mouth to the stomach. Lead candidate ESO-101 has received Orphan Drug Designation from the U.S. Food & Drug Administration (FDA) for the treatment of EoE and is in clinical development for this indication. EsoCap has developed a unique, proprietary drug delivery platform enabling the efficient topical application of drug substances for the local treatment of diseases of the upper gastrointestinal tract. EsoCap has a strong and broad intellectual property position. For more information, please visit and follow EsoCap on LinkedIn and Twitter. Contacts: Isabelle Racamier, CEO EsoCap AG Malzgasse 9 4052 Basel Switzerland isabelle.racamier@esocapbiotech.com Media Inquiries: MC Services AG Katja Arnold, Dr. Dennis Burger Phone: +49 89 210288-0 esocap@mc-services.eu References 1 C Rosenbaum et al, 2021. Functionality and Acceptance of the EsoCap System—A Novel Film-Based Drug Delivery Technology: Results of an In Vivo Study. Pharmaceutics, 13 (6): 828. 2 Krause et al., 2020. The EsoCap-system – An innovative platform to drug targeting in the esophagus. Journal of controlled release, 327:1–7. 3 Burton et al., 1995. Intragastric Distribution of Ion-exchange Resins: a Drug Delivery System for the Topical Treatment of the Gastric Mucosa. J. of Pharmacy and Pharmacology, 47: 901-906.

EsoCap's novel targeted delivery platform is designed to increase mucosal contact time and drug deposition in the esophagus ESO-101, EsoCap's lead product candidate, consists of a capsule with a rolled-up, thin mucoadhesive film filled with the anti-inflammatory corticosteroid, mometasone furoate Topline results from ACESO study expected in late 2023 BASEL, Switzerland, Sept. 05, 2023 (GLOBE NEWSWIRE) -- EsoCap AG, the Swiss biotech company dedicated to improving the lives of patients with serious diseases of the upper gastrointestinal tract, reports the completion of patient recruitment in the Phase II ACESO trial. The randomized, placebo-controlled, double-blind study is evaluating the efficacy, tolerability and safety of ESO-101 in 43 adult patients with active eosinophilic esophagitis (EoE) in five European countries. Patients have been randomized 2:1 and are being treated for 28 days. EoE is a rare, chronic, local immune-mediated esophageal disease, characterized clinically by symptoms related to esophageal dysfunction including swallowing disorders, food impaction, heartburn and vomiting, and histologically by eosinophil-predominant inflammation. The first product candidate, ESO-101, is loaded with mometasone furoate, an anti-inflammatory corticosteroid and well-known active pharmaceutical ingredient (API) that is approved for the topical treatment of asthma and skin conditions, making it well suited as a loco-regional treatment for EoE. Upon drinking the capsule through a specially designed drinking cup, the film unrolls and adheres to the patient's esophageal mucosa, where it slowly dissolves, releasing mometasone furoate. The objectives of the ACESO Phase II trial are to evaluate the efficacy of ESO-101 based on histological response and clinical symptoms. "Reaching completion of patient enrollment reflects the high level of interest in our clinical development program. We look forward to upcoming data readouts, which will guide our future strategic development plans,” said Isabelle Racamier, Chief Executive Officer. “Our technology is highly flexible. Multiple relevant agents, including biologics and other innovative compounds, can be incorporated into the film, making our drug delivery platform suitable for several clinical indications, including reflux disease, Barrett’s disease and esophageal cancer – all areas of high unmet medical need.” “Eosinophilic esophagitis is a chronic disease with few therapeutic options. This limitation in treatment arises from the distinctive anatomical and functional attributes of the esophagus, which result in an exceedingly brief transit period within the esophageal tract. As a result, achieving effective topical treatment for the esophagus presents a significant challenge,” explained Dr. Alfredo J. Lucendo, Department of Gastroenterology, Hospital General de Tomelloso, Spain, and coordinating principal investigator of the ACESO trial. “We are confident that ESO-101 holds the promise via increased mucosal contact time of enhancing the efficacy of drugs within the esophagus, thereby providing significant advantages to individuals suffering from EoE.” In a previous study in healthy volunteers, EsoCap's proprietary and unique targeted delivery technology for the upper gastrointestinal tract was shown to significantly increase contact time with the mucosa and was well tolerated. About eosinophilic esophagitis Eosinophilic esophagitis (EoE), is a rare, chronic, local immune-mediated esophageal disease, characterized clinically by symptoms related to esophageal dysfunction, and histologically by eosinophil-predominant inflammation. The symptoms of EoE include swallowing disorders, food impaction, vomiting, and heartburn. EoE is the leading cause of dysphagia and food impaction in children and young adults. The only treatment options for the condition are extremely strict diets, off-label treatment with steroids, or off-label proton pump inhibitors, or an orodispersible budesonide tablet available only in limited territories. These treatment options remain suboptimal for the vast majority of affected patients. Recently a monoclonal antibody targeting Th2 cytokines has been approved to treat EoE in a limited treatment resistant patient population. Currently, about half a million patients worldwide suffer from this disease. The prevalence of EoE is about 5 in 10,000. The current incidence is estimated at about 5 cases per 100,000. The incidence of EoE increases with age and peaks at 30-50 years. About ACESO The ACESO trial is a multicenter, randomized, double-blinded, placebo-controlled clinical Phase II trial evaluating the efficacy, tolerability, and safety of ESO-101 in adult patients with active EoE. Patients are treated once daily for 28 days. The trial’s primary objective is to evaluate efficacy based on histological response. Secondary objectives include efficacy based on histological response and clinical symptoms, efficacy based on clinical response assessed by patient-reported outcomes, efficacy based on endoscopic response, patient-reported treatment satisfaction, as well as evaluation of tolerability and safety. About ESO-101 The EsoCap system is a unique drug delivery system for the upper gastrointestinal tract, consisting of a capsule holder containing a hard gelatin capsule, with a rolled, thin mucoadhesive film, a sinker, and a soluble retainer. The capsule holder is screwed onto the lid of a drinking cup to facilitate swallowing while drinking from the cup. Upon swallowing, the film unrolls and sticks to the esophageal mucosa, where it dissolves, with a contact time of up to 15 minutes1,2 significantly longer than the mucosal contact time of pharmaceutical dosage forms, such as orodispersible tablets (less than one minute)3. The use of mometasone furoate as a swallowed aerosol formulation has been studied previously in several trials assessing its effect on EoE in adults. In these trials, mometasone furoate was shown to significantly decrease esophageal eosinophilic inflammation and improve clinical symptoms. ESO-101 was designed as a locoregional, esophagus-adjusted drug formulation and novel delivery system to optimize mucosal contact time and maximize esophageal deposition of mometasone furoate. ESO-101 has the potential to provide significant clinical benefits to EoE patients. About EsoCap EsoCap AG is a privately funded company based in Basel, Switzerland. EsoCap’s vision is to improve the lives of patients with serious diseases of the upper gastrointestinal tract through development of a unique and innovative topical drug delivery platform. Effective topical treatment of the esophagus is extremely difficult to achieve with the current standard of care, due to the ultra-short drug contact time of one to two seconds from the mouth to the stomach. Lead candidate ESO-101 has received Orphan Drug Designation from the U.S. Food & Drug Administration (FDA) for the treatment of EoE and is in clinical development for this indication. EsoCap has developed a unique, proprietary drug delivery platform allowing the efficient topical application of drug substances for the local treatment of diseases of the upper gastrointestinal tract. EsoCap has a strong and broad intellectual property position. For more information, please visit and follow EsoCap on LinkedIn and Twitter. Contacts: Isabelle Racamier, CEO EsoCap AG Malzgasse 9 4052 Basel Switzerland isabelle.racamier@esocapbiotech.com Media Inquiries: MC Services AG Katja Arnold, Dr. Dennis Burger Phone: +49 89 210288-0 esocap@mc-services.eu References 1 C Rosenbaum et al, 2021. Functionality and Acceptance of the EsoCap System—A Novel Film-Based Drug Delivery Technology: Results of an In Vivo Study. Pharmaceutics, 13 (6): 828. 2 Krause et al., 2020. The EsoCap-system – An innovative platform to drug targeting in the esophagus. Journal of controlled release, 327:1–7. 3 Burton et al., 1995. Intragastric Distribution of Ion-exchange Resins: a Drug Delivery System for the Topical Treatment of the Gastric Mucosa. J. of Pharmacy and Pharmacology, 47: 901-906.