Core Viewpoint - The FDA has granted Breakthrough Therapy Designation to izalontamab brengitecan (iza-bren) for the treatment of locally advanced or metastatic non-small cell lung cancer (NSCLC) patients who have progressed after EGFR-TKI and platinum-based chemotherapy, indicating significant clinical benefits over existing therapies [1][2]. Group 1: FDA Breakthrough Therapy Designation - The designation is based on clinical research data from multiple studies, including BL-B01D1-101, BL-B01D1-203, and BL-B01D1-LUNG-101 [2]. - This recognition validates the reliability of existing data and highlights the unmet clinical needs faced by patients after EGFR-TKI and platinum-based chemotherapy [2][4]. Group 2: Clinical Context and Market Need - NSCLC accounts for approximately 80% of all lung cancer cases and remains a leading cause of cancer-related deaths globally [3]. - In Western populations, 10% to 15% of NSCLC patients have EGFR mutations, while in Asian populations, this figure can be as high as 50% [3]. Group 3: Drug Development and Clinical Trials - Iza-bren is a globally innovative EGFR×HER3 dual antibody ADC that has entered Phase III clinical trials [4]. - The drug is currently involved in over 40 clinical trials across various tumor types in China and the U.S., with several Phase III trials ongoing for NSCLC, small cell lung cancer, breast cancer, and nasopharyngeal carcinoma [5].

格隆汇8月18日丨百利天恒(688506.SH)公布，公司全资子公司SystImmune, Inc.（以下简 称"SystImmune"）与合作伙伴百时美施贵宝（以下简称"BMS"）宣布，美国食品药品监督管理局（以下 简称"FDA"）已授予izalontamabbrengitecan（iza-bren）突破性疗法认定（Breakthrough Therapy Designation），用于既往 EGFR-TKI 及含铂化疗治疗失败后的 EGFR19 外显子缺失或21 外显子L858R 置换突变的局部晚期或转移性非小细胞肺癌。 本次授予是公司与百时美施贵宝（BMS）正在合作开发的iza-bren 基于BL-B01D1-101（中国）、BL- B01D1-203（中国）和 BL-B01D1-LUNG-101（美国/欧洲）的临床研究数据，首次获得美国 FDA 突破 性疗法认定。本次突破性疗法认定的授予充分验证了现有数据的可靠性，同时彰显了 EGFR TKI 和铂类 化疗治疗后患者面临的重大未满足临床需求。虽然 EGFR TKI 在一线治疗中显示出临床疗效，但大多数 患者在约 18 个月后仍会出现疾病进展，后续治疗方 ...

本文源自：金融界AI电报 百利天恒公告，全资子公司SystImmune与合作伙伴百时美施贵宝宣布，美国食品药品监督管理局已授 予izalontamab brengitecan（iza-bren）突破性疗法认定，用于既往EGFR-TKI及含铂化疗治疗失败后的 EGFR19外显子缺失或21外显子L858R置换突变的局部晚期或转移性非小细胞肺癌。本次突破性疗法认 定的授予基于公司在中国和美国进行的多项临床试验数据。 ...

Core Insights - Dyne Therapeutics has received Breakthrough Therapy Designation from the FDA for DYNE-251, aimed at treating Duchenne muscular dystrophy (DMD) through exon 51 skipping, with data from the DELIVER trial expected in late 2025 and a potential BLA submission anticipated in early 2026 [1][2][6] Company Overview - Dyne Therapeutics is focused on developing therapies for genetically driven neuromuscular diseases, including DMD and myotonic dystrophy type 1 (DM1) [9] - The company is advancing clinical programs targeting muscle and the central nervous system to address the root causes of these diseases [9] Product Details - DYNE-251 is an investigational therapeutic designed to produce near full-length dystrophin in muscle and the central nervous system, which is crucial for functional improvement in DMD patients [5][8] - The DELIVER trial is a global, randomized, placebo-controlled, double-blind Phase 1/2 clinical trial assessing the safety, tolerability, and efficacy of DYNE-251 in DMD patients with specific mutations [4][5] Clinical Trial Information - The DELIVER trial has completed enrollment of 32 patients in the Registrational Expansion Cohort, with the primary endpoint being the change in dystrophin protein levels at 6 months [6][4] - The trial's multiple ascending dose portion identified a registrational dose of 20 mg/kg of DYNE-251 administered every four weeks [4] Regulatory Designations - DYNE-251 has received multiple designations from the FDA, including Fast Track, Orphan Drug, and Rare Pediatric Disease designations, which facilitate expedited development and review processes [6][7] - The Breakthrough Therapy Designation allows for enhanced FDA support, including senior-level involvement and early communication regarding trial design and regulatory strategy [6][2] Disease Context - Duchenne muscular dystrophy (DMD) is a rare, progressive neuromuscular disorder primarily affecting males, with approximately 12,000 individuals in the U.S. and 16,000 in the EU affected [8] - There is a significant unmet need for new treatment options that can deliver functional improvements for DMD patients [8]

Core Viewpoint - Sichuan Kelun Pharmaceutical Co., Ltd. announced that its subsidiary, Sichuan Kelun Botai Biopharmaceutical Co., Ltd., received breakthrough therapy designation from the National Medical Products Administration (NMPA) for its TROP2 ADC product, sac-TMT, in combination with PD-L1 monoclonal antibody, Tagolizumab, for the first-line treatment of locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) without driver gene mutations [2][3]. Group 1: Product and Clinical Development - Sac-TMT is a novel TROP2 ADC developed by Kelun Botai, targeting advanced solid tumors such as NSCLC, breast cancer (BC), gastric cancer (GC), and gynecological tumors [3][5]. - The drug utilizes a new linker and has a drug-antibody ratio (DAR) of 7.4, employing a topoisomerase I inhibitor as an effective payload to induce DNA damage in tumor cells [3][5]. - Sac-TMT has previously received four other breakthrough therapy designations for various indications, including triple-negative breast cancer (TNBC) and EGFR mutation-positive NSCLC [3][4]. Group 2: Regulatory and Market Position - Sac-TMT is the first domestically developed ADC with global intellectual property rights and the first TROP2 ADC approved for lung cancer indications globally [5]. - The drug has already been approved for two indications in China, including treatment for advanced TNBC and EGFR mutation-positive NSCLC after prior treatment [4][5]. - Kelun Botai has initiated eight registration clinical studies in China, while Merck has started 14 ongoing global Phase III clinical studies for sac-TMT in various cancer types [5]. Group 3: Collaborations and Partnerships - In May 2022, Kelun Botai granted Merck exclusive rights to develop, use, manufacture, and commercialize sac-TMT outside Greater China [4]. - Tagolizumab, the PD-L1 monoclonal antibody used in combination with sac-TMT, is the first approved PD-L1 antibody for first-line treatment of nasopharyngeal carcinoma in China [6].

Core Viewpoint - The company Kolon Biotech has received breakthrough therapy designation from the NMPA for its antibody-drug conjugate sac-TMT in combination with the PD-L1 monoclonal antibody for the treatment of advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) without driver gene mutations, indicating significant clinical advantages over existing treatments [1][2]. Group 1 - The breakthrough therapy designation aims to expedite the research, development, and promotion of innovative treatments to address urgent medical needs [1]. - This marks the fifth breakthrough therapy designation for sac-TMT, with previous recognitions for treating various cancers including triple-negative breast cancer (TNBC) and hormone receptor-positive breast cancer [2]. - The results of the phase 2 OptiTROP-Lung01 clinical study for sac-TMT combined with Tagoli monoclonal antibody in treating advanced or metastatic non-squamous NSCLC patients will be presented at the 2025 ASCO annual meeting [2].

Summary of IDEA Biosciences Conference Call Company Overview - **Company**: IDEA Biosciences - **Focus**: Development of innovative therapies for cancer treatment, with six clinical programs currently and a target of nine by year-end [2][1]. Key Value Drivers - **Lead Program**: Derobicertib, targeting metastatic human melanoma, currently in a registrational trial [2][1]. - **Enrollment Status**: Completed enrollment for the trial, with over 320 patients enrolled, exceeding the target of 250 [12][13]. - **Primary Endpoint**: Median progression-free survival (PFS) is crucial for potential accelerated approval, with historical PFS around 2-3 months and a target of over 5.5 months for the current study [3][8]. Clinical Data and Updates - **PFS Results**: Previous studies showed a PFS of approximately 7 months, with a specific setting achieving nearly 11 months [10][11]. - **Overall Survival (OS)**: Anticipated update on OS data from the phase two study, with historical OS in treatment-naive settings around 12-13 months [17][18]. - **Market Opportunity**: Targeting the HLA A2 negative population, with an estimated annual incidence of 4,000 to 5,000 patients in the US and Europe [20][19]. Commercial Strategy - **Commercial Organization**: Building a commercial team with key hires, including a Chief Commercial Officer and heads of various departments [24][25]. - **Regulatory Strategy**: Fast track designation from the FDA for expedited review, with ongoing discussions regarding NDA submission [14][16]. Pipeline and Future Developments - **Additional Programs**: Focus on MTAP deletion space and DLL3, with several data catalysts expected [5][6][51]. - **Combination Therapies**: Emphasis on rational combinations, particularly with PRMT5 inhibitors in lung cancer [70][71]. - **IND Filings**: Targeting multiple INDs, with a goal of one IND per year, and a record of four INDs planned for the current year [86][87]. Financial Position - **Cash Reserves**: Reported over $1 billion in cash, providing a strong financial position for ongoing and future clinical activities [88][87]. Conclusion - IDEA Biosciences is positioned for significant growth with multiple clinical programs, a robust commercial strategy, and a strong financial foundation, aiming to address unmet needs in cancer treatment through innovative therapies and strategic partnerships.

（於 開 曼 群 島 註 冊 成 立 的 有 限 公 司） （股 份 代 號：01530） 自願公告 抗VEGF/PD-1雙特異性抗體藥物707注射液 獲國家藥品監督管理局突破性療法認定 本公告由三生制药（「本公司」，連 同 其 附 屬 公 司，統 稱（「三生制药」））自 願 刊 發。 三生制药自主研發的抗VEGF/PD-1雙特異性抗體（研 發 代 碼：707注 射 液）於2025年4 月17日 獲 國 家 藥 品 監 督 管 理 局 納 入 突 破 性 治 療 品 種，適 應 症 為 一 綫 治 療PD-L1表 達 陽 性 的 局 部 晚 期 或 轉 移 性 非 小 細 胞 肺 癌。 707注射液是三生制药基於CLF2專利平台自主開發的靶向VEGF/PD-1雙特異性抗 體，目 前 正 於 中 國 開 展 多 項 臨 床 研 究，其 中 一 綫 治 療PD-L1表達陽性的局部晚期 或轉移性非小細胞肺癌已獲國家藥監局藥品審評中心（「CDE」）批准開展III期臨床 研 究。此 外，707注 射 液 正 於 國 內 開 展 聯 合 化 療 一 綫 治 療 晚 期 非 小 細 胞 肺 癌、轉 移 性結直腸癌及晚期婦科 ...