Bexmarilimab

本期看点： 1. Faron Pharmaceuticals旗下Clever-1靶向单抗bexmarilimab联合标准治疗，在携带双等位基因TP53突变的初治高危骨髓增生异常综合征（HR-MDS）患者达到中位总生存期（OS）8.8个月的积极结果。 2. Dyne Therapeutics日前宣布，美国FDA已批准其抗体片段偶联siRNA疗法DYNE-302的IND申请，将启动用于治疗面肩肱型肌营养不良症（FSHD）的1期临床试验。 Bexmarilimab：公布1/2期试验结果 Faron Pharmaceuticals公布了BEXMAB试验的首次OS数据分析结果。经过中位14.9个月的随访，bexmarilimab联合azacitidine在携带双等位基因TP53突变的初治HR-MDS患者中取得了8.8个月的中位OS。该亚组患者通常预后较差，此次结果与历史数据基本一致；考虑到该亚组患者具有复杂的细胞遗传学特征，这一结果具有一定的鼓舞性。单等位基因TP53突变患者和TP53野生型患者的中位OS尚未达到。研究仍在持续开展生存随访，多名患者目前仍存活并处于随访阶段，相关数据有望随着随访时间延长进一步成熟。 BEXMAB是一项开放标签的1/2期临床试验，旨在评估抗Clever-1免疫疗法bexmarilimab联合标准治疗azacitidine，用于HR-MDS患者的治疗效果。在入组的21例初治HR-MDS患者中，48%携带TP53突变，这是一项与较差生存结局相关的不良预后因素。在等位基因频率数据已知的TP53突变患者中，33%为单等位基因TP53变异，67%为双等位基因TP53变异。 Bexmarilimab是一款由Faron Pharmaceuticals全资拥有的在研免疫疗法，通过靶向髓系细胞功能并激活免疫系统，旨在克服对现有治疗的耐药性并改善临床结局。该药物可结合巨噬细胞表面的免疫抑制性受体Clever-1，从而干预其促进肿瘤生长、转移及免疫逃逸的作用。 DYNE-302：IND申请获得FDA许可 Dyne Therapeutics日前宣布，美国FDA已许可DYNE-302的IND申请，允许公司启动一项治疗面肩肱型肌营养不良症的1期临床试验。DYNE-302基于该公司前两个临床项目所采用的FORCE平台开发。这两个项目分别为治疗适合外显子51跳跃治疗的杜氏肌营养不良（DMD）的zeleciment rostudirsen（z-rostudirsen，亦称DYNE-251），以及治疗1型强直性肌营养不良症（DM1）的zeleciment basivarsen（z-basivarsen，亦称DYNE-101）。 FSHD是一种罕见、进行性遗传性肌肉疾病，目前尚无获批疗法。骨骼肌中DUX4基因的异常去抑制及由此导致的异常表达是推动该病发生发展的重要因素，可导致肌肉损伤和功能丧失，进而限制患者的日常活动，并造成沉重的身体、情绪和经济负担。 DYNE-302是一款在研抗体片段偶联寡核苷酸疗法，由靶向转铁蛋白受体1（TfR1）的抗原结合片段（Fab）与siRNA偶联而成，旨在将对DUX4 mRNA具有高度特异性的siRNA递送至肌肉组织，从而抑制DUX4表达及其下游转录程序。在FSHD临床前模型中，DYNE-302可显著降低骨骼肌中的DUX4转录水平，并在重度疾病模型中显著逆转肌纤维损伤、改善肌肉功能。这些研究结果提示，通过DYNE-302靶向DUX4 mRNA，FSHD患者已经存在的骨骼肌损伤可能具有一定的逆转潜力。 MGC026：公布1期试验进展 MacroGenics公布了B7-H3靶向新型抗体偶联药物（ADC）MGC026正在开展的1期临床研究最新进展。MGC026采用基于拓扑异构酶I抑制剂的连接子-载荷组合，拟用于治疗晚期实体瘤患者。该研究的剂量递增部分已于2025年第四季度完成，受试者每3周接受一次给药，评估剂量范围为1-9 mg/kg。目前，研究正进一步评估7.5 mg/kg、每3周一次的给药方案，涉及复发或转移性头颈部鳞状细胞癌（SCCHN）、子宫内膜癌、黑色素瘤和软组织肉瘤4个瘤种特异性队列。该研究目前正在美国、澳大利亚和英国的临床中心开展。 其中，SCCHN队列采用Simon两阶段设计，计划入组40例患者。由于第一阶段已达到预设的缓解阈值，MacroGenics目前正在开展该队列第二阶段的患者入组，其余3个队列也在按计划推进。截至2026年7月8日，剂量递增阶段和正在进行的队列扩展阶段共入组74例患者。数据截止时，研究中未报告间质性肺病或眼部毒性病例，同时在多个适应症中观察到MGC026的抗肿瘤活性信号。 IDE892：公布1/2期试验结果 IDEAYA Biosciences宣布，其评估IDE892的1/2期临床试验已启动第二部分单药扩展研究。IDE892是一款具有“best-in-class”潜力的甲硫腺苷（MTA）协同型PRMT5抑制剂，拟用于治疗MTAP缺失型实体瘤，重点关注非小细胞肺癌（NSCLC）和胰腺导管腺癌（PDAC）。目前，IDE892已达到预计具有疗效的目标人体暴露水平，并实现24小时靶标EC90覆盖，因此启动了单药扩展研究。与此同时，剂量递增研究仍在继续，已完成多个剂量队列的评估，最大耐受剂量（MTD）尚未达到。 根据新闻稿，IDE892对MTA-PRMT5协同结合的选择性较SAM-PRMT5协同结合高约1400倍，其对CYP3A4的半数抑制浓度（IC50）超过45 μmol/L，且对7种主要细胞色素P450酶未显示时间依赖性抑制。目前，IDE892与IDE397的联合剂量递增研究正在MTAP缺失型NSCLC和PDAC患者中开展；IDEAYA Biosciences还计划于2026年下半年，在MTAP缺失型PDAC患者中推进IDE892与泛RAS抑制剂联合方案的首例患者入组。 BAT8013：1期试验首例患者给药 百奥泰（Bio-Thera Solutions）宣布，其潜在“best-in-class”、CD25抗体偶联药物BAT8013已启动1期临床研究并完成首例患者给药。该研究是一项多中心、开放标签的1期临床试验，将在晚期实体瘤患者中评估BAT8013的安全性和耐受性，并确定其推荐2期剂量。CD25又称白细胞介素-2受体α链（IL-2Rα），是一种由IL2RA基因编码的55 kDa跨膜糖蛋白，主要表达于活化的T细胞和B细胞、调节性T细胞（Treg）以及多种血液系统恶性肿瘤细胞。BAT8013由百奥泰自主研发的抗CD25抗体与专有连接子-载荷组合构成，其中包括在全身循环中稳定且可裂解的连接子，以及一种小分子拓扑异构酶I抑制剂。 CD25在实体瘤微环境中的Treg细胞上呈差异性高表达，因此，BAT8013有望通过清除Treg细胞，增强PD-1、PD-L1单克隆抗体等免疫肿瘤药物的治疗效果。系列临床前研究显示，BAT8013具有良好的稳定性和安全性，与百奥泰PD-1抑制剂BAT1308联合使用时表现出较高的抗肿瘤活性。其携带的小分子拓扑异构酶I抑制剂具有较强的细胞膜穿透能力，在靶向杀伤Treg细胞后，释放的载荷还可进一步杀伤邻近的Treg细胞，产生旁观者效应。 参考资料： [1] Dyne Therapeutics Announces U.S. FDA Clearance of Investigational New Drug (IND) Application for DYNE-302 in Facioscapulohumeral Muscular Dystrophy (FSHD). Retrieved July 28, 2026 from https://www.globenewswire.com/news-release/2026/07/28/3334144/0/en/dyne-therapeutics-announces-u-s-fda-clearance-of-investigational-new-drug-ind-application-for-dyne-302-in-facioscapulohumeral-muscular-dystrophy-fshd.html [2] EuBiologics reports positive phase 1 results for RSV vaccine, advances toward phase 2. Retrieved July 28, 2026 from https://www.koreabiomed.com/news/articleView.html?idxno=32564 [3] ProMIS Neurosciences Reports Positive Blinded Six-Month Interim Safety and Biomarker Data for PMN310 in the PRECISE-AD Phase 1b Alzheimer’s Disease Trial. Retrieved July 28, 2026 from https://www.globenewswire.com/news-release/2026/07/28/3334059/0/en/promis-neurosciences-reports-positive-blinded-six-month-interim-safety-and-biomarker-data-for-pmn310-in-the-precise-ad-phase-1b-alzheimer-s-disease-trial.html [4] Hopstem Receives U.S. FDA IND Clearance and Fast Track designations for hNPC01 in Hemorrhagic Stroke and Traumatic Brain Injury-Expanding Clinical Development Across Three Major Brain Injury Indications. Retrieved July 29, 2026 from https://www.globenewswire.com/news-release/2026/07/29/3334982/0/en/hopstem-receives-u-s-fda-ind-clearance-and-fast-track-designations-for-hnpc01-in-hemorrhagic-stroke-and-traumatic-brain-injury-expanding-clinical-development-across-three-major-bra.html [5] Insilico Medicine Receives FDA Fast Track Designation for ISM6331, the AI-driven Pan-TEAD Inhibitor, in Advanced Mesothelioma. Retrieved July 29, 2026 from https://www.prnewswire.com/news-releases/insilico-medicine-receives-fda-fast-track-designation-for-ism6331-the-ai-driven-pan-tead-inhibitor-in-advanced-mesothelioma-302836507.html [6] MannKind Reports Positive Phase 1b INFLO-1 Results, Demonstrating Safety and Tolerability of Nintedanib DPI and Clinical Validation of Its DPI Technology in IPF. Retrieved July 29, 2026 from https://www.globenewswire.com/news-release/2026/07/29/3335054/29517/en/mannkind-reports-positive-phase-1b-inflo-1-results-demonstrating-safety-and-tolerability-of-nintedanib-dpi-and-clinical-validation-of-its-dpi-technology-in-ipf.html [7] Ocugen Announces that the U.S. Food and Drug Administration Has Granted OCU410 Regenerative Medicine Advanced Therapy (RMAT) Designation for Treatment of Geographic Atrophy, Secondary to Dry Age-Related Macular Degeneration. Retrieved July 29, 2026 from https://www.globenewswire.com/news-release/2026/07/29/3335110/0/en/ocugen-announces-that-the-u-s-food-and-drug-administration-has-granted-ocu410-regenerative-medicine-advanced-therapy-rmat-designation-for-treatment-of-geographic-atrophy-secondary-.html [8] Bio-Thera Solutions Announces First Patient Dosed in Phase 1 Study for BAT8013, an Antibody Drug Conjugate Targeting CD25 for the Treatment of Advanced Solid Tumors. Retrieved July 30, 2026 from https://www.prnewswire.com/news-releases/bio-thera-solutions-announces-first-patient-dosed-in-phase-1-study-for-bat8013-an-antibody-drug-conjugate-targeting-cd25-for-the-treatment-of-advanced-solid-tumors-302833333.html [9] MacroGenics Provides Clinical Update on Ongoing Phase 1 Study for MGC026. Retrieved July 30, 2026 from https://ir.macrogenics.com/news-releases/news-release-details/macrogenics-provides-clinical-update-ongoing-phase-1-study [10] Inside Information: Faron announces encouraging first overall survival data from frontline higher-risk (HR) MDS patients in the BEXMAB phase 1/2 trial. Retrieved July 30, 2026 from https://faron.com/releases-and-publications/inside-information-faron-announces-encouraging-first-overall-survival-data-from-frontline-higher-risk-hr-mds-patients-in-the-bexmab-phase-1-2-trial/ [11] IDEAYA Biosciences Announces IDE892, a Potential Best-in-Class MTA-Cooperative PRMT5 Inhibitor, Initiates Part 2 Monotherapy Expansion in the Phase 1/2 Study in MTAP-Deleted Pancreatic and Lung Cancers. Retrieved July 30, 2026 from https://www.prnewswire.com/news-releases/ideaya-biosciences-announces-ide892-a-potential-best-in-class-mta-cooperative-prmt5-inhibitor-initiates-part-2-monotherapy-expansion-in-the-phase-12-study-in-mtap-deleted-pancreatic-and-lung-cancers-302834359.html [12] Revagenix Doses First Participant in Phase 1 Trial of Rev-56, an Inhaled Therapy for Non-CF Bronchiectasis. Retrieved July 30, 2026 from https://www.revagenix.com/press-releases/revagenix-doses-first-participant-in-phase-1-trial-of-rev-56 免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，聚焦全球生物医药健康创新

Inside Information: After a median follow-up of 14.9 months, median overall survival has not yet been reached in frontline HR MDS without TP53 mutations, nor has it yet been reached for monoallelic TP53 mutated patients. For patients with biallelic TP53 mutations the median OS is 8.8 months in line with historical results in this highly aggressive population. TURKU, FI / ACCESS Newswire / July 27, 2026 / Faron Pharmaceuticals Ltd. ((HEL:FARON)(LSE:FARN), a global, clinical-stage biopharmaceutical company focused on creating innovative cancer treatments that leverage the patient's own immune system, today announced that it has conducted its first overall survival (OS) data cut from treatment-naïve higher-risk myelodysplastic syndrome (HR-MDS) patients enrolled in the BEXMAB trial. At this time, after a median follow-up of 14.9 months, the only subgroup to have hit the median is the biallelic TP53 mutated population. This subgroup, historically associated with very poor outcomes, achieved a median OS of 8.8 months, which is in line with historical data and reassuring given the complex cytogenetics within the BEXMAB biallelic patient population. Other subgroups: monoallelic TP53 mutated and wildtype patients have still not reached their median OS. The BEXMAB trial is an open-label Phase 1/2 study investigating bexmarilimab , Faron's novel anti-Clever-1 immunotherapy, in combination with standard-of-care azacitidine in patients with higher-risk myelodysplastic syndromes. Of the 21 treatment-naïve HR-MDS patients, 48% harboured TP53 mutations, a well-established adverse prognostic factor associated with inferior survival outcomes. Of the TP53 mutated patients (with known allele frequency data), 33% had monoallelic TP53 alterations, and 67% had biallelic TP53 alterations. Survival follow-up is ongoing, and several patients remain alive and under follow-up, providing the opportunity for further maturation of the dataset over time. ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); " These results further support the potential of bexmarilimab in frontline TP53 wild-type and mutated HR-MDS patients, where significant unmet medical need remains," said Dr. Petri Bono, Chief Medical Officer of Faron Pharmaceuticals. "The data provide additional support as we continue preparations for BEXERA, our planned randomized study evaluating bexmarilimab in combination with azacitidine in frontline HR-MDS patients. We look forward to presenting the detailed full BEXMAB dataset at an upcoming scientific meeting later this year." For more information, please contact: IR Partners, Finland (Media) Kare Laukkanen +358 50 553 9535 / +44 7 469 766 223 kare.laukkanen@irpartners.fi FINN Partners, US (Media) Alyssa Paldo +1 847 791-8085 alyssa.paldo@finnpartners.com Cairn Financial Advisers LLP (Nominated Adviser and Broker) Sandy Jamieson, Jo Turner +44 (0) 207 213 0880 Sisu Partners Oy (Certified Adviser on Nasdaq First North) Juha Karttunen Jukka Järvelä +358 (0)40 555 4727 +358 (0)50 553 8990 About bexmarilimab Bexmarilimab is Faron's wholly owned, investigational immunotherapy designed to overcome resistance to existing treatments and optimize clinical outcomes, by targeting myeloid cell function and igniting the immune system. Bexmarilimab binds to Clever-1, an immunosuppressive receptor found on macrophages leading to tumor growth and metastases (i.e. helps cancer evade the immune system). By targeting the Clever-1 receptor on macrophages, bexmarilimab alters the tumor microenvironment, reprogramming macrophages from an immunosuppressive (M2) state to an immunostimulatory (M1) one, upregulating interferon production and priming the immune system to attack tumors and sensitizing cancer cells to standard of care. About Faron Pharmaceuticals Ltd. Faron (HEL:FARON)(LSE:FARN) is a global, clinical-stage biopharmaceutical company, focused on tackling cancers via novel immunotherapies. Its mission is to bring the promise of immunotherapy to a broader population by uncovering novel ways to control and harness the power of the immune system. The Company's lead asset is bexmarilimab , a novel anti-Clever-1 humanized antibody, with the potential to remove immunosuppression of cancers through reprogramming myeloid cell function. Bexmarilimab is being investigated in Phase 1/2 clinical trials as a potential therapy for patients with hematological cancers in combination with other standard treatments. Further information is available at Forward-Looking Statements Certain statements in this announcement are, or may be deemed to be, forward-looking statements. Forward looking statements are identified by their use of terms and phrases such as "believe", "could", "should", "expect", "hope", "seek", "envisage", "estimate", "intend", "may", "plan", "potentially", "will" or the negative of those, variations or comparable expressions, including references to assumptions. These forward-looking statements are not based on historical facts but rather on the Directors' current expectations and assumptions regarding the Company's future growth, results of operations, performance, future capital and other expenditures (including the amount, nature and sources of funding thereof), competitive advantages, business prospects and opportunities. Such forward-looking statements reflect the Directors' current beliefs and assumptions and are based on information currently available to the Directors. A number of factors could cause actual results to differ materially from the results and expectations discussed in the forward-looking statements, many of which are beyond the control of the Company. In addition, other factors which could cause actual results to differ materially include the ability of the Company to successfully license its programs within the anticipated timeframe or at all, risks associated with vulnerability to general economic and business conditions, competition, environmental and other regulatory changes, actions by governmental authorities, the availability of capital markets or other sources of funding, reliance on key personnel, uninsured and underinsured losses and other factors. Although any forward-looking statements contained in this announcement are based upon what the Directors believe to be reasonable assumptions, the Company cannot assure investors that actual results will be consistent with such forward-looking statements. Accordingly, readers are cautioned not to place undue reliance on forward-looking statements. SOURCE: Faron Pharmaceuticals View the original press release on ACCESS Newswire