Afamelanotide Acetate

本周药研简讯 • GSK抗PD-1单抗Jemperli获FDA加速批准，用于II/III期MSI-H/dMMR局部晚期直肠癌，成为该人群首个且唯一获批的免疫疗法，单药治疗有望让患者免于化疗、放疗和手术。 • 罗氏与翰森制药达成ADC合作协议，支付4亿美元首付款、交易总额最高150亿美元，翰森将贡献两个临床前ADC并按罗氏提出的靶点开发新分子，这是罗氏8月底以来第6笔中国交易。 • Medicovestor的首创新类FRα靶向化学免疫ADC药物MC001获FDA胰腺癌快速通道认定，继10月初IND获批后再获监管加持，即将启动国际多中心I期研究。 • 德国默克宣布以约13亿美元全现金收购英国RNAi公司Silence Therapeutics，获得针对真性红细胞增多症的潜在同类最佳siRNA药物divesiran，交易溢价达130%。 • EMA人用药品委员会推荐批准Revolution Medicines的胰腺癌药物Rasonque，但适应人群限制比FDA更严格，仅推荐用于至少接受过一线治疗后的患者。 • Rapport Therapeutics的癫痫候选药RAP-219在双相情感障碍躁狂发作II期研究中险胜主要终点，有望切入超10亿美元市场机会。 • Veradermics的口服缓释米诺地尔VDPHL01在男性型脱发第二项III期研究中达到主要终点，分析师称结果与首项研究一致且"方向上更好"。 • Disc Medicine的罕见光敏性疾病药物bitopertin在被FDA拒绝数月后宣布III期研究成功，将重新与监管机构沟通上市申请。 • ICER发布2022-2025年新药上市定价分析：净上市价格大幅飙升，多数新药定价远超其成本效益指南阈值。 • 投融资动态密集：Nouscom完成1.35亿美元D轮融资推进林奇综合征个体化免疫疗法；OrbiMed募集近30亿美元双基金含10亿美元亚洲基金；Ultragenyx两周内二度出售优先审评凭证获2.03亿美元。NEWS ANALYSIS药研重磅解读01 Jemperli单药获批直肠癌，免疫治疗首次让患者"免于化疗放疗手术" GSK宣布FDA加速批准抗PD-1单抗Jemperli（dostarlimab），用于未经治疗的II/III期MSI-H/dMMR局部晚期直肠癌成人患者，成为该适应症首个且唯一获批的免疫疗法。 获批依据是II期AZUR-1研究（154例）：研究者评估的12个月持续临床完全缓解率（cCR12）达68%，独立中心评审数据仍在成熟中（目前47%）；76%的可评估患者治疗后一年以上无癌症证据，中位随访超过两年无应答者复发。MSI-H/dMMR约占直肠癌的5-10%，现有标准治疗（化疗+放疗+手术）可导致肠道、泌尿、性功能障碍及不孕等长期后果，Jemperli单药（每3周500mg静脉输注、共9个周期6个月）有望让这部分患者避免三重治疗负担。该申请先后获得快速通道、突破性疗法认定、优先审评和国家优先凭证，并通过Project Orbis接受多国并行审评。继MSI-H实体瘤泛适应症之后，PD-1抗体正凭借生物标志物精准定位向"器官保留"治疗范式推进，国内多家PD-1企业在dMMR肠癌新辅助场景已有布局，该获批确立了新的全球参照标准。（GSK）02 罗氏150亿美元牵手翰森，ADC中国交易潮再下一城 翰森制药集团宣布与罗氏达成抗体偶联药物（ADC）合作：罗氏支付4亿美元首付款，叠加开发与商业里程碑后交易总额最高可达150亿美元，翰森将贡献两个临床前ADC，并按罗氏提出的靶点启动新ADC开发，双方未披露具体药物与适应症。 这是罗氏自8月底以来第6笔中国交易，据BioPharma Dive统计，其今年与中国药企的合作节奏已远超同行。ADC是中国创新药出海最强势的赛道：从早期科伦博泰、百利天恒与默沙东、BMS的重磅交易，到如今罗氏持续扫货，跨国药企对中国ADC平台的分子设计、连接子载荷与临床推进效率已形成高度共识。对罗氏而言，此举是在其HER2 ADC、Trop2等自有管线之外快速补齐下一代ADC货架；对翰森则是继多笔出海交易后再获MNC背书。"首付款+里程碑"的平台型合作模式正成为中国ADC企业估值的核心支撑，也预示2026年BD交易向临床前资产和平台授权进一步前置。（Roche/Hansoh）03 MC001获FDA胰腺癌快速通道，二聚体"化学免疫ADC"新机制登场 Medicovestor宣布FDA授予其首创新类FRα靶向化学免疫ADC药物MC001胰腺癌快速通道认定，这是继2025年6月孤儿药认定、2026年10月初IND获批（30天审查期无临床暂停）之后的又一监管里程碑，公司即将启动国际多中心I期研究，评估其在晚期或转移性胰腺癌及其他FRα表达实体瘤中的安全性与初步疗效。 MC001源自公司专有的ADoBind抗体支架平台：以高亲和力抗FRα IgG1抗体（携带S444C重链突变）通过链间二硫键交联成四价二聚体，经可酶切连接子偶联MMAE载荷，药物抗体比约5，兼具靶向细胞毒递送与抗体介导的固有免疫效应（ADCC）双重机制。FRα在高达80%的胰腺导管腺癌中过表达且与侵袭性相关；2026年AACR年会公布的临床前数据显示，单剂MC001在等效摩尔剂量下对肿瘤生长的抑制作用约为单体ADC的5倍，抑瘤持续超过30天并改善生存，且优于已获批的FRα ADC药物Elahere。胰腺癌五年生存率不足13%，是ADC攻坚最迫切的瘤种之一，二聚体支架能否在临床复制"化学+免疫"协同，值得持续关注。（Medicovestor）04 默克13亿美元收购Silence，siRNA罕见病赛道溢价创新高 德国默克（Merck KGaA）宣布将以每ADS 20美元现金收购英国siRNA公司Silence Therapeutics，股权价值约13亿美元、企业价值11亿美元，较其收盘价溢价130%，为今年大型交易中第二高溢价，预计2027年一季度完成交割。 交易核心资产是divesiran——一款靶向肝脏TMPRSS6 mRNA的siRNA疗法，通过减少铁调素抑制通路将铁输送从骨髓重定向，从而延缓红细胞过度生成，皮下注射每3个月一次，已具备III期就绪状态，用于治疗需要定期放血的真性红细胞增多症（PV）。8月公布的II期SANRECO研究中，divesiran组第18-36周应答率达88%（安慰剂组19%），6周给药组高达93.8%，华尔街分析师认为其具备"同类最佳"潜力，可挑战武田/Protagonist刚获批的Mimrylo（rusfertide）——后者峰值销售预期10-20亿美元。这是默克两年内第三笔重大收购（此前39亿美元收购SpringWorks、113亿美元收购Bio-Techne待交割），CEO Kai Beckmann称正"将管线向高专精度与未满足需求领域转型"。长效siRNA正成为罕见血液病领域继JAK抑制剂、肝素模拟肽之后的第三极，交易也再次确认RNAi平台资产的并购热度。（Merck KGaA/Silence）05 Rasonque获EMA推荐但适应症收窄，欧美监管口径分化 欧洲药品管理局（EMA）人用药品委员会（CHMP）上周五推荐批准Revolution Medicines的胰腺癌药物Rasonque，但适用人群限制比FDA更严格：EMA仅推荐用于至少接受过一线其他治疗后的患者，而FDA的批准范围同时覆盖该人群以及不适合一线化疗的患者。 推荐决定待欧盟委员会正式批准后生效，此后27个成员国还需与Revolution逐一完成价格谈判，医生方可开始用药。Rasonque是靶向KRAS突变的新机制药物，胰腺癌中约九成患者携带KRAS突变，此前该靶点长期被认为"不可成药"；其在FDA以突破性疗法认定获得快速批准，从申报到获批仅约一个月，创胰腺癌领域纪录。欧美监管在适应人群上的分歧意味着Revolution需要在欧洲补充更多后线治疗证据或开展上市后研究，商业化节奏也将受各国定价谈判拖累。对国内KRAS G12D/G12C抑制剂及pan-RAS管线企业，Rasonque的监管路径为"小人群快速获批+大适应症渐进扩展"策略提供了参考样本。（Revolution Medicines）06 RAP-219双相躁狂II期险胜，癫痫药物跨界精神科 Rapport Therapeutics宣布其原本开发用于癫痫的候选药物RAP-219在治疗双相情感障碍躁狂发作的II期研究达到主要终点，尽管部分分析师此前预期不高，药物仍以微弱优势"险胜"，为公司打开超过10亿美元的市场机会。 双相躁狂现有治疗以锂盐、丙戊酸盐和抗精神病药为主，起效慢、代谢副作用与依从性问题突出，新机制药物需求明确；癫痫与双相障碍共享神经元兴奋性失调的病理基础，抗癫痫药物跨界情绪稳定已有拉莫三嗪等成功先例。Rapport将RAP-219从癫痫适应症横向拓展至精神科，体现了中枢神经领域"一药多适应症"的平台化开发策略。"险胜"的措辞提示效应量有限，后续III期的剂量选择与终点设计将是关键，详细数据的成色将决定华尔街是否买单。对国内CNS管线企业，该结果再次验证离子通道/神经兴奋性调节靶点在精神科适应症拓展的可行性。（Rapport Therapeutics）07 VDPHL01第二项III期告捷，口服米诺地尔挑战脱发市场 Veradermics宣布其缓释口服米诺地尔VDPHL01在男性型脱发第二项III期研究（304研究）中达到主要终点，实现"早期、持续且稳健"的毛发生长；Cantor Fitzgerald分析师评价结果不仅与首项III期研究"一致"，而且"方向上更好"。 米诺地尔是经典外用脱发药物，但外用剂型依从性差、局部刺激明显，低剂量口服米诺地尔（oral minoxidil）近年在皮肤科超说明书使用中快速流行，却缺乏大规模随机对照证据与监管批准。VDPHL01以缓释制剂控制血药浓度、降低心血管副作用风险，若获FDA批准将成为首个有III期数据支持的处方口服米诺地尔。市场分歧在于：廉价仿制米诺地尔随处可得，处方版的溢价空间何在？分析师认为，具备"差异化疗效与安全性档案"的获批产品在脱发这一高支付意愿消费医疗领域可能快速放量。脱发赛道正从外用OTC向处方口服、从米诺地尔向雄激素受体降解剂等多机制演进，VDPHL01的两项III期连胜使其处于领跑位置。（Veradermics）08 bitopertin III期成功，Disc罕见病药物获"二次机会" Disc Medicine宣布其治疗红细胞生成性原卟啉症（EPP）的候选药物bitopertin在III期研究中取得成功，这是在该罕见光敏性疾病疗法被FDA拒绝数月之后，公司计划重新与监管机构沟通上市路径。 EPP是一种遗传性血红素合成障碍疾病，患者暴露于日光后即产生剧烈烧灼样疼痛，严重影响生活质量，此前全球仅有输血支持和阿法诺肽（Scenesse）等有限选择。bitopertin是甘氨酸转运体1（GlyT1）抑制剂，通过减少甘氨酸供给来限制原卟啉IX的过量蓄积，从源头缓解光毒性。首次申报遭FDA拒绝后，公司以新III期数据重启监管沟通，凸显罕见病药物开发中确证性试验设计与监管预期的博弈。若bitopertin最终获批，将成为EPP疾病修饰治疗的重要突破，也为"被拒药物如何翻身"提供案例——关键在于针对FDA关切点重新设计终点与人群。（Disc Medicine）09 ICER：新药上市定价依然过高，成本效益红线屡被突破 美国临床与经济研究所（ICER）发布2022-2025年新药上市定价分析：新药净上市价格（扣除回扣与折扣后）在这一时期显著飙升，绝大多数药物的定价远高于其成本效益指南阈值。 ICER以质量调整生命年（QALY）为标尺评估药物价值，其结论虽不直接约束定价，但常被美国医保方（PBM、商业保险）与国会政策讨论引用，也是《通胀削减法案》药价谈判背景下各方博弈的公共参照系。在MNC普遍面临专利悬崖、将资源押注肿瘤与罕见病高价药的当下，上市定价水涨船高几乎是结构性趋势；而ICER的持续警示可能强化支付方对"高定价、小人群"药物的准入审查与结果对赌合同（value-based contract）要求。对进入美国市场的中国创新药，定价策略需同时考虑ICER类卫生技术评估的舆论压力与商业保险的准入谈判现实，罕见病与肿瘤资产的定价空间正在被更精细的价值证据要求所约束。（ICER）10 一周资本动向：Nouscom融资1.35亿、OrbiMed募资30亿、PRV二度变现 本周生物医药投融资与资本运作密集：个体化癌症免疫疗法公司Nouscom完成1.35亿美元D轮融资，用于推进针对林奇综合征（遗传性结直肠癌高发人群）的个体化新抗原免疫疗法；顶级医疗基金OrbiMed一次性募集近30亿美元双基金，其中包括一只10亿美元的亚洲专项基金；Ultragenyx在不到两周内第二次出售优先审评凭证（PRV），成交价2.03亿美元。 三则动态分别映射当前资本市场的三条主线：其一，预防性/早发肿瘤免疫（林奇综合征癌前干预）正从概念走向大额融资支持的关键临床阶段；其二，尽管全球生物科技IPO窗口仍未完全打开，头部医疗PE对亚洲创新药资产的兴趣持续升温，OrbiMed的10亿美元亚洲基金即为信号；其三，PRV二级市场交易常态化为罕见病药企提供了可预期的非稀释性现金流，凭证定价在1.5-2亿美元区间企稳。对国内Biotech而言，亚洲专项基金的扩容意味着license-out之外，股权层面的跨境资本渠道也在拓宽。（Nouscom/OrbiMed/Ultragenyx）关于赛业生物 赛业生物成立于2006年，是国家高新技术企业及专精特新“小巨人”企业，作为深耕生物医药源头创新的全球化平台，致力于以新一代AI驱动的全人抗体药物研发与转化平台全面赋能全球创新药研发。 公司以自主基因编辑技术为核心，打造了HUGO-Ab®全人源抗体小鼠平台，融合共轻链技术、纳米抗体技术及自研AbSeek®智能抗体计算平台，构建起抗体HUGO-LAb™分子库，围绕肿瘤、自免、代谢等重点疾病方向，建立靶点研究、抗体发现、分子筛选与功能验证的一体化数据体系，并持续沉淀具有开发潜力的候选分子与技术数据资产。通过打通靶点验证、抗体筛选、序列优化和体内外验证全闭环，赛业生物革新传统抗体研发模式，实现可预测、高效率、高成药性的规模化抗体研发。同时配套HUGO-GT®全基因组人源化小鼠平台与完善的临床前CRO全链条服务，精准支撑靶点验证与功能研究，显著提升生物药临床转化效率。未来，赛业生物将持续迭代核心技术，依托全产业链优势，助力全球药企加速创新药研发与落地。 点击 「阅读原文」 立即咨询！

Positive APOLLO trial topline results demonstrated statistically significant and clinically meaningful benefit across all study endpointsSuccessfully met co-primary endpoints of reduction in protoporphyrin IX (PPIX) and increased total time in lightAll secondary endpoint results were also positive and statistically significant, including reductions in phototoxic reactions and improvements on multiple measures of light toleranceExploratory endpoints of quality-of-life improvement were also statistically significantBitopertin was generally well-tolerated with no new safety signals observed in APOLLODisc plans to resubmit an NDA to FDA by end of 2026, with an FDA decision anticipated by mid-2027Disc to discuss topline results in a corporate webcast on October 19, 2026 at 8:00am ET WATERTOWN, Mass., Oct. 19, 2026 (GLOBE NEWSWIRE) -- Disc Medicine, Inc. (NASDAQ:IRON), a clinical stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, today announced topline results from the APOLLO Phase 3 trial of bitopertin in erythropoietic protoporphyria (EPP), including X-linked protoporphyria (XLP). The trial met all its primary and secondary endpoints. “Today represents a potential breakthrough in the science and treatment of EPP,” said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc Medicine. “We could not have hoped for a better outcome, and we are grateful to the patients, caregivers, and physicians who have partnered with us throughout the clinical development of bitopertin. We look forward to engaging with regulators with the goal of bringing an important, innovative medicine to this community as efficiently as possible.” “This is the first time a potential treatment for EPP has shown robust evidence of whole blood PPIX reduction alongside unequivocal improvement on important measures of disease severity: increased time in light, reduced phototoxic reactions, and improved Patient Reported Outcome scores,” said Herbert L. Bonkovsky, M.D., Director of Gastroenterology & Internal Medicine, Professor Emeritus of Medicine at Wake Forest University School of Medicine, and a Principal Investigator of APOLLO. “These results are compelling evidence of the clinical benefit of a mechanism aimed at reducing the production of PPIX, the fundamental cause of disease,” added Will Savage, M.D., Ph.D., Chief Medical Officer of Disc Medicine. “It has been an incredible journey from establishing the original hypothesis of targeting heme synthesis in cellular models to seeing that biology translate into the clear and meaningful clinical effects from APOLLO. We plan to share more detailed data with additional insights from APOLLO at a future medical conference.” APOLLO is a randomized, double-blind pivotal Phase 3 trial evaluating the safety and efficacy of bitopertin versus placebo in adults and adolescents aged 12 or older with EPP or XLP. The trial enrolled 183 patients across the United States, Canada, Europe, and Australia, who were stratified by baseline light tolerance and geography and randomized 1:1 to receive 60 mg oral bitopertin or placebo once daily for 6 months. Summary of topline results: Bitopertin achieved statistical significance on both co-primary endpoints vs. placebo: Reduction in whole-blood metal-free PPIX from baseline (-41.4% vs. placebo, p<0.0001)Increase in average monthly total time in light without pain during month 6 (+12.2 hours vs. placebo, p=0.0010) Bitopertin achieved statistical significance on all secondary endpoints vs. placebo, including: Reduction in incidence rate of phototoxic reactions over the 6-month treatment period vs. placebo (p<0.0001)Increase in cumulative total time in light without pain over the 6-month treatment period vs. placebo (p<0.0001)Increase in change from baseline in average daily sunlight exposure time to prodrome vs. placebo (p=0.0077, adults only) Bitopertin demonstrated additional quality of life benefits vs. placebo, including improved Patient Global Impression of Change (PGIC) score (p<0.0001) and Patient Global Impression of Severity (PGIS) score (p<0.0001)Bitopertin was generally well-tolerated. Consistent with prior studies, dizziness was the most commonly reported treatment emergent adverse event (TEAE) in the bitopertin group (50%) and was mild or moderate and generally transient. There were no TEAEs above Grade 3. The APOLLO study adds to an extensive clinical data package supporting bitopertin’s potential in EPP. Based on these results, Disc plans to resubmit a New Drug Application (NDA) to the U.S. FDA by the end of 2026, with an FDA decision anticipated by mid-2027. Management will host a corporate webcast this morning, Monday, October 19 at 8:00am EST to discuss the APOLLO topline results and next steps. Please register for the event on the Events and Presentations page of Disc’s website (https://ir.discmedicine.com/). About Bitopertin Bitopertin is an investigational, clinical-stage, orally administered inhibitor of glycine transporter 1 (GlyT1) that is designed to modulate heme biosynthesis. GlyT1 is a membrane transporter expressed on developing red blood cells and is required to supply sufficient glycine for heme biosynthesis and support erythropoiesis. Disc is planning to develop bitopertin as a potential treatment for a range of hematologic diseases including erythropoietic porphyrias, where it has potential to be the first disease-modifying therapy. Bitopertin has been studied in multiple clinical trials in patients with EPP, including the Phase 2 open-label BEACON trial, the Phase 2 double-blind, placebo-controlled AURORA trial, the open-label extension HELIOS trial, and the APOLLO trial. Bitopertin is an investigational agent and is not approved for use as a therapy in any jurisdiction worldwide. Disc obtained global rights to bitopertin under a license agreement from Roche in May 2021. About EPP Erythropoietic protoporphyria (EPP), including X-linked Protoporphyria (XLP), is a rare, debilitating and potentially life-threatening disease caused by mutations that affect heme biosynthesis, resulting in the accumulation of a toxic, photoactive intermediate called protoporphyrin IX (PPIX). This causes severe reactions when patients are exposed to sunlight, characterized by excruciating pain, edema, burning sensations and potential blistering and disfigurement. PPIX also accumulates in the hepatobiliary system and can result in complications including gallstones, cholestasis, and liver damage in 20-30% of patients and in extreme cases liver failure. Current standard of care involves extreme measures to avoid sunlight, including restricting outdoor activities to nighttime, use of protective clothing and opaque shields, and pain management. This has a significant impact on the psychosocial development, quality of life, and daily activities of patients, particularly in young children and families. There is currently no cure for EPP and only one FDA-approved therapy, a surgically implanted synthetic hormone designed to stimulate melanin production called Scenesse® (afamelanotide). About Disc Medicine Disc Medicine is a clinical-stage biopharmaceutical company committed to discovering, developing, and commercializing novel treatments for patients who suffer from serious hematologic diseases. We are building a portfolio of innovative, potentially first-in-class therapeutic candidates that aim to address a wide spectrum of hematologic diseases by targeting fundamental biological pathways of red blood cell biology, specifically heme biosynthesis and iron homeostasis. For more information, please visit www.discmedicine.com. Disc Cautionary Statement Regarding Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, express or implied statements regarding Disc’s expectations with respect to: the next stages of its development of bitopertin; anticipated engagement with regulators with respect to bitopertin, including the timing of an NDA resubmission and an FDA decision; and the potential therapeutic benefits of bitopertin as a treatment for EPP, if approved. The use of words such as, but not limited to, “believe,” “expect,” “estimate,” “project,” “intend,” “future,” “potential,” “continue,” “may,” “might,” “plan,” “will,” “should,” “seek,” “anticipate,” or “could” or the negative of these terms and other similar words or expressions that are intended to identify forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on Disc’s current beliefs, expectations and assumptions regarding the future of Disc’s business, future plans and strategies, clinical results and other future conditions. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. Disc may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and investors should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements as a result of a number of material risks and uncertainties including but not limited to: the adequacy of Disc’s capital to support its future operations and its ability to successfully initiate and complete clinical trials; the nature, strategy and focus of Disc; the difficulty in predicting the time and cost of development of Disc’s product candidates; Disc’s plans to research, develop and commercialize its current and future product candidates; the timing of initiation of Disc’s planned preclinical studies and clinical trials; the timing of the availability of data from Disc’s clinical trials; Disc’s ability to identify additional product candidates with significant commercial potential and to expand its pipeline in hematological diseases; the timing and anticipated results of Disc’s preclinical studies and clinical trials and the risk that the results of Disc’s preclinical studies and clinical trials may not be predictive of future results in connection with future studies or clinical trials and may not support further development and marketing approval; the content and timing of decisions made by the FDA and other regulatory authorities; and the other risks and uncertainties described in Disc’s filings with the Securities and Exchange Commission, including in the “Risk Factors” section of Disc’s Annual Report on Form 10-K for the year ended December 31, 2025, and in subsequent Quarterly Reports on Form 10-Q. Any forward-looking statement speaks only as of the date on which it was made. None of Disc, nor its affiliates, advisors or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as result of new information, future events or otherwise, except as required by law. Media Contact Peg RusconiDeerfield Grouppeg.rusconi@deerfieldgroup.com Investor Relations Contact Kevin LuiPrecision AQ kevin.lui@precisionaq.com