1. PURPOSE

This document describes KOUSAI´s position, and reflects our Corporate Policy on how patients in medical need may be able to access our new medicines before they are fully approved for use on prescription. 

2. SCOPE

We cover two areas in this document:

1.    Where patients participate in one of our clinical studies, we explain how we may provide continued treatment for patients after their study treatment, or after the whole study, has completed.

2.    Where patients are suffering from a disease that is not possible to be treated with existing medicines, or patients are not able to participate in one of our clinical studies, we explain how we handle requests for compassionate or emergency use of our new medicines.

3.    POLICY

3.1 Criteria

Where there is a possible need for patients to gain early access to a new medicine that is not generally available for prescription, KOUSAI will consider the need for early access using the following criteria: 

•      Where it is expected that there is a need for ongoing treatment with one of our new medicines, we recognize that this must be through normal health services once the new medicine is available for prescription. As such we will only conduct clinical studies and provide early access outside of studies, in those countries where we intend to make the medicine available through normal prescription channels after its approval.

•      We recognize that we must have a minimum level of evidence that the new medicine is likely to work before considering early access and must not provide excessive risk to any patients who may receive the new medicine early.

•      While addressing this patient need, we will ensure that if we agree to provide early access to our new medicines, that there is sufficient medicine available to ensure the supply will not run out for those patients receiving it early, or for those patients in our clinical studies. This is because we want to ensure that when providing early access, it does not prevent us completing clinical studies and regulatory approval that would lead to wider access through normal routes of prescription.

Any early access programs we provide will be in line with the laws and requirements of the country involved. 

3.2 Continued treatment after clinical studies

To be sure that our new medicines will work effectively and be safe enough to be prescribed for the diseases patients are suffering from, we study them rigorously to see whether they provide adequate relief. The results of these clinical studies allow the regulatory authorities to independently assess whether to allow the new medicines to be prescribed. As this can take several years, patients who participate in our clinical studies may have to wait for a new medicine to become available on prescription. 

Where there may be a benefit for a patient to continue to be treated after the clinical study has ended, but before it is available on prescription, we will decide if we have enough information to justify early access. This will depend on the severity of the disease we are studying, how much evidence we have that the medicine works as expected and is sufficiently safe, and whether alternative effective medicines are already available on prescription. If we plan for continued treatment after a clinical study, we will include this information in our study documents so that it is clear to each patient whether this is a possibility before they start in the study. 

We understand that it can be difficult to decide to participate in a clinical study, so will ensure we make this information as clear as possible. To be sure we can assess whether the new medicine continues to work effectively and is safe enough to continue to be used during early access, we will ask for the patient’s consent for their physician to collect some relevant information from them on an ongoing basis while they receive the medicine. 

3.3 Compassionate or emergency use outside of clinical studies

The best way to see if a new medicine is sufficiently safe and effective is to run clinical studies. As such we prefer that, where possible, patients have the opportunity to participate in our clinical studies. We recognize however that not everyone has this opportunity. Where patients who cannot enroll in a clinical study are suffering from serious or immediately life threatening diseases, and there are no satisfactory alternative treatments available, we will consider early access if there is enough evidence available in their condition (as described above) and whether there is a possibility of them being able to access the medicine on prescription in the future. 

In the same way as after a clinical study, we want to be sure we can assess whether the new medicine continues to work and is safe enough to continue to be used. As such we will ask for the patient’s consent for their physician to collect some relevant information from them on an ongoing basis while they receive the medicine. 

3.4 Discontinuation of early access

When a new medicine receives approval from the regulatory authorities for the disease we are studying, and is available for prescription in the patient’s country, we will phase out the early access to allow the health system to prescribe the new medicine in the normal manner. 

If however the medicine is approved but not available for prescription for some reason, and there is no alternative medicine available, we will work with country authorities to discuss if we can jointly set up a patient-assistance program to allow patients who are benefitting from it, who do not have alternative treatment options, to continue to receive the new medicine. Patient safety is our primary concern, and we respect patient safety by continually evaluating all the evidence that we have available to ensure that early access use of new medicines remains effective and appropriately safe. If however the evidence demonstrates that the new medicine is not going to be as sufficiently effective or safe as we had hoped, we will discontinue all early access and work with patients and their physicians to help them safely transition patients to other treatments. When regulatory authorities do not approve the use of the new medicine for prescription, because it is considered insufficiently safe or effective, and KOUSAI discontinues its work to achieve regulatory approval, we will work with patients and their physicians to help them safely transition patients to other treatments.

Contacts: Yali Quan

E-mail: clinicaltrial@kousai.vip

Tel: 021-57527655

2025年12月25日，中国创新生物制药企业恒赛生物宣布一项重磅喜讯——其自主研发的原创树突状细胞疫苗（DC疫苗）KSD-101注射液正式获得美国食品药品监督管理局（FDA）授予的快速通道资格（Fast Track Designation, FTD），用于治疗复发或难治性（R/R）EBV 阳性淋巴瘤。KSD-101成为我国首个获得FTD认定的DC疫苗产品，标志着该疗法在解决未满足的临床需求方面获得国际监管机构的高度认可，并将加速其在美国的临床开发和上市审评进程，为全球复发或难治性EBV阳性淋巴瘤疾病患者带来了全新的治疗希望。

FTD认定旨在加速针对严重疾病且存在未满足医疗需求的药物的开发与审评，通过提供更频繁的监管沟通、滚动审评等一系列便利措施，助力相关药物早日惠及患者。KSD-101此次获得FTD，是基于其作为全球首款靶向EBV的广谱DC疫苗的创新性，以及其在早期临床研究中表现出的显著治疗潜力。

EB病毒是一种在全球范围内感染率超过95%的常见病毒，被世界卫生组织明确认定为首个与人类肿瘤相关的重要病毒。其感染可引发EBV相关淋巴增殖性疾病（EBV-LPD）、鼻咽癌、胃癌等多种严重疾病。目前，针对EBV相关淋巴增殖性疾病等患者，临床上仍缺乏有效的治疗方案，存在显著的未被满足的医疗需求。KSD-101作为我国首款获得FDA新药临床试验(IND)许可的原创DC疫苗，使用患者自身来源的单核细胞诱导分化为成熟的树突状细胞，并负载EBV相关类肿瘤复合抗原，可精准呈递EBV潜伏期及裂解期蛋白LMP2A、 EBNA3A/3B 、 EBNA4 、BMLF1等多种病毒抗原表位，高效激活CD4⁺辅助性T细胞与CD8⁺细胞毒性T细胞，从而构建全面且强大的抗病毒与抗肿瘤免疫应答。

KSD-101单药治疗复发难治的EBV相关血液肿瘤的I期临床研究中，客观缓解率（ORR）高达91.67%，其中完全缓解率（CR）83.33%，疾病控制率（DCR）100%，同时展现出持久应答和良好的安全性。这一突破性数据为KSD-101的临床价值提供了坚实支撑。

KSD-101是恒赛生物依托其自主研发的 “Eco-DC Vax”技术平台开发的首款自体DC疫苗，为我国首个获得美国FDA IND批件的原创DC疫苗产品。其作用机制是通过采集患者自身的单核细胞，在体外诱导分化为树突细胞，并负载EBV相关的特异性复合抗原，制备成个体化的治疗性疫苗。该疫苗回输至患者体内后，可精准激活并训练患者自身的免疫系统，使其识别和杀伤表达EBV抗原的肿瘤细胞，并建立起持久的抗肿瘤免疫记忆。

恒赛生物成立于2018年，是致力于树突细胞疫苗（DC疫苗）研发与产业化的科技创新型企业、国家高新技术企业。公司已成功构建 Eco-DCVax 平台，旨在打破产业化技术壁垒、开发具有自主产权的全球First-in-Class的安全高效、给药便利的高品质DC疫苗产品。所开发产品——治疗性树突细胞疫苗，为复发难治性肿瘤、慢性病毒感染、自身免疫性疾病等临床亟需有效治疗手段的疾病提供治疗新途径、新策略。

公司创始人兼CEO刘慧宁博士表示：“此次KSD-101获得FDA快速通道资质认定，是对我们研发实力与产品价值的重要肯定。恒赛生物将以此为契机，充分利用快速通道带来的监管便利，加速推进KSD-101的全球临床开发进程，早日为国内外EBV相关疾病患者提供更有效的治疗选择。”

作为国内DC疫苗研发领域的引领者，恒赛生物已实现KSD-101的中美双报，其国内IND申请已正式被中国国家药监局受理。此次获得FDA快速通道资质认定，不仅将加速KSD-101的海外临床推进与上市进程，也将进一步提升国产原创生物药在全球肿瘤免疫领域的影响力，为我国生物医药产业的国际化发展注入新动力。的原创DC疫苗产品。其作用机制是通过采集患者自身的单核细胞，在体外诱导分化为树突细胞，并负载EBV相关的特异性复合抗原，制备成个体化的治疗性疫苗。该疫苗回输至患者体内后，可精准激活并训练患者自身的免疫系统，使其识别和杀伤表达EBV抗原的肿瘤细胞，并建立起持久的抗肿瘤免疫记忆。

—  END  —

2025年8月30日上午，乐城先行区生物医学新技术政策推介会暨第一批企业集中签约大会在博鳌乐城永不落幕国际创新药械展隆重举行。海南省人民政府、海南省工信厅、海南省卫健委、海南省医保局、海南省药监局、海南国际经发局、乐城先行区管理局、琼海市人民政府、乐城医疗机构及生物医药企业共同出席见证。

本次大会上，乐城管理局与13家国内头部生物医药企业达成签约合作。恒赛生物联合创始人、首席运营官程相国先生作为获批项目企业代表参与签约，标志着恒赛生物DC疫苗项目正式落地乐城先行区。

作为全国唯一的“医疗特区”和海南自贸港建设的重要功能平台，乐城近年来充分运用国家赋予的“国九条”等特殊政策，逐步构建起覆盖国际创新药械快速引进、临床应用、注册审批及生产制造的全链条服务体系。同时，积极把握医疗科技革命和产业变革新机遇，在全国率先开展细胞治疗、基因编辑等生物医学新技术临床转化应用。

恒赛生物DC疫苗安全性良好、应用广泛（治疗及防复发）、无需清淋预处理、门诊可施治、治疗成本较低，有望提升临床可及性与患者依从性，完美契合乐城先行区生物医学新技术产业政策。恒赛生物作为国内首批获准在乐城开展DC疫苗转化应用的企业，未来将结合自身发展需求和乐城的政策与平台优势，与乐城先行区深度融合、共同发展。

公司在研管线均为没有任何基因编辑的自体DC疫苗，适应症涵盖血液肿瘤和实体瘤。核心管线KSD-101为全球首个靶向EBV广谱的DC疫苗，同时也是中国首个获得美国FDA IND批件的原创DC疫苗。

公司依托自主构建的Eco-DC Vax平台，系统整合抗原预测、免疫学验证、工艺优化与质量控制四大模块，具备“一药多靶”和平台延展性。目前在上海自贸区临港先片区设有建筑面积 6300平、产能约5000人份/年的综合研发生产基地，配备行业顶尖设施，具备从科研到产业化的硬实力。恒赛生物自创立以来秉持“让肿瘤患者无痛治愈”的朴素愿望， 旨在打破产业化技术壁垒、开发具有自主产权的全球First-in-Class的安全高效、给药便利的高品质DC疫苗产品，为复发难治性肿瘤、慢性病毒感染、自身免疫性疾病等临床亟需有效治疗手段的疾病提供治疗新途径、新策略。

2025年8月15日，恒赛生物临港研发生产基地启用仪式在临港新片区生命蓝湾智造园十期隆重举行。 仪式以“恒创前沿・赛领健康”为主题，邀请了临港管委会、行业专家、股东代表、合作伙伴、园区领导与恒赛生物全体员工共同见证这一重要时刻。

灵蛇献瑞启新篇，菁英共赴春日宴。值此灵蛇盘踞、万物萌新之际，恒赛生物全体员工欢聚一堂，于浙江舟山举办2025年年会暨年度总结活动。恒赛团队踏浪而行，以协作淬炼默契，以欢笑凝聚共识，在碧海金沙间共绘新春序章。

破开万顷波涛，团队共赴碧海征途。出海巡游，体验渔获，收网时频传的捷报，鱼跃舱中的丰收画卷，恰似团队乘风破浪、硕果盈枝的奋斗缩影

夕阳下，拔河赛场呐喊震天，绳索两端拉锯的是万众一心的坚韧；你画我猜妙趣横生，笔触间流淌的是心有灵犀的默契；长绳翻飞中跃动的身影，恰似百舸争流的进取之姿。汗水与欢笑交织，凝聚成势不可挡的向心力。

夜幕垂落，烟花如银河倾泻，璀璨光华照亮每一张仰望的脸庞。恒赛生物愿做暗夜追光者，以今日之热血为序章，争当明日之行业明星，让团队光芒如星火燎原，辉映未来征途！

栉风沐雨结硕果，笃行不怠续华章。恒赛生物创始人兼CEO刘慧宁博士在年终总结会议上发表精彩致辞，回顾了过去一年恒赛生物的累累硕果，肯定了每一位团队成员的付出，展望未来树突细胞疫苗的无限可能。

联合创始人程相国先生以公司的价值观为纽带，滋养团队认同，共筑长青基业。聚百川灵秀、激细胞潜能、赋医者仁术、创团队恢弘的理念深入人心。

恒赛生物研发部、质量部、质检部、生产部、临床部、行政部、信息部、商务部等负责人对过去一年的工作进行详尽总结，数据如珠玑落盘，项目似锦绣铺陈。

一马当先者，必得头筹；兢兢业业者，终获殊荣。颁奖典礼上，优秀员工们以专业能力，凭出色表现赢得了属于他们的高光时刻。恒赛生物的今天离不开每一位成员的奋斗，以榜样为镜，在新的一年里继续携手同行，共创辉煌！期待更多成员站上荣耀舞台，与恒赛生物共同成长！

以信念为帆，破旧岁之浪；

以协作为桨，开新程之局；

以创新为锚，定行业之标！

愿恒赛生物如星火燎原辉映长空，

以创新为翼再书华章；

愿追梦者们似春潮奔涌势破千钧，

凭同心之力共攀巅峰！

END

2025年3月3日，恒赛生物自主研发的KSD-201 DC疫苗治疗晚期肾细胞癌项目在华中科技大学同济医学院附属同济医院（简称“武汉同济医院”）正式启动。该项目依托恒赛生物全球领先的Eco-DC Vax技术平台，联合武汉同济医院泌尿外科专家团队，旨在为复发难治性肾癌患者提供新型免疫治疗方案。这是恒赛生物继EBV相关鼻咽癌后，又一个进入临床研究的实体瘤在研管线

启动会现场

武汉同济医院泌尿外科作为“中国泌尿肿瘤十强”之一，其多学科团队（MDT）在肾癌微创手术、个体化综合治疗领域居国内领先地位。肾细胞癌（RCC）是起源于肾小管上皮的恶性肿瘤，约占肾脏恶性肿瘤的85%。透明细胞肾细胞癌（ccRCC）是RCC最常见的病理亚型。ccRCC在所有RCC病理类型中预后最差，复发及转移的潜能最高，而且对放疗、化疗均不敏感。晚期ccRCC是一种无法治愈的疾病，易转移、易复发、难根治，靶向治疗疗效及生存获益个体化差异明显，不良反应严重，易耐药。因此，ccRCC一直以来都是困扰众多患者的难题，给患者及其家庭带来了沉重的负担。

启动会现场，以主要研究者王少刚教授为首的泌尿外科专家团队和恒赛生物临床团队共同探讨了DC疫苗治疗ccRCC疾病IIT研究的重点和难点，双方明确了项目目标和人员分工，展现出对项目未来发展的高度期待和坚定信心，为后续项目高效推进奠定了坚实的基础。

恒赛生物创始人兼CEO刘慧宁博士表示：“DC疫苗的独特优势在于诱导长期免疫记忆，通过精准的特异性免疫反应发挥持久的抗肿瘤作用。此次与武汉同济医院的合作，将加速技术从实验室到临床的转化，期待DC疫苗为更多肾癌患者提供安全、便利和持久有效的治疗方案。”

KSD-201是负载广谱的透明细胞肾细胞癌相关抗原的人单核来源的自体树突细胞疫苗。通过细胞因子诱导单核细胞转化为树突细胞，用广谱的透明细胞肾细胞癌相关抗原负载树突细胞并诱导其成为成熟的树突细胞，制备成树突细胞疫苗，是一种肿瘤治疗性疫苗。通过皮下注射后能在人体内激活患者的免疫功能，从而达到治疗晚期透明细胞肾细胞癌的效果。

恒赛生物成立于2018年，是致力于树突细胞疫苗（DC疫苗）研发与产业化的科技创新型企业、国家高新技术企业。公司已成功构建Eco-DCVax 平台，旨在打破产业化技术壁垒、开发具有自主产权的全球First-in-Class的安全高效、给药便利的高品质DC疫苗产品。所开发产品——治疗性树突细胞疫苗，为复发难治性肿瘤、慢性病毒感染、自身免疫性疾病等临床亟需有效治疗手段的疾病提供治疗新途径、新策略。

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恒赛生物位于上海临港蓝湾的研发中心和中试车间于2021年投入使用，中山研发中心正在建设中。公司首发管线KSD-101已顺利完成中美双报，即将正式进入注册临床阶段。公司现已和多家国内外科研机构和三甲医院建立稳定的合作关系，开展临床研究和技术合作，以全面验证DC疫苗的临床安全性和有效性。期竣工、高效运营！”