Mabwell Therapeutics, Inc.

中国好BD，在这里相聚！药时代学苑「第3期」创新药BD高阶研讨会欢迎您！回顾2023年Q1季度BD交易，为何早期项目正变得越来越抢手？犹记得2023年开年之时，医药BD人还沉浸在对2022年的复盘和对2023年的憧憬之中。当时，大多数BD人认为2023年将会是一个丰收年。转眼2023年第二季度已经过半，中国医药BD人的成绩如何，是否如大家在开年时所期盼呢？带着这个问题，我们对2023年第一季的BD交易进行了回顾。经过梳理我们发现，2022年1-3月共发生45笔与中国药企有关的交易（包括license in、license out以及国内企业合作）；而2023年1-3月，只发生27个与中国药企有关的交易。从交易数量上来看，今年医药行业的回暖热度似乎不及预期，但从交易趋势来看，目前发生的27个BD交易中，license in共5项，license out共15项，国内/境内合作共6项，中国药企出海依旧是大趋势。在披露了临床进展的19个项目中，有7个项目处于临床前或IND阶段，3个项目处于临床I期，这些项目数量加起来约占项目总数的一半。对于大部分处于新药研发和BD交易初期阶段的国内药企来说，这些成功案例依然值得重点关注和研究。本篇文章对2023年第一季度临床前及临床I期的项目做了盘点，期望能够为大家在新药立项和BD交易工作上带来新的思路。也期望大家能够通过药时代BD投融资平台找到合适的项目（项目表单请参考文末图片）。积极地合作，谨慎地付款据不完全统计，此次BD交易初期阶段的10个项目中，已披露金额的7项交易总额达52.14亿美元，而已披露的首付款金额普遍较低，均占交易额的2-7%左右，这也反映出创新药交易双方所持有的谨慎态度以及处于初期阶段管线研发的难度和风险。从靶点来看，主要还是集中在肿瘤热门靶点领域，如EGFR、TAAxCD3、CD19、PD-1xCD40和Claudin18.2，占交易项目的一半，此外还包括醛固酮合成酶、TYK2/JAK1和GIPR&GLP-1R非肿瘤靶点。从药物种类来看，初期阶段的药物不乏研发技术相对成熟、难度相对较低的小分子药物，这类药物高达4项，同时我们也看见涌现了种类丰富、研发热度和难度都很高的抗体、ADC和细胞治疗药物，各占3、2和1项。独特的作用机制，更优秀的数据在早期交易中，最引人注目的是葛兰素史克（GSK）就药明生物“1款双抗+3款TCE”达成的过百亿（15亿美元）“巨额交易”，显示出GSK对双抗领域的一片看好。紧跟其后的是阿斯利康就康诺亚的一款靶向Claudin 18.2的ADC达成11.88亿美元交易，目前国内外关于Claudin 18.2靶点的研发热度日益高涨，研发管线几乎包含了所有热门药物研发类型，如ADC、单克隆抗体、双特异性抗体和细胞治疗药物。排名第三的是Biohaven与杭州高光制药JAK1/TYK2抑制剂的授权，交易金额达9.7亿美元。值得注意的是，自从去年12月武田制药以高达60亿美元从Nimbus Therapeutic购买了TYK2抑制剂NDI-034858后，将本就是热门靶点的TYK2抑制剂推向研发高潮，这项交易也达到了小分子药物研发的高光时刻，国内外纷纷推进了TYK2抑制剂研发布局。从目前热门、交易额大的项目中，可以窥见新药授权交易趋势，走在国际研发前沿的生物技术公司纷纷刷新生物技术产品，而传统大药企要么自主布局要么也通过购买管线提前布局在热门疾病和热门靶点领域。01案例一转让方：药明生物受让方：GSK交易项目：1款双抗+3款TCE靶点：TAAxCD3适应证：肿瘤项目阶段：临床前交易金额：15亿美元2023年1月5日，药明生物通过官网宣布公司与GSK就一款双抗达成独家许可协议（包含全球独家研究、开发、生产和商业化权利），并可基于该平台选择至多3款其他TCE、双抗/多抗药物。该款双抗分别靶向肿瘤细胞表面的肿瘤相关抗原（TAA）和T细胞表面抗原CD3，通过与肿瘤细胞及T细胞结合，特异性激活T细胞，从而杀伤肿瘤细胞，产生抗肿瘤活性。根据协议，药明生物将获得4000万美元首付款和最高达14.6亿美元的四款TCE抗体的研究、开发、注册和商业化里程碑付款。药明生物也有资格获得基于净销售额的分级销售提成。双方高层均认为该款产品具有同类最优（Best-in-Class）的潜力，有望满足巨大的未满足的临床需求，为多种癌症患者提供潜在治疗方案。02案例二转让方：康诺亚(子公司：KYM BiosciencesInc)受让方：阿斯利康交易项目：CMG901（ADC）靶点：Claudin 18.2适应证：肿瘤项目阶段：Ia期交易金额：11.88亿美元2023年2月23日，康诺亚发布公告称，公司非全资附属公司KYM Biosciences Inc.（KYM）与AstraZeneca签订全球独家许可协议。AstraZeneca将获得CMG901的研究、开发、注册、生产及商业化的独家全球许可。根据协议，KYM将获得6300美元的预付款，并在达成若干开发、监管及商业里程碑之后，收取最多11.25亿美元的潜在付款，以及基于净销售额的分级特许权使用费。据了解，CMG901是一款靶向Claudin 18.2的ADC，是首个在中国及美国均取得临床试验申请许可的同靶点ADC，Linker采用可裂解的连接子，payload为甲基澳瑞他汀E (MMAE)。该产品在2022年4月获美国FDA孤儿药资格认定及快速通道资格，用于复发/难治性胃癌及胃食管结合部腺癌。项目目前处于临床I期，并于2023年1月公布了Ia期的剂量递增试验的最新结果。03案例三转让方：高光制药受让方：Biohaven交易项目：BHV-8000（TLL-041）靶点：TYK2/JAK1适应证：神经系统疾病项目阶段：I期交易金额：9.7亿美元2023年3月22日，Biohaven与杭州高光制药共同宣布，达成在大中华区以外地区的独家开发与许可协议，Biohaven拟在上述地区引进TLL-041。根据协议，Biohaven将支付2000万美元首付款（现金+股票）、9.5亿美元里程碑及分级特许权使用费。Biohaven将在2023年启动临床I期，双方在全球范围内协作进行临床开发。TLL-041是全球首个具有通过血脑屏障能力的高选择性双靶点TYK2/JAK1抑制剂。TYK2和JAK1是处理一系列促炎症细胞因子的STAT通路调节剂;抑制这些激酶可能在减少神经系统疾病的炎症方面发挥重要作用，包括帕金森病、阿尔茨海默病、肌萎缩性侧索硬化症和多发性硬化症。与选择性较低或无选择性的JAK抑制剂相比，TLL-041的高选择性有可能提供更好的安全性。Biohaven公司CEO Vlad Coric博士认为TLL-041通过血脑屏障的能力及对TYK2/JAK1双重抑制作用，使之具有独特和极具吸引力的潜力。公司将会积极推动该项目进入临床，发掘神经免疫调节的潜力，以治疗新疗法的严重神经系统疾病。04案例四转让方：信诺维受让方：AmMax Bio交易项目：实体瘤ADC项目靶点：未披露适应证：肿瘤项目阶段：临床前交易金额：8.71亿美元根据信诺维官网发布的消息，双方在2023年1月10日就一款用于实体瘤治疗的ADC项目达成合作，把除大中华区以外的研发和商业化权益独家转让给AmMax Bio。该ADC采用新一代的linker、payload和抗体，在多种动物模型中展现优越的抗肿瘤活性。临床前的疗效和毒理研究表明该ADC具有更宽的治疗窗口，其他数据表明其临床疗效和安全性将比同类ADC有大幅提升。根据协议条款，信诺维将会在协议执行后获得8.71亿美元的付款，包含首付款、开发、注册及销售里程碑。AmMax Bio的CEO Larry Hsu博士表明，该项目将会在2023年下半年推进到临床阶段。差异化布局助力困境突围随着创新药细分赛道的热烈竞争，新药研发赛道变得越发拥挤。近两年ADC和双抗药物无疑是继CAR-T治疗最火的两大赛道，但靶点同质化严重，创新药想要创新破圈尤为困难。产品研发的成功与否，差异化的研发和突出的优势是一个重要的因素。迈威生物瞄准罕见病（铁稳态相关疾病）进行单抗研发，有望获得孤儿药资格；百奥赛图的双靶点“PD-1xCD40”双抗药物，具有独特的作用机理并获得了出色的临床前数据，让其临床潜力值得期待；值得注意的是，今年5月10日，明济生物研发的抗PD-L1xCD40双特异性抗体（实体瘤）也获批Ⅰ期临床试验；环状RNA技术在细胞疗法领域的巨大潜力，先声集团（先博生物）购入了Orna Therapeutics的环状RNA疗法。环状RNA技术虽然在细胞疗法中的应用仍处于早期阶段，并且面临较多待解决的技术难点和挑战，但其吸引了大量的资本投入。自2021年以来，CircRNA领域已获得超过8亿美元融资。风险与收益并存，尽管存在风险，但成功开发出可靠的环状RNA细胞疗法将带来巨大的回报。可能先声集团也正是看中了环状RNA技术在细胞疗法中的潜在价值和前景，这项战略举措可能表明先声集团在细胞疗法领域的长期战略规划，并希望通过此举扩大其在细胞疗法领域的竞争优势。然而，需要注意的是，环状RNA技术仍需要进一步的研究和发展，以解决其面临的技术挑战和确保其安全性和有效性。05案例五转让方：迈威生物受让方：DISC MEDICINE. INC.交易项目：9MW3011（单抗）靶点：未披露适应证：铁稳态相关疾病项目阶段：IND交易金额：4.125亿美元2023年1月20日，迈威医药发布公告称，公司美国子公司（迈威（美国））与DISC MEDICINE, INC.（DISC）拟就在研品种9MW3011签署《独家许可协议》。迈威（美国）将许可DISC在除大中华区和东南亚以外的所有区域内的独家开发、生产和商业化及其他方式开发9MW3011的权利。根据协议，DISC需向迈威（美国）支付一次性不可退还的首付款1000万美元，高达4.125亿美元的首付款及里程碑付款，以及基于净销售额的最高近2位数百分比的特许使用费。9MW3011是迈威医药在美国自主研发的创新靶点单克隆抗体，靶点主要表达在肝细胞膜表面。通过特异性结合，可以上调肝细胞表达铁调素（Hepcidin）的水平，抑制铁的吸收和释放，降低血清铁水平，从而调节体内的铁稳态。该产品已获得NMPA和美国FDA批准开展临床试验。适应证包括多种罕见病，如β-地中海贫血、真性红细胞增多症等与铁稳态相关的疾病。目前，相关适应证尚无成熟有效的大分子治疗药物，因此9MW3011有望在未来获得孤儿药资格，并且有望成为全球范围内首个调节体内铁稳态的大分子药物。06案例六转让方：百奥赛图 (子公司：祐和医药)受让方：微芯生物(子公司：微芯新域)交易项目：YH008（双抗）靶点：PD-1xCD40适应证：肿瘤项目阶段：IND交易金额：5.96亿人民币2023年2月27日，百奥赛图发布公告称，公司子公司祐和医药与微芯生物就YH008达成在大中华区（中国大陆、香港、澳门和台湾）的临床开发及商业化授权的协议。根据协议，微芯生物将通过子公司微芯新域向祐和医药支付4000万人民币首付款，不超过3.6亿元的潜在研发里程碑付款，不超过1.96亿人民币的潜在销售里程碑付款，以及基于净销售额的特权使用费。据了解，YH008是一款百奥赛图自主研发并拥有知识产权的免疫疗法双抗，IND申请已在中国获得NMPA受理，并在美国获得FDA批准。YH008作用靶点为PD-1xCD40，该靶点组合为全球首创。体外及体内实验结果显示，YH008可在富含肿瘤特异性T细胞的肿瘤微环境中条件性激活免疫通路，从而避免全身非特异性的激活。此外，通过采用无效应功能的IgG1亚型，避免由Fc受体介导的非特异性激活。在作用机理上，YH008兼顾了免疫激活和免疫抑制阻断两条通路，并在疗效上产生协同。独特的作用机理和出色的临床前数据，让其临床潜力值得期待。微芯新域抗体早期研发中心负责人刘斌博士表示，该产品与微芯新域在研产品具有很好的互补性，公司将会迅速将该产品推进到临床阶段。07案例七转让方：Orna Therapeutics受让方：先声集团（先博生物）交易项目：isCAR技术的某些项目靶点：CD19适应证：肿瘤项目阶段：临床前交易金额：未披露2023年1月5 日，先博生物（先声集团子公司）与Orna Therapeutics共同宣布，就基于环状RNA技术开发的细胞疗法达成合作，包括Orna Therapeutics拥有的主要资产ORN-101（靶向CD19原位CAR）。Orna Therapeutics将在中国开展研究者发起的临床，以加速特定项目在患者中的验证。协议执行后，Orna Therapeutics将收到先博生物的预付款，并有望获得开发、注册和销售里程碑及后续的销售分成。Orna专有的 isCAR 技术包括两个部分，分别是表达嵌合抗原受体的环状RNA和定制的脂质纳米颗粒（LNP）。据了解，环状RNA具有更高的稳定性，Orna 的环状RNA-LNP平台被认为具有跨适应证的潜力，包括肿瘤、遗传病和传染病。Orna 的主导项目 ORN-101 是一种原位 CAR 疗法，旨在改变患者体内的免疫细胞。通过LNP将环状RNA封装在其中，可以避免患者淋巴细胞耗竭并实现更可靠的剂量控制。临床前数据证明该疗法可以抑制和根除动物模型中的肿瘤，表明该疗法有望超越细胞疗法。聚焦未满足的临床需求创新的生物技术如ADC（抗体药物偶联物）和双特异性抗体（BiTE）等药物在某种程度上对小分子药物的研发构成了一定的冲击，但小分子药物研发技术相对较为成熟，风险相对较小，仍然有其独特的优势和发展空间，在药物研发领域仍然扮演着重要的角色。尤其是在非肿瘤药物领域，小分子药物仍然是主要的研发方向和治疗选择。在2023年Q1的BD交易中，小分子创新药物占1/3，而在初期阶段的10个项目中占近一半。要使小分子创新药物在BD交易中脱颖而出，可能需要更明确的治疗优势、更明确的市场机会，包括目标疾病的患病率、市场规模、竞争环境、市场增长预测等方面的数据支持以及优秀的临床阶段和数据支持。08案例八转让方：和誉医药受让方：艾力斯交易项目：ABK3376（小分子）靶点：EGFR适应证：肿瘤项目阶段：临床前交易金额：1.879亿美元2023年3月1日，上海和誉生物医药与上海艾力斯医药就新一代小分子表皮生长因子受体酪氨酸激酶抑制剂（EGFR-TKI）ABK3376签订合作协议。前者授予后者在中国区域（中国大陆、香港、澳门及台湾地区）研究、开发、制造、使用及销售的独家许可。根据上海艾力斯医药发布的公告，公司有权在达到行权条件时选择行使海外权益，授权区域将扩大到全球范围。而针对此笔授权，上海艾力斯需支付最高不超过1.879亿美元的付款，包含首付款、开发及销售里程碑付款及基于净销售的许可提成费。据了解，ABK3376是和誉医药自主研发的、具有知识产权的高选择性、可入脑的新一代EGFR口服小分子抑制剂，具有强大的肿瘤细胞抑制和杀伤活性，在实验模型体内显示出抑制并缩小肿瘤的能力。该产品不仅可以高效抑制三代EGFR-TKI耐药后产生的C797S突变，还具有高效穿透血脑屏障的特性。ABK3376目前处于临床前开发阶段，临床前研究显示无论单药还是与伏美替尼联用，都取得了积极结果，有望成为精准治疗伴有 EGFRC797S 耐药突变非小细胞肺癌的新一代靶向治疗药物。09案例九转让方：SCOHIA PHARMA,Inc受让方：华东医药(子公司：中美华东)交易项目：SCO-094及其衍生产品靶点：GIPR&GLP-1R适应证：糖尿病、肥胖和NASH项目阶段：I期交易金额：未披露2023年2月22日，华东医药全资子公司中美华东与日本SCOHIA PHARMA, Inc共同宣布，将就GIPR&GLP-1R双激动剂SCO-094及其衍生产品在全球范围内扩大战略合作。早在2021年6月，双方便签订了产品独家许可协议，中美华东获得SCO-094在中国、韩国、澳大利亚等25个亚太国家和地区的独家开发、生产和商业化权益。此次签署的新合作协议，将合作范围进一步扩大，中美华东将获得SCO-094及其衍生产品在全球（包括日本）的独家开发、生产及商业化权益。SCO-094是一种GIPR&GLP-1R双靶点激动剂，通过选择性激活GLP-1（Glucagon-like peptide-1，胰高血糖素样肽-1）受体和GIP（Glucose-dependent insulinotropic polypeptide，葡萄糖依赖性促胰岛素多肽）受体，激动下游通路，产生控糖、减重等生物学效应。研究表明，SCO-094具有较强靶点结合活性和细胞生物活性。动物模型显示其具有强大的降糖效果，减肥及改善肝功能、降低甘油三酯和抑制肝脏脂肪变性作用。临床前数据支持其对糖尿病、减肥、NASH等适应证的临床开发。该项目目前已在英国开展Ⅰ期临床试验。10案例十转让方：PhaseBio受让方：箕星药业交易项目：PB6440（小分子）靶点：醛固酮合成酶适应证：高血压项目阶段：临床前交易金额：未披露根据箕星药业官网公布的消息，箕星药业通过购买获得了PhaseBio药业PB6440的全球权益。PB6440目前处于临床前阶段，是新一代高选择性醛固酮合成酶抑制剂。主要针对包括高血压在内的心血管领域未满足的临床需求。因为直接作用于醛固酮合成酶，因而避免了由于非特异性盐皮质激素受体阻断而导致的副作用。这种高活性和高选择性特征使其具有BIC药物的潜力。虽然全球尚未有醛固酮合成酶抑制剂药物用于高血压领域，中国也没有开展这类药物的临床研究，箕星药业CEO牟艳萍女士依然表示将会积极推动PB6440的临床开发。结语通过梳理以上项目，我们发现每一个成功交易的项目，都有它独特的优势或者差异化的方向。即使拥挤如Claudin 18.2，现在依然有成功合作的机会。根据药时代BD团队的观察，这些差异化的方向可以被归纳为靶向性、选择性是否足够好，能否在安全性方面为患者带来获益；速度是否领先，研发进度排在世界第一梯队；数据是否优秀，能否显示出BIC的潜力；作用机制是否独特，能否带来更优的临床效果等。我们药时代BD团队也在不断寻找和推广优质项目，在2023年第一季度，我们也代理了如下临床前和临床I期的项目：如果您对以上项目感兴趣或者有项目买卖需求，欢迎前来咨询和洽谈合作。我们将以最快的速度帮您推进项目合作。联系人：Richard电话：13817995747微信：balance002邮箱：bd@drugtimes.cn封面图来源：pixabay2023年1月1日至今，~20笔中国新药出海交易横空出世，总金额高达上百亿美元！一时间，喜讯频传，令行业、企业和每一位同药振奋！曾经提出“百靶竞技，百炼创新，百家上市，百药争先”的药时代团队再一次真切感受到：中国新药研发正当时！中国新药出海正当时！中国新药BD正当时！在这个BD如火如的好时代里，企业对于BD的要求不断提高，如何与时俱进，学习最新BD知识，掌握最新BD技能，成为摆在每一位BD同药面前的一个实际而迫切的问题。为了帮助企业培养优秀的BD高级人才，为了帮助BD人才成长进步，药时代学苑全力打造创新药BD高级研讨会，致力于成为中国制药界ZUI有实力和活力的学习平台。点击这里，报名参加，一起快乐学习！

Two ongoing Phase 2 studies of bitopertin in EPP; initial safety and efficacy data from open-label BEACON trial to be presented at the European Hematology Association (EHA) Congress in June 2023; topline data from both BEACON and AURORA studies expected by end of 2023Two separate, ongoing Phase 1b/2 studies of DISC-0974 in patients with anemia of chronic kidney disease who are not receiving dialysis (NDD-CKD) and also in patients with myelofibrosis and anemia; interim data expected by end of 2023In-licensed anti-TMPRSS6 monoclonal antibodies from Mabwell Therapeutics; Phase 1 trial expected to start 2H 2023Completed $62.5 million financing led by Bain Capital Life Sciences WATERTOWN, Mass., May 15, 2023 (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 reported financial results for the first quarter ending March 31, 2023 and provided an update on recent program and corporate developments. “We have had an excellent start to 2023 with each of our development programs on-track; multiple clinical studies ongoing, including new indications such as CKD anemia and Diamond-Blackfan anemia; and growing our pipeline by in-licensing a third, Phase-1 ready program,” said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc. “We are positioned to deliver a series of important catalysts across our portfolio through the rest of 2023 and into next year, beginning with the presentation of initial data from the BEACON study at EHA.” Recent Business Highlights and Upcoming Milestones: Bitopertin: GlyT1 Inhibitor (Heme Synthesis Modulator) 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. Continued enrollment of BEACON, an open-label Phase 2 clinical study of bitopertin in patients with EPP and X-linked protoporphyria (XLP). Interim data will be presented at EHA Congress on June 9, 2023, with an accompanying management call at 7:30 am ET the same day.Disc expects topline data from BEACON by end of 2023. Continued enrollment for AURORA, a Phase 2 randomized, placebo-controlled clinical study of bitopertin in adults with EPP. Disc expects topline data from AURORA by end of 2023.Received a positive opinion on Orphan Designation for bitopertin for treatment of EPP from the European Committee for Orphan Medical Products in January 2023. Orphan Drug Designation was previously granted by the FDA in 2022.Announced a collaboration with NIH to study bitopertin in patients with Diamond-Blackfan Anemia in March 2023; the study is expected to initiate mid-year 2023. DISC-0974: Anti-Hemojuvelin Antibody (Hepcidin Suppression) DISC-0974 is an investigational anti-hemojuvelin monoclonal antibody (mAb) and is designed to suppress hepcidin production and increase serum iron levels in patients suffering from anemia of inflammation. Initiated and enrollment is ongoing for a Phase 1b/2 clinical study of patients with anemia of chronic kidney disease who are not receiving dialysis (NDD-CKD) in February 2023; interim data expected by end of 2023Continued enrollment in a Phase 1b/2 clinical study in MF patients with severe anemia on stable background therapy; interim data expected by end of 2023 MWTX-003: Anti-TMPRSS6 Antibody (Hepcidin Induction) MWTX-003 is an investigational, anti-TMPRSS6 (Transmembrane Serine Protease 6, also known as Matriptase-2) monoclonal antibody designed to increase hepcidin production and suppress serum iron. Entered into an exclusive licensing agreement with Mabwell Therapeutics in January 2023 to obtain an exclusive license to rights outside of Greater China for a portfolio of monoclonal antibodies targeting TMPRSS6 including a Phase 1-ready drug candidate, MWTX-003.The IND was accepted in November 2022 and Disc plans to initiate a Phase 1 study of MWTX-003 in healthy volunteers during the second half of 2023.Disc plans to develop MWTX-003 initially as a treatment for polycythemia vera as well as other indications. Corporate: Completed a $62.5 million registered direct offering of our common stock led by Bain Capital Life Sciences, with participation from Access Biotechnology and OrbiMed, in February 2023. First Quarter 2023 Financial Results: Cash Position: Cash and cash equivalents were $236.4 million as of March 31, 2023 compared to $194.6 million as of December 31, 2022. The increase was due to $62.5 million in gross proceeds from a registered direct offering led by Bain Capital Life Sciences in February 2023, as well as $15.0 million in gross proceeds from ATM offerings completed in Q1 2023. Disc expects its cash and cash equivalents to fund its operational plans into 2025.Research and Development Expenses: R&D expenses were $20.2 million for the quarter ending March 31, 2023, as compared to $7.8 million for the quarter ending March 31, 2022. The increase in R&D expenses were primarily driven by a one-time $10.0 million upfront payment under the Mabwell license agreement and the progression of Disc’s portfolio, including increased headcount, the advancement of DISC-0974 into an additional Phase 1b/2 clinical study, and bitopertin’s ongoing two Phase 2 clinical studies.General and Administrative Expenses: G&A expenses were $4.9 million for the quarter ending March 31, 2023, as compared to $2.1 million for the same period in 2022. The increase in G&A expenses was primarily due to increased headcount and legal and market research costs.Net Loss: The net loss was $22.8 million for the first quarter of 2023, as compared to $9.9 million for the first quarter of 2022. The increase was primarily due to higher operating costs in the current period to support the continued advancement of the Company’s pipeline. About Disc Medicine Disc Medicine (NASDAQ:IRON) 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. Available Information Disc announces material information to the public about the Company, its products and services, and other matters through a variety of means, including filings with the U.S. Securities and Exchange Commission (SEC), press releases, public conference calls, webcasts and the investor relations section of the Company website at ir.discmedicine.com in order to achieve broad, non-exclusionary distribution of information to the public and for complying with its disclosure obligations under Regulation FD. 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 its AURORA Phase 2 and BEACON Phase 2 clinical studies of bitopertin, and anticipated study of bitopertin in Diamond-Blackfan Anemia, its Phase 1b/2 clinical study of DISC-0974 in NDD-CKD patients with anemia, its anticipated Phase 1 study of MWTX-003 and potential development of MWTX-003 as a treatment for polycythemia vera and other indications, projected timelines for the initiation and completion of its clinical trials, anticipated timing of release of data, and other clinical activities; Disc’s business plans and objectives; and Disc’s beliefs about operating expenses and that it will have capital to fund Disc into 2025. 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 other risks and uncertainties described in the “Risk Factors” section of our Annual Report on Form 10-K for the year ended December 31, 2022 and other documents filed by Disc from time to time with the Securities and Exchange Commission, as well as discussions of potential risks, uncertainties, and other important factors in Disc’s subsequent filings with the Securities and Exchange Commission. 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. DISC MEDICINE, INC. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (In thousands, except share and per share amounts) (Unaudited) ​Three Ended March 31, ​ 2023 ​ 2022 Operating expenses:​ ​ Research and development​$20,180 ​$7,821 General and administrative​ 4,945 ​ 2,139 Total operating expenses​ 25,125 ​ 9,960 Loss from operations​ (25,125)​ (9,960) Other income (expense), net 2,367 ​ 107 Income tax expense​ (23)​ — Net loss​$(22,781)​$(9,853) Weighted-average common shares outstanding-basic and diluted​ 18,954,914 ​ 923,750 Net loss per share-basic and diluted​$(1.20)​$(10.67) DISC MEDICINE, INC. CONDENSED CONSOLIDATED BALANCE SHEETS (In thousands) (Unaudited) ​March 31, December 31, ​2023​2022 Assets​ ​ Cash and cash equivalents​$236,422​$194,611 Other current assets​ 5,716​ 3,880 Total current assets​ 242,138​ 198,491 Non-current assets​ 1,642 1,714 Total assets​$243,780​$200,205 Liabilities and Stockholders’ Equity​ ​ Current liabilities​$9,980​$22,578 Non-current liabilities​ 945​ 1,027 Total liabilities​ 10,925​ 23,605 Total stockholders’ equity​ 232,855​ 176,600 Total liabilities and stockholders’ equity​$243,780​$200,205 Media Contact Peg RusconiVerge Scientific Communicationsprusconi@vergescientific.com Investor Relations Contact Suzanne Messere Stern Investor Relationssuzanne.messere@sternir.com

Mabwell (688062.SH), an innovative biopharmaceutical company with the whole industry chain layout, announced its Nectin-4 targeted site-specific ADC asset (R&D code: 9MW2821) demonstrated promising clinical data. The preliminary data show positive therapeutic signals in solid tumors, and good safety profile at the recommended phase II dose (RP2D). Developed with site specific conjugate technology, 9MW2821 is China's first, global second Nectin-4 targeted ADC approved for clinical study. The multiple ongoing clinical studies include more than 10 different solid tumors, evaluate safety, tolerability, pharmacokinetics, and preliminary antitumor activity. The preliminary data show that under RP2D, among 12 UC (Urothelial Carcinoma) patients the ORR (Objective Response Rate) was 50% and DCR (Disease Control Rate) was 100%, and among 6 CC (Cervical Carcinoma) patients, the ORR was 50% and DCR was 100%. Mabwell is promoting the enrollment of multiple cohorts of UC, CC, prostate cancer, HER-2 negative breast cancer, and non-small cell lung cancer. Details will be published in upcoming academic meetings. Mabwell has also achieved several breakthroughs in new ADC technology platform, optimizing ADC platform by developing IDDC™, a new generation ADC site-specific conjugate technology platform, which is composed of multiple systematized core patented technologies including site-specific conjugate process DARfinity™, special designed linker IDconnect™, novel payload Mtoxin™, and conditional release structure LysOnly™. Developed Based on mentioned systematic patent technologies, the next generation ADCs have better structural homogeneity, quality stability, pharmacodynamics and tolerability, as well as dominant advantage of differentiation. At present, the advantage of IDDC™ platform has been validated in several products under development. Clinical trial application for Trop-2 targeted ADC (R&D code: 9MW2921) has been accepted by NMPA. Clinical trial application for B7-H3 targeted ADC (R&D code: 7MW3711) is expected to be submitted soon. By continuously advancing development of multiple ADC candidates, it is expected that Mabwell will have 3 to 5 ADC products in clinical stage by 2024. Mabwell has established complete industry chain for ADC, including early discovery, pre-clinical development and pilot scale production. The large-scale commercialized site is expected to be ready for production trial run at Q2, 2023. About Mabwell Mabwell (688062.SH), an innovation-driven biopharmaceutical company, has the whole industrial chain of R&D, manufacturing, and commercialization. We provide more effective and accessible therapy and innovative medicines to fulfill global medical needs. Since 2017, an advanced R&D system which covers target discovery, early discovery, druggability, preclinical, clinical research and manufacturing transformation was established. Mabwell has 17 pipeline products in different R&D stages based on a world-class and state-of-the-art R&D engine, including 13 novel drug candidates and 4 biosimilars. We focus on the therapeutic areas of auto-immune diseases, oncology, metabolic disorders, ophthalmologic diseases and infectious diseases, etc. Of these, 2 products have been approved and commercialized, 1 product has been filed for MA approval, 3 products are in pivotal trials. We have also undertaken 1 national major scientific and technological special project for "Significant New Drugs Development", 2 projects for National Key R&D Programmes, and several provincial and municipal science and technological innovation projects. Mabwell's Taizhou factory possesses robust in-house manufacturing capability compliant with international GMP standards regulated by the NMPA, FDA and EMA, and has passed the EU QP Audit. The large-scale manufacturing base located in Shanghai is under construction. Our mission is "Explore Life, Benefit Health" and our vision is "Innovation, from ideas to reality". For more information, please visit www.mabwell.com. SOURCE: Mabwell