Bitopertin

10月，全球及中国医药监管体系政策的发布，持续推动临床研发与审评审批效率提升：国际上，英国MHRA开发知识中心和GMP合规检查器两个AI工具，加速临床试验审评；美国FDA授予首批9张“局长国家优先审评券”，审评周期缩短至1-2个月。国内方面，“十五五”规划建议通过 “制度协同、技术赋能、基层强基”，对医药、医疗、医保三大领域形成差异化影响，且三者相互支撑、形成闭环；国务院发布《生物医学新技术临床研究和临床转化应用管理条例》，从顶层设计强化临床研究规范；工信部等七部门发布服务型制造创新发展实施方案，推动先进制造业与现代服务业融合；国家药监局相关部门和CDE发布多项政策及指导原则，涉及儿童用药研发、药品质量抽查检验、药物临床试验质量管理等方面，进一步明确技术要求、优化审评路径；此外，国际医药创新公园规划方案在北京亦庄正式发布，将助力医药创新发展。 10月7日，MHRA发文称其通过重大流程改革和人工智能（AI）技术的应用，将临床试验审批时间缩短了一半以上。根据《英国临床药理学杂志》（British Journal of Clinical Pharmacology, BJCP）最新研究，MHRA的IND审批时间从平均91天缩短至41天。过去一年，超过4600项IND申请中有99%在法定时限内完成，且大多数审批远早于目标时间。低风险研究的快速审批通道可在14天内完成审批。 原文链接： https://www.gov.uk/government/news/uk-clinical-trial-approval-times-twice-as-fast-with-ai-and-reforms 10月16日，FDA发布新闻稿，宣布首批“局长国家优先审评券”（CNPV）授予Pergoveris、Teplizumab、Cytisinicline、DB-OTO、Cenegermin-bkbj、RMC-6236、Bitopertin、Ketamine、Augmentin XR等9个药品。FDA将通过一个专门的多学科团队对候选药物进行快速审评，采用团队审评的方式，与申办者频繁互动以澄清问题。当各部门完成各自审评工作后，FDA将召集团队进行为期一天的“肿瘤委员会风格”（tumor board style）的会议，集中讨论审评结果。 原文链接： https://www.fda.gov/news-events/press-announcements/fda-awards-first-ever-national-priority-vouchers-nine-sponsors 10月20—23日，中国共产党第二十届中央委员会第四次全体会议审议通过了《中共中央关于制定国民经济和社会发展第十五个五年规划的建议》。其中第 42 条通过 “制度协同、技术赋能、基层强基”，对医药、医疗、医保三大领域形成差异化影响，且三者相互支撑、形成闭环。建议还提出，前瞻布局未来产业，推动生物制造、脑机接口等成为新的经济增长点；并明确完善新型举国体制，采取超常规措施，全链条推动生物制造等重点领域关键核心技术攻坚战取得决定性突破。 原文链接： https://www.gov.cn/zhengce/202510/content_7046050.htm 10月10日，国务院公布《生物医学新技术临床研究和临床转化应用管理条例》（国务院令[2025]第818号）将于2026年5月1日生效。该条例对生物医学新技术的定义、临床研究的备案程序与实施、后续临床转化的申请与应用等事项进行了系统性规范，标志着细胞与基因治疗等生物医学技术的监管从相对模糊且缺乏明确商业化的路径阶段逐渐向清晰规范化方向发展。 原文链接： https://www.gov.cn/zhengce/content/202510/content_7043790.htm 10月11日，工信部等七部门发布《深入推动服务型制造创新发展实施方案（2025—2028年）》。该方案明确，到2028年，打造服务型制造升级版，完成20项标准制定，打造50个领军品牌，建设100个创新发展高地，服务型制造典型模式广泛普及、新模式不断涌现。方案部署了7项主要任务，包括加强关键共性技术攻关和模式创新、培育壮大重点生产性服务业、分类推进服务型制造模式推广应用、推进标准体系建设、激发经营主体发展活力、打造服务型制造创新发展高地、夯实服务型制造发展底座。 原文链接： https://www.miit.gov.cn/zwgk/zcwj/wjfb/tz/art/2025/art_347dfcaa34b147458d9e52f0e9f238e1.html 10月11日，CDE发布《儿童用药研发常见/共性问题及一般性答复》。该文件对通过改进剂型、工艺、给药途径等，提升药物的安全性、有效性及儿童用药依从性的改良型新药，提出更确定的支持信号。 原文链接： https://www.cde.org.cn/main/news/viewInfoCommon/bccd09f6a6a311ffb5827be1e0389df0 10月28日，国家药监局综合司发布《药品质量抽查检验管理办法》（征求意见稿）。现行《药品质量抽查检验管理办法》为2019年8月发布的，本次修订主要为了匹配2019年《药品管理法》修订的新要求，新增了“网络抽检”独立章节，并将“复验”章节从“监督管理”中独立。 原文链接： https://www.nmpa.gov.cn/xxgk/zhqyj/zhqyjyp/20251028103307152.html 10月28日，国家药监局综合司公开征求《药物临床试验质量管理规范（修订稿征求意见稿）》意见，旨在保证药物临床试验过程规范，保护试验参与者的权益、安全和福祉，确保数据和结果科学、真实、可靠。该意见稿与2020版GCP相比本次修订保留了总则、伦理审查委员会、主要研究者和药物临床试验机构、申办者、附则5个章节，增加了数据治理1个章节。考虑到全文实施E6（R3）中文版，只保留我国特有的术语及其定义，删除试验方案、研究者手册、必备文件管理3个章节。 原文链接： https://www.nmpa.gov.cn/xxgk/zhqyj/zhqyjyp/20251028164306197.html 10月28日，CDE发布《主方案设计的药物临床试验指导原则（征求意见稿）》，旨在指导申办者科学、规范地开展采用主方案设计的药物临床试验，提高药物研发效率，加速创新药物上市，保障受试者权益。 原文链接： https://www.cde.org.cn/main/news/viewInfoCommon/a67f5834244b8671399c14279434ae42 10月28日，CDE发布《化学药品批准后药学变更管理方案技术指导原则（试行）》，该指导原则把将ICH Q12 的 PACMP（Post Approval Change Management Protocol） 真正落到中国化学药领域：企业可以用预先获批的“变更方案”来实施未来的特定药学变更，在满足预先约定的研究验证与可接受标准时，有机会降低变更管理类别并缩短审评周期。 原文链接： https://www.cde.org.cn/main/news/viewInfoCommon/0889d723de12de0bb82f616ef34376e3 10月25日，在2025国际生物医药产业创新北京论坛全体大会上，北京市发布国际医药创新公园（BioPark）规划方案，提出高标准规划总部集聚区、医工融合区等四大产业功能区，构筑高端产业链体系，打造全球医药健康创新合作枢纽。BioPark已吸引包括礼来、辉瑞、拜耳、美敦力等跨国巨头，覆盖疫苗、创新药、高端医疗器械等多个赛道，逐步构建“全球+本土”协同生态圈。例如，2024年，礼来中国医学创新中心与美国本土以外的首家创新孵化器落地北京亦庄的BioPark。 点击查看原文链接

Submitted a New Drug Application (NDA) for accelerated approval of bitopertin in erythropoietic protoporphyria (EPP) on September 29, 2025; Awarded the FDA Commissioner’s National Priority Voucher (CNPV), which is designed to shorten the expected NDA review period to 1-2 months from NDA acceptance Acceleration of ongoing efforts to support the potential earlier approval and commercialization of bitopertin for EPP in the US, expected in late 2025 or early 2026 Initial data from Phase 2 study of DISC-0974 in patients with anemia of myelofibrosis (MF) to be presented at the American Society of Hematology (ASH) conference in December Progressing ongoing Phase 2 study of DISC-3405 in polycythemia vera (PV) and initiated a Phase 1b study of DISC-3405 in sickle cell disease (SCD) in October Strong financial position ending Q3 with approximately $616 million in cash, cash equivalents, and marketable securities; completed a public offering in October 2025, with net proceeds of approximately $211M expected to extend cash runway into 2029 WATERTOWN, Mass., Nov. 06, 2025 (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 third quarter ended September 30, 2025, and provided a review of recent program and corporate developments. “We are incredibly proud of the progress across our entire pipeline this quarter, most notably around our NDA submission for bitopertin in EPP at the end of September and subsequent receipt of the CNPV,” said John Quisel, J.D., Ph.D., Chief Executive Officer and President of Disc. “Achieving this milestone is a strong testament to our team’s commitment to execution and dedication to bringing a potential new treatment option to the EPP patient community. We continue to work with the Agency on their review of our application, and we are furthering our commercial infrastructure in preparation for a potential launch.” “Beyond bitopertin, our broader pipeline is progressing well, as we expect new data from the Phase 2 trial of DISC-0974 in MF anemia by the end of the year and data from studies of DISC-3405 in PV and SCD next year. With a strong balance sheet providing cash runway into 2029, we are well-positioned to prepare for the anticipated launch of bitopertin and ultimately advancing towards our goal of delivering new treatment options to patients suffering from hematological diseases.” Recent Highlights and Anticipated Milestones: Bitopertin: GlyT1 Inhibitor (Heme Synthesis Modulator) Submitted NDA for bitopertin in EPP seeking priority review and accelerated approval with reduction in protoporphyrin IX (PPIX) as the surrogate endpoint for approval supported by clinical results from BEACON and AURORA Phase 2 trials Received the CNPV, which is designed to shorten the NDA review process to 1-2 months from NDA acceptance Accelerating activities to support a potential US approval and launch in late 2025 or early 2026 Progressing confirmatory Phase 3 APOLLO clinical trial of bitopertin in adults and adolescents with EPP DISC-0974: Anti-Hemojuvelin Antibody (Hepcidin Suppression) Initial data from Phase 2 RALLY-MF trial of DISC-0974 in patients with anemia of myelofibrosis (MF) to be presented at ASH Annual Meeting in December, with topline data expected in 2026 Data from recently completed multiple-dose cohorts of Phase 1b study of DISC-0974 in patients with anemia of NDD-CKD to be presented at ASN Kidney Week DISC-0974 was generally well-tolerated with substantial decreases in hepcidin, increases in iron, and improvements in markers of erythropoiesis Meaningful hemoglobin increases observed in only a subset of patients were in part driven by those with higher baseline erythropoietin (EPO) levels Disc is assessing options for the program based on full analysis of the data Phase 2 study in patients with inflammatory bowel disease (IBD) and anemia anticipated to begin in Q1 2026 DISC-0974 was generally well-tolerated with substantial decreases in hepcidin, increases in iron, and improvements in markers of erythropoiesis Meaningful hemoglobin increases observed in only a subset of patients were in part driven by those with higher baseline erythropoietin (EPO) levels Disc is assessing options for the program based on full analysis of the data DISC-3405: Anti-TMPRSS6 Antibody (Hepcidin Induction) Progressing ongoing Phase 2 study of DISC-3405 in patients with PV with initial data expected in 2026 Initiated Phase 1b study of DISC-3405 in patients with SCD in October 2025 with initial data expected in 2026 Third Quarter 2025 Financial Results: Cash Position: Cash, cash equivalents, and marketable securities were $615.9 million as of September 30, 2025. In October 2025, Disc completed a public offering with net proceeds of approximately $211 million, extending cash runway into 2029. Research and Development Expenses: R&D expenses were $50.3 million for the three months ended September 30, 2025, as compared to $24.7 million for the three months ended September 30, 2024. The increase in R&D expenses was primarily driven by the progression of Disc’s portfolio, including bitopertin’s clinical studies and drug manufacturing, the advancement of the DISC-0974 program, and increased headcount, as well as a payment of a $10.0 million milestone upon first administration to a patient in the Phase 2 trial of DISC-3405. Selling, General and Administrative Expenses: SG&A expenses were $17.4 million for the three months ended September 30, 2025, as compared to $8.2 million for the three months ended September 30, 2024. The increase in SG&A expenses was primarily due to increased headcount including establishing infrastructure to support potential commercialization. Net Loss: Net loss was $62.3 million for the three months ended September 30, 2025, as compared to $26.6 million for the three months ended September 30, 2024. The increase was primarily due to higher operating costs in the current period to support the continued advancement of our 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 the next stages of its development programs for bitopertin, DISC-0974 and DISC-3405, including projected timelines for the initiation and completion of its clinical trials, anticipated timing of release of data, and other clinical activities; the registrational pathway for bitopertin, including the potential for accelerated approval, benefits of the CNPV, expected review period and the timing of approval, if granted; anticipated discussions with regulatory agencies; ongoing preparations for the potential launch of bitopertin; and the strength of its financial position and its anticipated cash runway. 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, 2024, 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 Rusconi Deerfield Group peg.rusconi@deerfieldgroup.com Investor Relations Contact Christina Tartaglia Precision AQ christina.tartaglia@precisionaq.com