Tovorafenib

EMERYVILLE, Calif., Sept. 22, 2025 (GLOBE NEWSWIRE) -- XOMA Royalty Corporation (Nasdaq: XOMA) today announced its Board of Directors has authorized the following cash dividends to holders of XOMA Royalty’s Series A and Series B Cumulative Preferred Stock: Holders of the 8.625% Series A Cumulative Perpetual Preferred Stock (Nasdaq: XOMAP) shall receive a cash dividend equal to $0.53906 per share. Holders of depositary shares, each representing 1/1000 of a share of XOMA Royalty’s 8.375% Series B Cumulative Perpetual Preferred Stock (Nasdaq: XOMAO), shall receive a cash dividend equal to $0.52344 per depositary share. The preferred dividends will be paid on or about October 15, 2025, to respective holders of record at the close of business on October 3, 2025. About XOMA Royalty CorporationXOMA Royalty is a biotechnology royalty aggregator playing a distinctive role in helping biotech companies achieve their goal of improving human health. XOMA Royalty acquires the potential future economics associated with pre-commercial and commercial therapeutic candidates that have been licensed to pharmaceutical or biotechnology companies. When XOMA Royalty acquires the future economics, the sellers receive non-dilutive, non-recourse funding they can use to advance their internal drug candidate(s) or for general corporate purposes. XOMA Royalty has an extensive and growing portfolio of assets (asset defined as the right to receive potential future economics associated with the advancement of an underlying therapeutic candidate). For more information about XOMA Royalty and its portfolio, please visit www.xoma.com or follow XOMA Royalty Corporation on LinkedIn. EXPLANATORY NOTE: Any references to “portfolio” in this press release refer strictly to milestone and/or royalty rights associated with a basket of drug products in development. Any references to “assets” in this press release refer strictly to milestone and/or royalty rights associated with individual drug products in development. As of the date of this press release, the commercial assets in XOMA Royalty’s milestone and royalty portfolio are VABYSMO® (faricimab-svoa), OJEMDA™ (tovorafenib), MIPLYFFA™ (arimoclomol), XACIATO™ (clindamycin phosphate) vaginal gel 2%, IXINITY® [coagulation factor IX (recombinant)], and DSUVIA® (sufentanil sublingual tablet). All other assets in the milestone and royalty portfolio are investigational compounds. Efficacy and safety have not been established. There is no guarantee that any of the investigational compounds will become commercially available. Investor Contact Juliane Snowden XOMA Royalty Corporation +1-646-438-9754 juilane.snowden@xoma.comXOMA Royalty Media ContactKathy VincentKV Consulting & Managementkathy@kathyvincent.com

编者按：低级别胶质瘤（LGG）是一类起源于中枢神经系统的原发性脑瘤。根据世界卫生组织（WHO）统计，儿童脑瘤是继白血病之后第二常见的儿童恶性肿瘤类型，其中低级别胶质瘤占据较高比例。虽然这类肿瘤生长相对缓慢，但仍会对患者的神经功能、生活质量和长期生存带来重大影响。手术、放疗和化疗是主要治疗手段，但复发风险和治疗相关副作用仍是临床难题。近年来，靶向治疗、免疫治疗等新型疗法不断取得突破，为患者带来更多希望。突破背后，是科学家、患者群体以及产业的持续耕耘与协作。长期以来，药明康德始终支持创新儿童癌症疗法的研发，依托“一体化、端到端”的CRDMO模式，助力合作伙伴将科学突破高效转化为创新药物。在这个儿童癌症宣传月（Childhood Cancer Awareness Month），本文将带大家走进一位低级别胶质瘤儿童患者的故事。这是科学、坚守与希望的汇聚，也是患者群体、科学家与产业携手为儿童癌症患者开辟新路径的生动写照。 不用上学是许多孩子梦寐以求的事，但对加比（Gabby）来说却不是这样。在她10岁那年，她因病在医院度过了5天，而这段经历彻底改变了她的生活。 因流感住院，却被诊断出脑瘤 加比的故事始于她10岁那年，她因流感住院5天。回到家时，加比因为太累了而无法去上学。而第二天，她突然瘫倒在地，加比的妈妈曼迪（Mandy）急忙将孩子送到急诊做CT扫描，想帮女儿找出病因。 在感觉像是经过了一个世纪的等待后，加比的医生拿到了结果。“抱歉让你久等了。”医生告诉曼迪，“我刚安排直升机把她送往另一家医院。” “你女儿的大脑里有一个鸡蛋大小的肿瘤，她需要手术！” 经过两次手术、一系列并发症以及在儿科重症监护室（PICU）16天的治疗，加比被确诊为儿科低级别胶质瘤。 从此，无休止的检查和治疗成了加比生活中的日常，照顾加比成了曼迪的全职工作，她不得不辞去原本的工作。加比的父亲在打两份工的同时，还要照顾家里的农场。加比的兄弟姐妹不得不更早成熟，在力所能及的范围内支持父母和姐姐。 每次带加比去做脑部扫描，曼迪都会紧张不已，担心检查结果带来坏消息。加比又经历了几次脑部手术，但艰难的治疗并未就此结束。虽然每个孩子的病程不同，但大约一半接受手术的LGG患者会复发，需要额外治疗。不幸的是，加比就是其中之一。在本应充满生日派对、学校社团活动和睡衣派对的青春期，加比不得不一次又一次接受化疗。她每隔一周就要去一次医院，错过了许多课程，也错过了很多童年的快乐时光。 虽然加比的健康状况一度稳定，但是在2021年，一次扫描带来了噩耗：脑中的肿瘤又长大了。曼迪知道必须采取行动。她记得之前曾与加比的肿瘤科医生讨论过另一种治疗方案，于是她翻阅医学期刊，找到了医生曾经提到的在研药物——由Day One Biopharmaceuticals开发的tovorafenib。 ▲Tovorafenib分子结构（图片来源：PubChem） 她了解到，tovorafenib正在针对携带BRAF基因融合或V600E突变的LGG儿童患者进行研究。这种药物可以在家中每周口服一次，而且是专为儿童设计的。 这是许久以来曼迪第一次燃起希望。而这款给加比带来希望的药物也走过漫长而曲折的开发历程。 患者群体、科学家和产业携手，让“老药”焕发青春 儿科LGG药物的开发面对多重挑战，由于LGG肿瘤生长速度缓慢，难以在动物模型中检测药物疗效。有的时候科学家尚未确定药物能否缩小肿瘤，小鼠就因寿命限制而死亡。而且儿童肿瘤药物开发通常滞后于成人肿瘤药物的开发，只有在针对成人患者的药物获批后，研究人员才启动在儿童患者群体中的研究。 这是儿童癌症患者父母们致力于改变的现状。在2007年，一群儿童脑瘤患者的父母加入了致力于通过骑车活动，为丹娜法伯癌症研究所（Dana-Farber Cancer Institute）的癌症研究捐款的Pan-Mass Challenge（PMC），并且寻求研究所对儿童癌症药物开发的支持。在患者团队的号召下，丹娜法伯癌症研究所专门成立了一个汇集Broad研究所与哈佛医学院医生、科学家的实验室，致力于探索儿童癌症药物开发的新策略。 研究团队发现在儿科LGG肿瘤中经常携带RAS/RAF/MAPK信号通路的突变，这意味着靶向这一信号通路可能成为疗法开发的突破口。经过广泛的临床前研究，科学家们发现一款RAF抑制剂展现出良好的抗癌效果，这就是后来的tovorafenib。他们启动了首个在儿科LGG患者中的临床试验，并且获得了初步积极结果。 Tovorafenib最初由渤健（Biogen）和Sunesis Pharmaceuticals联合开发，2011年，武田（Takeda）获得了这款药物的开发权益，并启动临床试验评估它治疗黑色素瘤的效果。然而临床试验结果并不理想，tovorafenib在成人癌症患者中的研发项目被中止，其在儿科LGG患者中的研发进程一度岌岌可危。 在这一关键时刻，武田公司中致力于儿科脑瘤药物开发的科学家们并没有放弃，他们说服武田将tovorafenib的开发权益授权给Day One Biopharmaceuticals公司。Day One Biopharmaceuticals公司创建的最初目标是开发针对儿童癌症患者的新药，其研发策略之一是从针对成年患者的“老药”中发掘治疗儿童癌症的“潜力股”，而tovorafenib正是这样一款与公司研发策略相匹配的药物。 Day One Biopharmaceuticals公司从武田获得了tovorafenib的开发权并且在2021年启动名为FIREFLY-1的2期临床试验，在携带BRAF变异的儿科LGG患者中评估tovorafenib的效果。 曼迪与加比的肿瘤科医生见面，确认加比符合参加这一临床试验的资格。 “超级英雄”的胜利 加比于2021年10月开始使用tovorafenib。她坚持服用了两年，在大部分时间里每月接受一次实验室检查，每3个月做一次MRI扫描来监测疗效。曼迪说：“她能在家服药就已经是个很大的胜利，这样她就不必每周去诊所接受治疗而缺课了。” 加比也确实经历了一些不良反应：皮疹、恶心、呕吐、毛发颜色变化和疲劳。有两次，她在服药后呕吐。随着时间推移，她的头发、睫毛和眉毛变成了白色。加比坦然接受了与众不同的自己，她说：“我看起来像个超级英雄！” 两年后，加比于2023年11月完成了tovorafenib治疗。她的肿瘤缩小并停止生长。现在，她每月复诊一次，病情依然保持稳定，并定期做MRI扫描以确保情况未发生变化。 2024年，美国FDA加速批准tovorafenib上市，商品名为Ojemda。它成为首个获批用于6个月及以上、携带BRAF基因变异的复发或难治性儿科LGG患者的处方药。 改变儿童癌症患者的生活 Tovorafenib的成功，是患者群体、科研人员和产业界多方共同努力的成果。这些年来，不止在儿科低级别胶质瘤领域，整个儿童癌症的治疗格局都在不断取得进展。根据世界卫生组织（WHO）的数据，截至2022年，全球范围内已有440款针对儿童癌症的药物处于研发阶段，其中靶向疗法占比最高（135款），其次是免疫疗法（108款），而小分子药物也约占三分之一。这些不断涌现的创新，正在为儿童癌症患者带来前所未有的治疗希望。 ▲儿童癌症疗法统计（图片来源：参考资料[7]） 作为全球医药创新的赋能者，药明康德很高兴能与合作伙伴携手，加速多款儿童癌症创新疗法问世，造福更多病患。长期以来，药明康德都在支持全球合作伙伴从药物研究（R）、开发（D）到商业化生产（M）各个阶段的需求，通过独特的一体化、端到端CRDMO模式，助力更多药物加速从实验室来到患者身边。 以治疗儿童癌症的小分子药物为例，药明康德化学服务平台能支持各种化学药物的分子形式及类别，满足从药物发现到商业化生产各个阶段、各个规模的各种物料需求。药明康德生物学业务平台作为综合性的早期发现和转化生物学赋能平台，能为多个疾病领域的新药研发提供从早期发现到临床研究阶段的生物学解决方案。药明康德测试业务平台可为包括儿童癌症在内多个疾病领域的药物提供全生命周期的一体化研发测试服务及全方位的临床研究服务，助力合作伙伴的药物成功申报IND、NDA以及通过核查上市。 在这个儿童癌症宣传月，让我们再次将目光投向这些勇敢的孩子和他们的家庭。每一次探索与坚持，都是为了让更多像加比一样的患者迎来新的希望。未来，药明康德将继续依托其一体化、端到端的CRDMO平台，携手全球合作伙伴，加速儿童癌症新药的研发与上市，把更多科学突破转化为真正改变生命的疗法，让更多孩子能够拥抱健康的明天。 Advancing Hope for Children with Cancer: WuXi AppTec’s End-to-End Support for Therapeutic Innovation Pediatric low-grade glioma (pLGG) is the most common brain tumor in children, representing nearly one-third of all pediatric central nervous system (CNS) tumors. While survival rates are generally favorable, pLGG is not without significant burdens, as children often face long-term neurological complications, impaired quality of life, and frequent relapses. As molecular insights reveal key oncogenic drivers such as MAPK/ERK and mTOR pathway alterations, new opportunities for targeted therapies are emerging. WuXi AppTec, through its fully integrated CRDMO (Contract Research, Development, and Manufacturing Organization) model, is enabling global partners to translate scientific breakthroughs into innovative medicines. By providing seamless support from discovery and preclinical research to IND-enabling studies and GMP manufacturing, WuXi AppTec helps accelerate progress toward safer and more effective treatments for pediatric cancers. Understanding the Challenge: Unmet Needs in Pediatric Low-Grade Glioma pLGG accounts for approximately 30–40% of pediatric CNS tumors, making it the single most common brain tumor type in children. Classified as a chronic rather than an acutely lethal disease, it nonetheless imposes lifelong consequences. Patients may suffer from seizures, vision loss, motor deficits, and neurocognitive impairment—all of which profoundly affect both the children and their families. Surgery remains the primary treatment option, but in many cases—especially tumors involving the brainstem or optic pathways—complete resection is not possible. For children with unresectable or recurrent disease, chemotherapy and radiotherapy are often necessary. While these approaches can prolong survival, they frequently cause severe long-term side effects, including endocrine dysfunction, secondary malignancies, and developmental delays. Recent advances have transformed the understanding of pLGG biology. Aberrant activation of the MAPK/ERK signaling pathway, often through BRAF gene fusions or mutations, is a hallmark event. Dysregulation of mTOR signaling also contributes to tumor growth. These findings have opened the door to targeted therapies, but critical challenges remain: achieving durable responses, minimizing toxicity, and identifying reliable preclinical models to predict clinical benefit. Breakthroughs in Motion: Science, Solutions, and Support As a trusted partner to global innovators, WuXi AppTec plays a pivotal role in the development of next-generation therapies for pediatric cancers through its fully integrated CRDMO model. We provide seamless support across the entire drug development spectrum, enabling faster, more cost-effective progress from discovery to commercialization. For small-molecule innovation, WuXi AppTec’s Chemistry Services Platform supports all categories of chemical compounds and production scales. This infrastructure helps partners meet material needs from lead optimization to clinical supply and beyond. Meanwhile, WuXi AppTec’s Biology Business Unit offers an integrated platform for early discovery and translational biology, empowering researchers to evaluate new targets and mechanisms in pediatric cancers with precision and speed. WuXi AppTec’s Testing Division enhances these capabilities with comprehensive R&D testing and clinical research services. From preclinical safety assessments to IND/NDA-enabling studies, the team helps streamline regulatory submissions and approval processes. This integrated model not only accelerates timelines but also enhances efficiency—helping transform innovative ideas into lifesaving therapies. Expanding Frontiers: Toward a Healthier Tomorrow for Children In recent years, progress has been made not only in the field of pLGG, but also across the broader landscape of childhood cancer treatment. According to data from the World Health Organization (WHO), as of 2022, a total of 440 drugs targeting childhood cancers were in development worldwide. Among them, targeted therapies accounted for the largest share (135 drugs), followed by immunotherapies (108 drugs), while small-molecule drugs also represented about one-third. These continuous innovations are bringing unprecedented treatment hope to pediatric cancer patients. WuXi AppTec is proud to contribute to this transformation. By providing integrated, end-to-end CRDMO solutions, we empower our partners to accelerate the development of novel therapies for pediatric cancers. With a steadfast commitment to scientific excellence, speed-to-clinic execution, and patient impact, WuXi AppTec stands with the global community in working toward a future where children with cancer can look forward to healthier, brighter lives. 参考资料： [1] Meet Gabby: The Shield-maiden With the Sweetest Hugs. Retrieved August 14, 2025, from https://patientworthy.com/2025/05/15/meet-gabby-the-shield-maiden-with-the-sweetest-hugs-2/. [2] Families and PMC funding play major role in new FDA-approved pediatric brain cancer drug. Retrieved August 14, 2025, from https://www.cbsnews.com/boston/news/pan-mass-challenge-funding-fda-pediatric-brain-cancer-drug-boston/ [3] A new dawn for tovorafenib. Retrieved August 14, 2025, from https://www.oncologypipeline.com/apexonco/new-dawn-tovorafenib [4] Turning “One Day” into “Day One”. Retrieved August 15, 2025, from https://pcrf-kids.org/2024/02/16/special-guest-author-blackman/ [5] A History Making FDA Drug Approval for the Most Common Brain Tumor in Children. Retrieved August 15, 2025, from https://www.pnocfoundation.org/toverafanib-fda-approval [6] Cleveland et al., (2025). The global landscape and development pipeline for childhood cancer medicines: a comprehensive analysis with dashboard generation. The Lancet Child & Adolescent Health. https://doi.org/10.1016/S2352-4642(25)00142-7 [7] Childhood cancer drug current landscape and pipeline characteristics. Retrieved August 15, 2025, from https://www.who.int/observatories/global-observatory-on-health-research-and-development/monitoring/pediatric-cancer-drug-pipeline-characteristics 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