Muscular Dystrophy, Duchenne

点击蓝字关注我们本周，大热点不多，但小热点不少。首先是审评审批方面，多个药申报上市，比如和黄医药的赛沃替尼片以及强生的古塞奇尤单抗注射液。此外，国外方面也值得一说，很值得关注的就是恒瑞医药TROP2-ADC被FDA授予快速通道资格，华东医药的索米妥昔单抗注射液成为首个和唯一一个在美国获批用于治疗卵巢癌的ADC药物；研发方面，多个药物启动关键临床以及获得重要进展，这里提一下，再鼎医药超3.3亿美元引进的KRAS抑制剂在中国启动Ⅲ期临床，一线治疗肺癌；交易及投融资方面，不少交易发生，但最引起关注的就是葆元医药被收购。本周盘点包括审评审批、研发、交易及投融资三大板块，统计时间为3.25-3.29，包含28条信息。 审评审批NMPA上市申请1、3月27日，CDE官网显示，和黄医药的赛沃替尼片（savolitinib，沃瑞沙）新适应症申报上市，拟用于一线及二线治疗MET外显子14跳跃突变非小细胞肺癌（NSCLC）患者。赛沃替尼是一种口服的高选择性小分子c-Met抑制剂，2011年，和黄医药与阿斯利康达成一项全球许可协议，将共同开发沃瑞沙并促进其商业化。2、3月27日，CDE官网显示，强生的古塞奇尤单抗注射液新适应症申报上市，拟用于治疗中重度活动性克罗恩病（CD）成年患者，此外该适应症已被纳入优先审批。古塞奇尤单抗是全球首个获批的白介素23（IL-23）抑制剂，于2019年12月在中国获批上市，用于治疗中重度斑块状银屑病成人患者。3、3月27日，CDE官网显示，Santhera制药和曙方医药的Vamorolone口服混悬液申报上市，拟用于治疗四岁及以上杜氏肌营养不良（DMD）患者。Vamorolone与糖皮质激素结合的受体相同但改变了其下游活性，2022年1月，曙方医药从Santhera获得Vamorolone在中国（包括中国香港、中国澳门和中国台湾）用于杜氏肌营养不良和其他罕见病适应症的独家开发和商业化权益。4、3月28日，CDE官网显示，安斯泰来的Nectin-4ADC药物维恩妥尤单抗（enfortumabvedotin）和默沙东的PD-1单抗帕博利珠单抗（pembrolizumab）新适应症申报上市，联合用于一线治疗既往未经治疗的局部晚期或转移性尿路上皮癌（la/mUC）成年患者。5、3月29日，CDE官网显示，和黄医药的呋喹替尼胶囊新适应症申报上市，拟用于联合信迪利单抗用于既往系统性抗肿瘤治疗后疾病进展且不适合进行根治性手术治疗或根治性放疗的晚期pMMR或nonMSI-H子宫内膜癌患者。此前，该适应症已被纳入优先审评。呋喹替尼是一种高选择性、强效的口服VEGFR1/2/3抑制剂。临床批准       6、3月25日，CDE官网显示，石药集团的司美格鲁肽注射液获批临床，用于减少热量饮食和增加体力活动的基础上对成人超重或肥胖患者的体重管理。石药集团的司美格鲁肽产品属于中国化药注册分类2.2类，目前全球尚无化学合成的司美格鲁肽产品上市。7、3月28日，CDE官网显示，驯鹿生物的伊基奥仑赛注射液获批临床，拟扩大适应症用于治疗既往经过1-2线治疗且来那度胺耐药的复发/难治性多发性骨髓瘤患者。伊基奥仑赛注射液是全人源靶向BCMA嵌合抗原受体自体T细胞注射液，于2023年6月在中国上市。优先审评8、3月27日，CDE官网显示，BMS的纳武利尤单抗（Opdivo）+伊匹木单抗（Yervoy）双重免疫治疗组合拟纳入优先审批，用于一线治疗不可切除或转移性微卫星高度不稳定性（MSI-H）或错配修复缺陷型（dMMR）结直肠癌（CRC）患者。突破性疗法9、3月25日，CDE官网显示，罗氏（Roche）的Inavolisib拟纳入突破性治疗品种，用于治疗与哌柏西利和内分泌疗法联合用药适用于治疗PIK3CA突变、激素受体（HR）阳性、人表皮生长因子受体2（HER2）阴性的局部晚期或转移性乳腺癌成人患者。Inavolisib是一款靶向PI3Kα突变体的选择性抑制剂。10、3月25日，CDE官网显示，恒瑞医药的氟唑帕利胶囊和甲磺酸阿帕替尼片拟纳入突破性治疗品种，氟唑帕利单药或联合阿帕替尼治疗gBRCA突变的HER2阴性乳腺癌。氟唑帕利属于一种新型口服PARP抑制剂，阿帕替尼属于一种高选择性的靶向血管内皮细胞生长因子受体2（VEGFR2）的抗血管生成药物。FDA上市批准11、3月25日，FDA官网显示，阿斯利康的ravulizumab-cwvz（Ultomiris）新适应症获批上市，用于治疗抗水通道蛋白4（AQP4）抗体阳性（Ab+）视神经脊髓炎（NMOSD）。Ultomiris是一款长效补体C5抑制剂，最早于2018年12月获FDA批准上市，这是首款也是唯一一款能够使得此类患者摆脱复发的长效C5补体抑制剂。12、3月25日，FDA官网显示，华东医药全资子公司中美华东美国合作方AbbVie的索米妥昔单抗注射液（ELAHERE）获完全批准，用于治疗既往接受过1-3线系统性治疗的叶酸受体α（FRα）阳性的铂类耐药的上皮性卵巢癌、输卵管癌或原发性腹膜癌成年患者。13、3月25日，FDA官网显示，再生元收到CD3/CD20双抗odronextamab治疗复发/难治性（R/R）滤泡性淋巴瘤（FL）和R/R弥漫性大B细胞淋巴瘤（DLBCL）的BLA申请发出的完整回复函（CRL）。再生元表示唯一原因是确证临床的入组状态问题，而与疗效或安全性、临床设计、标签或生产问题无关。14、3月26日，FDA官网显示，默沙东的Sotatercept（Winrevair）获批上市，用于治疗肺动脉高压（PAH）。Sotatercept是由Acceleron Pharma开发的一款first-in-class ACVR2A-Fc融合蛋白。15、3月27日，FDA官网显示，Akebia Therapeutics的vadadustat（Vafseo）获批上市，用于治疗至少接受3个月透析的慢性肾病（CKD）贫血患者。vadadustat是一款小分子缺氧诱导因子-1脯氨酰羟化酶（HIF-PH）抑制剂，通过增加内源性促红细胞生成素的产生，改善铁的吸收和动员以及下调铁调素来促进红细胞生成。孤儿药资格16、3月28日，FDA官网显示，华昊中天的优替德隆注射液被授予孤儿药资格，用于治疗乳腺癌脑转移。优替德隆为一种基于合成生物学技术研发上市的化疗新分子，其与紫杉类作用机制相似，已于2021年在中国获批上市。快速通道资格17、3月25日，FDA官网显示，凡恩世（PhanesTherapeutics）的PT886被授予快速通道资格，用于治疗转移性Claudin18.2阳性胰腺癌患者。此前，PT886已于2022年获得了FDA授予用于治疗胰腺癌的孤儿药资格。PT886是一款在研的双特异性抗体，靶向Claudin18.2和CD47，拟开发用于治疗胃癌、胃食管交界处癌和胰腺癌。18、3月28日，FDA官网显示，恒瑞医药的注射用SHR-A1921被授予快速通道资格（FTD)。这是恒瑞第3款获得美国FDA快速通道资格认定的产品。SHR-A1921是一款靶向TROP-2肿瘤相关抗原的抗体药物偶联物（ADC）。研发临床状态19、3月25日，药物临床试验登记与信息公示平台显示，安进（Amgen）和百济神州共同启动了一项国际多中心（含中国）Ⅲ期临床研究（DeLLphi-306），以评价局限期小细胞肺癌放化疗后进行tarlatamab治疗的有效性和安全性。tarlatamab（AMG757）是一款靶向DLL3和CD3的BiTE产品。20、3月27日，药物临床试验登记与信息公示平台显示，康乐卫士生物和成大生物登记了一项重组十五价人乳头瘤病毒疫苗（大肠埃希菌）Ⅰ期临床试验。这是一项随机、盲法、安慰剂和阳性对照的I期临床试验，旨在评价在9-45周岁健康女性受试者中接种重组十五价人乳头瘤病毒疫苗（大肠埃希菌）的安全性、耐受性和免疫原性。21、3月29日，药物临床试验登记与信息公示平台显示，再鼎医药和Mirati公司（已被BMS收购）启动了一项国际多中心（含中国）Ⅲ期临床研究，旨在评估adagrasib联合帕博利珠单抗一线治疗具有KRASG12C突变且TPS≥50%的非小细胞肺癌（NSCLC）患者的有效性和安全性。Adagrasib是一款具有高度特异性的强力口服KRASG12C抑制剂。临床数据22、3月25日，Axsome宣布，AXS-12（瑞波西汀）治疗发作性睡病的Ⅲ期SYMPHONY研究达到了主要终点，即AXS-12可以显著降低发作性睡病患者的猝倒频率。AXS-12是一种高选择性且强效的去甲肾上腺素再摄取（NET）抑制剂和皮质多巴胺调节剂。此前，AXS-12已被获批用于治疗抑郁症。23、3月25日，康方生物宣布，其与合作伙伴SummitTherapeutics发布了PD-1/VEGF双抗依沃西联合化疗或单药治疗脑转移非小细胞肺癌（NSCLC）的疗效分析结果。结果显示，依沃西对NSCLC脑转移的颅内肿瘤控制效果极具临床潜力，有望成为NSCLC脑转移患者的全新高效治疗选择。24、3月26日，恒瑞医药宣布，CameL-sq研究的4年长期随访结果。最新生存数据显示：卡瑞利珠单抗联合化疗一线治疗晚期驱动基因阴性鳞状非小细胞肺癌4年总生存期（OS）率为33.9%，较化疗组（14.3%）提升了近20%。这也意味着，CameL-sq研究中卡瑞利珠单抗联合化疗组中有超过1/3的晚期肺鳞癌患者生存期突破4年，为更多患者带来长生存的希望。25、3月27日，Novocure宣布，肿瘤电场治疗（TTFields）的Ⅲ期METIS研究达到主要终点。在1–10个病灶的新诊断非小细胞肺癌脑转移患者中，与仅接受支持治疗的患者相比，使用肿瘤电场治疗联合支持治疗的患者在颅内进展时间方面有统计学意义的改善。再鼎医药拥有该肿瘤电场疗法在大中华区的开发权益。交易及投融资26、3月25日，艾伯维宣布，与LandosBiopharma达成了一项收购协议，从而获得了first-in-class口服NLRX1激动剂（NOD样受体家族成员）NX-13。NX-13具有双重作用机制（MOA），兼具抗炎和促进上皮修复功能。目前NX-13治疗UC的随机对照Ⅱ期NEXUS临床试验正在美国和欧洲招募患者(NCT05785715)。27、3月26日，NuvationBio宣布，与葆元医药达成最终协议，NuvationBio以全股票交易方式收购葆元医药。NuvationBio是一家生物制药公司，旨在通过开发差异化和新颖的治疗候选药物以满足肿瘤治疗中亟需的要求。葆元医药是一家致力于为癌症患者开发新型精准疗法的全球性临床阶段生物制药公司。28、3月27日，Moderna宣布，与黑石生命科学公司达成了一项开发和商业化资助协议，以推进公司的流感疫苗项目。根据协议条款，黑石集团将为Moderna提供高达7.5亿美元的研发资金。而Moderna未来需要向黑石集团支付累计商业里程金和低个位数版税。【智药研习社近期课程预告】来源：CPHI制药在线声明：本文仅代表作者观点，并不代表制药在线立场。本网站内容仅出于传递更多信息之目的。如需转载，请务必注明文章来源和作者。投稿邮箱：Kelly.Xiao@imsinoexpo.com▼更多制药资讯，请关注CPHI制药在线▼点击阅读原文，进入智药研习社~

TUCSON, Ariz., March 28, 2024 /PRNewswire/ -- Critical Path Institute's (C-Path) Duchenne Regulatory Science Consortium (D-RSC) is excited to announce the launch of a groundbreaking model-based Clinical Trial Simulator (CTS), specifically designed to improve design of efficacy studies for potential therapies for Duchenne muscular dystrophy (DMD). This pioneering Drug Development Tool is set to positively impact the medical research community by significantly optimizing clinical trial design. Continue Reading DMD is a rare, fatal, X-linked, muscle wasting, and progressive disease that predominantly affects boys but has been shown to manifest in some female carriers. Research and development in this area have been challenging due to the complexities associated with the disease. This Clinical Trial Simulator will positively impact DMD research by significantly optimizing clinical trial design. Post this Developed by C-Path's Quantitative Medicine Program and D-RSC, together with an extensive group of collaborators, this simulator utilizes advanced models of the longitudinal changes in the velocity at which individuals can complete specified, timed functional tests. These tests, which include the supine-stand, 4-stair climb, 10-meter walk/run test, or 30-foot walk/run test, are frequently used as clinical trial endpoints. Additionally, the tool models longitudinal changes in forced vital capacity and the North Star Ambulatory Assessment total score, providing a comprehensive approach to trial design for simulation. A key feature of the DMD Clinical Trial Simulator is its incorporation of relevant sources of variability such as baseline severity at study start, age, steroid use, genetic mutation and study type. This ensures that the simulations are as realistic and informative as possible, paving the way for more effective and efficient clinical trial designs. In November 2022, D-RSC received a Letter of Support from the European Medicines Agency, and the Clinical Trial Simulator is currently being reviewed by the U.S. Food and Drug Administration (FDA). "The launch of the DMD Clinical Trial Simulator represents a significant step forward in our fight against Duchenne muscular dystrophy," said Ramona Belfiore-Oshan, Ph.D., Executive Director for D-RSC. "By using quantitative methodologies that allow the improvement of trial design in silico, we can accelerate the development of effective therapies for DMD and provide a learning platform for application of model informed drug development in other rare diseases." "The DMD Clinical Trial Simulator is poised to become a valuable resource for researchers and pharmaceutical companies involved in DMD research," commented C-Path CEO Klaus Romero, M.D. M.S., FCP. "Its ability to simulate various scenarios and outcomes will not only improve the accuracy of clinical trials but also reduce the time and resources required to bring new therapies to market." The CTS will be made publicly available on c-path.org, here, and hosted on the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®), C-Path's designated analytics hub for data analysis and generation of drug development tools across multiple rare diseases. "We advance the RDCA-DAP platform as a place for innovation for rare disease drug development and are excited to offer open access to C-Path's drug development tools on RDCA-DAP. Granting access to the D-RSC Clinical Trial Simulator through RDCA-DAP creates a unique opportunity for innovative trial designs in DMD," said Alexandre Bétourné, Pharm.D., Ph.D., Executive Director, RDCA-DAP. About D-RSC: D-RSC (Duchenne Regulatory Science Consortium) is comprised of a team of experts dedicated to advancing the understanding and treatment of Duchenne muscular dystrophy through innovative research and technology solutions. About Critical Path Institute Critical Path Institute (C-Path) is an independent, nonprofit established in 2005 as a public-private partnership, in response to the FDA's Critical Path Initiative. C-Path's mission is to lead collaborations that advance better treatments for people worldwide. Globally recognized as a pioneer in accelerating drug development, C-Path has established numerous international consortia, programs and initiatives that currently include more than 1,600 scientists and representatives from government and regulatory agencies, academia, patient organizations, disease foundations and pharmaceutical and biotech companies. With dedicated team members located throughout the world, C-Path's global headquarters is in Tucson, Arizona and C-Path's Europe subsidiary is headquartered in Amsterdam, Netherlands. For more information, visit c-path.org. FDA Acknowledgement Contacts: Roxan Triolo Olivas C-Path 520.954.1634 [email protected] Kissy Black C-Path 615.310.1894 [email protected] SOURCE Critical Path Institute

CORAL GABLES, Fla., March 28, 2024 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst") (Nasdaq: CPRX), a commercial-stage, patient-centric biopharmaceutical company focused on in-licensing, developing, and commercializing novel high-quality medicines for patients living with rare and difficult-to-treat diseases, today announced its endorsement of the inaugural Lambert-Eaton myasthenic syndrome ("LEMS") Awareness Day. This landmark event, now officially observed annually on March 30th, signifies a meaningful milestone for the LEMS community. The chosen date holds profound historical significance, coinciding with the publication of Dr. Edward Lambert and Dr. Lee Eaton's pioneering research on LEMS, which has greatly advanced the understanding and treatment of this rare neuromuscular disorder. "Creating an awareness day for LEMS is a huge win for our community," said Price Wooldridge, Founder and Board Member of the LEMS Family Association. "This brings us one step closer to our goal, improving the awareness of our rare condition in the larger population. We couldn't be more thrilled to celebrate the milestone of this first LEMS Awareness Day together." "In the realm of rare diseases, awareness is the key that unlocks paths to diagnosis and treatment. By shedding light on LEMS, we pave the way for better support, understanding, and care for those who need it most," stated Richard J. Daly, CEO of Catalyst. "At Catalyst, we are proud to stand alongside the LEMS Family Association in honoring this pivotal event. Together, we forge pathways of support and understanding for those affected by rare diseases, illuminating a brighter future for all." LEMS is a rare neuromuscular disease that affects at least 3,600 and potentially up to 5,400 LEMS patients in the United States. For more information, please visit www.lemsfamily.org and www.lemsaware.com. About Catalyst Pharmaceuticals, Inc.With exceptional patient focus, Catalyst is committed to developing and commercializing innovative first-in-class medicines that address rare and difficult-to-treat diseases. Catalyst's flagship U.S. commercial product is FIRDAPSE® (amifampridine) Tablets 10 mg, approved for the treatment of Lambert-Eaton myasthenic syndrome ("LEMS") for adults and for children ages six to seventeen. In January 2023, Catalyst acquired the U.S. commercial rights to FYCOMPA® (perampanel) CIII, a prescription medicine approved in people with epilepsy aged four and older alone or with other medicines to treat partial-onset seizures with or without secondarily generalized seizures and with other medicines to treat primary generalized tonic-clonic seizures for people with epilepsy aged 12 and older. Further, Canada's national healthcare regulatory agency, Health Canada, has approved the use of FIRDAPSE for the treatment of adult patients in Canada with LEMS. On July 18th, 2023, Catalyst acquired an exclusive license for North America for AGAMREE® (vamorolone) oral suspension 40 mg/mL, a novel corticosteroid treatment for Duchenne Muscular Dystrophy. AGAMREE previously received FDA Orphan Drug and Fast Track designations and was approved by the FDA for commercialization in the U.S. on October 26th, 2023. AGAMREE became available in the U.S. by prescription on March 13th, 2024. For more information about Catalyst Pharmaceuticals, Inc., visit the Company's website at www.catalystpharma.com. For Full Prescribing and Safety Information for FIRDAPSE®, visit www.firdapse.com. For Full Prescribing Information, including Boxed WARNING for FYCOMPA®, please visit www.fycompa.com. For Full Prescribing Information for AGAMREE®, please visit https://www.agamree.com. Forward-Looking StatementsThis press release contains forward-looking statements, as that term is defined in the Private Securities Litigation Reform Act of 1995. Forward-looking statements involve known and unknown risks and uncertainties, which may cause Catalyst's actual results in future periods to differ materially from forecasted results. A number of factors, including those factors described in Catalyst's Annual Report on Form 10-K for the fiscal year 2023 and its other filings with the U.S. Securities and Exchange Commission ("SEC"), could adversely affect Catalyst. Copies of Catalyst's filings with the SEC are available from the SEC, may be found on Catalyst's website, or may be obtained upon request from Catalyst. Catalyst does not undertake any obligation to update the information contained herein, which speaks only as of this date. Source: Catalyst Pharmaceuticals, Inc.