CORRECTION: Capricor Therapeutics
CORRECTION: Capricor Therapeutics
SAN DIEGO, June 25, 2024 (GLOBE NEWSWIRE) -- In a release issued under the headline "Capricor Therapeutics Announces for the Treatment of Duchenne Muscular Dystrophy" on Tuesday, June 25th by Capricor Therapeutics, please note that the words "Pre-BLA Meeting with FDA for Deramiocel" were omitted from the headline. The corrected release follows.
请注意,2024年6月25日星期二,Capricor Therapeutics发布的题为“Capricor Therapeutics宣布治疗杜兴肌萎缩症”的新闻稿中省略了“与FDA进行Deramiocel的BLA前会议”的话,该正确的新闻稿如下。
Capricor Therapeutics Announces Pre-BLA Meeting with FDA for Deramiocel for the Treatment of Duchenne Muscular Dystrophy
Capricor Therapeutics宣布将于2024年第三季度与美国食品和药物管理局(FDA)进行Deramiocel (CAP-1002)治疗杜兴肌萎缩症(DMD)的BLA (生物许可证申请)前会议。
Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has scheduled a Pre-BLA (Biologics License Application) meeting with the Company in the third quarter of 2024 for deramiocel (CAP-1002), for the treatment of Duchenne Muscular Dystrophy (DMD). Capricor's goal for this meeting will be to finalize its BLA filing plans based on all currently available data as well as to work with the FDA to outline the rolling BLA submission timeline.
Capricor TherapeuticsCapricor Therapeutics是一家生物技术公司,致力于开发用于治疗罕见疾病的细胞和外泌体转化性疗法。该公司的目标是细胞疗法将结合生物技术、制造和半导体技术。
"We continue to move rapidly towards potential approval of deramiocel for the treatment of DMD," said Linda Marbán, Ph.D., Capricor's chief executive officer. "We now have formally scheduled our Pre-BLA meeting with the FDA, which will finalize our BLA filing plans and discuss available options to expedite the filing of our BLA. We recognize the FDA's willingness to bring impactful therapies to market as quickly as possible due to the enormous unmet needs of patients with DMD. While we are delighted with the approval of gene therapy for DMD, we believe that it will take multiple therapies to combat DMD effectively. Based on the need to address the secondary consequences of DMD, we believe deramiocel can serve as a potential anchor therapy for DMD patients."
“我们继续快速向杜兴肌萎缩症治疗的可能批准迈进,”Capricor的首席执行官Linda Marban博士表示,“我们现在已经正式安排了与FDA的BLA前会议,这将最终确定我们的BLA申请计划,并讨论可用的选项以加速我们的BLA的提交。我们认识到FDA愿意尽快将重要的治疗方法引入市场,因为DMD患者的需求极大。虽然我们对DMD基因治疗的批准感到高兴,但我们认为,需要多种治疗方法才能有效地对抗DMD。基于应对DMD的副作用的需求,我们认为Deramiocel可以成为治疗DMD患者的潜在锚点疗法。”
Additionally, Capricor will present its latest update of the HOPE-2 open label extension (OLE) 36-month data, both skeletal and cardiac, at the upcoming Parent Project Muscular Dystrophy (PPMD) 30th Annual Conference being held June 27-29, 2024.
此外,Capricor将在即将举行的Parent Project肌萎缩症(PPMD) 30年度会议上,展示HOPE-2开放标签延伸(OLE) 36个月的骨骼和心脏最新数据。th2024年6月27日-29日举行。
About Duchenne Muscular Dystrophy
关于杜兴肌萎缩症
Duchenne muscular dystrophy (DMD) is a devastating genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles with mortality at a median age of approximately 30 years. It is estimated that DMD occurs in approximately one in every 3,500 male births and that the patient population is estimated to be approximately 15,000 to 20,000 in the United States. DMD pathophysiology is driven by the impaired production of functional dystrophin, which normally functions as a structural protein in muscle. The reduction of functional dystrophin in muscle cells leads to significant cell damage and ultimately causes muscle cell death and fibrotic replacement. Treatment options are limited and there is no cure.
杜氏肌营养不良症(DMD)是一种具有遗传性的灾难性疾病,其特征为骨骼、心脏和呼吸肌肉的渐进性衰弱和慢性炎症,中位数约在30岁左右死亡。据估计,DMD在每3500个男性新生儿中发生一次,患者人口在美国估计约为15000至20000人。DMD的病理生理学是由于功能性的肌营养素受损而导致的,肌营养素通常作为肌肉中的结构蛋白。肌细胞中功能性肌营养素的减少导致明显的细胞损伤,最终导致肌细胞死亡和纤维化替换。治疗选择有限,目前还没有治愈方法。
About Capricor Therapeutics
关于Capricor Therapeutics
Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases. At the forefront of our innovation is our lead product candidate, deramiocel (CAP-1002), an allogeneic cardiac-derived cell therapy. Extensive preclinical and clinical studies have shown deramiocel to demonstrate immunomodulatory, antifibrotic, and regenerative actions specifically tailored for dystrophinopathies and heart disease. Deramiocel is currently advancing through Phase 3 clinical development for the treatment of Duchenne muscular dystrophy (DMD). Capricor is also harnessing the power of our exosome technology, using our proprietary StealthX platform in preclinical development focused on the areas of vaccinology, targeted delivery of oligonucleotides, proteins and small molecule therapeutics to potentially treat and prevent a diverse array of diseases. At Capricor, we stand committed to pushing the boundaries of possibility and forging a path toward transformative treatments for those in need. For more information, visit capricor.com, and follow Capricor on Facebook, Instagram and Twitter.
Capricor Therapeutics, Inc.(纳斯达克:CAPR)是一家生物技术公司,致力于推进细胞和外泌体转化性疗法,重新定义罕见疾病的治疗模式。我们的创新前沿产品候选药是Deramiocel(CAP-1002),这是一种异基因心脏衍生细胞疗法。广泛的临床前研究和临床研究已经表明,Deramiocel具有针对骨骼肌和心脏疾病特定的免疫调节、抗纤维化和再生作用。Deramiocel目前正在进行第III期临床开发,用于治疗杜兴肌萎缩症(DMD)。Capricor还正在利用我们的外泌体技术,利用我们专有的StealthX平台,在疫苗学、寡核苷酸、蛋白和小分子疗法的靶向传递等预临床开发领域开发应用,以潜在地治疗和预防多种疾病。在Capricor,我们致力于推动可能性的界限,开辟通往有需求病患的变革性治疗的道路。欲了解更多信息,请访问capricor.com,并关注Capricor的Facebook、Instagram和Twitter。
Cautionary Note Regarding Forward-Looking Statements
关于前瞻性声明的谨慎说明
Statements in this press release regarding the efficacy, safety, and intended utilization of Capricor's product candidates; the initiation, conduct, size, timing and results of discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including the ability to obtain regulatory approvals or otherwise bring products to market; manufacturing capabilities; dates for regulatory meetings; statements about our financial outlook; the ability to achieve product milestones and to receive milestone payments from commercial partners; plans regarding current and future collaborative activities and the ownership of commercial rights; scope, duration, validity and enforceability of intellectual property rights; future revenue streams and projections; expectations with respect to the expected use of proceeds from the recently completed offerings and the anticipated effects of the offerings; and any other statements about Capricor's management team's future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words "believes," "plans," "could," "anticipates," "expects," "estimates," "should," "target," "will," "would" and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements. More information about these and other risks that may impact Capricor's business is set forth in Capricor's Annual Report on Form 10-K for the year ended December 31, 2023, as filed with the Securities and Exchange Commission on March 11, 2024, and in our Quarterly Report on Form 10-Q for the quarter ended March 31, 2024, as filed with the Securities and Exchange Commission on May 14, 2024. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements.
本新闻稿中的有关Capricor产品候选药物的功效、安全性和预期用途、发现工作和临床试验的启动、进行、规模、时间表和结果的表述、临床试验招募速度、有关产品的监管文件、未来研究和临床试验的计划、包括能否获得监管批准或其他将产品引入市场的能力的监管进展情况、制造能力、监管会议的日期、我们的财务展望、实现产品里程碑和从商业合作伙伴那里获得里程碑付款的能力、当前和未来合作活动的范围、持续时间、有效性和可执行性的知识产权权利、未来的收入流和预测、有关最近完成的配股所募集资金的预期用途及配股的预期效果的表述,以及Capricor管理团队未来的期望、信念、目标、计划或前景的任何其他表述,均构成《1995年私人诉讼改革法》下的前瞻性声明。任何非历史事实的陈述(包括包含“相信”、“计划”、“可能”、“预测”、“期望”、“估计”、“应”、“目标”、“将”、“将要”及类似表述的陈述)也应被视为前瞻性陈述。有许多重要因素可能导致实际结果或事件与此类前瞻性声明所示不符。有关这些及其他可能影响Capricor业务的风险的更多信息,请参阅Capricor 2023年12月31日提交给证券交易委员会的10-K年度报告,以及我们提交给证券交易委员会的2024年3月31日提交的10-Q季度报告。本新闻稿中所有前瞻性陈述均基于Capricor现有的信息,Capricor不承担更新这些前瞻性陈述的义务。
Capricor has entered into an agreement for the exclusive commercialization and distribution of deramiocel (CAP-1002) for DMD in the United States and Japan with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval. Deramiocel is an Investigational New Drug and is not approved for any indications. None of Capricor's exosome-based candidates have been approved for clinical investigation.
Capricor已与日本新药股份有限公司 (美国子公司: NS Pharma,Inc.)签订协议,独家商业化和分销deramiocel(CAP-1002)用于治疗杜兴肌萎缩症(DMD)在美国和日本,需经监管部门批准。Deramiocel是一种 Investigational New Drug,尚未获得任何适应症的批准。Capricor的任何外泌体为基础的候选药物都没有获得临床研究批准。(NS Pharma关于库存制品制造商Biologics
For more information, please contact:
更多信息,请联系:
Capricor Company Contact:
AJ Bergmann, Chief Financial Officer
abergmann@capricor.com
858.727.1755
Capricor公司联系方式:
AJ Bergmann,首席财务官
abergmann@capricor.com
858.727.1755