--FDA Grants Pre-BLA Meeting Request and Rolling BLA Submission after Review of HOPE-2 and HOPE-2 OLE 3-Year Results--

--FDA在审查HOPE-2和HOPE-2 OLE 3年结果后，批准BLA会议前请求和滚动BLA提交--

SAN DIEGO, June 11, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced the successful completion of a Type-B meeting with the U.S. Food and Drug Administration (FDA) on next steps for the Biologics License Application (BLA) submission with its lead asset, CAP-1002 for the treatment of Duchenne muscular dystrophy (DMD).

2024年6月11日，圣地亚哥（环球通讯社）--Capricor Therapeutics（NASDAQ:CAPR）是一家开发用于治疗罕见疾病的转化细胞和外泌体疗法的生物技术公司，今天宣布成功完成与美国食品和药物管理局（FDA）的B型会议，讨论生物制剂许可证申请（BLA）提交的下一步步骤，其主要资产CAP-1002用于治疗杜兴氏肌营养不良症（DMD）。

Additionally, Deramiocel has been selected as the proposed International Nonproprietary Name (INN) for CAP-1002 by the World Health Organization..

此外，Deramiocel已被世界卫生组织选为CAP-1002的拟议国际非专有名称（INN）。。

“The last year has been transformative for Capricor as we have moved rapidly towards potential approval for CAP-1002 for the treatment of DMD,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “The most recent meeting had several very important outcomes; the first of which was that the FDA has agreed to a pre-BLA meeting based on their review of our clinical data from HOPE-2, HOPE-2 open label extension (OLE) and available data from HOPE-3.

Capricor首席执行官琳达·马尔班（LindaMarbán）博士说：“去年对Capricor来说是一个变革，因为我们已经迅速取得了CAP-1002治疗DMD的潜在批准。”。“最近的一次会议取得了几个非常重要的成果；首先，FDA根据他们对HOPE-2、HOPE-2开放标签扩展（OLE）的临床数据以及HOPE-3的可用数据的审查，同意召开BLA前会议。

This key agreement will allow us to begin our BLA filing with a rolling submission planned to initiate in the third quarter of 2024.”.

这项关键协议将使我们能够以计划于2024年第三季度启动的滚动提交开始提交BLA。”。

The rolling submission process allows for the submission of pre-agreed components of the BLA to be submitted as they are completed, which can streamline the regulatory review process and de-risk the BLA. Capricor plans to further outline the submission schedule with the FDA in the coming months.

滚动提交流程允许提交预先商定的BLA组成部分，以便在完成后提交，这可以简化监管审查流程并降低BLA的风险。Capricor计划在未来几个月与FDA进一步概述提交时间表。

Dr. Marbán continued, “We continue to make significant progress on this program as we are awaiting final data in the fourth quarter of 2024 from our already enrolled HOPE-3 pivotal trial and prepare for potential commercialization, including completion of our commercial manufacturing facility and actively preparing for our pre-BLA meeting and rolling BLA submission.

Marbán博士继续说道：“我们在这个项目上继续取得重大进展，因为我们正在等待2024年第四季度已经登记的HOPE-3关键试验的最终数据，并为潜在的商业化做好准备，包括完成我们的商业制造设施，并积极准备我们的BLA前会议和滚动BLA提交。

Furthermore, we are looking forward to our presentation of additional 3-year data from our HOPE-2 OLE study at the upcoming PPMD conference which we believe will add further evidence of the efficacy and safety of CAP-1002.”.

此外，我们期待着在即将举行的PPMD会议上提交HOPE-2 OLE研究的额外3年数据，我们相信这将进一步证明CAP-1002的有效性和安全性。”。

Deramiocel (CAP-1002) for the treatment of DMD has received Orphan Drug Designation and the regulatory pathway for this drug is supported by RMAT (Regenerative Medicine Advanced Therapy Designation). In addition, if Capricor were to receive FDA marketing approval for Deramiocel for the treatment of DMD, Capricor would be eligible to receive a Priority Review Voucher (PRV) based on its previous receipt of a rare pediatric disease designation.

用于治疗DMD的Deramiocel（CAP-1002）已获得孤儿药物指定，该药物的监管途径得到了RMAT（再生医学高级治疗指定）的支持。此外，如果Capricor获得FDA对Deramiocel治疗DMD的上市批准，Capricor将有资格根据其先前收到的罕见儿科疾病名称获得优先审查凭证（PRV）。

Capricor retains full rights to the PRV, if received..

Capricor保留PRV的全部权利（如果收到）。。

About Duchenne Muscular Dystrophy

关于杜兴氏肌营养不良症

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 15,000-20,000 in the United States.

杜兴氏肌营养不良症（DMD）是一种破坏性遗传疾病，其特征是骨骼肌，心脏和呼吸肌进行性无力和慢性炎症，中位年龄约为30岁。据估计，DMD大约每3500名男性婴儿中就有一名发生，在美国，患者人口估计约为15000-20000。

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..

DMD病理生理学是由功能性肌营养不良蛋白的产生受损所驱动的，该蛋白通常在肌肉中起结构蛋白的作用。肌细胞中功能性肌营养不良蛋白的减少会导致严重的细胞损伤，并最终导致肌细胞死亡和纤维化替代。治疗选择有限，无法治愈。。

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.

Capricor Therapeutics，Inc.（纳斯达克股票代码：CAPR）是一家生物技术公司，致力于推进基于转化细胞和外泌体的疗法，以重新定义罕见疾病的治疗前景。我们创新的最前沿是我们的主要候选产品Deramiocel（CAP-1002），一种同种异体心脏衍生细胞疗法。

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).

广泛的临床前和临床研究表明，Deramiocel具有专门针对肌营养不良症和心脏病的免疫调节，抗纤维化和再生作用。Deramiocel目前正在进行治疗杜兴氏肌营养不良症（DMD）的3期临床开发。

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.

Capricor还利用我们的外泌体技术的力量，在临床前开发中使用我们专有的StealthX™平台，专注于疫苗学领域，靶向递送寡核苷酸，蛋白质和小分子疗法，以潜在地治疗和预防各种疾病。

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..

在Capricor，我们致力于突破可能性的界限，为有需要的人开辟一条变革性治疗之路。有关更多信息，请访问capricor.com，并在Facebook、Instagram和Twitter上关注capricor。。

Cautionary Note Regarding Forward-Looking Statements

关于前瞻性声明的警示说明

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.

本新闻稿中关于Capricor候选产品的功效，安全性和预期用途的声明；发现工作和临床试验的开始，行为，规模，时间和结果；临床试验的注册速度；关于监管文件，未来研究和临床试验的计划；涉及产品的监管发展，包括获得监管批准或以其他方式将产品推向市场的能力；制造能力；监管会议日期；关于我们财务前景的报表；实现产品里程碑和从商业合作伙伴处获得里程碑付款的能力；关于当前和未来合作活动以及商业权利所有权的计划；知识产权的范围、期限、有效性和可执行性；未来收入流和预测；对最近完成的发行收益的预期用途和发行的预期效果的预期；以及关于Capricor管理团队未来期望、信念、目标、计划或前景的任何其他声明，构成1995年《私人证券诉讼改革法案》含义内的前瞻性声明。

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.

任何不属于历史事实陈述的陈述（包括包含“相信”、“计划”、“可能”、“预期”、“期望”、“估计”、“应该”、“目标”、“将会”、“将会”和类似表述的陈述）也应视为前瞻性陈述。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的结果或事件存在重大差异。

More information about these and other risks that may impact Capricor’s business is set fo.

有关这些风险和其他可能影响Capricor业务的风险的更多信息，请参见。

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 (CAP-1002) is an Investigational New Drug and is not approved for any indications.

Capricor已与日本新屋株式会社（美国子公司：NS Pharma，Inc.）签订了一份协议，在美国和日本独家商业化和分销用于DMD的Deramiocel（CAP-1002），但需获得监管部门的批准。Deramiocel（CAP-1002）是一种研究性新药，未被批准用于任何适应症。

None of Capricor’s exosome-based candidates have been approved for clinical investigation..

Capricor的基于外泌体的候选人均未被批准用于临床研究。。

For more information, please contact:

有关更多信息，请联系：

Capricor Company Contact:

Capricor公司联系人：

AJ Bergmann, Chief Financial Officer

首席财务官AJ Bergmann

abergmann@capricor.com

abergmann@capricor.com

858.727.1755

858.727.1755