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. Capricor Therapeutics Announces Pre-BLA Meeting with FDA for Deramiocel for the Treatment of Duchenne Muscular Dystrophy Capricor Therapeutics (NASDAQ: CAPR), a biot ...

SAN DIEGO, June 25, 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 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 final ...

SAN DIEGO, June 25, 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 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 fina ...

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

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

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q ☒ Quarterly Report Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934 for the quarterly period ended March 31, 2024 or ☐ Transition Report Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934 for the transition period from to Commission File Number: 001-34058 CAPRICOR THERAPEUTICS, INC. (Exact Name Of Registrant As Specified In Its Charter) Delaware 88-0363465 (State or o ...

财务数据和关键指标变化 - 公司现金、现金等价物和有价证券总额为3.99亿美元,较2023年12月31日的3.95亿美元有所增加 [33] - 公司在第一季度从日本新药公司收到了1000万美元的里程碑付款 [34] - 公司还通过ATM融资计划在第一季度及之后筹集了约350万美元的资金,平均价格为每股5.75美元 [34] - 根据公司当前的经营计划和预测,预计现金可持续到2025年第一季度,不包括日本新药公司未来可能的里程碑付款 [34] 各条业务线数据和关键指标变化 - 公司正在推进其主要资产CAP-1002用于治疗杜氏肌肉营养不良症(DMD)的临床开发 [5][6] - 公司完成了HOPE-3 III期临床试验Cohort A的入组,共61名受试者被随机分配到CAP-1002组和安慰剂组 [7] - Cohort A在2023年12月完成了预设的中期无效性分析,这一里程碑触发了公司从日本新药公司获得的1000万美元付款 [8] - 公司正在评估Cohort B的扩展,包括在欧洲招募受试者的可能性 [11] 各个市场数据和关键指标变化 - 公司正在与日本新药公司及其美国子公司NS Pharma积极准备CAP-1002的潜在商业化 [16] - 公司计划将CAP-1002的价格定在目前获批的外显子跳读疗法的价格范围内或以上 [18] - 公司预计CAP-1002的潜在适应症人群为7500-10000名DMD患者 [19] - 公司正在与欧洲药品管理局(EMA)和日本药品医疗器械综合机构(PMDA)就全球拓展CAP-1002进行初步接触和策划 [39][40] 公司战略和发展方向及行业竞争 - 公司正在推进CAP-1002用于治疗DMD的临床开发,并在制造、BLA申报和商业化准备等方面取得进展 [6] - 公司正在评估扩大Cohort B临床试验至欧洲的可能性,以支持CAP-1002在全球范围内的开发 [11] - 公司正在与FDA就CAP-1002的BLA申报进行多次会议和沟通,包括获得滚动提交BLA的批准 [12][13] - 公司正在开发其外泌体平台技术,包括StealthX疫苗和ARG1酶替代治疗等项目 [25][26][28] - 公司正在评估通过合作或独立的方式拓展CAP-1002在欧洲和其他地区的权益 [22] 管理层对经营环境和未来前景的评论 - 管理层认为CAP-1002有望成为DMD治疗的长期或终生治疗选择,并有望获得较高的价格和报销 [17][18] - 管理层认为CAP-1002有望成为DMD患者的首选补充疗法,可以与基因调控或外显子跳读疗法协同使用 [23] - 管理层对公司外泌体平台技术的未来发展前景持乐观态度,认为该技术有广泛的应用潜力 [25][26] - 管理层表示公司将继续专注于推进CAP-1002的商业化准备,并谨慎利用非稀释性资本机会支持外泌体项目 [24][30] 问答环节重要的提问和回答 问题1 **Kristen Kluska 提问** 询问公司与日本新药公司的里程碑付款安排,以及是否有可能在2024年第四季度获得积极数据触发付款 [37][38] **Linda Marban 回答** 公司暂不能透露具体的里程碑付款安排,但表示这些付款将有助于公司在获批后推进商业化 [38] 问题2 **Joe Pantginis 提问** 询问公司是否考虑将CAP-1002用于治疗杜氏肌肉营养不良症相关心肌病的可能性,以及FDA可能关注的指标 [52][53][54][55][56][57][58] **Linda Marban 回答** 公司正在与FDA和行业专家密切合作,探讨将CAP-1002用于治疗DMD相关心肌病的可能性,并希望能够获得FDA支持,将心功能指标纳入CAP-1002的潜在适应症 [58][59] 问题3 **Aydin Huseynov 提问** 询问公司是否考虑将CAP-1002用于贝克尔肌营养不良症的可能性,以及这会如何影响与日本新药公司的财务关系 [66][67] **Linda Marban 回答** 公司正在评估将CAP-1002用于贝克尔肌营养不良症等其他适应症的可能性,这不受与日本新药公司的合作协议限制,公司有自主权进行拓展 [67]

Capricor Therapeutics (CAPR) came out with a quarterly loss of $0.31 per share versus the Zacks Consensus Estimate of a loss of $0.11. This compares to loss of $0.31 per share a year ago. These figures are adjusted for non-recurring items. This quarterly report represents an earnings surprise of -181.82%. A quarter ago, it was expected that this biotechnology company would post a loss of $0.23 per share when it actually produced a loss of $0.02, delivering a surprise of 91.30%. Over the last four quarters, ...

Exhibit 99.1 Capricor Therapeutics Reports First Quarter 2024 Financial Results and Provides Corporate Update -Phase 3, HOPE-3 Trial (Cohort A) of CAP-1002 in Duchenne Muscular Dystrophy Fully Enrolled; On Track to Report Top-Line Data in Q4 2024- -Positive Type-B CMC FDA Meeting held in Q1; Company Aligned with FDA on Demonstration of Non- Clinical Comparability, A Major Milestone on Path to CMC Clearance for BLA- -Received First Milestone Payment of $10 Million under U.S. Distribution and Commercializatio ...

-Preclinical findings show exosome-based therapeutic approach for the treatment of arginase-1 deficiency (ARG1-D)- -Presentation to be featured in an oral session being held on May 9, 2024- SAN DIEGO, May 09, 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 that an abstract featuring new preclinical data highlighting the therapeutic potential of its StealthX™ ...