A Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial Evaluating the Efficacy and Safety of Human Allogeneic Cardiosphere-Derived Cells for the Treatment of Duchenne Muscular Dystrophy

HOPE-3 is a two cohort, Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the efficacy and safety of a cell therapy called CAP-1002 in study participants with Duchenne muscular dystrophy (DMD) and impaired skeletal muscle function. Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either CAP-1002 or placebo every 3 months for a total of 4 doses during the first 12-months of the study. All participants will be eligible to receive 4 doses of CAP-1002 for an additional 12 months as part of an open-label extended assessment period.

开始日期2022-06-22

申办/合作机构

Capricor, Inc.

NCT04623671 / Completed临床2期

A Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of Intravenous Infusion of CAP-1002 in Patients With COVID-19 (INSPIRE)

This is a randomized, double-blind, placebo-controlled, Pilot, Phase 2 Exploratory study that will enroll subjects with a clinical diagnosis of COVID-19 confirmed by laboratory testing and who are in severe or critical condition as indicated by life-support measures.

开始日期2020-11-15

申办/合作机构

Capricor, Inc.

NCT04428476 / Active, not recruiting临床2期

Open-Label Extension of the HOPE-2 Duchenne Muscular Dystrophy Trial

This Phase 2, multi-center, open-label extension trial will provide CAP-1002 to subjects that were enrolled in the HOPE-2 trial and completed 12 months of follow-up. The trial will explore the safety and efficacy of sixteen intravenous administrations of CAP-1002, each separated by three months. Subjects will undergo a targeted screening during a 30-day screening period, eligible subjects will then undergo baseline safety and efficacy assessments on Day 1 prior to their first infusion of CAP-1002.
Subjects will complete trial assessments at Screening; Day 1; Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45 and 48. Safety and efficacy assessments will be conducted prior to CAP-1002 administration at the Day 1, Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42 and 45 trial visits, unless otherwise indicated.
All CAP-1002 infusions will be conducted in an outpatient setting at the investigative site on Day 1 and Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42 and 45. Subjects will be observed in the outpatient setting for at least two hours post infusion and then discharged the same day, if medically cleared by the site Investigator.

开始日期2020-07-20

申办/合作机构

Capricor, Inc.

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100 项与 Deramiocel 相关的专利（医药）

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项与 Deramiocel 相关的文献（医药）

2023-01-02·Expert Opinion on Biological Therapy

Biological and genetic therapies for the treatment of Duchenne muscular dystrophy

Review

作者: Yokota, Toshifumi ; Wilton-Clark, Harry

INTRODUCTIONDuchenne muscular dystrophy is a lethal genetic disease which currently has no cure, and poor standard treatment options largely focused on symptom relief. The development of multiple biological and genetic therapies is underway across various stages of clinical progress which could markedly affect how DMD patients are treated in the future.AREAS COVEREDThe purpose of this review is to provide an introduction to the different therapeutic modalities currently being studied, as well as a brief description of their progress to date and relative advantages and disadvantages for the treatment of DMD. This review discusses exon skipping therapy, microdystrophin therapy, stop codon readthrough therapy, CRISPR-based gene editing, cell-based therapy, and utrophin upregulation. Secondary therapies addressing nonspecific symptoms of DMD were excluded.EXPERT OPINIONDespite the vast potential held by gene replacement therapy options such as microdystrophin production and utrophin upregulation, safety risks inherent to the adeno-associated virus delivery vector might hamper the clinical viability of these approaches until further improvements can be made. Of the mutation-specific therapies, exon skipping therapy remains the most extensively validated and explored option, and the cell-based CAP-1002 therapy may prove to be a suitable adjunct therapy filling the urgent need for cardiac-specific therapies.

2022-03-01·The Lancet1区 · 医学

Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial

1区 · 医学

Article

作者: McDonald, Craig M ; Collado, Jorge ; Henricson, Erik K ; Prasad, Poonam ; Ascheim, Deborah D ; Dayan, Jonathan G. ; Rogy, Siegfried ; Surampudi, Prasanth ; Hor, Kan N ; Furlong, Pat ; Eagle, Michelle ; Mayer, Oscar H ; Varadhachary, Arun S ; Williams, Paula ; Marbán, Eduardo ; Vega, Melisa ; Sarwary, Omaid ; Taylor, Michael D ; Evans, Maya ; Edmondson, Angie ; Rybalsky, Irina ; Jhawar, Sanjay ; Wells, Julie ; Kusmik, Kyle ; Van Eyk, Jennifer ; Janas, Joanne ; Harmelink, Matthew M ; Ruckdeschel Smith, Rachel ; Baek, Jayoon ; Butterfield, Russell ; Rodriguez, Aixa ; Marbán, Linda ; Rehborg, Rebecca ; Hendrix, Suzanne ; Giordano, Anthony F. ; Filar, Lauri ; Finkel, Richard S ; Nicorici, Alina ; Goude, Erica ; Hogan, Nathaniel ; Newton, Andrew ; Wezel, Germaine ; Chouteau, Wendy ; Pyzik, Erika ; Abedi, Mehrdad ; Duke, Julie ; Tian, Cuixia ; Connolly, Anne ; Civitello, Matthew ; Anderson, Kristan ; Johnson, Hannah ; Rogers, Russell G. ; Muntoni, Francesco ; Joyce, Nanette C. ; Brown, Michelle ; Anthonisen, Colleen

BACKGROUNDCardiosphere-derived cells (CDCs) ameliorate skeletal and cardiac muscle deterioration in experimental models of Duchenne muscular dystrophy. The HOPE-2 trial examined the safety and efficacy of sequential intravenous infusions of human allogeneic CDCs in late-stage Duchenne muscular dystrophy.METHODSIn this multicentre, randomised, double-blind, placebo-controlled, phase 2 trial, patients with Duchenne muscular dystrophy, aged 10 years or older with moderate upper limb impairment, were enrolled at seven centres in the USA. Patients were randomly assigned (1:1) using stratified permuted blocks to receive CAP-1002 (1·5 × 10⁸ CDCs) or placebo intravenously every 3 months for a total of four infusions. Clinicians, caregivers, patients, and clinical operations personnel were fully masked to treatment groups. The primary outcome was the change in mid-level elbow Performance of Upper Limb version 1.2 (PUL 1.2) score at 12 months, assessed in the intention-to-treat population. Safety was assessed in all individuals who received an investigational product. This trial is registered with ClinicalTrials.gov, NCT03406780.FINDINGSBetween March 1, 2018, and March 31, 2020, 26 male patients with Duchenne muscular dystrophy were enrolled, of whom eight were randomly assigned to the CAP-1002 group and 12 to the placebo group (six were not randomised due to screening failure). In patients who had a post-treatment PUL 1.2 assessment (eight in the CAP-1002 group and 11 in the placebo group), the mean 12-month change from baseline in mid-level elbow PUL1.2 favoured CAP-1002 over placebo (percentile difference 36·2, 95% CI 12·7-59·7; difference of 2·6 points; p=0·014). Infusion-related hypersensitivity reactions without long-term sequelae were observed in three patients, with one patient discontinuing therapy due to a severe allergic reaction. No other major adverse reactions were noted, and no deaths occurred.INTERPRETATIONCAP-1002 cell therapy appears to be safe and effective in reducing deterioration of upper limb function in patients with late-stage Duchenne muscular dystrophy. Various measures of cardiac function and structure were also improved in the CAP-1002 group compared with the placebo group. Longer-term extension studies are needed to confirm the therapeutic durability and safety of CAP-1002 beyond 12 months for the treatment of skeletal myopathy and cardiomyopathy in Duchenne muscular dystrophy.FUNDINGCapricor Therapeutics.

2020-07-01·Basic Research in Cardiology

Allogeneic cardiosphere-derived cells (CAP-1002) in critically ill COVID-19 patients: compassionate-use case series

Article

作者: Zaman, Tanzira ; Makkar, Raj R ; Singh, Siddharth ; Akhmerov, Akbarshakh ; Chen, Peter ; Friedman, Oren ; Chakravarty, Tarun ; Marbán, Eduardo ; Ebinger, Joseph E ; Gheorghiu, Mitch ; Falk, Jeremy ; Marbán, Linda

AbstractThere are no definitive therapies for patients with severe acute respiratory syndrome coronavirus-2 (SARS-CoV-2) infection. Therefore, new therapeutic strategies are needed to improve clinical outcomes, particularly in patients with severe disease. This case series explores the safety and effectiveness of intravenous allogeneic cardiosphere-derived cells (CDCs), formulated as CAP-1002, in critically ill patients with confirmed coronavirus disease 2019 (COVID-19). Adverse reactions to CAP-1002, clinical status on the World Health Organization (WHO) ordinal scale, and changes in pro-inflammatory biomarkers and leukocyte counts were analyzed. All patients (n = 6; age range 19–75 years, 1 female) required ventilatory support (invasive mechanical ventilation, n = 5) with PaO2/FiO2 ranging from 69 to 198. No adverse events related to CAP-1002 administration were observed. Four patients (67%) were weaned from respiratory support and discharged from the hospital. One patient remains mechanically ventilated as of April 28th, 2020; all survive. A contemporaneous control group of critically ill COVID-19 patients (n = 34) at our institution showed 18% overall mortality at a similar stage of hospitalization. Ferritin was elevated in all patients at baseline (range of all patients 605.43–2991.52 ng/ml) and decreased in 5/6 patients (range of all patients 252.89–1029.90 ng/ml). Absolute lymphocyte counts were low in 5/6 patients at baseline (range 0.26–0.82 × 103/µl) but had increased in three of these five patients at last follow-up (range 0.23–1.02 × 103/µl). In this series of six critically ill COVID-19 patients, intravenous infusion of CAP-1002 was well tolerated and associated with resolution of critical illness in 4 patients. This series demonstrates the apparent safety of CAP-1002 in COVID-19. While this initial experience is promising, efficacy will need to be further assessed in a randomized controlled trial.

107

项与 Deramiocel 相关的新闻（医药）

2024-10-19

·药明康德

使94%患儿病情稳定的基因疗法；缓解持续三年的细胞疗法…… | CGT周报

▎药明康德内容团队编辑近期，全球细胞和基因疗法（CGT）领域迎来系列进展。美国FDA批准的首款脑性肾上腺脑白质营养不良（CALD）基因疗法Skysona的长期研究结果积极，中位随访时间超过6年，94%患者的神经功能没有下降。用于治疗杜氏肌营养不良（DMD）的细胞疗法deramiocel在一项临床试验中使患者的心脏和骨骼肌功能在三年期间趋于稳定，其上市申请有望在今年年底前完成递交。首个进入人体临床试验的工程化B细胞疗法ISP-001的初步结果积极，首位接受治疗的1型黏多糖贮积症（MPS I）患者已停止使用标准疗法。本文将节选其中部分重要进展做简单介绍，仅供读者参阅。图片来源：123RF ———✦研发进展✦——— ◇ 近日，《新英格兰医学杂志》（NEJM）上刊登了两篇论文，公布了美国FDA批准的首款脑性肾上腺脑白质营养不良基因疗法Skysona（elivaldogene autotemcel，也称为eli-cel）的长期随访结果。首批在临床试验中接受eli-cel治疗的CALD患儿的中位随访时间已超过6年。长期疗效数据显示，eli-cel能够控制这种神经系统疾病的进展，94%患者的神经功能没有下降，超过80%的患者没有出现严重残疾。 CALD是一种罕见的X染色体连锁遗传性脑部疾病，主要发生于男孩。该病患儿因ABCD1基因突变导致过氧化物酶体功能缺失，大量异常的极长链脂肪酸（VLCFAs）会沉积于大脑白质和肾上腺，从而导致患儿进行性、不可逆的神经功能丧失，最终过早死亡。Eli-cel利用Lenti-D慢病毒载体，在体外将ABCD1基因的功能性拷贝导入到患者自身的造血干细胞中，再输回到患者体内，从而促进VLCFAs分解、避免毒性积累。 ◇ Capricor Therapeutics公司公布了其主打细胞疗法deramiocel（CAP-1002）治疗不能行动的晚期DMD患者的HOPE-2开放标签扩展（OLE）试验的三年积极结果。HOPE-2试验达成其主要终点，即在测试上肢功能的指标PUL 1.2上，接受CAP-1002治疗的病患有显著疗效（2.6分差异，P=0.014）。OLE试验的分析显示，接受deramiocel治疗患者的心脏和骨骼肌功能在三年期间趋于稳定。Capricor预计在今年年底前完成递交该疗法用于治疗DMD相关心肌病患者的生物制品许可申请（BLA）。 CAP-1002是一种同种异体的细胞疗法，它们由心脏来源细胞（CDCs）组成，CDCs是一种含有心脏祖细胞的特殊细胞群。它们通过释放包含微RNA、非编码RNA和蛋白的外泌体，改善DMD患者肌肉瘢痕或纤维化以及心脏功能。CAP-1002还显示出有效的免疫调节活性，可能促进细胞再生。目前美国FDA已授予CAP-1002再生医学先进疗法认定与孤儿药资格。 ◇ MeiraGTx公司公布了在研帕金森病基因疗法AAV-GAD在随机双盲、安慰剂对照临床试验MGT-GAD-025中的顶线数据。在这项试验中，帕金森病患者接受双侧丘脑底核AAV-GAD输注或安慰剂。初步数据显示，AAV-GAD达到了安全性和耐受性的主要终点。此外，接受AAV-GAD治疗26周后，患者在运动能力和生活质量方面均获得统计显著并具有临床意义的改善。 AAV-GAD是一款在研基因疗法，能将编码谷氨酸脱羧酶（GAD）的转基因递送至丘脑底核，以增加人脑中的主要抑制性神经递质GABA的产生。GAD是GABA合成中的限速酶，因此通过基因疗法增加丘脑底核GAD的表达，将导致运动回路正常化，并改善帕金森病的症状，同时不影响其他可能造成现有疗法并发症的大脑区域。 ◇ Beacon Therapeutics公司公布了其在研基因疗法AGTC-501在治疗X连锁视网膜色素变性（XLRP）的2期临床试验SKYLINE中获得的积极结果。在接受治疗24个月后，接受高剂量AGTC-501治疗的眼睛的缓解率达到57%。接受低剂量AGTC-501治疗的眼睛的缓解率为25%。AGTC-501表现出良好的安全性和耐受性，治疗伴发不良事件的大多为轻度或中度。 XLRP是一种遗传性单基因隐性疾病，会导致男孩和年轻男性视力逐渐丧失。XLRP主要由视网膜色素变性GTPase调节剂（RPGR）基因突变引起。AGTC-501表达全长RPGR蛋白，从而解决XLRP引起的感光细胞损伤，包括视杆细胞和视锥细胞的丧失。图片来源：123RF ◇ Immusoft公司公布了首位接受其工程化B细胞疗法ISP-001治疗的1型黏多糖贮积症患者的积极结果。根据新闻稿，ISP-001是首个进入人体临床试验的工程化B细胞疗法。该疗法使用非病毒载体将表达α-L-艾杜糖苷酶的转基因引入B细胞中，能够让经过改造的B细胞长期大量生产治疗性蛋白，从而改善患者症状。初步数据显示，该患者的疾病生物标志物指标获得改善，在接受一次ISP-001治疗8个月后，已停止使用标准酶替代疗法（ERT）。 ◇ 中国国家药监局药品审评中心（CDE）官网最新公示，合源生物纳基奥仑赛注射液获批一项临床试验默示许可，针对适应症为难治性系统性红斑狼疮相关的免疫性血小板减少症（SLE-ITP）。纳基奥仑赛为靶向CD19嵌合抗原受体自体T（CD19 CAR-T）细胞注射液。根据合源生物新闻稿，这也是该产品在自身免疫性疾病治疗领域获得的第一张IND批件。今年7月，《新英格兰医学杂志》在线发表了纳基奥仑赛治疗SLE-ITP的案例报道。1名此前曾接受过大量治疗的SLE-ITP女性患者在接受纳基奥仑赛注射液回输后的第28天，血液中的CD19阳性B细胞被清除，第三个月时B细胞获得重建。患者的血小板计数在治疗后的六个月内持续上升，并达到临床完全缓解。此外，患者停用了糖皮质激素和所有免疫抑制剂。 ◇ 原天生物1类生物制品新药YT001注射液在中国获批临床，拟开发治疗膝骨关节炎（KOA）。根据原天生物新闻稿介绍，YT001是其研发的一款间充质干细胞治疗产品。间充质干细胞具有免疫调节作用，能够缓释炎症反应，促进损伤修复，其免疫调节和损伤修复功能可帮助调节关节腔内紊乱微环境，缓解软骨的降解。 ◇ 因诺惟康宣布，其自主研发的可玻璃体内给药的基因疗法IVB103注射液获得了CDE的临床许可，旨在治疗新生血管性年龄相关性黄斑变性（nAMD）。IVB103通过该公司的EASI-FIND腺相关病毒（AAV）发现平台开发，具有“best-in-class”的潜力。今年7月，IVB103注射液获得了FDA的IND许可，用于同样的适应症。 ◇ 瑞风生物申报的RM-101注射液在中国获批临床，拟开发用于治疗USH2A基因外显子13突变所致的Usher综合征2型相关视网膜色素变性（RP）或非综合征型RP。Usher综合征是与视网膜色素变性有关的最常见综合征，USH2A基因突变导致视网膜色素变性，患者通常在青少年时期逐渐出现夜盲和周边视野缺失，最终可能导致全盲。根据瑞风生物公开资料介绍，RM-101是基于AAV的基因编辑类药物，能特异靶向USH2A基因转录的RNA，调控可变剪接生物学过程，诱导功能正常蛋白的表达恢复。该产品通过视网膜下注射给药，有望实现一次性给药并长期有效。 ◇ 恒瑞医药子公司瑞宏迪医药申报的1类新药RGL-193注射液在中国获批临床，拟开发治疗帕金森病。公开资料显示，RGL-193是瑞宏迪医药针对帕金森病自主研发的一款AAV双基因药物，采用立体定向技术注入患者脑内。它可以提高左旋多巴的转化效率，同时还起到保护和修复受损多巴胺能神经元的作用，具有潜在的延缓病情进展、减少口服抗帕金森病药量的效果。 ◇ 艺妙医疗申报的CAR-T细胞注射液IM96在中国获批临床，拟开发用于治疗结直肠癌。根据艺妙医疗新闻稿介绍，IM96是其与北京大学肿瘤医院合作开发用于治疗转移性结直肠癌的全新一代抗肿瘤药物，是一款靶向鸟苷酸环化酶2C（GUCY2C）的CAR-T疗法。该产品的临床前研究数据于今年6月入选美国临床肿瘤学会（ASCO）年会，研究显示IM96针对结直肠癌的最佳客观缓解率（ORR）达到50%，且仅有5.0%的患者出现神经毒性和≥3级的细胞因子释放综合征（CRS）。 ———✦融资、合作、M&A✦——— 图片来源：123RF ◇ Tolerance公司宣布完成了1720万美元的种子轮融资，本轮融资由Columbus Venture Partners领投，Criteria Bio Ventures、Sessa Capital、BioAdvance、Ben Franklin Technology Partners等投资者参投。该公司正在开发一种以同种异体或“现货型”胸腺诱导多能干细胞（iPSC）为基础的细胞疗法平台和胸腺疗法，旨在延缓和预防人体免疫系统中的重要器官胸腺的衰退，并在胸腺功能丧失时恢复其功能，以治疗由免疫耐受异常引起的免疫介导疾病，包括癌症、自身免疫疾病、移植排斥、感染、免疫缺陷和过敏。 ◇ 睿健医药宣布完成超亿元B轮融资，该资金将用于加速其帕金森病治疗产品NouvNeu001/NouvNeu003，以及眼科治疗产品NouvSight001的中美临床推进，加强公司多款创新管线的研发推进和临床能力的搭建，以及加大公司产业化建设的力度。 NouvNeu001是一款源于iPSC，化学诱导分化的多巴胺能神经元前体细胞。新闻稿指出，NouvNeu001是帕金森领域中，全球首个进入临床阶段的基于化学诱导的通用型细胞治疗产品，其IND申请已在中美两地获批。其早发型帕金森产品NouvNeu003的1期临床试验也即将完成剂量拓展阶段，实现睿健医药覆盖帕金森病全病程的重要一步。NouvSight001是睿健医药团队依托公司独特的“AI+化学诱导”平台开发的首款iPSC来源眼科通用型细胞治疗产品，旨在针对视网膜色素变性系列适应症。该产品于今年3月获得了美国FDA授予的孤儿药资格。 ◇ Eterna Therapeutics公司与Factor Bioscience公司宣布达成一项独家许可与合作协议，旨在加速开发治疗肿瘤、罕见病和自身免疫疾病的候选细胞疗法。根据协议条款，Eterna公司获得了一项全球独家许可，利用Factor公司的细胞重编程和基因编辑技术，开发某些基于iPSC的细胞疗法产品，尤其是iPSC衍生的间充质干细胞（iMSC），该细胞经过工程化处理可表达某些细胞因子。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料（可上下滑动查看） [1] Florian Eichler, et al., (2024). Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy. The N Engl J Med, DOI: 10.1056/NEJMoa2400442 [2] Christine N. Duncan, et al., (2024). Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy. The N Engl J Med, DOI: 10.1056/NEJMoa2405541 [3] Capricor Therapeutics Announces Positive Long-Term Data from HOPE-2 OLE Study in Duchenne Muscular Dystrophy at 2024 World Muscle Society Congress. Retrieved October 11, 2024 from https://www.globenewswire.com/news-release/2024/10/11/2962050/0/en/Capricor-Therapeutics-Announces-Positive-Long-Term-Data-from-HOPE-2-OLE-Study-in-Duchenne-Muscular-Dystrophy-at-2024-World-Muscle-Society-Congress.html [4] Beacon Therapeutics Announces Positive 24-Month Data from Phase 2 SKYLINE Trial of AGTC-501 in Patients with X-Linked Retinitis Pigmentosa. Retrieved October 15, 2024, from https://www.prnewswire.com/news-releases/beacon-therapeutics-announces-positive-24-month-data-from-phase-2-skyline-trial-of-agtc-501-in-patients-with-x-linked-retinitis-pigmentosa-302275688.html [5] MeiraGTx Announces Positive Data from Randomized, Sham-controlled Clinical Bridging Study of AAV-GAD for the Treatment of Parkinson’s Disease. Retrieved October 15, 2024, from https://investors.meiragtx.com/news-releases/news-release-details/meiragtx-announces-positive-data-randomized-sham-controlled [6] Immusoft to Announce Positive Phase 1 Data for First Engineered B Cell Therapy in a Clinical Trial. Retrieved October 15, 2024, from https://www.prnewswire.com/news-releases/immusoft-to-announce-positive-phase-1-data-for-first-engineered-b-cell-therapy-in-a-clinical-trial-302255551.html [7] 合源生物纳源瑞达®新增自身免疫性疾病适应症新药临床试验（IND）申请获默示许可. Retrieved October 11, 2024, from https://mp.weixin.qq.com/s/9-w9DlWNPKFMdZ8KjxGz7g [8]中国国家药监局药品审评中心（CDE）官网. Retrieved October 13, 2024, from https://www.cde.org.cn/main/xxgk/listpage/4b5255eb0a84820cef4ca3e8b6bbe20c [9] Tolerance Bio, Inc. Launches With $17.2 Million in Seed Financing to Advance Thymus-Based Therapies for Immune-Mediated Diseases. Retrieved October 18, 2024, from https://www.businesswire.com/news/home/20241008134130/en/Tolerance-Bio-Inc.-Launches-With-17.2-Million-in-Seed-Financing-to-Advance-Thymus-Based-Therapies-for-Immune-Mediated-Diseases#:~:text=Tolerance%20Bio%2C%20Inc.%20Launches%20With%20%2417.2%20Million%20in,manipulation%20platforms%20to%20prevent%20and%20treat%20immune%20diseases [10] 睿健医药完成超亿元B轮融资，加速国际化进程. Retrieved October 18, 2024, from https://mp.weixin.qq.com/s/gIxbOkl79mn2_OSK0bAs1w [11] Eterna Therapeutics and Factor Bioscience Announce Exclusive License and Collaboration Agreement to Accelerate Cell Therapy Development for Oncology, Autoimmune, and Rare Diseases. Retrieved October 18, 2024, from https://www.globenewswire.com/news-release/2024/10/17/2964799/0/en/Eterna-Therapeutics-and-Factor-Bioscience-Announce-Exclusive-License-and-Collaboration-Agreement-to-Accelerate-Cell-Therapy-Development-for-Oncology-Autoimmune-and-Rare-Diseases.html [12] 因诺惟康IVB103注射液获得CDE临床许可. Retrieved October 18, 2024, from https://mp.weixin.qq.com/s/OU_bxfT9u4a_-Cs6WreoDQ 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。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：本文来自药明康德内容团队，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

孤儿药基因疗法细胞疗法临床研究

2024-10-18

·GlobeNewswire-Health Care

Capricor Therapeutics Announces Closing of Underwritten Public Offering and Full Exercise of the Underwriters’ Option to Purchase Additional Shares

SAN DIEGO, Oct. 18, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, announced today the closing of its previously announced underwritten public offering of 5,073,800 shares of its common stock, including the exercise in full of the underwriters’ option to purchase additional shares to cover over allotments, bringing the total gross proceeds to Capricor from the offering to approximately $86.3 million, before deducting underwriting commissions and other offering expenses payable by Capricor. All of the shares of common stock sold in the offering, including the over-allotment, were offered by Capricor. The closing of the offering occurred on October 18, 2024. Piper Sandler & Co. and Oppenheimer & Co. Inc. acted as the joint book-running managers for the offering. The Company intends to use the net proceeds from this offering on the continued development of its product candidates, manufacturing of its product candidates, working capital and general corporate purposes. The securities were offered by the Company pursuant to an effective shelf registration statement on Form S-3 (File No. 333-280229) that was originally filed with the Securities and Exchange Commission (“SEC”) on June 14, 2024 and declared effective on October 16, 2024. The offering was made only by means of a prospectus and related prospectus supplement. A preliminary prospectus supplement relating to the offering was filed with the SEC on October 16, 2024. The final prospectus supplement relating to and describing the terms of the offering was filed with the SEC on October 17, 2024 and is available on the SEC's website at www.sec.gov. Electronic copies of the prospectus supplement and the accompanying prospectus may also be obtained from Piper Sandler & Co. at 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, Attention: Prospectus and Oppenheimer & Co. Inc. at Attention: Syndicate Prospectus Department, 85 Broad Street, 26th Floor, New York, NY 10004, by telephone at (212) 667-8055, or by email at EquityProspectus@opco.com. This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction. About 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. Capricor is also harnessing the power of its exosome technology, using its 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. Cautionary Note Regarding Forward-Looking Statements 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; potential future agreements; 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 June 30, 2024, as filed with the Securities and Exchange Commission on August 8, 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. 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. For more information, please contact: Capricor Media Contact:Raquel ConaKCSA Strategic Communications rcona@kcsa.com212.896.1204 Capricor Company Contact:AJ Bergmann, Chief Financial Officerabergmann@capricor.com 858.727.1755

临床3期细胞疗法

2024-10-17

·Endpoints

Viatris, Lexicon ink licensing deal; Capricor’s $75M offering

Plus, news about Kezar Life Sciences and Concentra Biosciences: Viatris, Lexicon Pharmaceuticals sign licensing deal for cardio drug: Per the agreement , Viatris will pay $25 million upfront to Lexicon in exchange for the rights to sotagliflozin in all markets outside of the US and Europe. Lexicon may also receive regulatory- and sales-based milestones and tiered royalties. The FDA last year approved the drug to reduce the risk of cardiovascular death, hospitalization for heart failure, and urgent heart failure. — Katherine Lewin Capricor Therapeutics prices $75M public stock offering: The rare disease biotech is selling 4.4 million of its shares at $17.00 apiece. The funds are set to go toward the development and manufacturing of its investigational drugs, as well as “working capital and general corporate purposes.” Capricor’s lead candidate, deramiocel, is a cell therapy currently in Phase 3 development for the treatment of Duchenne muscular dystrophy. Earlier this month, Capricor announced positive longer-term data from the ongoing HOPE-2 trial and that it had filed for approval with the FDA. — Katherine Lewin Kezar Life Sciences rejects Concentra’s unsolicited offer: The Bay Area biotech’s board said “no” to Kevin Tang’s Concentra Biosciences, which had offered $1.10 per share $KZR and 80% of potential asset sale proceeds to buy the company. Concentra made the offer last week after Kezar stopped a Phase 2b in lupus nephritis. On Thursday, Kezar also reiterated that it expects to have Phase 2a data in autoimmune hepatitis in the first half of next year. Kezar’s stock was down about 10% on Thursday morning. — Kyle LaHucik

引进/卖出临床2期细胞疗法临床3期临床结果

100 项与 Deramiocel 相关的药物交易

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研发状态

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
杜氏肌营养不良症	申请上市	美国	Capricor Therapeutics, Inc.	2024-10-09
左心室功能不全	临床1期	美国	Capricor, Inc.	2012-11-13
心肌梗塞	临床前	美国	Capricor, Inc.	2012-11-13
新型冠状病毒感染	药物发现	美国	Capricor Therapeutics, Inc.	2020-11-01
心脏衰竭	药物发现	美国	Medical University of South Carolina	2016-09-11
心肌疾病	药物发现	美国	Capricor, Inc.	2015-07-19
心肌梗塞	药物发现	美国	Capricor, Inc.	2012-11-13
肌营养不良	药物发现	美国	Lonza Houston, Inc.	-

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04428476 (HOPE-2 OLE, GlobeNewswire) 人工标引	临床2期	杜氏肌营养不良症	-	Deramiocel	鑰窪鬱艱繭淵築網艱願(壓遞艱積淵獵觸鹽鑰膚) = 遞繭憲築餘範鹹獵網選範獵簾壓艱願顧淵淵壓 (膚製簾夢觸築構製廠構 )	积极	2024-10-11
NCT02941705 (RegressHFpEF, CTgov)	临床2期	射血分数保留型心力衰竭 \| 纤维化 \| 舒张期心力衰竭	27	Allogeneic Derived Cells (RECIEVED CELLS)	鹹願遞艱艱築衊遞鬱憲(糧齋觸艱壓餘齋窪齋鹽) = 衊夢鏇觸襯糧夢憲鑰鏇獵齋淵簾鬱窪鹽鑰製築 (艱壓餘衊襯淵壓壓蓋壓, 壓淵憲選鏇齋積顧鏇鏇 ~ 網遞襯艱鹹蓋製願觸廠) 更多	-	2024-07-25
				Placebo/Control Arm (CONTROL ARM)	鹹願遞艱艱築衊遞鬱憲(糧齋觸艱壓餘齋窪齋鹽) = 艱獵鬱鬱糧築夢選醖繭獵齋淵簾鬱窪鹽鑰製築 (艱壓餘衊襯淵壓壓蓋壓, 製蓋廠範製膚繭選製醖 ~ 醖繭鏇積鹹範鑰網廠窪) 更多
NCT04428476 (HOPE-2-OLE, Biospace) 人工标引	临床2期	杜氏肌营养不良症	20	CAP-1002	(鑰遞淵觸襯糧選積襯繭) = 顧鬱築製襯餘膚範選膚範窪構鹹鬱願觸範網衊 (範獵願醖鏇膚醖網膚築 )	积极	2024-06-04
				External Comparator	(鑰遞淵觸襯糧選積襯繭) = 鑰遞衊夢淵網窪蓋鬱獵範窪構鹹鬱願觸範網衊 (範獵願醖鏇膚醖網膚築 )
NCT01458405 (ALLSTAR, CTgov)	临床1/2期	左心室功能不全 \| 心肌梗塞	135	placebo (Randomized Treatment Cohort: Placebo)	壓製糧範壓鬱餘夢積簾(構蓋顧糧壓鏇獵廠蓋製) = 鬱壓簾壓製糧壓衊選築齋繭蓋鏇鬱鏇繭鏇廠鬱 (繭觸鬱鏇夢願選簾繭願, 壓築構餘築構觸獵壓獵 ~ 鹹壓蓋鹹積範壓範醖網) 更多	-	2024-04-09
				CAP-1002 (Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells)	壓製糧範壓鬱餘夢積簾(構蓋顧糧壓鏇獵廠蓋製) = 鏇壓襯窪膚糧蓋襯淵糧齋繭蓋鏇鬱鏇繭鏇廠鬱 (繭觸鬱鏇夢願選簾繭願, 鬱鏇網鏇簾繭遞憲淵鑰 ~ 鬱蓋製鬱壓齋糧壓鑰選) 更多
NCT05126758 (HOPE-2-OLE, MDA2024) 人工标引	临床3期	杜氏肌营养不良症	12	CAP-1002	(憲積網鬱顧醖繭繭觸獵) = 壓製衊糧齋襯繭簾製範窪製遞窪廠壓襯範齋網 (夢醖鹹選鏇範選醖衊願 ) 更多	积极	2024-03-03
NCT04428476 (Corporate Publications) 人工标引	临床2期	杜氏肌营养不良症	13	CAP-1002 (continuous CAP-1002)	積廠網網顧壓夢鑰襯選(鹽淵繭選構艱鏇齋窪觸) = CAP-1002, when used over time, is slowing DMD’s devastating effects and may be disease modifying. 廠鹽範鏇膚齋膚衊廠築 (遞範簾鏇網鏇壓構顧窪 ) 更多	积极	2022-06-27
				CAP-1002 (from placebo to CAP-1002)
NCT03406780 (HOPE-2, Pubmed \| Lancet) 人工标引	临床2期	杜氏肌营养不良症	19	CAP-1002	(窪蓋襯鹽醖夢構衊網積) = No other major adverse reactions were noted 襯夢鹽膚廠廠簾範構蓋 (獵製鬱襯繭襯鬱鑰齋醖 ) 更多	积极	2022-03-12
				Placebo
NCT02485938 (HOPE-Duchenne, Pubmed \| Neurology) 人工标引	临床1/2期	杜氏肌营养不良症	25	CAP-1002	齋廠遞夢艱遞繭範蓋築(蓋鏇淵夢廠窪願獵願構) = Mid-distal PUL improved at 12 months in 8 of 9 lower functioning CAP-1002 patients, and no controls 鑰積鏇壓簾鏇築選遞壓 (夢廠鹹鹽廠選遞醖夢願 )	积极	2019-02-19
				usual care
NCT02293603 (Biospace) 人工标引	临床1期	扩张型心肌病 \| 心脏衰竭	-	CAP-1002	(餘夢艱網糧壓網鬱蓋憲) = 壓壓襯壓觸襯艱鹹簾廠鏇構構鹽築遞鹽醖憲鑰 (範觸網製膚獵選鹽醖鹽 ) 更多	积极	2015-11-09