Core Insights - Capricor Therapeutics announced positive top-line results from its pivotal Phase III HOPE-3 clinical trial for its investigational cell therapy Deramiocel in treating Duchenne Muscular Dystrophy (DMD), leading to a 371% increase in stock price to $29.96 on December 3, 2025 [1][5]. Group 1: Clinical Trial Results - The HOPE-3 trial was a randomized, double-blind, placebo-controlled study involving 106 patients with an average age of approximately 15 years [5][24]. - Deramiocel demonstrated a 54% reduction in disease progression in upper limb function (PUL v2.0) compared to placebo (p=0.029) and a 91% reduction in the decline of left ventricular ejection fraction (LVEF) (p=0.041) [6][11]. - The safety and tolerability of Deramiocel were consistent with previous clinical experiences [7][13]. Group 2: Regulatory Context - Capricor plans to respond to the Complete Response Letter (CRL) received from the FDA, believing that the HOPE-3 results, along with data from the HOPE-2 trial, will address the clinical questions raised by the FDA [10][15]. - The FDA previously rejected the approval based on the Phase II trial's inability to demonstrate efficacy for the pre-specified primary endpoint, but Capricor argues that the primary endpoint was indeed met with appropriate statistical methods [15]. Group 3: Implications for Patients - The results from HOPE-3 signify a new possibility for DMD patients, focusing on immune modulation to slow muscle and heart decline rather than gene repair or protein supplementation [17]. - While Deramiocel cannot reverse the disease, it may provide longer functional ability, more stable heart function, and improved daily life for patients [17]. Group 4: About Duchenne Muscular Dystrophy - Duchenne Muscular Dystrophy (DMD) is a severe X-linked genetic disorder characterized by progressive degeneration of skeletal, respiratory, and cardiac muscles, primarily affecting boys [18]. - Approximately 15,000 individuals in the U.S. are affected by DMD, with heart degeneration leading to cardiomyopathy and heart failure being the primary cause of death [18]. Group 5: About Deramiocel - Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs) known for their immune-modulating and anti-fibrotic properties, which have been shown to maintain cardiac and skeletal muscle function in DMD patients [21]. - The therapy has received orphan drug designation from the FDA and EMA for DMD treatment and has been recognized as a regenerative medicine advanced therapy (RMAT) in the U.S. [21]. Group 6: About Capricor Therapeutics - Capricor Therapeutics is a biotechnology company focused on advancing transformative cell and exosome therapies to redefine the treatment landscape for rare diseases, with Deramiocel being its leading product in late-stage clinical development for DMD [27].

Capricor Therapeutics (NasdaqCM:CAPR) Update / Briefing December 03, 2025 08:00 AM ET Company ParticipantsAJ Bergmann - CFONathan Hogan - Director of BiostatisticsCraig McDonald - Scientific AdvisorLinda Marban - CEOKristen Kluska - Managing DirectorCatherine Novack - DirectorJoe Pantginis - Director of Equity ResearchJohn Soslow - Professor of PediatricsConference Call ParticipantsNone - AnalystLeland Gershell - Managing Director and Senior AnalystAydin Huseynov - Equity Research AnalystBoobalan Pachaiyapp ...

新京报讯(记者戴轩)杜氏肌营养不良症(DMD)治疗领域迎来进展。今日(7月22日)，创新治疗药物伐莫洛 龙在首都医科大学附属北京儿童医院神经内科实现临床应用，这是该药物自获批进入中国市场后，在全 国医疗机构内开出的首张处方。 患儿悠悠在4岁时确诊DMD，之后通过服用泼尼松、地夫可特等糖皮质激素药物，开展运动康复进行治 疗。熊晖介绍，DMD患儿要长期服用激素，面临骨质疏松、不长个儿等副作用。伐莫洛龙拥有抗炎作 用，但副作用比糖皮质激素小，可以降低对于儿童生长发育方面的负面影响。 伐莫洛龙口服混悬于2024年12月获得国家药品监督管理局批准，用于治疗四岁及以上的DMD患者。 DMD在男性中完全外显，主要表现为进行性、对称性肌无力，近端重于远端。患儿通常表现为走路 晚，易摔倒，走路慢，到3岁左右步态异常开始变得明显；开始时运动发育仍不断进步，但逐渐表现出 爬楼、跑、跳和起立等行动的困难，并逐渐出现腰前凸、双侧腓肠肌假性肥大等体征，4-5岁过了平台 期之后，运动能力开始倒退，10-12岁丧失独立行走能力，需借助轮椅行动，往往在30岁前因呼吸系统 并发症或心力衰竭而死亡。 今年9岁的悠悠在5年前确诊DMD，之后一直通过 ...