Dive Brief:

简报：

Capricor Therapeutics shares soared more than 300% early Wednesday as investors bet a new study will be enough for the company to win approval of its experimental cell therapy for Duchenne muscular dystrophy.

Capricor Therapeutics的股价在周三早盘飙升超过300%，投资者押注一项新的研究将足以使该公司获得其针对杜氏肌营养不良症的实验性细胞疗法的批准。

The Food and Drug Administration in July

食品药品监督管理局七月

rejected Capricor’s initial application

拒绝了Capricor的初始申请

for the medicine, deramiocel, saying that more data was needed. After a meeting with regulators, Capricor announced in September that the agency was

对于药物deramiocel，称需要更多数据。在与监管机构会面后，Capricor于九月宣布该机构正在

willing to reconsider

愿意重新考虑

its decision with new information from an already completed clinical trial titled Hope-3.

它用来自一个已经完成的名为Hope-3的临床试验的新信息来决定。

Now,

现在，

freshly released results

新鲜出炉的结果

from that Phase 3 trial show deramiocel offers patients “significant skeletal and cardiac benefits.” Importantly, the study succeeded on two main goals, helping preserve both upper limb function and the heart’s ability to pump blood.

从该第 3 阶段试验结果显示，deramiocel 为患者提供了“显著的骨骼和心脏益处”。重要的是，该研究在两个主要目标上取得了成功，即帮助保持上肢功能和心脏泵血能力。

Dive Insight:

深入洞察：

Capricor far surpassed investor and analyst expectations with its results, according to Cantor Fitzgerald analyst Kristen Kluska. Before the announcement, sentiment was “skewing mixed or low.” Cantor’s discussions with investors found that those who were positive on the stock believed the company’s therapy would succeed in meeting one of the major endpoints, but not both, Kluska wrote in a note to clients..

据康托尔菲茨杰拉德分析师克里斯汀·克鲁斯卡称，Capricor 的业绩远超投资者和分析师的预期。克鲁斯卡在一份致客户的报告中写道，在公告发布之前，情绪“偏向混合或低落”。康托尔与投资者的讨论发现，那些对股票持乐观态度的人认为该公司的疗法将会成功达成其中一个主要终点，但并非两者全部。

The latest data represents a significant turnaround for Capricor, whose stock had been battered through much of the year and took a further dive following the July rejection. After closing at $6.36 on Tuesday, shares traded as high as $40 apiece early Wednesday. The settled back down, to hover around $25 apiece, by late morning..

最新的数据对Capricor来说是一个显著的转折，其股票在今年的大部分时间里一直受到重创，并在7月遭到拒绝后进一步暴跌。在周二以6.36美元收盘后，周三早盘股价一度高达每股40美元。到临近中午时，回落到每股约25美元。

If approved, deramiocel would be the first drug available to treat Duchenne cardiomyopathy, the leading cause of death in patients with the muscle-wasting disease. “We expect many physicians will elect to use this therapy, particularly given a strong safety profile,” Kluska wrote. Prior talks with doctors suggested they were intrigued by the treatment but needed to see more data, she added.

如果获得批准，deramiocel 将成为首个可用于治疗杜氏肌营养不良症心肌病的药物，而心肌病是这种肌肉消耗性疾病患者的主要死因。克拉丝卡写道：“我们预计许多医生会选择使用这种疗法，特别是鉴于其良好的安全性。”她补充道，此前与医生的对话表明，他们对该治疗很感兴趣，但需要看到更多数据。

“Today’s update provides this.”.

“今天的更新提供了这个。”

The next step for Capricor is to submit a response to the FDA’s earlier rejection letter incorporating the latest data. The company noted its “prior alignment” with the agency on the path forward and its belief that the results should be enough to support approval.

Capricor 下一步是向 FDA 提交针对先前拒绝信的回复，并纳入最新数据。该公司表示，其与该机构在前进路径上“先前已达成一致”，并认为这些结果应足以支持获批。

The FDA’s rejection in July took Capricor by surprise and drew the attention of

FDA在七月份的拒绝让Capricor感到意外，并引起了

The Wall Street Journal

《华尔街日报》

editorial board, which chastised the agency for “torpedoing promising treatments.” The paper criticized FDA official Vinay Prasad, who oversees gene and cell therapies, for pushing out veteran leaders who had helped shepherd deramiocel through the application process.

编辑委员会谴责该机构“扼杀有希望的治疗方法”。该报批评了负责基因和细胞疗法的FDA官员维奈·普拉萨德，指责他排挤那些曾帮助德拉米奥赛尔通过申请流程的老资格领导者。

Deramiocel is derived from special heart cells that have been shown to help preserve cardiac and skeletal muscle function. The drug has the potential to “meaningfully improve the course of Duchenne muscular dystrophy,” Capricor CEO Linda Marbán said in the company’s statement.

Deramiocel 源自特殊的心脏细胞，这些细胞已被证明有助于保护心脏和骨骼肌功能。Capricor 首席执行官琳达·马尔班在公司声明中表示，该药物有潜力“显著改善杜氏肌营养不良症的病程”。