Sarepta Stock Has Been Battered. Positive Gene Therapy Data Sends Shares 5% Higher. -- Barrons.com
Sarepta Stock Has Been Battered. Positive Gene Therapy Data Sends Shares 5% Higher. -- Barrons.com
DJ Sarepta Stock Has Been Battered. Positive Gene Therapy Data Sends Shares 5% Higher. -- Barrons.com
DJ Sarepta股票遭受重创。积极的基因治疗数据推动股价上涨5%。--Barrons.com
By Josh Nathan-Kazis
乔什·内森-卡齐斯著
Shares of rare-disease biotech Sarepta Therapeutics were up 5.4% in premarket trading on Friday after the company released data on Thursday that showed one of its experimental gene therapies remained effective two years after it was administered.
罕见疾病生物技术公司Sarepta Treeutics的股价周五盘前交易中上涨5.4%，此前该公司周四发布的数据显示，其一种实验性基因疗法在接受治疗两年后仍然有效。
The gene therapy, known as SRP-9003, treats a disorder called limb-girdle muscular dystrophy type 2E. The treatment is meant to cause the cells of patients suffering from the disease to manufacture a protein they cannot otherwise make.
这种名为SRP-9003的基因疗法治疗一种名为2E型肢体带状肌营养不良症。这种疗法的目的是使患有这种疾病的患者的细胞产生一种原本无法制造的蛋白质。
The company said the new data showed that expression of that protein was higher after two years than it had been after 60 days and that functional improvements in the patients were as good as they had been at 18 months. It's the first long-term data on a gene therapy for any form of muscular dystrophy, the company said, and will help ease concerns about the durability of the treatment, which remains an open question for most gene therapies.
该公司表示，新数据显示，两年后该蛋白的表达高于60天后，患者的功能改善与18个月时一样好。该公司表示，这是关于任何形式的肌营养不良症的基因疗法的第一个长期数据，将有助于缓解人们对治疗持久性的担忧，对于大多数基因疗法来说，这仍然是一个悬而未决的问题。
What's more, analysts said that the data was good news not only for SRP-9003, but also for Sarepta's (ticker: SRPT) other gene therapy programs, which have had a disastrous year.
更重要的是，分析人士说，这些数据不仅对SRP-9003是好消息，对Sarepta(股票代码：SRPT)的其他基因治疗项目也是好消息，这些项目经历了灾难性的一年。
Shares of Sarepta fell 50% in early January after the company announced that its Duchenne muscular dystrophy gene therapy had not successfully improved muscle strength in patients. The stock, which was trading at $168.95 on Jan. 7, fell to $82.29 on Jan. 8 and has not recovered since. It closed at $83 on Thursday.
Sarepta的股价在1月初下跌50%，此前该公司宣布其Duchenne肌营养不良症基因疗法未能成功改善患者的肌肉力量。该股在1月7日的交易价格为168.95美元，1月8日跌至82.29美元，此后一直没有回升。周四收报83美元。
"We believe today's LGMD results provided some much needed positive news for SRPT's gene therapy programs," SVB Leerink analyst Joseph P. Schwartz wrote in a note out late Thursday.
SVB Leerink分析师约瑟夫·P·施瓦茨(Joseph P.Schwartz)周四晚些时候在一份报告中写道：“我们相信，今天的LGMD结果为SRPT的基因治疗计划提供了一些亟需的积极消息。”
Muzho analyst Difei Yang wrote that the long-term efficacy of SRP-9003 could reaffirm the scientific hypotheses behind the Duchenne muscular dystrophy gene therapy, known as SRP-9001. "We believe the new SRP-9003 data, including sustained functional improvements relative to natural history, supports the hypothesis that gene therapy in muscular diseases like LGMD and DMD can enable functional benefits for patients," Yang wrote. "While additional placebo-controlled data is needed to fully validate this hypothesis, we believe these new data are nonetheless encouraging."
Muzho分析师Difei Yang写道，SRP-9003的长期疗效可能会重申Duchenne肌营养不良症基因疗法(SRP-9001)背后的科学假设。杨写道：“我们相信，新的SRP-9003数据，包括相对于自然历史的持续功能改善，支持了LGMD和DMD等肌肉疾病的基因治疗可以为患者带来功能益处的假设。”“虽然还需要更多的安慰剂对照数据来充分验证这一假设，但我们相信这些新数据还是令人鼓舞的。”
Sarepta shares were trading at $87.48 in premarket trading on Friday. The stock is down 51.3% so far this year, and 4.6% over the past 12 months.
在周五的盘前交易中，Sarepta的股价为87.48美元。该股今年迄今下跌51.3%，过去12个月累计下跌4.6%。
"This data is the first look at longer-term expression data with any gene therapy for muscular dystrophy," the company's chief scientific officer, Louise Rodino-Klapac, said in a statement. "The meaningful and sustained levels of beta-sarcoglycan protein expression at two years and continued strength of the functional outcomes measured are tremendously positive and support continued advancement of this investigational treatment for patients."
该公司首席科学官路易丝·罗迪诺-克拉帕克(Louise Rodino-Krapac)在一份声明中说：“这些数据是首次看到任何肌肉营养不良基因疗法的长期表达数据。”在两年内，有意义的和持续的β-肌聚糖蛋白表达水平和所测量的功能结果的持续强度是非常积极的，并支持继续推进这项针对患者的研究治疗。“
Write to Josh Nathan-Kazis at josh.nathan-kazis@barrons.com
写信给Josh Nathan-Kazis，电子邮件：josh.nathan-Kazis@Barrons.com
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March 19, 2021 08:26 ET (12:26 GMT)
2021年3月19日东部时间08：26(格林尼治标准时间12：26)
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