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股票遭受重創。積極的基因治療數據推動股價上漲5%。--Barrons.com
By Josh Nathan-Kazis
喬什·內森-卡齊斯著
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.
罕見疾病生物技術公司Sarepta Treeutics的股價週五盤前交易中上漲5.4%，此前該公司週四發佈的數據顯示，其一種實驗性基因療法在接受治療兩年後仍然有效。
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.
這種名為SRP-9003的基因療法治療一種名為2E型肢體帶狀肌營養不良症。這種療法的目的是使患有這種疾病的患者的細胞產生一種原本無法制造的蛋白質。
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.
該公司表示，新數據顯示，兩年後該蛋白的表達高於60天后，患者的功能改善與18個月時一樣好。該公司表示，這是關於任何形式的肌營養不良症的基因療法的第一個長期數據，將有助於緩解人們對治療持久性的擔憂，對於大多數基因療法來説，這仍然是一個懸而未決的問題。
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，分析人士説，這些數據不僅對SRP-9003是好消息，對Sarepta(股票代碼：SRPT)的其他基因治療項目也是好消息，這些項目經歷了災難性的一年。
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的股價在1月初下跌50%，此前該公司宣佈其Duchenne肌營養不良症基因療法未能成功改善患者的肌肉力量。該股在1月7日的交易價格為168.95美元，1月8日跌至82.29美元，此後一直沒有回升。週四收報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分析師約瑟夫·P·施瓦茨(Joseph P.Schwartz)週四晚些時候在一份報告中寫道：“我們相信，今天的LGMD結果為SRPT的基因治療計劃提供了一些亟需的積極消息。”
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分析師Difei Yang寫道，SRP-9003的長期療效可能會重申Duchenne肌營養不良症基因療法(SRP-9001)背後的科學假設。楊寫道：“我們相信，新的SRP-9003數據，包括相對於自然歷史的持續功能改善，支持了LGMD和DMD等肌肉疾病的基因治療可以為患者帶來功能益處的假設。”“雖然還需要更多的安慰劑對照數據來充分驗證這一假設，但我們相信這些新數據還是令人鼓舞的。”
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.
在週五的盤前交易中，Sarepta的股價為87.48美元。該股今年迄今下跌51.3%，過去12個月累計下跌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."
該公司首席科學官路易絲·羅迪諾-克拉帕克(Louise Rodino-Krapac)在一份聲明中説：“這些數據是首次看到任何肌肉營養不良基因療法的長期表達數據。”在兩年內，有意義的和持續的β-肌聚糖蛋白表達水平和所測量的功能結果的持續強度是非常積極的，並支持繼續推進這項針對患者的研究治療。“
Write to Josh Nathan-Kazis at josh.nathan-kazis@barrons.com
寫信給Josh Nathan-Kazis，電子郵件：josh.nathan-Kazis@Barrons.com
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March 19, 2021 08:26 ET (12:26 GMT)
2021年3月19日東部時間08：26(格林尼治標準時間12：26)
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