DJ Sarepta Stock Has Been Battered. Positive Gene Therapy Data Sends Shares 5% Higher. -- Barrons.com
By Josh Nathan-Kazis
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.
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.
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.
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.
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.
"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.
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."
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.
"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."
Write to Josh Nathan-Kazis at josh.nathan-kazis@barrons.com
(END) Dow Jones Newswires
March 19, 2021 08:26 ET (12:26 GMT)
Copyright (c) 2021 Dow Jones & Company, Inc.