By Manas Mishra
Sarepta Therapeutics’ shares jumped 60 percent on Tuesday after promising results from a gene therapy study positioned the specialty drugmaker as a potential leader in the market for Duchenne muscular dystrophy (DMD) treatments
Results from Sarepta’s early-stage study that tested three DMD patients showed that they experienced an average 87 percent reduction in serum creatine kinase (CK) — an enzyme strongly associated with the muscle damage caused by DMD.
The data also showed that the level of micro-dystrophin – a protein that helps keep muscles intact – in patients was about 38 percent of normal levels, significantly higher than Wall Street’s expectations of 10 percent.
“I have been waiting my entire 49-year career to find a therapy that dramatically reduces CK levels and creates significant levels of dystrophin,” Jerry Mendell, the study’s principal investigator, said in a statement.
Analysts expressed optimism that Sarepta – which already has approval for a DMD treatment – could become a market leader in treating the disease if its experimental gene therapy is successful.
“Given today’s data, it is really hard to believe this study won’t be positive, ushering in a new paradigm that we expect will transform outcomes for patients diagnosed with this horrible disease,” said Brian Skorney, an analyst at Baird.
DMD is a rare, genetic disorder that hampers muscle movement mainly in men, affecting one in every 3,500 to 5,000 males.
A lack of dystrophin can cause life-threatening damage to the heart, and over time, death, often at a young age
Cambridge, Massachusetts-based Sarepta is among a clutch of drugmakers trying to develop successful gene therapies that hold promise for people with rare genetic diseases including DMD and spinal muscular atrophy.
The therapies, which aim to treat patients by manipulating genes at a cellular level, have received greater attention from companies and investors in recent years after high-profile failures in the late 1990s and early 2000s.
Just this year, Swiss drugmaker Novartis bought AveXis Inc for $8.7 billion, seeking to add a potentially lucrative gene therapy to its portfolio of rare-disease treatments.
Sarepta’s stock climbed as much as 67.7 percent to a record high of $176.60, before trimming gains to around 33 percent.
Shares of other companies developing gene therapies, including Solid Biosciences and Spark Therapeutics, also surged on Tuesday.
(Reporting by Manas Mishra in Bengaluru; Editing by Sai Sachin Ravikumar)
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