Sarepta Therapeutics shares pop on “significant milestone” in muscular dystrophy drug trial
Sarepta Therapeutics (NASDAQ:SRPT) shares soared to a yearly high Wednesday, as it announced that its Duchenne Muscular Dystrophy (DMD) drug eteplirsen trial results could lead to “first-ever opportunities” in the treatment and underlying causes of the disease.
The company said that treatment with its lead DMD drug candidate, eteplirsen, met the primary efficacy endpoint of an increase in novel dystrophin. Dystrophin deficiency has been definitively established as one of the root causes of muscular dystrophy.
Sarepta also said that the trials achieved a “significant clinical benefit” on the primary clinical outcome, the 6-minute walk test, over the placebo/delayed treatment in a phase IIb extension trial in DMD patients.
In recent years, the 6-minute walk test has been adapted to evaluate functional capacity in neuromuscular diseases and has served as the basis for regulatory approval of a number of drugs for rare diseases.
Shares of the company surged up 175 per cent, or $26.37, on the news as at about 10:40 a.m., trading at $41.51.
“We are extremely excited about these data, as they demonstrate that longer-term treatment with eteplirsen is translating to continued and unprecedented increases in both dystrophin production and clinical benefit across various subgroups of DMD patients involved in this study,” said president and CEO Chris Garabedian.
"On a broader scale, these results signify the promise and tremendous potential of our RNA-based technology to impact and modulate disease at the genetic level, which may lead to first-ever opportunities to target serious and life-threatening rare conditions at the origin of disease.”
Duchenne muscular dystrophy is a rare, degenerative neuromuscular disorder causing severe, progressive muscle loss and a premature death. One of the most common fatal genetic disorders, it affects roughly one in every 3,500 boys worldwide.
Sarepta said that eteplirsen administered once weekly at either 30 mg/kg or 50 mg/kg for 48 weeks resulted in a “statistically significant” increase in dystrophin-positive fibers to 47.0 per cent of normal, compared to 38 per cent in the placebo/delayed treatment cohort.
Dr. Jerry Mendell, director of the Centers for Gene Therapy and Muscular Dystrophy at Nationwide Children's Hospital, and principal investigator of the phase IIb study, said that the data represents a “significant milestone and a defining moment of progress and hope” for DMD patients.
“By addressing the underlying cause of DMD, eteplirsen has demonstrated unparalleled effects on enabling dystrophin production and slowing the progression of the disease as measured by the 6-minute walk test, with no treatment associated adverse events," he added.
“While eteplirsen is targeted to DMD patients with a specific genetic mutation, I think the implications for all DMD patients with related genetic mutations are clearly evident.”
The company said that the phase IIb eteplirsen study included 12 boys between seven and 13 years of age and was conducted at Nationwide Children's Hospital in Columbus, Ohio over the course of the 24 weeks.
Sarepta Therapeutics is focused on developing RNA-based therapeutics to improve and save the lives of people affected by serious and life-threatening rare and infectious diseases.
The company's pipeline includes its lead program eteplirsen, for DMD, as well as potential treatments for some of the world's most lethal infectious diseases.