Dive Brief:

• Children with Duchenne muscular dystrophy who received Sarepta Therapeutics’ gene therapy Elevidys in a clinical trial continued to perform better on tests of motor function than historical data suggests they should, and the benefits appear to compound with time, the company said Monday.
• According to Sarepta, patients in the study, Embark, had greater reductions on three measures of function than a matched historical control group, with the gap “significantly widening” between two and three years after treatment. Doug Ingram, Sarepta’s CEO, said the data is an opportunity to “rebalance the discussion” surrounding Elevidys, sales of which have slowed amid safety concerns and newly restrictive labeling. 
• In research notes published Monday, multiple Wall Street analysts viewed the data as a positive development for the company. They also noted, though, that investors will be more focused on whether the results translate to sales growth. Sarepta shares, which have lost much of their value over the last year, rose by double digits in morning trading. 

Dive Insight:

The data Sarepta released Monday come from long-term follow-up from Elevidys’ pivotal study. 

In that study, Elevidys missed its main goal, which was to demonstrate an improvement in motor function, versus placebo recipients, after a year. However, Sarepta has continued to track people in the trial and compared their trajectory with historical data to see whether the benefits of treatment might better show up over a longer period of time.  

Sarepta is now claiming such an outcome. In a statement Monday, it said that the difference between Elevidys recipients and an external control group on a widely used measure of motor function had grown from a non-significant 0.7 points at 52 weeks to a statistically significant 4 points at three years. The gap between the two groups also grew on other evaluations, such as timed tests for walking or getting up from the floor.  

The results “mirror my observations personally, from my treated patients within the clinical setting, that the beneficial impacts of Elevidys really have been very tangible and measurable, and the distinction from my untreated patients is quite clear,” said Crystal Pound, the chief of neurology at Children’s Hospital of The King’s Daughters in Norfolk, Va, who spoke on a Monday conference call hosted by Sarepta.

Sarepta said that trial monitors have detected no new safety signals in the study, “reinforcing the consistent and manageable safety profile seen in ambulatory patients treated with Elevidys to date.” Elevidys treatment was associated with liver failure and the deaths of two drug recipients, leading to its temporary removal from market last summer and restricted use among sicker patients who’ve lost the ability to walk. 

Still, the controversy has impacted Elevidys’ use. Elevidys was projected to be a blockbuster, but Sarepta had to revise its forecasts and saw sales decline throughout the year. Earlier this month, Sarepta revealed that preliminary fourth quarter revenue shrank to $110 million, down from $132 million the previous quarter and $282 million in the three-month period before shipments were paused.

Analysts were skeptical on Monday that the new update might change the narrative surrounding Elevidys.

Feedback from treating physicians “suggests most patients that were initially eligible and waiting for treatment with Elevidys have been already provided the therapy, and that treatment going forward is likely to follow an incidence-like pattern of mostly newly diagnosed, and perhaps a few hesitant [patients and families],” wrote RBC Capital Markets analyst Brian Abrahams in a note to clients.

“While today's data do provide the company with some additional talking points in educating physicians, we would not foresee it dramatically changing clinician views around the therapy,” Abrahams wrote.

The data “reinforces” the durable benefits of Elevidys, but is likely to have a “modest” impact on sales, added William Blair analyst Sami Corwin.