Sarepta's Elevidys shows benefit in older Duchenne patients

Sarepta Therapeutics (NASDAQ: SRPT) presented new data at the 31st Annual Congress of the World Muscle Society showing that its gene therapy Elevidys (delandistrogene moxeparvovec) produced statistically significant functional benefits in ambulatory Duchenne muscular dystrophy patients aged 8 to 12 over two years, compared to a well-matched external control group.

The analysis drew from the EMBARK and ENDEAVOR studies, comparing 25 treated patients against 99 external controls. Treated patients showed a 3.32-point improvement on the North Star Ambulatory Assessment (p=0.0022), a 0.041 rise/second improvement in time-to-rise-from-floor velocity (p=0.0019), and a 0.195 meters/second improvement in 10-meter walk run velocity (p=0.0114). The safety profile was consistent with prior data, with nausea and vomiting among the most common treatment-related adverse events.

A separate analysis of patients aged 2 to under 3, drawn from the ENDEAVOR and ENVOL studies, showed dystrophin expression levels at 12 weeks that were higher than those previously observed in patients aged 4 and older. Mean western blot results reached 93.9% of control in ENDEAVOR Cohort 6 (N=6) and 72.8% in ENVOL Cohort B (N=3). Sarepta noted that the use of Elevidys in patients under age 4 remains investigational and has not been evaluated by regulatory authorities.

"The older ambulatory patient data demonstrate that delandistrogene moxeparvovec treatment can provide functional benefit that's statistically significant at a stage when decline often becomes more pronounced," said Craig McDonald, M.D., professor and chair of the UC Davis Health Department of Physical Medicine and Rehabilitation and an investigator on both studies.

In the U.S., Elevidys is approved for ambulatory patients aged 4 and older with a confirmed mutation in the DMD gene. The drug carries a boxed warning for acute serious liver injury and acute liver failure, and is contraindicated in patients with deletions in exon 8 and/or exon 9 of the DMD gene.

You May Also Be Interested In

• Merck and Moderna report Phase 3 melanoma trial results
• RBC Capital Reiterates Sector Perform Rating on Merck (MRK)
• Carnival Corp. (CCL) Declares $0.15 Quarterly Dividend; 2.2% Yield