Sarepta Therapeutics Inc. (NASDAQ:SRPT) is a commercial-stage biotechnology company engaged in developing unique RNA-targeted therapies and gene therapies for treating rare diseases. It is currently working on a wide range of disorders such as Duchenne muscular dystrophy (DMD), Limb-girdle muscular dystrophies (LGMDs), and Mucopolysaccharidosis, among others. One of its key programs include SRP-9001, which is an investigational gene transfer therapy for curing DMD.
Most of its stock movement over the past year was driven by developments related to different therapies in its pipeline, particularly SRP-9001. The company announced positive safety and efficacy data for SRP-9001 in June. The Journal of the American Medical Association (JAMA), which published its clinical trial results, suggested back then that the therapy is expected to become a safe treatment for children with DMD. One month later, the U.S. Food and Drug Administration (FDA) granted fast track designation (FTD) and rare pediatric disease (RPD) designation to SRP-9001.
Sarepta in September disclosed 2 years follow up results from four participants with DMD who received SRP-9001. The company reported that “Trial participants exhibited a mean 7.0-point improvement on the North Star Ambulatory Assessment (NSAA) compared to baseline.”
The company last week released another update related to SRP-9001. Sarepta reported that a controlled trial of gene therapy did not achieve statistical significance on the primary functional endpoint. SRPT stock lost about half its value in the previous trading session following the update.
Several research firms downgraded Sarepta over its disappointing clinical trial results. For instance, JPMorgan cut its ratings for the stock from “Outperform” to “Underperform” on Friday. Separately, Barclays lowered its price target for SRPT from $192 per share to $125 per share.
Sarepta stock marginally rebounded in the current trading session from Friday’s sharp dive. At 1:23 p.m. ET, its stock rose 9.90 percent to $90.44 on a heavy volume of approx. 7.8 million shares, as compared to the daily average volume of about 1.14 million shares.