Sarepta Still Strong On Gene Therapy As Muscular Dystrophy Treatment - (Investor's Business Daily via NewsPoints Desk)

  • JMP Securities analyst Liisa Bayko suggested that if approved, Sarepta Therapeutics' experimental Duchenne muscular dystrophy (DMD) gene therapy, called micro-dystrophin, could chip away at sales of Exondys 51 and other exon-skipping drugs in the company's pipeline, reported Investor's Business Daily.

  • "Management indicated the price for AveXis' (now Novartis') gene therapy for spinal muscular atrophy at $2 million is too low, so we bookmark it between $2 million and $8 million which represents the net present value of 15 years' worth of exon skipping," Bayko said.

  • According to the news source, Sarepta anticipates it will gain approval for the exon-skipping drug golodirsen by year's end, while it also sees approval for a similar drug called casimersen roughly nine months later. The company has indicated that it is likely micro-dystrophin will erode some of those sales.

  • Sarepta has some preclinical evidence showing there could be a benefit of using exon-skipping drugs prior to gene therapy, and it also sees exon-skipping drugs as monthly maintenance therapy following gene therapy.

  • "Either of these strategies would support substantial upside to our base case, where we assume the gene therapy cannibalizes revenues from the exon skippers," Bayko said.

  • The company will likely accelerate enrollment in its next micro-dystrophin study, which will dose 24 more children with DMD and test them for functional improvements at the year mark, she added.

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