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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