Sarepta Therapeutics Inc Q1 2021 Earnings Call Transcript

The following is excerpted from the question-and-answer section of the transcript.

Question: Gil Joseph Blum - Needham & Company, LLC, Research Division - Analyst : Congratulations on your progress on the pipeline. So we've discussed the potential for accelerated approval in the U.S. for SRP-5051. Maybe going on the global aspect of this, would there be challenges in enrolling a randomized study for SRP-5051, assuming a placebo control, for example?

Question: Gil Joseph Blum - Needham & Company, LLC, Research Division - Analyst : Absolutely. So when thinking of a global study for SRP-5051, could there be potential challenges in enrollment if there's a placebo arm?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : Congrats on the quarter. So on 9001, you mentioned that the linear qPCR titering method is achieving expression levels which were greater than you saw in Part 1 of Study 102. Looking at my notes, I think you said that you saw no correlation between dystrophin expression and function in Part 1. So can you confirm whether you saw a threshold relationship with expression levels? And could you maybe put some levels around that? And then just another quick one. Could -- is there any interest on your part in pursuing a filing strategy with the FDA based on the 4- to 5-year olds, obviously, assuming your data looks okay.