The following is excerpted from the question-and-answer section of the transcript.
(Questions from industry analysts are provided in full, but answers are omitted - download the transcript to see the full question-and-answer session)
Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst
: Congrats on the data. First, somewhat of a housekeeping question on patient numbers. I think there's 65 patients across cohort 1
for 103, and then Studies 102 and 101, in the pooled analysis, there were 52. I didn't quite see the footnotes on the slide. Could you
just help me with that delta?
And then just a quick one on the functional data for 103, can you talk about any differences or any meaningful differences in ages
and baseline characteristics for the additional 9 patients we got today versus the original 11 that comprise the 24-week data we saw
at the micro-dystrophin data.
Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst
: On the -- sorry, just on the target dose issue, what was the primary reason for patients not receiving the target dose?
Louise R. Rodino-Klapac - Sarepta Therapeutics, Inc. - Executive VP, Chief Scientific Officer and Head of Research & Development
Yes. So in Study 102, if you'll recall, we -- when we retrospectively titered those patients using our now validated methods, it was
noted that there was some variability in the lot using the clinical material in Part 1 of that study, and that's why there was some
patients that receive less than the target dose.
Question: Kristen Brianne Kluska - Cantor Fitzgerald & Co., Research Division - Analyst
: Congrats on these data that you presented. Could you talk more about the importance of the earlier intervention here, especially
that you had followed up with patients for over 4 years, and you've also looked at different patients across different age groups?
And I understand that PPMD very recently submitted a nomination package to add DMD to the newborn screening panel?
Question: Yun Zhong - BTIG, LLC, Research Division - Analyst
: So I think on your discussion with the FDA on the accelerated approval pathway, outside the functional data that you presented this
morning, do you think you would need or do you plan to submit anything specifically to support a correlation between
micro-dystrophin expression? I assume that, that will be the surrogate end point and clinical benefit.
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JULY 06, 2022 / 12:30PM, SRPT.OQ - Sarepta Therapeutics Inc to Discuss New Clinical Data and Integrated
Analysis Call
And also on using the external control, there was a previous question on the steroid use, but I wonder, can you remind us when the
external control data were collected? And anything that can potentially may have improved over the years in terms of standard of
care outside that would use might affect the validity of the comparison, please?
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