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
: A big congrats on the progress. So a few from my side. Regarding the discussions around the accelerated pathway, can you give us any color on
what was discussed regarding the sort of titering or product consistency issues in Part 1? And sort of what gets FDA comfortable with this? On the
expression versus function data, this is something you've been teasing us with for a while. Now FDA has seen it, when should we expect to see it?
And then just finally, the letter that you got from FDA, who is the signatory on the letter?
Question: Yun Zhong - BTIG, LLC, Research Division - Analyst
: Great. So a follow-up question on the non-ambulatory patient. Was that a study -- the new study required by the FDA during your discussion with
the agency? And based on your answers to the previous question, I guess, it's not going to be part of the confirmatory study required again by the
FDA? And secondly, I just wanted to confirm the time line. I believe you talked about potential Accelerated Approval around midyear 2023. So that
should be before you get top line data from the EMBARK Phase III study. But what happens if the EMBARK Phase III study misses the primary
endpoint? Has that possibility come up at all during your discussion with the FDA? Would there be any room of flexibility depending on how the
data look like?
Question: Zhiqiang Shu - Joh. Berenberg, Gossler & Co. KG, Research Division - Analyst
: Great. I want to add my congrats to the progress as well. Just a few clarification questions. First, on the -- based on your communication with FDA
I guess, has FDA explicitly encourage you to file based on Accelerated Approval? And secondly, I wanted to ask, Ian, around the SG&A uptick expense
in the second quarter. You mentioned the -- that was primarily driven by noncash stock options. Should we assume the expenses in Q3 and Q4
should come down substantially?
Question: Kristen Brianne Kluska - Cantor Fitzgerald & Co., Research Division - Analyst
: Congrats on a very exciting quarter for the company. Most of my questions have been asked, so I'll change topics a little bit here in gene therapy.
So based off the path you took with SRP-9001 and your discussions with the agency, has this changed how you might envision some of the trial
designs for the limb-girdle programs. And of course, I understand here that you'd want to see some supportive data before figuring out the path
forward. But any initial thoughts at this stage?
Question: Gavin Clark-Gartner - Evercore ISI Institutional Equities, Research Division - Analyst
: I just wanted to ask if you could give any more granularity around the status of the CMC work and thoughts on the time line for engaging with the
FDA. Is it maybe later this year saying or more like a 2023 event?
Question: Gavin Clark-Gartner - Evercore ISI Institutional Equities, Research Division - Analyst
: Yes. Yes, for limb-girdle.
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