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: Brian Chang - JPMorgan - Analyst
: Hi, Warner. Hi guys. Thanks for taking our questions this morning and, really congrats on the data. As we think through the phenotype of,
SPS, there's always these painful spasms, that is clearly a notable characteristic of the disease. Can you speak to the level of improvement
either in the frequency or the severity of the spasms? Or con any possible contraction in the in the patients. Can you talk about the influence
that nerve cells has in those patients and then we'll a quick follow-up.
Thank you.
Question: Brian Chang - JPMorgan - Analyst
: Great. And then, just, back to your first filing, in the first half next year, what are some gaining factors remaining before, the filing? What is
left to do, now that you have your pivotal result in hand?
Question: Thomas Smith - Leerink Partners LLC - Analyst
: Hey guys, good morning. Congrats on the really stellar data here and thanks for taking our questions. Maybe I could just start with, maybe
two for Dr. PK. Can I just ask you to elaborate on how you're thinking about the profile here and the balance between efficacy and safety
with MIV cell and just help us understand within your center, how many patients do you think are candidates for MIV cell and how are you
Question: Thomas Smith - Leerink Partners LLC - Analyst
: Sure, yeah, no, I was just, hoping you could comment on, sort of within your center, Dr. PK how many patients do you think are candidates
for MIV cell and how are you thinking about the positioning relative to off-label, treatment options?
Question: Thomas Smith - Leerink Partners LLC - Analyst
: Got it. That's super helpful. Yeah, I appreciate the insights and then maybe one for the company just from a regulatory perspective based
on the transformative efficacy and the huge unmet need in this population, could you comment on, I guess your level of engagement with
FDA and whether you plan to pursue, one of the commissioner national priority vouchers? Thanks so much.
Question: Michael Ulz - Morgan Stanley & Co Ltd - Analyst
: Good morning. Thanks for taking the question and congratulations on the data as well. Maybe just one on the time 25 ft walk. There were
a couple patients that didn't meet that 20% threshold. Just curious if those patients also had a benefit and if there's anything, unique maybe
in the baseline characteristics, for that group of patients. Thanks.
Question: Derek Archila - Wells Fargo Securities LLC - Analyst
: Hey, good morning and congrats on the data. Thanks for taking the questions. Maybe just first, as you look at the baseline characteristics
from the trial, how do you think they reflect the patients that you see or, some of the physicians see in the real world?
So that's question number one and then I just wanted to confirm on the neutropenia, I presume this is largely attributed to the preconditioning
regimen, so I just wanted to confirm that and then I have a follow-up as well. Thanks.
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Question: Derek Archila - Wells Fargo Securities LLC - Analyst
: Very helpful. And then I just wanted to follow-up on the prior comments in terms of like BLA submission. I just wanted to know in terms of
CMC where you stand there and is that kind of a potential factor or gating factor on the BLA submission.
Thank you.
Question: Sami Corwin - William Blair Capital Partners - Analyst
: Good morning. Congrats on the data and thanks for taking my questions. I noticed that over half of the patients have follow-up beyond 24
weeks. So I guess I was curious if you continue to see stability or continued improvements in the 25 ft walk test beyond that time point. And
then for Dr. Piquet, how important is durability of response and how long would you want to see improvement sustained for?
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