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: Gena Wang - Barclays Capital Inc. - Analyst
: Congrats on the update. Okay. I will stick to just one question. So with these data sets, how would you envision this impact the commercial uptake
and also incorporation into the label?
Question: Tazeen Ahmad - BofA Securities, Inc. - Analyst
: Doug, you talked about your thoughts about the competitive landscape for gene therapy going forward, with potential next-gen therapy from
other companies. How do you think today's data presents itself vis-a-vis the barrier to entry for future gene therapies going forward? Thanks.
Question: Tazeen Ahmad - BofA Securities, Inc. - Analyst
: Brian Abrahams, RBC Capital Markets.
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JANUARY 27, 2025 / 1:30PM, SRPT.OQ - Sarepta Therapeutics Inc To Provide Corporate Update
Question: Andrew Tsai - Jefferies - Analyst
: I appreciate the update. So just one quick question is, do you think your partner, Roche, can leverage this data set as part of their ongoing MAA
filing in the EU as well as for their other ex-US submissions? Thanks.
Question: Kostas Biliouris - BMO Capital Markets - Analyst
: Good morning, Sarepta, and congrats on the update. A quick question from us. Can you comment on the ambulatory status of patients in year
two, and sorry if you clarified that before? We are wondering whether there was any patient during the entire previous study who transitioned
from ambulatory to nonambulatory status. Thank you.
Question: Gil Blum - Needham & Company, LLC - Analyst
: So maybe a question for Louise. As it relates to the treatment kinetics, it's look like even after a year, you got roughly similar delta benefit, maybe
a little smaller. And before and at the end, at MSA, it looks like pretty much most patient groups reached a similar level. I'd appreciate your comments
there, especially as it relates to the differences in NMR. Thank you.
Question: Ritu Baral - TD Cowen - Analyst
: One question and one quick follow-up. Can you talk about the steroid dose given at week 52? Was it equal between both patient groups? And
how do they compare to the initial steroid dose given at study start? And just a quick follow-up. Doug, do you anticipate that today's data will be
used in any payer discussions? Have any Medicaid plans asked for the two-year data in evaluation for continuing coverage or current coverage,
just to make things easier for patients?
Question: Kristen Kluska - Cantor Fitzgerald & Co., Inc. - Analyst
: Since the field has really moved to understand that markers -- end points, excuse me, like NSAA are going to require a lot longer than a years' worth
of data, now that you have two years' data from a good number of cohort, are you seeing that the patient that perhaps didn't have as robust as an
effect at year one are starting to catch up and benefit more through months 12 to 24?
Question: Sami Corwin - William Blair & Company, L.L.C. - Analyst
: Congrats on the data. Some of your earlier gene therapy competitors have communicated that they think that percent positive dystrophin fibers,
this could be better early surrogate marker of long-term benefit as opposed to micro-dystrophin expression as measured by western blot. So I
guess I just wanted to get your thoughts there and if it would be possible for you to retrospectively analyse percent positive dystrophin fibers in
the study? Thank you.
Question: Daniel Smith - H.C. Wainwright & Co., LLC - Analyst
: This is Dan on for Mitch. Congratulations on the data. So we now have one-year follow-up for Cohort 2. And at the same one-year follow-up mark
for Cohort 1, the NSA wasn't statistically significant and was only increased 0.65, which was much less than Cohort-2 at 2.34. So what do you think
is driving the considerable differences between Cohort 1 and Cohort 2? And separately, how might unique side effects to the AAV Capsid
immunologically have affected the integrity of the crossover cohort functional finding? Thank you.
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JANUARY 27, 2025 / 1:30PM, SRPT.OQ - Sarepta Therapeutics Inc To Provide Corporate Update
Question: Gavin Clark-Gartner - Evercore ISI - Analyst
: I'm just wondering what you saw on biomarkers such as creatine kinase. Thank you.
Question: Uy Ear - Mizuho Financial Group - Analyst
: Congrats on the data. Just curious, wondering if you also saw other benefits other than time function test? Just the ability -- better improvement
in bleedings or cardiovascular problems and whether you've seen any improvement in those functions?
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JANUARY 27, 2025 / 1:30PM, SRPT.OQ - Sarepta Therapeutics Inc To Provide Corporate Update
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