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: Andrew Berens - Leerink Partners LLC - Analyst
: Hi, can you hear me?
Question: Andrew Berens - Leerink Partners LLC - Analyst
: Great. Thanks, Andy. Congrats on the data and all the progress. I guess ISM slides 12 to 15 are particularly striking and suggesting that bezu
has a broad effect on symptoms and the biomarkers correlate with symptoms. It's different from what ava has shown in the past. I'm wondering
if the doctor could comment whether she thinks this is going to translate into an improved quality of life for the ISM patients and when both
of these drugs are on the market, how will she decide which drugs she's going to use on a new patient that's naive to therapy and presents
in her clinic, and then I have a question on ASM after that.
Question: Andrew Berens - Leerink Partners LLC - Analyst
: Yes.
Question: Andrew Berens - Leerink Partners LLC - Analyst
: Okay, thank you very much for the in-depth response. And then just on the ASM data, just wondering if Andy could give us a little more
color on the unconfirmed patients, the number of patients that have had a response maybe that haven't confirmed yet but still may confirm,
and then when we might see the 20 patients, the combination patients presented, and if you think those patients may eventually end up in
the label.
Question: Andrew Berens - Leerink Partners LLC - Analyst
: Right. Well, congrats again on all the progress and execution.
Question: Anupam Rama - JPMorgan Chase & Co - Analyst
: One for Dr. Rein. Look, this question was almost just asked of you, and I know you called it a loaded question on stage on Saturday morning.
I was just wondering if you could dig in a little bit on how you think about the segments of patients that might be more appropriate for
bezuclastinib in both the non-advanced and advanced setting versus avapritinib based on the totality of what you know today.
And then Andy and team, congrats on all the progress. Just wondering what are the final gating steps here to getting that NDA submitted
in non-advanced in the SUMMIT data. I think you've reiterated that that's happening this month, so thanks so much.
Question: Michael Schmidt - Guggenheim Securities LLC - Analyst
: Congrats on the data as well. Just maybe to follow up on SUMMIT, so just obviously great to see consistency in effect really in treatment
effect across all symptom domains, but are there any particular subsets in the score that are particularly burdensome for patients, a particular
impact to have improvement perhaps relative to avapritinib as we look at symptomatic improvement? So that's my first question, anything
that stands out there.
And then on the correlation with PD markers essentially and outcomes on slide 17, really interesting to see the correlation especially with
some of the measurable things like serum tryptase reduction, and I was just wondering in clinical practice, having that correlation, can that
be used to guide therapy in any way? For example, seeing perhaps serum tryptase reduction first but not yet a clinical response and keeping
patients on longer on therapy, having that additional measurement, can that be used in clinical practice to guide therapy was the other
question I had.
Question: Christopher Raymond - Raymond James - Analyst
: Maybe one for Dr. Rein on the treatment choices here. I know you've answered a couple of previous questions on how you choose between
these drugs once they're both on the market, but maybe just a question here and another wrinkle. A big part of the treatment decision tree
with ava is whether to up-dose from 25 to 50 mg. We've heard from a number of treating physicians that this can be an issue for patients
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who are worried about cognitive effects, et cetera. So maybe with both drugs on the market, how big of a factor is that for you and how
important would it be to not have to deal with that? And then I have a follow-up for management.
Question: Christopher Raymond - Raymond James - Analyst
: And then maybe, Andy, so you guys now have objectively a superior drug to Ayvakit in both non-advanced and in ASM here. I think we all
know the price that's been set for Ayvakit, and I know you're not going to talk about the price specifically, but you have flexibility here now
given the different doses. Should we be thinking about a meaningfully different price point for these indications?
Question: Sam Slutsky - LifeSci Capital LLC - Analyst
: Great work on the update. Just two for me, just on the correlation between the PD markers and symptom benefit in non-advanced SM, any
hypothesis on why your data shows this for avapritinib didn't, and then I guess to this point, could it be possibly because you have more
data points that deeper reductions contribute to the analysis?
And then the second question is for the patients who had prior avapritinib in both your advanced SM and non-advanced SM studies, just
any general statements on how they did?
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