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: Tim Lugo - William Blair & Company, LLC - Analyst
: Sure. Thank you for that overview. And I will point out to anyone on the line, the Company did host an excellent KOL symposium on Monday
digging into 1101. We also wrote a note on that, so that is also a great place to start if you are new to the story. So maybe ahead of the X-TOLE data,
which I know is kind of the next big event for the Company, can you just talk a bit about the treatment landscape in focal epilepsy? And I do get
questions -- I just got one yesterday from a client asking me, what's the bar for success coming out of X-TOLE?
Question: Tim Lugo - William Blair & Company, LLC - Analyst
: I think something that came across from your Monday event was kind of the patient interest in the open-label extension, which sounds like it's
been very strong. I think it was over 95% or so have rolled over. And I think Jackie mentioned that even extending the OLE almost is also kind of a
sign that at least patients -- or physicians want their patients to be on this therapy. I guess, what are you kind of gleaming from the OLE and that
extension which kind of makes you confident that this drug does seem to be, at least, incredibly tolerable and combinable and all these nice
properties you mentioned?
Question: Tim Lugo - William Blair & Company, LLC - Analyst
: And is there a dose in the OLE? Like how do you (multiple speakers)?
Question: Tim Lugo - William Blair & Company, LLC - Analyst
: And I think you've done a great job in discussing what makes a successful therapy in this indication outside of, just -- is it 30% seizure reduction, is
it 40%. Can you talk a bit about -- I think years ago, there was a discussion with the KOL community around seizure freedom. And cenobamate had
great data around seizure freedom, but it doesn't look like it's gained traction in the market. Is this swinging back to ease of use in your opinion?
Question: Tim Lugo - William Blair & Company, LLC - Analyst
: And ultimately, about the goal of being the easy add-on therapy in the second-line -- and we've seen that in a number of CNS indications, and in
epilepsy too, where just that ease of use just makes it so attractive for a neurologist to add onto whatever therapy might not be getting optimal
benefits.
Question: Tim Lugo - William Blair & Company, LLC - Analyst
: That's a great segue into -- I've really been intrigued by the dosing schedule and the nighttime dosing. Does that provide ease of compliance? Is
that the mechanism for avoiding AEs? Are there sleep benefits? I'm really intrigued by how that might help the outcomes.
Question: Tim Lugo - William Blair & Company, LLC - Analyst
: And it definitely leads us into the mood improvements that the compound seems to be having. Can you talk a bit about MDD and anhedonia? And
also how that is also fantastic for epilepsy patients where that's a major issue?
Question: Tim Lugo - William Blair & Company, LLC - Analyst
: And how are you going to tease out some of this in X-TOLE? Did you -- I know the MDD's anhedonia's signal came interest studies. So was any of
that built into the study or is that really -- we'll look for, obviously, the two Phase 2s and then maybe whatever is next for 1101 to really tease this
out? Or will we see some of that in X-TOLE?
Question: Tim Lugo - William Blair & Company, LLC - Analyst
: Sure, sure. And also, what is the timing to kick off the company-led Phase 2 in MDD? I know that those could be a little faster than the Mount
Sinai-Baylor trial.
Question: Tim Lugo - William Blair & Company, LLC - Analyst
: And X-TOLE is pretty large in terms of patient size. Is there a potential that this could be one of the registrational studies? I know we are jumping
ahead, obviously, but knock on wood it's going to be a success. And when do you clarify with Phase 3 and what that looks like?
Question: Tim Lugo - William Blair & Company, LLC - Analyst
: It's interesting you bring that up as we know that the cenobamate X US-alone market seem to be worth $600 million or so. So as I get questions
about valuation -- that's something I point to often is, what is a much smaller market than the US market. It still looks to be at least $600 million or
so in value on the strategic side. So we will be eagerly anticipating your decisions X US as well. And I guess before we -- it looks like we are almost
bumping up to time, I apologize for that. But can you talk a bit about 496 and 007 and what are the next data points we should be looking out for
those programs?
Question: Tim Lugo - William Blair & Company, LLC - Analyst
: That's very interesting. Thank you for the overview. And when I get questions, I often point to -- that are very X-TOLE focused -- I think it's important
to note that you have such a large pipeline. And the Neurocrine program is rapidly progressing. Can you just talk about -- I mean, that's going to
be in Phase 2 within a quarter, so what is the status of the collaboration and what also has helped to push it really into Phase 2 so quickly? And I
apologize, we only have a couple minutes left but I would just love to hear more about it before we sign off.
Question: Tim Lugo - William Blair & Company, LLC - Analyst
: That's incredibly exciting. Well, with that, we are out of time. So thank you so much for attending this year's conference. It's very much appreciated.
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