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: Myles Robert Minter - William Blair & Company L.L.C., Research Division - Analyst
: Yes. Thanks for the introductions, everyone. One of the things that's absolutely fascinated me in neuropsychiatry is that specific mechanisms of
actions of drugs can have broad implications for multiple indications. So it leads me to my first sort of top-level question for the panelists here is
that when you've got a neuropsychiatry asset with a particular mechanism that could have such broad implications, how do you decide what
indications to initially chase versus which ones to expand into? And does that have implications for potential business development relationships
you can do down the line? Maybe we can start with Kyle, considering your portfolio is largely in-licensed from Takeda.
Question: Myles Robert Minter - William Blair & Company L.L.C., Research Division - Analyst
: Marcio, do you want to comment on that?
Question: Myles Robert Minter - William Blair & Company L.L.C., Research Division - Analyst
: And then, Shawn, yes, with the Pherin class, like obviously, you've gone into social anxiety disorder, and that (inaudible) indications are neglected
in some way, but particularly SAD. So maybe you can explain the rationale there. And we've also got numerous questions in for VistaGen about
potential business development deals and if we can expect something like that in the near term.
Question: Myles Robert Minter - William Blair & Company L.L.C., Research Division - Analyst
: And then maybe on to Norbert, you've sort of approached it as an NMDA receptor modulator platform, but developing individual assets for
indications within that platform. Can you talk about that rationale rather than just producing one asset and trying to leverage that for multiple
indications?
Question: Myles Robert Minter - William Blair & Company L.L.C., Research Division - Analyst
: No, that's fair enough. I want to touch on trial design and endpoint, because it's one thing to have an active compound, it's another thing to actually
prove that (inaudible), which is obviously what the regulators are looking at. So my overarching question is that the FDA seems to be pretty lenient
in terms of adaptive trial designs, randomized withdrawals, enriching patient populations. But they also come out and state that they want that
trial to be representative of the ultimate patient population that you're going to be treating.
So where is that balance struck? How sort of valid are the endpoints we use in neuropsychiatry to actually conferring patient benefit? And what
are the key considerations in designing those trials to make sure you strike that right balance of giving the drug the best chance to show a positive
outcome, but also in the correct patient population that we can actually use that evidence to inform on prescribing? Maybe I can start with Marcio
on that one, and we can go around the panel.
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JULY 15, 2021 / 4:00PM, NBIX.OQ - Neurocrine Biosciences Inc at William Blair Biotech Focus Conference
(Virtual)
Question: Myles Robert Minter - William Blair & Company L.L.C., Research Division - Analyst
: And then maybe to Shawn, like you've got the PALISADE trial up and running now. And that struck me as interesting, considering that's actually a
laboratory sort of setting public speaking challenge. So I'm wondering how those sort of FDA interactions went to sort of set that as the primary
test and then use the label with scale. It's the primary endpoint there. But like how do you think that, that controls for the trial success rate, I guess,
and versus how your Pherin drugs could actually be used in the real world setting?
Question: Myles Robert Minter - William Blair & Company L.L.C., Research Division - Analyst
: And Norbert, you very recently just had some FDA interactions on the PTSD program. So can you give us an update there and your thoughts on
the language provided there?
Question: Myles Robert Minter - William Blair & Company L.L.C., Research Division - Analyst
: I think, Kyle, I'm particularly interested in luvadaxistat, and we had some data on negative symptoms of schizophrenia, but it seems like you were
able to do some sort of signal on cognition. I think when we look at trials of cognitive symptoms in schizophrenia, they use an endpoint called the
matrix, which is kind of constantly evolving and may have less regulatory validation here. So how do you think about pursuing indications like
that? What are your sort of talks with the FDA about that endpoint selection sort of involve? And how do you get comfortable about designing a
Question: Myles Robert Minter - William Blair & Company L.L.C., Research Division - Analyst
: Yes. I think that's great. We are pushing up on time. I did want to mention that the placebo response is clearly important. I mean we've seen some
really well conducted trials in schizophrenia by like Karuna and recently Cerevel that have shown historically minimal rates. And then we've seen
recent waterfall data that seems to be historically high, like, and that's just clearly impacted that demonstrated effect size of agents.
But maybe the last question, I have to ask it, and it's more focused on the neurology side. But with the recent approval of Aduhelm, do we have
any predictions for how the agency is going to react in the neuropsychiatry space and how the division of neurosciences is going to perceive new
drug applications? Are they going to be more or less flexible? Maybe just an opinion here. I'll start with Marcio and pass it around.
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JULY 15, 2021 / 4:00PM, NBIX.OQ - Neurocrine Biosciences Inc at William Blair Biotech Focus Conference
(Virtual)
Question: Myles Robert Minter - William Blair & Company L.L.C., Research Division - Analyst
: Well said. Any other comments from the panel before we wrap it up?
Question: Myles Robert Minter - William Blair & Company L.L.C., Research Division - Analyst
: All right. Well said. Super exciting programs going on at all 4 companies here and real leaders in the neuropsych space. So I applaud your efforts,
and it's great to see you all flying the flag there. So with that, I'll close panel, and enjoy the rest of the conference. Thanks, everyone.
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JULY 15, 2021 / 4:00PM, NBIX.OQ - Neurocrine Biosciences Inc at William Blair Biotech Focus Conference
(Virtual)
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