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: Ash Verma - UBS - Analyst
: Yeah, absolutely. I mean, there is a lot to unpack here.
Unidentified Company Representative
Kyle, maybe it would be interesting just for the audience just to get a little bit of background on you and how things have transpired. Even over
the last seven years as a company, we've gone from not revenue generating to revenue generating.
And I think Kyle is just heritage with the company. I think it would be a good perspective. If you don't mind sharing a bit, Kyle.
Question: Ash Verma - UBS - Analyst
: Excellent. That's great. Great start. So yeah, I wanted to sort of talk about some of the recent developments that have happened in this story, and
just this field in general for muscarinic. So as you might have seen some data coming out from (inaudible)your competitor program to the extent
that it has any kind of like a read across to you. I wanted to understand that.
So in terms of like this (inaudible) data yesterday, not only did it like surprise negatively on such a high placebo response, but also their own like
efficacy was much lower versus what they showed in their earlier Phase 1b trial. So I wanted to understand like what are the implications of that
for you, because you are effectively like starting a new study in this domain. And if from a trial conduct perspective, like are there some learnings
that you identified that might be relevant for your program?
Question: Ash Verma - UBS - Analyst
: Just like a quick question or follow-up on this. So I think as companies like similar to you, like trying to do like a Phase 2 meeting -- end of Phase 2
meeting with the FDA, right? Is there a possibility that you are either AbbVie, and this is I'm just asking you your assumption, either AbbVie or you
might be asked by the FDA that mechanistically or from -- how much of a placebo response we are seeing, it might not make sense to continue
development.
Question: Ash Verma - UBS - Analyst
: Great. So maybe just like a switching topics. So you have this potential upcoming launch of crinecerfont, maybe let's talk about that a little bit. I
know on the third quarter conference call, you mentioned that like you still positive or upbeat about like patient adds.
But in terms of like the revenue realization, it might be a little bit slower because of like the how the commercial insurance is going to work out.
Can you elaborate on that dynamic a little bit? And I guess like the most important one thing for me is that would you be providing like patient
numbers to the street on where you are?
Unidentified Company Representative
I mean, that's a lot in that question. But yeah, absolutely, there's going to be a lot that we learn in this launch. I mean, this is the first medicine in
this entire patient population that's been launched in 70 years. So we don't have it all figured out.
But what we do know is that there's a lot of patient demand at the moment. But given that 60% to 70% of these patients are actually commercial
patients. There's more to like there's more than likely going to be some form of a block on that new medicine.
And as a result of that, there's going to be a delay in reimbursed scripts. So early on, there may be patients taking the medication that is not
reimbursed. And so that's where your question comes into. Would we be willing to provide patient numbers who are on therapy?
That's something that we're still trying to think through from a disclosure perspective. The other aspect that we think about that might impair
ultimate adoption. And the recent reality of the space is that these patients by and large only see their clinician one to two times per year.
So when you think about the just natural flow of them seeing their clinicians, there's not going to be a bolus effect of patients that are knocking
on the door from day one that will get this prescribed. It will be prescribed as they come in, as clinicians get experience with this medication, how
do they down titrate any of the glucocorticoid.
So there's still a lot to be learned in the commercial market. But all the feedback that we're getting, whether it's from patients, whether it's from
clinicians, people can't wait to get their hands on the medicine to see how it can start helping these patients with congenital adrenal hyperplasia.
Question: Ash Verma - UBS - Analyst
: I know you've previously talked about these like 8,000 patients in the CARES foundation, which are kind of like activated patients right, out of the
30,000 total prevalence. Is that still the assumption that like those to your point that like they would they be early adopters and might be in active
care and physician that that's like the low hanging fruit that you might get a quick ramp in.
Unidentified Company Representative
Well, I just say for any parent or anybody with the age, it's like a four letter word. You don't understand what it truly is, and how it wreaks havoc,
and the burden of taking really high dose steroids three or four times a day, and the ramifications it has on the life.
And I would just say the CARES Foundation was established, I don't know, 15 or 20 years ago, and their whole focus has been just on creating some
network for these patients. Some establishment of clinicians who actually specialize or care about treating patients with CAH, because up until the
1960s, all of these patients died, because they couldn't produce cortisol.
And as a result, they would have this huge surge of androgens and ultimately would pass away. So at the advent of the hydrocortisone, that's really
where things have started. So over the last 15 years, 20 years, education awareness, patient advocacy has really ramped up in CARES has been a
huge help in this regard.
So when you ask who could be the earliest adopters, I think that kids in particular are going to be those who are going to be the most motivated
to seek treatment. However, any kid or any parent, if they ask a clinician and they're going to listen to the clinician.
And so I think getting clinician expertise, getting their insight. Many of them participated in the trials, these parents or these patients are ultimately
going to rely on what the clinicians say. So we do have a motivated patient population.
We do have the defined patients that are part of the CARES Foundation. But I really think the adoption is going to ultimately come down to the
clinicians. How comfortable are they prescribing the medicine? What types of patients are they going to try this on first? But as Kyle said, with the
rich data that we saw in both efficacy, and there's very little downside from what we saw in terms of safety.
Our sense is that up to 80% of patients would be typical candidates for this type of medication. So we're enthusiastic about the long term potential.
It may take a little while for this to convert to revenue, but would expect this to be a quicker ramp to peak than per se as compared to the tardive
dyskinesia market.
Question: Ash Verma - UBS - Analyst
: Got it. Great. So I guess the other thing that I wanted to ask about the pricing. So Matt, going back to your comment at the conference call, and on
the third quarter, I think you mentioned that you expect on a year over year basis like pricing per script to be flat year over year.
Just like if you can talk about quickly on the pushes and pulls. I know you have the Medicare Part D redesign, any kind of like price increase and
contracting, anything that you would highlight on those factors.
Question: Ash Verma - UBS - Analyst
: So just one last question on INGREZZA. So I think there's an increasingly like investors are trying to understand, like let's say 10 (inaudible) of stero
ends up on the list for 2027. What is the sort of the if there is any indirect impact of that to you? And I understand that you have the small by the
exception, so that takes about the impact too further away. But just in terms of the competitive indirect pricing impact.
Question: Ash Verma - UBS - Analyst
: Great. Just one last quick question for me and we can wrap it up. So I guess positively surprised by the ASR that you're now $300 million, it's
becoming a little bit more common in the biotech land to see that -- for you, do you think that this is sort of like a one-time rest or can buy backs
become more prominent part of your capital allocation strategy going forward?
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NOVEMBER 12, 2024 / 9:15PM, NBIX.OQ - Neurocrine Biosciences Inc at UBS Global Healthcare Conference
Unidentified Company Representative
This was clearly opportunistic. We're buyers of our stock at this level for sure. And I was thankful the Board was supportive of wanting to move
forward with a direct shot like this in retiring the shares. We know, I think most of biotech knows that long term shareholder value is going to be
driven by revenue growth and advance in and expanding our pipeline.
And so looking forward, Ash, the large majority of our capital is still going to be invested behind R&D potential business development into the
future. But of course, this will always be on the radar if we do have excess capital in our stock price, feels very dislocated.
I think our Board will continue to be supportive, but with that as a backdrop, knowing that investing as a growth company is the utmost priority
for us as a company.
Question: Ash Verma - UBS - Analyst
: Excellent. Thanks a lot for your insights. Thanks for taking the time to participate in this fireside chat. I'm very happy that we have you here, and
looking forward to more interaction like this.
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