Neurocrine Biosciences, Inc. ($NBIX)

Thank you everyone for joining us here at the 46th William Blair Annual Growth Stock Conference. I haven't been here for all 46 years, but I have been here for close to 10. My name is Myles Minter. I'm a senior biotech analyst here at the firm. I cover the neurosciences and then genetic medicines, and it is my pleasure today to introduce Matt Abernethy, the CFO; and also to my right, Todd Tushla, Head of IR at Neurocrine Sciences, fresh from the West Coast. So thanks very much for attending.

Myles, did disclose that he just came from Scotland. Is that right?

I did. I did.

And my last name is Abernethy, and that's Scottish. You can get some cookies ever go to that town, but really glad to be here. I went to business school in Chicago. And so always nice to see San Diego, ask weather in Chicago. Really nice to be able to be here with you guys today. A little bit of fun, forward-looking statements. I will be making some, so direct you to the SEC filings for the related risk factors and uncertainties associated with our company and then also our industry. We have changed quite a bit. I was just talking to a gentleman before this. We were a single product company, now a multiproduct company, generating significant cash flow, significant data readouts coming in '27 and '28. As a biotech company that had been a single product company for quite some time, I can tell you, it's very nice to be able to speak to you guys today and to be able to share with you guys the story of the great things that we have going on at Neurocrine. We're a neuroscience company focused in endocrinology, psychiatry, neurology, immunology were based in San Diego, California, have over 2,500 employees at this point. Half of those are in San Diego. The other half are spread across the United States, marketing our products, so we have a lot of fun. And if you ever find yourself in San Diego, would love to host you at our new campus. We have a lot of magic going on there, which you can see going into our early-stage research and development pipeline that we'll see in just a little bit. So very much looking forward to talking with you guys today. So we have 3 pillars, just like every good presentation has to have 3 pillars. The first 1 is our commercial products. These are generating significant cash flow at this point, growing quite significantly. We had 45% year-over-year growth in Q1 and this allows us the ability to invest in R&D, which is the lifeblood of any company within Biotech. And then lastly, that's translating to very nice financial returns, where we've been generating significant cash flow over $200 million of non-GAAP net income in Q1 alone, and we've recently deployed our cash to acquire Soleno Therapeutics, which was for $2.9 billion. It's the biggest acquisition Neurocrine has ever made, but very exciting for us. On the commercial product side of the equation, we have INGREZZA. That was approved in 2017. That was our only approved medicine until December of 2024, but it's been the lifeblood of our company. It's allowed us to reinvest, build the company to where you see it today and our guidance range is $2.7 billion to $2.8 billion this year with INGREZZA. And that implies mid-teens underlying volume growth in a very attractive market, offset by a little bit of price pressure. The second medicine that I mentioned earlier was CRENESSITY that's for congenital adrenal hyperplasia. That's a rare endocrine product. We launched that last year. It's been on the market for 5 quarters now. The patients had no other option other than to take high-dose steroids for their condition for their entire life. And this had $150 million of sales alone in the first quarter, and the team has done an incredible job with CRENESSITY. Last, this is VYKAT XR. This is the 1 that came to us from that $2.9 billion acquisition. This is for a very rare devastating disease called Prader-Willi syndrome. It was approved in March of 2025. It's been on the market about a year at this point. And similar to CRENESSITY off to a great start, sales are around $100 million in Q1. So when you add all 3 of these products up, over $900 million in sales in the first quarter alone. For a finance guy, this next part is important, 97% plus gross margins on these products. So you think about the cash flow that's coming from growing sales, the ability to reinvest back into research and development at a rate of 30% to 35% without sacrificing ultimate profitability is something that we've been doing, and we've created a very diversified pipeline at this point, which I'm excited to share with you guys about a bit later in the presentation. And then lastly, financially, we did do that acquisition for Soleno, $2.9 billion. We pretty much funded that all cash and it will be immediately accretive on a non-GAAP basis to our shareholders on an EPS basis. So overall, very fortunate to be in the position that we find ourselves in today. So let me tell you a little bit about INGREZZA, and I was talking to an individual here who actually was selling an antipsychotic early in the 60s or 70s. And unfortunately, with antipsychotic use, you have a 10% likelihood of developing a debilitating disorder called tardive dyskinesia. It shows up in the hands, mouth, the base, the trunk, and it's just an unfortunate byproduct of taking an antipsychotic. And up until 2017, there were absolutely no treatment options for these patients. So if you think about dealing with your underlying mental health condition and then adding a movement disorder on top of that, it's not unsurprising that the stigma that comes with that isolation, the low self worth. And so it's been so rewarding since 2017 to be able to start developing this market. We estimate that there's around 800,000 patients with tardive dyskinesia but only 2% were diagnosed in 2017 because there were no reasons to make a formal diagnosis because you couldn't do anything about it. Even if you stop the underlying antipsychotic, those movements would stay. So a lot of our effort over the last 9 years has really surrounded diagnosis. And that's one major challenge of -- I get asked all the time. Why don't you have more than 1 out of 10 on a VMAT2 inhibitor today? It really comes down to the basics of making the diagnosis. And why might that be difficult? These patients are being seen by psychiatrists. The psychiatrists rightfully so are caring more about the patient's underlying mental health condition, working on a computer screen, not looking intently at their patients. So it's not surprising that it's not top of list for a psychiatrist. So a lot of our call frequency with our sales force is simply to put and keep tardive dyskinesia on the radar. The second piece really has to do with the psychiatrist and getting confidence in making a movement disorder diagnosis. That's something that they're not necessarily trained on, and that's something that we help support them in getting confidence making the diagnosis. The second piece is that -- the second phenomenon is that patients who have tardive dyskinesia, they have no idea who to talk to about it. You wouldn't think about going into the psychiatrist's office and talking to your psychiatrist about a movement disorder. I'd liken that to going into the dentist's office and asking the dentist to look at your ankle. So if you see our commercial, the INGREZZA jingle commercial on TV, I just know we are trying to motivate patients to actually bring it up with the psychiatrists that can I actually get help, because if they don't bring it up, the clinician may feel like it doesn't bother them, so they're not going to spend the time to ultimately treat. So overall, it's been quite rewarding to be able to develop the market in this way. But those two fundamental aspects still remain the same challenge in 2026 as it was in 2017, diagnosis and patient activation. So how has that translated financially? You can see a pretty linear growth patient by patient, adding them on therapy, staying on therapy. Now we're at a place where we're at $2.7 billion to $2.8 billion in sales. And as I said at the outset, this implies the mid-teens underlying growth offset by a little bit of price. But it's very rare to see, Myles, if any other product growing at this clip 9 months or 9 years after launch. And it really comes back to -- I think I can go backwards, yes, there we go. It really goes back to the right hand of this slide. This is what gives us confidence that growth is going to continue. There's still so many patients with tardive dyskinesia that need help, and it's been a real reward for Neurocrine to be able to help this many patients. We have IP protection that goes out till 2030. So durability of these revenues is quite strong. Now as I said at the outset, INGREZZA, funded our company and the first product that came as a result of this funding was CRENESSITY. This is an internally advanced compound. It was called croneserfant, now it's called CRENESSITY. This absolutely changed the game for patients with a disease called classic congenital adrenal hyperplasia. That's a mouthful, so we say CAH. But essentially, what it is, is the patient is born without the ability to produce cortisol. And without cortisol, you die. And up until 1950s, 1960s, all patients with CAH would ultimately pass away until hydrocortisone was developed. So since they can't produce the cortisol you supplement it with synthetic cortisol, hydrocortisone and ultimately, that's been how you maintain the life of these children and then into adulthood. There's about 20,000 patients, but you can imagine androgens being out of control, plus high-dose steroids for your entire life. That is not a good recipe for success. So we got approval in December of 2024. First in 70 years to give an option that's not a steroid to patients. And it's been just a tremendous launch thus far. I was at a conference a few weeks ago and was asked tell me why has CRENESSITY been so successful? And I would just tell you that it's basic -- that's a three-ingredient recipe. One, your product has to absolutely meet an unmet need. Here, that was quite clear. Second, it really has to make a difference. And you can see the difference quite quickly. And what we've been hearing in the real world is that the results are at least as good as what you see in the clinical trials, if not better, on both the efficacy and safety. And last, a patient has stay on therapy. And what we have found both from reimbursement has been over 80% of all prescriptions are getting reimbursed, so no payer pushback and also tolerability and benefit is quite clear. Patients are staying on therapy, and it's making a difference in their life. So how does this translate to revenue? You can see in Q1 of 2025 a year ago, we're pretty much an INGREZZA only company, and that had been the case for the previous 7 years. You then fast forward to last quarter, you can see $150 million in CRENESSITY sales, about 10% of patients with classic CAH have been treated at this point. And really, the diversification and scale in year-over-year growth is starting to look quite attractive. And for us, as a company, it's amazing, but also for patients just thinking about helping a segment of patients and in particular, kids who''re taking high-dose steroids. I think we all know. If you have had Poison Ivy, if you grew up in the Midwest, like me, you start taking steroids, you want to taper off of them as quickly as you can. Think about that your lifeline for the rest of your life. This has been very nice to be able to see androgens going down and then also seeing the glucocorticoids going down. Last comment here, which is probably the most personal comment I'd make. My son has CAH. And so when I talk about the value to patients, this was something I was working in the medical device industry and somebody reached out to me about the Neurocrine job, and I said I never heard of Neurocrine. Probably called it neurocrine, but looked at the pipeline and then I saw they were working on a medicine for my son. And so 7 years later, we got approved. He was the first commercial patient on therapy. So wanted to share that with you equally special for me. So moving on to the next product, VYKAT XR. This came to us from the Soleno Therapeutics acquisition. This is for a devastating disorder called Prader-Willi syndrome, and I'll tell you a little bit about that syndrome here in a few minutes. But before we do that, we spent $2.9 billion of shareholder capital. So what is that -- what was the strategic rationale? The first piece is that this is our second rare endocrine product. So CRENESSITY was our first. This gives us start of a franchise here. Very similar call points. So strategically fits right into our wheelhouse commercially. The second is it adds to both growth and the diversification mission that we've been on to build an enterprise, not just a single product company. And then lastly, this is cash flow positive from day 1, and it's going to be quite accretive in value creation for shareholders given the IP landscape here. We believe it's going to last for quite some time. So there were really three key aspects that we needed to figure out in diligence. The first one was about the Prader-Willi's market. What was it like? Is there a significant unmet need? And that's the easiest box to the check on. Everything that we found about the PWS community was one that there's extreme need to be able to help those patients. The second question that we had to answer was the safety and efficacy aspect of VYKAT XR. Was it providing benefit? Were the safety signals consistent with what's in the label and what we've seen in clinical studies? We talked to parents. We talked to KOLs. We talked to patients themselves. And overall, we found this as a great product helping these patients. And then lastly, the IP. I personally didn't want to buy something that was going to fall off into generic landscape in the early 2030s. This is something I wanted strategic value from. And we went through and looked at both the filed and also on published patents or published patents and unpublished patents, and we were able to get a lot of comfort around IP extending into the mid-2040s, which will become more known, publicly likely sometime later this year. So overall, that was a strategic rationale and some of the aspects that we went through in diligence and trying to convince ourselves of VYKAT XR which is truly changing the game for patients with Prader-Willi syndrome. Prader-Willi's, I had never heard of it until we started diligencing this product. It affects about 10,000 to 12,000 patients. And it's this unrelenting obsessive food-seeking behavior. Think about your hungriest moment that you've ever been and that's how these patients are for their entire lives, and it's devastating, and it leads to a lot of disruption at the home life and the family and with the patients individually. And this was the first approved treatment in -- ever for these patients. It's been on the market since March of 2025. So about a year now, and you have around 10% of patients who have also been helped with VYKAT XR in the first year or so of launch. So what does this do for Neurocrine? You can look at the middle pie chart. To be able to see $250 million out of our $900 million coming from something on INGREZZA, that's a nice picture for us. And it's not to say we don't like INGREZZA, we love INGREZZA. And we think INGREZZA is going to continue to grow into the future. But as we think about managing our business and investments and concentration risk, having two rare endocrine products in our commercial channel has been quite significant. And I think this is just the beginning. And then lastly, you'll start seeing it accrete into our P&L over the second half of this year. And I think you'll also agree with us in understanding both the scale benefit as well as the profit profile that will come from this acquisition. So financially, this is an easy picture to interpret. Revenue growing from $570 million last year to $815 million this year, over 40% year-over-year growth, quite significant with a big chunk of that incremental growth coming from CRENESSITY and we feel like we're just getting started with CRENESSITY, as I said earlier. On the R&D front, reinvesting around 30% to 35% back into R&D based upon the quality of the products that we have and that translates to a residual of $200 million of non-GAAP net income. And you can see at the bottom, the $2.6 billion of cash, that's before we acquired VYKAT XR. This really reflects our core capital allocation priorities. The #1 priority and I think everybody in biotech would say this, who has a commercial product, top line revenue growth matters significantly. So we invest significantly to drive top line revenue growth. That then gets us to the second priority, which is to be able to afford funding the right R&D programs to build the company. And then our third is to use our capital to deploy towards business development in a smart way that will drive returns for our shareholders. And I think you're seeing that play out in how we're operating our company and that allocation framework has been something we've articulated over the last 3 or 4 years. We are patient in our acquisition process. And obviously, with Soleno, I think it will only accelerate our forward growth from here. So now let's look at this pipeline. It's pretty cool. If you would have seen a Neurocrine pipeline just 5 years ago, we would have been looking at probably 3 to 5 products. Now we have close to 20 clinical programs. So the scale sticks out to you. And this isn't reckless, this is intentional. We need scale. We need smart scale that's diversified in different approaches and we need programs that will give us data quickly to let us know if there's a there, there or if you can that program and move on to the next, and that's something that you can see showing up in our pipeline. If you go left to right, the left is earlier, that's preclinical to clinical. We have probably 50 other programs, not on here that are preclinical that Jude and team have queued up for us. But we have to be very selective on how much we want to fund and pace it appropriately. But you can see the color. The color diversification represents different therapeutic areas that we find ourselves going into whether it's psychiatry, neurology, endocrinology or immunology and then the symbols. The symbols reflect whether it's a small molecule or a large molecule. And you can see on the far right, which is our later stage all small molecule. On the left, being able to get into different modalities has been an important step for us. So this has been quite the journey and kudos to Jude and his team for all the value that I know are going to be created for shareholders as well as for patients. If you go to the far right, our 2 marquee Phase III programs. The first 1 listed here is direclidine, which is a muscarinic agonist. If you remember the acquisition of Karuna by BMS and the product, Cobempi. This is the same pathway. And this is a medicine that is in a Phase III trial right now and that will read out in 2027, one of the trials and then 2028 for the second trial. In addition to that, you have Osavampator, which is an AMPA potentiator. This is being studied in major depressive disorder. And this is a trial, we'll have 3 trials reading out in 2027. So if you fast forward over the next 12 to 18 months to have answers on those 2 programs and the transformation that could have for our company is quite significant. The 1 program I called out earlier stage that gets a lot of investor interest has to do with our obesity program. It's the second one to the bottom in the Phase I. This is a CRF 2 agonist. It's a different approach, and we're really looking at trying to reduce weight, but primarily in visceral fat as compared to lean muscle mass and so we'll be able to get through our Phase Ib trial in 2027. We'll have data later in the year and that we'll be able to highlight progress on that patient population, which would be a healthy overweight or healthy obese patients. And so it does give a good directional picture as to are we seeing what we saw in animal models translating into humans. And so that will be another important milestone. So I'll wrap it up here. Neurocrine has been on an amazing journey. And for me, personally, being part of it, going from single product to multiproduct, multiple cash flow generating assets and being able to invest in meaningful pipeline programs has just been very rewarding. You can see, as I said earlier, 3 commercial products. But I want you to think about the patients. The tardive dyskinesia patient taking INGREZZA. There was never an option for those patients to get [Audio Gap] TD. Similarly with CAH, with CRENESSITY, the only option was to take high-dose steroids for their entire lives. Now you have CRENESSITY to be able to help them. Lastly, on VYKAT XR, never an approved treatment option to help patients with Prader-Willi syndrome. Now we have something to help those patients. So I think we can talk -- I can talk in numbers that this is $900 million, great gross profit to invest in the next medicine. But we take a step back in a very proud way to think we are truly impacting the lives of patients. And that's what makes investing in life science fund or at least operating company fund. There is high risk, but there is great reward on the other end and glad I could present Neurocrine Biosciences to you guys today.

Matt requested a standing ovation, so you go one first. We've got 4 minutes for you to Q&A before we break out. We'll definitely take a question from the audience to kick us off, please.

[Technical Difficulty].

So the question has to do with, would a GLP-1 help PWS patients in addition to VYKAT XR? And so what VYKAT XR is doing is aiding the brain aspect to this, and it's not necessarily helping on the metabolic side of the equation. You're trying to give this to patients before they progress to becoming very obese or diabetic or whatnot. So over time, you could imagine, though, patients who are benefiting from VYKAT XR and their life is stable, that they would be a potential candidate to also be on a GLP-1, but that would be a medical decision. But that was 1 of the questions that we had is would a GLP-1 cure or help people with PWS? It would allow them to potentially manage their weight a little bit more, but not the mental obsessive unrelenting behaviors that causes most of the destruction in these patients' lives.

Maybe 1 on INGREZZA, just IP out to 2038, still a growing franchise. I think you painted a really nice picture of the case for organic growth with that franchise as well. Investors do bring up the competitor and the potential pricing restructure that might go on in 2027 for that product. How does that sort of factor into your long-term growth potential of INGREZZA?

I think regardless of how you skin this. This market is all -- this is going to be driven by volume. How many more patients can you actually be treating with the VMAT2 inhibitor between ourselves and our competitors. So that's number one. I think volume is going to continue to be strong for quite some time. There will be some price pressure as you go through the Inflation Reduction Act, in particular, for us. We will be more than likely selected for negotiation in 2027. And then that price would take effect in 2029. Fortunately, for us, we know the outcome of that price because we have a small biotech exemption that's going to be between 25% and 34% price impact. And so there will be price pressure in 2029 but still a very durable revenue stream that would continue on from there. On the competitive front in 2027, our competitors price kicks in from the government. They've already been negotiated. Our product is pretty closely priced to theirs after their MFP. So right now, we're the lower priced product in the market. And so I think we'll be much closer to parity at that point. So I think any price that we would have to give to stay on formulary and to maintain access would be expected to be very manageable and modest.

And just a very quick one on capital allocation. I think you mentioned multiple times here at the presentation, 30% to 35% back into R&D. You're a company that's generating $750 million to $1 billion in free cash flow and theoretically, that is growing substantially. What about inorganic versus organic R&D? Are we expecting more Soleno like deals or maybe hands in pocket for the time being?

Well, my hands were literally in my pocket. But I think the good part is for Neurocrine right now. We have so much growth on the top line and so much in the pipeline, we don't feel a burning need to go and do something. With that said, we do have the capital flexibility if we wanted to do something. But we like to set up right now with 3 growing products, a pipeline with meaningful data catalysts over the next 12 to 18 months. But if the right opportunity did present itself, it's up to us to look at it to determine can we drive shareholder value with doing that acquisition. But -- sorry, last piece. To be a successful biopharma company, you have to do both. I think if you aren't pursuing anything internally. You're going to miss out and vice versa. And that's been part of the journey of building our pipeline over the last couple of years with Jude Onyia and team a real investment in our early-stage R&D development.

Well said. Thanks very much for joining us today, Matt and Todd here as well. The breakout session will be in Jenny B upstairs. Thanks very much.