Jazz Pharmaceuticals at Bank of America 2025: Strategic Growth Amid Challenges

On Wednesday, 14 May 2025, Jazz Pharmaceuticals (NASDAQ:JAZZ) participated in the Bank of America 2025 Healthcare Conference, providing a strategic overview of its Q1 2024 performance and future outlook. The company highlighted robust growth in its neuroscience products but acknowledged challenges in its oncology portfolio. Jazz’s leadership expressed confidence in achieving its full-year guidance despite market concerns.

Key Takeaways

• Jazz Pharmaceuticals reported Q1 2024 revenue of approximately $898 million, steady with Q1 2023.
• Strong performance in neuroscience products, notably Xywav and Epidiolex, with significant patient additions.
• Oncology sales faced an 11% decline, attributed to shipping calendar dynamics and increased competition.
• The acquisition of Chimerix and the potential of dordavaprone for treating H3K27M mutant diffused glioma were discussed.
• Strategies to mitigate potential tariff impacts and the anticipation of Xyrem generics in 2026 were outlined.

Financial Results

• Q1 2024 revenue stood at approximately $898 million, maintaining parity with the previous year.

• Neuroscience products, including Xywav and Epidiolex, showed robust growth:

- Xywav added 450 net patients, primarily in idiopathic hypersomnia and narcolepsy.

- Epidiolex experienced double-digit growth despite inventory drawdowns.

• Oncology sales decreased by 11% year-over-year, influenced by:

- Only 12 shipping weeks compared to 13 in the prior year.

- Competitive pressures affecting products like Zepzelca due to new study results.

Operational Updates

• The acquisition of Chimerix was finalized in April, enhancing Jazz’s portfolio with dordavaprone.
• Dordavaprone is a promising treatment for H3K27M mutant diffused glioma, with a PDUFA date expected in August.
• Jazz is well-prepared to handle potential tariffs on imported pharmaceuticals into the US in 2025 through inventory and production adjustments.

Future Outlook

• Zepzelca data from the Phase III IMFORT trial will be presented at ASCO, with FDA submission already made.
• The zanidatumab Phase III trial in GEA is on track for a second-half 2025 readout.
• Jazz is preparing for the entry of Xyrem generics in 2026, focusing on the unique benefits of Xywav.
• An interim readout of the ACTION trial for dordavaprone is expected by year-end.

Q&A Highlights

• Jazz is emphasizing the low-sodium benefits of Xywav in response to upcoming Xyrem generics.
• Positive regulatory engagement with the FDA on dordavaprone, with the PDUFA date on schedule.
• Orexin therapies are viewed as complementary to oxybate therapy, not competitive.

In conclusion, Jazz Pharmaceuticals remains optimistic about its strategic direction and growth prospects. For more detailed insights, readers are encouraged to refer to the full conference call transcript.

Full transcript - Bank of America 2025 Healthcare Conference:

Jason Gerberry, Interviewer, BofA: We’re going get going here with our next company presenter. At the BofA Annual Healthcare Conference. I’m pleased to be introducing Jazz Pharmaceuticals. We’ve got CFO, Phil Johnson and Amal Bertrand, Head of Clinical R and D Oncology at Jazz. My name is Jason Gerberry.

I cover MidCap Biotech and Specialty Pharma at BofA. And so thank you both for joining us. And Phil, I think maybe the logical first place to start, coming off of 1Q results, Jazz historically has some seasonality in 1Q, felt like a pretty strong reaction by the Street in terms of the reaction to the print, even though I thought you reaffirmed the guidance. So maybe just is the high points around 1Q and how you’re feeling about kind of the full year numbers, maybe some of the oncology. It was sort of a broad based, maybe underperformance relative to consensus, but it sounds like you feel good about the full year outlook.

Phil Johnson, CFO, Jazz Pharmaceuticals: Yes. No, I appreciate the question. And first of all, thanks for having us again this year. Great to be out, great slate of investors to meet with, looking forward to the continued interactions over the course of today. So first quarter results, we had about $898,000,000 in revenue, roughly in line with our first quarter of twenty twenty four.

That was really based on strong growth in our neuroscience products with XiWave and Epidiolex in particular. Jason, as you mentioned, XiWave does have a seasonality that affects the first quarter where we see a significant number of patients that are getting their insurance reauthorized and not resulting in paying scripts until that reauthorization occurs. We actually feel really good about the underlying trends we’re seeing there. We added four fifty net patients compared to the end of the year, three twenty five of those in idiopathic hypersomnia. And even with both branded and authorized generic competition, one hundred and twenty five net patient adds in narcolepsy as well.

And we continue to feel really good about how we’re competing with the only low sodium oxybate in our positioning there. Epidiolex has strong growth, double digits. That was affected by some inventory drawdown that was a bit greater in the first quarter than we typically see. We typically see that spread over the first and second quarters, but we feel very good about the progress there. And then oncology was certainly light relative to consensus and did post an 11% decline year on year.

A little over half of that decline was driven by a dynamic here in The U. S. It doesn’t happen often, but it does happen from time to time. Just based on the calendar, we actually only had 12 shipping weeks in the quarter, not 13. So that drove over half of that decline.

We’ve had some dynamics competitively that have been affecting a couple of our products, Rylase, where we talked about the expectation that the COG protocol change for pediatric ALL patients, we expect that to normalize here as we move into subsequent quarters. And then we had some additional competition that we saw for Zepzelca in addition to IMDELTRA in the second line. Great data that came out in the Adriatic study with Imfinzi in first line, limited stage. We’re not in first line, but what that does mean is that those patients are taking longer to progress to second line and therefore for some period of time, lower opportunity for us there. We’re really looking forward to presenting at ASCO in June the data from our Phase III IMFORT trial, looking at Zepzelc in the first line maintenance setting, having that published as well, rapidly seek NCCN treatment guideline inclusion.

We’ve already made the submission to the FDA and then begin promotion once that would be approved. So we feel good about the mid to longer term trajectory for Zepzelca.

Jason Gerberry, Interviewer, BofA: Yep.

Phil Johnson, CFO, Jazz Pharmaceuticals: We also are really pleased that we closed in April the acquisition of Chimerix, bringing dordavaprone into Jazz. This is a product that if approved would be the first treatment effectively, pharmaceutical treatment for these patients. This is a specific type of brain cancer. This is H3K27M mutant diffused glioma. It’s a lot for a finance guy to say.

But these are patients who have really poor prognosis. Typically, these are younger patients from time of diagnosis might have a year to live from a time of recurrence of their disease, maybe only five months. So really looking forward to not only presenting some of this data at ASCO as well, but the upcoming PDUFA in August and potentially then rapidly bringing this treatment option to patients who are suffering from this severe disease. Also feel good about momentum we have in our oncology portfolio, which Jamal can talk about more. We’ve got a number of different things coming up for zanidatumab, where we’ve got a CHMP recommendation for approval in Europe in second line BTC.

Typically that’s a sixty seven day period roughly to get the EC to then make their decision, but confident in the position we have there, looking forward to marketing that in Europe. We’ve got data we’ll present at ASCO also for zanidatumab, not just for Zepzelca and or davaprone. This particular case, some data I think I’m particularly interested for people to have a chance to see is in our phase two study in GEA. This is a study that Zym and Jazz have been running. In the past, we presented data multiple times on this, but never were in a position to actually show a number for overall survival because it wasn’t yet mature.

So we’ll have updated data to show there. It underscores what we’ve been seeing over time in that study our confidence in the first line Phase III study that is still on track to read out here in the second half of the year, probably the most widely cited and catalyst Jazz moving forward. Maybe two last things real quick. There’s been a lot going on in Washington that can affect the sector and our company. We talked on our earnings call about one of those, which is tariffs, have positioned the company well so that if tariffs do come in on imported pharmaceuticals into The US, there really be no, if any, impact to 2025 financials.

And therefore our guidance, obviously, these last longer, 2026, we’ll need to look at. We do have ways of mitigating that, not just with inventory, like we have the 2025 exposure, but also where products are produced. Our biggest exposure would be with CyWave. We do have U. S.

Production third party capacity to be able to do that. And then we have some other mitigating strategy maybe for other parts of the portfolio. And last, I’d say even with the 900 or so million dollars that went out the door to acquire Chimerix, we’re still in a really strong financial position. So whether that’s investing in our current marketed products, investing to rapidly move products through our pipeline, or engaging in additional corporate development to bring in, whether that’s through licensing or acquisition, other assets from outside our walls to bolster our future growth prospects. We’re in a really strong position to do that.

So feel very good about the year overall. We did confirm our top line revenue guidance had really very modest tweaks. Other than that, as far as an operational perspective, we pulled down our R and D slightly based on the early readout of a couple of studies, Phase IV studies for Xywav. The other changes that made the quarter a bit messy, I think for people to understand, really related to the Chimerix acquisition and then some legal settlements that we announced earlier.

Jason Gerberry, Interviewer, BofA: Yep. So basically flattish top line growth year on year in 1Q, a lot of drags, still have mid single digit growth midpoint for the full year. So kind of next three quarters, you’ll start to see an uptick in growth as some of these lag factors kind of come off and you get to a more normalized growth for each of key franchises. Exactly. Okay.

So tariffs, real quickly, we don’t need to belabor the point. I think the points on Xywav were very encouraging, at least from our perspective. You get a few questions about Epidiolex. I know The US and The UK have worked towards a trade deal, but I don’t know if we have much clarity around pharma specifically. But any viewpoints there?

Phil Johnson, CFO, Jazz Pharmaceuticals: Yeah. We don’t have clarity yet on tariffs on pharma. We keep hearing it’s going to come, it’s going to come. I expect at some point there will be something. I don’t think it’s just going to be talk without any action.

I think it’s encouraging that The UK and The US were able come to an agreement relatively quickly and seems amicably hopefully that portends something good for what may happen with further tariff discussions between the two countries, including on pharmaceuticals. Obviously, things have been highly volatile, so all I say is we’re monitoring that with interest and we’ll be able to make appropriate actions, I think, based on what happens with both The UK as well as other countries

Jason Gerberry, Interviewer, BofA: maybe for Amal, just FDA disruption, you have two action dates on two cancer drugs. So is the messaging status quo with your interactions with the review teams as you go into these PDUFA for both Subselka and the recently acquired drug?

Amal Bertrand, Head of Clinical R and D Oncology, Jazz Pharmaceuticals: Yes, mean really we haven’t seen any disruption thankfully, and things continue to go on time. So it’s very encouraging to see that there have been no delays or disruption from this.

Jason Gerberry, Interviewer, BofA: Yep, okay. And maybe, Phil, some high level questions before we go into the product specifically, but there will be true Xyrem generics next year. Xyrem, the brand, was even lower than I think it was like $3,040,000,000 dollars in 1Q. So annualizing below $200,000,000 I imagine it’s going be even the exit rate will be even lower given kind of how that has evolved. So I guess the question is what do you think true generics look like?

Because you’re going to kind of explain that to investors. I’m sure you’re going get question a lot today. You know, my understanding is they would have to drive indirect substitution with a product that they’re not substitutable for in Xywav if there was to be a meaningful impact of those or compete with HICMA effectively if HICMA chooses to remain in the current arrangement that you have.

Phil Johnson, CFO, Jazz Pharmaceuticals: Yeah. So you said there will be generics. Our working assumption is that’s likely to be the case, but we don’t know for sure. Just real quick to recap, there are two dynamics at play, one related to the main authorized generic player, HICMA, the other two other generics. Let me start with the other generics first.

Regardless of what HICMA decides to do, those generics do have the ability to come into the market with their own true generic starting in 2026 effectively. HICMA has had for some time and still has the ability to come in earlier than that date. If they were to do that, the others also could advance their entry into the market. So maybe the calculus for HCPMA is a little complicated by that fact. Let’s assume that we get into 2026 and we do have HCPMA and other generics coming into the market.

What would be the impact to Jazz? So first, Hickma can either market the authorized generic or a true generic, but not both. So if they’re marketing a true generic, that means that our authorized generic revenue would go away. We would presume that the Xyrem additional branded sales, whatever is still left, would likely come down pretty substantially in the face of true generics. And then the question mark is what does happen to Xywav because these are not generics of Xywav, they’re generics of Xyrem.

Xywav is the only low sodium oxybate in the market, whether that’s current branded or authorized generics or potentially then generics. So it’s not AB rated. We’re well positioned there. The FDA itself has commented on the significant health benefit, safety benefit that accrues of having low sodium as opposed to high sodium. We think this is relevant for physicians and patients and is the reason why we’re not only building the IH market steadily but also faring very well on narcolepsy with branded and authorized generic competition.

And I think increasingly payers hopefully will value this as an attribute of the product as well. So what will happen to Xywav next year is the biggest question mark. I think it’s highly dependent as we look at different analogs on how many generics come in, what the timing of that is, what their pricing strategy is. We would expect in any event there’d probably be some disruption to Xywav. But again, feel that we’re well positioned.

We’ll continue to drive home the messaging that makes our product unique and differentiated that others cannot provide to patients and sort of see where the where we land is going in 2026.

Jason Gerberry, Interviewer, BofA: When the settlement was originally structured with HICMA, I remember there were terms, right? Like where there was a first term, the second term of the deal, and that affected royalties and different things like that. So is there a point of clarity, the arrangement with Hikma, like do they have to re up on the arrangement and that gives you then clarity for a new set period of time? Just wondering how to think about that dynamic.

Phil Johnson, CFO, Jazz Pharmaceuticals: Yeah. So the set points you’re referring to were on the royalty rate that effectively was up to the current rate starting early last year. And then the current contract current agreement does go through the end of twenty twenty seven. So they’re able to continue with authorized generic under these terms through that time if they choose to.

Jason Gerberry, Interviewer, BofA: Okay, but they could break free from that if they choose to launch their own Yeah,

Phil Johnson, CFO, Jazz Pharmaceuticals: they’ve able to do that ever since they had signed the agreement.

Jason Gerberry, Interviewer, BofA: Yeah, okay. All right, so maybe shifting gears to zanatetamab and the phase three GEA, that’s going to be a still a second half twenty twenty five update hasn’t been narrowed at all, but no PFS delays announced on the the most recent earnings call. I guess how how confident are you now in terms of I imagine you’re looking at the PFS event accrual that, you know, that’s going to be a firm data update timeline?

Amal Bertrand, Head of Clinical R and D Oncology, Jazz Pharmaceuticals: Yeah, mean, as time goes by, we’re more and more confident, but we’re still very confident in the second half of twenty twenty five so that there hasn’t been any changes there.

Jason Gerberry, Interviewer, BofA: Okay. When we looked at Keynote eight eleven, the last major GEA phase three in this space, I think from study start to data, that would kind of imply something like in the fall, like September ish timeframe. Is that reasonable analog, or would you say, hey, each of these trials has some nuances and differences and it’s probably hard to be too precise?

Amal Bertrand, Head of Clinical R and D Oncology, Jazz Pharmaceuticals: Yeah, mean if you look at all the trials that have read out for that control arm with Herceptin, the TOGA trial, Jacob, and the KEYNOTE-eight eleven, they’ve been consistent, give or take a month there. So, from the control arm perspective, we don’t expect any major surprises. So what we’re hoping on is that delay is probably coming from the two arms that contains enzanitamab.

Jason Gerberry, Interviewer, BofA: And so I know that a question that you guys get a lot is around IHC three plus as a demographic enrollment dynamic. And you could look to very small data set that Inheritu had, which had crazy high PFS, right? And they had like 90% IH3 plus As a proportion of patients, your Phase two data was in the high eighty percents, I believe. So on the one hand, I think investors maybe have concern if you go to a Phase three, are you going to have similar high rates of IH3 enrollment? And does that as a maybe prognostic factor maybe not benefit the Toger regimen as much?

Because when we looked at a lot of like phase three trials, the performance has kind of gradually come up I feel like on Toger regimen, not that much. And in all those trials I think the IHC three representation in those studies has creeped up as well.

Amal Bertrand, Head of Clinical R and D Oncology, Jazz Pharmaceuticals: Yeah, I mean they’ve been consistent roughly across the different trials in the 70% range that are or a bit more even that are IHC three plus. So, I don’t expect any other major differences there. Now, do stratify by IHC two plus and three plus so we’re hoping that that’s going be equivalent between the arms.

Phil Johnson, CFO, Jazz Pharmaceuticals: I say it’s not speaking to the composition of IHC two plus or three plus but you’re just looking at the data from the control arms in those various studies and the doublet we’ve seen sort of six point seven to eight point one months of progression free The last update that we had provided last year on the phase two study, it’s not the phase two, on the phase two was fifteen point two months. So even with degradation potentially in phase three, we still feel like the data we’ve been seeing to date is indicating that this molecule is acting very significantly in this patient population, driving long progression free survival, long duration of response, which sort of underpins our confidence in a Phase III readout later this year.

Jason Gerberry, Interviewer, BofA: Yeah, okay.

Amal Bertrand, Head of Clinical R and D Oncology, Jazz Pharmaceuticals: And this has been verified by two studies run by BeiGene and one by ZymWorks, which one we will be having updated data at ASCO that we’re excited to share.

Jason Gerberry, Interviewer, BofA: So on the co primary endpoint, right, you have PFS but you also have no OS detriment. And my feedback that we hear from clinicians is they want to see maybe at least a hazard ratio on OS similar to Keynote eight eleven, at least in your triplet arm containing the PDX agent. Is that the right way to think about it? And in general when we get the initial update, I think you’re saying the OS data will be more mature now given some of these PFS delays. So I’m trying to get a sense of when we get this initial update how much of a read through we’ll have to OS.

My sense is that if the PFS hazard ratio is very low, that tends to correlate pretty well with OS and that should have some read across or if we should just expect mature OS data later this year.

Amal Bertrand, Head of Clinical R and D Oncology, Jazz Pharmaceuticals: Yeah, just to clarify that the primary endpoint for the study are dual primaries. They’re not co primary, meaning they’re not independently tied to each other. So, we just have to be positive on one, right? And we’re very confident with the PFS. The OS is going to depend on the maturity of the events.

In terms of approval based on PFS in the frontline setting, is an approvable endpoint, and we’re not concerned there. But yes, absolutely, they’ll need to be follow-up with the OS data like any of the other phase three studies that I’ve read out. Okay.

Jason Gerberry, Interviewer, BofA: And with respect to OS, you wouldn’t expect that to be muddied at all by the post progression therapies that are available to patients. Mean this is a European trial, if I understand they’re going have access to Inheritu post progression, but they could get a zany based regimen could easily kind of convert to Inheritu as could a someone who’s getting the comparator therapies.

Amal Bertrand, Head of Clinical R and D Oncology, Jazz Pharmaceuticals: Yeah, that’s correct. I mean, based on the data that we have from the phase two studies and the OS that will be presented at ASCO that we’re seeing, we feel confident that that’s not going to be affecting the OS data at survival for patients progressing onto the different arms. So, we don’t anticipate that to be a major risk factor, especially based on the known OS data from HER2 that we know. And to your point as well, most of the patients were well, all of the patients were ex US for the three zero one trial, so.

Jason Gerberry, Interviewer, BofA: How representative do you think that ASCO OS data is going to be from your smaller supportive study?

Amal Bertrand, Head of Clinical R and D Oncology, Jazz Pharmaceuticals: Yeah, mean, you know, it is definitely a smaller study, but we did enroll in The US, Canada, as well as South Korea for that study. So, you know, European and Asian population, it should be fairly similar.

Jason Gerberry, Interviewer, BofA: Yep. So maybe speaking to data scenarios with the GEA study and focusing on the Zany chemo arm, arm B, as we understand it, How that I feel like everything is tied to that. Right? Like that has to win, that has to show that that’ll give doctors the truest apples to apples comparison to and then and then there can be arguments to XANI should be incorporated into whatever type of regimen that you have, right, as long as you show some incremental benefit with your PDX treatment arm. So I’m curious, you know, we oftentimes hear of like two, three months PFS delta, right, as sort of a clinically meaningful threshold.

Would you disabuse us of that notion, or do you view things differently?

Amal Bertrand, Head of Clinical R and D Oncology, Jazz Pharmaceuticals: I mean, again, going back to the data that we’ve presented for the for the Phase II studies and, you know, a doubling of what we’ve seen historically for Herceptin combination as chemotherapy, we’re hopeful that it’s going to be better than that. You know traditionally yes, three months has been a minimum.

Jason Gerberry, Interviewer, BofA: Yeah, mean I guess the reason I say it that way is you know there’s always the phase two to phase three effect size compression. How do we interpret some of the data sensors and the median duration of response? And you know so I guess kind of contextualizing those data points, small data sample as well, I imagine Inheritu is going to come down a lot from its phase two PFS benefit as well.

Amal Bertrand, Head of Clinical R and D Oncology, Jazz Pharmaceuticals: Yeah. Yeah, mean traditionally we do see a shortening of PFS transition from phase two to phase three, and again, the phase two is a single arm trial. That being said, we’ve looked at it independently in two different studies. The response rates that we see are also very compelling, the duration of response, and now mature OS data that gives us reason to believe that even if there is some attrition, not going to be significant to affect those study results. And the study is well powered for that too.

Jason Gerberry, Interviewer, BofA: Yeah, and I guess the other risk factor, you will call it that, is the Jacob trial and what we saw with Herceptin Perjeta, which sort of similarly targets what know zaniditanib does. There’s the mechanistic rationale as to maybe why zaniditanib through the clustering of the receptors may drive more of an anticancer effect. We can look to the xenograft data, we can look to maybe BTC. What are some of the data points that you feel like most strongly make the argument as to why zanitamab will achieve different outcomes than the modest PFS that Herceptin Perjeta saw in the frontline GEA setting.

Amal Bertrand, Head of Clinical R and D Oncology, Jazz Pharmaceuticals: Yeah, I mean, so just you know, state that we are comparing ourselves just to Herceptin in the three zero three Yeah, yeah. But we do have some data preclinically that do show that ozanidatumab in preclinical models is superior to the combination of both Herceptin and birtuzumab, and if you do some cross trial comparisons, we don’t like to do that based on the data that we have from the BTC trials and other patients with cancer where there has been data for the combination of pertuzumab and Herceptin, we’re encouraged that the response rates are significantly better than the two together. So, some indirect clinical data, mechanistic data, and preclinical data that’s showing us superiority against that combination.

Jason Gerberry, Interviewer, BofA: If the data and GEA as you hope, how do you start to feel about breast cancer study, right? Because you’re in another HER2 amplified tumor setting in a setting where you’re compared to Herceptin chemo, so same comparator, the same type of malignancy setting, does that offer strong read through to the metastatic breast cancer study, or do you feel like that’s maybe a more complicated study in sort of the postin HER2 setting? I know you have some data in postin HER2, but

Amal Bertrand, Head of Clinical R and D Oncology, Jazz Pharmaceuticals: Yeah, I mean, we, you know, this is not the only subset of data that we have from the GEA. We do have several other breast cancer studies that have been run where we’ve seen the activity of Herceptin with different combinations and really encouraged of zanidetamab, sorry, with different combinations and really encouraged by that data for breast cancer. So, definitely the GA positive trial will give it a big boost because then we’re directly comparing it, but there’s also ways to believe that we have compelling data on the breast cancer space that should allow us to have a positive study for the metastatic breast trial. Okay.

Jason Gerberry, Interviewer, BofA: You also have the pan tumor study that’s going to be registrational. Can you remind us how many different settings and is this upside to that? Think you guys talked about a $2,000,000,000 I imagine that’s mainly breast cancer and GEA of pan tumor. How big can pan tumor be? What’s going to be the cadence of data flow around that?

I imagine it’s all going to be one NDA or BLA submission, right?

Amal Bertrand, Head of Clinical R and D Oncology, Jazz Pharmaceuticals: Yeah, that’s right.

Jason Gerberry, Interviewer, BofA: When it’s completed. So is the will there be incremental data sets along the way or more of just one data update to the street with this type of study?

Amal Bertrand, Head of Clinical R and D Oncology, Jazz Pharmaceuticals: Probably one data update is what we’re anticipating. Now that study has three different cohorts. One of them is cohort one, which is in all HER2 expressing cancer that have progressed on treatment but have not received the prior HER2 agent, and then we have two other cohorts for GA and breast cancer, and those are, you know, how we assess those may change depending on the data for the GA trial and the breast cancer trial. Okay.

Phil Johnson, CFO, Jazz Pharmaceuticals: And in terms of the 2,000,000,000 number that you referred to, we talked about before, that does include BTC, GEA, pan tumor and the metastatic breast. So

Jason Gerberry, Interviewer, BofA: maybe shifting gears to your core sleep franchise and given that there’s going to be a couple orexin data sets coming up here, we’re getting this question more in the context of how the ripple effect competitively to other modalities including oxybate. I think what we hear and kind of what we understand like these agents should work in narcolepsy type one, that’s pretty straightforward. More I guess the open question is narcolepsy type two and idiopathic hypersomnia, IH being kind of your big growth source. So when we think about NG2 NIH, weight promoters are available generically, So when you look at orexins as competitive threats, I mean sometimes KOLs call them souped up weight promoters. So do you feel like do you see the orexins as competitive in NT2 or IH?

I get the logic in NT1 and it’s kind of hitting a root cause of the disease, but maybe just thinking about the competitive interplay in those other settings.

Phil Johnson, CFO, Jazz Pharmaceuticals: So I guess the short answer would be we see these therapies being complementary to oxybate therapy and not competitive. Again, I think there’s been promise that people have posited that the orexins will actually help to, while they’re taken during the day, normalize and restore quality of sleep during the night to get at the underlying reason for some of the disability effectively that patients with narcolepsy and IH have. That’s not what we’ve seen so far in the data, though. What we’ve seen in the data so far is that they actually are very potent daytime waking agents, maybe the most potent that could come to market and be available, but not actually helping with quality of nighttime sleep and addressing the underlying cause of the diseases. And in some cases, actually for longer half life molecules or if it’s dosed later in the day, leading to some level of insomnia early in the night that’s not helpful in the patient having restorative, less disturbed nighttime sleep.

So again, we know the promise is out there that some people have said for what orexins could do in that promise. If it were to hold true, there could be competition with oxybate. But again, that’s not what we’re seeing so far in the data. It sees it being really potent, effective daytime waking agents that would be used in combination with oxybate therapy.

Jason Gerberry, Interviewer, BofA: Maybe I’m jumping around topics here in the last two minutes, but Amal, the Chimerix acquisition, you know, the late line given the short lifespan of these patients, it seems like the real opportunity if you can break in the frontline, the phase three action trial is ongoing. I think there’s an interim, the first interim in second half. You know, I don’t know, has that come and gone yet or confidence that that can kind of yield a positive result in early interims or would you say, hey, we’re going to need to wait probably for second or final analysis in this setting?

Amal Bertrand, Head of Clinical R and D Oncology, Jazz Pharmaceuticals: Yeah, it’s hard to speculate on that one, to be honest. But so far, the trial seems on track for that initial interim readout for the ACTION trial in, I believe it was towards the end of this year. But, I mean, the bigger population is definitely the frontline trial, and we’ll see the results of the action trial soon, so there’s not going to be that much of a lag hopefully between

Phil Johnson, CFO, Jazz Pharmaceuticals: the two. I will say, without committing, there’s going to be a change to protocol. We are working with our Chimerix colleagues to look at the protocol for the study. If I just go back to our prior licensing deal, do a Zynworks, you may recall that we worked with them as well and looked at the powering for the study that was underway. And we decided significantly increase from seven fourteen to nine eighteen patients recruitment for the trial.

Trial was well powered for PFS, but we wanted it to be more sufficiently powered for overall survival. So I just want to let you know, we are looking actively with our colleagues at ChimeraX on do we feel like we have the right setup for this particular trial for it to be successful? If we determine, yes, we do, then we probably make any changes to the protocol. If we think there are ways we can enhance the protocol that can increase chances of success, we would do that.

Jason Gerberry, Interviewer, BofA: And with regulatory for the second line opportunity, just sort of the puts and takes or pros and cons here, it is a genetically predefined patient population that tends to afford some latitude. I think with respect to not having a control arm in your study. There are a couple of data sets here. So I don’t know, just your overall confidence and approval as you guys diligence the opportunity.

Amal Bertrand, Head of Clinical R and D Oncology, Jazz Pharmaceuticals: Yeah, I mean, so far we have been encouraged by the fact that the FDA hasn’t requested an outcome, which means that hopefully it’s not going to be something controversial, and we’re still on track for that PDUFA date. So, you know, engagement with the FDA has been good. Okay.

Jason Gerberry, Interviewer, BofA: Well, we’re out of time, so thank you both for joining us.

Phil Johnson, CFO, Jazz Pharmaceuticals: Thank you. Really appreciate Thank you.

Jason Gerberry, Interviewer, BofA: All right.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.