All right, let's go ahead and get started. Welcome, everyone, to the 44th Annual J.P. Morgan Healthcare Conference. My name is Anupam Rama. I am one of the senior biotech analysts here at J.P. Morgan. I'm joined by my squad: Priyanka Grover, Joyce Zhou, and Rathi Pinhasi. The next presenting company is Kiniksa. I'm presenting on behalf of the company. We have CEO Sanj Patel. Sanj?
Thank you, Anupam. It's always a pleasure. And thank you to J.P. Morgan for hosting us today. I was just thinking, Anupam, we've been doing this for quite a long time together. I think it's almost 20 years, at least 18 years.
Yeah.
I still think we're pretty sprightly and chipper. What do you think?
Yeah, I think so.
Brilliant. I agree. I agree. I am so excited to share our immense progress that we've made this year at Kiniksa, particularly with ARCALYST in recurrent pericarditis. And we have a number of other value-creating opportunities in front of us. We've got a compelling program in KPL-387. This program is now enrolling and dosing patients as part of a phase two, phase three study in recurrent pericarditis. I'm joined today by Ross Moat, who's our Chief Corporate and Commercial Officer. I know he's chomping at the bit to tell you all about the excellent commercial execution with ARCALYST and the revenue growth. Also today, we have Dr. John Paolini, who's our Chief Medical Officer. John will be joining us for the Q&A session. And it's thanks to the entire team that Kiniksa is now a well-capitalized, growth-oriented company.
We've made significant progress on our goal of making a generational impact on the lives of patients. As we start this year, as usual, we have massive goals, but we're squarely focused on creating value. Before we crack on, please note that we will be making forward-looking statements today that are subject to risks and uncertainties. A review of those statements and risk factors is found on this slide, as well as under the heading of risk factors in our SEC filings. The team at Kiniksa has a successful track record of execution across all stages of drug development. We've got proven experience in discovery, clinical, commercialization, and business development, particularly in rare and specialty diseases. In the four and a half years since we launched ARCALYST in recurrent pericarditis, we're now a thriving commercial organization.
We're delivering significant revenue growth and, in fact, changing the treatment paradigm for this debilitating disease. Just this morning, we announced our full year 2025 unaudited net revenue. That was $677.5 million. We also provided guidance for the full year 2026, which was $900-$920 million. At Kiniksa, we're advancing our clinical portfolio through very careful and thoughtful data-driven decisions. We've got extremely disciplined capital allocation and a strong commercial mindset. Pertinently, we have a robust financial position that gives us the ability to invest in ARCALYST, our pipeline, and do strategic business development. Kiniksa has created and proven the market in recurrent pericarditis with the growing franchise with ARCALYST. For us, that's just the beginning. We're focused on expanding and advancing our leadership in this disease.
Aside from ARCALYST, KPL-387 could be an important advancement and treatment option for patients with recurrent pericarditis. Also, this molecule could potentially expand penetration into the addressable market by enabling monthly dosing with an autoinjector. The KPL-387 development plan builds on the extensive experience we have in recurrent pericarditis. The team is drawing upon our expertise from the successful RHAPSODY study, which was the phase 3 pivotal study that enabled the sBLA for ARCALYST. This program consists of a pivotal study and supplemental studies designed to provide a robust data package for both physicians and patients. We expect to have data from the dose-focusing portion of the phase 2/3 study in the second half of this year. We ultimately aim to have this molecule commercialized and approved in the 2028 and 2029 timeframe.
This slide gives you an overview of our commercial and clinical stage portfolio. You see ARCALYST at the top. We still believe there's an awful lot of growth and revenue that we can capture with ARCALYST in recurrent pericarditis. But we're also focused, as you've heard, on getting KPL-387 to the market as fast as humanly possible. This morning, in a press release, we announced that we plan to be in the clinic with KPL-1161 by the end of this year. And that's our Fc-modified IL-1 alpha and beta inhibitor. So as I hand it over to Ross, I think, and I hope you'll see the sheer energy and excitement we have about Kiniksa's future. We certainly know we've got the right strategy. We are focused. And most importantly, we've got a team that knows how to execute. With that, Ross.
Thank you, Sanj, and I certainly share your excitement for what we've achieved so far at Kiniksa. But we have got so much more work to do. And what I'm going to share with you in the slides ahead is why we are even more excited about the future. The ARCALYST commercialization continues at pace, and we have significant opportunity ahead with a clear line of sight to future blockbuster status. We have established the recurrent pericarditis market and so far delivered around $1.5 billion in cumulative net revenue launched to date by introducing ARCALYST as a transformative steroid-sparing treatment option targeting the key drivers of the disease. And we have built an effective commercial engine to identify patients and to change the treatment paradigm. As Sanj said, in 2025, we delivered a full year net revenue of $677.5 million, which is a 62% year-over-year growth.
This morning, we guided to 2026 net product revenue of between $900 and $920 million. In 2026, we are aiming to unlock the next phase in growth for ARCALYST by positioning ARCALYST as the second line standard of care immediately after the failure of NSAIDs and Colchicine for recurrent pericarditis and to increase the utilization in the multiple recurrence patient population, as well as driving greater adoption in the first recurrence patient group. As the knowledge and experience of prescribing ARCALYST has increased over time, we have seen growth in market share as well as use of ARCALYST earlier on in the disease. We've seen steady growth into the multiple recurrence population. At the end of 2025, we'd reached around 18% penetration into the 14,000 multiple recurrent patient group. That's up from around 13% at the end of 2024, which shows good growth, but more importantly, significant opportunity ahead.
On top of the initial target population of patients, we have an additional opportunity with patients in their first recurrence of the disease, which is around 26,000 patients in any given year, and a sizable portion of this patient group could be appropriate for biologic treatment. In fact, around 20% of patients on ARCALYST say that they were prescribed ARCALYST when they were on the first recurrence of the disease, so ARCALYST is evolving the treatment landscape for recurrent pericarditis. On the left-hand side of this slide, you can see data from RESONANCE, which is our real-world evidence disease registry, which demonstrates ARCALYST has increasingly become the second line treatment option at expert centers across the U.S., and that year-on-year growth has been matched by a decline in the utilization of corticosteroids as the second line treatment choice.
On the right-hand side of this slide, you can see that we now have the ACC Concise Clinical Guidance, which is the first U.S. formal guidance publication, which places interleukin-1 pathway inhibition such as ARCALYST as the second line treatment choice after NSAIDs and colchicine and prior to corticosteroids in the autoinflammatory disease that is recurrent pericarditis. With growing utilization of ARCALYST at expert centers, as well as now the ACC Concise Clinical Guidance in place, we aim to replicate this new treatment paradigm nationwide. We have built a robust commercial engine to do just that. We have built a well-established cardiovascular-focused field team and utilized continuous optimization of our field territories based upon where we know recurrent pericarditis patients interact with the healthcare system.
We've also used innovative targeting techniques, particularly around the utilization of AI-driven field alerts to share with our teams not just which physicians to be calling upon, but importantly, when to be calling upon those physicians. And we're in regular communication with many recurrent pericarditis patients who we know have the ability to self-advocate to their healthcare professional for targeted treatment. Additionally, we've seen the increase in pericardial disease centers, up from just two at the time of our launch to now 18 centers nationwide providing specialist care for recurrent pericarditis patients. And we've seen substantial growth in ARCALYST prescriptions at those pericardial disease centers, which is outpacing the growth at other sites across the country. So overall, our deep experience in this market sets a solid foundation for our future growth.
As I move to KPL-387, one of our pipeline programs, this program aims to address key patient needs and to expand the interleukin-1 inhibition market for recurrent pericarditis with a target product profile of KPL-387 of a highly efficacious, well-tolerated drug in a streamlined presentation with reduced dosing frequency and in a patient-friendly administration. This target product profile has been very well received by both patients and healthcare professionals. On the patient side, around 75% of all recurrent pericarditis patients surveyed say that they prefer the KPL-387 target profile over current commercial and investigational therapies. Additionally, around 75% of ARCALYST naive patients state an increased willingness to take an injectable therapy if it's presented in an autoinjector format.
On the healthcare professional side, greater than 90% of healthcare professionals say they are highly likely to prescribe KPL 387 in the context of available commercial and current investigational therapies. You can see that Kiniksa is poised to continue our growth by helping many, many more recurrent pericarditis patients in the years to come. Hopefully, you've heard from the presentation that Kiniksa is well positioned for both future success and value generation. We are focused on maximizing the current commercial opportunity. We provided our net revenue guidance this morning for 2026 of between $900 and $920 million. We are also focused on advancing our clinical portfolio by progressing KPL 387 through both mid-stage and pivotal trials. For KPL 1161, as you heard earlier from Sanj, this is now expected to enter the clinic by the end of this year.
We are also focused on maintaining a strong financial profile, and with growing ARCALYST revenue and with a year-end cash reserves of around $414 million, this enables optionality for investment in additional value creation opportunities for the business. Hopefully, you've heard from this presentation how excited we are about the future that we are building at Kiniksa. I'd like to thank everyone in this room for attending today and those listening online and hand back to Anupam to moderate the Q&A session. Thank you.
Thank you, guys. I'll ask the first couple of questions, and then obviously, if there are questions in the audience, feel free to raise your hand, and I'm happy to call on you. I wanted to dig in a little bit on the guidance, which was ahead of consensus for this year. If you could really drill in on what's driving the strong guidance in terms of patient starts, duration, moving the product upstream, what are the levers that we should be thinking about?
Thanks, Anupam. So maybe I'll take this as for Ross again. So yeah, we provided the revenue guidance this morning, and I think of a strong revenue guidance of $900-$920 million showing pretty significant growth year-on-year coming off the back of another year of very good growth in 2025. So it shows our optimism for the future. As you said, it was ahead of the analysts' expectations and the consensus. There are many things that obviously go into the guidance, but we provide guidance in a way that is to the best of our knowledge at the time and what we believe the market is shaping up to be for ARCALYST. So that includes everything from new starts to duration of therapy and the trajectory that we've either been on and expect to be on throughout the year.
The thing that we are really focused on this year as we kind of kick into the year is that there's been a little bit of a shift from talking about the number of recurrences that a patient has suffered before they then get access or prescriptions for ARCALYST more towards actually the place in therapy of where ARCALYST is utilized. And I think what we've seen from both the RESONANCE data, our real-world evidence data, and then also now from the ACC Concise Clinical Guidance is really placing it solidly as the second line therapy, which is a completely new standard of care. And it's something that we've really been promoting for some time, but the data is starting to move in that direction, as are the national publications.
Making sure that we disseminate that data and really change the paradigm for these patients moving forward is incredibly important.
How have the guidelines in this RESONANCE data changed over the course of the last few months? What are you seeing in terms of second line uptakes since these updates, even if they're anecdotal?
Yeah, thank you. So clearly, we were pleased with the guidance that came out, and that was back in August of 2025, and it's something that really we've been messaging to for quite some time, but the fact is here in recurrent pericarditis that these patients are looked after by a wide degree of cardiologists and rheumatologists, but mainly cardiologists across the country, so disseminating data to a significant number of physicians takes time to work its way through, particularly in a rare flaring disease like recurrent pericarditis, but we were very pleased with the guidance. Clearly, we're using our promotional efforts to try to emphasize the guidance across the country, and while there has been somewhat of a growth in pericardial disease centers around the country, the fact is that this message needs to get out to many, many physicians to really change the treatment paradigm.
That takes some time. Our medical affairs team play a huge role in disseminating data, as do obviously our promotional commercial team, both in person and through digital marketing. These are all things that we've been very focused on, but we know it takes some time to come into a marketplace and really change what's historically been done within this market. I think we're making good progress, but really to the tone of the presentation, that we just have a significant opportunity ahead. Even though we've had a good year in 2025, this is not an organization that rests on its laurels as we move forward.
What makes a pericardial center and what defines that? And you're saying it's growing. So what's driving that growth? Is it you guys? Is it the community coming together? What's driving this growth in the actual centers that treat this?
Sure. It's multifactorial. So when the program started, if dial all the way back to the 2017-2018 timeframe, there were only two centers that were focused on pericardial disease. And it was the process of running the clinical trials, not only globally, but expanding within the United States the number of centers that were enrolling these patients, that was the first step. And then from there, as we launched the RESONANCE registry, there were actually up to 29 centers that were focused on doing research, collecting information about clinical practice in recurrent pericarditis.
That creates a special mindset or focus in the mind of the clinician about how to approach this disease and, importantly, how to be evidence-based or data-driven in the treatment of disease, which means going back to the actual clinical trials, which, as you remember, in RHAPSODY, where half of the patients that entered the trial were, in fact, in a second line treatment paradigm. That was an intentional step that we took when we designed the program to move the treatment paradigm forward. From there, then becomes the initiative with the American Heart Association, etc., where as an organization, they took up the concept of building awareness and training clinicians in terms of how to treat this disease.
And so it started off with 15 centers, grew to 18 centers, and now these groups really contain the necessary expertise in the diagnosis and management of pericardial disease. And that then sets the tone. It sets the example for other centers to follow because as they see the improved outcomes that come from centers like that, they want to have those kinds of outcomes for their patients as well. And that's the snowball that builds the momentum in changing the paradigm of the disease, as Ross and Sanj have mentioned.
Questions from the audience? Maybe we could talk a little bit about treatment duration. When I think about RHAPSODY, that data was pretty evident that symptoms came back if you went off drug, and so there was a view that it could be more of a chronic treatment. You've drawn duration all the way up to three years, but it is also an episodic disease, so how do we think about duration from three years?
Sure, so a couple of things. First, about the epidemiology, and maybe second, about the cadence of pericarditis recurrence, so in terms of the epidemiology of the disease, we have published data that show that the median duration of disease in patients who have two or more recurrences is three years, one third of patients suffering at five years, a quarter of patients still suffering at eight years, and the data set ends at eight years, so that's the median. We have orthogonal data that point to 3.8 years, for example, in the Italian population, so that tells you about how long this disease can last, and patients who have significant risk factors are usually pitched over towards those longer disease durations. Now, for example, when RHAPSODY was starting, you point to how those patients flared. That was, of course, after only 12 weeks of treatment.
And in fact, the median duration of treatment in RHAPSODY writ large was nine months in the primary study, which, as you mentioned, we started to expand ever increasingly outward, showing, for example, that at 18 months, there's still a high flare rate because the disease is still present. So if you withdraw therapy, the disease comes back. And then that was shown again in an Italian cohort after 23 months of treatment. And that's all layered on top of patients having had a year and a half of disease even before entering the trial. So what you're hearing is this was a well-studied population that were five years out into their disease duration and still needing even more therapy. So what does that mean with regard to the treatment duration?
It really points to the concept that if you maintain treatment, so for example, as you looked at the long-term extension, as long as patients remained on continuous therapy and there were no interruptions, the flare rate in the clinical trials was essentially zero. And we even showed that at a recent analysis of the European Society of Cardiology. On the other hand, if you take the older way of treating, which was treat episodically, this is where you get the multiple recurrences. And in fact, patients who are managed with corticosteroids, because you can't stay on them continuously, have to go through serial tapers.
And it was published in two groups, both at the Cleveland Clinic as well as by an Italian group, that that is actually the driver of why these patients have, quote, "so many recurrences." It's the iatrogenic aspect of serial steroid tapers and being subtherapeutic on treatment that then exposes the underlying disease. And that's why, as Ross was saying, the fundamental treatment paradigm shift is not only to use it second line, but also then to maintain therapy throughout the treatment duration so that the flare rate can be really brought down to the minimum level possible. And I think that's kind of how you redefine what patients can expect when they're on an effective suppressive therapy.
Ross, I know you're probably saving a few things for the earnings call, but usually you have some commentary on trends with new prescribers, repeat prescribers. What are you seeing in 4Q? And probably more importantly, where do you see this going? And one of the things I've struggled with is if you're a first-time prescriber, what makes you a repeat prescriber?
Yeah, it's a great question, Anupam, so we did share in the press release this morning that we did see substantial growth in the number of new prescribers in quarter four last year, around 325 additional new prescribers coming in in the quarter, taking the total to more than 4,150 total prescribers, and I guess the way to place that in context is that there were around 25,000 to 30,000 physicians potentially seeing a recurrent pericarditis patient in any given year, so we're making good progress. Switching on additional 325 new prescribers in a given quarter is good progress, but there is just so much more to do in this disease area, and we've got the right team who are up for the challenge of doing just that. Out of that 4,150-ish prescribers, we now have around 29% of those physicians who have prescribed for two or more patients.
So a big part of what becomes a going from a first-time prescriber to a repeat prescriber is ultimately, on one hand, this is a rare flaring disease, so often it's a matter of time waiting for the next patient to come to that particular physician to have the opportunity to prescribe again. But I think the most important thing that we are very focused on is making sure that physicians, one, they know how to prescribe the drug and how to differentiate recurrent pericarditis from the index first episode of pericarditis, and they recognize that this is a time where the patient needs to have a different treatment modality to help with their disease over the years to come.
And then making sure that they have a good experience, that they know how to prescribe the drug, they know how to go through the prior authorization, they see their patient get onto the drug, and then very importantly, they see the type of effect that the patient has on therapy. And by gaining that positive feedback loop and that halo effect enables physicians, one, to be very confident that their patients will get onto drug, they have a great effect, they will look for the next patients that can be supported with ARCALYST, but also it has an element of peer-to-peer education.
When we are trying to create this new wave of how to treat the disease, and now with the ACC Concise Clinical Guidance back in a similar treatment regimen, these are all really important steps for how we educate general cardiologists now of how to identify the patients and what the new way of treating the disease is. One by one, we are switching more and more physicians on. They are becoming repeat prescribers on an ever-increasing rate. The opportunity ahead to continue to go much, much further on both the total prescribing level and the repeat prescribing level is very much there for us.
Final questions on ARCALYST before we move to 387? So maybe on 387, just remind us what the size and scope of the phase two dose focusing portion is and what we would anticipate to learn there in the top line.
Sure. KPL-387, the phase 2 program is ongoing as a dose focusing study. What that means is that it's intended to define the dose level that will be carried forward into the phase 3 pivotal trial. The way that we've approached that is in a four-arm study enrolling approximately 80 patients. These four dose arms are really around trying to identify or clarify that dose to go forward. It's centered on or anchored on the 300 milligram once-monthly dose level, given the fact that the phase 1 data showed that a 300 milligram subcutaneous dose administered would carry forward serum concentrations above the target concentration for about 57 days. That creates sufficient buffer for once-monthly dosing.
Then the scientific question is to ask if you give more drug than that, so in other words, giving the 300 milligrams every two weeks, do you get any additional benefit or not by having done that? Then it also asks the scientific question if you give less drug than that, where are the weaknesses and how does that help you understand the PK/PD relationships of drug concentration and disease activity? And so that's done not with all of the arms, but certainly with the 100 milligram biweekly dose level and the 100 milligram monthly dose level. Importantly, there's no placebo in this arm, and that's because for flaring patients, it's important for the trial to provide a treatment opportunity, if you will, for those patients.
And so both the 100 and the 300 milligram dose levels are intended to give initial drug levels that should be sufficient to suppress disease activity. And of course, that's part of the trial in order to define all of those elements of dose response. So what you see that I'm pointing to is a totality of evidence approach. It's not about any one particular arm, but rather looking across all of the arms of the study as a way of demonstrating to the regulatory authorities that the dose level that we have taken forward or plan to take forward into phase 3 represents the minimum effective dose level, if you will, that would be sufficient for treating this disease long term. So the data that would come forward from that, as Sanj and Ross mentioned, in the second half of 2026, would be focused on that objective.
Given your experience with RHAPSODY, as well as what you're experiencing in the dose-finding portion, once you identify a dose and go into the pivotal, how do you think about that enrollment curve?
So the advantage of how we have set up the phase 2/3 study is, in fact, part of our strategy to move as fast as humanly possible to bring the program forward to patients. So once the dose level has been identified and we pull the trigger on the phase 3 program, the program in its totality will already be in all of those centers around the world. And so that enables us to really catapult forward into the phase 3 program and drive enrollment into the pivotal study in a way that's really different than if you try to build a phase 3 program from the ground up, which is the usual way of doing it where there's like a year, a year and a half lag between the phase 2 and phase 3.
That's one of the innovations that we've brought with this program to the ultimate goal of bringing KPL-387 forward.
John, you talked about enrolling this trial as fast as humanly possible, and I'm looking at a screen that says your motto, "Every second counts." So thank you guys so much for.
Thank you, Anupam.
Sanj and Team.
Thank you, Anupam.