Welcome, everybody, to the JPMorgan Healthcare Conference. My name is Anupam Rama. I'm one of the Senior Biotech Analysts here at JPMorgan. I'm joined by my squad: Priyanka Grover, Malcolm Kuno, and Ritika Pai. Our next presenting company is Kiniksa, and presenting on behalf of the company, we have CEO Sanj Patel. Sanj?
Thank you, Anupam. It's always a pleasure, and thank you to JPMorgan for hosting us today. It's good to see everyone here in person. Kiniksa is a well-capitalized, growth-oriented company that puts patients at the center of everything we do. We are building a global generational company by focusing on developing life-changing medicines for patients that are suffering from debilitating diseases. Today, you'll see that we've built a solid commercial organization with the launch of our first product. That product, ARCALYST, has been on the market now for over three and a half years, and it's still growing really well. I'm joined today by Ross Moat, who's our Chief Commercial Officer. I know he's jolly keen to tell you about our commercial execution with ARCALYST, but also how we plan to leverage that commercial organization for other potential commercial opportunities.
Before we get started, please note that we will be making forward-looking statements today that are subject to risks and uncertainties. A review of those risk factors and statements is found on this slide, as well as under the heading of risk factors in our SEC filings. The team at Kiniksa has a solid track record of performance across all stages of drug development since starting the company just eight and a half years ago, and ARCALYST was approved by the FDA in March of 2021, and it's still growing very, very well. In fact, that was only three and a half years after our first clinical studies in recurrent pericarditis, and today, you'll see the scale of the revenues we now have and also the potential for growth this year and going forward.
And it's really thanks to very thoughtful, data-driven decisions, careful capital allocation, and rational commercial mindset across our entire corporate strategy that has allowed us to get to where we are today. In terms of our business development strategy, we've shown that we can find these hidden gems that are ripe for innovation and bring them to market quickly and successfully. And in fact, business development is still a key part of our core strategy. And thanks to our strong financial profile, we continue to look for assets that have strong biologic rationale and validated mechanisms. And we are particularly interested in late-stage rare and specialty programs. This slide four gives you a snapshot of our advancing commercial and clinical portfolio. ARCALYST is the only FDA-approved treatment for recurrent pericarditis.
We've built that market with this IL-1 alpha and IL-1 beta cytokine trap, which is now approved for three indications, also including CAPS and DIRA. We fully intend to capture the additional market and remain the market leaders. We're also innovating with ARCALYST. We have an ongoing collaborative study with the Mayo Clinic looking at cardiac sarcoidosis. Within our clinical portfolio, we have an anti-CD40 program called abiprubart, and that's in a phase II study with Sjögren’s. There's also, by the way, some undisclosed programs that I look forward to sharing with you in the future. Today, we're going to focus mostly, though, on our commercial organization and ARCALYST. I'm happy to report that we've continued to have success with our collaborations.
In fact, Huadong Medicine, a large pharma company in China that we have a partnership with, gained approval for ARCALYST in recurrent pericarditis and CAPS in late 2024. Before I dive further into ARCALYST, I want to touch upon our clinical portfolio, which really builds upon the foundation of success that we've had to date. Abiprubart is a monoclonal antibody that inhibits the signaling between CD40 and CD154. That mechanism has been implicated in a number of autoimmune diseases. We are enrolling and dosing patients as part of that phase 2b study in Sjögren’s disease. That disease represents a potential significant market because there are currently no FDA-approved therapies. There has been some external de-risking of the mechanism through other assets. But it's our high-concentration liquid formulation that provides the potential for differentiation through monthly sub-q dosing.
It is, in fact, the only asset being tested in this disease with a monthly sub-q dose. Kiniksa offers a unique and compelling value proposition. And it's the team's commercialization that's led to significant growth. And in fact, the franchise is already profitable. You can see from this slide that we've had tremendous growth since we launched in April of 2021, seen by marked growth year upon year. And I think a lot of this has been down to very thoughtful investments in our field force, continuing the physician and patient disease education, importantly having effective market access, and continually building upon the brand awareness for ARCALYST. We actually just reported net revenues for 2024. Those are $416.4 million, which represents 79% year-on-year growth. And we've disclosed that we expect our 2025 net revenue to be between $560 million-$580 million.
Before I hand over to Ross, I must say that we are incredibly excited about the future at Kiniksa. We've got a team that knows how to execute commercially, clinically, and we know how to find these hidden gems that are ripe for innovation. I know we have the ability to help many more patients that are suffering from these diseases and also create tremendous value. I'll hand it ov er to Ross for the rest of the presentation.
Thank you, Sanj. Thank you. I share your excitement by what we've achieved so far at Kiniksa, but we have got so much more work to do. We have built a robust commercial organization that has delivered a successful launch serving thousands of patients suffering from recurrent pericarditis. The best news is we are only just getting started.
Our team has driven a robust and sustained growth, and we are well positioned to continue that growth in the years to come. In 2024, we delivered $416.4 million in net revenue, which is a 79% year-on-year growth, and that's been driven by continued increases to our prescriber base. At the end of 2024, we had more than 2,850 individual unique prescribers of ARCALYST for recurrent pericarditis, and of that group, around 730 prescribers have prescribed for two or more patients. Duration of therapy has also continued to grow since launch, most recently to around 27 months as a total average duration of therapy, and importantly, we are only at around 13% penetration into the 14,000 target population, which is that patient group that have two or more recurrences, and as Sanj said, we are happy to provide the net revenue guidance for 2025 of between $560 million and $580 million.
Our team is experienced at both building and executing on commercial strategy. In fact, we led an efficient and effective launch, which drove to profitability for our franchise within just the first three quarters of our launch. We've maintained profitability for the franchise every single quarter since. Today, I'm going to be focused on three key pillars of our commercial strategy. Firstly, driving patient identification, which we do through increasing disease awareness and ensuring that physicians have a proactive mindset in how they diagnose and treat for this disease. That means not just treating for an individual flare of the disease, but treating throughout the disease duration in order to prevent future flares. Secondly, we're focused on healthcare professional and patient adoption. This means ensuring a positive experience when physicians are prescribing and patients are initiated on ARCALYST.
And we do that in large part through our robust patient service support program called Kiniksa One Connect, who are there to support our patients all the way throughout their entire journey on ARCALYST. And finally, we're focused on improving the patient journey. All too often, patients suffer from a diagnostic odyssey, meaning that they suffer multiple misdiagnoses before they get the recurrent pericarditis diagnosis. We are focused on supporting the creation of an efficient network of care, including regional centers of excellence, to provide better support to recurrent pericarditis patients. And in fact, in 2024, we sponsored an initiative with the American Heart Association called Addressing Recurrent Pericarditis, which is a multi-year initiative and is already proven successful, having gone from just two centers of excellence in the U.S. to now 15 centers providing expert care supporting recurrent pericarditis patients.
Our strategy is already shifting ARCALYST utilization earlier on in the disease. If you look at the left-hand side of this slide, you can see that physicians report a growing consideration to prescribe ARCALYST across all stages of the disease, including a third of physicians that say they would consider prescribing ARCALYST on just the first recurrence. That's important because that's where there is an additional 26,000 patients on top of the 14,000 patients that we're targeted on that have two or more recurrences. On the right-hand side of this slide, you can see that that intention to prescribe is translating across into actual prescribing behavior, where we see that around 15% of all patients that are prescribed ARCALYST are on the first recurrence, and around 85% of patients are on two or more recurrences. ARCALYST has evolved the treatment paradigm for recurrent pericarditis patients.
If you look at RESONANCE, which is our disease registry capturing real-world evidence across expert centers in the U.S., you can see from the left that ARCALYST has increasingly become the second-line treatment of choice after NSAIDs and colchicine at leading centers in the U.S. On the right-hand side, you can see a marked decline in the use of corticosteroids as a second-line treatment option. We believe this is important as key centers in the U.S. lead the way with a new treatment paradigm for other centers to follow. So to close, Kiniksa is very well positioned for both near-term and long-term success. We are focused on maximizing the recurrent pericarditis opportunity, as well as advancing our clinical portfolio and maintaining a strong financial profile, which means we have the ability and optionality to invest in further value creation across our business.
Kiniksa is one of a very few biotech companies with proven experience across business development, clinical, regulatory, and now commercial launch success. I hope you can hear from Sanj and I that we are very excited about the future for Kiniksa. We'd like to thank you very much for joining us today, and we'll hand over to Anupam for the Q&A session.
Thank you, sir. I just want to remind everybody that if you'd like to ask a question, raise your hand, and I'm happy to call on you. You can also send a question to the portal, and I'll get it, or you can email me. All three of those work. I'll just start out with a couple of questions. First, on the guidance that you gave, which came in ahead of kind of where street consensus was for 2025, just wondering if you can unpack that a little bit of what's driving the growth in terms of new patients, treatment duration, that type of thing.
Yeah, thanks, Anupam. So yeah, we're pleased to provide that guidance. As you say, it was quite some way ahead of analyst consensus prior to the guidance that we provided this morning. And we think that's just reflective of where we are at the time of launch, the success that we're having, the momentum that we've built up. As we said in the pre-prepared remarks, we're pleased to see that we just consistently add to our prescribing base, whether that's through new prescribers or people coming with repeat prescribers and just making more and more inroads into the opportunity. But we're still at 13% penetration into the 14,000 patient population forgetting about the additional upside of the extra 26,000 patients that are on their first recurrence. So we feel like we've made good progress, but have just a huge amount more to do. Duration of therapy has grown, as you've said.
Whether that will continue to grow or not remains to be seen, but it's something that we will track and continue to report on as we see the latest data. There's certainly room for growth because we do see in the natural history of the disease that patients suffer from recurrent pericarditis generally for three years as an average. That's for patients that have two or more recurrences, and a third of patients still suffer at five years out from the disease, and a quarter of the patients still at eight years out from the disease, so this is definitely a multi-year disease for most patients, so we think it's promising that we're now through to 27 months as a total average duration of therapy. We'll see how that evolves over time.
We are just very focused on continuing to expand our prescriber base, generate more repeat prescribers, and just get further and further into the number of patients that we can help with this disease.
Maybe you could expand a little bit about treatment duration, maybe just more as how physicians view the product and how that's evolved from an acute therapy to maybe is it viewed now more as a chronic therapy?
Yeah, so maybe I'll take that. And John, if you want to add, feel free to, please. But I think there has been a transition since our launch. And as I said during the prepared remarks, we strongly believe that we have changed the treatment paradigm for recurrent pericarditis with the advent of ARCALYST as the only approved therapy in the U.S. People used to treat this disease really as short-term as possible, treating for individual flares, trying to get patients off therapies, particularly if it was corticosteroids, which were kind of used as a second-line therapy, if you like, prior to ARCALYST being available. So you can see the reasons for that because of toxicity, elongating the disease, just having lots of difficulties trying to get patients off therapy.
But with the advent of ARCALYST and the approval of ARCALYST and physicians, both cardiologists and rheumatologists becoming more and more aware both of the disease as well as the treatment, we think that that's making people are being made very comfortable with how to prescribe it. They've gone through it first time, now very often multiple times, of how to prescribe the drug, how to get the approval for patients. And people's mindsets are really changing that this is a multi-year disease and it really needs to be treated as a multi-year disease. And that's exactly how ARCALYST was designed and approved. And as we saw from RHAPSODY as our phase III study and the long-term extension. So we are very much encouraging physicians to use ARCALYST throughout the entirety of the disease with the quite stellar efficacy profile that we have with ARCALYST.
And then, how should we be thinking about, you mentioned in the presentation, just the growth in use in patients with first occurrence versus multiple occurrence and how's that trend going to change, you think?
Yeah, so it's certainly grown over time. And just maybe in the last six months or so, we started to share that data, that there are more and more patients in that first recurrence group. And as the data becomes more robust, we felt that that was something that was worthwhile sharing. And it's indicative of, again, how the treatment paradigm is shifting. ARCALYST has a very broad label, and the label is just for recurrent pericarditis, both for the treatment and the prevention of future recurrences or the reduction in risk of future recurrences. And physicians are really taking that on board. And maybe you start off with a patient, and as time goes on and you get used to prescribing this and seeing the effect that it has on patients, it generates more confidence in how to continue to do that.
Over time, people have moved that earlier on in the disease, which we think is a great thing for patients.
Questions from the audience? Yeah.
In this context of multi-year disease, I noticed that the preparation steps for ARCALYST are quite extensive. It's something like 54 steps in the prescribing information to reconstitute the medication. Are you making plans to, or do you see a need to evolve the product presentation to be more convenient for users?
We have a lot of experience and a lot of confidence in ARCALYST as a lyophilized formulation. And while the package information is particularly detailed to make sure that patients have clear written instructions on how to do that, an important part of the initial process is really to kind of socialize that process of handling the injections. And so, in fact, the fact that there's a loading dose allows kind of a C1, D1 approach where the clinician gives one and then the patient can then follow. And then Ross, with the team of Kiniksa One Connect, has been doing a great job with clinician educators of really taking the patients to the other side of that. So instead of thinking of it as 54 written steps, it's really a fairly simple process of reconstituting the medication.
So in that sense, I think as it comes to the patient experience and how they handle the weekly administration of the drug, it tends to be fairly self-reinforcing. I don't know, Ross, if you want to?
Yeah, I think that's exactly right and well said. And often we do surveys with our patients as well of how they're getting on. We've invested heavily in our patient services program, which exactly provides the support that John is talking about. And generally, patients feedback to us that they get on very well with the preparation and generally find it easy to take. And I think a couple of measurements or metrics that are indicative of that. One is the duration that people are staying on this for a long period of time, and then also the compliance rates, which has generally been 85%-90% compliance rates aggregated ever since launch. So generally, we have very good feedback on that side.
Questions from the audience?
How are you thinking about kind of the ROI that you've seen thus far from the most recent sales force expansion, and how is the marketing message going to evolve over time for ARCALYST?
Maybe I'll start, Ross, and then you can jump in. I mean, we think about it very carefully. I mean, as you may know, we've made some incremental investments in the field force over time on a number of levels. I mean, most basically the field force size from when we launched at around 29 reps, 30 reps to 50 reps or so, and then most recently we announced around 85 reps or so. So we're constantly looking at that size. And as you all know, it's a very dispersed target population as far as the physicians are concerned around the country. So we've been really doing a lot of analytical work, just making sure we get to the appropriate deciles and that we are doing as much disease education as possible. But there's more than that.
I mean, aside from just the field force, we've talked about digital marketing and making sure we reach patients that way. We've got a number of initiatives with the AHA developing centers of excellence and making sure that it's just we find these new cardiologists that are willing to sort of create their own networks and helping reach other physicians all around the country. That's been really important. We've got a couple of celebrity campaigns that we recently announced last year, which is again helping to really further the disease education, so we really see it as a multi-pronged approach, and you can't take your eye off the ball in any one of those areas. There's no one-size-fits-all. You've just got to make sure you invest and you execute and you do the analytics. That's why I was talking about data-driven decision making.
It can't be just made in a vacuum. So it's an incredibly comprehensive approach to making sure the launch continues to go the way it is going. Anything to that?
Yeah, exactly right. I mean, we're just very mindful around where we are, where it's most effective to place our resources, knowing that patients are quite widely spread around the U.S., acknowledging that there is more of an advent in centers of excellence kind of growing up now and increasing. But we're very mindful around where is it best to place in-person resources to really educate and help to keep driving that change in the treatment paradigm versus where is it best to kind of spend digital efforts and web-based marketing, webinars, conferences, peer-to-peer support, and lots of other initiatives.
So yeah, we're very conscious about being very effective and efficient in what we do, which is exactly what you see from how we drove to profitability in just three quarters of our launch and how we've maintained that and grown that substantially in the quarters after.
Are you seeing encouraging prescribing from that next decile of doctors that you were targeting with the sales force expansion? Could you quantify that a little bit?
Yes, we certainly have. But I mean, we've seen, I think, continued prescribing across all deciles. I mean, we're very focused in a targeted approach with the use of our field team on the highest deciles, but then through other activities, we generate a lot of prescribing in the lower deciles as well. I think that's always been the case since launch. We've still got a lot of high decile doctors to still switch on to prescribing ARCALYST. Sometimes it just takes time to get there and providing peer-to-peer education and support and sharing how that's going. Sometimes it just takes time. It's not that we've fulfilled all the high decile targets and now we're going much, much lower down.
When you look at it on an aggregated basis of new prescribers versus repeat prescribers, you actually see that about 45% of all of the ARCALYST prescriptions are coming from repeat prescribers now. So we've really got a very healthy mix, I would say, across the deciles, but also from new prescribers, from repeat prescribers, and just have a lot of opportunity ahead to continue both the breadth and the depth of prescribing.
Questions from the audience? Maybe I was thinking we could switch gears to abiprubart. How's enrollment going in the phase 2b study in Sjögren’s and any timelines to consider for data?
Yeah, no, thanks for the question, Anupam. So as you know, the phase 2b clinical trial, abiprubart and Sjögren’s disease is ongoing, and we're certainly very excited about that, certainly with the involvement also with Sjögren’s awareness as well and the fact that this is a situation where there's no approved therapy and so a real unmet medical need. And so in that sense, having clinical trials as being an available way of testing new therapies is important. In terms of the actual timelines themselves, as you know, in general, we tend not to provide specific guidance of that other than to direct people to clinicaltrials.gov, where those listings are provided.
In general, though, if you look to the literature of how long these trials take to enroll, certainly there are clinical precedents showing that this is usually a couple-year process to get through to the other side of these trials.
Sanj, you kind of talked in your presentation pretty openly about business development and kind of the construct of what you'd be looking for. How active is that relative to focusing on the ARCALYST launch, focusing on execution on abiprubart?
Yeah, I mean, it's incredibly active. I mean, obviously, suffice to say, we've done well on the commercial and the clinical execution side, but what you don't see is all the efforts on the business development side. Clearly, the company got started from a blank piece of paper by business development, but we've got a very, very, very high bar for the assets we bring in. Clearly, ARCALYST has done well and has had vixarelimab and abiprubart, which we outlicensed for a very large upfront amount of money. And we're really proud of the things we brought in, but we continue to look at both in-licensing and out-licensing. We've got a fairly decent focus on it as far as allocation of our time. Eben continues to look at all the areas that we think are ripe or hidden gems. And they go from early stage to late stage.
I suppose most of our focus is really on mid to late stage assets, but it's a very important part of our corporate strategy, and providing something hits our bar, that very high bar, we'll look forward to announcing it.
Questions from the audience? I noticed that you have an IST ongoing with the Mayo Clinic on cardiac sarcoidosis.
Sarcoidosis. We like to have diseases that are very hard to pronounce.
Yeah, I got the cardiac part right, so maybe as you get some of this IST data, could that evolve into something more company-sponsored if you see the right sort of data?
Yeah, so I mean, we're always data-driven in terms of thinking about where to go next, and of course, the fact that ARCALYST is a way of blocking IL-1 alpha and IL-1 beta and so completely abrogating that part of the IL-1 driven pathway and autoinflammation is really foundational, and as a result, we think about other forms of innate immune system inflammation, and specifically in the cardiac space, cardiac sarcoidosis is really one of those canonical diseases, if you will, where innate inflammation plays a part. What we're particularly excited about this clinical trial is the fact that while there's some external proof of concept for IL-1 pathway inhibition and biomarker reduction, this is really the first study to look directly at the myocardium and look for reductions in inflammation by imaging, and so that's kind of the next step that's essentially hypothesis generating, if you will.
Obviously, to get to the other side would require more registrational type trials and potentially outcomes, and so this is kind of the next step in generating that evidence base, and we'll take a look at it and see where it goes, but this is a very efficient way to generate hypothesis-generating data and consider the next steps.
Okay. Any final questions? All right. Thank you, Sanj Patel.
Thanks, Anupam. Thanks.