Hi there. Welcome to Evercore ISI's Healthcare Conference. My name is Lisa Bayko, one of the Evercore ISI analysts here, and very pleased to have a fireside chat with Kiniksa. Kiniksa is a name I've been covering since its inception, and here with me today is Ross and Eben, Chief Operating Officer Eben and Chief Commercial Officer Ross. Let's start with a brief company overview to get everyone on the same page.
Yeah, thank you very much, Lisa. So thank you very much for hosting us and to Evercore as well. It's a pleasure to be here again. Thank you to everyone in the audience. We're happy to go through both the ARCALYST performance and the continued successful commercialization that we have with ARCALYST and recurrent pericarditis, as well as looking at our pipeline as well. So we're looking forward to the conversation, particularly around our phase II-B study of abiprubart as a CD40 inhibitor in Sjögren’s disease. So happy to go through those. Before we start, we will be making forward-looking statements today, which are subject to risk and uncertainties, and a copy of those can be seen in detail in our SEC filings. So with that, we're happy to go straight into the questions.
Okay, wonderful. So let's talk about, you know, you haven't reported fourth quarter or in the fourth quarter. Talk about sort of what we've seen historically for abiprubart, kind of, you know, as we talk about seasonality and all that kind of stuff.
For ARCALYST?
That's for ARCALYST.
Yeah, ARCALYST. Yeah, so we'll start with our commercial, and then we'll go on to abiprubart when you're ready on the clinical side. So yeah, so thank you. I mean, you know, the commercialization has continued to be successful through the end of Q3, as most recently reported. We continue to be happy with the performance and both all the metrics that underline the performance, whether that's the prescriber growth. We've obviously delivered net revenue increases every single quarter since the time of launch. And we're fairly nascent in the opportunity as well. We're around 11% penetrated into the 14,000 patient population. So on one hand, being pleased with that, on the other hand, also acknowledging the upside that we have ahead and the huge amount of work that we still have to do to deliver for patients in this disease space.
In terms of seasonality, I guess on one hand, there's kind of the gross-to-net seasonality, which we've seen historically since the time of our launch, where we haven't had massive swings in the gross-to-net. But historically, having seen, you know, a bit of a higher rate in Q1, it trailing off in Q2 and Q3 and Q4, kind of ticking up a little bit again towards the end of the year. So there's a little bit of seasonality related to gross-to-net. Outside of gross-to-net dynamics, we don't necessarily see any particular seasonality, certainly around the epidemiology. There is maybe to some degree around pericarditis as the acute index episode of pericarditis, which is often following viral infections, but not necessarily pulling through into the recurrent pericarditis population. So nothing really of note there.
So I think on our side, it's really being focused on the continued execution that we've had since the time of launch and continuing to play that through into the marketplace and the opportunity that we have.
What are the key levers you have to increase that 11% penetrance up higher?
Yeah, so as I said, you know, we're pleased with the 11% mark, and it shows that there's substantial headroom ahead. But, you know, that's also kind of delivered good success so far in the launch. We continue to be focused on raising disease awareness. It's something that often goes underdiagnosed and misdiagnosed. These patients often suffer, well, on average, 2.7 misdiagnoses before they get an accurate recurrent pericarditis diagnosis. So disease awareness continues to be incredibly important for us as kind of a fundamental, you know, core pillar to the work that we do as part of our strategy. And that plays through in many different ways, both in terms of our field team, whether that's our commercial team or our medical team, in-person interactions with the healthcare professional community, and also on the patient side as well from direct marketing.
But also through peer-to-peer education conferences, we make sure that we are very focused around our in-person utilization of our resources, making sure we're highly targeted in our approach because the patient population is relatively dispersed across the U.S. So we need to make sure we use our in-person resources very wisely. But at the same time, focusing on digital marketing, conferences, peer-to-peer education and support, and making sure that we go much broader than that as well. And I think we've seen the success of that play through. If you look at the prescriber number, for example, which is one of the key metrics that we have been sharing on our quarterly earnings call, most recently from Q2 to Q3, that grew by around 250 individual prescribers to more than 2,550 prescribers, unique prescribers since the time of launch. So that has continued to grow steadily.
And when you overlay that, we're thinking that, you know, we're targeting around 11,000 individual prescribers with our field team. And certainly not all of those in the 2,500 group are within the 11,000 target group. So again, it's another way of showing that, you know, we've seen good steady progression since the time of launch, but acknowledge that there's a huge opportunity ahead to continue to switch on new prescribers and also generate repeat prescribers and making sure that they have a good prescribing experience, that the patient gets onto drug and the patient reports back, you know, good efficacious messages to the doctor, which will then encourage them to look for and seek treatment for further patients.
I think you've checked the box there. I mean, all the feedback we get on sort of the experience with the product is very positive. It seems like finding the patients is the key thing. Can you talk about this repeat prescriber concept? What is the average number of prescriptions per prescriber at this point?
Yeah, it's a great question. So we haven't shared the average number directly. We've just shared that there's around 2,550 individual prescribers, and around 640 of those have written for two or more patients. So acknowledging that this is a rare and flaring disease, often there's some lag between someone becoming an initial prescriber and then having the opportunity to find the next patients and become a repeat prescriber down the line. But we're confident that if we continue to make sure that they have a good prescribing experience and patients get onto treatment and see a good positive effect being on treatment, it will come with time. And we've seen it gradually growing. So as I say, it's around 640 repeat prescribers, which is about 25% of the total population.
Is there any effort to sort of consolidate the market into some sort of, you know, kind of like centers of excellence or this kind of thing to maybe, you know, concentrate the field a little bit more? Is that any particular?
Yeah, so I think there's been an emerging area where, you know, in the early stages of the launch of ARCALYST in recurrent pericarditis, there were probably only a couple of key centers, recognized centers, if you like, focusing on pericardial diseases. That landscape has evolved over time, and there have become more, and there's a collaboration or sponsorship that we have with the American Heart Association called Addressing Recurrent Pericarditis, where there are now 15 recognized centers around the U.S. that have expertise and a specialist interest within pericardial diseases and recurrent pericarditis specifically, so we're certainly seeing the landscape, you know, evolve over time.
You know, one of the key levers in these kind of markets too is Field Force, right? If you can expand the message. You've gone through a couple of expansions already. Do you feel like you're right size? Should we expect in the future for maybe further expansion? I mean, to some degree, it would make a lot of sense. I'm just curious how you're thinking about it.
Yeah, so it's something that we always look at and will always be very data-driven in our approach to the market. You know, going back three and a half years ago now, when we first launched in recurrent pericarditis, we had a very focused approach with a team of around 30 representatives in the field, and our key focus at that time was to make sure we got a good launch under our belts, that we were educating physicians and their offices in how to diagnose patients and how to prescribe ARCALYST and going through the prior authorization process and making sure that they had a good experience with that, and our focus was addressing the market and driving towards profitability for the collaboration that we have as quickly as possible, which we achieved at around three quarters into the launch.
And then we took the decision to further invest in the field team. As you said, we've done that a couple of times now, most recently at the start of this year, having around 85 representatives in the field team. And it's something we'll always look at, whether it's right sized or not. But we're very data-driven in our approach, and we will take the decisions that we believe will create the best value and generation of value for the organization.
Okay. One thing that we've seen has been, I think, a really big success and source of sort of a lot of upside has been duration of therapy, which has been much longer than I think even the maybe more liberal estimates initially. Do you see this as a chronic therapy, or is this a therapy that it will be for some period of time? Like, where is this going? Because every time you report, the duration of therapy seems to lengthen, right? So are patients staying on this really at this point chronically, or how should we think about that?
Yeah, so we certainly see this as a chronic therapy, and we see recurrent pericarditis as a chronic disease, in particular with patients that are suffering for two or more recurrences. If you look at the natural history of the disease, for patients that suffer for two or more recurrences, the median duration of the disease is around three years. And then still a third of the patients are suffering from the disease at five years out from their initial episode, and a quarter of the patients at eight years. So for those patients, it's definitely a multi-year chronic disease. And ARCALYST has really been designed to be treated throughout the duration of the disease. So you're right, we have seen the duration grow over time.
I think when we first launched, it was around six to nine months' worth of duration of therapy, and that's grown most recently to 27 months in total. You know, we see some patients that stop therapy and suffer from the disease still and go back onto therapy. Thankfully, it's a very easy process generally for them to do that. We see it as a chronic therapy, and we really focus on educating physicians that this is a chronic disease and the drug needs to be treated throughout the course of the disease.
And then as you kind of expand the scope into sort of first recurrence, how does that affect, do you think, the duration of therapy? Will first recurrent patients, you know, treat for a shorter period of time, or is there no difference for those who are maybe like highly refractory?
Yeah, so it's interesting that we really focus our commercial efforts on those patients that have two or more recurrences because that's the patient group that have the highest burden of the disease. They're the group that have the most similarities with RHAPSODY as the phase III clinical trial study. And the patients that we think there's a really clear call to action for physicians to, you know, offer a real targeted therapy to help patients that are at that stage. And then the patient group that generally have the longest duration of disease as well. So that's our key target both. But we're also very aware and pleased that we have a very broad label with ARCALYST. It's agnostic to the number of flares a patient has had. It's just for recurrent pericarditis, the treatment of recurrent pericarditis and the prevention of future flares.
So physicians are certainly, you know, taking it upon themselves to prescribe within the first recurrence as well. And we're pleased about that. So it's good that they utilize the breadth of the label. A lot of the patients that are in the first recurrence do have generally shorter duration of disease as a median of around six months with those patients that have one recurrence. But that's the group per se. Within that group, there are patients that have higher risk factors that I think physicians are starting to tease out to the patients that may have the longer durations of therapy and need a more targeted approach. So whether that's patients that have significant effusion or cardiac tamponade or constriction, those patients are being treated with ARCALYST now as well. It's around 15% of the total patients that are on ARCALYST are on their first recurrence.
So we're pleased about that, that people are using the breadth of the label. We continue to be focused on two or more recurrences and make good progress so far.
Should we expect you to pre-announce at our friends' conference in January or?
I think, you know, we're still making decisions on that around exactly what we will do at the turn of the year.
Okay, great. Now turning to abiprubart. Amgen and Novartis have progressed into late-stage trials for Sjögren’s disease, and you're kind of coming as a, in a way, a fast follower to that. Can you talk about kind of the market opportunity in the context of, you know, being a fast follower into this market?
Yeah, sure. So we're very excited about KPL- 404 and in particular Sjögren’s, right? This is a very large market, no currently approved therapies. And I think the, I mean, you mentioned those two other companies there in your lead in. Those are large companies that don't chase small markets. And so when we look at the overall opportunity, we think it's very large with a very unserved patient population. And we think KPL-404 is really well positioned, you know, particularly within the CD40 class, CD154 class, but also amongst the other potential mechanisms.
Can you talk about some of the differentiating features if maybe go into a little more detail?
Yeah, sure. So I think in terms of differentiation, there are really two key points. One is the mechanism itself, right? This is a central control node of innate and adaptive immunity. It sits right at the central node where those two systems talk to each other. And so it has a broader potential set of actions across two different types of immunity than other mechanisms that may only target one. You know, whether that ultimately manifests into clinical efficacy, I think we see a little bit of that with the data that exists today in Sjögren’s. The agents that have previously gone before us and blocked CD40 or CD154 show numerically higher ESSDAI reductions versus placebo than some other mechanisms like FcRn or the BAFF depletion. And so that's one.
And I think the second point that we really shouldn't overlook is our program has a high concentration of liquid formulation, which enables subcutaneous development. And many of these other mechanisms that are maybe further ahead in development are high dose IV only, which is incredibly inconvenient for the patient. So I think the combination of those two things and ultimately, you know, the data will play out the way they play out. But as of right now, we think CD40 is really in a great spot compared to the existing data in the field.
Tell us about your ongoing study?
Yeah, so we're studying a phase II-B study in Sjögren’s disease, 24-week endpoint overall. The endpoint is ESSDAI, which is comprised of patient population with severe, moderate to severe systemic involvement, as well as the sicca symptoms that patients normally complain about. And that study is enrolling with two dose groups, one biweekly subcutaneous and the other monthly. So we think, you know, coming out of that study, we'll be in a great position to be competitive against the field.
Do you feel like you need to see efficacy on par with Amgen and Novartis, or is something less than that acceptable? I mean, you are a sub-Q administered drug that might afford you some liberties. I'm just curious how you're thinking.
Yeah, no, that's a great question. And I think, you know, we'll have to see the totality of the data, and it really comes down to overall risk-benefit. And it's also very difficult to compare across trials, right? Cross-trial comparisons are fraught with challenges. So I think it'll be the totality of the data to whether we get to a go, no go on that. But I think basically the way it's shaping up, you know, we really like our trial design. We really like our sub-Q approach with biweekly and weekly dosing, and we'll go from there.
Do you see read-through from your initial RA trial as sort of a read-through on potency and how this molecule acts into Sjögren’s? Is there kind of a connection there? Because the Amgen data, I thought on whole in RA looked a little, you know, stronger. But I don't know if that means what the implications are for Sjögren’s. I mean, completely different disease. So curious.
Yeah, no, great question. I think the way we view the data are, they give us a lot of confidence in our study, especially dosing subcutaneously versus their high dose IV. When you look at the sort of the numerical data, again, cross-trial comparisons, but when you look at our RA data versus the dazodalibep data, they are similar to, I think we are slightly numerically better. And I think the key part of that and where we get excited about the translation is that using their high IV monthly doses, they reduced rheumatoid factor, a clear pharmacodynamic marker of effect. And so did we to the same degree and to the same level dosing subcutaneously. And so translating those RA results to Sjögren’s disease, we are certainly, we believe inhibiting the mechanism to its full degree with our biweekly and monthly dose versus their IV approach.
How is enrollment going and when can we expect data?
That is a great question. We haven't disclosed that. We generally like to enroll the study before we start providing guidance. So more to come. ClinicalTrials.gov is a great reference for that. But suffice it to say, the trial is active and enrolling patients.
Okay. And do you feel like you're kind of full now with, you know, your commercial product and, you know, I think a mid to late stage asset coming along nicely, or do you feel like you need, you know, or are interested in other assets that would complement your portfolio?
Look, we are always interested in additional value creation. I think we've got a scalable organization that can, for the right asset or for the right indication, or we can certainly execute against that.
Okay, great. So maybe as we wrap up, tell us a little bit more about your cash runway, sort of how you're thinking about the path to profitability and what we can look forward to in 2025 from Kiniksa.
Yeah, so in terms of our cash position at the end of Q3, we had $223.8 million cash on hand. And we've said that, you know, we plan to be cash flow positive on an annual basis. So we're pleased with our positioning, I think from a financial point of view, as well as the execution on the commercial side and the portfolio that we have, particularly with abiprubart in Sjögren’s disease. And as Eben said, also always looking at business development and value creation for the future.
Okay, great. Well, thanks for joining us today. Really appreciate it.
Thank you, Lisa.