管理層發言
Good day, and thank you for standing by. Welcome to the Kiniksa Pharmaceuticals First Quarter 2026 Earnings Conference Call. Please be advised today's conference is being recorded. I would now like to hand the conference over to your speaker today, Jonathan Kirshenbaum, Investor Relations. Please go ahead.
Thank you, operator. Good morning, everyone, and thank you for joining Kiniksa's call to discuss our first quarter 2026 financial results and recent portfolio execution. A press release highlighting these results can be found on our website under the Investors section. After the agenda, our Chief Executive Officer, Sanj K. Patel, will start with an introduction. From there, Ross Moat, our Chief Operating Officer, will provide an update on ARCALYST commercial execution. Then Kiniksa's Chief Medical Officer, Dr. John Paolini, will review our KPL-387 development program and the ongoing Phase II/III clinical trial in recurrent pericarditis. After that, Mark Ragosa, our Chief Financial Officer, will review our first quarter 2026 financial results. And finally, Sanj will share closing remarks and kick off the Q&A session, for which Eben Tessari, our Chief Strategy Officer, will also be on the line.
Before getting started, please note that we will be making forward-looking statements today that are subject to risks and uncertainties that may cause actual results to differ materially from such statements. A review of these statements and risk factors can be found on this slide as well as under the caption Risk Factors contained in our SEC filings. These statements speak only as of the date of this presentation, and we undertake no obligation to update such statements, except as required by law. With that, I'll turn it over to Sanj.
Thanks, Jonathan, and good day, everyone. Kiniksa continues to build strength across the business, which is driven by both our commercial progress with ARCALYST and the advancement of our pipeline programs, including KPL-387 and KPL-1161. On the commercial side, the end of the first quarter marks the fifth anniversary of the FDA approval for ARCALYST in recurrent pericarditis. Through our consistent and effective execution over those past five years, we've established and developed the market for this debilitating disease. This has enabled a fundamental shift in the treatment paradigm for patients and led to significant growth for the ARCALYST franchise. Within our clinical portfolio, we continue to advance the KPL-387 Phase II/Phase III study in recurrent pericarditis. Data of the Phase II dose-focusing portion of the study are on track for the second half of this year. We also expect to start the Phase III portion of the program by the end of this year.
In addition, we are advancing KPL-1161 closer to the clinic. This is our Fc-modified IL-1 alpha and beta inhibitor with a target profile of quarterly dosing. And as we've previously shared, we plan to start a Phase I study by the end of this year. Our robust financial position, together with profitable ARCALYST revenue growth gives us the ability to invest in value creation across the business. Commercially, ARCALYST continues to be on a robust trajectory five years from launch, and we intend to capture the additional opportunity that remains across the recurrent pericarditis market. Adoption of long-term IL-1 alpha and beta inhibition with ARCALYST is expanding in the approximately 40,000 patients each year in the United States who experience recurrent pericarditis flares. In the first quarter of this year, this expanding adoption contributed to ARCALYST sales growing to $214.3 million.
Looking to the rest of the year, the marked increase in both the breadth and depth of prescribing we observed in the first quarter provides momentum going forward. As a result, we've now raised our full year 2026 revenue guidance to $930 million to $945 million from our previous guidance of $900 million to $920 million. In summary, Kiniksa is a well-capitalized, growth-oriented company that is well positioned to maximize the substantial ARCALYST commercial opportunity that is available to us. The company's portfolio of programs have numerous milestones throughout the rest of the year and that also have the potential to create meaningful value.
Thank you, Sanj. Our continued commercial execution has driven strong revenue growth in Q1, leading to an ARCALYST net revenue of $214.3 million, which represents an increase of more than $76 million compared to the first quarter 2025 and approximately $12 million over Q4 of last year. This revenue growth was driven by strong underlying commercial metrics, which outpaced the Q1 industry-wide headwinds related to co-pay resets and changes in insurance plans. In particular, growth was achieved by two key commercial dynamics. Firstly, we saw an acceleration in the growth of new prescribers through the quarter, which resulted in the highest quarterly increase in new patient enrollments since launch. This bodes particularly well for the rest of the year as the new larger prescriber base, along with the durability of average duration of therapy has enabled us to increase our full year revenue guidance from between $900 million to $920 million to between $930 million and $945 million.
Secondly, in Q1, our gross-to-net increased compared to the prior quarter as expected. However, it was lower than Q1 of 2025. This was mainly driven by changes to our co-pay support program where we made enhancements to our assistance program design, which reduced the average co-pay payout per patient relative to prior Q1s. With the momentum created early in the year, combined with our strong underlying commercial foundation, we believe we are well positioned to continue driving ARCALYST growth through the rest of the year and believe there is substantial opportunity ahead to support many more recurrent pericarditis patients. In Q1, thanks to the strong execution from our team, approximately 400 new prescribers wrote ARCALYST for the first time, representing the highest quarter-on-quarter increase launched to date. This brings the total number of prescribers to more than 4,550. As a reminder, with more than 25,000 health care professionals seeing recurrent pericarditis patients in a given year, there is substantial opportunity ahead.
We also saw growth in the number of health care professionals who became repeat prescribers during Q1, resulting in approximately 1,320 prescribers in total who have now prescribed ARCALYST multiple times. The acceleration we've seen in both the breadth and the depth of prescribing reflects our continued commercial execution as well as the growing understanding and adoption of interleukin-1 alpha and beta inhibition as the treatment choice following the prior use of NSAIDs and colchicine, as recommended in the 2025 ACC Concise Clinical Guidance. Earlier this month, we announced the initiation of our highly targeted direct-to-consumer campaign, Heart's Home. This campaign is designed to identify and target patients who may be suffering with recurrent pericarditis and not currently taking ARCALYST with the aim of empowering them to discuss ARCALYST with their healthcare provider. Through digital innovation, including the use of AI, we are able to deploy DTC in a way that's cost effective, highly targeted and ultimately applicable for a rare disease market.
We have focused on utilizing our existing patient database and added search optimization and machine learning models informed by de-identified claims, demographics, and consumer market data to define an enriched population of potential recurrent pericarditis patients to deliver tailored content, opposed to a traditional DTC approach of broad-scale and high-cost marketing. The centerpiece of our campaign is a connected TV commercial that is directed to potential patients through their individual streaming accounts on platforms such as YouTube and Hulu, as well as across social media channels. This campaign is informed by our market research, which demonstrated that when a recurrent pericarditis patient inquires about ARCALYST to their provider, the healthcare professional is receptive to the inquiry, and it results in ARCALYST being prescribed in around 80% of cases. As previously mentioned, our ARCALYST franchise is growing, is profitable and has significant opportunity ahead, and this has allowed us to make disciplined investment decisions to expand our reach to capture the opportunity and help more patients.
We are focused on many different initiatives to accelerate growth, including continued execution across our commercial organization, broader digital marketing under which the DTC campaign sits, and peer-to-peer education to expand awareness among clinicians. With that, I'll turn the call over to John to cover our KPL-387 development program.
Thank you, Ross. As a brief refresher, we leveraged our extensive clinical experience with the IL-1 signaling pathway when designing the integrated development program for KPL-387 shown here, broken down by phase of development. The core component of the program is the Phase III placebo-controlled event-driven randomized withdrawal study. Just as in RHAPSODY, the pivotal study, which supported ARCALYST approval in recurrent pericarditis, the primary efficacy endpoint for Phase III will measure the reduction in risk of pericarditis recurrence as the primary demonstration of KPL-387 efficacy for the label. We believe from our regulatory interactions that this study will be sufficient to support registration as a single pivotal study. As a reminder, to maximize operational efficiency, we combined the Phase II dose-focusing trial and the Phase III pivotal trial into a single integrated Phase II/III protocol so that Phase III could initiate independently of Phase II execution, and we added long-term extensions to all trial activities.
We previously guided that we expect data from the dose-focusing study outlined here in red in the second half of this year. And today, we guided that we expect to initiate the Phase III portion of the study by the end of this year. The Phase II dose-focusing study builds on insights from previous clinical trials with rilonacept, and it is designed to define the KPL-387 PK/PD relationship as well as to support the data-driven approach for affirming the dose level for the Phase III pivotal trial. Looking at the rilonacept Phase II precedent in the left panels, the single active arm study demonstrated that IL-1 pathway inhibition with once-weekly rilonacept resulted in rapid and sustained reductions in reported pain and inflammation in patients with active recurrent pericarditis and elevated C-reactive protein over the initial six-week treatment period as well as the subsequent long-term extension through 24 weeks.
Now looking forward, the KPL-387 Phase II dose-focusing study, which is assessing four dose levels in up to 20 patients per arm mirrors the rilonacept Phase II study in terms of study population, number of patients per arm and the primary endpoint, which is time to treatment response. The study framework has been adjusted for the specific attributes of the long-acting pharmacokinetics of KPL-387. Thus, development stage appropriate data, which are expected in the second half of this year, are designed to provide useful information on the cadence and magnitude of initial response as well as the duration of action of KPL-387 dose levels, affirming the dose level for Phase III and informing Phase III outcomes measures. I will now turn it over to our Chief Financial Officer. Mark?
Thanks, John. This morning, I will cover our first quarter 2026 financial performance. As always, you can find our detailed financial information in today's press release. In the first quarter of 2026, we continue to build strong momentum across the business, advancing ARCALYST, progressing our clinical portfolio and maintaining a strong financial position. Starting on the left-hand side of this slide with our income statement. As you've heard from Sanj and Ross, ARCALYST revenue grew 56% year-over-year to $214.3 million in the first quarter. This growth was driven by strong expansion in new prescribers and new patient enrollments, which more than offset the impact of industry-wide seasonal headwinds. Operating expense growth year-over-year was driven by several factors. Higher cost of goods sold due to ARCALYST revenue growth, increased collaboration expenses aligned with higher ARCALYST revenue and collaboration profit, higher R&D primarily due to increased clinical and manufacturing costs associated with the development of KPL-387 and additional SG&A, primarily driven by investment associated with the commercialization of ARCALYST, including personnel and leveraging new technologies to enhance our targeting strategy and reach additional patients and HCPs.
As a result of the strong revenue growth against more moderate expense growth, net income increased significantly to $22.6 million in the first quarter of 2026 compared to $8.5 million in the first quarter of 2025. Turning to the right-hand side of the slide, you'll find the calculation for ARCALYST collaboration profit, which drives total collaboration expenses. In the first quarter of 2026, ARCALYST collaboration profit continued to grow faster than sales on a year-over-year basis, up 73% to $151.2 million. Finally, at the bottom of the slide, we ended the first quarter with a $468.1 million cash balance, representing $54 million of net cash generation for the period. We expect to remain cash flow positive on an annual basis under our current operating plan, enabling us to continue to help patients while creating additional value in both the near and long term.
Thanks, Mark. As you've heard, Kiniksa is well positioned to build significant future value as we grow our IL-1 alpha and beta inhibition franchise. We are dedicated to helping as many patients as possible with ARCALYST and to advancing the development of our clinical portfolio in order to bring additional therapies to patients suffering from debilitating diseases. With that, I'll now turn the call back to the operator for questions.
分析師問答
Our first question comes from Nick Lorusso with TD Cowen.
Congrats on the strong quarter. Can you discuss what you have seen in terms of increased demand from the early days of the DTC campaign, acknowledging that it is still pretty early on? And what other plans do you have to accelerate demand in the future, either via patients or prescriber targeting?
Yes, Nick, this is Ross. Thank you very much. As you said, it's early days for the DTC campaign. We just announced it recently and are focusing on DTC in a very targeted way to reach patients we believe have recurrent pericarditis and to inform and educate them on how to discuss ARCALYST with their healthcare providers. One thing I can share is that when patients proactively speak to their healthcare professionals about ARCALYST, it gets prescribed in around 80% of cases, yet only about 14% of recurrent pericarditis patients are unaided aware of ARCALYST. So we know there's a big awareness gap. Patients are widely dispersed across the country, so our approach is to identify those patients and serve very targeted, tailored messages to empower them to speak to their providers. We're excited about the campaign, but it's early. We're focused on multiple initiatives to accelerate growth. As a reminder, we were around 18% penetrated into the 14,000 patient population, not accounting for patients that are even in their first recurrence.
We've also seen strong growth in first-recurrence patients over time, so the opportunity is significant. We're executing across our commercial organization, focusing on digital marketing — of which the DTC campaign is a part — and peer-to-peer education to grow how recurrent pericarditis is treated. We're excited about the future and have a multifaceted approach to grow the breadth and depth of prescribing and help more patients.
Our next question comes from Anupam Rama with JPMorgan.
And congrats on a strong quarter here. For KPL-387 and the second half dose focus update, John, I was wondering if you could comment a little bit as when you look at the totality of the range of endpoints and assessments that you're going to be looking at in Phase II, how do you think about which ones are most important in sort of the ultimate dose selection moving to Phase III?
Anupam, thank you for that question. With regard to the Phase II trial, the value is showing the kind of data we generated previously in the Phase II program with rilonacept in terms of three critical elements: cadence and magnitude, which is the time of onset of action and degree of suppression of pain and inflammation measured with C-reactive protein; and the durability of response. Carrying that forward to the KPL-387 program, our models indicate that regardless of the dose level selected, we expect to see high drug levels modeled to have a rapid cadence in onset of action and magnitude of effect in suppressing the initial inflammatory response. The key scientific question across dose levels is the duration of action of the different doses. We integrate those three elements to build the PK/PD relationship and affirm what we believe to be the therapeutic concentration and the dose level to carry forward into Phase III. So it's really the integration of those three critical elements.
Our next question comes from David Nierengarten with Wedbush Securities.
Just a couple of quick ones for me. First off, on the DTC ad, is it fair to model in the incremental spend year-over-year on marketing as the DTC component? Or is there some additional sales guys or other folks that you hired or other expenses going in there? And then the second one on 387. On the transition study, the treatment duration is 16 weeks, which is different than the 12 or 24 weeks that you've looked at in Phase I or Phase II. Is there any reason you picked 16 weeks versus having a little bit more apples-to-apples duration comparison, at least for patients who are moving from ARCALYST to 387?
Maybe David, on the first one, I think as you heard on the call, SG&A did go up as a result of personnel-related expenses as well as sales and marketing initiatives, which Ross has covered. We continue to invest responsibly in the commercialization of ARCALYST as shown by collaboration profit continuing to grow faster than revenue. We haven't provided specific guidance on spend, but it's worth noting that on a percentage of sales basis, SG&A has been fairly consistent over the last year.
Regarding the transition to KPL-387 monotherapy dosing and administration study, the 16-week treatment duration for the posology portion is appropriate for this type of study. It's designed to look at well-controlled patients as they move from their prior therapies to KPL-387. Patients are transitioning from regimens of NSAIDs and colchicine, from corticosteroids, and from IL-1 pathway inhibitors, including anakinra and rilonacept. Previously with rilonacept, time to monotherapy was under eight weeks in the RHAPSODY program. This program is designed to move patients off those other therapies onto KPL-387 and achieve monotherapy within that window. Additional doses are administered to achieve steady state by the week 16 time point, and importantly, patients transition to a long-term extension where they can continue to receive KPL-387 for up to two total years. So it's a well-designed study to inform clinical practice for transitioning patients to KPL-387.
Maybe a quick follow-up. Do you look at patients by prior treatment to determine if anyone has a new attack of pericarditis and stratify accordingly? How are you thinking about differences in prior treatments for the transition?
That's a reasonable approach. If you look at the ARCALYST label, it covers the various therapies that patients can transition from: NSAIDs and colchicine, corticosteroids, and others. We reported in prior trials how patients responded across different treatments and the time to monotherapy. Similarly, in this trial, we'll look at the different types of prior regimens patients came from and ensure the transition to KPL-387 is robust. It's important to point out that the anticipated Phase III dose level of KPL-387 supports once-monthly dosing, which covers most of that initial transition period depending on the prior therapy.
Our next question comes from Edward Nash with Canaccord Genuity.
Really great quarter. Congratulations. I wanted to ask: with regards to what's driving the biggest change in new patient starts, you said the awareness gap has shrunk and you've seen increasing physician adoption. What effect has reimbursement or referral patterns had on these new patient starts?
Thanks, Ed. There are many things driving increases we've seen, not just in Q1 but over time. Q1 was the highest quarter-on-quarter growth we've had in new prescribers and the highest number of new patient enrollments since launch. Reimbursement continues to be very strong across payer mixes for new patients as well as revalidation of scripts after the typical one-year period. Regarding referrals, there are about 18 centers that act as centers of excellence or pericardial disease-specific clinics, supported in part by initiatives to grow expertise and share best practices. But recurrent pericarditis patients remain broadly dispersed, so we need many touchpoints to educate physicians. We use peer-to-peer education, our sales team, and digital marketing initiatives like the DTC campaign to increase awareness. With only around 14% of patients having unaided awareness of ARCALYST, empowering patients with knowledge can play a substantial role in helping awareness and enabling patients to ask their physicians about ARCALYST, which can be one of several drivers supporting growth.
Our next question comes from Paul Choi with Goldman Sachs.
Congratulations on the strong quarterly results. First, could you elaborate on the co-payment commentary for the quarter? Is this specific to this quarter, or could it be more structurally favorable to gross-to-net over the long term? Second, on KPL-387: with regard to the transition switch study, can you comment if you have a fairly confident view on the dose going forward for Phase III, or are you still testing multiple doses there?
I'll take the gross-to-net question. We haven't provided specific guidance on gross-to-net. We do not expect major fluctuations relative to 2025, but we do anticipate that co-pay support will be favorable to gross-to-net on an annual basis, with the majority of the impact having taken place in the first quarter. Historically, gross-to-net has been highest in Q1, lower in Q2 and Q3, and then shifts higher again in Q4 due to industry dynamics. That's the pattern we expect to generally return to, absent any prior period reserve adjustments.
I'll add some color on the co-pay dynamics. We made enhancements to our co-pay assistance program at the beginning of the year which reduced the average co-pay payment per patient, contributing to a more favorable gross-to-net versus Q1 of last year, although higher than Q4 of last year. We reduced the maximum amount of co-pay payments we make per patient and implemented a machine learning solution to proactively identify patients on non-traditional payment plans, such as maximizer plans, so we can better manage payments in cases where the manufacturer would otherwise be paying until funds are exhausted before insurers pick up coverage. Lowering the co-pay amount for those plans reduced the maximum amount we paid per patient before full coverage under insurers kicked in, thereby improving gross-to-net.
Regarding the transition to monotherapy study, details of the study architecture are on clinicaltrials.gov, but specific dose level information is not disclosed there. We will have more to say at a later date about the design and dose levels being studied.
Our next question comes from Geoff Meacham with Citi.
Just had two quick ones. Ross, on the commercial side, is there a tipping point for adding more patients to the first recurrence segment? Would you want to wait longer on awareness and DTC visibility, or do you have to navigate reimbursement hurdles in that more upstream segment? And for Sanj: with positive cash flow and consistent profitability, how do you think about maximizing value from here? Would you want to be in Phase III for 387 before you take another look at business development, or how are you thinking about it?
Thanks, Geoff. As mentioned earlier, we were around 18% penetrated into the 2-plus recurrence group and about 20% of prescriptions are now in the first recurrence group, which has grown over time. The 2025 ACC Concise Clinical Guidance is helpful, as it places the use of IL-1 inhibition prior to corticosteroids, moving ARCALYST further upstream. ARCALYST has a broad, label-agnostic indication relative to the number of flares a patient has had, and we have broad patient coverage across the labeled indication for the majority of plans. So we feel well positioned and believe there's a significant opportunity ahead in both the first recurrence and 2-plus recurrence segments.
Geoff, thanks for the question. Our team focuses on creating value and executing. There's a lot of focus right now on completing the Phase II study later this year and initiating the Phase III study this year for KPL-387, which is exciting. We're also focused on capturing further growth with ARCALYST and advancing KPL-1161 into the clinic this year. We balance internal development with business development opportunities and maintain a high bar for any deal. Capital allocation and efficiency are priorities for us; we've been pragmatic, leveraging digital and AI to be efficient in our commercial investments. We'll continue to evaluate options to create value, whether through internal development or business development, and we will be disciplined in our approach.
Our next question comes from Roger Song with Jefferies.
This is Fiona on for Roger. Congrats on the strong quarter. Quick one on KPL-387: any meaningful difference in formulation versus ARCALYST? Do you plan to use an autoinjector? And down the line, if 387 gets approved, how do you plan your commercial strategy around incorporating and potentially transitioning patients to 387?
There is a difference: KPL-387 is a liquid formulation that allows the total dose to be delivered in a single syringe subcutaneously. With the extended pharmacokinetics shown in Phase I, that profile supports once-monthly dosing. Once you have that profile, it becomes favorable for the development of an autoinjector. We have not discussed autoinjector details in depth at this time.
Nothing to add to John's remarks other than we'll continue to execute on the ongoing clinical trials — Phase II dose-focusing and initiating Phase III this year — as quickly and as well as possible.
Our next question comes from Eva Fortea with Wells Fargo.
Congrats on the quarter. Two quick ones: first, how should we think about R&D expense for the rest of the year and into 2027 as 387 Phase III and 1161 Phase I are initiated? Second, you've guided to initiating the Phase III pivotal portion for 387 by year-end 2026. Are there any key steps or milestones you need to clear to initiate the study, or is it just a matter of seeing the Phase II data before moving forward?
Similar to earlier questions, we haven't provided explicit R&D guidance. On a percentage of sales basis, R&D has been fairly consistent over the last year. The timing of clinical trials and manufacturing of clinical supply are the key variables. We disclosed ongoing investments to advance KPL-387 into Phase III and KPL-1161 into Phase I in 2026. Longer trials tend to be more expensive, but keep in mind sales growth and our fairly consistent R&D-to-sales ratio when modeling.
Eva, regarding the Phase II/III transition: Phase II data are expected in the second half of 2026 and we are on track to receive dose level confirmation data in that timeframe. Because the Phase II dose-focusing portion and the Phase III pivotal portion are integrated into a single protocol, the Phase III pivotal trial can begin independently of Phase II execution.
I am not showing any further questions at this time. I'll turn the call back over to Sanj for any further remarks.
Thanks, operator. Thank you for all the questions and for joining the call today. We look forward to the remainder of the year and providing additional updates in the future. Thank you.
Thank you, ladies and gentlemen. This concludes today's presentation. We thank you for your participation. You may now disconnect, and have a wonderful day.