Kiniksa Pharmaceuticals International PLC (KNSA) 2025 Q2 法說會逐字稿

Thank you for standing by and welcome to the Kiniksa Pharmaceutical second-quarter 2025 earnings conference call. (Operator Instructions) As a reminder, today's program is being recorded.

感謝您的耐心等待，歡迎參加 Kiniksa Pharmaceutical 2025 年第二季財報電話會議。（操作說明）提醒各位，今天的節目正在錄製中。

And now I'd like to introduce your host for today's program, Jonathan Kirshenbaum, Investor Relations. Please go ahead, sir.

現在，我謹向大家介紹今天節目的主持人，投資者關係部的喬納森·柯申鮑姆。請繼續，先生。

Thank you, operator. Good morning, everyone, and thank you for joining Kiniksa's call to discuss our second-quarter 2025 financial results and recent portfolio execution. A press release highlighting these results can be found on our website under the Investors section.

謝謝接線生。各位早安，感謝各位參加 Kiniksa 的電話會議，討論我們 2025 年第二季度的財務業績和最近的投資組合執行情況。有關這些結果的新聞稿可在我們網站的「投資者」欄位下找到。

As for the agenda for today's call, our Chief Executive Officer, Sanj K. Patel, will start with an introduction and overview of our business. Ross Moat, Kiniksa's Chief Commercial Officer, will provide an update on ARCALYST commercial execution, then Mark Ragosa, our Chief Financial Officer, will review our second-quarter 2025 financial results. Finally, Sanj will share closing remarks and kick off the Q&A session, for which John F. Paolini, our Chief Medical Officer; and Eben Tessari, our Chief Operating Officer, will also be on the line.

至於今天電話會議的議程，我們的執行長桑吉·K·帕特爾將首先介紹和概述我們的業務。Kiniksa 商務長 Ross Moat 將介紹 ARCALYST 的商業執行情況，然後我們的財務長 Mark Ragosa 將回顧我們 2025 年第二季的財務表現。最後，Sanj 將發表閉幕致詞並開始問答環節，我們的首席醫療官 John F. Paolini 和首席營運官 Eben Tessari 也將在線參與問答。

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 these statements. A review of such 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 the date of this presentation, and we undertake no obligation to update such statements, except as required by law.

在開始之前，請注意，我們今天將做出一些前瞻性陳述，這些陳述存在風險和不確定性，可能導致實際結果與這些陳述有重大差異。您可以在本投影片以及我們提交給美國證券交易委員會的文件的「風險因素」標題下找到這些聲明和風險因素的審查。這些聲明僅代表本次發布之日的情況，除法律要求外，我們不承擔更新這些聲明的義務。

With that, I will turn it over to Sanj.

這樣，我就把麥克風交給桑吉了。

Thanks, Jonathan, and good morning, everyone. I'm happy to review Kiniksa's second quarter financial results and the highlights across our portfolio. Kiniksa continues to build upon the strength across our business, which is driven by both our commercial execution with ARCALYST and our pipeline development programs, including KPL-387. ARCALYST continues to generate strong revenue growth. Our continued execution across key commercial drivers and increased penetration across the pericarditis population led to a net revenue of $156.8 million in the second quarter.

謝謝你，喬納森，大家早安。我很高興能回顧 Kiniksa 第二季度的財務表現以及我們投資組合中的亮點。Kiniksa 繼續鞏固我們業務的實力，這得益於我們在 ARCALYST 的商業執行以及我們的管線開發計劃，包括 KPL-387。ARCALYST持續維持強勁的營收成長。我們持續推進關鍵商業驅動因素，並持續提高在心包膜炎人群中的滲透率，使得第二季淨收入達到 1.568 億美元。

This represents a growth of $19 million over the first quarter. In just over four years since the launch of ARCALYST, as the first and only FDA-approved therapy for recurrent pericarditis, Kiniksa has generated over $1 billion in cumulative net sales. Surpassing this milestone is a result of our effective commercial strategy and our team who work relentlessly to bring this highly efficacious therapy to thousands of patients suffering from this debilitating disease. Kiniksa is well positioned to continue maximizing the potential of ARCALYST. For full year 2025, we're raising our ARCALYST net sales guidance to between $625 million and $640 million from $590 million to $605 million.

這比第一季成長了1900萬美元。自 ARCALYST 上市以來，短短四年多時間，作為首個也是唯一一個獲得 FDA 批准的複發性心包膜炎療法，Kiniksa 的累計淨銷售額已超過 10 億美元。取得這一里程碑式的成就，得益於我們有效的商業策略以及我們團隊的不懈努力，他們致力於將這種高效療法帶給成千上萬遭受這種衰弱性疾病折磨的患者。Kiniksa 已做好充分準備，繼續最大限度地發揮 ARCALYST 的潛力。我們將 2025 年全年 ARCALYST 淨銷售額預期從 5.9 億美元至 6.05 億美元上調至 6.25 億美元至 6.4 億美元。

Importantly, growing ARCALYST revenue continues to support our robust balance sheet, providing capacity for continued investment in value-creating opportunities across the business without the need to access the capital markets.

重要的是，ARCALYST 不斷增長的收入持續支撐著我們穩健的資產負債表，使我們有能力繼續投資於業務中創造價值的機會，而無需進入資本市場。

Turning to our pipeline. We've now initiated and have begun recruiting in the Phase 2/Phase 3 clinical trial of KPL-387 in current pericarditis. This next slide highlights the design of the Phase 2, Phase 3 clinical trial. In designing this study, we've leveraged our experience with that RHAPSODY, which was the successful Phase 3 pivotal trial, supporting FDA approval of ARCALYST in recurrent pericarditis.

接下來我們來看看我們的管道。我們現在已經啟動了 KPL-387 治療目前心包膜炎的 2 期/3 期臨床試驗，並開始招募受試者。下一張投影片重點介紹了第二期、第三期臨床試驗的設計。在設計這項研究時，我們借鑒了 RHAPSODY 的經驗，RHAPSODY 是一項成功的 3 期關鍵性試驗，支持 FDA 批准 ARCALYST 用於治療復發性心包膜炎。

This study consists of three overlapping parts, which have been combined into a single protocol. The Phase 2 dose focusing portion, the Phase 3 double-blind placebo-controlled pivotal portion and the long-term extensions. We're now recruiting patients in the dose focusing portion of the study and expect data in the second half of next year. From there, we'll continue to move as fast as possible and our goal is to deliver this treatment option to patients in the '28, '29 time frame. Thanks to the excellent work of our teams, Kiniksa is the leader in the recurrent pericarditis.

本研究由三個相互重疊的部分組成，並合併為單一的方案。第二階段劑量聚焦部分、第三階段雙盲安慰劑對照關鍵部分和長期擴展部分。我們現在正在招募患者參與研究的劑量聚焦部分，預計明年下半年獲得數據。接下來，我們將繼續以最快的速度推進，我們的目標是在 2028 年、2029 年為患者提供這種治療方案。由於我們團隊的出色工作，Kiniksa 在復發性心包膜炎領域處於領先地位。

Importantly, we are committed to driving additional innovation for these patients and maintaining our leadership position. Our physician and patient market research shows an IL-1 alpha and beta inhibitor with the target profile of KPL-387 could be a meaningful treatment option for patients with recurrent pericarditis.

重要的是，我們致力於為這些患者推動更多創新，並保持我們的領先地位。我們的醫生和患者市場調查顯示，具有 KPL-387 標靶特性的 IL-1 α 和 β 抑制劑可能是複發性心包膜炎患者的一種有意義的治療選擇。

Specifically, the potential for a once-monthly dosing of a liquid formulation in an auto-injector could drive further adoption as well as potentially enhance both duration and compliance. We continue to make solid progress in the second quarter, both commercially and clinically, and we continue to crack on across the portfolio.

具體來說，每月一次使用自動注射器注射液體製劑的潛力可能會推動其進一步普及，並有可能提高治療持續時間和依從性。第二季度，我們在商業和臨床方面都繼續取得穩步進展，並且我們繼續在整個產品組合中取得進展。

With that, I'll turn it over to Ross.

接下來，我將把麥克風交給羅斯。

Thank you, Sanj. Strong execution in Q2 led to significant revenue growth to $156.8 million, representing a 52% year-over-year increase compared to Q2 of last year. This performance was driven by expansion in both the breadth and depth of the prescriber base, which led to the highest number of quarterly new patient enrollments since launch and resulted in a substantial increase to our active commercial patients. Additionally, we've seen good persistence from the Medicare Part D patients who transitioned to commercial therapy at the start of the year due to the affordability changes associated with the Inflation Reduction Act. We are seeing this patient cohort follow similar metrics to other groups of patients on ARCALYST.

謝謝你，桑吉。第二季強勁的執行力帶動了營收的顯著成長，達到 1.568 億美元，比去年同期成長了 52%。這一業績的取得得益於處方醫生群體的廣度和深度的擴大，這使得自產品上市以來，季度新患者註冊人數達到最高水平，並導致我們的活躍商業患者數量大幅增加。此外，我們看到，由於通貨膨脹削減法案帶來的經濟負擔能力變化，年初轉而接受商業治療的 Medicare Part D 患者也保持了良好的堅持性。我們發現，該患者群體遵循與 ARCALYST 上其他患者群體相似的指標。

And while the onetime bolus of patients observed in Q1 will not repeat, we have seen an increase in new Medicare Part D patients initiating commercial therapy versus the previous years. As a result of the increase in active patients, our penetration into the multiple recurrence population increased from approximately 13% at the end of last year to approximately 15% at the end of Q2. Ultimately, this growth reflects that patients and healthcare professionals continue to report high degrees of satisfaction with ARCALYST, and we've built a robust foundation of commercial fundamentals.

雖然第一季觀察到的一次性患者激增的情況不會重現，但與往年相比，我們看到開始接受商業治療的新的 Medicare Part D 患者人數有所增加。由於活躍患者數量的增加，我們在多次復發人群中的滲透率從去年年底的約 13% 增加到第二季末的約 15%。最終，這一增長反映出患者和醫療保健專業人員對 ARCALYST 的滿意度持續很高，並且我們已經建立了強大的商業基礎。

For example, in Q2, our payer approval rate remained greater than 90%. Total duration of therapy was approximately 30 months on average, patient compliance with therapy remains strong at over 85%, and we continue to see ARCALYST used earlier in the course of the disease. Importantly, our strong Q2 performance highlights the progress we've made but more importantly, we continue to be even more excited about the significant opportunity ahead with ARCALYST.

例如，在第二季度，我們的付款方批准率保持在 90% 以上。治療總持續時間平均約為 30 個月，患者對治療的依從性仍然很高，超過 85%，我們繼續看到 ARCALYST 在疾病早期階段被使用。重要的是，我們強勁的第二季業績凸顯了我們所取得的進步，但更重要的是，我們對 ARCALYST 未來帶來的巨大機會感到更加興奮。

On this slide, I'm going to highlight how ARCALYST has continued to shift the treatment paradigm to become the standard of care for recurrent pericarditis. Our promotional efforts have been focused on educating patients and healthcare professionals to recognize recurrent pericarditis as an interleukin-1 alpha and beta mediated disease best managed with targeted immunomodulation.

在這張投影片中，我將重點介紹 ARCALYST 如何持續改變治療模式，成為復發性心包膜炎的標準治療方法。我們的宣傳工作一直致力於教育患者和醫療保健專業人員，讓他們認識到復發性心包膜炎是一種由白細胞介素-1α和β介導的疾病，最好透過標靶免疫調節進行治療。

Since launch, we've seen continuous robust increases in both the new and repeat prescribers every single quarter. This growth not only speaks to the effectiveness of our educational efforts but it also illustrates how receptive physicians have been to this evolved paradigm that utilizes a targeted highly efficacious and well-tolerated treatment.

自推出以來，我們看到每季新處方醫生和重複處方醫生的數量都持續強勁增長。這一增長不僅說明了我們教育工作的有效性，也說明了醫生們對這種採用有針對性、高效且耐受性良好的治療方法的改進範式的接受程度。

In Q2, more than 325 additional health care professionals wrote their first ARCALYST prescription representing one of the highest quarter-on-quarter increases to date and bringing the total number of prescribers to more than 3,475. Additionally, repeat prescribing also continued to increase with more than 120 ARCALYST prescribers writing for their second patients. Finally, we've also seen an increase in prescribing earlier in the disease. Of all the patients on ARCALYST, around 20% were prescribed ARCALYST while on their first recurrence and roughly 80% when they had two or more recurrences. This highlights the growing physician appreciation for the value ARCALYST provides in preventing their patients from suffering future flares.

第二季度，超過 325 名醫療保健專業人員首次開立了 ARCALYST 處方，這是迄今為止季度環比增幅最大的一次，使開處方的醫生總數超過 3475 人。此外，重複處方也持續增加，超過 120 位 ARCALYST 處方醫生為他們的第二個病人開立處方。最後，我們也發現，在疾病早期階段就開處方的情況增加。在所有接受 ARCALYST 治療的患者中，約 20% 的患者在第一次復發時接受了 ARCALYST 治療，約 80% 的患者在兩次或兩次以上復發時接受了 ARCALYST 治療。這凸顯了醫生們越來越認識到 ARCALYST 在預防患者未來病情復發的價值。

In addition to more patients receiving ARCALYST at every stage of the disease, there has been a marked increase in the number of dedicated pericardial disease centers where patients are able to access expert care for healthcare providers, well versed in their disease. We have sponsored the AHAs addressing recurrent pericarditis initiative as part of our ongoing efforts to shorten the treatment journey for patients by providing expert care close to home. There are also several more dedicated pericardial clinics outside of this initiative and our aim is to continue supporting this growth to help patients gain an earlier diagnosis and appropriate treatment of their disease.

除了在疾病的各個階段接受 ARCALYST 治療的患者人數顯著增加外，專門治療心包膜疾病的中心數量也顯著增加，患者可以在這些中心獲得精通該疾病的醫療保健提供者的專家護理。我們贊助了美國心臟協會 (AHA) 的複發性心包膜炎防治計劃，這是我們持續努力的一部分，旨在透過在患者家中附近提供專家護理來縮短他們的治療過程。除了這項計劃之外，還有幾家專門的心包診所，我們的目標是繼續支持這種發展，以幫助患者更早診斷和適當治療他們的疾病。

As the treatment approach continues to change across the country, there's a growing body of published literature recommended IL-1 pathway inhibitors, such as ARCALYST to be used ahead of corticosteroids, which is well aligned with our commercial positioning of ARCALYST. Furthermore, looking at data from RESONANCE, our real-world evidence disease registry, which is driven by expert pericardial centers across the country, ARCALYST has increasingly become the second-line treatment choice after NSAIDs and colchicine.

隨著全國各地治療方法的不斷變化，越來越多的文獻建議在皮質類固醇之前使用 IL-1 通路抑制劑（如 ARCALYST），這與我們對 ARCALYST 的商業定位非常吻合。此外，根據 RESONANCE（我們由全國各地的專家心包中心推動的真實世界證據疾病登記處）的數據，ARCALYST 已逐漸成為 NSAIDs 和秋水仙鹼之後的二線治療選擇。

In Q2, we delivered $156.8 million in net revenue as well as increase the franchise profitability. As a result, we are pleased to increase our 2025 net revenue guidance by $35 million between the midpoint of the prior range and of the new range. This takes us far expecting between $590 million to $605 million to now expecting between $625 million and $640 million. This guidance indicates year-on-year net revenue growth of $215 million at the midpoint compared to full year 2024. This would be the highest annual increase in net revenue to date.

第二季度，我們實現了 1.568 億美元的淨收入，並提高了特許經營的獲利能力。因此，我們很高興地將 2025 年淨收入預期提高 3500 萬美元，新預期範圍的中點與先前的預期範圍的中點相差無幾。這使我們的預期從 5.9 億美元到 6.05 億美元大幅提高到 6.25 億美元到 6.4 億美元。該預測表明，與 2024 年全年相比，淨收入年增 2.15 億美元（按年中位數計算）。這將是迄今為止淨收入年度增幅最高的一次。

As you can hear, we are excited about the future of ARCALYST as well as the progress of our pipeline. We are determined to bring future launches of novel therapies to patients who are suffering from debilitating diseases.

正如你所聽到的，我們對 ARCALYST 的未來以及我們研發管線的進展感到非常興奮。我們決心將未來推出的新療法帶給那些飽受疾病折磨的患者。

And with that, I'll turn the call over to Mark to discuss our financial results. Mark?

接下來，我將把電話交給馬克，讓他來討論我們的財務表現。標記？

Thanks, Ross. This morning, I will cover our second quarter 2025 financial performance. You can find our detailed financial information in today's press release. There are a few items I'd like to call your attention to.

謝謝你，羅斯。今天上午，我將介紹我們2025年第二季的財務表現。您可以在今天的新聞稿中找到我們詳細的財務資訊。有幾件事我想提請您注意。

First, starting with our income statement on the left-hand side of the slide. ARCALYST revenue grew 52% year-over-year in the second quarter to $156.8 million, driven primarily by strong growth in new patient enrollments, prescribers and active commercial patients.

首先，請看投影片左側的損益表。ARCALYST 第二季度營收年增 52% 至 1.568 億美元，主要得益於新患者註冊、處方醫生和活躍商業患者的強勁增長。

Operating expenses grew 26% year-over-year in the second quarter driven primarily by cost of goods sold and collaboration expenses from continued ARCALYST revenue growth and SG&A in support of ARCALYST commercialization. Lastly, due to strong revenue growth, coupled with more moderate expense growth, net income was $17.8 million in the second quarter compared to a net loss of $3.9 million a year ago.

第二季營運費用年增 26%，主要原因是 ARCALYST 收入持續成長帶來的銷售成本和合作費用，以及支援 ARCALYST 商業化的銷售、一般及行政費用。最後，由於營收強勁成長，加上支出成長較為溫和，第二季淨利為 1,780 萬美元，而去年同期淨虧損為 390 萬美元。

Second, the right-hand side of the slide provides the calculation of ARCALYST collaboration profit, which grew 75% year-over-year in the second quarter to $104.8 million, driven by sales volume and disciplined commercial investments.

其次，投影片的右側提供了 ARCALYST 合作利潤的計算結果，第二季年增 75% 至 1.048 億美元，這得益於銷售量和有紀律的商業投資。

Third, at the bottom of this slide, our cash balance increased by approximately $40 million to $307.8 million in the second quarter and we continue to expect our current operating plan to remain cash flow positive on an annual basis.

第三，在本頁底部，我們的現金餘額在第二季度增加了約 4000 萬美元，達到 3.078 億美元，我們繼續預計我們目前的營運計劃將保持年度正現金流。

As you've heard from Sanj and Ross, both commercial and clinical execution in the second quarter added to Kiniksa significant momentum across its business. Combined with financial discipline and a strong balance sheet, Kiniksa remains well positioned to continue to help patients as well as to create additional value in both the near and long term.

正如你從 Sanj 和 Ross 那裡聽到的，第二季度的商業和臨床執行情況都為 Kiniksa 的業務帶來了顯著的成長勢頭。憑藉財務紀律和強勁的資產負債表，Kiniksa 仍然處於有利地位，能夠繼續幫助患者，並在近期和長期內創造更多價值。

And with that, I'll turn the call back to Sanj for closing remarks.

接下來，我將把電話轉回桑吉，請他作總結發言。

Thanks, Mark. As you've heard, Kiniksa continues to execute both clinically and commercially and is well positioned to build significant future value. We are dedicated to helping as many patients as possible with ARCALYST and to advancing the development of our clinical portfolio, which includes KPL-387, the liquid formulation IL-1 receptor antagonist, which has a target profile of monthly dosing. Our ultimate goal is to bring additional treatment options and therapies to patients suffering from debilitating diseases with unmet need.

謝謝你，馬克。正如你所聽到的，Kiniksa 在臨床和商業方面都持續取得進展，並已做好充分準備，在未來創造巨大的價值。我們致力於幫助盡可能多的患者使用 ARCALYST，並推進我們的臨床產品組合的開發，其中包括 KPL-387，這是一種液體製劑 IL-1 受體拮抗劑，其目標是每月給藥一次。我們的最終目標是為患有嚴重疾病且治療需求尚未滿足的患者帶來更多的治療選擇和療法。

I'll now turn the call back to the operator for questions. Thank you.

現在我將把電話轉回給接線員，以便回答問題。謝謝。

Anupam Rama, JPMorgan.

Anupam Rama，摩根大通。

Congrats on the quarter. I know you highlighted 15% penetration into the multiple recurrence setting at the end of 2Q. Wondering if you could provide some commentary on kind of the trends that you're seeing in the first recurrent setting. I think the slide said about 20% of total prescriptions are coming from this setting?

恭喜你本季取得佳績。我知道你在第二季末強調了多重復發場景下 15% 的滲透率。想請您就您在第一個循環設定中觀察到的趨勢提供一些評論。我記得幻燈片上說，大約 20% 的處方來自這種場所？

Yes. Thanks very much, Anupam. This is Ross. Thank you for the question. So you're right that in the 2-plus recurrence group, as a reminder, that's the 14,000 patient population in any given year.

是的。非常感謝，阿努帕姆。這是羅斯。謝謝你的提問。所以你說得對，在復發次數超過 2 次的群體中，提醒一下，每年有 14,000 名患者。

We've seen continuous increase since the launch into the penetration into that group. Most recently, going to 15% versus last reported at the end of 2024 of 13%. So seeing some nice increase within that group, and that remains our key target base as the patient groups who are suffering the most had the highest burden of the disease, also most closely aligned with the data in RHAPSODY as well. But we've also seen, as the treatment paradigm has changed over time, significant growth in early on in the disease. So those patients are still very much within label with the broad label that we have, just for recovering pericarditis agnostic to the number of flares.

自產品上市以來，我們看到該群體滲透率持續成長。最新數據顯示，到 2024 年底，這一比例將達到 15%，而先前報告的為 13%。因此，我們看到該群體中出現了一些不錯的成長，而這仍然是我們的主要目標群體，因為遭受痛苦最嚴重的患者群體承受著最沉重的疾病負擔，這也與 RHAPSODY 的數據最為吻合。但隨著治療模式的不斷變化，我們也看到，在疾病早期階段，治療效果顯著提升。因此，這些患者仍然完全符合我們廣泛的標籤定義，即心包膜炎的恢復，而與發作次數無關。

And there's an additional group of 26,000 patients in that first recurrence group, which is a significant opportunity for us. And I think what we're seeing now with around 20% of all the ARCALYST patients that were prescribed to the drug when they were on their first recurrence is greater confidence, familiarity, knowledge of how to prescribe, how to look after patients while they're on ARCALYST, and just greater comfort for healthcare professionals having greater experience with the drug and having seen the impact that it has on patients in the real-world setting to utilize this drug early on in the disease.

此外，在第一個復發組中還有 26,000 名患者，這對我們來說是一個重要的機會。我認為，現在我們看到，大約 20% 的 ARCALYST 患者在第一次復發時就被處方了這種藥物，他們對如何開處方、如何在患者服用 ARCALYST 期間照顧他們有了更大的信心、更熟悉、更了解，醫療保健專業人員也因為擁有更多使用該藥物的經驗，並看到了它在真實世界中對患者的影響，而感到更加安心，從而能夠使用這種藥物。

I think as well as an increase in understanding the recurrent pericarditis is a disease, which is mediated by interleukin-1 alpha and beta, and in order to control the disease and prevent future flares, patients are having to needlessly suffer those future flares, the utilization of an inhibitor of interleukin 1 alpha, beta that's very well tolerated with high efficacy, is being very well received by both patients and health care professionals. So we are pleased to see an increase across really the whole population, which is a total of 40,000 patients in totality when you include the first recurrence on top of the 2 plus recurrence groups.

我認為，隨著人們對復發性心包膜炎的認識不斷加深，認識到這種疾病是由白細胞介素-1α和β介導的，為了控制病情並防止未來復發，患者不得不承受不必要的痛苦，因此，使用耐受性良好且療效高的白細胞介素-1α、β抑製劑受到了患者和醫療保健專業人員的廣泛歡迎。因此，我們很高興看到整個人群的病例數都有所增加，如果將首次復發病例加上兩次或兩次以上復發病例，患者總數將達到 40,000 人。

Geoff Meacham, Citi.

傑夫‧米查姆，花旗銀行。

I just want to, I guess, follow up on Anupam's question. I guess when you look at the patients who've dropped off, maybe over, say, the past year or so, was the dosing frequency a big driver? I guess I'm trying to get a sense for what the new start outlook could be for 387. And I'm under the assumption that you'll have a pretty good switch rate as well looking from ARCALYST?

我想就Anupam的問題做個後續回答。我想，如果你觀察一下那些在過去一年左右停止治療的患者，給藥頻率是否是主要原因？我想了解一下 387 號航班重新開始後的前景如何。我假設從 ARCALYST 的情況來看，你們的切換率也會相當不錯？

Thanks, Jeff. So we're seeing the patients continuing to stay on therapy for quite some time. We've seen an average of 30 months in total. Patients are also pretty compliant to therapy overall at 85% or more, and patients reacting very well while on ARCALYST. So we're pleased about that, and we believe that there's a significant opportunity ahead for ARCALYST as we continue to switch on more and more physicians.

謝謝你，傑夫。所以我們看到患者需要持續接受治療相當長一段時間。我們看到的平均總時長為 30 個月。患者對治療的整體依從性也相當高，達到 85% 或更高，且患者在使用 ARCALYST 期間反應良好。因此我們對此感到高興，我們相信，隨著越來越多的醫生加入，ARCALYST 將迎來巨大的發展機會。

And when you take the penetration numbers of 15%, I think that shows that we've had good growth up until this moment in time but the opportunity ahead is significant, and that's without taking into account the first recurrence patient. So we're very focused on continuing the growth of ARCALYST. Maybe, I'll pause here. Sanj, do you want to comment on 387?

當你看到 15% 的滲透率時，我認為這表明我們迄今為止取得了良好的成長，但未來的機會是巨大的，而且這還沒有考慮到第一個復發患者。因此，我們非常專注於繼續推動 ARCALYST 的發展。或許，我該在這裡稍作停留。Sanj，你想對第387條發表評論嗎？

Yes. No, thanks, Jeff. I mean, obviously, we're very excited about 387, and we are definitely tracking on as far as the Phase 2, Phase 3 study is concerned. Obviously, that will be data dependent. But clearly, we've shown we know to commercialize in this space.

是的。不用了，謝謝你，傑夫。我的意思是，很顯然，我們對 387 感到非常興奮，而且我們肯定會在 2 期和 3 期研究方面取得進展。顯然，這取決於數據。但很顯然，我們已經證明我們有能力在這個領域實現商業化。

We've developed an awful lot of great contacts and relationships with physicians. So that will be key. But ultimately, depending on data but we certainly know how to do it. And we've -- as I said, we've leveraged a lot of the learnings we had from RHAPSODY and from ARCALYST earlier on. So we'll continue to keep working on.

我們與醫生們建立了許多非常好的聯繫和關係。所以這一點至關重要。但最終還是要看數據，不過我們當然知道該怎麼做。正如我之前所說，我們充分利用了從 RHAPSODY 和 ARCALYST 中學到的許多經驗。所以我們會繼續努力。

Paul Choi, Goldman Sachs.

Paul Choi，高盛。

Congrats on the quarter. My first question is, just given the pace of growth here, how do you think about potentially further expansion of the sales force and/or some sort of larger form of marketing/DTC to continue to expand awareness and drive penetration?

恭喜你本季取得佳績。我的第一個問題是，鑑於目前的發展速度，您認為是否有可能進一步擴大銷售隊伍和/或採取某種更大規模的營銷/DTC形式來繼續擴大知名度並提高市場滲透率？

And my second question is with 387, how are you thinking about potential in office utilization in the future as part of the paradigm? Do you vision this potentially being more administered in office? And just how you're thinking about self-administration versus physician administration down the road here?

我的第二個問題是關於 387，作為該模式的一部分，您如何看待未來辦公室利用率的潛力？您認為這項工作有可能更多地由辦公室負責管理嗎？那麼，您未來是如何考慮患者自行用藥和醫師用藥這兩種方式的呢？

Thanks, Paul. This is Sanj. Maybe I'll make a few comments and pass it over to Ross if he has anything to add. But yes, very excited about the growth that we've had in ARCALYST without a doubt. And as far as the sales force is concerned, obviously, as we've always said, we do an awful lot of analytical work on what the rightsizing is, looking at the territories.

謝謝你，保羅。這是桑吉。我可能會發表一些意見，然後把話題轉給羅斯，看看他有沒有什麼補充。是的，毫無疑問，我們對 ARCALYST 取得的成長感到非常興奮。至於銷售團隊，顯然，正如我們一直所說，我們做了大量的分析工作，研究如何合理調整人員規模，並考察各個區域的情況。

And we've done that, as you know, since launch just over four years ago. And as you've heard in the past, we have increased that. So at the moment, really, we've not made any comments as to exactly what we've done on the size of it. Last reported was around 85. We're certainly continuing to look at what's needed as far as growth is concerned but I think they're being utilized incredibly well.

如你所知，自從四年多前公司成立以來，我們一直都在這樣做。正如你之前聽到的，我們已經增加了這個數字。所以目前，我們還沒有就具體規模問題發表任何評論。上次報告的年齡約為 85 歲。我們當然會繼續關注成長方面的需求，但我認為他們目前都得到了非常有效的利用。

And as far as DTC and other things, clearly, our marketing groups had a great impact in the launch so far. We're certainly don't rests on our laurels. We're certainly looking at in addition to what we've done as far as sales force is concerned in our existing materials and disease education, physician education, we continue to look at other ways.

至於 DTC 和其他方面，很顯然，到目前為止，我們的行銷團隊在產品發布中發揮了巨大的作用。我們當然不會固步自封。除了我們在銷售團隊方面所做的工作，以及我們現有的材料、疾病教育和醫生教育之外，我們當然也在考慮其他方法。

I'm sure Ross can go into more detail, but we've certainly got a massive focus on digital marketing and looking at other ways we can really apply sort of not just the metrics but also some of the new technologies that are out there to identify patients that are very much in need. So it's a very exciting area for us.

我相信羅斯可以更詳細地介紹一下，但我們確實非常重視數位行銷，並正在尋找其他方法，不僅可以運用各種指標，還可以運用一些新技術來識別真正需要幫助的患者。所以這對我們來說是一個非常令人興奮的領域。

As you can see, it's still growing. There's an awful lot more we can do. We certainly are tapping into that in the future. But maybe Ross, you can comment on how we're looking at marketing and expanding our efforts there.

正如你所看到的，它還在生長。我們還有很多事情可以做。我們未來肯定會利用這一點。不過羅斯，或許你可以談談我們如何看待行銷以及如何擴大這方面的投入。

Yes, absolutely. Thanks, Sanj. Thank you, Paul. Yes, I think the key thing here on what Sanj is we are an organization that never rests on our laurels, we're quite happy with how the launch has gone to date but we have so much more to do as an organization. And we're very innovative in our approach, constantly evolving and refreshing what we do and finding better ways of doing things.

是的，絕對的。謝謝你，桑吉。謝謝你，保羅。是的，我認為 Sanj 的關鍵在於我們是一個從不滿足於現狀的組織，我們對迄今為止的發布情況相當滿意，但作為一個組織，我們還有很多工作要做。我們的方法非常具有創新性，不斷發展和改進我們的工作方式，並尋找更好的做事方法。

And as we get more and more into this market and increase our understanding of the market, we find that we can get more effective over time as we just have way to understanding. The utilization of newer technologies is also important to us. We have looked utilizing AI in our tucked-in targeting strategy and in a variety of different digital marketing environments. And that's proven very successful for us, and we haven't shared great detail on that but we're utilizing a lot of new technologies now, which is really paying dividends and helping us to get out there to physicians and to patients and making a difference in this marketplace. So we constantly evolve and look at new ways of doing things to get better and better over time.

隨著我們越來越深入了解這個市場，隨著時間的推移，我們會發現，隨著我們對市場的理解不斷加深，我們的效率也會越來越高。對我們來說，運用新技術也很重要。我們已經研究瞭如何在我們的定向行銷策略和各種不同的數位行銷環境中利用人工智慧。事實證明，這對我們來說非常成功，雖然我們沒有透露太多細節，但我們現在正在利用許多新技術，這確實帶來了回報，幫助我們接觸到醫生和患者，並在這個市場中發揮作用。因此，我們會不斷發展，尋找新的做事方法，以便隨著時間的推移變得越來越好。

To the second part of your question, Paul, regarding in-office versus outpatient use, the vast majority of ARCALYST is in outpatients under pharmacy benefits. With KPL-387, obviously, as Sanj has said, everything is data dependent, and we'll see as we progress further but with a target profile of monthly and in a liquid formulation with the potential to go to an auto injector. That also plays nicely into the -- where the patients are, which is ultimately not wanting to be in hospital suffering from this disease but being treated appropriately and kept at home and preventing flares and preventing them going into a hospital for the future. So whether that precipitates a change in kind of in office versus outpatients and patients administered in their own home is to be seen but we don't see a substantial call for in hospital utilization.

Paul，關於你問題的第二部分，即在診間使用與門診使用，ARCALYST 的絕大部分是在門診病患中透過藥局福利使用的。顯然，正如Sanj所說，KPL-387 的一切都取決於數據，隨著我們進一步的進展，我們將看到結果，但目標是每月一次，採用液體配方，並有可能用於自動注射器。這也很好地契合了——患者的現狀，他們最終都不想因為這種疾病而住院，而是希望得到適當的治療，待在家裡，防止病情復發，避免將來再次住院。因此，這是否會促使門診治療與居家治療方式改變還有待觀察，但我們目前並未看到住院需求大幅增加。

Eva Fortea, Wells Fargo.

伊娃‧福特亞，富國銀行。

Two quick ones from us. So on ARCALYST from the 30-month average therapy duration, can you give us a sense in terms of numbers on how -- are you seeing a shorter treatment duration the patients in first recurrence versus patients in second and beyond? And the second question is on 387, can you discuss how you're thinking about like the balance between remaining cash flow positive on an annual basis and initiating studies in new indications beyond the recurrent pericarditis?

我們先簡單介紹兩點。那麼，根據 ARCALYST 30 個月的平均治療持續時間，您能否用數字告訴我們，您是否觀察到首次復發患者的治療持續時間比第二次及以後復發患者的治療持續時間更短？第二個問題是關於 387，您能否討論一下您是如何考慮在保持年度正現金流和啟動除復發性心包炎之外的新適應症研究之間取得平衡的？

Thanks, Eva. Thank you very much for the question. So I'll certainly take the first part and then hand over to someone else to cover the second part. Yes, we're not really seeing any meaningful differences in terms of duration from the different cohorts, including patients on the first recurrence versus second, third, fourth, fifth recurrence groups. But of course, the data is always evolving and building and as more patients starting to be initiated on ARCALYST earlier on in the disease.

謝謝你，伊娃。非常感謝您的提問。所以我一定會負責第一部分，然後把第二部分交給其他人負責。是的，我們並沒有看到不同隊列在持續時間上有任何實質的差異，包括首次復發患者與第二次、第三次、第四次、第五次復發患者。當然，數據總是不斷發展和積累，而且越來越多的患者在疾病早期就開始接受 ARCALYST 治療。

We just don't have some of that data yet but it will build, and we'll report as we see it later down the line. But from what we see so far, no significant differences. The average is 30 months. The median of the initial duration of therapy is around 17 months and the restart rate remains around 45%.

我們目前還沒有一些數據，但數據會逐漸積累，我們會在稍後看到數據後進行報告。但就目前來看，並沒有明顯的差異。平均為30個月。初始治療持續時間的中位數約為 17 個月，重新開始治療的比例約為 45%。

I think importantly, of all those patients that started as ARCALYST in our launch quarter back in Q2 of 2021 now, around 10% of all of those patients that started way back then are still on therapy, meaning their initial treatment of therapy and have just stayed on throughout, which I think is testament to the effect and how well tolerated ARCALYST could be for many of these patients. So no significant differences today. It's obviously something that we will keep an eye on and report as we see.

我認為很重要的一點是，在 2021 年第二季度我們推出 ARCALYST 時開始接受治療的所有患者中，大約有 10% 的患者仍在接受治療，這意味著他們完成了最初的治療療程並一直堅持了下來，我認為這證明了 ARCALYST 的療效以及它對許多患者的良好耐受性。所以今天沒有明顯變化。這顯然是我們將會密切關注並隨時報告的事情。

And Eva, as far as your second question regarding cash flow and further investment, I think at this point in our life cycle, we are focused on continuing to create value. And importantly, as we've talked about in the past, we do think that through commercial execution and continued financial discipline, we do have the capacity to continue to create value across our business, whether it's to further maximize the opportunity with ARCALYST to further advance our pipeline and/or to pursue strategic initiatives.

伊娃，關於你第二個問題，即現金流和進一步投資，我認為在我們目前的生命週期階段，我們專注於繼續創造價值。更重要的是，正如我們過去所討論過的，我們認為透過商業執行和持續的財務紀律，我們有能力繼續在整個業務中創造價值，無論是進一步最大限度地利用 ARCALYST 的機會來進一步推進我們的產品線和/或推行戰略舉措。

(Operator Instructions) Roger Song, Jefferies.

（操作說明）羅傑‧宋，傑富瑞。

Congrats for the quarter. Maybe two quick ones for the pipeline, the first one, KPL-387. So understanding you're doing the Phase 2 portion to decide the dosing. Just curious about what is the target efficacy safety profile particularly in the context of comparison to ARCALYST, you can make a decision to decide those and how likely you will move multiple dose into the Phase 3 portion, something like induction maintenance? And then a quick one for the 1161 IND enabling right now? And then what's the current thinking about the potential indication for this quarterly IL-1?

恭喜你本季取得佳績。或許可以快速地為管道做兩個測試，第一個是 KPL-387。所以，我理解你正在進行第二階段試驗，以確定劑量。我只是好奇目標療效安全性概況是什麼，尤其是在與 ARCALYST 進行比較的情況下，您可以據此做出決定，以及您有多大可能將多劑量方案推進到 3 期部分，例如誘導維持治療？那麼，現在能否快速解答一下關於 1161 IND 啟用的問題呢？那麼，目前對於季度 IL-1 檢測的潛在適應症有何看法？

Yes. Thanks for that question. We were actually both of those questions. So regarding the profile of KPL-387 what we're looking for in the dose focusing portion of the trial is to select the dose that we'll use in the pivotal portion of the trial. And so in that sense, the ability to treat the acute flare as it happens and then prevent the subsequent flare is really the profile that we established with ARCALYST and that we're looking for similar profile, if you will, with regard to the KPL-387 efficacy.

是的。謝謝你的提問。我們其實都回答了這兩個問題。因此，關於 KPL-387 的特性，我們在試驗的劑量聚焦部分所尋找的，是選擇我們將在試驗的關鍵部分使用的劑量。因此，從這個意義上講，能夠及時治療急性發作並防止後續發作，正是我們在 ARCALYST 中確立的特性，也是我們希望 KPL-387 療效能夠達到的類似特性。

So once we've selected that dose, we then carry that forward into the randomized withdrawal portion, pivotal section of the trial, which bears a remarkable similarity in terms of its design, in terms of the end points that we have structured. And so at that point, it will be data-driven in terms of the -- what the actual profile of KPL-387 is but we're very confident in the study design and is being able to show the strength of KPL-387 and its target profile of one monthly dose.

因此，一旦我們選定了劑量，我們就將其帶入隨機撤藥部分，這是試驗的關鍵部分，其設計以及我們設定的終點都與先前部分有顯著的相似之處。因此，到那時，我們將以數據為依據來確定 KPL-387 的實際特性，但我們對研究設計非常有信心，並且能夠展示 KPL-387 的效力及其每月一次給藥的目標特性。

Regarding the second question of 1161, at this point, we've not announced any specific indication. We realize that there's broad potential of having an IL-1 alpha and IL-beta inhibitor pathway inhibition, if you will, that has a target profile of dosing every three months. So that really opens the possibility of a range of chronic lifelong diseases that are auto-inflammatory.

關於 1161 號提案的第二個問題，目前我們還沒有公佈任何具體指示。我們意識到，IL-1α 和 IL-β 抑制劑路徑抑制具有廣泛的潛力，其目標給藥方案為每三個月給藥一次。因此，這確實為一系列自體發炎性慢性終身疾病的可能性打開了大門。

So we'll continue to do that work. But in the meantime, our focus is on the IND-enabling studies so that we can begin the first in-human study as soon as possible. Thank you so much.

所以我們會繼續做這項工作。但同時，我們的重點是 IND 申報研究，以便我們能夠盡快開始第一次人體試驗。太感謝了。

David Nierengarten, Wedbush Securities.

大衛‧尼倫加滕 (David Nierengarten)，韋德布希證券公司 (Wedbush Securities)。

I had two and maybe one is kind of a follow-up to the last one. But is there any specific kind of efficacy boundaries that you're looking for out of the 387 study? So of course, ARCALYST showed a near 100% drop-off in recurrences. I mean is that the kind of efficacy bar we should be thinking about? Or could it be a little bit lower because of the more convenient dosing?

我寫了兩篇，其中一篇可能是上一篇的後續。但是，您希望從 387 項研究中得出哪些特定的療效界線？因此，ARCALYST 的復發率當然下降了近 100%。我的意思是，這是我們應該考慮的那種功效標準嗎？或者，由於給藥方式更便捷，劑量可能會略低一些？

And then I had another question on just emerging competition. There's a potential oral competitor out there that's going to report out data this half. Is there any thinking on that or any thoughts on how we should think about the potential competition emerging?

然後我還有一個關於新興競爭的問題。有一家潛在的口服競爭對手將在本半年公佈數據。對此大家有什麼想法嗎？或者說，對於我們該如何看待可能出現的競爭，大家有什麼建議嗎？

Sure. Thanks, David. Appreciate the questions. So regarding the efficacy profile in the Phase 2 studies, the dose focusing portion, we're really looking across a range of different dose levels in order to understand the performance characteristics of KPL-387. And so in that sense, we intend to use the totality of the data in order to define the dose level that we'll take forward. So at this point, it's a little early to describe exactly what the expectation is precisely, but rather to say that I think this will be a very informative study based upon its design that will help us optimize the performance of the drug.

當然。謝謝你，大衛。感謝提問。因此，關於 2 期研究中的療效概況，劑量聚焦部分，我們正在研究一系列不同的劑量水平，以了解 KPL-387 的性能特徵。因此，從這個意義上講，我們打算利用所有數據來確定我們將要採取的劑量水平。所以現在要準確描述預期結果還為時過早，但我認為這項研究的設計將非常有啟發性，有助於我們優化藥物的性能。

Regarding competition that's on the horizon, we remain the leaders in the space of recurrent pericarditis for the reason that we have really done the deep work in understanding the mechanism of this disease. And we've identified the fact that IL-1 alpha and IL-1 beta inhibition is a critical element for maintaining control of the disease, an important effect to be able to maintain control of the disease as monotherapy.

至於即將到來的競爭，我們仍然是復發性心包膜炎的領導者，因為我們確實對這種疾病的機制進行了深入的研究。我們已經確定，IL-1 α 和 IL-1 β 抑制是維持疾病控制的關鍵因素，這是作為單一療法維持疾病控制的重要效果。

And so we continue to look with interest to see other data as they emerge but understanding those. So we say those mechanisms will have to define themselves in the context of the fact that, as I mentioned, we understand that complete control of the disease requires control of both cytokines.

因此，我們將繼續饒有興趣地關注其他數據的出現，並努力理解這些數據。因此，我們認為這些機制必須在這樣的背景下定義：正如我所提到的，我們明白，要完全控制這種疾病，就需要控制這兩種細胞激素。

Thank you. And this does conclude the question-and-answer session of today's program. I'd like to hand the program back to Sanj Patel for any further remarks.

謝謝。今天的問答環節到此結束。我想把發言權交還給桑吉·帕特爾，請他再補充一些內容。

Thank you, operator, and thanks, everybody, for the questions and joining the call today. We look forward to the remainder of the year and to providing additional opportunities and updates in the future. So very excited, and thank you very much.

謝謝接線員，也謝謝各位今天參加電話會議並提出問題。我們期待今年剩下的日子，並希望在未來提供更多機會和最新資訊。太激動了，非常感謝！

Thank you, ladies and gentlemen, for your participation in today's conference. This does conclude the program. You may now disconnect. Good day.

感謝各位女士、先生參加今天的會議。節目到此結束。您現在可以斷開連線了。再會。