Travere Therapeutics Inc (TVTX) 2025 Q3 法說會逐字稿

Good afternoon, and welcome to the Travere Therapeutics' Third Quarter 2025 Financial Results Conference Call. Today's call is being recorded.

下午好，歡迎參加 Travere Therapeutics 2025 年第三季財務業績電話會議。今天的通話將會被錄音。

At this time, I would like to turn the conference call over to Nivi Nehra, Vice President, Corporate Communications and Investor Relations. Please go ahead, Nivi.

此時，我謹將電話會議交給企業傳播與投資者關係副總裁 Nivi Nehra。請繼續，妮維。

Thank you, operator. Good afternoon, and welcome to Travere Therapeutics' Third Quarter 2025 Financial Results and Corporate Update Call. Thank you, all, for joining.

謝謝接線生。下午好，歡迎參加 Travere Therapeutics 2025 年第三季財務業績及公司最新進度電話會議。謝謝各位的參與。

Today's call will be led by Dr. Eric Dube, our President and Chief Executive Officer. Eric joined in the prepared remarks by Dr. Jula Inrig, our Chief Medical Officer; Peter Heerma, our Chief Commercial Officer; and Chris Cline, our Chief Financial Officer. Dr. Bill Rote, our Chief Research Officer, will join us for the Q&A.

今天的電話會議將由我們的總裁兼執行長埃里克·杜貝博士主持。艾瑞克與首席醫療官朱拉·因里格博士、首席商務官彼得·赫爾馬和首席財務官克里斯·克萊恩一起發表了事先準備好的演講。我們的首席研究官比爾·羅特博士將參加問答環節。

Before we begin, I'd like to remind everyone that statements made during this call regarding matters that are not historical facts are forward-looking statements within the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are not guarantees of performance. They involve known and unknown risks, uncertainties and assumptions that may cause actual results, performance and achievements to differ materially from those expressed or implied by the statement. Please see the forward-looking statement disclaimer on the company's press release issued earlier today, as well as the Risk Factors section in our Forms 10-Q and 10-K, filed with the SEC. In addition, any forward-looking statements represent our views only as of the date such statements are made, October 30, 2025, and Travere specifically disclaims any obligation to update such statements to reflect future information, events or circumstances.

在開始之前，我想提醒大家，本次電話會議中提及的非歷史事實的陳述，均屬於 1995 年《私人證券訴訟改革法案》安全港條款所界定的前瞻性陳述。前瞻性陳述並非業績保證。它們涉及已知和未知的風險、不確定性和假設，可能導致實際結果、績效和成就與聲明中明示或暗示的內容有重大差異。請參閱本公司今天稍早發布的新聞稿中的前瞻性聲明免責聲明，以及我們向美國證券交易委員會提交的 10-Q 表格和 10-K 表格中的「風險因素」部分。此外，任何前瞻性聲明僅代表我們截至該等聲明發布之日（2025 年 10 月 30 日）的觀點，Travere 特此聲明不承擔任何更新該等聲明以反映未來資訊、事件或情況的義務。

With that, let me now turn the call over to Eric.

那麼，現在讓我把電話交給艾瑞克。

Thank you, Nivi, and good afternoon, everyone. The third quarter marked exceptional progress across our three key priorities: delivering strong commercial execution in IgA nephropathy, preparing for a potential FDA approval in FSGS, and successfully advancing the manufacturing scale-up of pegtibatinase to support restarting enrollment in the pivotal HARMONY study in 2026.

謝謝你，妮維，大家午安。第三季我們在三個關鍵優先事項上取得了顯著進展：在 IgA 腎病領域實現了強勁的商業執行，為 FSGS 的潛在 FDA 批准做好了準備，並成功推進了 pegtibatinase 的生產規模擴大，以支持在 2026 年重新啟動關鍵的 HARMONY 研究的招募工作。

The core driver of our performance is FILSPARI's continued growth in IgA nephropathy, where we delivered sustained commercial excellence in the third quarter. Physicians continue to confidently adopt FILSPARI as a foundational nephroprotective therapy for their patients. This confidence reflects consistent real-world outcomes, robust long-term data reinforcing FILSPARI's differentiated profile and its recent inclusion in the KDIGO guidelines for earlier first-line use to optimize nephroprotection in IgAN. Additionally, in August, the FDA approved a modification to the FILSPARI REMS program, removing the embryo-fetal toxicity REMS and reducing the liver monitoring frequency to quarterly, which aligns with routine clinical practice and our clinical trial experience. This change not only simplifies care for physicians and patients, but also reinforces FILSPARI's long-term safety profile.

我們業績的核心驅動力是 FILSPARI 在 IgA 腎臟病領域的持續成長，我們在第三季度實現了持續的商業卓越表現。醫生們繼續滿懷信心地將 FILSPARI 作為患者的基礎腎臟保護療法。這種信心反映了真實世界中一致的結果、強有力的長期數據，這些數據強化了 FILSPARI 的差異化特性，並且最近將其納入 KDIGO 指南，用於更早的一線治療，以優化 IgAN 的腎臟保護。此外，8 月，FDA 批准了 FILSPARI REMS 計劃的修改，取消了胚胎-胎兒毒性 REMS，並將肝臟監測頻率降低至每季一次，這與常規臨床實踐和我們的臨床試驗經驗相符。這項變更不僅簡化了醫生和患者的護理，而且還增強了 FILSPARI 的長期安全性。

Our US performance continues to be complemented by strong progress from our partners globally. In Europe and the UK, CSL Vifor is expanding access, following full regulatory approvals and the progress has been culminated in the recent achievement of a meaningful market access milestone.

我們在美國市場的優異表現，也得益於全​​球合作夥伴的強勁進展。在歐洲和英國，CSL Vifor 在獲得全面監管批准後，正在擴大市場准入範圍，並且最近取得了意義重大的市場准入里程碑式的進展。

In Japan, Renalys completed enrollment in its registrational trial in IgAN and remains on track to deliver top line data in quarter four. The company also reached an agreement with the PMDA of Japan to initiate 2 Phase III trials for sparsentan in FSGS and Alport syndrome and recently announced its planned acquisition by Chugai, a leading innovator in renal and rare disease research in Japan. Together, these milestones underscore FILSPARI's expanding global footprint and the growing excitement around its long-term potential to transform care for renal rare kidney diseases.

在日本，Renalys 已完成 IgAN 註冊試驗的患者招募，並預計在第四季度公佈主要數據。該公司還與日本藥品和醫療器材管理局 (PMDA) 達成協議，啟動針對 FSGS 和 Alport 綜合徵的 2 項 III 期臨床試驗，並於近期宣布計劃被日本腎臟和罕見疾病研究領域的領先創新者中外製藥收購。這些里程碑共同凸顯了 FILSPARI 不斷擴大的全球影響力，以及人們對其在改變罕見腎臟疾病治療方面長期潛力的日益增長的興奮之情。

Beyond our progress in IgAN, addressing the urgent need for an approved medication in FSGS is both central to our mission and represents the next pillar of growth for Travere. Today, there are no FDA-approved medicines for this disease. Patients often experience rapid disease progression with many reaching kidney failure within just a few years of diagnosis, often requiring a transplant. Even then, the disease recurs in approximately half of transplant recipients. The consequences are devastating for patients and their families. Earlier and more effective treatment is desperately needed, which is why the opportunity to bring FILSPARI forward in FSGS is so meaningful for this community who have waited far too long.

除了我們在 IgAN 方面取得的進展之外，解決 FSGS 領域急需核准藥物的問題，既是我們使命的核心，也是 Travere 的下一個成長支柱。目前，尚無獲得美國食品藥物管理局（FDA）批准的治療這種疾病的藥物。患者病情往往進展迅速，許多患者在確診後幾年內就會出現腎衰竭，通常需要腎臟移植。即使如此，大約一半的移植受者仍會復發這種疾病。這對患者及其家人來說後果是毀滅性的。迫切需要更早、更有效的治療，因此，對於等待了太久的 FSGS 患者群體來說，將 FILSPARI 應用於 FSGS 治療具有非常重要的意義。

In September, the FDA communicated that an advisory committee is no longer needed for our sNDA in FSGS. We have been pleased with the progress of our review and our ongoing engagement with the agency to date. Pending approval, FILSPARI will become the first and only approved medication for FSGS, representing a landmark moment for this community, and given the urgent need for an effective approved medication, a transformational opportunity for Travere. Our teams are fully prepared to execute a rapid launch upon approval, building upon the commercial foundation we've established in IgA nephropathy.

9 月，FDA 通知我們，對於我們針對 FSGS 的補充新藥申請 (sNDA)，不再需要諮詢委員會。我們對審查工作的進展以及迄今為止與該機構的持續合作感到滿意。待批准後，FILSPARI 將成為第一個也是唯一一個獲批用於治療 FSGS 的藥物，這對該群體來說是一個里程碑式的時刻，鑑於目前迫切需要一種有效的獲批藥物，這對 Travere 來說也是一個變革性的機會。我們的團隊已做好充分準備，一旦獲得批准，即可迅速啟動，並依賴我們在 IgA 腎臟病領域建立的商業基礎。

Beyond FILSPARI, we have successfully manufactured the first commercial scale batches of pegtibatinase and are looking forward to an expected restart of the pivotal HARMONY study of pegtibatinase in classical HCU next year. PEG-t remains a promising potentially disease-modifying investigational therapy that could address a substantial gap for patients living with this rare metabolic disorder.

除了 FILSPARI 之外，我們已經成功生產了第一批商業規模的 pegtibatinase，並期待明年重啟 pegtibatinase 在經典 HCU 中的關鍵性 HARMONY 研究。PEG-t 仍然是一種很有前景的潛在疾病改善研究療法，可以解決患有這種罕見代謝疾病的患者的重大需求缺口。

I'll now turn the call over to Jula for a clinical update. Jula?

現在我將把電話交給朱拉，請她報告臨床狀況。朱拉？

Thank you, Eric. One of the most significant milestones this quarter was the inclusion of dual endothelin angiotensin receptor antagonism in the updated KDIGO guidelines for IgA nephropathy, a strong external validation of FILSPARI's role as foundational treatment. KDIGO includes FILSPARI as a first-line option for patients who are at risk of IgA nephropathy progression, recognizing it as the only therapy with proven efficacy versus optimized RAS inhibition.

謝謝你，埃里克。本季度最重要的里程碑之一是將雙重內皮素血管緊張素受體拮抗劑納入更新後的 KDIGO IgA 腎病指南，這有力地驗證了 FILSPARI 作為基礎治療的作用。KDIGO 將 FILSPARI 列為 IgA 腎病變進展風險患者的第一線治療方案，認為它是唯一經證實療效優於優化 RAS 抑制的療法。

The guidelines also recommend simultaneous treatment of the two drivers of IgA nephropathy progression, targeting both the upstream immune activation that causes pathogenic IgA deposition and the downstream glomerular injury that leads to nephron loss.

該指南還建議同時治療 IgA 腎病進展的兩個驅動因素，即上游導致致病性 IgA 沉積的免疫活化和下游導致腎單位喪失的腎小球損傷。

This holistic framing of disease management aligns with FILSPARI's mechanism of action as the only fully approved non-immunosuppressive nephroprotective treatment, which can be combined with immune-targeted medications to optimize long-term outcomes for patients living with IgA nephropathy.

這種疾病管理的整體框架與 FILSPARI 的作用機制一致，FILSPARI 是唯一獲得完全批准的非免疫抑制性腎臟保護治療藥物，可與免疫標靶藥物聯合使用，以優化 IgA 腎病患者的長期療效。

Across our KOL engagements following the publication of the guidelines, nephrologists have described the new KDIGO framework as a true paradigm shift that validates early and comprehensive intervention. We believe this recognition cements FILSPARI's position as foundational care in IgA nephropathy, guiding a new era of evidence-based treatment sequencing.

在指南發布後，我們與關鍵意見領袖進行了多次交流，腎臟科醫生們都認為新的 KDIGO 框架是一個真正的範式轉變，它驗證了早期和全面乾預的必要性。我們相信，這項認可鞏固了 FILSPARI 在 IgA 腎臟病基礎護理領域的地位，引領了實證治療順序的新時代。

A further testament to our leadership in rare kidney disease is our focus on data generation and dissemination, as exemplified by numerous scientific presentations and engagements at recent congresses, including our 11 upcoming presentations at ASN Kidney Week. A few highlights of this data include the Phase II SPARTAN trial in RAS inhibitor naive patients with IgA nephropathy, demonstrating that irrespective of baseline proteinuria levels. FILSPARI consistently reduced proteinuria and led to significant reductions in urinary biomarkers of disease activity, including reductions in immune system and complement activation markers, indicating potential disease-modifying qualities of FILSPARI.

我們致力於數據生成和傳播，這進一步證明了我們在罕見腎臟病領域的領先地位。我們在最近的大會上進行了大量的科學演講和交流，包括即將在 ASN 腎臟週上進行的 11 場演講。此數據的一些亮點包括針對 RAS 抑制劑初治的 IgA 腎病患者的 II 期 SPARTAN 試驗，結果表明，無論基線蛋白尿水平如何，均有效。FILSPARI 持續降低蛋白尿，並顯著降低尿液中疾病活動的生物標記，包括降低免疫系統和補體活化標記物，顯示 FILSPARI 具有潛在的疾病改善特性。

We also have two new presentations from the Phase III PROTECT trial in IgA nephropathy. One, evaluating efficacy across historical histopathology from kidney biopsies and another assessing outcomes based on time from IgA nephropathy diagnosis. Both presentations reinforce the SPARTAN findings and align with the KDIGO recommendations, showing that earlier treatment of patients with FILSPARI can lead to greater nephroprotection.

我們還有來自 IgA 腎病 III 期 PROTECT 試驗的兩項新報告。一項研究透過腎臟切片的歷史組織病理學進行療效評估，另一項研究則根據 IgA 腎病診斷的時間評估療效。兩項報告均強化了 SPARTAN 研究結果，並與 KDIGO 建議一致，顯示對患者進行 FILSPARI 早期治療可帶來更大的腎臟保護作用。

We also continue to generate and present real-world and long-term data across a broad spectrum of IgA nephropathy disease severity, demonstrating FILSPARI's consistent benefit in reducing proteinuria and preserving kidney function.

我們也持續產生並展示涵蓋各種 IgA 腎病嚴重程度的真實世界和長期數據，證明 FILSPARI 在減少蛋白尿和保護腎功能方面具有持續的益處。

In FSGS, as Eric highlighted in his opening remarks, we are pleased with the progress of our review. The agency remains engaged on our submission. And from our perspective, the process continues to be similar to our experience during the IgAN NDA review.

正如 Eric 在開場白中所強調的那樣，我們對 FSGS 的審查進展感到滿意。該機構仍在處理我們的申請。從我們的角度來看，這個過程與我們在 IgAN NDA 審查期間的經歷類似。

Ahead of a potential approval in January 2026, our Medical Affairs teams are deeply engaged, expanding disease education, strengthening nephrologist awareness around the importance of proteinuria in FSGS disease progression and responding to queries regarding how the DUPLEX data could translate into real-world benefit for this underserved patient population.

在 2026 年 1 月可能獲得批准之前，我們的醫學事務團隊正在積極參與，擴大疾病教育，加強腎臟科醫生對 FSGS 疾病進展中蛋白尿重要性的認識，並回應有關 DUPLEX 數據如何轉化為對這一服務不足的患者群體的實際益處的疑問。

At ASN, we are presenting several new analyses from the DUPLEX study, including a late-breaking analysis that demonstrates that patients treated with FILSPARI achieved proteinuria levels of less than 0.7 grams per gram more frequently versus maximum labeled dose irbesartan. And patients who achieved this threshold had a lower risk of kidney failure, irrespective of treatment arm. This analysis demonstrates further alignment and supports the conclusions of the PARASOL working group that lower levels of proteinuria translate into meaningful improvements in kidney outcomes.

在 ASN 會議上，我們將展示 DUPLEX 研究的幾項新分析，其中包括一項最新分析，該分析表明，與最大標籤劑量厄貝沙坦相比，接受 FILSPARI 治療的患者更頻繁地達到每克尿蛋白水平低於 0.7 克。達到此閾值的患者，無論接受何種治療，發生腎衰竭的風險都較低。該分析進一步證實了 PARASOL 工作小組的結論，即較低的蛋白尿水平可轉化為腎臟預後的顯著改善。

We also have data that extrapolates the antiproteinuric treatment effect of FILSPARI versus irbesartan seen in the two-year DUPLEX trial into longer term kidney failure outcomes from the UK Rare Disease Renal Registry or RaDaR. And we also have subgroup analyses of pediatric patients and patients with collagen four genetic mutations, demonstrating a consistent antiproteinuric treatment effect with FILSPARI versus irbesartan in these two high-risk difficult-to-treat patient populations.

我們也擁有數據，可以將兩年 DUPLEX 試驗中觀察到的 FILSPARI 與厄貝沙坦相比的抗蛋白尿治療效果外推到英國罕見疾病腎臟登記處 (RaDaR) 的長期腎衰竭結果。我們也對兒科患者和膠原蛋白 4 基因突變患者進行了亞組分析，結果表明，在這兩類高風險難治性患者群體中，FILSPARI 與厄貝沙坦相比，具有一致的抗蛋白尿治療效果。

With no approved medicine for patients with FSGS today, the opportunity to bring FILSPARI forward is both urgent and transformative. The supportive data from DUPLEX and our regulatory momentum give us confidence in the path ahead.

目前尚無核准用於治療 FSGS 患者的藥物，因此，推出 FILSPARI 的機會既緊迫又具有變革意義。DUPLEX 的利多數據和監管方面的進展讓我們對未來的道路充滿信心。

With our goal to provide FILSPARI as a foundational treatment for patients with IgA nephropathy and ultimately those with FSGS, we are pleased that the FDA approved modifications to our REMS program, removing the embryo fetal REMS and reducing the frequency of liver monitoring to quarterly.

為了實現將 FILSPARI 作為 IgA 腎病患者的基礎治療，並最終用於治療 FSGS 患者的目標，我們很高興 FDA 批准了我們 REMS 計劃的修改，取消了胚胎胎兒 REMS，並將肝臟監測的頻率降低到每季度一次。

The feedback we have heard from nephrologists is that these changes are welcomed. The monitoring frequency aligns with how they care for their patients in clinical practice. And these changes can help increase access for the subset of patients for whom monthly testing was an impediment.

我們從腎臟科醫生那裡得到的回饋是，他們歡迎這些改變。監測頻率與他們在臨床實踐中照顧患者的方式一致。這些改變有助於提高部分患者的就醫機會，因為每月一次的檢測對他們來說是一個障礙。

Turning to our pegtibatinase development program for the treatment of classical HCU. We recently presented long-term data at the ICIEM Congress from Cohort 6 in our Phase I/II COMPOSE open-label extension. At the 2.5 milligrams per kilogram twice weekly target dose, patients treated with pegtibatinase achieved sustained and clinically meaningful reductions in total homocysteine and methionine over an additional year of follow-up, remarkable results in the context of an open-label study. Importantly, we have successfully manufactured the first commercial scale batches of pegtibatinase and have generated data to support FDA interactions. This progress positions us for an expected restart of enrollment in the pivotal Phase III HARMONY study next year, reinforcing our commitment to advancing the only investigational therapy with disease-modifying potential for patients with classical HCU.

轉向我們的pegtibatinase開發項目，用於治療經典HCU。我們最近在 ICIEM 大會上展示了來自 I/II 期 COMPOSE 開放標籤擴展研究第 6 組隊列的長期數據。在每週兩次、每次 2.5 毫克/公斤的目標劑量下，接受培格替巴汀酶治療的患者在額外一年的隨訪中，總同型半胱氨酸和蛋氨酸水平持續且具有臨床意義地降低，這在開放標籤研究中取得了顯著成果。重要的是，我們已經成功生產了第一批商業規模的培格替巴汀酶，並產生了支持與 FDA 互動的數據。這項進展使我們有望在明年重新啟動關鍵的 III 期 HARMONY 研究的招募工作，從而鞏固了我們推進唯一具有改善經典 HCU 患者疾病狀況潛力的研究性療法的承諾。

I will now turn the call over to Peter for a commercial update. Peter?

現在我將把電話轉給彼得，讓他帶來最新的商業資訊。彼得？

Thank you, Jula. I am very pleased to share that the third quarter marked another period of strong commercial performance and continued momentum for FILSPARI in IgA nephropathy, reinforcing its position as a foundational therapy.

謝謝你，朱拉。我非常高興地宣布，第三季標誌著 FILSPARI 在 IgA 腎臟病領域又一次取得了強勁的商業業績和持續的成長勢頭，鞏固了其作為基礎療法的地位。

FILSPARI net product sales reached approximately $91 million in the third quarter, representing another quarter of strong growth, driven by consistent demand and deepening engagement among new and experienced prescribers.

FILSPARI 第三季淨產品銷售額達到約 9,100 萬美元，再次實現強勁成長，這得益於持續的需求以及新舊處方醫生參與度的提高。

Demand for FILSPARI remains robust with 731 new patient start forms received during the quarter despite experiencing summer seasonality as is typical in the summer months. In fact, in September, we recorded our highest daily patient start form rate since launch and we are seeing that trend continue into October.

儘管夏季月份通常會出現季節性波動，但 FILSPARI 的需求依然強勁，本季收到了 731 份新的患者開始治療申請表。事實上，9 月我們創下了自推出以來最高的每日患者開戶申請率，我們看到這一趨勢在 10 月仍在繼續。

Throughout the quarter, we saw durable utilization among existing nephrologists and a continuation of new prescribers. Importantly, we are seeing a steady increase in the number of practices treating multiple patients with FILSPARI, which highlights growing confidence in the therapy's profile and real-world performance.

整個季度，我們看到現有腎臟科醫生持續使用該產品，並且不斷有新的醫生開立處方。重要的是，我們看到越來越多的診所使用 FILSPARI 治療多名患者，這凸顯了人們對該療法的療效和實際表現越來越有信心。

As the IgA nephropathy treatment landscape evolves, we continue to hear consistent feedback from the nephrology community, reinforcing that physicians view FILSPARI as the preferred novel therapy, not only because of its proteinuria efficacy. But because it delivers a meaningful long-term improvement in kidney outcomes while allowing patients to maintain a normal lifestyle through a once-daily oral regimen. And we are encouraged by the response of the nephrology community to the modification of our REMS program. This simplification makes FILSPARI treatment even more convenient, particularly for newly diagnosed or lower-risk patients as quarterly monitoring is consistent to nephrology clinical practice.

隨著 IgA 腎病治療格局的演變，我們不斷聽到來自腎臟病學界的回饋，這進一步表明，醫生們認為 FILSPARI 是首選的新療法，不僅僅是因為它對蛋白尿的療效。但它能從長遠角度顯著改善腎臟疾病的治療效果，同時也能讓患者透過每日一次的口服療法維持正常的生活方式。腎臟病學界對我們 REMS 計畫修改的回應令我們倍感鼓舞。這種簡化使得 FILSPARI 治療更加方便，特別是對於新確診或低風險患者而言，因為每季監測與腎臟病臨床實踐一致。

We are pleased to see continued uptake of FILSPARI among patients with lower proteinuria levels, reflecting growing recognition that patients above 0.5 gram per gram remain at risk of progression in alignment with our broader label and the KDIGO guidelines. Patient satisfaction is strong as evidenced by consistently high compliance and persistence.

我們很高興看到 FILSPARI 在蛋白尿水平較低的患者中持續得到應用，這反映出人們越來越認識到，蛋白尿水平高於 0.5 克/克的患者仍有病情進展的風險，這與我們更廣泛的標籤和 KDIGO 指南相一致。患者滿意度很高，從持續較高的依從性和堅持性即可看出。

As we continue to expand FILSPARI's reach, our patient services and fulfillment programs remain an important contributor. We have maintained broad payer coverage with easing of prior authorization requirements to reflect FILSPARI's broader label, long-term evidence and positioning in the guidelines.

隨著 FILSPARI 業務範圍的不斷擴大，我們的患者服務和履行計劃仍然是重要的貢獻因素。我們透過放寬事先授權要求，維持了廣泛的支付方覆蓋範圍，以反映 FILSPARI 更廣泛的標籤、長期證據和在指南中的定位。

Turning to FSGS. If approved, FILSPARI will become the first approved medicine for FSGS, a leading cause of kidney failure. Given the high degree of overlap between the FSGS and the IgA nephropathy prescriber base, we will be able to build upon strong brand awareness and familiarity of FILSPARI with many physicians that have already had experience with the product.

接下來討論FSGS。如果獲得批准，FILSPARI 將成為第一個獲準用於治療 FSGS（腎衰竭的主要原因之一）的藥物。鑑於 FSGS 和 IgA 腎病處方醫生群體高度重疊，我們將能夠利用 FILSPARI 強大的品牌知名度和眾多已使用過該產品的醫生的熟悉度。

Given the high unmet need for an approved medication and the progressive nature of FSGS, we believe this could be an even bigger opportunity with a more rapid uptake versus our launch in IgA nephropathy. We know the FSGS community is eagerly awaiting an effective medicine. And we will be ready to launch in January, if approved.

鑑於目前對已獲批准藥物的巨大未滿足需求以及 FSGS 的進展性，我們認為這可能是一個更大的機會，其市場接受度將比我們在 IgA 腎病領域的推出更快。我們知道 FSGS 患者群體正熱切期盼一種有效的藥物。如果獲得批准，我們將在1月份做好發布準備。

In summary, the third quarter represents another quarter of exquisite execution and continued growth for FILSPARI in IgA nephropathy. The combination of clinical product differentiation, early intervention, strong prescriber confidence and a consistent patient experience continues to drive momentum and position FILSPARI as a foundational and nephroprotective choice among IgA nephropathy therapies.

總而言之，第三季代表 FILSPARI 在 IgA 腎臟病領域又一個季度的出色執行和持續成長。臨床產品差異化、早期介入、處方醫生的強大信心以及一致的患者體驗，這些因素共同推動了 FILSPARI 的發展勢頭，使其成為 IgA 腎病治療中的基礎性和腎臟保護性選擇。

With our strong commercial foundation and expanding real-world experience, we remain confident in FILSPARI's ability to deliver sustainable growth and long-term leadership in rare kidney disease care. I am sincerely proud of the continued performance of our commercial teams and the dedication they bring every day to support patients and physicians. Their success in establishing FILSPARI in IgA nephropathy gives us great confidence in our ability to execute effectively in FSGS, and we will be ready if approved.

憑藉我們強大的商業基礎和不斷積累的實際經驗，我們仍然對 FILSPARI 在罕見腎病治療領域實現可持續增長和長期領先地位的能力充滿信心。我由衷地為我們商業團隊的持續出色表現以及他們每天為支持患者和醫生所付出的奉獻精神感到自豪。他們在 IgA 腎病領域成功應用 FILSPARI，這讓我們對有效實施 FSGS 治療充滿信心，如果獲得批准，我們將做好準備。

Let me now turn the call over to Chris for the financial update. Chris?

現在我把電話交給克里斯，讓他報告財務狀況。克里斯？

Thank you, Peter, and good afternoon. This quarter, we delivered another strong set of financial results with continued significant revenue growth and disciplined financial investments. As Peter mentioned, our top line expansion reflects the strength of our underlying FILSPARI business and the consistent execution across our key commercial initiatives, momentum that we believe sets us up for durable growth ahead.

謝謝你，彼得，下午好。本季度，我們憑藉著持續顯著的營收成長和審慎的財務投資，再次取得了強勁的財務表現。正如彼得所提到的，我們的營收成長反映了我們 FILSPARI 業務的強勁實力以及我們在關鍵商業舉措方面的持續執行，我們相信這種勢頭將為我們未來的持續成長奠定基礎。

We also further strengthened our financial foundation by repaying our remaining 2025 convertible notes and significant value was generated from our partnerships, including the recently achieved $40 million market access milestone from CSL Vifor and the announced acquisition of Renalys by Chugai, both great examples of how our collaborations continue to create value and validate the potential of FILSPARI globally.

我們也透過償還剩餘的 2025 年可轉換票據進一步鞏固了財務基礎，並且透過合作夥伴關係創造了顯著價值，包括最近從 CSL Vifor 獲得的 4000 萬美元市場准入里程碑，以及宣布中外製藥收購 Renalys，這兩個例子都很好地說明了我們的合作如何繼續創造價值並驗證 FILSPARI 在全球範圍內的潛力。

Starting with revenue. In the third quarter, we generated US net product sales of $113.2 million. FILSPARI continued to grow significantly in the third quarter, generating $90.9 million in US net product sales, which represents an increase of more than 155% year-over-year. From a gross to net perspective, FILSPARI had a onetime benefit of less than $2 million during the quarter. And we continue to anticipate higher discounts in the fourth quarter.

首先從營收說起。第三季度，我們在美國實現了淨產品銷售額 1.132 億美元。FILSPARI 第三季持續維持顯著成長，在美國的淨產品銷售額達到 9,090 萬美元，年成長超過 155%。從毛利到淨利的計算來看，FILSPARI 在本季獲得了不到 200 萬美元的一次性收益。我們預計第四季折扣力度會更大。

Elsewhere, DILI contributed $22.3 million in US net product sales. And we also recognized $51.7 million of license and collaboration revenue, which results in total revenue of $164.9 million for the quarter. Included in the license and collaboration revenue line this quarter is a $40 million market access milestone that was achieved by CSL Vifor. We recently received payment, which will be reflected in our cash balance in the fourth quarter. Also included in the license and collaboration this quarter is $9.3 million in noncash revenue that resulted from the relinquishment of our option to acquire Renalys in anticipation of their agreement to be acquired by Chugai.

在其他方面，DILI 為美國貢獻了 2,230 萬美元的淨產品銷售額。此外，我們也確認了 5,170 萬美元的授權和合作收入，使得該季度的總收入達到 1.649 億美元。本季度授權和合作收入中包含 CSL Vifor 實現的 4,000 萬美元市場准入里程碑。我們最近收到了款項，這筆款項將在第四季度的現金餘額中體現出來。本季授權和合作還包括 930 萬美元的非現金收入，這是由於我們放棄了收購 Renalys 的選擇權，以期他們同意被 Chugai 收購。

Moving to operating expenses. Our research and development expenses for the third quarter of 2025 were $51.9 million compared to $51.7 million for the same period in 2024. On a non-GAAP adjusted basis, R&D expenses were $47.8 million compared to $48.4 million for the same period in 2024. Selling, general and administrative expenses for the third quarter were $86.5 million compared to $65.6 million for the same period in 2024. On a non-GAAP adjusted basis, SG&A expenses were $63.5 million for the third quarter compared to $49.7 million for the same period in 2024. The increase in SG&A is primarily attributable to investments in preparations for a potential launch in FSGS in January, increased amortization expense related to FILSPARI royalties as well as an increased investment in supporting commercial efforts for FILSPARI in IgA nephropathy following full approval.

接下來是營運費用。2025 年第三季度，我們的研發費用為 5,190 萬美元，而 2024 年同期為 5,170 萬美元。按非GAAP調整後，研發費用為4,780萬美元，而2024年同期為4,840萬美元。第三季的銷售、一般及行政費用為 8,650 萬美元，而 2024 年同期為 6,560 萬美元。依非GAAP調整後，第三季銷售、一般及行政費用為6,350萬美元，2024年同期為4,970萬美元。銷售、一般及行政費用的增加主要歸因於為 1 月份 FSGS 的潛在上市做準備的投資、與 FILSPARI 特許權使用費相關的攤銷費用增加，以及在 FILSPARI 獲得全面批准後，為支持 IgA 腎病商業化工作而增加的投資。

Total other income net for the third quarter of 2025 was less than $1 million compared to $1.3 million for the same period in 2024. Net income for the third quarter of 2025 was $25.7 million or $0.29 per basic share compared to a net loss of $54.8 million or $0.70 per basic share for the same period in 2024. On a non-GAAP adjusted basis, net income for the third quarter of 2025 was $52.8 million or $0.59 per basic share compared to a net loss of $35.6 million or $0.46 per basic share for the same period 2024.

2025 年第三季其他淨收入總額不足 100 萬美元，而 2024 年同期為 130 萬美元。2025 年第三季淨收入為 2,570 萬美元，即每股基本收益 0.29 美元，而 2024 年同期淨虧損為 5,480 萬美元，即每股基本虧損 0.70 美元。依非GAAP調整後，2025年第三季淨收入為5,280萬美元，即每股基本虧損0.59美元，而2024年同期淨虧損為3,560萬美元，即每股基本虧損0.46美元。

As of September 30, 2025, we had cash, cash equivalents and marketable securities totaling approximately $254.5 million. This balance reflects our repayment of the remaining $69 million in 2025 convertible notes. And as I highlighted earlier, it does not yet reflect the proceeds of the $40 million milestone payment from Vifor and it also does not yet include any proceeds from the recently announced acquisition of Renalys by Chugai.

截至 2025 年 9 月 30 日，我們擁有現金、現金等價物及有價證券總計約 2.545 億美元。該餘額反映了我們償還剩餘的 6,900 萬美元 2025 年可轉換票據。正如我之前強調的那樣，它還沒有反映出 Vifor 支付的 4000 萬美元里程碑付款，也沒有包括最近宣布的 Chugai 收購 Renalys 的任何收益。

As we move forward, we are well positioned to sustain our momentum in IgA nephropathy, execute a successful launch in FSGS if approved and advance the reinitiation of enrollment in our pegtibatinase Phase III study next year. Importantly, we're doing all of this from a position of financial strength with no near-term need for additional capital to execute on our core objectives. This foundation gives us confidence in our ability to execute on our key priorities and continue advancing our mission for patients.

展望未來，我們已做好充分準備，保持我們在 IgA 腎病領域的良好勢頭，如果獲得批准，將在 FSGS 領域成功推出產品，並於明年重新啟動 pegtibatinase III 期研究的患者招募工作。重要的是，我們是在財務實力雄厚的情況下開展所有工作的，近期不需要額外的資金來實現我們的核心目標。這項基礎使我們有信心執行我們的關鍵優先事項，並繼續推進我們為患者服務的使命。

I'll now turn it over to Eric for his closing comments. Eric?

現在我將把發言權交給艾瑞克，請他作總結陳詞。艾瑞克？

Thank you, Chris. In Q3, we made tremendous strides across all of our programs. And I am proud of how every employee shows up with passion and focus to advance our mission. One great example is our pegtibatinase team, who has diligently solved scale-up challenges so that we are positioned to restart the HARMONY trial next year.

謝謝你，克里斯。第三季度，我們所有專案都取得了巨大進步。我為每位員工都充滿熱情、專注地投入推進我們使命的工作而感到自豪。一個很好的例子就是我們的 pegtibatinase 團隊，他們勤奮地解決了規模化難題，使我們能夠在明年重新啟動 HARMONY 試驗。

October is HCU awareness month. And it is a fitting reminder of how much work is still needed to allow families affected by HCU to live with a little less worry and a bit more hope. We've entered the final months of 2025 confident in our ability to sustain FILSPARI's growth in IgAN to successfully execute on a potential approval and launch in FSGS and to advance our pipeline with focus. We have the right people, a strong financial foundation and the momentum to bring incredible innovation to the rare disease communities that have been waiting far too long.

十月是HCU宣傳月。這恰當地提醒我們，要讓受 HCU 影響的家庭少一點憂慮，多一些希望，還有很多工作要做。進入 2025 年的最後幾個月，我們有信心維持 FILSPARI 在 IgAN 領域的成長，成功獲得 FSGS 的潛在批准並上市，並有重點推進我們的研發管線。我們擁有合適的人才、雄厚的財力和強勁的發展勢頭，能夠為等待已久的罕見疾病群體帶來令人難以置信的創新。

I'll now turn the call over to Nivi for Q&A. Nivi?

現在我將把電話交給Nivi進行問答環節。尼維？

Thank you, Eric, operator, we can now open up the line for Q&A.

謝謝接線員艾瑞克，我們現在可以開通問答環節了。

(Operator Instructions)

（操作說明）

Joe Schwartz, Leerink Partners.

Joe Schwartz，Leerink Partners。

Great, thanks. Congrats on another strong quarter of execution. With the new label approved in August, can you quantify either qualitatively or quantitatively the early impact of the REMS adjustment? Are you seeing new prescribers or a new patient base that might have been more reluctant previously. It seems like with such a strong beat this quarter, you might not be seeing any competitive impacts? Or are you seeing any at all and it was just offset by the updated label? Any color you could provide would be great.

太好了，謝謝。恭喜你們又一個季度業績出色。新標籤已於 8 月獲得批准，您能否定性或定量地量化 REMS 調整的早期影響？您是否遇到了新的處方醫生或新的患者群體，他們以前可能比較猶豫不決？本季業績如此強勁，似乎不會受到任何競爭影響？或者你根本看不到任何內容，只是被更新後的標籤掩蓋了？您提供的任何顏色都行。

Thanks, Joe. Peter, why don't you take that question?

謝謝你，喬。彼得，你來回答這個問題吧？

Yes. Thanks, Joe. It's a good question. I think you're asking two questions. One is what is the impact of the REMS modification so far? And two, are you seeing any impact of competitive dynamics? I think overall, I would say we see very consistent demand since we had our full approval last year. And that consistency have not been impacted by launches of new products that came into the marketplace. So I think very robust continuation of growth.

是的。謝謝你，喬。問得好。我認為你問了兩個問題。一是 REMS 修改迄今為止產生了什麼影響？第二，您是否看到競爭動態產生了任何影響？總的來說，我認為自去年獲得全面批准以來，市場需求一直非常穩定。而且，這種穩定性並未受到市場上新產品上市的影響。所以我認為成長勢頭將非常強勁地持續下去。

I think to your first question with regards to the REMS modification, I think that is certainly a tailwind that we are having and that has been very positively received by the nephrology community. What we are seeing is that we have a continuation of new prescribers, while we also continue to expand within experienced prescribers. And I think especially the REMS modification from a monthly base to a quarterly base in the first year really helps for those patients that are not as sick at the higher proteinuria levels, but still are at significant risk of progression of disease. Those patients may not see the physician on a monthly base or may not do traditional testing on a monthly base, but certainly do it at a quarterly base. So I think the timing of the REMS modification fits very nicely in the expansion of the patient population that we are seeing.

關於您提出的第一個問題，即 REMS 修改，我認為這無疑是我們目前面臨的一個有利因素，並且受到了腎臟病學界的積極歡迎。我們看到的是，一方面不斷有新的處方醫生加入，另一方面，經驗豐富的處方醫生也不斷壯大。我認為，尤其是在第一年將 REMS 的評估頻率從每月改為每季度，對於那些尿蛋白水平較高但病情並不嚴重的患者，以及那些仍有重大疾病進展風險的患者來說，真的很有幫助。這些患者可能不會每月都去看醫生，也不會每月都進行傳統檢查，但肯定會每季進行檢查。所以我認為 REMS 修改的時機與我們所看到的患者群體擴大情況非常吻合。

Very helpful. Any insight into any competitive pressures at all? Or have you not detected any?

很有幫助。有沒有人了解是否有任何競爭壓力？還是你根本沒偵測到？

Yes. As I mentioned, we have seen very consistent demand. I would say Q3, we saw less of an impact of seasonality than we saw last year and that in a more competitive landscape. So I would say that gives you a color of our execution and performance in Q3.

是的。正如我之前提到的，我們看到了非常穩定的需求。我認為第三季度，季節性因素的影響比去年有所減弱，而且市場競爭也更加激烈。所以，我認為這可以讓你大致了解我們第三季的執行情況和業績。

Yes. That's great, Peter. And the only thing that I would offer in addition, Joe, is that not only did we see the modification of REMS, as you alluded to, which makes it just that much easier for physicians and patients. But we also saw the publication of the KDIGO guidelines that further reinforce the positioning of FILSPARI. And I think both of those in combination, of course, with the phenomenal execution of Peter's team continues to reinforce our strong position within this market.

是的。太好了，彼得。喬，我唯一要補充的是，正如你所提到的，我們不僅看到了 REMS 的修改，這使得醫生和病人都更容易操作。但我們也看到了 KDIGO 指南的發布，這進一步鞏固了 FILSPARI 的市場地位。我認為，這兩點結合起來，當然再加上彼得團隊出色的執行力，將繼續鞏固我們在這個市場中的強大地位。

Thank you.

謝謝。

Thank you.

謝謝。

Laura Chico, Wedbush.

勞拉·奇科，韋德布希。

Good afternoon. Thanks very much for taking the question. Just two quick ones for me. First, with respect to FILSPARI at this point, do you have a sense as to what the typical baseline proteinuria level is at start of prescribing? I think Peter made a comment about perhaps some patients coming in now with a lower level. Second, are you detecting any off-label use in the FSGS setting at this point?

午安.非常感謝您回答這個問題。我只需要回答兩個問題。首先，就目前 FILSPARI 而言，您是否了解開始服用該藥物時典型的基線蛋白尿水平是多少？我認為彼得曾說過，現在可能有些病人的血糖值較低。其次，目前您是否在 FSGS 設定中偵測到任何標籤外使用情況？

Thank you very much.

非常感謝。

Thanks, Laura, for those questions. I'll take the second one regarding FSGS. We do see some limited prescribing and use in FSGS. We, of course, do nothing to promote that. But we are seeing some physicians make that choice.

謝謝勞拉提出這些問題。我選擇關於FSGS的第二個答案。我們確實看到一些有限的處方和使用，用於治療 FSGS。我們當然不會採取任何措施來推廣這種做法。但我們看到一些醫生做出了這樣的選擇。

I will turn it over to Peter to ask your question or answer your question about baseline UPC.

我將把時間交給 Peter，讓他來問你關於基礎 UPC 的問題或回答你的問題。

Yes. Thanks, Laura. So what we have seen since we had a full approval last year in September is that we have seen consistently the baseline proteinuria levels are well below 1.5 gram per gram. And it's what you would expect. I mean, the larger patient population, about 65% of the patient population have proteinuria levels below 1.5. And we're making good inroads in penetrating that market segment. And what you would expect is that you will see a continuation of lower proteinuria levels at initiation.

是的。謝謝你，勞拉。自從去年九月獲得全面批准以來，我們看到的是，基線蛋白尿水平一直遠低於每克 1.5 克。這也在你的意料之中。我的意思是，在較大的患者群體中，大約 65% 的患者尿蛋白水平低於 1.5。我們在打入這個細分市場方面取得了不錯的進展。可以預見的是，在治療初期，蛋白尿量會持續降低。

Thanks very much.

非常感謝。

Anupam Rama, JPMorgan.

Anupam Rama，摩根大通。

Hey guys, thanks so much for taking the question. Congrats on the quarter. Just in the context of the beat that you guys had here with FILSPARI, how do we think about sort of the quarter-over-quarter declines in patient start forms? I know you mentioned some summer seasonality, but there were those tailwinds from guidelines and REMS. What are the considerations there? Anything to note on gross to net or inventory?

各位，非常感謝你們回答這個問題。恭喜你本季取得佳績。就你們之前提到的 FILSPARI 的情況而言，我們該如何看待病患起始表格數量較上季下降的問題呢？我知道你提到了一些夏季季節性因素，但指南和 REMS 也帶來了一些有利因素。這裡需要考慮哪些因素？關於毛利淨利或庫存方面有什麼需要注意的嗎？

Yes. Maybe I can frame this and then have Peter and Chris offer anything further. I think the strong performance in demand in Q3 really reflects that underlying expectation. And I'll have Peter talk about some of the trends within the quarter that we saw. But it really is about the seasonality. While we didn't see as much impact this year as we did last year, we certainly did see some of that in terms of the slower months.

是的。或許我可以把這個問題整理一下，然後讓彼得和克里斯再提供其他建議。我認為第三季強勁的需求表現確實反映了這一潛在預期。接下來，我會請彼得談談我們在本季觀察到的一些趨勢。但實際上，這主要還是季節性因素造成的。雖然今年受到的影響不如去年那麼大，但在淡季月份，我們確實看到了一些類似的影響。

Peter, maybe you can allude to that. And Chris, you can talk about the gross to net impact in Q3.

彼得，或許你可以暗示一下。克里斯，你可以談談第三季毛利與淨利的對比情況。

Yes. Happy to comment on that, Anupam, and thanks for that question. I'm actually really pleased with the performance and the demand we saw in Q3. In particular, what I outlined during the call, September, we had the strongest daily patient start form generation and that trend has continued in October. So I think very strong demand. And as I mentioned earlier, we have seen less of an impact of seasonality in a more competitive environment. So I think the performance is really strong. And yes, I couldn't be more proud of the team to continue to execute in the way they do.

是的。樂意就此發表評論，Anupam，感謝你的提問。我對第三季的業績和市場需求都非常滿意。特別是我在電話會議中概述的內容，9 月份我們每天的患者入院申請表生成量最高，而且這一趨勢在 10 月份仍在繼續。所以我認為需求非常強。正如我之前提到的，在競爭更加激烈的環境中，季節性因素的影響有所減弱。所以我覺得這場演出非常精彩。是的，我為團隊能夠繼續以這樣的方式執行任務而感到無比自豪。

Anupam, on the gross to net factor for this quarter, we did highlight that there was less than $2 million benefit. And really, that's just working through the first year here in Part D and having the true-ups as we go throughout the year.

Anupam，關於本季毛利與淨利的比率，我們確實強調了收益不到 200 萬美元。實際上，這只是在D部分第一年的工作，我們會在這一年中進行調整。

Looking ahead, we've guided to throughout the year that the back half may have higher gross to net. That remains the same for the fourth quarter. But we're still right around that guidance of around 20% for the year. And the fundamentals, as Eric and Peter highlighted, very strong. So we're looking forward to the end of the year here.

展望未來，我們全年都在預測下半年毛利潤與淨利比可能更高。第四季情況依然如此。但我們今年的成長目標仍在20%左右。正如埃里克和彼得所強調的那樣，基本面非常強勁。所以我們很期待今年的結束。

Thanks a lot.

多謝。

Tyler Van Buren, TD Cowen.

泰勒·範·布倫，TD·考恩。

Hi, this is Francis on for Tyler. What can we expect in terms of communication leading up to the FSGS PDUFA date in January? Is it possible that you'll disclose if and when you're in labeling discussions?

大家好，我是法蘭西斯，替泰勒為您報道。在 1 月份 FSGS PDUFA 日期之前，我們可以期待哪些方面的溝通？您能否透露您是否參與過標籤討論以及何時參與？

Francis, thanks for the question. It's been our practice not to comment on ongoing FDA interactions. And like we did during our IgAN review, we'll be entering a quiet period as we approach the PDUFA date. So you wouldn't expect any updates from us during that time. But we will provide and look forward to providing updates on January 13.

法蘭西斯，謝謝你的提問。我們一貫的做法是不對正在進行的與FDA的互動發表評論。就像我們在 IgAN 審查期間所做的那樣，隨著 PDUFA 日期的臨近，我們將進入一段靜默期。所以在此期間，您不會期待我們發布任何更新資訊。但我們將提供最新消息，並期待在1月13日提供更新資訊。

Yigal Nochomovitz, Citigroup.

Yigal Nochomovitz，花旗集團。

Hi guys, thank you very much for taking the question. So I wanted to ask about REMS and KDIGO. I'm just curious, when you're in the field now with the new message around the reduced REMS and the better KDIGO guidelines, how many of the practitioners are sort of aware of these changes or were informed outside of the channels through Travere? Or is it really that the information is coming from Travere in terms of learning about the better REMS and the KDIGO? Just how is that information flowing? It would be interesting to understand a little better.

大家好，非常感謝你們回答這個問題。所以我想問一下關於REMS和KDIGO的問題。我只是好奇，當您現在帶著關於減少 REMS 和改進 KDIGO 指南的新資訊在現場工作時，有多少從業人員了解這些變化，或者透過 Travere 等管道以外的途徑獲悉這些變化？或者，關於更好的 REMS 和 KDIGO 的資訊真的來自 Travere 嗎？這些資訊是如何流通的？如果能更深入了解一下就太好了。

Thank you.

謝謝。

Yes, Peter, do you want to take that? And then, Jula, do you have anything further from your engagement with KOLs? Peter?

是的，彼得，你想拿嗎？那麼，Jula，你與KOL的互動還有什麼其他收穫嗎？彼得？

Happy to take that one. I mean it was a year ago that KDIGO disclosed the draft guidelines. And I think familiar, the key opinion leaders and the thought leaders, they were well familiar with the KDIGO guidelines. But what we are seeing now is the full publication that it really trickles down to the community nephrologists as well. And so that publication really helps there. And our team is certainly -- it fits nicely in our educational efforts with physicians.

我樂意接受這個任務。我的意思是，KDIGO 公佈指導方針草案已經是一年前的事了。而且我認為，關鍵意見領袖和思想領袖們對 KDIGO 指南非常熟悉。但我們現在看到的是，完整的出版物也真正滲透到了社區腎臟科醫生那裡。所以，那份出版品確實很有幫助。我們的團隊當然也——這與我們面向醫生的教育工作非常契合。

With regards to the REMS modification, that is really up to us to communicate to physicians. And like I said in the prepared remarks, I'm really pleased with the reaction and the response we got from physicians of that modification in the first year and how this fits very nicely with their clinical practice, not having to have that monthly monitoring, but doing it at a quarterly base from the get-go. And like I said -- I mean, this is something that they are doing on a quarterly basis anyway. So there is no additional burden for the physician, neither for the patient.

關於 REMS 的修改，這確實需要我們與醫生溝通。正如我在準備好的發言稿中所說，我對第一年醫生們對這一修改的反應和反饋非常滿意，而且這一修改與他們的臨床實踐非常契合，無需每月進行監測，而是從一開始就按季度進行監測。就像我說的——我的意思是，無論如何，這是他們每季都會做的事情。因此，醫生和患者都不會增加額外的負擔。

Okay. Then on pegtibatinase, just very quickly, is the scale-up basically a completed project now? Or is there any more work to do to make sure you have enough supply for the whole HCU market?

好的。那麼關於培替巴汀酶，簡單來說，規模化生產現在基本上已經完成了嗎？或者，是否還有其他工作要做，以確保有足夠的供應來滿足整個HCU市場的需求？

Thanks, Yigal. Bill, why don't you take that one?

謝謝你，伊加爾。比爾，你為什麼不接下這個任務呢？

Certainly. Well, we're very pleased to have completed our first commercial batches. This enables us to engage with the FDA as was planned, which enables the restart of the study in the next year. We will continue additional manufacturing campaigns in parallel with the study running to do the further characterization work that's required for the BLA and to build stock for launch. But the key milestone is getting to this scale of manufacture, so that we can restart enrollment in the Phase III study.

當然。我們很高興完成了首批商業生產。這樣一來，我們就可以按計劃與 FDA 進行溝通，使這項研究能夠在明年重新啟動。我們將繼續進行額外的生產活動，與研究同步進行，以完成 BLA 所需的進一步表徵工作，並為上市生產儲備物資。但關鍵的里程碑是達到這樣的生產規模，這樣我們就可以重新啟動 III 期研究的招募工作。

Gavin Clark-Gardner, Evercore.

Gavin Clark-Gardner，Evercore。

I'm sorry to go back to kind of the net price discussion. But even if I take a couple of million out there for the onetime net price boost, I think the revenue was still a little bit higher than some investors were anticipating based on the PSS trajectory. I'm just curious like is this volume of PSS trajectory that you got, like this quarter and last quarter, which is fairly consistent. Is the revenue growth you're seeing based on that something we should be extrapolating going forward? And like how much is the Q4 gross to net impact? Thanks so much.

很抱歉又回到淨價這個話題了。但即使我扣除幾百萬美元的一次性淨價上漲，我認為收入仍然比一些投資者根據 PSS 的發展軌跡所預期的要高一些。我只是好奇，你得到的 PSS 軌蹟的這個量級，例如本季和上個季度，是否相當穩定。基於此，您看到的營收成長是否值得我們進一步推論？第四季毛利與淨利比是多少？非常感謝。

Thanks, Gavin. Chris, why don't you take that?

謝謝你，加文。克里斯，你為什麼不拿走？

Sure. So I think one of the things that Peter has mentioned along the way is that we've continued to refine our pull-through process and we've really made good progress there. So I think that's part of what's driving the revenue growth that has been able to outpace the PSS growth over time. We've also seen very strong compliance and persistence. I think, again, that's another testament to the overall profile for FILSPARI.

當然。所以我覺得彼得一路以來提到的其中一點是，我們一直在不斷改進我們的推廣流程，我們在這方面取得了很大的進展。所以我認為這在一定程度上推動了營收成長，使其能夠隨著時間的推移超過產品服務價格（PSS）的成長。我們也看到了非常強烈的服從性和堅持性。我認為這再次證明了 FILSPARI 的整體實力。

On the gross to net front, we haven't broken it down specifically by quarter. But the third quarter was similar to the second quarter, slightly lower. We would expect that to increase in the fourth quarter. And overall for the year, we're expecting to come out right around the 20% mark. So that's about as much of the guidance as we can provide at this point. But hopefully, that gives you a better sense for how to model that out, Gavin.

在毛利潤與淨利方面，我們還沒有按季度進行具體細分。但第三季與第二季類似，略有下降。我們預計第四季這一數字會上升。總的來說，我們預計全年成長率將在 20% 左右。所以，目前我們能提供的指導就這麼多了。但希望這能讓你更了解如何建模，加文。

Yeah, that's really helpfu. Thanks guys.

是的，這真的很有幫助。謝謝各位。

Thank you.

謝謝。

Mohit Bansal, Wells Fargo.

莫希特·班薩爾，富國銀行。

Thanks for taking my question. Congrats on the progress. So in FSGS, I think we might see some data from Novartis soon with atrasentan from their basket trial. Can you talk about advantages you see with a dual ERA mechanism in this indication compared with an agent like atrasentan, which doesn't have the RAS inhibitor component, especially this being an indication where there is not as high background use of RAS inhibitors compared with IgAN?

謝謝您回答我的問題。恭喜你取得進展。所以，我認為在 FSGS 方面，我們可能很快就會看到諾華公司關於阿曲生坦的籃子試驗的一些數據。您能否談談與阿曲生坦（一種不含 RAS 抑制劑成分的藥物）相比，雙重 ERA 機制在該適應症中的優勢？尤其是在 RAS 抑制劑的背景使用率不如 IgAN 高的情況下。

Thank you.

謝謝。

Thanks for the question. Jula, why don't we have you answer that?

謝謝你的提問。朱拉，為什麼不讓你來回答這個問題呢？

Certainly, it is quite important in FSGS, which is a true podocytopathy that's at the heart of the disease to target it with both endothelin and angiotensin II together to have the greatest nephroprotective potential. And we also see that with the magnitude of proteinuria reduction we see in this patient population of FILSPARI being used. We see about a 50% reduction in proteinuria that's durable out to two years. And that's where we have the confidence that this is the right way to target these patients to provide them long-term kidney protection. I understand there might be some use of single agents. I won't comment on the lack of data that we have regarding atrasentan. We really haven't seen anything to date.

當然，對於 FSGS（一種真正的足細胞病，是該疾病的核心）來說，同時使用內皮素和血管緊張素 II 進行治療非常重要，這樣才能發揮最大的腎臟保護作用。我們也看到，使用 FILSPARI 的患者族群中，蛋白尿減少的幅度很大。我們發現蛋白尿減少了約 50%，且效果可持續兩年。正因如此，我們才有信心，這是針對這些患者提供長期腎臟保護的正確方法。我了解到可能有一些使用單一代理的方法。我不會對我們目前缺乏的關於阿曲生坦的數據發表評論。到目前為止，我們真的什麼都沒看到。

So I can't comment on what that gap might leave behind when you don't target both mechanisms. We know when we target both mechanisms, we have -- we get more patients into complete remission as well as greater reductions in proteinuria and FSGS, and that's what really matters.

因此，我無法評論如果不針對這兩種機制，可能會造成什麼後果。我們知道，當我們同時針對這兩種機制時，我們就能讓更多患者達到完全緩解，並大幅減少蛋白尿和 FSGS，而這才是真正重要的。

Got it, thank you.

明白了，謝謝。

Prakhar Agarwal, Cantor.

Prakhar Agarwal，坎托爾。

Hi, thank you for my questions. So Novartis during their earnings said that they have 20% NBRx share, 10% of that is coming from Venrefa and the rest is from Fibralta. So maybe if you can expand on where you are seeing Venrefa and Fibralta as gaining share? And then another follow-up on IgAN. You said September was the strongest month, and October you're also seeing good consistent demand. So should we expect the new patient start forms to increase sequentially in 4Q?

您好，感謝您提出的問題。諾華在財報中表示，他們擁有 20% 的 NBRx 股份，其中 10% 來自 Venrefa，其餘來自 Fibralta。那麼，您能否詳細說明您認為 Venrefa 和 Fibralta 在哪些方面會獲得市場份額？然後是另一篇關於IgAN的後續報導。你說九月是銷售最強勁的月份，十月份的需求也保持良好且穩定。那麼我們是否應該預期第四季新病患入院申請表數量會逐季增加？

Thank you.

謝謝。

Thank you for the questions. Peter, why don't you take those?

謝謝大家的提問。彼得，你為什麼不拿走那些？

Yes, I'm happy to take that question. I mean what we have seen -- and I mentioned that before, is that we see very consistent and steadily growing demand since we had our full approval in September last year. And the launch of atrasentan or iptacopan has not really changed that. I mean, iptacopan was launched basically at the same time as we have full approval. Atrasentan was launched like six months ago. But it hasn't really changed our trajectory and our continuation of the momentum. So I couldn't be more pleased with the execution and what we are seeing. And I think now with the REMS modification as well as the KDIGO guidelines, I think those are additional momentum builders for us. And so I remain very confident in a more competitive landscape.

是的，我很樂意回答這個問題。我的意思是，我們已經看到——我之前也提到過——自從去年九月獲得全面批准以來，我們看到需求一直非常穩定且穩定成長。而阿曲生坦或伊普他可潘的上市並沒有真正改變這種情況。我的意思是，iptacopan 的推出基本上與我們獲得全面批准的時間是同時進行的。阿特拉生坦大約六個月前上市。但這並沒有真正改變我們的發展軌跡和我們繼續保持發展勢頭的現狀。所以我對最終的執行效果和我們目前看到的成果非常滿意。我認為，隨著 REMS 修改和 KDIGO 指南的出台，這些都為我們帶來了額外的動力。因此，即使在競爭日益激烈的市場環境中，我仍然充滿信心。

Yes. And just to add with regard to whether you can expect sequential increase we've not provided guidance. What Peter shared in the past is, I think, two really important components of that. One is we expect that demand to be above 700 in terms of that quarterly demand. We certainly have seen that as he talked about. But also we think about the large opportunity to be able to have these patients move from RAS inhibition to dual inhibition with something like FILSPARI or the addition of ERA.

是的。另外，關於是否可以預期業績會持續成長，我們尚未提供相關指引。我認為，彼得過去分享的內容，包含了其中兩個非常重要的組成部分。第一，我們預計該季度需求量將超過 700。正如他所說，我們確實看到了這一點。但我們也考慮到，如果能夠讓這些患者從 RAS 抑製過渡到使用 FILSPARI 之類的藥物進行雙重抑制，或者添加 ERA，那將是一個很大的機會。

Most of these patients still are on only RAS. So there is a tremendous opportunity for growth. We're clearly making that progress. We're seeing those occur. And I don't want to speak about other companies' performance. They're clearly helping to be able to increase the shift from RAS inhibition. But as you can see, we've not really seen an impact from their launches.

這些患者中的大多數目前仍然只接受 RAS 治療。因此，發展機會巨大。我們顯然正在取得進展。我們看到這種情況正在發生。我不想評論其他公司的表現。它們顯然有助於增加從 RAS 抑制向 RAS 抑制的轉變。但正如你所看到的，我們並沒有真正看到他們的產品發布帶來的影響。

Maury Raycroft, Jeffries.

莫里‧雷克羅夫特，傑弗里斯。

Hi. Congrats on the quarter. You mentioned that your increased SG&A for the third quarter includes additional investment in preparation for a potential FSGS launch. Can you talk more about how you're prepping for the launch and how we should think about SG&A expectations going forward?

你好。恭喜你本季取得佳績。您提到第三季增加的銷售、一般及行政費用包括為可能推出 FSGS 而進行的額外投資。您能否詳細介紹一下您為產品發布所做的準備工作，以及我們應該如何看待未來的銷售、一般及行政費用預期？

Sure. Peter, why don't you take the question with regard to how your team is preparing for the approval? And Chris, you can talk about SG&A.

當然。彼得，你為什麼不回答一下關於你的團隊如何準備審批的問題呢？克里斯，你可以談談銷售、管理及行政費用。

Yes, Maurice, first of all, I think it's good to realize that this is basically the same prescriber base in FSGS as what we have seen for IgA nephropathy. Basically, the only nephrology segment that we haven't called upon is the pediatric nephrologists. But overall, there's a high level of overlap. So we build upon strength and high brand familiarity. We will have an incremental increase in our commercial footprint to really continue that momentum in IgA nephropathy while also enabling the early uptake that we are envisioning for FILSPARI. So we are building upon strength. And like I said, we have that incremental increase of our commercial footprint.

是的，莫里斯，首先，我認為應該意識到，FSGS 的處方醫生群體基本上與我們在 IgA 腎臟病中看到的相同。基本上，我們唯一還沒聯絡到的腎臟科專家就是兒科腎臟病專家。但總體而言，兩者之間存在高度重疊。因此，我們依賴自身實力和較高的品牌知名度。我們將逐步擴大商業版圖，以繼續保持 IgA 腎病領域的成長勢頭，同時實現我們設想的 FILSPARI 的早期推廣。所以我們正在鞏固優勢。正如我所說，我們的商業版圖正在逐漸擴大。

Maurice, as you can take from Peter's comments, with bringing on some additional sales team members and some other support services here, we do expect to see an incremental increase in SG&A. We started to onboard a number of those people this quarter. But really, you'll see more of that effect in 4Q and going forward. And then around the time of launch, you would also anticipate that we'll have an increase in investment level as we're really making sure that we're providing the right resources to have a very strong start out of the gate early next year. So incremental increases as we go, but we are building from a very strong base. And we're going to be able to leverage a lot of synergies from Peter's team that's performing quite well right now.

莫里斯，正如彼得的評論所表明的那樣，隨著銷售團隊增加一些成員和其他支援服務，我們預計銷售、一般及行政費用將逐步增加。本季我們開始陸續招收其中一些人。但實際上，這種影響會在第四季及以後更加明顯。然後，在產品發布前後，預計投資水平也會提高，因為我們正在確保提供合適的資源，以便在明年初取得強勁的開局。所以我們會逐步增加，但我們的基礎非常堅實。我們將能夠充分利用彼得團隊目前表現出色所帶來的綜效。

Got it. Okay, thanks for taking.

知道了。好的，謝謝。

Thanks, Maury.

謝謝你，莫里。

Jason Zemansky, Bank of America.

傑森‧澤曼斯基，美國銀行。

Good afternoon. Congrats on the great progress. I wanted to revisit the efforts to now completely remove the REMS. I guess, first, given the acceleration in patient starts here and therefore, overall exposure to FILSPARI, have your time lines changed at all? And then I guess, any other updates on this front now that the original REMS modification has occurred?

午安.祝賀你們取得巨大進展。我想重新審視徹底取消 REMS 的努力。我想，首先，考慮到患者數量的加速成長以及因此而來的 FILSPARI 整體暴露量增加，你們的時間安排是否有所改變？那麼，既然最初的 REMS 修改已經完成，這方面還有其他更新嗎？

Thanks.

謝謝。

Thanks, Jason. Bill, why don't you take that question?

謝謝你，傑森。比爾，你來回答這個問題吧？

Sure. And we're excited about the REMS modification that was granted in August. And I think we've seen the tailwinds that that provides and the positive feedback from physicians and patients. Our strategy has always been for ultimate removal of the REMS. And with our prior interactions with the agency, we've approached it with a two-step process with seeing the frequency change first and then removal second.

當然。我們對 8 月獲批的 REMS 修改感到非常興奮。我認為我們已經看到了這帶來的利多因素，以及來自醫生和患者的正面回饋。我們的策略始終是最終取消風險評估和緩解策略（REMS）。根據我們先前與該機構的接觸，我們採取了兩步驟的方法，首先觀察頻率變化，然後再進行移除。

As we've noted in the past, the FDA has been anchored on our PMR study, which requires exposure across about 3,000 patients for two years. So our process really hasn't changed. Consistent with our approach, we'll continue to engage with the agency and align with them on our next steps.

正如我們過去所指出的，FDA 一直以我們的 PMR 研究為依據，該研究需要對大約 3,000 名患者進行為期兩年的暴露試驗。所以我們的操作流程其實沒有改變。按照我們一貫的做法，我們將繼續與該機構保持溝通，並與他們就下一步行動達成協議。

Alex Thompson, Stifel.

Alex Thompson，Stifel。

Hey, great, thanks for taking our question. Maybe a follow-up on the commentary on some off-label FSGS use. I wonder if you could comment as to whether those patients are coming in at about 2x the IgAN dose or if they're still early in their treatment course and maybe not titrate up fully yet.

嘿，太好了，謝謝你回答我們的問題。或許可以對一些非適應症用藥的 FSGS 情況進行後續評論。我想請您評論一下，這些患者入院時的 IgAN 劑量是正常劑量的兩倍左右，還是他們仍處於治療早期，劑量尚未完全增加？

Thanks.

謝謝。

Alex, thanks for the question. So we do have limited insight into some of that information. And I would not want to generalize around the dosing at this point. I think what's important is that upon an approval, we would make sure that physicians are appropriately educated on the label, on the target dose. And of course, as we have with IgAN, we've got strong patient services support for the patients and their offices to ensure that they're at the appropriate dose.

Alex，謝謝你的提問。所以，我們對其中一些資訊的了解確實有限。目前我不想就劑量問題進行概括。我認為重要的是，一旦獲得批准，我們將確保醫生們對藥品標籤和目標劑量進行適當的教育。當然，就像我們對待 IgAN 一樣，我們為患者及其診所提供了強大的患者服務支持，以確保他們獲得適當的劑量。

Joe Pantginis, H.C. Wainwright.

喬·潘特吉尼斯，H.C. 溫賴特。

Hi, thanks and good afternoon. So first, I want to talk more about the expenses that you mentioned earlier, but to the totality of the expenses going forward. I won't ask you to project profitability timing. But I guess, can you directionally speak to especially R&D going forward as you're going to bringing PEG back into the clinic and how we should sort of view that offset by FILSPARI revenues?

您好，謝謝，下午好。首先，我想更詳細地談談您之前提到的費用，以及未來所有費用的總和。我不會要求你預測獲利時間。但我想，您能否就未來的研發方向談談，特別是當您將 PEG 重新引入臨床時，我們應該如何看待 FILSPARI 的收入抵消這部分投入？

Secondly, I'm just curious with regard to Renalys and Chugai, any change in time lines for development of sparsentan in Japan, South Korea and Taiwan?

其次，我很好奇Renalys和Chugai，日本、韓國和台灣的稀疏豆開發時間表是否有任何變化？

Joe, thanks for the questions. I'll quickly address the last one and then turn it over to Chris to answer the questions on expenses. No change in time lines. We've been incredibly impressed with the speed and quality of work from Renalys and we have a high regard for Chugai Pharmaceuticals. We would expect that they would be just as focused when they initiate the FSGS and Alport syndrome programs. We can't speak for them. But what I can say is what we've seen thus far has been very impressive. Chris?

喬，謝謝你的提問。我會快速解答最後一個問題，然後把問題交給克里斯來回答有關費用的問題。時間線不變。我們對 Renalys 的工作速度和品質印象深刻，並且對 Chugai Pharmaceuticals 也抱有很高的敬意。我們期望他們在啟動 FSGS 和 Alport 綜合徵專案時也能同樣專注。我們無法代表他們發言。但我可以說，到目前為止我們所看到的景象非常令人印象深刻。克里斯？

Joe, on the R&D front for operating expenses, we're in the midst of the budgeting process now. So I'll be able to come back with a little bit more clarity on that post 4Q. But you are right that we do expect to have additional investments for pegtibatinase as that clinical operation really ramps up once we restart. And we're looking at investments there to have that be the fastest enrollment and time line to top line data while maintaining quality that we can.

喬，關於營運費用的研發方面，我們目前正在進行預算編制工作。所以，我會在第四季結束後，就此問題給予更清晰的解釋。但您說得對，隨著我們重啟臨床運作並真正擴大規模，我們預計會為培替巴汀酶追加投資。我們正在考慮在這方面進行投資，以期在保證品質的前提下，實現最快的招生速度和最快獲得主要數據的時間線。

For sparsentan, there are -- as you might imagine, with DUPLEX and PROTECT, we do see a ramp down in activity in that. But there are also other evidence generation efforts that could potentially be helpful both in IgA nephropathy, but then also in FSGS pending approval here where we believe we can help generate even more value.

對於稀疏的，正如你可能想像的那樣，隨著 DUPLEX 和 PROTECT 的出現，我們確實看到其活動逐漸減少。但還有其他證據產生工作可能對 IgA 腎病有所幫助，在 FSGS 獲得批准之前，我們相信我們可以幫助創造更大的價值。

The last thing I'll highlight with FILSPARI that's still going to be an investment is going to be the transplant studies that recently kicked off and are in the recruiting phase now. So there are still investments that we need to make on the R&D front. But to your point or question around the context of the revenue, we expect revenue to continue to grow very nicely and be able to support our efforts here.

最後，我要重點介紹 FILSPARI 的一項投資，那就是最近啟動的移植研究，目前正處於招募階段。因此，我們仍需要在研發方面進行投資。但就您提出的收入方面的問題而言，我們預計收入將繼續保持良好的成長勢頭，並能夠支持我們在這方面的努力。

Thank you.

謝謝。

Ladies and gentlemen, this concludes the question-and-answer session of today's conference call. I'll hand the call back over to Nivi.

女士們、先生們，今天的電話會議問答環節到此結束。我會把電話轉回給妮維。

Thank you, everyone, for joining today's call. Have a great rest of your day.

感謝各位參加今天的電話會議。祝您今天餘下的時間過得愉快。

This concludes today's conference call. Thank you for participating. You may now disconnect.

今天的電話會議到此結束。感謝您的參與。您現在可以斷開連線了。