Travere Therapeutics Inc (TVTX) 2025 Q2 法說會逐字稿

Good afternoon and welcome to the Travere Therapeutics second-quarter 2025 financial results conference call. Today's call is being recorded.

下午好，歡迎參加 Travere Therapeutics 2025 年第二季財務業績電話會議。今天的通話正在錄音。

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

現在，我想將會議交給企業傳播和投資者關係副總裁 Nivi Nehra。請繼續，Nivi。

Thank you, operator. Good afternoon and welcome to Travere Therapeutics' second-quarter 2025 financial results and corporate update call. Thank you all for joining. Today's call will be led by Dr. Eric Dube, our President and Chief Executive Officer. Eric will be 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.

謝謝您，接線生。下午好，歡迎參加 Travere Therapeutics 2025 年第二季財務業績和公司最新情況電話會議。感謝大家的加入。今天的電話會議將由我們的總裁兼執行長 Eric Dube 博士主持。我們的首席醫療官 Jula Inrig 博士、我們的首席商務官 Peter Heerma 和我們的財務長 Chris Cline 將與 Eric 一起發表準備好的演講。我們的首席研究長 Bill Rote 博士將加入我們的問答環節。

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 provision 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 statements. Please see the forward-looking statement disclaimer in the company's press release issued earlier today as well as the risk factors section in our Form 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, August 6, 2025, and Travere specifically disclaims any obligation to update such statements to reflect future information, events, or circumstances.

在我們開始之前，我想提醒大家，本次電話會議中關於非歷史事實的陳述屬於 1995 年《私人證券訴訟改革法案》安全港條款範圍內的前瞻性陳述。前瞻性陳述並不保證業績。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、表現和成就與陳述中表達或暗示的結果、表現和成就有重大差異。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 和 10-K 表中的風險因素部分。此外，任何前瞻性陳述僅代表我們截至該陳述作出之日（2025 年 8 月 6 日）的觀點，Travere 明確否認有義務更新此類陳述以反映未來的資訊、事件或情況。

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

說完這些，現在讓我把電話轉給 Eric。

Thank you, Nivi. Good afternoon and thank you all for joining us. The second quarter of 2025 was a standout for Travere, a reflection of strong strategic execution and continued momentum in delivering against our mission. At the heart of this progress is FILSPARI, where we are advancing our leadership in rare kidney disease by deepening our impact in IgA nephropathy and laying the foundation for potential expansion into FSGS.

謝謝你，Nivi。下午好，感謝大家的參與。2025 年第二季對於 Travere 來說是一個突出的成績，體現了強大的策略執行力和持續完成使命的勢頭。這項進展的核心是 FILSPARI，我們透過深化在 IgA 腎病變領域的影響力，鞏固我們在罕見腎臟病領域的領導地位，並為潛在擴展到 FSGS 領域奠定基礎。

In IgAN, we delivered our strongest commercial quarter to date with continued demand driven by both new and repeat prescribers. As the IgAN treatment landscape evolves, physicians are increasingly adopting FILSPARI as foundational care, recognizing the meaningful and consistent outcomes it delivers and doing so earlier in the disease process. This growing confidence reflects our ongoing strategy to establish FILSPARI as the new foundational therapy for IgA nephropathy. There are several pillars to this strategy that we expect to be further solidified through year-end.

在 IgAN 領域，我們實現了迄今為止最強勁的商業季度，新處方者和重複處方者的需求持續成長。隨著 IgAN 治療領域的不斷發展，醫生越來越多地採用 FILSPARI 作為基礎治療，認識到它帶來的有意義且一致的結果，並在疾病過程的早期就開始這樣做。這種日益增長的信心反映了我們持續的策略，即將 FILSPARI 確立為 IgA 腎病的新基礎療法。我們預計該策略有幾個支柱將在年底前進一步鞏固。

First, the continued generation of robust clinical evidence to support the use of FILSPARI across a broad range of patients and in combination with other classes of medicines. Second, broad access aligned with the full approval indication statement, payer coverage, and an expected modification of the liver-monitoring REMS and removal of the pregnancy testing REMS. And finally, the continued real-world clinical experience and growing recommendation by nephrologists and treatment guidelines that recognize FILSPARI’s ability to reduce proteinuria and reinforce the benefits of getting patients to complete remission.

首先，不斷產生強有力的臨床證據來支持在廣泛的患者中使用 FILSPARI 並與其他類別的藥物合併使用。其次，廣泛的使用範圍與完整的批准適應症聲明、付款人覆蓋範圍以及對肝臟監測 REMS 的預期修改和對妊娠測試 REMS 的取消相一致。最後，持續的真實世界臨床經驗和腎病專家越來越多的推薦以及治療指南都認可了 FILSPARI 降低蛋白尿的能力並強化了使患者完全緩解的益處。

I am pleased with the tremendous progress that our team has made to date, which sets us up for sustained growth in an expanding IgAN market. Our performance in Q2 reflects the strength of this positioning and the growing use of FILSPARI as a foundational nephroprotective treatment in IgAN, even as new treatments enter the market. While still early days in the evolving IgAN treatment landscape, FILSPARI’s performance is directly aligned with the expectations that we have set over the last few years and validation of its path to foundational use. Jula and Peter will review the progress of our strategy in greater detail.

我很高興看到我們的團隊迄今為止的巨大進步，這為我們在不斷擴大的 IgAN 市場中持續成長奠定了基礎。我們在第二季度的表現反映了這一定位的實力，以及 FILSPARI 作為 IgAN 基礎腎臟保護治療的日益廣泛的應用，即使新的治療方法進入市場。儘管在不斷發展的 IgAN 治療領域中仍處於早期階段，但 FILSPARI 的表現與我們過去幾年設定的期望以及其基礎應用路徑的驗證直接一致。Jula 和 Peter 將更詳細地回顧我們策略的進展。

We've also seen strong progress outside of the US by our partners CSL Vifor and Renalys as they look to expand access to FILSPARI internationally. We've entered the second half of the year from a position of strength, and we're ready to build on this momentum. The sNDA review process for potential second indication for FILSPARI in FSGS is advancing as expected. We're preparing for what could be an extraordinary milestone for Travere and the FSGS community. If approved, FILSPARI would become the first-ever treatment approved for this underserved patient community, a breakthrough for patients and a near- and long-term growth opportunity for the company given that there are no other FSGS therapies likely to be available in the near future.

我們也看到我們的合作夥伴 CSL Vifor 和 Renalys 在美國以外取得了長足的進步，他們希望擴大 FILSPARI 在國際上的覆蓋範圍。我們以強勁的勢頭進入了下半年，並準備繼續保持這一勢頭。FILSPARI 在 FSGS 中的潛在第二種適應症的 sNDA 審查流程正在按預期推進。我們正在為特拉維爾和 FSGS 社區的一個非凡里程碑做準備。如果獲得批准，FILSPARI 將成為首個獲批用於此醫療資源匱乏患者群體的治療方法，這對患者來說是一個突破，鑑於近期可能沒有其他 FSGS 療法可用，這對公司來說也是一個近期和長期的成長機會。

Beyond FILSPARI, we continue to advance our pipeline with a drive to transform paradigms in rare diseases like classical homocystinuria, and we remain on track to reinitiate enrollment in the HARMONY study for pegtibatinase next year. With a robust and growing foundation in IgAN, the potential to be the first approved medicine in FSGS, and a pipeline built for continued innovation, we remain confident in the road ahead.

除了 FILSPARI 之外，我們還將繼續推進我們的產品線，致力於改變經典型高胱氨酸尿症等罕見疾病的治療模式，並且我們仍有望在明年重新啟動聚乙二醇化巴汀酶的 HARMONY 研究的招募工作。憑藉在 IgAN 領域強大且不斷發展的基礎、成為 FSGS 領域首個獲批藥物的潛力以及為持續創新而建立的管道，我們對未來的道路充滿信心。

I will now hand the call over to Jula for an update on our clinical programs. Jula?

我現在將把電話交給 Jula，請她介紹我們的臨床計畫的最新進展。朱拉？

Thank you, Eric. As a nephrologist, it's incredibly rewarding to witness the continued innovation in IgA nephropathy, a disease where not long ago, patients had very few options. We are expanding the body of evidence that supports the foundational role of FILSPARI across a broad spectrum of IgA nephropathy patients at risk of disease progression, ranging from those who are newly diagnosed to those with recurrent disease post transplant.

謝謝你，埃里克。作為腎臟病專家，見證 IgA 腎病治療領域的不斷創新是令人欣慰的，不久前，患者對這種疾病的選擇還很少。我們正在擴大證據範圍，以支持 FILSPARI 在廣泛的有疾病進展風險的 IgA 腎病患者中發揮基礎性作用，這些患者包括新診斷的患者和移植後復發的患者。

FILSPARI’s unique ability to target two pathways causing kidney injury, endothelin-1 and angiotensin II, provides patients with IgA nephropathy a non-immunosuppressive treatment option to help preserve kidney function. We were pleased to engage with the medical and patient community at several recent congresses, where we presented important new data that continue to reinforce the nephroprotective effects, strong safety profile, and emerging disease-modifying potential of FILSPARI.

FILSPARI 的獨特能力在於針對導致腎損傷的兩種途徑——內皮素-1 和血管緊張素 II，為 IgA 腎病患者提供了一種非免疫抑制治療選擇，以幫助保護腎功能。我們很高興在最近的幾次會議上與醫療和患者社區進行了交流，我們在會議上展示了重要的新數據，這些數據繼續強化了 FILSPARI 的腎臟保護作用、強大的安全性以及新興的疾病改良潛力。

Select data at these congresses include: in the Phase 2 SPARTAN trial, when used first-line in treatment-naive IgA nephropathy patients, FILSPARI-treated patients achieved an approximately 70% proteinuria reduction, with nearly 60% of patients in the study reaching complete proteinuria remission and stable eGFR through 24 weeks. Also, in the SPARTAN study, an analysis of patient samples showed that FILSPARI-treated patients with IgA nephropathy demonstrated rapid and sustained reductions in urinary B-cell activating factor or BAFF and complement factor C5b-9, as well as reductions in pro-inflammatory and profibrotic biomarkers.

這些會議上精選的數據包括：在 2 期 SPARTAN 試驗中，當對初治 IgA 腎病患者進行一線治療時，接受 FILSPARI 治療的患者蛋白尿減少了約 70%，研究中近 60% 的患者在 24 週內達到蛋白尿完全緩解和 eGFR 穩定。此外，在 SPARTAN 研究中，對患者樣本的分析表明，接受 FILSPARI 治療的 IgA 腎病患者的尿液 B 細胞活化因子或 BAFF 和補體因子 C5b-9 迅速且持續減少，促炎和促纖維化生物標誌物也減少。

When taken in combination with our preclinical data showing FILSPARI protects from mesangial deposition of IgA, these data suggest a potential role of optimal dual inhibition of endothelin-1 and angiotensin II as modifying the underlying disease.

結合我們的臨床前數據，表明 FILSPARI 可防止系膜細胞 IgA 沉積，這些數據表明內皮素-1 和血管緊張素 II 的最佳雙重抑制具有改變潛在疾病的潛在作用。

In the Phase 3 PROTECT open-label extension that is ongoing, patients transitioning from maximally titrated irbesartan to FILSPARI after the double-blind period also achieved approximately 50% reductions in proteinuria and a relatively stable eGFR out to one year. Together with the kidney function preservation FILSPARI provides, these new data further reinforce FILSPARI’s disease-modifying potential in IgA nephropathy without immunosuppression.

在正在進行的 III 期 PROTECT 開放標籤擴展研究中，雙盲期後從最大滴定劑量的厄貝沙坦過渡到 FILSPARI 的患者也實現了蛋白尿減少約 50%，並且在一年內實現了相對穩定的 eGFR。結合 FILSPARI 提供的腎功能保護，這些新數據進一步證實了 FILSPARI 在無需免疫抑制的情況下治療 IgA 腎病的疾病改良潛力。

As the IgAN treatment paradigm evolves, we expect combination therapy to become the new standard to help more patients reach complete proteinuria remission, which is recognized as a treatment goal in the draft update to the KDIGO guidelines. FILSPARI remains uniquely positioned as the only medicine to replace RAAS inhibitors as a more effective foundational therapy in IgA nephropathy, with a profile that is complementary to the emerging therapeutic classes.

隨著 IgAN 治療模式的發展，我們預計聯合治療將成為新的標準，幫助更多患者達到完全蛋白尿緩解，這在 KDIGO 指南更新草案中被確認為治療目標。FILSPARI 仍具有獨特的優勢，是唯一能夠取代 RAAS 抑制劑，成為 IgA 腎病變更有效的基礎療法的藥物，其特性與新興治療類別相輔相成。

We're also approaching the August 28 PDUFA date for the removal of the embryo-fetal toxicity REMS and the potential modification of the liver monitoring REMS to quarterly. With no cases of high (inaudible) to date, we remain confident in the safety profile of FILSPARI to support this change.

我們也即將在 8 月 28 日的 PDUFA 日期之前取消胚胎-胎兒毒性 REMS，並可能將肝臟監測 REMS 修改為每季一次。到目前為止，還沒有出現高（聽不清楚）病例，我們仍然對 FILSPARI 的安全性充滿信心，相信其能夠支持這項改變。

Turning to FSGS, our sNDA seeking full approval of FILSPARI in FSGS was accepted by the FDA, with a PDUFA date of January 13, 2026. As a reminder, the work by the independent PARASOL Group established proteinuria as a valid surrogate endpoint for kidney failure in FSGS and helped pave the way for regulatory consideration of proteinuria reduction for full approval. The sNDA review process is advancing as expected, and we're preparing for an advisory committee to discuss FILSPARI as the first potential approved medicine for FSGS.

談到 FSGS，我們尋求 FILSPARI 在 FSGS 治療中全面批准的 sNDA 已獲得 FDA 接受，PDUFA 日期為 2026 年 1 月 13 日。需要提醒的是，獨立 PARASOL 小組的工作將蛋白尿確立為 FSGS 腎衰竭的有效替代終點，並為監管機構考慮全面批准減少蛋白尿鋪平了道路。sNDA 審查流程正在按預期推進，我們正在準備成立一個諮詢委員會來討論 FILSPARI 作為首個可能獲批的 FSGS 藥物。

Our team is actively preparing to present the strong data from DUPLEX and DUET in the context of the independent PARASOL findings. For example, earlier this quarter, we presented new analysis from the DUPLEX study that confirmed the PARASOL findings. In DUPLEX, significantly more FILSPARI-treated patients achieved either partial or complete remission compared to irbesartan. Importantly, those patients who achieved partial or complete proteinuria remission in the study, irrespective of treatment arm, had a 67% to 77% lower risk of kidney failure respectively. These data represent the first trial-level evidence in support of the independent PARASOL analysis of proteinuria as a validated surrogate endpoint in FSGS.

我們的團隊正在積極準備在獨立的 PARASOL 研究結果的背景下展示來自 DUPLEX 和 DUET 的強大數據。例如，本季度早些時候，我們展示了 DUPLEX 研究的新分析，證實了 PARASOL 的研究結果。在 DUPLEX 中，與接受厄貝沙坦治療的患者相比，接受 FILSPARI 治療的患者中實現部分或完全緩解的患者明顯增多。重要的是，在研究中，那些達到蛋白尿部分或完全緩解的患者，無論治療組如何，腎衰竭的風險分別降低了 67% 至 77%。這些數據代表了支持獨立 PARASOL 分析將蛋白尿作為 FSGS 中經過驗證的替代終點的第一個試驗級證據。

Lastly, on our HCU program, we continue to be excited about the potential of our investigational enzyme replacement therapy, pegtibatinase. We have made strong progress on our manufacturing scale-up to support our Phase 3 trial and a future commercial launch and are on track towards restarting patient enrollment in the Phase 3 HARMONY study next year.

最後，在我們的 HCU 計畫中，我們繼續對正在研究的酵素替代療法聚乙二醇丁醚酶的潛力感到興奮。我們在擴大生產規模方面取得了長足進展，以支持我們的 3 期試驗和未來的商業發布，並有望於明年重新開始 3 期 HARMONY 研究的患者招募。

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

我現在將把電話轉給彼得，詢問商業更新情況。彼得？

Thank you, Jula. I'm pleased to report that our commercial team executed exceptionally well in Q2 as demonstrated by our strong performance metrics. FILSPARI net product sales reached approximately $72 million in the US in the second quarter, representing significant year-over-year growth. This performance was driven by strong demand, expansion and deepening of prescriber base, as well as further efficiencies in our fulfillment process, and full therapy compliance and persistence.

謝謝你，朱拉。我很高興地報告，我們的商業團隊在第二季表現非常出色，這可以從我們強大的績效指標中看出。FILSPARI 淨產品銷售額第二季在美國達到約 7,200 萬美元，較去年同期成長顯著。這項業績的推動因素包括強勁的需求、處方者基礎的擴大和深化、履行流程的進一步效率以及全面的治療依從性和持久性。

As the only fully approved non-immunosuppressive kidney-targeted therapy for IgA nephropathy, FILSPARI remains uniquely positioned to replace the historical standards of care in this evolving treatment landscape. The growing recognition of FILSPARI’s nephroprotective profile, not just from returning prescribers, but also from nephrologists who are new to the brand, resulted in 745 new patient forms this quarter, approximately a 43% increase compared to the same period last year.

作為唯一獲得全面批准的非免疫抑制性腎臟標靶治療 IgA 腎病的藥物，FILSPARI 在這一不斷發展的治療領域中仍具有獨特的優勢，可以取代歷史治療標準。FILSPARI 的腎臟保護特性得到了越來越多的認可，不僅來自回頭客，也來自新接觸該品牌的腎病專家，導致本季度新患者人數達到 745 人，與去年同期相比增長了約 43%。

At a time when new treatment options are emerging for IgA nephropathy, feedback from our field teams and market research emphasize physician confidence in FILSPARI’s established position in clinical practice. Nephrologists regularly cite FILSPARI’s consistent, rapid, and sustained proteinuria efficacy with long-term kidney preservation benefits, a safety profile that allows for chronic use, and a patient-friendly once-daily oral administration that optimally inhibits two critical kidney-damaging pathways as key reasons for making FILSPARI their foundational therapeutic option.

在 IgA 腎病治療新方案不斷湧現的時代，我們現場團隊和市場研究的回饋強調了醫生對 FILSPARI 在臨床實踐中既定地位的信心。腎臟病專家經常引用 FILSPARI 的一致、快速和持續的蛋白尿療效，以及長期腎臟保護益處、允許長期使用的安全性，以及患者友好的每日一次口服給藥，可最佳地抑制兩種關鍵的腎臟損害途徑，這些都是將 FILSPARI 作為其基礎治療選擇的關鍵原因。

Importantly, many physicians are choosing FILSPARI earlier in the treatment journey. This reflects a growing emphasis on earlier intervention and a shift away from legacy approaches, such as the off-label use of RAAS inhibitors or steroids. Based on our research, about 70% of the addressable patients with IgA nephropathy have elevated proteinuria levels below 1.5 gram per gram. With the proteinuria threshold being removed and the FILSPARI label at full approval, FILSPARI is particularly well positioned in this large patient segment, allowing for sustainable demand potential.

重要的是，許多醫生在治療早期就選擇了 FILSPARI。這反映出人們越來越重視早期幹預，並逐漸擺脫傳統方法，例如 RAAS 抑制劑或類固醇的標籤外使用。根據我們的研究，約70%的可治療的IgA腎病患者的蛋白尿水平升高至每克1.5克以下。隨著蛋白尿閾值的取消和 FILSPARI 標籤的全面批准，FILSPARI 在這一龐大的患者群體中佔據了特別有利的地位，具有可持續的需求潛力。

As we look at the remainder of the year, as with any rare disease product, we may see some variability quarter over quarter, but we expect to continue identifying new prescribers and that the use of FILSPARI will further deepen as foundational care among current prescribers. We believe this will be driven by continuous positive experience, consistent with FILSPARI’s superior clinical profile relative to historical standards of care, and also driven by the anticipated final KDIGO guideline publication and the potential REMS modifications, which may further simplify access for patients.

展望今年剩餘時間，與任何罕見疾病產品一樣，我們可能會看到季度間出現一些變化，但我們預計將繼續發現新的處方者，並且 FILSPARI 作為現有處方者的基礎護理的使用將進一步深化。我們相信，這將由持續的積極經驗推動，與 FILSPARI 相對於歷史護理標準的卓越臨床特徵相一致，同時也由預期的最終 KDIGO 指南出版物和潛在的 REMS 修改推動，這可能會進一步簡化患者的就醫途徑。

Turning to FSGS. If approved, FILSPARI would become the first approved therapy for FSGS, a progressive and often debilitating rare kidney disease with significant unmet needs. Given the feedback from nephrologists and the broader FSGS community, we believe FSGS could be an even bigger opportunity with a more rapid uptake versus what we have experienced with our launch in IgA nephropathy. We are actively expanding and preparing our commercial organization to be ready to serve this patient community, beginning with a potential approval early next year.

轉向 FSGS。如果獲得批准，FILSPARI 將成為首個核准的 FSGS 治療藥物，FSGS 是一種進行性且常常使人衰弱的罕見腎臟疾病，存在大量未滿足的醫療需求。考慮到腎臟病專家和更廣泛的 FSGS 社群的回饋，我們相信 FSGS 可能是一個更大的機會，與我們在 IgA 腎臟病領域推出產品時所經歷的相比，FSGS 的吸收速度更快。我們正在積極擴大和準備我們的商業組織，以便為這個患者群體提供服務，從明年年初可能獲得批准開始。

In summary, Q2 was another quarter of strong execution and meaningful growth for FILSPARI amidst the evolving IgA and FSGS treatment landscape. We remain confident in our strategy and encouraged by the feedback from the medical community and the continued momentum in US adoption. With a growing body of clinical and real-world evidence, expanding prescriber engagement, and the potential to serve more rare kidney disease patients through future label expansions, FILSPARI is well positioned to continue its leadership as foundational treatment.

總而言之，在不斷變化的 IgA 和 FSGS 治療環境中，第二季是 FILSPARI 又一個執行力強勁、成長有意義的季度。我們對我們的策略仍然充滿信心，並受到醫學界的反饋和美國持續採用勢頭的鼓舞。隨著臨床和現實世界證據的不斷增加、處方者參與度的不斷提高以及透過未來標籤擴展為更多罕見腎病患者提供服務的潛力，FILSPARI 完全有能力繼續保持其作為基礎治療的領導地位。

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

現在，讓我將電話轉給克里斯，請他報告財務更新。克里斯？

Thank you, Peter, and good afternoon all. Our financial foundation continues to strengthen. In the second quarter, net product sales grew approximately 82% over the same period last year, and we continue to make disciplined investments in areas of high growth such as in building further momentum for FILSPARI in IgA nephropathy, preparing our organization for a potential launch of FILSPARI in FSGS, and advancing manufacturing to restart enrollment in the pivotal pegtibatinase study.

謝謝你，彼得，大家下午好。我們的財務基礎不斷加強。第二季度，淨產品銷售額比去年同期成長了約 82%，我們繼續在高成長領域進行嚴格的投資，例如為 FILSPARI 在 IgA 腎病領域的發展創造進一步的勢頭，為我們組織在 FSGS 中推出 FILSPARI 做好準備，並推進製造以重新啟動關鍵的聚乙二醇巴汀酶研究的招募。

Beginning with revenue, in the second quarter we generated US net product sales of $94.8 million. As Peter highlighted, FILSPARI continued to grow significantly in the second quarter, generating $71.9 million in US net product sales. Thiola and Thiola EC contributed $23 million in net product sales for the second quarter, and these medicines continue to be a meaningful option for patients living with cystinuria. But, as we've highlighted previously, we anticipate more generic competition in the coming quarters.

從收入開始，第二季我們在美國實現了 9,480 萬美元的淨產品銷售額。正如彼得所強調的，FILSPARI 在第二季度繼續大幅成長，在美國實現了 7,190 萬美元的淨產品銷售額。Thiola 和 Thiola EC 為第二季度貢獻了 2,300 萬美元的淨產品銷售額，這些藥物繼續成為胱胺酸尿症患者有意義的選擇。但是，正如我們之前強調的那樣，我們預計未來幾季將會有更多的仿製藥競爭。

During the second quarter, we also recognized $19.6 million of license and collaboration revenue, resulting in total revenue of $114.4 million. Included in the license and collaboration revenue line this quarter is the previously announced one-time $17.5 million milestone payment from CSL Vifor, which resulted from the conversion of conditional approval of FILSPARI to full approval in Europe earlier this year.

第二季度，我們也實現了 1,960 萬美元的許可和合作收入，總收入達到 1.144 億美元。本季度的許可和合作收入包括先前宣布的 CSL Vifor 的 1750 萬美元一次性里程碑付款，這筆款項源於今年早些時候歐洲對 FILSPARI 的有條件批准轉為完全批准。

Turning to operating expenses, our research and development expenses for the second quarter of 2025 were $49.4 million compared to $54.3 million for the same period in 2024. The decrease in R&D is largely attributable to reduced clinical activity in the Phase 3 HARMONY study while we optimize our manufacturing efforts. On a non-GAAP adjusted basis, R&D expenses were $45.4 million compared to $50.6 million for the same period in 2024.

談到營運費用，我們 2025 年第二季的研發費用為 4,940 萬美元，而 2024 年同期為 5,430 萬美元。研發費用的減少主要歸因於我們優化製造工作的同時，第三階段 HARMONY 研究中臨床活動的減少。以非公認會計準則調整後，研發費用為 4,540 萬美元，而 2024 年同期為 5,060 萬美元。

Selling, general, and administrative expenses for the second quarter were $76.2 million compared to $64.8 million for the same period in 2024. The increase in SG&A is largely attributable to increased amortization expense related to FILSPARI royalties, as well as increased investment to support the ongoing launch in IgA nephropathy following full approval and preparing for a potential FSGS launch in January. On a non-GAAP adjusted basis, SG&A expenses were $55.5 million compared to $48.3 million for the same period in 2024.

第二季的銷售、一般和行政費用為 7,620 萬美元，而 2024 年同期為 6,480 萬美元。銷售、一般及行政費用的增加主要歸因於與 FILSPARI 特許權使用費相關的攤銷費用增加，以及在獲得全面批准後為支持 IgA 腎病的持續推出而增加的投資，以及為 1 月份潛在的 FSGS 推出做準備。依非公認會計準則調整後，銷售、一般及行政費用為 5,550 萬美元，而 2024 年同期為 4,830 萬美元。

Total other expense net for the second quarter of 2025 was immaterial compared to net expense of $1.9 million in the same period in 2024. Net loss for the second quarter of 2025 was $12.8 million or $0.14 per basic share, compared to $70.4 million or $0.91 per basic share for the same period in 2024. On a non-GAAP adjusted basis, net income for the second quarter of 2025 was $11.9 million or $0.13 per basic share, compared to a net loss of $50.1 million or $0.65 per basic share for the same period in 2024.

2025 年第二季的其他支出淨總額與 2024 年同期的 190 萬美元淨支出相比微不足道。2025 年第二季淨虧損為 1,280 萬美元，即每股基本虧損 0.14 美元，而 2024 年同期淨虧損為 7,040 萬美元，即每股基本虧損 0.91 美元。以非公認會計準則調整後，2025 年第二季淨收入為 1,190 萬美元，即每股基本收益 0.13 美元，而 2024 年同期淨虧損為 5,010 萬美元，即每股基本收益 0.65 美元。

As of June 30, 2025, we had cash, cash equivalents, and marketable securities totaling approximately $319.5 million. As I highlighted earlier, this cash balance reflects the net proceeds of the $17.5 million payment we received from CSL Vifor during the quarter, and we remain on track to potentially achieve additional milestone payments tied to key market access achievements later this year and sales-based achievements in the future, which should further enhance our financial flexibility.

截至 2025 年 6 月 30 日，我們的現金、現金等價物及有價證券總額約為 3.195 億美元。正如我之前強調的那樣，該現金餘額反映了我們在本季度從 CSL Vifor 收到的 1750 萬美元付款的淨收益，並且我們仍有望在今年晚些時候實現與關鍵市場准入成就相關的額外里程碑付款以及未來基於銷售的成就，這將進一步增強我們的財務靈活性。

Looking ahead, we expect continued revenue growth driven by robust underlying demand for FILSPARI in IgA nephropathy. With a strong balance sheet and potential additional incoming milestone payments as well as a disciplined approach to investing in our key growth drivers, we are well positioned to execute on our strategy and deliver sustainable value for both the near and long term.

展望未來，我們預計，由於 IgA 腎病領域對 FILSPARI 的強勁潛在需求，收入將持續成長。憑藉強勁的資產負債表和潛在的額外里程碑付款，以及對關鍵成長動力進行投資的嚴謹方法，我們完全有能力執行我們的策略並在短期和長期內創造可持續的價值。

I now turn the call back over to Eric for his closing comments. Eric?

現在我將電話轉回給 Eric，請他作最後發言。艾瑞克？

Thank you, Chris. As you've heard, our team has delivered strong performance across our priorities for the first half of 2025. We are well positioned and eager to continue this momentum. My team and I are deeply motivated to support the rare disease communities we serve. Recently, I had the honor of attending the annual SPARK patient meeting of the IgA Nephropathy Foundation. At the meeting, I heard many stories of resilience and hope. This community has been advocating and hoping for a future with therapies developed for them that can stave off kidney failure. We are proud to be part of this journey, and we are committed to the same goal for the FSGS and HCU communities.

謝謝你，克里斯。正如您所聽到的，我們的團隊在 2025 年上半年的各項重點工作中都表現出色。我們已做好準備並渴望延續這股勢頭。我和我的團隊非常積極地支持我們所服務的罕見疾病社區。最近，我有幸參加了IgA腎病基金會的年度SPARK患者會議。在會議上，我聽到了許多關於堅韌和希望的故事。這個社區一直在倡導並希望未來能夠開發出可以預防腎衰竭的治療方法。我們很自豪能夠成為這趟旅程的一部分，並且我們致力於為 FSGS 和 HCU 社群實現同一個目標。

Now, let me turn the call over to Nivi for Q&A. Nivi?

現在，讓我將電話轉給 Nivi 進行問答。尼維？

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

謝謝你，埃里克。接線員，我們現在可以開通問答熱線了。

(Operator Instructions)

（操作員指示）

Joseph Schwartz, Leerink Partners.

Leerink Partners 的 Joseph Schwartz。

This is Will on for Joe. Thanks for taking our questions, and congrats on the strong progress this quarter. So one for FSGS, as you prepare for the AdCom, what do you anticipate the major topics could be, and how do you plan to respond to the FDA’s inquiries here? Do you have a sense for who might be on the panel itself? And given this group of experts will likely include nephrologists and cardiologists, how do you plan to effectively message your case to these two different groups of physicians?

我是威爾，請喬代為提問。感謝您回答我們的問題，並祝賀您本季的強勁進展。那麼對於 FSGS，在您為 AdCom 做準備時，您預計主要議題是什麼，以及您計劃如何回應 FDA 的詢問？您是否知道小組成員可能是誰？鑑於這群專家可能包括腎臟科醫師和心臟科醫師，您打算如何有效地將您的病例傳達給這兩組不同的醫師？

Good point. Anytime a rare disease goes before an advisory committee, there's certainly an educational component that we need to do about the disease and the pathophysiology. Importantly, there's both nephrologist and cardiologist part of the panel, and they all know that proteinuria is harmful, and they know the importance of blocking both the RAAS system and endothelin-1 to reduce cardiovascular and renal outcomes. So we certainly are prepared to educate the mix of panelists, I think, importantly about the biologic plausibility supporting proteinuria as a validated endpoint why eGFR is challenging in FSGS and then followed by the strong clinical data showing sparsentan meaningfully and significantly reduce proteinuria compared to an active comparator.

說得好。每當一種罕見疾病提交諮詢委員會時，我們肯定需要對該疾病和病理生理學進行教育。重要的是，小組中有腎臟病專家和心臟病專家，他們都知道蛋白尿是有害的，並且他們知道阻斷 RAAS 系統和內皮素-1 對減少心血管和腎臟後果的重要性。因此，我認為，我們當然準備向小組成員進行重要教育，讓他們了解支持蛋白尿作為驗證終點的生物學合理性，為什麼 eGFR 在 FSGS 中具有挑戰性，然後是強有力的臨床數據，表明與活性對照藥相比，Sparsentan 可以顯著降低蛋白尿。

Anupam Rama, JPMorgan.

摩根大通的 Anupam Rama。

Just wondering if you could comment a little bit on the cadence and level of engagement you've had with the agency heading into the REMS update for FILSPARI, just given the evolving landscape and what we've seen from a regulatory perspective in other cases in the sector.

我只是想知道，考慮到不斷變化的情況以及我們從監管角度看到的該行業其他案例的情況，您是否可以稍微評論一下您與該機構在 FILSPARI 的 REMS 更新方面的合作節奏和程度。

Certainly, we've been monitoring the overall landscape with regard to the FDA. Bill, why don't you comment on what we've seen with our progress.

當然，我們一直在關注 FDA 的整體情況。比爾，為什麼不評論一下我們所看到的進展呢？

Yeah, certainly. What we've seen is basically through the lens of two sNDAs, both with the sNDA for the modification of the REMS frequency and the removal of the embryo-fetal toxicity REMS as well as the sNDA for FSGS. Our interactions have been progressing as we'd expect, and the back-and-forth has been very similar to what we experienced just last year with the NDA for full approval in IgA nephropathy. The frequency of interaction and the types of questions, it feels just the same. So we certainly see, on our part, a very engaged and active review team.

是的，當然。我們所看到的基本上是透過兩個 sNDA 的視角，這兩個 sNDA 都用於修改 REMS 頻率和消除胚胎-胎兒毒性 REMS，以及用於 FSGS 的 sNDA。我們的互動正如我們預期的那樣進展順利，並且反覆的過程與我們去年在 IgA 腎病的 NDA 全面批准過程中經歷的非常相似。互動的頻率，提問的類型，感覺都是一樣的。因此，我們當然看到，我們擁有一支非常投入且積極的審查團隊。

Laura Chico, Wedbush Securities.

勞拉‧奇科 (Laura Chico)，韋德布希證券公司。

Another regulatory question if I might be able to. I'm curious at what point would you gain insight into the timing of the advisory committee panel meeting? It sounded from the prior comments that the interactions are occurring on a regular cadence, as would be anticipated, but we detected another possible shift in CDER headcount. So just trying to understand, is there any expected timeline that you would receive notice for the advisory committee meeting?

如果我可以的話，還有另一個監管問題。我很好奇您什麼時候會了解諮詢委員會小組會議的時間表？從先前的評論來看，這些互動正在以預期的方式定期發生，但我們發現 CDER 員工人數可能發生了另一種變化。所以只是想了解一下，您是否會收到諮詢委員會會議通知的預計時間表？

Bill, why don't you take that one?

比爾，你為什麼不拿那個？

Certainly. We don't know at this stage what the date of the advisory committee will be, but once we do know it, we will certainly provide that update. Given that we have a PDUFA date of January 13, it's reasonable to anticipate that the advisory committee should be taking place sometime in Q4.

當然。目前我們還不知道諮詢委員會的會議日期，但一旦知道，我們一定會提供最新消息。鑑於 PDUFA 日期為 1 月 13 日，因此可以合理地預期諮詢委員會應該在第四季度的某個時候召開會議。

Tyler Van Buren, TD Securities.

泰勒·範布倫，道明證券。

Hi, this is Frances on for Tyler. Congratulations on a really amazing quarter. So just wondering, what would it take after the REMS PDUFA to get the REMS potentially removed entirely, and on what timeline would you expect that to be?

大家好，我是法蘭西斯，為泰勒服務。恭喜您取得如此出色的季度業績。所以我只是想知道，在 REMS PDUFA 之後需要做什麼才能完全刪除 REMS，以及您預計這會在什麼時間表上實現？

Okay. Bill, another one for you.

好的。比爾，再給你一個。

Sure. Our strategy has always been to have the ultimate removal of the REMS. And per our prior interactions with the agency, we've always approached this as a two-step process, seeking first to lessen the testing frequency and then full removal as a second step. Historically, the FDA has been anchored on completing our PMR following 3,000 patients for two years. And we're going to continue the dialogue to evaluate opportunities to potentially remove the REMS ahead of that following the PDUFA date on the 28th of this month.

當然。我們的策略一直是最終消除 REMS。根據我們之前與該機構的互動，我們一直將此視為一個兩步驟流程，首先尋求減少測試頻率，然後第二步完全取消。從歷史上看，FDA 一直致力於在兩年內追蹤 3,000 名患者並完成 PMR。我們將繼續對話，評估在本月 28 日 PDUFA 日期之前取消 REMS 的可能性。

Vamil Divan, Guggenheim Securities.

瓦米爾·迪萬，古根漢證券公司。

Hi, this Arseniy on for Vamil. Congrats on a great quarter. You presented new data from the SPARTAN study at recent conferences. How have these studies been received by the broad community, and do you anticipate further real-world evidence or biomarker data to support FILSPARI’s positioning as a disease-modifying therapy?

大家好，我是 Arseniy，代表 Vamil。恭喜本季取得優異成績。您在最近的會議上展示了來自 SPARTAN 研究的新數據。社會各界對這些研究的接受度如何？您是否預計會有進一步的真實世界證據或生物標記數據來支持 FILSPARI 作為疾病修飾療法的定位？

Great. Jula, why don't you comment on what your team has been hearing when the data have been presented?

偉大的。Jula，當數據呈現時，為什麼不評論一下你的團隊聽到了什麼？

Yeah. It has been received very positively. There's a lot of excitement about the data that we're generating, both, as you mentioned, in combination with SGLT2 inhibitors that it's safe, and we have good efficacy when used in combination. And then to your point around the SPARTAN study, we've been increasingly showing data showing the effect of FILSPARI on reducing disease-modifying biomarkers such as the soluble CD163, which we released last year and then, as I mentioned on the call, additional data reducing inflammation, B-cell activation, and complement activation. And so people are now starting to think that FILSPARI works directly within the kidney on reducing the inflammation in the kidney, and that is part of the role for why it’s nephroprotective over the long term.

是的。它受到了非常積極的評價。我們產生的數據令人非常興奮，正如您所說，與 SGLT2 抑制劑結合使用是安全的，並且聯合使用具有良好的療效。然後關於您提到的 SPARTAN 研究，我們越來越多地展示數據，表明 FILSPARI 對減少疾病修飾生物標記（例如我們去年發布的可溶性 CD163）的效果，然後，正如我在電話中提到的，還有減少發炎、B 細胞活化和補體活化的額外數據。因此，人們現在開始認為 FILSPARI 直接作用於腎臟，減少腎臟炎症，這也​​是它長期保護腎臟的作用部分原因。

And then your second part was additional data. We certainly want to validate this. We have a large Phase 3 study from which we have biomarkers that we can confirm and validate this, and we plan to present that data in future congresses.

然後你的第二部分是附加資料。我們當然想驗證這一點。我們有一個大型的第三階段研究，其中有生物標記可以確認和驗證這一點，我們計劃在未來的會議上展示這些數據。

Maury Raycroft, Jefferies.

莫里‧雷克羅夫特 (Maury Raycroft)，傑富瑞 (Jefferies)。

Hi, this is [Ferdinand] for Maury. Congrats on the solid Q2 number. Is there more perspective you can provide on the dynamics and breakdown of the growth contributions from stocking, new patients, and persistence rates? How could this look for the rest of the year?

大家好，我是 Maury 的 [Ferdinand]。恭喜您第二季業績穩健。您能否提供更多關於庫存、新患者和持續率對成長貢獻的動態和細分的觀點？今年剩下的時間狀況會如何？

Peter, I'll turn that one to you.

彼得，我會把這個交給你。

Yeah. Indeed very strong performance both on the demand side as well as the revenue side. I think your question is mainly referring to the revenue side. We didn’t see stocking. This was really like performance-driven growth.

是的。需求方面和收入方面的表現確實非常強勁。我認為你的問題主要是指收入方面。我們沒有看到長襪。這確實像是績效驅動的成長。

And then on the persistence rate and the new patients versus the carryover patients from last quarters?

那麼持續率和新患者與上個季度的結轉患者相比如何呢？

Yeah, so the persistence rate continues to be very high. There has been no change in what we've seen. And as Peter mentioned in his prepared comments, really all the fundamentals that we look at, particularly lead indicators of performance moving forward for the rest of the year, all remain very strong. So it really is the underlying performance. And as you would imagine, over time, much of the volume becomes continuing patients, and growth from adding new patients is precisely the dynamic that we're seeing, and we expect that to be sustainable for the foreseeable future.

是的，所以持久率仍然很高。我們所看到的並沒有任何改變。正如彼得在他的準備好的評論中提到的那樣，我們所關注的所有基本面，特別是今年剩餘時間內業績的領先指標，都仍然非常強勁。所以這確實是潛在的表現。正如您所想的，隨著時間的推移，大部分患者都會成為持續的患者，而增加新患者的成長正是我們所看到的動態，我們預計這種成長在可預見的未來將是可持續的。

Yigal Nochomovitz, Citigroup.

花旗集團的 Yigal Nochomovitz。

I had a question on FSGS. As you may know, there was a paper published in October 2024 called Proteinuria as an Endpoint in Clinical Trials for FSGS, and they make a very specific point in there with respect to the reference to a, quote, sustained CR. And I'm just wondering if you could comment as to your understanding of what that means and whether it's associated with any particular time point or not.

我對 FSGS 有疑問。您可能知道，2024 年 10 月發表了一篇名為《蛋白尿作為 FSGS 臨床試驗的終點》的論文，其中就持續 CR 的參考提出了非常具體的觀點。我只是想知道您是否可以評論一下您對這意味著什麼的理解以及它是否與任何特定的時間點相關。

I know that in the study, you obviously had a 2.5 times better CR response rate versus irbesartan in the double-blind period. I'm just wondering if that is consistent with the definition of sustained CR, or if sustained CR refers to a specific point in time as opposed to over the entire course of the trial.

我知道在研究中，在雙盲期內，您的 CR 反應率顯然比厄貝沙坦高出 2.5 倍。我只是想知道這是否與持續 CR 的定義一致，或者持續 CR 是指特定時間點而不是整個試驗過程。

Jula, I'll turn over to you.

朱拉，我把麥克風交給你了。

I was part of that manuscript, so happy to answer it. So complete remission can occur at one point in time or it can be durable. And there's not a -- we didn’t define exactly what that meant, but I can tell you when we look at the DUPLEX data, and you mentioned the 2.5 times greater rate of complete remission, what we commented at least within the DUPLEX manuscript in the New England Journal is that 85% of those patients stayed below their baseline rates of proteinuria so had some level of durability of complete remission response.

我參與了該手稿的撰寫，很高興能回答這個問題。因此，完全緩解可以在某個時間點發生，也可以持續很長時間。我們沒有明確定義這意味著什麼，但我可以告訴你，當我們查看 DUPLEX 數據時，你提到了 2.5 倍的完全緩解率，我們至少在《新英格蘭雜誌》的 DUPLEX 手稿中評論說，85% 的患者蛋白尿發生率低於基線水平，因此完全緩解反應具有一定程度的持久性。

We did look at it at any point in time during our DUPLEX trial data. We haven’t specifically elucidated others other than what we have published to date. If there's additional questions around durability, there's lots of ways in which you can define that. Our focus is on what the FDA wants, and so that’s what we’ve given to them. And any questions that they have around additional ways in which we can analyze the data, we can certainly respond to them around that.

我們確實在 DUPLEX 試驗數據期間的任何時間點都查看過它。除了迄今為止已發表的內容外，我們還沒有具體闡明其他內容。如果對耐用性還有其他疑問，那麼有很多方法可以定義它。我們關注的是 FDA 想要什麼，所以我們就把這些給了他們。如果他們對我們分析數據的其他方法有任何疑問，我們當然可以回答他們。

Maybe just two additional things I’ll add, Yigal. Sorry, Yigal. Anything you wanted to clarify before I add something?

也許我只想補充兩件事，伊加爾。抱歉，伊加爾。在我添加內容之前您想澄清什麼嗎？

No, I was just saying that was a super helpful answer.

不，我只是說這是一個非常有用的答案。

That's great. And while this isn’t specific to complete remission, if you look at our corporate deck, slide 31, you’ll see that the patients that were able to get to FPRE, which was the pre-specified endpoint within our trial, that is sustained whether you look at 9 months or 2 years. And the treatment effect continues to be consistent and significant. So I think, in the ways that we’ve looked at it, as Jula talked about it, you have a reduction in proteinuria that is rapid, that is sustained, and that is consistent.

那太棒了。雖然這並不特定於完全緩解，但如果您查看我們的公司簡報第 31 張投影片，您會看到能夠達到 FPRE（這是我們試驗中預先指定的終點）的患者，無論您觀察 9 個月還是 2 年，這種狀態都能持續下去。且治療效果持續穩定、顯著。因此我認為，正如 Jula 所說的那樣，從我們的角度來看，蛋白尿的減少是快速的、持續的、一致的。

The other thing that I’ll point out about that publication which was really informative, it occurred before PARASOL. And what's important is to make sure that we recognize that the analyses and conclusions from PARASOL are the largest -- they reflect the largest analyses of registry data within FSGS. So it's certainly consistent and informative, but it's really important to keep in mind that the thresholds that we put forth in PARASOL represent a very robust dataset.

關於該出版物，我要指出的另一件事是，它確實很有資訊量，它出現在 PARASOL 之前。重要的是確保我們認識到 PARASOL 的分析和結論是最大的——它們反映了 FSGS 內註冊資料的最大分析。因此，它肯定是一致的和資訊豐富的，但真正重要的是要記住，我們在 PARASOL 中提出的閾值代表了一個非常強大的數據集。

Mohit Bansal, Wells Fargo.

富國銀行的 Mohit Bansal。

Congrats on the progress. I would like to understand, I mean, if you have a good cadence of patient start forms here, can you comment on what is the conversion rate of those forms to the number of patients that are on the drug? And how would you characterize where are you in terms of the penetration in this market? How do you see it evolving now with more competition, but at the same time, more awareness of that competition?

恭喜你取得進展。我想了解，我的意思是，如果您這裡有良好的患者開始表格節奏，您能否評論一下這些表格與服用該藥物的患者數量的轉換率是多少？您如何描述您在該市場的滲透率？隨著競爭日益激烈，但同時人們對競爭的認識也日益加深，您如何看待它的發展？

Okay. Great. I think we’ve got three questions. So Peter, why don’t you take all of those: conversion rate, penetration rate, and then how you think that will evolve with the market?

好的。偉大的。我想我們有三個問題。那麼彼得，為什麼不把這些都考慮進去呢：轉換率、滲透率，然後你認為這些會如何隨著市場而發展？

Let me start with the last part on the evolution of the market and how we see it. I think the market, we have spoken about in the past, especially after our full approval, we anticipate that the market potential is about 70,000 addressable patients for FILSPARI in the US. And about 70% of those patients have elevated proteinuria levels below 1.5 grams per grams. So if you talk about penetration, it really depends on what segment are you talking about. Are you talking about the segment 1.5 or greater, or are you talking about the full market potential.

讓我從最後一部分開始，談談市場的發展以及我們如何看待它。我認為，我們過去曾談到市場，特別是在我們獲得全面批准之後，我們預計 FILSPARI 在美國的市場潛力約為 70,000 名可尋址患者。其中約 70% 的患者蛋白尿量升高至每克 1.5 克以下。因此，如果您談論滲透率，這實際上取決於您所談論的部分。您談論的是 1.5 或更大的細分市場，還是整個市場潛力。

And after our full approval and (inaudible) proteinuria threshold in our label, we now have access to the broader market. And I think that's really where the opportunity reside and will continue to reside as well. In that context, I’m really happy with the progress that we are making that we are seeing also. And I mentioned that in the last earnings call as well, the median proteinuria level by patient start form is moving to the left, and what I mean with that it's now below 1.5 and continues to trend in that same direction. So I think we are entering the market segment where most of the potential is, and that allows for sustainable growth as well.

在我們獲得全面批准並且（聽不清楚）我們的標籤中的蛋白尿閾值之後，我們現在可以進入更廣泛的市場。我認為這確實是一個機會所在，而且機會還將繼續存在。從這個角度來看，我對我們所取得的進展感到非常高興。我在上次財報電話會議上也提到過，患者開始時的蛋白尿中位數水平正在向左移動，我的意思是現在它低於 1.5 並且繼續朝著同一方向發展。因此我認為我們正在進入最具潛力的市場領域，這也有利於實現永續成長。

So that's how we see the market evolving as well as the penetration component. With regards to conversion, I think based on my experience in rare disease as well as what I've seen from rare disease benchmarks, I would say that we are at the top-end best practice on conversion, as you would expect in rare disease.

這就是我們所看到的市場發展以及滲透部分。關於轉化，我認為根據我在罕見疾病方面的經驗以及我從罕見疾病基準中看到的情況，我想說我們在轉化方面處於最佳的頂級實踐，正如你在罕見疾病中所期望的那樣。

And Mohit, one thing that I’ll add, just if you look at the cumulative PSFs from initial approval, we still have less than 10% of the overall addressable population. Now, we've had very strong performance, and this is one of the strongest rare kidney disease launch uptakes over the last five years, but that penetration rate reflects that we have significant room to grow. And this is where we talked about the sustainable growth we expect, it’s because we’ve been successful, and it represents a relatively small penetration rate to date.

Mohit，我要補充一點，如果你看一下從最初批准開始累積的 PSF，你會發現我們可涵蓋的總人口仍然不到 10%。現在，我們的業績非常強勁，這是過去五年來罕見腎臟病領域最強勁的上市項目之一，但這一滲透率反映出我們還有很大的成長空間。這就是我們談論的我們期望的可持續增長，這是因為我們已經取得了成功，並且迄今為止滲透率相對較小。

Got it. And then if I could ask a follow-up. I mean, from the patient start point of view, given that there is another player on the market, how should we think about the patient start form cadence going forward given that you have competition here? So that's the part we'd love to understand your thoughts and expectations there.

知道了。然後我是否可以問一個後續問題。我的意思是，從患者開始的角度來看，考慮到市場上還有另一個參與者，考慮到這裡有競爭，我們應該如何考慮患者開始形成的節奏？因此，我們希望了解您的想法和期望。

Yeah, but we’ve not provided guidance, so I just want to be cautious of not doing that. But I will reflect on what Peter has talked about in the last two quarters, which is that the new baseline for new patient start forms is around 700. We clearly achieved, exceeded that in this first quarter of having a direct competitor and growing treatment options within the space. We expect this opportunity to continue.

是的，但我們沒有提供指導，所以我只是想謹慎不要這樣做。但我會反思彼得在過去兩個季度談到的內容，即新患者開始表格的新基準約為 700。我們在第一季明顯取得了成就，甚至超越了直接競爭對手，並且在該領域增加了治療選擇。我們期望這個機會能夠持續下去。

And Peter can certainly talk more about the dynamics that we expect with a new treatment option within the endothelin class. Peter?

當然，彼得可以多談談我們對內皮素類新治療方案的預期動態。彼得？

Yeah, just to put an exclamation point on some of the points you made. I think, Mohit, within the evolving treatment paradigm, we delivered our strongest quarter to date, both from a demand perspective as well as from a revenue perspective. Even though it's early days since we saw some of those new entrants coming to the market, everything we have seen so far is consistent with our expectations.

是的，只是想對你提出的一些觀點加一個感嘆號。莫希特，我認為，在不斷發展的治療模式下，無論從需求角度還是從收入角度來看，我們都創造了迄今為止最強勁的季度業績。儘管我們看到一些新進入者進入市場還為時過早，但迄今為止我們所看到的一切都符合我們的預期。

And new classes and new therapies coming to the market, one, further reinforces the urgency to treat patients earlier and more aggressively. And, two, it also grows the endothelin market. But I think the market is big enough. To Eric's earlier point, all the products will be used, but given the strong profile of FILSPARI and the recognized profile in the long-term data that we have, I’m confident that we will remain the market leader.

而新類別和新療法的上市，進一步強調了對患者進行更早、更積極治療的迫切性。其次，它也促進了內皮素市場的成長。但我認為市場夠大。正如 Eric 之前所說，所有產品都會被使用，但鑑於 FILSPARI 的強大形像以及我們擁有的長期數據中公認的形象，我相信我們將繼續保持市場領先地位。

Prakhar Agrawal, Cantor.

普拉卡爾‧阿格拉瓦爾 (Prakhar Agrawal)，領唱。

Congrats on the quarter. So another on FSGS. In the Phase 3, I know you have disclosed eGFR at the end of the trial, but we’re not seeing the eGFR curves in FSGS. So maybe qualitatively, could you comment on what to expect when the curves will be presented during the AdCom and the latest thinking on what the FDA would like to see on eGFR here?

恭喜本季取得佳績。另一個是關於 FSGS 的。在第 3 階段，我知道您在試驗結束時揭露了 eGFR，但我們沒有看到 FSGS 中的 eGFR 曲線。因此，也許從定性角度來看，您能否評論一下在 AdCom 期間展示曲線時的預期情況，以及 FDA 希望在這裡看到的 eGFR 的最新想法？

And secondly, on IgAN, if you could comment on the gross-to-net trends you saw in 2Q and expectations for the rest of the year?

其次，關於 IgAN，您能否評論一下第二季的毛利率與淨利率趨勢以及今年剩餘時間的預期？

Okay. Jula, why don't you take these questions on EGFR. And Chris, you can take the gross-to-net question.

好的。Jula，為什麼不回答這些關於 EGFR 的問題呢？克里斯，你可以回答總額與淨額的問題。

Certainly. With the PARASOL analysis and recommendation and the move to focus on proteinuria as the endpoint for full approval in FSGS, we haven’t spent much time publishing our eGFR data, and I don’t anticipate that to be a large focus of our advisory committee. Of course, we certainly can show the curves. It's consistent with what you would anticipate. We had a washout of RAAS inhibitors for two weeks, and then we initiated the two therapies, sparsentan and irbesartan.

當然。由於 PARASOL 的分析和建議以及將蛋白尿作為 FSGS 完全批准終點的舉措，我們並沒有花太多時間發布我們的 eGFR 數據，而且我預計這不會成為我們諮詢委員會關注的重點。當然，我們當然可以展示曲線。這與您的預期一致。我們停用 RAAS 抑制劑兩週，然後開始使用 sparsentan 和 irbesartan 兩種療法。

We had an acute decline in eGFR, and then it's relatively thereafter. And you have some decline as you can see by the absolute change over time, but nothing surprising. If we show our eGFR curve, it just hasn't been our focus because we're moving to proteinuria which is a better surrogate with less variability for predicting avoidance of kidney failure. So that has been our focus.

我們的 eGFR 急劇下降，之後相對下降。從隨時間推移的絕對變化可以看出，確實出現了一些下降，但這並不奇怪。如果我們展示 eGFR 曲線，那麼它就不再是我們的重點，因為我們正在轉向蛋白尿，它是預測避免腎衰竭的更好的替代指標，且變異性更小。這就是我們的重點。

(multiple speakers) The FDA has not requested eGFR as part of our Type C meeting. So again, it's just not reflective of what we've interpreted from PARASOL, but specifically, the area of focus from our Type C meeting that we've discussed previously. Sorry, Chris, go ahead.

（多位發言者）FDA 並未要求將 eGFR 作為我們 C 類會議的一部分。所以，這不僅反映了我們對 PARASOL 的解讀，也具體反映了我們之前討論過的 C 類會議的重點領域。抱歉，克里斯，請繼續。

Thanks for the question on gross-to-net. And as you would expect, we did see some relief this quarter relative to last quarter once we got through the typical beginning-of-the-year dynamic. And as we look ahead, we may see some incremental increases in discounts in 3Q, 4Q, but we’re squarely in line with where we've guided for the year being in the low 20%. So everything is shaping up as expected.

感謝您提出有關毛利與淨利之比的問題。正如您所預料的，一旦我們度過了典型的年初動態，本季的情況相對於上一季確實有所緩解。展望未來，我們可能會看到第三季和第四季的折扣有所增加，但與我們對今年的指導水準（20% 以下）完全一致。一切都如預期的順利。

Jason Zemansky, Bank of America.

美國銀行的傑森·澤曼斯基。

Congrats on the progress. Maybe at this point in the launch, do you have a greater sense of where the headwinds or bottlenecks are in terms of uptakes, whether it’s on the prescriber side or patient, payer, logistical, or administrative? Just trying to get a sense of what some of the potential near-term levers are.

恭喜你取得進展。也許在發布的這個階段，您是否對吸收方面的阻力或瓶頸有了更深入的了解，無論是在處方方還是患者、付款人、物流還是行政方面？只是想了解近期的一些潛在槓桿是什麼。

And then maybe just as a quick follow-up to one of your earlier comments regarding these new segments of the market, but is there a meaningful or measurable difference in the use of drug between these new patients who may be a little bit healthier or not as advanced?

然後也許只是對您之前關於這些新市場部分的評論的快速跟進，但是這些可能更健康或不那麼先進的新患者在使用藥物方面是否存在有意義或可衡量的差異？

Peter, why don't you take both of those?

彼得，你為什麼不把這兩個都拿走？

With regard to your first question on potential bottlenecks, I think -- I’m really pleased with the progress that we’re making. But when I think about like our launch in the last 2.5 years, I think the main issue we had to overcome was the urgency to treat and change treatment for those patients. And what really will help is more treatment options coming to the market together with the KDIGO guideline to reinforce that same methods that patients should be recognized and treated more aggressively early on. It will help to further develop this market. So I think this is really the market in development.

關於您提出的第一個有關潛在瓶頸的問題，我認為——我對我們取得的進展感到非常高興。但當我回想起我們在過去兩年半的研發歷程時，我認為我們必須克服的主要問題是對這些患者進行治療和改變治療方法的緊迫性。真正有幫助的是更多的治療方案與 KDIGO 指南一起進入市場，以強化相同的方法，即應儘早識別患者並更積極地治療。這將有助於進一步開發這個市場。所以我認為這確實是一個正在發展的市場。

On your second question with regards to like the new patient segment like the lower proteinuria, still elevated proteinuria levels but lower than 1.5, I think this is a segment where I really see an opportunity for us with the REMS modification. And that is more in line with the clinical practice of three monthly monitoring of lab values. The higher proteinuria level patients like 1.5 or greater, it is not uncommon that I actually do monthly lab testing. But when you talk about patients at the lower proteinuria levels, it is more common that they are seen every three months by the nephrologists. And as part of the routine monitoring, they do the measurements.

關於您的第二個問題，關於像蛋白尿較低的新患者群體，蛋白尿水平仍然升高但低於 1.5，我認為對於這個群體來說，我真正看到了 REMS 修改給我們帶來的機會。這更符合每三個月監測一次實驗室值的臨床實務。對於蛋白尿水平較高的患者，例如 1.5 或更高，我實際上每月進行一次實驗室測試並不罕見。但是對於蛋白尿水平較低的患者來說，更常見的情況是每三個月去看一次腎臟科醫生。作為例行監測的一部分，他們會進行測量。

So I think our REMS modification is right on time where we are in the launch, so that's why I see the opportunity moving forward.

因此，我認為我們的 REMS 修改恰逢其時，因此我看到了向前發展的機會。

Greg Harrison, Scotiabank.

加拿大豐業銀行的格雷格·哈里森。

Hi everyone, thank you for taking our question and congrats on the quarter. Regarding patient start forms this quarter, could you comment on the distribution as the quarter progressed? Did you see any lumpiness or acceleration, and how should we think about that going forward?

大家好，感謝您回答我們的問題，並祝賀本季取得的成績。關於本季的患者開始表格，您能否評論一下本季進展中的分佈？您是否看到了任何不平衡或加速，我們應該如何看待未來的發展？

Thanks for the question. As I mentioned earlier, I’m really pleased with the strong performance this quarter. As is typical in rare disease, there’s often variability month to month, but overall, what we saw was consistent throughout the quarter—and that continued into July as well.

謝謝你的提問。正如我之前提到的，我對本季的強勁表現感到非常滿意。正如罕見疾病的典型情況一樣，每個月的情況通常都會有所不同，但總體而言，我們看到的情況在整個季度都是一致的，而且這種情況也持續到了 7 月。

Greg Harrison, Scotiabank.

加拿大豐業銀行的格雷格·哈里森。

Congratulations on the quarter. This is Joe Thomas on for Greg. Just taking on the patient start forms in the quarter, I wonder if you might be able to comment on the distribution as the quarter went along. Did you see any lumpiness or any acceleration as the quarter was going and how should we think about that going forward?

恭喜本季。這是喬·托馬斯 (Joe Thomas) 代替格雷格 (Greg)。僅就本季的患者開始表格而言，我想知道您是否可以對本季的分佈發表評論。您是否看到本季出現任何波動或加速，我們應該如何看待未來的發展？

Peter, why don't you take that one?

彼得，你為什麼不拿那個？

As I mentioned already, I'm really pleased with the strong performance this quarter. As is typical in rare disease, there is often variability month over month, but overall, what we have seen is consistent demand during the quarter, and that continued in July as well.

正如我已經提到的，我對本季的強勁表現感到非常滿意。正如罕見疾病的典型情況一樣，每個月的情況通常都會有所不同，但總體而言，我們看到本季的需求是一致的，7 月也保持了這種狀態。

Alex Thompson, Stifel.

亞歷克斯湯普森，Stifel。

Maybe shifting over to Europe, to the extent to which you have visibility on CSL launch, when should we expect to see meaningful royalty revenue ex-US?

也許轉移到歐洲，就您對 CSL 發布的了解程度而言，我們什麼時候可以在美國以外看到有意義的特許權使用費收入？

We do want to defer to CSL Vifor to comment on their performance. Chris, I'll let you discuss a bit more on expectations for royalty.

我們確實想聽聽 CSL Vifor 對他們表現的評論。克里斯，我讓你進一步討論一下對皇室的期望。

Yeah, happy to do that. As Eric mentioned, we’ll defer to CSL Vifor on any kind of guidance. But as you would imagine, they're now through the early stages of going through the country-by-country process. And as that happens and they gain reimbursement, we would expect revenues to begin picking up overseas and in return royalties coming through to us, so more to come as CSL Vifor continues to navigate that and report. And as soon as we have more that we can share, we’ll be happy to do so.

是的，很高興這麼做。正如 Eric 所提到的，我們將聽從 CSL Vifor 的任何指導。但正如您所想像的，他們現在正處於逐國審查進程的早期階段。隨著這種情況的發生以及他們獲得報銷，我們預計海外收入將開始回升，而版稅也將轉交給我們，因此隨著 CSL Vifor 繼續探索和報告，未來還會有更多收入。一旦我們有更多可以分享的訊息，我們就會很樂意這樣做。

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

謝謝。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 Nivi。

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

感謝大家參加今天的電話會議。祝您今天剩餘時間愉快。

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。