Akebia Therapeutics Inc (AKBA) 2023 Q2 法說會逐字稿

Good day, and welcome to Akebia's Second Quarter 2023 Financial Results Call. (Operator Instructions) As a reminder, this call is being recorded. I would now like to introduce your host for today, Mercedes Carrasco, Senior Director of Corporate Communications and Investor Relations. You may begin.

美好的一天，歡迎參加 Akebia 2023 年第二季財務業績電話會議。 （操作員說明）謹此提醒，此通話正在錄音。現在我想介紹今天的主持人，梅賽德斯·卡拉斯科，企業傳播和投資者關係高級總監。你可以開始了。

Thank you, and welcome to Akebia's Second Quarter 2023 Financial Results and Business Update Conference Call. Please note that a press release was issued earlier today, Friday, August 25, detailing our second quarter financial results, and that release is available on the Investors section of our website. For your convenience, a replay of today's call will also be available on our website after we conclude.

謝謝，歡迎參加 Akebia 2023 年第二季財務表現與業務更新電話會議。請注意，今天早些時候（即 8 月 25 日星期五）發布了一份新聞稿，詳細介紹了我們第二季度的財務業績，該新聞稿可在我們網站的投資者部分查看。為了您的方便，今天電話會議結束後，我們還將在我們的網站上提供今天電話會議的重播。

Joining me for today's call, we have John Butler, Chief Executive Officer; Dr. Steve Burke, Chief Medical Officer; and Ellen Snow, Chief Financial Officer.

與我一起參加今天的電話會議的有執行長約翰·巴特勒 (John Butler)； Steve Burke 博士，首席醫療官；和財務長艾倫·斯諾。

I'd like to remind everyone that this call includes forward-looking statements. Each forward-looking statement on this call is subject to risks and uncertainties that could cause actual results to differ materially from those described in these statements. Additionally, additional information describing these risks is included in the financial results press release that we issued on August 25 as well as in the Risk Factors and Management Discussion and Analysis section of our most recent annual and quarterly reports filed with the SEC. The forward-looking statements on this call speak only as to the original date of this call, and except as required by law, we do not undertake any obligation to update or revise any of these statements.

我想提醒大家，這次電話會議包含前瞻性陳述。本次電話會議中的每項前瞻性聲明都存在風險和不確定性，可能導致實際結果與這些聲明中所述的結果有重大差異。此外，描述這些風險的更多資訊包含在我們 8 月 25 日發布的財務業績新聞稿中，以及我們向 SEC 提交的最新年度和季度報告的風險因素和管理層討論與分析部分中。本次電話會議的前瞻性陳述僅涉及本次電話會議的原始日期，除非法律要求，否則我們不承擔更新或修改任何這些陳述的義務。

As described in the press release, we intend to file an amendment to our 2022 annual report on Form 10-K to reflect corrections to our previously issued financial statements due to recently identified accounting errors related to the recording and reporting of return reserves for Auryxia as well as our second quarter Form 10-Q.

如新聞稿所述，我們打算對10-K 表格2022 年年度報告進行修訂，以反映由於最近發現的與Auryxia 收益準備金記錄和報告相關的會計錯誤而對我們之前發布的財務報表進行的更正：以及我們第二季的 10-Q 表格。

Please note that comments made during this call regarding the company's financials reflect revised financial statements for the years ended December 31, 2022, 2021 and 2020 and the first quarter of 2023. As outlined in the press release and to be described in the amendment to the 2022 Form 10-K and the second quarter Form 10-Q. With that, I'd like to introduce our CEO, John Butler.

請注意，本次電話會議期間發表的有關公司財務狀況的評論反映了截至 2022 年 12 月 31 日、2021 年和 2020 年以及 2023 年第一季度的修訂後財務報表。2022 年 10-K 表格和第二季10-Q 表格。接下來，我想介紹一下我們的執行長約翰·巴特勒。

Thanks, Mercedes, and thanks, everyone, for joining us. It's a pleasure to be here with you today. Although it's a little later than we expected to be together, the extra time spent was necessary. We're very pleased with the revisions to our financial statements that we had to work through are not material, and they don't impact the value of the company. You'll hear from Ellen shortly, but I want to thank her and her team for the work done over the past several weeks that helped to ensure we are best positioned to maximize value as we approach several important catalysts.

謝謝梅賽德斯，謝謝大家加入我們。很高興今天能和大家在一起。雖然比我們預計的見面時間晚了一些，但多花的時間也是必要的。我們對財務報表的修改感到非常滿意，我們必須完成的修改並不重要，而且不會影響公司的價值。您很快就會收到艾倫的來信，但我要感謝她和她的團隊在過去幾週所做的工作，這些工作有助於確保我們在接近幾個重要催化劑時處於最佳位置，以實現價值最大化。

First, let's focus on the operational progress that we've made over the past few months, working through towards the potential approval of our second product for patients with kidney disease, vadadustat. We are now on a clear path to potentially gain U.S. approval for vadadustat as a treatment for anemia due to chronic kidney disease or CKD in adult patients on dialysis. In fact, as we shared earlier, we've received the minutes from our recent end of dispute Type A meeting with the FDA, which reflect our productive discussion with the agency and support our optimism that we will resubmit our new drug application or NDA for vadadustat by the end of this quarter.

首先，讓我們專注於我們在過去幾個月中取得的營運進展，努力爭取我們的第二種用於腎臟病患者的產品 vadadustat 的潛在批准。我們現在正處於一條明確的道路上，有可能獲得美國批准 vadadustat 用於治療成年透析患者因慢性腎臟病或 CKD 引起的貧血。事實上，正如我們之前分享的，我們已經收到了最近與FDA 的A 類爭議結束會議的紀要，這反映了我們與FDA 的富有成效的討論，並支持我們的樂觀態度，即我們將重新提交我們的新藥申請或NDA vadadustat 將於本季末發布。

With that timing for resubmission, we would expect a decision from the FDA on the vadadustat NDA in March of 2024. We have Dr. Steve Burke here to answer any questions about our interactions with the FDA or our timeline for NDA resubmission at the end of the call. And I'll also discuss initial plans for an anticipated launch next year, if approved.

根據重新提交的時間，我們預計 FDA 將於 2024 年 3 月就 vadadustat NDA 做出決定。我們請 Steve Burke 博士在這裡回答有關我們與 FDA 的互動或我們在年底重新提交 NDA 的時間表的任何問題。的電話。如果獲得批准，我還將討論明年預計推出的初步計劃。

What's especially encouraging for our team at Akebia is that the momentum in the U.S. regulatory discussions follows tangible progress in markets around the globe. Vadadustat is approved in 34 countries. In the past quarter alone, the European Commission, the United Kingdom Medicines and Healthcare Products Regulatory Agency and Swiss Agency for Therapeutic products approved Vafseo, vadadustat, for the treatment of symptomatic anemia associated with CKD in adults on chronic maintenance dialysis in the EU, United Kingdom and Switzerland, respectively.

對我們 Akebia 團隊來說特別令人鼓舞的是，美國監管討論的勢頭緊跟著全球市場的實際進展。 Vadadustat 在 34 個國家獲得批准。僅在上個季度，歐盟委員會、英國藥品和保健品監管局和瑞士治療產品局就批准了 Vafseo（vadadustat）用於治療歐盟、美國慢性維持性透析成人 CKD 相關的症狀性貧血分別是王國和瑞士。

Further, Akebia expects a regulatory opinion on vadadustat in Australia this year. I want to credit our team who assumes responsibility for the regulatory processes outside the U.S. mid last year and worked efficiently to secure local approvals to help to enable Akebia to deliver a new therapeutic option to patients in need. To that end, we had previously discussed our priority was to secure a partner in Europe who would bring Vafseo to market. Here again, we met our objective and signed a license agreement with Medice.

此外，Akebia 預計今年澳洲將對 vadadustat 提出監管意見。我要讚揚我們的團隊，他們在去年年中承擔了美國境外監管流程的責任，並有效率地工作以獲得當地批准，幫助 Akebia 為有需要的患者提供新的治療選擇。為此，我們之前討論過我們的首要任務是在歐洲找到一個合作夥伴，將 Vafseo 推向市場。在這裡，我們再次實現了我們的目標，並與 Medice 簽署了許可協議。

Medice is a fully integrated pharmaceutical company based in Germany with a strategic focus on the dialysis market. The license agreement grants Medice the exclusive rights to market and sell Vafseo in the European economic area, the United Kingdom, Switzerland and Australia for the treatment of anemia in patients with CKD. The agreement included an upfront payment of $10 million. And in addition, we're eligible for commercial milestone payments up to an aggregate of $100 million and tiered royalty payments ranging from 10% to 30% of Medice's net sales.

Medice 是一家總部位於德國的綜合製藥公司，策略重點是透析市場。該授權協議授予 Medice 在歐洲經濟區、英國、瑞士和澳洲行銷和銷售 Vafseo 的獨家權利，用於治療 CKD 患者的貧血。該協議包括預付款 1000 萬美元。此外，我們還有資格獲得總計高達 1 億美元的商業里程碑付款以及 Medice 淨銷售額 10% 至 30% 的分級特許權使用費。

We believe Medice is situated to maximize the European market potential for vadadustat. They understand the unique country-by-country pricing and access dynamics related to dialysis. They are committed and driven to succeed, and we continue to support their efforts. We expect Medice to launch as soon as possible in 2024.

我們相信 Medice 能夠最大限度地發揮 vadadustat 的歐洲市場潛力。他們了解與透析相關的各國獨特的定價和獲取動態。他們致力於並努力取得成功，我們將繼續支持他們的努力。我們預計 Medice 將於 2024 年盡快推出。

Building on the regulatory success we've had in other markets, we're eager to advance the regulatory approval process for vadadustat in the U.S. An approval in the U.S. would represent our most significant commercial market opportunity and allow us to target the 550,000 U.S. patients with anemia due to CKD who are receiving dialysis treatment. I'll note that the resubmission is a very focused filing. As part of the resubmission, we'll submit safety data collected since our original submission in 2021, including data from two alternate dosing studies FO2CUS and MODIFY. We'll also submit post-marketing data from Japan, where tens of thousands of patients have received the drug over the past 2 years.

基於我們在其他市場取得的監管成功，我們渴望推進 vadadustat 在美國的監管審批流程。美國的批准將代表我們最重要的商業市場機會，並使我們能夠瞄準 550,000 名美國患者因CKD 導致貧血且正在接受透析治療的人。我要指出的是，重新提交的文件非常有針對性。作為重新提交的一部分，我們將提交自 2021 年最初提交以來收集的安全數據，包括兩項替代劑量研究 FO2CUS 和 MODIFY 的數據。我們也將提交來自日本的上市後數據，在過去的兩年裡，日本有數萬名患者接受了該藥物。

Now working through a potential launch timeline. Again, we expect to resubmit the NDA by the end of the quarter. Since we're submitting data that was not in the original NDA, our expectation is that our resubmission would undergo a 6-month review. And this review period would commence upon submission. As such, we anticipate a new PDUFA date likely in March of 2024. Upon a potential approval, we'll quickly file for TDAPA reimbursement, which is a 6-month process. After receiving TDAPA designation, we expect the product to be widely available for patients. We're actively preparing for a potential launch of vadadustat and are reengaging around the commercial opportunity.

現在正在研究可能的發佈時間表。同樣，我們預計將在本季結束時重新提交 NDA。由於我們提交的數據未包含在原始 NDA 中，因此我們預計重新提交的數據將接受為期 6 個月的審查。此審查期將從提交後開始。因此，我們預計新的 PDUFA 日期可能為 2024 年 3 月。一旦獲得批准，我們將快速申請 TDAPA 報銷，這是一個為期 6 個月的過程。在獲得 TDAPA 指定後，我們預計該產品將廣泛供患者使用。我們正在積極準備 vadadustat 的潛在上市，並圍繞商業機會重新展開合作。

Let me summarize. We estimate that approximately 88% of the nearly 550,000 patients on dialysis are treated with an erythropoiesis-stimulating agent or an ESA for anemia, an injectable that is currently the standard of care. Medications used to treat dialysis patients in the U.S. are paid for as part of a bundled payment made to dialysis providers.

讓我總結一下。我們估計，在近 55 萬名透析患者中​​，約有 88% 接受紅血球生成刺激劑或 ESA 治療貧血，這是目前標準治療的注射劑。在美國，用於治療透析患者的藥物是作為向透析提供者捆綁付款的一部分支付的。

Now to promote innovative drug use for Medicare patients on dialysis, CMS created a transitional add-on payment adjustment over TDAPA. The add-on payment would cover the cost of vadadustat when a physician prescribes the drug for 2 years after receiving the TDAPA designation on top of the regular bundled payment. The TDAPA payment would allow providers to incorporate innovative products like vadadustat into their treatment protocols, while still receiving the full bundled payment for treatment of their PPS or prospective payment system, Medicare patients.

現在，為了促進 Medicare 透析患者的創新藥物使用，CMS 針對 TDAPA 創建了過渡性附加付款調整。當醫生在獲得 TDAPA 指定後在常規捆綁付款之外開出該藥 2 年內的藥物時，附加付款將涵蓋 vadadustat 的費用。 TDAPA 付款將允許提供者將 vadadustat 等創新產品納入他們的治療方案中，同時仍收到為其 PPS 或未來支付系統、醫療保險患者進行治療的全額捆綁付款。

And we believe that we've created a favorable environment for adoption of the product as we prepare for a potential launch. We have a collaboration with Vifor CSL that provides access to up to 60% of the dialysis market through existing Vifor CSL relationships, which includes Fresenius Kidney Care and most small to midsized providers. Vifor CSL has a relationship with Fresenius for the procurement of therapeutic products used in their network, and vadadustat would be made available through that collaboration.

我們相信，在我們準備潛在的發佈時，我們已經為該產品的採用創造了有利的環境。我們與 Vifor CSL 合作，透過現有的 Vifor CSL 關係（其中包括 Fresenius Kidney Care 和大多數中小型提供者）進入高達 60% 的透析市場。 Vifor CSL 與 Fresenius 建立了採購其網路中使用的治療產品的關係，並且 vadadustat 將透過該合作提供。

We believe Vifor's unique relationship and our experience in the dialysis market will create a favorable environment for pull-through within the dialysis centers. Together, we believe we are well positioned for a successful launch if vadadustat is approved. Our collaboration with Vifor CSL is a profit share, where we retain approximately 2/3 of the profit from vadadustat, net of certain prespecified costs with Vifor CSL keeping the remaining 1/3.

我們相信 Vifor 獨特的關係和我們在透析市場的經驗將為透析中心內的拉動創造有利的環境。我們相信，如果 vadadustat 獲得批准，我們就能夠成功推出該產品。我們與 Vifor CSL 的合作是利潤分成，我們保留 vadadustat 約 2/3 的利潤，扣除某些預先指定的成本，Vifor CSL 保留剩餘的 1/3。

For the remaining 40% of the dialysis market that Vifor CSL does not have rights to sell to, we retain 100% of the economics. So one of the most common questions I get these days is on launch costs. I'll note that we are also really strong position here. The most significant launch expenses relates to people and products. But we have the commercial organization placed today with only incremental additions needed, and we already have the products on the shelf to launch as soon as we receive approval. We believe our additional costs will be truly incremental.

對於 Vifor CSL 無權出售的剩餘 40% 透析市場，我們保留 100% 的經濟利益。因此，這些天我收到的最常見問題之一是發射成本。我要指出的是，我們在這裡也擁有非常強大的地位。最重要的啟動費用與人員和產品有關。但我們今天已經建立了商業組織，只需要增量添加，並且我們已經將產品放在貨架上，一旦獲得批准即可推出。我們相信我們的額外成本將是真正增量的。

Now to speak more about our financial position, I'm pleased to introduce Ellen Snow, who joined our team as CFO and Treasurer last month. Ellen's impact has been near immediate as she brought vast accounting and financial management expertise into the process to close the quarter and put the organization on the right footing by strengthening our product return reserves accounting process that was described in the press release filed this morning. This financial discipline will be especially important as we prepare to launch vadadustat in the U.S. if approved. Ellen?

現在，為了更多地談論我們的財務狀況，我很高興地介紹 Ellen Snow，她上個月加入我們的團隊，擔任財務長和財務主管。艾倫的影響幾乎是立竿見影的，因為她將豐富的會計和財務管理專業知識帶入了本季度結束的流程中，並通過加強我們今天早上提交的新聞稿中描述的產品退貨儲備會計流程，使組織處於正確的基礎上。如果獲得批准，當我們準備在美國推出 vadadustat 時，這種財務紀律將特別重要。艾倫？

Thanks, John. First, let me say that I'm extremely pleased to have joined Akebia last month. I saw significant potential and upside for the organization associated with the pending potential vadadustat FDA approval as well as opportunities to optimize the company's cost structure and better leverage the cash flows from Auryxia towards improving the balance sheet and creating shareholder value. I'm grateful to have a talented, hard-working and dedicated accounting team who have worked tirelessly over the last few weeks as we identified and corrected errors in the accounting.

謝謝，約翰。首先，我很高興上個月加入 Akebia。我看到了該組織與即將獲得 FDA 批准的 vadadustat 相關的巨大潛力和優勢，以及優化公司成本結構和更好地利用 Auryxia 現金流來改善資產負債表和創造股東價值的機會。我很感激有一個才華橫溢、勤奮和敬業的會計團隊，他們在過去幾周里不知疲倦地工作，幫助我們發現並糾正了會計中的錯誤。

As previously announced during the second quarter close process, we identified errors related to the accruals for product returns. We concluded, the errors represent a material weakness in internal controls over financial reporting and deficiencies we are working to remediate. We also intend to revise our financial statements in our 2022 annual report on Form 10-K to correct the impact of the errors which will primarily impact the balance sheet and the affected years, including liabilities for product returns, accounts receivable and goodwill as well as immaterial changes to the prior year revenues. We expect to file our amended Form 10-K and second quarter Form 10-Q on Monday.

正如先前在第二季結帳流程中宣布的那樣，我們發現了與產品退貨應計費用相關的錯誤。我們的結論是，這些錯誤代表了財務報告內部控制的重大缺陷以及我們正在努力糾正的缺陷。我們還打算修改 2022 年 10-K 年度報告中的財務報表，以糾正錯誤的影響，這些錯誤將主要影響資產負債表和受影響的年份，包括產品退貨負債、應收帳款和商譽以及對上年度收入無重大變動。我們預計將於週一提交修訂後的 10-K 表格和第二季的 10-Q 表格。

Now let's move to our current financial position. Importantly, we believe our cash runway will fund our current operating plan for at least 12 months. Cash and cash equivalents as of June 30, 2023, were $53.6 million. Total revenue was $56.4 million for the second quarter of 2023. Of that, $42.2 million was Auryxia net product revenue, and $14.1 million was Other revenue. Other revenue consists primarily of the $10 million upfront payment in connection with the Medice license agreement.

現在讓我們看看我們目前的財務狀況。重要的是，我們相信我們的現金跑道將為我們目前的營運計劃提供至少 12 個月的資金。截至 2023 年 6 月 30 日，現金及現金等價物為 5,360 萬美元。 2023 年第二季的總收入為 5,640 萬美元。其中，Auryxia 淨產品收入為 4,220 萬美元，其他收入為 1,410 萬美元。其他收入主要包括與 Medice 授權協議相關的 1,000 萬美元預付款。

Auryxia net product revenue increased 21.6% over the previous quarter. The growth was due to the timing of purchases in certain Auryxia customers and expected cyclical demand from the first quarter to the second quarter. Auryxia revenue decreased 2.5% compared to the same quarter of 2022. The decrease compared to the prior year period is due in part to the impact of the shifting payer mix and a volume decrease partially caused by the contracting dynamics and phosphate binder market erosion.

Auryxia 淨產品營收較上一季成長 21.6%。這一成長是由於某些 Auryxia 客戶的採購時間以及第一季至第二季的預期週期性需求。與2022 年同季相比，Auryxia 收入下降了2.5%。與去年同期相比，收入下降的部分原因是付款人結構變化的影響，以及合約動態和磷酸鹽黏合劑市場侵蝕造成的銷量下降。

We expected quarterly swings in the Auryxia revenue but had also expected the binder market to rebound. Based on our analysis, we believe the binder market is leveling, but there's evidence of a long tail impact of COVID in the dialysis community.

我們預計 Auryxia 營收將出現季度波動，但也預期黏合劑市場將會反彈。根據我們的分析，我們認為黏合劑市場正在趨於平穩，但有證據顯示新冠疫情對透析產業產生長尾影響。

We are in a position to affirm our 2023 net product revenue guidance of $175 million to $180 million. Our team continues a high-touch engagement with key accounts, and we continue to carefully review the trends. We remain focused on maximizing Auryxia revenue through its loss of exclusivity in March of 2025.

我們可以確認 2023 年淨產品收入指引為 1.75 億至 1.8 億美元。我們的團隊繼續與重要客戶保持密切接觸，並繼續仔細審視趨勢。我們仍然專注於透過 2025 年 3 月失去獨家經營權來最大化 Auryxia 的收入。

Regarding expenses, we reported a decrease in cost of goods sold and R&D expenses compared to the second quarter of 2022. Cost of goods sold decreased slightly to $17.3 million in the second quarter of 2023 from $18.6 million in the second quarter of 2022. R&D expenses decreased from $26 million to $20.2 million, a more than 20% decrease as a result of reduced spending on vadadustat development, R&D headcount and outsourced contract services.

關於費用，我們報告與2022 年第二季相比，銷售商品成本和研發費用有所下降。銷售商品成本從2022 年第二季的1,860 萬美元小幅下降至2023 年第二季的1,730 萬美元。研發費用由於 vadadustat 開發、研發人員和外包合約服務支出減少，從 2,600 萬美元減少到 2,020 萬美元，降幅超過 20%。

We have curtailed non-headcount-related expenses growth by continuing to find ways of operating more efficiently, placing an increased scrutiny on all areas of operating expenses and taking measures such as reducing our office footprint with the assignment of the Seaport office lease, which was completed at the end of the second quarter.

我們透過繼續尋找更有效率的營運方式、加強對所有營運支出領域的審查以及採取措施（例如透過轉讓海港辦公室租賃來減少辦公室佔地面積）來抑制與員工人數無關的支出成長。第二季末完成。

We are deliberate about managing our expenses in an effort to extend our runway which will be achieved in part by keeping 2023 headcount relatively flat with current levels.

我們正在深思熟慮地管理我們的開支，以努力延長我們的跑道，這在一定程度上可以透過保持 2023 年員工人數與當前水平相對持平來實現。

In the time I've spent with Akebia, it's clear that everyone on the team is dedicated to managing resources while continuing to deliver on their individual and group objectives. Revenue from Auryxia continues to provide cash for operations, and our teams are motivated by the progress we've made across the business this quarter as we all work to prepare for the launch of vadadustat in the U.S. next year, if approved.

在我與 Akebia 合作的時間裡，很明顯，團隊中的每個人都致力於管理資源，同時繼續實現他們的個人和團隊目標。 Auryxia 的收入繼續為營運提供現金，我們的團隊受到本季度業務取得的進展的激勵，因為我們都在努力準備明年在美國推出 vadadustat（如果獲得批准）。

With that, let's open the line for questions.

接下來，讓我們打開提問熱線。

(Operator Instructions) Our first question comes from Allison Bratzel with Piper Sandler.

（操作員說明）我們的第一個問題來自 Allison Bratzel 和 Piper Sandler。

So first, just hoping you could frame for us from a high-level topics that were addressed at the Type A meeting, any remaining gating factors to submission for vadadustat and just your confidence that FDA will find the resubmission approvable and dialysis without the generation of additional clinical data? And then I have a follow-up.

因此，首先，希望您能為我們從 A 類會議上討論的高級別主題、提交 vadadustat 的任何剩餘限制因素以及您對 FDA 會發現重新提交可批准和透析的信心，而不會產生額外的臨床數據？然後我有一個後續行動。

Sure, Ally. Thanks for the question. I mean it was -- I think the Type A meeting was a pretty technical meeting in a lot of ways. Steve was here and attended the meeting. Steve, maybe give a little color?

當然，艾麗。謝謝你的提問。我的意思是——我認為 A 類會議在很多方面都是一個非常技術性的會議。史蒂夫在這裡參加了會議。史蒂夫，也許給點顏色？

Sure. This is Steve Burke, the Chief Medical Officer. The meeting was -- it's like a pre-NDA meeting where you meet with the agency to describe how you're going to present the requested data and is that acceptable to them. So for instance, like when we finish an NDA review, we have a 120-day safety update. So we have to do another safety update. And so we just clarified, for instance, the window in which any new data would be collected and reported. So John alluded earlier to the MODIFY and FO2CUS studies, what we were going to present, what we're going to provide to them and was that acceptable, how we were going to present Japanese post-marketing surveillance data, the actual components and how it would be analyzed and things of this nature.

當然。我是首席醫療官史蒂夫·伯克。這次會議就像 NDA 前的會議，您與該機構會面，描述您將如何提供所要求的數據以及他們是否可以接受。例如，當我們完成 NDA 審查時，我們有 120 天的安全性更新。所以我們必須進行另一次安全性更新。例如，我們只是澄清了收集和報告任何新數據的視窗。所以約翰早些時候提到了 MODIFY 和 FO2CUS 研究，我們將要展示什麼，我們將向他們提供什麼以及是否可以接受，我們將如何展示日本的上市後監測數據，實際的組成部分以及如何它將被分析以及這種性質的事物。

They also had asked some questions about some additional analysis they wanted of the Phase III data. But all of this is pretty modest in scope. But as John indicated, is new data, and therefore, we expect they will get a 6-month review clock because of that. I think we're satisfying all the requests and have really excellent clarity on what they want and expect from us and that we're able to deliver that. And there's no need for us to generate new data -- clinical data.

他們也詢問了一些關於他們想要對第三階段數據進行額外分析的問題。但所有這些的範圍都相當有限。但正如約翰指出的那樣，這是新數據，因此，我們預計他們將因此獲得 6 個月的審查時間。我認為我們滿足了所有的要求，並且非常清楚地了解他們對我們的需求和期望，並且我們能夠實現這一點。我們不需要產生新的數據——臨床數據。

Yes. I would say that it was an amazingly collaborative meeting kind of given that the CRL from last year, it was very focused on what we need to put through. And even going into it, most of the questions that we asked -- we posed to them, they found our approach reasonable. So there's really more clarification than anything else. So we feel pretty good about where things are and very much on track for our filing.

是的。我想說，考慮到去年的 CRL，這是一次令人驚奇的協作會議，它非常關注我們需要完成的事情。即使深入探討，我們向他們提出的大多數問題，他們也認為我們的方法是合理的。因此，確實比其他任何事情都更加澄清。因此，我們對事情的進展感到非常滿意，我們的歸檔工作也非常順利。

That's helpful. So now just hoping you could also walk us through your expectations for (inaudible) launch will look like kind of between the March PDUFA and the receipt of TDAPA designation during that timeframe. And then separately, if you could just kind of frame or compare contrast for us whether KORSUVA's launch experience under TDAPA, is it good analog for vadadustat? Any color or perspective there would be helpful.

這很有幫助。因此，現在希望您也能向我們介紹一下您對（聽不清楚）發布的期望，這看起來像是在 3 月份 PDUFA 和在此時間範圍內收到 TDAPA 指定之間。另外，如果您能為我們提供框架或比較 KORSUVA 在 TDAPA 下的發布體驗，它對 vadadustat 來說是一個很好的模擬嗎？任何顏色或視角都會有幫助。

Yes. No, thanks for that question, Ally. So a couple of things that are really important to communicate. While we will make the product available during the post-launch pre-TDAPA is really available for dialysis providers to purchase the product and develop protocols or the like. We don't expect widespread adoption until you have the TDAPA designation, and they'll have the product reimbursed outside of the bundle. So that's -- I mean I think that should be a clear expectation for everyone. If we see any dollars, it will be very modest, and you'll really see that uptake at TDAPA.

是的。不，謝謝你的提問，艾莉。因此，有幾件事對於溝通來說非常重要。雖然我們將在發布後提供該產品，但 TDAPA 確實可供透析提供者購買產品並開發協議等。在您獲得 TDAPA 指定之前，我們預計不會廣泛採用，並且他們將在捆綁包之外對產品進行報銷。所以，我的意思是，我認為這應該是每個人的明確期望。如果我們看到任何美元，那將是非常有限的，而且你會真正看到 TDAPA 的吸收。

I think the KORSUVA question is a really important one as well, and I appreciate you asking that. The KORSUVA launch is a very different product and a very different opportunity than vadadustat, I think for a number of reasons. When you look at KORSUVA, this is a product that was approved for pruritus, for severe itching, which is in a small number of patients. And the average pricing that's published now for KORSUVA is about $135 per dialysis session. And there's nothing in the bundle to cover pruritus other than $0.25 for BENADRYL, right? So the $135, they'd be reimbursed for -- on a cost basis as well. But when you speak to physicians about that experience and that desire to use the product, they really worry about, well, when TDAPA ends, there's no mechanism for me to continue to use this product to benefit patients. And think about it, a bundled payment is about $280 per dialysis session, and you have $135 for KORSUVA is a real worry from a financial perspective about having to use that, continuing to use that product.

我認為 KORSUVA 問題也是一個非常重要的問題，我很感謝你提出這個問題。我認為，與 vadadustat 相比，KORSUVA 的推出是一款非常不同的產品，也是非常不同的機會，原因有很多。當你看到 KORSUVA 時，你會發現這是一種被批准用於治療搔癢症、嚴重搔癢的產品，這種情況只發生在少數患者身上。目前公佈的 KORSUVA 平均定價約為每次透析 135 美元。除了 0.25 美元的 BENADRYL 之外，捆綁包中沒有任何東西可以治療瘙癢，對嗎？因此，他們將獲得 135 美元的報銷——也是基於成本。但當你與醫生談論這種經歷和使用該產品的願望時，他們真的擔心，當 TDAPA 結束時，我沒有任何機制可以繼續使用該產品來造福患者。想想看，每次透析療程的捆綁付款約為 280 美元，而您有 135 美元購買 KORSUVA，從財務角度來看，不得不使用該產品、繼續使用該產品確實令人擔憂。

So you're just not seeing patients who could benefit from the product benefit. And so that's one of the reasons why we've been trying to push CMS to create a continued add-on payment beyond TDAPA that follows the drug and haven't been successful yet. Now vadadustat is very different, right, because there's money in the bundle for ESAs today, the pricing will be very different. While it may be premium to ESAs, it's certainly not going to be $135 per dialysis session and that those dollars that are in the bundle will continue to be in the bundle post-TDAPA. So now our pricing may have to change from a contracting perspective post-TDAPA. But it's not as if people are going to stop treating anemia or not treat anemia, right? They're going to do that, which they're not doing with pruritus. So TDAPA is very much a positive for us, it has been more of a challenge for KORSUVA.

所以你只是沒有看到可以從產品的好處中受益的患者。這就是為什麼我們一直試圖推動 CMS 在 TDAPA 之外創建持續的附加付款方式，該附加付款方式是在藥物之後進行的，但尚未成功。現在 vadadustat 非常不同，對吧，因為今天 ESA 捆綁包裡有錢，定價將會非常不同。雖然它可能比 ESA 溢價，但每次透析療程肯定不會是 135 美元，而且捆綁包中的這些美元在 TDAPA 後仍將繼續包含在捆綁包中。因此，現在我們的定價可能必須在 TDAPA 後從合約角度進行變更。但這並不是說人們會停止治療貧血或不治療貧血，對嗎？他們會這麼做，但他們不會因為癢而這麼做。所以 TDAPA 對我們來說是非常積極的，但對 KORSUVA 來說更是一個挑戰。

Our next question comes from Ed Arce with H.C. Wainwright.

我們的下一個問題來自 Ed Arce 和 H.C.溫賴特。

Great. First, I just wanted to ask again on the Type A meeting. John, you and I have talked about this a number of times since the letter in May, specifically about the two issues from the CRL, the [thrombotic] events and the risk of (inaudible). I wanted to get your thoughts on how those meeting minutes now that you have them in hand are fully aligned with the path that was offered in that letter from Dr. Stein back in May. And then I have a follow-up.

偉大的。首先，我只是想再問一下關於A類會議的問題。約翰，自從 5 月的信函以來，你和我已經多次討論過這個問題，特別是關於 CRL 的兩個問題，即[血栓]事件和（聽不清楚）的風險。我想了解您對這些會議紀要的看法，既然您已經掌握了這些會議紀要，那麼它們與斯坦博士五月份的那封信中提供的路徑完全一致。然後我有一個後續行動。

Yes. I think I can say unequivocally that they're fully aligned. I mean I think we have -- as I said, and Steve, you kind of give more detail on, I mean, a lot of that meeting is kind of how you're going to present the data. It's more technical. But we had questions about our labeling, right, like that. I mean that's always a good sign, right? So it was -- there's nothing to indicate in that meeting that there was anything that was misaligned with the letter that we received from Dr. Stein. As I've said all along, I think that letter describes kind of the guardrails in the path for us and for the division. And I think that was reflected in the discussions.

是的。我想我可以明確地說，他們是完全一致的。我的意思是，我認為我們已經 - 正如我所說，史蒂夫，你提供了更多細節，我的意思是，會議的很多內容都是你將如何呈現數據。這更具技術性。但我們對我們的標籤有疑問，對吧，就像這樣。我的意思是這總是一個好兆頭，對吧？所以，在那次會議上沒有任何跡象表明我們從史丹博士那裡收到的信件有任何不一致之處。正如我一直以來所說的那樣，我認為這封信描述了我們和該部門前進道路上的護欄。我認為這反映在討論中。

It was -- again, it was a very collaborative discussion. I mean it's fair to say you never are quite sure what you're going to -- when you get when you go into those meetings. And I think (inaudible) agrees, I mean, we were quite pleased with the quality of the focus on the issues and the collaborative nature of the discussion and makes us feel quite good about our path forward.

這又是一次非常合作的討論。我的意思是，公平地說，當你參加這些會議時，你永遠不確定自己要做什麼。我認為（聽不清楚）同意，我的意思是，我們對問題的關注品質和討論的協作性質感到非常滿意，這讓我們對我們的前進道路感到非常滿意。

Great. And then I'd like to ask if you could remind us of the objective of having the MODIFY and FO2CUS studies included in the resubmission.

偉大的。然後我想問您是否可以提醒我們將 MODIFY 和 FO2CUS 研究納入重新提交的目的。

Yes. So for this resubmission, that's an important question. I mean, obviously, we think the MODIFY and FO2CUS studies will be quite helpful in securing 3x weekly dosing in our label after our initial approval and -- but it's clear they won't be looking at that 3x weekly dosing regimen for this initial approval. The reason for adding to MODIFY and FO2CUS data is to -- you basically have to add all of your safety data to your file, right, which (inaudible) '21 when we originally filed and now we simply have to update all of the safety data that we have.

是的。因此，對於這次重新提交，這是一個重要的問題。我的意思是，很明顯，我們認為MODIFY 和FO2CUS 研究對於在我們初步批准後確保我們的標籤中每週3 次給藥非常有幫助，但很明顯，他們不會在初次批准時考慮每週3次給藥方案。添加 MODIFY 和 FO2CUS 數據的原因是 - 您基本上必須將所有安全數據添加到您的文件中，對吧，我們最初提交時（聽不清楚）'21，現在我們只需更新所有安全數據我們擁有的數據。

So they won't be considering efficacy from those studies at all, while we know that they were successful. And we think we're expecting they'll support our 3x weekly filing, and we're working to publish them quickly, it won't be in the label.

因此，儘管我們知道它們是成功的，但他們根本不會考慮這些研究的功效。我們認為我們預計他們會支持我們每週 3 次的歸檔，我們正在努力快速發布它們，它不會出現在唱片公司中。

Okay. Great. Actually, if I could squeeze one more in, question on Auryxia, probably for Ms. Snow. Given that the half year net sales is now about $78 million, and you're reaffirming your guidance of $175 million to $180 million for the full year. I'm just wondering if you could help us understand what gives you the confidence to reach that guidance especially given what you described as a decline in the phosphate binder market.

好的。偉大的。事實上，如果我可以再插一句，關於 Auryxia 的問題，可能是問斯諾女士的。鑑於半年淨銷售額現在約為 7800 萬美元，並且您重申全年 1.75 億至 1.8 億美元的指引。我只是想知道您是否可以幫助我們了解是什麼讓您有信心達到該指導，特別是考慮到您所描述的磷酸鹽結合劑市場的下滑。

Yes. Thanks, Ed. I appreciate the question. So a number of things. There are -- if you look at the dynamics of sales over the past couple of years, it's kind of that September to December timeframe really is kind of where we see a significant step-up. To be sure, I would say that our -- we had an expectation of our price being a bit higher, but our payer mix has skewed towards [Part D] a little bit more than we expected. So I would say my expectation is probably more on the lower end of our guidance range. But when you look at -- there's a number of qualitative things as well. One of the large dialysis providers hadn't let our sales reps in to see people at all, and everything was being done virtually. That's just changed in the last month. And I think everyone could agree that sales reps can be much more effective when they're face-to-face with people. So we expect that to be helpful as well.

是的。謝謝，艾德。我很欣賞這個問題。所以有很多事情。如果你看看過去幾年的銷售動態，你會發現 9 月到 12 月的時間範圍確實是我們看到顯著成長的時期。可以肯定的是，我想說的是，我們的預期價格會更高一些，但我們的付款人組合偏向 [D 部分] 的程度比我們預期的要高一些。所以我想說，我的預期可能更多地處於我們指導範圍的下限。但當你觀察時——也有很多定性的東西。一家大型透析提供者根本不讓我們的銷售代表會見人員，一切都是虛擬完成的。這在上個月才發生了變化。我認為每個人都會同意，銷售代表在與人面對面時會更有效率。所以我們希望這也會有所幫助。

And a number of other -- those types of dynamics. And as Ellen said, we think that the binder market has still been under pressure, but it is flattening, if you will, we think. This is obviously, we'll continue to monitor this, but as it flattens -- I mean patients still need to be treated, right? So as they come in the funnel, I think there's opportunity there. And we also expect competitive product to be launched in the back half of this year as well, and that will come, I think, with a lot of focus on phosphate control, and that's kind of one of those rising tides (inaudible) boats at moments as well. So I think that there's a number of those types of issues that allow us to maintain that guidance, albeit I would say -- I would think more towards the lower end of the range where earlier in the year, I was expecting the higher end.

還有許多其他類型的動態。正如艾倫所說，我們認為黏合劑市場仍然面臨壓力，但我們認為，如果你願意的話，它正在趨於平緩。顯然，我們將繼續監測這種情況，但隨著情況趨於平緩——我的意思是患者仍然需要接受治療，對吧？因此，當他們進入漏斗時，我認為那裡有機會。我們也預計有競爭力的產品也將在今年下半年推出，我認為，這將在很大程度上關注磷酸鹽控制，這就是那些漲潮（聽不清楚）的船之一也有一些時刻。因此，我認為有許多此類問題使我們能夠維持這一指導，儘管我會說 - 我會更多地考慮範圍的低端，而在今年早些時候，我預計會出現較高端。

Our next question comes from Julian Harrison with BTIG.

我們的下一個問題來自 BTIG 的 Julian Harrison。

First, just on the 3x weekly dosing regimen, again, you studied in FO2CUS. So I'm curious if you could talk more about your strategy there and how soon a potential label for vadadustat maybe be amended with this dosing regimen?

首先，您再次在 FO2CUS 中研究了每週 3 次的給藥方案。所以我很好奇您是否可以更多地談談您的策略以及 vadadustat 的潛在標籤多久可以透過這種給藥方案進行修改？

Sure. So thanks, Julian, for the question. Welcome to the call. So 3x weekly is very helpful for treating patients in the dialysis center, right? You give them the dose during your dialysis session, you ensure compliance from the patient, you don't have to send them at home with drug. So we've always felt that, that was something that was important, and that's something that we could potentially add to the label that we never expected to have it in the label at launch. We will publish the data. We expect it to be presented at the ASN meeting in October from FO2CUS. FO2CUS was the large study that really looked at that 3x weekly dosing.

當然。謝謝朱利安提出的問題。歡迎來電。那麼每週 3 次對於治療透析中心的患者非常有幫助，對吧？您在透析過程中給予他們劑量，確保患者的依從性，您不必將藥物送回家。因此，我們一直認為，這是很重要的事情，而且我們有可能將其添加到標籤中，而我們從未期望在發佈時將其添加到標籤中。我們將公佈數據。我們預計 FO2CUS 將在 10 月的 ASN 會議上發布該報告。 FO2CUS 是一項大型研究，真正關注每週 3 次給藥。

Then once we have our NDA approved in March, of course, as we've been talking about and expect, then we'll have a conversation with the FDA about supplemental NDA where we'll submit for 3x weekly dosing. Again, when you think about the low-hanging fruit for the product adoption initially, it's home patients where daily dosing works just fine. And I would say those patients who are on the highest doses of ESAs where they're having the risk of exposure to high doses of ESAs and added cost as well to the dialysis provider. And even though those might be in-center patients, I think they'd be willing to dose them once a day and send them home a drug, or rely on the publication and choose to use 3x weekly dosing.

當然，一旦我們的新藥申請在三月獲得批准，正如我們一直在談論和期望的那樣，我們將與 FDA 就補充新藥申請進行對話，我們將提交每週 3 次給藥的申請。同樣，當您最初考慮產品採用的容易實現的目標時，家庭患者的每日給藥效果很好。我想說的是那些服用最高劑量 ESA 的患者，他們面臨接觸高劑量 ESA 的風險，並增加了透析提供者的成本。即使這些患者可能是中心內的患者，我認為他們也願意每天服用一次藥物並將藥物送回家，或依賴出版物並選擇每週服用 3 次劑量。

Of course, we wouldn't be promoting 3x weekly dosing until it's in our label, but that's certainly something that physicians can choose to do with the data that supports it. And then as I said, we work to have the 3x weekly dosing regimen added to our label as quickly as we can.

當然，我們不會提倡每週 3 次給藥，除非它出現在我們的標籤中，但這當然是醫生可以選擇使用支持它的數據來實現的。然後正如我所說，我們努力盡快將每週 3 次的給藥方案添加到我們的標籤中。

Okay. Got it. That's helpful. And then earlier in the call, you gave a great overview on your current commercial preparations. Sorry if I missed it, but I was just curious if you're talking at all about how much you're manufacturing at risk ahead of potential approval of vadadustat.

好的。知道了。這很有幫助。在電話會議的早些時候，您對目前的商業準備工作進行了很好的概述。抱歉，如果我錯過了，但我只是好奇你是否在談論在 vadadustat 可能獲得批准之前你的生產有多少風險。

Yes. Thanks for that question, Julian. We -- as I mentioned, the two biggest costs are people and product. And product is a really significant one. Remember, we had product on the shelf ready to launch, back last year. And our quality team has done a magnificent job of continuing to generate data, and we're able to push out the expiry date on that product to 4 years. So that product that's on the shelf today will be able to support our launch. So we don't have to do any incremental manufacturing and take that incremental cost to be ready to launch the drug. And of course, that drug is also going to support Medice and the European launch as well.

是的。謝謝你提出這個問題，朱利安。正如我所提到的，我們最大的兩個成本是人員和產品。產品是非常重要的。請記住，我們去年就已經準備好推出的產品。我們的品質團隊在持續產生數據方面做得非常出色，我們能夠將產品的有效期延長至 4 年。因此，今天上架的產品將能夠支援我們的發布。因此，我們不需要進行任何增量生產，也不需要花費增量成本來準備推出該藥物。當然，該藥物也將支持 Medice 和歐洲的上市。

There are no further questions. I'd like to turn the call over to John Butler for closing remarks.

沒有其他問題了。我想將電話轉給約翰·巴特勒（John Butler）做總結發言。

Thanks, Michelle. We are extremely pleased with the progress we've made last quarter as we made significant strides towards our purpose to better the lives of people impacted by kidney disease. We're preparing for very near-term potential catalysts. Most immediately, the resubmission of the vadadustat NDA next month. With this momentum, we believe we are now poised to disrupt the market for the anemia of CKD in dialysis patients if vadadustat is approved. Everyone at Akebia is excited to be in that position and remains motivated by our potential to help patients. The team is focused on the work we need to do in the very near future to get us there. And I look forward to continuing to update you as we move through resubmission, acceptance and ultimately, potential approval and launch of vadadustat in the U.S. Thank you all for joining us today.

謝謝，米歇爾。我們對上季度的進展感到非常滿意，因為我們在改善受腎臟疾病影響的人們的生活的目標方面取得了重大進展。我們正在為近期的潛在催化劑做準備。最直接的是下個月重新提交 vadadustat 保密協議。憑藉這一勢頭，我們相信，如果 vadadustat 獲得批准，我們現在將擾亂透析患者 CKD 貧血市場。 Akebia 的每個人都對能夠擔任這一職位感到興奮，並且仍然對我們幫助患者的潛力充滿動力。團隊專注於我們在不久的將來需要做的工作，以實現這一目標。我期待著在我們重新提交、接受以及最終在美國批准和推出 vadadustat 的過程中繼續向您通報最新情況。感謝大家今天加入我們。

Thank you for your participation. This does conclude the program, and you may now disconnect. Everyone, have a great day. Goodbye.

感謝您的參與。這確實結束了程序，您現在可以斷開連接。大家，祝你有美好的一天。再見。