Roivant Sciences Ltd (ROIV) 2024 Q2 法說會逐字稿

Good day and thank you for standing by. Welcome to the Roivant second-quarter 2024 earnings conference call. (Operator Instructions) Please be advised that today's conference is being recorded. I would now like to hand the conference over to your speaker today, Stephanie Lee. Please go ahead.

美好的一天，感謝您的支持。歡迎參加 Roivant 2024 年第二季財報電話會議。（操作員指示）請注意，今天的會議正在錄製中。我現在想把會議交給今天的發言人史蒂芬妮李。請繼續。

Good morning, and thanks for joining today's call to review Roivant's financial results for the second quarter ended September 30, 2024. I'm Stephanie Lee with Roivant. Presenting today, we have Matt Gline, CEO of Roivant; and Ben Zimmer, CEO of Priovant.

早安，感謝您參加今天的電話會議，審查 Roivant 截至 2024 年 9 月 30 日的第二季財務業績。我是 Roivant 的史蒂芬妮李。今天的演講嘉賓是 Roivant 執行長 Matt Gline；和 Priovant 執行長 Ben Zimmer。

For those dialing in via our conference call, you can find the slides being presented today as well as the press release announcing these updates on our IR website at www.investor.roivant.com. We'll also be providing the current slide numbers that we present to help you follow along.

對於透過我們的電話會議撥打電話的人，您可以在我們的投資者關係網站 www.investor.roivant.com 上找到今天演示的幻燈片以及宣布這些更新的新聞稿。我們還將提供目前的幻燈片編號，以幫助您跟進。

I'd like to remind you that we'll be making certain forward-looking statements during today's presentation. We strongly encourage you to review the information that we filed with the SEC for more information regarding these forward-looking statements and related risks and uncertainties.

我想提醒您，我們將在今天的演示中做出某些前瞻性陳述。我們強烈建議您查看我們向 SEC 提交的信息，以獲取有關這些前瞻性陳述以及相關風險和不確定性的更多信息。

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

有了這個，我會把它交給馬特。

Thank you, Stephanie. Thank you, to the operator, and thank you, everyone, for dialing in this morning. We actually have a relatively full update. So I'm excited to share, and there's at least one major new thing we're going to talk about, which is the 52-week data from our NIU study, which I'm pretty excited about.

謝謝你，斯蒂芬妮。謝謝接線員，也謝謝大家今天早上撥通電話。我們實際上有一個相對完整的更新。所以我很高興與大家分享，我們至少要討論一件重大的新事情，那就是我們 NIU 研究的 52 週數據，我對此感到非常興奮。

So starting on slide 5. Look, I think there's really two major areas of focus for us right now. And the first is clinical trial execution. We have a lot of ongoing trials, all of which are important that are going to generate interesting data coming in the near future here. Obviously, today, we're talking about the 52-week data for brepocitinib in NIU in the Phase 2 study. We're also -- the Phase 3 study is ongoing with the first patients enrolled and with Fast Track Designation having been granted. We presented this quarter batoclimab in Graves' disease.

那麼就從投影片 5 開始。聽著，我認為我們現在確實有兩個主要關注領域。首先是臨床試驗的執行。我們正在進行許多試驗，所有這些試驗都很重要，將在不久的將來產生有趣的數據。顯然，今天我們討論的是 brepocitinib 在 NIU 2 期研究中的 52 週資料。我們也—第三階段研究正在進行中，第一批患者已入組，並已獲得快速通道指定。本季我們介紹了巴托利單抗治療格雷夫茲病的方法。

We are now getting our programs up and running for IMVT-1402 in both Graves' disease and difficult-to-treat rheumatoid arthritis as well as a number of other programs to be starting soon. We have initiated our Phase 2 study of Mosli in PH-ILD.

我們現在正在啟動和運行 IMVT-1402 治療格雷夫茲病和難治性類風濕性關節炎的項目，以及即將啟動的許多其他項目。我們已經啟動了 Mosli 治療 PH-ILD 的 2 期研究。

And then we have clinical data coming, including namilumab in sarcoidosis by the end of this quarter. Batoclimab data coming next year in MG, CIDP and TED and brepocitinib in dermatomyositis top line data coming in the middle of next year as well. So that should be all exciting updates in the near future, all focused on clinical execution.

然後我們將收到臨床數據，包括本季末用於結節病的奈米魯單抗。MG、CIDP 和 TED 的 Batoclimab 數據將於明年公佈，皮肌炎的 brepocitinib 數據也將於明年年中公佈。因此，這應該是在不久的將來所有令人興奮的更新，所有這些更新都集中在臨床執行上。

On slide 6, the evolution of the business continues in some other ways as well with the Dermavant deal having closed just a week or so ago, now allowing us to refocus on clinical execution, as I just mentioned, while maintaining a large share of potential upside from VTAMA. We talked about that not long ago. We've made some progress with our LNP litigation with a Markman hearing in the Pfizer/ BioNTech case in December with a trial less than a year from now and the Moderna case clinically scheduled.

在幻燈片 6 中，業務的發展以其他一些方式繼續進行，Dermavant 交易僅在一周左右前完成，現在使我們能夠重新專注於臨床執行，正如我剛才提到的，同時保持很大的潛力份額VTAMA的優勢。我們不久前討論過這個。我們的 LNP 訴訟取得了一些進展，12 月在輝瑞/ BioNTech 案件中舉行了馬克曼聽證會，距離現在不到一年的審判，Moderna 案件也已進入臨床日程。

We've continued our plan of returning capital to shareholders. We've so far repurchased an aggregate of $754 million worth of stock as of 9/30, including $106 million in the quarter reporting today with an ongoing commitment to be prudent and thoughtful in deploying this capital. And then, of course, and I know this will be a topic of discussion, we have ongoing business development activities with multiple negotiations for potential in-licensing of new programs that we are really excited about. This really does remain one of the most exciting environments for that activity that we've ever been in.

我們繼續實施向股東返還資本的計畫。截至 9 月 30 日，我們已回購了總計價值 7.54 億美元的股票，其中包括今天季度報告中的 1.06 億美元，並持續承諾謹慎、深思熟慮地部署這筆資金。當然，我知道這將是一個討論的話題，我們正在進行業務開發活動，並就新項目的潛在許可進行多次談判，我們對此感到非常興奮。這確實仍然是我們經歷過的最令人興奮的活動環境之一。

As we've been saying for a little while here, on slide 7, the next chapter for us is really anchored by our late-stage pipeline. That includes IMVT-1402 and batoclimab together our FcRn franchise. That includes brepocitinib, which we'll be talking about a fair amount today, our TYK2 and JAK1 dual inhibitor. That includes namilumab and Mosli and a number of other programs that we expect and hope to be bringing in, in the near future.

正如我們在投影片 7 上已經說過的那樣，我們的下一章實際上是由我們的後期管道奠定的。其中包括 IMVT-1402 和 batoclimab 以及我們的 FcRn 特許經營權。其中包括 brepocitinib，我們今天將大量討論它，我們的 TYK2 和 JAK1 雙重抑制劑。其中包括 namilumab 和 Mosli 以及我們期望並希望在不久的將來引入的許多其他項目。

Altogether on slide 8, we think that really does give us one of the most exciting development stage clinical pipelines out there with a path to a $10 billion-plus peak sales portfolio spanning multiple therapeutic areas, obviously, including I&I and pulmonary hypertension as well as others with the first approvals potentially coming in the next couple of years as well as many Phase 2 and Phase 3 data readouts and a significant wave of approvals across new indications in the '26 to '30 timeframe. So really looking forward to the next handful of years here as our portfolio has time to mature and develop.

總而言之，在幻燈片8 上，我們認為這確實為我們提供了最令人興奮的開發階段臨床管道之一，並有望實現跨越多個治療領域的超過100 億美元的峰值銷售組合，顯然包括I&I 和肺動脈高壓以及其他藥物可能會在未來幾年內獲得首次批准，以及許多第二階段和第三階段的數據讀出，以及在 26 至 30 年時間範圍內針對新適應症的大量批准。因此，我們非常期待未來幾年的發展，因為我們的產品組合有時間成熟和發展。

So with that as the 30,000-foot overview, what I want to do next is make sure we give due time to the most significant of our updates for today, which is the 52-week data from our NIU Phase 2 study. And so I'm going to give just a really brief introduction here, and then I'm going to hand it over to Ben Zimmer, who's going to take you through the updates on that program in more detail.

因此，作為 30,000 英尺的概述，我接下來要做的是確保我們為今天最重要的更新留出足夠的時間，即來自我們 NIU 第二階段研究的 52 週數據。因此，我將在這裡做一個非常簡短的介紹，然後我將把它交給 Ben Zimmer，他將帶您更詳細地了解該計劃的更新。

So look, on slide 10, I'm going to steal only a tiny bit of Ben's thunder. Basically, as you'll recall, in the spring, we presented the 24-week data, and it looked to be lot like it supported a potential best-in-indication efficacy profile, a data of a kind that the NIU feel bluntly had not seen before.

所以看，在幻燈片 10 上，我只會搶走 Ben 的一小部分風頭。基本上，正如您所記得的，在春季，我們提供了 24 週數據，看起來很像是支持潛在的最佳適應症療效概況，NIU 直言不諱地認為這種數據以前從未見過。

And the hope really was just to maintain that level of efficacy through 52 weeks and I can say we've definitely hit that bar. We really only had one additional subject in each dose arm of the treatment failure, and we had sustained improvement from baseline on important metrics like retinal vascular leakage, CST and macular edema.

我們真正的希望只是在 52 週內保持這種療效水平，我可以說我們肯定已經達到了這個標準。在治療失敗的每個劑量組中，我們實際上只增加了一名受試者，並且我們在視網膜血管滲漏、CST 和黃斑水腫等重要指標上較基線持續改善。

So really exciting data. No new safety or tolerability signals. The safety database at this point, as you know, comprises 1,400 exposed subjects and patients and is consistent with approved and widely tried JAK inhibitors. And we've got Fast Track Designation from FDA in NIU with patients currently enrolling. So that's the best (inaudible) overview, but the data is pretty exciting.

非常令人興奮的數據。沒有新的安全性或耐受性訊號。如您所知，目前的安全資料庫包含 1,400 名暴露的受試者和患者，並且與已批准和廣泛嘗試的 JAK 抑制劑一致。我們在 NIU 獲得了 FDA 的快速通道指定，目前正在招募患者。這是最好的（聽不清楚）概述，但數據非常令人興奮。

So I want to hand it over to Ben Zimmer, as I said, the CEO of Priovant, who's going to take you through the next slides here and walk through the actual data. Ben, take it away.

因此，正如我所說，我想將其交給 Priovant 執行長 Ben Zimmer，他將帶您瀏覽下一張投影片並瀏覽實際數據。本，把它拿走。

Great. Thanks, Matt. Before going into the 52-week data, I just wanted to briefly highlight two tailwinds in I&I that we are really excited about as it relates to the brepo Phase 3 programs in NIU as well as in dermatomyositis. And these are outlined on slide 11.

偉大的。謝謝，馬特。在討論 52 週數據之前，我只想簡單強調 I&I 中的兩個我們非常興奮的順風車，因為它與 NIU 的 brepo 第三階段項目以及皮肌炎相關。這些都在幻燈片 11 中進行了概述。

The first on the left-hand side is you can see that as a category, JAK inhibitors have roughly doubled since 2020 in terms of both treated patients and revenue. And I think this really speaks to the fact that the benefit risk profile of these agents and physicians and patients' comfort with that benefit risk profile is quite established at this point, and that even includes in indications with significantly less morbidity than those in which Priovant is operating.

左側第一個是您可以看到，作為一個類別，自 2020 年以來，JAK 抑制劑在治療的患者數量和收入方面增加了一倍。我認為這確實說明了這樣一個事實，即這些藥物和醫生的獲益風險狀況以及患者對這種獲益風險狀況的舒適感在這一點上已經相當確定，甚至包括與Priovant 相比發病率顯著降低的適應症。

And then in terms of those indications where Priovant is operating is orphan diseases with high morbidity. We've also seen over the last few years, several launches in those, including the one played out on the right-hand side of this slide that have really validated this category of indication as a source, not only blockbuster revenue, but blockbuster revenue that can be achieved in a very quick timeframe given the prevalence of the disease, high morbidity, high unmet need and concentrated prescriber base with orphan pricing.

然後，就 Priovant 所經營的適應症而言，是發病率高的孤兒疾病。在過去的幾年裡，我們還看到了其中的幾項發布，包括這張幻燈片右側的發布，它們確實驗證了這一類別的指示作為來源，不僅是重磅收入，而且是重磅收入鑑於該疾病的流行、發病率高、未滿足的需求高以及採用孤兒藥定價的集中處方者基礎，這一目標可以在非常短的時間內實現。

And so if you turn to slide 12, you see that NIU really fits this phenotype quite on the money around 70,000 to 100,000 patients in terms of prevalence, very high morbidity, fourth leading cause of blindness in the developed world among the working age population, very little available for patients and concentrated prescriber base. Most of these patients are seeing dedicated uveitis specialty centers.

因此，如果你翻到投影片12，你會發現NIU 確實非常符合這種表型，從患病率、發病率非常高、發達國家勞動年齡人口中第四大失明原因來看，大約有70,000 到100,000 名患者。這些患者中的大多數都在專門的葡萄膜炎專科中心就診。

And turning to slide 13, we see really supporting this a new claims analysis that we did with IQVIA, which reconfirms once again the large number of patients receiving biologic therapy and TNF therapy in particular. In 2022, about 40,000 patients with NIU receiving a TNF inhibitor, including Humira and off-label TNF inhibitors, that number you can see is growing quite significantly and includes between off-label TNF and other biologics, so a large number of off-label therapies. And we know both from real-world experience and from clinical trials that Humira is only effective in fewer than 50% of patients with NIU.

轉向投影片 13，我們看到我們與 IQVIA 進行的新索賠分析確實支持了這一點，該分析再次證實大量患者正在接受生物治療，特別是 TNF 治療。到2022 年，大約有40,000 名NIU 患者接受TNF 抑制劑治療，包括Humira 和標籤外TNF 抑制劑，您可以看到這個數字正在顯著增長，其中包括標籤外TNF 和其他生物製劑，因此大量標籤外藥物療法。我們從現實經驗和臨床試驗中得知，修美樂僅對不到 50% 的 NIU 患者有效。

So I think the data here really speaks to both the large commercial opportunity in the TNF-refractory population but also with all of these off-label therapies, the urgency that physicians and patients feel to find something that does work and the willingness to try that and try it aggressively. And we think that speaks to the additional opportunity for brepocitinib given our data potentially in the broader non-anterior NIU population.

因此，我認為這裡的數據確實說明了 TNF 難治性人群中存在巨大的商業機會，也說明了所有這些標籤外療法、醫生和患者迫切需要找到有效的藥物以及嘗試的意願並積極嘗試。鑑於我們的數據可能涉及更廣泛的非前部 NIU 人群，我們認為這說明了 brepocitinib 的額外機會。

So with that context, I'll turn to the data itself. Just a brief reminder on slide 14 of the design of the Phase 2 NEPTUNE study, as this enrolled patients with active non-interior NIU, randomized 2:1 to brepo 45 milligrams and 15 milligrams, consistent with past studies, including the registrational study for Humira as well as the treatment paradigm in NIU, given how sick these patients are and are in need of immediate therapy to avoid the risk of blindness. All patients in both arms received a 2-week, 60-milligram per day steroid burst and then are rapidly tapered off those steroids. And the primary goal of the study then is to prevent treatment failure.

因此，在這種背景下，我將轉向數據本身。只是在幻燈片 14 上簡要提醒 2 期 NEPTUNE 研究的設計，因為該研究納入了活動性非內部 NIU 患者，按 2:1 隨機分配至 brepo 45 毫克和 15 毫克，與過去的研究一致，包括註冊研究考慮到這些患者病情嚴重且需要立即治療以避免失明的風險，修美樂以及NIU 的治療模式。雙臂的所有患者都接受了為期 2 週、每天 60 毫克的類固醇爆發治療，然後迅速逐漸減量。研究的主要目標是防止治療失敗。

Notably, as a reminder, in the NEPTUNE study, the taper occurred over 6 weeks to week 8, while in precedent studies, including the Humira VISUAL I study, it occurred over 13 weeks. So the study was designed really to set actually a higher bar for brepocitinib may get more difficult for it to demonstrate efficacy. And so against that backdrop, we're very excited with the results that were generated.

值得注意的是，在 NEPTUNE 研究中，逐漸減少發生在 6 週至第 8 週期間，而在包括 Humira VISUAL I 研究在內的先例研究中，逐漸減少發生在 13 週內。因此，這項研究的目的實際上是為 brepocitinib 設定更高的標準，這可能會使其更難以證明療效。因此，在這種背景下，我們對所產生的結果感到非常興奮。

And turning now to slide 15, to walk through that. We start with the primary endpoint from the NEPTUNE study. And on the left-hand side of the slide, you see the -- what was the primary endpoint, which is a 24-week treatment failure rate, which we've shared previously. And then on the right side, we have the updated data at week 52. In both cases, measured here against the historical placebo rate from the VISUAL I study, as Matt mentioned, only one additional patient met treatment failure criteria in each arm.

現在轉到投影片 15，詳細介紹一下。我們從 NEPTUNE 研究的主要終點開始。在幻燈片的左側，您可以看到主要終點是什麼，即 24 週治療失敗率，我們之前已經分享過。然後在右側，我們有第 52 週的更新數據。正如馬特所提到的，在這兩種情況下，根據 VISUAL I 研究的歷史安慰劑率進行衡量，每組中只有一名患者符合治療失敗標準。

So excellent data sustained up to week 52. You'll recall that Humira's failure rate at week 24 was over 50% using this analysis that includes discontinuations with 62%. And although AbbVie did not specifically report a 1-year failure rate, their rate based on the data that was published was well in excess of 70% under this analysis.

如此出色的數據一直持續到第 52 週。您可能還記得，根據此分析，Humira 在第 24 週的失敗率超過 50%，其中停藥率為 62%。儘管艾伯維沒有具體報告 1 年失敗率，但根據本次分析，根據已發布的數據，他們的失敗率遠遠超過 70%。

And probably the most effective way to compare to Humira now, which we do on slide 16, now that we have 1-year data from the NEPTUNE study is against what was the prespecified primary endpoint in the VISUAL I study, which was median time to treatment failure.

現在，與 Humira 進行比較的最有效方法（我們在幻燈片 16 上進行了比較），現在我們擁有 NEPTUNE 研究的 1 年數據，即與 VISUAL I 研究中預先指定的主要終點進行比較，即中位數時間治療失敗。

And you see that on slide 16, with 3 months for the placebo arm in VISUAL I, 5.6 months for the active arm. So again, reinforcing that the median patient fails before 6 months. So more than half of patients, even just at the 6-month mark, let alone the 1-year mark are not able to receive benefit. And then you see in contrast in brepo, the low-dose arm, median time to treatment failure, 9.3 months. And in the high-dose arm, as we did not get to a 50% treatment failure rate even at 1 year, it's not estimable over 12 months. So a very exciting data on treatment failure.

您可以在幻燈片 16 上看到，VISUAL I 中安慰劑組為 3 個月，活性組為 5.6 個月。再次強調，平均患者在 6 個月前就失敗了。因此，超過一半的患者，即使在 6 個月大關，更不用說 1 年大關，都無法受益。然後你會看到低劑量組 brepo 的對比，治療失敗的中位數時間為 9.3 個月。在高劑量組中，由於我們即使在 1 年時也沒有達到 50% 的治療失敗率，因此無法估計超過 12 個月的情況。這是關於治療失敗的非常令人興奮的數據。

And on slide 17, we also see that clinical data supported by what's really the gold standard biomarker for measuring ocular inflammation, which is wide-field fluorescein angiography needs measurements of retinal vascular leakage. And you see here on the left side, data for the high-dose arm, brepo 45 milligrams; on the right side, the low-dose arm, brepo 15 milligrams. The top row is the week 24 data, which we presented at the Euretina conference this fall.

在幻燈片 17 上，我們也看到由測量眼部發炎的真正黃金標準生物標記（即廣視野螢光素血管攝影）支援的臨床數據需要測量視網膜血管滲漏。您可以在左側看到高劑量組的數據，brepo 45 毫克；右側為低劑量手臂，brepo 15 毫克。最上面一行是第 24 週的數據，我們在今年秋天的 Euretina 會議上展示了這些數據。

And then the bottom is the new 52-week data today. And you see really great data, particularly from the high-dose arm, no patients worsening, most of the patients improving and that improvement from week 24, which was already quite significant, not only being sustained up to 52 weeks, but actually even increasing slightly. And then you see at both time points, a very clear dose response as it relates to the 15-milligram data. So very exciting data here that I think resonates quite a bit with the ophthalmology community in particular.

然後底部是今天新的 52 週數據。你會看到非常好的數據，特別是來自高劑量組的數據，沒有患者病情惡化，大多數患者都在改善，並且從第24 週開始改善已經非常顯著，不僅持續了長達52 週，而且實際上甚至還在增加輕微地。然後您會在兩個時間點看到與 15 毫克數據相關的非常清晰的劑量反應。這裡的數據非常令人興奮，我認為特別引起了眼科界的共鳴。

Turning to slide 18. I wanted to share an update as well on our macular edema data. Think this is one where the 52-week data is particularly important because macular edema in uveitis patients really develops over time as the medium- to longer-term consequence of inflammation.

轉到投影片 18。我還想分享我們的黃斑水腫數據的更新。我認為 52 週數據尤其重要，因為葡萄膜炎患者的黃斑水腫確實是隨著時間的推移而發展為發炎的中長期後果。

So even if inflammation can be gotten under control to the point that it's not impairing visual acuity in the short term, low levels of inflammation over time can lead to macular swelling and ultimately macular edema, which, of course, is one of the causes of blindness in uveitis patients and significantly impaired vision otherwise.

因此，即使發炎可以得到控制，在短期內不會損害視力，但隨著時間的推移，低水平的發炎也會導致黃斑腫脹，最終導致黃斑水腫，這當然是黃斑水腫的原因之一。炎患者失明，否則視力明顯受損。

And so week 24, as you'll recall, we had quite exciting data in the 45-milligram arm of the 10 patients who did not have macular edema at baseline. None developed it. And of the 7 patients who had it at baseline, 3 out of those 7 had resolution. But at the time, we were very eager to see the extent to which that held up at 1 year and very excited to share that it held up, as you can see here, quite well with no new patients developing macular edema and the 3 who had resolution maintaining that resolution.

因此，您可能還記得，第 24 週，我們在 10 名基線時沒有黃斑水腫的患者的 45 毫克劑量組中獲得了非常令人興奮的數據。沒有人開發它。在基線時患有此病的 7 名患者中，7 名中的 3 名已緩解。但當時，我們非常渴望看到這種情況在1 年時的持續程度，並且非常高興地分享這一情況，正如您在這裡所看到的，沒有新患者出現黃斑水腫，而且3 名患者也沒有出現黃斑水腫。

And again, this compares quite favorably to Humira, which is generally viewed by physicians in the real world as not being super effective at preventing the onset of macular edema. And you see that's actually supported by data from the VISUAL I study, where at 1 year of patients who did not have macular edema at baseline, half of them had developed it at 1 year. And in the placebo group, that was at 6 months. So again, a small number of patients. We're excited to see what the Phase 3 data looks like, but certainly very promising in terms of the potential of the drug.

而且，這與修美樂相比相當有利，現實世界的醫生普遍認為修美樂在預防黃斑水腫的發生方面並不是非常有效。你會看到，這實際上得到了 VISUAL I 研究數據的支持，在基線時沒有黃斑水腫的患者中，有一半在 1 歲時出現了黃斑水腫。在安慰劑組中，那是在 6 個月大時。再次，患者數量很少。我們很高興看到第三階段的數據，但就該藥物的潛力而言，肯定非常有希望。

And then just very briefly on slide 9 (sic - slide 19) I want to further reinforce the robustness of this by looking at mean CST over time. And you see here in addition to the great data in the 45-milligram arm, a clear dose response with the 15-milligram arm, which is great to see as well as time course data that is consistent with what you'd hope to see, particularly looking at the 45-milligram arm, very rapid onset of effect in the first few weeks, even while the steroid taper has already begun at week 2, and then that is clearly sustained over time out to 1 year.

然後，在投影片 9（原文如此 - 投影片 19）上，我想透過觀察一段時間內的平均 CST 來進一步增強其穩健性。除了 45 毫克組的出色數據之外，您還可以在此處看到 15 毫克組的清晰劑量反應，這非常值得一看，而且時間過程數據與您希望看到的一致，特別是觀察45 毫克劑量組，即使在第2 週就已經開始逐漸減少類固醇，但在最初幾週內起效非常迅速，然後這種效果明顯持續到1 年。

So very excited about the Phase 2 data, and we're already excited after 6 months, even more excited now after 1 year. And at Priovant, we're already really focused now on the Phase 3, not the Phase 2 and excited to share, as Matt mentioned, and as we announced a few months ago that enrollment of that study is underway and off to a great start.

對第二階段的數據感到非常興奮，6 個月後我們已經很興奮，1 年後我們更加興奮。在Priovant，我們現在已經真正專注於第3 階段，而不是第2 階段，並且很高興與大家分享，正如Matt 提到的，正如我們幾個月前宣布的那樣，該研究的註冊正在進行中，並且有一個好的開始。

You see on slide 20 here, the schematic for the study, very closely modeled on the Phase 2, as you would -- as one would imagine. We are advancing only the 45-milligram dose into Phase 3 with patients randomized 1:1 for brepo 45 against placebo.

您可以在投影片 20 上看到該研究的示意圖，它非常接近第二階段的模型，正如您所想像的那樣。我們只將 45 毫克劑量推進到第 3 階段，患者以 1:1 隨機分配 brepo 45 與安慰劑。

The study is a single protocol, so technically one study, but with two identical sub studies. So it will be reported out as two studies, CLARITY 1 and CLARITY 2, 150 subjects per sub study, so 300 subjects total. And the primary endpoint will be time to treatment failure, so the same primary endpoint as the VISUAL I study, and that will be over period 1, which is 1 year of placebo-controlled data. And then secondary endpoints will include all of the same measurements as the NEPTUNE study.

該研究是一項單一方案，因此從技術上講是一項研究，但有兩項相同的子研究。因此，它將報告為兩項研究，CLARITY 1 和 CLARITY 2，每個子研究 150 名受試者，因此總共 300 名受試者。主要終點將是治療失敗的時間，因此與 VISUAL I 研究相同的主要終點，將在第 1 期，即 1 年的安慰劑對照數據。然後次要終點將包括與 NEPTUNE 研究相同的所有測量結果。

And notably, as well, we are maintaining the rapid steroid taper that we've seen -- that we've done in the NEPTUNE study based on the great data we had in spite of that taper, we saw no need to change that and are excited to be bringing that forward and really hopefully setting a new standard for uveitis clinical trials in terms of what therapies should be expected to demonstrate. And we did have a productive meeting with the FDA over the summer, and this design incorporates their input, and we're confident that if we're able to deliver successful results, it will support an NDA filing.

值得注意的是，我們正在維持我們所看到的類固醇的快速減量——我們在海王星研究中所做的基於我們擁有的大量數據，儘管有這種減量，我們認為沒有必要改變這一點，我們很高興能夠提出這一點，並真正希望為葡萄膜炎臨床試驗制定新的標準，以預期治療效果。今年夏天我們確實與 FDA 進行了一次富有成效的會議，這個設計吸收了他們的意見，我們相信，如果我們能夠取得成功的結果，它將支持 NDA 備案。

So all in all, really a lot of excitement around NIU, around the NIU program and brepo's potential there. I did want to highlight briefly before handing it back to Matt, as Matt mentioned, actually, although we're very excited about the Phase 3 NIU program, even before that, we're going to have a readout of the Phase 3 dermatomyositis program, which is fully enrolled and will be reading out next year.

總而言之，NIU、NIU 計畫和 brepo 的潛力確實令人興奮。在將其交還給馬特之前，我確實想簡單地強調一下，正如馬特提到的，實際上，儘管我們對第3 階段NIU 計劃感到非常興奮，但即使在那之前，我們也將讀出第3 階段皮肌炎計劃，已全部註冊，並將於明年宣讀。

And I just want to highlight on slide 21 that similar to NIU, this again really meets the criteria of the attributes that we've seen being conducive to a very rapid blockbuster revenue potential indication. Again, prevalence of around 40,000 adults in the US, very high percentage of these patients are in the active treatment funnel, as I'll walk through on the next slide.

我只想在投影片 21 上強調，與 NIU 類似，這再次真正符合我們所看到的有利於非常快速的重磅收入潛力指示的屬性標準。同樣，在美國約有 40,000 名成年人患有該病，這些患者中很大一部分處於積極治療漏斗中，正如我將在下一張幻燈片中介紹的那樣。

This is a very high morbidity indication with really nothing available for patients in terms of modern therapies, mostly just steroids and IVIG. And again, a concentrated prescriber base, and our claims analysis suggests that about half of these patients are treated at a few hundred tertiary centers of excellence.

這是一個非常高的發病率適應症，對於現代療法來說，患者實際上沒有任何可用的方法，大多數只是類固醇和 IVIG。同樣，處方者基礎集中，我們的理賠分析表明，這些患者中約有一半在數百個三級卓越中心接受治療。

And on slide 22, this provides just a bit more color on kind of what these patients are currently being treated on and where they stand. As you see on the left here, as mentioned before, around 35 patients in 2022 actively treated with late-stage therapies for dermatomyositis.

在幻燈片 22 上，這為這些患者目前正在接受的治療類型以及他們的處境提供了更多的資訊。正如您在左側看到的，如前所述，2022 年約有 35 名患者積極接受皮肌炎的後期療法。

Notably, all of the steroids referenced here are oral or injectable and in no cases, did we count topical steroids. And you can see on the left-hand side of the slide that over 50% of these patients are treated with polypharmacy. So I think it really goes to how sick they are and how limited the efficacy of the currently available treatments are.

值得注意的是，這裡提到的所有類固醇都是口服或註射的，在任何情況下，我們都沒有計算外用類固醇。您可以在幻燈片左側看到超過 50% 的患者接受多種藥物治療。所以我認為這實際上取決於他們的病情有多嚴重以及目前可用治療方法的功效有多有限。

And then you see on the right-hand side, a different cut of the same data, which just looks at for any patients on each of these supposedly steroid-sparing therapies in terms of ISTs, biologics and IVIG that they're not really achieving that goal in terms of steroid sparing with over 50% of patients or some groups roughly 50% receiving greater than 10 milligrams per day of oral steroids chronically. So we're really excited about the DM data readout, and we think that if brepo is approved, this claims analysis really supports a large bolus of potential demand for rapid adoption early in the launch.

然後你在右側看到相同數據的不同部分，它只是查看接受這些所謂的類固醇節約療法的患者在 IST、生物製劑和 IVIG 方面的表現，但他們並沒有真正實現超過50% 的患者或大約50 % 的某些族群每天長期接受超過10 毫克的口服類固醇，從而實現了這一目標。因此，我們對 DM 數據讀出感到非常興奮，我們認為，如果 brepo 獲得批准，該聲明分析確實支持了在發布初期快速採用的大量潛在需求。

So before handing it back to Matt, really just to wrap up my section on slide 23, we've been working on really just, as Matt mentioned, development execution over the last few years with Priovant. And I think starting next year, that really tees us up to have some major value inflection with the upcoming Phase 3 data in DM, NIU study to follow soon behind that. We are also planning to start studies in additional orphan indications in 2025. And as I mentioned at the beginning, doing all of this into an environment with quite a few commercial tailwinds.

因此，在將其交還給 Matt 之前，實際上只是為了總結幻燈片 23 上的部分，正如 Matt 提到的那樣，我們在過去幾年中一直在與 Priovant 一起進行開發執行。我認為從明年開始，這確實讓我們做好了準備，即將到來的 DM、NIU 研究的第三階段數據將產生一些重大的價值變化。我們也計劃在 2025 年開始其他孤兒適應症的研究。正如我在一開始提到的，所有這些都是在一個有相當多商業順風的環境中進行的。

So really excited about what's ahead. And with that, I'll hand it back to Matt.

對未來感到非常興奮。有了這個，我會把它交還給馬特。

Thank you, Ben. And as I said, really excited about that data as well. So I appreciate all the work Priovant team has done there and looking forward to what's coming. So I'm going to now read through a couple of other updates that are rehashing some things that have happened over the past couple of months that we are excited about, starting with a reminder of what's going on in our anti-FcRn franchise.

謝謝你，本。正如我所說，這些數據也讓我感到非常興奮。因此，我感謝 Priovant 團隊在那裡所做的所有工作，並期待即將發生的事情。因此，我現在將閱讀其他一些更新，這些更新正在重述過去幾個月中發生的一些讓我們感到興奮的事情，首先提醒我們反 FcRn 專營權中正在發生的事情。

On slide 25, I know everyone is familiar with this data, but we're really thrilled with it. We've put out some really, really good proof-of-concept data in Graves' disease, which we think positions IMVT-1402 to be a potentially best-in-class and first-in-class program in that indication. As you'll remember, we had an over 75% response rate in patients who were uncontrolled on ATDs with over 50% of patients becoming ATD-free, being able to completely titrate their ATD dose and getting to normal T3 and T4 levels by 12 weeks.

在投影片 25 上，我知道每個人都熟悉這些數據，但我們對此感到非常興奮。我們已經針對格雷夫茲病發布了一些非常非常好的概念驗證數據，我們認為這些數據使 IMVT-1402 成為該適應症中潛在的同類最佳和同類首創項目。您可能還記得，對於 ATD 未控制的患者，我們的緩解率超過 75%，超過 50% 的患者不再使用 ATD，能夠完全滴定 ATD 劑量，並達到正常 T3 和 T4 水平 12幾週。

We continue to support our premise that deeper IgG reduction is important with our sort of 680 dose with deeper IgG reductions driving meaningfully higher response rates. which gives us a position for IMVT-1402 to be potentially best-in-class. And that's against the backdrop of a very high unmet need with 25% to 30% of Graves disease patients uncontrolled or intolerant to ATDs and really with no pharmacologic options.

我們繼續支持我們的前提，即更深層的 IgG 減少對於我們的 680 劑量非常重要，更深層的 IgG 減少可顯著提高反應率。這使我們有可能成為同類最佳的 IMVT-1402。這是在需求未被滿足的情況非常高的背景下，25% 至 30% 的格雷夫茲病患者對 ATD 無法控製或不耐受，並且實際上沒有藥物選擇。

And now as Immunovant has announced, the 1402 IND has been cleared by FDA, enabling a straight to pivotal transition. I won't go through the data again in great detail, but on '26 and '27 and '28, we repeat that data. It's just -- it's really exciting data for a disease with really no other options for this portion of the population.

現在，Immunovant 宣布，1402 IND 已獲得 FDA 批准，實現了直接向關鍵過渡。我不會再詳細介紹這些數據，但在 26 年、27 年和 28 年，我們會重複這些數據。這只是——對於一種對於這部分人群來說確實沒有其他選擇的疾病來說，這確實是令人興奮的數據。

The other announcement from Immunovant on slide 29 is the first of several new indications now that Immunovant is planning to get started in, which is in difficult-to-treat rheumatoid arthritis. So this is a subset of the RA population between 5% to 20% of the patient population that has failed at least three therapies. And what we've seen from another FcRn program for nipocalimab is that specifically in ACPA positive RA patients, this is associated with severe disease and poor outcomes.

投影片29上的Immunovant的另一項公告是Immunovant目前計劃開始的幾個新適應症中的第一個，即難以治療的類風濕性關節炎。因此，這是 RA 族群的一個子集，佔至少三種療法失敗的患者族群的 5% 至 20%。我們從 nipocalimab 的另一個 FcRn 項目中看到，特別是在 ACPA 陽性 RA 患者中，這與嚴重疾病和不良預後相關。

And the in-class data from nipocalimab suggests that those patients respond to FcRn therapy even when it delivers comparatively lower IgG suppression than we believe 1402 will be able to deliver. This study design is built to read out quickly with a quick answer. It's got an open-label read-in with a randomized withdrawal design that means that all of these patients are going to get on therapy.

nipocalimab 的同類數據表明，即使 FcRn 療法提供的 IgG 抑制比我們認為的 1402 能夠提供的相對較低，這些患者也會對 FcRn 療法產生反應。本研究設計旨在快速讀出並快速給出答案。它有一個開放標籤讀入和隨機退出設計，這意味著所有這些患者都將接受治療。

So we think enrollment should be a relatively straightforward proposition, and we'll get data quickly, which is something we think is valuable for the overall 1402 program. And again, we think our deeper IgG suppression is going to help meaningfully given the data that we've observed from others.

因此，我們認為註冊應該是一個相對簡單的提議，我們會快速獲取數據，我們認為這對整個 1402 計劃很有價值。再次強調，根據我們從其他人觀察到的數據，我們認為更深入的 IgG 抑制將產生有意義的幫助。

If you look at slide 30, just a reminder on that patient population. This is a big population of patients. People think about RA, they think about sort of the full bolus of the 1.5 million US RA patients. But for a subset of those patients, they just can't get treatment. They try multiple lines of therapy. It's just not working. And we're focused specifically on those patients who are ACPA autoantibody positive. This is a subset of patients that is quite sick and really lacks for good options.

如果您查看投影片 30，您會發現，這只是對患者群體的提醒。這是一個龐大的患者群體。人們想到 RA，就會想到美國 150 萬 RA 患者的全部劑量。但對其中一部分患者來說，他們就是無法得到治療。他們嘗試多種療法。它只是不起作用。我們特別關注 ACPA 自身抗體陽性的患者。這是一部分病情嚴重且確實缺乏良好選擇的患者。

So again, I won't go into all the detail here. The Immunovant team did a great call with the KOL just last week on this. But on slide 31, as a reminder, the two indications we've announced so far for 1402 are Graves' disease and RA. Graves' disease where we ought to be first-in-class in RA, where we think we have clear potential to be best-in-class. We have a number of other indications coming.

再說一次，我不會在這裡詳細討論所有細節。上週，Immunovant 團隊就此事與 KOL 進行了一次精彩的通話。但在投影片 31 上，提醒一下，我們迄今為止宣布的 1402 的兩個適應症是格雷夫茲病和 RA。格雷夫茲病，我們應該在 RA 領域處於領先地位，我們認為我們有明顯的潛力成為同類最佳。我們還有許多其他跡象。

The Immunovant team has indicated that five INDs have been cleared by FDA. So we're excited to talk about more of those indications, some of which are known to the public and some of which are not yet known to the public in the weeks and months to come. And I am as or more excited for some of those as I am for some of the ones we've already announced. So stay tuned, but excited to see that program developing.

Immunovant 團隊表示，FDA 已批准 5 個 IND。因此，我們很高興能夠談論更多這些跡象，其中一些是公眾已知的，而另一些在未來幾週和幾個月內尚未為公眾所知。我對其中一些項目的興奮程度與我對我們已經宣布的一些項目的興奮程度一樣，甚至更加興奮。所以請繼續關注，但很高興看到該程式的開發。

And then finally, on the clinical side, and again, I won't go through it in great detail because we spent time on it on the call earlier this fall. As you recall, this quarter, we unveiled Mosliciguat, which is our Phase 2 inhaled sGC activator for pulmonary hypertension patients with interstitial lung disease.

最後，在臨床方面，我不會詳細介紹它，因為我們在今年秋天早些時候的電話會議上花了很多時間。您還記得，本季度，我們推出了 Mosliciguat，這是我們的 2 期吸入 sGC 活化劑，用於治療患有間質性肺病的肺動脈高壓患者。

Really excited for the potential of that program. I've talked to many folks about it since we unveiled it. I think it could be a really great program for these patients, again, with few treatment options in a market that's now been validated by the very strong continued launch of Tyvaso.

對該計劃的潛力感到非常興奮。自從我們推出它以來，我已經和很多人談論過它。我認為，對於這些患者來說，這可能是一個非常好的項目，因為市場上幾乎沒有治療選擇，但現在已經透過 Tyvaso 的強勁持續推出得到了驗證。

As a reminder, on slide 34, Mosli has shown among the highest PVR reductions ever seen in either a single or repeat dose setting in a way that makes us excited for the possible translation of that to overall improvement in these PH-ILD patients.

提醒一下，在幻燈片 34 上，Mosli 在單次或重複劑量設定中顯示有史以來最高的 PVR 降低，這讓我們對可能將其轉化為這些 PH-ILD 患者的整體改善感到興奮。

And we're not totally sure even that we've seen the best of that PVR data on slide 35. You can see the PVRs continue to improve at the time point where they were measured. And we've got -- and this is maybe one of the most important pieces and there's one piece of new data I'll share in a moment here.

我們甚至無法完全確定我們是否在幻燈片 35 上看到了最好的 PVR 數據。您可以看到 PVR 在測量的時間點持續改善。我們已經得到了——這可能是最重要的部分之一，我稍後將在這裡分享一項新數據。

A really great dosing profile and formulation with sort of one pump once-a-day dosing for these patients. And one of the things that we think is really important here is that as an inhaled sGC activator, we don't have systemic vasodilation. And we've gotten some questions as we've been out talking about the program about how we are confident in that.

對於這些患者來說，這是一個非常好的劑量配置和配方，每天一次泵一次給藥。我們認為這裡真正重要的一件事是，作為吸入 sGC 活化劑，我們沒有全身血管舒張。當我們談論該計劃時，我們收到了一些關於我們對此有何信心的問題。

Obviously, the systemic exposure data that we've talked about is very helpful. We do not see meaningful levels of systemic exposure in assays. But the other evidence we have, this is the first time we put this out on slide 36, is that this is from a different Phase 1 study of Mosli. This is from a multiple ascending dose study in healthy volunteers. And what you would expect if you were getting systemic vasodilation is an impact on systemic systolic blood pressure.

顯然，我們談到的系統暴露資料非常有幫助。我們在檢測中沒有看到有意義的全身暴露水平。但我們擁有的其他證據（這是我們第一次將其放在第 36 張幻燈片上）是，這是來自 Mosli 的另一個第一階段研究。這是來自對健康志願者的多次遞增劑量研究。如果您進行全身血管舒張，您會期望對全身收縮壓產生影響。

And you can see on slide 36, we've got a couple of dose arms here. This study did not go all the way up to 4 milligrams, but it's got about 0.5 milligram, 1 milligram and 2 milligrams. And you can see there's really no discernible pattern. And in fact, you would expect if we had systemic exposure reductions in systolic blood pressure. And in fact, the lowest dose arm here is placebo. So we -- this gives us additional confidence that this drug is not having a systemic impact.

您可以在幻燈片 36 上看到，我們這裡有幾個劑量臂。這項研究並沒有一直達到 4 毫克，但大約有 0.5 毫克、1 毫克和 2 毫克。您會發現實際上沒有明顯的模式。事實上，您會期望我們的收縮壓會出現全身性的降低。事實上，這裡的最低劑量組是安慰劑。所以我們——這讓我們更相信這種藥物不會產生系統性影響。

So with that, I'm just going to turn a little bit to the future. And then in just a minute, we'll open the line up to questions. So on slide 38, I talked about this in August, but I am just super excited for where we are right now. In the next 18 months or this slide goes at '24, we just have -- we have a number of really exciting large value creation opportunities, including things like the near-term data of the Namilumab in sarcoidosis, which as we said, is a little bit higher risk, it would be a huge opportunity if successful as well as multiple late-stage readouts. Ben talked about brepocitinib in DM.

因此，我將稍微轉向未來。稍後我們將開始提問。在幻燈片 38 上，我在 8 月談到了這一點，但我對我們現在的處境感到非常興奮。在接下來的 18 個月內，或這張投影片在 24 歲時，我們有許多真正令人興奮的巨大價值創造機會，包括 Namilumab 治療結節病的近期數據，正如我們所說，風險稍高一點，如果成功的話將是一個巨大的機會以及多個後期讀數。Ben 談到了 DM 中的 Brepocitinib。

We have a number of really important, we think, highly value-creating readouts from batoclimab and Immunovant. We have important updates in our LNP litigation. And then at the tail end of this period, the second half of 2026, we get that Phase 2 data from Mosli.

我們認為，我們有許多來自 batoclimab 和 Immunovant 的非常重要的、高度創造價值的讀數。我們的 LNP 訴訟有重要更新。然後在這一時期的末尾，即 2026 年下半年，我們從 Mosli 獲得了第二階段的數據。

So this is a period even with our existing pipeline without thinking about business development that is stacked with catalysts. And then as I've said a few times, we just could not be more excited for the business development environment we're in. And although everything takes a little bit longer than you want it to, I am chomping at the bit waiting to share some of the things we're working on. So continue to give us a minute and we're ready to share that. I think you'll agree, it's pretty exciting.

因此，這是一個即使我們現有的管道也沒有考慮充滿催化劑的業務發展的時期。正如我多次說過的，我們對所處的業務開發環境感到非常興奮。雖然一切都比你想要的要花更長的時間，但我還是迫不及待地想分享我們正在做的一些事情。請繼續給我們一分鐘時間，我們已準備好分享這一點。我想你會同意的，這非常令人興奮。

A brief financial update on slide 40, just to say, continue to be careful on managing burn. I think you'll see, especially with Dermavant now out of the picture, these numbers will come down in the coming quarters and with some one-time expenses behind us, R&D expense of $143 million or adjusted R&D non-GAAP of $132 million, G&A of $203 million, non-GAAP of $142 million, and that includes some pretty significant one-time expenses and overall loss from continuing operations of $237 million or adjusted of $219 million.

第 40 張投影片上的簡短財務更新只是說，繼續謹慎管理燒錢。我想你會看到，特別是Dermavant 現在已經退出舞台，這些數字將在未來幾個季度下降，並且我們已經消除了一些一次性費用，研發費用為1.43 億美元，或調整後的非公認會計準則研發費用為1.32 億美元，G&A 為 2.03 億美元，非 GAAP 為 1.42 億美元，其中包括一些相當大的一次性費用以及持續營運造成的 2.37 億美元或調整後的 2.19 億美元的總體損失。

We ended the quarter in a very strong cash position with $5.4 billion of cash. Again, most of the dollars we spent in the recent months have been on the share repurchase, including the Sumitomo dollars and no debt on the balance sheet following the close of the Dermavant transaction. The credit facility was repaid at closing and Organon acquired all the remaining debt and a share count that continues to come down over time as we repurchase shares.

本季結束時，我們的現金狀況非常強勁，擁有 54 億美元的現金。同樣，我們最近幾個月花費的大部分美元都用於股票回購，包括住友美元，並且在 Dermavant 交易結束後資產負債表上沒有任何債務。信貸額度在交易結束時得到償還，歐加農收購了所有剩餘債務，並且隨著我們回購股票，股票數量不斷減少。

So with that, I'll just end by pointing you to slide 42, which lays out the timeline for our upcoming catalysts. And I will end there. I'll say thank you again to everybody who's listening this morning, and I will hand it over to the operator to begin Q&A.

因此，最後我將向您指出幻燈片 42，其中列出了我們即將推出的催化劑的時間表。我就到此結束。我要再次向今天早上收聽的每個人表示感謝，然後我會將其交給接線員開始問答。

(Operator Instructions)

（操作員說明）

Louise Chen, Cantor.

路易絲·陳，康托爾。

Hi. Congratulations on all the progress and the data today. Thanks for taking my questions. So I had two on brepocitinib. I wanted to ask you what set of efficacy you'd like to see in your Phase 3 NIU trial? And then also for brepo and HS, where do you stand and what's the latest update there? Thank you.

你好。祝賀今天取得的所有進展和數據。感謝您回答我的問題。所以我服用了兩次佈雷波西替尼。我想問您希望在第 3 期 NIU 試驗中看到什麼樣的療效？那麼對於 brepo 和 HS，您的立場如何？謝謝。

Yeah. I'll let Ben take both those questions other than I'll say what I hope I will say is we're very happy with the efficacy we've seen in Phase 2, and I think it gives us a pretty wide margin relative to the competitor programs like Humira, but Ben take it away.

是的。我會讓 Ben 回答這兩個問題，但我希望我要說的是，我們對第二階段看到的功效非常滿意，我認為這給了我們相當大的餘地競爭對手的項目如修美樂（ Humira），但本把它拿走了。

Yeah. I would agree with that. I think anything that even approximates the Phase 2 would be terrific. And I think even if there's some standard drop off in efficacy that one often sees between Phase 2 and Phase 3. This is a space where there's very little available for patients, TNF inhibitors are widely used in spite of their quite limited efficacy.

是的。我同意這一點。我認為任何接近第二階段的東西都會很棒。我認為，即使在第二階段和第三階段之間經常看到療效出現一些標準下降。這是一個可供患者使用的藥物很少的領域，儘管 TNF 抑制劑的功效相當有限，但仍被廣泛使用。

And so I think anything that gets the drug approved would support widespread adoption and certainly anything that supports a potential better product profile than Humira, which support widespread adoption potentially even in the first-line setting.

因此，我認為任何獲得該藥物批准的藥物都將支持廣泛採用，當然任何支持比 Humira 更好的潛在產品概況的藥物都將支持廣泛採用，即使在一線環境中也可能支持廣泛採用。

And then on HS and Ben, you can jump in if you have any comments as well. But I guess what I'd say is -- I can say this from the outside, I've been really happy with the work that the private team has done on indication expansion, and we have some other ideas.

然後關於 HS 和 Ben，如果您有任何意見也可以加入。但我想我想說的是——我可以從外部說，我對私人團隊在適應症擴展方面所做的工作非常滿意，而且我們還有一些其他想法。

HS is a great indication. We have very good Phase 2 data in it. It's a competitive field with a lot of other mechanisms, and there are some great places to take brepocitinib that may be less competitive, but we're continuing to consider a wide variety of indications. And certainly, HS remains on our radar.

HS 是一個很好的指示。我們有非常好的第二階段數據。這是一個競爭激烈的領域，有許多其他機制，並且有一些很好的地方可以服用 brepocitinib，但競爭可能不那麼激烈，但我們正在繼續考慮各種適應症。當然，HS 仍然在我們的關注範圍內。

Yeah, don't really have much to add to that.

是的，其實沒什麼好補充的。

Thank you. Thanks for the questions. Really appreciate it.

謝謝。感謝您的提問。真的很感激。

(Operator Instructions)

（操作員說明）

Brian Cheng, JPMorgan.

布萊恩鄭，摩根大通。

Hey, guys. Thanks for taking our questions this morning. Maybe just a question on CLARITY design. Are you requiring patients to have a certain steroid dose for entry? And are there any certification strategy that we should make note of? And also on the sub studies between CLARITY-1 and 2, what is the difference here? Is there a geographical location difference?

嘿，夥計們。感謝您今天早上提出我們的問題。也許只是關於 CLARITY 設計的問題。您是否要求患者服用一定劑量的類固醇才能進入？有什麼認證策略值得我們注意嗎？還有 CLARITY-1 和 2 之間的子研究，這裡有什麼不同？有地理位置差異嗎？

Great. I'll let Ben take all of those.

偉大的。我會讓本拿走所有這些。

Yeah. So in terms of steroids, there's no specific requirement. Patients are allowed on any background steroid dose of up to 40 milligrams per day or no steroids at all. And again, with the notion that because there's the 2-week burst at the start of the study, that kind of neutralizes whatever the background regimen was before.

是的。所以就類固醇而言，沒有具體要求。患者每天可以使用任何劑量不超過 40 毫克的類固醇，或完全不使用類固醇。再說一遍，因為研究開始時有兩週的爆發，所以這會抵消先前的背景治療方案。

In terms of stratification, no particular stratification of material note. And in terms of the two sub studies, sites will be assigned to one or the other in some geographies like the United States and certain other larger geographies will have sites in both sub-studies and then there are certain countries that will only be in one or the other.

從分層來看，材料註釋沒有特別的分層。就這兩項子研究而言，在某些地區（例如美國）中，地點將被分配給其中一個或另一個，而某些其他較大的地區將在這兩個子研究中都有地點，然後某些國家只會在一個子研究中或其他。

Thanks, Brian. Appreciate it.

謝謝，布萊恩。欣賞它。

Yaron Werber, TD Cowen.

亞龍·韋伯，TD·考恩。

Hey. Good morning, guys. This is Joyce on for Yaron. Thank you for taking my quesition. Can you talk about your thoughts around pricing for brepocitinib, of course, orphan price point here, but how should we think about pricing by indication? Thank you.

嘿。早安，夥計們。這是喬伊斯為亞龍發言。感謝您回答我的問題。您能否談談您對 brepocitinib 定價的想法，當然，這裡是孤兒藥價格點，但我們應該如何考慮按適應症定價？謝謝。

Thanks. Look, I appreciate the question. Thank you for asking. Look, it's obviously premature to have a firm view on pricing for a program at this stage. We're focused on orphan disease with high unmet needs. So we think a pretty wide range of prices is supportable.

謝謝。聽著，我很欣賞這個問題。謝謝你的詢問。看，現階段對專案的定價有堅定的看法顯然還為時過早。我們專注於需求未被滿足的孤兒疾病。因此，我們認為相當廣泛的價格範圍是可以支持的。

What I would say and let Ben to answer as well, is that the only thing I'd say is other competitors in dermatomyositis have talked about net pricing in current sort of the high $100,000 range. And I think that's a useful benchmark for us as we think about the range of possibilities in that indication. But other than that, I think a pretty light range of if possible. Ben?

我要說的是，也讓 Ben 來回答，我唯一要說的是，皮肌炎領域的其他競爭對手已經談到了目前 10 萬美元高位的淨定價。我認為，當我們考慮該指標的可能性範圍時，這對我們來說是一個有用的基準。但除此之外，我認為如果可能的話，範圍相當小。本？

Yeah. I now would echo what Matt said. I think if you look at benchmarks for recent orphan launches, including both biologics and small molecules that have been at similar price points, that's obviously the kind of band and range we'll be looking at, but we don't have any sort of firm decisions or plans at this point in time.

是的。我現在會附和馬特所說的話。我認為，如果你看看最近推出的孤兒藥的基準，包括價格相似的生物製品和小分子，這顯然是我們將要考慮的那種帶和範圍，但我們沒有任何形式的此時的堅定決定或計劃。

Thank you.

謝謝。

Andy Chen, Wolfe Research.

安迪陳，沃爾夫研究中心。

Hey, good morning. Thank you for taking the question. On uveitis Phase 3, can you talk about what placebo response you're assuming? So in the Humira trial, I think they saw 13 weeks or 3 months. But in your trial, you have a more stringent tapering. So your tapering is 8 weeks versus Humira trial, which was, I think, 15 weeks. So in other words, are you assuming 13 weeks, is that going to be the placebo response on the primary endpoint? Is it going to be less than 13 weeks? Or should we be assuming that it's going to be less than 13 weeks? Thank you.

嘿，早安。感謝您提出問題。關於葡萄膜炎第三階段，您能談談您假設的安慰劑反應是什麼嗎？因此，在 Humira 試驗中，我認為他們持續了 13 週或 3 個月。但在你的試驗中，你有更嚴格的減量。所以你的減量時間是 8 週，而 Humira 試驗是 15 週。換句話說，您是否假設 13 週，這將是主要終點的安慰劑反應嗎？會不會少於13週？或者我們應該假設這將少於 13 週？謝謝。

That's a great question. Ben?

這是一個很好的問題。本？

Yeah. I mean I think as the base case, our assumption was a similar placebo rate to what was seen in VISUAL I. We think there's opportunity potentially for it to be even higher for the reasons you said with the taper, but we didn't want to assume that.

是的。我的意思是，我認為作為基本情況，我們的假設是與VISUAL I 中看到的類似的安慰劑率。希望假設。

In general, the study is actually overpowered. So even if the placebo rate ends up being significantly higher or the failure rate is significantly lower in the placebo group than we expected and we saw in VISUAL 1, we'd still have an opportunity to detect the difference just being humble about the fact that this is an area where there's really just one precedented study, we wanted to err on the side of being conservative, and that's why we're running as large a program as we are.

總的來說，這項研究實際上是壓倒性的。因此，即使安慰劑組的安慰劑率最終顯著高於我們的預期，或者安慰劑組的失敗率顯著低於我們在視覺1 中看到的預期，我們仍然有機會發現差異，只需謙虛地對待這一事實：這個領域實際上只有一項先例研究，我們寧願保守一點，這就是為什麼我們要運行如此大的專案。

And one thing that's patient-friendly about the study as a reminder is that it's an event-driven study. And as people fail, they'll move right over to the repo, which the patients will like about the study. Thanks, Andy. That was a great question.

這項研究對患者友善的一點是，它是一項事件驅動的研究。當人們失敗時，他們會直接轉移到儲存庫，患者會喜歡這項研究。謝謝，安迪。這是一個很好的問題。

Douglas Tsao, H.C. Wainwright.

雪兒 (Douglas Tsao)溫賴特。

Hi, good morning. Thank you for taking the questions. Matt, just on that last one, do patients automatically switch to brepo if they have treatment dose or do they have the option to switch? And is there a sort of alternative protocol that they can pursue?

嗨，早安。感謝您提出問題。馬特，就最後一項而言，如果患者接受治療劑量，他們是否會自動切換到 brepo，或者他們可以選擇切換？他們可以追求某種替代協議嗎？

They automatically switch to brepo. Obviously, they can drop out of the study if they wish and do not hesitate. But if they want to stay in the study, the kind of first fare, obviously, we don't know whether patients are on placebo or drug or neither do the investigator.

他們會自動切換到 brepo。顯然，如果他們願意並且毫不猶豫，他們可以退出研究。但如果他們想留在研究中，顯然，我們不知道患者是否服用安慰劑或藥物，研究人員也不知道。

So the first rescue medication in the event of failure is brepocitinib along with some other different options that the investigators have to deploy along with that. And then if the patient then fails for a second time in the open-label period, then there's an even wider array of options that the physician has available. And then at that point, the patient can choose whether or not they want to stay on drug and can still remain in the study.

因此，如果失敗，第一個救援藥物是布雷波西替尼，以及研究人員必須部署的其他一些不同的選擇。然後，如果患者在開放標籤期內第二次失敗，那麼醫生可以有更廣泛的選擇。然後，患者可以選擇是否繼續用藥並仍留在研究中。

Okay. Great. Thanks. And just as a follow-up for Matt. From a business development standpoint, obviously, with Mosli and the creation of namilumab we have sort of gone beyond what has been a short-term focus on I&I. I'm just curious how you're thinking about Roivant from a therapeutic category standpoint right now? And does this move respiratory like a big focus for adding additional assets? Or you're just going to continue to be as you put sort of rip off the economics and dig out the best opportunities as you identify them? Thank you.

好的。偉大的。謝謝。就像馬特的後續行動一樣。顯然，從業務發展的角度來看，隨著 Mosli 和 namilumab 的創建，我們已經超越了對 I&I 的短期關注。我只是好奇您現在從治療類別的角度如何看待 Roivant？這是否會引起人們對增加額外資產的關注？或者你會繼續這樣，你會利用經濟學原理，在發現最佳機會時挖掘它們？謝謝。

Yeah. Thanks, Doug. It's a great question. It's one you've been focused on and rightly so. I think we are pretty ruthlessly focused on doing the best things we can. The analogy that I've been using that you might have heard lately is that we exist in the excess dough outside of other people's cookie cutters. And so we're not the snowman, we're not the Christmas tree. We're the dough in between the two, and that means we've kind of got to be flexible in terms of therapeutic area. But we think there's a lot of really great cookie to bake out of that dough as well.

是的。謝謝，道格。這是一個很好的問題。這是您一直關注的問題，也是正確的。我認為我們非常無情地專注於做我們能做的最好的事情。我一直在使用的類比你最近可能聽說過，那就是我們存在於其他人的餅乾切刀之外的多餘麵團中。所以我們不是雪人，我們不是聖誕樹。我們是兩者之間的麵團，這意味著我們必須在治療領域方面保持靈活性。但我們認為用這個麵團也可以烤出很多很棒的餅乾。

Okay, great. Thank you.

好的，太好了。謝謝。

Dennis Ding, Jefferies.

丹尼斯丁，傑弗里斯。

Good morning. This is Anthea on for Dennis. Two on DM. Could you talk about your plan to share the full lupus data? And if you see any overlaps between lupus and DM? And then also, what's the willingness from doctors to prescribe JAKs in DM and how much off-label use is there currently, if any? Thank you.

早安.這是丹尼斯的安西婭。DM 上有兩個。能談談您分享完整狼瘡數據的計畫嗎？您是否發現狼瘡和 DM 之間有任何重疊？另外，醫生在 DM 中開 JAK 的意願如何？謝謝。

Yeah, thanks. That's -- it's a great question. I'll take a part of it and then hand it over to Ben. Look, my only reminder is well, two things. One is a reminder, Pfizer designed and ran the DM study was -- sorry, the lupus study. It was one of the last ones from their original brepocitinib program and I guess on the overlap point, and I am sure Ben will say something similar.

是的，謝謝。這是一個很好的問題。我會拿一部分然後交給本。聽著，我唯一的提醒是，有兩件事。提醒一下，輝瑞設計並進行的 DM 研究是－抱歉，是狼瘡研究。這是他們最初的 brepocitinib 計劃中的最後一個，我猜是在重疊點上，我相信 Ben 也會說類似的話。

We have so much data in brepocitinib across such a breadth of indications at this point that any one study in any one indication is really not as informative as the overall bolus of data on how efficacious the drug is. So I'm not sure we see a lot of specific commonality between SLE and DM or any prediction from SLE related to DM.

目前，我們擁有如此多適應症的布雷波西替尼數據，以至於任何一種適應症的任何一項研究實際上都不如關於該藥物有效性的整體數據。所以我不確定我們是否看到 SLE 和 DM 之間有很多具體的共通性，或者 SLE 與 DM 相關的任何預測。

But Ben, you want to take that as well as the off-label JAK use question?

但是 Ben，您想接受這個問題以及標籤外 JAK 使用問題嗎？

Yeah, I don't see a ton of read-through there. I mean I think one lesson from these sort of rheumatic diseases in general and our lupus data was an example of that is you have to be very focused on managing placebo response, and that's something I can't say that in the DM study, where we designed it and are running it ourselves, we've been extremely focused on that, including with the mandatory steroid taper in the study and a very high operational focus from our team on ensuring adherence to that taper among other kind of study execution related steps we're doing.

是的，我在那裡沒有看到大量的閱讀。我的意思是，我認為從一般風濕病和我們的狼瘡數據中得到的一個教訓是，你必須非常專注於管理安慰劑反應，這是我在 DM 研究中不能說的，其中我們設計了它並自己運行它，我們一直非常關注這一點，包括研究中的強制性類固醇逐漸減少，以及我們團隊非常高度的操作重點，以確保在其他類型的研究執行相關步驟中遵守該逐漸減少我們正在做。

As far as JAKs in DM, yeah, they're used pretty extensively. There was a recent publication that was kind of a literature review of case reports and there were 600 published case reports roughly in that across DM and Juvenile DM in that. And I think if you talk to KOLs and other prescribers and other DM treaters, you'll see that they do use JAK inhibitors. So I think it's certainly an area of a lot of comfort.

就 DM 中的 JAK 而言，是的，它們的使用相當廣泛。最近有一份出版物是對病例報告的文獻綜述，其中大約有 600 份已發表的病例報告，涉及 DM 和青少年 DM。我認為，如果您與 KOL 和其他處方者以及其他糖尿病治療者交談，您會發現他們確實使用 JAK 抑制劑。所以我認為這肯定是一個非常舒適的領域。

Most of the treating physicians here will be rheumatologists and dermatologists, a few neurologists as well. But both rheums and derms are also obviously very comfortable with JAK inhibitors from other indications as well. And as I mentioned before, both rheumatic and derm indications with less morbidity than DM, JAK inhibitors that are on label for those indications are widely prescribed at this point.

這裡的大多數主治醫生都是風濕科醫生和皮膚科醫生，還有一些神經科醫生。但大黃肌和真皮顯然也對其他適應症的 JAK 抑制劑非常滿意。正如我之前提到的，風濕病和皮膚適應症的發生率都比 DM 低，標籤上針對這些適應症的 JAK 抑制劑目前已被廣泛使用。

And so I think there's a lot of excitement in the physician community about JAK inhibition. I think there's a lot of excitement about a TYK2/JAK1 inhibitor in particular, given the alignment of that particular mechanism to the pathobiology of DM and really just the prospect of having a once-daily oral approved therapy that's efficacious and targets the underlying disease, that would be a new and important development for the field.

因此，我認為醫生界對 JAK 抑制非常興奮。我認為 TYK2/JAK1 抑制劑尤其令人興奮，因為該特定機制與 DM 病理學的一致性，並且實際上是一種每日一次口服批准療法的前景，該療法有效且針對潛在疾病，這將是該領域的一個新的重要發展。

Thank you.

謝謝。

Corinne Johnson, Goldman Sachs.

科琳·約翰遜，高盛。

Hey, good morning. This is Craig on for Corinne. So the first question here is given the emergence of Humira biosimilars, how do you expect brepo could be positioned if it potentially gains approval? And then from there, will you recruit or target patients that are refractory to anti-TNF-type medicines within CLARITY?

嘿，早安。這是為科琳代言的克雷格。因此，這裡的第一個問題是考慮到 Humira 生物相似藥的出現，如果 brepo 可能獲得批准，您預計它會如何定位？然後，您會在 CLARITY 中招募或對抗 TNF 類藥物抗藥性的患者嗎？

Yeah. Ben, do you want to take first crack at both of those?

是的。本，你想先試試這兩個嗎？

Yeah.

是的。

So what was -- the first question was just how do we try to position brepo relative to Humira biosimilar?

那麼第一個問題是我們如何嘗試將 brepo 相對於 Humira 生物相似藥定位？

Yeah.

是的。

And then the second was will we recruit refractory patients?

第二個問題是我們會招募難治性患者嗎？

Yeah.

是的。

So on the first one -- and you know, look, I think our base case kind of view of the market here is to be used predominantly in the TNF refractory population. And I think we're going into this with a lot of excitement about brepo's potential in NIU, even if that is the only population into which we're launching. I think, as I mentioned before, Humira's failure rate is high and its use is high. And I think the biosimilar -- with biosimilars available, we'd expect the use to be at least as high and the failure rate to be at least as high.

因此，關於第一個 - 你知道，看，我認為我們對市場的基本案例觀點是主要用於 TNF 難治人群。我認為我們對 brepo 在 NIU 中的潛力感到非常興奮，即使這是我們要推出的唯一人群。我認為，正如我之前提到的，修美樂的失敗率很高，而且使用率很高。我認為生物相似藥——有了生物相似藥，我們預計使用率至少會一樣高，失敗率至少也會一樣高。

And so I think that will only lead to an expansion of the TNF refractory market. I also think that this is a rare disease, very high unmet need, one of the leading causes of blindness in the United States. If our data is actually differentiated from Humira, there is going to be a very significant outcry from patients and physicians to use this drug first line because people don't want to go blind, and they want to use whatever the best available treatment is to prevent that.

因此我認為這只會導致 TNF 耐火材料市場的擴大。我也認為這是一種罕見的疾病，未滿足的需求非常高，是美國失明的主要原因之一。如果我們的數據實際上與 Humira 不同，那麼患者和醫生將會強烈抗議使用這種藥物作為第一線藥物，因為人們不想失明，他們希望使用任何最好的可用治療方法來治療防止這種情況發生。

And then just the study --

然後就是研究--

Yeah. And then, no. So there's no particular stratification or requirement in that regard. That's something we discussed with FDA. It's not something they are focused on in the study. I think our expectation is that we will be enrolling a number of patients who have been on prior TNF therapy, just given the extent to which these drugs are used, and we'll be tracking that and be able to analyze those subgroups, but it's not something that we're, in any way, stratifying for prespecifying.

是的。然後，不。因此，在這方面沒有特定的分層或要求。這是我們與 FDA 討論的事情。這不是他們研究的重點。我認為我們的期望是，我們將招募一些先前接受過 TNF 治療的患者，只要考慮到這些藥物的使用程度，我們將對其進行追蹤並能夠分析這些亞組，但這是無論如何，我們都不是為了預先指定而分層的。

Yeah. And I would just reiterate, tolerance for ocular inflammation is very low. And I think if brepocitinib proves as the Phase 2 data suggests may to be a best-in-class agent with a pretty wide margin, there's just going to be a lot of demand to use it in the earlier setting as people can get comfortable because that's how you treat this disease most effectively.

是的。我想重申一下，對眼部發炎的耐受性非常低。我認為，如果brepocitinib 被證明像2 期數據所表明的那樣，可能是一種同類最佳藥物，具有相當大的利潤空間，那麼在早期環境中就會有很多需求使用它，因為人們會感到舒服，因為這就是最有效治療這種疾病的方法。

So we'll see. It's going to be a conversation with FDA and so on. But we feel confident given the profile of the Phase 2 data that we have a good shot at a bigger population even the refractory population, acknowledging also refractory population is very large.

所以我們拭目以待。這將是與 FDA 等的對話。但鑑於第二階段的數據概況，我們有信心，我們有很好的機會獲得更大的人口，甚至是難治的人口，同時承認難治的人口也非常大。

Great. Thank you very much.

偉大的。非常感謝。

I would now like to hand the conference back over to Matthew Gline for any further remarks.

現在我想將會議交回給馬修·格萊恩（Matthew Gline），讓他發表進一步的評論。

Great. Thank you, operator. Thanks, everyone, for listening this morning. Thanks, obviously, to Ben and the Priovant team for the Phase 2 study NIU and for getting the Phase 3 going. Thanks to all the patients and investigators. Thanks to the entire Roivant team who gets these results together and then moves this forward every quarter. Looking forward to a little bit of a busy end of the year with the namilumab data coming and then a very busy 2025. So we'll talk to you all very soon. Thank you. Have a good day.

偉大的。謝謝你，接線生。謝謝大家今天早上的收聽。顯然，要感謝 Ben 和 Priovant 團隊進行 NIU 的第二階段研究以及第三階段的發展。感謝所有患者和研究人員。感謝整個 Roivant 團隊，他們將這些結果匯總在一起，然後每個季度都向前推進。隨著 namilumab 數據的到來，我們期待今年有點忙碌，然後是非常忙碌的 2025 年。所以我們很快就會和大家談談。謝謝。祝你有美好的一天。

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。