Belite Bio Inc (BLTE) 2025 Q1 法說會逐字稿

Ladies and gentlemen, thank you for joining us and welcome to the Belite Bio first-quarter 2025 earnings call. (Operator instructions)

女士們，先生們，感謝你們的到來，歡迎參加 Belite Bio 2025 年第一季財報電話會議。（操作員指示）

I will now hand the conference over to Julie Fallon. Please go ahead.

現在我將會議交給茱莉法倫。請繼續。

Hello, and thank you for joining us to discuss Belite Bio's first quarter 2025 financial results.

您好，感謝您加入我們討論 Belite Bio 2025 年第一季的財務表現。

Joining the call today are Dr. Tom Lin, Chairman and CEO of Belite Bio; Doctor Hendrick Scholl, Chief Medical Officer, Doctor Nathan Mata, Chief Scientific Officer, and Hao-Yuan Chuang, Chief Financial Officer.

今天參加電話會議的有 Belite Bio 董事長兼執行長 Tom Lin 博士；首席醫療官 Hendrick Scholl 博士、首席科學官 Nathan Mata 博士和財務長 Hao-Yuan Chuang。

Before we begin, let me point out that we will be making forward-looking statements that are based on our current expectations and beliefs. These statements are subject to certain risks and uncertainties, and actual results may differ materially.

在我們開始之前，請允許我指出，我們將根據我們當前的期望和信念做出前瞻性的陳述。這些聲明受一定風險和不確定性的影響，實際結果可能有重大差異。

We encourage you to consult the risk factors discussed in our SEC filings for additional detail.

我們鼓勵您查閱我們提交給美國證券交易委員會的文件中討論的風險因素以獲取更多詳細資訊。

Now, I'll turn the call over to Dr. Lin.

現在，我將把電話轉給林博士。

Thank you for joining today's call to discuss our first quarter of 2025 financial results. We continue to make good progress in this quarter towards advancing Tinlarebant in patients living with Stargardt disease and Geographic Strophy. For those who are new to our story, Tinlarebant is a first in class oral therapy intended to reduce the accumulation of toxic vitamin A byproducts, which is implicated in the progression of retinal lesions in patients with Stargardt disease and geographic atrophy.

感謝您參加今天的電話會議討論我們 2025 年第一季的財務業績。本季度，我們在推進 Tinlarebant 對患有 Stargardt 病和地圖樣萎縮症患者的治療方面繼續取得良好進展。對於剛了解我們故事的人來說，Tinlarebant 是一種一流的口服療法，旨在減少有毒維生素 A 副產物的積累，這種副產物與 Stargardt 病和地圖狀萎縮患者的視網膜病變進展有關。

We believe this approach will be effective in slowing or halting lesion growth, which would ultimately preserve vision. To give you some perspective on the importance of this potential therapy, Tinlarebant has been granted rare pediatric disease and fast track designations in the US and pioneer drug designations in Japan. It has also been granted orphan drug designation in the US, Europe, and Japan.

我們相信這種方法能夠有效減緩或阻止病變的生長，最終保住視力。為了讓您對這種潛在療法的重要性有所了解，Tinlarebant 已在美國獲得罕見兒科疾病和快速通道稱號，並在日本獲得先鋒藥物稱號。它也在美國、歐洲和日本被授予孤兒藥稱號。

We believe this speaks to this significant unmet need for both indications, as currently there is no approved treatment for Stargardt disease and no approved oral treatment for geographic atrophy. And more importantly, we are uniquely positioned as we are already in global Phase 3 trials for both indications.

我們認為這說明這兩種適應症都存在著巨大的未滿足需求，因為目前還沒有批准治療 Stargardt 病的方法，也沒有批准治療地圖樣萎縮的口服藥物。更重要的是，我們具有獨特的優勢，因為我們已經針對這兩種適應症進行了全球 3 期試驗。

So with that, let me provide a high level overview of the recent progress we have had made. We have two studies underway with Tinlarebant patients living with Stargardt disease.

因此，讓我對我們最近的進展進行一個高層次的概述。我們正在對患有 Stargardt 病的 Tinlarebant 患者進行兩項研究。

These are the Phase 3 Dragon trail and the Phase 2, 3 Dragon II trial. As part of the Phase 3 Dragon trial, we recently announced that the Data Safety Monitoring Board has completed its interim analysis, which is based on all subjects having completed the one year assessment period.

這些是第 3 階段 Dragon 試驗和第 2 階段、第 3 階段 Dragon II 試驗。作為第三階段 Dragon 試驗的一部分，我們最近宣布資料安全監測委員會已經完成了其中期分析，該分析基於所有受試者都完成了一年的評估期。

The DSMB recommended that the trial proceed without sample size increase or modifications. So essentially maintained the sample size at 104 subjects. In addition, they recommended that we submit the data for further regulatory review for drug approval. With the DSMB's review done, completion of a trial is on track for end of this year.

DSMB 建議繼續進行試驗，無需增加樣本量或進行修改。因此樣本量基本上維持在 104 個物件。此外，他們建議我們提交數據以供進一步監管審查以獲得藥品批准。隨著 DSMB 審查的完成，試驗預計將在今年年底完成。

The Dragon II trial continues to progress rapidly. We have enrolled 16 of our targeted enrollment of approximately 60 subjects, including about 10 Japanese subjects. Then from a Japanese subject is intended to expedite a new drug application in Japan, to which we have already been granted a pioneer drug designation.

龍飛船二號試驗持續快速進展。我們計劃招生約60名學生，目前已招收16名，其中包括約10名日文學生。然後從日本的主題來打算加快在日本的新藥申請，我們已經獲得了先鋒藥物的稱號。

In GA, we also continue to make progress in our clinical global Phase 3 Phoenix trial, which has already enrolled 464 subjects to date. We expect Phoenix trial to be fully enrolled for 500 subjects in Q3 this year.

在 GA，我們的全球臨床 3 期 Phoenix 試驗也持續取得進展，迄今已招募 464 名受試者。我們預計 Phoenix 試驗將在今年第三季招募 500 名受試者。

To summarize, with the excellent progress in our Phase 3 trials and promising interim results from Phase 3 Stargardt study, 2025 is off to a great start from a clinical perspective and our balance sheet is also strong with a four year cash runway. (inaudible) well positioned in advancing to the nerve band as potentially the first oral treatment for people living with degenerative retinal disease.

總而言之，憑藉我們第 3 階段試驗的出色進展和第 3 階段 Stargardt 研究的良好中期結果，從臨床角度來看，2025 年是一個良好的開端，我們的資產負債表也很強勁，擁有四年的現金流。 （聽不清楚）該藥物有望成為治療退化性視網膜疾病患者的首個口服神經帶藥物。

I'd like to now turn over the presentation to Nathan.

現在我想把演講交給 Nathan。

Thank you, Tom. So here I'll talk about the dragon clinical trials, both the Dragon I and Dragon II, as well as the in term analysis from the Dragon II study as Tom just mentioned. Here's an overview of the clinical trial designs in the Phase 3 trials for Dragon and Dragon II. These trials are designed nearly identically. There's only three differences in the trial designs, and those are highlighted in the top three rows, the first being the number of subjects, 104 in Dragon versus 60 in Dragon II.

謝謝你，湯姆。所以在這裡我將討論龍臨床試驗，包括龍 I 和龍 II，以及湯姆剛才提到的龍 II 研究的長期分析。以下是 Dragon 和 Dragon II 第 3 階段試驗的臨床試驗設計概述。這些試驗的設計幾乎相同。試驗設計只有三個不同之處，這些不同之處在前三行突出顯示，第一個是受試者的數量，Dragon 中為 104 人，而 Dragon II 中為 60 人。

The global nature of the Phase 3 design for Dragon versus a more localized geography for Dragon II in Japan, US, and UK, and the randomization is 2 to 1 in Dragon versus 1 to 1 in Dragon II because of the difference in the sample sizes. Other than that, all of the other parameters of the study are identical, and of course, the endpoint is exactly the same. That is slowing the growth of the atrophic lesions as measured by FAF photography.

Dragon 的第三階段設計具有全球性，而 Dragon II 的設計則更具在地化特點，主要集中在日本、美國和英國。由於樣本數不同，Dragon 的隨機性為 2 比 1，而 Dragon II 的隨機性為 1 比 1。除此之外，研究的所有其他參數都是相同的，當然，終點也完全相同。這減緩了 FAF 攝影所測量到的萎縮性病變的生長。

This is the FDA accepted endpoint. The other thing I should mention is that these studies both include the same dose, which is a 5 mg daily dose that reduces retinal binding protein [4] to about 80% below the baseline value. And at the very bottom there you can see the key inclusion criteria. All subjects in the dragon studies are 12 to 20 years old, and they have clinically and medically confirmed diagnosis of Stargardt disease.

這是 FDA 接受的終點。另外值得一提的是，這些研究都採用了相同的劑量，即每日 5 毫克的劑量，可將視網膜結合蛋白 [4] 降低至基線值以下約 80%。在最底部，您可以看到關鍵的納入標準。所有參與龍研究的對象年齡都在 12 至 20 歲之間，並且均已透過臨床和醫學確診患有 Stargardt 病。

Importantly, about the dragon trial, as Tom mentioned, there was an interim analysis. This was a pre-specified sample size reestimation in which the DSMB would take an unmasked look at the study data to determine whether or not there was a trend for efficacy in a so-called promising zone. This is a statistically identified window of conditional power that tells us there's a trend for efficacy.

重要的是，關於龍的審判，正如湯姆所提到的，有一個中期分析。這是一個預先指定的樣本量重新估計，其中 DSMB 將對研究數據進行未掩蔽的觀察，以確定所謂的有希望區域是否存在療效趨勢。這是一個統計上確定的條件功效窗口，它告訴我們功效存在趨勢。

If in fact there were a trend for efficacy noted by the DSMB, we would then be allowed to add 30 additional subjects to the study that would preserve and enrich the observation made at the interim so that we would have a better chance of seeing a statistically significant difference at the end of the second year.

如果 DSMB 確實指出了療效趨勢，那麼我們將被允許在研究中增加 30 名額外的受試者，以保留和豐富在此期間進行的觀察，以便我們有更好的機會在第二年末看到統計上顯著的差異。

That analysis, the interim analysis was triggered when the last patient had met his or her 12 month visit. The DSMB then took a look at the data and they decided there was no modification of the study that would be required and that we continue the study without a sample size increase. So that told us that we were not in this conditional zone of power called a promising zone.

當最後一位患者完成 12 個月的訪視時，就會觸發該分析，即中期分析。隨後，DSMB 查看了數據，並決定無需對研究進行任何修改，並且我們將繼續進行研究，而無需增加樣本量。所以這告訴我們，我們並不是處於這個被稱為有希望區的有條件的權力區。

We were either on the opposite side of that, which would be futile, or we're on the very positive side, which would be the overly efficacious side or unexpected efficacy. And in fact, we knew that we're probably very efficacious because the DSMB did provide an additional comment that they recommend we submit the data for further regulatory review for drug approval.

我們要么站在相反的一邊，這將是徒勞的，要么站在非常積極的一邊，這將是過於有效的一邊或意想不到的功效。事實上，我們知道我們可能非常有效，因為 DSMB 確實提供了額外的評論，他們建議我們提交數據以供進一步監管審查以獲得藥物批准。

That comment would not have been made if in fact we were on the futile side of that promising zone. We feel very optimistic and encouraged about this outcome. Importantly, at the time of the interim analysis, the overall withdrawal rate was less than 10%, so only 10% of 104 subjects withdrew of course, we don't know the breakout between placebo and active treatment because again this is blended data, but to have less than a 10% dropout when the majority of the study data has been analyzed is quite significant.

如果我們實際上處於這個充滿希望的區域的徒勞一側，那麼就不會發表這樣的評論了。我們對這一結果感到非常樂觀和鼓舞。重要的是，在中期分析時，總體退出率低於 10%，因此 104 名受試者中只有 10% 退出，當然，我們不知道安慰劑和積極治療之間的差異，因為這又是混合數據，但在分析了大部分研究數據後，退出率低於 10% 是非常重要的。

And importantly, the withdrawal due to ocular adverse events was only 3.8%, so only 4 of 104 subjects withdrawing because of ocular EEs. This is particularly important because the nature of our MOA would predict that there would be ocular adverse events. We know now from these study data that is very well tolerated and of course, mild in transit. That's very important.

重要的是，因眼部不良事件而退出研究的比例僅為 3.8%，因此 104 名受試者中只有 4 名因眼部 EE 而退出研究。這一點尤其重要，因為我們的 MOA 的性質預示著會發生眼部不良事件。我們現在從這些研究數據中知道，其耐受性非常好，當然，運輸過程也很溫和。這非常重要。

And then finally when we look at visual acuity, we find that visual acuity was stabilized in a majority of subjects with a mean change from baseline of less than three letters under both standard and low luminance throughout the two year study.

最後，當我們觀察視力時，我們發現在兩年的研究中，大多數受試者的視力都保持穩定，在標準和低亮度下，與基線的平均變化小於三個字母。

Here's the breakdown of the treatment emergent adverse events in the dragon trial. It's important to note that systemically there was only one drug-related adverse event, and that was acne, and this can happen in teens and preteens when vitamin A is diminished in the skin because vitamin A does help clean pores. So for diminishing it perhaps pores are not so clean, but it's a very mild AE to have systemically.

以下是龍試驗中治療出現的不良事件的細目。值得注意的是，系統性地只有一種與藥物相關的不良事件，那就是痤瘡，這種情況可能發生在青少年和青春期前兒童身上，因為維生素 A 確實有助於清潔毛孔。因此，對於減少它來說，毛孔可能不是那麼乾淨，但對於系統來說，這是一種非常溫和的不良反應。

Other than that, clinically significant findings in relation to vital signs were nothing in relation to physical exams, cardiac health, or organ function. What you see on the table are the outcomes from the two ocular AEs that we expected Xanthopsia and delayed dark notation. As mentioned, these were reported as mild and of course, transient.

除此之外，與生命徵象相關的臨床重要發現與身體檢查、心臟健康或器官功能無關。您在表格中看到的是我們預期的黃視症和延遲暗戀這兩種眼部不良事件的結果。如上所述，據報導這些症狀是輕微的，當然也是短暫的。

You can see on the right hand side the frequency and number of patients presenting with those AEs. The night vision impairment you see there, which is also mild is a more severe exacerbation of delayed dark adaptation. We had that in 15 reports of it. And then of course, we have a non-ocular headache in some subjects, which of course, can be manifest when subjects strain to use their visual acuity while experiencing these ocular AEs.

您可以在右側看到出現這些不良事件的患者的頻率和數量。您看到的夜視障礙也是輕微的，但卻是暗適應延遲的更嚴重的表現。我們在 15 份報告中都提到了這一點。當然，有些受試者會出現非眼部頭痛，這當然可以在受試者在經歷這些眼部不良事件時努力使用視力時表現出來。

But overall a very well tolerated and safe profile from an adverse event perspective. With respect to the visual acuity, I mentioned it was stabilization throughout the study trial.

但從不良事件的角度來看，整體而言，其耐受性非常好，而且很安全。關於視力，我提到在整個研究試驗過程中它是穩定的。

Since we have our CMO, Dr. Hendrik Scholl on the line, I'd like to get his clinical opinion on the BCVA data. Dr. Scholl?

由於我們的首席行銷長 Hendrik Scholl 博士在線，所以我想聽聽他對 BCVA 數據的臨床意見。肖爾博士？

Thank you, Nathan. The chart shows the EDTRS letter score in the study eye in orange and the fellow eye in gray over 24 months. This is blended data, meaning the Tinlarebant group and the placebo group are shown as one group.

謝謝你，內森。圖表顯示，研究眼的 EDTRS 字母評分為橘色，對側眼的 EDTRS 字母評分為灰色，持續 24 個月。這是混合數據，意味著 Tinlarebant 組和安慰劑組顯示為一個組。

The early treatment diabetic retinopathy study, or briefly ETDRS visual acuity testing is a standardized method used to measure visual acuity and is the gold standard in clinical trials and research settings. The EDTRS chart includes 14 lines with 5 letters per line and the maximum score on EDTRS visual acuity testing is 100.

早期治療糖尿病視網膜病變研究，簡稱 ETDRS 視力測試，是一種用於測量視力的標準化方法，是臨床試驗和研究環境中的黃金標準。EDTRS 圖表包含 14 行，每行 5 個字母，EDTRS 視力測驗的最高分數為 100。

Their significant intercession variability of ETDRS acuity measurements in normal subjects. Test retest variability typically falls within about five letters, and in individuals with macular degeneration, variability tends to be greater, namely about eight letters. What we see on the chart is that there was not even a single letter loss on average. Clearly this allows us to conclude that there was no significant loss of visual acuity, and it is fair to say there was stabilization of visual acuity in the Dragon trial.

它們對正常受試者的 ETDRS 視力測量具有顯著的干預變異性。重測變異性通常在五個字母左右，而對於患有黃斑部病變的個體，變異性往往較大，即大約八個字母。我們在圖表上看到，平均而言，甚至沒有丟失一個字母。顯然，這讓我們得出結論，視力沒有明顯下降，可以公平地說，在 Dragon 試驗中視力是穩定的。

I hand back over to Nathan.

我把麥克風交還給 Nathan。

Thank you, Hendrick. So moving forward now, a few words on our Phase 3 trial and geographic atrophy, which is called Phoenix. This is the overview of the trial design of Phoenix. You can see on the right hand side the various criteria for the study. This study is enrolling up to 500 subjects. It's a global study. The design is essentially the same as that for the Stargardt trials.

謝謝你，亨德里克。現在我們繼續前進，簡單談談我們的第三階段試驗和地理萎縮（稱為鳳凰城）。這是 Phoenix 試驗設計的概述。您可以在右側看到研究的各種標準。這項研究招募了多達 500 名受試者。這是一項全球性研究。設計與 Stargardt 試驗的設計基本相同。

In fact, the dose is exactly the same, 5 mg daily. It produces the same pharmacodynamic effect in older healthy adults as it does in young adolescent children, so we can use the same dose. It has the same endpoint. There's going to be the same trial duration. We will also be including an interim analysis. The timing and specifications of that analysis have not been worked out, but that will be conducted. And of course, that trial is still enrolling. We expect to close that enrollment sometime this summer, as again, as I said, up to 500 subjects.

事實上，劑量完全相同，每天 5 毫克。它在年長的健康成年人和青少年兒童身上產生的藥效學作用相同，因此我們可以使用相同的劑量。它具有相同的端點。試用期將會相同。我們也將進行中期分析。該分析的時間和規格尚未確定，但將會進行。當然，該試驗仍在招募中。我們預計將在今年夏天的某個時候結束招生，正如我所說，最多可招收 500 名學生。

So with that, I'll turn over to Hao-Yun for the financial results.

因此，我將把財務表現轉交給郝雲。

Thank you, Nathan. For Q1 2025, we had R&D expenses of $9.4 million compared to $6.8 million for the same period last year. The increase was primarily attributable to the share-based compensation and the slightly higher clinical trial expenses related to the Phoenix trial.

謝謝你，內森。2025 年第一季度，我們的研發費用為 940 萬美元，去年同期為 680 萬美元。成長主要歸因於股權激勵以及與鳳凰城試驗相關的略高的臨床試驗費用。

Regarding G&A expenses, it was $6.1 million compared to $1.6 million for the same period last year. The increase was also due to share-based compensation grant. Overall, we had a net loss of $14.3 million compared to a net loss of $7.9 million for the same period last year.

至於一般及行政開支，為 610 萬美元，去年同期為 160 萬美元。這一增長也歸因於股權激勵獎勵。整體而言，我們的淨虧損為 1,430 萬美元，而去年同期的淨虧損為 790 萬美元。

One thing to note is that as the majority of the increase of the expenses came from the share-based compensation, which was about $6.7 million and was not cash related, the operating cash outflow was only about $8.3 million.

需要注意的一點是，由於費用增加的大部分來自股權激勵費用（約 670 萬美元），且與現金無關，因此經營現金流出僅約 830 萬美元。

As we raised $15 million through the registered direct offering in February and received about $5.6 million from employee stock option exercise, we had a cash increase of $12.3 million for the quarter and leave us with $157.4 million in cash, liquidity funds, time deposit, and US Treasury bills as of end of Q1.

由於我們在 2 月透過註冊直接發行籌集了 1,500 萬美元，並透過員工股票選擇權行使獲得了約 560 萬美元，因此本季我們的現金增加了 1,230 萬美元，截至第一季末，我們的現金、流動資金、定期存款和美國國庫券餘額為 1.574 億美元。

We still expect four years of cash one way, and we expect to be able to complete all of our current clinical trials with this current cash. Thank you.

我們仍然期望單程獲得四年的現金，並且我們希望能夠利用這些現金完成我們目前的所有臨床試驗。謝謝。

Back to you operator.

回到你的操作員。

We will now begin the question and answer session. (Operator instructions)

我們現在開始問答環節。（操作員指示）

Mark Goodman, Leerink Partners.

Leerink Partners 的 Mark Goodman。

This is Basma on for Mark. Thank you for taking our question. Could you please, our first question is about the Phoenix trial. Could you provide us a update about the discontinuation rates, and how the enrollment is ongoing in this trial?

這是巴斯瑪為馬克上場的。感謝您回答我們的問題。請問，我們的第一個問題是關於鳳凰審判的。您能否向我們提供有關中止率的最新情況以及該試驗的招募進度？

And the second question, we have, it's about any updates on the regulatory meetings for the end points-- for the I'm sorry for the trial requirements for for Stargardt disease you mentioned that you were supposed to be working on, you're planning to meet up with us, the FDA and other regulatory agencies to finalize the plan, the development plan. That's it for us. Thank you so much.

第二個問題，我們有，是關於終點監管會議的任何更新 - 對於您提到的您應該進行的 Stargardt 病的試驗要求，我很抱歉，您計劃與我們、 FDA 和其他監管機構會面以最終確定計劃和開發計劃。對我們來說就是這樣。太感謝了。

Well, thank you. So I'll take those questions. So the first question is regarding the Phoenix dropout rates, is that right?

好的，謝謝你。所以我將回答這些問題。所以第一個問題是關於 Phoenix 的輟學率，對嗎？

Yes, correct.

是的，正確。

So, the dropout rate is approximately around about 20%. This is way below, what's been reported in previous studies, such as the derivative of vitamin A, Phase 3 that was reported, I think, earlier this year, there's a dropout of about 30%, and I believe that (inaudible) and other, anti-complements inhibitors in GA they were much more over 30% to 50%, I believe.

因此，輟學率大約在 20% 左右。這遠低於以往研究報告的結果，例如，我認為，今年早些時候報告的維生素 A 衍生物第 3 階段的退出率約為 30%，而且我相信（聽不清楚）以及其他抗補體抑製劑在 GA 中的退出率要高得多，我相信超過 30% 到 50%。

Hendrik, could you shed some light on that?

亨德里克，你能解釋一下嗎？

Yes, that is correct. I mean, it's not surprising that the injectables come with a higher risk and a higher dropout rate. And [amyostat] led to severe, night blindness, and there was also a higher dropout rate in the trial using [amyostat]as a visual cycle modulator.

是的，正確。我的意思是，注射劑具有更高的風險和更高的退出率，這並不奇怪。而 [amyostat] 會導致嚴重的夜盲症，且在使用 [amyostat] 作為視覺週期調節劑的試驗中，退出率也較高。

Yeah. So what's most surprising is that even the due rate of vitamin A had a more than 30% dropout rate. So we're way below that. So that's pretty encouraging, considering that we're conducting the trial in very elderly patients. I think the average is around about 80 year old. So, compliance is an issue, but we still have it within that 20%. So we're doing pretty well.

是的。因此最令人驚訝的是，即使是維生素A的應得率也有30%以上的輟學率。所以我們遠低於這個水平。考慮到我們是在非常年長的患者中進行試驗，這是非常令人鼓舞的。我認為平均年齡大約是80歲。因此，合規性是一個問題，但我們仍然將其控制在 20% 以內。所以我們做得很好。

And then the second question was, the Stargardt IA that we'll be discussing that with regulators. Is that correct, if I remember correctly?

第二個問題是，我們將與監管機構討論 Stargardt IA。如果我沒記錯的話，這是正確的嗎？

Yes, correct.

是的，正確。

Yeah, so we have, we have, Been scheduling meetings with the regulators, and we are doing so right now. We'll be meeting a few of those, we've scheduled some meetings in the short term. We'll be updating that as we go along. So right now we don't have much to report back on.

是的，我們一直在安排與監管機構的會議，我們現在正在這樣做。我們將會與其中一些人會面，我們已經安排了短期內的一些會議。我們將隨時更新。因此目前我們沒有太多可報告的資訊。

Got it. Thank you so much.

知道了。太感謝了。

Jennifer Kim, Cantor.

珍妮佛金（Jennifer Kim），領唱。

Hi, thanks for taking my questions. Maybe to start off in Stargardt, can you just talk about the recent interactions you've had with the FDA and any thoughts on maybe any perceived regulatory risk given the changes at the agency?

你好，謝謝你回答我的問題。也許從 Stargardt 開始，您能否談談您最近與 FDA 的互動，以及考慮到該機構的變化，您對任何可能的監管風險有何看法？

And then my second question is, with the Phase 3 data, I think it's coming in early 2026, can you just remind us what the goalpost is on efficacy and safety?

我的第二個問題是，根據第三階段的數據，我認為它將在 2026 年初出現，您能否提醒我們功效和安全性的目標是什麼？

So I'll take the first one. So, we haven't met up with the FDA yet. We'll be meeting up with them soon, so that, that's been scheduled. And then what's the other question?

所以我會選第一個。所以，我們還沒有與 FDA 會面。我們很快就會與他們會面，所以這已經安排好了。那麼另一個問題是什麼？

If you have any thoughts on, I guess, regulatory risk, given specifically that the changes that have been going on at the agency.

我想，如果您對監管風險有任何想法，請特別考慮該機構正在發生的變化。

No, I think we don't have any risk given that the data speaks for itself. So, even, I mean, even though there's some changes within the FDA, I don't think that's going to affect us, especially I think the division that we've met up with.

不，我認為我們沒有任何風險，因為數據說明了一切。所以，即使，我的意思是，即使 FDA 內部發生了一些變化，我也不認為這會影響我們，尤其是我認為我們已經遇到的部門。

Well, the division that, that's been giving us guidance throughout the whole study, especially during the Phase 3, most of them are still there. So I don't think there's any issues with the personnel change within the FDA.

嗯，該部門在整個研究過程中一直為我們提供指導，特別是在第三階段，他們中的大多數人仍然在那裡。所以我認為 FDA 內部的人事變動沒有任何問題。

Okay, and then can I ask on the goalpost for the Phase 3, the 24 month Phase 3 data?

好的，那麼我可以問一下第 3 階段的目標，也就是第 3 階段 24 個月的資料嗎？

Oh, I'll tell you for the Dragon. I think it will complete by Q3 this year.

哦，我會告訴你龍的事。我認為它將在今年第三季完成。

Nathan, you want to shed some light on that?

內森，你想解釋一下嗎？

I think Jennifer may be asking about what exactly we're we're looking for in terms of efficacy and safety, correct, Jennifer?

我認為詹妮弗可能會問我們在功效和安全性方面到底尋求什麼，對嗎，詹妮弗？

Oh, okay. So, Nathan, you want to answer this then?

哦好的。那麼，內森，你想回答這個問題嗎？

I was just going to mention that this study is powered to detect a 35% treatment effect between placebo and active. So that's what we're looking for. I mean, sometimes you get there, sometimes you don't, but based upon what the DSMB looked at and told us, in terms of safety and efficacy at the interim, which, by the way, as I mentioned, the majority of data were actually available at that time.

我只是想說，這項研究的目的是檢測安慰劑和活性藥物之間 35% 的治療效果。這就是我們所尋找的。我的意思是，有時你能達到目的，有時不能，但根據 DSMB 的觀察和告訴我們的情況，就當時的安全性和有效性而言，順便說一句，正如我提到的，當時大多數數據實際上都是可用的。

We're expecting that we'll be getting very close to our anticipated treatment effect size of 30% to 35%. Again, that's what the study's powered for. In terms of safety, I think the outlook is very positive, given again what we see at the interim less than a 10% overall withdrawal rate and 3.8% with respect to ocular AEs, again, because the majority of the study data had already been evaluated.

我們預計我們將非常接近預期的 30% 到 35% 的治療效果。再次強調，這正是這項研究的目的。在安全性方面，我認為前景非常樂觀，因為我們再次看到，目前總體退出率不到 10%，眼部不良事件發生率為 3.8%，同樣，因為大多數研究數據已經過評估。

By the time of the interim analysis, roughly 70% to 75% of the data was evaluated. I don't think that's going to change very much by the end of the year. So I expect those dropout rates to be fairly consistent, and as I said before, you never know about efficacy, but based upon what the DSMB saw at the interim, I'm expecting that we'll get very close to our anticipated treatment effect size.

到中期分析時，大約 70% 到 75% 的數據已被評估。我認為到今年年底這種情況不會有太大變化。因此我預計這些退出率會相當一致，正如我之前所說，你永遠不知道療效如何，但根據 DSMB 在此期間看到的情況，我預計我們會非常接近我們預期的治療效果大小。

Got it. That's helpful. Thanks, guys.

知道了。這很有幫助。謝謝大家。

Yi Chen, HCW.

陳毅，居家護理工作者。

All right, thank you for taking my questions. In view of President Trump's recent policy of most favored nation drug pricing, could you comment on your potential strategy regarding approval on launching of the drug in ex US territories, particularly Japan, and whether that will affect your US drug pricing and market prospects. Thank you.

好的，感謝您回答我的問題。鑑於川普總統最近推出的最惠國藥品定價政策，您能否評論一下您關於在美國以外領土（特別是日本）推出該藥品的潛在策略，以及這是否會影響您的美國藥品定價和市場前景。謝謝。

Yes, thanks for that. Well, first of all, since Tinlarebant has not launched yet, it's not going to affect us immediately. We're still observing how this is going to impact the industry as a whole, and then what type of drugs are affected or drugs, pediatric drugs, et cetera.

是的，謝謝。首先，由於 Tinlarebant 尚未推出，所以它不會立即對我們產生影響。我們仍在觀察這將如何影響整個行業，以及哪些類型的藥物會受到影響，例如兒科藥物等等。

Right now, we're still monitoring what's going on. So we don't have an immediate answer for that. I don't think anyone has an immediate answer for that, but we're still monitoring how it goes.

目前，我們仍在監視事態發展。所以我們對此還沒有立即的答案。我認為沒有人能立即給出答案，但我們仍在關注事態的進展。

Thank you. And a quick question on the financial side. Will the operating expenses continue to rise during the remainder of 2025 based on the level recorded in the first quarter?

謝謝。關於財務方面，我有一個簡短的問題。2025 年剩餘時間的營運費用是否會在第一季的基礎上繼續上漲？

I think it'll be about slightly higher than the Q1. We did, I think we did guide the market this year and the next year, we expect it to be higher expenses given that most of the milestone of the studies, all three studies expect to be reached in the next one, two years. Yeah, so the expenses will be higher this and next year, but, moving forward, you will come down back to the previous level.

我認為它會比第一季略高一些。我們確實，我認為我們確實引導了今年和明年的市場，我們預計費用會更高，因為研究的大部分里程碑，所有三項研究預計都將在未來一兩年內達到。是的，所以今年和明年的開支會更高，但是，展望未來，你會回到以前的水平。

Okay. Thank you.

好的。謝謝。

Thank you.

謝謝。

Bruce Jackson, Benchmark.

布魯斯傑克遜，基準。

Hi, good afternoon, and thanks for taking my questions. Can you hear me okay?

大家好，下午好，感謝您回答我的問題。你聽見我說話嗎？

Perfect.

完美的。

Just to follow up on that last question, we had the increase in stock comp during the quarter. Is that also expected to be a little bit higher going forward?

為了回答最後一個問題，我們在本季增加了股票補償。預計未來這數字還會更高嗎？

Well, so, it will really depends on the allocation of the expenses. So, you will have part of our (inaudible) will be only vested based on so-called, the development milestone. So, it's a little bit hard to say what exactly when, it will be allocated at and for the those that are going to be best based on time, it will be allocated by the period of the duration of the options. So it's a little bit hard to really, you give you a figure, but I think, given, we did have some of the ease up that just happened in that quarter. So, I don't expect it's going to be that high moving forward this year.

嗯，所以，這實際上取決於費用的分配。因此，我們的部分（聽不清楚）將僅基於所謂的發展里程碑而歸屬。因此，很難說具體什麼時候分配，對於那些基於時間最佳的選擇權，它將根據選擇權的持續時間進行分配。所以實際上很難給出一個數字，但我認為，考慮到那個季度我們確實出現了一些緩解。所以，我預計今年的漲幅不會那麼高。

Okay, great. And then last question from me is on manufacturing. Obviously there's a push to move manufacturing to the United States for pharmaceuticals. How do you, how are you set up right now in terms of inventory and how is your supply chain structured geographically?

好的，太好了。我的最後一個問題是關於製造業的。顯然，人們正在推動將藥品生產轉移到美國。您目前的庫存安排是怎麼樣的？您的供應鏈在地理上的結構是怎麼樣的？

Oh yeah, good question. So Tinlarebant is manufactured in the US, as well as in other geography. So, terrorism is not going to affect us anyway.

哦，是的，好問題。因此，Tinlarebant 不僅在美國生產，在其他地區也生產。所以，無論如何，恐怖主義不會影響我們。

Okay, super. All right. Thank you very much.

好的，太棒了。好的。非常感謝。

Thank you.

謝謝。

Michael Okunewitch, Maxim.

邁克爾·奧庫內維奇，馬克西姆。

Thank you. Thanks for taking my questions today, guys. So, I guess the first question I would like to ask is just if you could provide any more detail on the timing or any of the conditions that you would need to meet for that interim (inaudible) and geographic atrophy.

謝謝。謝謝你們今天回答我的問題。所以，我想我要問的第一個問題是，您是否可以提供有關時間安排或您需要滿足的任何條件的更多細節，以實現暫時（聽不清楚）和地理萎縮。

So we believe that, we've, so speaking on behalf of the MMA team, it's probably just me, and Hendrik. We are meeting with the regulators, we believe that what we got, what it takes to get us over the line, but, until we've met up having an official response from them, we're not going to disclose anything as of now.

所以我們相信，我們代表 MMA 團隊發言，可能只有我和亨德里克。我們正在與監管機構會面，我們相信我們所得到的資訊以及幫助我們跨越界限所需的條件，但是，在我們收到他們的正式回覆之前，我們現在不會透露任何資訊。

All right, fair enough. And then, are there any additional sample size re-estimations for the Phoenix study, or is that 500 patient count final as it looks like we're going to wrap up enrollment in the next couple of months.

好吧，夠公平。那麼，鳳凰研究是否有任何額外的樣本量重新估計，或者 500 名患者的數量是否是最終的，因為我們似乎將在接下來的幾個月內完成招募。

Yeah, so we're expecting to wrap up the enrollment because, the enrollment is going very smoothly as of now. We're making good progress. We don't believe that we would need extra additional subjects. We're just adding it on, just in case because we're having smooth enrollments, then why not just enroll more of that to, enroll more subjects to boost up our success.

是的，我們預計招生工作很快就會完成，因為目前招生工作進展非常順利。我們正在取得良好進展。我們認為我們不需要額外的附加科目。我們只是將其添加進去，以防萬一，因為我們的招生很順利，那麼為什麼不招收更多的學生，招收更多的科目來提高我們的成功率呢。

Nathan, do we have anything to add on?

內森，我們還有什麼要補充的嗎？

No, in fact, right now there's no plan to do a sample size re-estimation in Phoenix. As Tom said, we're sort of hedging our bets now that enrollment is going smoothly to enroll as many subjects as possible at the very start, so that we won't have to do a sample size re-estimation at the interim.

不，事實上，目前沒有計劃在鳳凰城進行樣本量重新估計。正如湯姆所說，既然招生工作現在進展順利，我們就採取一種規避風險的策略，在一開始就招收盡可能多的受試者，這樣我們就不必在中期重新估計樣本量。

All right. Thank you very much.

好的。非常感謝。

Thanks, Michael.

謝謝，麥可。

There are no further questions, so this concludes today's call. Thank you for joining us. You may now disconnect.

沒有其他問題了，今天的電話會議到此結束。感謝您加入我們。您現在可以斷開連線。