Liquidia Corp (LQDA) 2024 Q2 法說會逐字稿

Good morning, and welcome, everyone, to the Liquidia Corporation second quarter 2024 financial results and corporate update conference call. My name is Lisa, and I will be your conference operator today. [Operator Instructions] I would now like to remind everyone this conference call is being recorded.

早安，歡迎大家參加 Liquidia Corporation 2024 年第二季財務表現及公司更新電話會議。我叫麗莎，今天我將擔任你們的會議操作員。[操作員說明] 我現在想提醒大家，本次電話會議正在錄音。

I will now turn – hand the call over to Jason Adair, Chief Business Officer. Please go ahead.

現在我將把電話轉給首席商務官賈森·阿代爾 (Jason Adair)。請繼續。

Thank you, Lisa. It's my pleasure to welcome everyone to Liquidia Corporation's second quarter 2024 financial results and corporate update call. Joining the call today are Chief Executive Officer, Dr. Roger Jeffs; Chief Operating Officer and CFO, Michael Kaseta; Chief Medical Officer, Dr. Rajeev Saggar; Chief Commercial Officer, Scott Moomaw; and General Counsel, Rusty Schundler.

謝謝你，麗莎。我很高興歡迎大家參加 Liquidia Corporation 2024 年第二季財務業績和公司最新情況電話會議。今天加入電話會議的還有執行長 Roger Jeffs 博士；營運長兼財務長 Michael Kaseta；首席醫療官 Rajeev Saggar 博士；商務長 Scott Moomaw；和總法律顧問 Rusty Schundler。

Before we begin, please note that today's conference call will contain forward-looking statements, including those statements regarding future results, unaudited and forward-looking financial information, as well as the company's future performance and/or achievements. These statements are subject to known and unknown risks and uncertainties which may cause our actual results or performance to be materially different from any future results or performance expressed or implied on this call. For additional information, including a detailed discussion of our risk factors, please refer to the company's documents filed with the Securities and Exchange Commission, which can be accessed on our website.

在我們開始之前，請注意，今天的電話會議將包含前瞻性聲明，包括有關未來業績、未經審計和前瞻性財務資訊以及公司未來業績和/或成就的聲明。這些陳述受到已知和未知的風險和不確定性的影響，這些風險和不確定性可能導致我們的實際結果或業績與本次電話會議中明示或暗示的任何未來結果或業績存在重大差異。如需了解更多信息，包括對我們風險因素的詳細討論，請參閱公司向美國證券交易委員會提交的文件，這些文件可在我們的網站上訪問。

I would now like to turn the call over to Roger for our prepared remarks, after which he will open the call for your questions. Roger?

我現在想將電話轉給羅傑，讓他聽我們準備好的發言，然後他將開始電話會議，回答你們的問題。羅傑？

Thank you, Jason. Good morning, everyone. Thank you for joining us today. While we and patients still anxiously await FDA action on the YUTREPIA NDA seeking approval for both pulmonary arterial hypertension, PAH, and pulmonary hypertension associated with interstitial lung disease, or PH-ILD, we remain hopeful that we are close to achieving this. As a reminder, the FDA has hit no legal impediments since April 1st to take action on the amendment as submitted seeking approval for both PAH and PH-ILD.

謝謝你，傑森。大家早安。感謝您今天加入我們。雖然我們和患者仍在焦急地等待FDA 對YUTREPIA NDA 採取行動，尋求批准治療肺動脈高壓、PAH 和與間質性肺部疾病(PH-ILD) 相關的肺動脈高壓，但我們仍然希望我們即將實現這一目標。需要提醒的是，自 4 月 1 日以來，FDA 沒有遇到任何法律障礙，對提交的尋求批准 PAH 和 PH-ILD 的修正案採取行動。

What I can say today is that we have been in active and constructive communication with the FDA over these past four months, but we will not comment on the specifics of our conversations with the FDA. To be crystal clear, our medical and commercial teams remain on heightened alert, ready to launch YUTREPIA immediately upon approval. Our sales team continues to call on key PAH accounts, strengthening relationships and educating them on Liquidia. And importantly, we are staging commercial product for rapid distribution to specialty pharmacies upon approval.

今天我能說的是，在過去的四個月裡，我們一直在與 FDA 進行積極和建設性的溝通，但我們不會就我們與 FDA 對話的具體細節發表評論。需要明確的是，我們的醫療和商業團隊仍保持高度警惕，準備在獲得批准後立即推出 YUTREPIA。我們的銷售團隊繼續拜訪主要的 PAH 客戶，加強關係並對他們進行 Liquidia 教育。重要的是，我們正在推出商業產品，以便在獲得批准後快速分發到專業藥房。

Moving to our clinical programs. The open-label ASCENT study of YUTREPIA in PH-ILD patients continues to ramp up in the number of active clinical sites, with parallel increases in patient screening and patient enrollment. We are pleased with the preliminary feedback from physicians suggesting that patients can readily titrate YUTREPIA to escalating therapeutic levels in these PH-ILD patients. While the ASCENT data needs to mature more, our early patient experience today suggests that the benefits of PRINT-formulated treprostinil delivered via low-effort inhaler parallels a very good experience observed in PAH patients.

轉向我們的臨床計劃。YUTREPIA 在 PH-ILD 患者的開放標籤 ASCENT 研究持續增加活躍臨床中心的數量，同時患者篩檢和患者入組數量也隨之增加。我們對醫生的初步回饋感到滿意，這些回饋表明患者可以輕鬆地將 YUTREPIA 滴定至這些 PH-ILD 患者的治療水平。雖然 ASCENT 數據需要進一步成熟，但我們今天的早期患者經驗表明，透過省力吸入器輸送 PRINT 配方的曲前列環素的益處與在 PAH 患者中觀察到的非常好的體驗相似。

For example, the median dose of YUTREPIA for patients currently enrolled beyond eight weeks in the ASCENT trial is 185.5 micrograms per treatment session, or approximately 21 breath equivalents of Tyvaso per session, with a top dose of 318 micrograms or approximately 36 breath equivalents. These doses are several orders of magnitude beyond the recommended 9 to 12 breath dose target of Tyvaso and exemplify the paradigm-shifting potential of YUTREPIA for PAH and PH-ILD patients, especially as it relates to tolerability and potentially durability. We plan to submit additional clinical data from the ASCENT trial at future medical conferences, so more to come on that.

例如，目前參加 ASCENT 試驗超過 8 週的患者的 YUTREPIA 中位數劑量為每次治療 185.5 微克，或每次治療約 21 次 Tyvaso 呼吸當量，最高劑量為 318 微克或約 36 次呼吸當量。這些劑量比 Tyvaso 建議的 9 至 12 次呼吸劑量目標高出幾個數量級，並例證了 YUTREPIA 對 PAH 和 PH-ILD 患者的範式轉移潛力，特別是因為它與耐受性和潛在的耐久性有關。我們計劃在未來的醫學會議上提交更多 ASCENT 試驗的臨床數據，因此還會有更多數據。

With respect to our sustained-release liposomal formulation of inhaled treprostinil, L606, the preliminary safety data and exploratory efficacy data from the first 28 patients switching from Tyvaso or Tyvaso DPI in our open-label clinical study has been highly encouraging. We continue to observe favorable tolerability and titratability profile of twice daily dosing of L606, likely attributable to the sevenfold lower Cmax, but with a similar systemic exposure over a 24-hour period compared with a 4 times a day dosing of inhaled treprostinil, all while using a rapid portable handheld breath actuated nebulizer.

關於我們的吸入曲前列環素L606 緩釋脂質體製劑，在我們的開放標籤臨床研究中，來自首批28 名從Tyvaso 或Tyvaso DPI 轉換的患者的初步安全性數據和探索性療效數據非常令人鼓舞。我們繼續觀察到每日兩次L606 給藥的良好耐受性和滴定性曲線，這可能歸因於Cmax 低七倍，但與每日4 次吸入曲前列環素相比，24 小時內的全身暴露量相似，同時使用快速便攜式手持式呼吸驅動霧化器。

The long-term safety data generated from this study has helped solicit favorable scientific advice from the European Medicines Agency, or the EMA, last month on our pivotal trial design, which was very consistent with the FDA's feedback from our Type C meeting in December. While we continue to observe these patients in the open-label study, our focus will now shift to our efforts to initiate the registrational global trial in patients with PH-ILD later this year.

這項研究產生的長期安全性數據幫助我們在上個月向歐洲藥品管理局 (EMA) 徵求了關於我們關鍵試驗設計的有利科學建議，這與 FDA 在 12 月 C 類會議上的反饋非常一致。雖然我們繼續在開放標籤研究中觀察這些患者，但我們現在的重點將轉向今年稍後啟動針對 PH-ILD 患者的註冊全球試驗。

At this time, I will turn the call over to Mike to summarize the second quarter financials results.

此時，我將把電話轉給麥克，總結第二季的財務表現。

Thank you, Roger, and good morning, everyone. I will briefly address our second quarter financial results found in today's press release. As you will see, revenue was $3.7 million for the second quarter of 2024, compared with $4.8 million in the same quarter 2023. Revenue was tied to our promotional agreement with Sandoz to commercialize treprostinil injection. The decrease was primarily due to lower sales quantities in the current year as compared to the same period in the prior year. Cost of revenue increased to $1.5 million for the second quarter 2024, compared to $0.7 million in the same quarter for 2023, with the increase being primarily due to our sales force expansion during the fourth quarter of 2023.

謝謝你，羅傑，大家早安。我將簡要介紹今天新聞稿中的第二季財務業績。正如您將看到的，2024 年第二季的收入為 370 萬美元，而 2023 年同一季度的收入為 480 萬美元。收入與我們與山德士就曲前列環素注射液商業化的促銷協議掛鉤。減少的主要原因是本年度銷量較上年同期減少。2024 年第二季的收入成本增加至 150 萬美元，而 2023 年同一季度的收入成本為 70 萬美元，增加的主要原因是我們在 2023 年第四季銷售團隊的擴張。

Research and development expenses were $9.4 million in the second quarter of 2024, compared with $17.7 million in 2Q 2023 which included a $10 million upfront license fee to Pharmosa for the exclusive license to L606 in North America. We saw a $1.4 million decrease in expenses related to our YUTREPIA program driven by expensing prelaunch inventory costs in the prior year. These decreases were offset by a $1.7 million increase in clinical expenses related to our L606 program and a $1.5 million increase in personnel expenses related to increased head count.

2024 年第二季的研發費用為 940 萬美元，而 2023 年第二季的研發費用為 1,770 萬美元，其中包括向 Pharmosa 支付的 1,000 萬美元的預付費用，用於 L606 在北美的獨家許可。由於上一年啟動前庫存成本的支出，我們發現與 YUTREPIA 計劃相關的費用減少了 140 萬美元。這些減少被與我們的 L606 計畫相關的臨床費用增加 170 萬美元和與人數增加相關的人員費用增加 150 萬美元所抵消。

General and administrative expenses were $20 million in the second quarter of 2024, compared to $9.2 million in the same quarter for 2023. The increase of $10.8 million was primarily due to a $6.3 million increase in personnel expenses, which includes stock-based compensation, a $2.2 million increase in commercial and consulting expenses, and a $0.9 million increase in legal fees related to our ongoing YUTREPIA-related litigation. In summary, we incurred a net loss for the three months ended June 30, 2024 of $27.9 million, or $0.37 per basic and diluted share, compared to a net loss of $23.5 million, or $0.36 per basic and diluted share, for the three months ended March 31, 2023. We ended the second quarter of 2024 with $133 million cash on hand, and remain well positioned financially to achieve our corporate objectives this year.

2024 年第二季的一般和管理費用為 2,000 萬美元，而 2023 年第二季的一般和管理費用為 920 萬美元。增加 1,080 萬美元主要是由於人事費用增加 630 萬美元，其中包括股票薪酬、商業和諮詢費用增加 220 萬美元，以及與我們正在進行的 YUTREPIA 相關訴訟相關的法律費用增加 90 萬美元。總之，截至2024 年6 月30 日的三個月，我們的淨虧損為2,790 萬美元，即每股基本股和稀釋股0.37 美元，而這三個月的淨虧損為2,350 萬美元，即每股基本股和稀釋股0.36 美元截至 2023 年 3 月 31 日。截至 2024 年第二季末，我們手頭現金為 1.33 億美元，並在財務上保持良好狀態，可實現今年的企業目標。

With that, I'd like to now turn the call back over to Roger.

有了這個，我現在想把電話轉回給羅傑。

At this time, I will turn the call over to Mike to summarize the second quarter financial results.

此時，我將把電話轉給麥克，總結第二季的財務表現。

Thank you, Mike. As you've just heard, it's been an active summer on several fronts since our last call. We are fully prepared for the potential launch of YUTREPIA with a team of dedicated professionals who are poised to reshape and grow the market for inhaled treprostinil upon YUTREPIA's approval. The market opportunity for inhaled treprostinil is currently at a $1.5 billion run rate, with the potential to grow in excess of $3 billion in the coming years. This market remains keen for a competitive alternative, especially one with the dosing and tolerability advantages that YUTREPIA can potentially provide once approved.

謝謝你，麥克。正如您剛剛聽到的，自從我們上次通話以來，今年夏天在多個方面都表現活躍。我們已為 YUTREPIA 的上市做好了充分準備，擁有一支敬業的專業團隊，他們準備在 YUTREPIA 獲得批准後重塑和發展吸入曲前列環素市場。吸入曲前列環素的市場機會目前為 15 億美元，未來幾年有可能成長超過 30 億美元。該市場仍然熱衷於一種有競爭力的替代品，尤其是一種具有劑量和耐受性優勢的替代品，一旦獲得批准，YUTREPIA 就可能提供這種優勢。

With that, I would now like to open the call to questions. Operator, first question please.

現在我想開始提問。接線員，第一個問題請。

Thank you. [Operator Instructions] Julian Harrison of BTIG.

謝謝。[操作指示] BTIG 的 Julian Harrison。

Hi, good morning. Thank you for taking my questions and congrats on the recent progress. I'm wondering if we could briefly review why higher inhaled treprostinil exposure should be beneficial both in PAH and PH-ILD.

嗨，早安。感謝您回答我的問題並祝賀我最近的進展。我想知道我們是否可以簡要回顧為什麼較高的吸入曲前列環素暴露對於 PAH 和 PH-ILD 都有益。

Yes, Julian. Thanks very much for the question. So I'll start and then I'll ask Rajeev to add some additional color. So what we know historically from the use of prostacyclins is that – and one of the beauties of prostacyclins in particular is the ability to continually titrate to effect over time, because, unfortunately, these patients have an advancing disease that continues without relent. So the only way – the only therapeutic class that can address this progressive disease and remain a sort of an ability to tweak, are prostacyclins. So if you look at treprostinil in its various forms, parenteral, oral, inhaled, you can see that the ability to titrate is a hallmark of the therapy. But what had been limiting prior to YUTREPIA in particular, as you see with Tyvaso, is the inability to titrate above a fairly low therapeutic ceiling.

是的，朱利安。非常感謝您的提問。所以我會開始，然後我會要求 Rajeev 添加一些額外的顏色。因此，我們從歷史上使用前列環素的過程中了解到，前列環素的優點之一是能夠隨著時間的推移不斷滴定以達到效果，因為不幸的是，這些患者的病情正在不斷惡化，而且不會有任何緩解。因此，唯一的方法——唯一能夠解決這種進行性疾病並保持某種調整能力的治療類別是前列環素。因此，如果您觀察曲前列環素的各種形式（胃腸外、口服、吸入），您會發現滴定能力是此療法的標誌。但在 YUTREPIA 之前，尤其是在 Tyvaso 中所看到的限制是無法在相當低的治療上限之上進行滴定。

So what YUTREPIA has done, and this is why I say it's paradigm shifting, it's giving you all the benefits of the parenteral and oral products, but giving it directly to the site of action to limit the systemic side effects. So you now have a highly flexible therapeutic with high utility that can really be dosed to effect, also while minimizing the side effect. So I think this is why we're so excited about YUTREPIA. We think it brings a different sort of utility to the marketplace in terms of treprostinil use. And we think this therapeutic profile will lead it to become both best-in-class and first-in-choice when considering starting a prostacyclin, be it parenteral, oral or inhaled. Rajeev, I don't know if you have any additional comments.

YUTREPIA 所做的就是為什麼我說它是範式轉變，它為您提供了腸外和口服產品的所有好處，但將其直接給予作用部位以限制全身副作用。因此，您現在擁有了一種高度靈活且實用的治療方法，可以真正按劑量達到效果，同時最大限度地減少副作用。所以我認為這就是我們對 YUTREPIA 如此興奮的原因。我們認為，就曲前列環素的使用而言，它為市場帶來了不同類型的效用。我們認為，在考慮開始使用前列環素（無論是腸胃外、口服或吸入）時，這種治療特性將使它成為同類最佳和首選。Rajeev，不知道您是否還有其他意見。

Yes, Julian, yes, I think Roger answered most of it correctly. I would just sort of add, is that we learned a lot from the INCREASE study in PH-ILD. I think in that study, the data highlighted that patients that achieve more than at least 9 breaths, and especially as you get to higher doses, those patients – the clinical observations was that they walked farther, and they had also improvements in many of the secondary outcomes. Also, I think this is a very heterogeneous group of patients, both in PAH and PH-ILD, with various degrees of severity. And in many of these patients, the disease, as Roger highlighted, it's progressive. The opportunity to continue to titrate and match disease severity and temper that down, I think, is going to be a clear advantage and a great armamentarium for clinicians as well as for the outcomes for patients.

是的，朱利安，是的，我認為羅傑回答了大部分。我想補充一點，我們從 PH-ILD 的 INCREASE 研究中學到了很多。我認為在那項研究中，數據強調了那些呼吸次數至少超過9 次的患者，尤其是當您接受更高劑量時，這些患者- 臨床觀察結果是他們走得更遠，而且他們在許多方面也有改善。另外，我認為這是一個非常異質的患者群體，無論是 PAH 還是 PH-ILD，都有不同程度的嚴重程度。正如羅傑所強調的，在許多患者中，這種疾病是進行性的。我認為，繼續滴定和匹配疾病嚴重程度並降低其嚴重程度的機會，對於臨床醫生以及患者的治療結果來說將是一個明顯的優勢和一個很好的武器庫。

Thanks for the question.

謝謝你的提問。

Okay, great. And I have one more, if I may. I was just curious if you have a good sense for when we might expect a comprehensive data disclosure from the ASCENT trial. And can we maybe review what the key unanswered questions are that you plan to address with that trial?

好的，太好了。如果可以的話，我還有一份。我只是好奇你是否清楚我們何時可以期待 ASCENT 試驗的全面數據披露。我們能否回顧一下您計劃透過該試驗解決哪些關鍵的未解答問題？

Yeah. Maybe, Rajeev, if you could comment on that.

是的。也許，拉吉夫，如果你能對此發表評論的話。

Yeah, Julian. Thanks very much for the question. So I'll start and then I'll ask Rajeev to add some additional color. So what we know historically from the use of prostacyclins is that – and one of the beauties of prostacyclins in particular is the ability to continually titrate to effect over time, because, unfortunately, these patients have an advancing disease that continues without relent. So the only way – the only therapeutic class that can address this progressive disease and remain a sort of an ability to tweak, are prostacyclins. So if you look at treprostinil in its various forms, parenteral, oral, inhaled, you can see that the ability to titrate is a hallmark of the therapy. But what had been limiting prior to YUTREPIA in particular, as you see with Tyvaso, is the inability to titrate above a fairly low therapeutic ceiling.

是的，朱利安。非常感謝您的提問。所以我會開始，然後我會要求 Rajeev 添加一些額外的顏色。因此，我們從歷史上使用前列環素的過程中了解到，前列環素的優點之一是能夠隨著時間的推移不斷滴定以達到效果，因為不幸的是，這些患者的病情正在不斷惡化，而且不會有任何緩解。因此，唯一的方法——唯一能夠解決這種進行性疾病並保持某種調整能力的治療類別是前列環素。因此，如果您觀察曲前列環素的各種形式（胃腸外、口服、吸入），您會發現滴定能力是此療法的標誌。但在 YUTREPIA 之前，尤其是在 Tyvaso 中所看到的限制是無法在相當低的治療上限之上進行滴定。

So what YUTREPIA has done, and this is why I say it's paradigm shifting, it's giving you all the benefits of the parenteral and oral products, but giving it directly to the site of action to limit the systemic side effects. So you now have a highly flexible therapeutic with high utility that can really be dosed to effect, also while minimizing the side effect. So I think this is why we're so excited about YUTREPIA. We think it brings a different sort of utility to the marketplace in terms of treprostinil use. And we think this therapeutic profile will lead it to become both best-in-class and first-in-choice when considering starting a prostacyclin, be it parenteral, oral or inhaled. Rajeev, I don't know if you have any additional comments.

YUTREPIA 所做的就是為什麼我說它是範式轉變，它為您提供了腸外和口服產品的所有好處，但將其直接給予作用部位以限制全身副作用。因此，您現在擁有了一種高度靈活且實用的治療方法，可以真正按劑量達到效果，同時最大限度地減少副作用。所以我認為這就是我們對 YUTREPIA 如此興奮的原因。我們認為，就曲前列環素的使用而言，它為市場帶來了不同類型的效用。我們認為，在考慮開始使用前列環素（無論是腸胃外、口服或吸入）時，這種治療特性將使它成為同類最佳和首選。 Rajeev，不知道您是否還有其他意見。

Okay, great. And I have one more, if I may. I was just curious if you have a good sense for when we might expect a comprehensive data disclosure from the ASCENT trial. And can we maybe review what the key unanswered questions are that you plan to address with that trial?

好的，太好了。如果可以的話，我還有一份。我只是好奇你是否清楚我們何時可以期待 ASCENT 試驗的全面數據披露。我們能否回顧一下您計劃透過該試驗解決哪些關鍵的未解答問題？

es, Julian. So again, just to remind everyone, ASCENT is a study studying the safety and tolerability, and the secondary outcomes would be exploratory efficacy of YUTREPIA in patients with PH-ILD, who have not been treated. The patients have to enroll with the baseline right heart catheterization. The study is actually for 24 months, and then we follow the patient out to one year. So what is very important here in the study is that what we're looking for is to maintain the patients on a – the optimal dose of YUTREPIA, and also to show durability. Because what we do know is that after 16 weeks in INCREASE study, many of these patients start to have a significant amount of instability. So we wanted to show patients definitely have durability.

是的，朱利安。再次提醒大家，ASCENT 是一項研究安全性和耐受性的研究，次要結果是 YUTREPIA 對未經治療的 PH-ILD 患者的探索性療效。患者必須參加基線右心導管檢查。該研究實際上持續 24 個月，然後我們對患者進行一年的追蹤。因此，本研究中非常重要的是，我們所尋求的是讓患者維持 YUTREPIA 的最佳劑量，並顯示出持久性。因為我們所知道的是，在 INCREASE 研究 16 週後，許多患者開始出現明顯的不穩定。所以我們想向患者展示絕對具有耐久性。

Our focus really, coming out in the next set of congresses that are approaching here, is really to highlight some of this exploratory clinical efficacy data. We are following clinical variables such as walk distance in terms of six-minute walk. We're also looking at various forms of questionnaires. And finally, we're also looking at effects on the lung parenchyma and the lung vasculature by using CT chest imaging, really to highlight really where the effect is, particularly on the pulmonary vasculature, with the use of YUTREPIA.

在即將舉行的下一組大會上，我們的重點實際上是強調一些探索性臨床療效數據。我們正在追蹤臨床變量，例如以六分鐘步行為單位的步行距離。我們也正在研究各種形式的問卷。最後，我們也透過使用 CT 胸部影像來研究對肺實質和肺脈管系統的影響，真正強調使用 YUTREPIA 的效果，特別是對肺脈管系統的影響。

I just want to highlight, as Roger said, is that at least right now, the median dose for those patients now past the eight-week mark is now 185.5 micrograms of YUTREPIA. This is just to highlight that, if you compare this to our sentinel INSPIRE study in PAH, by two years, patients were on – about a third of the patients were on 159 micrograms. And so what we're seeing here is that providers and patients in particular are able to tolerate YUTREPIA. And there is also, just to go back to our last question, a very clear understanding by providers that dose – the higher the dose, the potential to benefit of the patient. And I think we look forward to highlighting some of these clinical parameters in the very near future.

正如羅傑所說，我只是想強調的是，至少現在，那些已經過了八週的患者的 YUTREPIA 中位數劑量現在是 185.5 微克。這只是為了強調，如果將此與我們的 PAH 前哨 INSPIRE 研究進行比較，兩年後，患者的劑量為 159 微克——大約三分之一的患者服用了 159 微克。因此，我們在這裡看到的是，提供者和患者尤其能夠耐受 YUTREPIA。回到我們的最後一個問題，醫療服務提供者對劑量有一個非常清晰的認知——劑量越高，患者受益的潛力就越大。我認為我們期待在不久的將來強調其中一些臨床參數。

Thanks, Rajeev. So Julian, as you can hear very robust study. We're trying to complete it by year-end, to specifically answer your question. And then we'll look to publish that in 2025. Operator, next question.

謝謝，拉吉夫。朱利安，你可以聽到非常有力的研究。我們正努力在年底前完成它，以專門回答您的問題。然後我們將在 2025 年發布該內容。接線員，下一個問題。

Excellent. Thank you, Roger.

出色的。謝謝你，羅傑。

Serge Belanger of Needham.

李約瑟的謝爾蓋·貝蘭格 (Serge Belanger)。

Hi, good morning. Thanks for taking my question. I have a couple of legal ones. First one, on the UT case against FDA, I believe, in the motion for dismissal, both the agency and Liquidia completed their briefs. So just curious if you have any visibility on time lines for the next steps here, whether we'll get a hearing or judge-based rule on the briefs. And related to this case, how closely is FDA following this dismissal motion? And how could that be a gating factor for them rendering a decision on the YUTREPIA NDA? Thanks.

嗨，早安。感謝您提出我的問題。我有幾個合法的。第一個，關於 UT 針對 FDA 的案件，我相信，在駁回動議中，該機構和 Liquidia 都完成了他們的陳述。因此，我想知道您是否對下一步的時間表有任何了解，我們是否會就案情摘要進行聽證會或基於法官的規則。與此案相關的是，FDA 對這項駁回動議的關注程度如何？這怎麼可能成為他們就 YUTREPIA NDA 做出決定的限制因素？謝謝。

Thanks Serge, Thanks for the question. Rusty?

謝謝塞爾吉，謝謝你的提問。生鏽了？

Yes, Serge, thanks for the question. So on the – taking your first question first. On the motion to dismiss, next steps, we don't know yet. The court could have an oral argument or it could rule on the briefs. We don't have an indication either way yet from the court. So really can't provide any guidance there just because we're waiting for the court to decide on that. On the second point, obviously, it's tough for us to comment on what's in the FDA's said. And I think as Roger said in his opening remarks, I think we want to be careful not to comment on our communications with the FDA. So obviously, once the FDA has taken definitive action, obviously, we will announce that. But in the meantime, we're not going to comment on our communications with the FDA.

是的，塞爾日，謝謝你的提問。那麼，先回答你的第一個問題。關於駁回動議的後續步驟，我們還不知道。法院可以進行口頭辯論，也可以對案情摘要做出裁決。我們還沒有從法庭得到任何跡象。所以真的無法提供任何指導，因為我們正在等待法院對此做出決定。顯然，關於第二點，我們很難對 FDA 的說法發表評論。我認為正如羅傑在開場白中所說，我認為我們要小心，不要對我們與 FDA 的溝通發表評論。顯然，一旦 FDA 採取明確行動，我們就會宣布這一點。但同時，我們不會對與 FDA 的溝通發表評論。

Kambiz Yazdi of Jefferies.

傑富瑞 (Jefferies) 的卡姆比茲·亞茲迪 (Kambiz Yazdi)。

Good morning team. For the registrational study for L606, can you remind us the key features for that study design, and when is that slated to start? And then maybe just a few finer points on the ASCENT study. How many more sites have you onboarded since the last update? And how many patients so far have you treated in the ASCENT study? Thank you.

早上好，團隊。對於 L606 的註冊研究，您能否提醒我們該研究設計的主要特點以及計劃何時開始？然後也許只是關於 ASCENT 研究的一些細節。自上次更新以來，您又登入了多少個網站？到目前為止，您在 ASCENT 研究中治療了多少名患者？謝謝。

Good questions. Rajeev, both of those are in your court, please.

好問題。拉吉夫，這兩個人都在你的法庭上，請你。

Yes. Hi, Kambiz So regarding the global single placebo-controlled efficacy study with L606, specifically in patients with pulmonary hypertension associated with interstitial lung disease, we plan to initiate that study by the end of the year. I think we're working feverishly to do all the necessary steps to make sure that our protocols are appropriately submitted and viewed by agencies. As Roger highlighted, we've had favorable feedback from both the Type C meeting with the FDA and also from the scientific advice from the European Medicines Agency. So I think we remain pretty confident in our plans.

是的。您好，Kambiz 因此，關於 L606 的全球單一安慰劑對照療效研究，特別是針對與間質性肺部疾病相關的肺動脈高壓患者，我們計劃在今年年底啟動研究。我認為我們正在積極採取一切必要措施，以確保我們的協議得到各機構的適當提交和查看。正如 Roger 所強調的那樣，我們從 FDA 的 C 類會議以及歐洲藥品管理局的科學建議中獲得了積極的回饋。所以我認為我們對我們的計劃仍然充滿信心。

We've already highlighted the primary endpoint in this study is going to be 6-minute walk distance, with a whole host of secondary efficacy endpoints that I think would be important to support our target profile. I think we're extremely confident, based on the safety data that's emerging from the open-label study in the United States. Again, just highlighting the liposomal technology on top of the treprostinil, I think really has shown to minimize cost and really supports dosing and titratability of this drug, I think, which is going to be a game changer, along with a reduced frequency to twice a day.

我們已經強調了這項研究的主要終點將是 6 分鐘步行距離，還有一系列次要功效終點，我認為這些終點對於支持我們的目標概況非常重要。根據美國開放標籤研究得出的安全數據，我認為我們非常有信心。再次強調，曲前列環素之上的脂質體技術，我認為確實已經證明可以最大限度地降低成本，並且真正支持這種藥物的劑量和滴定性，我認為這將改變遊戲規則，同時將頻率減少到兩倍一天。

So we remain very, very excited about the feedback that KOLs throughout the global market has been showing for support of the study. There's extremely significant amount of unmet need, I think, definitely outside the U.S., in addition to the U.S. in that regard.

因此，我們對全球市場的 KOL 為支持這項研究而提供的回饋感到非常非常興奮。我認為，除了美國之外，在美國之外肯定還有大量未滿足的需求。

In terms of ASCENT, we have – we're close to about tripling the number of sites by the end of this month, and we remain quite confident on completing the study by the end of the year. We anticipate to have close to about 15 patients by the end of this month. Just to highlight that the majority of the sites that have been just recently initiated have come on over the past 45 days, and we anticipate the remainder of sites to come on within the next 30 to 60 days as well. Then by that time, I think we'll have – we'll be on full ramp to support the rest of the study.

就 ASCENT 而言，我們已經——到本月底，我們的站點數量幾乎增加了兩倍，而且我們對在年底前完成這項研究仍然充滿信心。我們預計到本月底將有近 15 名患者。需要強調的是，大多數最近啟動的網站都是在過去 45 天內上線的，我們預計其餘網站也將在未來 30 到 60 天內上線。到那時，我想我們將會全力以赴支持研究的其餘部分。

Great. Thank you. Thank you, Rajeev. Thanks for the questions, Kambiz. As you can see, really positive data flow from both the L606 open-label study and the ASCENT trial, we're obviously very excited to get into the registrational study with L606 in PH-ILD patients, as Rajeev said. And I think the fact now that we're seeing the ability to titrate as easily as we are and as quickly as we are, as well as to see the durability of the 2 times a day format, that's an exciting Phase 3 program with a very positive predictive future. Same for the ASCENT trial, I think when you see this rapid ability to dose these patients, quite differentiating, and I think something that the market will lean on once we get to market. So thanks for the question, Kambiz. Next question please.

偉大的。謝謝。謝謝你，拉吉夫。謝謝你的提問，卡姆比茲。正如您所看到的，L606 開放標籤研究和 ASCENT 試驗都提供了非常積極的數據，我們顯然非常高興能在 PH-ILD 患者中開展 L606 註冊研究，正如 Rajeev 所說。我認為現在的事實是，我們已經看到了像我們一樣輕鬆、快速地進行滴定的能力，並且看到了每天 2 次形式的持久性，這是一個令人興奮的第 3 階段計劃，其中非常積極地預測未來。ASCENT 試驗也是如此，我認為當你看到這種快速給這些患者用藥的能力時，就非常與眾不同，而且我認為一旦我們進入市場，市場就會依賴這一點。謝謝你的提問，Kambiz。請下一個問題。

Matt Kaplan of Ladenburg Thalmann.

拉登堡塔爾曼的馬特卡普蘭。

Hi, good morning guys. Thanks for taking my question. I guess since we're past the kind of legal impediments for FDA approval, I guess, with your interaction with the FDA, has the FDA identified any material differences, which would prevents approval and would, I guess, necessitate a CRL?

嗨，大家早安。感謝您提出我的問題。我想，既然我們已經克服了 FDA 批准的法律障礙，我想，在您與 FDA 的互動中，FDA 是否發現了任何實質差異，這將阻止批准，並且我猜需要 CRL？

Hi, Matt. It's Roger. Thanks for the question. As I said in the intro, we're really not going to comment on specific discussions with the FDA. I think one way to look at this is, yes, we are past the time when there was no legal impediment for approval, that being April 1st, four months past that and while that's frustrating, I think if you want to put a positive spin on that, four months is in the rearview mirror, so there's been four months to work to a solution. So we think we're that much closer to a decision and an action. And we won't comment on direct communications with that, but we remain optimistic that we've had constructive discussions with the agency. And we remain highly focused on approval for both indications. So that's – as we submitted it, we feel it was appropriate as amended, and we are seeking approval for both PAH and PH-ILD. We haven't backed away from that position and that remains our focus. So, apologies that I can't give specifics, but hopefully we'll have an action soon and we can talk about that in great detail with you. Operator, next question.

嗨，馬特。是羅傑。謝謝你的提問。正如我在介紹中所說，我們真的不會對與 FDA 的具體討論發表評論。我認為看待這個問題的一種方式是，是的，我們已經過了批准沒有法律障礙的時代，即4 月1 日，四個月過去了，雖然這令人沮喪，但我認為如果你想積極樂觀的話在這個問題上，四個月的時間已經過去了，所以有四個月的時間來尋找解決方案。所以我們認為我們距離做出決定和採取行動已經更近了。我們不會就與該機構的直接溝通發表評論，但我們仍然樂觀地認為我們與該機構進行了建設性討論。我們仍然高度關注這兩種適應症的批准。因此，當我們提交該文件時，我們認為修訂後的文件是適當的，並且我們正在尋求 PAH 和 PH-ILD 的批准。我們沒有放棄這一立場，這仍然是我們的重點。因此，很抱歉我無法提供具體細節，但希望我們很快就會採取行動，並且可以與您詳細討論。接線員，下一個問題。

Thanks.

謝謝。

Our next question will be coming from the line of Jason Gerberry of Bank of America. Your line is open.

我們的下一個問題將來自美國銀行的 Jason Gerberry。您的線路已開通。

Hi, guys. Good morning. Thanks for taking my questions. So two for me. Just on the cash burn language in the 10-Q, about just roughly around a year of cash runway. I assume that that assumes an approval and launch costs. Can you talk about, in the alternative scenario, if there are delays, any flexibility to preserve the cash runway, any flexibility or levers that you could pull?

嗨，大家好。早安.感謝您回答我的問題。所以對我來說是兩個。僅就 10-Q 中的現金消耗而言，大約只有大約一年的現金跑道。我認為這需要批准和啟動成本。您能否談談在另一種情況下，如果出現延誤，保留現金跑道的任何靈活性，您可以採取的任何靈活性或槓桿？

And then my second question is just on United's citizens' petition, is there any plans to submit any correspondence clarifying LGM's role as an importer or – I assume that most of this is going to be handled behind the scenes, if at all. But just curious if there is any plans to submit any response? Thanks.

我的第二個問題是關於美聯航公民請願書，是否有計劃提交任何信件來澄清 LGM 作為進口商的角色，或者——我認為其中大部分將在幕後處理（如果有的話）。但只是好奇是否有計劃提交任何回應？謝謝。

Yes, great. Thanks, Jason. So Mike, if you'll address the cash burn question, and Rusty, if you could talk to the citizens' petition, please?

是的，太好了。謝謝，傑森。麥克，請您解決燒錢問題，拉斯蒂，請您談談公民請願書，好嗎？

Yes. So Jason, thanks for the question. I mean I think where we sit now, we are sitting at $133 million of cash. We're very confident in our cash position. We talked about all of our objectives for the rest of this year and going forward with ASCENT, without getting L606 initiated, and also supporting the launch of YUTREPIA. We've always taken pride of having a strong balance sheet. We feel that we still have that. And we're very confident in our ability to deliver.

是的。傑森，謝謝你的提問。我的意思是，我認為我們現在擁有 1.33 億美元的現金。我們對我們的現金狀況非常有信心。我們討論了今年剩餘時間的所有目標，以及在不啟動 L606 的情況下繼續推進 ASCENT，並支持 YUTREPIA 的推出。我們一直為擁有強大的資產負債表而感到自豪。我們覺得我們仍然擁有這一點。我們對我們的交付能力非常有信心。

Now with that being said, we'll always focus on what's at hand, and depending on where things stand, we can make decisions, we will be able to do that. But our focus right now really is to deliver on all three of those objectives, of getting YUTREPIA launched, getting L606 pivotal trial initiated, and continuing the ASCENT trial. So when you look at our cash balance, I think our burn from year-end to Q1 to Q2, we've always been disciplined in how we invest. We will continue to do that and look forward to hopefully a YUTREPIA launch here in the not too distant future.

話雖如此，我們將始終關注手頭上的事情，根據情況的不同，我們可以做出決定，我們將能夠做到這一點。但我們現在的重點實際上是實現所有這三個目標，即啟動 YUTREPIA、啟動 L606 關鍵試驗以及繼續 ASCENT 試驗。因此，當你查看我們的現金餘額時，我認為從年底到第一季到第二季的資金消耗，我們一直在投資方式上遵守紀律。我們將繼續這樣做，並期待在不久的將來 YUTREPIA 在這裡推出。

And Jason, thanks for the question on the citizens' petition. We do not currently anticipate filing a public response to the citizens' petition. Obviously, any issues that – there will be direct communications between us and the FDA, which, as Roger said before, we won't comment on. The other thing I'd point out is, I mean, even United Therapeutics had to file an amended citizens' petition to cure some of the misstatements on the original citizens' petition that is public. But again, we won't have a public response, or at least we're not anticipating one at this time.

傑森，感謝您提出有關公民請願書的問題。我們目前預計不會對公民的請願書做出公開回應。顯然，任何問題我們和 FDA 之間都將進行直接溝通，正如羅傑之前所說，我們不會對此發表評論。我要指出的另一件事是，我的意思是，即使是聯合治療公司也必須提交一份修改後的公民請願書，以糾正原始公開的公民請願書上的一些錯誤陳述。但同樣，我們不會做出公開回應，或至少我們目前不期待公開回應。

Okay. Thank you.

好的。謝謝。

Thank you. And this does conclude today's Q&A session. And I would now like to turn the call back over to Roger for closing remarks.

謝謝。今天的問答環節到此結束。現在我想將電話轉回給羅傑，讓他發表結束語。

Please go ahead.

請繼續。

Great. Well, again, I want to thank everybody for joining us today. As you've clearly heard, we remain confident in the approvability of YUTREPIA in the near term for both PAH and PH-ILD and the competitive profile once launched. We look forward to updating you in the near future. Thank you

偉大的。好吧，我想再次感謝大家今天加入我們。正如您所清楚聽到的，我們對 YUTREPIA 在近期內對 PAH 和 PH-ILD 的批准以及一旦推出後的競爭狀況仍然充滿信心。我們期待在不久的將來為您提供最新消息。謝謝

Thank you for joining today's conference call. You may disconnect.

感謝您參加今天的電話會議。您可以斷開連線。