Soleno Therapeutics Inc (SLNO) 2025 Q2 法說會逐字稿

Greetings and welcome to the Soleno second quarter 2025 earnings conference call.

大家好，歡迎參加 Soleno 2025 年第二季財報電話會議。

(Operator Instructions) It's now my pleasure to turn the call over to Brian Ritchie of LifeSci Advisors. Please go ahead, Brian.

（操作員指示）現在我很高興將電話轉給 LifeSci Advisors 的 Brian Ritchie。請繼續，布萊恩。

Thank you. Good afternoon, everyone, and thank you for joining us to discuss Soleno Therapeutics second quarter 2025 financial and operating results. Please note, we'll be making certain forward-looking statements today. We refer you to Soleno's SEC filings for discussion of the risks. That may cause actual results to differ from the forward-looking statements on the call with me today for Seleno are Anish Bhatnagar, Seleno's Chairman and Chief Executive Officer; Meredith Manning, Seleno's Chief Commercial Officer; and Jim Mackaness, Seleno's Chief Financial Officer.

謝謝。大家下午好，感謝您加入我們討論 Soleno Therapeutics 2025 年第二季財務與營運績效。請注意，我們今天將做出某些前瞻性陳述。我們請您參閱 Soleno 提交給美國證券交易委員會 (SEC) 的文件，以了解相關風險。這可能會導致實際結果與今天與我一起參加 Seleno 電話會議的前瞻性陳述不同，與會者包括 Seleno 董事長兼首席執行官 Anish Bhatnagar、Seleno 首席商務官 Meredith Manning 和 Seleno 首席財務官 Jim Mackaness。

With that, I will now turn the call over to Anish.

說完這些，我現在將電話轉給 Anish。

Thank you, Brian. And thank you, everyone, for joining us for a second quarter financial results call. For those who follow us closely, you will know that we announced results for the second quarter ending June 30, 2025, on July 10. Therefore, many of you will be familiar with certain key metrics we'll be discussing today.

謝謝你，布萊恩。感謝大家參加我們的第二季財務業績電話會議。對於那些密切關注我們的人來說，您會知道我們在 7 月 10 日公佈了截至 2025 年 6 月 30 日的第二季業績。因此，你們中的許多人都會熟悉我們今天要討論的某些關鍵指標。

With that said, I am very pleased to share our significant progress during the second quarter, highlighted by the commercial launch of VYKAT XR and commencement of treating people living with Prader-Willi syndrome or PWS. Meredith will review the company's commercialization progress to date, and Jim will cover the company's financial statements for the second quarter. We will then open the call for questions.

話雖如此，我很高興與大家分享我們在第二季度取得的重大進展，其中最突出的是 VYKAT XR 的商業發布和開始治療患有普拉德-威利綜合徵或 PWS 的患者。梅雷迪斯將回顧公司迄今的商業化進展，吉姆將介紹公司第二季的財務報表。然後我們將開始提問。

We achieved a major milestone for the PWS community and for Seleno in March when we launched VYKAT XR, the first FDA approved medicine for the treatment of hyperphagia in adults and children four years of age and older with PWS. The approval of VYKAT XR was based on our comprehensive clinical development program in which participants received double blind and or open label VYKAT XR for a mean duration of 3.3 years. Primary evidence of efficacy came from a 16-week randomized withdrawal phase 3 multi-center double blind placebo controlled trial.

今年 3 月，我們推出了 VYKAT XR，這是第一個獲得 FDA 批准用於治療 PWS 成人和 4 歲及以上兒童暴食症的藥物，這對 PWS 社區和 Seleno 來說是一個重要的里程碑。VYKAT XR 的批准是基於我們全面的臨床開發計劃，該計劃參與者接受雙盲和/或開放標籤 VYKAT XR 治療，平均持續時間為 3.3 年。療效的主要證據來自於16週的隨機撤藥3期多中心雙盲安慰劑對照試驗。

Following FDA approval on March 26, VYKAT XR was available on April 14, ahead of plan. We've been extremely pleased with the initial reception and demand from the PWS community, which we believe speaks to the urgent need for an FDA approved therapy to treat the hallmark feature of PWS, which is hyperphagia.

繼 3 月 26 日獲得 FDA 批准後，VYKAT XR 於 4 月 14 日提前上市。我們對 PWS 社群的初步反應和需求感到非常高興，我們相信這表明迫切需要一種經 FDA 批准的療法來治療 PWS 的標誌性特徵，即暴食症。

As we announced at that time, concurrent with product availability, prescriptions of VYKAT XR had been delivered to the first individuals living with PWS. Since that time, we have seen steady growth in both patients starts and unique prescribers. Total net revenue was $32.7 million in the second quarter. Which in part reflects underlying demand for the drug, but also the significant efforts of our experienced commercial team whose launch out outreach efforts to patients, physicians, and payers set the stage for a strong and successful launch.

正如我們當時宣布的那樣，隨著產品上市，VYKAT XR 處方已送達首批 PWS 患者。自那時起，我們看到患者數量和獨特處方數量都在穩步增長。第二季總淨收入為3,270萬美元。這在一定程度上反映了對該藥物的潛在需求，也反映了我們經驗豐富的商業團隊的巨大努力，他們向患者、醫生和付款人開展的推廣工作為強勁而成功的推出奠定了基礎。

As Meredith will describe in a moment, we have made steady progress with both commercial and government payers. We continue to engage with payers to ensure that they understand the severe complications and high unmet needs associated with PWS and the inherent value proposition that VYKAT XR offers. Establishing broad payer reimbursement is among our highest priorities going forward, and the compelling efficacy and safety data from our clinical trial program is clearly resonating.

正如梅雷迪斯稍後將要描述的那樣，我們在商業和政府付款方面都取得了穩步進展。我們繼續與付款人合作，以確保他們了解與 PWS 相關的嚴重併發症和大量未滿足的需求以及 VYKAT XR 提供的內在價值主張。建立廣泛的付款人報銷制度是我們未來的首要任務之一，而我們臨床試驗計劃中令人信服的療效和安全性數據顯然引起了共鳴。

I would like to once again recognize the substantial contributions of the entire PWS community, including study participants and their families, the study investigators and study site team members, as well as the two major PWS advocacy organizations, the Foundation for Prabli Research and the Prader-Willi Syndrome Association USA. I would also like to thank the Soleno team members who worked so tirelessly to get us to this point.

我想再次感謝整個 PWS 社區的巨大貢獻，包括研究參與者及其家人、研究調查員和研究現場團隊成員，以及兩個主要的 PWS 倡導組織——普拉布利研究基金會和美國普拉德-威利綜合徵協會。我還要感謝 Soleno 團隊成員，他們孜孜不倦地工作，使我們達到了這一點。

I would now like to provide a brief update on our activities and support of potential approval of DCCR in Europe. As we market DCCR in the US as VYKAT XR. PWS is a global disease that impacts hundreds of thousands of patients all over the world in an effort to make DCCR available to as many of these patients as possible. In parallel with our US commercial launch, we have continued to make progress along regulatory pathways and other geographies, the most important of which is the EU.

現在，我想簡要介紹一下我們的活動以及對歐洲 DCCR 潛在批准的支持。我們在美國將 DCCR 以 VYKAT XR 的名稱銷售。PWS 是一種全球性疾病，影響著全世界數十萬名患者，我們努力讓盡可能多的患者能夠使用 DCCR。在我們在美國進行商業推廣的同時，我們也繼續在監管途徑和其他地區取得進展，其中最重要的是歐盟。

As we have stated previously, Europe also has a high unmet need among patients with PWS. Based on widely cited prevalence data, it is estimated that approximately 9,000 patients living with PWS in the EU 4 in the UK. We have conducted market research with many PWS experts, patient advocacy leaders, care home executives, etc. Where we have confirmed the prevalence numbers.

正如我們之前所說，歐洲的 PWS 患者也存在大量未滿足的需求。根據廣泛引用的盛行率數據，估計英國歐盟 4 國中約有 9,000 名 PWS 患者。我們與許多 PWS 專家、患者權益領袖、養老院管理人員等進行了市場調查，並確認了盛行率。

Today, in most major markets in the EU, early diagnosis is common. Our research suggests that there is significant structured care for people living with PWS across Europe, with variations that will impact our go to market strategy by country.

如今，在歐盟的大多數主要市場，早期診斷已經很常見。我們的研究表明，歐洲各地為患有 PWS 的人提供了重要的結構化護理，但各國的差異將影響我們針對不同國家的市場策略。

Additionally, as with the US, the PWS community has strong thought leader support, and patient care is often concentrated around centers of excellence, even more so than in the US. In May we were pleased to announce the submission and EMA validation of our marketing authorization application. Gaining approval to market DCCR in the EU would represent a meaningful ex expansion of our commercial market and remains a priority for us while we continue to progress our US launch.

此外，與美國一樣，PWS 社區擁有強大的思想領袖支持，患者護理通常集中在卓越中心，甚至比美國更集中。五月份，我們很高興地宣布我們的行銷授權申請已提交並獲得 EMA 驗證。獲得在歐盟銷售 DCCR 的批准將代表我們商業市場的一次有意義的擴張，並且在我們繼續推進美國市場推廣的同時仍然是我們的首要任務。

I will now turn the call over to Meredith to provide an update on the launch. Meredith.

現在我將把電話轉給梅雷迪斯，讓她提供有關發布會的最新進展。梅雷迪絲。

Thank you, Aneesh, and good afternoon, everyone. As Anish previously mentioned, we are still early in our early phase of launch, and the response from both families and providers has been exceptionally encouraging. This tells us that our outreach, whether to caregivers, individuals with PWS, physicians, centers of excellence, payers, or advocacy groups, is truly resonating.

謝謝你，Aneesh，大家下午好。正如 Anish 之前提到的，我們仍處於發布的早期階段，家庭和提供者的反應都非常令人鼓舞。這告訴我們，我們的外展活動，無論是針對護理人員、PWS 患者、醫生、卓越中心、付款人還是倡導團體，都確實引起了共鳴。

Our disciplined execution and strong, clear messaging are helping us to build clinical conviction in the field. Taken together, these factors reinforce our confidence in the significant potential ahead. At this point I would like to provide an update on the key performance indicators that we that we believe are helpful in tracking our progress.

我們嚴謹的執行和強大而清晰的訊息傳遞正在幫助我們在該領域建立臨床信念。綜合起來，這些因素增強了我們對未來巨大潛力的信心。此時，我想提供我們認為有助於追蹤我們進展的關鍵績效指標的最新資訊。

The first of these is patient start forms. As we announced in our preliminary results press release on July 10, we received 646 patient start forms from launch through June 30. A vast majority of people who have started treatment as expected are younger, so that's between 4 and 26 years of age. However, a majority of these individuals are older than what we saw in the clinical trial C601. Which was approximately 13.5 years.

其中第一個是患者開始表格。正如我們在 7 月 10 日的初步結果新聞稿中宣布的那樣，從啟動到 6 月 30 日，我們收到了 646 份患者開始表格。絕大多數如預期開始接受治療的人都比較年輕，年齡在 4 至 26 歲之間。然而，這些人中的大多數比我們在臨床試驗 C601 中看到的要老。大約是13.5年。

The second key performance indicator is the number of prescribers. From launch through June 30, that number is 295 unique prescribers. More than a third of the top 300 prescribers who are the primary prescribers for approximately 2000 patients have written prescriptions. And we are very heartened to see a large number of start forms coming from prescribers who we did not expect to see writing start forms this early in our launch.

第二個關鍵績效指標是開處方者的數量。從推出到 6 月 30 日，這個數字是 295 個獨立處方者。在為大約 2,000 名患者開立處方的主要前 300 名處方者中，超過三分之一的人開出了處方。我們非常高興地看到，有大量來自處方醫生的開始表格，我們沒想到他們在我們推出的初期就寫好了開始表格。

We believe the launch momentum and positive results directly reflects the success of our strategic launch efforts, initiatives that targeted pediatric and adult endocrinologists, geneticists, and psychiatrists who directly treat or influence a substantial portion of our addressable market. Our field force is prioritizing deep engagement with top tier providers, each of whom treats multiple individuals with PWS.

我們相信，發布的勢頭和積極成果直接反映了我們戰略發布努力的成功，這些舉措針對的是直接治療或影響我們很大一部分潛在市場的兒科和成人內分泌學家、遺傳學家和精神病學家。我們的現場人員優先考慮與頂級供應商進行深入合作，每個供應商都為多名 PWS 患者提供治療。

Not only to further strengthen their experience and confidence with VYKAT XR, but also because these key clinicians play a pivotal role in influencing practice patterns and the broader adoption of new therapies within the PWS community.

這不僅進一步加強了他們對 VYKAT XR 的經驗和信心，而且因為這些關鍵臨床醫生在影響 PWS 社群的實踐模式和更廣泛地採用新療法方面發揮關鍵作用。

At the same time, we are increasingly encouraged by the strong response from physicians beyond this core group. This demonstrates not only growing awareness of VYKAT XR but also expanding recognition of the therapeutic need to treat hyperphagia.

同時，核心群體之外的醫生們的強烈反應也讓我們倍受鼓舞。這不僅表明人們對 VYKAT XR 的認識不斷提高，而且對治療暴食症的治療需求的認識也不斷擴大。

The third performance indicator is payer policies. As Anish mentioned, securing broad coverage for VYKAT XR is a core focus for us and is fundamental to the success of our launch. We have seen rapid and broad coverage surpassing recent rare disease launches, with approximately 33% of all insured lives now covered, representing just over $100 million lives covered in the United States.

第三個績效指標是付款人政策。正如 Anish 所提到的，確保 VYKAT XR 的廣泛覆蓋是我們關注的核心，也是我們成功推出產品的基礎。我們已經看到了快速而廣泛的覆蓋範圍，超越了最近推出的罕見疾病保險，目前約有 33% 的受保人得到了保障，相當於在美國獲得了超過 1 億美元的保障。

We are encouraged with the payer coverage policies established for VYKAT XR to date, including several from major insurers. And it's worth highlighting that we are receiving coverage across all channels, commercial, Medicaid and Medicare. And to see such broad coverage so early in the launch is outstanding. These positive outcomes are a direct result of our proactive engagement.

我們對迄今為止為 VYKAT XR 制定的付款人承保保單感到鼓舞，其中包括幾家主要保險公司的保單。值得強調的是，我們正在透過商業、醫療補助和醫療保險等所有管道獲得報告。在發布初期就獲得如此廣泛的報道是非常了不起的。這些正面成果是我們積極參與的直接結果。

Efforts that enabled payers to move quickly in recognizing the value of VYKAT XR and the urgency of addressing PWS related hyperphasia. The coverage decisions we have seen so far demonstrate that payers recognize the seriousness of PWS, understand the unique challenges of hyperphasia, and appreciate the meaningful value VYKAT XR delivers.

這些努力使付款人能夠迅速認識到 VYKAT XR 的價值以及解決 PWS 相關多語症的緊迫性。到目前為止，我們看到的承保決定表明，付款人認識到 PWS 的嚴重性，理解多語症的獨特挑戰，並欣賞 VYKAT XR 帶來的重大價值。

I will now turn the call over to Jim for a review of the company's financial statements for the second quarter.

我現在會把電話轉給吉姆，讓他審查公司第二季的財務報表。

Thank you, Meredith. We used $12.6 million of cash in operating activities during the three months ended June 30, 2025, and had$293.8 million of cash equivalents of marketable securities at the end of the quarter. Subsequent to the quarter end, we raised an additional $230 million of gross proceeds through an underwriter offering of our common stock, bringing our total pro forma cash balance following the financing to more than $500 million.

謝謝你，梅雷迪斯。在截至 2025 年 6 月 30 日的三個月內，我們在經營活動中使用了 1,260 萬美元的現金，並且在本季末擁有 2.938 億美元的有價證券現金等價物。在本季末之後，我們透過承銷商發行普通股又籌集了 2.3 億美元的總收益，使融資後我們的總備考現金餘額超過 5 億美元。

This balance sheet strength ensures that we are sufficiently well capitalized to execute on an effective US launch of VYKAT XR and become cash flow positive while in parallel progressing towards regulatory approvals and commercialization, either on a stand-alone basis or with partners in the EU and other geographies.

這種資產負債表實力確保我們擁有充足的資本，能夠有效地在美國推出 VYKAT XR，並實現正現金流，同時繼續推進監管部門的批准和商業化，無論是獨立進行還是與歐盟和其他地區的合作夥伴一起進行。

Turning now to a few income statement items, total net revenue for the second quarter ended June 30, 2025, was $32.7 million. As VYKAT XR was approved in March of this year, the company generated no revenue in the second quarter ended June 30, 2024. Cost of goods sold was $0.7 million for the second quarter ended June 30, 2025.

現在來看幾個損益表項目，截至 2025 年 6 月 30 日的第二季總淨收入為 3,270 萬美元。由於VYKAT XR於今年3月獲得批准，該公司在截至2024年6月30日的第二季度沒有產生任何收入。截至 2025 年 6 月 30 日的第二季度，銷售成本為 70 萬美元。

Please note that prior to the FDA approval, costs associated with manufacturing VYKAT XR were expensed as research and development expenses. As such, a portion of the cost of goods sold during the period included inventory at zero cost. Going forward, as we continue to sell VYKAT XR, we will deplete our zero-cost inventory. And replenish it without cost inventory and consequently cost of goods sold as a percentage of revenue will increase.

請注意，在獲得 FDA 批准之前，製造 VYKAT XR 的相關成本被計入研發費用。因此，該期間銷售商品成本的一部分包括零成本庫存。展望未來，隨著我們繼續銷售 VYKAT XR，我們將耗盡我們的零成本庫存。並補充無成本庫存，因此銷售成本佔收入的百分比將會增加。

Research and development expense for the second quarter ended June 30, 2025, with $9.1 million, which includes $2.4 million of non-cash stock-based compensation compared to $12.3 million, which includes $2.7 million of non-cash stock-based compensation for the same period of 2024.

截至 2025 年 6 月 30 日的第二季研發費用為 910 萬美元，其中包括 240 萬美元的非現金股票薪酬，而 2024 年同期的研發費用為 1,230 萬美元，其中包括 270 萬美元的非現金股票薪酬。

The cadence of our research and development expenditures fluctuate depending upon the state of our clinical programs, timing of manufacturing and other projects as we've moved through submission, approval, and now preparation for commercialization.

我們的研發支出節奏隨著臨床項目的狀態、製造時間和其他項目的提交、批准以及現在的商業化準備而波動。

Selling general administrative expense for the second quarter ended June 30, 2025, was $28.2 million which includes $7.3 million of non-cash stock-based compensation compared to $10.9 million, which includes 4.5 million of non-cash stock-based compensation for the same period of 2024. The increase reflects our ongoing investment in additional personnel and new programs to support the VYKAT XR commercial launch and in support of our increased business activity.

截至 2025 年 6 月 30 日的第二季度，銷售一般管理費用為 2,820 萬美元，其中包括 730 萬美元的非現金股票薪酬，而 2024 年同期為 1,090 萬美元，其中包括 450 萬美元的非現金股票薪酬。這一成長反映了我們對額外人員和新項目的持續投資，以支持 VYKAT XR 的商業發布和支持我們不斷增加的業務活動。

Total other income net was $1.8 million for the three months ended June 30, 2025, compared to total other net income of [$3.0 million] in the same period of 2024. Net loss was approximately $4.7 million or $0.09 per basic and diluted shares for the second quarter ended June 30, 2025, and $21.9 million or $0.57 per basic and diluted share for the same period in 2024.

截至 2025 年 6 月 30 日的三個月，其他淨收入總額為 180 萬美元，而 2024 年同期的其他淨收入總額為 [300 萬美元]。截至 2025 年 6 月 30 日的第二季淨虧損約為 470 萬美元，即每股基本虧損和稀釋虧損均為 0.09 美元；2024 年同期淨虧損約為 2,190 萬美元，即每股基本虧損和稀釋虧損均為 0.57 美元。

This concludes the financial overview, and I'll now turn the call back over to Anish for closing remarks. Anish.

財務概覽到此結束，現在我將把電話轉回給 Anish 做結束語。阿尼什。

Thank you, Jim. In closing, while we are still early in the launch of VYKAT XR, we are pleased with the trajectory we're on. In the second quarter, we saw extraordinary momentum in the number of SAT forms, patients on active drug, number of patients on paid drug, and lives covered. While we're not able to share details at this time since we are far from steady state, we continue to be encouraged that VYKAT XR is a groundbreaking therapy, and we believe it will soon be the standard of care for people living with PWS related hyperphagia.

謝謝你，吉姆。最後，雖然我們仍處於 VYKAT XR 發布的早期階段，但我們對目前的發展軌跡感到滿意。在第二季度，我們看到 SAT 表格數量、使用活性藥物的患者數量、使用付費藥物的患者數量以及覆蓋的生命數量都出現了顯著的增長勢頭。雖然由於我們遠未達到穩定狀態，我們目前無法分享細節，但我們仍然對 VYKAT XR 是一種突破性的療法感到鼓舞，我們相信它很快就會成為患有 PWS 相關暴食症的人的護理標準。

And with that, we'll now open the call to questions, operator.

接線員，我們現在開始回答問題。

(Operator Instructions)

（操作員指示）

Yasmeen Rahimi, Piper Sandler.

亞斯明·拉希米、派珀·桑德勒。

Good afternoon team. Thank you so much for the updates. I guess many of our clients were wondering if you could comment on how do you see sort sortly the monthly script in July compared to the earlier months and how do you think it will change going into the remainder of August and September, appreciate any color around that. And then also maybe second one to squeeze in is like what is sort of the time to fill currently and how do you think it would change over time, and I'll jump back in the queue.

大家下午好。非常感謝您的更新。我想我們的許多客戶都想知道您是否可以評論一下，與前幾個月相比，您如何看待 7 月份的月度腳本排序，以及您認為 8 月剩餘時間和 9 月的腳本排序將如何變化，請您對此進行評論。然後也許第二個要擠進去的就像目前要填補的時間是什麼樣的以及你認為它會如何隨著時間而變化，我會跳回隊列。

Okay. Thanks, Yasmin. I'll take the July question and as we're not addressing data after the end of the quarter on this call. But what I can tell you is that we have confidence that VYKAT XR is on its way to being the standard of care for people living with PWS. It is going to be a therapy that's going to be meaningful. And it's going to be something that PWS patients are going to be on for a long time to come. But Meredith, I'll let you address the time to fill question.

好的。謝謝，Yasmin。我將回答七月份的問題，因為我們不會在本次電話會議上討論本季結束後的數據。但我可以告訴你們的是，我們有信心 VYKAT XR 將成為 PWS 患者的照護標準。這將是一種有意義的治療方法。這將是 PWS 患者未來很長一段時間內需要堅持的治療方法。但是梅雷迪斯，我會讓你花時間回答問題。

Yeah, thank you. Thanks, Yasmin for the question. I think with regard to time to fill and something that we've mentioned in the past is it takes a while for the payer policies to come in and and have a steady state and while we're very pleased and super encouraged with the 33% coverage live you know we still expect policies to come in and for that to grow. So we've seen pretty rapid turnaround time based on the fact that we do have very strong policies favorable policies coming in, and we have that 33% coverage as more policies come in, the turnaround time could potentially slow.

是的，謝謝。謝謝 Yasmin 提出的問題。我認為關於填補時間以及我們過去提到過的事情是，付款人政策需要一段時間才能出台並達到穩定狀態，雖然我們對 33% 的覆蓋率感到非常高興和鼓舞，但你知道我們仍然希望政策能夠出台並實現增長。因此，我們看到了相當快的周轉時間，因為我們確實有非常強有力的政策優惠政策，而且我們的覆蓋率是 33%，隨著更多政策的出台，週轉時間可能會變慢。

And then hopefully as we move into the later months, after a full year, we're looking at reaching a steady state and something that we're shooting for that we've seen in other rare disease or other therapeutic areas around approximately 30 days of turnaround time is pretty standard.

然後希望隨著我們進入後幾個月，經過整整一年，我們能夠達到穩定狀態，並且我們在其他罕見疾病或其他治療領域也看到了類似的目標，大約 30 天的周轉時間是相當標準的。

Thank you so much.

太感謝了。

Ry Forseth with Guggenheim.

古根漢美術館的 Ry Forseth。

Hi, this is Ry from WI's team. Thanks for taking our questions. Are there any emerging pain points during the patient start form process that you see, as addressable in the next couple quarters? And, our second question is, how are early compliance trends tracking?

大家好，我是 WI 團隊的 Ry。感謝您回答我們的問題。您認為在患者開始填寫表格的過程中是否存在一些新出現的痛點，可以在接下來的幾個季度內解決嗎？我們的第二個問題是，早期合規趨勢如何追蹤？

So, on the pain points front, as this is a very strong start. I mean, we, 646 patients start in the first quarter is a very large number. We try to track things like seasonality, holidays, vacation summer holidays, things like that, and this is the first drug launched for hyperphagia, so we don't know how it's going to be. It's hard for us to predict the pain points with the start forms, but again, we'll, the next couple of quarters will be pretty educational, so we'll keep you posted on that.

因此，就痛點而言，這是一個非常強勁的開端。我的意思是，第一季就有 646 名患者，這是一個非常大的數字。我們嘗試追蹤諸如季節、假日、暑假等因素，這是第一款針對暴食症推出的藥物，所以我們不知道它會如何。我們很難預測開始形式的痛點，但同樣，接下來的幾季將會非常有教育意義，所以我們會隨時向您通報情況。

And your second question was comp compliance. Yeah, so it's too early to tell, right? Because we haven't seen that much data, but what I can tell you is that discontinuation rates are substantially lower than what we saw even in clinical trials. So, as you may remember, in our trials, we've seen very high compliance rates in part because some of these, some of the people living with PWS can also have obsessive compulsive tendencies and they tend to want to stay on drug as well. So we don't expect that to change significantly and certainly too early to tell from the data.

您的第二個問題是公司合規性。是的，現在說還太早，對嗎？因為我們還沒有看到那麼多數據，但我可以告訴你的是，停藥率遠低於我們在臨床試驗中看到的水平。所以，您可能還記得，在我們的試驗中，我們看到了非常高的依從率，部分原因是其中一些患有 PWS 的人也可能有強迫症傾向，他們也傾向於繼續服藥。因此，我們預計這種情況不會發生重大變化，而且從數據上判斷還為時過早。

Thank you very much.

非常感謝。

Kristen Kluska, Cantor.

克里斯汀·克魯斯卡（Kristen Kluska），領唱。

Hi everyone, congrats on a very strong quarter. My first question, I just wanted to get a sense of what you're seeing on safety. You've obviously collected a lot of real world evidence now. And then second, I know you're not commenting on specific revenue trends, but clearly, I think the two numbers were a lot higher than a lot of us modeled and what the investment community was looking for. So can you just give us any. Broad sense of how we should be thinking about the rest of the year so people perhaps don't go over their skis either thank you.

大家好，恭喜本季業績強勁。我的第一個問題是，我只是想了解您對安全的看法。您現在顯然已經收集了大量現實世界的證據。其次，我知道您沒有評論具體的收入趨勢，但顯然，我認為這兩個數字比我們許多人模擬的和投資界所期望的要高得多。那麼你能給我們任何資訊嗎？我們應該從廣義上考慮今年剩餘的時間，這樣人們就不會再滑雪了，謝謝。

Sure. Thanks, Kristen. Thanks for the question. So, on the safety side, as many of you I'm sure know, monitoring for safety data in the postmarketing setting is quite different from clinical trial settings. So one typically relies on reports from caregivers or healthcare providers, and the patients that you're treating are also often not as controlled and may have more comorbidities, etc.

當然。謝謝，克里斯汀。謝謝你的提問。因此，在安全方面，我相信你們中的許多人都知道，上市後環境中的安全資料監測與臨床試驗環境中的安全資料監測有很大不同。因此，人們通常依賴護理人員或醫療保健提供者的報告，並且所治療的患者通常也不受控制，並且可能患有更多合併症等。

So we are pretty early in the launch, but that said, I can tell you that we have not seen anything in the postmarketing setting that is different from the clinical trial setting. So there are no new safety signals. And once again, just to reiterate what we have seen with the discontinuation rates at this time are substantially lower than what we've seen in the clinical trials.

因此，我們處於發布的早期階段，但話雖如此，我可以告訴你，我們在上市後環境中沒有看到任何與臨床試驗環境不同的情況。因此沒有新的安全訊號。再次重申，我們目前看到的停藥率遠低於臨床試驗中看到的停藥率。

And Jim, I'll let you take the revenue question.

吉姆，我讓你回答收入問題。

Sure, yes. Hi Kristen. Yeah, well, as there are quite a number of moving parts between the start form and ultimately the revenue. So I think, what we saw was the fact that everything seemed to be clicking very well out of the gate. So that's excellent. We're obviously sort of mindful of maybe things normalizing over the next couple of quarters, but at the moment it just seems to be a very strong start with everything all coming together. So good momentum at a Q2.

當然，是的。你好，克里斯汀。是的，因為從起始形式到最終收入之間有相當多的活動部分。所以我認為，我們看到的事實是，一切似乎都很順利。這太棒了。我們顯然很擔心未來幾季情況會恢復正常，但目前看來，一切似乎都已步入正軌，開局非常強勁。因此第二季的勢頭良好。

Tyler Van Buren, TD Cowen.

泰勒範布倫 (Tyler Van Buren)，TD Cowen。

Hey guys, thanks very much and congratulations on the tremendous progress made during the quarter. So, regarding the $33 million of ICA sales for the quarter, can you help us understand to what extent there was an initial patient bolus or stocking in this number, and maybe just a follow up, I think you mentioned. That you're commercially, you're seeing older patients in the 13.5 years in the trial. So, does that mean on average that these commercial patients are heavier in weight, above that [61 kg] and higher than the average price of $466,000 estimated based on that?

嘿，夥計們，非常感謝，並祝賀你們在本季度取得的巨大進展。那麼，關於本季 3300 萬美元的 ICA 銷售額，您能否幫助我們了解這個數字中有多少是初始患者推注或庫存，也許只是後續行動，我想您提到過。從商業角度來看，在 13.5 年的試驗中，您會看到年齡較大的患者。那麼，這是否意味著這些商業患者的平均體重更重，高於這個數字 [61 公斤]，並且高於基於此估算的 466,000 美元的平均價格？

Thanks, Tyler. Jim, you want to take the bolus stocking question?

謝謝，泰勒。吉姆，你想回答關於丸劑長襪的問題嗎？

Yeah, so specifically to the stocking question, Tyler, yeah, we, as we have one distribution partner, Panther. They have been managing their business, I'd say very astutely. They order weekly from us. They obviously do carry a little bit of inventory, probably 7 to 10 days, but we've not really seen anything that was out of the norm, so they've just been a very repetitive once a week stocking proposition from us, and that's continued. So I said don't think there's any anomalies there.

是的，具體到庫存問題，泰勒，是的，我們有一個分銷合作夥伴，Panther。我想說，他們一直在非常精明地管理自己的業務。他們每周向我們訂貨。他們顯然確實有少量庫存，大概需要 7 到 10 天，但我們並沒有看到任何異常情況，因此他們只是每週重複一次我們的備貨建議，並且這種情況一直持續下去。所以我說，不要認為那裡有任何異常。

I think on the bonus question, yeah, we do think there was a fantastic set of start forms coming in in Q2. I think we've commented before. Hard to imagine that that will continue linearly going forward if you like, so there'll be some moderation there, but everything at the moment seems to indicate a very strong launch going forward.

我認為關於附加問題，是的，我們確實認為第二季出現了一組非常棒的開始形式。我想我們之前已經評論過了。如果你願意的話，很難想像這種情況會繼續線性發展，所以會有一些緩和，但目前的一切似乎都表明未來的發展非常強勁。

And Tyler, to your question about patient weights, as you correctly pointed out, the patients that we're seeing, the majority of them are above the average age in [601], which is more than 13.5 years. So I think it's a reasonable assumption that they are heavier as well.

泰勒，關於患者體重的問題，正如您正確指出的那樣，我們所見到的患者中，大多數都高於 [601] 的平均年齡，即 13.5 歲以上。所以我認為它們也更重是一個合理的假設。

Thank you.

謝謝。

[James Condulis, People].

[詹姆斯·康杜利斯，《人物》]

Hey, thanks so much for taking my question, and congrats on the corner. Just one from us just curious, these patients and start forms you're seeing obviously a great start. Is this all kind of coming, are these patients on sort of like their normal cadence of visiting doctors or are these kind of patients, calling in and trying to get in kind of off cycle? Just curious if you have any color on that dynamic and kind of on that more broadly, all the kind of cylinders seem to be firing here so curious what. Is the rate limiter here kind of going forward if there's any that's obvious to you thanks so much.

嘿，非常感謝您回答我的問題，恭喜您取得好成績。我們只是好奇，這些病人和開始的形式，你看到的顯然是一個很好的開始。這些都是這樣嗎？這些病人是否按照正常的步調去看醫生？或是這些病人打電話來，試圖以某種非週期的方式就診？只是好奇您是否對這種動態有任何了解，並且更廣泛地說，所有類型的氣缸似乎都在這裡點火，所以很好奇。這裡的速率限制器是否會向前發展，如果有的話，這對您來說很明顯，非常感謝。

Meredith, go ahead.

梅雷迪斯，說吧。

Yeah, happy to take that and you know it's been really nice to get out in the field and be with our field team and also meet with many of the clinicians we're seeing both to answer your question both patients who were very proactive as they were anticipating the launch of VYKAT getting. Some of their appointments set up within an early time frame, but we're also seeing that some of the PWS experts are very busy with the launch and therefore it is taking time to get some of the other patients coming in.

是的，很高興接受這個採訪，您知道，能夠走出去，和我們的現場團隊在一起，並與我們看到的許多臨床醫生會面，這真的很棒，可以回答您的問題，兩位患者都非常積極主動，因為他們期待著 VYKAT 的推出。他們的一些預約是在早期時間內安排的，但我們也看到一些 PWS 專家非常忙於啟動，因此需要一些時間才能讓其他患者進來。

I think something that we're very excited to see is many of these clinics did not have a set PWS clinic day prior to launch and so as we're seeing the. The groundwork being laid across these various different clinics, they are setting up their PWS clinic days and we feel like that that will allow for a little bit more steady patient visits and patient cycle to come in so we're still seeing good significant opportunity ahead with the way that the landscape is really. Measuring out their process and their logistics.

我認為我們非常高興地看到，許多診所在推出前一天並沒有設立 PWS 診所，因此我們看到。這些不同的診所正在奠定基礎，他們正在設置 PWS 診所日，我們覺得這將使患者就診量和患者週期更加穩定，因此，就目前的情況來看，我們仍然看到未來存在著巨大的機會。衡量他們的流程和物流。

Leland Gershell, Oppenheimer.

利蘭·格謝爾，奧本海默。

Hi, good afternoon, guys, and thanks for the date and tremendous progress. I wanted to ask, maybe a little bit in connection with the last question. Patients with PWS come at different levels of severity, like in all diseases. Wondering if you're seeing a pattern where perhaps the more severe patients are the ones who are getting VYKAT earlier, as in, Q2, and currently, and if we should maybe think about, a broader uptake as, physicians become more familiar with the drug. I also want to ask with respect to, Europe, I know that's probably going to be in next year, but, you're sort of touching profitability here on the US business, wondering if you could just comment on what could be maybe a more efficient. Launch, in Europe with respect to OpEx and how that may affect your thoughts on profitability and cash flow going forward. Thanks.

大家下午好，感謝你們的安排和巨大的進步。我想問一下，也許與最後一個問題有一點關聯。與所有疾病一樣，PWS 患者的病情嚴重程度也不同。想知道您是否看到一種模式，也許病情較重的患者是較早服用 VYKAT 的患者，例如在 Q2 和目前，我們是否應該考慮，隨著醫生對該藥物越來越熟悉，這種藥物的接受範圍會更加廣泛。我還想問一下關於歐洲的問題，我知道這可能是在明年，但是，您有點觸及美國業務的盈利能力，想知道您是否可以評論一下什麼可能更有效率。在歐洲推出營運支出，以及這可能會如何影響您對未來獲利能力和現金流的看法。謝謝。

Sure. Thanks, Leland. So in terms of severity, we don't actually collect that information actively. What we see is a diagnosis of PWS and presence of hyperphagia as sort of on label and the age of four years, and that patient would be on label. So, we don't get that level of detail. But I suspect there's probably a combination of things happening. One is the idea that if there are severe patients, there's probably physicians who were calling them in earlier and getting them on drugs, but there's definitely an element of more motivated families likely with younger kids who are also pushing their way to getting therapy earlier. And we think this phenomenon of a lot of the non-KOL prescribers may well be the idea that the KOL practices are so full that some of these more motivated families are going to peripheral providers, their local lendos, etc. And getting prescriptions from there.

當然。謝謝，利蘭。因此，就嚴重性而言，我們實際上不會主動收集這些資訊。我們看到的是 PWS 的診斷和存在暴食症，就像標籤上寫的一樣，年齡為四歲，而該患者符合標籤上寫的條件。所以，我們無法獲得那麼詳細的資訊。但我懷疑可能發生了多種因素共同作用的結果。一種想法是，如果有重症患者，醫生可能會更早地打電話給他們並給他們開藥，但肯定也有一些更有動力的家庭，例如孩子年紀較小的家庭，也在努力讓他們儘早接受治療。我們認為，許多非 KOL 處方者的這種現像很可能是因為 KOL 實踐過於繁忙，以至於一些更有動力的家庭會去外圍提供者、當地的 lendo 等，並從那裡獲取處方。

In terms of the EU business and OECs, Jim, you want to take that?

關於歐盟業務和 OEC，吉姆，你想接受這個嗎？

Yeah, sure. So, I think Leeland as you said, I mean that goes back to the fact that we have the $500 million of balance sheet strength. So it allows us the optionality when we're looking at Europe, we've mentioned partners along the way, but we now also have the ability if we wish to continue to do it on our own.

是的，當然。所以，我認為正如你所說，Leeland，我的意思是這可以追溯到我們擁有 5 億美元資產負債表實力的事實。因此，當我們關注歐洲時，它為我們提供了選擇權，我們一路上提到了合作夥伴，但如果我們希望繼續自己做，我們現在也有能力了。

We do think it's a concentrated market. Some of the ways that rare diseases are addressed within the European countries do present sort of smaller call points than even in the US, so we've still got to work out the details on exactly the size of the sort of the sales force, the commercial team that would be needed if we do go on our own, but we think it's manageable and as I said, we just point back to the $500 million because it gives us that option to do it if we choose.

我們確實認為這是一個集中的市場。歐洲國家治療罕見疾病的某些方法確實比美國治療的呼叫點要小，所以我們仍然需要確定如果我們真的獨立開展業務，需要什麼樣的銷售隊伍和商業團隊的具體規模，但我們認為這是可以管理的，正如我所說的，我們只是回到 5 億美元，因為如果我們選擇的話，它給了我們這樣做的選擇。

All right, thank you.

好的，謝謝。

[Brian Scorney, Baird].

[布萊恩·斯科尼，貝爾德]

Hey, good afternoon, and thanks for taking my question and congrats on in my entire career being the first company, to have a sequentially down operating expenditures into the first quarter of launch. Very impressive. So, to that point, Jim, I'm just wondering, looking at, the OpEx, if this is a fair go forward number to model for the US business and you know you only it says that operationally spent $12.5 million in cash.

嘿，下午好，感謝您回答我的問題，並祝賀您成為我整個職業生涯中第一家在推出後的第一季度連續降低營運支出的公司。非常令人印象深刻。所以，吉姆，就這一點而言，我只是想知道，看看營運支出，這是否是一個公平的未來數字，可以為美國業務建模，而且您知道它只說營運上花費了 1250 萬美元現金。

I'm just wondering if I look at just the income statement did. $32.7 million in sales, about $40 million in in OpEx, but if I back out, stock-based compact's $10 million less. So I would have thought that it would actually be operationally cash flow positive. So you know what, what's in the cash flow from operations here, that's not kind of summing up with the income statement.

我只是想知道，如果我只看損益表，銷售額為 3270 萬美元，營運支出約為 4000 萬美元，但如果我退出，基於股票的契約就會減少 1000 萬美元。所以我原本以為它的營運現金流其實是正的。所以你知道嗎，這裡的經營活動現金流並不是與損益表相加的。

Go ahead, Jim. Yeah, I'm not sure I understand the tail end of that question, but Brian, let me provide, color that we've, I think we've provided in the past. So, our cash OpEx, I think we've mentioned in the past we think about $120 million to $130 million for this year. We may find that there's a reason to sort of increase that a little bit, but there wouldn't be much more than [140], and that would just be doing things like I said, putting some early sort of initiatives in Europe, as we've mentioned.

繼續吧，吉姆。是的，我不確定我是否理解了這個問題的結尾，但是布萊恩，讓我提供我們過去提供的顏色。因此，我們的現金營運支出，我想我們過去曾提到過，我們認為今年的現金營運支出約為 1.2 億至 1.3 億美元。我們可能會發現有理由稍微增加這個數字，但不會超過 [140]，這只是像我說的那樣，在歐洲採取一些早期舉措，正如我們所提到的。

There's also some life cycle management stuff we might fire up as well. So still very well managed for 2025. And then if we go into 2026, that'll increment, and I think again we mentioned sort of probably go north of $150 million, but it certainly will be managed, not too much aggressively above that. A lot depends on the size of the commercial footprint and if we decide to go along.

我們也可能啟動一些生命週期管理的東西。因此，到 2025 年，管理仍會非常好。然後，如果我們進入 2026 年，這個數字就會增加，我想我們再次提到可能會超過 1.5 億美元，但肯定會得到管理，不會超過這個數字太多。這很大程度上取決於商業足跡的規模以及我們是否決定繼續下去。

And then I think to your point, then you've just got to layer the revenues from the launch and we've sort of said that's why we've commented in the past. If we think what we'd refer to as a modestly successful, I would suggest Q2 is exceeding that, but if we have a modestly successful launch, that's how we see ourselves getting to cash flow positive in somewhere in the near term. Hopefully that provides you the guidance you're looking for.

然後我想，正如你所說，你只需要從發布開始就分層獲得收入，我們過去也說過，這就是我們評論的原因。如果我們認為這是一個適度的成功，那麼我認為第二季度的表現會超過這個數字，但如果我們有一個適度成功的發布，那麼我們就能在短期內實現正現金流。希望這能為您提供您所尋求的指導。

Yes, that's perfect. Thanks, Jim.

是的，太完美了。謝謝，吉姆。

Yale Jen, Laidlaw.

耶魯詹，萊德勞。

Good afternoon and thanks for taking the questions and add my congrats as well. Just a little bit in terms of the revenue, breakdowns, in terms of the patients that the titrating phase, the cells versus the maintenance cells, the number is quite different. So I'm just curious whether most of the patients in this quarter are already the patient in the trial, so they are actually immediately moved to the maintenance or the, there are many more new patients, newer patients that actually in the earlier titration stage and thanks.

下午好，感謝您回答問題，也向您表示祝賀。就收入、細目分類而言，就滴定階段的患者而言，細胞與維持細胞的數量有很大差異。所以我只是好奇這個季度的大多數患者是否已經是試驗中的患者，所以他們實際上會立即轉移到維持治療階段，或者，實際上還有更多的新患者、較新的患者處於早期滴定階段，謝謝。

Yeah, thanks, yeah. So, as you may remember, only about 60 patients were on the long-term study in the US. The rest were in the UK. So, if you look at 646 [satrt] forms, that's a pretty small fraction of it. And as we've said in the past, virtually all of them will be on, are on commercial drug at this time. So, it's fair to say that in this quarter, a number of patients They are still in the titration phase or at least part of the quarter they were in the titration phase.

是的，謝謝，是的。所以，您可能還記得，美國祇有大約 60 名患者參與了這項長期研究。其餘的都在英國。因此，如果你看一下 646 [satrt] 形式，那隻是其中很小的一部分。正如我們過去所說的那樣，他們現在幾乎都在服用商業藥物。因此，可以公平地說，在本季度，許多患者仍處於滴定階段，或至少是處於滴定階段的一部分。

Okay, great. And maybe just one more question in terms of any breakdown in terms of the payers between government and the commercial. And the thanks.

好的，太好了。也許還有一個問題，關於政府和商業付款人的細分。並致謝。

Too early to tell, Yale. I think we're not quite a steady state yet, but we'll just remind you of the actual split in the population. It's about [1/3] commercial, [13 Medicare, and 13 Medicaid]. So we don't have numbers to provide for this quarter because of all the moving parts.

耶魯，現在說這個還太早。我認為我們還沒有達到穩定的狀態，但我們只是想提醒大家人口的實際分佈。大約三分之一是商業廣告，[13 醫療保險和 13 醫療補助]。由於所有變動因素，我們無法提供本季的數據。

Okay, great. Thanks a lot, and again, congrats, a great order.

好的，太好了。非常感謝，再次祝賀，這是一個很棒的訂單。

Thank you.

謝謝。

Ram Selvaraju, HC Wainwright.

拉姆·塞爾瓦拉朱、HC·溫賴特。

Hi, this is Jay on for Ram. Congrats on that first commercial quarter you guys doing great. So, first, have you seen any reluctance on the part of, your prescribers to deploy, in patients who have, PWS and are also diagnosed with diabetes?

大家好，我是 Jay，代表 Ram。恭喜你們在第一個商業季度取得了巨大的成功。那麼，首先，您是否發現您的處方醫生不願意為患有 PWS 且被診斷患有糖尿病的患者部署藥物？

We don't get sort of real-time information on these things, but I think it would be reasonable for a pair to be, for a provider to be hesitant if someone has uncontrolled diabetes. If it's diabetes that is controlled, then there's no reason to be reluctant to use DCCR. Of course, you need to be careful and do all the appropriate monitoring for the label, but the drug can certainly be used in patients who have type 2 diabetes.

我們沒有獲得這些事情的即時信息，但我認為，如果有人患有不受控制的糖尿病，醫療服務提供者猶豫不決是合理的。如果控制住了糖尿病，那就沒有理由不願意使用 DCCR。當然，您需要小心並對標籤進行所有適當的監測，但該藥物肯定可以用於患有第 2 型糖尿病的患者。

Okay, great. And just as a follow up, so looking to the future, are you thinking about expanding your portfolio beyond VYKAT XR, and if so, would this potentially include opportunistic and licensing and in what areas?

好的，太好了。作為後續問題，展望未來，您是否考慮將您的產品組合擴展到 VYKAT XR 之外，如果是，這是否可能包括機會主義和許可，以及在哪些領域？

Yeah, I think it's fair to say that as of today we are laser focused on the launch of VYKAT XR, and we don't want to deviate from that. But I think in the long term it's fair to say that we would as a company need to diversify from just VYKAT XR and we will look for opportunities that are likely adjacent to where we are today, but that is certainly not something we would do in the very short term.

是的，我認為可以公平地說，從今天起，我們的重點將集中在 VYKAT XR 的推出上，我們不想偏離這一點。但我認為從長遠來看，公平地說，我們作為一家公司需要從 VYKAT XR 轉向多元化，我們會尋找可能與我們目前所處位置相鄰的機會，但這肯定不是我們在短期內會做的事情。

Okay makes sense. Okay, thank you.

好的，有道理。好的，謝謝。

There are no further questions at this time. I will now turn the call over to Anish for closing remarks.

目前沒有其他問題。現在我將把電話交給阿尼什 (Anish) 來做結束語。

Well, thank you all again for calling in and we look forward to talking to you at the end of the next quarter.

好吧，再次感謝大家的來電，我們期待在下個季度末與您交談。

(Operator Instructions)

（操作員指示）