Soleno Therapeutics Inc (SLNO) 2025 Q1 法說會逐字稿

Greetings, and welcome to the Soleno Therapeutics first-quarter 2025 earnings conference call.

大家好，歡迎參加 Soleno Therapeutics 2025 年第一季財報電話會議。

(Operator Instructions)

（操作員指示）

As a reminder, this conference is being recorded. It is now my pleasure to turn the call over to Brian Ritchie of LifeSci Advisors. Please go ahead, Brian.

提醒一下，本次會議正在錄製中。現在我很高興將電話轉給 LifeSci Advisors 的 Brian Ritchie。請繼續，布萊恩。

Thank you, operator. Good afternoon, everyone, and thank you for joining us to discuss Soleno Therapeutics' first-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 a discussion of the risks that may cause actual results to differ from the forward-looking statements.

謝謝您，接線生。大家下午好，感謝您加入我們討論 Soleno Therapeutics 2025 年第一季度的財務和營運業績。請注意，我們今天將做出某些前瞻性陳述。我們請您參閱 Soleno 向美國證券交易委員會 (SEC) 提交的文件，以了解可能導致實際結果與前瞻性聲明不同的風險。

On the call with me today from Soleno are Anish Bhatnagar, Soleno's Chairman and Chief Executive Officer; Meredith Manning, Soleno's Chief Commercial Officer; and James MacKaness, Soleno's Chief Financial Officer. Anish will begin with a review of the company's progress during the first quarter and subsequent period, including FDA approval of VYKAT XR and commercial launch. Meredith will then review the company's commercial progress to date, and Jim will cover the company's financial statements for the first quarter. We will then open the call for questions.

今天與我一起參加電話會議的還有 Soleno 董事長兼首席執行官 Anish Bhatnagar； Soleno 首席商務官 Meredith Manning；以及 Soleno 首席財務官 James MacKaness。Anish 將首先回顧公司在第一季及後續期間的進展，包括 FDA 對 VYKAT XR 的批准和商業發布。隨後，梅雷迪斯將回顧公司迄今的商業進展，吉姆將介紹公司第一季的財務報表。然後我們將開始提問。

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 our very first quarterly results earnings call. It's been a momentous couple of months for our company, highlighted by our announcement on March 26 that the FDA approved VYKAT XR, previously known as diazoxide choline extended-release tablets or DCCR, as the first medicine for the treatment of hyperphagia in people four years of age and older with Prader-Willi syndrome or PWS.

謝謝布萊恩，也謝謝大家參加我們第一次的季度業績電話會議。對我們公司來說，過去的幾個月意義重大，尤其值得一提的是，我們於 3 月 26 日宣布，FDA 批准 VYKAT XR（之前稱為二氮嗪膽鹼緩釋片或 DCCR）作為首個用於治療四歲及以上患有普拉德-威利綜合徵或 PWS 的人的暴食症的藥物。

This approval is the culmination of many years of tireless work by the entire Soleno team, without whom none of this would have been possible. I would also like to recognize the significant contributions of the entire PWS community, including study participants and their families, the study investigators and study site team members, as well as the major PWS advocacy organizations, the Foundation for Prader-Willi Research, and the PWSA USA and the UK. We are very pleased to offer VYKAT XR for individuals and families who have been waiting for a treatment option for the symptoms related to this devastating disease since it was first recognized in 1956.

這項批准是整個 Soleno 團隊多年不懈努力的成果，沒有他們的努力，這一切都不可能實現。我還要感謝整個 PWS 社區的重大貢獻，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，以及主要的 PWS 倡導組織、普拉德威利研究基金會以及美國和英國的 PWSA。我們非常高興為那些自 1956 年首次發現這種毀滅性疾病以來一直在等待治療相關症狀的個人和家庭提供 VYKAT XR。

Following approval and reflecting our progress in establishing our commercial readiness, we were able to move very quickly to launch. We announced on April 14 that the first patient had received and is currently being treated with VYKAT XR, and this was about a week ahead of our internal forecast. Meredith will provide some additional color on the initial launch in a moment.

在獲得批准並反映我們在建立商業準備方面取得的進展後，我們能夠非常迅速地啟動。我們在 4 月 14 日宣布，第一位患者已經接受 VYKAT XR 治療，目前正接受該治療，這比我們的內部預測提前了大約一周。梅雷迪斯稍後將提供有關首次發布的更多詳細資訊。

For those who may be new to the story, I would like to briefly remind you of the key characteristics of PWS and discuss the impact that hyperphagia can have on the lives of people with PWS, their caregivers, their families, and the health care professionals who treat them. PWS is a rare genetic disease.

對於那些可能不熟悉這個故事的人，我想簡要地提醒您 PWS 的主要特徵，並討論暴食症對 PWS 患者、他們的護理人員、他們的家人以及治療他們的醫療保健專業人員的生活的影響。PWS 是一種罕見的遺傳疾病。

It occurs spontaneously in about 1 in 15,000 to 1 in 25,000 live births due to the deletion or lack of expression of a certain set of genes in chromosome 15. This translates to approximately 300,000 to 400,000 individuals living with PWS around the world.

由於 15 號染色體上某組基因的缺失或表現不足，大約每 15,000 個到每 25,000 個活產嬰兒中就會有 1 個自發性出現這種情況。這意味著全世界大約有 30 萬至 40 萬名 PWS 患者。

By the age of around seven or eight years, although sometimes as early as four, individuals with PWS typically will begin to exhibit the hallmark characteristic of the disease, hyperphagia, which is an insatiable desire to eat. This is essentially your brain telling you that you're starving despite having eaten. The only thing families and caregivers have been able to do to try to control hyperphagia is restrict access to food, such as locking refrigerators, trash cans, and pantries.

大約在七、八歲時，儘管有時早在四歲時，患有 PWS 的人通常就會開始表現出這種疾病的標誌性特徵，即暴食症，即對進食有無法滿足的慾望。這實際上是你的大腦在告訴你，儘管已經吃過東西了，但你仍然很餓。家人和照護人員能夠做的唯一控制暴食症的事情就是限制獲取食物，例如鎖上冰箱、垃圾桶和食品儲藏室。

Constant food preoccupation contributes to significant behavioral problems that can substantially disrupt daily life for those living with PWS and their families. Caregiver burden increases after the onset of hyperphagia and, in fact, has been measured to be higher than the burden experienced by caregivers for patients with Alzheimer's.

持續的對食物的過度關注會導致嚴重的行為問題，從而嚴重擾亂 PWS 患者及其家人的日常生活。暴食症發作後，護理人員的負擔會增加，事實上，經過測量，其負擔比阿茲海默症患者的照護者所承受的負擔還要高。

So, hyperphagia is a truly terrible condition for which no approved treatments have existed until the approval and launch of VYKAT XR. Recapping our label, the indication is for the treatment of hyperphagia in adults and pediatric patients four years of age and older with PWS. We believe that our label reflects VYKAT XR's favorable safety and tolerability profile, contains no box warning, no contraindication for diabetes, no exclusions for severity of hyperphagia, or no requirement for a risk evaluation and mitigation strategy or REMS program.

因此，暴食症是一種真正可怕的疾病，在 VYKAT XR 獲得批准和上市之前，尚無任何核准的治療方法。回顧我們的標籤，該藥物的適應症是治療患有 PWS 的成人和四歲及以上兒科患者的暴食症。我們相信，我們的標籤反映了 VYKAT XR 良好的安全性和耐受性特徵，不包含盒子警告、沒有糖尿病禁忌症、沒有對暴食症嚴重程度的排除，或不需要風險評估和緩解策略或 REMS 計劃。

VYKAT XR is to be taken orally once daily. Dosing is weight-based. There is a titration period of about six weeks, after which all people taking VYKAT XR should be on or close to their maintenance dose. The label has clear directions on how physicians should modify the dose if needed to address any side effects and minimize any dose interruptions. The commercial opportunity for VYKAT XR as a first-to-market therapy is significant.

VYKAT XR 每日口服一次。劑量以體重為準。滴定期約為六週，此後所有服用 VYKAT XR 的人都應達到或接近維持劑量。標籤上有明確的說明，說明醫生應如何在必要時調整劑量以解決任何副作用並盡量減少劑量中斷。VYKAT XR 作為首個上市療法，其商業機會十分巨大。

We have used existing claims data to confidently identify approximately 12,000 individuals diagnosed with PWS in the United States, of which approximately 10,000 should represent our total on-label addressable market. This excludes those individuals with PWS who are younger than four years old, others who may not be experiencing hyperphagia, or who may have comorbidities that make them ineligible for VYKAT XR.

我們利用現有的索賠數據，自信地識別出美國約 12,000 名被診斷患有 PWS 的個人，其中約 10,000 人應該代表我們整個標籤可尋址市場。這不包括那些四歲以下的 PWS 患者、可能沒有暴食症的患者或可能患有合併症而不適合使用 VYKAT XR 的患者。

As mentioned, as soon as we received FDA approval, we were prepared to begin accepting VYKAT XR start forms immediately, and patient treatments commenced in mid-April. This is a testament to the efforts of Meredith Manning, our CCO, and our world-class commercial team, whose prelaunch efforts to patients, physicians, and payers set the stage for a strong and successful launch, and to all of our employees who have worked so hard to prepare the company for this day.

如上所述，一旦我們獲得 FDA 批准，我們就準備好立即開始接受 VYKAT XR 啟動表格，並於 4 月中旬開始患者治療。這證明了我們的首席商務官梅雷迪斯·曼寧 (Meredith Manning) 和我們世界一流的商業團隊的努力，他們為患者、醫生和付款人所做的前期努力為強勁而成功的發布奠定了基礎，也證明了我們所有為公司這一天的準備而辛勤工作的員工。

I will now turn the presentation over to Meredith to discuss the early days of the launch and a few performance indicators that we plan to provide for the next several quarters to assist you in tracking our progress. Meredith?

現在，我將把演講交給梅雷迪斯 (Meredith)，討論發布的早期階段以及我們計劃在接下來的幾個季度提供的一些績效指標，以幫助您追蹤我們的進度。梅雷迪斯？

Thank you, Anish, and good afternoon, everyone. As Anish indicated, the launch readiness activities that we have been diligently executing for the past several months have ensured that we are well-positioned to deliver this much-needed treatment to patients as quickly as possible. We previously discussed the three pillars of activity that we view as crucial to a successful launch.

謝謝你，阿尼什，大家下午好。正如阿尼什所指出的，過去幾個月我們一直在努力執行的啟動準備活動確保我們能夠盡快為患者提供這種急需的治療。我們之前討論過我們認為對成功發布至關重要的三大活動支柱。

First, to establish VYKAT XR as the standard of care in hyperphagia in PWS. Second, to deliver operational excellence, and finally, to communicate the compelling VYKAT XR value proposition. And while it is still very early in the launch cycle of a new therapy, I am nonetheless very pleased with our launch efforts to date. We are seeing awareness on the part of people living with PWS and their caregivers, a willingness to prescribe on the part of treating physicians, and a recognition of the unmet need on the part of payers.

首先，將 VYKAT XR 確立為 PWS 患者暴食症的治療標準。其次，實現卓越的營運；最後，傳達引人注目的 VYKAT XR 價值主張。儘管新療法的發布週期還處於非常早期的階段，但我對我們迄今為止的發布工作感到非常滿意。我們看到，PWS 患者及其照護者的意識有所提高，主治醫生願意開處方，付款人也意識到了未滿足的需求。

We outlined several performance indicators we plan to provide over the next several quarters to help track our progress. The first of these is patient start forms. As of the end of the day yesterday, we have received 268 start forms, and that's in 29 business days since approval.

我們概述了計劃在未來幾季提供的幾個績效指標，以幫助追蹤我們的進展。其中第一個是患者開始表格。截至昨天結束，我們已經收到了 268 份開始表格，自批准以來已用了 29 個工作天。

And while it's early, it is very encouraging. We look forward to future quarterly updates when we will have an opportunity to collect more data and identify trends that are emerging. On April 14, we announced the first shipment of VYKAT XR to individuals living with hyperphagia in PWS. Since then, many patients have received their first dose of VYKAT XR and have started treatment. This reflects our dedication to ensuring timely access to treatment.

儘管還為時過早，但這已經非常令人鼓舞了。我們期待未來的季度更新，屆時我們將有機會收集更多數據並識別正在出現的趨勢。4 月 14 日，我們宣布向患有 PWS 暴食症的患者運送第一批 VYKAT XR。自那時起，許多患者已經接受了第一劑 VYKAT XR 並開始治療。這反映了我們致力於確保及時接受治療的決心。

The second performance indicator that we will be providing is the number of prescribers, which is 131 unique prescribers as of yesterday. Our field force is prioritizing engagement with top-tier providers with high patient volumes, which includes pediatric and adult endocrinologists, geneticists, and psychiatrists. We have also been impressed with the adoption of VYKAT XR from a broad base of prescribers, including those practicing in the community.

我們將提供的第二個績效指標是開處方者的數量，截至昨天，共有 131 名開處方者。我們的現場人員優先與擁有大量患者的頂級供應商合作，其中包括兒科和成人內分泌學家、遺傳學家和精神病學家。我們也對 VYKAT XR 受到廣大處方醫師（包括社區執業醫師）的青睞印象深刻。

This expanded reach indicates that our efforts to drive awareness of VYKAT XR availability, to communicate the efficacy and safety profile, and the need for a therapy across a broad prescribing spectrum are strong. Finally, in the future, we will be covering a detailed update on payer coverage.

擴大覆蓋範圍表明，我們為提高人們對 VYKAT XR 可用性的認識、傳達其功效和安全性以及對廣泛處方範圍內的治療的需求做出了巨大努力。最後，在未來，我們將介紹付款人覆蓋範圍的詳細更新。

That will be covered live and some color on payer policies, as this is critical to patients initiating and remaining on therapy. We previously talked about our payer outreach, which was mostly focused on emphasizing the complexity of hyperphagia in PWS patients, the consequences of not controlling hyperphagia, and the need for a new therapeutic option. We are very pleased with the feedback we have received from payers as a result of this outreach, and we are confident the VYKAT XR value proposition is resonating with commercial and government payers alike.

我們將現場報道這一點，並對付款人政策進行一些說明，因為這對開始和繼續治療的患者至關重要。我們之前討論過我們的付款人拓展，主要集中在強調 PWS 患者暴食症的複雜性、不控制暴食症的後果以及對新治療選擇的必要性。我們對此次推廣活動從付款人那裡收到的回饋感到非常滿意，我們相信 VYKAT XR 的價值主張會引起商業和政府付款人的共鳴。

While formal coverage policies can take anywhere from three to six months and sometimes longer to develop and implement, we do expect that VYKAT XR will achieve broad payer coverage in the United States. We are fully committed to ensuring that VYKAT XR is available to all eligible patients and that affordability is not a barrier to treatment.

雖然正式的覆蓋政策可能需要三到六個月甚至更長的時間來製定和實施，但我們確實預計 VYKAT XR 將在美國實現廣泛的付款人覆蓋。我們全力致力於確保所有符合條件的患者都能獲得 VYKAT XR，且負擔能力不會成為治療的障礙。

To this end, we have stood up Solena ONE, our single point of contact for patient services. Solena ONE was operational day one upon approval and is dedicated to supporting caregivers and health care providers in minimizing and navigating payer access obstacles. I will now turn the call over to James for a review of the company's financial statements for the first quarter.

為此，我們建立了 Solena ONE，作為患者服務的單一聯繫點。Solena ONE 在獲得批准的第一天就開始運營，致力於支持護理人員和醫療保健提供者盡量減少和克服付款人訪問障礙。現在我將把電話轉給詹姆斯，讓他審查公司第一季的財務報表。

Thank you, Meredith. We used $32.8 million of cash in operating activities during the three months ended March 31, 2025, and ended the period with $290 million of cash, cash equivalents, and marketable securities compared with $318.6 million as of December 31, 2024.

謝謝你，梅雷迪斯。在截至 2025 年 3 月 31 日的三個月內，我們在經營活動中使用了 3,280 萬美元的現金，期末現金、現金等價物和有價證券為 2.9 億美元，而截至 2024 年 12 月 31 日為 3.186 億美元。

As we have stated previously, based on our current operating plan, we believe our cash, cash equivalents, and marketable securities are sufficient to fund our operations through cash flow breakeven. Furthermore, with the approval of VYKAT XR, we now have an additional $75 million available to us in two tranches over the next 18 months under our loan agreement. Turning now to a few income statement items.

正如我們之前所說，根據我們目前的營運計劃，我們相信我們的現金、現金等價物和有價證券足以透過現金流盈虧平衡為我們的營運提供資金。此外，隨著 VYKAT XR 的批准，根據貸款協議，我們在未來 18 個月內將分兩批額外獲得 7,500 萬美元。現在來看看幾個損益表項目。

The company had not commercialized VYKAT XR in the three months ended March 31, 2025, and accordingly generated no revenue for the period.

該公司在截至 2025 年 3 月 31 日的三個月內尚未將 VYKAT XR 商業化，因此該期間未產生任何收入。

Research and development expenses for the first quarter ended March 31, 2025, were $13.5 million, which includes $4.3 million of noncash stock-based compensation. That compares to $14.6 million, which includes $2.4 million of noncash stock-based compensation for the same period of 2024. The cadence of our research and development expenditures fluctuates 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.

截至 2025 年 3 月 31 日的第一季研發費用為 1,350 萬美元，其中包括 430 萬美元的非現金股票薪酬。相較之下，2024 年同期的非現金股票薪酬為 1,460 萬美元，其中包括 240 萬美元。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, and administrative expense for the first quarter ended March 31, 2025, was $29.3 million, which includes $10.4 million of noncash stock-based compensation, and that compares to $8.5 million, which includes $4.0 million of noncash stock-based compensation for the same period of 2024.

截至 2025 年 3 月 31 日的第一季度，銷售、一般和行政費用為 2,930 萬美元，其中包括 1,040 萬美元的非現金股票薪酬，而 2024 年同期為 850 萬美元，其中包括 400 萬美元的非現金股票薪酬。

The increase reflects our ongoing investment in additional personnel and new programs in preparation for commercial launch and in support of our increased business activity. Total other income net was $2.0 million for the three months ended March 31, 2025, compared to total other income net of $2.1 million in the same period of 2024. Our net loss was approximately $43.8 million or $0.95 per basic and diluted share for the first quarter ended March 31, 2025, and $21.4 million or $0.59 per basic and diluted share for the same period in 2024. This concludes the financial overview, and I'll now turn the call back over to Anish for closing remarks. Anish?

這一成長反映了我們為準備商業發布和支持我們不斷增加的業務活動而對額外人員和新項目的持續投資。截至 2025 年 3 月 31 日的三個月，其他淨收入總額為 200 萬美元，而 2024 年同期的其他淨收入總額為 210 萬美元。截至 2025 年 3 月 31 日的第一季度，我們的淨虧損約為 4,380 萬美元，即每股基本虧損和稀釋虧損均為 0.95 美元；2024 年同期，我們的淨虧損約為 2,140 萬美元，即每股基本虧損和稀釋虧損均為 0.59 美元。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. While we are very pleased with what we are seeing in the early days of our US commercial launch, PWS is a global disease that impacts hundreds of thousands of patients around the world. In an effort to make VYKAT XR available to as many of these patients as possible, in parallel with our US commercial launch, we continue to progress along regulatory pathways in other geographies, the most important of which is the EU.

謝謝你，吉姆。雖然我們對美國商業發布初期所看到的情況感到非常高興，但 PWS 是一種全球性疾病，影響著全球數十萬名患者。為了讓盡可能多的患者能夠使用 VYKAT XR，在我們在美國進行商業化推廣的同時，我們將繼續在其他地區推動監管途徑，其中最重要的是歐盟。

We have stated previously that Europe also has a high unmet need among other patients with PWS. And as with the US, the PWS community has strong thought leader support, and patients are often concentrated around centers of excellence. Based on widely cited prevalence data, we estimate that there are as many as 9,500 patients with PWS in the EU4 and the UK.

我們先前曾指出，歐洲在其他 PWS 患者中也存在大量未滿足的需求。與美國一樣，PWS 社群擁有強大的思想領袖支持，患者通常集中在卓越中心周圍。根據廣泛引用的盛行率數據，我們估計歐盟四國和英國有多達 9,500 名 PWS 患者。

As we have stated previously, the submission of an MAA to the EMA is planned for the first half of this year. Before we open the call for questions, I want to close by reiterating how pleased we are with the early response we are seeing from the PWS community to the availability of VYKAT XR in the US.

正如我們之前所說，我們計劃在今年上半年向 EMA 提交 MAA。在我們開始提問之前，我想最後重申一下，我們對 PWS 社群對美國 VYKAT XR 上市的早期反應感到非常高興。

With that said, however, I remind everyone that start forms are a leading indicator, and there is a lag between receiving a start form and our specialty pharmacy partner purchasing product from us, which is when we record revenue.

話雖如此，但我還是要提醒大家，開始表格是一個領先指標，在收到開始表格和我們的專業藥房合作夥伴從我們這裡購買產品之間存在滯後，而這正是我們記錄收入的時候。

Therefore, as is typical in these types of launches, we expect revenues to start up modestly. It will take time to execute a full commercial launch, which includes patients scheduling visits with their health care providers, payers determining coverage policies, and the sales team being fully oriented to their territories. I look forward to sharing more details on our launch trajectory during our second quarter results call in August.

因此，正如此類產品的典型發布一樣，我們預計收入將會適度增長。全面商業發布需要時間，其中包括患者安排與醫療保健提供者的訪問、付款人確定承保政策以及銷售團隊完全面向其領域。我期待在八月的第二季業績電話會議上分享有關我們發布軌蹟的更多細節。

And with that, we will now open the call to your questions. Operator?

現在，我們將開始回答大家的提問。操作員？

(Operator Instructions) Yasmeen Rahimi, Piper Sandler.

（操作員指示）Yasmeen Rahimi、Piper Sandler。

Congrats on an outstanding quarter. Two questions. The first one is just, could you quantify how soon we could start converting the starting forms into revenue? The second question is, what do you project the time to fill to be at this point, and how will it change over the course of 2025? And I respect the wishes of two questions, and I'll jump back into the queue.

恭喜您本季取得了出色的成績。兩個問題。第一個問題是，您能否量化我們多久可以開始將起始表格轉換為收入？第二個問題是，您預計目前需要填補的時間是多少，以及 2025 年將如何變化？我尊重兩個問題的願望，我會重新回到隊列中。

Jim, do you want to take the first question?

吉姆，你想回答第一個問題嗎？

Yes, sure. Yes. So obviously, there are a number of steps that have to go through to get the start form into the hands of the specialty pharmacy and qualify, if you like, for the commercial drug because you're talking about the commercial drug. And then they, in turn, have to turn around and obviously place the order. And we anticipate that they will be cautious, if you like, in the inventory build that they'll want to take.

是的，當然。是的。因此，顯然，需要經過多個步驟才能將起始表格交到專科藥房手中並獲得商業藥物的資格（如果您願意的話），因為您談論的是商業藥物。然後，他們必須轉身並下訂單。我們預計，他們在建立庫存時會非常謹慎。

So, that's why we're just being mindful and saying we think modest revenues for Q2.

所以，這就是為什麼我們只是謹慎地認為第二季的收入會適中。

Thank you for the question. We're very happy with the speed at which we're able to convert the current start forms over to ship product. It's too soon right now. We're only 29 days in to give specific details on the turnaround time, but we're seeing what's ordinary with other rare disease launches, and we'll look forward to giving more information on the next call.

謝謝你的提問。我們對能夠將目前的起始表單轉換為出貨產品的速度感到非常滿意。現在還太早。我們只有 29 天的時間來提供有關週轉時間的具體細節，但我們看到其他罕見疾病產品的上市情況很正常，我們期待在下次電話會議上提供更多資訊。

Debjit Chattopadhyay, Guggenheim Securities.

古根漢證券的 Debjit Chattopadhyay。

Congrats. I have a couple. If I remember correctly, about 127 patients were enrolled in the Phase III program. What percentage of those patients are reflected in the current start forms?

恭喜。我有一對。如果我沒記錯的話，大約有 127 名患者參加了第三階段計畫。目前的開始表格中反映了這些患者中的多少百分比？

Meredith, do you want to take that?

梅雷迪斯，你想接受這個嗎？

Yes, happy to take that. So, as you'll recall, in the randomized withdrawal period, we had a total of 77 patients, 60 of whom are here in the United States. And so, the majority of those patients, we've received their start form, and we're looking at converting them over. So, we're very pleased with how fast our clinical operations team is executing on converting those patients.

是的，很高興接受。所以，你會記得，在隨機停藥期內，我們總共有 77 位患者，其中 60 位在美國。因此，我們已經收到了大多數患者的起病表格，並且正在考慮將其轉診。因此，我們對臨床營運團隊轉化這些患者的速度感到非常滿意。

And in terms of the free drug that patients are receiving right now, how should we think about it? How many days of free drug before they reimburse therapy (inaudible)?

那麼就患者目前正在接受的免費藥物而言，我們該如何看待它？報銷治療費用前可以免費用藥幾天（聽不清楚）？

Yes. So, I'll just remind everybody that we're really early in the launch, and it takes easily six months for many payers to arrive at a full coverage policy, sometimes a little bit longer. But we're very pleased with the reimbursement that we're seeing come in. We have a couple of programs. One, we have our bridge program with the clinical trial patients.

是的。因此，我只想提醒大家，我們才剛開始實施，許多付款人很容易就需要六個月的時間才能製定出全面覆蓋的政策，有時甚至需要更長的時間。但我們對收到的報銷金額感到非常滿意。我們有幾個項目。首先，我們與臨床試驗患者有橋樑計劃。

And then, if needed, if we're seeing more of an excessive delay on reimbursement or approval of the start form, then we have what we call a quick start. And we're looking at providing a 28-day prescription.

然後，如果需要，如果我們發現報銷或批准開始表格的延遲過長，那麼我們就會採取所謂的快速啟動措施。我們正在考慮提供 28 天的處方。

Kristen Kluska, Cantor.

克里斯汀·克魯斯卡（Kristen Kluska），領唱。

Let me also add my congrats on a great start here. My two questions. First, can you help us understand some of the ways we should be thinking about coverage decisions on a state-by-state level and how policies may differ? Then my second question is that the International PWSP Organization Conference is taking place next month, always a huge conference for these patients. So, I was hoping you could give some color about ways that Solena will be present there now that you have an approved therapy to talk about.

我還要對這裡的良好開端表示祝賀。我的兩個問題。首先，您能否幫助我們了解我們應該如何考慮各州的覆蓋範圍決策以及政策可能有何不同？我的第二個問題是，國際 PWSP 組織會議將於下個月舉行，對於這些患者來說，這始終是一個大型會議。因此，我希望您能提供一些關於 Solena 在那裡出現的方式，因為您現在已經有了一種已獲批准的治療方法可以討論。

Let me take the second part of it, Meredith, you can take the first one then. So, we definitely will have a significant presence at this United and Hope conference. It's the first time the international organization, as well as the two US-based organizations, are having a combined conference. It's a large conference, and it's going to be very well attended. Our commercial teams, med affairs teams, as well as clinical development teams will be there.

讓我來討論第二部分，梅雷迪斯，那麼你可以討論第一部分。因此，我們肯定會在這次聯合希望會議上發揮重要作用。這是該國際組織以及兩個美國組織首次舉辦聯合會議。這是一次大型會議，出席人數將會非常多。我們的商業團隊、醫療事務團隊以及臨床開發團隊都會出席。

We have several presentations and abstracts. So, we would like to see you all there. I know many of you are planning to be there as well. So, we're happy to engage and tell you more about what we are doing while we are there. Meredith, do you want to take the first part?

我們有幾個簡報和摘要。所以，我們很高興在那裡見到你們。我知道你們中的許多人也計劃去那裡。因此，我們很高興與您合作並向您詳細介紹我們在那裡所做的事情。梅瑞迪斯，你想參加第一部分嗎？

So, talking about state by state, Solena, we're participating in the MDRP program, the Medicaid program, which allows for states to start uploading VYKAT XR in the system as of May 1. But I'm sure you know that states vary. Some come on early and decide their policy within a month or so, and others can take up to July, August, or the second half of the year. So, we're pleased because we've already seen several states upload VYKAT XR in their system, and we're seeing coverage across various different states.

因此，就各州而言，索萊納，我們正在參與 MDRP 計劃，即醫療補助計劃，該計劃允許各州從 5 月 1 日起開始在系統中上傳 VYKAT XR。但我確信你知道各州的情況有所不同。有些人很早就開始行動並在一個月左右的時間內決定他們的政策，而其他人則可能需要等到七月、八月或下半年。因此，我們很高興，因為我們已經看到幾個州在其係統中上傳了 VYKAT XR，並且我們看到了不同州的覆蓋範圍。

Leland Gershell, Oppenheimer.

利蘭·格謝爾，奧本海默。

Great to see the early launch numbers. Maybe a question for Meredith, just teeing off your comment about the fact that you're seeing prescriptions coming from docs who may be outside of the initial targeted group of high prescribers. Could you share a bit more color on what you're seeing with respect to docs who may not have been in that 300 or so that you had targeted, what the maybe more community-type interest is looking like?

很高興看到早期發布的數據。也許我想問梅雷迪斯一個問題，只是想針對你的評論，你看到的處方來自可能不屬於最初目標高處方人群的醫生。您能否更詳細地介紹一下，對於可能不在您目標的 300 名左右的醫生，您觀察到的情況以及可能更多的社區類型的興趣是什麼樣的？

And then also, I wanted to ask with respect to Europe, if you could share now, maybe a bit early, as it will be time until approved there, but what look like in Europe in terms of dispersion of patients and how they're treated? Does it look like the states in terms of a smaller group of specialists who tend to treat those patients? Or is it a bit different from what it is in the States?

另外，我想問一下關於歐洲的情況，現在可以分享一下嗎？可能有點早，因為還需要一段時間才能獲得批准，但就歐洲的患者分佈和治療情況而言，情況如何？從治療這些患者的專科醫生人數較少這一點來看，它是否與各州的情況類似？或者它與美國的情況有點不同？

Why don't you take the first one? I'll take the second.

為什麼不選擇第一個呢？我要第二個。

So, thanks for the question, Leland. We are very encouraged and very pleased by the broad spectrum of writing that we've seen. We definitely anticipated a lot of excitement from the community, whether it be thought leaders, also community treating physicians who see PWS. I think what we can attribute that to is the fact that we had a lot of strong prelaunch awareness efforts and an opportunity to partner very closely with many thought leaders, as well as advocacy in the community to hold various different types of webinars upon launch.

所以，謝謝你的提問，Leland。我們看到如此廣泛的寫作風格，這讓我們感到非常鼓舞和高興。我們確實預料到社區會非常興奮，無論是思想領袖，還是治療 PWS 的社區治療醫生。我認為我們可以將其歸因於這樣一個事實：我們在發布前進行了大量的宣傳宣傳，並有機會與許多思想領袖密切合作，同時在社區中倡導在發佈時舉辦各種不同類型的網路研討會。

We have a lot of promotional materials and digital assets up and running. So, we believe that not only our sales force, but also our omnichannel efforts have enabled us to reach a broad spectrum of those physicians, including those in the community.

我們有大量宣傳材料和數位資產正在運行。因此，我們相信，不僅我們的銷售隊伍，而且我們的全通路努力使我們能夠接觸到廣泛的醫生，包括社區中的醫生。

Leland, regarding Europe, in general, I would say that the care for PWS in Europe is similar. But I would say that in some cases, it's probably more organized than the US. So, for example, when you think about a place like France, our understanding based on conversations and visiting some of the centers there is that virtually every individual with PWS there is seen as one of the few centers of excellence.

Leland，關於歐洲，總的來說，我認為歐洲對 PWS 的照顧是相似的。但我想說，在某些情況下，它可能比美國更有組織。因此，例如，當您想到法國這樣的地方時，根據我們的對話和訪問那裡的一些中心，我們發現幾乎每個患有 PWS 的人都被視為少數卓越中心之一。

And virtually, all of them are tracked by a centralized system. We believe something similar exists in Germany. And we think that the care of those patients is quite similar to here, with growth hormone being administered to the ones who are qualified for it, and really no other treatments being available. So, the environment for the use of a drug like Vykat is quite similar to what you would expect here.

實際上，所有這些都由一個集中式系統進行追蹤。我們相信德國也有類似的情況。我們認為這些患者的護理與這裡非常相似，對符合條件的患者註射生長激素，並且實際上沒有其他治療方法。因此，使用 Vykat 等藥物的環境與您預期的非常相似。

Brian Skorney, Baird.

布萊恩·斯科尼，貝爾德。

On the initial launch metrics. I hate to put you guys on the spot and make a direct comparison to another drug launch. But since (inaudible) is on your Board, I can't imagine that this conversation hasn't come up. But at 268 start forms over a little more than a month, you seem to be pacing Skyclarys almost exactly. They had 500 start forms in a little over two months.

關於初始發布指標。我不想讓你們為難，也不想直接將這款藥物與另一種新藥的上市進行比較。但既然 (聽不清楚) 是你們的董事會成員，我無法想像這個對話沒有出現過。但在一個多月的時間裡，您已經完成了 268 份起始表格，看起來您的步調幾乎與 Skyclarys 完全一致。他們在短短兩個多月的時間裡就收到了 500 份入職表格。

Their first quarter sales were almost half of what your full year consensus estimates here are right now. So, I'm just wondering why shouldn't Vykat, given the start form, given similar reimbursement dynamics, why would it track lower than Skyclarys? Because that's a much better launch curve than where consensus is currently modeling you at.

他們第一季的銷售額幾乎是目前全年普遍預期的一半。所以，我只是想知道，考慮到起始形式、考慮到類似的報銷動態，為什麼 Vykat 的價格會低於 Skyclarys？因為這是一個比目前共識模型更好的啟動曲線。

Brian, thanks for putting us on the spot. Jim wants to take the question.

布萊恩，謝謝你讓我們當場回答這個問題。吉姆想回答這個問題。

Well, the one thing I'd comment on, Brian, is that obviously, each company has its own dynamics. I would suggest that with our weight-based pricing, one has to be sensitive to the weight profile of the patients. And if you remember, we anticipated that we might have a younger set of patients coming through. So, that's one variable that we have to consider.

嗯，布萊恩，我想評論的一件事是，顯然每家公司都有自己的動態。我建議，採用基於體重的定價時，必須對患者的體重狀況保持敏感。如果您還記得的話，我們預計可能會有一群更年輕的患者前來就診。所以，這是我們必須考慮的變數。

Yes. And if I may, I would also just mention that I think Skyclarys had a lot of their commercial team in place for many years prior to launch, and their field team was out a couple of months prior. Remember that we had half of our sales team come on at the end of January. And then, we most recently brought on the rest of our field team, fully trained, and they're out as of April 27.

是的。如果可以的話，我還想提一下，我認為 Skyclarys 的許多商業團隊在產品發布前很多年就已經到位，而他們的現場團隊在產品發布前幾個月就已經出發了。請記住，我們的銷售團隊有一半的人是在一月底加入的。最近，我們召集了其餘的現場團隊成員，對他們進行了全面培訓，他們將於 4 月 27 日起撤出。

So, I think if you could give us some time to get our team out there to sell, I'm confident that we'll produce similar as well. Additionally, I would also bring your attention to the fact that we had a drug in channel by April 14. Skyclarys didn't. So, we are also operationalizing and converting start forms into paid patients.

因此，我認為如果您能給我們一些時間讓我們的團隊出去銷售，我相信我們也會生產類似的產品。此外，我還想提請您注意，我們在 4 月 14 日之前就已經在渠道中擁有一種藥物。Skyclarys 沒有。因此，我們也正在將開始表格操作化並轉化為付費患者。

Brian, I think it's also important to note that our prescription base, as Meredith has mentioned, is 131 physicians already. So, we think it's a broad-based build, which will sustain us over time. We're not as in the details with Skyclarys, maybe you are, but no direct comparisons to be made, but we certainly feel pretty enthusiastic about what we've seen so far.

布萊恩，我認為還需要注意的是，正如梅雷迪斯所提到的，我們的處方基礎已經有 131 名醫生了。因此，我們認為這是一個廣泛的建設，它將長期維持我們。我們並不像您那樣了解 Skyclarys 的細節，但沒有直接的比較，但我們對迄今為止所看到的情況感到非常興奮。

James Condulis, Stifel.

詹姆斯·康杜利斯（James Condulis），Stifel。

Congrats again on a great quarter, and following up on that last kind of discussion point in looking at other rare disease launches, is there a general ballpark on the percentage of patient start forms that actually convert to paid drug? It typically all patients actually convert, and it's just a matter of when. Like, just curious if you can provide any color there?

再次恭喜您度過了一個美好的季度，並根據最後一個討論點，在研究其他罕見疾病藥物的發佈時，患者起始表格實際轉化為付費藥物的百分比是否有一個大致的估計值？通常所有患者都會轉變，只是時間問題。例如，只是好奇您是否可以提供任何顏色？

And along those lines, as you think about the cadence of adding start forms throughout the next 2Q and beyond, do you expect it to accelerate or hold steady? Just like curious, any color you can provide there.

並且沿著這個思路，當您考慮在接下來的第二季度及以後添加開始表格的節奏時，您預計它會加速還是保持穩定？就像好奇一樣，您可以在那裡提供任何顏色。

Meredith?

梅雷迪斯？

So, I think it's too early to give a percentage, but we definitely are expecting to be comparable to other very strong rare disease launches. We have our internal metrics, but I think it's too early to share what we're looking at.

因此，我認為現在給出百分比還為時過早，但我們肯定期望能與其他非常強勁的罕見疾病產品相提並論。我們有內部指標，但我認為現在分享我們正在關注的內容還為時過早。

I think it's also fair to say that the unmet need here is pretty significant. No other treatments are available. So, we should expect to be at least comparable to other rare disease launches.

我認為也可以公平地說，這裡未滿足的需求相當大。沒有其他治療方法。因此，我們至少應該預期其能與其他罕見疾病藥物的上市相提並論。

Myriam Belghiti, LifeSci Capital.

Myriam Belghiti，生命科學資本。

Congrats on the update. A little bit off topic here. I was wondering if you have any comments on how or if the evolving tariff policies are affecting your business operations during the launch.

恭喜更新。這裡有點題外話。我想知道您是否對不斷變化的關稅政策如何影響您在發布期間的業務運營有何評論？

Yes, we have looked through our supply chain, and I would suggest that, really, we don't see any significant impact with the way the tariffs are currently being phrased. We do most of our manufacturing in the US, so we feel that it's not something that's going to impact us to any great extent.

是的，我們已經檢查了我們的供應鏈，我認為，我們確實沒有看到目前的關稅措辭方式帶來任何重大影響。我們的大部分製造業務都在美國進行，因此我們認為這不會對我們造成太大影響。

So, the drug substance and drug product vendors are both US-based. We do source some starting materials from outside the US. But as Jim said, no major impact.

因此，藥物成分和藥物產品供應商均位於美國。我們確實從美國以外採購一些起始材料。但正如吉姆所說，沒有重大影響。

Yale Jen, Laidlaw.

耶魯詹，萊德勞。

Congrats on the progression. Just two questions here. The first one is, could you give us a little bit of color for new patients, what are the steps of the procedures to ultimately get the drug prescribed? Do they need to start with the request of the start form, or do they need to visit the physician to get a prescription? And how does that work? And then I have a quick follow-up.

恭喜你有進步。這裡只有兩個問題。第一個問題是，您能否為新患者稍微介紹一下，最終獲得處方藥的程序步驟是什麼？他們是否需要從開始表格的請求開始，還是需要去看醫生才能獲得處方？那它是如何運作的呢？然後我有一個快速的後續問題。

Yes. So, the best way to think of it is that either the patient/caregiver will reach out to a physician, or the physician will reach out to them. They would typically need to see the physician. The physician would need to fill out a start form. The start form would go to the specialty pharmacy, and the specialty pharmacy would then take it from there to start the process of providing drugs to the patients.

是的。因此，最好的思考方式是，要么患者/照護者會聯繫醫生，要么醫生會聯繫他們。他們通常需要去看醫生。醫生需要填寫一份開始表格。起始表格將發送到專科藥房，然後專科藥房將從那裡開始為患者提供藥物的流程。

And maybe just one question. In terms of European development, I know you guys were talking before about whether you want to launch the product by yourself or seek a partner. Was there anything close to a decision at this point?

也許只有一個問題。關於歐洲發展，我知道你們之前討論過是否要自己推出產品還是尋求合作夥伴。此時是否已經接近做出決定了？

So, we continue down the path of assessing both options, whether we partner with it and there is a fair bit of interest in partnering, or we do it ourselves, and we are continuing to assess that as well. For us, the key next step has been to get the MAA submission in place with the EMA, and that should be happening in this quarter. And the decisions to commercialize and how to do it best will follow that. So we'll certainly keep you posted.

因此，我們將繼續評估這兩種選擇，無論是與其合作（並且有相當多的合作興趣），還是我們自己做，我們也將繼續評估這一點。對我們來說，下一步的關鍵是向 EMA 提交 MAA，這應該會在本季完成。商業化的決策以及如何最好地實現商業化將隨之而來。因此我們一定會及時向你通報最新情況。

(Operator Instructions) Ram Selvaraju, H.C. Wainwright.

（操作員說明）Ram Selvaraju, H.C.溫賴特。

This is Jade on for Ram. Again, congrats on the fantastic launch. So I just wanted to ask a little bit about how you see the competitive landscape right now in Prader-Willi hyperphagia. Do you really see that there are any noteworthy potential rivals in late-stage development? How are you guys thinking about this?

這是 Jade 為 Ram 所作所為。再次恭喜這次出色的發布。所以我只想問一下您如何看待目前普拉德威利暴食症的競爭格局。您是否確實看到後期開發中存在任何值得注意的潛在競爭對手？你們覺得這個怎麼樣？

So, I would say the late-stage rival, the obvious one, is Acadia with carbetocin, which, as you know, has already been studied in a large Phase II as well as a large Phase III study. It was submitted to the FDA and got an advisory committee, where it was voted down, received a CRL, and they're running another study at this time. I would say that is the later-stage competitor.

因此，我想說，後期競爭對手，顯而易見的，是含有卡貝縮宮素的 Acadia，正如你所知，它已經在大規模 II 期和 III 期研究中進行了研究。它被提交給 FDA 並得到了一個諮詢委員會的批准，但被否決了，收到了 CRL，他們目前正在進行另一項研究。我想說那是後期競爭對手。

It is, as you know, a three-times-a-day nasal product that requires temperature control. The other one, Aardvark, is apparently starting a Phase 3 study. The data that is publicly available is short-term, early data. So, it's hard for us to gauge what that will look like in a controlled study. Those are probably the two late-stage competitors.

如您所知，這是一種每天使用三次的鼻腔產品，需要溫度控制。另一家公司 Aardvark 顯然正在啟動第三階段研究。公開的數據是短期、早期數據。因此，我們很難判斷在一項受控研究中會出現什麼情況。這可能是兩個後期競爭對手。

And how are you seeing the ex-US opportunity for this drug shaping up? I know you've said about submitting the MMA, but how do you see this doing in Europe?

您如何看待這種藥物在美國以外市場的發展機會？我知道您曾經提到提交 MMA，但您認為這在歐洲會如何發展？

Yes. We think the opportunity is very significant. The genetics of Prader-Willi syndrome are such that the birth incidence is the same regardless of geography or ethnicity. So, you will see the same number of patients who have PWS based on the population. So, if you look at the EU four plus the UK, you're seeing about the same number as the US.

是的。我們認為這個機會非常重要。普拉德-威利症候群的遺傳學特徵是，無論地理或種族如何，出生發生率都是相同的。因此，根據人口數量，您會看到相同數量的 PWS 患者。因此，如果你看看歐盟四個國家加上英國，你會看到這個數字與美國大致相同。

In the EU 27, it's obviously a proportionately larger number. The unmet need is the same everywhere. So, nothing really exists to treat hyperphagia. So, we think it's a pretty meaningful market. In terms of rare disease pricing, we see that while Europe is a challenging place for pricing, rare diseases are the one place that appears to be an exception.

在歐盟27國中，這個數字顯然比例更大。未滿足的需求在任何地方都是一樣的。因此，實際上沒有任何東西可以治療暴食症。所以，我們認為這是一個非常有意義的市場。在罕見疾病定價方面，我們發現，雖然歐洲是一個定價困難的地方，但罕見疾病似乎是個例外。

So, we are pretty enthusiastic about it in Europe as well as in other geographies.

因此，我們對此在歐洲和其他地區都非常熱衷。

At this time, we have no other questions registered. So, I will turn the call back over to Anish.

目前，我們還沒有收到其他問題。因此，我將把電話轉回給 Anish。

Well, thank you, everyone, for listening in today, and we look forward to talking again for the second quarter call.

好吧，謝謝大家今天的收聽，我們期待在第二季電話會議上再次交談。

Thank you, sir. Ladies and gentlemen, this does indeed conclude your conference call for today. Once again, thank you for attending. And at this time, we ask that you please disconnect your lines.

謝謝您，先生。女士們、先生們，今天的電話會議到此結束。再次感謝大家的出席。此時，我們請求您斷開線路。