Soleno Therapeutics Inc (SLNO) 2025 Q3 法說會逐字稿

Good afternoon and thank you for standing by. Welcome to Soleno Therapeutics Third quarter 2025 Financial and operating results conference call and webcast. [Operator Instructions]

下午好，感謝您的耐心等待。歡迎參加 Soleno Therapeutics 2025 年第三季財務和營運業績電話會議和網路直播。[操作說明]

I would now like to introduce Brian Ritchie of LifeSci Advisors. Please go ahead.

現在我謹向大家介紹 LifeSci Advisors 的 Brian Ritchie。請繼續。

Thank you. Good afternoon, everyone, and thank you for joining us to discuss Soleno Therapeutics Third quarter 2025 Financial and Operating Results. Please note, we will be making certain forward-looking statements today.

謝謝。各位下午好，感謝各位參加本次關於 Soleno Therapeutics 2025 年第三季財務與營運業績的討論。請注意，我們今天將發表一些前瞻性聲明。

We refer you to Solano's SEC filings for a discussion of the risks that may cause actual results to differ from the forward-looking statements.

有關可能導致實際結果與前瞻性聲明差異的風險的討論，請參閱 Solano 向美國證券交易委員會提交的文件。

On the call with me today for Soleno are Anish Bhatnagar, Soleno's Chairman and Chief Executive Officer, Meredith Manning, Soleno's Chief Commercial Officer, and Jim Mackaness Soleno's Chief Financial Officer.

今天與我一起參加 Soleno 公司電話會議的有 Soleno 董事長兼首席執行官 Anish Bhatnagar、Soleno 首席商務官 Meredith Manning 和 Soleno 首席財務官 Jim Mackaness。

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

接下來，我將把電話交給阿尼什。

Thank you, Brian, and thank you everyone for joining us for our third quarter results call this afternoon.

謝謝布萊恩，也謝謝各位今天下午參加我們的第三季業績電話會議。

Following my brief opening remarks, Meredith will review the company's commercialization progress to date, and Jim will cover the company's financial statements for the third quarter. We will then open the call for questions.

在我簡短的開場白之後，梅雷迪思將回顧公司迄今為止的商業化進展，吉姆將介紹公司第三季的財務報表。接下來我們將開放提問環節。

We had an outstanding third quarter building on the strong launch momentum from our second quarter. Our total net revenue more than doubled from the second quarter to USD66 million and the company achieved profitability with a positive net income of USD26 million for the third quarter.

在延續第二季強勁開局勢頭的基礎上，我們第三季取得了非常出色的成績。第三季度，公司淨收入較第二季度翻了一番多，達到 6,600 萬美元，並實現盈利，淨利潤為 2,600 萬美元。

Our leading indicators, including patients start forms, unique prescribers, and numbers of lives covered, reflect growing awareness of VYKAT XR's potential to improve hyperphagia in those with PWS.

我們的主要指標，包括患者開始使用表格、獨特的處方醫生和覆蓋人數，都反映出人們對 VYKAT XR 改善 PWS 患者過度進食的潛力的認識不斷提高。

As the first and only FDA approved treatment for patients 4 years and older, for the primary feature of PWS, which is hyperphagia, VYKAT XR is providing a meaningful solution to individuals living with PWS, their caregivers, and physicians.

作為首個也是唯一一個獲得 FDA 批准的、針對 4 歲及以上患者治療 PWS 的主要特徵——過度進食的藥物，VYKAT XR 為 PWS 患者、他們的護理人員和醫生提供了一個有意義的解決方案。

I would like to take a moment to reiterate the complexity of Prader-Willi syndrome. In addition to the hallmark symptom of hyperphagia, people with PWS suffer from a broad range of potentially serious comorbidities, as well as significant behavioral problems.

我想藉此機會重申普拉德-威利症候群的複雜性。除了過度進食這種標誌性症狀外，PWS 患者還患有多種潛在的嚴重合併症，以及嚴重的行為問題。

And while people with PWS are living longer, some into their 50s and older, the mean age of death unfortunately still stands at around 30 years old.

雖然患有普瑞德威利症候群 (PWS) 的人壽命越來越長，有些人甚至活到了 50 多歲或更大，但不幸的是，平均死亡年齡仍然在 30 歲左右。

Patients with PWS experience a very high burden of disease compared to the general population. In particular, comorbidities associated with fluid overload, diabetes, respiratory failure, and congestive heart failure are common.

與一般人群相比，普拉德-威利症候群（PWS）患者承受著非常高的疾病負擔。尤其常見的合併症包括體液超負荷、糖尿病、呼吸衰竭和充血性心臟衰竭。

In a recently published registry of patients with PWS in Sweden, Gaeke and colleagues observed a greater than 20-fold increase in heart failure, a tenfold increase in venous thrombosis, and a five-fold increase in atrial fibrillation and pulmonary embolism.

在最近發表的瑞典 PWS 患者登記冊中，Gaeke 及其同事觀察到心臟衰竭增加了 20 倍以上，靜脈血栓形成增加了 10 倍，心房顫動和肺栓塞增加了 5 倍。

In a 40 year survey of mortality in patients with PWS, Butler and colleagues reported that respiratory failure and cardiac disease and cardiac failure together accounted for nearly half of all fatalities.

Butler 及其同事對 PWS 患者的死亡率進行了 40 年的調查，結果顯示，呼吸衰竭、心臟病和心臟衰竭加起來佔所有死亡人數的近一半。

The most common causes of death are respiratory failure, uncontrolled hyperphagia, and hyperphagia-related behaviors, cardiac causes, infection, obesity, and pulmonary embolism, with each accounting for greater than 5% of deaths.

最常見的死亡原因是呼吸衰竭、無法控制的過度進食和與過度進食相關的行為、心臟原因、感染、肥胖和肺栓塞，每一種原因造成的死亡人數都超過 5%。

A recent study in 2020 revealed that the mortality rate in people with PWS is substantially higher than the general US population at 2.7%. This translates to over 300 fatalities per year, assuming a population of approximately 12,000 people living with PWS in the US.

2020 年的一項最新研究表明，PWS 患者的死亡率遠高於美國一般人群，為 2.7%。這意味著每年將有超過 300 人死亡，假設美國約有 12,000 人患有普瑞德威利症候群 (PWS)。

The Wear XR clinical program established both substantial evidence of efficacy and a safety profile deemed approvable by the FDA based on a comprehensive phase three clinical program in 127 patients with over 400 patient years of exposure, including patients with nearly six years of continuous treatment.

Wear XR 臨床計畫在 127 名患者中進行了全面的第三期臨床研究，累計用藥時間超過 400 人年，其中包括接受近六年連續治療的患者，該計畫已證實其具有充分的療效和安全性，並獲得了 FDA 的批准。

At the end of Q3, at approximately six months in market, we had 764 active patients. Our real world experience, including efficacy, side effects, and discontinuation rates related to therapy, have been in line with our expectations.

第三季末，也就是產品上市約六個月後，我們有 764 位活躍患者。我們在實際應用上的經驗，包括療效、副作用和治療中斷率，都與我們的預期相符。

As discussed in VYKAT XR's FDA approved label, the most common adverse events reported in our clinical trials were hypertrichosis, edema, hyperglycemia, and rash.

正如 VYKAT XR 的 FDA 核准標籤中所述，我們在臨床試驗中報告的最常見不良事件是多毛症、水腫、高血糖和皮疹。

Most adverse events were self-limiting, with some needing dose adjustments, interruption, or other concomitant treatment. In particular, regarding fluid retention related adverse events post launch.

大多數不良事件都是自限性的，有些需要調整劑量、中斷治療或其他伴隨治療。特別是關於上市後與體液滯留相關的不良事件。

We see on a percent basis the incidence being lower than what we saw in clinical trials in spite of the post launch patients being more complex and having more comorbidities.

儘管上市後的患者病情更加複雜，合併症更多，但我們從百分比來看，其發生率低於我們在臨床試驗中看到的發生率。

Since approval, the discontinuation rate of VYKAT XR related to AEs was approximately 8% at the end of the third quarter, and the total discontinuation was approximately 10%.

自核准以來，截至第三季末，VYKAT XR 與不良事件相關的停用率約為 8%，總停用率約為 10%。

While the discontinuation rate has increased, it remains below our expected long-term rate based on our clinical trial data.

雖然停藥率上升，但仍低於我們根據臨床試驗數據預期的長期停藥率。

It is worth noting that we did see a disruption in our launch trajectory in the wake of a short seller report that was released in mid-August, mostly in the form of a lower number of start forms and increased discontinuations for non-serious adverse events.

值得注意的是，在 8 月中旬發布了一份做空機構報告後，我們的產品上市進程受到了乾擾，主要表現為啟動表格數量減少以及因非嚴重不良事件而終止的表格數量增加。

As we have anticipated, we have started to see patients return to therapy, often as withdrawal of VYKAT XR can bring a rapid return of PWS symptoms.

正如我們所預料的那樣，我們已經開始看到患者恢復治療，這通常是因為停用 VYKAT XR 會導致 PWS 症狀迅速復發。

We continue to educate physicians and families in the compelling clinical profile of VYKAT XR and we dedicated significant resources to these activities during the third quarter.

我們繼續向醫生和患者家屬普及 VYKAT XR 的卓越臨床特性，並在第三季度投入了大量資源用於這些活動。

Our team of patient and community educators known as the PACE team is educating families and caregivers on therapy expectations, administration, and monitoring at the time of first dose, and throughout the patient journey.

我們的患者和社區教育團隊（即 PACE 團隊）正在對患者家屬和照護人員進行教育，內容涵蓋首次用藥時的治療預期、用藥方法和監測，以及整個治療過程中的治療效果。

We're also hosting live community events in collaboration with advocacy organizations, healthcare providers, and caregivers of individuals already on VYKAT XR so they can share their experience with other caregivers contemplating initiating treatment.

我們還與倡導組織、醫療保健提供者和已接受 VYKAT XR 治療的患者的護理人員合作，舉辦現場社區活動，以便他們能夠與其他正在考慮開始治療的護理人員分享他們的經驗。

We're continuing our HCP education initiatives, including facilitating physician to physician programs, which allows physicians to learn more about VYKAT XR from experts who have experienced treating patients with PWS related hyperphagia.

我們將繼續推進 HCP 教育計劃，包括促進醫生之間的交流項目，讓醫生能夠從有治療 PWS 相關過度進食患者經驗的專家那裡了解更多關於 VYKAT XR 的信息。

Selena has received positive feedback on our engagement with the stakeholders in the PWS community, and we believe this will continue to fuel our growth and allow us to establish VYKAT XR as a standard of care for hyperphagia and those living with PWS.

Selena 收到了關於我們與 PWS 社區利益相關者互動的積極反饋，我們相信這將繼續推動我們的發展，並使我們能夠將 VYKAT XR 確立為治療多食症和 PWS 患者的標準療法。

I would now like to provide a brief update on our activities in support of potential approval of DCCR in Europe. As we market DCCR in the US as BAR.

現在我想簡要介紹一下我們為支持 DCCR 在歐洲獲得潛在批准所開展的活動。我們在美國以 BAR 的名義銷售 DCCR。

In parallel with our US commercial launch, we have continued to progress along regulatory pathways and other geography, the most prominent of which is the EU. In May we announced the submission and EMA validation of our marketing authorization application.

在我們於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，其中最突出的是歐盟。5月份，我們宣布已提交市場授權申請並獲得EMA驗證。

We received day 120 questions during the past quarter and are preparing our responses at this time. The nature of the questions are generally similar to what we discussed with the US FDA during the approval process.

上個季度我們收到了關於第 120 天的問題，目前正在準備答案。這些問題的性質與我們在審批過程中與美國FDA討論的問題大致相似。

Gaining approval to market DCCR in the EU would represent a meaningful expansion of our addressable market and remains a priority for us while we continue to progress our US launch.

獲得歐盟對 DCCR 的上市批准將顯著擴大我們的目標市場，在我們繼續推動美國上市的同時，這仍然是我們的首要任務。

Based on widely cited prevalence data, it is estimated that there are as many as 9,500 people living with PWS in France, Germany, Italy, Spain, and the UK combined.

根據廣泛引用的盛行率數據估計，法國、德國、義大利、西班牙和英國加起來共有多達 9500 人患有普拉德威利症候群 (PWS)。

Diagnostic rates are high. Patient care is often concentrated around centers of excellence, and as with the US, the PWS community has strong thought leader support. We will continue to keep you apprised of our progress there and in other territories.

診斷率很高。病患照護通常集中在卓越中心，而且與美國一樣，PWS 群體也得到了思想領袖的大力支持。我們將繼續向您通報我們在當地和其他地區的進展。

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

現在我將把電話交給梅雷迪思，請她介紹一下發表會的最新情況。梅雷迪絲。

Thank you, Aneesh, and good afternoon, everyone. As Anish mentioned, since approval, we've made remarkable progress in launching VYKAT XR.

謝謝你，阿尼什，大家下午好。正如 Anish 所提到的，自從獲得批准以來，我們在推出 VYKAT XR 方面取得了顯著進展。

Our success driving broad awareness and adoption reflects discipline execution grounded in effectively introducing VYKAT XR to the prescriber community, individuals with PWS, their caregivers and payers.

我們成功地推動了廣泛的認知和採用，這反映了我們以有效向處方醫生群體、PWS 患者、他們的護理人員和支付方介紹 VYKAT XR 為基礎的嚴謹執行。

We also attribute our launch success to Soleno's steadfast commitment to educating stakeholders, sharing individuals' experience on VYKAT XR therapy, and robust payer access. Soleno field teams are dedicated to ensuring comprehensive educational support on therapeutic expectations.

我們也將上市成功歸功於 Soleno 對利害關係人的堅定承諾、分享個人使用 VYKAT XR 療法的經驗以及強大的支付方管道。Soleno 現場團隊致力於為患者提供全面的治療預期教育支援。

Appropriate dosing and comprehensive monitoring, all critical factors for successfully integrating VYKAT XR into clinical practice and optimizing patient outcomes.

適當的劑量和全面的監測，都是將 VYKAT XR 成功整合到臨床實踐中並優化患者療效的關鍵因素。

It is worth noting that many prescribers and academic centers are still in the process of setting up their designated PWS clinics.

值得注意的是，許多處方醫生和學術中心仍在籌建其指定的 PWS 診所。

As a reminder, VYKAT XR is indicated to treat hyperphasia in adults and children four years of age and older with Prader-Willi syndrome.

提醒大家，VYKAT XR 適用於治療患有普拉德-威利症候群的成人和四歲以上兒童的語言過多症。

And hyperphasia is defined as extreme hunger, constant thoughts about food, and constant urge to eat that cannot be satisfied with food.

而食慾亢進的定義是：極度飢餓、對食物的持續思考、無法透過食物滿足的持續進食慾望。

Throughout the last several months, we have been hearing from families who are seeing meaningful benefit after completing titration and finding their optimal dose.

在過去的幾個月裡，我們不斷收到來自家庭的回饋，他們在完成劑量調整並找到最佳劑量後，獲得了顯著的益處。

While all experiences with VYKAT XR are unique, these experiences continue to reinforce the possibilities and real world impact of VYKAT XR.

雖然所有與 VYKAT XR 的體驗都是獨一無二的，但這些體驗不斷強化了 VYKAT XR 的可能性和現實世界的影響。

And through our responsibility to comprehensive education, we always encourage people to review the full prescribing information and medication guide for important safety information, which can be found on vykatxr.com.

出於我們對全面教育的責任，我們始終鼓勵人們查看完整的處方資訊和用藥指南，以獲取重要的安全信息，這些信息可以在 vykatxr.com 上找到。

Our commitment to patient services and market access underpins these efforts, ensuring timely access and reimbursement across all payer channels. I will now share the results of our key performance indicators, patient start forms, unique prescribers, and number of covered lives.

我們對病患服務和市場准入的承諾支撐著這些努力，確保在所有支付管道都能及時獲得治療和報銷。接下來，我將分享我們的關鍵績效指標、病患開戶表格、獨立處方醫師和投保人數的結果。

Cumulative patient start forms from launch through September 30 totaled 1,043, of which 397 were in the third quarter.

從上市到 9 月 30 日，累計患者入院申請表總數為 1,043 份，其中第三季有 397 份。

We previously commented that obtaining 646 patient start forms in our first quarter of launch was outstanding and included a bolus. We recognized the rapid uptake was due to strong operational excellence, a large unmet need, and pent up demand that carried into the early part of Q3.

我們之前評論過，在產品上市的第一季獲得 646 份患者啟動表格非常出色，其中包括一筆推注。我們意識到，快速成長的市場需求是由於卓越的營運能力、龐大的未滿足需求以及延續到第三季初的積壓需求。

By the end of Q3, 764 individuals were actively treated with ViCA XR. We believe.

截至第三季末，共有 764 人接受了 ViCA XR 的積極治療。我們相信。

That as more and more success stories are shared with the community, they will help create a firm foundation for continued growth.

隨著越來越多的成功案例與社區分享，這將有助於為持續發展奠定堅實的基礎。

The second performance indicator is the number of prescribers. We are continuing to make great strides with expanding our prescriber base. In Q3, we added an additional 199 new prescribers, bringing total unique prescribers as of September 30 to 494.

第二個績效指標是處方醫生的數量。我們在擴大處方醫生群體方面持續取得長足進展。第三季度，我們新增了 199 位處方醫生，截至 9 月 30 日，處方醫生總數達到 494 位。

Third quarter performance highlights significant progress within our key accounts. Over 50% of our top 300 providers have submitted start forms, signaling strong adoption.

第三季業績凸顯了我們在重點客戶方面取得的顯著進展。超過 50% 的前 300 家供應商已提交啟動表格，這表明市場反應熱烈。

Importantly, a significant share of SAR forms originated from healthcare providers who are associated with our KOL network. This is underscoring the effectiveness of our ability to educate physicians who play a pivotal role in influencing practice patterns.

值得注意的是，相當一部分 SAR 表格來自與我們 KOL 網路相關的醫療保健提供者。這凸顯了我們在教育醫生方面的有效性，而醫生在影響醫療實踐模式方面發揮關鍵作用。

We are also seeing strong adoption among community treaters, highlighting our expanded reach and continued growth beyond our core targets.

我們也看到社區治療人員也積極採用我們的產品，這凸顯了我們覆蓋範圍的擴大以及在核心目標之外的持續成長。

We have further strengthened our messaging this quarter by spotlighting real patient experiences, launching a new campaign, Make space for what matters that highlights VYKAT XR as a treatment that has the potential to lessen the relentless burden of hyperphasia, creating mental space for individuals living with PWS to focus on what truly matters to them.

本季度，我們透過重點介紹真實的患者體驗，進一步加強了我們的宣傳訊息，並推出了一項名為「為重要的事情騰出空間」的新活動，該活動強調 VYKAT XR 是一種有可能減輕多語症帶來的持續負擔的治療方法，為患有 PWS 的人們創造精神空間，讓他們能夠專注於對他們真正重要的事情。

Our third performance indicator is payer policies. We have been working diligently to secure broad coverage for VYKAT XR, resulting in policies that cover approximately 132 million lives at the end of the third quarter, including coverage policies with appropriate criteria from the top three national PBMs.

我們的第三個績效指標是支付方政策。我們一直在努力確保 VYKAT XR 獲得廣泛的覆蓋，截至第三季度末，已有約 1.32 億人獲得了保單保障，其中包括來自三大全國性藥品福利管理機構 (PBM) 的符合相應標準的承保保單。

We have achieved broad access coverage across all channels, commercial, Medicaid and Medicare, with a strong uptick in state Medicaid coverage where we had received reimbursed claims from approximately 40 state Medicaid programs through Q3.

我們已在所有管道（商業、醫療補助和醫療保險）實現了廣泛的覆蓋，其中州醫療補助覆蓋率大幅上升，截至第三季度，我們已收到來自約 40 個州醫療補助計劃的報銷索賠。

As I stated during our last earning earnings call, these positive coverage decisions demonstrate payers recognizing the seriousness of PWS and that that pairers recognize the seriousness of PWS, understand the true unmet need in treating hyperphasia, and appreciate the meaningful value VYKAT XR can deliver.

正如我在上次財報電話會議上所說，這些積極的醫保覆蓋決定表明，支付方認識到 PWS 的嚴重性，而醫保匹配方也認識到 PWS 的嚴重性，了解治療多語症方面真正的未滿足需求，並認識到 VYKAT XR 能夠帶來的有意義的價值。

This is great progress this early in launch because one of the perceived barriers to adoption. Among prescribers is the lack of coverage or the lengthy reimbursement path.

在產品發布初期就取得這樣的進展非常了不起，因為人們普遍認為這是推廣應用的一大障礙。處方醫生面臨的問題是缺乏醫療保險或報銷流程冗長。

As we move forward, we continue to invest in stakeholder awareness, education, and access resources to ensure every individual being treated with VYKAT XR, their family, and clinician feels supported throughout the treatment journey.

展望未來，我們將繼續投資於利害關係人的意識、教育和資源獲取，以確保每個接受 VYKAT XR 治療的患者、他們的家人和臨床醫生在整個治療過程中都能感受到支持。

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

現在我將把電話交給吉姆，讓他來審閱公司第三季的財務報表。

Thank you, Meredith.

謝謝你，梅雷迪思。

Total net revenue for the third quarter ended September 30, 2025 with USD66.0 million, which was more than doubled from USD32.7 million in the second quarter of 2025, and we achieved profitability with a positive net income of USD26.0 million for the quarter.

截至 2025 年 9 月 30 日的第三季度，公司淨收入總額為 6,600 萬美元，比 2025 年第二季的 3,270 萬美元翻了一番還多，並且實現了盈利，該季度淨利潤為 2,600 萬美元。

In addition, we generated USD43.5 million of cash from operating activities during the three months ended September 30.

此外，截至 9 月 30 日的三個月內，我們從經營活動中產生了 4,350 萬美元的現金。

At the end of the third quarter, we had USD556.1 million of cash equivalents and marketable securities. This includes the USD230 million of gross proceeds that we raised to an underwritten offering of our common stock in July.

第三季末，我們擁有 5.561 億美元的現金等價物和有價證券。這其中包括我們在 7 月透過承銷發行普通股籌集的 2.3 億美元總收益。

Our strong balance sheet ensures that we are sufficiently capitalized to continue to execute on an effective US launch of VYKAT XR while in parallel progressing towards regulatory approval and commercialization either on a stand-alone basis or with partners in the EU and other geographies.

我們強大的資產負債表確保我們有足夠的資金繼續有效地在美國推出 VYKAT XR，同時獨立或與歐盟及其他地區的合作夥伴一起推進監管批准和商業化。

As a reminder, VYKAT XR was approved in March of this year and therefore the company generated no revenue in the third quarter ended September 30, 2024.

提醒一下，VYKAT XR 於今年 3 月獲得批准，因此該公司在截至 2024 年 9 月 30 日的第三季沒有產生任何收入。

Cost of goods sold was USD1.1 million for the third quarter. Please note that prior to the FDA approval, costs associated with manufacturing VYKAT XR were expensed as research and development expenses.

第三季銷售成本為110萬美元。請注意，在獲得 FDA 批准之前，與 VYKAT XR 生產相關的成本已作為研發費用計入支出。

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 with at cost inventory, and consequently, cost of goods sold as a percentage of revenue will increase.

因此，該期間銷售成本的一部分包含了成本為零的存貨。展望未來，隨著我們繼續銷售 VYKAT XR，我們將耗盡零成本庫存，並以成本價庫存進行補充，因此，銷售成本佔收入的百分比將會增加。

Research and development expense for the third quarter was USD8.4 million, which includes USD2.2 million of non-cash stock-based compensation compared to USD30.1 million, which includes USD18.5 million of non-cash stock-based compensation for the same period of 2024.

第三季的研發費用為 840 萬美元，其中包括 220 萬美元的非現金股票選擇權費用；而 2024 年同期，研發費用為 3,010 萬美元，其中包括 1,850 萬美元的非現金股票選擇權費用。

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 move through submission, approval, and now commercialization.

我們的研發支出節奏會隨著臨床專案的進展、生產時間以及其他專案而波動，因為我們正在經歷提交、批准和現在的商業化過程。

Selling general and administrative expense for the third quarter ended September 30, 2025 was USD33.8 million, which includes USD7.8 million of non-cash stock-based compensation compared to USD49.2 million, which includes USD38.1 million of non-cash stock-based compensation for the same period of 2024.

截至 2025 年 9 月 30 日的第三季度，銷售一般及行政費用為 3,380 萬美元，其中包括 780 萬美元的非現金股票選擇權費用；而 2024 年同期，銷售一般及行政費用為 4,920 萬美元，其中包括 3,810 萬美元的非現金股票選擇權費用。

The increase in expense after removing stock-based compensation reflects our ongoing investment in additional personnel and new programs to support VYKAT XR commercial launch and in support of our increased business activities.

扣除股票選擇權費用後，支出增加反映了我們為支持 VYKAT XR 商業上市和支持我們不斷增長的業務活動而對額外人員和新項目進行的持續投資。

Total other income net was USD3.9 million for the three months ended September 30, 2025, compared to total other income net of USD3.6 million in the same period of 2024.

截至 2025 年 9 月 30 日止三個月的其他淨收入為 390 萬美元，而 2024 年同期其他淨收入為 360 萬美元。

Net income was approximately USD26.0 million or USD0.49 per basic and USD0.47 per diluted share for the third quarter ended September 30, 2025 compared to a net loss of USD76.6 million or USD1.83 per basic I diluted share for the same period in 2024.

截至 2025 年 9 月 30 日的第三季度，淨收入約為 2,600 萬美元，即每股基本收益 0.49 美元，每股攤薄收益 0.47 美元；而 2024 年同期淨虧損為 7,660 萬美元，即每股基本收益或每股攤薄虧損 1.83 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，現在我將把電話交還給阿尼什，請他作總結發言。阿尼什。

Thank you, Jim. In closing, we're very pleased with the trajectory we're on, and we will continue to work tirelessly to make the safe and effective therapy available to as many people living with PWS related hyperphagia as possible.

謝謝你，吉姆。最後，我們對目前的發展方向非常滿意，我們將繼續不懈努力，使盡可能多的患有 PWS 相關過度進食症的人都能獲得安全有效的治療。

We had an outstanding Q3 marked by noteworthy advancements in each of our key metrics from start forms to new prescribers to lives covered, all resulting in the doubling of our revenues from Q2 and leading to the company being cash flow positive.

第三季我們取得了卓越的成績，從起始表格到新處方醫生再到覆蓋人數，我們各項關鍵指標均取得了顯著進展，最終使我們的收入比第二季度翻了一番，並使公司實現了正現金流。

We look forward to continuing to deliver on the successful launch we have seen to date. And with that, we'll now open the call for questions.

我們期待繼續保持迄今為止的成功動能。接下來，我們將開始接受提問。

[Operator Instructions]

[操作說明]

Thank you. Our first question comes from the line of Paul Shoy from Goldman Sachs. Your line is open.

謝謝。我們的第一個問題來自高盛的保羅·肖伊。您的線路已開通。

Hi, congrats on the good performance in the quarter, with regard to to to the sales.

您好，恭喜貴公司本季銷售業績優異。

My first question for the team is, can you maybe comment on the restart rate with regard to the discontinuations you're seeing any color from the field in terms of how many patients are restarting and sort of the time to restart there would be helpful and then second.

我向團隊提出的第一個問題是，你們能否就重新開始治療的比例發表一下看法？關於你們觀察到的治療中斷情況，能否提供一些現場信息，例如有多少患者重新開始治療以及重新開始治療所需的時間？第二個問題是：

In terms of the number of patients on active drug that you disclosed with the press release, can you maybe comment on just how many of those are still waiting for insurance clearance versus the the patient start forms, any color there helping us connect the dots would be great. Thanks for taking the questions.

關於您在新聞稿中披露的正在接受活性藥物治療的患者人數，您能否說明一下其中有多少人仍在等待保險審批，又有多少人已經提交了患者用藥申請表？任何有助於我們了解情況的資訊都將不勝感激。謝謝您回答問題。

Sure. Thanks, Paul. So, on the restart, this is early. So we're just starting to see it now. There's a handful of people who've already started and we've anecdotally heard of others who are planning to start. So this is not a metric we can give meaningful numbers on, but it's early.

當然。謝謝你，保羅。所以，重啟還為時過早。所以我們現在才開始看到這種情況。已經有少數人開始了，我們也聽說還有一些人計劃開始。所以，我們目前還無法就此指標給出有意義的數字，但現在下結論還為時過早。

Your second question around number of patients on active drug, Meredith, do you want to answer that? Yeah.

梅雷迪思，你的第二個問題是關於正在服用活性藥物的患者人數，你想回答這個問題嗎？是的。

So, I think Paul what you're looking at is active versus or paid versus free and that's still a number that we're not reaching steady state and evolving so all of the 764 have claims that are being reimbursed is that the is that exactly what you're asking for?

所以，保羅，我認為你關注的是活躍用戶與付費用戶與免費用戶之間的對比，而這個數字我們還沒有達到穩定狀態，它還在不斷發展變化。所以，所有 764 例索賠都得到了報銷，這是否就是你想要的？

Maybe just some clarity, any color you can offer on the lag time between the start forms and the patients actually getting getting coverage that would be helpful too just so we can sort of triangulate maybe, how many patients might be backfilled in in the quarter to come.

或許您能提供一些更清晰的信息，例如從提交申請表到患者實際獲得保險覆蓋之間的滯後時間，這將對我們有所幫助，以便我們能夠大致估算出在接下來的一個季度中可能會有多少患者需要補種保險。

Yeah, Jim. Go ahead.

是的，吉姆。前進。

Yeah, so, I've been working closely with the specialty pharmacy on this. So, you get your start forms and then obviously there's a discontinuation cancellation that we've spoken to and then we're seeing the fill rate, somewhere around 30 days.

是的，我一直在與這家特藥藥房密切合作處理這件事。所以，你收到開始表格後，顯然會有中止取消的情況，我們已經和他們談過了，然後我們看到填充率大約在 30 天左右。

It's plus and minus, obviously depending on where the patient comes from and the amount of time to get through the benefits, but probably we're carrying about 1 month in backlog if you like, of start forms.

這有利有弊，顯然取決於患者來自哪裡以及處理福利所需的時間，但如果你願意的話，我們可能積壓了大約一個月的申請表格。

Okay great thank you for taking the questions.

好的，非常感謝你回答這些問題。

Thanks, Paul.

謝謝你，保羅。

Our next question is from Tyler Van Buren from PD call and your line is open.

下一個問題來自警局的泰勒·範·布倫，您的線路已接通。

Hey guys, congratulations on another strong quarter of sales and achieving profitability. Curious to hear you elaborate on the impact on discontinuations and lower patient start forms due to the short report during the quarter, and I guess how you're confident that it had an impact.

各位，祝賀你們又一個季度銷售業績強勁並實現盈利。我很想聽您詳細說明由於本季度報告較短而導致的停藥和患者起始表格數量減少的影響，以及您如何確信它產生了影響。

And then, is this impact going away as we enter Q4? What can you tell us about the early launch momentum observed into Q4 here and into year end?

那麼，隨著我們進入第四季度，這種影響會消失嗎？您能跟我們談談目前第四季以及年底前觀察到的早期產品上市勢頭嗎？

Yeah. Thanks, Tyler. So what we can tell you is that as we look at the quarter, we saw, I would say, a decrease in the August, September time frame.

是的。謝謝你，泰勒。因此，我們可以告訴大家的是，當我們回顧本季時，我們發現，我認為，在 8 月和 9 月期間，出現了下降。

Now, realize there's complexity of summer as well as the short report around the same time. So, with the slower August, September, we're also seeing no meaningful changes into October.

現在，要意識到夏季的複雜性，以及同期發布的簡短報告。因此，隨著 8 月和 9 月的銷售放緩，我們也沒有看到 10 月出現任何實質的變化。

So we think that there is an effect and we unfortunately think that this is on patients who've had the non-serious adverse events, probably the people who would have benefited tremendously had they stayed on therapy.

所以我們認為確實存在這種影響，但不幸的是，我們認為這種影響體現在那些出現非嚴重不良反應的患者身上，而這些患者如果堅持治療，可能會從中受益匪淺。

So, as Meredith has mentioned, as I also talked to, we're making a lot of efforts in reaching out to these people we're having individuals who are on drugs, talking to other patients who were on drugs.

正如梅雷迪思所提到的，也正如我和她談過的，我們正在努力接觸這些人，我們正在讓吸毒者與其他曾經吸毒的患者交談。

Yesterday we had a webinar with about 80+ families on that listening to a patient who's been on VYKAT XR and their experiences.

昨天我們舉辦了一場網路研討會，有 80 多個家庭參加，聽取了一位服用 VYKAT XR 的患者講述他們的經歷。

So we think that it's hard to say exactly when the effect would go away, but it's certainly something that we're making serious efforts on.

因此，我們認為很難確切地說出這種影響何時會消失，但這無疑是我們正在認真努力解決的問題。

And and when you say no meaningful impact in October, are you saying that October looks similar to August and September, or that there's no meaningful impact from the report and that you're seeing some of whatever rebound in October?

你說10月份沒有產生實質影響，是指10月份的情況與8月和9月類似，還是說這份報告沒有產生實質影響，而你看到的是10月份出現了一些反彈？

I'm saying it's looking somewhat similar to what we saw in September.

我的意思是，情況看起來和我們九月看到的情況有些類似。

Okay. Thank you.

好的。謝謝。

Our next question is from Maurice right there from Guggenheim. Your line is open.

我們的下一個問題來自古根漢美術館的莫里斯。您的線路已開通。

Hi, this is Morris on for DevJet. Congrats on a strong quarter. I have two questions. First of all, could you, elaborate a little bit on the average dose, across all patients that are currently on drugs?

大家好，我是DevJet的Morris。恭喜你們本季業績出色。我有兩個問題。首先，您能否詳細說明一下目前正在接受藥物治療的所有患者的平均劑量？

And secondly, for your existing prescribers, could you estimate what percent of the PWS patients are currently on VYKAT XR?

其次，對於您現有的處方醫生，您能否估計目前有多少百分比的 PWS 患者正在服用 VYKAT XR？

What I'm trying to get at is there's still room to grow within those existing prescribers or the future growth need to come from new prescribers?

我想表達的是，現有處方醫生是否還有發展空間，還是未來的成長需要來自新的處方醫生？

Thank you.

謝謝。

Yeah, I'll take the second part of it, and Meredith can elaborate on it. There's definitely room to grow. We have about 1,000 start forms right now. That's about 10% of the tap.

好的，我負責後半部分，梅雷迪斯可以補充詳細說明。還有很大的發展空間。我們現在大約有1000份入學申請表。那大約是水龍頭出水量的10%。

So I'd say across the board there is room to grow, and that's particularly the case with our KOL accounts where Meredith mentioned that more than half of them have written scripts, but as we had anticipated.

所以我覺得各方面都有發展空間，尤其是我們的 KOL 帳戶，Meredith 提到其中超過一半的人都寫過劇本，但這正如我們所預料的那樣。

Their practices are pretty full, and they are, they appear to be prescribing more when they see patients in their regular cadence of one to two times a year.

他們的診療工作非常繁忙，而且，當他們按常規頻率（每年一到兩次）接診病人時，似乎會開出更多的處方。

So we expect that to remain and we expect that over time we'll be getting access to those patients, but Meredith.

所以我們預計這種情況會持續下去，我們也預計隨著時間的推移，我們將能夠接觸到這些患者，但梅雷迪思。

Go ahead. Yeah, adding on to what Aneesh said, we're very pleased with the broad prescriber base and we continue to add new prescribers on a daily basis, so we know that there's, significant amount of room for growth and as Anish stated on the numbers with regard to the TAM.

前進。是的，補充一下 Aneesh 的說法，我們對廣泛的處方醫生群體非常滿意，而且我們每天都在增加新的處方醫生，所以我們知道還有很大的增長空間，正如 Anish 就 TAM 數據所指出的那樣。

You had asked about the average dosing, so again that's still an evolving number. One thing that we shared last earnings call, which we'll continue to reiterate, is that the majority of our patient population is coming in between four and 26 age.

您問過平均劑量，所以這仍然是一個不斷變化的數字。我們在上一次財報電話會議上分享過一點，我們將繼續重申，那就是我們的大多數患者年齡在 4 歲到 26 歲之間。

And we are continuing to make progress in the younger adults, so the 27 to 45, if you will. And we are seeing the average weight coming in a little bit heavier than what we saw in our clinical trial.

我們在年輕成年人群（即 27 至 45 歲人群）中也持續取得進展。我們發現平均體重比我們在臨床試驗中看到的要重一些。

Thank you.

謝謝。

Our next question is from Kristen Kuka from Canter. Your line is open.

我們的下一個問題來自坎特大學的克里斯汀·庫卡。您的線路已開通。

Yeah, hi, this is Rick Miller on for Kristen. Thanks for taking our questions.

大家好，我是瑞克米勒，替克莉絲汀為你報道。謝謝您回答我們的問題。

So you mentioned the discontinuations being up after, some of the non-serious AEs after the short report. Can you give us a sense for kind of the kind of the profile of the AEs that were kind of leading to these discontinuations is this strictly sort of the on label safety profile that we're used to or anything else you could talk about there, thank you.

所以你提到，在短期報告發布後，一些非嚴重不良事件導致停產。您能否大致介紹一下導致這些停產的不良事件的特徵？這是否完全符合我們通常所了解的標籤安全特性？或者您還有其他想說的話嗎？謝謝。

Sure. So, yes, these are on label AEs, typically, low levels of peripheral edema or you can have hyperglycemia. And when I was referring to non-serious adverse events, as you can look at fairs and you can see that a a vast majority of events that are reported are non-serious.

當然。是的，這些都是說明書上列出的不良反應，通常表現為輕微的周邊水腫或高血糖。當我提到非嚴重不良事件時，你可以看看展會，你會發現報告的事件絕大多數都是非嚴重事件。

So I think what's happened is a concern that's been created because of the adverse event profile of the drug, which is non-serious, most patients are able To sort of power through and we'll start to see the benefits and we're seeing that in real life.

所以我認為，現在的問題是，這種藥物的不良反應雖然不嚴重，卻引發了人們的擔憂。大多數患者都能挺過來，我們會開始看到藥物的益處，而且我們在現實生活中也看到了這一點。

We've had numerous anecdotes of people who have stayed with low levels of edema, some levels of hyperglycemia, and have done really well from an efficacy perspective. So yes, the adverse events remain sort of on label, and what we are seeing is predominantly non-serious.

我們收到過很多這樣的軼事：有些人雖然水腫程度較低，血糖值也有些高，但從療效的角度來看，他們的治療效果非常好。所以，是的，不良反應基本上符合預期，而且我們看到的主要是非嚴重不良反應。

If Our next question is from Yasmin Rahimi from Piper Center. Your line is open.

下一個問題來自Piper Center的Yasmin Rahimi。您的線路已開通。

Hi team, congrats on a strong quarter. I guess, team, when you look at, given that August and September was impacted by the short report, like What is this, is the hesitation among patients that are under the care of general endocrinology?

各位同事，恭喜你們本季業績優異。我想，各位，考慮到八月和九月受到短期報告的影響，這是否意味著接受普通內分泌治療的患者有所猶豫？

Is there a quantification on who are the type of patients or the type of physicians that need additional outreach and communication? Like, what is that profile look like where you guys are targeting to really have in-depth education for that sort of question one.

是否有量化數據來確定哪些類型的患者或哪些類型的醫生需要額外的宣傳和溝通？例如，你們的目標群體是什麼樣的？你們希望他們能對這類問題進行深入的教育。

And then question two is like, how do you, obviously, I think that Tyler's question that that, October is looking like more like September.

然後第二個問題是，你如何，顯然，我認為泰勒的問題是，十月看起來更像是九月。

How will you continue to communicate when we're going to When are we going to be out of this, I guess, fear or worry by this report, like any visibility like that, the rest of the quarter could be very much rebounding substantially, and what are your, what would your disclosures be around it.

我們將如何繼續溝通？我們什麼時候才能擺脫這份報告帶來的恐懼或擔憂？就像任何類似的可見性一樣，本季剩餘時間可能會大幅反彈，你們會就此披露哪些資訊？

So we don't expect to do sort of intra-quarter disclosures and as you can imagine, Yasmine that there's a lot of complexity in treating hyperphagia and PWS. We're the first drug and we're learning what the market's like and the prescribers are learning how to use the drug.

因此，我們預計不會進行季度內披露，正如你所想，Yasmine，治療過度進食和 PWS 非常複雜。我們是第一個推出這種藥物的，我們正在了解市場狀況，處方醫生也在學習如何使用這種藥物。

So, we realized that last quarter was perhaps a short report, perhaps it was summer, perhaps it was people going to summer camp.

所以，我們意識到上個季度的報告可能比較短，可能是因為正值夏季，可能是因為人們都去參加夏令營了。

And now this quarter we're going to have Thanksgiving and Christmas, which obviously have meaning for everyone, but for PWS and those with hyperphagia, it has a very different meaning.

這個季度我們將迎來感恩節和聖誕節，這對每個人來說顯然都意義非凡，但對於普瑞德-威利症候群患者和患有過度進食症的人來說，它的意義卻截然不同。

So we have to navigate this. We have to see what it looks like and we have to see how prescribers also get accustomed to it, as we said earlier.

所以我們必須想辦法解決這個問題。我們必須看看它的實際效果，也要看看處方醫生如何適應它，正如我們之前所說。

There are some hospitals, institutions that are trying to create PWS clinics to sort of, figure out how to best administer VYKAT XR and sort of make sure that they can follow these patients. Meredith, would you like to add something?

一些醫院和機構正在嘗試建立 PWS 診所，以便弄清楚如何最好地管理 VYKAT XR，並確保能夠追蹤這些患者。梅瑞迪思，你還有什麼要補充的嗎？

Yeah, I think, Yasmine, we're really pleased about the program that actually we launched in August. More of your traditional speakers bureau program, and we've had really strong adoption with that.

是的，雅斯敏，我覺得我們對八月啟動的這個計畫非常滿意。更偏向傳統的演講者經紀項目，我們在這方面取得了非常好的迴響。

So we have recruited some of the national PWS experts to be speakers in the program, which allows for both virtual and in-person as well as an opportunity to do expert on demand, and we've seen really strong interest in that and you asked about the profile of the prescribers who are being educated.

因此，我們招募了一些全國性的 PWS 專家擔任該計畫的演講嘉賓，該計畫既可以進行線上演講，也可以​​進行線下演講，還可以提供專家按需演講的機會。我們看到大家對此表現出了非常濃厚的興趣，您問到了接受訓練的處方醫生的概況。

I think what you're leaning towards, and you're correct in thinking that as we're adding these new prescribers on a daily basis who are out in the community who potentially have only one or two PWS patients.

我認為你傾向於這種觀點，而且你的想法是正確的，因為我們每天都在增加新的處方醫生，他們可能在社區裡只有一兩個普瑞德-威利綜合徵（PWS）患者。

Those are the ones who really need additional education on the disease state in general, as well as how to integrate VYKAT XR into their practice.

這些人確實需要接受關於疾病狀態的額外教育，以及如何將 VYKAT XR 融入他們的實踐中。

So we've received, as Anish said in his prepared comments, really positive feedback on these programs.

正如阿尼什在事先準備好的演講稿中所說，我們收到了對這些項目的非常正面的回饋。

And, additionally we're continuing to educate caregivers and families so we've had live patient programs and we'll continue to do that in Q4, as well as webinars all received very well.

此外，我們還在繼續對護理人員和家屬進行教育，因此我們舉辦了現場患者項目，並將在第四季度繼續舉辦，此外還有網路研討會，這些都受到了很好的反響。

Thank you.

謝謝。

Our next question is from Leland Kershaw from Oppenheimer. Your line is open.

我們的下一個問題來自奧本海默公司的利蘭·克肖。您的線路已開通。

Hey, good afternoon, thanks for taking our questions. We just want to maybe understand, with respect to the treating physician population of patients who are on VYKAT XR, you're having close to 500 unique prescribers, at the, end of Q3.

嗨，下午好，謝謝你們回答我們的問題。我們只是想了解一下，關於正在接受 VYKAT XR 治療的醫生群體，截至第三季末，你們有近 500 位不同的處方醫生。

But in your investment materials, you said that about 300 providers are primarily creatures of about 2,100 PWS patients.

但在您的投資資料中，您提到大約 300 家供應商主要服務約 2100 名 PWS 患者。

So just going back to something we've talked about in the past, is it the case that we should think about these reports of adverse events as more likely to occur amongst patients who are being cared for by those who are less specialized in preability treatment and therefore maybe less.

所以，回到我們過去討論過的一個問題，我們是否應該認為這些不良事件的報告更有可能發生在那些由不太擅長預防性治療的醫生照顧的患者身上，因此這些患者可能接受的治療較少。

Astute at at managing some of the side effects wondering if you could share your insights there. Thank you.

我很擅長應付一些副作用，不知道您是否可以分享您的經驗。謝謝。

Yeah, thanks Leland. I think it's fair to say that just a reminder, the adverse event profile and if you look at edema as an example or food retention related events, in our clinical trials is about, 20%.

謝謝你，利蘭。我想說，提醒一下，不良事件概況，例如水腫或食物滯留相關事件，在我們的臨床試驗中發生率約為 20%。

What we are seeing now, at least the reported events are actually lower than that. So it's actually a pretty small minority that has these events, and most of the events that are happening are low grade events.

我們現在看到的，至少已報告的事件數量實際上低於這個數字。所以實際上只有極少數人會遇到這類事件，而且發生的事件大多是低階事件。

So, these are most often things that may not even need treatment. So, what you're really probably concerned about is significant adverse events or serious adverse events.

所以，這些情況通常可能根本不需要治療。所以，你真正可能擔心的是重大不良事件或嚴重不良事件。

And those we do worry that pa physicians who are out there who don't have experience in using VYKAT XR and have one patient on it and choose a patient who had significant comorbidities, etc.

我們確實擔心，有些醫生沒有使用 VYKAT XR 的經驗，只有一個病人正在服用，卻選擇了有嚴重合併症的病人等等。

And how would they manage the side effects. So we do, we are concerned about that, and that's something as Meredith mentioned, we have a significant effort in trying to mitigate.

他們將如何應對副作用？所以，我們確實很關注這個問題，正如梅雷迪思所提到的那樣，我們正在努力減輕這個問題的影響。

So we have our field teams, our MSLs who go out, have conversations with these physicians about how to manage these things better.

所以我們有一個現場團隊，我們的醫學聯絡官，他們會走出去，與這些醫生進行交流，討論如何更好地管理這些問題。

And we have physician to physician programs where there's an expert on demand thing where you can call a physician who has significant experience and that's actually been used very successfully recently.

我們還有醫生對醫生的項目，提供專家諮詢服務，您可以聯繫到經驗豐富的醫生，而且最近這項服務也取得了非常成功的應用。

Great and just wanted to ask you, is, the time to securing reimbursement is that changed from the past is that improved, increased, or is it the is about the same in terms of going from start form to pull through thanks.

太好了，我只是想問一下，從開始填寫表格到最終拿到報銷款所需的時間與過去相比是否有所變化？是縮短了、增加了，還是差不多？謝謝。

There it is. Yeah, so Leland, I think on our last earnings call we mentioned that we were looking at a turnaround time targeting for 30 days.

找到了。是的，Leland，我想我們在上次財報電話會議上提到過，我們的目標是在 30 天內完成周轉。

We're that's still our target that's still optimal, but as you know during the first year of launch it can vary depending on the channel but that's exactly what we're targeting is the 30 days.

我們的目標仍然是30天，這仍然是最佳狀態，但正如你所知，在產品上市的第一年，情況可能會因通路而異，但這正是我們努力的目標，即30天。

Great thanks very much.

非常感謝。

Our next question is from Brian Scorny from Beer. Your line is open.

我們的下一個問題來自 Beer 的 Brian Scorny。您的線路已開通。

Hey, good afternoon, everyone. Thank you for taking the question. Yeah, I just wanted to try to get a little more clarification on your comments on the disruption that occurred over the summer and how it's specifically manifesting in the numbers.

大家好，下午好。感謝您回答這個問題。是的，我只是想更清楚地了解您對夏季發生的混亂情況的評論，以及這種混亂情況是如何具體體現在數據中的。

Was there both a slowdown in start forms that you're saying and an increase in discontinuations and implying that you would otherwise have more than 400 start forms.

你所說的起始形式減少和終止形式增加，是否意味著你原本會有超過 400 個起始形式？

And or lower than 8% discontinuation due to AEs otherwise, or is there another figure in terms of missed refills that isn't specifically being called out on on a quantification measure?

或者，因不良事件導致的停藥率低於 8% 嗎？或者，在漏服藥方面，還有其他數據沒有在量化指標中明確指出嗎？

So, thanks, Brian. I think where I was trying to go with this is that we saw a decrease in August, September and we realized it's also summer.

謝謝你，布萊恩。我想表達的是，我們在八月和九月看到了下降，我們也意識到現在正是夏天。

So we think it was some combination of summer people in camps as well as the short report that caused the start forms to decrease. Now, we obviously cannot pinpoint that, such and so didn't come in because of they read something or whatever.

所以我們認為，夏令營的暑期人員以及簡短的報告等因素共同導致了入學申請表數量的減少。現在，我們顯然無法確定具體原因，某某人沒有進來是因為他們讀了什麼書或其他什麼原因。

But I do think that could, that was likely a factor. We don't have any specific metrics and refills, etc. But what we are thinking through is.

但我認為那很可能是個因素。我們沒有特定的指標和補貨計劃等等。但我們正在思考的是…

If you have patients who have non-serious adverse events who discontinue and anecdotally call into the specialty pharmacy and say, I read something that I didn't like and I'm concerned and I'm not going to continue on drugs, that's what we are basing the idea on that perhaps it's, these anecdotal things that are out there and misleading that are leading to discontinuation that wouldn't have occurred.

如果患者出現非嚴重不良反應後停止用藥，並私下致電專科藥房說：“我讀到了一些我不喜歡的東西，我很擔心，我不會繼續服用這些藥物”，那麼我們就會基於這樣的想法：也許正是這些流傳的、具有誤導性的軼事導致了原本不會發生的停藥。

Our next question is from James Conduis from Stavi. Your line is open.

我們的下一個問題來自 Stavi 的 James Conduis。您的線路已開通。

Hey, thanks for taking my question. I wanted to ask one on efficacy. Obviously it takes some time to see it, but we're now approaching six months kind of plus or so.

嘿，謝謝你回答我的問題。我想問一個關於療效的問題。顯然，這需要一些時間才能看到結果，但現在已經過去六個月左右了。

And just curious what your early kind of learnings are there, what you've heard, how you kind of think that's playing into the current discontinuation rates and what that may do to to discontinuation rates over time as as kind of patients stay on drugs for longer.

我很好奇你早期了解到的情況，你聽到了什麼，你認為這會如何影響目前的停藥率，以及隨著患者服藥時間延長，這會對停藥率產生怎樣的影響。

Any color there would be appreciated. Thanks.

任何顏色都歡迎。謝謝。

So, James, thank you for asking about efficacy. We are starting to hear anecdotes across the board on good things that are happening to these patients. There's tons of examples, we got a call the other day about a child who went without food for nine hours.

詹姆斯，謝謝你詢問療效方面的問題。我們開始陸續聽到關於這些患者身上發生的良好情況的軼事。例子數不勝數，前幾天我們接到一個電話，說有個孩子九個小時沒吃東西了。

We talked to a mom about a child who sat in temple at a memorial service for several hours calmly without any tantrums.

我們和一位母親談論她的孩子在寺廟參加追悼會時，平靜地坐了好幾個小時，沒有發脾氣。

We heard from an adult who lives in a group home who will, after, years be able to travel alone to see their family in Florida alone. So it is starting to happen.

我們訪問了一位住在集體宿舍的成年人，他表示多年後將能夠獨自前往佛羅裡達州探望家人。事情正在發生。

We're seeing things happen across the board and we think it will make a difference to these discontinuation rates. And as I was mentioning, we've started a series of patient webinars where those who are on drugs and their families are able to share their experiences with others.

我們看到各方面都在變化，我們認為這將對停售率產生影響。正如我剛才提到的，我們已經開始舉辦一系列患者網路研討會，讓正在吸毒的人及其家人能夠與他人分享他們的經驗。

And the first one of those literally had, 100 plus people who signed up for it. So there is a lot of interest, and this is exactly the sort of thing that will turn things around.

第一個活動確實吸引了 100 多人報名參加。所以大家對此很感興趣，而這正是能夠扭轉局面的事。

Great I appreciate it.

太好了，謝謝。

Thanks. Our next question is from Derek Artila from Wells Fargo. Your line is open.

謝謝。下一個問題來自富國銀行的德里克·阿蒂拉。您的線路已開通。

Hey, thanks for taking the questions and congrats on the progress, I guess first I guess. Obviously you're saying what you're seeing in October is kind of resembling, September trends.

嘿，謝謝你回答這些問題，也祝賀你的進展，我想這是第一點吧。顯然，你的意思是說，你在十月看到的趨勢與九月的趨勢有些相似。

I guess is the bolus over or do we think that it resumes as people get more comfortable or the messaging gets better, education gets better.

我猜想，這波衝擊是不是已經結束了，還是說隨著人們越來越適應，或是訊息傳遞和教育程度提高，衝擊又會再次出現？

So that's question number one. And then just quickly on the EU, I know you mentioned about the 120 day questions.

這是第一個問題。然後，關於歐盟，我再簡單提一下，我知道你提到了120天的問題。

I don't know if you can kind of characterize some of those requests, but do they improve your confidence of an eventual approval in Europe? Thanks.

我不知道您是否可以對這些要求進行一些概括，但它們是否能提高您最終在歐洲獲得批准的信心？謝謝。

Sure, so in terms of the bolus, we, this is one of those situations where when you have the first drug for an indication, it's hard to tell what it's supposed to look like.

當然，就推注劑量而言，這種情況就是，當你第一次使用某種適應症的藥物時，很難說它應該是什麼樣子。

So we're finding out as we go, but if you remember some of the conversations we had prior to launch, we said we don't know if there will be a bolus, but what we are expecting to see is a slow, steady build up over time with a time of 10,000 patients. There's a lot of them out there and it takes time for physicians to get accustomed to using a drug.

所以我們也在摸索前進，但如果你還記得我們在上市前的一些談話，我們說過我們不知道是否會有快速增長，但我們預計會看到隨著時間的推移，患者數量會緩慢、穩定地增長，最終達到 10,000 人。市面上有許多這類藥物，醫生需要時間才能習慣使用一種藥物。

For an indication that has not really had a treatment before, so it's hard for us to comment on, does the bolus come back or not. I think by definition a bolus does not come back, and we're looking for a steady state, that will continue over time and will continue to build a solid base of revenue that we have.

對於以前從未接受過治療的適應症，我們很難評論，藥物是否會反彈。我認為，根據定義，大劑量注射不會再次生效，我們追求的是穩定狀態，這種狀態會隨著時間的推移而持續，並繼續為我們現有的穩固收入基礎奠定基礎。

On the EU, I can't really give you any more details on the questions, but I will say that they feel very much similar to the discussions that we had with the FDA.

關於歐盟方面的問題，我無法提供更多細節，但我可以說，這些問題與我們之前和美國食品藥物管理局（FDA）的討論非常相似。

Mostly around efficacy, the design of the studies, the sequential nature of the studies, the fact that the same patients have been used in in through the multiple studies, etc.

主要圍繞在療效、研究設計、研究的順序性、多項研究中使用同一批患者等問題。

But, I would say that, as we get these responses and as we get responses to these responses from the EU, we'll have a better sense of where it's going.

但是，我認為，隨著我們收到這些回复，以及歐盟對這些回應的回應，我們將更了解事態的發展方向。

Our next question is from Gail Chen from Laidlaw. Your line is open.

我們的下一個問題來自萊德勞公司的蓋爾陳。您的線路已開通。

Thanks for taking the questions and congrats on a very good great quarters. And basically I have two here. The first one is sort of related to the efficacy.

感謝您回答問題，並祝賀您度過了一個非常出色的季度。我這裡基本上有兩個。第一個問題與療效有關。

That you guys have talked, you, in the press release, you indicated you have a 100 patients have more than one year treatment and the many of those has a multiple years of treatments.

你們在新聞稿中提到，你們有 100 名患者接受了一年以上的治療，其中許多患者接受了多年的治療。

I just wonder whether you'll be able to do some studies of them to see over such a longer period of treatments, are those patients have been improved and how much those improve, maybe this will be useful. Longer-term historical analysis and have a follow-up.

我只是想知道您是否能夠對他們進行一些研究，看看在如此長的治療期內，這些患者的病情是否有所改善，以及改善了多少，也許這會很有用。進行更長期的歷史分析並跟進。

Yeah, I mean, we have, as been running a long-term open label study. Then there was a randomized withdrawal, and the patients went back on drugs.

是的，我的意思是，我們一直在進行一項長期的開放標籤研究。然後進行了隨機撤藥，患者們又重新開始服藥。

So we've had the ability to follow these patients very carefully for a very long period of time and as it's very unusual in a rare disease to have such long-term data.

因此，我們能夠非常仔細地對這些患者進行長時間的追蹤觀察，而對於罕見疾病來說，擁有如此長期的數據是非常不尋常的。

And we have seen, improvements of many different kinds in these patients, and this is obviously anecdotal and doesn't apply to everyone, but only yesterday we were looking at, we were talking to a patient who is running a triathlon or preparing for a triathlon.

我們已經看到這些患者在許多方面都有所改善，這顯然只是個例，並不適用於所有人，但就在昨天，我們還在和一位正在參加鐵人三項比賽或正在準備參加鐵人三項比賽的患者交談。

This is a person who's in college. We are aware of a person who's a sous chef. We're aware of numerous people who have graduated from high school.

這是個大學生。我們知道有一位副廚師長。我們知道有很多高中畢業生。

So, the long-term effects will vary by patient, but we think that taking away the hyperphagia, which is the hallmark symptom of the disease, will over a period of time really alter their lives.

因此，長期效果因人而異，但我們認為，消除這種疾病的標誌性症狀——過度進食，會在一段時間內真正改變他們的生活。

Okay, great. That's very helpful. Maybe one more question here, which is about the patient size that you already have on your a over 1,000 starting from and you suggested maybe 10,000 patients that give and take.

好的，太好了。那很有幫助。這裡可能還有一個問題，那就是關於你們的患者規模，你們目前已經有超過 1,000 名患者，而你們建議最終可能達到 10,000 名患者左右。

And that, at this stage, do you feel that you need to have additional effort to finding more patients, new patients? Or how would you prescribe your effort in, on that regard, thanks.

那麼，在現階段，您是否覺得需要付出更多努力來尋找更多新患者？或者，您會如何安排在這方面的努力呢？謝謝。

Meredith.

梅雷迪絲。

Yeah, so I think as we said previously, in our earnings call that we have a claims database where we're confidently able to identify approximately 12,000 claims, and individuals who have PWS and that when we look at that information that brings our TAM down to around 10,000.

是的，正如我們之前在財報電話會議上所說，我們有一個理賠資料庫，我們可以自信地從中識別出大約 12,000 例理賠案例和患有 PWS 的個人，當我們查看這些資訊時，我們的 TAM 就下降到了大約 10,000。

So we know where the patients are and where they're being treated. I think one thing that we're very excited about that we're working on is more around machine learning to identify specifically when these patients might be coming into the physician's office to really optimize our effective targeting for the sales team.

這樣我們就能知道病人在哪裡，在哪裡接受治療。我認為我們正在努力實現的一個令人興奮的目標是利用機器學習來具體識別這些患者何時會到醫生辦公室就診，從而真正優化我們銷售團隊的有效目標定位。

But with regard to traditional rare disease patient finding, we have the claims so we know where they're being treated.

但就傳統的罕見疾病患者查找而言，我們有理賠記錄，所以我們知道他們在哪裡接受治療。

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

好的，太好了。非常感謝，再次恭喜！

Thank you.

謝謝。

Our next question is from Catherine De La Rosso from LifeSci. Your line is open.

我們的下一個問題來自 LifeSci 的 Catherine De La Rosso。您的線路已開通。

Hi, congrats on the quarter and thanks for taking the question. So I was just curious, if you have a sense of the proportion of patients who are able to reach, or be maintained on their on labeled dose versus, those who undergo dose reduction.

您好，恭喜您本季取得佳績，感謝您回答這個問題。所以我很好奇，您是否了解能夠達到或維持其標籤劑量的患者比例，以及需要減少劑量的患者比例。

And I guess, apologies if I missed this, but for those who discontinue treatment, do you have a sense of how long they're on therapy before they're coming off and kind of expectations for that going forward?

我想，如果我錯過了什麼，請見諒。對於那些停止治療的人，您是否了解他們在停止治療前需要接受多長時間的治療，以及他們對未來的預期？

Yeah, I can address the second part of it. So we see the discontinuations happening earlier in treatment. So they're either in titration or just after. So think of it as sort of the first three months.

是的，我可以回答第二個問題。因此，我們看到治療中斷的情況發生在治療的早期階段。所以它們要么正在進行滴定，要么剛剛完成滴定。所以你可以把它看作是最初的三個月。

By the way, of the active patients, we think about 2/3 are, beyond the three-month time frame at this time.

順便說一下，在活躍患者中，我們認為目前約有 2/3 的患者已經超過三個月的治療期限。

Do you want to address the first question, Meredith? Yeah.

梅瑞迪思，你想回答第一個問題嗎？是的。

I think it's a little more complicated than that with regard to reducing dose because if you look at our label, really the therapeutic window, if you will, is between, I think like three to five nigs per kg, so. Any dosing around there is is considered on label dosing.

我認為在減少劑量方面，情況要複雜一些，因為如果你看一下我們的標籤，真正的治療窗口，如果你願意這麼說的話，我認為是在每公斤體重三到五尼格之間。該範圍內的任何劑量均視為標籤所示劑量。

So I think if you are looking at on target dose for a given weight band then I think it's fair to say that there's a very small minority that undergo do reductions in general you would expect people to get to the target doses.

所以我認為，如果你要觀察特定體重範圍內的目標劑量，那麼可以說，只有極少數人需要減量，一般來說，人們應該都能達到目標劑量。

Great, thanks so much.

太好了，非常感謝。

[Operator Instructions]

[操作說明]

Our next question is from Ram Silvera from HC Plainwright. Your line is open.

下一個問題來自 HC Plainwright 公司的 Ram Silvera。您的線路已開通。

Hi, this is Jay on for Ram. Thanks for taking our questions and congrats on the profitability this quarter. That actually leads me to my first question.

大家好，我是 Jay，為您帶來 Ram 的報道。感謝您回答我們的問題，並祝賀貴公司本季實現盈利。這其實引出了我的第一個問題。

Do you have some sort of idea when you might be in a position to start providing annualized revenue guidance? And secondly, as I'm sure Rhythm is pursuing an expansion into PWS first at Melatide.

您預計何時能夠開始提供年度營收預期？其次，我相信 Rhythm 首先會在 Melatide 上尋求向 PWS 領域擴張。

Do you think of that purely as a competitor drug, or do you think there's a possibility for, synergism between between the two drugs there?

你認為它純粹是一種競爭藥物，還是認為這兩種藥物之間有可能存在協同作用？

I'll answer the second part. Jim can take the first part. In terms of rhythms drug, we still have to see efficacy, as the one study that's been conducted with the drug in PWS, which was a large randomized phase 2 study, was negative for weight loss and hyperphagia.

我來回答第二個問題。吉姆可以承擔第一部分。就節律藥物而言，我們仍需觀察其療效，因為目前針對 PWS 進行的一項大型隨機 2 期研究顯示，該藥物對減輕體重和過度進食沒有效果。

I believe they are conducting an open label study, so we'll have to see what the data shows. In terms of whether it's competitive or potentially synergistic, it can be potentially synergistic because the two mechanisms are different from each other.

我相信他們正在進行一項開放標籤研究，所以我們得看看數據會顯示什麼。就其是否具有競爭性或潛在協同效應而言，它具有潛在協同效應，因為這兩種機制彼此不同。

So, for on the competition piece we'll have to see the data and certainly at least theoretically potentially synergistic.

所以，關於競爭方面，我們得看看數據，當然，至少在理論上，它們之間可能存在協同效應。

Yeah, and with regards to guidance I'd say it's a bit early at this stage. We're obviously looking for a little bit of maturation in the various components of the business, but we'll continue to investigate that.

是的，至於指導意見，我認為現在談論還為時過早。我們顯然希望公司各個組成部分都能更成熟一些，但我們會繼續對此進行調查。

Okay, thank you so much.

好的，非常感謝。

There are no questions at this time. I would like to have the call back to Anish. Please go ahead.

目前沒有問題。我希望阿尼什能給我回電。請繼續。

Thank you all for listening in today. Have a good evening.

感謝各位今天的收聽。祝你晚上愉快。

This concludes today's conference call. Thank you for participating. You may now disconnect.

今天的電話會議到此結束。感謝您的參與。您現在可以斷開連線了。