Mesoblast Ltd (MESO) 2025 Q2 法說會逐字稿

Good morning, everybody. I'm Silviu Itescu. I'm the Chief Executive of Mesoblast. Together with me this morning is Marcelo Santoro, our Chief Commercial Officer; and Andrew Chaponnel, our Interim Chief Financial Officer. Today, we're presenting our financial results and operational update for the half year ended December 31, 2024.

大家早安。我是 Silviu Itescu。我是 Mesoblast 的執行長。今天早上與我一起的還有我們的首席商務官馬塞洛·桑托羅 (Marcelo Santoro) 和我們的臨時首席財務官安德魯·查波內爾 (Andrew Chaponnel)。今天，我們將公佈截至 2024 年 12 月 31 日的半年財務表現和營運更新。

If we could go to slide 4, please. Mesoblast is the global leader in allogeneic cellular medicines for inflammatory diseases. We have one product already FDA approved, Ryoncil. We have multiple locations globally. We're listed duly on the ASX and NASDAQ.

請翻到幻燈片 4。Mesoblast 是治療發炎疾病的同種異體細胞藥物領域的全球領導者。我們有一款產品已獲得 FDA 批准，即 Ryoncil。我們在全球擁有多個辦事處。我們已在澳洲證券交易所和納斯達克上市。

We have more than 1,000 patents and patent applications that support our products. Beyond our first approved product, Ryoncil, we have two other major products in Phase 3, and we have a whole pipeline sitting behind these. We have scalable manufacturing that have been FDA inspected, and we have a supply chain capability that allows us to meet the global needs commercially.

我們擁有超過 1,000 項專利和專利申請來支持我們的產品。除了我們第一個獲批的產品 Ryoncil 之外，我們還有另外兩個主要產品處於第三階段，我們擁有完整的產品線。我們擁有經過 FDA 檢查的可擴展製造能力，並且擁有能夠滿足全球商業需求的供應鏈能力。

Next slide, please. Our platform technology is based on a shared mechanism of action across all of our products. Our mesenchymal lineage precursor/stromal cells are highly purified to very high concentrations in final cryopreserved vials. These cells have on their surface a range of receptors for inflammatory cytokines, including interferon gamma, TNF, IL-17, IL-6, and IL-1 and others. And when the cells are placed in regions of severe inflammation where these cytokines play major disease roles, they're able to respond to inflammation with the release of multiple anti-inflammatory factors that act in concert to turn off the damaging inflammation that results in severe diseases and potentially life-threatening outcomes.

請看下一張投影片。我們的平台技術是基於所有產品的共享作用機制。我們的間質譜系前驅細胞/基質細胞在最終冷凍保存的小瓶中被高度純化至非常高的濃度。這些細胞表面有一系列發炎細胞激素受體，包括幹擾素γ、TNF、IL-17、IL-6、IL-1等。當這些細胞被放置在這些細胞因子發揮主要疾病作用的嚴重炎症區域時，它們能夠通過釋放多種抗炎因子來應對炎症，這些抗炎因子協同作用，以關閉導致嚴重疾病和潛在危及生命的後果的破壞性炎症。

Next slide, please. This slide provides a snapshot of our clinical product pipeline. Our platform technology based on remestemcel, our first-generation product, trade name is Ryoncil, has now been approved by the FDA for the treatment of children with severe steroid-refractory acute graft versus host disease. I'll be talking a lot more about this product, which today we've announced pricing for and that physicians can access. This product is also being developed for adults with steroid-refractory GVHD and will be developed for life cycle extension into inflammatory bowel disease in both children and adults.

請看下一張投影片。這張投影片提供了我們臨床產品線的快照。我們基於第一代產品remestemcel的平台技術，商品名是Ryoncil，目前已獲得FDA批准，用於治療患有嚴重類固醇難治性急性移植物抗宿主疾病的兒童。我將會更多地談論這個產品，今天我們已經公佈了它的定價，醫生可以使用它。該產品還針對患有類固醇抗性 GVHD 的成年人進行開發，並將開發用於延長兒童和成人發炎性腸道疾病的生命週期。

Our second-generation technology platform is termed rexlemestrocel. These cells are immunoselected using monoclonal antibodies to high purity and potency. And this technology is being developed for inflammatory cardiovascular disease and inflammatory back pain. More about that later.

我們的第二代技術平台稱為 rexlemestrocel。這些細胞透過單株抗體進行免疫選擇，以獲得高純度和效力。並且該技術正在被開發用於治療發炎性心血管疾病和發炎性背痛。稍後會詳細介紹。

Next slide, please. Now, I'd like to turn to Andrew Chaponnel, who's going to be discussing our financial results for the period ended December 31, 2024.

請看下一張投影片。現在，我想請安德魯·查波內爾 (Andrew Chaponnel) 討論我們截至 2024 年 12 月 31 日的財務表現。

Thanks, Silviu. Turning to slide 8 for the financial highlights for the year. Our cash balance at December 31, 2024, was USD38 million with pro forma cash of approximately USD200 million after the successful completion of a global private placement, which raised USD161 million. Net operating cash spend was USD20.7 million for the first half of FY 2025, which was a 22% reduction on the first half of FY 2024. As a result of FDA approval of Ryoncil in December, a $23 million provision against the value of inventory manufactured and expensed in prior periods was reversed and is now recognized as an inventory asset on the balance sheet.

謝謝，西爾維烏。請翻到幻燈片 8 查看今年的財務亮點。截至 2024 年 12 月 31 日，我們的現金餘額為 3,800 萬美元，在成功完成全球私募（籌集 1.61 億美元）後，預計現金約為 2 億美元。2025財年上半年淨營業現金支出為2,070萬美元，較2024財年上半年減少22%。由於 FDA 於 12 月批准了 Ryoncil，針對前期生產和費用化的庫存價值的 2300 萬美元撥備被撤銷，現在被確認為資產負債表上的庫存資產。

Turning to the next slide, you will see our P&L statement. The BLA approval in December resulted in noncash balance sheet adjustments, including the write-off of the value of inventory. Starting with the line items most affected by the noncash balance sheet adjustments, let's look at the results of both the revaluation of contingent consideration and the revaluation of the warrant liability. The increase in these line items in the current half year were as a result of FDA approval. Within contingent consideration on FDA approval, the probability of success of pediatric GVHD increased to 100% and resulted in a noncash remeasurement increasing by $4 million to USD4.3 million for half one FY 2025 compared to $0.3 million for half one FY 2024.

翻到下一張投影片，您將會看到我們的損益表。12 月的 BLA 批准導致了非現金資產負債表調整，包括庫存價值的註銷。從非現金資產負債表調整影響最大的項目開始，讓我們看看或有對價重估和認股權證負債重估的結果。本半年這些項目的增加是由於獲得了 FDA 的批准。在 FDA 批准的附帶考慮範圍內，兒科 GVHD 的成功機率增加到 100%，並導致 2025 財年上半年的非現金重估增加 400 萬美元至 430 萬美元，而 2024 財年上半年為 30 萬美元。

Within revaluation of warrant liability as a result of FDA approval and the consequential share price appreciation, our warrant remeasurement increased by USD16 million to USD12 million for half one FY 2025 compared to a gain of $4.4 million for half one FY 2024. Within manufacturing, as a result of the FDA approval, the USD23 million provision against the value of inventory manufactured and expensed in prior periods was reversed. And as a result, we are now recognizing USD24 million of inventory on our balance sheet.

由於 FDA 批准和隨之而來的股價上漲而導致的認股權證負債重估，我們的認股權證重估在 2025 財年上半年增加了 1600 萬美元，達到 1200 萬美元，而 2024 財年上半年的收益為 440 萬美元。在製造業方面，由於獲得了 FDA 的批准，針對前期生產併支出的庫存價值計提的 2300 萬美元準備金被沖銷。因此，我們現在在資產負債表上確認了 2400 萬美元的庫存。

The increase in expenditure for both our R&D and management and admin was due to noncash share-based payments primarily for STI in lieu of cash-based payments. As described above, the BLA approval resulted in a number of noncash balance sheet adjustments, which drove the loss after tax of USD47.9 million for the half year. Pleasingly, for the same half year period, our total operating cash flows were only USD20.7 million, a reduction of $5.9 million from the comparative half year.

我們的研發和管理支出的增加主要是由於 STI 以非現金股份支付代替了現金支付。如上所述，BLA 批准導致了一些非現金資產負債表調整，從而導致半年稅後虧損 4,790 萬美元。令人高興的是，在同一半年期間，我們的總經營現金流僅為 2,070 萬美元，比去年同期減少了 590 萬美元。

Back to you for the call, Silviu.

回到你的電話，Silviu。

Thanks, Andrew. We can go to slide 10. I'd like to talk now about Ryoncil, our launch strategy, pricing and other guidance. Ryoncil is the first mesenchymal stromal cell therapy approved by the FDA. Next slide, please. The first FDA-approved, off-the-shelf therapy for children aged two months and older, including adolescents and teenagers with steroid-refractory acute GVHD, a life-threatening condition with high mortality rates.

謝謝，安德魯。我們可以轉到第 10 張投影片。現在我想談談 Ryoncil、我們的發布策略、定價和其他指導。Ryoncil 是第一個獲得 FDA 批准的間質基質細胞療法。請看下一張投影片。這是 FDA 批准的首個現成療法，適用於治療兩個月及以上兒童，包括患有類固醇難治性急性 GVHD 的青少年，這是一種危及生命的疾病，死亡率很高。

Next slide. We have the opportunity to address a very critical unmet need in children two months and older. Across the US, approximately 10,000 allogeneic bone marrow transplants are performed annually. Acute graft-versus-host disease occurs in about 50% of patients. Approximately half of these fail to respond to steroids.

下一張投影片。我們有機會解決兩個月及以上兒童的一個非常關鍵的未滿足的需求。在美國，每年約進行 10,000 例異基因骨髓移植手術。約50％的患者會出現急性移植物抗宿主疾病。其中約有一半對類固醇沒有反應。

And for those who failed to respond to steroids, mortality is very high, and there are significant extended hospital stay costs. We believe that the addressable market in the US is approximately 375 new children per year with life-threatening steroid-refractory acute graft-versus-host disease.

對於那些對類固醇沒有反應的患者，死亡率非常高，住院費用會大幅增加。我們認為，美國的潛在市場是每年約有 375 名患有危及生命的類固醇難治性急性移植物抗宿主疾病的新生兒童。

Next slide, please. In our Phase 3 trial that underpinned FDA approval, Ryoncil delivered high overall response rates at day 28, which is a measure well established to predict long-term survival in this disease. Overall response rates were 70% at day 28, significantly higher than is achievable with other therapy for this disease.

請看下一張投影片。在我們獲得 FDA 批准的 3 期試驗中，Ryoncil 在第 28 天實現了較高的總體反應率，這是預測該疾病長期存活率的完善指標。第 28 天的整體反應率為 70%，明顯高於該疾病的其他療法。

Importantly, 89% of the children enrolled in this trial had the most severe form of the disease, Grade C/D, which involves the gastrointestinal tract and liver in addition to skin. Ryoncil treatment was not discontinued or interrupted in any patient for any laboratory abnormality, and the full course was completed without interruption in more than 85% of patients. This is very different from the profile with other therapies used in these children with very severe disease as well as in adults with acute graft-versus-host disease.

重要的是，參加本次試驗的 89% 兒童患有最嚴重的疾病形式，即 C/D 級，除了皮膚外，還涉及胃腸道和肝臟。沒有任何患者因為實驗室異常而停止或中斷 Ryoncil 治療，超過 85% 的患者順利完成整個療程。這與患有非常嚴重疾病的兒童以及患有急性移植物抗宿主疾病的成人所使用的其他療法的情況非常不同。

Next slide, please. Now, the cost of treating children with steroid-refractory GVHD who subsequently die is very high. The cost of treating a child who dies within 12 months of a transplant from steroid-refractory GVHD is approximately $2.5 million. And notably, it's $1.8 million higher than for those children with steroid-refractory GVHD who actually remain alive.

請看下一張投影片。目前，治療患有類固醇抗性移植物抗宿主疾病並隨後死亡的兒童的費用非常高。治療一名因類固醇難治性 GVHD 在移植後 12 個月內死亡的兒童的費用約為 250 萬美元。值得注意的是，這比患有類固醇抗性移植物抗宿主疾病但仍存活的兒童的治療費用高出 180 萬美元。

Next slide, please. Well, Ryoncil has demonstrated long-term survival free from acute GVHD. In the long-term follow-up of Ryoncil by the Center for International Blood and Marrow Transplant Research, CIBMTR, in the 51 patients who were followed long term, notably 88% of whom had life-threatening Grade C/D disease. two-year survival was 51%. And beyond that, survival was relatively plateaued with four-year survival of 49%.

請看下一張投影片。嗯，Ryoncil 已證明能夠長期生存，不患急性 GVHD。國際血液和骨髓移植研究中心 (CIBMTR) 對 Ryoncil 進行了長期隨訪，在 51 名長期隨訪的患者中，值得注意的是，其中 88% 患有危及生命的 C/D 級疾病。兩年存活率為 51%。除此之外，存活率相對穩定，四年存活率為 49%。

Notably, only 14%, seven children have died due to acute graft versus host disease through four years and beyond. One would have expected a much higher number to have died from acute GVHD if treated by other therapies.

值得注意的是，只有 14% 的兒童在四年或更長時間內死於急性移植物抗宿主疾病。如果採用其他療法治療，人們預計死於急性 GVHD 的人數會更多。

Next slide, please. What is the value of Ryoncil in treating pediatric patients with acute GVHD? Well, the total benefits of patient outcomes using Ryoncil range between $3.2 million to $4.1 million. And this is based on health economic models for lifetime, ultra-rare disease and high-impact, short-term therapies, including Quality of Life Years gained. And the benefits comprise long-term survival, cost offsets and cost savings.

請看下一張投影片。Ryoncil 對治療兒童急性 GVHD 患者有何價值？那麼，使用 Ryoncil 的患者的總收益在 320 萬美元到 410 萬美元之間。這是基於針對終生、極為罕見的疾病和高影響、短期治療的健康經濟模型，包括所獲得的生活品質年限。其好處包括長期生存、成本抵銷和成本節約。

Next slide, please. So for treating pediatric patients with acute GVHD, the recommended dosage of Ryoncil based on our FDA-approved label is 2 million cells per kilogram body weight given intravenously twice per week for four consecutive weeks. The wholesale acquisition cost of Ryoncil is $194,000 per intravenous infusion across all body weights.

請看下一張投影片。因此，對於治療患有急性 GVHD 的兒科患者，根據我們 FDA 批准的標籤，Ryoncil 的建議劑量為每公斤體重 200 萬個細胞，連續四周每週靜脈注射兩次。Ryoncil 的批發採購成本為每次靜脈輸注 194,000 美元（適用於所有體重）。

Next slide, please. This is the mandatory hub that has been established, termed MyMesoblast. This is set up to assist patients with insurance coverage, financial assistance and access programs, ensuring that no patient is left behind in receiving this potentially life-saving therapy, a comprehensive patient services hub, which provides access and helps both patients, their families and institutions.

請看下一張投影片。這是已建立的強制性中心，稱為 MyMesoblast。該中心的設立旨在為患者提供保險、經濟援助和醫療服務，確保所有患者都能接受這種可能挽救生命的治療，此外，該中心還設有一個綜合性患者服務中心，為患者、患者家屬和醫療機構提供醫療服務。

Next slide, please. Ryoncil is being made available for pediatric GVHD in the United States in March. We are approaching this in a staged manner, targeting post-transplant centers with highest volume and with established experience using the Ryoncil product. We're establishing and have established already a targeted sales force with experience in bone marrow transplant centers. 15 of the highest volume centers account for 50% of the patients and the 45 highest volume centers account for 80% of patients. And you'll hear more about this from Marcelo Santoro in the Q&A session.

請看下一張投影片。Ryoncil 將於 3 月在美國上市，用於治療兒童 GVHD。我們正在分階段實施這項計劃，目標是數量最多且擁有使用 Ryoncil 產品豐富經驗的移植後中心。我們正在建立並已經建立了一支具有骨髓移植中心經驗的目標銷售團隊。 15 個最高容量中心佔病患的 50%，而 45 個最高容量中心佔病患的 80%。您還可以在問答環節中從馬塞洛·桑托羅 (Marcelo Santoro) 那裡聽到更多有關此內容的介紹。

Beyond acute GVHD, we have a strategy to expand the label and establish a life cycle for the product. slide 21, please. In particular, we're focusing on inflammatory bowel disease, inflammatory Crohn's and ulcerative colitis. The pathology and the clinical aspects of these diseases are very similar to the inflammatory bowel complications of acute graft-versus-host disease. And the ability to respond to Ryoncil is similar and has been evaluated in previous studies.

除了急性 GVHD 之外，我們還有擴大標籤和建立產品生命週期的策略。請看第 21 張投影片。我們特別關注發炎性腸道疾病、發炎性克隆氏症和潰瘍性結腸炎。這些疾病的病理學和臨床表現與急性移植物抗宿主疾病的發炎性腸道併發症非常相似。對 Ryoncil 的反應能力也類似，這在先前的研究中已經被評估。

The unmet need is large in both adults and children. And in particular, more than 60% of patients fail to respond or subsequently lose response to anti-TNF agents, which are the first line of biologics in this patient population. About 25% of all inflammatory bowel disease patients are of pediatric age. And in these patients, an anti-TNF agent is the only approved therapy. This represents potentially as many as 7,000 children, 50% to 60% of whom are likely to be refractory to anti-TNF therapies and where potentially Ryoncil may have -- may make a difference.

成人和兒童中未滿足的需求都很大。尤其是超過 60% 的患者對抗 TNF 藥物沒有反應或隨後失去反應，而抗 TNF 藥物是該患者群體的一線生物製劑。所有發炎性腸道疾病患者中約有 25% 為兒科年齡。對於這些患者，抗 TNF 藥物是唯一核准的治療方法。這意味著可能有多達 7,000 名兒童受到影響，其中 50% 至 60% 可能對抗 TNF 療法具有抵抗力，而 Ryoncil 可能會發揮作用。

Next slide, please. A recent pilot study in adults demonstrated positive outcomes with Ryoncil directly injected submucosally in biologic refractory patients. Ryoncil Was delivered by direct endoscopic injection to areas of inflammation. In addition, we have data showing that remestemcel induces early remission in Crohn's disease adults who failed a single anti-TNF agent following the course of intravenous treatment. Given the effectiveness of Ryoncil in treating children with gastrointestinal-related GVHD with inflammation of the gut and the existing data in adult Crohn's disease patients, we plan to further evaluate the immunomodulatory effects of Ryoncil on GI inflammation in medically refractory pediatric Crohn's disease and ulcerative colitis patients.

請看下一張投影片。最近針對成人進行的一項初步研究表明，對於生物製劑難治性患者，直接在黏膜下注射 Ryoncil 可產生積極的效果。Ryoncil 透過內視鏡直接注射到發炎區域。此外，我們有數據顯示，對於單一抗 TNF 藥物治療失敗的克隆氏症成人患者，remestemcel 可在靜脈治療後誘導早期緩解。鑑於 Ryoncil 在治療患有腸道發炎的胃腸道相關 GVHD 兒童方面的有效性以及成人克隆氏症患者的現有數據，我們計劃進一步評估 Ryoncil 對藥物難治性兒童克隆氏症和潰瘍性結腸炎患者的胃腸道發炎的免疫調節作用。

Next slide, please. In addition to inflammatory bowel disease, we have a strategy to expand Ryoncil use in adult patients with GVHD. This continues to be an unmet need, particularly in patients who fail ruxolitinib, the only approved drug in adults with GVHD. This accounts for about 40% to 45% of all ruxolitinib-treated patients. In addition, survival of these patients who fail ruxolitinib is very dismal, around 20% to 30% by 100 days.

請看下一張投影片。除了發炎性腸道疾病外，我們還有一個策略，即擴大 Ryoncil 在患有 GVHD 的成年患者中的使用。這仍然是一個未滿足的需求，特別是對於使用蘆可替尼治療失敗的患者而言，蘆可替尼是唯一獲準用於治療成人 GVHD 的藥物。這佔所有接受蘆可替尼治療的患者的約 40% 至 45%。此外，蘆可替尼治療失敗的患者的存活率非常低，100天存活率約為20%至30%。

And for this patient population, there are no approved therapies. Treatment in a pilot study of third-line agents using Ryoncil demonstrated 73% survival at day 100. And that provides us with the confidence to move forward into an appropriate pivotal study in adults of Ryoncil who are refractory to ruxolitinib. We're collaborating with the Blood and Bone Marrow Transplant Clinical Trials Network, an NIH-funded body responsible for approximately 80% of all US transplants, to conduct this pivotal trial. And you'll hear more about that in due course.

對於這群患者，目前尚無核准的治療方法。在使用 Ryoncil 進行三線藥物治療的初步研究中，結果顯示第 100 天的存活率為 73%。這讓我們有信心繼續對對蘆可替尼有抵抗力的成年人進行 Ryoncil 的適當關鍵研究。我們正在與血液和骨髓移植臨床試驗網絡合作進行這項關鍵試驗，這是美國國立衛生研究院資助的機構，負責美國約 80% 的移植手術。您稍後將會聽到更多有關此內容的消息。

Next slide, please. Let's move forward to the second platform technology, rexlemestrocel, immunoselected STRO3-positive cells that are expanded and used for local delivery into areas of inflammatory tissues. slide 25. We move to the use of rexlemestrocel for chronic inflammatory low back pain due to degenerative disc disease. This is a disease that affects more than 7 million patients across the US, a similar number of patients across the EU5.

請看下一張投影片。讓我們繼續討論第二個平台技術，rexlemestrocel，免疫選擇的STRO3陽性細胞被擴增並用於局部遞送到發炎組織區域。幻燈片25。我們開始使用 rexlemestrocel 治療因退化性椎間盤疾病引起的慢性發炎性下背痛。這種疾病影響了美國超過 700 萬名患者，歐盟 5 國的患者數量也差不多。

After failure of nonsteroidal agents and other conservative therapies, there are minimal treatment options. And in fact, 50% of opioid prescriptions are for this particular indication. And we all know the problems with opioid addiction and the epidemic of opioid over usage and over prescription across the US. We have established that there's durable improvement in pain from a single injection of our cells in prior studies and currently are in a confirmatory Phase 3 trial.

非類固醇藥物和其他保守療法失敗後，治療選擇就很少了。事實上，50% 的鴉片類藥物處方都是針對這特定症狀的。我們都知道阿片類藥物成癮問題以及美國境內阿片類藥物過度使用和過度處方的流行病。我們在先前的研究中已經證實，單次注射我們的細胞就能持久改善疼痛，目前正處於確認性的 3 期試驗階段。

If we go to the next slide, slide 26. This is the patient journey. And really, the point of this slide is to demonstrate how rapidly patients go through conservative approaches, and go through opioids and then really what's left are interventional therapies with all of the related adverse complications. We aim to be establishing a best-in-care, best-in-class approach to the treatment of inflammatory back pain as soon as conservative treatments have failed.

如果我們轉到下一張投影片，即第 26 張投影片。這是患者的旅程。實際上，這張投影片的目的是展示患者如何快速地接受保守治療和鴉片類藥物治療，然後剩下的就是介入治療以及所有相關的不良併發症。我們的目標是，一旦保守治療失敗，就建立最佳照護、一流的方法來治療發炎性背痛。

slide 27 summarizes the outcomes in the first Phase 3 trial, where as you can see here, the comparison was the change in pain from baseline, in red, of our product, rexlemestrocel in combination with a carrier and comparison at month 12, which was the primary end point of the study in terms of pain reduction against the placebo control in green. That difference is highly significant. And just to put this into context, the minimal pain reduction that is seen in the green at 12 months is roughly what you would expect to see with opioid usage.

投影片 27 總結了第一階段 3 期試驗的結果，如您所見，比較的是我們的 rexlemestrocel 與載體組合使用後，相對於基線的疼痛變化（紅色），以及第 12 個月的比較，這是研究的主要終點，與安慰劑對照相比，疼痛減輕的程度（綠色）。這種差異非常顯著。為了更清楚地說明這一點，12 個月後綠色部分所見的最小疼痛減輕大致與您預期使用鴉片類藥物時所見的疼痛減輕大致相同。

That difference between the two is a very large difference in terms of mean pain reduction. But I think it's important to note that this is mean pain reduction for the group as a whole and that 50% of patients treated with rexlemestrocel showed complete remission or no pain at all at 12 months. And these patients who were improved with pain at 12 months showed very durable, long-term maintenance of pain-free outcomes through 36 months.

就平均疼痛減輕程度而言，兩者之間的差異非常大。但我認為值得注意的是，這是整個群體的平均疼痛減輕程度，並且 50% 接受 rexlemestrocel 治療的患者在 12 個月內完全緩解或完全沒有疼痛。這些在 12 個月時疼痛得到改善的患者在 36 個月內表現出非常持久的無痛效果。

Moreover, 40% of patients were on opioids at commencement of the study. And despite the fact that they were told and their physicians were told not to change medications, almost 30% of patients in the treated arms were able to completely come off opioids versus mid-single digits in the control arm, and this was a significant outcome. So we're currently enrolling this trial. We're increasing the enrollment rates through various interventions, including increasing sites from 15 to 40 sites, and we'll be updating the market shortly in due course.

此外，40% 的患者在研究開始時正在服用鴉片類藥物。儘管他們和他們的醫生都被告知不要更換藥物，但治療組中幾乎有 30% 的患者能夠完全停用阿片類藥物，而對照組中只有個位數的患者能夠完全停用阿片類藥物，這是一個顯著的結果。所以我們目前正在進行這項試驗。我們正在透過各種幹預措施來提高入學率，包括將站點從 15 個增加到 40 個，並且我們將在適當的時候更新市場資訊。

Next slide, please. slide 29. Let's move to rexlemestrocel for ischemic heart failure. Heart failure with low ejection fraction due to underlying ischemia continues to be a major problem in the Western world, in the US more broadly, increasing in prevalence and associated with the high risk of death, heart attacks and strokes.

請看下一張投影片。第 29 張投影片。讓我們轉向使用 rexlemestrocel 來治療缺血性心臟衰竭。由於潛在缺血導致的低射血分數心臟衰竭仍然是西方世界（更廣泛地說是美國）的一個主要問題，其盛行率正在上升，並且與死亡、心臟病發作和中風的高風險有關。

Heart failure with low ejection fraction occurs in about 50% of all patients with heart failure. 60% of these patients have heart failure due to ischemia. They are at highest risk of cardiac events, including death. The target market is very large in the US, likely to be around 1 million patients with ischemic heart failure and inflammation.

約50%的心臟衰竭患者會出現低射血分數的心臟衰竭，其中60%的患者因缺血而出現心臟衰竭。他們發生心臟病甚至死亡的風險最高。目標市場在美國非常大，可能有大約 100 萬名患有缺血性心臟衰竭和發炎的患者。

Slide 30, please. This is a complex slide. We have shown this previously where the patient journey for heart failure is on the left-hand side, New York Heart Association class I and class II, the bulk of heart failure patients. These patients are on a whole range of different oral medications. But inexorably, over five years to 10 years of the disease, they move into the class III, class IV and end-stage spectrum.

請翻到第 30 張投影片。這是一張複雜的幻燈片。我們之前已經展示過這一點，其中心力衰竭患者的病程在左側，紐約心臟協會 I 級和 II 級，大多數心臟衰竭患者。這些患者正在服用各種不同的口服藥物。但不可避免的是，在生病五年到十年後，它們會進入 III 級、IV 級和終末期。

Despite being on maximal oral therapy, they move into this area of high risk for progression to death. We have performed two large, randomized controlled studies in this patient population: the DREAM trial, 537 patients in class III; and the LVAD MPC trial of 159 patients, both randomized controlled studies that is in end-stage patients being kept alive by an artificial device.

儘管接受了最大程度的口服治療，他們仍然進入了死亡的高風險狀態。我們在該患者群體中進行了兩項大型隨機對照研究：DREAM 試驗，涉及 537 名 III 類患者；LVAD MPC 試驗，涉及 159 名患者，這兩項隨機對照研究均針對透過人工裝置維持生命的終末期患者。

Next slide, please, slide 31. This slide shows the effect on cardiovascular death of a single MPC injection in our Phase 3 trial: on the left-hand side in patients who are categorized on the basis prespecified of a simple blood test for inflammation called CRP; and on the right-hand side, a slightly more fancy measurement of inflammation, measuring a cytokine called interleukin-6.

請看下一張投影片，第 31 張。這張投影片展示了我們 3 期試驗中單次 MPC 注射對心血管死亡的影響：左側是根據預先指定的簡單血液發炎檢測 CRP 進行分類的患者；右側是稍微複雜一點的發炎測量方法，即測量一種名為白細胞介素 6 的細胞因子。

What you see in both the left and the right panels is that control patients have a very high risk of cardiovascular death over a five-year follow-up period. And in both analyses, a single intracardiac injection of 150 million MPCs or rexlemestrocels significantly reduced the risk of cardiovascular death by approximately 80% in the CRP categorization on the left and by about 60% in patients determined by high or low IL-6 levels.

從左側和右側面板可以看到，在五年的追蹤期內，對照組患者發生心血管死亡的風險非常高。在這兩項分析中，單次心內注射 1.5 億個 MPC 或 rexlemestrocels 可顯著降低左側 CRP 分類患者的心血管死亡風險約 80%，降低 IL-6 水平高或低確定的患者心血管死亡風險約 60%。

What this demonstrates is that despite the fact that patients are apparently well treated with a range of approved drugs for heart failure, which improves their symptoms and reduces their hospitalizations due to symptomatic shortness of breath, they remain -- particularly if they've got measurable inflammation, they remain at high risk for death, which -- over the subsequent three, four, five years of follow-up. And that a single injection of rexlemestrocel substantially reduces that long-term risk of death.

這表明，儘管患者似乎透過一系列已獲批准的心臟衰竭藥物得到了良好的治療，這些藥物改善了他們的症狀並減少了由於呼吸困難症狀而住院的次數，但他們仍然——特別是如果他們有可測量的炎症，他們仍然面臨很高的死亡風險——在隨後的三、四、五年的隨訪中。並且一次注射 rexlemestrocel 可以大大降低長期死亡風險。

Next slide, 32. Also from the recently published paper in the European Journal of Heart Failure, we analyzed composite end points, which were prespecified from the DREAM trial of either 2-point MACE on the left or 3-point MACE on the right, MACE being defined as time to either cardiovascular death or heart attack or stroke. 2-point MACE is just time to heart attack or stroke. And what you see in both analyses is that overall, patients showed a significant reduction in heart attacks or stroke. That was notable, particularly in the setting of ischemia, particularly in the setting of inflammation.

下一張投影片，32。此外，根據最近發表在《歐洲心臟衰竭雜誌》上的論文，我們分析了複合終點，這些終點是根據 DREAM 試驗預先指定的，左側為 2 點 MACE，右側為 3 點 MACE，MACE 定義為心血管死亡或心臟病發作或中風的時間。 2 點 MACE 只是心臟病發作或中風的時間。從這兩項分析中可以看出，總體而言，患者的心臟病發作或中風的發生率顯著降低。這是值得注意的，特別是在缺血的情況下，特別是在發炎的情況下。

And most notably, the greatest risk reduction, 90% risk reduction of heart attacks or stroke and almost 50% risk reduction in heart attack, stroke or death in those patients who had both ischemia and inflammation. And that represents clearly the highest risk population and the population most likely to respond to a single treatment of our cells.

最值得注意的是，對於同時患有缺血和發炎的患者來說，風險降低幅度最大，心臟病發作或中風的風險降低了 90%，心臟病發作、中風或死亡的風險降低了近 50%。這顯然代表了最高風險族群和最有可能對我們的細胞進行單一治療產生反應的族群。

We can go to slide 33, please. So based on meetings with the FDA, we have a clear pathway towards accelerated approval for Revascor in adults with heart failure with low ejection fraction. I've highlighted the outcomes from the DREAM trial over a minimum -- median follow-up of 30 months and beyond. We also have results from the second LVAD study, MPC number two, which demonstrated that at 12 months of follow-up, there was a significant increase in the proportion of LVAD patients with ischemic heart failure who were successfully weaned and then had a reduction in both hospitalizations and mortality.

請翻到第 33 張投影片。因此，根據與 FDA 的會議，我們有明確的途徑加速批准 Revascor 用於治療射血分數較低的心臟衰竭成人患者。我重點介紹了 DREAM 試驗至少 30 個月及以上的中位數追蹤結果。我們還有第二項 LVAD 研究（MPC 2 號）的結果，研究顯示，在 12 個月的追蹤中，成功脫機的 LVAD 缺血性心臟衰竭患者比例顯著增加，住院率和死亡率均降低。

Based on the data from both of these trials, when we met with the FDA, we were informed that the totality of the trial results from these studies may support an accelerated approval pathway in ischemic heart failure patients with low ejection fraction. On that basis, we intend to meet with the FDA further, get clarification on our various components of it. What needs to go into a BLA filing to discuss clinical data, particularly the data that would be required for a confirmatory study in order to file for accelerated approval in ischemic patients with heart failure.

根據這兩項試驗的數據，當我們與 FDA 會面時，我們被告知這些研究的整體試驗結果可能支持對射血分數低的缺血性心臟衰竭患者進行加速審批。在此基礎上，我們打算進一步與 FDA 會面，澄清其各個組成部分。在 BLA 申請中需要討論哪些臨床數據，特別是為了申請加速批准缺血性心臟衰竭患者而進行的確認性研究所需的數據。

Slide 34. And so I won't talk in a lot more detail on additional areas of focus in cardiovascular disease. But as I've previously highlighted, we have also obtained data in children with congenital heart disease and the hypoplastic left heart syndrome, where we have both an RMAT orphan drug designation and a rare pediatric disease designation based on the data that's been accrued to date. And we will be having meetings with the FDA to discuss whether the controlled study can be used to support regulatory approval for this life-threatening condition using Revascor.

幻燈片 34。因此，我不會詳細談論心血管疾病的其他重點領域。但正如我之前強調的那樣，我們還獲得了患有先天性心臟病和左心發育不全綜合徵的兒童的數據，根據迄今為止積累的數據，我們獲得了 RMAT 孤兒藥稱號和罕見兒科疾病稱號。我們將與 FDA 舉行會議，討論是否可以使用對照研究來支持使用 Revascor 治療這種危及生命的疾病的監管批准。

And my final slide, if we can go now to slide 36, is where we see our key objectives for each of our programs over the coming 6 to 12 months. And really, we have three major programs and products and objectives. For Ryoncil, which is developed for pediatric and adult inflammatory diseases, Ryoncil will be available for use in steroid-refractory acute GVHD at US hospitals this quarter. Studies will commence in both pediatric and adult label extension indications, as I've talked about, including adult GVHD and pediatric and adult inflammatory bowel disease. Revascor for heart failure in adults with low ejection fraction and heart failure and in children with congenital heart disease are being prepared for meetings with the agency for accelerated approval filing.

我的最後一張投影片（如果我們現在可以翻到第 36 張投影片的話）是我們在未來 6 到 12 個月內每個專案的關鍵目標。實際上，我們有三個主要計劃、產品和目標。對於為兒童和成人發炎疾病開發的 Ryoncil，Ryoncil 將於本季在美國醫院用於治療類固醇難治性急性 GVHD。正如我所談到的，研究將針對兒科和成人標籤擴展適應症展開，包括成人 GVHD 以及兒科和成人發炎性腸道疾病。Revascor 用於治療低射血分數心臟衰竭成人患者和先天性心臟病兒童患者的心臟衰竭，目前正在準備與該機構會面，以加快審批申請。

And finally, rexlemestrocel for chronic low back pain. We have a Phase 3 trial that we have and we'll continue to actively progress and invest in order to accelerate enrollment across multiple sites across the US. This has a 12-month primary end point of pain reduction subsequent to which we could be in a position to file for approval.

最後，rexlemestrocel 可用於治療慢性下背痛。我們正在進行第三階段試驗，我們將繼續積極推進和投資，以加快在美國多個地點的招募。其主要終點是減輕疼痛，持續 12 個月，之後我們就可以申請批准了。

And on that note, I think I'll stop and thank you very much. I'd like to open it up to questions, please.

就此而言，我想我會停下來並非常感謝你。我想開放提問環節。

(Operator Instructions) Edward Tenthoff, Piper Sandler.

（操作員指示）Edward Tenthoff，Piper Sandler。

A lot of really exciting success going on here. I wanted to get a sense just with respect to the Ryoncil launch, and I appreciate the color on the pricing. How large is the sales force? And because you've sort of had the expanded access program in place, how many centers are already trained on using Ryoncil? So just trying to get a sense of how quickly this could really be launched in the US. And then I have a quick follow-up question.

這裡發生了許多真正令人興奮的成功。我只是想了解一下 Ryoncil 的發布情況，我很欣賞其定價。銷售團隊有多大？由於你們已經實施了擴展訪問計劃，有多少中心已經接受過使用 Ryoncil 的培訓？所以只是想了解這項技術在美國能以多快的速度真正推出。然後我有一個快速的後續問題。

I'll just say that Ryoncil is already the standard of care in children with steroid-refractory acute GVHD. We have been providing it, as you mentioned, under expanded access. Quite a number -- most physicians and most transplant centers are very familiar with the product and are waiting -- literally waiting for this product to be commercially available so they can use it freely.

我只想說，Ryoncil 已經是類固醇難治性急性 GVHD 兒童的標準治療方法。正如您所說，我們一直在擴大存取權限範圍內提供它。相當多的人——大多數醫生和大多數移植中心都非常熟悉該產品並且正在等待——確切地說是在等待該產品商業化，以便他們可以自由使用。

I'll ask Marcelo Santoro to talk a little bit about his commercial team, which is already in place. As I mentioned earlier, 80% of the transplants are performed across 45 sites. And Marcelo, maybe you can talk about your strategy to get it across all those sites, please?

我將請馬塞洛·桑托羅 (Marcelo Santoro) 談談他的商業團隊，該團隊已經到位。正如我之前提到的，80% 的移植手術是在 45 個地點進行的。馬塞洛，您能談談將其推廣到所有網站的策略嗎？

No, that's great. So thank you very much, Silviu. Thank you for the question. It's a good one. So I think as Silviu mentioned, right, so we've built and are continuing to build a world-class sales force with transplant experience.

不，那太好了。非常感謝你，Silviu。謝謝你的提問。這是個好主意。所以我認為正如 Silviu 所提到的那樣，我們已經建立並正在繼續建立一支具有移植經驗的世界級銷售團隊。

It's a small, appropriately sized sales force of nine total key account managers along with the head of sales force that will be focusing on this 45 key transplant centers. Obviously, we'll treat each transplant center as a key target, a key customer for us. That represents 80% of the potential. And onboarding has already started. It actually started before we even hired the sales force.

這是一支規模適中的小型銷售隊伍，共有 9 名大客戶經理和一名銷售主管，主要負責這 45 個主要移植中心。顯然，我們會將每個移植中心視為我們的重點目標、重點客戶。這代表了80%的潛力。入職培訓已經開始。事實上，它在我們僱用銷售人員之前就已經開始了。

We've been in discussions with a lot of the centers at the moment so that by the time the product is available at the end of the month, we're ready to roll.

目前我們已經與許多中心進行了討論，以便在月底產品上市時，我們已經準備好了。

Yes. I would say the other point that's important is we've already had inquiries, inbound inquiries from at least 5 to 10 -- or at least 5 to 10 children, very sick, who are waiting for product as we speak.

是的。我想說的另一點是，我們已經收到了至少 5 到 10 個詢問，或者至少 5 到 10 個病得很重的孩子的詢問，他們正在等待產品。

Wow, that's great. Well, it just shows you how important a product this really is and life-saving product this is. My second question had to do with respect to the chronic lower back pain ongoing trial, and again, this being another really important product. How far are you guys along in terms of enrollment? And when do you anticipate actually reporting data from that Phase 3 trial? Is your goal to launch that yourselves in the US or seek a partner?

哇，太棒了。嗯，這只是向您展示了這個產品有多重要，以及它是多麼能救命的產品。我的第二個問題與慢性下背部疼痛的持續試驗有關，這也是另一個非常重要的產品。你們的入學進程如何了？您預計何時會實際報告第三階段試驗的數據？你們的目標是自己在美國推出這個產品還是尋求合作夥伴？

Well, I would say that if we're successful, this is a huge market opportunity. I mentioned earlier, there's about 7 million people in the US, same type of number in EU5 to meet the criteria that patients are being enrolled at for this trial. And if successful, a single injectable will be a major immunomodulatory pain management therapeutic. You can imagine that the sales force required for targeting this patient population is substantial.

嗯，我想說，如果我們成功了，這是一個巨大的市場機會。我之前提到過，美國大約有 700 萬人符合參與此試驗的病患標準，與歐盟 5 國的人數相同。如果成功的話，一次注射就將成為主要的免疫調節疼痛管理治療方法。你可以想像，針對這群患者所需的銷售團隊是相當龐大的。

In Europe, we already have a commercial partner in Grünenthal, the largest, number one company in the pain space. They have the expertise across the major jurisdictions there, both in terms of regulatory and reimbursement. We would presumably enter into a similar partnership in the US rather than invest our own in the distribution.

在歐洲，我們已經與 Grünenthal 建立了商業合作夥伴，Grünenthal 是疼痛治療領域規模最大、排名第一的公司。他們在當地主要司法管轄區擁有監管和報銷方面的專業知識。我們可能會在美國建立類似的合作夥伴關係，而不是在分銷方面進行自己的投資。

You're going to see a ramp-up of enrollment in short order. We've invested substantially in sites. The number of centers that are coming on board, that are onboard now is approximately 15. We expect over the coming four weeks to get up to about 40. And what happens is that as the physicians have more and more experience in terms of screening, evaluating, turning through the backlog of patients, it becomes easier and you get that sort of hockey stick takeoff.

您很快就會看到入學人數的增加。我們已經在場地上投入了大量資金。即將加入的中心數量，目前已加入的中心數量約為 15 個。我們預計未來四周將達到約 40 個。實際情況是，隨著醫生在篩檢、評估、處理積壓病人方面的經驗越來越豐富，事情變得越來越容易，你就能獲得那種曲棍球棒式的起飛。

And so we have something like 20 patients currently in screening and that on a weekly basis, that turns into an additional 15 to 20 new patients. So these numbers are rapidly increasing. So we have a target enrollment by -- towards the end of this year. But if we can compress it and bring it faster in, then we'll certainly try to do so.

因此，我們目前有大約 20 名患者接受篩檢，每週都會新增 15 至 20 名患者。所以這些數字正在迅速增加。因此，我們的目標是在今年年底前完成招生。但如果我們可以壓縮它並使其更快實現，那麼我們肯定會嘗試這樣做。

Michael Okunewitch, Maxim Group.

Michael Okunewitch，Maxim 集團。

Congratulations on all the exciting progress.

恭喜所有令人興奮的進展。

Thank you.

謝謝。

I guess just to kick things off, quickly looking at the math, with a WACC price of $194,000 per injection, 8 injections, that's about $1.5 million per quarter of treatment. Am I getting that right? And then just what sort of feedback have you gotten from payers on this pricing level?

我想只是為了開始，快速看一下數學，每次注射的 WACC 價格為 194,000 美元，注射 8 次，那麼每季的治療費用約為 150 萬美元。我說得對嗎？那麼，您從付款人那裡得到了關於這個定價水平的什麼樣的反饋？

Well, again, let me start by saying that based on health economic models, which reflect the net positive benefits of treatment with Ryoncil of between $3.2 million to $4.1 million, we have set the price per infusion at $194,000. The recommended dose for a child with steroid-refractory GVHD is twice weekly infusions of 2 million cells per kilogram for four weeks. So really, the price that we've set per infusion is based on the economic value of the treatment and the product is available this quarter for physicians to order.

好吧，首先我要說的是，根據健康經濟模型，使用 Ryoncil 治療的淨正效益在 320 萬美元到 410 萬美元之間，我們將每次輸液的價格定為 194,000 美元。對於患有類固醇抗性 GVHD 的兒童，建議劑量為每週兩次輸注每公斤 200 萬個細胞，持續四週。因此，我們設定的每次輸液價格實際上是基於治療的經濟價值，而該產品本季可供醫生訂購。

The question around how physicians see the product here is entirely based on the clinical efficacy and on the results delivered to date and on the long-term survival benefits given the high mortality rate of this disease and the absence of any other treatment other than Ryoncil for children under 12. So I think everybody is pretty keen to get hold of the product. But Marcelo, you might want to chime in. You were at Tandem. You led our clinical commercial interactions with all the payers and with the various clinicians.

關於醫生如何看待該產品的問題完全基於臨床療效、迄今為止的結果以及考慮到該疾病的高死亡率以及除了 Ryoncil 之外沒有任何其他針對 12 歲以下兒童的治療方法，該產品的長期生存益處。所以我認為每個人都非常渴望得到該產品。但是馬塞洛，你可能會想插嘴一下。你當時在 Tandem。您領導了我們與所有付款人和各個臨床醫生的臨床商業互動。

So let's address both, right? So thank you, Silviu. So first of all, Tandem, we saw Tandem as our scientific launch. We had multiple activities during the convention. I think we could see the enthusiasm for the products and the level of questions asked in terms of availability, when people can actually start prescribing the product.

那麼讓我們來解決這兩個問題，對嗎？所以謝謝你，Silviu。首先，我們將 Tandem 視為我們的科學發射。大會期間我們舉辦了多項活動。我認為，當人們真正開始開立該產品處方時，我們可以看到人們對該產品的熱情以及在可用性方面提出的問題程度。

For us, it was very, very encouraging. And the feedback that we received from most centers was also very well, very important for us, right? So Tandem was a successful scientific launch.

對我們來說，這是非常非常令人鼓舞的。而且我們從大多數中心收到的回饋對我們來說也非常重要，對嗎？因此，Tandem 是一次成功的科學發射。

Now, in terms of engagement with payers, obviously, we've been engaging with all payers for quite some time now. I think there is an appreciation for the low number of kids that is affected by this condition, right? So it's 375 kids. And also like this, as Silviu mentioned, this price is fair when you consider the benefit that Ryoncil provides to these kids.

現在，就與付款人的互動而言，顯然我們已經與所有付款人互動了相當長一段時間。我認為，受這種疾病影響的兒童數量很少，這是值得慶幸的，對嗎？所以一共有 375 個孩子。而且正如 Silviu 提到的那樣，如果考慮到 Ryoncil 為這些孩子帶來的利益，這個價格是公平的。

So overall, I think the discussions have been very positive. I think there's an appreciation for the burden of the disease, and there is also an appreciation for the long-term survival that Ryoncil offers these patients. So we are very optimistic, and we're looking forward to next steps.

所以總的來說，我認為討論非常積極。我認為人們認識到這種疾病的負擔，也認識到 Ryoncil 為這些患者提供的長期生存。因此我們非常樂觀，並期待下一步。

All right. And then just one more for me. I wanted to see if you had any thoughts regarding December's FDA draft guidance on accelerated approvals, in particular, how that could pertain to the class IV heart failure program. Is there an expectation that you'll need to start up the class II/III confirmatory ahead of that filing? And then just what are your thoughts on timing and next steps to get that confirmatory study going?

好的。然後我再說一次。我想了解您對 12 月 FDA 關於加速審批的指導草案有何看法，特別是這與 IV 類心臟衰竭計畫有何關係。您是否預計需要在提交申請之前啟動 II/III 類確認程序？那麼，您對啟動此驗證性研究的時間安排和後續步驟有何看法？

Yes. I think this is the key -- the right key question, and we expect to be meeting with the FDA in the coming month or so or two months or so time frame to clarify exactly that. We've put into the so-called briefing poster to be reviewed by FDA, the clinical trial design and the components of a confirmatory study that we would want to do post-accelerated approval. And so the details of that are really what we want to discuss with the FDA.

是的。我認為這是關鍵——正確的關鍵問題，我們預計將在未來一個月或兩個月左右與 FDA 會面，以明確這一點。我們已將臨床試驗設計以及我們希望在加速批准後進行的確認性研究的組成部分放入 FDA 審查的所謂簡報海報中。因此，我們真正想與 FDA 討論的是這方面的細節。

We would expect that given the severity of the patients with advanced and end-stage heart failure and the mortality benefits that we've shown, that the FDA would want us to put the product on market as soon as possible. And the arrangements or the discussions with the FDA around the start-up and the agreement on the confirmatory trial design itself, I think, would be a gating event. I'll come back to the Street as soon as we have more clarity on that.

我們預計，考慮到晚期和末期心臟衰竭患者的嚴重程度以及我們已經證明的死亡率益處，FDA 希望我們盡快將產品推出市場。我認為，圍繞啟動事宜與 FDA 進行的安排或討論以及就確認性試驗設計本身達成的協議將是一個門控事件。一旦我們對此有了更清晰的認識，我就會回到華爾街。

There are no further questions at this time. I'll now hand back to Dr. Itescu for closing comments.

目前沒有其他問題。現在我將把主題交還給 Itescu 博士，請他做最後評論。

Great. Look, I want to thank everybody on the line who's listened to our presentation today. We couldn't be more excited about how rapidly we're delivering this product to the children who need it. There's a lot of work that is going on behind the scenes at every level in the company from commercial to manufacturing to regulatory and the amount of effort that's going into doing this right and doing it in a way that we will save lives. And we're going to work with our partners, the physicians, the institutions and the families to make sure that we deliver a top-quality product that is going to save a lot of lives.

偉大的。瞧，我想感謝今天在線上聆聽我們演講的所有人。我們對於能夠如此迅速地將產品送到需要它的孩子們手中感到無比興奮。從商業到製造到監管，公司各個層面都在幕後進行大量工作，為了正確地完成這項工作並以拯救生命的方式完成這項工作，我們付出了巨大的努力。我們將與我們的合作夥伴、醫生、機構和家庭一起努力，確保提供能夠挽救大量生命的優質產品。

Today was a summary of the activities in the last 6 months, and I think you're going to hear a lot more from us in short order as we move forward to start putting out our product and making it available in the marketplace to physicians and health care providers. Thank you all.

今天是過去 6 個月活動的總結，隨著我們開始推出我們的產品並將其推向市場供醫生和醫療保健提供者使用，我想您很快就會聽到我們的更多消息。謝謝大家。

Thank you. That does conclude our conference for today. Thank you for participating. You may now disconnect.

謝謝大家。今天的會議就到此結束。感謝大家的參與。您現在可以斷開連線。