MorphoSys AG (MOR) 2023 Q1 法說會逐字稿

Ladies and gentlemen, thank you for standing by. Welcome, and thank you for joining the First Quarter Interim Statement 2023 of MorphoSys. (Operator Instructions) I would now like to turn the conference over to Julia Neugebauer. Please go ahead.

女士們先生們，感謝你們的支持。歡迎並感謝您參與 MorphoSys 2023 年第一季度中期報表。 （操作員指示）我現在想將會議轉交給 Julia Neugebauer。請繼續。

Ladies and gentlemen, good afternoon or good morning. My name is Julia Neugebauer, Head of Investor Relations at MorphoSys, and it is my pleasure to welcome you to our first quarter 2023 financial results conference call.

女士們先生們，下午好或早上好。我是 MorphoSys 投資者關係主管 Julia Neugebauer，我很高興歡迎您參加我們的 2023 年第一季度財務業績電話會議。

Joining me on the call today are Jean-Paul Kress, Chief Executive Officer; Tim Demuth, Chief Research and Development Officer; and Joe Horvat, U.S. General Manager, who will join for the Q&A.

今天與我一起參加電話會議的是首席執行官 Jean-Paul Kress； Tim Demuth，首席研發官；和美國總經理 Joe Horvat 將參加問答。

Before we begin, I'd like to remind you on Slide 2 that some of the statements made during the call today are forward-looking statements, including statements regarding our expectations for the commercialization of our products and our development plans, and expectations for the compounds in our pipeline as well as the development plans of our collaboration partners.

在我們開始之前，我想在幻燈片 2 上提醒您，今天電話會議中所做的一些陳述屬於前瞻性陳述，包括有關我們對產品商業化和開發計劃的預期的陳述，以及對我們的管道中的化合物以及我們的合作夥伴的開發計劃。

These forward-looking statements are subject to a number of risks and uncertainties that may cause our actual results to differ materially, including those described in MorphoSys' 20-F and annual report, all for the year ended December 31, 2022, and from time to time in other SEC documents of MorphoSys. It is important to keep in mind that our statements in this webcast speak as of today.

這些前瞻性陳述受到許多風險和不確定性的影響，可能導致我們的實際結果出現重大差異，包括 MorphoSys 的 20-F 和年度報告中描述的風險和不確定性，所有這些風險和不確定性均截至 2022 年 12 月 31 日止年度，並且從那時起與 MorphoSys 的其他 SEC 文件中的時間相同。重要的是要記住，我們在本次網絡廣播中的聲明僅適用於今天。

On Slide 3, you will find the agenda for today's call. Jean-Paul will begin with an overview and give an outlook. After that, Tim will share an update on our clinical development work, and then we will provide a summary of our first quarter 2023 financial results. Following our prepared remarks, we will open the call for your questions.

在幻燈片 3 上，您將找到今天電話會議的議程。讓-保羅將從概述開始並給出展望。之後，Tim 將分享我們臨床開發工作的最新情況，然後我們將提供 2023 年第一季度財務業績的摘要。在我們準備好的發言之後，我們將開始電話詢問您的問題。

With that, I now hand the call over to Jean-Paul.

現在，我將電話轉交給讓-保羅。

Good morning and good afternoon, everyone. Thanks for joining us today. We had a strong first quarter marked by numerous achievements. We are more focused than ever on the opportunities ahead of us this year, and I'm confident that we will deliver.

大家早上好，下午好。感謝您今天加入我們。我們的第一季度表現強勁，取得了眾多成就。今年我們比以往任何時候都更加關注擺在我們面前的機遇，我相信我們能夠實現這一目標。

Pelabresib, our investigational BET inhibitor is a potential best and first-in-class foundational first-line treatment for patients with myelofibrosis. It represents our largest and most immediate opportunity. This quarter, we announced that we completed enrollment of our Phase III MANIFEST-2 study of pelabresib in myofibrosis ahead of schedule. As a result, the top line data from the trial are now expected by the end of 2023, months earlier than previously anticipated.

Pelabresib 是我們的研究性 BET 抑製劑，是骨髓纖維化患者潛在的最佳、一流的基礎一線治療藥物。它代表了我們最大、最直接的機會。本季度，我們宣布提前完成了 pelabresib 治療肌纖維化的 III 期 MANIFEST-2 研究的入組。因此，目前預計該試驗的主要數據將在 2023 年底之前公佈，比之前的預期提前了幾個月。

This advancement of the trial time line also provides us with the opportunity to bring pelabresib to patients much earlier. The MANIFEST-2 study enrolling ahead of schedule underscores that there is a significant need for better treatment options for patients with myelofibrosis. Further, it shows the enthusiasm of investigators and treating physicians for pelabresib. In speaking with physicians who treat myelofibrosis patients, we constantly hear that depth and durability of responses to treatment are limited with current first-line therapy.

試驗時間的提前也為我們提供了更早為患者提供 pelabresib 的機會。提前入組的 MANIFEST-2 研究強調，骨髓纖維化患者迫切需要更好的治療選擇。此外，它還顯示了研究人員和治療醫生對 pelabresib 的熱情。在與治療骨髓纖維化患者的醫生交談時，我們不斷聽到目前一線治療的治療反應深度和持久性受到限制。

Results from our Phase II MANIFEST study of pelabresib in myelofibrosis suggest that pelabresib in combination with a JAK inhibitor may offer prolonged improvements in both spleen size and symptom severity at and beyond 24 weeks. Further to this, in the MANIFEST study, changes in biomarkers correlated with improvements in clinical measures of treatment success suggesting a potential disease-modifying effect of pelabresib. The body of data presented on pelabresib to date, reiterates its potential to address the critical needs of myelofibrosis patients. We are laser-focused on delivering the top line data from the Phase III MANIFEST-2 study by the end of this year.

我們對 pelabresib 治療骨髓纖維化的 II 期 MANIFEST 研究結果表明，pelabresib 與 JAK 抑製劑聯合使用可能會在 24 週及之後長期改善脾臟大小和症狀嚴重程度。此外，在 MANIFEST 研究中，生物標誌物的變化與治療成功的臨床指標的改善相關，表明 pelabresib 具有潛在的疾病緩解作用。迄今為止關於 pelabresib 的大量數據重申了其滿足骨髓纖維化患者關鍵需求的潛力。我們致力於在今年年底之前提供 III 期 MANIFEST-2 研究的主要數據。

We also see great potential for pelabresib beyond myelofibrosis, and we will continue to explore it in treating patients with other myeloid diseases. Monjuvi continues to address critical needs of patients living with relapsed or refractory diffuse large B-cell lymphoma, also known as DLBCL. In the first quarter, Monjuvi net sales were USD 20.8 million, representing an 11% year-over-year growth and on track with our 2023 guidance. At the 2023 AACR Annual Meeting, we presented final 5-year follow-up data from the Phase II L-MIND study. These data show that Monjuvi plus lenalidomide offers prolonged and durable responses in adults with relapsed or refractory DLBCL with 40% of patients who received the regimen still alive after 5 years.

我們還看到了 Pelabresib 在骨髓纖維化之外的巨大潛力，我們將繼續探索它在治療其他骨髓疾病患者中的應用。 Monjuvi 繼續滿足復發性或難治性瀰漫性大 B 細胞淋巴瘤（也稱為 DLBCL）患者的關鍵需求。第一季度，Monjuvi 淨銷售額為 2080 萬美元，同比增長 11%，符合我們 2023 年的指導方針。在 2023 年 AACR 年會上，我們展示了 II 期 L-MIND 研究的最終 5 年隨訪數據。這些數據表明，Monjuvi 聯合來那度胺對患有復發性或難治性 DLBCL 的成人患者提供長期且持久的緩解，接受該方案的患者中有 40% 在 5 年後仍然存活。

The durable responses and consistent safety profile observed in the 5-year analysis further support the Monjuvi regimen as a potentially curative option for appropriate patients. We believe the largest opportunity for Monjuvi is in the first-line DLBCL setting. Last month, we announced that enrollment of the Phase III frontMIND study is also complete with more than 880 patients enrolled in the trial. The study is exploring tafasitamab plus lenalidomide in addition to R-CHOP, the current standard of care for this patient population versus R-CHOP alone as a first-line treatment for patients with high intermediate and high-risk DLBCL.

5 年分析中觀察到的持久反應和一致的安全性進一步支持 Monjuvi 方案作為適當患者的潛在治療選擇。我們認為 Monjuvi 最大的機會是一線 DLBCL 治療。上個月，我們宣布 III 期 frontMIND 研究的入組工作也已完成，共有 880 多名患者入組。該研究正在探索除 R-CHOP 之外，將他法西他單抗加來那度胺作為該患者群體的當前護理標準，與單獨使用 R-CHOP 作為高、中、高危 DLBCL 患者的一線治療進行比較。

We look forward to sharing data from the trial in the second half of 2025. We have a rich set of pivotal catalysts over the next 2 years, starting with the pelabresib Phase III data in first-line myofibrosis later this year. To ensure we are set up for success, we continue to take steps to optimize our cost structure and further strengthen our financial position. For example, we recently purchased part of our convertible bonds that are due in 2025 to reduce our debt. We did this to take advantage of the market dynamics as the bond is trading with significant discount. As a result, we were able to buy back approximately 19% of our outstanding principal amount at a lower cost. We continue to concentrate our investments on our most advanced clinical programs that will create near-term value.

我們期待在 2025 年下半年分享試驗數據。我們在未來 2 年擁有豐富的關鍵催化劑，從今年晚些時候用於一線肌纖維化的 pelabresib III 期數據開始。為了確保我們取得成功，我們繼續採取措施優化成本結構並進一步加強我們的財務狀況。例如，我們最近購買了部分 2025 年到期的可轉換債券，以減少債務。我們這樣做是為了利用市場動態，因為債券的交易折扣很大。因此，我們能夠以較低的成本回購約 19% 的未償還本金。我們繼續將投資集中在最先進的臨床項目上，這些項目將創造短期價值。

I would now like to turn the call over to Tim to provide the development update. Tim, over to you.

我現在想將電話轉給蒂姆，以提供開發更新。蒂姆，交給你了。

Thank you, Jean-Paul. We continue to advance our ongoing mid- to late-stage clinical programs and make exceptional progress. We'll start with pelabresib. The Phase III MANIFEST-2 study is our #1 priority. MANIFEST-2 is a global multicenter double-blind study of more than 400 patients who were naive to JAK inhibitors. Patients were randomized 1:1 to pelabresib in combination with ruxolitinib or placebo plus ruxolitinib. The primary endpoint of the study is the proportion of patients who achieve a 35% or greater reduction in spleen volume at week 24, known as SVR35. The key secondary endpoint is the proportion of patients achieving a 50% or greater improvement in total symptom score at week 24.

謝謝你，讓-保羅。我們繼續推進正在進行的中後期臨床項目並取得非凡進展。我們將從 Pelabresib 開始。 III 期 MANIFEST-2 研究是我們的第一要務。 MANIFEST-2 是一項全球多中心雙盲研究，納入了 400 多名未接受過 JAK 抑製劑治療的患者。患者按 1:1 的比例隨機分配至 Pelabresib 聯合魯索替尼或安慰劑加魯索替尼。該研究的主要終點是第 24 週時脾臟體積減少 35% 或更多的患者比例，稱為 SVR35。關鍵的次要終點是第 24 週總症狀評分改善 50% 或以上的患者比例。

This is known as TSS50 and is measured by the Myelofibrosis Symptom Assessment Form version 4.0. The MANIFEST-2 study is supported by findings from the Phase II MANIFEST trial of pelabresib in combination with ruxolitinib in patients with myelofibrosis, including those who were JAK inhibitor naive. Updated results from MANIFEST were presented at the ASH meeting in December 2022 and were recently published in the Journal of Clinical Oncology. These results suggest that pelabresib in combination with ruxolitinib provides prolonged improvement in both spleen size and symptom severity at and beyond 24 weeks. In the MANIFEST study, changes in biomarkers correlated with improvements in clinical measures of treatment success.

這稱為 TSS50，通過骨髓纖維化症狀評估表 4.0 版進行測量。 MANIFEST-2 研究得到了 pelabresib 聯合魯索替尼治療骨髓纖維化患者（包括未接受過 JAK 抑製劑治療的患者）的 II 期 MANIFEST 試驗結果的支持。 MANIFEST 的最新結果已在 2022 年 12 月的 ASH 會議上公佈，並於最近發表在《臨床腫瘤學雜誌》上。這些結果表明，pelabresib 與 ruxolitinib 聯合使用可在 24 週及之後長期改善脾臟大小和症狀嚴重程度。在 MANIFEST 研究中，生物標誌物的變化與治療成功的臨床指標的改善相關。

This included SVR35, TSS50 and hemoglobin increases indicative of improved anemia, suggesting a disease-modifying effect of pelabresib. Examined biomarkers included bone marrow scarring, known as fibrosis and the frequency of the JAK2 allele that is known to drive disease activity. All patients who had clinical responses plus reduced allele frequency and improvement in bone marrow fibrosis were naive to JAK inhibitors. Based on the body of data we have presented thus far, our confidence in pelabresib and the Phase III MANIFEST-2 study is high, and we look forward to releasing the top line data from the trial data this year.

其中包括 SVR35、TSS50 和血紅蛋白增加，表明貧血有所改善，表明 Pelabresib 具有緩解疾病的作用。檢查的生物標誌物包括骨髓疤痕（稱為纖維化）和已知會導致疾病活動的 JAK2 等位基因的頻率。所有具有臨床反應、等位基因頻率降低和骨髓纖維化改善的患者均未曾接受過 JAK 抑製劑治療。根據我們迄今為止提供的數據主體，我們對 pelabresib 和 III 期 MANIFEST-2 研究充滿信心，我們期待今年發布試驗數據的頂線數據。

Moving on to tafasitamab. As Jean-Paul mentioned, at the 2023 AACR annual meeting, final data from our Phase II L-MIND study were presented during a late-breaking oral presentation spotlighting 5-year efficacy and safety results in patients with relapsed or refractory DLBCL. That's the data cutoff. The overall response rate of patients enrolled in the study was 58%, and complete response was observed in 41% of patients. The median overall survival was 34 months with 40% of patients alive at 5 years. The regimen was well tolerated and no new safety signals were identified. The prolonged and durable responses seen at 5 years among patients in this study are very meaningful and show that the Monjuvi treatment regimen has curative potential.

繼續使用他法西他單抗。正如 Jean-Paul 提到的，在 2023 年 AACR 年會上，我們的 II 期 L-MIND 研究的最終數據在最新的口頭報告中公佈，重點關注復發或難治性 DLBCL 患者的 5 年療效和安全性結果。這就是數據截止。參加該研究的患者總體緩解率為 58%，其中 41% 的患者出現完全緩解。中位總生存期為 34 個月，其中 40% 的患者在 5 年後仍存活。該方案耐受性良好，沒有發現新的安全信號。本研究中患者在 5 年時觀察到的長期且持久的反應非常有意義，表明 Monjuvi 治療方案具有治療潛力。

Beyond the currently approved indication, we're also exploring tafasitamab in 2 Phase III studies, frontMIND in first-line DLBCL and inMIND in relapsed or refractory follicular or marginal zone lymphoma, which is being driven by our partner Incyte. For about 50% of patients with high intermediate and high-risk DLBCL, the standard of care first-line therapy, R-CHOP is ineffective. And the prognosis for patients with relapsed or refractory disease is very poor. We are investigating the potential of adding tafasitamab and lenalidomide to R-CHOP to increase the DLBCL cure rate in the first line and help more patients avoid relapse. At the ASCO 2023 Annual Meeting in early June we will present data that reinforces the strong potential of our pipeline.

除了目前批准的適應症之外，我們還在 2 項 III 期研究中探索 tafasitamab，frontMIND 用於一線 DLBCL，inMIND 用於復發或難治性濾泡性或邊緣區淋巴瘤，該研究由我們的合作夥伴 Incyte 推動。對於約 50% 的高、中、高危 DLBCL 患者，標準治療一線治療 R-CHOP 無效。復發或難治性疾病患者的預後很差。我們正在研究在 R-CHOP 中添加他法西他單抗和來那度胺以提高一線 DLBCL 治愈率並幫助更多患者避免復發的潛力。在 6 月初舉行的 ASCO 2023 年會上，我們將展示增強我們產品線強大潛力的數據。

Our presentations feature proof-of-concept data on pelabresib in essential thrombocythemia and tulmimetostat, our investigational next-generation dual inhibitor of EZH2 and EZH1 in a broad array of advanced tumors. In our Phase II MANIFEST study, pelabresib is also being investigated in patients with essential thrombocythemia in addition to myelofibrosis. These indications are both myeloproliferative neoplasms, which are types of blood cancers that begin with a genetic change in bone marrow stem cells. One arm of the MANIFEST study is exploring pelabresib as monotherapy in patients with high-risk essential thrombocythemia who are refractory or intolerant to hydroxyurea. The chemotherapeutic agents most used to treat this disease.

我們的演講以 Pelabresib 治療原發性血小板增多症和 tulmimetostat 的概念驗證數據為特色，tulmimetostat 是我們在一系列晚期腫瘤中研究的下一代 EZH2 和 EZH1 雙重抑製劑。在我們的 II 期 MANIFEST 研究中，除了骨髓纖維化之外，還在患有原發性血小板增多症的患者中研究了 pelabresib。這些適應症都是骨髓增生性腫瘤，這是一種始於骨髓幹細胞基因變化的血癌。 MANIFEST 研究的一個分支正在探索 Pelabresib 作為單一療法治療對羥基脲難治或不耐受的高危原發性血小板增多症患者。最常用於治療這種疾病的化療藥物。

Proof-of-concept data from this arm of the Phase II study will be presented during a poster discussion session. Tulmimetostat is being evaluated in a Phase I/II trial in patients with advanced solid tumors or lymphomas, including ARID1A mutated ovarian carcinoma and endometrial carcinoma, BAP1-mutated mesothelioma, and peripheral T-cell lymphoma. Tulmimetostat was designed to improve on first generation EZH2 inhibitors through increased potency, longer residence time on target and a longer half life offering the potential for enhanced antitumor activity. Preliminary results from the Phase II portion of the study, evaluating tulmimetostat across multiple tumor types will be presented during a poster session.

第二階段研究的這一部分的概念驗證數據將在海報討論會上展示。 Tulmimetostat 正在 I/II 期試驗中對晚期實體瘤或淋巴瘤患者進行評估，包括 ARID1A 突變的卵巢癌和子宮內膜癌、BAP1 突變的間皮瘤和外周 T 細胞淋巴瘤。 Tulmimetostat 旨在通過提高效力、延長靶標停留時間和延長半衰期來改進第一代 EZH2 抑製劑，從而提供增強抗腫瘤活性的潛力。該研究的 II 期部分的初步結果（評估 tulmimetostat 對多種腫瘤類型的影響）將在海報會議期間公佈。

In summary, the data we are presenting at ASCO 2023 showcases the wealth of potential opportunities that our pipeline offers to address the critical needs of people living with blood cancers, including myeloid malignancies and those patients with solid tumors.

總之，我們在 ASCO 2023 上展示的數據展示了我們的產品線為滿足血癌患者（包括骨髓惡性腫瘤和實體瘤患者）的關鍵需求提供的大量潛在機會。

With that, I now turn the call over to Julia to review our financials.

現在，我將電話轉給朱莉婭，以審查我們的財務狀況。

Thank you, Tim. We're pleased to share our financial results for the first quarter of 2023. Monjuvi sales were $20.8 million in the first quarter of 2023, reflecting a year-over-year growth of 11%, driven by higher demand. On a sequential basis, sales declined by 18%, largely due to the inventory dynamics and demand seasonality. We continue to be excited for the Minjuvi opportunity outside of the U.S., which our partner Incyte is responsible for. In the first quarter of 2023, we recorded EUR 0.7 million or $0.8 million in royalty revenues for Minjuvi.

謝謝你，蒂姆。我們很高興分享 2023 年第一季度的財務業績。Monjuvi 在 2023 年第一季度的銷售額為 2080 萬美元，在需求增加的推動下，同比增長 11%。銷售額環比下降 18%，主要是由於庫存動態和需求季節性。我們仍然對 Minjuvi 在美國以外的機會感到興奮，這是我們的合作夥伴 Incyte 負責的。 2023 年第一季度，Minjuvi 的特許權使用費收入為 70 萬歐元（80 萬美元）。

Total revenues in the first quarter of 2023 were EUR 62.3 million compared to EUR 41.5 million in the same period a year ago. This increase resulted mainly from higher revenues from the sale of clinical vials. Total cost of sales were EUR 21 million in the first quarter of 2023 compared to EUR 7.9 million a year ago. The year-over-year increase resulted primarily from expenses related to vial sales to our partner Incyte. Cost of sales specific to Monjuvi U.S. product sales were EUR 3.1 million in the first quarter of 2023.

2023 年第一季度的總收入為 6230 萬歐元，而去年同期為 4150 萬歐元。這一增長主要是由於臨床藥瓶銷售收入增加。 2023 年第一季度的總銷售成本為 2100 萬歐元，而去年同期為 790 萬歐元。同比增長主要是由於向我們的合作夥伴 Incyte 銷售小瓶的相關費用。 2023 年第一季度 Monjuvi 美國產品銷售的具體銷售成本為 310 萬歐元。

Turning to operating expenses. R&D expenses in the first quarter of 2023 were EUR 83.1 million compared to EUR 65 million for the first quarter of 2022. The growth primarily reflects the additional costs incurred due to the positive development of the patient recruitment in the major ongoing clinical studies, and a onetime effect resulting from severance payments in connection with the restructuring of the research area. Selling expenses decreased to EUR 16.9 million in the first quarter of 2023 compared to EUR 21.9 million for the same period in 2022. The year-over-year decline was driven by streamlining and focusing on selling efforts. G&A expenses in the first quarter of 2023 were EUR 10.9 million compared to EUR 14.6 million in the first quarter of 2022.

轉向運營費用。 2023 年第一季度的研發費用為 8310 萬歐元，而 2022 年第一季度為 6500 萬歐元。這一增長主要反映了由於正在進行的主要臨床研究中患者招募的積極進展而產生的額外成本，以及與研究領域重組有關的遣散費所產生的一次性影響。 2023 年第一季度銷售費用下降至 1690 萬歐元，而 2022 年同期為 2190 萬歐元。同比下降的原因是精簡和專注於銷售工作。 2023 年第一季度的一般管理費用為 1,090 萬歐元，而 2022 年第一季度為 1,460 萬歐元。

For the first quarter of 2023, we reported a consolidated net loss of EUR 44.4 million compared to a net loss of EUR 122.7 million in the first quarter of 2022. The lower consolidated net loss in 2023 was driven mainly by the recognition of finance income in relation to the financial liabilities from future payments to Royalty Pharma and by additional finance income derived from the repurchase of a portion of outstanding convertible bonds.

2023 年第一季度，我們報告的綜合淨虧損為 4440 萬歐元，而 2022 年第一季度的淨虧損為 1.227 億歐元。2023 年綜合淨虧損較低的主要原因是確認了 2023 年的財務收入與未來向Royalty Pharma 付款的金融負債以及回購部分未償還可轉換債券產生的額外財務收入有關。

Turning to our balance sheet. We ended the first quarter of 2023 with cash and investments of EUR 791.5 million compared to EUR 907.2 million at the end of 2022. Our solid cash position enables us not only to reach the pivotal data milestone for the Phase III study of pelabresib now expected by the end of 2023, but to also provide a cash runway of at least 12 months beyond the pivotal data readout.

轉向我們的資產負債表。截至 2023 年第一季度末，我們的現金和投資為 7.915 億歐元，而 2022 年底為 9.072 億歐元。我們穩健的現金狀況使我們不僅能夠實現目前預計的 Pelabresib III 期研究的關鍵數據里程碑2023 年底，但也提供了關鍵數據讀出後至少 12 個月的現金跑道。

Turning to our guidance for 2023. We are reiterating our guidance that was provided at the beginning of January this year. All aspects of our guidance remain the same.

談到我們對 2023 年的指導。我們重申今年 1 月初提供的指導。我們指導的各個方面都保持不變。

With that, I now turn the call back to Jean-Paul.

現在，我將電話轉回讓-保羅。

Before we go into Q&A, I want to conclude a few words. We made exceptional progress this quarter, and we are more focused than ever to build on this great momentum and drive our strong mid- to late-stage pipeline forward. With pelabresib, we have the opportunity to bring a foundational first-line treatment to patients living with myelofibrosis with the potential to improve the standard of care. Now that our Phase III MANIFEST-2 study has completed enrollment earlier than anticipated, we will release top line data later this year.

在我們進入問答之前，我想總結幾句話。本季度我們取得了非凡的進展，我們比以往任何時候都更加專注於鞏固這一巨大勢頭，推動我們強大的中後期產品線向前發展。借助 pelabresib，我們有機會為骨髓纖維化患者提供基礎的一線治療，並有可能提高護理標準。現在我們的 III 期 MANIFEST-2 研究已比預期提前完成入組，我們將在今年晚些時候發布頂線數據。

On Wednesday, June 21, we will host the virtual event that provides an in-depth overview of pelabresib and its potential. Dr. John Mascarenhas, Professor of Medicine and Director of the Adult Leukemia Program at The Tisch Cancer Institute at Mount Sinai, New York, will speak and be available for questions. Further details about this session will be provided closer to the date. We are well financed to deliver on our priorities, and we will continue to focus our resources on our most advanced clinical programs that will create near-term value.

6 月 21 日星期三，我們將舉辦虛擬活動，深入概述 pelabresib 及其潛力。紐約西奈山蒂施癌症研究所的醫學教授兼成人白血病項目主任 John Mascarenhas 博士將發表講話並回答問題。有關本次會議的更多詳細信息將在臨近日期時提供。我們有充足的資金來實現我們的優先事項，我們將繼續將資源集中在最先進的臨床項目上，以創造近期價值。

With that, I would like to open the call for questions. Operator, please open the line.

至此，我想開始提問。接線員，請開通線路。

(Operator Instructions) And the first question comes from Jason Butler from JMP Securities.

（操作員說明）第一個問題來自 JMP 證券的 Jason Butler。

Congrats on the progress. Just one on MANIFEST-2. Can you -- now you've completed enrollment. Can you comment on the comparability of the patient population in MANIFEST-2 to arm 3 of the MANIFEST-1 trial? And then wondering if you could just walk us through a little bit more detail of your commercial preps for pelabresib and what you're doing to build awareness ahead of data.

祝賀取得的進展。 MANIFEST-2 上只有一個。你可以嗎——現在你已經完成了註冊。您能否評論一下 MANIFEST-2 試驗中患者群體與 MANIFEST-1 試驗第 3 組患者群體的可比性？然後想知道您是否可以向我們介紹一下您為 pelabresib 進行商業準備的更多細節，以及您正在採取哪些措施來在數據之前建立意識。

Thanks, Jason. Tim will take the first part of the question and Joe will answer the second part.

謝謝，傑森。蒂姆將回答問題的第一部分，喬將回答第二部分。

Yes. Jason, this is Tim. In terms of comparability between the population of MANIFEST-2 and arm 3 in the MANIFEST, generally, the populations are very comparable. That was intentional. So we can really use manifest as a very good benchmark for what to expect in MANIFEST-2. So highly comparable patient populations there.

是的。傑森，這是蒂姆。就MANIFEST-2的群體和MANIFEST中的arm 3之間的可比性而言，總體來說，群體非常具有可比性。那是故意的。因此，我們確實可以將清單用作 MANIFEST-2 中預期內容的非常好的基準。那裡的患者群體具有高度可比性。

And Jason, on the commercial readiness, a couple of thoughts here. First and foremost, we have our organization, commercial organization already interacting, as you know, with space for Monjuvi and there is a very high level of overlap in the target, so we don't have to start from scratch. Obviously, we make adjustments with time. We have time for that. What is really important now is to continue to engage with the key opinion leaders, and we've been doing that through our development program, and we've basically interacted with the most relevant sites worldwide, including very much so in the U.S. for that.

賈森，關於商業準備情況，我有一些想法。首先也是最重要的是，我們的組織、商業組織已經與 Monjuvi 的空間進行了互動，而且目標有很高的重疊度，所以我們不必從頭開始。顯然，我們會隨著時間的推移進行調整。我們有時間這樣做。現在真正重要的是繼續與關鍵意見領袖接觸，我們一直通過我們的開發計劃來做到這一點，我們基本上已經與世界各地最相關的網站進行了互動，包括在美國的網站上。 。

But at the same time, now we are having our medical affairs organization work with the field on engaging on the beta and raising excitement. So it's going very well. And we constantly hear a very strong endorsement of our regimen and the desire to continue these interactions and engagement. And again, the fact that we have enrolled that swiftly and ahead of time is a really, really strong indicator of the interest for this time.

但與此同時，現在我們正在讓我們的醫療事務組織與該領域合作，參與測試並提高興奮度。所以一切進展順利。我們不斷聽到對我們的治療方案的強烈認可以及繼續這些互動和參與的願望。再說一次，我們如此迅速、提前註冊這一事實確實非常強烈地表明了人們對這一次的興趣。

And the question comes from James Quigley from Morgan Stanley.

這個問題來自摩根士丹利的詹姆斯·奎格利。

I got 3, please. So on pelabresib and thinking about other endpoints beyond SVR35 and TSS50, so in terms of additional data on bone marrow fibrosis and survival data. At what point can we expect this data to come through? And it's not listed as a second endpoint on clinicaltrials.gov, but how important could these additional data be in terms of driving uptake or from your conversations, does that not matter, it's more of a nice to have for the future?

我有3個，拜託。因此，關於 pelabresib 並考慮 SVR35 和 TSS50 之外的其他終點，因此就骨髓纖維化和生存數據的附加數據而言。我們預計什麼時候可以得到這些數據？它沒有被列為 ClinicalTrials.gov 上的第二個終點，但這些額外的數據在推動採用或從您的對話中有多重要，這不重要嗎？對於未來來說，擁有它更加美好？

Secondly, on Monjuvi curative potential, in the L-MIND- 5-year update, are there any patients that have stop therapy and not rebounded. So are there any patients that have gone into a complete response manage to stop therapy or come off therapy and not see the accounts of return. I'm just thinking about that in terms of demonstrating curative potential.

其次，關於Monjuvi的治療潛力，在L-MIND-5年的更新中，是否有患者停止治療並且沒有反彈。那麼是否有任何已經完全緩解的患者在停止治療或結束治療後卻看不到恢復情況呢？我只是從展示治療潛力的角度考慮這一點。

And finally, on R&D, there's a bit of a step up in Q1 due to the Phase III trials. If you can give us a sense of what portion of your current R&D is late stage or is related to late-stage assets. Obviously, you stopped the early-stage research, but how should we think about how R&D develops over time? You gave a little hint with the essential thrombocytopenia, but should this step down over time as those trials run off?

最後，在研發方面，由於第三階段試驗，第一季度取得了一些進展。您能否讓我們了解您當前研發的哪些部分處於後期階段或與後期資產相關。顯然，你停止了早期研究，但我們應該如何思考研發隨著時間的推移如何發展？您對基本的血小板減少症給出了一些暗示，但是隨著這些試驗的結束，這種情況是否應該逐漸減弱？

Thanks, James. I'll start by the last question and Tim will address the pela and the Monjuvi questions. Regarding the evolution of our focus and capital allocation towards the R&D spectrum, you're correct, we are obviously now very much focusing on our late-stage opportunities, and there will be a phasing with that. Right now, you can probably assume that we are at the peak in terms of our Phase III efforts. But we've got the pela MANIFEST-2 study. We've got the frontMIND study. And together with inMIND -- with Incyte, we have the inMIND study for follicular lymphoma.

謝謝，詹姆斯。我將從最後一個問題開始，蒂姆將解決佩拉和蒙朱維問題。關於我們的重點和資本分配向研發領域的演變，你是對的，我們現在顯然非常關注我們的後期機會，並且將會分階段進行。現在，您可能會認為我們的第三階段工作正處於頂峰。但我們有 pela MANIFEST-2 研究。我們有 frontMIND 研究。我們與 inMIND 和 Incyte 一起開展針對濾泡性淋巴瘤的 inMIND 研究。

But that will kind of decrease in the future. So -- but we're not saying that we must start new things, but if you take the [ISO] picture that we're probably peaking now. And so we will actually see an OpEx reduction towards R&D and development in the future. So yes, so that's the answer to that. And indeed, we have greatly reduced our expenses in preclinical to make sure that we could skew the opportunities that we mentioned. Tim, on the 2 other questions?

但未來這種情況會有所減少。所以——但我們並不是說我們必須開始新的事情，但如果你拍一下 [ISO] 圖片，我們現在可能已經達到了頂峰。因此，我們實際上會看到未來研發和開發的運營支出有所減少。所以是的，這就是答案。事實上，我們已經大大減少了臨床前的費用，以確保我們能夠利用我們提到的機會。蒂姆，關於另外兩個問題？

Yes. Thank you. So James, on the first question, pela, other endpoints beyond SVR and TSS. So yes, there are other endpoints in the study, as you mentioned, just to come back to the primary endpoint and the key secondary. Obviously, health authorities are expecting, and we know that from interactions with them, are expecting us to demonstrate winning on SVR35 and TSS50, both at week 24. Those are the accepted and expected regulatory endpoints. And we feel very good about meeting those endpoints. If you remember, we particularly increased the sample size from 330 to 400 patients to be particularly well powered for that key secondary endpoint, the TSS50.

是的。謝謝。 James，關於第一個問題，pela，SVR 和 TSS 之外的其他端點。所以，是的，正如您提到的，研究中還有其他終點，只是回到主要終點和關鍵次要終點。顯然，衛生當局期望我們在第 24 週在 SVR35 和 TSS50 上取得勝利，而且我們從與他們的互動中得知，他們期望我們在 SVR35 和 TSS50 上取得勝利。這些是公認的和預期的監管終點。我們對實現這些終點感到非常高興。如果您還記得，我們​​特別將樣本量從 330 名患者增加到 400 名患者，以便為關鍵的次要終點 TSS50 提供特別有力的支持。

As regards bone marrow fibrosis, that's clearly an important translational endpoint and endpoints that very much speaks to potentially altering the natural history of the disease when we're talking at the disease that is carrying the bone marrow and being able to demonstrate reversal of that fibrosis, I think, helps a lot understanding further trajectory. And there is a lot of reason to believe that improvement in bone marrow fibrosis will translate to long-term cycle. And those are, of course, endpoints that we're assessing in the study. You mentioned they are not listed on clinicaltrials.gov, yet, they are definitely part of the protocol. And we will analyze this data as soon as we're done with analysis of the top line data and then have this ready for presentation at an available scientific conference in the near future.

至於骨髓纖維化，這顯然是一個重要的轉化終點，當我們談論攜帶骨髓的疾病並能夠證明纖維化的逆轉時，這在很大程度上可能會改變疾病的自然史我認為，這對理解進一步的軌蹟有很大幫助。有很多理由相信骨髓纖維化的改善將轉化為長期週期。當然，這些是我們在研究中評估的終點。您提到它們沒有在 ClinicalTrials.gov 上列出，但它們絕對是協議的一部分。我們將在完成對主要數據的分析後立即分析這些數據，然後準備在不久的將來在可用的科學會議上進行演示。

As it comes to tafasitamab and the curative potential. You asked about are there patients who stopped treatment with CR and continue to show that survival benefit. Absolutely, yes. If I can just refer you back to that AACR presentation that Professor (inaudible) was giving just a few weeks ago. In Florida, there are a number of patients who actually complete and discontinued treatment and who remain in complete response despite discontinuing or completing treatment. Again, really supporting the potential for cure in these patients treated with tafa/len. And we're very excited about these findings, and so are some of the investigators who we're speaking with about this data.

談到他法西他單抗及其治療潛力。您詢問是否有停止 CR 治療並繼續顯示生存獲益的患者。絕對沒錯。我可以請您回顧一下教授（聽不清）幾週前所做的 AACR 演講嗎？在佛羅里達州，有許多患者實際上完成並停止了治療，並且儘管停止或完成了治療，但仍保持完全緩解。再次，真正支持這些接受 tafa/len 治療的患者的治愈潛力。我們對這些發現感到非常興奮，我們正在談論這些數據的一些研究人員也是如此。

And the next question comes from Zain Ebrahim from JPMorgan.

下一個問題來自摩根大通的 Zain Ebrahim。

Just 2 for me, please. So my first question would be on MANIFEST-2. You brought forward the timing for the study a few times now, and that's because of completing enrollment, but is there any chance of that readout being brought forward even further? Or with this, we can now expect the data in 2023? And my second question would be on the myelofibrosis competitive landscape. So one of a potential competitor for pela, momelotinib that's due for approval in June, possibly in second line, but I think there's some discussion at the moment as to whether that could receive a line agnostic approval. How are you thinking about positioning of pela and Jakafi combination relative to momelotinib and also other potential competitors that we've seen.

請給我 2 個。所以我的第一個問題是關於 MANIFEST-2 的。您現在已經多次提前了研究時間，那是因為完成了註冊，但是是否有可能進一步提前該讀數？或者說，我們現在可以期待 2023 年的數據了嗎？我的第二個問題是關於骨髓纖維化的競爭格局。因此，Pela 的潛在競爭對手之一 Momelotinib 將於 6 月獲得批准，可能是二線藥物，但我認為目前存在一些關於是否可以獲得與生產線無關的批准的討論。您如何看待 pela 和 Jakafi 組合相對於 momelotinib 以及我們見過的其他潛在競爭對手的定位？

Zain, thanks for your question. This is Jean-Paul. From MANIFEST-2, the timing is already greatly, greatly improved compared to what we had in mind over 1 year ago or 1.5 years ago or 2 years ago when we acquired Constellation. I mean, there not many companies or studies, Phase III studies in oncology, especially who have been beating time line. So I think we've done extraordinarily well here, and we don't want to signal not any potential upside. It's a couple of more months. I think now it's about making sure that we deliver the right quality together within this time line.

扎因，謝謝你的問題。這是讓-保羅。從 MANIFEST-2 開始，與我們 1 年前、1.5 年前或 2 年前收購 Constellation 時的想法相比，時機已經大大改善了。我的意思是，沒有多少公司或研究，腫瘤學的第三階段研究，特別是那些一直在趕時間的公司或研究。所以我認為我們在這方面做得非常好，我們不想表明沒有任何潛在的好處。還有幾個月了。我認為現在的重點是確保我們在這個時間內共同提供正確的質量。

So that's for the time line. Regarding the positioning of pelabresib in the landscape. First of all, the landscape is evolving very much and there has been a decade of basically same standard of care with monotherapy, JAK inhibition, building up a very big deal of unmet need. And if unmet need is really, really giving us the opportunity to make a big change here, and this is what we hear constantly from this space.

這就是時間線。關於 Pelabresib 在景觀中的定位。首先，情況正在發生很大的變化，十年來，單一療法、JAK 抑制的護理標準基本相同，形成了大量未滿足的需求。如果未滿足的需求確實給了我們在這裡做出重大改變的機會，這就是我們從這個領域不斷聽到的。

There is awful lot of expectations for a first-line combination in myelofibrosis and pelabresib from what we know from the data and what we as feedback is the longer wait of the opportunity to establish this [topline] change for helping patients in first line because the very important point here is that as much as you hear about many other important products coming up, mostly in the JAK inhibition segment, actually, the real unmet need is topline change in first-line in combination, which should increase and improve the quality of life of the patient and intimate survival of the patients. They need to live longer and better lives and we can provide that with our regimen.

從我們從數據中了解到的情況來看，人們對骨髓纖維化和 Pelabresib 的一線組合抱有很大的期望，而我們的反饋是，等待建立這種[頂線]改變以幫助一線患者的機會的時間更長，因為這裡非常重要的一點是，儘管您聽說了許多其他重要產品的出現，主要是在 JAK 抑制領域，但實際上，真正未滿足的需求是一線組合的頂線變化，這應該會增加和提高質量患者的生命和患者的親密生存。他們需要活得更長、更好，而我們可以通過我們的養生方案來實現這一點。

The other agents are probably going to see some segments of the market. And I would end my comments by saying that you can't exclude that we will not partner with some of them. We're exploring some combination. So we started by the beginning, which is establishing a strong registration with pelabresib plus ruxolitinib, but there are other possibilities which would put us at a cornerstone in the treatment of Myelofibrosis.

其他代理商可能會看到一些市場細分。在我的評論結束時，我想說的是，你不能排除我們不會與其中一些公司合作。我們正在探索一些組合。因此，我們從頭開始，正在與 pelabresib 加魯索替尼建立強有力的註冊，但還有其他可能性可以使我們成為骨髓纖維化治療的基石。

(Operator Instructions)

（操作員說明）

And the next question comes from Vineet Agrawal from Citi.

下一個問題來自花旗集團的 Vineet Agrawal。

Vineet here from Citi. I just want to check if you have shared any data on the weight of the patients on the pelabresib combo. I am just trying to understand how important is the weight gain for these myelofibrosis patients. And just on biomarker analysis, I think Navitoclax showed 50% of patients reaching more than 20% reduction in JAK2 allele frequency. I'm wondering if you could comment on your positioning there from the biomarker analysis perspective.

來自花旗銀行的 Vineet。我只是想檢查一下您是否分享了有關使用 pelabresib 組合的患者體重的任何數據。我只是想了解體重增加對於這些骨髓纖維化患者有多重要。僅就生物標誌物分析而言，我認為 Navitoclax 顯示 50% 的患者 JAK2 等位基因頻率降低了 20% 以上。我想知道您是否可以從生物標誌物分析的角度評論一下您在那裡的定位。

Vineet, thanks for the questions. Tim, go ahead.

維尼特，謝謝你的提問。蒂姆，繼續吧。

Yes. On the first question, have we shown data regarding weight gain in manifest? The answer is, no, we haven't done that. It's something the team is looking into. As far as it goes in terms of the JAK allele burden, we have a presentation on translational data from the arm 2 and arm 3 of the MANIFEST study showing that pelabresib very clearly reduces that JAK2 allele burden in our study. I don't think that we can do reasonably across trial comparison.

是的。關於第一個問題，我們是否在清單中顯示了有關體重增加的數據？答案是，不，我們還沒有這樣做。這是團隊正在研究的事情。就 JAK 等位基因負擔而言，我們介紹了 MANIFEST 研究第 2 組和第 3 組的翻譯數據，表明 pelabresib 非常明顯地降低了我們研究中的 JAK2 等位基因負擔。我認為我們無法合理地進行試驗比較。

But the overall reduction in the JAK2 allele burden is certainly very impressive and something that, again, fits together in to that picture of modifying disease activity, particularly when you put this together with some of the cytokine data that was presented also at EHA last year, together with the megakaryocytes de-clustering and the fibrosis improvement, I think a pattern is emerging that suggests very strongly that pela, ruxolitinib have a very positive effect on modifying the disease.

但 JAK2 等位基因負擔的總體減少無疑是非常令人印象深刻的，並且再次符合改變疾病活動的情況，特別是當你將其與去年也在 EHA 上提出的一些細胞因子數據放在一起時連同巨核細胞去簇化和纖維化改善，我認為正在出現一種模式，強烈表明佩拉、魯索替尼對改變疾病具有非常積極的作用。

So there are no further questions at this time, and I hand back to you, Julia Neugebauer for closing comments.

因此，目前沒有其他問題，我將請朱莉婭·紐格鮑爾 (Julia Neugebauer) 發表結束評論。

Ladies and gentlemen, this concludes today's conference call. If any of you would like to follow up, MorphoSys Investor Relations team is available for the remainder of the day. Once again, thank you for joining our call. Have a good day, and goodbye.

女士們、先生們，今天的電話會議到此結束。如果你們中的任何人想跟進，MorphoSys 投資者關係團隊將在當天剩餘時間為您提供服務。再次感謝您加入我們的通話。祝你有美好的一天，再見。

Ladies and Gentleman, the conference is now concluded, and you may disconnect your telephone. Thank you for joining, and have a pleasant day. Good afternoon.

女士們、先生們，會議現已結束，您可以掛斷電話了。感謝您的加入，祝您度過愉快的一天。下午好。