Traws Pharma Inc (TRAW) 2024 Q2 法說會逐字稿

Ladies and gentlemen, thank you for standing by. Welcome to Traws Pharma, Inc. corporate update call. (Operator Instructions) As a reminder, this call is being recorded today, August 15, 2024. At this time, I would like to turn the call over to Bruce Mackle of Lifesci Advisors.

女士們先生們，謝謝你們的支持。歡迎致電 Traws Pharma, Inc. 公司更新資訊。（操作員說明）謹此提醒，此通話將於今天（2024 年 8 月 15 日）錄音。此時，我想將電話轉給 Lifesci Advisors 的 Bruce Mackle。

Thank you, operator, and welcome, everyone, to Traws Pharma, Inc.'s corporate update conference call. This morning, Traws issued a press release reporting its second quarter 2024 financial results and corporate update. If you have not yet seen this press release, it is available in the Investors & Media section of the company's website at www.trawspharma.com. Following my introduction, we will hear from Traws' Chief Executive Officer, Dr. Werner Cautreels; and Chief Financial Officer, Mark Guerin.

謝謝操作員，歡迎大家參加 Traws Pharma, Inc. 的公司最新情況電話會議。今天上午，Traws 發布了一份新聞稿，報告了 2024 年第二季的財務表現和公司最新情況。如果您尚未看到本新聞稿，可以在本公司網站 www.trawspharma.com 的投資者和媒體部分查看。在我的介紹之後，我們將聽取 Traws 首席執行官 Werner Cautreels 博士的介紹；和財務長馬克·蓋林。

Before we begin, I would like to remind everyone that statements made during this conference call will include forward-looking statements under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, which involve risks and uncertainties that can cause actual results to differ materially.

在開始之前，我想提醒大家，本次電話會議中的陳述將包括1995 年《私人證券訴訟改革法案》安全港條款下的前瞻性陳述，其中涉及風險和不確定性，可能導致實際結果有所不同重大。

Forward-looking statements speak only as of the date they are made as the underlying facts and circumstances may change. Except as required by law, Traws disclaims any obligation to update these forward-looking statements to reflect future information, events or circumstances. For more information on forward-looking statements, please review the disclaimer in today's press release and the risk factors in the company's SEC filings.

前瞻性陳述僅代表發布之日的情況，因為基本事實和情況可能會改變。除法律要求外，Traws 不承擔更新這些前瞻性聲明以反映未來資訊、事件或情況的義務。有關前瞻性陳述的更多信息，請查看今天新聞稿中的免責聲明以及公司向 SEC 提交的文件中的風險因素。

With that, I will turn the call over to Werner.

這樣，我會將電話轉給維爾納。

Thanks, Bruce, and thanks to everyone for joining us today. The last several months have been transformational and very positive for Traws Pharma. Thanks to the excellent execution of everyone on our teams, we have substantially advanced our clinical pipeline. While the data analysis is still ongoing, we believe that we will have a very good picture of the preliminary pharmacokinetics, preferred dosing plans, and safety for each of our product candidates.

謝謝布魯斯，也謝謝大家今天加入我們。過去幾個月對 Traws Pharma 來說是轉型期，也是非常正面的。由於我們團隊中每個人的出色執行力，我們已經大大推進了我們的臨床管道。雖然數據分析仍在進行中，但我們相信我們將對每種候選產品的初步藥物動力學、首選劑量計劃和安全性有一個很好的了解。

During the call today, our CFO, Mark Guerin, and I, we will summarize the latest progress with our clinical pipeline, including expected upcoming milestones. We will briefly review our financials and the transaction that created Traws Pharma and then also take your questions.

在今天的電話會議中，我們的財務長 Mark Guerin 和我將總結我們臨床管道的最新進展，包括預期即將到來的里程碑。我們將簡要回顧我們的財務狀況和創建 Traws Pharma 的交易，然後回答您的問題。

Traws Pharma's mission is to build solutions for important medical challenges and alleviate the burden of viral infections and cancer. In April, we completed the merger agreement that created Traws Pharma. This transaction expanded our investor base to include recognized healthcare investors, OrbiMed and Torrey Pines, and also broadens our portfolio.

Traws Pharma 的使命是為重要的醫療挑戰建立解決方案並減輕病毒感染和癌症的負擔。4 月，我們完成了創建 Traws Pharma 的合併協議。此次交易擴大了我們的投資者基礎，將知名醫療保健投資者 OrbiMed 和 Torrey Pines 納入其中，並擴大了我們的投資組合。

Our pipeline of potentially best-in-class oral small molecule medicines now includes antiviral therapies with two programs for serious respiratory infections, influenza, and COVID-19, as well as oncology with two agents that target essential steps in the cell cycle for solid tumor cancers.

我們潛在的同類最佳口服小分子藥物產品線現在包括抗病毒療法，其中有兩種治療嚴重呼吸道感染、流感和COVID-19 的方案，以及腫瘤學治療，有兩種針對實體瘤細胞週期中重要步驟的藥物癌症。

In the coming months, we look forward to completing and reporting the top line data from each of our ongoing clinical studies and to sharing plans for the initiation of Phase 2 studies in our antiviral program. At the same time, we are progressing our investigator-sponsored trials, or IST, strategy in oncology. We believe that these data could pave the way for key readouts in 2025.

在接下來的幾個月中，我們期待完成並報告我們正在進行的每項臨床研究的主要數據，並分享我們抗病毒計畫第二階段研究的啟動計畫。同時，我們正在推動研究者資助的試驗（IST）腫瘤學策略。我們相信這些數據可以為 2025 年的關鍵讀數鋪路。

I'm excited about our portfolio based on each candidates' differentiated and potential best-in-class profile. With that, I'd like briefly to take you through each of the programs, starting with our program for influenza, including avian or bird flu.

我對我們基於每位候選人的差異化和潛在的一流個人資料的投資組合感到興奮。接下來，我想簡單介紹每個項目，首先是我們的流感項目，包括禽流感項目。

Tivoxavir marboxil is our influenza candidate. It targets the influenza cap-dependent endonuclease, which is highly conserved across flu strains, including bird flu, making it a potential universal agent for flu. We believe that our compound may provide an important contribution to alleviate influenza, including seasonal and pandemic infections, both as a treatment as well as in certain prophylactic settings.

Tivoxavi marboxil 是我們的流感候選藥物。它針對流感帽依賴性核酸內切酶，該酶在包括禽流感在內的流感病毒株中高度保守，使其成為流感的潛在通用製劑。我們相信，我們的化合物可以為緩解流感（包括季節性和大流行感染）做出重要貢獻，無論是作為治療還是在某些預防環境中。

Preclinical data and top line results from single-ascending dose, or SAD, studies suggest that tivoxavir marboxil has the potential to achieve our target product profile as a single-dose treatment that is well-tolerated and active against influenza A or B, also known as pandemic-potential viruses, as well as against oseltamivir and baloxavir resistant viruses.

單劑量遞增（SAD）研究的臨床前數據和頂線結果表明，tivoxavir marboxil 作為單劑量治療藥物有潛力實現我們的目標產品特徵，該治療方法具有良好的耐受性，並且對甲型或乙型流感（也已知）具有活性作為潛在大流行病毒，以及針對奧司他韋和巴洛沙韋抗藥性病毒。

We are dosing the first cohorts in a Phase 1 extension study in Australia. This placebo-controlled trial is being conducted in healthy volunteers and is evaluating three tivoxavir marboxil doses, including two doses from the previous successful Phase 1 dose escalation study at 80 and 120 milligrams and one new increased dose, 240 milligrams. The study will assess pharmacokinetics, pharmacodynamics, and safety and is expected to define the dosing plan for a future Phase 2 study.

我們正在澳洲進行第一階段擴展研究，對第一批患者進行給藥。這項安慰劑對照試驗正在健康志願者中進行，正在評估三種tivoxavir marboxil 劑量，包括先前成功的1 期劑量遞增研究中的兩種劑量（80 毫克和120 毫克）和一種新增加的劑量（ 240 毫克）。該研究將評估藥物動力學、藥效學和安全性，並有望確定未來 2 期研究的給藥計畫。

Next steps for this program are to announce top line Phase 1 dose extension studies results from Australia expected in Q4 of this year and prepare for the initiation of a Phase 2 study expected to begin in Q4 of this year or Q1 of 2025.

該計畫的後續步驟是預計今年第四季公佈澳洲第一階段的劑量擴展研究結果，並為預計今年第四季或 2025 年第一季開始的第二階段研究做好準備。

Ratutrelvir is our COVID candidate. It targets Mpro, which is the 3CL protease. We believe that the major differentiator is that the compound does not require combination use of CYP-inhibitor, such as ritonavir. CYP-inhibitors are used to slow down the metabolism of the active compound as demonstrated by nirmatrelvir.

Ratutrelvir 是我們的新冠候選藥物。它的目標是 Mpro，即 3CL 蛋白酶。我們認為主要的差異在於該化合物不需要合併使用 CYP 抑制劑，例如利托那韋。CYP 抑制劑用於減緩活性化合物的代謝，如 nirmatrelvir 所證明的那樣。

But CYP-inhibitors can also result in potentially significant drug-drug interactions with other medications as can be observed in the older, more vulnerable patient populations. We are aiming for a 10-day once-a-day treatment. Clinical trial-enabling studies suggest that ratutrelvir should be well-tolerated and is active against COVID strains that may be resistant to approved agents such as nirmatrelvir and that includes Delta and Omicron strains.

但 CYP 抑制劑也可能導致與其他藥物潛在的顯著藥物交互作用，這可以在老年、較脆弱的患者群體中觀察到。我們的目標是每天一次，為期 10 天的治療。臨床試驗支持研究表明，ratutrelvir 應具有良好的耐受性，並且對可能對 nirmatrelvir 等已批准藥物產生抗藥性的 COVID 菌株具有活性，其中包括 Delta 和 Omicron 菌株。

We just completed dosing in a Phase 1 single-ascending dose, SAD, and multiple-ascending dose, MAD, study in healthy volunteers in Australia. The study SAD segment was designed to assess five doses ranging from 15 milligrams to 600 milligrams. And the MAD segment was designed to evaluate two dose levels, 150 milligram and 600 milligram, dosed daily for 10 days. We expect the study to provide pharmacokinetics, pharmacodynamics, and safety data that should define the dosing plan for an upcoming Phase 2 efficacy study.

我們剛在澳洲健康志願者中完成了 1 期單次遞增劑量 (SAD) 和多次遞增劑量 (MAD) 研究的給藥。SAD 研究部分旨在評估 15 毫克至 600 毫克之間的五種劑量。MAD 部分旨在評估兩種劑量等級：150 毫克和 600 毫克，每天給藥，持續 10 天。我們預計該研究將提供藥物動力學、藥效學和安全性數據，為即將進行的 2 期療效研究確定給藥計畫。

So the next step for this program are to announce top line results from Phase 1 SAD and MAD studies, and that is expected in Q4 of this year, and prepare for the initiation of a Phase 2 efficacy study expected to begin in Q4 of this year or Q1 of 2025.

因此，該計劃的下一步是公佈 1 期 SAD 和 MAD 研究的主要結果（預計在今年第四季度），並為啟動預計在今年第四季度開始的 2 期療效研究做好準備或2025 年第一季度。

Now switching gears, I'd like to provide you with a brief update on our oncology programs, starting with narazaciclib. Narazaciclib is a multi-kinase inhibitor targeting CDK4+ and other kinases with potential for use in multiple solid tumors.

現在換個主題，我想向您提供我們腫瘤學課程的簡短更新，從 narazaciclib 開始。Narazaciclib 是一種針對 CDK4+ 和其他激酶的多激酶抑制劑，有可能用於多種實體瘤。

We recently completed Phase 1/2 dose escalation studies for the compound, evaluated both as a monotherapy and in combination with letrozole in patients with recurrent metastatic low-grade endometrioid endometrial cancer and other gynecologic malignancies. The studies were designed to define the dose-limiting toxicity and maximal tolerated dose of the combination that should result in the recommended Phase 2 dose for further clinical trials.

我們最近完成了該化合物的 1/2 期劑量遞增研究，對複發性轉移性低級別子宮內膜樣子宮內膜癌和其他婦科惡性腫瘤患者作為單一療法和與來曲唑聯合療法進行了評估。這些研究旨在確定聯合用藥的劑量限制毒性和最大耐受劑量，從而得出建議的 2 期劑量用於進一步的臨床試驗。

The next steps for narazaciclib will utilize the investigator-sponsored trial, or IST, strategy. Upcoming milestones includes the release of top line data from the recently completed Phase 1/2 dose escalation studies at an upcoming medical meeting and identify the recommended Phase 2 dose and initiation of ISTs in multiple myeloma and breast cancer. And that's from the second half of this year and beyond.

narazaciclib 的下一步將利用研究者資助的試驗（IST）策略。即將到來的里程碑包括在即將舉行的醫學會議上發布最近完成的 1/2 期劑量遞增研究的主要數據，並確定建議的 2 期劑量和在多發性骨髓瘤和乳腺癌中啟動 IST。這是今年下半年及以後的情況。

Our second oncology candidate is rigosertib. Rigosertib is also a multi-kinase inhibitor targeting cell cycle proteins, including PLK-1, with potential use in the ultra-rare disease of advanced squamous cell carcinoma complicating recessive dystrophic epidermolysis bullosa. And that is also known as RDEB-associated SCC.

我們的第二個腫瘤學候選者是rigosertib。Rigosertib 也是一種針對細胞週期蛋白（包括 PLK-1）的多激酶抑制劑，可用於治療極為罕見的晚期鱗狀細胞癌併發隱性營養不良性大皰性表皮鬆解症。這也稱為 RDEB 相關 SCC。

We have been utilizing an IST strategy to develop rigosertib in this ultra-rare disease. Data from these studies have been presented at international medical meetings, including more recently at the Society of Investigative Dermatology held in July of this year, which highlighted ongoing studies conducted at the University Hospital in Salzburg, Austria and Thomas Jefferson University in Philadelphia. We have been encouraged by the ongoing investigator interest for rigosertib and support the IST-led RDEB program, including compassionate use filings in both US and other countries.

我們一直在利用 IST 策略來開發 rigosertib 來治療這種極為罕見的疾病。這些研究的數據已在國際醫學會議上公佈，包括最近在今年 7 月舉行的皮膚病研究學會上公佈，該會議重點介紹了奧地利薩爾茨堡大學醫院和費城托馬斯傑斐遜大學正在進行的研究。我們受到研究者對 rigosertib 持續興趣的鼓舞，並支持 IST 主導的 RDEB 計劃，包括在美國和其他國家/地區進行同情使用申請。

With that, I would like to turn it over to our CFO. Mark?

有了這個，我想把它交給我們的財務長。標記？

Thank you, Werner, and good morning, everyone. Traws' financial results for the quarter ended June 30, 2024, represents the first post-transaction quarterly report of the combined company. The press release issued this morning includes explanatory statements related to the financial results for this last quarter. I'll refer you to our recent 10-Q filing for a review of the full financial statements. You can also access the press release and 10-Q on the Investor Relations section of our website.

謝謝維爾納，大家早安。Traws 截至 2024 年 6 月 30 日的季度財務表現是合併後公司的第一份交易後季度報告。今天早上發布的新聞稿包括與上一季財務業績相關的解釋性聲明。我將向您推薦我們最近提交的 10-Q 報告，以審查完整的財務報表。您也可以在我們網站的投資者關係部分存取新聞稿和 10-Q。

Turning to our financials. The net loss that we reported for the quarter ended June 30, 2024, of $123.1 million, or $4.87 per basic and diluted common share, reflects a noncash charge of $117.5 million related to in-process R&D from Onconovas' April 2024 acquisition of Trawsfynydd. Traws Pharma closed the second quarter of 2024 with cash and equivalents of $16.9 million compared to $20.8 million as of December 31, 2023.

轉向我們的財務狀況。我們報告的截至2024 年6 月30 日的季度淨虧損為1.231 億美元，即每股基本和稀釋普通股4.87 美元，反映了Onconovas 2024 年4 月收購Trawsfynydd 時與正在進行的研發相關的1.175 億美元非現金費用。截至 2024 年第二季度，Traws Pharma 現金及等價物為 1,690 萬美元，截至 2023 年 12 月 31 日為 2,080 萬美元。

We continue to close attention to our spending level while progressing our compounds in the clinic to reach the milestones that Werner discussed earlier. Based on these efforts and our current projections, we believe our cash will be sufficient to fund our ongoing clinical trials and operations through yearend.

我們繼續密切關注我們的支出水平，同時在臨床上推進我們的化合物以達到沃納之前討論的里程碑。根據這些努力和我們目前的預測，我們相信我們的現金將足以資助我們年底正在進行的臨床試驗和營運。

Building further on Werner's comments, we believe the transaction and concurrent $14 million private placement meaningfully expanded our portfolio and enhanced the company's investor base with the addition of OrbiMed and Torrey Pines.

進一步根據 Werner 的評論，我們相信此次交易和同時進行的 1400 萬美元私募有意義地擴大了我們的投資組合，並透過 OrbiMed 和 Torrey Pines 的加入增強了公司的投資者基礎。

I would like to turn the call back to Werner.

我想把電話轉回維爾納。

Thanks, Mark. In closing, and with thanks to Mark and his team and thanks to our experienced product developments and clinical operations team, we believe that the progress we have made so far, this transformational year is completely consistent with our expectations and plan.

謝謝，馬克。最後，感謝馬克和他的團隊，感謝我們經驗豐富的產品開發和臨床營運團隊，我們相信，我們迄今為止所取得的進展，這一轉型的一年完全符合我們的期望和計劃。

Over the next six-plus months, we expect to advance our antivirals into Phase 2 studies and define the clinical strategy in oncology. We believe that each of our upcoming milestones has the potential to solidify the target product profile for each program.

在接下來的六個多月裡，我們預計將我們的抗病毒藥物推進到第二期研究，並確定腫瘤學的臨床策略。我們相信，即將到來的每個里程碑都有可能鞏固每個計劃的目標產品概況。

And again, starting with flu. For our single-dose compound, tivoxavir marboxil, we expect to announce top line Phase 1 dose extension results from Australia in Q4 of this year and initiate the Phase 2 study in Q4 of this year or Q1 of 2025. For COVID, our CYP-independent COVID compound, ratutrelvir, we expect to also announce top line results from our Phase 1 SAD and MAD studies in Australia in Q4 of this year and initiate the Phase 2 efficacy study in Q4 of this year or Q1 of 2025.

再次，從流感開始。對於我們的單劑量化合物 tivoxavir marboxil，我們預計將於今年第四季度公佈來自澳洲的頂級 1 期劑量擴展結果，並於今年第四季或 2025 年第一季啟動 2 期研究。對於新冠病毒，我們的CYP 獨立的新冠病毒化合物，ratutrelvir，我們預計將於今年第四季度公佈我們在澳大利亞進行的1 期SAD 和MAD 研究的主要結果，並於今年第四季度或2019 年第一季度啟動2 期療效研究。

In oncology, for narazaciclib, we intend to release the top line results from our Phase 1/2 study at an upcoming medical meeting. In addition, we plan to identify the recommended Phase 2 dose and initiate ISTs in multiple myeloma and breast cancer from the second half of this year onwards. For rigosertib, we plan to support the IST-led program, including compassionate use filings for patients with RDEB.

在腫瘤學方面，對於 narazaciclib，我們打算在即將舉行的醫學會議上發布 1/2 期研究的主要結果。此外，我們計劃從今年下半年開始確定建議的 2 期劑量，並啟動多發性骨髓瘤和乳癌的 IST。對於 rigosertib，我們計劃支持 IST 主導的項目，包括 RDEB 患者的同情使用申請。

We thank you for joining us today and look forward to upcoming -- to updating you on our continued progress. Before we open the call for questions, I would like to express our gratitude to the investigators, patients and their families and everyone at the study sites. Their contribution has been very important to the progress of our clinical research.

我們感謝您今天加入我們，並期待接下來的活動，向您介紹我們的持續進展。在我們開始提問之前，我想向研究人員、患者及其家屬以及研究地點的每個人表示感謝。他們的貢獻對我們臨床研究的進展非常重要。

With that, operator, we would like to begin the Q&A session. Please go ahead.

接線員，我們就這樣開始問答環節。請繼續。

(Operator Instructions) Ahu Demir, Ladenburg Thalmann.

（操作員說明）Ahu Demir，Ladenburg Thalmann。

Hello, this is Chong for Ahu Demir. Thank you for taking our question. We have a couple of questions. And the first one is regarding the influenza program. So we know we will expect to see a Phase 1 study in the fourth quarter of this year. Could you please give us some color on the details of the data, like how many patients will be on PK, PD, and the safety data?

大家好，我是 Ahu Demir 的 Chong。感謝您提出我們的問題。我們有幾個問題。第一個是關於流感計劃。所以我們知道我們預計將在今年第四季看到第一階段研究。您能否給我們一些關於資料細節的說明，例如有多少患者將接受 PK、PD 和安全性資料？

Yeah, thank you for your question. Indeed, we are currently dosing the first cohort in that human volunteer study. It's a Phase 1 study. It's a single dose as we have intended also for further therapeutic use and it's placebo-controlled, and I think a total of about 24 or 26 human volunteers will be included. That will complete the study as we have already available from a previous trial.

是的，謝謝你的提問。事實上，我們目前正在對人類志願者研究的第一批患者進行給藥。這是第一階段研究。這是單劑量，因為我們也打算用於進一步的治療用途，並且它是安慰劑對照的，我認為總共將包括大約 24 或 26 名人類誌願者。這將完成研究，因為我們已經從先前的試驗中獲得了這些資訊。

Then how about the Phase 2 study design, what population do you plan to target? And what is the timeline for the Phase 2? When do we expect to see the data?

那麼二期研究設計怎麼樣，計畫針對哪些族群？第二階段的時間表是什麼？我們預計什麼時候可以看到數據？

We are currently analyzing the Phase 1 data. And that will take still a little while. So before we have the full analysis of the Phase 1 data, I think it's premature to talk about the final design of our Phase 2 trial. And we will certainly report on that when those data are becoming available.

我們目前正在分析第一階段的數據。這還需要一段時間。因此，在我們對第一階段數據進行全面分析之前，我認為現在談論第二階段試驗的最終設計還為時過早。當這些數據可用時，我們肯定會報告這一點。

Great. And our next question is regarding the narazaciclib. So what is the status of the program in the endometrial? Like how many patients get involved? And will you plan to advance the nara in the endometrial Phase 2?

偉大的。我們的下一個問題是關於 narazaciclib。那麼該程序在子宮內膜中的狀況如何呢？例如有多少患者參與其中？您打算在子宮內膜第2階段推進奈拉嗎？

From the Phase 1/2 studies, the total patient numbers was, I think, approximately 35 or 40. Again, there, the analysis is ongoing. The endometrial is currently not our priority. And as explained, we will follow an IST strategy in multiple myeloma and breast with the investigator-supported studies.

從 1/2 期研究來看，我認為患者總數約為 35 或 40 人。同樣，分析仍在進行中。子宮內膜目前不是我們的首要任務。如所解釋的，我們將在研究者支持的研究中遵循多發性骨髓瘤和乳癌的 IST 策略。

So can you please give us a rationale, like, why we want to initiate the nara in the multiple myeloma and breast cancer? Did you see any early sign from the Phase 1 of the study?

那麼您能否給我們一個基本原理，例如為什麼我們要在多發性骨髓瘤和乳癌中啟動奈拉？您是否從該研究的第一階段中看到了任何早期跡象？

The profile, the pharmacological profile of the compound has generated high interest with the investigators in those two areas because of the multiple targets profile of the compound. And that was the motivation for them to propose such a study.

由於該化合物的多靶點概況，該化合物的藥理學概況引起了這兩個領域的研究人員的高度興趣。這就是他們提出這樣研究的動機。

Okay. Thank you. That's very helpful.

好的。謝謝。這非常有幫助。

(Operator Instructions) I'm showing no further questions in queue. Ladies and gentlemen, thank you for your participation on today's conference call. This concludes today's event. You may now disconnect.

（操作員說明）我在隊列中沒有顯示任何其他問題。女士們、先生們，感謝您參加今天的電話會議。今天的活動到此結束。您現在可以斷開連線。