TRACON Pharmaceuticals Inc (TCON) 2024 Q1 法說會逐字稿

Good day, ladies and gentlemen, and welcome to TRACON Pharmaceuticals first quarter 2024 earnings conference call. At this time, all callers are in a listen only mode. After the speakers' prepared remarks, we will conduct a question and answer session and instructions will be given at that time.

女士、先生們，大家好，歡迎參加 TRACON Pharmaceuticals 2024 年第一季財報電話會議。此時，所有來電者均處於只聽模式。演講者發言結束後，我們將進行問答環節，屆時會給予相關說明。

During today's call, we will be making certain forward-looking statements, including statements regarding expected timing of clinical trials and results, regulatory activities, plans for future clinical trials, financing opportunities, our development plans and strategies, potential cost savings and other benefits deliverable through our product development platform or PDP, ability to enter into additional licensing agreements, ability to generate non-dilutive capital using the PDP, market size estimates, and whether the company's stock will remain listed on NASDAQ.

在今天的電話會議上，我們將做出一些前瞻性聲明，包括有關臨床試驗和結果的預期時間、監管活動、未來臨床試驗計劃、融資機會、我們的發展計劃和戰略、透過我們的產品開發平台或PDP可實現的潛在成本節約和其他收益、簽訂額外許可協議的能力、利用PDP產生非稀釋性資本的能力、市場規模估計以及公司股票是否會繼續在達克的聲明公司中上市的聲明。

These statements are subject to various risks that are described in our filings made with the Securities Exchange Commission, including our Annual Report on Form 10-K for the year ended December 31, 2023 and subsequent Quarterly Reports on Form 10-Q. You are cautioned not to place undue reliance on these forward-looking statements and unless required by applicable law, we disclaim any obligation to update such statements.

這些聲明受到各種風險的影響，這些風險已在我們的文件中進行了描述，包括截至 2023 年 12 月 31 日止年度的 10-K 表格年度報告以及隨後的 10-Q 表格季度報告。請注意，不要過度依賴這些前瞻性陳述，除非適用法律要求，否則我們不承擔更新此類陳述的任何義務。

Now I would like to turn the call over to Dr. Charles Theuer, President and CEO of TRACON Pharmaceuticals. Dr. Theuer?

現在，我想把電話交給 TRACON Pharmaceuticals 的總裁兼執行長 Charles Theuer 博士。Theuer 博士？

Good afternoon and thank you for joining TRACON's first quarter 2024 financial results and business update call. I will begin with an update on our pipeline and then review our recent activities. Following that, Scott Brown, our Chief Financial Officer, will discuss our financial results for the three months ended March 31, 2024. Finally, we will conclude by taking your questions.

下午好，感謝各位參加TRACON 2024年第一季財務業績及業務更新電話會議。我將首先報告我們的產品線進度情況，然後回顧我們最近的活動。接下來，我們的財務長史考特布朗將討論截至 2024 年 3 月 31 日止三個月的財務表現。最後，我們將以回答大家的問題作為結尾。

I will begin with an update on our continued progress with the ongoing Phase 2 ENVASARC pivotal trial. In April, the Independent Data Monitoring Committee recommended the trial continue as planned following a review of interim safety and efficacy data from 73 patients in Cohort C of single agent enva treatment.

首先，我將報告我們正在進行的 ENVASARC 二期關鍵性試驗的最新進展。4 月，獨立數據監測委員會在審查了 C 組 73 名接受單藥 enva 治療的患者的中期安全性和有效性數據後，建議試驗按計劃繼續進行。

The objective response rate in patients treated with single-agent enva was 11% by investigator review and 5.5% by blinded independent central review or four responses. Enva monotherapy was generally well-tolerated without a single drug-related serious adverse event.

單藥恩瓦治療患者的客觀緩解率，經研究者評估為 11%，經盲法獨立中心評估為 5.5% 或 4 次緩解。Enva 單藥治療整體耐受性良好，未發生一例與藥物相關的嚴重不良事件。

Importantly, median duration response by independent central review was greater than six months. We have completed accrual of 82 patients dosed with enva as a single agent at 600 milligrams and expect to report final data in the third quarter.

值得注意的是，經獨立中心審查，中位數持續時間反應超過六個月。我們已經完成了 82 名接受 600 毫克單藥恩伐他汀治療的患者的招募工作，預計將在第三季公佈最終數據。

As a reminder, in order to statistically exceed the 4% objective response rate of Votrient, the only FDA approved treatment for patients with refractory UPS or MFS, the primary endpoint in ENVARSAC must show objective responses in 9 out of 82 patients or 11% objective response rate confirmed by independent central review.

提醒一下，為了在統計學上超過 Votrient 4% 的客觀緩解率（Votrient 是唯一獲得 FDA 批准用於治療難治性 UPS 或 MFS 患者的藥物），ENVARSAC 的主要終點必須顯示 82 名患者中有 9 名出現客觀緩解，或客觀緩解率達到 11%，並經獨立中心審查確認。

Median duration of response of greater than six months is a key secondary endpoint. Our goal in ENVARSAC is to demonstrate that enva has the potential to be both safer and more efficacious than Votrient, a drug with a black box warning for fatal liver toxicity.

中位反應持續時間超過六個月是一項關鍵的次要終點。我們在 ENVARSAC 中的目標是證明 enva 有可能比 Votrient 更安全、更有效，Votrient 是一種帶有致命肝毒性黑框警告的藥物。

We plan to approach the FDA to discuss a BLA filing strategy if we determine nine responses by independent central review. As a reminder, we have received fast-track designation for enva in the sarcoma subtypes of UPS and MFS that have progressed on one or two prior lines of therapy and received orphan drug designation in soft tissue sarcoma based on activity observed in ENVASARC. These designations provide important advantages that might expedite regulatory review of enva.

如果透過獨立中央審查確定有九份回复，我們將與 FDA 接洽，討論生物製品許可申請策略。提醒一下，我們已獲得 enva 在 UPS 和 MFS 肉瘤亞型中的快速通道資格，這些亞型在一線或兩線治療後病情進展；並且基於 ENVASARC 中觀察到的活性，我們獲得了軟組織肉瘤孤兒藥資格。這些認定具有重要優勢，可能會加快對 Enva 的監管審查。

ENVASARC is designed to provide safety and efficacy data in the refractory sarcoma subtypes of UPS and MFS. We also have a strategy to pursue the approval of enva in front-line sarcoma. Doxorubicin is the most commonly approved therapy used for the treatment of newly diagnosed sarcoma patients. We therefore, plan to initiate a trial of enva and doxorubicin in the frontline setting of the common sarcoma subtypes, including UPS and MFS following the completion of enrollment in the pivotal ENVASARC trial and prior to the expected BLA submission subject to positive results from ENVASARC, including achieving the primary endpoint.

ENVASARC 旨在提供 UPS 和 MFS 這兩種難治性肉瘤亞型的安全性和有效性資料。我們也制定了策略，爭取讓恩瓦（enva）獲準用於第一線肉瘤治療。多柔比星是目前最常用的核准用於治療新診斷肉瘤患者的療法。因此，我們計劃在關鍵性 ENVASARC 試驗完成入組後，並在預期 BLA 提交之前，啟動一項針對常見肉瘤亞型（包括 UPS 和 MFS）一線治療的 enva 和阿黴素試驗，前提是 ENVASARC 試驗取得積極結果，包括達到主要終點。

The goal of that trial will be to determine the subtypes of sarcoma that best respond to the combination of enva and doxorubicin. Assuming positive results in the ENVASARC pivotal trial, we expect the FDA will require a randomized trial to demonstrate a survival benefit. We expect this potential Phase 3 post-approval trial will compare single agent doxorubicin to doxorubicin with enva with progression-free survival as the endpoint. This trial would be expected to enroll patients with UPS and MFS as well as other sarcoma subtypes expected to respond to therapy with enva and doxorubicin.

該試驗的目標是確定哪些亞型的肉瘤對恩維拉和阿黴素聯合治療的反應最好。假設 ENVASARC 關鍵性試驗取得正面結果，我們預期 FDA 將要求進行隨機試驗以證明存活獲益。我們預期這項潛在的 3 期上市後試驗將比較單藥多柔比星與多柔比星聯合恩維司群，以無惡化存活期為終點。預計該試驗將招募患有 UPS 和 MFS 以及其他預期會對恩維拉和阿黴素治療產生反應的肉瘤亞型的患者。

We expect to discuss the design of a front-line trial with the FDA at the time of a pre-BLA meeting to review the expected submission of data from ENVASARC for potential accelerated approval of enva in refractory sarcoma, assuming positive results from the ENVASARC pivotal trial. It is important to understand the sales potential in sarcoma with enva at parity pricing is not solely the forecasted $200 million in peak annual enva revenues anticipated if approved in refractory UPS and MFS. Our clinical development strategy is designed to create the opportunity for enva to broadly benefit patients with sarcoma in the frontline, adjuvant and neoadjuvant settings by seeking supplemental BLAs.

我們預計在 BLA 前會議期間與 FDA 討論一線試驗的設計，以審查 ENVASARC 預期提交的數據，從而在 ENVASARC 關鍵性試驗取得積極結果的情況下，加速批准 enva 用於治療難治性肉瘤。重要的是要了解，如果恩維他（Enva）在難治性UPS和MFS中獲批，其在肉瘤領域的銷售潛力，不僅僅是預計的2億美元年度高峰收入。我們的臨床開發策略旨在透過尋求補充生物製品許可申請 (BLA)，為 Enva 創造機會，使其能夠在初級、輔助和新輔助治療中廣泛惠及肉瘤患者。

We will now turn to our DNA damage repair inhibitor, TRC102, that is financially supported through a cooperative research and development agreement with the National Cancer Institute. The NCI is sponsoring an ongoing randomized Phase 2 trial assessing TRC102 in stage three non-squamous non-small cell lung cancer in combination with chemo radiation.

接下來，我們將介紹我們的 DNA 損傷修復抑制劑 TRC102，該抑制劑透過與美國國家癌症研究所的合作研發協議獲得資金支持。美國國家癌症研究所 (NCI) 正在資助一項正在進行的隨機 2 期試驗，評估 TRC102 與化療和放療聯合用於治療第三期非鱗狀非小細胞肺癌的療效。

The two arm trial will enroll 78 patients to assess the benefit of adding TRC102 to current standard of care treatment of pemetrexed, cisplatin and radiation therapy, followed by consolidative durvalumab maintenance treatment. The primary endpoint of the trial is progression-free survival, and the trial is designed to detect an improvement in PFS at one year from 56% to 75%. 15 sites are now open for enrollment in the US and final results are expected in 2025.

這項雙臂試驗將招募 78 名患者，以評估在培美曲塞、順鉑和放射療法的當前標準治療方案中加入 TRC102，然後進行鞏固性度伐利尤單抗維持治療的益處。該試驗的主要終點是無惡化存活期，旨在檢測一年後無惡化存活率從56%提高到75%。目前美國有15個研究中心正在招募患者，最終結果預計2025年公佈。

I will now shift from our pipeline update to discuss our product development platform or PDP of CRO independent research. We executed a license or a PDP for an upfront payment of $3 million in November of last year to a biotech company that recognize the value of internalizing its clinical operations to reap the benefits of CRO independent clinical trial implementation that we enjoy at TRACON. The license of the PDP is expected to allow them to run clinical trials as we do at TRACON for an estimated cost of approximately $100,000 per patient for oncology trials.

接下來，我將從我們的產品線更新轉向討論我們的產品開發平台（PDP），即獨立於 CRO 的研究。去年 11 月，我們與一家生技公司簽訂了一份授權協議或產品開發計畫 (PDP)，預付金額為 300 萬美元。該公司認識到將臨床營運內部化的價值，從而能夠像我們在 TRACON 一樣，獲得獨立於 CRO 的臨床試驗實施所帶來的好處。PDP 的許可證預計將允許他們像我們在 TRACON 一樣進行臨床試驗，腫瘤試驗的估計費用約為每位患者 10 萬美元。

As a typical CRO charges $300,000 or more per patient in this indication, the potential savings from licensing our PDP on a 100 patient trial could be up to approximately $20 million for a partner in addition to expected advantages of increased speed of trial execution and pace of enrollment that we enjoy at TRACON by running trials using our in-house team.

由於典型的 CRO 在此適應症中對每位患者收取 30 萬美元或更多費用，因此，對於合作夥伴而言，授權我們的 PDP 進行 100 例患者的試驗，除了我們 TRACON 通過使用內部團隊開展試驗所享有的試驗執行速度加快和入組速度加快等預期優勢外，還可能節省高達約 2000 萬美元。

As we have noted in the past, we expect to further supplement our cash position through opportunities for non-dilutive capital enabled through our CRO independent PDP that we believe positions us as one of the most efficient clinical development organizations.

正如我們過去所指出的，我們希望透過我們獨立的 CRO PDP 提供的非稀釋性資本機會，進一步補充我們的現金儲備。我們相信，這將使我們成為最高效的臨床開發組織之一。

We expect to continue to leverage our platform in two ways that provide potential non-dilutive capital to TRACON. First, we plan to continue to evaluate drug candidates whereby TRACON captures revenue by performing clinical trials at a lower fixed cost compared to a CRO, but still at a premium to our costs using a pay-for-performance model. This is an aligned structure we used in the past, for example, with Johnson & Johnson.

我們預計將繼續以兩種方式利用我們的平台，為 TRACON 提供潛在的非稀釋性資本。首先，我們計劃繼續評估候選藥物，TRACON 透過進行臨床試驗來獲取收入，與 CRO 相比，其固定成本較低，但仍高於我們採用按績效付費模式的成本。這是我們過去曾經使用過的合作模式，例如與強生公司合作時就曾經採用過。

Second, we plan to continue to execute non transferable licenses to our PDP, whereby we are paid to share our proprietary capabilities and know-how to enable another company's independently internalized clinical operations and use these new capabilities to avoid contracting with CROs to execute clinical trials. As has been the experience at TRACON, we believe such an investment to result in substantial time and cost savings for our partner.

其次，我們計劃繼續執行不可轉讓的許可協議，向我們的 PDP 提供許可，透過分享我們的專有能力和專業知識，我們獲得報酬，以支持另一家公司獨立開展內部臨床運營，並利用這些新能力避免與 CRO 簽訂合約來執行臨床試驗。根據 TRACON 的經驗，我們相信這樣的投資將為我們的合作夥伴節省大量的時間和成本。

We believe that overtime our PDP has earned strong credibility as a compelling solution for companies who wish to become CRO independent and reap the rewards of conducting trials faster at higher quality and at lower cost compared to trials typically contracted to CROs.

我們相信，隨著時間的推移，我們的PDP已經贏得了強大的信譽，成為希望擺脫CRO依賴、以更快的速度、更高的品質和更低的成本開展試驗的公司的理想解決方案，而這些試驗通常外包給CRO。

As previously announced, on March 7, 2024, we had a hearing with Nasdaq regarding a letter of noncompliance that we received on June 8, 2023 regarding two deficiencies. We presented a compliance plan to cure our deficiencies to Nasdaq at the hearing on March 7.

正如先前宣布的那樣，2024 年 3 月 7 日，我們與納斯達克舉行了聽證會，討論我們於 2023 年 6 月 8 日收到的關於兩項缺陷的不合規信函。我們在 3 月 7 日的聽證會上向納斯達克提交了一份合規計劃，以彌補我們的不足。

On March 20, the Nasdaq hearings panel granted our request for continued listing on the Nasdaq capital market, subject to us regaining compliance with all applicable continued listing requirements. We effected a reverse stock split on April 9 to cure one of the Nasdaq deficiencies and are considering alternatives to cure the other Nasdaq deficiency, which requires us to have a market value for our listed securities of at least $35 million or meet the stockholders' equity requirement of $2.5 million by June 3. At this time, Scott will provide an update on our financials.

3月20日，納斯達克聽證小組批准了我們繼續在納斯達克資本市場上市的請求，但前提是我們必須重新符合所有適用的繼續上市要求。我們於 4 月 9 日進行了反向股票分割，以彌補納斯達克的一項不足，目前正在考慮其他方法來彌補納斯達克的另一項不足，即到 6 月 3 日，我們上市證券的市值必須至少達到 3500 萬美元，或者股東權益必須達到 250 萬美元。屆時，斯科特將向我們報告財務狀況。

Thank you, Charles, and good afternoon, everyone. TRACON's research and development expenses were $1.9 million for the three months ended March 31, 2024 compared to $5 million for the comparable period of 2023. The decrease was due to termination of cohort D of the ENVASARC pivotal trial in 2023.

謝謝你，查爾斯，大家下午好。截至 2024 年 3 月 31 日止的三個月，TRACON 的研發費用為 190 萬美元，而 2023 年同期為 500 萬美元。下降的原因是 ENVASARC 關鍵性試驗的 D 組於 2023 年終止。

General and administrative expenses were $1.4 million for the three months ended March 31, 2024, compared to $2.3 million for the comparable period of 2023. The decrease was due to lower legal expenses. Our net loss was $3.2 million for the three months ended March 31, 2024 compared to $8.5 million for the comparable period of 2023.

截至 2024 年 3 月 31 日止三個月的一般及行政費用為 140 萬美元，而 2023 年同期為 230 萬美元。下降的原因是法律費用降低。截至 2024 年 3 月 31 日止的三個月，我們的淨虧損為 320 萬美元，而 2023 年同期淨虧損為 850 萬美元。

Turning to the balance sheet, at March 31, 2024, our cash, cash equivalents and restricted cash totaled $8 million compared to $8.6 million at December 31, 2023.

從資產負債表來看，截至 2024 年 3 月 31 日，我們的現金、現金等價物和受限現金總額為 800 萬美元，而截至 2023 年 12 月 31 日為 860 萬美元。

With that, I will turn the call back over to Charles.

這樣，我就把電話轉回查爾斯了。

Thank you, Scott. As you have heard, our corporate strategy is proceeding as planned. Allow me to recap two key expected events. First, in the third quarter, we expect to report final data from the ENVASARC pivotal trial. Second, we expect to continue to leverage our product development platform to generate non-dilutive capital through either an additional license or by capturing revenue by replacing a CRO and executing clinical trials for partners at a lower cost compared to a CRO, but still at a premium to our costs using a pay-for-performance model.

謝謝你，斯科特。正如你們所聽到的，我們的公司策略正在按計劃進行。請容許我回顧一下兩個關鍵的預期事件。首先，我們預計在第三季公佈 ENVASARC 關鍵性試驗的最終數據。其次，我們期望繼續利用我們的產品開發平台，透過獲得額外許可或透過取代 CRO 並為合作夥伴執行臨床試驗來創造非稀釋性資本。與 CRO 相比，我們的成本更低，但仍會高於我們的成本，並採用績效付費的模式。

Thank you for your time and attention and we are now available to answer your questions.

感謝您抽出寶貴時間關注，我們現在可以回答您的問題了。

Thank you. (Operator Instructions) James Molloy, Alliance Global Partners.

謝謝。（操作說明）James Molloy，Alliance Global Partners。

Hi, guys. This is Matt Vinnytsia on for Jim Molloy. I just had a couple of questions. First, regarding the PDP. Can you go over a little bit more about the margins you would expect on a clinical trial that you guys would run through PDP for you guys and for your client?

嗨，大家好。這裡是馬特·文尼西亞，他代替吉姆·莫洛伊為您報道。我還有幾個問題。首先，關於人民民主黨。您能否再詳細介紹一下，您透過PDP為貴公司和您的客戶所進行的臨床試驗中，預期利潤率為何？

Appreciate the question. Thanks so much. I can give an example that serves as a precedent case. For example, we have run in the past the Phase 1 trial for I-Mab, whereby we were paid $9 million to conduct a clinical trial, again, a Phase 1 trial. Our total expense of that trial was a bit under $3 million. So in other words, we can run a trial at roughly $100,000 a patient in oncology. We know CROs are bidding those trials at roughly $300,000 per patient.

感謝您的提問。非常感謝。我可以舉一個可以當先例的例子。例如，我們過去曾進行過 I-Mab 的 1 期試驗，為此我們獲得了 900 萬美元的報酬，進行一項臨床試驗，這同樣是一項 1 期試驗。那次試驗的總費用略低於 300 萬美元。換句話說，我們可以在腫瘤科進行一項試驗，每位患者的費用約為 10 萬美元。我們知道，合約研究組織 (CRO) 對這些試驗的投標價格約為每位患者 30 萬美元。

So for example, for a 30 patient trial, we can do it at $3 million, which is $100,000 per patient. And we can guarantee the total cost of the trial to a partner at $300,000 a patient, which for 30 patients is about $9 million. It's a benefit to our partner because that's a guaranteed price. It won't go up, which, based on change orders, [to] see our bid price of $300,000 a patient, could go up substantially beyond that. We'll guarantee a $300,000 a patient knowing the profit margin there is about 200% for us.

例如，對於一項 30 名患者的試驗，我們可以花費 300 萬美元，即每位患者 10 萬美元。我們可以向合作夥伴保證，試驗的總成本為每位患者 30 萬美元，30 名患者的總成本約為 900 萬美元。這對我們的合作夥伴有利，因為這是一個有保障的價格。價格不會上漲，但根據變更訂單來看，我們為每位患者開出的報價為 30 萬美元，實際價格可能會大幅上漲。我們保證每位患者能獲得 30 萬美元，因為我們知道從中可以獲得約 200% 的利潤。

Got it. All right. So 200%. Thank you. And then could you go over the potential solutions in terms of regaining compliance with the minimums by early June, I think you had talked about, and how that would align with the timeline for the final ENVASARC data?

知道了。好的。所以是200%。謝謝。那麼，您能否再談談在 6 月初之前重新達到最低標準的潛在解決方案（我想您之前也談過），以及這將如何與 ENVASARC 最終數據的時間表相一致？

Yeah. Great question. So with respect to the Nasdaq compliance to do one or two things, either increase the market cap to $35 million or regain compliance through increasing the stockholder equity to $2.5 million, our preferred approach is to leverage the PDP that if we can leverage the PDP and gain revenue by, for instance, as you just pointed out, doing a trial for someone else, including a significant upfront payment to do that or by licensing the PDP as we did in November, that's the preferred option for us to have capital come in the company that would potentially cure the stockholder equity deficit. Other options would potentially be fundraising as well, but our preferred approach clearly is leveraging the PDP through business development.

是的。問得好。因此，關於納斯達克要求我們做一兩件事才能符合規定，要么將市值提高到 3500 萬美元，要么通過將股東權益提高到 250 萬美元來重新獲得合規性，我們更傾向於利用 PDP。如果我們能夠利用 PDP 並獲得收入，例如，正如您剛才指出的那樣，為其他人進行試用，包括為此支付一大筆預付款，或者像我們在 11 月份那樣通過授權 PDP 來實現，那麼這將是我們獲得公司資金的首選方案，這有可能彌補股東權益的不足。其他選擇可能包括籌款，但我們更傾向於透過業務發展來利用 PDP。

Got it. Okay. And can you go over how that would align with the timeline for ENVASARC? And what --? Sorry.

知道了。好的。您能否詳細說明這與 ENVASARC 的時間安排是如何連結的？還有什麼——？對不起。

No, appreciable the question. Sorry. So ENVASARC data, we're expecting final data in Q3. So that's just past June, it's when Q3 starts. We haven't given a more definitive time within Q3. But I would expect it to be just after the Nasdaq compliance date of June 3.

不，這個問題很值得思考。對不起。因此，ENVASARC 數據的最終數據預計將在第三季公佈。所以那時剛過六月，第三季就開始了。我們尚未給出第三季度更確切的時間。但我預計會在納斯達克合規日期 6 月 3 日之後不久。

Thank you. (Operator Instructions) So then I'll hand the call back over to CEO and President, Dr. Charles Theuer, for any closing remarks.

謝謝。（操作員指示）那麼，接下來我將把電話轉回給執行長兼總裁查爾斯·圖爾博士，請他作總結發言。

I'd like to thank the audience for your time and attention and your questions and we look forward to talking with you again next quarter.

感謝各位聽眾抽出時間聆聽並提出問題，我們期待下個季度再次與大家交流。

Ladies and gentlemen, thank you for participating. This does conclude today's program, and you may now disconnect.

女士們、先生們，感謝各位的參與。今天的節目到此結束，您可以斷開連線了。