Bio Path Holdings Inc (BPTH) 2024 Q2 法說會逐字稿

Good morning, ladies and gentlemen. Welcome to the Bio-Path Holdings second-quarter 2024 earnings conference call. (Operator Instructions)

早安，女士們先生們。歡迎參加 Bio-Path Holdings 2024 年第二季財報電話會議。（操作員說明）

At this time, I'd like to turn the floor over to Will O'Connor of Stern Investor Relations. Please proceed.

現在，我想請史特恩投資者關係部的威爾·奧康納發言。請繼續。

Thank you, operator. Welcome to the Bio-Path Holdings conference call and webcast to review the company's second-quarter 2024 financial results and to provide an update on recent pipeline and corporate developments. Earlier we issued a press release, which outlines the topics that we plan to discuss on today's call. The release is available at biopathholdings.com.

謝謝你，接線生。歡迎參加 Bio-Path Holdings 電話會議和網路廣播，回顧該公司 2024 年第二季的財務業績，並提供有關最新產品線和公司發展的最新資訊。早些時候，我們發布了一份新聞稿，概述了我們計劃在今天的電話會議上討論的主題。此版本可在 biopathholdings.com 上取得。

With me today from Bio-Path are President and CEO, Peter Nielsen; and Senior Vice President of Finance, Accounting and Administration, Anthony Price. Before I begin the call, I'd like to remind you that today's discussion will contain forward-looking statements that involve risks and uncertainties. These risks and uncertainties are outlined in today's press release and in the company's recent filings with the Securities and Exchange Commission, which we urge you to read. Our actual results may differ materially from what is discussed on today's call.

今天和我在一起的有 Bio-Path 總裁兼執行長 Peter Nielsen；財務、會計和行政高級副總裁 Anthony Price。在開始通話之前，我想提醒您，今天的討論將包含涉及風險和不確定性的前瞻性陳述。這些風險和不確定性在今天的新聞稿和公司最近向美國證券交易委員會提交的文件中進行了概述，我們強烈建議您閱讀這些文件。我們的實際結果可能與今天電話會議中討論的內容有重大差異。

With that, I'll now turn the call over to Bio Path's CEO, Peter Nielsen.

現在，我將電話轉給 Bio Path 的執行長 Peter Nielsen。

Thanks, Will. Good morning, everyone and thank you for joining us. The first half of 2024 was an undeniably productive period for Bio Path. I'm extremely proud of our team, which continues to efficiently operate on all cylinders and has executed our strategy to achieve both clinical and corporate objectives. I'll begin this morning with the progress we have made with our lead product candidate, prexigebersen.

謝謝，威爾。大家早安，感謝您加入我們。不可否認，2024 年上半年是 Bio Path 的豐產期。我為我們的團隊感到非常自豪，他們繼續高效運作，並執行我們的策略以實現臨床和企業目標。今天早上我將首先介紹我們的主要候選產品 prexigebersen 所取得的進展。

In June, we were honored to have a presence at two of the most prestigious medical meetings in the field of oncology. The American Society of Clinical Oncology or ASCO Annual Meeting in Chicago and the European Hematology Association, or EHA Congress in Madrid. At both meetings, we showcased interim results from our Phase 2 study of prexigebersen in combination with frontline therapy, decitabine and venetoclax for the treatment of acute myeloid leukemia, or AML.

六月，我們很榮幸參加了腫瘤學領域兩個最負盛名的醫學會議。在芝加哥舉行的美國臨床腫瘤學會 (ASCO) 年會和在馬德里舉行的歐洲血液學會 (EHA) 大會。在這兩次會議上，我們展示了 prexigebersen 聯合一線療法、地西他濱和 Venetoclax 治療急性髓系白血病 (AML) 的 2 期研究的中期結果。

At ASCO, we were recognized with an oral presentation of our results. As you know, the study is an amended Stage 2 of our Phase 2 trial in AML. It is an open-label two-stage multi-center study of prexigebersen in combination with decitabine and venetoclax in two cohorts of patients with previously untreated AML and refractory relapsed AML.

在 ASCO，我們透過口頭介紹我們的研究結果而獲得認可。如您所知，該研究是我們 AML 第二階段試驗的第二階段修訂版。這是一項開放標籤、兩階段多中心研究，在兩組既往未經治療的 AML 和難治性復發性 AML 患者中，將 prexigebersen 聯合地西他濱和 Venetoclax 進行研究。

The third cohort includes the treatment of refractory relapsed AML patients who are venetoclax resistant or intolerant with the two-drug combination of prexigebersen and decitabine. The primary endpoint for this study will be the number of patients who achieve complete remission, which includes complete remission with incomplete hematologic recovery, and complete remission with partial hematologic recovery.

第三組包括治療對 Venetoclax 有抗藥性或不耐受 Prexigebersen 和地西他濱兩種藥物組合的難治性復發性 AML 患者。本研究的主要終點是實現完全緩解的患者數量，包括完全緩解但血液學不完全恢復，以及完全緩解但部分血液學恢復。

The interim efficacy data presented at ASCO and EHA were from the initial interim analysis of Cohort 1 and Cohort 2. These compelling data showed that prexigebersen-based combination therapy was not only safely administered in cohort 1 and cohort 2 to high-risk newly diagnosed, and refractory relapsed AML patients considered unsuitable for standard chemotherapy, but also demonstrated efficacy signals better than current therapies.

ASCO 和 EHA 上提供的中期療效數據來自隊列 1 和隊列 2 的初始中期分析。這些令人信服的數據表明，基於prexigebersen 的聯合療法不僅可以在隊列1 和隊列2 中安全地用於被認為不適合標準化療的高風險新診斷和難治性復發AML 患者，而且還顯示出比目前療法更好的療效訊號。

This is particularly encouraging as refractory relapse patients are a challenging population in which current treatment options are suboptimal. It was a pleasure to present these data to an audience of world-leading oncologists, who treat AML patients and understand the continued great need for new therapeutic options. During the second quarter we also announced the exciting development of a molecular biomarker package to accompany prexigebersen treatment.

這是特別令人鼓舞的，因為難治性復發患者是一個具有挑戰性的族群，目前的治療方案並不理想。我們很高興向世界領先的腫瘤學家展示這些數據，他們治療 AML 患者並了解對新治療方案的持續巨大需求。在第二季度，我們還宣布了令人興奮的分子生物標記包的開發，以配合 prexigebersen 治療。

The goal here is to identify patients with a genetic profile more likely to respond to treatment thereby improving the probability of success for this program. The emerging role of biomarkers has enhanced cancer treatment development over the past decade and has become a more common companion to many oncology programs. We will keep you up-to-date as this cutting-edge project advances. Turning now to BP1002 program, which targets Bcl-2.

這裡的目標是識別具有更可能對治療產生反應的基因譜的患者，從而提高該計劃的成功機率。生物標記的新興作用在過去十年中促進了癌症治療的發展，並已成為許多腫瘤學計畫更常見的伴侶。隨著這個尖端專案的進展，我們將隨時向您通報最新情況。現在轉向 BP1002 計劃，該計劃的目標是 Bcl-2。

As you know, Bcl-2 is responsible for driving cell survival and up to 60% of all cancers. High expression of Bcl-2 has been correlated with poor prognosis for patients diagnosed with AML. Venetoclax has shown activity against the antiapoptotic protein Bcl-2 and works by neutralizing the protein's BH3 domain. It is an improved treatment for chronic lymphocytic leukemia, or CLL patients and untreated AML patients. However with the exception of some patients treated with allogeneic hematopoietic cell transplantation, disease relapse invariably occurs oftentimes due to BH3 domain mutation over time.

如您所知，Bcl-2 負責促進細胞存活和高達 60% 的癌症。Bcl-2 的高表現與診斷為 AML 的患者預後不良相關。Venetoclax 已顯示出抗凋亡蛋白 Bcl-2 的活性，並透過中和該蛋白的 BH3 結構域發揮作用。它是對慢性淋巴細胞白血病或 CLL 患者和未經治療的 AML 患者的改進治療方法。然而，除部分接受異體造血細胞移植治療的患者外，由於BH3結構域隨著時間的推移發生突變，疾病常常會復發。

BP1002 also targets the BCL-2 protein. However, BP1002 activity is based on blocking the Bcl-2 messenger RNA and not the BH3 domain. As a result, we believe that BP1002 could provide an alternative for venetoclax patients who have relapsed, including AML patients who previously received venetoclax treatments.

BP1002 也針對 BCL-2 蛋白。然而，BP1002 的活性是基於阻斷 Bcl-2 信使 RNA，而不是 BH3 結構域。因此，我們相信 BP1002 可以為復發的 Venetoclax 患者提供替代方案，包括先前接受 Venetoclax 治療的 AML 患者。

During the second quarter, we announced completion of the second dose cohort for the dose escalation portion of our Phase I/Ib clinical trial of BP1002 to treat relapsed -- refractory relapsed AML, including venetoclax resistant patients.

在第二季度，我們宣布完成了 BP1002 的 I/Ib 期臨床試驗的劑量遞增部分的第二個劑量隊列，以治療復發性難治性復發 AML，包括 Venetoclax 抗藥性患者。

A total of three value patients per dosing cohort are scheduled to be treated with BP1002 monotherapy in a standard three-plus-three design. Unless there is a dose-limiting toxicity, which would require an additional three patients tested. The first dose cohort consisted of a starting dose of 20-milligram per square meter and there is no dose-limiting toxicities.

每個給藥隊列總共有三名有價值的患者計劃按照標準的三加三設計接受 BP1002 單藥治療。除非有劑量限制性毒性，否則需要另外三名患者進行測試。第一個劑量組的起始劑量為每平方公尺 20 毫克，沒有劑量限制性毒性。

The approved treatment cycle is two doses per week, over four weeks for a total of eight doses administered over 28 days. The Phase Ib portion of the study is expected to commence after completion of BP1002 monotherapy cohorts, and will assess the safety and efficacy of BP1002 in combination with decitabine in refractory relapsed AML patients. We look forward to keeping you apprised of our progress here.

核准的治療週期為每週兩劑，為期四週，總共在 28 天內注射八劑。研究的 Ib 期部分預計將在 BP1002 單藥治療組完成後開始，並將評估 BP1002 與地西他濱聯合治療難治性復發性 AML 患者的安全性和有效性。我們期待隨時向您通報我們的進展。

Next, let's turn to our Phase I/Ib clinical trial of BP1001-A in patients with solid tumors, including ovarian, endometrial, pancreatic and triple negative breast cancer. Some of the most challenging cancers to treat with today's therapeutic toolkit. BP1001-A is a modified product from prexigebersen sharing the same drug substance with enhanced nanoparticle properties. This trial is being conducted at several leading cancer centers and will initially evaluate the safety in solid tumor patients.

接下來，讓我們來看看 BP1001-A 在實體腫瘤患者中進行的 I/Ib 期臨床試驗，包括卵巢癌、子宮內膜癌、胰臟癌和三陰性乳癌。使用當今的治療工具包治療一些最具挑戰性的癌症。BP1001-A 是 prexigebersen 的改良產品，具有相同的藥物成分，但具有增強的奈米顆粒特性。該試驗正在幾個領先的癌症中心進行，最初將評估實體瘤患者的安全性。

Patients diagnosed with recurrent ovarian and endometrial cancer often have poor outcomes, and it is our hope that we may provide clinical benefit to such patients. We look forward to cohort completion and data readout from this study potentially later this year.

被診斷為復發性卵巢癌和子宮內膜癌的患者通常預後不佳，我們希望能為此類患者提供臨床益處。我們期待今年稍後這項研究的隊列完成和數據讀出。

Finally, let's review the progress we've made with BP1003, which targets the STAT3 protein. STAT3 is a transcription factor that regulates various tumorigenic processes such as tumor proliferation, metastasis and drug resistance. It is overexpression and aberrant activation characterize many cancers including breast, lung, ovarian, liver and colon cancer. Activation of the STAT3 pathway in breast and ovarian cancer cells promotes tumor initiation migration and taxol resistance.

最後，讓我們回顧一下針對 STAT3 蛋白的 BP1003 所取得的進展。STAT3是一種轉錄因子，調節腫瘤增殖、轉移和抗藥性等多種致瘤過程。它的過度表現和異常活化是許多癌症的特徵，包括乳腺癌、肺癌、卵巢癌、肝癌和結腸癌。乳癌和卵巢癌細胞中 STAT3 路徑的活化促進腫瘤起始遷移和紫杉醇抗藥性。

STAT3 also promotes 5-FU resistance in colorectal cancer cells. Its role in numerous malignancies made STAT3 a potential cancer therapeutic target. BP1003 is a novel liposome-incorporated STAT3 antisense oligodeoxynucleotide that efficiently reduces STAT3 expression and enhances the sensitivity of breast and ovarian cancer cells the taxol and 5-FU. These results are in-line with previous work in which BP1003 plus gemcitabine displayed enhanced antitumor activity in pancreatic ductal adenocarcinoma.

STAT3 也促進大腸直腸癌細胞對 5-FU 的抗藥性。STAT3 在多種惡性腫瘤中的作用使其成為潛在的癌症治療標靶。BP1003是一種新型脂質體摻入的STAT3反義寡脫氧核苷酸，可有效降低STAT3表現並增強乳癌和卵巢癌細胞對紫杉醇和5-FU的敏感性。這些結果與先前的研究結果一致，其中 BP1003 聯合吉西他濱在胰腺導管腺癌中顯示出增強的抗腫瘤活性。

Together, these results strongly suggest BP1003 combination therapy is a novel strategy for patients with advanced solid tumors. After an extended period of testing, we have identified a method for oligo detection and plasma that we expect will enable us to complete final safety testing, needed to finalize an investigational new drug, or IND application for submission to the FDA. We are particularly excited to launch our first in-human validation of this cutting-edge therapy in an especially challenging cancer indication that has limited treatment options.

總之，這些結果強烈表明 BP1003 聯合療法對於晚期實體瘤患者來說是一種新策略。經過長時間的測試，我們已經確定了一種寡核苷酸檢測和血漿方法，我們預計該方法將使我們能夠完成最終的安全測試，這是完成研究性新藥或向FDA 提交IND 申請所需的。我們特別高興能夠在治療選擇有限的特別具有挑戰性的癌症適應症中啟動這種尖端療法的首次人體驗證。

Before I turn the call over to Anthony for his review of our financial quarter results, I would like to highlight that we've strengthened our balance sheet in recent weeks with a $4 million financing. This additional funding provides the financial underpinning from which to execute on our clinical development plan.

在我將電話轉交給安東尼審查我們的財務季度業績之前，我想強調一下，我們最近幾週透過 400 萬美元的融資加強了我們的資產負債表。這筆額外資金為我們執行臨床開發計畫提供了財務基礎。

With that, I'll now turn the program over to Anthony Price for a brief review of our financials along with balance sheet highlights. Anthony?

現在，我將把該計劃移交給安東尼·普萊斯，以簡要回顧我們的財務狀況以及資產負債表要點。安東尼？

Thanks, Peter. The company reported a net loss of $1.9 million or $1.16 per share for the three months ended June 30, 2024, compared to a net loss of $4.2 million or $10.64 per share for the 3 months ended June 30, 2023.

謝謝，彼得。該公司報告，截至2024年6月30日止三個月的淨虧損為190萬美元，即每股1.16美元，而截至2023年6月30日止三個月的淨虧損為420萬美元，即每股10.64美元。

Research and development expense for the three months ended June 30, 2024, decreased to $1.9 million compared to $3.1 million for the three months ended June 30, 2023, primarily due to decreased manufacturing expenses related to drug product releases, partially offset by an increase in expense related to our clinical trial for BP1002 in lymphoma, due to increased patient enrollment in 2024.

截至2024 年6 月30 日止三個月的研發費用減少至190 萬美元，而截至2023 年6 月30 日止三個月的研發費用為310 萬美元，主要是由於與藥品發布相關的製造費用減少，部分被藥品發布的增加所抵消。

General and administrative expense for the three months ended June 30, 2024, and June 30, 2023, was $1.2 million. As of June 30, 2024 the company had cash of $4.0 million compared to $1.1 million as of December 31, 2023.

截至2024年6月30日和2023年6月30日的三個月的一般和管理費用為120萬美元。截至 2024 年 6 月 30 日，該公司擁有現金 400 萬美元，而截至 2023 年 12 月 31 日為 110 萬美元。

The Net cash used in operating activities for the six months ended June 30, 2024, was $4.3 million compared to $6.9 million for the comparable period in 2023. Net cash provided by financing activities for the six months ended June 30, 2024, was $7.2 million.

截至 2024 年 6 月 30 日的六個月經營活動使用的淨現金為 430 萬美元，而 2023 年同期為 690 萬美元。截至 2024 年 6 月 30 日的六個月，融資活動提供的淨現金為 720 萬美元。

With that, I'll now turn the call back over to Peter.

這樣，我現在將把電話轉回給彼得。

Thanks, Anthony. As you can see from the data we presented last quarter, we are well on our way to establishing our DNAbilize platform as a better path for cancer patients. We are paving the way for a revolutionary treatment of disease by taking a gentler path.

謝謝，安東尼。正如您從我們上季度提供的數據中看到的，我們正在努力建立 DNAbilize 平台，為癌症患者提供更好的治療途徑。我們正在透過採取更溫和的方式為疾病的革命性治療鋪平道路。

As positive data continues to flow from our clinical trials, we are becoming even more excited about bringing these potentially life-saving therapies to some of the most fragile oncology patients. We know this is an ambitious commitment, but we have confidence in our approach and conviction in our mission to give previously untreatable cancer patients a fighting chance.

隨著我們的臨床試驗不斷湧現出積極的數據，我們對於將這些可能挽救生命的療法帶給一些最脆弱的腫瘤患者變得更加興奮。我們知道這是一項雄心勃勃的承諾，但我們對我們的方法充滿信心，也堅信我們的使命是為以前無法治癒的癌症患者提供一個奮鬥的機會。

With that, operator, we are ready to open the call for questions.

接線員，我們已經準備好開始提問了。

Ladies and gentlemen, at this time, we'll begin the question-and-answer session.

女士們、先生們，現在我們開始問答環節。

(Operator Instructions)

（操作員說明）

And ladies and gentlemen, we appear to have no questions today. So I will turn the floor back over to you, Peter Nielsen, for any closing comments.

女士們先生們，我們今天似乎沒有問題。因此，我將把發言權交還給彼得·尼爾森（Peter Nielsen），請您發表結束語。

Thank you again for joining us and for your continued support of Bio Path. Have a great day.

再次感謝您加入我們並感謝您對 Bio Path 的持續支持。祝你有美好的一天。

And ladies and gentlemen, thank you again for joining us today. This concludes today's presentation. We do thank you for joining. Have a great rest of the day.

女士們、先生們，再次感謝你們今天加入我們。今天的演講到此結束。我們非常感謝您的加入。祝你這一天好好休息。