Biora Therapeutics Inc (BIOR) 2023 Q2 法說會逐字稿

Greetings, and welcome to the Biora Therapeutics's second-quarter 2023 earnings call. (Operator Instructions) As a reminder, this conference is being recorded.

大家好，歡迎參加 Biora Therapeutics 的 2023 年第二季財報電話會議。 （操作員指示）謹此提醒，本次會議正在錄製中。

It's now my pleasure to introduce your host, Chuck Padala, Managing Director with LifeSci Advisors. Thank you. You might begin.

現在我很高興向您介紹主持人，查克·帕達拉 (Chuck Padala)，他是 LifeSci Advisors 的董事總經理。謝謝。你可以開始了。

Thank you, operator. Good afternoon, and welcome to the Biora Therapeutics's second quarter 2023 corporate update and financial results conference call. Joining me on the call are Adi Mohanty, Chief Executive Officer; and Eric d'Esparbes, Chief Financial Officer.

謝謝你，接線生。下午好，歡迎參加 Biora Therapeutics 2023 年第二季公司更新與財務業績電話會議。與我一起參加電話會議的還有執行長 Adi Mohanty；艾瑞克‧德斯帕布 (Eric d'Esparbes)，財務長。

Before I turn the call over to Mr. Mohanty, I would like to remind you that today's call will include forward-looking statements within the meaning of the Federal Securities Law, including, but not limited to, the types of statements identified as forward-looking in our quarterly report on Form 10-Q that we filed or will file later today and our subsequent reports with the SEC, which are available on our website in the Investor's section.

在我將電話轉交給莫漢蒂先生之前，我想提醒您，今天的電話會議將包括聯邦證券法含義內的前瞻性陳述，包括但不限於被確定為前瞻性陳述的類型查看我們今天晚點時候提交或即將提交的10-Q 表格季度報告以及我們向SEC 提交的後續報告，這些報告可在我們網站的投資者部分找到。

These forward-looking statements represent our views only as of the date of this call and involve substantial risks and uncertainties, including many that are beyond our control. Please note that the actual results could differ materially from those projected in any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ materially from those expressed in the forward-looking statements, as well as risks related to our business, please see the company's periodic reports filed with the SEC.

這些前瞻性陳述僅代表我們截至本次電話會議之日的觀點，涉及重大風險和不確定性，包括許多我們無法控制的風險和不確定性。請注意，實際結果可能與任何前瞻性陳述中的預測有重大差異。有關可能導致實際結果與前瞻性陳述中表達的結果存在重大差異的風險和不確定性以及與我們業務相關的風險的進一步描述，請參閱公司向 SEC 提交的定期報告。

With that, I will now turn the call over to Adi Mohanty, CEO of Biora Therapeutics. Adi?

現在，我將電話轉給 Biora Therapeutics 執行長 Adi Mohanty。阿迪？

Thanks, Chuck. And thank you, everyone, for joining us. During the second quarter, we continued to make excellent progress with our NaviCap platform, advancing toward our planned IND filing in Q3, and with our BioJet platform, where we continued our progress on device development and testing. We also further strengthened our intellectual property position for both platforms.

謝謝，查克。感謝大家加入我們。在第二季度，我們的 NaviCap 平台繼續取得出色進展，朝著我們計劃在第三季度提交 IND 的目標邁進，而我們的 BioJet 平台則繼續在設備開發和測試方面取得進展。我們也進一步加強了這兩個平台的知識產權地位。

First, an update on our NaviCap Targeted Therapeutics platform, with our lead program BT-600 in ulcerative colitis. Currently available therapeutics for ulcerative colitis or UC, work by entering the bloodstream and circulating systemically throughout the body with a limited amount of drug reaching the colon, which is the site of the disease. Research suggests there is a minimum amount of drug needed in the tissue at the site of disease to achieve better therapeutic outcomes. Reaching or exceeding this threshold in the tissue should improve patient outcomes for UC.

首先，更新我們的 NaviCap 標靶治療平台，其中包括我們治療潰瘍性結腸炎的主導項目 BT-600。目前治療潰瘍性結腸炎或 UC 的方法是透過進入血液並在全身進行系統循環來發揮作用，其中有限數量的藥物到達結腸（疾病部位）。研究表明，疾病部位的組織需要最少量的藥物才能達到更好的治療效果。達到或超過組織中的這個閾值應該可以改善 UC 患者的預後。

Our NaviCap platform uses a device about the size fish-oil pill, that once swallowed is designed to deliver drug directly to the colon using our GI tract localization technology. The commercially available version of tofacitinib is approved in capsule form for the treatment of UC. Like other drugs, tofacitinib is taken up systemically with a limited amount reaching the area of disease in the colon. By delivering drug where it is needed at the site of disease, we believe our BT-600 program can deliver sufficient drug to the tissue of the targeted area to improve efficacy.

我們的 NaviCap 平台使用魚油丸大小的設備，一旦吞嚥，就會利用我們的胃腸道定位技術將藥物直接輸送到結腸。託法替尼的市售版本以膠囊形式被批准用於治療 UC。與其他藥物一樣，託法替布被全身吸收，到達結腸病變區域的劑量有限。透過將藥物輸送到疾病部位需要的地方，我們相信我們的 BT-600 計劃可以將足夠的藥物輸送到目標區域的組織，以提高療效。

This localized delivery, along with our proprietary formulation, has shown in animal studies the ability to deliver the right amount of drug to the tissue even with a lower overall dose than the currently approved dose for tofacitinib. We also expect our approach the result in reduced systemic exposure, which may improve safety for this patient population.

這種局部遞送以及我們的專有配方在動物研究中表明，即使總體劑量低於目前批准的託法替布劑量，也能夠將適量的藥物遞送到組織。我們也期望我們的方法能夠減少全身暴露，這可能會提高該患者群體的安全性。

So we're extremely focused on getting NaviCap into the clinic. And everything we're working on right now with this program is intended to enable our planned September IND filing. I'm happy to report that we are on track with our plans.

因此，我們非常專注於將 NaviCap 引入診所。我們目前在該計劃中所做的一切都是為了實現我們計劃的 9 月 IND 申請。我很高興地報告，我們的計劃正在按計劃進行。

All of the analyses from our talk study, which was conducted earlier this year, have been completed. We continue to believe that these results support our assessment of readiness for Phase 1. As we shared previously, all plan devices were administered without any adverse events or safety signals, even at the highest 25-milligram dose of tofacitinib.

我們今年早些時候進行的談話研究的所有分析都已經完成。我們仍然相信這些結果支持我們對第一階段準備情況的評估。正如我們之前分享的那樣，所有計劃裝置的使用都沒有任何不良事件或安全信號，即使是最高25 毫克劑量的託法替布也是如此。

We measured drug levels in colon tissue 24-hours after administration of the last dose. And observed drug levels supporting our ability to reach the appropriate tissue drug concentration. We observed that the drug did not accumulate over time, which is a favorable sign of safety. We also observed peak drug levels in blood that are approximately 1/3 of what would be expected with the equivalent dose of commercially available tofacitinib.

我們在最後一次給藥後 24 小時測量了結腸組織中的藥物含量。觀察到的藥物水平支持我們達到適當的組織藥物濃度的能力。我們觀察到該藥物不會隨著時間的推移而積累，這是安全性的一個有利跡象。我們也觀察到血液中的最高藥物濃度約為同等劑量市售託法替布的預期水準的 1/3。

As we prepare for our Phase 1 trial, we have conducted another device function study, which demonstrated outstanding performance and supports our assessment of device function in humans. We recently announced preliminary results from this study. And we subsequently completed the study as planned after 16 healthy volunteers had been administered the NaviCap device. Our Phase 1-ready device performed as intended in 15 out of 16 subjects.

在我們準備第一階段試驗時，我們進行了另一項設備功能研究，該研究展示了出色的性能並支持我們對人體設備功能的評估。我們最近公佈了這項研究的初步結果。在 16 名健康志願者使用 NaviCap 設備後，我們隨後按計劃完成了研究。我們的第一階段就緒設備在 16 名受試者中的 15 名中表現符合預期。

The NaviCap device accurately identified entry into the colon, triggered release of its non-drug payload, and achieved distribution throughout the colon. The greater than 90% performance is consistent with our previous human studies. It exceeds our minimum device function requirements, and it gives us confidence as we head toward the clinic.

NaviCap 設備可準確識別進入結腸的入口，觸發其非藥物有效負載的釋放，並實現整個結腸的分佈。超過 90% 的性能與我們先前的人體研究一致。它超出了我們的最低設備功能要求，讓我們在走向診所時充滿信心。

From the latest study, we generated a series of images that show the NaviCap device progressing through the GI tract, activating upon entry to the colon, and dispersion of the payload throughout the colon. We've included these time lapse images as a video in our corporate deck on our website. I invite you to take a look at that.

根據最新研究，我們產生了一系列圖像，顯示 NaviCap 設備在胃腸道中前進，在進入結腸時激活，以及有效負載在整個結腸中的分散。我們已將這些延時圖像作為影片包含在我們網站上的公司幻燈片中。我邀請你看一下。

The study once again showed the robustness of our NaviCap platform, which performed as designed across a range of expected differences in GI motility between study participants. Looking ahead to the planned Phase 1 study, we hope to see data that confirm the learnings from our animal studies and confirm our hypothesis that BT-600 can safely deliver tofacitinib locally to the colon resulting in sufficient colonic tissue levels and low systemic exposure.

該研究再次證明了我們的 NaviCap 平台的穩健性，該平台在研究參與者之間胃腸道運動的一系列預期差異中表現出色。展望計劃中的1 期研究，我們希望看到數據證實我們的動物研究成果，並證實我們的假設，即BT-600 可以安全地將託法替佈局部遞送到結腸，從而產生足夠的結腸組織水平和較低的全身暴露。

The Phase 1 study design includes a colon tissue biopsy in all subjects at the end of the study. Tissue biopsy is not typical for a Phase 1 study in healthy volunteers. But we feel this information will be extremely valuable in informing the design of our future studies in patients with UC. This proposed trial design has been discussed with the FDA, and they have been supportive of it so far.

第一階段研究設計包括在研究結束時對所有受試者進行結腸組織活檢。組織切片檢查對於健康志願者的一期研究來說並不典型。但我們認為這些資訊對於我們未來針對 UC 患者的研究設計非常有價值。這項擬議的試驗設計已經與 FDA 進行了討論，到目前為止他們一直在支持它。

We expect to exit Phase 1 with safety data but also with critical data on potential exposure in both serum and tissue, which could be much more informative than a typical Phase 1 trial. In addition to progress towards the IND, we also had some nice wins on the IP front recently. Having been awarded a group of US and European patents directed to methods of treating ulcerative colitis using our NaviCap device, to deliver well known IBD drugs such as Janus kinase inhibitors, anti-integrins, and IL-12/23 inhibitors.

我們期望在退出第一階段時提供安全性數據，同時也提供有關血清和組織中潛在暴露的關鍵數據，這些數據可能比典型的第一階段試驗提供更多資訊。除了在 IND 方面取得進展外，我們最近在智慧財產權方面也取得了一些不錯的成果。已獲得一組美國和歐洲專利，涉及使用我們的 NaviCap 設備治療潰瘍性結腸炎的方法，以提供眾所周知的 IBD 藥物，如 Janus 激酶抑製劑、抗整合素和 IL-12/23 抑製劑。

Our extensive IP will allow us to organically expand our pipeline to several potential targets. We believe the NaviCap platform could transform treatment options for many other diseases of the GI tract. And that progress with BT-600 will unlock significant new opportunities for Biora.

我們廣泛的智慧財產權將使我們能夠有機地將我們的產品線擴展到多個潛在目標。我們相信 NaviCap 平台可以改變許多其他胃腸道疾病的治療選擇。 BT-600 的進展將為 Biora 帶來重大的新機會。

To summarize, we're on track and remain confident in our progress toward a September IND filing for the NaviCap program. We believe we can potentially address the large unmet need for UC patients by solving one of the primary treatment gaps, which is the inability to achieve high-enough drug levels in the diseased tissue without systemic toxicity.

總而言之，我們正在步入正軌，並對 9 月 NaviCap 計畫 IND 申請的進展充滿信心。我們相信，我們可以透過解決主要治療差距之一來解決 UC 患者未滿足的巨大需求，即無法在患病組織中達到足夠高的藥物水平而不產生全身毒性。

Moving on to our BioJet Systemic Therapeutics platform. Our goal with the BioJet platform is to provide needle free, oral delivery of large molecules. It is based on the device the size of a multivitamin, that once swallowed, is designed to deliver drug into the small intestine. Using liquid jet injection to maximize systemic uptake.

繼續我們的 BioJet 系統治療平台。我們 BioJet 平台的目標是提供大分子的無針口服輸送。它基於多種維生素大小的裝置，一旦吞嚥，就會將藥物輸送到小腸中。使用液體噴射注射最大限度地提高全身吸收。

We believe the BioJet platform can transform patient care by improving patient convenience, which has been shown to affect patient compliance. We see this leading to better disease management and associated patient outcomes across a range of chronic use indications.

我們相信 BioJet 平台可以透過提高患者便利性來改變患者護理，這已被證明會影響患者的依從性。我們認為這可以在一系列長期使用適應症中帶來更好的疾病管理和相關患者結果。

During the second quarter, we presented recent data generated with the BioJet platform at the American Diabetes Association Scientific Sessions, where we assess the bioavailability of semaglutide delivered via our BioJet device in an animal model. In two studies, we achieved more than double our target average bioavailability of 15%, which tells us that so far based on the data the BioJet platform can deliver existing liquid drug formulations with bioavailability similar to injection. We base our claims on data generated in animals where we use IV delivery as our control because IV is considered the gold standard, particularly when it comes to bioavailability.

第二季度，我們在美國糖尿病協會科學會議上展示了 BioJet 平台產生的最新數據，我們在動物模型中評估了透過 BioJet 設備輸送的索馬魯肽的生物利用度。在兩項研究中，我們實現了目標平均生物利用度15% 的兩倍多，這告訴我們，到目前為止，根據數據，BioJet 平台可以提供現有的液體藥物製劑，其生物利​​用度與注射劑相似。我們的主張是基於我們使用靜脈注射作為對照的動物產生的數據，因為靜脈注射被認為是黃金標準，特別是在生物利用度方面。

Using the IV standard as a measuring stick, our bioavailability would be quite impressive, compared to other oral delivery modes. However, some of our competitors use subcutaneous injection as their competitor arm. Subcutaneous injection is known to achieve about 60% of the bioavailability of IV administration. If we compare our BioJet platform using the measuring stick of subcu injection, we see that we're achieving bioavailability similar to injection for the molecules we have tested, which is meaningfully better than what others have achieved.

使用 IV 標準作為衡量標準，與其他口服給藥方式相比，我們的生物利用度將非常令人印象深刻。然而，我們的一些競爭對手使用皮下注射作為他們的競爭對手。已知皮下注射的生物利用度約為靜脈注射的 60%。如果我們使用 subcu 注射的量尺來比較我們的 BioJet 平台，我們會發現我們所測試的分子實現了與注射相似的生物利用度，這比其他人所實現的要好得多。

We will be presenting more BioJet generated data at the European Association for the Study of Diabetes annual meeting, which will be help in October. As planned, during Q2, we have continued development and testing of the autonomously triggered version of our latest BioJet device. We have successfully adapted our previous autonomous trigger designs to this latest device. After several rounds of design evolution and testing, we've been able to achieve our device function targets while continuing to exceed our performance target of 15% bioavailability.

我們將在歐洲糖尿病研究協會年會上展示更多 BioJet 產生的數據，這將在 10 月有所幫助。按照計劃，在第二季度，我們繼續開發和測試最新 BioJet 設備的自動觸發版本。我們已成功地將先前的自主觸發器設計應用於這款最新設備。經過幾輪的設計改進和測試，我們已經能夠實現我們的設備功能目標，同時繼續超過 15% 生物利用度的性能目標。

We're now repeating these tests so we can have a robust data set for discussions with our pharma collaborators. We remain encouraged by interactions with our collaborators, and look forward to further progress and subsequently advancing toward our goal of achieving meaningful partnerships for our BioJet platform.

我們現在正在重複這些測試，以便我們能夠擁有可靠的數據集，以便與我們的製藥合作者進行討論。我們仍然對與合作者的互動感到鼓舞，並期待取得進一步進展，並隨後朝著為我們的 BioJet 平台實現有意義的合作夥伴關係的目標邁進。

While many organizations have been working to solve the challenges of oral delivery of large molecules, our approach stands out because of our liquid jet delivery method, which is not only able to achieve excellent bioavailability. But is also able to deliver an existing liquid formulation. Reformulating a drug is time consuming and expensive. So the ability to use existing liquid formulations gives us another potential competitive advantage.

雖然許多組織一直在努力解決大分子口服給藥的挑戰，但我們的方法因其液體噴射給藥方法而脫穎而出，該方法不僅能夠實現出色的生物利用度。但也能夠提供現有的液體配方。重新配製藥物既耗時又昂貴。因此，使用現有液體配方的能力為我們帶來了另一個潛在的競爭優勢。

Looking ahead to the future of disease management, we think it will involve more complex molecules such as dual and triple agonist for diabetes management and also combination therapies, which are all harder to make into pills. We think our BioJet platform will be well positioned to deliver these more complex therapies.

展望疾病管理的未來，我們認為它將涉及更複雜的分子，例如用於糖尿病管理的雙重和三重激動劑以及聯合療法，這些都更難製成藥丸。我們認為我們的 BioJet 平台將能夠很好地提供這些更複雜的療法。

Another potential advantage of our technology is the ability to achieve uptake of drugs in hard-to-reach areas like the liver. A large number of disease targets reside in the liver, and it is a key area for gene expression therapies. For example, RNA therapies that rely on liver-targeted delivery of antisense oligonucleotides.

我們技術的另一個潛在優勢是能夠在肝臟等難以到達的區域實現藥物的吸收。大量的疾病標靶存在於肝臟中，它是基因表現治療的關鍵領域。例如，RNA 療法依賴反義寡核苷酸的肝臟標靶遞送。

Other oral delivery technologies have not been able to address this challenge. We are awaiting data from studies we completed with one of our collaborator that may provide useful information on our ability to deliver these types of therapies. And we look forward to sharing more of this in the coming weeks.

其他口服給藥技術尚無法應對這項挑戰。我們正在等待與一位合作者完成的研究數據，這些數據可能會提供有關我們提供此類療法的能力的有用資訊。我們期待在未來幾週內分享更多相關內容。

We recently announced further progress on the IP front related to the BioJet platform as well. With additional patents covering key features of our liquid jet injection technology, with approximately 38 issued patents and pending applications covering our delivery platform and methods for using liquid jet delivery to treat patients, we are in a strong competitive position to participate in delivery of the $100 billion-plus GLP-1 agonist market. As well as many other large molecules such as proteins, peptides, and nucleic acids.

我們最近也宣布了與 BioJet 平台相關的知識產權方面的進一步進展。憑藉涵蓋我們液體噴射注射技術關鍵特徵的其他專利，大約38 項已發布專利和待審申請涵蓋我們的交付平台和使用液體噴射交付治療患者的方法，我們在參與交付100 美元的項目方面處於強大的競爭地位超過十億的 GLP-1 激動劑市場。以及許多其他大分子，如蛋白質、勝肽和核酸。

To summarize our anticipated milestones for our NaviCap platform, we remain on track filing an IND for BT-600 in September. And we anticipate initiating our Phase 1 trial before the end of the year. For our BioJet platform, we're confirming the performance of our autonomous next generation BioJet device. And we expect to continue generating preclinical data during the third quarter of 2023.

為了總結我們 NaviCap 平台的預期里程碑，我們仍在按計劃於 9 月提交 BT-600 的 IND。我們預計在今年年底前啟動第一階段試驗。對於我們的 BioJet 平台，我們正在確認下一代自主 BioJet 設備的效能。我們預計將在 2023 年第三季繼續產生臨床前數據。

We expect to continue testing the BioJet device with the molecules of our pharma collaborators in the coming months. We look forward to sharing additional BioJet preclinical data at the European Association for the Study of Diabetes annual meeting in October.

我們預計在未來幾個月內繼續使用我們製藥合作者的分子測試 BioJet 設備。我們期待在 10 月的歐洲糖尿病研究協會年會上分享更多 BioJet 臨床前數據。

With that, I'll now turn the call over to Eric for a review of our financial results and capital market activities.

現在，我將把電話轉給埃里克，要求他審查我們的財務表現和資本市場活動。

Thanks, Adi. And good afternoon, everyone. During the second quarter, operating expenses, excluding stock-based compensation expenses, remained stable at $12.5 million, with continued investment in device development and preclinical activities.

謝謝，阿迪。大家下午好。第二季度，隨著對設備開發和臨床前活動的持續投資，營運費用（不包括股票補償費用）保持穩定在 1,250 萬美元。

Breaking this down, G&A expenses in the second quarter were $7.7 million, excluding stock-based compensation expenses; while R&D expenses in the second quarter were $5.2 million, excluding stock-based compensation expenses. We've been continuing to streamline our G&A expenses and making great progress in focusing on investments on our R&D programs. While more than 30% of our G&A spend in Q2 was still composed of legacy spend, we expect that will gradually tail off by early next year. Our core OpEx expense remains very much focused towards our R&D programs, which makes sense, as we rapidly move towards the clinic.

細分來看，第二季的一般管理費用為 770 萬美元，不包括股票薪酬費用；而第二季的研發費用為520萬美元，不包括股票補償費用。我們一直在繼續精簡一般管理費用，並在專注於研發項目的投資方面取得了巨大進展。雖然我們第二季超過 30% 的一般管理支出仍由遺留支出組成，但我們預計到明年初這一比例將逐漸減少。我們的核心營運支出仍然主要集中在我們的研發項目上，隨著我們迅速走向臨床，這是有道理的。

While spending will continue to fluctuate from month-to-month as we progress towards the clinic, we maintain our guidance of an average monthly operating cash burn of approximately $4 million. We successfully raised $8 million in gross equity proceeds during the second quarter, which funded the majority of our Q2 operating expenses leading to only a slight reduction in our cash balance to $26.5 million as of June 30, 2023. We also continue to make progress with monetizing remaining legacy assets, which will also add incremental funding.

雖然隨著診所的進展，支出將繼續每月波動，但我們維持每月平均營運現金消耗約 400 萬美元的指引。我們在第二季成功籌集了800 萬美元的總股本收益，這為我們第二季的大部分營運支出提供了資金，導致截至2023 年6 月30 日我們的現金餘額僅略有減少至2,650 萬美元。我們也繼續在以下方面取得進展：將剩餘的遺留資產貨幣化，這也將增加增量資金。

Finally, we continue to work actively on optimizing our capital structure to maintain and enhance our public company profile. And we expect to have some updates soon.

最後，我們持續積極優化資本結構，以維持和提升我們的上市公司形象。我們預計很快就會有一些更新。

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

現在，我將把電話轉回給阿迪。

Thanks, Eric. Biora continues to make strides with both our NaviCap-targeted oral delivery platform, where we are focused on entering with our BT-600 program; and our BioJet Systemic oral delivery platform, where we're focused on confirming the performance of our next gen device and progressing with pharma collaborations. We look forward to providing further updates, as we achieve these milestones.

謝謝，埃里克。 Biora 繼續在我們的 NaviCap 目標口服給藥平台上取得進展，我們專注於進入我們的 BT-600 計畫；以及我們的 BioJet Systemic 口服給藥平台，我們專注於確認下一代設備的性能並推動製藥合作。當我們實現這些里程碑時，我們期待提供進一步的更新。

With that, operator, we're now ready for question.

接線員，我們現在準備好提問了。

Thank you. We will now be conducting a question-and-answer session.

謝謝。我們現在將進行問答環節。

(Operator Instructions) Joe Pantginis, H.C. Wainwright.

（操作員說明）Joe Pantginis, H.C.溫賴特。

Good afternoon, gentlemen. Thank you for taking the question. Very nice to see the recent NaviCap data. So I wanted to focus on that for a couple of moments, if you don't mind.

下午好，先生們。感謝您提出問題。很高興看到最近的 NaviCap 數據。如果你不介意的話，我想集中討論一下這個問題。

So I guess, the focus of my question is on business development. So, obviously, you're dealing with UC right now and UC-associated drugs. You're also getting the level of bioavailability that you'd like to see and you're getting the tissue targeting. So I guess my two questions are, how do these data impact your overall business development strategy? And also what is the potential, scientific or clinical impact, based on the type of drug that you use within the NaviCap system? Thanks a lot.

所以我想，我的問題的重點是業務發展。所以，顯然，您現在正在處理 UC 和 UC 相關藥物。您還可以獲得您希望看到的生物利用度水平，並獲得組織靶向。所以我想我的兩個問題是，這些數據如何影響您的整體業務發展策略？根據您在 NaviCap 系統中使用的藥物類型，潛在的科學或臨床影響是什麼？多謝。

Yeah. Thanks for the question, Joe. So it's a slightly complex question when you talk about how does it impact our BD strategy. Clearly, we're really excited about how it's performing.

是的。謝謝你的提問，喬。因此，當您談論它如何影響我們的業務發展策略時，這是一個稍微複雜的問題。顯然，我們對它的表現感到非常興奮。

So our NaviCap platform are Phase 1-ready devices, we just ran it. It is working amazingly well. So our GI tract localization technology figures out exactly where we want the drug to be dropped. So the programming is set. We can tweak that if we want, and do different drugs in different parts of the GI tract.

因此，我們的 NaviCap 平台是第一階段就緒設備，我們剛剛運行它。它運行得非常好。因此，我們的胃腸道定位技術可以準確地確定我們想要將藥物投放到的位置。這樣程序就設定好了。如果我們願意的話，我們可以進行調整，並在胃腸道的不同部位使用不同的藥物。

There's various ways we could do a lot more with this platform. But we're staying focused on trying to get to this UC trial where, for those who may know the UC area, you would know that tofacitinib is a very powerful molecule. It works extremely well. Problem is, it works so well that a huge amount of this becomes toxic to the body. And in order to get the right amount of drug in the colon, you got to dose up to the point where it's toxic and you get a great response.

我們可以透過多種方式利用這個平台做更多事情。但我們仍然專注於嘗試進行 UC 試驗，對於可能了解 UC 領域的人來說，您會知道託法替布是一種非常強大的分子。它運作得非常好。問題是，它的作用非常好，以至於大量的這種物質會對身體產生毒性。為了在結腸中獲得適量的藥物，您必須將劑量達到有毒的程度，並且您會得到很好的反應。

So solving the problem of not having systemic toxicity by having too much going around in the blood. But getting enough drug to the tissue, would make this a substantially different therapeutic option that many physicians would love to have and certainly the patients.

因此解決了血液中循環過多而不會產生全身毒性的問題。但是，將足夠的藥物輸送到組織中，將使其成為許多醫生（當然還有患者）希望擁有的完全不同的治療選擇。

So what we're seeing is with the way we have delivered it, and this is through the animal studies, we get something in the range of a third or quarter of the amount of drug that you would normally get in the blood when we're getting a lot more in the tissue, which makes sense, right? We have a formulation that is a liquid formulation. That when it's dropped in the colon, coats to colon, and you get most of that uptake through that colon tissue, and getting a lot more in that tissue than any other method.

所以我們看到的是我們提供藥物的方式，這是透過動物研究，我們得到的藥物量是通常在血液中獲得的藥物量的三分之一或四分之一。組織中得到了更多，這是有道理的，對嗎？我們有一種液體配方。當它落入結腸時，覆蓋到結腸，並且您通過結腸組織獲得大部分吸收，並且在該組織中獲得的量比任何其他方法都要多。

So we want to stay focused on getting a little bit further along. We think it -- we don't need to go much further, right? If we can show in our upcoming trial that we get the kinds of blood concentrations we're getting in animals; the kinds of tissue concentrations we're getting in animals; that we get confirmation that you can get a large amount of drug in the tissue the amount that is required and you can use a much smaller dose of tofacitinib; and get less systemic exposure but still get some, all of those things allow us to then have the opportunity to discuss how we keep going forward.

因此，我們希望繼續專注於取得更進一步的進展。我們認為──我們不需要走得更遠，對嗎？如果我們能夠在即將進行的試驗中證明我們獲得了動物身上的血液濃度；我們在動物身上得到的組織濃度類型；我們得到確認，您可以在組織中獲得所需量的大量藥物，並且您可以使用小得多劑量的託法替布；減少系統性風險，但仍然得到一些，所有這些都讓我們有機會討論如何繼續前進。

For now, we're planning to then move on to using the drug in patients. We want to stay focused on UC, because we think that it not only is a great target as you mentioned, right? Right now, there's been a couple of three really great business transactions in the UC arena. Worth billions of dollars for companies that have had early-stage drugs that large companies want.

目前，我們計劃繼續在患者身上使用該藥物。我們希望繼續關注 UC，因為我們認為它不僅是您提到的一個偉大目標，對嗎？目前，UC 領域已經發生了三筆非常出色的商業交易。對於擁有大公司想要的早期藥物的公司來說價值數十億美元。

But it unlocks our entire platform. I know there's a long answer. But what it says is if we continue just a little bit further and show that the platform works and the particular approach for UC works, we could do something in the UC space. We could do a lot more with the entire platform.

但它解鎖了我們的整個平台。我知道答案很長。但它所說的是，如果我們繼續更進一步並證明該平台有效且 UC 的特定方法有效，我們可以在 UC 領域做一些事情。我們可以利用整個平台做更多事情。

We also got some new IP that allows us to do a lot more drugs of various kinds in the GI tract. Most of these you might be familiar with. So we unlock many different paths forward, and we will remain open to many of those options in a short few months.

我們也獲得了一些新的智慧財產權，使我們能夠在胃腸道中生產更多種類的藥物。其中大多數您可能很熟悉。因此，我們開啟了許多不同的前進道路，並且我們將在短短幾個月內對其中許多選擇保持開放。

Great. Appreciate the answer, Adi.

偉大的。感謝您的回答，阿迪。

Julian Harrison, BTIG.

朱利安·哈里森，BTIG。

Hi, thank you for taking my questions. And congrats on the progress. On BT-600, I'm curious how soon you could proceed to dosing in ulcerative colitis patients? And then, are there any device-related considerations or parameter changes when you make that move from healthy volunteers to UC patients?

你好，謝謝你回答我的問題。並祝賀取得的進展。對於 BT-600，我很好奇你們多久可以開始對潰瘍性結腸炎患者進行給藥？然後，當您從健康志願者轉移到 UC 患者時，是否有任何與設備相關的考慮因素或參數變化？

Yeah. Thanks, Julian for the question. Second part, I'll take first, which is really interesting, right? There's been a lot of questions about, hey, when you put this in humans, does it work? Because -- and yes, we have done it.

是的。謝謝朱利安的提問。第二部分，我先看，這真的很有趣，對吧？有很多問題，嘿，當你把這個放在人類身上時，它有效嗎？因為——是的，我們已經做到了。

So far, we have done more than 80 human subjects. And interestingly, we've actually done it with patients. So we've had UC patients and, you can find it on our website. We have some data where we've used this device with the card programming in UC patients of various different severity.

到目前為止，我們已經完成了 80 多個人體實驗。有趣的是，我們實際上已經在患者身上做到了這一點。我們有 UC 患者，您可以在我們的網站上找到它。我們有一些數據顯示，我們已將此設備與卡片編程一起用於各種不同嚴重程度的 UC 患者。

So we've gone from less severe, the male score going from 2 to 6 to 8. So we've gone to more severe patients. Amazingly, it worked every single time. So even in the environment where they've got inflammation, bleeding, motility issues, it worked.

因此，我們從不太嚴重的患者開始，男性評分從 2 分上升到 6 分，再到 8 分。因此，我們轉向了更嚴重的患者。令人驚訝的是，它每次都有效。因此，即使在有發炎、出血、運動問題的環境中，它也能發揮作用。

So we know that the device would work and we did it without, obviously, the drug payload. So the device would work in UC patients. The device works in healthy patients, with over 80 subjects now. We know that we're getting a greater than 90% performance functionally. And it's doing the right thing at the right time.

所以我們知道該設備可以工作，而且我們顯然沒有藥物有效載荷。因此該設備適用於 UC 患者。該設備適用於健康患者，目前已有 80 多名受試者。我們知道我們的功能性能達到了 90% 以上。而且是在對的時間做對的事。

So we don't think we will need to tweak the device going from our Phase 1, where we will be using healthy volunteers to the patients themselves. What I think will be more interesting, and which we want to get to is, is there accumulation in the tissue?

因此，我們認為我們不需要從第一階段開始調整設備，我們將使用健康的志願者來治療患者本身。我認為更有趣的是，我們想要了解的是，組織中是否存在累積？

As I mentioned, right? In the animals, we saw that there wasn't any. But getting to that answer, and getting some of the other, which we're not worried about getting but will be just a good confirmation to get in the Phase 1 is, what the systemic exposure is. What all the other safety PK/PD data we can gather.

正如我所提到的，對嗎？在動物身上，我們發現沒有。但是，得到這個答案，並得到其他一些答案，我們並不擔心得到這個答案，但這只是進入第一階段的一個很好的確認，即係統性風險是什麼。我們可以收集哪些其他安全性 PK/PD 資料。

All of those would be helpful inputs. So that we can quickly propose a in-patient study. And we intend to do that. We think that in terms of derisking, when we get just this Phase 1 data, lots of people will begin to appreciate what we're going to be able to do in patients.

所有這些都會是有用的投入。這樣我們就可以快速提出住院研究。我們打算這樣做。我們認為，就降低風險而言，當我們獲得第一階段數據時，許多人就會開始意識到我們將能夠在患者身上做些什麼。

Very helpful. Thank you.

很有幫助。謝謝。

(Operator Instructions) Mayank Mamtani, B. Riley Securities

（操作員指示）Mayank Mamtani，B. Riley Securities

Hey, guys [Madison] on for Mayank. thanks for taking our call. Congrats on the progress. For the device function study. Could you talk to any differences to keep in mind, as we think about the Phase 1 BT-600 initiation, particularly in regards to the biopsy? Just wondering how performing the biopsy may impact any execution goals, enrollment goals.

嘿，夥計們[麥迪遜]為 Mayank 效力。感謝您接聽我們的電話。祝賀取得的進展。用於裝置功能研究。當我們考慮 BT-600 的 1 期啟動時，特別是在活檢方面，您能否談談需要記住的任何差異？只是想知道進行活檢可能會如何影響任何執行目標、註冊目標。

And then any color on the FDA's perspective would be helpful. For example, if you sense any pushback or hesitation? And then lastly, could you remind us of the timeline that the FDA has to respond once you submit your IND in September? Thanks.

從 FDA 的角度來看，任何顏色都會有所幫助。例如，您是否感覺到任何阻力或猶豫？最後，您能否提醒我們，一旦您在 9 月提交 IND，FDA 必須做出回應的時間表？謝謝。

Yeah, thanks for the question. It is an important one because we are fully baked in is the taking the biopsy. We would love to get the answer on what the tissue concentrations are. Obviously, what we're doing is a single-tissue biopsy at the very end of the MAD study. So it's not like we're taking it routinely. That would be more difficult in healthy volunteers as opposed to patients.

是的，謝謝你的提問。這很重要，因為我們已經完全了解了活檢的情況。我們很想知道組織濃度是多少的答案。顯然，我們正在做的是在 MAD 研究最後進行單組織活檢。所以我們並不像常規那樣服用它。與患者相比，健康志願者的情況會更加困難。

So what we want is a answer on the tissue, we will do that at the end. That is not as onerous. And we're working with a very top notch clinical trial operator who has absolutely confirmed that this is something they can do.

所以我們想要的是紙巾上的答案，我們將在最後這樣做。這並不那麼繁重。我們正在與一位非常頂尖的臨床試驗操作員合作，他絕對證實這是他們可以做到的事情。

As far as the FDA interaction, as few months ago, we actually had a Type C interaction with the FDA where we had detailed all what we wanted to do. We got very detailed responses from them That is what gives us comfort that they were quite supportive of our approach and what we were planning to do. They had some helpful input on some small areas of the trial design, which we have incorporated, or are incorporating.

就 FDA 的互動而言，幾個月前，我們實際上與 FDA 進行了 C 類互動，我們詳細說明了我們想要做的所有事情。我們得到了他們非常詳細的答复，這讓我們感到安慰，他們非常支持我們的方法和我們計劃做的事情。他們對試驗設計的一些小領域提供了一些有用的意見，我們已經納入或正在納入這些意見。

And so we feel pretty good that our interactions with the agencies have been good. The clinical trial operator has indicated that this is something that is very doable. We're working very closely with the people who would actually run the trial. And if you remember a few months ago, we got our new Chief Medical Officer who is also quite familiar with, what we're trying to execute.

因此，我們與各機構的互動良好，對此我們感覺非常好。臨床試驗操作者表示這是非常可行的。我們正在與實際進行試驗的人員密切合作。如果你還記得幾個月前，我們新任首席醫療官也非常熟悉我們正在努力執行的任務。

So we're pretty comfortable. We feel good about where we are. When we file, as you know, a typical IND after filing, it's about a 30-day period of time before you can say, yes, let's go ahead and start executing on the trial itself. So we feel, sometime later this year, we will be activating the sites in actually doing some treating of people this year. And hopefully getting the data fairly soon.

所以我們很舒服。我們對自己所處的位置感覺良好。如您所知，當我們在提交後提交典型的 IND 時，大約需要 30 天的時間，您才能說，是的，讓我們繼續開始執行試驗本身。因此，我們認為，在今年稍後的某個時候，我們將激活這些站點，在今年實際對人們進行一些治療。希望盡快獲得數據。

Alright. Great. Well, I appreciate the color.

好吧。偉大的。嗯，我很欣賞這個顏色。

John Vandermosten from Zacks.

Zacks 的約翰范德莫斯坦。

Thank you, and good afternoon. What are some of the primary endpoints you anticipate FDA will look for when you get to a pivotal trial and UC program? And are there any precedent for the NaviCap platform or what the FDA might look for when you get to that next stage?

謝謝你，下午好。當您進行關鍵試驗和 UC 計畫時，您預計 FDA 會尋找哪些主要終點？ NaviCap 平台是否有任何先例，或者當您進入下一階段時 FDA 可能會尋找什麼？

Yes, Joe. I think there's been a lot of UC trials. It's pretty well defined. It's very well understood.

是的，喬。我認為 UC 試驗已經有很多了。它的定義非常好。這是很好理解的。

Things like how quickly you get that initial response, how many weeks it takes, they're extremely well defined -- tons of trials have been done in display in this space. So that is really not that big a question. We think we can easily show also comparative data, because there's just so much data out there on how different molecules are performed.

像你多快得到初步反應、需要多少週的時間，它們都有非常明確的定義——在這個領域已經進行了大量的試驗。所以這確實不是一個大問題。我們認為我們可以輕鬆顯示比較數據，因為有太多關於不同分子如何發揮作用的數據。

Pretty easy to do. Very straightforward. We don't think that's going to be a question.

很容易做到。非常簡單。我們認為這不會成為一個問題。

When it comes to the specifics of NaviCap, no. There is no other such thing out there. There is no localization technology that is only available that you can just take a pill every day, and it goes to the right spot and drops the payload. There is a part of that that makes it a more interesting than almost any other typical drug trial, right?

至於 NaviCap 的具體細節，答案是否定的。那裡沒有其他這樣的事情。沒有一種定位技術只能讓你每天服用一顆藥丸，它就會到達正確的位置並掉落有效負載。其中一部分使它比幾乎所有其他典型的藥物試驗更有趣，對嗎？

Most of the endpoints will be drug endpoints. And so that's pretty common. I think what'll be more interesting is early on, the functionality and safety around just having a device go through every day and come out every day or as these patients are taking them, which is where there will be some amount of additional data collection around just any impact of the device, which we think we'll get very early on.

大多數終點將是藥物終點。所以很常見。我認為更有趣的是早期的功能和安全性，即設備每天經過並每天出現，或者當這些患者服用它們時，這將是一些額外數據收集的地方圍繞該設備的任何影響，我們認為我們會很早就了解這一點。

Like I said, we've already got over 80 subjects that we've done these devices with, collecting all of that into a database safety, around being able to use a device every day. We've done over 600 of these in the talks trial we did with the dogs where we saw.

就像我說的，我們已經完成了 80 多個主題，我們已經使用這些設備，將所有這些主題收集到資料庫安全中，圍繞著每天能夠使用設備。在我們對所見狗進行的談話試驗中，我們已經做了 600 多次這樣的事情。

There was no impact from whether it was a device going through and how it operated. Whether it was stuck in there with two or three of them at the same time. All various kinds of ways that the device might perform. We saw, and there was no issue.

它是否是正在經歷的設備以及它的操作方式都沒有影響。無論是兩個人還是三個人同時被困在那裡。設備可能執行的各種方式。我們看到了，沒有任何問題。

So we'll see some more of that with the Phase 1. We will see -- so I think we will collect all of that. That is the additional data we will have to collect, as the device aspect of it.

所以我們會在第一階段看到更多的內容。我們會看到——所以我認為我們會收集所有這些。這是我們必須收集的額外數據，作為其設備方面。

But when it comes to the Phase 3 outcomes, typical standard UC trial outcomes, which are really well established with lots and lots of different data. So we think by the time we get there, it'll be quite straightforward.

但當談到第 3 階段的結果時，典型的標準 UC 試驗結果確實是透過大量不同的數據建立起來的。所以我們認為，當我們到達那裡時，事情會變得非常簡單。

Thank you. I appreciate the detail. And then moving to BioJet, what are the next steps that we anticipate there before an IND is ready for that program?

謝謝。我很欣賞細節。然後轉向 BioJet，在 IND 為該計劃做好準備之前，我們預計接下來的步驟是什麼？

Yeah. So with our BioJet program, we've taken a slightly different attack instead of getting to quickly a IND and a clinical trial on our own. We want to leverage these conversations we've had. And we do have three current collaborators, really large companies that we're working with very closely, where we want to establish what the BioJet platform could do using their molecules.

是的。因此，透過我們的 BioJet 計劃，我們採取了略有不同的攻擊方式，而不是自行快速進行 IND 和臨床試驗。我們希望利用我們已經進行的這些對話。我們目前確實有三個合作者，他們都是非常大的公司，我們正在與他們密切合作，我們希望確定 BioJet 平台可以使用他們的分子做什麼。

So where we are is we had our research device that worked last year. We evolved that to a next-generation clinical ready device this year. We did an instant trigger mechanism so that everything inside the pill could be tested. We've then, in the last few months, added on the autonomous trigger. Because last year we had already used an autonomous trigger.

所以我們的研究設備去年就發揮了作用。今年我們將其發展為下一代臨床就緒設備。我們採用了即時觸發機制，以便可以測試藥丸內的所有內容。然後，在過去的幾個月裡，我們增加了自主觸發器。因為去年我們已經使用了自主觸發器。

So we've got now data with the autonomous trigger that looks like, yes, it's working. We can get the autonomous triggered work with the device. We've generated some data in a few weeks or less, we will have additional data that will now make a robust-enough package to talk with our collaborators, the existing ones and others, and start using their molecules.

現在我們已經獲得了帶有自主觸發器的數據，看起來，是的，它正在工作。我們可以讓設備自主觸發工作。我們在幾週或更短的時間內產生了一些數據，我們將獲得更多數據，這些數據現在將形成一個足夠強大的包，以便與我們的合作者、現有合作者和其他人進行交談，並開始使用他們的分子。

So one of them has already allowed us to use. So we have an antisense. I think this is the one name we've been allowed to use as Ionis. And I say allowed to use as the others. We want to work with them in full trust of their requirements, which is we can share their names as we go further.

所以其中一個已經允許我們使用。所以我們有一個反義。我認為這是我們被允許使用的唯一一個名字：Ionis。我說允許像其他人一樣使用。我們希望與他們合作，並充分信任他們的要求，即我們可以在進一步發展時分享他們的名字。

But we have used an antisense device, antisense molecule. We'll use other molecules from those collaborators. We will have that data in the coming weeks months. We think that information will allow us to go into something of a more meaningful partnership with these or other collaborators. And we think that is the first step of establishing what the BioJet platform can do.

但我們使用了反義裝置，反義分子。我們將使用這些合作者的其他分子。我們將在未來幾週內獲得這些數據。我們認為這些資訊將使我們能夠與這些或其他合作者建立更有意義的合作夥伴關係。我們認為這是建立 BioJet 平台功能的第一步。

And then proceed with the molecules that we do have in our own trials, and our own plans, for our own molecules later on. So in the near-term, we want to make sure that we get some confirmatory autonomous trigger data in the next short few weeks. And start working with the collaborators molecules in the next few months. And then progress to meaningful partnerships with these or other collaborators in the coming months.

然後繼續研究我們在自己的試驗中確實擁有的分子，以及我們自己的計劃，稍後我們自己的分子。因此，在短期內，我們希望確保在接下來的幾週內獲得一些確認性的自主觸發數據。並在接下來的幾個月內開始與合作者分子合作。然後在未來幾個月內與這些或其他合作者建立有意義的合作關係。

And that's what we have planned. We want to make sure that we stay focused. We say resource efficient until we get to next year, where we can expand both the platforms, right? By then, we will have confirmation of both our platforms and organically be able to expand our pipeline substantially as our resources grow.

這就是我們的計劃。我們希望確保我們保持專注。我們說資源高效，直到明年我們可以擴展這兩個平台，對嗎？到那時，我們將確認我們的兩個平台，並隨著我們的資源成長，有機地能夠大幅擴展我們的管道。

Great. And then regarding the collaborations. Do any of those collaborations that I need from working in certain therapeutic areas with other prospective partners?

偉大的。然後是關於合作。我是否需要在某些治療領域與其他潛在合作夥伴合作？

Yeah, that's a good question. At this point, we have not allowed ourselves to be tied down that way. So unless we get to a meaningful partnerships, we will not allow ourselves to be tied down. These give us plenty of room to do anything that we need to do at this point. And hopefully, we will be able to get further down the road where we can talk about which areas we are constraining ourselves from, because we've struck a different meaningful partnership.

是的，這是一個好問題。在這一點上，我們不允許自己被這樣束縛。因此，除非我們建立有意義的合作關係，否則我們不會讓自己受到束縛。這些給了我們足夠的空間來做我們目前需要做的任何事情。希望我們能夠進一步討論我們在哪些領域受到限制，因為我們已經建立了不同的有意義的合作關係。

Great. Thank you, Adi. Appreciate it.

偉大的。謝謝你，阿迪。欣賞它。

There are no further questions at this time. I'd like now to turn the floor back over to Mr. Adi, for closing comments. Please, sir, you can go ahead.

目前沒有其他問題。現在我想請阿迪先生發表總結意見。先生，請您繼續。

Thank you all for joining us today. We're encouraged by the progress we're making, and we look forward to keeping you updated on our progress. Have a good evening. Thank you.

感謝大家今天加入我們。我們對所取得的進展感到鼓舞，並期待向您通報我們的最新進展。祝你晚上愉快。謝謝。

This concludes today's teleconference. You might disconnect your lines at this time. Thank you for your participation.

今天的電話會議到此結束。此時您可以斷開線路。感謝您的參與。