Titan Pharmaceuticals Inc (TTNP) 2020 Q3 法說會逐字稿

Thank you for holding, and welcome to the Titan Pharmaceuticals Third Quarter 2020 Financial Results Conference Call. Please be advised that this call is being taped at the company's request and will be archived on the company's website starting later today.

感謝您召開並歡迎參加泰坦製藥 2020 年第三季財務業績電話會議。請注意，本次通話是應公司要求進行錄音的，並將於今天晚些時候開始在公司網站上存檔。

At this time, I would like to turn the call over to Dr. Marc Rubin, Executive Chairman of Titan Pharmaceuticals. Please go ahead.

現在，我想把電話轉給泰坦製藥公司執行主席馬克·魯賓博士。請繼續。

Thank you, Sarah. And as always, thank you to all of you for joining us today. Welcome to the Titan Pharmaceuticals Third Quarter 2020 Conference Call.

謝謝你，莎拉。一如既往，感謝大家今天加入我們。歡迎參加泰坦製藥 2020 年第三季電話會議。

Before we begin, I want to inform you that we have filed our quarterly report on Form 10-Q with the SEC, and the press release issued earlier today provides a summary of the financial and operational results for the third quarter ending September 30, 2020, and can also be found on our website at www.titanpharm.com.

在開始之前，我想通知您，我們已向 SEC 提交了 10-Q 表格季度報告，今天早些時候發布的新聞稿提供了截至 2020 年 9 月 30 日的第三季度財務和運營業績摘要，也可以在我們的網站www.titanpharm.com 上找到。

Joining me on the call today from Titan is Dr. Kate DeVarney, our President and Chief Operating Officer. As you all know, Sunil Bhonsle has recently made the decision to retire from the company. And we would like to take this opportunity to thank him for his unwavering dedication and many, many, many contributions to Titan over the years. We all wish him the best for his very, very well-earned retirement. Moving forward, Kate and I will oversee the company's product development activities.

今天從 Titan 與我一起參加電話會議的是我們的總裁兼營運長 Kate DeVarney 博士。眾所周知，Sunil Bhonsle 最近決定從公司退休。我們想藉此機會感謝他多年來堅定不移的奉獻精神以及為泰坦做出的許多、許多、許多貢獻。我們都祝他在退休後一切順利。展望未來，凱特和我將監督公司的產品開發活動。

The relevance of our prior operating results is diminished, in that Titan has transitioned back from a commercial to a development stage company. So rather discussing -- rather than discussing this today, I'd like to draw your attention to the aforementioned filings. And I'll now turn the call to Jennifer Kiernan, who will review the required cautions regarding forward-looking statements. Jennifer?

我們先前的經營績效的相關性已經減弱，因為泰坦已經從商業公司轉變回開發階段的公司。因此，與其討論——而不是今天討論這個問題，我想提請您注意上述文件。現在我將把電話轉給詹妮弗·基爾南，她將審查有關前瞻性陳述的必要注意事項。詹妮弗？

Thank you, Marc. I want to remind everyone that certain matters that will be discussed today other than historical information may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Such statements include, but are not limited to, any statements relating to our product commercialization and development programs and any other statements that are not historical facts. Such statements involve risks and uncertainties that could negatively affect our business, operating results, financial conditions and stock price.

謝謝你，馬克。我想提醒大家，今天討論的除歷史資訊之外的某些事項可能包含1933 年《證券法》第27A 條和1934 年《證券交易法》第21E 條含義內的前瞻性陳述。 ：但不限於與我們的產品商業化和開發計劃有關的任何陳述以及任何其他不屬於歷史事實的陳述。此類陳述涉及風險和不確定性，可能對我們的業務、經營業績、財務狀況和股價產生負面影響。

Factors that could cause actual results to differ materially from management's current expectations include those risks and uncertainties relating to our ability to raise capital; the winding down of U.S. commercial activities related to Probuphine; the regulatory approval process; the development, testing, production and marketing of our drug candidates; patent and intellectual property matters; and strategic agreements and relationships. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law.

可能導致實際結果與管理層當前預期存在重大差異的因素包括與我們籌集資金的能力相關的風險和不確定性；結束與普羅布啡相關的美國商業活動；監管審批流程；我們候選藥物的開發、測試、生產和行銷；專利和智慧財產權事務；以及戰略協議和關係。我們明確表示不承擔任何義務或承諾公開發布對此處包含的任何前瞻性陳述的任何更新或修訂，以反映我們的期望的任何變化或任何此類陳述所依據的事件、條件或情況的任何變化，除非法律。

I'll now turn the call back over to you, Marc.

我現在將把電話轉回給你，馬克。

Thanks, Jennifer. And we'd like to focus today's call on a brief overview and update of our 2 key assets, JT-09 and nalmefene. We completed a public offering 2 weeks ago and have a meeting of stockholders scheduled for November 30. So the details of all recent developments at Titan are available via a number of recently filed disclosures with the SEC.

謝謝，珍妮佛。我們希望今天的電話會議重點介紹我們的兩種關鍵資產 JT-09 和納美芬的簡要概述和更新。我們在兩週前完成了公開發行，並計劃於 11 月 30 日召開股東大會。

Our recent restructuring aims to position the company for future growth and is expected to result in substantial reductions in our fixed operating costs. It also means that Titan is back to being a development stage company, which really represents our core expertise. And we have 2 key assets, one of which is JT-09, a kappa opioid receptor agonist peptide that we recently acquired from JT Pharma. I will briefly begin by describing this program before turning the call over to Kate, who will go on to give a brief overview of nalmefene.

我們最近的重組旨在使公司為未來的成長做好準備，並預計將大幅降低我們的固定營運成本。這也意味著泰坦重新成為一家發展階段的公司，這真正代表了我們的核心專業知識。我們有兩個關鍵資產，其中一個是 JT-09，這是我們最近從 JT Pharma 收購的 kappa 鴉片受體激動劑勝肽。我將首先簡要描述該計劃，然後將電話轉給凱特，她將繼續簡要概述納美芬。

The acquisition of JT-09 was based on work that we actually began several years ago to assess the feasibility of delivering JT-09 through peptide-infused ProNeura implants in animal models. And we had initially focused this work on looking at providing a nonaddictive treatment for chronic pain. But recently, we have pivoted to focus and explore the feasibility of using JT-09 implants for the treatment of chronic pruritus, initially looking at the potential for treating chronic pruritus in patients with end-stage kidney disease.

JT-09 的收購是基於我們幾年前開始的工作，即評估透過注射到勝肽的 ProNeura 植入物在動物模型中遞送 JT-09 的可行性。我們最初將這項工作的重點放在尋找一種非成癮性的慢性疼痛治療​​方法。但最近，我們重點關注並探索使用 JT-09 植入物治療慢性搔癢的可行性，初步研究了治療末期腎病患者慢性搔癢的潛力。

Chronic pruritus in general is a very debilitating condition, defined as itching of the skin that lasts for longer than 6 weeks, and an estimated 23 million to 44 million Americans suffer from chronic pruritus caused by a number of cutaneous as well as systemic conditions. And current treatments for chronic pruritus include antihistamines, corticosteroids, a number of over-the-counter lotions, all of which are relatively ineffective. And many have undesirable side effect profiles, varying, of course, based on the etiology of the pruritus itself.

一般來說，慢性搔癢症是一種非常令人衰弱的疾病，定義為持續超過6 週的皮膚瘙癢，估計有2300 萬至4400 萬美國人患有由多種皮膚和全身疾病引起的慢性瘙癢症。目前治療慢性搔癢症的方法包括抗組織胺、皮質類固醇、許多非處方洗劑，所有這些都相對無效。許多藥物都有不良副作用，當然，根據搔癢症本身的病因不同，副作用也會有所不同。

The antipruritic effect of kappa opioid agonist is thought to be related to their binding to kappa opioid receptors on keratinocytes, immune cells and peripheral itch neurons. The efficacy of a kappa opioid agonist for chronic pruritus was first demonstrated definitively in humans by Toray Industries using a highly potent small molecule kappa agonist called nalfurafine. Toray's application for nalfurafine was approved in Japan for the treatment of pruritus in end-stage kidney disease and in chronic liver disease.

κ阿片受體激動劑的止癢作用被認為與其與角質形成細胞、免疫細胞和周邊搔癢神經元上的κ阿片受體的結合有關。東麗公司使用一種名為納芙拉芬的高效小分子 kappa 激動劑，首次在人體中明確證明了 kappa 阿片受體激動劑對慢性搔癢的療效。東麗的納芙拉芬申請在日本獲得批准，用於治療末期腎病和慢性肝病引起的搔癢。

Pruritus does affect about 40% of patients who have end-stage kidney disease. So it is very prevalent, and it's associated with very poor quality of life, poor sleep, depression and actually overall increased mortality. Because Toray's nalfurafine is a small molecule that penetrates into the central nervous system, there have been some CNS-related adverse events observed. Nalfurafine for the treatment of chronic pruritus is approved only in Japan.

大約 40% 的末期腎病患者確實會出現搔癢症狀。所以它非常普遍，並且與生活品質非常差、睡眠品質差、抑鬱以及實際上總體死亡率上升有關。由於東麗的納芙拉芬是一種滲透到中樞神經系統的小分子，因此觀察到了一些與中樞神經系統相關的不良事件。用於治療慢性搔癢的納芙拉芬僅在日本獲得批准。

More recently, Cara Therapeutics demonstrated in both Phase II and Phase III clinical trials the efficacy of a selective kappa opioid receptor agonist peptide, they call it CR845, in the treatment of pruritus associated with end-stage kidney disease in patients undergoing dialysis. And Cara has announced plans to submit a new drug application in the U.S. in the second half of 2020.

最近，Cara Therapeutics 在 II 期和 III 期臨床試驗中證明了選擇性 kappa 鴉片受體激動劑勝肽（他們稱之為 CR845）在治療透析患者與末期腎臟病相關的搔癢方面的功效。卡拉已宣布計劃於2020年下半年在美國提交新藥申請。

We believe that based on our early animal data, subcutaneous implementation of JT ProNeura rods could potentially deliver therapeutic concentrations of JT-09 for 6 months or longer following a single in-office procedure. This could potentially eliminate the need for multiple weekly injections by delivering relatively nonfluctuating therapeutic levels of medication over a long period of time. We are now working towards establishing proof-of-concept of the JT-09 peptide, and we expect to get this proof-of-concept data in the second quarter of 2021. If successful, we then plan on conducting investigational new drug enabling safety and pharmacology studies. Of course, we will keep you updated on our progress and share key data as we generate it.

我們相信，根據我們的早期動物數據，皮下注射 JT ProNeura 棒有可能在單次診室手術後提供 6 個月或更長時間的 JT-09 治療濃度。透過在很長一段時間內提供相對穩定的治療水平的藥物，這可能會消除每週多次注射的需要。我們現在正在努力建立 JT-09 肽的概念驗證，我們預計在 2021 年第二季度獲得該概念驗證數據。當然，我們會及時向您通報我們的進展，並在產生關鍵數據時分享這些數據。

I now would like to pass the call to Kate, who's going to discuss our nalmefene development program. Kate?

我現在想把電話轉給凱特，她將討論我們的納美芬發展計畫。凱特？

Thank you, Marc.

謝謝你，馬克。

As you know, we are developing a nalmefene implant for the prevention of opioid relapse following detoxification. This program is based in part on FDA's 1995 approval of an injectable formulation of nalmefene for the management and reversal of opioid overdose, including respiratory depression. In addition, oral nalmefene was approved by the European Medicines Agency in 2013 for treating alcohol dependence.

如您所知，我們正在開發納美芬植入物，用於預防戒毒後鴉片類藥物復發。該計劃部分基於 FDA 1995 年批准的納美芬注射製劑，用於治療和逆轉阿片類藥物過量（包括呼吸抑制）。此外，口服納美芬於2013年獲得歐洲藥品管理局核准用於治療酒精依賴。

In September of 2019, the National Institute on Drug Abuse, or NIDA, awarded us approximately $8.7 million grant over 2 years for this program. The NIDA grant provides funds for the completion of implant formulation development, GMP manufacturing and the nonclinical studies required for filing an IND. We are in regular communication with NIDA regarding the progress of this program, and we remain on track to meet our next milestone, which is filing the IND in the first half of 2021.

2019 年 9 月，美國國家藥物濫用研究所 (NIDA) 在兩年內為該計畫提供了約 870 萬美元的資助。 NIDA 撥款為完成植入物配方開發、GMP 製造以及提交 IND 所需的非臨床研究提供資金。我們正在與 NIDA 就該計劃的進展情況進行定期溝通，並且我們仍有望實現下一個里程碑，即在 2021 年上半年提交 IND。

Now acceptance of the IND by the FDA will allow us to apply to NIDA for the remaining portion of the grant, which is approximately $6.3 million. We have received clear guidance from the FDA that this product should follow the 505(b)(1) regulatory approval pathway, and that's due to the lack of safety data on nalmefene for a long-acting formulation. The FDA also advised on the nonclinical studies that will be required to file the IND. So based on this input, we plan to conduct an additional study as well as to increase the duration of an ongoing study and collect all the nonclinical chronic toxicology data that's required. NIDA has accepted our plan to reallocate previously approved funds to conduct these studies.

現在 FDA 接受 IND 將使我們能夠向 NIDA 申請剩餘部分的撥款，大約為 630 萬美元。我們已收到 FDA 的明確指導，該產品應遵循 505(b)(1) 監管審批途徑，這是由於缺乏長效製劑納美芬的安全數據。 FDA 也就提交 IND 所需的非臨床研究提供了建議。因此，根據這一意見，我們計劃進行一項額外的研究，並延長正在進行的研究的持續時間，並收集所需的所有非臨床慢性毒理學數據。 NIDA 已接受我們重新分配先前批准的資金來進行這些研究的計劃。

So in summary, we believe that our new corporate strategy refocused on the potential of our ProNeura platform has both near and long-term value-generating opportunities. We're working toward achieving development milestones in both of our ProNeura-based programs by the middle of next year and look forward to updating you on our progress.

總而言之，我們相信，我們的新企業策略重新聚焦於 ProNeura 平台的潛力，具有近期和長期的價值創造機會。我們正在努力在明年年中之前實現兩個基於 ProNeura 的專案的開發里程碑，並期待向您通報我們的最新進展。

Thank you all for participating on this call. As always, we really appreciate your ongoing support, and we look forward to reporting continued progress in our ProNeura portfolio as we move forward. Thank you.

感謝大家參與本次電話會議。一如既往，我們非常感謝您持續的支持，我們期待在我們前進的過程中報告 ProNeura 產品組合的持續進展。謝謝。

The conference has now concluded. Thank you for attending today's presentation. You may now disconnect.

會議現已結束。感謝您參加今天的演講。您現在可以斷開連線。