aTyr Pharma Inc (ATYR) 2023 Q4 法說會逐字稿

Good afternoon, ladies and gentlemen, and welcome to the aTyr Pharma fourth quarter and full year 2023 conference call.

女士們、先生們，下午好，歡迎參加 aTyr Pharma 2023 年第四季和全年電話會議。

At this time, all participants are in a listen only mode.

此時，所有參與者都處於只聽模式。

(Operator Instructions) As a reminder, this conference is being recorded for replay purposes.

（操作員說明）謹此提醒，本次會議正在錄製以供重播之用。

It is now my pleasure to hand the conference call over to Ashlee Dunston, aTyr's Director of Investor Relations and Public Affairs.

現在我很高興將電話會議交給 aTyr 投資者關係和公共事務總監 Ashlee Dunston。

Ms. Dunston, you may begin.

鄧斯頓女士，您可以開始了。

Thank you, and good afternoon, everyone.

謝謝大家，大家下午好。

Thank you for joining us today to discuss aTyr's fourth quarter and full year 2023 operating results and corporate updates.

感謝您今天加入我們討論 aTyr 第四季和 2023 年全年營運表現和公司最新動態。

We are joined today by Dr. Sanjay Shukla, our President and CEO, and Ms. Jill Broadfoot, our CFO.

今天，我們的總裁兼執行長 Sanjay Shukla 博士和財務長 Jill Broadfoot 女士也加入了我們的行列。

On the call, Sanjay will provide an update on our corporate strategy, including our clinical program for Efzofitimod and research and discovery program.

在電話會議上，桑傑將提供我們公司策略的最新訊息，包括我們的 Efzofitimod 臨床計劃以及研究和發現計劃。

Jill will review our financial results and our current financial position before handing it back to Sanjay to open the call up for any questions.

吉爾將審查我們的財務業績和當前的財務狀況，然後將其交還給桑傑以提出任何問題。

Before we begin, I would like to remind everyone that except for statements of historical facts, the statements made by management and responses to questions on this conference call are forward-looking statements under the Safe Harbor provisions of the Private Securities Litigation Reform Act of 1995.

在開始之前，我想提醒大家，除了歷史事實的陳述外，管理層在本次電話會議上的陳述和對問題的回答均屬於1995年《私人證券訴訟改革法案》安全港條款下的前瞻性陳述。

These statements involve risks and uncertainties that can cause actual results to differ materially from those in such forward-looking statements.

這些陳述涉及風險和不確定性，可能導致實際結果與此類前瞻性陳述中的結果有重大差異。

Please see the forward-looking statement disclaimer in the company's press release issued this afternoon as well as the risk factors in the company's SEC filings and included in our most recent Annual Report on Form 10-K subsequently filed Quarterly Reports on Form 10-Q and in our other SEC filings.

請參閱今天下午發布的公司新聞稿中的前瞻性聲明免責聲明，以及公司向SEC 提交的文件中包含的風險因素，以及我們最新的10-K 表格年度報告以及隨後提交的10-Q 表格季度報告中的風險因素。

Undue reliance should not be placed on forward-looking statements, which speak only as of the day they are made as facts and circumstances underlying these forward-looking statements may change.

不應過度依賴前瞻性聲明，這些聲明僅代表發布之日的情況，因為這些前瞻性聲明所依據的事實和情況可能會發生變化。

Except as required by law, aTyr Pharma disclaims any obligation to update these forward-looking statements to reflect future information events or circumstances.

除法律要求外，aTyr Pharma 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。

I will now turn the call over to Sanjay.

我現在將把電話轉給 Sanjay。

Thank you, Ashlee.

謝謝你，阿什莉。

Good afternoon, everyone, and thank you for joining us for our fourth quarter and full year 2023 results conference call.

大家下午好，感謝您參加我們的 2023 年第四季和全年業績電話會議。

At aTyr, we are leveraging evolutionary intelligence to translate tRNA synthetase biology into new therapies for fibrosis and inflammation.

在 aTyr，我們正在利用進化智能將 tRNA 合成酶生物學轉化為纖維化和發炎的新療法。

Our lead therapeutic candidate, Efzofitimod, is a first-in-class biologic immunomodulator based on a naturally occurring, long enriched splice variant of the tRNA synthetases, HARS.

我們的主要候選治療藥物 Efzofitimod 是一種一流的生物免疫調節劑，基於天然存在的、長富集的 tRNA 合成酶剪接變體 HARS。

Efzofitimod selectively modulates activated myeloid cells via neuropilin-2 or NRP-2 to resolve inflammation without immune suppression and potentially prevent the progression of fibrosis.

Efzofitimod 透過 Neuropilin-2 或 NRP-2 選擇性調節活化的骨髓細胞，在不抑制免疫的情況下解決炎症，並可能防止纖維化的進展。

We're developing Efzofitimod as a treatment for patients with interstitial lung disease or ILD, a group of rare immune-mediated disorders that can cause chronic inflammation and fibrosis of the lungs.

我們正在開發 Efzofitimod 作為治療間質性肺病或 ILD 患者的藥物，這是一組罕見的免疫介導疾病，可導致肺部慢性發炎和纖維化。

2023 was an important year for aTyr as we progressed and expanded our Efzofitimod clinical development program, which now includes two ongoing clinical studies, the Phase 3 EFZO-FIT study in patients with pulmonary sarcoidosis, a major form of ILD; and the Phase 2 EFZO-CONNECT study in patients with ILD related to systemic sclerosis, which is known as SSc or more commonly, scleroderma.

2023 年對aTyr 來說是重要的一年，因為我們取得了進展並擴大了Efzofitimod 臨床開發計劃，該計劃目前包括兩項正在進行的臨床研究：針對肺結節病（ILD 的一種主要形式）患者的3 期EFZO-FIT 研究；以及針對肺結節病（ILD 的一種主要形式）患者的3 期EFZO-FIT 研究。 EFZO-CONNECT 2 期研究針對與系統性硬化症相關的 ILD 患者，這種疾病被稱為 SSc，或更常見的是硬皮症。

Throughout the past year, we have also greatly enhanced our mechanistic understanding of the way in which Efzofitimod is confirming its anti-inflammatory effects.

在過去的一年裡，我們也大大增強了對 Efzofitimod 確認其抗發炎作用的機制的理解。

NRP-2 is highly expressed on activated immune cells doing an inflammatory response, notably myeloid cells, including monocytes and macrophages.

NRP-2 在產生發炎反應的活化免疫細胞上高度表達，特別是骨髓細胞，包括單核細胞和巨噬細胞。

By binding NRP-2, Efzofitimod guides the differentiation of monocytes at the site of inflammation into a macrophage subtype that is less pro-inflammatory to resolve aberrant inflammation.

透過結合 NRP-2，Efzofitimod 引導發炎部位的單核細胞分化為促發炎性較低的巨噬細胞亞型，從而解決異常發炎。

Dysregulated inflammation is a hallmark of myelo-driven diseases such as ILD, where persistent, uncontrolled inflammation can lead to the progression of fibrosis.

發炎失調是骨髓驅動疾病（例如間質性肺病）的一個標誌，持續的、不受控制的發炎會導致纖維化進展。

With this new understanding, we now have even greater clarity and confidence as to why Efzofitimod may represent a breakthrough in treatment for ILD.

有了這個新的認識，我們現在對為什麼 Efzofitimod 可能代表 ILD 治療的突破有了更加清晰和信心。

Our lead indication for Efzofitimod is pulmonary sarcoidosis, the most prevalent form of ILD, where approximately 70% of patients will have symptomatic disease and nearly 20% will develop lung fibrosis.

我們 Efzofitimod 的主要適應症是肺結節病，這是最常見的 ILD 形式，大約 70% 的患者會出現症狀性疾病，近 20% 的患者會出現肺纖維化。

Current standard of care is primarily oral corticosteroids, a highly toxic treatment that has limited clinical evidence, is broadly immunosuppressive, and comes with side effects resulting in a high disease burden for patients.

目前的護理標準主要是口服皮質類固醇，這是一種劇毒治療方法，臨床證據有限，具有廣泛的免疫抑製作用，並具有副作用，導致患者承受較高的疾病負擔。

EFZO-FIT is a global pivotal Phase 3 study evaluating Efzofitimod compared to placebo in the context of a four steroid taper in patients with pulmonary sarcoidosis.

EFZO-FIT 是一項全球關鍵 3 期研究，在肺結節病患者逐漸減少四種類固醇的情況下，評估 Efzofitimod 與安慰劑的比較。

This study is currently enrolling at more than 90 centers in nine countries.

目前，這項研究正在 9 個國家的 90 多個中心進行招募。

We're pleased with the progress we've made thus far with this study, which is expected to be the largest interventional study ever conducted in sarcoidosis.

我們對這項研究迄今為止所取得的進展感到高興，預計這將是有史以來針對結節病進行的最大規模的介入研究。

Completing enrollment in EFZO-FIT is our primary focus and we anticipate doing so in the second quarter of this year.

完成 EFZO-FIT 的註冊是我們的首要任務，我們預計在今年第二季完成。

In the past few months as patients have completed the 52-week EFZO-FIT study, we've received multiple inquiries from study principles -- study principal investigators, or PIs, whose patients have requested to continue treatment once they completed the trial.

在過去幾個月，隨著患者完成為期 52 週的 EFZO-FIT 研究，我們收到了來自研究負責人（研究主要研究者或 PI）的多次詢問，他們的患者要求在完成試驗後繼續治療。

While aTyr PIs and patients are all blinded to what treatment patients received as part of the study, either Efzofitimod or placebo, the feedback we've received has suggested that some patients have performed well and want to continue on study drug rather than returning to the treatment regimen they had prior to the study.

雖然 aTyr PI 和患者都對患者在研究中接受的治療（Efzofitimod 或安慰劑）一無所知，但我們收到的反饋表明，一些患者表現良好，希望繼續使用研究藥物，而不是返回治療他們在研究之前接受的治療方案。

For some patients that may entail resuming or increasing steroid dose, which many patients are reluctant to do.

對於某些患者來說，這可能需要恢復或增加類固醇劑量，但許多患者不願意這樣做。

Based on this feedback, we decided to implement an individual patient expanded access program or TAP for patients who complete EFZO-FIT.

根據此回饋，我們決定為完成 EFZO-FIT 的患者實施個別患者擴展訪問計劃或 TAP。

This individual patient EAP is design to allow access to Efzofitimod for patients who all have or are in the process of completing EFZO-FIT beyond the duration of the clinical trial.

此個別患者 EAP 旨在允許所有已經或正在完成 EFZO-FIT 的患者在臨床試驗結束後獲得 Efzofitimod。

The company PIs and patients will remain blinded to treatment that occurred as part of EFZO-FIT.

公司 PI 和患者對作為 EFZO-FIT 一部分的治療將保持不知情。

Safety is a key component of any EAP.

安全性是任何 EAP 的關鍵組成部分。

We were able to implement this program based on the existing safety database from prior Efzofitimod clinical studies and additional safety and tolerability data from a Data Safety & Monitoring Board or DSMB review of data from EFZO-FIT, which included the evaluation of patients that completed 52 weeks of treatment.

我們能夠根據先前Efzofitimod 臨床研究的現有安全性資料庫以及來自資料安全與監測委員會或DSMB 對EFZO-FIT 資料審查的額外安全性和耐受性資料來實施該計劃，其中包括對完成52 例患者的評估數週的治療。

The DSMB review recommended the study proceed without modification, suggesting no major safety concerns.

DSMB 審查建議該研究無需修改即可繼續進行，表明不存在重大安全問題。

And while many types of EAPs are typically implemented after data from a study has been unblinded, we decided to implement this individual patient EAP early not only based on feedback and demand, but in part to continue to support those patients who have dedicated their time and entrusted us with their health by participating in this important study.

雖然許多類型的 EAP 通常是在研究資料揭盲後實施，但我們決定儘早實施個別病患 EAP，不僅是根據回饋和需求，而且部分是為了繼續支持那些投入時間和精力的病患。的研究，將他們的健康託付給我們。

This program reflects our ongoing commitment to the sarcoidosis community as we work to develop a safe and effective treatment for those in need.

該計劃反映了我們對結節病社區的持續承諾，我們致力於為有需要的人開發安全有效的治療方法。

Our second indication for Efzofitimod is SSc-ILD.

我們 Efzofitimod 的第二個適應症是 SSc-ILD。

SSc is a form of connective tissue disease where ILD commonly occurs and is a leading cause of mortality.

SSc 是一種結締組織疾病，其中 ILD 很常見，並且是導致死亡的主要原因。

Current treatment options are limited and like sarcoidosis, do not treat the underlying disease or improve quality of life.

目前的治療選擇有限，與結節病一樣，無法治療潛在疾病或改善生活品質。

EFZO-CONNECT is a Phase 2 proof-of-concept study evaluating Efzofitimod compared to placebo in patients with SSc-ILD.

EFZO-CONNECT 是一項 2 期概念驗證研究，在 SSc-ILD 患者中評估 Efzofitimod 與安慰劑的比較。

This study, which dosed the first patient last quarter, is currently open for enrollment at multiple centers in the US.

這項研究在上個季度對第一位患者進行了給藥，目前正在美國多個中心開放招募。

We're focused on generating data from this study in 2024, and we expect to provide an update on the study later this year.

我們的重點是在 2024 年從這項研究中產生數據，並預計在今年稍後提供該研究的更新。

We estimate that the two indications that comprise our current clinical program for Efzofitimod, pulmonary sarcoidosis and SSc-ILD collectively represent a potential $2 billion to $3 billion global market opportunity.

我們估計，構成我們目前 Efzofitimod 臨床計劃的兩個適應症、肺結節病和 SSc-ILD 共同代表了 20 億至 30 億美元的潛在全球市場機會。

This does not include any upside potential and other forms of the more than 200 ILDs, where Efzofitimod's unique mechanism of action to address complex immune pathology and desirable safety profile may be able to disrupt standard of care.

這不包括 200 多種 ILD 的任何上行潛力和其他形式，在這些 ILD 中，Efzofitimod 解決複雜免疫病理學的獨特作用機制和理想的安全性可能會破壞護理標準。

While our primary focus is our clinical program for Efzofitimod, we continue to leverage our intellectual property or IP estate covering domains from all 20 human tRNA synthetases and utilize our platform as an engine to generate new pipeline candidates.

雖然我們的主要重點是 Efzofitimod 的臨床項目，但我們繼續利用涵蓋所有 20 種人類 tRNA 合成酶領域的智慧財產權或 IP 財產，並利用我們的平台作為引擎來產生新的候選藥物。

tRNA synthetases are ancient essential proteins that have evolved novel domains to regulate diverse pathways, extracellularly, in humans.

tRNA 合成酶是古老的必需蛋白質，已進化出新的結構域來調節人類細胞外的多種途徑。

By identifying extracellular receptors and signaling pathways for these domains, we can elucidate the role these proteins play in cellular response and explore disease areas where they may have therapeutic benefit.

透過辨識這些結構域的細胞外受體和訊號通路，我們可以闡明這些蛋白質在細胞反應中發揮的作用，並探索它們可能具有治療益處的疾病領域。

Our two most advanced tRNA synthetases candidates in preclinical development are ATYR0101 and ATYR0750, both of which have specific interactions with targets that have implications in fibrosis.

我們處於臨床前開發階段的兩種最先進的 tRNA 合成酶候選物是 ATYR0101 和 ATYR0750，這兩種酶都與對纖維化有影響的目標有特定的相互作用。

These targets include latent transforming growth factor beta binding protein one or LTBP1 and fibroblast growth factor receptor four or FGFR4, respectively.

這些標靶分別包括潛在轉化生長因子β結合蛋白一或LTBP1和纖維母細胞生長因子受體四或FGFR4。

ATYR0101, which is derived from a domain of the tRNA synthetases DARS exerts its anti-fibrotic effects by selectively inducing a proptosis of myofibroblasts targeting a key hallmark of fibrosis pathology, which is the persistence of activated myofibroblasts.

ATYR0101 源自tRNA 合成酶DARS 的一個結構域，透過選擇性誘導肌成纖維細胞突出來發揮其抗纖維化作用，該作用針對纖維化病理學的關鍵標誌，即活化的肌成纖維細胞的持續存在。

This mechanism may support broad therapeutic application in indications like lung, liver, and kidney fibrosis.

這種機制可能支持肺、肝和腎纖維化等適應症的廣泛治療應用。

The hidden biology that we have been able to unlock from our platform continues to inspire us, including the way in which some of these appended domains like Efzofitimod interact extracellularly with previously under the radar targets like NRP-2 and in particular, its role as an immune regulator or bind more well-known targets in unique ways like ATYR0101 and ATYR0750.

我們能夠從我們的平台中解開的隱藏生物學繼續激勵我們，包括一些附加域（如 Efzofitimod）與先前雷達目標（如 NRP-2）在細胞外相互作用的方式，特別是它作為免疫調節劑或以獨特的方式結合更多眾所周知的靶標，如ATYR0101 和ATYR0750。

This platform, which is based on signaling pathways that have evolved over billions of years to maintain homeostasis is an excellent example of bio innovation and has the potential to disrupt traditional drug discovery, a process that is increasingly reliant on exploiting existing signaling pathways to generate [Me too] therapies.

該平台基於數十億年進化來維持體內平衡的信號通路，是生物創新的一個很好的例子，並且有可能顛覆傳統的藥物發現，而傳統的藥物發現過程越來越依賴於利用現有的訊號通路來產生[我也是]療法。

Our conviction to the potential of tRNA synthetases biology to lead the transformative medicines continues to grow stronger as our research advances.

隨著我們研究的進步，我們對 tRNA 合成酶生物學引領變革藥物的潛力的信念不斷增強。

I'll now turn it over to our Chief Financial Officer, Jill Broadfoot, to review our financial results.

現在我將把它交給我們的財務長 Jill Broadfoot，以審查我們的財務表現。

Thank you, Sanjay.

謝謝你，桑傑。

We ended 2023 with $101.7 million in cash, restricted cash, cash equivalents and investments.

截至 2023 年底，我們擁有 1.017 億美元的現金、限制性現金、現金等價物和投資。

Collaboration and license revenue related to the Kyorin agreement was $0.4 million for the year ended 2023, which consisted of drug product materials sold to Kyorin for the Japan portion of EFZO-FIT.

截至 2023 年，與 Kyorin 協議相關的合作和授權收入為 40 萬美元，其中包括出售給 Kyorin 的 EFZO-FIT 日本部分的藥品材料。

As a reminder, Kyorin, our partner for the development and commercialization of Efzofitimod demand for ILD in Japan, where they are responsible for costs related to EFZO-FIT in Japan.

提醒一下，Kyorin 是我們在日本 ILD 需求的 Efzofitimod 開發和商業化合作夥伴，他們負責日本 EFZO-FIT 的相關費用。

And purchase drug product material from us with a small markup.

並以小幅加價向我們購買藥品材料。

Under this agreement, we have received $20 million in upfront and milestone payments from this partnership to date, and we are eligible to receive up to an additional $155 million, which is primarily related to certain development and regulatory milestones.

根據該協議，迄今為止，我們已從該合作夥伴關係中收到了2000 萬美元的預付款和里程碑付款，並且我們有資格獲得最多1.55 億美元的額外資金，這主要與某些開發和監管里程碑相關。

Research and development expenses were $42.3 million for the year ended 2023, which consisted primarily of clinical trial costs for EFZO-FIT and EFZO-CONNECT studies manufacturing costs for the Efzofitimod program, and research and development costs for the Efzofitimod and discovery programs.

截至 2023 年底，研發費用為 4,230 萬美元，其中主要包括 EFZO-FIT 和 EFZO-CONNECT 研究的臨床試驗成本、Efzofitimod 專案的製造成本以及 Efzofitimod 和發現專案的研發成本。

General and administrative expenses were $13 million for the year ended 2023.

2023 年終了的年度一般及行政費用為 1,300 萬美元。

Based on our current operational plans and existing cash we, maintain our prior financial guidance and believe our cash runway is expected to be sufficient to fund the company through the filing of a biologics license application for Efzofitimod and pulmonary sarcoidosis.

根據我們目前的營運計劃和現有現金，我們維持先前的財務指導，並相信我們的現金跑道預計足以透過提交埃夫佐菲莫德和肺結節病的生物製劑許可申請為公司提供資金。

This takes into account the continued allocation of the majority of our resources to our Efzofitimod clinical development program, which is our main value driver for the company while also committing judicious resources to our tRNA synthetases pipeline candidates to maintain an active discovery program and advance our IP estate.

這考慮到我們繼續將大部分資源分配給我們的Efzofitimod 臨床開發計劃，這是我們公司的主要價值驅動力，同時也為我們的tRNA 合成酶管道候選者投入了明智的資源，以維持積極的發現規劃並推進我們的知識產權財產。

Furthermore, our forecast for our cash guidance does not include any potential future milestone payments from Kyorin or any proceeds that may result from additional potential partnerships or sources of non-dilutive funding.

此外，我們對現金指引的預測不包括 Kyorin 未來任何潛在的里程碑付款，也不包括額外潛在合作夥伴關係或非稀釋資金來源可能產生的任何收益。

So it does consider our proceeds from the prudent use of our at-the-market facility.

因此，它確實考慮了我們謹慎使用我們的市場設施的收益。

We implemented this operational plan more than a year ago, driven by our emphasis on maximizing efficiency and adapting to prevailing macroeconomic conditions, and this plan continues to be an effective way to meet our primary corporate objectives relative to optimal capital utilization.

出於對效率最大化和適應當前宏觀經濟條件的重視，我們在一年多前實施了這項營運計劃，並且該計劃仍然是實現我們與最佳資本利用相關的主要企業目標的有效方式。

Now, I'd like to turn the call back over to Sanjay before we open it up to Q&A.

現在，在我們進行問答之前，我想將電話轉回給 Sanjay。

Thanks, jill.

謝謝，吉爾。

As we look back on the past year, we're filled with optimism for the future.

回顧過去的一年，我們對未來充滿樂觀。

Our scientific expertise in understanding tRNA biology is unparalleled across the industry.

我們在了解 tRNA 生物學方面的科學專業知識在整個行業中是無與倫比的。

We're leveraging this unique biology and harnessing Ancient genetic codes developed throughout the course of billions of years in a trillion species.

我們正在利用這種獨特的生物學，並利用數萬億個物種在數十億年的過程中開發的古代遺傳密碼。

While other companies are just beginning to explore the untapped potential of tRNA biology we're leading the way with our decades of understanding this science, which we have leveraged to progress a Phase 3 therapeutic candidate.

雖然其他公司才剛開始探索 tRNA 生物學尚未開發的潛力，但我們憑藉數十年對這門科學的了解而處於領先地位，我們已利用這些知識來開發 3 期治療候選藥物。

It's not just our understanding of the endless frontier of tRNA synthetase biology that sets us apart.

讓我們與眾不同的不僅僅是我們對 tRNA 合成酶生物學無盡前沿的理解。

Scleroderma represents the reality, not just the concept, of a potential near-term, meaningful tRNA-based therapy that can change the lives of patients.

硬皮症代表了一種潛在的近期、有意義的基於 tRNA 的療法的現實，而不僅僅是概念，這種療法可以改變患者的生活。

We're on the cusp of a once-in-a-generation therapy for sarcoidosis engineered from our natural physiology and are marching towards the chance to transform the lives of ILD patients.

我們正處於根據自然生理學設計的千載難逢的結節病療法的風口浪尖，並正在朝著改變 ILD 患者生活的機會邁進。

tRNA sarcoidosis is only the starting point for how have Efzofitimod may be able to help millions of ILD patients.

tRNA 結節病只是 Efzofitimod 如何幫助數百萬 ILD 患者的起始點。

As the only biotech company in Phase 3 development for this indication, we note the accelerating pipeline of candidates not just for sarcoidosis, but also for ILD more broadly.

作為唯一一家處於此適應症第三階段開發的生物技術公司，我們注意到，不僅針對結節病，而且針對更廣泛的間質性肺病，候選藥物的研發管線正在加速。

Other biopharmaceutical companies who are following our lead also see the multibillion-dollar market opportunity in this space.

其他跟隨我們腳步的生物製藥公司也看到了這個領域數十億美元的市場機會。

While these companies have yet to show proof of concept have Efzofitimod well beyond that, with patients requesting to remain on treatment as they complete our current trial in sarcoidosis due to the benefits they are experiencing.

雖然這些公司尚未證明 Efzofitimod 的概念證明遠遠超出了這一範圍，但患者在完成我們目前的結節病試驗時要求繼續接受治療，因為他們正在體驗到療效。

So we believe it's just the beginning for us here at aTyr.

因此，我們相信這對 aTyr 來說只是一個開始。

New validated targets are emerging from our evolutionary intelligence or AI driven drug discovery platform.

我們的進化智慧或人工智慧驅動的藥物發現平台正在出現新的經過驗證的目標。

Alternatively, artificial intelligence or AI driven drug discovery is really in its infancy, and these unproven hyped approaches do not have the advantage of our AI drug discovery engine. aTyr is uncovering hidden functions embedded in our genetic code that were developed throughout evolution encompassing infinite real-life biological experiments in over a trillion species over billions of years.

另外，人工智慧或人工智慧驅動的藥物發現確實還處於起步階段，這些未經證實的大肆宣傳的方法並不具備我們人工智慧藥物發現引擎的優勢。 aTyr 正在揭示嵌入在我們遺傳密碼中的隱藏功能，這些功能是在進化過程中開發出來的，涵蓋了數十億年來在超過萬億個物種中進行的無數現實生活生物實驗。

Just think about that.

想想看。

The synthetase domains that we have mapped have developed over billions of years since the beginning of the tree of life.

我們繪製的合成酶結構域自生命樹誕生以來已經發展了數十億年。

Since the evolution of complex systems, all species, including humans share these same genetic domains that were created over billions of years of biological stress and strain, allowing all species to thrive, and overcome disease and dysfunction.

自從複雜系統的進化以來，包括人類在內的所有物種都共享這些相同的遺傳域，這些遺傳域是經過數十億年的生物壓力和應變而產生的，使所有物種都能蓬勃發展，並克服疾病和功能障礙。

As companies embark on computational models and approaches to quote-unquote, find new and novel targets. aTyr meanwhile has an IP library of hundreds of potentially efficacious proteins just waiting to be unleashed from our own genetic programming, leveraging novel domains that have been tested and validated since the beginnings of life, that we now look to target towards current day disease and dysfunction.

隨著公司開始採用計算模型和方法來引用-取消引用，尋找新的和新穎的目標。同時，aTyr 擁有一個包含數百種潛在有效蛋白質的IP 庫，正等待從我們自己的基因編程中釋放出來，利用自生命之初就經過測試和驗證的新結構域，我們現在希望將其針對當今的疾病和功能障礙。

I hope you can now better understand why we're so optimistic here at aTyr, not only in the short term with Efzofitimod, but also in the long-term potential aTyr to become the next transformative biotech company of our time.

我希望您現在能夠更好地理解為什麼我們對 aTyr 如此樂觀，不僅是對 Efzofitimod 的短期前景，而且是對 aTyr 成為我們這個時代下一個變革性生物技術公司的長期潛力。

We appreciate your interest at this time.

我們感謝您此時的興趣。

Jill and I will be happy to take your questions.

吉爾和我很樂意回答您的問題。

(Operator Instructions) Our first question comes from Gregory Renza with RBC Capital Markets.

（操作員說明）我們的第一個問題來自 RBC 資本市場的 Gregory Renza。

You may proceed.

您可以繼續。

Great.

偉大的。

Good afternoon, Sanjay, and Jill.

下午好，桑杰和吉爾。

Congrats on the progress.

祝賀取得的進展。

Thanks for the updates and thanks for taking my questions.

感謝您的更新並感謝您提出我的問題。

And Sanjay, maybe just going to the expanded access program, it's great to hear the color you've provided.

Sanjay，也許只是參加擴展訪問計劃，很高興聽到您提供的顏色。

I Just wanted to ask if you could maybe put into context the significance of this to you and to Efzofitimod, especially as these blinded patients are undergoing the considerable taper for the trial?

我只是想問您是否可以具體說明一下這對您和埃夫佐菲莫德的重要性，特別是當這些失明患者正在經歷相當大的試驗減量時？

And then secondly, just any other details that you are seeing, whether it's the patterns from the centers where that or what that demand is in order to have these patients and these trial participants to continue and with respect to the program and then, I have a follow-up as well.

其次，您所看到的任何其他細節，無論是中心的模式還是需求是什麼，以便讓這些患者和這些試驗參與者繼續進行該計劃，然後，我還有後續行動。

Thanks.

謝謝。

Sure, Greg, thanks for the question.

當然，格雷格，謝謝你的提問。

So really over the last, I would say six months, if you will we have been hearing I've been hearing directly from the call, I'd say at this point, dozens of PIs around the world about how happy they are with the performance of the study drug of the study drug.

所以，實際上，在過去的六個月裡，如果你願意的話，我們一直在直接從電話中聽到，我會說，在這一點上，世界各地的數十名 PI 都表達了他們對研究藥物的性能。

I'm going to highlight say that over and over again, because we don't know if successful it's Efzofitimod or Placebo, and how their patients are performing quite well in the trial.

我要一遍又一遍地強調這一點，因為我們不知道 Efzofitimod 或安慰劑是否成功，以及他們的患者在試驗中表現如何。

It had reached the point however, that patients now rolling off the trial, frankly, don't want to go back on steroids if they have been able to taper off or increase their steroids.

然而，坦白說，現在已經結束試驗的患者如果能夠逐漸減少或增加類固醇，就不想再重新使用類固醇。

Many of these patients have been on steroids for five, 10, sometimes 20 years.

其中許多患者已經服用類固醇 5 年、10 年，有時甚至 20 年。

So this has offered a real unique opportunity in this trial to live their lives with a lot less or no steroids.

因此，這在這次試驗中提供了一個真正獨特的機會，讓他們在生活中少吃或不吃類固醇。

So it had gotten to the point where I thought it's best for us to even think about MEAP really early on.

因此，我認為我們最好儘早考慮 MEAP。

Most of these programs are put in place after you unblind and you finish the trial.

大多數這些計劃是在您解除盲法並完成試驗後實施的。

But with data, obviously, we're looking at data next year.

但就數據而言，顯然我們會關註明年的數據。

We wanted to be able to really fulfill our mission to really be patient oriented patient first and look to implement this program.

我們希望能夠真正履行我們的使命，真正以患者為中心，患者至上，並希望實施該計劃。

Since we've put out the note, I think it's been received even with more interest.

自從我們發布了這張紙條以來，我認為它已經被人們更感興趣了。

This is certainly something that is a relief to a lot of these patients.

這無疑讓許多患者鬆了一口氣。

And I do think that it's a typical for a company to do this sort of thing ahead of data release.

我確實認為公司在數據發布之前做這種事情是很典型的。

I would much rather have patients wanting to remain in the trial and happy to get out of the trial, put it that way.

我更希望患者願意留在試驗中並樂意退出試驗，這樣說吧。

So I think for me clinically when I think about this clinically.

所以當我從臨床角度思考這個問題時，我會從臨床角度去思考。

I think we're doing the right thing here, ethically as well.

我認為我們在這裡做的是正確的事情，在道德上也是如此。

And I think it's probably the best biomarker you could ask for in my opinion.

我認為這可能是我認為最好的生物標記。

Yeah, that's helpful appreciate the color.

是的，這對欣賞顏色很有幫助。

And then just maybe shifting to EFZO-CONNECT just thinking about that, trial open for enrollment across these multiple centers.

然後考慮到這一點，可能會轉向 EFZO-CONNECT，在這些多個中心開放試用。

And that I think I heard you mention an update should come later this year.

我想我聽到你提到應該在今年晚些時候進行更新。

Just with respect to that uptake, what is your objective there?

就這種吸收而言，您的目標是什麼？

Of course, we're all interested in the 12-week skin assessment as well but just curious how we should be thinking about that.

當然，我們也對 12 週的皮膚評估感興趣，但只是好奇我們應該如何考慮這一點。

Is it more procedural in nature?

它本質上更加程序化嗎？

Or is it something where like to at least look and see some details of that program.

或至少想看看該程式的一些細節。

Thanks again and Congrats.

再次感謝並恭喜。

Sure, so we're just in that phase now where I think you'll see six, eight, 10 centers up and ready to enroll at this point.

當然，所以我們現在正處於這個階段，我想你會看到 6、8、10 個中心已經準備好註冊。

Getting that first patient in last quarter was important, but now we're getting into the meat of these sites being able to enroll.

上個季度獲得第一位患者很重要，但現在我們正在深入了解這些網站能夠註冊的內容。

And like I said, we'll have a good idea later this year around what kind of data we'll put out here.

就像我說的，我們將在今年晚些時候關於我們將在這裡發布什麼樣的數據有一個好主意。

There are opportunities with that data set to look at things, even a three month endpoint, which is some of the skin scoring that you pointed out there.

該資料集有機會觀察事物，甚至是三個月的終點，這是您在那裡指出的一些皮膚評分。

But like I said, I want to see how we do here in the first half of the year with regards to enrolling in that trial now that we are approaching more or less full activation or nearing full activation of the centers that we want to get up and enrolling and screening for that trial.

但就像我說的，我想看看我們在今年上半年在參加該試驗方面做得如何，因為我們正在或多或少地接近完全激活或接近完全激活我們想要建立的中心以及該試驗的招募和篩選。

Got it, thanks again.

明白了，再次感謝。

Thanks.

謝謝。

Thank you.

謝謝。

One moment for questions.

請稍等一下提問。

Our next question goes from Joe Pantginis with H.C. Wainwright, and you may proceed, everybody.

我們的下一個問題來自 Joe Pantginis 和 H.C.溫賴特，大家可以繼續了。

Hey everybody, good afternoon.

大家好，下午好。

Thanks for taking the questions.

感謝您提出問題。

Sanjay, I want to start more, start first also with the EAP.

Sanjay，我想開始更多，也先從 EAP 開始。

So it's pretty intriguing, like you said, to be able to have an EAP ahead of data.

因此，正如您所說，能夠在資料之前擁有 EAP 是非常有趣的。

And if I heard you correctly, both physicians and patients remain blinded even after exiting EFZO-FIT as to what they had been previously on.

如果我沒聽錯的話，即使在退出 EFZO-FIT 後，醫生和患者仍然對他們之前的經歷一無所知。

So first, was curious if you could provide any more color or anecdotes that the physicians are giving you with regard to wanting to be into the EAP.

首先，我想知道您是否可以提供醫生為您提供的關於想要加入 EAP 的更多資訊或軼事。

You alluded to, maybe quote that they were, patients are feeling better, but are there any more details you can share around that?

您提到，也許引用他們的話，患者感覺好多了，但是您是否可以分享更多細節？

Second, what are the general logistics of setting up an EAP?

其次，設立 EAP 的一般流程是什麼？

And related to that, what are the cost implications?

與此相關的是，成本影響是什麼？

Thanks.

謝謝。

Yeah, great questions, Joe. So I think just at a high level without sort of getting into specific endpoints, what it really comes down to is these physicians have to put these patients have to put these patients back on something which was presumably steroids as they entered the trial.

是的，很好的問題，喬。因此，我認為，在沒有進入具體終點的情況下，真正歸結為這些醫生必須讓這些患者重新服用一些在他們進入試驗時可能是類固醇的藥物。

And now the patients are refusing to want to do that.

現在患者拒絕這樣做。

So it speaks to the lack of options that we have here for sarcoidosis.

因此，這說明我們缺乏治療結節性多發性硬化症的選擇。

These patients, of course, are not so fibrotic that they need the antifibrotics, so now there's a quandary.

當然，這些患者的纖維化程度還沒有嚴重到需要抗纖維化藥物的程度，所以現在有一個困境。

Patients are doing well, and they've tasted kind of the best experience recently in their recent care, whether it's removing steroids partially or completely.

患者狀況良好，他們在最近的護理中嚐到了最好的體驗，無論是部分還是完全去除類固醇。

So I think we have to sort of step up here because many of the experts were sort of in a quandary now.

所以我認為我們必須採取行動，因為許多專家現在都陷入了困境。

So I think it speaks to the fact that, as I said, this is a really strong biomarker in my mind to have this kind of demand from centers and from patients.

所以我認為這說明了這樣一個事實：正如我所說，在我看來，來自中心和患者的這種需求是一個非常強大的生物標記。

With regards to how would this logistically be administered here in the US, it typically is a direct submission from the PI.

至於在美國如何進行後勤管理，通常是 PI 直接提交。

I would think of this as more like classical compassionate use as opposed to some of the EAPs we know of when drugs are approved and we're waiting for them to get to the market.

我認為這更像是經典的同情用藥，而不是我們所知道的一些藥物獲得批准並等待它們進入市場時的 EAP。

So this would be an individual PI at their center cross-referencing our ILD and submitting into the FDA.

因此，這將是他們中心的個人 PI 交叉引用我們的 ILD 並提交給 FDA。

Because we have some of those positive risk evaluations from a DSMB perspective, we feel as though we're also in good shape to support this vertical.

因為我們從 DSMB 的角度進行了一些積極的風險評估，所以我們覺得我們也處於支持這一垂直領域的良好狀態。

And we may provide small grants to kind of help patients with some of their travel or maybe even some infusion costs.

我們可能會提供小額贈款，以幫助患者支付部分旅行費用，甚至可能支付一些輸液費用。

That's going to be on a case-by-case level.

這將根據具體情況而定。

But this will be something that the centers themselves will bear a little bit more of the financial burden, if you will.

但如果你願意的話，中心本身將承擔更多的財務負擔。

We'll provide drug for free and we're in good shape from a drug supply perspective.

我們將免費提供藥品，從藥品供應的角度來看，我們的狀況良好。

But I do not expect, nor should any of the investors expect, that this will materially impact really our cash position.

但我並不期望，任何投資者也不應該期望這會對我們的現金狀況產生實質影響。

We have the drug, we have the drug supply.

我們有藥物，我們有藥物供應。

We'll help out with some administrative templates and things like that and maybe perhaps some small stipends to help patients.

我們將幫助提供一些管理模板和類似的東西，也許還提供一些小額津貼來幫助患者。

When you look at Europe, you look at other places around the world, that's going to be really a little bit more specific.

當你看看歐洲時，你看看世界上的其他地方，這確實會更具體一些。

Each country, for example, in Europe, some of them have more formal pathways that are more streamlined, similar to here in the US.

每個國家，例如在歐洲，其中一些國家有更正式、更精簡的途徑，類似於美國。

Other regions, we're working more closely with individual hospitals in determining how best to provide drug to those patients that want it.

在其他地區，我們正在與各個醫院更密切地合作，以確定如何最好地為需要藥物的患者提供藥物。

So it's an atypical program, but I also think it speaks to our commitment to really respond to this demand from patients and PIs.

所以這是一個非典型的計劃，但我也認為它體現了我們真正回應患者和 PI 需求的承諾。

Very, very helpful.

非常非常有幫助。

And then I guess when you consider being in a pivotal study right now that we'll go into, call it roughly mid next year and the Phase 2 SSc study, how would you sort of characterize at this moment your manufacturing needs to get beyond that and to potential commercialization for EFZO?

然後我想，當您考慮現在進行一項關鍵研究（我們將在明年年中左右進行）和第二階段 SSc 研究時，您目前會如何描述您的製造需要超越這一目標以及EFZO 的潛在商業化？

Yeah, so we had transitioned a few years ago to a more commercially oriented and scalable type of partnership.

是的，所以我們幾年前就轉變為更商業化和可擴展的合作夥伴關係。

So we're in really good shape here to essentially have drug manufactured, clear all the hurdles also with the FDA to support a launch with some of those initial batches and runs more or less mapped out here.

因此，我們的狀況非常好，基本上可以生產藥物，清除 FDA 的所有障礙，以支持其中一些初始批次的上市，並或多或少地在此處進行規劃。

Of course, -- if you go out five, seven years from now, I wouldn't say we have that type of capacity, but in the short term here to get through our clinical program and the early commercialization work, we're in good shape here.

當然，如果你從現在起五、七年後出去，我不會說我們有這種能力，但在短期內，為了完成我們的臨床計劃和早期商業化工作，我們處於這裡形狀好。

And we're also fortunate that the manufacturers here in the US, so we have good control, good line of sight.

而且我們也很幸運，製造商在美國這裡，所以我們有很好的控制力，良好的視線。

I know things are happening worldwide with manufacturers right now where there's a lot of controversy if it's not US based, but we're de-risked there as well by having a good manufacturing plan.

我知道現在世界各地的製造商都在發生這樣的事情，如果製造商不是位於美國，就會有很多爭議，但我們也透過制定良好的製造計劃來降低風險。

That we also spend a lot of time talking to regulators with to make sure --they want to make sure that we have that drug product ready for patients because there's going to be a high amount of demand assuming this drug gets approved.

我們也花了很多時間與監管機構交談，以確保他們希望確保我們為患者準備好該藥物產品，因為假設該藥物獲得批准，將會有大量需求。

These patients have been, as I said, waiting for a therapy their whole lives.

正如我所說，這些患者一生都在等待治療。

Well, that five to seven year comment would be a great problem to have, but thanks for all the comments, Sanjay.

好吧，五到七年的評論將是一個很大的問題，但感謝所有的評論，Sanjay。

Sure.

當然。

Thank you.

謝謝。

Our next question comes from Yasmeen Rahimi with Piper Sandler.

我們的下一個問題來自 Yasmeen Rahimi 和 Piper Sandler。

You may proceed.

您可以繼續。

Hey, Dean, this is [Jaekyung] for Yas.

嘿，Dean，這是 Yas 的 [Jaekyung]。

Congrats on the continued progress and thanks for taking our questions.

恭喜您持續取得進展，並感謝您提出我們的問題。

For EFZO-FIT, do you still need additional sites to bring enrollment to the finish line?

對於 EFZO-FIT，您是否還需要其他網站來完成註冊？

And is there actually an opportunity for additional enrollment beyond the 264 patients?

除了 264 名患者之外，是否真的有機會進行額外登記？

And second, could you provide more color on the percentage or number of patients continuing from EFZO-FIT to the EAP program?

其次，您能否提供更多關於從 EFZO-FIT 繼續到 EAP 計劃的患者百分比或數量的資訊？

Yeah, so 264 is kind of our goal.

是的，所以 264 是我們的目標。

A lot of that is based on our power calculations.

其中很大一部分是基於我們的功率計算。

It assumes that you can have a nominal dropout rate.

它假設你可以有一個名義上的輟學率。

So we're well-powered, actually overpowered in many ways compared to other Phase 3 trials. 92% powered here, 264 is our goal we still view that as a solid number there.

因此，與其他第三階段試驗相比，我們的能力很強，實際上在許多方面都超過了我們。這裡的供電率為 92%，264 是我們的目標，我們仍然認為這是一個可靠的數字。

It allows for also a buffer that if you had 10% of the patients discontinue for any reason, you'd still be able to hit your power calculation.

它還提供了一個緩衝，如果您有 10% 的患者因任何原因停止治療，您仍然能夠達到您的功效計算。

So we're still set with that number sometimes you can see trials that might be a few patients over or a few patients under.

因此，我們仍然會設定這個數字，有時您會看到試驗可能會超出或減少一些患者。

A lot of that has to do with patient screening right there at the end.

這很大程度上與最後的患者篩檢有關。

In our last trial, which was the 36 patient trial, we had eligible patients the day we stopped screening.

在我們的上一次試驗中，即 36 名患者的試驗中，我們在停止篩檢的當天就有了符合條件的患者。

We ended up having a few more patients, one more patient enrolled in that trial because they were screen eligible and we don't want sort of shut down enrollment if someone is already screened and is eligible.

我們最終又多了幾名患者，又多了一名患者參加了該試驗，因為他們符合篩選條件，如果有人已經經過篩選並且符合資格，我們不希望停止招募。

So I would say that we're still going to be right in that ballpark there of 264.

所以我想說，我們仍然處於 264 個大致範圍內。

With regards to how many are going to move into the EAP, we don't know yet.

至於有多少人將進入 EAP，我們還不知道。

We are continuing to generate requests.

我們正在繼續產生請求。

As you can imagine, as more and more patients finish the trial, we may get more patients interested.

正如你可以想像的那樣，隨著越來越多的患者完成試驗，我們可能會引起更多患者的興趣。

My early read on it is we're well built here to have a substantial number of patients.

我的早期解讀是，我們這裡建設得很好，可以容納大量患者。

If they want the drug, we're ready to provide it.

如果他們想要這種藥物，我們就準備好提供。

But I don't have an exact number, but we are prepared to potentially give the EAP program, have it there for a very, very large number of patients.

但我沒有確切的數字，但我們準備好提供 EAP 計劃，為非常非常多的患者提供服務。

We'll have to wait and see what kind of demand it continues.

我們將不得不等待，看看它會持續什麼樣的需求。

Early on right now, I will say, since we put the announcement, we probably had even more demand since we put the announcement out.

我想說，自從我們發佈公告以來，現在早些時候，我們可能有更多的需求。

Thank you so much for the color.

非常感謝你的顏色。

Thank you.

謝謝。

Our next question comes from Yale Jen Laidlaw & Co. You may proceed.

我們的下一個問題來自 Yale Jen Laidlaw & Co。

Good afternoon, and thanks for taking the questions.

下午好，感謝您提出問題。

I came in late, so maybe if you can reiterate a little bit in terms of EFZO-FIT enrollment.

我來晚了，所以也許您可以在 EFZO-FIT 註冊方面重申。

I know the press release indicated it's on track was there any other color of that?

我知道新聞稿表明它已步入正軌，還有其他顏色嗎？

Then I have another question.

那我還有一個問題。

No, Yale very much on track.

不，耶魯大學已經步入正軌。

Very much feeling good.

非常感覺良好。

You know, about Q2 this year, so very much on track.

你知道，今年第二季度，一切都步入正軌。

Okay, great, that's good.

好的，太好了，這很好。

And you mentioned a few preclinical programs and they look very interesting at this juncture.

您提到了一些臨床前項目，目前它們看起來非常有趣。

Was those the programs you intend to partner out at some point, or what will be the path you are thinking about moving forward with those preclinical programs?

這些是您打算在某個時候合作的專案嗎？

Well, I think it's, I mean, for everything as a biotech, like kind of where we are, you always have to look at partnering and those sorts of opportunities.

嗯，我認為，我的意思是，對於生物技術的一切，就像我們現在所處的位置一樣，你總是必須考慮合作和這類機會。

If there's no shortage of interest, obviously we have a rather novel platform here, and the platform now is generating more and more validated opportunities.

如果不缺乏興趣，顯然我們這裡有一個相當新穎的平台，而該平台現在正在產生越來越多的經過驗證的機會。

So that will be something that we'll look at.

所以這將是我們要關注的事情。

We always look at these sort of potential arrangements.

我們總是關注這些潛在的安排。

At the same time, we also are potentially, sitting on assets here that really are producing signals really never seen before.

同時，我們也有可能坐擁真正產生前所未見訊號的資產。

When you talk about being able to induce apoptosis of myofibroblasts, I don't think you'll hear too many companies really seeing those kinds of early signals.

當你談論能夠誘導肌成纖維細胞凋亡時，我認為你不會聽到太多公司真正看到這些早期訊號。

So that's where this biology, I think, it's important for everyone to understand.

因此，我認為，這就是每個人都必須理解的生物學原理。

There's hidden potential here, there's hidden potential efficacy here.

這裡有隱藏的潛力，這裡有隱藏的潛在功效。

And I think what aTyr is doing is really looking at these genetic codes and saying, where can they best be applied where there is disease and dysfunction.

我認為 aTyr 所做的實際上是在研究這些遺傳密碼，並說，在存在疾病和功能障礙的地方，它們可以最好地應用到哪裡。

Well?

出色地？

So unlike, you know, generations of MeToo therapies that have been out there that are not disease modifying, I think all of our approaches, Efzofitimod and even some of the pipeline approaches we have here are, we have a very, very high bar here.

因此，與已經存在的幾代 MeToo 療法不同，它們不能改變疾病，我認為我們所有的方法，Efzofitimod，甚至我們這裡的一些管道方法，我們在這裡有一個非常非常高的標準。

They have to be disease modifying, they have to really be showing activity here that is two standard deviations better than most-- opportunities out there.

它們必須能夠改變疾病，它們必須真正表現出比大多數機會好兩個標準差的活性。

And I think we want to be careful about-- giving away these gems too early, but we're open to talk to big players at all times.

我認為我們要小心——過早放棄這些寶石，但我們隨時願意與大玩家交談。

Okay, great.

好的，太好了。

Maybe just speaking one more question here, which is that, as you said, the trial is on track to complete enrollment second quarter.

也許在這裡再提一個問題，那就是，正如你所說，該試驗預計將在第二季完成註冊。

And just curious, in terms of the current in Japan, given they have smaller size, have they already completed any colors on that?

只是好奇，就日本目前的情況而言，鑑於它們的尺寸較小，他們是否已經完成了任何顏色？

And thanks.

謝謝。

Yeah, I can't really get into who's completed it.

是的，我實在無法了解誰完成了它。

I mean, it more or less is competitive enrollment.

我的意思是，這或多或少是競爭性招生。

There aren't hard caps per se, country by country.

各國本身並沒有硬性上限。

We have our goals on what we want, where we want to get to.

我們有我們想要的目標，我們想要到達的地方。

So I wouldn't think of it as a separate trial.

所以我不認為這是一個單獨的審判。

We've said in the past that a region like Japan, you might expect 25 to 30 patients there.

我們過去說過，像日本這樣的地區，可能會有 25 到 30 名患者。

So Japan, like the rest of the world that we're working in right now, very much on track, you know, to meet our projections to get this trial wrapped up here in Q2, at least enrollment.

因此，日本與我們目前正在進行的世界其他地區一樣，非常有希望達到我們的預測，在第二季完成這項試驗，至少在註冊方面。

Okay, great.

好的，太好了。

Thanks a lot and congrats on all the progress.

非常感謝並祝賀所有的進展。

Thank you, Yale.

謝謝你，耶魯。

Thank you.(Operator Instructions)

謝謝。

Our next question comes from Robert LeBoyer with NOBLE Capital Markets.

我們的下一個問題來自 NOBLE Capital Markets 的 Robert LeBoyer。

You may proceed.

您可以繼續。

Good afternoon and congratulations on all the progress.

下午好，祝賀所有進展。

I also have a question on the expanded access program and was wondering whether you're going to be compiling endpoints and other data that can be used in the BLA or whether you can present some of the extension some of the extension study data at medical meetings either before the data is announced or afterwards.

我還有一個關於擴展存取計劃的問題，想知道您是否要編譯可在 BLA 中使用的端點和其他數據，或者您是否可以在醫學會議上展示一些擴展研究數據無論是在數據公佈之前還是之後。

Yeah, it's a great question, Robert.

是的，這是一個很好的問題，羅伯特。

So just want to highlight one thing here is this is being conducted outside of our protocol and that's being done for a few reasons.

因此，我想強調一件事，這是在我們的協議之外進行的，並且這樣做有幾個原因。

Number one, we want to hit our BLA timelines and submit, finish the primary trial, if you will, the primary portion of the trial and submit that.

第一，我們希望在 BLA 時間表內完成並提交，完成主要試驗（如果您願意），完成試驗的主要部分並提交。

This secondary piece of the trial is sitting outside of our protocol.

該試驗的第二部分不在我們的方案範圍內。

Why are we doing it that way?

我們為什麼要這樣做？

Well, first of all, health authorities didn't mandate that we do any kind of open label extension.

嗯，首先，衛生當局沒有強制要求我們進行任何形式的開放標籤延伸。

There was no known safety risks observed early in the program, in our last program.

在我們上一個專案的專案早期，沒有觀察到已知的安全風險。

And we had long-term safety data already from this trial.

我們已經從這次試驗中獲得了長期安全數據。

So there was really no need to have a more formal open label extension or anything like that.

因此，確實沒有必要進行更正式的開放標籤擴展或類似的事情。

So this trial will be done outside of the boundaries of our protocol.

因此，這項試驗將在我們協議的範圍之外進行。

Now, already I would say many academics are really interested in looking at patients 18, 24 months out, perhaps with or without efzofitimod long-term.

現在，我已經說過，許多學者確實有興趣觀察 18、24 個月後的患者，可能長期服用或不服用 efzofitimod。

So I can't say that there would not be an academic consortium of some of our experts that would want to put out this data.

所以我不能說我們的一些專家組成的學術聯盟不會想要公佈這些數據。

I would support that.

我會支持這一點。

I think they could look at different things that within the confines of our own protocol, we haven't been able to look at-- to look at long-term, you know, steroid reduction effects.

我認為他們可以在我們自己的方案範圍內研究不同的事情，我們無法研究長期的，你知道的，類固醇減少的效果。

Some of the early promising things we see with regards to things like weight loss,-- it was always theoretical.

我們在減肥等方面看到的一些早期有希望的事情——它總是理論上的。

You get off steroids, you could help with weight.

你停止使用類固醇，可以幫助減輕體重。

You know, we're seeing some-- rather remarkable signals there it's early, it's anecdotal.

你知道，我們看到了一些相當顯著的訊號，現在還為時過早，這是軼事。

This might be something that we could look at, those investigators could look at in some sort of academic survey.

這可能是我們可以看到的東西，那些調查人員可以在某種學術調查中看到。

They may want to look at some sort of imaging data two or three years later.

他們可能想在兩三年後查看某種成像數據。

But right now we're focused on getting this drug approved, getting the BLA out.

但現在我們的重點是讓這種藥物獲得批准，獲得 BLA。

Those trickle of that kind of interesting data could almost be a headstart to Phase 4.

這些有趣的數據幾乎可以成為第四階段的先機。

That's the way I look at it.

我就是這麼看的。

We also don't want to spend money on that sort of-- downstream side of the fence right now.

我們現在也不想在圍欄的下游一側花錢。

But I think there's going to be some intriguing potential there to collect data, not only for the patients that continue in the trial, but also those that might have to revert back to steroids and see what happens with those patients.

但我認為，那裡將有一些有趣的潛力來收集數據，不僅適用於繼續試驗的患者，也適用於那些可能必須恢復使用類固醇並觀察這些患者會發生什麼情況的患者。

But I think it's a great question, Robert.

但我認為這是一個很好的問題，羅伯特。

Really nice how you're thinking about that.

你這麼想真是太好了。

And it's already things that-- experts are noodling over, potentially to do something outside of the trial.

專家已經在研究這些事情，可能會在試驗之外做一些事情。

Okay, great.

好的，太好了。

Thank you very much.

非常感謝。

Thank you.

謝謝。

I would now like to turn the call back over to Sanjay for any closing remarks.

我現在想將電話轉回給桑傑，讓其發表結束語。

Great.

偉大的。

Well, we thank everybody's interest.

好吧，我們感謝大家的興趣。

Great questions today.

今天的問題很好。

I know there's a lot of expectation of everyone's really looking forward to getting this trial wrapped up here we are too.

我知道每個人都非常期待這次審判在這裡結束，我們也是。

Appreciate everyone's interest here.

感謝大家對這裡的興趣。

Thank you for following us and look forward to talking to you in the future.

感謝您關注我們，並期待將來與您交談。

Thanks again.

再次感謝。

Thank you for your participation.

感謝您的參與。

You may now disconnect.

您現在可以斷開連線。