Crescent Biopharma Inc (CBIO) 2023 Q3 法說會逐字稿

Good morning and thank you for joining the GlycoMimetics Q3 2023 earnings call. At this time, all participants are in a listen only mode.

早安，感謝您參加 GlycoMimetics 2023 年第三季財報電話會議。此時，所有參與者都處於只聽模式。

Following management's remarks, we will hold a question and answer session (Operator Instructions). I would now like to turn the call over to Christian Dinneen-Long company counsel at GlycoMimetics. Please go ahead.

在管理階層發言後，我們將舉行問答環節（操作員說明）。我現在想將電話轉給 GlycoMimetics 的 Christian Dinneen-Long 公司法律顧問。請繼續。

Good morning. Today, we will review our business updates and financial results for the quarter ended September 30 2023. The press release this morning is available on the company's website at glycomimetics.com. This call is being recorded. The dial-in phone replay will be available for 24 hours after the closing of the call. The webcast replay will also be available for 30-days in the Investor Section of the company's website.

早安.今天，我們將回顧截至 2023 年 9 月 30 日的季度的業務更新和財務業績。此通話正在錄音。撥入電話重播將在通話結束後 24 小時內提供。網路廣播重播也將在公司網站的投資者部分提供為期 30 天的回放。

Joining me on the call today from GlycoMimetics are Harout Semerjian, Chief Executive Officer; Brian Hahn, Chief Financial Officer; and Dr. Edwin Rock, Chief Medical Officer. Today's call will include forward-looking statements based on our current expectations.Forward looking, statements may include, but are not limited to, statements about the company's product candidates uproleselan, and GMI -1687, along with statements about the progress and timing of clinical trials being conducted by us or our collaborators planned approach, central regulatory agency interactions are submissions.

今天與我一起參加 GlycoMimetics 電話會議的是執行長 Harout Semerjian；布萊恩‧哈恩，財務長；和首席醫療官 Edwin Rock 博士。今天的電話會議將包括基於我們目前預期的前瞻性陳述。計劃進行的試驗、中央監管機構的互動均已提交。

Development plans and activities, prelaunch preparations, the company's cash position and runway and our expectations regarding data readouts from clinical trials. Such statements represent management's judgment and intention as of today and involve assumptions, risks and uncertainties.

開發計劃和活動、上市前的準備工作、公司的現金狀況和跑道以及我們對臨床試驗數據讀數的期望。此類陳述代表管理階層截至目前的判斷和意圖，並涉及假設、風險和不確定性。

GlycoMimetics undertakes no obligation to update or revise any forward-looking statements. For information concerning the risk factors that could affect the company. Please refer to our filings with the SEC, which are available from the SEC or through the GlycoMimetics website.

GlycoMimetics 不承擔更新或修改任何前瞻性陳述的義務。有關可能影響公司的風險因素的資訊。請參閱我們向 SEC 提交的文件，這些文件可從 SEC 或透過 GlycoMimetics 網站取得。

I'll now turn the call over to Harout.

我現在將把電話轉給哈魯特。

Thank you Christian, and good Morning everyone. In the third quarter, we continued to make strong progress advancing our clinical pipeline as we evolve into a commercial stage organization. We remain committed to executing on our vision to deliver about uproleselan to AML patients in need of new treatment options.

謝謝克里斯蒂安，大家早安。第三季度，隨著我們發展成為商業階段組織，我們在臨床管道方面繼續取得強勁進展。我們仍然致力於實現我們的願景，為需要新治療方案的 AML 患者提供 uproleselan。

While we leverage our unique molecular biology approach to develop innovative medicines in additional diseases such as sickle cell.

我們利用獨特的分子生物學方法開發針對鐮狀細胞疾病等其他疾病的創新藥物。

Today, I would like to highlight three drivers that position us well to advance these programs and then coming here. First, we continue to expect top line results from our pivotal Phase 3 study of uproleselan relapsed and refractory AML by the the end of Q2 2024 and remain encouraged about the unprecedented median follow-up time.Should trial results be positive we expect US filing by end of 2024.

今天，我想重點介紹三個驅動因素，它們使我們能夠很好地推進這些計劃，然後來到這裡。首先，我們繼續預期我們的uproleselan 復發難治性AML 關鍵3 期研究將於2024 年第二季末取得頂線結果，並對前所未有的中位追蹤時間感到鼓舞。申請將於2024 年底。

Second, as part of our ongoing commitment to evaluate the utility of uproleselan across AML, researchers from MD Anderson will present updated clinical data from their investigator initiated trial in treated secondary AML at the American Society of Hematology meeting this December.+

其次，作為我們持續致力於評估uproleselan 在AML 中的效用的一部分，MD 安德森的研究人員將在今年12 月的美國血液學會會議上展示其研究者發起的治療繼發性AML 試驗的最新臨床數據。 +

Lastly, for GMI-1687 and our sickle cell disease program. The Phase 1a study remains on track for safety data by the end of Q1 2024. With addition of the time-based analysis option to our pivotal Phase 3 study of uproleselan in the relapsed refractory AML. We expect to report top-line results by the end of Q2 2024.

最後，針對 GMI-1687 和我們的鐮狀細胞疾病計畫。 1a 期研究仍將在 2024 年第一季末獲得安全數據。我們預計將在 2024 年第二季末報告營收結果。

We look forward to unveiling these data as this important milestone for patients, investigators and the company.

我們期待將這些數據公佈為患者、研究人員和公司的重要里程碑。

While recently FDA approved drugs have shown encouraging results in limited AML subpopulations. We're optimistic that your uproleselan has the potential to improve patient outcomes irrespective of mutation profile, cytogenetic risk or treatment backlog.

最近 FDA 批准的藥物在有限的 AML 亞群中顯示出令人鼓舞的結果。我們樂觀地認為，無論突變情況、細胞遺傳學風險或治療積壓如何，您的 uproleselan 都有可能改善患者的治療結果。

Building on the promise uproleselan in relapsed refractory AML , we are excited to explore its broader potential across different age groups and disease settings through studies run by partners and independent investigators.

基於 uproleselan 在治療復發難治性 AML 方面的前景，我們很高興透過合作夥伴和獨立研究人員進行的研究，探索其在不同年齡層和疾病環境中更廣泛的潛力。

For example, at the upcoming ASH Annual Meeting in San Diego, research from the MD Anderson Cancer Center will present updated clinical data from their Phase 1b 2 study, evaluating uproleselan with Cladribine and low-dose cytarabine in treated secondary AML patients.

例如，在即將於聖地牙哥舉行的ASH 年會上，MD 安德森癌症中心的研究人員將展示其1b 2 期研究的最新臨床數據，評估烏普羅塞蘭與克拉屈濱和低劑量阿糖胞苷治療繼發性AML 患者的效果。

We are grateful to be collaborating with leading organizations, including MD Anderson, the National Cancer Institute and the Dana-Farber Cancer Institute.

我們很高興能與 MD 安德森癌症中心、國家癌症研究所和丹納法伯癌症研究所等領先組織合作。

The commitment of our partners underscores uproleselan potential to improve and prolonged lives for a broad spectrum of AML patients.

我們合作夥伴的承諾強調了 uproleselan 在改善和延長廣大 AML 患者生命方面的潛力。

We look forward to providing updates as these investigator-initiated clinical trials continued. Finally, we expect to announce safety data from our Phase 1a study of GMI -1687 by end of Q1 2024. We expect these data will advance us towards our aspiration to develop GMI- 1687 has a patient controlled point of care therapy to interrupt early sickle cell pain crisis before need to seek emergency care. We will evaluate next steps shortly thereafter.

我們期待著隨著這些研究者發起的臨床試驗的繼續進行而提供最新資訊。最後，我們預計在2024 年第一季末公佈GMI -1687 1a 期研究的安全性數據。在早期鐮狀細胞疾病細胞疼痛危機之前需要尋求緊急護理。不久後我們將評估後續步驟。

As our clinical pipeline has progressed, we have strategically expanded our commercial and medical affairs capabilities, and we are now executing critical prelaunch activities, head of top line uproleselan readout expected in Q2 2024. Our educational disease awareness activities, target, academic and community hematologists that care for AML patients.

隨著我們的臨床管道取得進展，我們策略性地擴展了我們的商業和醫療事務能力，我們現在正在執行關鍵的啟動前活動，預計將於2024 年第二季度公佈頂線uproleselan 讀數。疾病意識活動、目標、學術和社區血液學家照顧 AML 患者。

Recently, we welcomed Dr. Gaetano Bonifacio as our Vice President of Global Medical Affairs and Debora Peralta as our Vice President of Commercial Operations. Gaetano and Debora bring decades of hematology launch experience and we are pleased to have the opportunity to leverage their expertise during this transformative point in our company's evolution.

最近，我們歡迎 Gaetano Bonifacio 博士擔任我們的全球醫療事務副總裁，Debora Peralta 擔任我們的商業營運副總裁。 Gaetano 和 Debora 帶來了數十年的血液學啟動經驗，我們很高興有機會在我們公司發展的這一變革時期利用他們的專業知識。

Now turning to our finances. We have a cash runway to fund operations into late Q4 2024, allowing us to continue executing our clinical development plan. On today's call, I'm happy to be joined by our CFO, Brian Hahn, and our CMO, Dr. Edwin Rock.

現在轉向我們的財務。我們擁有現金跑道，可以為 2024 年第四季末的營運提供資金，使我們能夠繼續執行我們的臨床開發計劃。我很高興邀請我們的財務長 Brian Hahn 和首席行銷長 Edwin Rock 博士參加今天的電話會議。

I'll now pass it over to Ed to share more details on our ongoing trials.

現在我將把它交給 Ed，分享我們正在進行的試驗的更多細節。

Thanks a Harout, and thanks to all of you on the line for joining our call today. As Harout mentioned, we expect to report top-line results of our Phase 3 trial by the end of Q2 2024. At that time, we'll have a clinically mature data set for time-based primary analysis of overall survival.

感謝哈魯特，也感謝所有在線的人今天加入我們的電話會議。正如 Harout 所提到的，我們預計在 2024 年第二季末報告 3 期試驗的主要結果。

If the 295 survival events that trigger primary the analysis are not already reached by that time.

如果此時尚未達到觸發主要分析的 295 個生存事件。

Median follow-up for our Phase 3 trial a few progressed well in relapsed and refractory AML stands now at 33 months and will be greater than three years at time of primary analysis that's unprecedented for therapeutic trial in relapsed and refractory AML.

我們的3 期試驗在復發性和難治性AML 方面進展良好，中位追蹤時間目前為33 個月，在初步分析時將超過三年，這對於復發性和難治性AML 的治療試驗來說是前所未有的。

Also, the substantial majority of surviving study patients received hematopoietic cell transplantation ended primary analysis a large majority of them will be at least two years out from their transplant. After two years post transplant disease relapse becomes infrequent. So it's at least two years of post transplant follow-up for almost all transplant recipients.

此外，絕大多數接受造血細胞移植的存活研究患者在初步分析結束後，其中大多數將在移植後至少兩年後進行。移植後兩年後，疾病復發變得罕見。因此，幾乎所有移植受者都需要至少兩年的移植後追蹤。

We're confident that our time-based primary analysis will provide adequate trial duration to demonstrate uproleselan benefit if present. Our biologic hypothesis is that edge point of view progressively and will lead to deeper more durable AML disease responses that help more people to and through potentially curative hematopoietic cell transplantation.

我們相信，我們基於時間的初步分析將提供足夠的試驗持續時間來證明 uproleselan 的益處（如果存在）。我們的生物學假設是，這種邊緣觀點將逐步導致更深入、更持久的 AML 疾病反應，從而幫助更多的人接受並透過潛在的治癒性造血細胞移植。

This effect may occur irrespective of specific gene mutations, cytogenetic risk or treatment backbone. Correspondingly, uproleselan and clinical activity is seen in both newly diagnosed and relapsed and refractory AML.

無論特定基因突變、細胞遺傳學風險或治療主幹如何，這種效應都可能發生。相應地，在新診斷的以及復發和難治性 AML 中都觀察到了 uproleselan 和臨床活性。

Importantly, uproleselan appears to generate noteably unremarkable safety profile, adding no additional toxicity when combined with other therapies.

重要的是，烏普羅塞蘭似乎產生了明顯不顯著的安全性，與其他療法聯合使用時不會增加額外的毒性。

So we are optimistic that uproleselan may one day be safely, combined with diverse other therapies that are broadly useful for most oral AML patients.

因此，我們樂觀地認為，烏普羅塞蘭有一天可能會安全地與對大多數口腔 AML 患者廣泛有用的其他多種療法相結合。

Partner and independent investigator studies are further exploring potential benefit of uproleselan across a AML subtypes. Most importantly, a randomized NCI Phase 23 trial conducted by the Alliance for Clinical Trials in Oncology is evaluating you for less land in newly diagnosed older patients with AML who are fit for intensive chemotherapy.

合作夥伴和獨立研究人員的研究正在進一步探索 uproleselan 在 AML 亞型中的潛在益處。最重要的是，腫瘤學臨床試驗聯盟進行的一項隨機 NCI 23 期試驗正在評估新診斷的老年 AML 患者是否適合接受強化化療。

This trial completed Phase 2 randomization of 267 patients in December 2021. Phase 2 analysis of event-free survival has been pending since then, and now almost to years since enrollment completion.

該試驗於 2021 年 12 月完成了 267 名患者的 2 期隨機分組。

And to see, I recently confirmed to us is that the event trigger for analysis hasn't yet been reached.

並且看到，我最近向我們確認的是，尚未達到分析的事件觸發器。

For reference. The Phase 2 portion of this trial was designed to demonstrate prolongation of median event-free survival from 7 to 11 months. We look forward to learning Phase 2 results when available. Significantly, NCI expanded our collaboration and now also supports a children's oncology group Phase 1 study conducted by their pediatric early phase clinical trial network.

以供參考。本試驗的第 2 期部分旨在證明中位無事件存活期從 7 個月延長至 11 個月。我們期待了解第二階段的結果。值得注意的是，NCI 擴大了我們的合作範圍，現在也支持由其兒科早期臨床試驗網絡進行的兒童腫瘤學小組一期研究。

This dose escalation trial will assess safety, pharmacokinetics and preliminary clinical activity uproleselan plus chemotherapy in pediatric patients with relapsed or refractory AML.

此劑量遞增試驗將評估 uproleselan 合併化療治療復發性或難治性 AML 兒科患者的安全性、藥物動力學和初步臨床活性。

We're glad to announce that the first patient enrolled this study in October, another investigator initiated trial, led by Dr. John Horan from the Dana-Farber Cancer Institute and Boston Children's Hospital also dosed its first patient earlier this year.

我們很高興地宣布，第一位患者於10 月加入了這項研究，由丹納法伯癌症研究所的John Horan 博士領導的另一位研究人員發起了試驗，波士頓兒童醫院也在今年早些時候時候對第一位患者進行了給藥。

Dr. Horan's trial is evaluating new progressively and with the pre-transplant regimen for pediatric and adult AML patients up to 39 years old.

Horan 醫師的試驗正在評估新的漸進式移植前治療方案，適用於 39 歲以下兒童和成人 AML 患者。

Today, we announced that at ASH in December, researchers from MD Anderson Cancer Center will present updated trial data on uproleselan in treated secondary AML.

今天，我們宣布，在 12 月的 ASH 上，MD 安德森癌症中心的研究人員將展示 uproleselan 在治療繼發性 AML 中的最新試驗數據。

This rare very high risk study population is defined by prior chemotherapy and an [anti- treatment] of and antecedent hematologic disorder. Phorgnosis is abysmal with expected median survival of less than five months. In the trial at MD Anderson investigators sought to generate a safe approach to marrow blast reduction, disease control and potential for transplant.

這種罕見的非常高風險的研究族群是由先前的化療和先前的血液疾病的[抗治療]來定義的。預後非常差，預計中位存活期不到五個月。在 MD 安德森的試驗中，研究人員試圖找到一種安全的方法來減少骨髓母細胞、控制疾病並提高移植潛力。

By combining uproleselan with low intensity chemotherapy of cladribine and cytarabine. Bad prognostic features in the study population include adverse cytogenetic risk and prior hypomethylating agent used in all valuable patients and prior hematopoietic cell transformation in 25% of them. Among 18 evaluable patients of data cut off, there were minimal therapy related toxicities 72% showed a reduction in bone marrow blasts and one patient had a potentially curative transplants.

透過將烏普羅塞蘭與克拉屈濱和阿糖胞苷的低強度化療相結合。研究族群的不良預後特徵包括不利的細胞遺傳學風險、所有有價值的患者均曾使用過低甲基化藥物，其中 25% 的患者曾發生過造血細胞轉化。在截止數據的 18 名可評估患者中，治療相關毒性極小，72% 的患者骨髓原始細胞減少，一名患者接受了潛在的治癒性移植手術。

These results in is notoriously difficult to treat disease underscore broad potential utility uproleselan across the AML spectrum. In addition, researchers from Washington University will present at ASH safety and signal generating data from their trial of uproleselan to reduce GI toxicities of melphalan chemotherapy given before transplant for multiple myeloma.

這些結果強調了 uproleselan 在整個 AML 譜系中的廣泛潛在效用。此外，華盛頓大學的研究人員將在 ASH 上展示他們的 uproleselan 試驗的安全性和信號生成數據，該試驗旨在降低多發性骨髓瘤移植前進行的美法崙化療的胃腸道毒性。

Beyond uproleselan we also made progress in our Phase 1a single ascending dose trial of GMI -1687 for second generation E-selectin antagonist. We will evaluate GMI -1687 as a potential outpatient self-administered subcutaneous therapy to interrupt sickle cell basil occlusive crises.

除了 uproleselan 之外，我們還在 GMI -1687 用於第二代 E-選擇素拮抗劑的 1a 期單劑量遞增試驗中取得了進展。我們將評估 GMI -1687 作為一種潛在的門診自我皮下療法，以中斷鐮狀細胞羅勒閉塞危機。

E-selectin plays a key mechanistic role in early progression of such acutely painful pathologic events. And if successful, GMI -1687 offers potential for a point of care patient controlled treatment option at time of pain onset.

E-選擇素在此類急性疼痛病理事件的早期進展中發揮關鍵的機製作用。如果成功的話，GMI -1687 可以為疼痛發作時的護理點患者控制治療選擇提供潛力。

In addition to patient benefit from pain control, such a point of care therapy has potential to reduce emergency room visits and hospitalizations of recipients.

除了患者從疼痛控制中受益之外，這種護理點治療還有可能減少接受者的急診室就診和住院。

As mentioned, we expect to have safety data in hand from this healthy volunteer study by the end of Q1 2024.

如前所述，我們預計到 2024 年第一季末可獲得這項健康志願者研究的安全數據。

Now I'll turn it over to Brian for a review of financial results.

現在我將把它交給布萊恩來審查財務結果。

Thank you Ed. As of September 30, 2023, GlycoMemitics cash- and- cash equivalents of $49.4 million as compared to $47.9 million as of December 31, 2022. This increase was due to the company's ability to raise additional cash earlier this year.

謝謝你艾德。截至 2023 年 9 月 30 日，GlycoMemitics 現金及現金等價物為 4,940 萬美元，而截至 2022 年 12 月 31 日為 4,790 萬美元。

The company's research and development expenses were $5.3 million for the quarter ended September 30, 2023, as compared to $4.9 million for the same period in 2022.

截至2023年9月30日的季度，該公司的研發費用為530萬美元，而2022年同期為490萬美元。

Decreased expenses were primarily due to the clinical development costs related to the Phase 1a trial of GMI -1687 in healthy adult volunteers, which was initiated in August 2023, partilally offset by decrease in stock based compensation costs due to lower headcount.

費用減少主要是由於與 2023 年 8 月在健康成年志願者中啟動的 GMI -1687 1a 期試驗相關的臨床開發成本，部分被由於員工人數減少而導致的基於股票的補償成本的減少所抵消。

The company's general administrative expenses increased to $4.5 million for the quarter ended September 30, 2023, as compared to $3.8 million for the same period in 2022. Increased expenses were primarily due to higher personnel related expenses and higher professional fees as a company advances uproleselan and preparing for potential regulatory filing and commercialization.

截至2023 年9 月30 日的季度，該公司的一般管理費用增加至450 萬美元，而2022 年同期為380 萬美元。專業費用增加。

I'd now like to turn the call back to Harout.

我現在想把電話轉回哈魯特。

Thank you, Brian. In summary, we are at a crucial stage in our company's evolution. In the coming months. We will remain laser focused on delivering the data, our Phase 3 study of uproleselan in the relapsed and refractory AML and continuing strategic prelaunch activities to accelerate our transition to a commercial side stage company after top-line results by end of Q2 2024.

謝謝你，布萊恩。總而言之，我們正處於公司發展的關鍵階段。在接下來的幾個月裡。我們將繼續專注於提供數據、uproleselan 在復發性和難治性AML 中的3 期研究，並繼續開展策略性預啟動活動，以在2024 年第二季末獲得頂線結果後加速我們向商業化階段公司的過渡。

We have the right team with the right experience in place, and I'm confident that we will be able to build upon our collective track record of successful commercial launches should data permit. Also, we continue to explore the uproleselan across the ammo spectrum.

我們擁有豐富經驗的合適團隊，我相信，如果數據允許，我們將能夠在成功商業發布的集體記錄基礎上再接再厲。此外，我們還將繼續探索整個彈藥領域的 uproleselan。

Thanks to studies conducted by institutions such as NCI. Dana Farber Cancer Institute and MD Anderson Cancer Center. Finally, we continued to progress our Phase 1 study of GMI -1687 towards initial data readout in Q1 2024.

感謝 NCI 等機構進行的研究。達納法伯癌症研究所和 MD 安德森癌症中心。最後，我們繼續推進 GMI -1687 的第一階段研究，爭取在 2024 年第一季讀出初始數據。

In closing, I want to thank our employees whose hard work and dedication drive uproleselan forward as well as the patients and investigators that make these trial if possible.

最後，我要感謝我們的員工，他們的辛勤工作和奉獻精神推動了 uproleselan 的發展，以及盡可能進行這些試驗的患者和研究人員。

I'd now like to open the lines to Q&A.

我現在想打開問答線路。

(Operator Instructions)

（操作員說明）

Roger Song, Jefferies.

羅傑·宋，杰弗里斯。

Great. Thank you, thanks for that for that update. A couple of questions on that. One thing is, can you share with us at how likely by the end of Q2 2024 you on the primary analysis to well-being event time-based just and awards how they get then accumulation since last update. Thank you.

偉大的。謝謝，謝謝您的更新。有幾個問題。一件事是，您能否與我們分享，到 2024 年第二季度末，您對基於時間的幸福事件的初步分析以及獎勵自上次更新以來如何獲得累積。謝謝。

Good morning, Roger. Excellent question. So as as we have mentioned over the last year, we have seen at two stages a slowdown in the number of events, which really led us to the collaboration with the FDA and the introduction of the time-based event trigger.

早安，羅傑。很好的問題。正如我們去年提到的，我們在兩個階段看到事件數量有所放緩，這確實促使我們與 FDA 合作並引入了基於時間的事件觸發器。

So currently, we are monitoring this very carefully, Roger. And what's happening is a lot of the patients as we are now almost two years out from full enrollment the follow up more a lot of times goes into quarterly rather than monthly. So we're evaluating that our next couple of months, and we should know by end of year and beyond.

所以目前，我們正在非常仔細地監控這一情況，羅傑。正在發生的事情是很多患者，因為我們距離全面入組還有近兩年的時間，更多的時候是每季度而不是每月進行追蹤。因此，我們正在評估接下來的幾個月，我們應該會在年底及以後知道。

But currently, what we are very pleased about is either through the events based trigger the time-based could trigger. We have a definitive cutoff by end of Q2 2024.

但目前，我們非常高興的是，要麼透過基於事件的觸發，要麼透過基於時間的觸發。我們將在 2024 年第二季末確定截止日期。

Excellent. I look forward to the next update. And also you guided you will submit that NDA by the end of this year, up by the end of 2024 if the primary analysis is positive. Just curious, any outstanding CMC and preclinical work you need to finish before you can file the NDA or anything else to step into that NDA filing?

出色的。我期待下一次更新。您還指導您將在今年年底之前提交 NDA，如果初步分析結果是正面的，則將在 2024 年底之前提交。只是好奇，在您提交 NDA 或其他任何內容以進入 NDA 備案之前，您需要完成任何未完成的 CMC 和臨床前工作嗎？

Yeah, Roger, we're in a very good shape. As we have been really in this trial, as you know, has taken longer than what any of us have anticipated driven by the fact that patients continue to live longer. Meanwhile we have done a very good job in terms of advancing CMC conversations, making sure that we have drug, making sure that we are preparing for an NDA filing with whatever we can do before we actually see the data as we continue to be blinded to that.

是的，羅傑，我們的狀態非常好。如您所知，正如我們實際進行的這項試驗所花費的時間比我們任何人預期的都要長，因為患者的壽命仍然更長。同時，我們在推進 CMC 對話方面做得非常好，確保我們有藥物，確保我們在真正看到數據之前盡一切努力準備 NDA 備案，因為我們仍然對數據視而不見。

So we think we have a foundation for very rapid enough move into an NDA filing after the data should the data be positive. So that's why we have now in a position to say that we're going to be doing that before end of the year.

因此，我們認為，如果數據呈陽性，我們就有了在數據公佈後足夠快地進入 NDA 備案的基礎。這就是為什麼我們現在可以說我們將在今年年底之前做到這一點。

Excellent. Yes, you do have some additional time to prepare our the work. Okay. Maybe just last one from us, if I can is in terms of GBI-1687, the healthy volunteer data by the end of 1Q next year, so how should we think about that data? What you really looking for from that data to move forward? And I see that dose response bio-marker, that would be helpful. Thank you.

出色的。是的，您確實有一些額外的時間來準備我們的工作。好的。如果可以的話，也許我們的最後一個數據是 GBI-1687，也就是明年第一季末的健康志工數據，那麼我們該如何看待這些數據？您真正想從這些數據中尋找什麼來推動前進？我看到劑量反應生物標記，這會很有幫助。謝謝。

Yes. Maybe I'll tackle some of the strategic perspective and if you want to add a few things about what we're going to be seeing.

是的。也許我會討論一些策略觀點，如果你想補充一些關於我們將要看到的內容的話。

So strategically, Roger, as you know, 1687 is our second generation E-selectin antagonist that is subcutaneously bioavailable, and that's why we're very excited to target sickle cell disease as our next area. As you know, we have a very deep tradition in that area with our first generation molecule.

因此，從戰略上講，Roger，如您所知，1687 是我們的第二代E-選擇素拮抗劑，具有皮下生物利用度，這就是為什麼我們非常興奮將鐮狀細胞疾病作為我們的下一個領域。如您所知，我們在該領域擁有非常深厚的傳統，我們的第一代分子。

So once we see and of course, the Phase 1a is really about safety. We want to make sure that we are doing all the building blocks to get to the next level and depending data and depending on where we are as well with not just the 301 our company-sponsored trial, but also the NCI sponsored Phase 2 in the frontline setting. Which continues to also take longer, depending on what what happens with these, we will be in a situation to say what do we want to do next and when after that. But if you wanted to maybe tackle the what's the Phase 1a

因此，一旦我們看到，當然，1a 階段確實是關於安全的。我們希望確保我們正在做所有的準備工作，以達到一個新的水平，並依賴數據和我們所處的位置，不僅是我們公司贊助的 301 試驗，還有 NCI 贊助的第 2 階段。這仍然需要更長的時間，具體取決於這些發生的情況，我們將處於一種情況下，我們要說下一步要做什麼以及之後什麼時候做。但如果你想解決什麼是階段 1a

Sure, Roger. As you know, key information from many first-in-human trial will include safety, data and pharmacokinetics, in particular, will the pharmacokinetics support achievement of a potential therapeutic level of the study drug.

當然，羅傑。如您所知，許多首次人體試驗的關鍵資訊將包括安全性、數據和藥物動力學，特別是藥物動力學是否支持研究藥物達到潛在治療水平。

Based on prior GlycoMimetics experience, we're confident that GMI -1687 will perform and will demonstrate that and share it when available.

根據先前的 GlycoMimetics 經驗，我們相信 GMI -1687 能夠發揮作用，並將證明這一點，並在可用時分享。

That's great. Thanks for taking the question. Thats it from my end.

那太棒了。感謝您提出問題。這就是我的結局。

Thank you, Roger.

謝謝你，羅傑。

Boris Peaker, TD Cowen.

鮑里斯·皮克，TD·考恩。

Great. Thanks taking my question. First, can you remind us maybe I missed that, how many patients in uproleselan study went on to transplant and also what the dropout rate wise?

偉大的。謝謝回答我的問題。首先，您能否提醒我們，也許我錯過了，uproleselan 研究中有多少患者繼續接受移植，以及退出率是多少？

Yes, good morning Boris in which, in which study?

是的，早安，鮑里斯在哪個、哪個研究室？

In your study specifically. But I guess if you notice for the NCI study as well, I guess that's a helpful, both of them.

具體在你的學習中。但我想，如果您也注意到 NCI 的研究，我想這對兩者都有幫助。

Yes. What we have announced Boris, as you know, is that in our Phase 2 trial in relapsed refractory, we have seen at 31% response rates. And as as I mentioned before, in our Phase 3, we are seeing a number north of that number. We have not disclosed the exact number. What we are saying, it's north of 31%.

是的。如你所知，鮑里斯，我們宣布的是，在我們針對復發難治性疾病的 2 期試驗中，我們看到了 31% 的緩解率。正如我之前提到的，在第三階段，我們看到的數字超出了這個數字。我們尚未透露具體數字。我們所說的，高於 31%。

And the dropout rate?

還有輟學率？

The dropout rate is 3%.A very low number.

輟學率為 3%。

My last question, if I may, NCI study, do you have a sense of why that's taking so long?

我的最後一個問題，如果可以的話，NCI 研究人員，您知道為什麼要花這麼長時間嗎？

Yeah we have our opinions, we have to wait for data to see what's happening. And as you know, the last patient enrolled in that trial in the Phase 2 population 267 patient was enrolled in December 2021. And that is in EFS primary endpoint for this up for the Phase 2.

是的，我們有自己的意見，我們必須等待數據才能看到發生了什麼。如您所知，最後一位參加此 2 期臨床試驗的患者（267 名患者）於 2021 年 12 月入組。

So we are we are very intrigued and hopeful that that's happening because, events are not reaching. And if that's the case, then that's good news to patients, obviously. So we'll see once they reach the events, we are we collaborate with NCI, we continue to have dialogues with them and they have told us that when they get to the events, they will let us know. And until now they have not done.

所以我們對這種情況的發生感到非常好奇和希望，因為事件還沒有發生。如果真是這樣，那麼這對患者來說顯然是個好消息。因此，一旦他們到達活動現場，我們就會與 NCI 合作，繼續與他們對話，他們告訴我們，當他們到達活動現場時，他們會讓我們知道。直到現在他們還沒有這樣做。

Great. Thanks for taking my questions.

偉大的。感謝您回答我的問題。

Thank you, Boris.

謝謝你，鮑里斯。

I'm showing no further questions at this time. I would now like to turn the call back to Harout for closing remarks.

我目前沒有提出任何進一步的問題。現在我想將電話轉回給哈魯特，讓其致閉幕詞。

Thank you, operator, and thank you to everyone for joining our call today.

謝謝接線員，也謝謝大家今天加入我們的通話。

We'll look forward to keeping you up-to-date on GlycoMimetics and seeing some of you at the Jefferies London Healthcare Conference or at the ASH in December. Thank you very much.

我們期待為您提供有關 GlycoMimetics 的最新信息，並期待在 12 月的 Jefferies 倫敦醫療保健會議或 ASH 上見到你們中的一些人。非常感謝。