Sangamo Therapeutics Inc (SGMO) 2025 Q2 法說會逐字稿

Good afternoon, and welcome to the Sangamo Therapeutics second quarter 2025 teleconference call. Please be advised that today's conference is being recorded.

下午好，歡迎參加 Sangamo Therapeutics 2025 年第二季電話會議。請注意，今天的會議正在錄製。

I would now like to turn the conference over to your speaker today, Louise Wilkie, Head of Investor Relations and Corporate Communications. Please go ahead.

現在，我想將會議交給今天的演講者、投資者關係和企業傳播主管 Louise Wilkie。請繼續。

Thank you. Good afternoon, everyone. Thank you for joining us on the call today. On this call are several members of the Sangamo executive leadership team, including Sandy Macrae, Chief Executive Officer; Nathalie Dubois-Stringfellow, Chief Development Officer; and Prathyusha Duraibabu, Chief Financial Officer.

謝謝。大家下午好。感謝您今天參加我們的電話會議。參加此次電話會議的有 Sangamo 執行領導團隊的幾位成員，包括執行長 Sandy Macrae、首席開發長 Nathalie Dubois-Stringfellow 和財務長 Prathyusha Duraibabu。

Slides from our corporate presentation can be found on our website, sangamo.com, and under the Presentations page of the Investors and Media section. This call includes forward-looking statements regarding Sangamo's current expectations. These statements include, but are not limited to, statements relating to Sangamo's cash runway, Sangamo's plans to obtain additional capital and its ability to continue to operate as a going concern, the therapeutic and commercial potential and value of Sangamo's product candidates and technologies; Sangamo's ability to establish and maintain collaborations and strategic partnerships, including for its Fabry disease program, the anticipated plans and time lines of Sangamo and its collaborators for clinical trials, clinical data presentations and releases, regulatory submissions and regulatory approvals, upcoming catalysts and milestones and other statements that are not historical fact.

我們的公司介紹的幻燈片可以在我們的網站 sangamo.com 以及投資者和媒體部分的簡報頁面上找到。本次電話會議包括有關 Sangamo 目前預期的前瞻性陳述。這些聲明包括但不限於與 Sangamo 的現金流量、Sangamo 獲得額外資本的計劃及其繼續作為持續經營企業運營的能力有關的聲明、Sangamo 候選產品和技術的治療和商業潛力及價值；Sangamo 建立和維持合作和戰略夥伴關係的能力，包括其法布里病計劃、Sangamo 及其合作者批准的臨床試驗預期計劃和催化劑的臨床試驗預期計劃和其他非催化劑的事實和展示歷史聲明、即將發布數據和非催化劑的臨床試驗預期計劃和催化劑

Actual results may differ materially from what we discuss today. These statements are subject to certain risks and uncertainties that are discussed in our filings with the SEC, specifically in our annual report on Form 10-K for the fiscal year ended December 31, 2024, and our quarterly report on Form 10-Q for the fiscal quarter ended -- sorry, June 30, 2025, and subsequent filings and reports that Sangamo makes from time to time with the SEC.

實際結果可能與我們今天討論的結果有重大差異。這些聲明受到我們向美國證券交易委員會提交的文件中討論的某些風險和不確定性的影響，特別是在我們截至 2024 年 12 月 31 日的財政年度的 10-K 表年度報告和截至 2025 年 6 月 30 日的財政季度的 10-Q 表季度報告以及 Sangamo 提交不時向美國證券委員會提交的後續文件和後續報告。

The forward-looking statements stated today are made as of today, and we undertake no duty to update such information, except as required by law. Please note that all forward-looking statements about our future plans and expectations are subject to our ability to secure adequate additional funding.

今天所述的前瞻性陳述是截至今天做出的，除非法律要求，否則我們不承擔更新此類資訊的義務。請注意，有關我們未來計劃和期望的所有前瞻性陳述都取決於我們獲得足夠額外資金的能力。

Now I'll turn the call over to our CEO, Sandy Macrae.

現在我將把電話轉給我們的執行長桑迪·麥克雷 (Sandy Macrae)。

Thank you, Louise, and good afternoon to everyone joining the call today. This quarter, we made important advances across both our clinical and preclinical pipeline. In June, we are happy to announce the positive top line results from our registrational STAAR in Fabry disease, taking us one step closer on the path towards potential approval of this promising treatment for Fabry disease patients.

謝謝你，路易絲，祝今天參加電話會議的各位下午。本季度，我們在臨床和臨床前研究方面都取得了重要進展。6 月，我們很高興地宣布，我們在法布瑞氏症註冊 STAAR 中取得了積極的頂線結果，這使我們在獲得這種有前景的法布瑞氏症患者治療方法的潛在批准的道路上又邁進了一步。

This month, we also became a clinical stage neurology genomic medicine company with the initiation of our first clinical site in the Phase 1/2 STAND study in chronic neuropathic pain. This is an important achievement, that means we are on track to generate clinical data for this program anticipated towards the end of 2026.

本月，我們也啟動了針對慢性神經性疼痛的 1/2 期 STAND 研究的首個臨床站點，成為臨床階段神經病學基因組醫學公司。這是一項重要的成就，這意味著我們預計在 2026 年底為該計劃產生臨床數據。

Finally, earlier this quarter, we held a productive meeting with the UK's Medicines and Healthcare products Regulatory Agency, or MHRA, for our preclinical prion disease program and are on track for a planned CTA submission for this program as early as mid-2026.

最後，本季度早些時候，我們與英國藥品和保健產品管理局（MHRA）就我們的臨床前朊病毒疾病項目舉行了富有成效的會議，並有望最早於 2026 年中期為該項目提交 CTA。

I'm proud of the progress and proud of my Sangamo colleagues who continue to work tirelessly to advance our pipeline while operating in such a challenging environment.

我為所取得的進步感到自豪，也為我的 Sangamo 同事感到自豪，他們在如此充滿挑戰的環境中繼續不懈地努力推進我們的管道建設。

Let me now hand directly to Nathalie Dubois-Stringfellow, our Chief Development Officer, to provide more context on these important programs. I will then close the call by summarizing the key takeaways from this quarter, and we'll put these updates into perspective. Nathalie?

現在，請允許我直接請我們的首席開發官 Nathalie Dubois-Stringfellow 提供更多有關這些重要項目的背景資訊。然後，我將在電話會議結束時總結本季的主要內容，我們將對這些更新進行全面分析。娜塔莉？

Thank you, Sandy. First, I am pleased to share details of the recent positive top line results from the registrational Phase 1/2 STAAR study evaluating isaralgagene civaparvovec or ST-920, our investigational gene therapy for the treatment of adults with Fabry disease.

謝謝你，桑迪。首先，我很高興分享註冊階段 1/2 STAAR 研究的最新積極頂線結果的詳細信息，該研究評估了 isaralgagene civaparvovec 或 ST-920，這是我們用於治療法布里病成人患者的研究性基因療法。

Following a single dose of ST-920, a positive mean annualized estimated glomerular filtration rate or eGFR slope of almost 2 was observed at 52 weeks across all 32 dose patients in this study. The FDA has agreed that mean eGFR slope will serve as the primary basis of approval under the accelerated approval pathway.

在接受單劑量 ST-920 治療後，本研究中所有 32 名劑量患者在 52 週時均觀察到接近 2 的平均年化估計腎小球濾過率或 eGFR 斜率的陽性值。FDA 已同意平均 eGFR 斜率將作為加速審批途徑下核准的主要依據。

Furthermore, a positive annualized eGFR slope of 1.7 was observed for the 19 patients who have achieved two years of follow-up. I want to take a moment to reflect upon this important accomplishment.

此外，在已完成兩年追蹤的 19 名患者中觀察到正年度 eGFR 斜率為 1.7。我想花點時間來反思這個重要成就。

As a reminder, the average untreated patient, Fabry patient experiences an annual decline in eGFR slope of minus 3 or minus 4. Achieving a positive mean eGFR slope across all 32 dose patients after one year and across the 19 patients who have reached two years is remarkable.

提醒一下，未經治療的法布瑞氏症患者平均每年的 eGFR 斜率會下降 3 或 4。所有 32 名接受劑量治療的患者在一年後均實現了正的平均 eGFR 斜率，而 19 名接受劑量治療的患者在兩年後也實現了正的平均 eGFR 斜率，這是非常了不起的。

As recommended by the FDA, we plan to compare the annualized mean eGFR slope of ST-920 with approved treatment for Fabry disease by performing a meta-analysis of published studies. According to observational studies, other marketed treatment options such as Replagal, Fabrazyme, and Galafold show a decline in annualized eGFR slope of minus 2.2 to minus 0.4.

根據 FDA 的建議，我們計劃透過對已發表的研究進行薈萃分析，比較 ST-920 與已批准的法布瑞氏症治療方法的年平均 eGFR 斜率。根據觀察性研究，其他市售治療方案如 Replagal、Fabrazyme 和 Galafold 顯示年化 eGFR 斜率下降了 -2.2 至 -0.4。

Key secondary endpoints in the ST-920 study were also positive. We continue to see strong durability in the study with elevated expression of alpha-Gal A activity maintained for up to 4.5 years for the longest treated patient and plasma lyso-Gb3 level that remain generally stable following the withdrawal of enzyme replacement therapy or ERT.

ST-920 研究的關鍵次要終點也呈現陽性。我們在研究中繼續看到了強大的持久性，對於接受治療時間最長的患者，其 alpha-Gal A 活性表達升高可維持長達 4.5 年，並​​且血漿溶血-Gb3 水平在停止酶替代療法或 ERT 後基本保持穩定。

We are excited to share for the first time a stabilization in cardiac endpoint, including a stabilization in cardiac function and morphological and biomarker data in the 32 patients with 52 weeks of follow-up. Measurement by MRI, including left ventricular mass, left ventricular mass index, left ventricle myocardial global longitudinal strain T1 and T2 mapping, end-diastolic; and endsystolic volume remained stable over one year.

我們很高興首次分享心臟終點的穩定，包括 32 名患者在 52 週的隨訪中的心臟功能穩定以及形態學和生物標記數據。透過 MRI 測量，包括左心室質量、左心室質量指數、左心室心肌整體縱向應變 T1 和 T2 映射、舒張末期；以及收縮末期容積在一年內保持穩定。

Furthermore, left ventricular ejection fraction measured by echo as well as cardiac biomarker such as troponin and NT-proBNP, have remained stable in all patients at one-year of follow-up. These data are striking, particularly given that cardiac function in Fabry patient tends to decline over time and is the leading cause of death in Fabry disease.

此外，透過超音波心動圖測量的左心室射血分數以及肌鈣蛋白和 NT-proBNP 等心臟生物標記在所有患者的一年追蹤中均保持穩定。這些數據令人震驚，特別是考慮到法布瑞氏症患者的心臟功能隨著時間的推移而下降，並且是法布瑞氏症死亡的主要原因。

Patients demonstrated a range of other clinical benefit, including improvement in disease severity reported in the first MSSI age-adjusted score and statistically and clinically significant improvement in the SF-36 quality of life scores, including a change of plus 15 in the role-physical score, plus 10 in the vitality score, and plus 9 in the bodily pain score at 52 weeks compared to baseline.

患者表現出一系列其他臨床益處，包括第一次 MSSI 年齡調整評分中報告的疾病嚴重程度的改善以及 SF-36 生活品質評分在統計和臨床上顯著改善，包括與基線相比，52 週時角色身體評分增加了 15 分，活力評分增加了 10 分，身體疼痛評分增加了 9 分。

Statistically significant improvement in the gastrointestinal symptom rating scale compared to baseline were also observed. I would like to particularly emphasize that ST-920 has been well tolerated in the study. The majority of adverse events were grade 1 or 2 in nature without the need for preconditioning. There was no safety-related study discontinuation or death.

與基線相比，胃腸道症狀評定量表也有統計學上顯著的改善。我想特別強調的是，ST-920 在研究中具有良好的耐受性。大多數不良事件本質上都是 1 級或 2 級，無需預先處理。沒有發生與安全相關的研究中止或死亡事件。

We believe that the totality of this compelling data demonstrate the potential for a single dose of ST-920 to treat the underlying pathology of Fabry disease and provide meaningful clinical benefit above current standard of care.

我們相信，所有這些令人信服的數據都表明，單劑量 ST-920 具有治療法布瑞氏症潛在病理的潛力，並且能夠提供高於目前治療標準的有意義的臨床益處。

ST-920 has shown the potential to transform the lives of patients, and we have observed additional clinical benefit in some, including the reduction and elimination in pain medication usage and the resumption of sweating, which has enabled these patients to perform physical task and exercise they were previously unable to do.

ST-920 已顯示出改變患者生活的潛力，我們在一些患者身上觀察到了額外的臨床益處，包括減少和消除止痛藥的使用以及恢復出汗，這使得這些患者能夠完成他們以前無法完成的體力任務和運動。

Following dosing with ST-920, all patients who came in the study on ERT were able to safely withdraw from ERT with one patient now off ERT for more than three years. In so doing, this patient have already avoided more than 1,000 biweekly ERT infusion, each of which can last up to six hours. What a transformation in the life of these Fabry disease patients.

服用 ST-920 後，所有參與 ERT 研究的患者都能夠安全地停止 ERT，其中一名患者已停止 ERT 三年多。這樣一來，該患者已經避免了每兩週進行 1,000 多次 ERT 輸注，每次輸注時間長達六個小時。這些法布瑞氏症患者的生活發生了巨大的變化。

Since the top line readout in June 2025, a physician has decided to resume ERT for one of their treated patients who had withdrawn from ERT. This patient who received ST-920 more than 2.5 years ago, maintained supraphysiological level of alpha-Gal A activity and their lyso-Gb3 levels were generally stable as of the top line readout date.

自 2025 年 6 月的頂線讀數以來，一位醫生決定為一位已停止 ERT 治療的患者恢復 ERT。該患者在 2.5 年多前接受了 ST-920 治療，截至第一行讀數日期，其 alpha-Gal A 活性維持在超生理水平，且其溶血-Gb3 水平基本穩定。

All of the other 17 patients who began the study on ERT and have withdrawn from ERT continue to remain off ERT as of today, with many experiencing benefit of ST-920 over and above what they were experiencing with ERT alone.

其餘 17 名開始接受 ERT 研究並已停止接受 ERT 治療的患者截至今天仍未接受 ERT 治療，其中許多人接受 ST-920 治療後獲得的益處超過了單獨接受 ERT 治療時獲得的益處。

All 32 patients have transitioned in the long-term follow-up study and the STAAR study is now complete. We continue to engage with the FDA ahead of our anticipated BLA submission under the accelerated approval pathway planned for as early as the first quarter of 2026. We are also looking forward to sharing additional clinical data at the 15th International Congress of Inborn Errors of Metabolism or ICIEM2025, taking place September 2 to 6, 2025, in Kyoto, Japan.

所有 32 名患者均已在長期追蹤研究中完成轉變，STAAR 研究現已完成。我們將根據計劃於 2026 年第一季加速審批的途徑，繼續與 FDA 保持溝通，爭取提交 BLA 申請。我們也期待在 2025 年 9 月 2 日至 6 日在日本京都舉行的第 15 屆國際先天性代謝錯誤大會 (ICIEM2025) 上分享更多臨床數據。

Before we move on and on behalf of our entire Fabry team at Sangamo, I want to take a moment to sincerely thank the patient and investigator who have participated in the STAAR study. Your dedication and commitment have been invaluable as we advance this treatment for such a debilitating and multifaceted condition towards registration. Thank you.

在我們繼續之前，我謹代表 Sangamo 的整個法布里病團隊，花點時間真誠地感謝參與 STAAR 研究的患者和研究人員。在我們推進這種針對如此衰弱和多方面的疾病的治療並使其獲得註冊的過程中，您的奉獻和承諾是無價的。謝謝。

Turning now to our neurology pipeline programs. As Sandy shared, this quarter, we became a clinical stage neurology genomic medicine company with the initiation of our first clinical site in the Phase 1/2 STAND study, evaluating ST-503, our investigational epigenetic regulator for patients with intractable pain due to idiopathic small fiber neuropathy or iSFN.

現在談談我們的神經病學管道項目。正如桑迪所分享的，本季度，我們成為一家臨床階段神經病學基因組醫學公司，並在 1/2 期 STAND 研究中啟動了我們的第一個臨床站點，評估 ST-503，這是我們針對因特發性小纖維神經病變或 iSFN 引起的頑固性疼痛患者進行研究的表觀遺傳調節劑。

This is an important milestone for Sangamo, and we're excited to be identifying patients in our first-ever neurology clinical trial. I want to thank everyone involved.

這對 Sangamo 來說是一個重要的里程碑，我們很高興能夠在我們的首次神經病學臨床試驗中確定患者。我要感謝所有參與的人。

We anticipate activating at least eight other clinical sites in the coming months, which we believe will further accelerate patient enrollment. We expect to dose the first patient in the fall of this year and anticipate having preliminary proof of efficacy data in the fourth quarter of 2026.

我們預計在未來幾個月內將啟動至少八個其他臨床站點，我們相信這將進一步加快患者入組速度。我們預計將於今年秋季為第一位患者進行給藥，並預計於 2026 年第四季獲得初步療效證明數據。

Our preclinical data for this program is compelling. By directly targeting the SCN9A gene, ST-503 has shown to precisely and potentially reduce the expression of Nav1.7 sodium channels in sensory neurons in animal models and significantly reduce pain hypersensitivity following a single intrathecal administration.

我們對該計畫的臨床前數據非常有說服力。透過直接針對 SCN9A 基因，ST-503 已被證明能夠精確且潛在地降低動物模型中感覺神經元中 Nav1.7 鈉通道的表達，並在單次鞘內給藥後顯著降低疼痛過敏。

ST-503 has been well tolerated in nonhuman primate with no off-target effect observed. And we plan to present updated nonclinical data at the 9th International Congress of Neuropathic Pain, taking place September 4 through 6 in Berlin, Germany.

ST-503 在非人類靈長類動物中耐受性良好，未觀察到脫靶效應。我們計劃在 9 月 4 日至 6 日在德國柏林舉行的第九屆國際神經性疼痛大會上展示最新的非臨床數據。

Finally, I am pleased to share progress in ST-506, our epigenetic regulator for the treatment of prion disease to be delivered intravenously using STAC-BBB. Earlier this quarter, we held a productive meeting with the UK's MHRA and aligned on the planned nonclinical safety study as well as the proposed clinical study design. We appreciated the collaborative nature of the discussion and their acknowledgment of the urgency to find a treatment for prion disease patient.

最後，我很高興與大家分享 ST-506 的進展，ST-506 是我們用於治療朊病毒疾病的表觀遺傳調節劑，將使用 STAC-BBB 透過靜脈注射的方式輸送。本季度早些時候，我們與英國 MHRA 舉行了富有成效的會議，就計劃中的非臨床安全性研究以及擬議的臨床研究設計達成了一致。我們讚賞討論的合作性質以及他們對尋找朊病毒病患者治療方法的緊迫性的認識。

We were also extremely proud to be selected to present during the prestigious Presidential Symposium at the recent ASGCT, Annual Meeting in New Orleans, where we showcased our potent combination of epigenetic regulation and capsid delivery technology in prion disease, including the profound survival benefit we observed when administered to post symptomatic mice.

我們也非常榮幸地被選中在最近於新奧爾良舉行的 ASGCT 年會上進行著名的總統研討會，在會上我們展示了我們在治療朊病毒疾病方面的表觀遺傳調控和衣殼遞送技術的強大組合，包括我們在對出現症狀的小鼠進行給藥時觀察到的顯著生存益處。

In addition, we described the sustained brain-wide suppression of prion protein expression in both mouse and nonhuman primate model, supporting the potential of ST-506 as a onetime therapeutic approach for prion disease.

此外，我們描述了小鼠和非人靈長類動物模型中朊病毒蛋白表達的持續全腦抑制，支持了 ST-506 作為朊病毒疾病一次性治療方法的潛力。

We have completed dose-range finding study and are preparing for the GLP tox study ahead of an anticipated CTA submission expected as early as mid-2026. I would like now to hand it back to Sandy for closing remarks. Sandy?

我們已經完成了劑量範圍探索研究，並正在為 GLP 毒性研究做準備，預計最早將於 2026 年中期提交 CTA。現在我想把發言時間交還給桑迪，請她做最後發言。沙？

Thank you, Nathalie. To close, we made strong pipeline advances this quarter. Firstly, we announced positive top line results from the registrational STAAR study in Fabry disease. We observed a positive mean annualized eGFR slope at 52 weeks across all dose patients in the study, which the FDA has agreed will serve as a primary basis of approval.

謝謝你，娜塔莉。最後，本季我們的管道業務取得了長足的進步。首先，我們宣布了法布瑞氏症註冊 STAAR 研究的正面頂線結果。我們觀察到研究中所有劑量患者在 52 週時的平均年化 eGFR 斜率均為正，FDA 已同意將其作為批准的主要依據。

Beyond renal function, we are pleased to observe a range of positive secondary endpoints and broader quality of life data, including a stabilization in cardiac endpoints. And importantly, ST-920 continued to be very well tolerated in the study without the need for preconditioning. We continue to engage with the FDA ahead of the planned BLA submission expected as early as the first quarter of 2026.

除了腎功能之外，我們很高興地觀察到一系列積極的次要終點和更廣泛的生活品質數據，包括心臟終點的穩定性。重要的是，ST-920 在研究中仍然具有良好的耐受性，無需預處理。我們將繼續與 FDA 保持聯繫，爭取最早於 2026 年第一季提交 BLA。

Secondly, this quarter, we became a clinical stage neurology genomic medicine company with the initiation of the first clinical site for the Phase 1/2 STAND study in chronic neuropathic pain. We expect to dose the first patient in the fall and anticipate having preliminary proof of efficacy data in the fourth quarter of 2026.

其次，本季度，我們成為一家臨床階段神經病學基因組醫學公司，並啟動了針對慢性神經性疼痛的 1/2 期 STAND 研究的第一個臨床站點。我們預計在秋季為第一位患者註射藥物，並預計在 2026 年第四季獲得初步療效證明數據。

And thirdly, we held a productive meeting with the MHRA for ST-506 in prion disease ahead of an anticipated CTA submission as early as mid '26.

第三，我們與 MHRA 就 ST-506 治療朊病毒病的會議富有成效，預計最早將於 26 年中期提交 CTA。

Moving now to broader business updates. Earlier this quarter, we completed an equity offering that we hope will bridge us to an anticipated Fabry commercialization agreement. Our current cash runway is expected to fund our planned operations into the fourth quarter of '25. And we remain highly focused on our critical task of securing a Fabry commercialization partner in the near term.

現在轉向更廣泛的業務更新。本季度早些時候，我們完成了股權發行，希望這將有助於我們達成預期的 Fabry 商業化協議。我們目前的現金流預計將為我們計劃在 25 年第四季的營運提供資金。我們仍然高度關注我們的關鍵任務，即在短期內找到法布里商業化合作夥伴。

We continue to advance business development negotiations for that potential Fabry commercialization agreement and are also engaging in broader business development discussions across our Sangamo pipeline and platforms, including our MINT platform.

我們繼續推進有關潛在 Fabry 商業化協議的業務發展談判，同時也在我們的 Sangamo 管道和平台（包括我們的 MINT 平台）上進行更廣泛的業務發展討論。

We remain focused on solving our long-term funding needs in order to enable us to advance our promising neurology genomic medicine pipeline.

我們仍然專注於解決我們的長​​期資金需求，以便我們能夠推進我們有前景的神經病學基因組醫學管道。

Operator, please open the line for questions.

接線員，請打開熱線以回答問題。

(Operator Instructions) Maury Raycroft, Jefferies.

（操作員指示）Maury Raycroft，Jefferies。

This is James on for Maury. Congrats on the progress. Just to start off, has the team already held the pre-BLA meeting with the FDA to discuss the potential path to approval using the 1-year eGFR data as a predictor for the 2-year eGFR benefit versus having to confirm the clinical benefit with two years of data from all patients. And if you had that meeting, could you share any takeaways from that discussion? Also, how important is the FDA alignment on the 2-year eGFR prediction in the context of the ongoing partner discussions?

這是詹姆斯 (James) 代替莫里 (Maury) 上場。恭喜你取得進展。首先，該團隊是否已經與 FDA 舉行了 BLA 前會議，討論使用 1 年 eGFR 數據作為 2 年 eGFR 益處的預測指標來獲得批准的潛在途徑，還是必須使用所有患者兩年的數據來確認臨床益處。如果您參加了那次會議，您能分享一下那次會議討論的要點嗎？此外，在正在進行的合作夥伴討論中，FDA 對 2 年 eGFR 預測的調整有多重要？

And I have a follow-up.

我還有一個後續問題。

Thank you for the question. So let me just restate, we held a meeting last year with the FDA where they agreed on accelerated approval. And at that meeting, they said that we could achieve accelerated approval with 1-year eGFR data and included a clause in it that said we might wish to submit 2-year data when that was available.

謝謝你的提問。因此，讓我重申一下，我們去年與 FDA 舉行了一次會議，他們同意加速批准。在那次會議上，他們表示，我們可以透過 1 年的 eGFR 數據獲得加速批准，並在其中包含一條條款，表示我們可能希望在 2 年數據可用時提交這些數據。

We currently have 32 patients at one year and 19 patients at two year. And the two data sets are very similar and complementary. We have not yet held our pre-BLA meeting because it's not the time to do it yet and have plans in place of when and how we're going to do that.

我們目前有 32 名一年期患者和 19 名兩年期患者。而且這兩組數據非常相似且互補。我們尚未舉行 BLA 前會議，因為現在還不是時候，我們還沒有計劃何時以及如何舉行會議。

The pre-BLA meeting is very much an operational meeting where you agree with the agency on what it is that you have to do to fulfill the BLA requirements, what sections, how it has to be presented, et cetera. We have no expectation that the agency will require anything other than the 1-year data for accelerated approval. And we are sure that we will end up providing them with yearly updates as these patients advance.

BLA 前會議本質上是一場營運會議，您將在會議上與機構商定為滿足 BLA 要求必須做什麼、包括哪些部分、如何呈現等等。我們預計該機構不會要求除 1 年數據之外的任何其他數據來獲得加速批准。我們確信，隨著這些患者的病情進展，我們最終會為他們提供年度更新資訊。

So just to remind you all, the latest -- the earliest patient is now 4.5 years out, and the data looks very consistent and very stable.

所以提醒大家，最新的——最早的患者現在已經 4.5 年了，數據看起來非常一致且非常穩定。

Got it. And then just another one. For the upcoming presentation SSIEM, what additional insights should we anticipate? Can we expect any details at the conference regarding the meta-analysis or new baseline characteristics such as proteinuria? Also, will you show individual eGFR trajectories or alpha-Gal levels for each patient or just the average?

知道了。然後又一個。對於即將舉行的 SSIEM 演示，我們應該期待哪些額外的見解？我們可以在會議上了解有關薈萃分析或蛋白尿等新基線特徵的任何詳細資訊嗎？此外，您會顯示每位患者的個別 eGFR 軌跡或 alpha-Gal 水準還是僅顯示平均值？

So I'll pass this on to Nathalie. But before I do that, I would say it would be really unusual in a patient data set of 32 patients where it's comparing the body of patients before compared to after to dissect out and show each individual patient. So we don't intend to show that at the presentation -- at the meeting in Japan. We may do that as part of a larger publication that we're working on at the moment. Nathalie?

所以我會把這個傳給娜塔莉。但在此之前，我想說，在 32 名患者的患者資料集中，比較患者治療前後的身體狀況，以解剖並展示每個患者的情況，這真的很不尋常。因此，我們不打算在日本的會議上展示這一點。我們可能會將其作為目前正在進行的更大出版物的一部分。娜塔莉？

Yes. Thank you, Sandy. We look forward to sharing additional clinical data at the ICIEM conference. We plan to present the top line data with additional details compared to the press release issued back in June. We encourage you to review the data presented at ICIEM that will also be available in our website once the embargo has lifted.

是的。謝謝你，桑迪。我們期待在 ICIEM 會議上分享更多臨床數據。我們計劃提供與 6 月發布的新聞稿相比更詳細的頂線數據。我們鼓勵您查看 ICIEM 上提供的數據，一旦禁運解除，這些數據也將在我們的網站上提供。

But Nathalie, our plans would be to say a little more about this.

但娜塔莉，我們的計劃是就此多說一點。

Absolutely. Absolutely. Yes. There will be more details on some of the endpoints. We're still finalizing the presentation, but there absolutely will be additional details.

絕對地。絕對地。是的。將會有關於某些端點的更多詳細資訊。我們仍在完成演示，但肯定會有更多細節。

(Operator Instructions) Ritu Baral, TD Cowen.

（操作員指示）Ritu Baral，TD Cowen。

This is Joshua Fleishman on the line for Ritu. Curious, how do you believe ST-503's efficacy in Nav1.7 will compare to the recent small molecule Nav1.8 inhibitors? And have recent trial outcomes in Nav1.8 changed your conviction in Nav1.7 as a target?

我是 Joshua Fleishman，代表 Ritu 進行接線。好奇，您認為 ST-503 在 Nav1.7 中的功效與最近的小分子 Nav1.8 抑制劑相比如何？最近 Nav1.8 的試驗結果是否改變了您對 Nav1.7 作為目標的信念？

So thank you very much for your question, and we've spent a lot of time looking at that data and discussing it and landed that we are even more convinced that NaV1.7 was the right target for us to go for. As you will -- as I think we've discussed before, because we are using a genomic way to target it and target the specific regulatory sequences of that gene, we could have gone for Nav1.8 or Nav1.7. And our belief was that the Nav1.7 control of the action potential that controls the pain signal was a more fundamental control.

非常感謝您的提問，我們花了很多時間查看並討論這些數據，最終我們更加確信 NaV1.7 是我們追求的正確目標。正如你所想——我想我們之前討論過，因為我們採用基因組學方法來靶向該基因，並靶向該基因的特定調控序列，所以我們可以選擇 Nav1.8 或 Nav1.7。我們認為，Nav1.7 對控制疼痛訊號的動作電位的控制是一種更根本的控制。

And one of the real pieces of evidence for that is that there are people out there that have got spontaneous mutations of Nav1.7 that just don't feel any pain. And there -- where it is very rare incidence is reported of Nav1.8 mutations, and they don't seem to have complete suppression of pain. So perhaps it isn't so surprising that the 1.8 results reported by Vertex were not as efficacious as had been hoped.

其中一個真實證據是，有些人的 Nav1.7 基因發生了自發性突變，因此感覺不到任何疼痛。據報導，Nav1.8 突變的發生率非常低，而且似乎無法完全抑制疼痛。因此，Vertex 報告的 1.8 結果並不像人們所希望的那樣有效，這也許並不奇怪。

So we are at the stage now of activating the study and hopefully, we'll have identified and recruited a patient soon. And we look forward to this. It's a dose-range finding study. And in our mouse studies, we see evidence of a dose-range response even in individual groups of mice. And we look forward to showing the suppression of pain because it's a really important unmet medical need.

因此，我們現在正處於啟動研究的階段，希望我們能很快確定並招募一名患者。我們對此充滿期待。這是一項劑量範圍探索研究。在我們的小鼠研究中，我們看到了劑量範圍反應的證據，即使在單一小鼠組中也是如此。我們期待展示對疼痛的抑製作用，因為這是一個非常重要的未滿足的醫療需求。

Great credit to Lilly and Vertex and others who are now pushing forward with non-opioid pain relief, but we believe Nav1.7 is the right target to go for.

禮來公司、福泰公司和其他正在大力推動非鴉片類止痛藥研發的公司值得稱讚，但我們相信 Nav1.7 是正確的目標。

Yanan Zhu, Wells Fargo.

朱亞南，富國銀行。

This is Kuan-Hung for Yanan. So our question is also around Fabry. We are wondering, have you done any survey to either patients or physicians to understand that with the current product profile, what could be the potential adoption rate?

這是延安的關鴻。所以我們的問題也是關於法布瑞氏症的。我們想知道，您是否對患者或醫生做過任何調查，以了解當前的產品概況，潛在的採用率是多少？

Can you just repeat that question again, please?

你能再重複一次這個問題嗎？

Sure. We are wondering, have you done any survey to either physicians or patients to understand that with the current product profile, what could be the potential adoption rate?

當然。我們想知道，您是否對醫生或患者做過任何調查，以了解目前產品概況的潛在採用率是多少？

Nathalie, you spent a lot of time with the patient support groups. What's your thoughts on this?

娜塔莉，你花了很多時間與病人支持小組在一起。您對此有何看法？

Yes. So from the patient advocacy group, they are waiting for a better solution for a long time. The current standard of care is burdensome, but -- and there is some small improvement in their disease, but it really does not address all the symptom of the disease. It's a biweekly infusion that lasts many hours, which really impact their daily life.

是的。因此從患者權益團體來看，他們長期在等待更好的解決方案。目前的護理標準很繁重，但是——他們的病情確實有一些小的改善，但它並沒有真正解決疾病的所有症狀。每兩週進行一次輸液，持續數小時，這對他們的日常生活有很大影響。

What we showed in our top line results for our patients that we have really improvement in the quality of life. It's a onetime injection and patients are uniformly saying this is what they're waiting for, for a long time.

我們的頂級結果顯示，患者的生活品質確實得到了改善。這是一次性注射，患者一致表示這是他們等待已久的注射。

So they're really eager to see this drug approved. Some of the patients, because it's a genetic condition, want their family to have access to the product as soon as possible. So we have an overwhelming response from the patient community.

所以他們非常渴望看到這種藥物獲得批准。由於這是一種遺傳疾病，一些患者希望他們的家人能夠盡快獲得該產品。因此，我們得到了患者群體的熱烈回應。

Our PI, our principal investigators that are taking care of those patients are also extremely enthusiastic. When they're reviewing the data, they're really very impressed by what we've accomplished. So we do believe that the adoption rate, both from the patient side and the doc side will be very impressive.

我們的 PI，照顧這些患者的首席研究員也非常熱情。當他們審查數據時，他們對我們所取得的成就印象深刻。因此，我們確實相信，無論是從患者方面還是醫生來看，採用率都會非常令人印象深刻。

And Nathalie, you met with cardiac experts recently, and they were very impressed.

娜塔莉，您最近會見了心臟科醫生，他們給您留下了深刻的印象。

Yes. We've met with cardiac experts to review our top line data with all the cardiac endpoints I mentioned. And first of all, they mentioned that we had many, many measures in the cardiac function that other study didn't have that was the most comprehensive set of data, and they were also very impressed with the data and the stabilization of the cardiac function. So we're focusing for the primary basis of approval on the eGFR slope and the renal function, but this is also a very important aspect of Fabry disease.

是的。我們已經與心臟科醫生會面，審查了我們最重要的數據以及我提到的所有心臟終點。首先，他們提到我們在心臟功能方面有很多其他研究所沒有的測量指標，這是最全面的數據集，他們對這些數據和心臟功能的穩定性也印象深刻。因此，我們將批准的主要依據集中在 eGFR 斜率和腎功能上，但這也是法布瑞氏症的一個非常重要的方面。

And the thing that -- when we go to these conferences, we often have Fabry patients come to us and tell us that they have received our treatment and how much their life has changed. And that kind of conversation spreads throughout the Fabry support groups and populations that we feel there's a real energy and anticipation.

當我們參加這些會議時，經常會有法布瑞氏症患者來找我們，告訴我們他們接受了我們的治療，他們的生活發生了多大的改變。這種對話在法布瑞氏症支持團體和人群中廣泛傳播，我們感覺到其中蘊含著真正的活力和期待。

And then the final thing I would say, as you look at the 17 patients who came in on ERT who have remained off of ERT, and that's over 1,000 infusions, and they feel better. And the SF-36 scores say that they are better on this treatment. And so I really look forward to solving the commercial partner and getting this medicine to patients as soon as possible.

最後我想說的是，你看，有 17 名接受 ERT 治療的患者已經停止接受 ERT 治療，雖然輸液次數超過 1,000 次，但他們感覺好多了。SF-36 評分錶明，這種治療的效果更好。因此，我非常期待解決商業合作夥伴問題並儘快將這種藥物提供給患者。

And I don't know if you can comment on this, but with the potential partners, do they share the same view? Or are they looking for something else, beyond what you have shared with the public?

我不知道您是否可以對此發表評論，但對於潛在的合作夥伴，他們是否有相同的看法？或者除了您與公眾分享的內容之外，他們還在尋找其他東西？

So uniformly, all of the partners have -- all the potential partners have said how excited they are of the data. They are completely convinced that this is a medicine that is both safe and shows an effectiveness and a benefit to patients. I don't think -- I am sure you, like us, are aware of the environment for gene therapy at the moment in the United States and the stability that we all hope for and look for from the agency.

因此，所有合作夥伴——所有潛在合作夥伴都一致表示他們對這些數據感到非常興奮。他們完全相信這是一種既安全又有效、對病人有益的藥物。我不認為——我相信您和我們一樣，了解美國目前基因治療的環境以及我們都希望並期待機構的穩定性。

Sangamo has had great interactions with the agency and continues to have them. And the partners just want to know that this is a stable place where their medicine will be well appreciated and taken forward.

Sangamo 與該機構一直保持著良好的互動，並將繼續保持這種互動。合作夥伴只想知道這是一個穩定的地方，他們的藥物將得到很好的認可和推廣。

There is a second piece that makes the Sangamo discussions a little unusual and that since we got accelerated approval last year, we have compressed the activities that take you to the BLA submission into a very short time, which means that there are lots of interactions and lots of data points and new information coming where partners who will have in the past been interested in seeing the top line data have wanted to know that we have, as we do have agreement on the CMC. So we feel that this product is increasingly derisked.

第二點使得 Sangamo 討論有些不尋常，自從我們去年獲得加速批准以來，我們將提交 BLA 的活動壓縮到了很短的時間內，這意味著會有大量互動、大量數據點和新信息，過去對查看頂級數據感興趣的合作夥伴希望知道我們已經擁有這些數據，因為我們確實就 CMC 達成了協議。因此我們認為該產品的風險正在逐漸降低。

And now for the partners, that gives them comfort to be able to move ahead. And we -- I'm very pleased with the pace of negotiations at the moment and look forward to finding a positive way through this.

對於合作夥伴來說，這讓他們感到安心，能夠繼續前進。我對目前的談判進度感到非常滿意，並期待找到積極的解決方案。

(Operator Instructions) Gena Wang, Barclays.

（操作員指示）巴克萊銀行的 Gena Wang。

This is Tony on for Gena. I guess just questions on upcoming updates in September. What data points should we be looking for, for the pain program in terms of how they would compare to existing NAV programs? And then also what incremental color should we expect for the Fabry update?

這是 Tony 代替 Gena 上場的。我想這只是關於九月即將推出的更新的問題。就疼痛計劃與現有 NAV 計劃的比較而言，我們應該尋找哪些數據點？那麼，對於 Fabry 更新，我們還能期待什麼增量色彩呢？

So the data that we'll be presenting at the conference in Berlin is preclinical data, and we will share more information about our GLP tox study in NHP and there will be also all the information on the mouse data and the non-GLP tox study. But the additional GLP showing the safety and efficacy of the product will be presented.

因此，我們將在柏林會議上展示的數據是臨床前數據，我們將分享更多有關 NHP 的 GLP 毒性研究的信息，還將提供有關小鼠數據和非 GLP 毒性研究的所有信息。但將提供顯示產品安全性和有效性的附加 GLP。

Luis Santos, H.C. Wainwright.

路易斯桑托斯、H.C. 溫賴特。

My question is mostly regarding your cash runway and cash burn associated with -- especially associated with the initiation of the STAND trial where you plan to continue activating more sites and dosing the first patient by the end of the year. How is that going to weigh on your cash burn? And also, do you have any updates on your MINT platform? Any recent additional data or coming?

我的問題主要是關於您的現金流和現金消耗——特別是與 STAND 試驗的啟動相關的問題，您計劃在年底前繼續啟動更多站點並為第一位患者進行給藥。這會對您的現金消耗產生什麼影響？另外，您的 MINT 平台有更新嗎？最近有任何附加數據或即將出現的數據嗎？

Louis, this is Prathyusha. I'll take the first one. So our intention is to continue taking the Nav1.7 program forward and dose patients as intended. As Sandy mentioned, our number one priority is finding a Fabry commercialization partner, and that will help us solve for our funding needs in both in the long term and the short term. And maybe let me turn it over to Greg to answer the question on the MINT platform.

路易斯，這是 Prathyusha。我要第一個。因此，我們的目的是繼續推進 Nav1.7 計劃並按預期為患者提供劑量。正如桑迪所提到的，我們的首要任務是找到法布里商業化合作夥伴，這將有助於我們解決長期和短期的資金需求。也許我可以把這個問題交給格雷格來回答，關於 MINT 平台的問題。

Yes. Thanks, Louis. We were happy to show the updates on the MINT platform at ASGCT recently. You probably saw lots of data there in relation to integration and -- or improvements in integration rates in primary cell types. So we're happy to share that data this year, and we continue to show that data to interested parties and engage in discussions with parties interested in collaborating with us.

是的。謝謝，路易斯。我們很高興最近在 ASGCT 上展示了 MINT 平台的更新。您可能看到了許多與原代細胞類型的整合或整合率提高有關的數據。因此，我們很高興今年分享這些數據，並且我們將繼續向有興趣的各方展示這些數據，並與有興趣與我們合作的各方進行討論。

And we have a number of ongoing discussions on the MINT platform.

我們在 MINT 平台上進行了許多討論。

And going back to the STAND trial, you said that you might have data by the end of next year. What kind of data should we expect?

回到 STAND 試驗，您說過您可能在明年年底之前獲得數據。我們應該期待什麼樣的數據？

So you can expect safety data from the patient and early efficacy data for the dose escalation trial.

因此，您可以預期獲得患者的安全數據和劑量遞增試驗的早期療效數據。

And Louis, just -- you can be sure we're doing all the standard pain study scores, sleep assessment scores, even suicidality scores because these are patients whose life is dominated and tragically dominated by this. We will be -- sorry, it would be a 12-week endpoint that we would be showing by the end of next year, but we'll be following them long term because we think the huge advantage of Nav1.7 as a genomic medicine is a long-term benefit that will bring to the patients.

路易斯，你可以肯定，我們正在進行所有標準的疼痛研究評分、睡眠評估評分，甚至自殺傾向評分，因為這些患者的生活都被這些疾病所主宰，甚至是悲慘地主宰。抱歉，我們將在明年年底之前展示一個 12 週的終點，但我們將長期追蹤它們，因為我們認為 Nav1.7 作為基因組藥物的巨大優勢是將為患者帶來的長期利益。

Yes. We expect to see a reduction.

是的。我們預計會看到減少。

Go ahead.

前進。

No, no, sorry, go ahead, please.

不，不，對不起，請繼續。

I was just going to say thank you for the added color. But if you have more color, always welcome.

我只是想對添加的顏色表示感謝。但如果您有更多顏色，我們隨時歡迎。

No, I think Sandy mentioned it. So we're good.

不，我認為桑迪提到過這一點。所以我們很好。

I am showing no further questions at this time. I would now like to turn the call back to Louise Wilkie for closing remarks.

我目前沒有其他問題。現在我想請路易斯‧威爾基 (Louise Wilkie) 致閉幕詞。

Thank you once again for joining us today and for your questions. As a reminder, you can access our presentation on the Investor Relations section of the Sangamo website. We look forward to keeping you updated on our future developments.

再次感謝您今天的參與和提問。提醒一下，您可以在 Sangamo 網站的投資者關係部分存取我們的簡報。我們期待向您通報我們未來的發展。

Thank you for your participation in today's conference. This does conclude the conference. You may now disconnect.

感謝大家參加今天的會議。會議到此結束。您現在可以斷開連線。

Thank you. We appreciate your help, Felicia.

謝謝。我們非常感謝你的幫助，費莉西亞。

Thank you, Felicia.

謝謝你，費莉西亞。