Sangamo Therapeutics Inc (SGMO) 2025 Q3 法說會逐字稿

Good morning and welcome to Sangamo Therapeutics third quarter 2025 conference call. Please be advised that today's conference is being recorded. 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.

早上好，歡迎參加 Sangamo Therapeutics 2025 年第三季電話會議。請注意，今天的會議正在錄影。現在我謹將會議交給今天的發言人，投資者關係和企業傳播主管路易絲·威爾基女士。請繼續。

Thank you. Good morning, everyone.

謝謝。各位早安。

Thank you for joining us on the call today. On this call are several members of the Sangama executive Leadership team, including Sandy Macrae, Chief Executive Officer, Nathalie Dubois-stringfellow, Chief Development Officer, Greg Davis, Head of Research and Technology, Pratyusha duraibabu, Principal Financial Officer.

感謝您今天參加我們的電話會議。參加此次電話會議的有桑加瑪執行領導團隊的幾位成員，包括執行長桑迪·麥克雷、首席發展官娜塔莉·杜波依斯-斯特林費洛、研發主管格雷格·戴維斯、首席財務官普拉蒂尤莎·杜萊巴布。

Slides from our corporate presentation can be found on our websites Sangamo.com and under the presentations page of the investors and media section. This call includes forward-looking statements regarding Sangamo's current expectations.

您可以在我們網站 Sangamo.com 的投資者和媒體板塊的簡報頁面找到我們公司簡報的幻燈片。本次電話會議包含有關 Sangamo 目前預期的前瞻性陳述。

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 Vibary Disease program, the anticipated plans and timelines 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.

這些聲明包括但不限於與 Sangamo 的現金儲備、Sangamo 獲取額外資金的計劃以及其持續經營能力相關的聲明。Sangamo 的產品候選藥物和技術的治療和商業潛力及價值，Sangamo 建立和維持合作與戰略夥伴關係的能力（包括其 Vibary 疾病項目），Sangamo 及其合作者在臨床試驗、臨床數據展示和發布、監管申報和監管批准方面的預期計劃和時間表，即將到來的催化劑和里程碑，以及其他非歷史事實的陳述。

Actual results may differ materially from what we discussed 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 10k for the fiscal year ended December 31, 2024, and our quarterly report on Form 10Q for the fiscal quarter ended September 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內容有重大差異。這些聲明受到某些風險和不確定性的影響，這些風險和不確定性已在我們向美國證券交易委員會提交的文件中進行了討論，特別是在我們截至 2024 年 12 月 31 日的財政年度的 10-K 表格年度報告、截至 2025 年 9 月 30 日的財政季度的 10-Q 季度報告以及對美國證券委員會的後續證券

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.

今天所作的前瞻性陳述僅代表截至今天的情況，除法律要求外，我們不承擔更新此類資訊的義務。請注意，所有關於我們未來計劃和預期的前瞻性聲明均取決於我們能否獲得足夠的額外資金。

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

現在我將把電話交給我們的執行長桑迪·麥克雷。

Thank you, Louise, and good morning to everyone joining the call today.

謝謝路易絲，也祝今天參加電話會議的各位早安。

This quarter we continue to advance our clinical and pre-clinical pipeline while managing our cash resources carefully.

本季度，我們將繼續推進臨床和臨床前研發管線，同時謹慎管理現金資源。

In September, we presented promising detailed clinical data from a registration of star study in Fari disease demonstrating the potential for ST 920 as a one-time durable treatment of the underlying pathology of Fari disease for all types of Fari disease patients.

9 月，我們公佈了一項針對 Fari 病的明星研究註冊的詳細臨床數據，這些數據很有希望，證明了 ST 920 有潛力成為針對所有類型 Fari 病患者的潛在病理的一次性持久治療方案。

And in October we were pleased to hold a meeting with the FDA to discuss the proposed efficacy and safety data package for a planned BLA submission where in the meeting minutes the FDA reaffirmed its October 2024 agreement to use EGFR slope as an endpoint to support an accelerated approval pathway.

10 月份，我們很高興與 FDA 舉行了一次會議，討論了計劃提交的 BLA 的擬議療效和安全性資料包。會議記錄顯示，FDA 重申了 2024 年 10 月達成的協議，即使用 EGFR 斜率作為終點，以支持加速審批途徑。

A particular highlight for me this quarter was attending the 15th annual Fari Family Education Conference that brought together more than 200 Fari patients, family members, and volunteers to provide educational presentations and gather insights from patients, including those who have received SC 920.

本季對我來說最特別的亮點是參加了第 15 屆 Fari 家庭教育年會，該年會匯聚了 200 多名 Fari 患者、家屬和志願者，旨在提供教育講座並收集患者的見解，其中包括接受過 SC 920 治療的患者。

This is an event we are privileged to attend each year, and it was humbling to spend time with this group of inspirational people of all age groups to learn more about their experiences with Fari disease.

每年我們都很榮幸能參加這個活動，能夠與這群各個年齡層、充滿鼓舞人心的人們共度時光，了解他們與法里氏病抗爭的經歷，我們深感榮幸。

I found it striking to hear firsthand the challenges these patients face, including their experiences with currently available treatments, yet also see the hope they have for treatment breakthroughs and their strong understanding of scientific advances and their unwavering support for one another.

親耳聽到這些患者面臨的挑戰，包括他們目前可用的治療方法，讓我印象深刻；同時，我也看到了他們對治療突破的希望，他們對科學進步的深刻理解，以及他們彼此之間堅定不移的支持。

I came away from the event more convinced than ever that patients are seeking an alternative to current standards of care alongside the peaks and troughs and associated symptoms that they can bring.

參加完這次活動後，我更加確信，患者正在尋求一種替代當前標準治療的方案，以應對治療過程中可能出現的波動和相關症狀。

Gene therapy, where the alpha-galli enzyme is expressed all day, every day by the liver, is a fundamentally different treatment modality to what is available today.

基因療法是指讓肝臟每天24小時不間斷地表達α-雞精酶，這與目前可用的治療方式有著根本的不同。

I was lucky to meet with some patients who received SC 920 as part of the STAR study, and their enthusiasm and excitement was overwhelming. They couldn't wait to tell me their experiences, and one patient approached me to share how SC 920 has completely transformed their life.

我很幸運地見到了一些作為 STAR 研究的一部分接受 SC 920 治療的患者，他們的熱情和興奮之情令人感動。他們迫不及待地想告訴我他們的經歷，其中一位患者走過來和我分享了 SC 920 如何徹底改變了他的生活。

We have a responsibility to bring this medicine to the Fabry community. In our prioritized neurology pipeline, we are excited now to be recruiting and enrolling patients in the phase 12 stand study in chronic neuropathic pain, our first ever neurology clinical study following the activation of the 1st 2 clinical sites.

我們有責任將這種藥物帶給法布瑞氏症患者族群。在我們優先發展的神經病學產品線中，我們很高興現在開始招募和登記慢性神經性疼痛 12 期臨床試驗的患者，這是我們在前 2 個臨床中心啟動後開展的首個神經病學臨床研究。

We also continue to advance our riam program ahead of the planned CTA submission next year.

我們也在繼續推進我們的 riam 項目，為明年計劃中的 CTA 提交做準備。

I would like to now hand directly over to Nathalie Dubois Stringfellow, our Chief Development Officer, to provide additional details on these important programs.

現在，我謹將麥克風交給我們的首席發展長娜塔莉·杜波依斯·斯特林費洛，讓她詳細介紹這些重要的項目。

Prathyusha Duraibabu, our principal financial officer, and I will then close the call by summing the key business and financial takeaways from this quarter.

接下來，我和財務長 Prathyusha Duraibabu 將總結本季的主要業務和財務要點，結束本次電話會議。

Nathalie.

娜塔莉。

Thank you, Sandy. First, I am pleased to share updates from our registrational phase 12A 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，我很高興與大家分享我們評估 Isaralgagene civaparvovec 或 ST 920（我們正在研究的用於治療成人法布里病的基因療法）的註冊 12A 期研究的最新進展。

This quarter, we presented encouraging detailed clinical data at the ICIEM 2025 conference in Kyoto, Japan. As we have shared previously, after 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 patient dosed in this study. Furthermore, a positive mean annualized EGFR slope of 1.7 was observed in the 19 patients who have achieved two years of follow-up.

本季度，我們在日本京都舉行的 ICIEM 2025 會議上展示了令人鼓舞的詳細臨床數據。正如我們之前分享的那樣，在單次注射 ST 920 後，本研究中所有 32 名接受治療的患者在 52 週時均觀察到平均年化估計腎小球濾過率 (EGFR) 斜率接近 2。此外，在 19 名完成兩年追蹤的患者中觀察到平均年化 EGFR 斜率為 1.7。

Supportive mean annualized EGFR slopes were also observed across a variety of patient subgroups, including gender, baseline ERT status, Fabri disease type, and baseline EGFR, showing consistency in effect across FABRI patient in the study.

在各種患者亞組中也觀察到了支持性的平均年化 EGFR 斜率，包括性別、基線 ERT 狀態、Fabri 疾病類型和基線 EGFR，表明該療法對研究中的 FABRI 患者俱有一致性療效。

For the first time we share cardiac data including stable cardiac morphology, stable cardiac function, and stability in early marker of cardiac damage in the 32 patients with at least 52 weeks of follow-up.

我們首次分享了心臟數據，包括穩定的心臟形態、穩定的心臟功能以及至少追蹤 52 週的 32 名患者的心臟損傷早期標記的穩定性。

These data are encouraging, particularly given that cardiac disease is a leading cause of death in Fabri disease patients.

這些數據令人鼓舞，尤其考慮到心臟病是法布瑞氏症患者的主要死因。

As we have outlined previously, a range of key secondary endpoints were also positive. We continue to see strong durability in the study, up to 4.5 years for the longest treated patient, and we are pleased to see an encouraging ongoing safety profile. Indeed, every day that passes, we accumulate more data, and as of today, we are pleased to have 3 patients with at least 4.5 years of follow-up.

正如我們之前概述的那樣，一系列關鍵次要終點也取得了積極成果。研究中我們持續觀察到了良好的持久性，接受治療時間最長的患者療效持續了 4.5 年，我們很高興看到其安全性持續良好。事實上，隨著時間的推移，我們每天都在累積更多的數據，截至今天，我們很高興地看到有 3 位患者接受了至少 4.5 年的追蹤。

We believe that these data demonstrate the potential of ST-920 to provide meaningful and long-lasting clinical benefit to a wide range of fibro disease patients, even above current standards of care.

我們相信這些數據表明，ST-920 有潛力為各種纖維化疾病患者帶來有意義且持久的臨床益處，甚至優於目前的治療標準。

In October, we held a meeting with the FDA to discuss the proposed efficacy and safety data package ahead of the planned BLA submission. We are pleased that the FDA meeting minutes reiterated the October 2024 agreement that we may use EGFR slope as an endpoint to support an accelerated approval pathway, agree on the adequacy of the safety package to support the BLA submission, and provided valuable input on the clinical data package.

10 月份，我們與 FDA 舉行了一次會議，討論了在計劃提交 BLA 之前提出的療效和安全性資料包。我們很高興 FDA 會議紀要重申了 2024 年 10 月的協議，即我們可以使用 EGFR 斜率作為終點來支持加速審批途徑，同意安全資料包足以支持 BLA 提交，並就臨床數據包提供了寶貴的意見。

We continue to prepare for anticipated BLA submission under the accelerated approval pathway planned for as early as the first quarter of 2026.

我們繼續為預計最早於 2026 年第一季根據加速審批途徑提交的生物製品許可申請 (BLA) 做準備。

Next, I'd like to focus on our prioritized neurology pipeline. As this quarter we commence, patient enrollment and recruitment in the phase 12 stent study evaluating ST 503, our investigational epigenetic regulator for patients with intractable pain due to small fiber neuropathy or SFN, following the activation of our first two clinical sites. SFN is a truly debilitating chronic neuropathic pain.

接下來，我想重點介紹我們優先發展的神經病學人才培養計劃。本季度，隨著我們前兩個臨床中心的啟動，我們將開始對 ST 503（一種用於治療小纖維神經病變或 SFN 引起的頑固性疼痛的在研表觀遺傳調節劑）進行 12 期支架研究的患者招募和登記工作。小纖維神經病變是一種真正令人痛苦的慢性神經性疼痛。

Impacting more than 650,000 people across the US, Europe, and Japan. We're thrilled to be recruiting patients for our first ever neurology genomic medicine clinical study and expect to dose the first patient in the coming months.

影響範圍遍及美國、歐洲和日本，超過 65 萬人。我們很高興能夠招募患者參與我們首個神經基因組醫學臨床研究，並預計在未來幾個月內給第一位患者用藥。

This quarter we are also pleased to present updated non-clinical data. At the 9th International Congress on Neuropathic Pain in Berlin, Germany, which demonstrated the durability, potency, and selectivity of ST 503 in non-human primates. Alongside a favorable safety profile.

本季我們也很高興地向大家介紹更新後的非臨床數據。在德國柏林舉行的第九屆國際神經性疼痛大會上，證明了 ST 503 在非人靈長類動物中的持久性、效力和選擇性。同時具備良好的安全性能。

We believe the preclinical data for this program is compelling, and we look forward to seeing how this translates into humans.

我們認為該計畫的臨床前數據令人信服，我們期待看到它在人體試驗中的效果。

We believe chronic pain is an area of strong market potential, and we're particularly encouraged by the FDA's recent draft guidance on the development of non-opioid analgesics for chronic pain, which seeks to accelerate safe and effective non-opioid treatment and to reduce prescription-related opioid misuse.

我們認為慢性疼痛是一個具有強大市場潛力的領域，我們尤其受到 FDA 最近發布的關於開髮用於治療慢性疼痛的非阿片類鎮痛藥的指導草案的鼓舞，該草案旨在加速安全有效的非阿片類藥物治療，並減少與處方相關的阿片類藥物濫用。

We stand with the FDA in seeking safe, effective alternative pain relief options. As a reminder, this is a dose escalation study which, if positive, could allow us to broaden into other potentially high value indication such as trigeminal neuralgia or oncology related chronic pain.

我們與美國食品藥物管理局站在一起，尋求安全有效的替代止痛方案。提醒大家，這是一項劑量遞增研究，如果結果積極，我們可以擴展到其他潛在的高價值適應症，例如三叉神經痛或腫瘤相關的慢性疼痛。

NA 1.7 is a well proven target with human genetic validation which we believe provide pain franchise potential. Finally, moving to ST 506, our epigenetic regulator for the treatment of prion disease to be delivered intravenously using our neurotropic STA BBB capsule.

NA 1.7 是一個經過充分驗證的靶點，並已通過人類遺傳學驗證，我們認為它具有治療疼痛的潛力。最後，讓我們來看看 ST 506，這是一種用於治療朊病毒病的表觀遺傳調節劑，將使用我們的神經營養性 STA BBB 膠囊進行靜脈注射。

This quarter we continue to advance clinical trial application or CTA enabling activities for the program ahead of our expected CTA submission as early as mid-2026.

本季我們將繼續推進該計畫的臨床試驗申請或 CTA 準備工作，預計最早將於 2026 年年中提交 CTA。

Following on from our productive meeting with the UK's MHRA earlier this year, where we align on non-clinical safety and the clinical study design, this quarter we were pleased to hold another productive interaction with the MHRA, this time to align on the planned chemistry, manufacturing and control CMC strategy for the anticipated CTA submission.

繼今年稍早與英國藥品和保健產品監管署 (MHRA) 舉行富有成效的會議，就非臨床安全性和臨床研究設計達成一致之後，本季度我們很高興與 MHRA 再次進行了富有成效的互動，這次是為了就預期提交的 CTA 申請的化學、製造和控制 (CMC) 策略達成一致。

In November we presented updated precall data at the prion 2025 conference which demonstrated the potential combination of epigenetic regulator and capsid delivery technology for the treatment of prion disease, including a profound survival extension that was observed in an aggressive mouse model of prion disease alongside widespread bread delivery and significant prion reduction in non-human primates.

11 月，我們在 prion 2025 會議上展示了更新的 precall 數據，證明了表觀遺傳調節劑和衣殼遞送技術在治療朊病毒病方面的潛在結合，包括在病毒病侵襲性小鼠模型中觀察到的顯著的生存期延長，以及廣泛的麵包遞送和非人靈長類動物中朊病毒的顯著減少。

Based on this compelling clinical data, we are preparing for the CTA submission to test 506 in the clinic. Given the rapidly deadly nature of prion disease with no currently available treatment option, we anticipate demand for this planned study to be high, with a short time frame to an expected data readout.

基於這些令人信服的臨床數據，我們正在準備提交 CTA 申請，以便在臨床上測試 506。鑑於朊病毒病具有快速致死的特性，且目前尚無有效的治療方案，我們預計這項計劃中的研究需求量會很高，預計在短時間內即可獲得數據結果。

Furthermore, this study would mark the first inhuman testing of our STA BBB capsid, which, if successful, could unlock a broader neurology pipeline for advancement, including with potential partners.

此外，這項研究將標誌著我們STA BBB衣殼首次進行非人體測試，如果成功，可能會開啟更廣泛的神經學發展路徑，包括與潛在合作夥伴的合作。

We have deliberately chosen two lead neurology assets that use different delivery mechanisms and bring different development risks. We believe both of them independently offer significant potential value and provide additional expansion opportunity into related neurological indications.

我們特意選擇了兩種採用不同給藥機制、具有不同研發風險的神經科領先資產。我們認為它們各自具有巨大的潛在價值，並為相關神經系統疾病的進一步拓展提供了機會。

I would like now to hand over to Prathyusha duraibabu, our principal financial officer, to provide a financial update. Prathyusha .

現在我將把發言權交給我們的財務長 Prathyusha duraibabu，請她介紹一下財務方面的最新情況。普拉蒂尤莎。

Thank you, Nathalie.

謝謝你，娜塔莉。

This quarter we continue to diligently prioritize and control spend while seeking ways to extend our cash runway as we continue business development discussions for a fabric commercialization agreement.

本季度，我們將繼續認真地優先考慮和控制支出，同時尋求延長現金流的方法，並繼續就布料商業化協議進行業務發展討論。

In October we received $6 million upon Pfizer's exercise of a buyout option from our 2008 license to use zinc finger modified cell lines.

10 月份，輝瑞公司行使了其 2008 年鋅指修飾細胞系許可的買斷選擇權，我們收到了 600 萬美元。

We also continue to engage in business development discussions across our Sangamo pipeline and platforms.

我們也將繼續就 Sangamo 的產品線和平台進行業務拓展的討論。

As of today, we believe our cash and cash equivalents, including the license fee received from Pfizer and proceeds from sales of common stock under our at the market offering program since September 30th, will be sufficient to fund our planned operations into the first quarter of 2026.

截至今日，我們相信，我們的現金及現金等價物，包括從輝瑞公司收到的許可費以及自 9 月 30 日以來根據我們的市場發行計劃出售普通股所得款項，將足以支持我們計劃運營至 2026 年第一季度。

Before handing back to Sandy, I'd like to emphasize that we remain resolutely focused on solving our long-term funding foundation to advance our promise it's in pipeline. In the near term, we seek to bridge to that future through a balanced approach.

在將發言權交還給桑迪之前，我想強調，我們仍然堅定地專注於解決我們的長​​期資金基礎問題，以推進我們正在籌備中的項目。短期內，我們將尋求透過平衡的方式來過渡到未來。

Actively pursuing non-diluted business development opportunities while exploring appropriate capital options.

在積極尋求不稀釋股權的業務發展機會的同時，探索合適的資本選擇。

I will now hand it back to Sandy for closing remarks, Sandy.

現在我將發言權交還給桑迪，請她作總結發言。

Thank you, Prathyusha.

謝謝你，普拉蒂尤莎。

To close, I'm pleased with the pipeline advances this quarter.

最後，我對本季管道建設的進展感到滿意。

We held a meeting with the FDA where in the meeting minutes the FDA reiterated its October 2024 guidance that we may use EGFR slope as an endpoint to support an accelerated approval pathway.

我們與 FDA 舉行了一次會議，會議記錄顯示，FDA 重申了其 2024 年 10 月的指導意見，即我們可以使用 EGFR 斜率作為終點來支持加速審批途徑。

We presented detailed data from our registrationalar study in Fabry disease, including a positive mean annualized EGFR slope at 52 weeks, and we're pleased to observe a wide range of other positive secondary endpoints.

我們展示了法布瑞氏症註冊研究的詳細數據，包括 52 週時 EGFR 年均斜率為正值，我們很高興觀察到其他一系列積極的次要終點。

Taken together, we believe in the potential for SG 920 to provide meaningful multi-organ clinical benefits above current standards of care.

綜上所述，我們相信 SG 920 有潛力提供比目前護理標準更有意義的多器官臨床益處。

The importance of this data has been, has also been recognized externally with the acceptance this week of 3 platform presentations on our Fabri disease program at the upcoming World Symposium in 2026.

這些數據的重要性也得到了外部的認可，本週，我們關於 Fabri 病計畫的 3 個平台演講被接受參加即將於 2026 年舉行的世界研討會。

This quarter we began recruiting and enrolling patients in the phase 12 stand study for chronic neuropathic pain following the activation of the first two clinical sites. We expect to dose the first patient in the coming months.

本季度，在首批兩個臨床中心啟動後，我們開始招募和登記慢性神經性疼痛 12 期臨床試驗的患者。我們預計在未來幾個月內給予第一位患者用藥。

And we continue to advance our prime program towards an anticipated CTA submission as early as mid-2026. As Prathyusha outlined, solving our long-term funding needs remains our number one priority.

我們將繼續推進我們的主要項目，預計最早將於 2026 年年中提交 CTA 申請。正如 Prathyusha 所概述的那樣，解決我們的長​​期資金需求仍然是我們的首要任務。

We continue to seek ways to raise additional capital alongside our focused effort to secure a fabric commercialization partner. Before closing for questions, I want to reflect on the recent Nobel Prize in Physiology or Medicine awarded to Mary Brumkow, Fred Ramsdale, and Simon Sakaguchi for their research on regulatory T cells, or TREGs.

我們將繼續尋求籌集更多資金的途徑，同時專注於尋找布料商業化合作夥伴。在結束提問環節之前，我想回顧一下最近授予瑪麗·布魯姆科、弗雷德·拉姆斯代爾和西蒙·坂口的諾貝爾生理學或醫學獎，以表彰他們對調節性 T 細胞（TREG）的研究。

At Sangamo we proudly advance the clinical development of T-REGs, being the first known company to dose a patient with an engineered CAR Treg.

在 Sangamo，我們很自豪地推進 T-REG 的臨床開發，是第一家已知使用工程化 CAR Treg 給患者給藥的公司。

In August of this year, we presented the clinical data from our TX 200 study in kidney transplantation at the World Transplant Congress in San Francisco, showing the clinical potential of CAR Tregs to create a toleogenic environment in the kidney alongside the ability to taper immunosuppression post TX 200 dosing.

今年 8 月，我們在舊金山舉行的世界移植大會上展示了 TX 200 在腎臟移植的臨床研究數據，顯示 CAR Tregs 具有在腎臟中創造耐受環境的臨床潛力，同時能夠在 TX 200 給藥後逐漸減少免疫抑制。

These are encouraging results that we believe demonstrate the potential for an engineered CAR T. Regs and organ transplantation. And we really want to say that we are thankful to everyone who has been involved in this first in human study.

這些結果令人鼓舞，我們相信它們證明了基因工程改造的 CAR T 調節細胞和器官移植的潛力。我們真心感謝參與這項首次人體研究的所有人。

While we're pleased with these innovative scientific advances, we remain focused on our promising neurology pipeline and look forward to sharing further novel scientific developments with you all.

雖然我們對這些創新性的科學進展感到高興，但我們仍專注於我們前景廣闊的神經學研發管線，並期待與大家分享更多新的科學進展。

Operator, please open the line for questions.

接線員，請開通提問線。

Thank you. At this time we will conduct a question-and-answer session. (Operator Instructions)

謝謝。屆時我們將進行問答環節。（操作說明）

Our next question comes from Gena Wang from Barclays. The floor is yours.

下一個問題來自巴克萊銀行的王吉娜。現在輪到你發言了。

Hi, thanks for taking my question. This is Hanghu from Barclays on behalf of Gena Wang. Just a couple of questions on, favorite disease. Could you share with us, what's the latest progress in your partnership deal negotiation and also, on the regulatory front, you shared your progress with FDA, but, in light of recent news from SABER, about the Unicure, is there any reason to your favorite disease drug program?

您好，感謝您回答我的問題。我是巴克萊銀行的杭虎，代表王佳娜。關於你最喜歡的疾病，我還有幾個問題。您能否與我們分享您在合作協議談判方面的最新進展？另外，在監管方面，您已經與 FDA 分享了您的進展，但鑑於 SABER 最近發布的關於 Unicure 的消息，您認為您最喜歡的疾病藥物計畫有什麼特別之處嗎？

So, I can't comment on the discussions Unicure had. What they're doing is important and difficult, and we simply wish them well for patients with Huntington's disease. But what I can tell you about is our interactions with the agency and the written minute meeting minutes that we have that confirm our ability to use the EGFR data for one year to get accelerated approval.

所以，我無法對Unicure內部的討論發表評論。他們所做的事情既重要又困難，我們衷心祝福他們能為亨廷頓舞蹈症患者帶來好運。但我可以告訴你們的是我們與該機構的互動以及我們擁有的書面會議記錄，這些記錄證實我們有能力使用 EGFR 數據一年以獲得加速批准。

And when we look at our data, it's the EGFR is very clear and compelling, but it's the overall body of data, the safety of the product, the ease of use the cardiac data, the SF 36, the kidney benefit, and I believe that's what the agencies see and that's why they have remain steadfast in endorsing the use of EGFR at one year to allow us accelerated approval and to file this next year.

當我們審視我們的數據時，EGFR 的結果非常清晰且令人信服，但更重要的是整體數據，包括產品的安全性、易用性、心臟數據、SF-36 評分以及腎臟獲益。我相信，這正是監管機構所看到的，也是他們堅持支持 EGFR 在一年內投入使用，以便我們能夠加速審批並在明年提交申請的原因。

We have also had other interactions with the agency on CMC and manufacturing, and I know those sometimes aren't quite as exciting as the clinical path forward. But for genomic medicines and for AV manufacturing, having the agencies's agreement on how you will manufacture it and what the packaging that is going to be required for approval is essential. And so we're very confident on that progress and to just to kind of reflect on that, now that the agency has reaffirmed our accelerated approval pathway, this can only be helpful for our business development discussions.

我們也與該機構就 CMC 和生產製造方面有過其他互動，我知道這些有時不如臨床進展那麼令人興奮。但對於基因組藥物和AV製造而言，獲得監管機構對生產方式和批准所需的包裝的同意至關重要。因此，我們對這一進展非常有信心，而且考慮到，既然該機構已經重申了我們的加速審批途徑，這對我們的業務發展討論肯定是有幫助的。

Thanks. How about the first part of this question, like could you share with us any progress and your status in partnership negotiation?

謝謝。關於這個問題的第一部分，您能否和我們分享您在合作談判方面取得的進展和現狀？

I'll just repeat what I said at the end there, that now that the FDA has reaffirmed both the CMC and the clinical pathway, it can only be helpful in those discussions.

我只想重複我剛才說的話，既然 FDA 已經重申了 CMC 和臨床路徑，這對這些討論只會有所幫助。

Thanks a lot.

多謝。

Thank you for your question.

謝謝你的提問。

Our next question Comes from a Maury Raycroft from Jefferies. The floor is yours.

我們的下一個問題來自傑富瑞集團的莫里‧雷克羅夫特。現在輪到你發言了。

Hi, good morning and thanks for taking my questions. Just kind of following up on the last questions, wondering if you're planning to have any additional meetings with FDA prior to, a pre-BLA meeting and do you need to have, do you plan on having a pre-BLA meeting and what additional clarification do you need from FDA at this point?

您好，早安，感謝您回答我的問題。我只是想跟進一下之前的問題，想知道您是否計劃在提交BLA申請之前與FDA舉行任何其他會議，例如BLA申請前會議？您是否需要舉行BLA申請前會議？您是否計劃舉行BLA申請前會議？目前您還需要FDA提供哪些澄清？

Nathalie, can you answer that?

娜塔莉，你能回答這個問題嗎？

As a result of the meeting we had with the FDA, we're really pleased with the clarity on the clinical and safety package required for the BLA submission, as Sandy mentioned, we also have clarity on the elements for the CMC strategy, and this interaction were recent, and we're exploring at this point if there are further topics that require a pre-BLA meeting.

由於我們與 FDA 進行了會面，我們對 BLA 申請所需的臨床和安全資料包的清晰度感到非常滿意。正如 Sandy 所提到的，我們也對 CMC 策略的要素有了清楚的了解。這次交流是最近進行的，目前我們正在探討是否還有其他主題需要召開 BLA 申請前會議。

Nathalie, this is a bit of a regulatory fine point. One doesn't have to have a pre-BLA meeting if you feel that all the questions have been answered.

娜塔莉，這涉及到一些監管方面的細枝末節。如果您覺得所有問題都已解答，則無需召開 BLA 預備會議。

Absolutely, and because we have AMA, we've had this year many interactions either, through meetings or correspondence where we had really good clarity on many of questions. So at this point we're really discussing internally if there is anything that remains to be discussed.

當然，而且因為我們有 AMA（Ask Me Anything，問我任何問題）環節，今年我們透過會議或信件進行了多次互動，對許多問題都有了非常清晰的了解。所以目前我們正在內部討論是否還有什麼需要討論的。

But there is the process works well, doesn't it, because it allows that ongoing interaction.

但是，這個過程之所以有效，不是嗎？因為它允許持續的互動。

Exactly, we were very pleased to hear on the responses from the FDA, and you know they were always on time and we've had no delays in our discussions.

沒錯，我們很高興聽到FDA的回复，而且你知道，他們的回覆總是很及時，我們的討論也從未出現過延誤。

Got it. That's helpful and just for the BD partners, assuming that this is kind of a three-way conversation where you're relaying the regulatory information to these BD partners, is there any additional clarity that you need on the regulatory front that the BD partners need in order to make a decision?

知道了。這很有幫助，僅針對 BD 合作夥伴而言，假設這是一個三方對話，您正在向這些 BD 合作夥伴傳達監管信息，那麼在監管方面，您是否還需要 BD 合作夥伴提供任何額外的澄清信息，以便他們做出決定？

I don't believe there are any questions left to answer. We have clarity on the primary endpoint, and the agency has seen our one-year data. So that was part of the reason we had that recent clinical meeting is because the last time we showed it to them, there was 18 patients at one year, and now there's 32 patients at 1 year and 19 patients at 2 years, and the certainty of the EGFR slope remains. We have which is something that the partners, potential partners would clearly want to know, and the CMC and manufacturing route is clear, and the manufacturing process is underway and locked and loaded.

我認為已經沒有什麼問題需要解答了。我們對主要終點有明確的認識，監管機構也已經看到了我們一年的數據。所以，我們最近召開臨床會議的部分原因是，上次我們向他們展示時，一年後有 18 名患者，而現在一年後有 32 名患者，兩年後有 19 名患者，EGFR 斜率的確定性依然存在。我們已經掌握了合作夥伴和潛在合作夥伴顯然想知道的信息，CMC 和製造路線很清晰，製造過程正在進行中，一切準備就緒。

Got it. Okay.

知道了。好的。

Thanks for taking my question.

謝謝您回答我的問題。

Thank you for your question.

謝謝你的提問。

Our next question comes from Wells Fargo. The floor is yours.

下一個問題來自富國銀行。現在輪到你發言了。

Hi, thanks for taking our question. This is Kuan Ang for yanna. So our question is also around the fabric program. Can you share with us in your, engagement with FDA has the topic of commissioner's National Priority Review voucher ever been mentioned, and do you think that could affect your BD discussions?

您好，感謝您回答我們的問題。這是 Kuan Ang 為 yanna 所做的祈禱。所以我們的問題也與布料項目有關。您能否與我們分享一下，在您與FDA的溝通中，是否曾提及專員的國家優先審評券（National Priority Review voice）這一話題？您認為這是否會影響您與FDA的BD討論？

Thank you.

謝謝。

Nathalie, can you comment on that?

娜塔莉，你能對此發表一下看法嗎？

Yes, no, we have not discussed specifically that, but we're looking into it.

是的，我們還沒有具體討論過這個問題，但我們正在調查。

Got it. Thank you. And have you shared all the details regarding the FDA meeting minutes with your potential partners and any caller, you can come on their reactions.

知道了。謝謝。你是否已將FDA會議紀錄的所有細節分享給你的潛在合作夥伴和任何來電者？你可以根據他們的反應來判斷。

So the, you can imagine when we got the minutes, we were pleased to share them with our partners, or to share the essence of them, and any partnership that moves ahead, the partners would definitely see the minutes as part of the process. So they always get to see all of the regulatory correspondence as part of any business development deal.

所以，你可以想像，當我們拿到會議紀錄時，我們很樂意與合作夥伴分享，或是分享會議紀錄的要點。任何進一步發展的合作關係，合作夥伴肯定都會將會議紀要視為流程的一部分。因此，作為任何業務拓展交易的一部分，他們總是可以看到所有監管方面的往來函件。

Got it. Thank you so much.

知道了。太感謝了。

Thank you for your question.

謝謝你的提問。

Our next question comes from Patrick Truchio from HC Wainwright. The floor is yours.

下一個問題來自 HC Wainwright 公司的 Patrick Truchio。現在輪到你發言了。

Good morning and thank you for taking our call. This is Luis Santos for Patrick. I first want to say it is, great to see the progress and the extension of your runway is commendable and reflects your, financial discipline, and we appreciate that. I want to ask a couple of questions, on 503 in neuropathic pain.

早安，謝謝您接聽我們的電話。這是路易斯·桑托斯給派崔克的信。首先我想說，很高興看到你們取得的進展，延長跑道長度的做法值得稱讚，這反映了你們的財務紀律，我們對此表示讚賞。我想問幾個關於神經性疼痛 503 的問題。

And regarding the stan study and the dosing that you said is going to start in the coming months, are there any additional challenges that we should consider recruiting this patient population and looking ahead into the readout in about a year? What would be a clear win for to move this program forward, and I have a follow-up.

關於您提到的史丹研究和即將於未來幾個月開始的給藥，在招募該患者群體以及展望大約一年後的讀數方面，我們是否應該考慮任何其他挑戰？什麼才是推動該專案向前發展的明確勝利？我還有後續問題。

Nathalie, can you comment about how we're doing with getting the study going?

娜塔莉，你能談談我們進行這項研究的進展嗎？

Sure, so we're pleased to have already two sites activated that are actually right now, screening patients and looking at the population. We've also broadened the population to SFM.

當然，我們很高興已經有兩個站點投入使用，目前正在篩選患者並觀察人群情況。我們也將目標族群擴大到了SFM。

Not just idiopathic small fighter neuropathy, which really allows a broader scope in terms of fighting patients. So we're very optimistic that we will be able to dose the first patient in the coming months. We're also very active in opening other sites. We are going to have up to 10 sites in the study.

不僅僅是特發性小戰士神經病變，這實際上為戰士患者提供了更廣泛的範圍。因此，我們非常樂觀地認為，我們將在未來幾個月內為第一位患者進行給藥。我們也積極籌建其他網站。這項研究我們將設立多達 10 個研究點。

And in terms of the success of the trial, of course this is a dose escalation to look at the safety and tolerability of the study, but we're also looking at efficacy in terms of the pain reduction from baseline using the PINRS, which is the overall Pain Intensity Numerical Rating Scale, which is broadly recognized and widely used as a preferred scale for pain measurement. And we will be monitoring this in a blinded fashion throughout the dose escalation.

至於試驗的成功與否，當然，這是一次劑量遞增試驗，旨在考察研究的安全性和耐受性，但我們也正在考察療效，即使用 PINRS（疼痛強度數字評分量表）評估疼痛較基線的減輕情況。 PINRS 被廣泛認可並廣泛用作疼痛測量的首選量表。我們將以盲法的方式在整個劑量遞增過程中監測此情況。

Nathalie, it's been interesting the number of patients that have spontaneously from other places written to us and tried to get into the trial. It's clear that there's a high unmet medical need here.

娜塔莉，很有趣的是，許多其他地方的患者自發性地給我們寫信，試圖參加試驗。很明顯，這裡存在著巨大的未滿足醫療需求。

Absolutely. People have reached out to Sagamore or have seen the site activated. In the clinicaltrial.gov page and I've been directly contacting sites to see if they could be eligible throughout the country.

絕對地。有人聯繫了薩加莫爾，或看到網站已啟用。我在 clinicaltrial.gov 頁面上進行了查詢，並且直接聯繫了全國各地的試驗點，詢問他們是否符合資格。

And Louise, to your other question about what does efficacy look like this is the first ever clinical dosing of something like this and we will understand it with every patient that we dose, it's not a medicine that is for every patient with neuropathic pain because it's a once and done treatment that will change their pain sensation forever and therefore we will understand the population better in the coming months as we dose these patients.

路易絲，關於你提出的療效問題，這是此類藥物的首次臨床給藥，我們會透過給每位患者用藥來了解其療效。這種藥物並非適用於所有神經性疼痛患者，因為它是一種一次性治療，將永遠改變他們的疼痛感覺。因此，在接下來的幾個月裡，隨著我們給這些患者用藥，我們將更了解該族群。

Sounds great. Thank you for that overview. And regarding your platform, can you discuss any ongoing partner interests in the stack DPD capsid, any additional deals that could extend your runway, and also with your min platform progressing?

聽起來不錯。謝謝你的概述。關於您的平台，您能否談談您在堆疊 DPD capsid 方面是否有任何正在進行的合作夥伴意向，是否有任何可以延長您運營週期的其他交易，以及您的最小平台的進展情況？

We're delighted with the progress of the capsid and the part, so yes, we are always talking to new people about the capsid.

我們對衣殼和該部件的進展感到非常滿意，所以是的，我們一直在與新人討論衣殼。

What's also important is the people that are working with the capsid, Genentech, Estelis, Lilly are also pleased and our relationship and discussions with them are going well. Greg, how's Mint doing?

同樣重要的是，與衣殼合作的基因泰克、Estelis、禮來等公司也對此感到滿意，我們與他們的關係和討論進展順利。格雷格，Mint最近怎麼樣？

Mint is doing well, so we continue to advance that platform in ways where we have minimal spend. So we're trying to de-risk that technology.

Mint 平台發展良好，因此我們將繼續以最少的投入推進該平台的發展。所以我們正在努力降低這項技術的風險。

From a molecular standpoint for ferment design towards genomic targets, and we're trying to use our money wisely in that to keep that moving forward, and we continue to advance business development negotiations for potential partnership and look forward to sharing more information on those when we're able to.

從分子層面來看，我們正在為基因組標靶設計發酵，並努力明智地利用資金來推動這一領域的發展。我們將繼續推動潛在的合作夥伴關係方面的業務發展談判，並期待在條件允許時分享更多相關資訊。

It's the success of the integration with the Mint platform is increasing every time. The team come and show me it, and it really is a fundamentally important piece of science that we hope to speak about more once in the future and hopefully put in the hands of others and Sangamo to make medicines from.

與 Mint 平台的整合越來越成功。團隊過來給我看了，這確實是一項非常重要的科學成果，我們希望將來能更多地談論它，並希望能夠把它交給其他人以及桑加莫公司用來製造藥物。

Great, thank you.

太好了，謝謝。

Thank you for your question.

謝謝你的提問。

This does conclude the question-and-answer session. I would now like to turn it back to Louise Wilkie for closing remarks. The floor is yours.

問答環節到此結束。現在我謹將發言權交還給路易絲·威爾基，請她作總結發言。現在輪到你發言了。

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.

再次感謝您今天收看我們的節目並提出問題。再次提醒，您可以在 Sangamo 網站的投資者關係版塊查看我們的簡報。我們期待隨時向您報告我們未來的發展進展。

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

感謝各位參加今天的會議。節目到此結束。您現在可以斷開連線了。