Sangamo Therapeutics, Inc.

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.

Thank you. Good morning, everyone. Thank you for joining us on the call today. On this call are several members of the Sangamo Executive Leadership Team, including Sandy McRae, Chief Executive Officer, Natalie Dubostring, Fellow Chief Development Officer, Greg Davis, Head of Research and Technology. Prathisha Durrababu, Principal Financial Officer and Principal Accounting Officer. 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 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. 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 10-K for the fiscal year ended December 31, 2024, and our quarterly report on Form 10-Q for the fiscal quarter ended September 30, 2025, and subsequent filings and reports that Sangamon makes from time to time with the SEC. 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. Now, I'll turn the call over to our CEO, Andy McRae.

Thank you, Louise, and good morning to everyone joining the call today. This quarter, we continue to advance our clinical and preclinical pipeline while managing our cash resources carefully. In September, we presented promising detailed clinical data from a registrational star study in Fabry disease, demonstrating the potential for ST920 as a one-time durable treatment of the underlying pathology of Fabry disease for all types of Fabry disease patients. 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. A particular highlight for me this quarter was attending the 15th annual Fabry Family Education Conference that brought together more than 200 Fabry patients, family members and volunteers to provide educational presentations and gather insights from patients, including those who have received SC920. 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 Fabry disease. I found it striking to hear firsthand the challenges these patients face, including their experiences with currently available treatments. You also see the hope they have for treatment breakthroughs and their strong understanding of scientific advances and their unwavering support for one another. 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. 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. I was lucky to meet with some patients who received SC920 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 SC920 has completely transformed their life. 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 1-2 STAND study in chronic neuropathic pain, our first-ever neurology clinical study, following the activation of the first two clinical sites. We also continue to advance our PRIAM program ahead of the planned CTA submission next year. I would like to now hand directly over to Nathalie Dubois-Stringfellow, our Chief Development Officer, to provide additional details on these important programmes. Prathusha Durrababu, our Principal Financial Officer, and I will then close the call by summarising the key business and financial takeaways from this quarter. Nathalie.

Thank you, Sandy. First, I am pleased to share updates from our Registrational Phase 1-2 STAR Study. evaluating Isaragal gene Sivaparvavec, or ST920, our investigational gene therapy for the treatment of adults with Fabry disease. 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 ST920, a positive mean annualized estimated glomerular filtration rate, or EGFR, slope of almost two, was observed at 52 weeks across all 32 patient dose 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. Supportive mean annualized EGFR slopes were also observed across a variety of patient subgroup, including gender, baseline ERT status, Fabry disease type, and baseline EGFR, showing consistency in effect across Fabry patients in the study. 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. These data are encouraging, particularly given that cardiac disease is a leading cause of death in Fabry disease patients. 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 four and a half 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 three patients with at least four and a half years of follow-up. We believe that these data demonstrate the potential of SC920 to provide meaningful and long-lasting clinical benefit to a wide range of Fabry disease patients, even above current standards of care. 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 provide a valuable input on the clinical data package. We continue to prepare for anticipated BLA submission under the accelerated approval pathway plan for as early as the first quarter of 2026. Next, I'd like to focus on our Priorized Neurology Pipeline. As this quarter, we commence patient enrollment and recruitment in the Phase 1-2 STANS study, evaluating ST503, 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 impacting more than 650,000 people across the U.S., Europe, and Japan. We are 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. 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 ST503 in non-human primates alongside a favorable safety profile. We believe the preclinical data for this program is compelling, and we look forward to seeing how this translates into humans. 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. 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. NAV1.7 is a well-proven target with human genetic validation, which we believe provide pain franchise potential. Finally, moving to ST506, our epigenetic regulator for the treatment of prion disease to be delivered intravenously using our neurotropic STAC-BBB capsid. 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. Following on from our productive meeting with the UK's MHRA earlier this year, where we aligned on nonclinical 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, or CMC, strategy for the anticipated CTA submission. In November, we presented updated pre-call data at the Prion 2025 conference, which demonstrated the potent 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 bred delivery and significant prion reduction in non-human primates. 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 timeframe to an expected data readout. Furthermore, this study would mark the first inhuman testing of our STAC-BBB capsid, which, if successful, could unlock a broader neurology pipeline for advancement, including with potential partners. 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. I would like now to hand over to Patricia Duraibabu, our principal financial officer, to provide a financial update. Patricia?

Thank you, Natalie. This quarter, we continue to diligently prioritize and control spend while seeking ways to extend our cash-run way as we continue business development discussions for a fabric commercialization agreement. 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. We also continue to engage in business development discussions across our Sangmo pipeline and platforms. 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 plan operations into the first quarter of 2026. 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 pipeline. In the near term, we seek to bridge to that future through a balanced approach. actively pursuing non-dilutive business development opportunities while exploring appropriate capital options. I will now hand it back to Sandy for closing remarks. Sandy?

Thank you, Pratusha. To close, I'm pleased with the pipeline advances this quarter. 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. We presented detailed data from our registrational STAR 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. Taken together, we believe in the potential for ST920 to provide meaningful multi-organ clinical benefits above current standards of care. The importance of this data has also been recognized externally. with the acceptance this week of three platform presentations on our Fabry Disease Programme at the upcoming World Symposium in 2026. This quarter, we began recruiting and enrolling patients in the Phase 1-2 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. and we continue to advance our prime program towards an anticipated CTA submission as early as mid-2026. As Prathusha outlined, solving our long-term funding needs remains our number one priority. 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 Brunkow, Fred Ramsdale and Simon Sakaguchi for their research on regulatory T-cells or Tregs. At Sangamal, we proudly advance the clinical development of Tregs, being the first known company to dose a patient with an engineered CAR Treg. In August of this year, we presented the clinical data from our TX200 study in kidney transplantation at the World Transplant Congress in San Francisco, showing the clinical potential of CAR Tregs to create a tolerogenic environment in the kidney, alongside the ability to taper immunosuppression post-TX200 dosing. These are encouraging results that we believe demonstrate the potential for an engineered CAR Tregs in organ transplantation. And we really want to say that we are thankful to everyone who has been involved in this first in human study. While we are 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. Operator, please open the line for questions.

Thank you. At this time, we will conduct a question and answer session. As a reminder, to ask a question, you will need to press star one one on your telephone and wait for your name to be announced. To withdraw your question, please press star one again.

Please stand by while we compile the Q&A roster. Our next question comes from Gina Wang from Barclays.

The floor is yours. Hi, thanks for taking my question. This is Hang Hu from Barclays on behalf of Gina Wang. Just a couple of questions on febrile 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 CBER about the unicorn, is there any race through to your febrile disease drug program?

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 meeting minutes that we have that confirm our ability to use the EGFR data for one year to get accelerated approval. When we look at our data, 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 SF36, the kidney benefit. And I believe that's what the agencies see. And that's why they have remained steadfast in endorsing the use of EGFR at one year to allow us accelerated approval and to file this next year. 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 agency's agreement on how you will manufacture it and what the package that is going to be required for approval is essential. And so we're very confident on that progress. And 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.

Yes, thanks. How about the first part of this question? Like, could you share with us any progress in your status in partnership negotiation?

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. Thanks a lot.

Thank you for your question.

Our next question comes from Murray Raycroft from Jefferies. The floor is yours.

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-BLI meeting, and do you need to have, do you plan on having a pre-BLI meeting, and what additional clarification do you need from FDA at this point?

Natalie, can you answer that?

Yeah. 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 element for the CMC strategy. And these interactions were recent, and we are exploring at this point if there are further topics that require a pre-BLA meeting.

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

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

But there is the previous... The RMAP process works well, doesn't it? Because it allows that ongoing interaction.

Exactly, exactly. Yeah, we were very pleased this year on the responses from the FDA. And, you know, they were always on time. And we've had no delays in our discussions.

Got it. That's helpful. And I guess just to, 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?

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 one year and 19 patients at two 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.

Got it. Okay. Thanks for taking my questions. Thank you for your question. Our next question comes from Wells Fargo.

The floor is yours. Hi, thanks for taking our question. This is Quan Ang for Yana. So our question is also around the fabric program. Can you share with us in your engagement with FDA, has the topic of commissioners national priority review voucher ever been mentioned? And do you think that could affect your BD discussions? Thank you.

Natalie, can you comment on that?

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

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

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.

Got it.

Thank you so much.

Thank you for your question. Our next question comes from Patrick Truccio from HC Wainwright. The floor is yours.

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 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 to move this program forward? And I have a follow-up.

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

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 SFN, not just idiopathic small-filer neuropathy, which really allows a broader scope in terms of finding 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. And in terms of the success of the trial, you know, 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 PI NRS. 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.

Natalie, 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.

Yeah, absolutely. People have reached out to Sangamo or have seen the site activated in the clinicaltrial.gov page and have been directly contacting sites to see if they could be eligible throughout the country.

And, Louise, to your other question about what does efficacy look like, this is the first ever clinical dosing of something like this, and I think 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.

That sounds great. Thank you for that overview. And regarding your platform, Can you discuss any ongoing partner interests in the StackDB capsid? Any additional deals that could extend your runway and also with your main platform progressing?

We're delighted with the progress of the capsid.

So yes, we are always talking to new people about the CAPSID. What's also important is the people that are working with the CAPSID, Genentech, Astellas, Lilly, are also pleased and our relationship and discussions with them are going well. Greg, how's Mint doing?

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 from a molecular standpoint for Mint 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 mint partnership and look forward to sharing more information on those when we're able to yeah 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 great thank you thank you for your question 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.

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 Sangama website. We look forward to keeping you updated on our future developments.

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