Good afternoon and welcome to the Sangamo Therapeutics First Quarter 2025 Teleconference 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, Vice President and Investor Relations and Corporate Communications. Please go ahead.

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; Prathyusha Duraibabu, Chief Financial Officer; and Nathalie Dubois-Stringfellow, Chief Development Officer. Slides from our corporate presentation can be found on our website, sangamo.com, and under the Presentations page of the Investors & Media section. This call includes forward-looking statements regarding Sangamo's current expectations. These statements include, but are not limited to, statements related to Sangamo's cash runway and operating expense guidance, the anticipated closing of the announced underwritten offering, Sangamo's plans to obtain additional capital and its ability to continue operate as a growing concern, the therapeutic and commercial potential and value of Sangamo's product candidates and technologies, Sangamo's ability to earn and receive payments from its collaboration and license agreements, 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 facts. 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 fiscal year ended December 31, 2024, and our quarterly report on Form 10-Q for the fiscal quarter ended March 31, 2025 and subsequent filings and reports that Sangamo 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, Sandy Macrae.

Thank you, Louise, and good afternoon to everyone joining the call. It's only been a couple of months since our fourth quarter call, but I'm pleased to share with you some company progress across a variety of areas including our capsid engineering platform, our neurology pipeline, our Fabry program and our finances. Beginning with our Capsid engineering platform, in April we announced our third capsid license agreement since we shared the discovery of our industry leading neurotropic delivery capsid STAC-BBB. We are pleased to sign an agreement with Eli Lilly and Company granting Lilly a worldwide exclusive license to STAC-BBB for up to five potential disease targets of the central nervous system. We have received the $18 million upfront license fee for the first target and are eligible to earn up to $1.4 billion in additional license target fees and milestone payments across all five potential disease targets as well as tiered royalties on potential net sales. We are thrilled to have signed this third important agreement, further demonstrating that we are a collaborator of choice for neurotropic capsids. With Genentech, Astellas and now Lilly, we have great partners in neuroscience for our technology and we continue to engage in discussions with new potential collaborators for STAC-BBB. Turning to our neurology pipeline programs, this quarter we continue to advance clinical study preparations for ST-503, our investigational epigenetic regulator for the treatment of chronic neuropathic pain. We are preparing for a Phase 1/2 study to assess the safety, tolerability and preliminary efficacy of a one-time dose of ST-503, our investigational epigenetic regulator that will be administered intrathecally to patients with intractable pain due to idiopathic small fiber neuropathy or ISFN. We plan to begin patient enrolment and dosing for the ST-503 study in mid-2025 and anticipate having preliminary proof of efficacy…

Thank you, Sandy. I'd like to begin by emphasizing our long-term vision for Sangamo as a neurology focused genomic medicine company. We strongly believe in the potential of our neurology pipeline to deliver transformational therapies for patients as well as significant value for our shareholders. Our goal is to appropriately fund the company to achieve clinical proof of concept data across both our chronic neuropathic pain and prion disease programs. These milestones represent critical potential value inflection points that could significantly change the trajectory of our company. To achieve these important milestones, we have a financial strategy with both short and long-term components. In the near-term, the equity financing we announced today is necessary to solidify Sangamo’s immediate financing needs. We believe the offering proceeds will extend our runway to late in the third quarter of this year. More importantly, this bridge financing provides us the time we believe that we need to secure the right Fabry commercial partnership, one that would fund our neurology mission in the near-term and create value for shareholders over the longer-term. We've already made significant progress in transforming our financial profile. In 2024 we reduced our non-GAAP operating expenses by 50% year-on-year by carefully focusing the organization on our most important priorities. We are leaving no stone unturned in seeking additional cost savings and are looking at ways to further reduce operating expenses to maximize the efficiency of the go forward neurology company. We are committed to operating as leanly as possible while continuing to advance our two neurology programs at a steady pace. We believe this disciplined approach to capital allocation will ensure that we have the specific talent and capabilities to execute on our neurology mission while extending our runway through our key value creating milestones. I'll now hand it back to Sandy for closing remarks.

Thank you, Prathyusha. I'm pleased with the progress we have made this year so far. We signed our third STAC-BBB License agreement, this time with Lilly reinforcing that Sangamo is a collaborator of choice for neurotropic capsids. We continue to advance our ST-503 program for the treatment of intractal pain due to ISFN ahead of the start of a planned patient enrolment and dosing in mid-2025. We achieved significant clinical and CMC derisking milestones in our pathway to anticipated BLA submission for our Fabry disease program and we have advanced multiple potential business development discussions across a range of Sangamo technologies including STAC-BBB, our Zinc Finger platform and our MINT platform, while securing additional capital to support our near-term efforts. We remain resolutely focused on solving our long-term funding needs and partnering our Fabry disease program in an effort to provide sustainable and long-term funding for our promising neurology genomic medicine pipeline. In closing, I would encourage you to engage with the data we'll be presenting at this week's ASGCT Annual Meeting which will be made available on the Sangamo website once data embargoes have lifted. These presentations will further demonstrate the scientific foundation of our pipeline and the potential of our therapeutic approach to address significant unmet medical needs. As always, we thank you for your continued support of Sangamo. Operator, please open the line for questions.

Thank you. [Operator Instructions] One moment while we compile our Q&A roster. Our first question is going to come from the line of Maury Raycroft with Jefferies. Your line is open. Please go ahead.

Hi, this is James on for Maury. Congrats on the progress and thanks for taking our questions. Just to start off, could you provide more color on what exactly you plan to show in the top line eGFR data? Do you plan to show just the eGFR slope or there be other quality of life endpoints? Also, do you plan to show an NHS analysis to contextualize the improvements you're seeing on eGFR and if so, can you provide some color on the statistical plan and P value threshold?

Thank you for your questions. Nathalie, can you talk to these?

Yes, yes. So we're really happy to have dosed all the patients that now have passed the one year amount required by the FDA for an accelerated approval regulatory pathway. We will of course share in the top line data the updated mean eGFR slope and we will comment on additional information at a later date.

It's fair to say, Nathalie, that at that time we'll also have 19 patients who will have achieved two years data.

Absolutely.

So it really is a robust data set. We understand the excitement people have to see this data in full and we will show as much as possible as we can of the top line data as we prepare it for the BLA submission.

Yes. And as to the statistical analysis, we are not commenting at this time, but we have agreement with the FDA on our Type B meeting with them. So, you know, we feel like we, it’s a standard way to doing it and I've agreed with the FDA on the path forward.

Got it, thanks. And just one really quick follow-up. Now that you're sort of in the final stages of securing a potential Fabry partnership, how many partners are you potential partners are you currently in conversations and what can you really tell us about those ongoing discussions at this point?

You can imagine that that's not something that we're able to comment, but it's fair to say there are multiple potential partners that we're talking to. And I think for all of them, the Type B meeting was very helpful because it gave a clear path for the CMC, which is something that is very important for any gene therapy BLA and approval. So we were very pleased with that result. We were very pleased with how straightforward the interaction with the agency was and how helpful they were because I know for people like you, predictability of the process and the approval process is very important.

Got it. Thank you so much for taking our questions. I'll hop back in the queue.

Thank you. One moment for our next question. Our next question is going to come from the line of Nicole Germino with Truist. Your line is open. Please go ahead.

Hi, good afternoon. Thanks for taking my question. So you’ve previously mentioned potential interesting partners for Fabry. Has the pace of those conversations gone -- have they changed in any way given the macro landscape with changes at FDA? And do you see any added pressure around cell and gene therapies in particular?

So, Nicole, sorry, your line was quite hard to hear here. I think I heard you ask us to comment on hemophilia A and the agency's attitude to gene therapy. Is that fair?

Yes and Fabry disease.

So, as I kind of alluded to in the last answer, the remarkable thing about the Agency interaction we just went through was how unremarkable it was and how helpful the Agency was. They send you answers to your written questions before the meeting. They came in on time and they were very detailed. They had all the right people at the most senior level in the meeting. And we got the answers, the formal minutes of the meeting within a week of the meeting, where they could take up to 30 days. And so in every form of interaction we've had, we have found them unchanged in how they are looking at our Fabry disease program. And when we look at the interactions that Pfizer had across the hume [ph], they also seemed to have been very positive about that. So we are not seeing any change. I'm sure, like us, you are very aware of the announcements and the concern about funding and about resourcing at the Agency, but we've not seen any evidence of that and it has not impacted anything to do with our program.

Okay, and then one quick question and follow-up. So given the current administration's concern around drug pricing, how do you think that will impact gene therapy uptake and pricing in U.S. and ex-U.S.?

I think there's a long way to go in those discussions around drug pricing. I think that the pharmaceutical industry is a very important industry in America. And I'm sure as someone who sits on the Board BIO, that BIO will be having very detailed and hopefully productive conversations with the administration to find a good way through this.

Great. Thank you so much, Sandy.

Thank you. And one moment for our next question. Our next question is going to come from the line of Yanan Zhu with Wells Fargo. Your line is open. Please go ahead.

Hi. Thanks for taking our question. This is [indiscernible]. Just to clarify on the prior questions on the regular path for Fabry, do you still plan to file based on 52-week eGFR data and is static versus baseline required for eGFR? Thank you.

Yes, absolutely. We are absolutely pursuing the agreement we had with the FDA to use 52 weeks eGFR for the entirety of the patient population in the Phase 1/2 study. We have collected all the data from the 32 patients and we are QC-ing it. So we expect to share the top line data at the end of the quarter and then this will be the basis for the data in our BLA.

And it's very important that we emphasize how much supportive data there is beyond the eGFR. We have got agreement with the Agency for submission based on that as the primary endpoint. But I think the thing that convinces the Agency and has made this such a compelling medicine for us is that every other indication index of success in the trial is going in the same direction. So CANE [ph] scores, Fabry scores SF-36.

Yes and the general safety is very good, very well tolerated. The patient in general are feeling better and our alpha-gal level remain high in all patients.

And some of these patients are now out at four plus years?

Four plus years, yes.

Sorry, maybe you already comment on it and is static versus baseline required for eGFR? Thank you.

We'll be looking at a variety of statistical methods to describe the difference in eGFR. But we said last week or the week before in our announcement that the eGFR remains positive. So the slope of the eGFR remains positive.

Got it. Thank you so much.

Thank you. One moment for our next question. Our next question comes from the line of Luis Santos with H.C. Wainwright. Your line is open. Please go ahead.

Hello everyone. Thank you for taking our questions and congratulations on the selection of your talk for the Presidential Symposium and your impressive presence at ASGCT. Regarding Fabry, I don't know if I missed this, but are the patients who were on ERT still all off ERT or were there any relapse?

Yes, all patient, the 18 patients that started on ERT out of our 32 patient are of still off ERT.

Very well. And for the -- I know that you mentioned you're not commenting so much on the statistical analysis plan, but the eGFR slope, you said it remains positive, but is there any threshold that we should be looking at as a minimum or is there any actual statistical significance? I think prior questions were asking about this already, but I'm not sure if I was clear on that.

We're not commenting on this right now. But what I can tell you is the mean eGFR slope remains positive.

And you will -- I know you're very aware that in normal people, sorry, people without Fabry disease, there's a gradual decline over time in their eGFR. In Fabry patients, there is an exaggerated decline in eGFR that is not fully addressed by ERT and it remains negative in patients that are on ERT. And so as part of our analysis, we'll look at the change from baseline and we'll be looking at compared to the data for other forms of treatment for Fabry disease. We would not have put out the press release that we did about the eGFR remains positive if we weren't confident that the results were what the Agency were looking for.

And that's helpful. Just wrap up that and clarify my question as well for the statistical analysis plan, even if you're not giving us more detail, you are submitting all the data available from all patients for the BLA submission. But which ones will be used for the which ones will be used for the statistical plan? Is it all of them or is it just a portion of them?

It's all of them.

So at the submission, for the BLA submission, it will be 32 patients at one year and 19 patients at two years.

That's helpful. Thanks so much.

Our pleasure.

Thank you. One moment for our next question. Our next question is going to come from the line of Luca Issi with RBC Capital Markets. Your line is open. Please go ahead.

Hi. Thanks so much for taking our question. This is Cassian [ph] for Luca and this will be a question on STAC-BBB and we recall that homology medicines had one of the first capsids delivered systematically which could cross the blood-brain barrier and demonstrated in a mouse model a dose dependent reduction in biomarker both systematically and in the CNS. So would you expect to see a similar dual impact with your STAC-BBB capsid and also do you expect similar preconditioning to your other capsid approaches, i.e. no prophylactic steroids, any color there would be very helpful? Thank you so much.

So we can really only comment on our own capsid and its effectiveness. We have not made the decision whether we will use steroid treatment in the clinical trial yet. We're very pleased with the effectiveness of STAC-BBB. We're very pleased to have seen the result in monkeys across with a variety of cargoes. And I would really commend you all to look at the presentation that Bryan will be giving at ASGCT where it really showcases both the effectiveness of STAC-BBB and the importance of the right cargo in it. And to be honest, that's why it's so pleasing that Lilly, Astellas and Genentech, who are all big names in the neuroscience field, have chosen to license our capsid directly.

Yes, and what we will present really is profound overall benefit of the treatment and disease mouse model and sustained brain wide suppression of prion protein expression in both mouse and nonhuman primate model, supporting its potential as a one-time therapeutic approach for prion disease. So it's very important that we have tested it in NHP and it's extremely well tolerated.

And you know, the Presidential Symposium, I think there's two presentations being given out of something like 4,000 abstracts that were presented. And it's a great credit to the work of Bryan Zeitler and David Ojala and Victoria and Bryan’s team that have all done so much work to drive something that hopefully next year will be a potential curative, sorry a potential treatment for patients with this awful, awful disease.

We'll be looking forward to the presentation at ASGCT. Thanks again and congrats on the presentation and abstracts.

Thank you. [Operator Instructions] Our next question comes from the line of Gena Wang with Barclays. Your line is open. Please go ahead. Ms. Wang, your line might be on mute. [Operator Instructions] I am showing no further questions at this time and I would like to hand the conference back over to Louise Wilkie for closing remarks.

Thank you once again for joining us and for your questions today. 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. Thank you.

This concludes today's conference call. Thank you for participating. You may now disconnect. Everyone, have a great day.