Sangamo Therapeutics, Inc.

Good afternoon and welcome to Zagnimo's Therapeutics First Quarter 2026 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, Head of Investor Relations and Corporate Communications. Ma'am, 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 McRae, Chief Executive Officer, Natalie Dubrov-Stringfellow, Chief Development Officer, and Nikunj Jain, Interim Chief Financial 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 of Sangamo and its collaborators for clinical trials, regulatory submissions, and regulatory approvals, 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, 2025, and the quarterly report on Form 10-Q for the fiscal quarter ended March 31, 2026, and subsequent filings and reports that Sancoma 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 McRae.

Thank you, Louise, and good afternoon to everyone joining the call today. In the short time since our fourth quarter earnings call, we have continued to advance the rolling submission of the BLA for ST920 in Fabry disease, and made progress across our differentiated neurology pipeline. Natalie will provide a detailed update on our development pipeline shortly. This quarter, Sangamo's common stock transitioned to trading on the OTCQB Venture Market, following the receipt of a delisting determination from Nasdaq Capital Markets due to non-compliance with Nasdaq's minimum bid requirements. We intend to appeal the determination at a hearing before Nasdaq in June. Our stock began trading on the OTCQB venture market earlier this month under the same ticker symbol SGMO. While we are disappointed by the NASDAQ delisting determination, the transition to the OTCQB market will allow Sangamo to maintain access to the public markets while we seek to advance our promising development pipeline. We remain focused on pursuing a number of opportunities to raise additional capital, including evaluating all strategic options to maximize the value of Sangamo's assets. Throughout this process, we have been and continue to be supported by a global investment bank. We continue to engage in business development discussions for a potential fabric commercialization agreement, and we'll share more information as appropriate. Before I hand over to Natalie, I'd like to share an update from Boston. where I've just returned, where we had three abstracts accepted this year's American Society of Gene and Cell Therapy annual meeting. ASGCT is an important forum for showcasing innovation in gene and cell therapy, and I was proud to see our talented scientists present our most recent advances in zinc finger epigenetic regulation, specifically for NAV1.7 and prion disease, alongside a platform presentation on our modular integrase technology. We have now achieved impressive levels of targeted integration in a variety of different cell types, moving us one step closer to our aspiration of achieving large-scale genome engineering to integrate large sequences of DNA into the genome. I would like now to hand over to Natalie. Natalie.

Thank you, Sandy. Thank you, Sandy. First, I am pleased to share updates from our registrational phase one two-star study evaluating isoragal gene Sivaparvavac or ST920, our investigational gene therapy for the treatment of adults with Fabry disease. As outlined during our recent fourth quarter earnings call, the rolling submission of a BLA to the US FDA seeking approval of ST920 under an accelerated approval pathway is in progress with the first two modules submitted. The team is actively advancing the activity required for the CMC module and we anticipate completing the BLA submission as early as this summer, subject to our ability to secure adequate additional funding. As a reminder, ST920 has a clear pathway to accelerated approval agreed with the FDA using mean annualized EGFR slope at 52 weeks across all 32-dose patients in the study. The FDA also affirmed to us in a very recent interaction that an additional confirmatory study will not be required and that we may submit 104-week data across all those patients when seeking traditional approval. In February, we also presented detailed clinical data via four-platform and poster presentation at the 22nd Annual World Symposium that took place in San Diego, California. We continue to believe that the data support the potential of ST920 as a one-time, well-tolerated and durable treatment to provide meaningful multi-organ clinical benefit that could fundamentally shift the Fabry treatment paradigm. All four presentations are available on the Sangamo website. Moving to our neurology pipeline, we have six clinical sites activated for the Phase I-II STANS study, evaluating ST503, our investigational epigenetic regulator for patients with intractable pain due to small fiber neuropathy, or SFN. These sites are working to identify patients. This quarter, we were also pleased to have a manuscript published in Science Translational Medicine detailing the preclinical safety and pharmacology of ST503 in human neurons, mice, and non-human primates. Finally, moving to ST506, our epigenetic regulator for the treatment of prion disease. This quarter, we continue to advance clinical trial application, or CTA, enabling activities. The good laboratory practice, or GLP toxicology study, has been completed and analysis is ongoing. In addition, we held a further productive interaction with the UK's Medicine and Healthcare Products Regulatory Agency, or MHRA, including alignment on diagnostic testing analytical validation, and non-clinical safety matters. I would like now to hand back to Sandy for closing remarks. Sandy?

Thank you, Natalie. In closing, we've made good progress this quarter. The rolling BLA submission for SC920 remains in progress with the first two modules submitted and with real clarity on the regulatory pathway reaffirmed very recently by the FDA. Six sites have been activated in the Phase 1-2 STAND study, evaluating ST503 for patients with chronic neuropathic pain. We held a productive meeting with the MHRA, including further alignment on key matters for the PRIAM program. And we showcased continual progress across our pipeline and platforms at ASGCT, including a platform presentation showing impressive advances in our MINT integrase technology. This remains an important period for Sangamo as we continue to advance our pipeline while actively pursuing strategic opportunities to strengthen our financial position and maximize the value of our assets. We will continue to provide relevant updates when we are able to. Operator, please open the line for questions.

Thank you. To ask a question, please press star 11 on your telephone and wait for your name to be announced. To withdraw your question, please press star 11 again. One moment for our first question. Our first question comes from the line of Murray Raycroft with Jefferies. Your line is open. Please go ahead.

Hi. This is James on for Murray. Thanks for taking our question. Congrats on getting the reaffirmation from the FDA on the 104 weekend point. Given that the last patient reached the two-year time point around April, can you walk us through the gating factors for analyzing that data set and disclosing the data set? Would you wait to present the 104-week data at a medical conference such as World next year, or could it be disclosed earlier after you file the BLA?

So, as you know, we're... I'm going to say three fifths the way through the rolling submission and the clinical module with the one year data is already submitted. We have a series of cleaning processes to gather the full two year data and we'll work out once we have that when the best place to submit it. But we can reassure you, Natalie, that we're now seeing patients out to five years worth of experience and it's looking very stable.

Yes, we absolutely show durability in the benefit for the patient at five years and beyond. We have now four patients that are beyond five years and with super physiological level of alpha-gal. and safety of the products also remains remarkable.

And the 17 patients that are off ERT, some of them are over three years off ERT now?

Yes, absolutely, yeah.

Great. Thank you for taking my question. I'll hop back on the queue.

Thank you, and one moment for our next question. Our next question comes from the line of Yanan Zhu with Wells Fargo. Your line is open. Please go ahead.

Hi. Thanks for taking our question. This is Kuaneng for Yanan. So with FDA's recent affirmation that two-year EGFR data can serve as a confirmatory evidence, can you talk about how that will affect your BD discussions and any updates on the BD discussion around the February program that you can share with us? Thank you.

I hope you can understand that we can't talk about those conversations. I am certain that the people who have been looking at this asset are pleased by the recent affirmation from the agency. Most importantly is to get accelerated approval as quickly as possible, and that's with the one-year data. Equally important is the agency signaling that a second study will not be necessary because that would be expensive and delay the process. And as we now gather the two-year data, not just for the EGFR, but for all the other benefits to the patients, we will plan when we can submit it. And I think I can assure you that the data is looking good. It's showing durability. And we look forward to sharing this at the appropriate time.

That's helpful. Thank you.

Thank you. And I would now like to hand the conference back over to Louise Wilkie for closing remarks.

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

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