Sangamo Therapeutics Q1 2026 Earnings Call - FDA Clears Path for Accelerated Fabry Approval Without Confirmatory Trial

Conference Operator: Good afternoon, and welcome to Sangamo 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.

Louise Wilkie, Head of Investor Relations and Corporate Communications, Sangamo Therapeutics: 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 Nikunj Jain, Interim Chief Financial Officer. Slides from our corporate presentation can be found on our website, sangamo.com, 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 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, 2025, and the quarterly report on Form 10-Q for the fiscal quarter ended March 31, 2026, 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.

Sandy Macrae, Chief Executive Officer, Sangamo Therapeutics: Thank you, Louise, 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 ST-920 in Fabry disease and made progress across our differentiated neurology pipeline. Nathalie 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 Market 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 Fabry commercialization agreement and will share more information as appropriate. Before I hand over to Nathalie, I’d like to share an update from Boston, where I’ve just returned, where we had three abstracts accepted to this year’s American Society of Gene & 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’d like now to hand over to Nathalie. Nathalie.

Nathalie Dubois-Stringfellow, Chief Development Officer, Sangamo Therapeutics: Thank you, Sandy. Thank you, Sandy. First, I am pleased to share updates from our registrational phase I/II STAAR study evaluating isaralgagene civaparvovec or ST-920, 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 U.S. FDA seeking approval of ST-920 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, we anticipate completing the BLA submission as early as this summer, subject to our ability to secure adequate additional funding. As a reminder, ST-920 has a clear pathway to accelerated approval agreed with the FDA using mean annualized eGFR slope at 52 weeks across all 32 dosed 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 WORLDSymposium that took place in San Diego, California. We continue to believe that the data support the potential of ST-920 as a one-time, well-tolerated, and durable treatment to provide meaningful multi-organ clinical benefit that could fundamentally shift the Fabry disease treatment paradigm. All four presentation are available on the Sangamo website. Moving to our neurology pipeline, we have six clinical site activated for the phase I/II STAND study evaluating ST-503, our investigational epigenetic regulator for patient 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 ST-503 in human neurons, mice, and non-human primates. Moving to ST-506, 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. We held a further productive interaction with the U.K.’s Medicines 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?

Sandy Macrae, Chief Executive Officer, Sangamo Therapeutics: Thank you, Nathalie. In closing, we’ve made good progress this quarter. The rolling BLA submission for ST-920 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 I/II STAND study evaluating ST-503 for patients with chronic neuropathic pain. We held a productive meeting with the MHRA, including further alignment on key matters for the Prion program. We showcase 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.

Conference Operator: Thank you. To ask a question, please press star 1 1 on your telephone and wait for your name to be announced. To withdraw your question, please press star 1 1 again. One moment for our first question. Our first question comes from the line of Maury Raycroft with Jefferies. Your line is open. Please go ahead.

James, Analyst, Jefferies: Hi. This is James on for Murray. Thanks for taking our question. Congrats on getting the reaffirmation from the FDA on the 104-week endpoint. Given that the last patient reached the 2-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 WORLDSymposium next year, or could it be disclosed earlier after you file the BLA?

Sandy Macrae, Chief Executive Officer, Sangamo Therapeutics: As you know, we’re I’m gonna say three-fifths the way through the rolling submission and the clinical module with the 1-year data is already submitted. We have a series of cleaning processes to gather the full 2-year data, and we’ll work out once we have that when the best place to submit it. We can reassure you, Nathalie, that we’re now seeing patients out to 5 years worth of experience and it’s looking very stable.

Nathalie Dubois-Stringfellow, Chief Development Officer, Sangamo Therapeutics: Yes, we absolutely show durability in the benefit for the patient, at 5 years and beyond. We have now 4 patients that are beyond 5 years and, with supraphysiological level of alpha-Gal. You know, safety of the products also remains remarkable.

Sandy Macrae, Chief Executive Officer, Sangamo Therapeutics: The 17 patients that are off ERT, some of them are over 3 years off ERT now?

Nathalie Dubois-Stringfellow, Chief Development Officer, Sangamo Therapeutics: Yes, absolutely. Yeah.

James, Analyst, Jefferies: Great. Thank you for taking my question. I’ll hop back in the queue.

Conference Operator: Thank you. 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.

Kwan, Analyst, Wells Fargo: Hi. Thanks for taking our question. This is Kwan for Yana. With FDA’s recent affirmation that 2-year eGFR data can serve as a confirmatory evidence, can you talk about how that’ll affect your BD discussions and any updates on the BD discussion around the Fabry program that you can share with us? Thank you.

Sandy Macrae, Chief Executive Officer, Sangamo Therapeutics: 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, that’s with the 1-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. As we now gather the 2-year data, not just for the EGFR, but for all the other benefits to the patients, we will plan when we can submit it. I can assure you that the data is looking good. It’s showing durability, we look forward to sharing this at the appropriate time.

Kwan, Analyst, Wells Fargo: All right. That’s helpful. Thank you.

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

Louise Wilkie, Head of Investor Relations and Corporate Communications, Sangamo Therapeutics: 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.

Conference Operator: This concludes today’s conference call. Thank you for participating, and you may now disconnect. Everyone, have a great day.