Sangamo Therapeutics, Inc.

Good afternoon, and welcome to the Sangamo Therapeutics Third Quarter 2024 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 of 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 McRae, Chief Executive Officer, Nathalie Dubois-Stringfellow, Chief Development Officer, and Prathisha Durrababu, 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 related to Sangamo's cash runway, plans to obtain additional capital, and ability to continue operating as a going concern, the therapeutic and commercial potential of Sangamo's product candidates and technologies, Sangamo's ability to earn and receive payments from its collaboration and license agreements, including the Genentech and Pfizer agreements, Sangamo's expectations regarding new collaborations and license agreements, 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 of the SEC, specifically in our annual report on Form 10-K for the fiscal year ended December 31, 2023, as supplemented by Sangra's quarterly reports on Form 10-Q for the quarter ending September 30, 2024, and subsequent filings and reports that Sangra 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. As we near the end of 2024, I'm extremely proud of the progress we are making this year. We are committed to translating groundbreaking science into medicines that transform the lives of patients and families afflicted with serious neurological diseases. The updates we will share today demonstrate several meaningful advances towards that goal and show how we believe Sangamo is well positioned for continued progress. First, Sangamo has transformed from a Phase 1-2 company to a pre-BLA company as a result of significant regulatory developments in our Fabry Disease Program. Second, Pfizer continues to engage in discussions with regulatory authorities concerning our Haemophilia A Program, both of which have the potential to provide a long-term financial foundation for our core neurology pipeline. Third, We signed a neurology epigenetic regulation and capsular delivery license agreement with Genentech and received $50 million in upfront license fees and milestone payments. And finally, we submitted our first ever IND application for a neurology indication. These developments demonstrate that Sangamo is steadily executing upon our strategy, is driving potential medicines towards patients in need, and is continuing to advance plans to put the company on a more stable financial footing. Natalie will share more context in a moment, but I want to start out by highlighting the significant clinical and regulatory progress made this quarter in our Fabry disease program, following alignment with the FDA on a clear regulatory pathway to accelerated approval. This pathway reduces the time to potential approval by three years and avoids the requirement for an additional lengthy and costly registrational study, which is incredibly impactful given the unmet medical need for patients with Fabry disease. We are delighted to have such a clear regulatory pathway that could bring this treatment to patients significantly sooner than originally anticipated and have begun to execute BLA readiness activities ahead of a submission anticipated in the second half of 2025. As you can imagine, this announcement has generated a lot of interest from external stakeholders, including patients who tell us about the remarkable change our treatment has brought to their lives, investors, and potential strategic partners. We continue to engage in ongoing business development discussions as we seek to get this treatment to patients as quickly as possible. Following the recent top line readout from Pfizer for the Phase 3 Affine trial in haemophilia A, We also move closer to potential regulatory submissions for this program, which has the potential to unlock up to $220 million in regulatory and commercial milestones for Sangamo over the next two years. Pfizer will present a detailed phase three data update at the upcoming American Society for Hematology, or ASH, annual meeting in December. And Pfizer have advised us that they are discussing this data with regulatory authorities. which is very encouraging. Taken together, the Fabry and Haemophilia A programs could provide Sangamo with a solid and long-term financial foundation for our core neurology pipeline. We're thrilled to be in the enviable position of seeing up to two potential BLE submissions in 2025 for product candidates developed by Sangamo. each of which leveraged the best of our science and capabilities and addressed significant unmet patient needs and commercial opportunities. This exciting momentum propels our neurology pipeline forward as we work to advance our science and technology for neurological indications. This quarter, we signed our first neurology epigenetic regulation and capsid delivery license agreement. We have granted Genentech an exclusive license to our highly potent zinc finger repressors that are directed to tau, a critical gene involved in Alzheimer's disease and other tauopathies, as well as an additional undisclosed second neurology target. For these same targets, we also granted Genentech an exclusive license to our industry-leading neurotropic delivery capsid, STAC-BBB, which has demonstrated potent blood-brain barrier penetration and brain transduction in non-human primates. We have received $50 million in upfront license fees and milestone payments from Genentech, which extended our cash runway to allow us to continue advancing other ongoing business development activities. This agreement has further drawn interest in our science and enhanced our ability to attract new, potentially valuable partnerships. We are currently advancing business development discussions with additional potential collaborators seeking to license our novel intravenous capsid, STAC-BBB, and we believe this capsid is a potentially valuable source of additional non-dilutive funding. This quarter, we also submitted our first ever IND application for a neurology indication. We expect our lead program, ST503 for intractable pain, to advance into the clinic in the middle of 2025, assuming clearance of the IND with the FDA, with our expected prior clinical trial authorization submission followed close behind by the end of 2025. Our cash runway remains unchanged and is sufficient to fund our planned operations into the first quarter of 2025, absent any potential funding from a Fabry partnership, haemophilia A milestone payments from Pfizer, or additional stacked BBB collaboration agreements. Alongside the two anticipated BLE submissions next year, we see a viable path to financial stability. I would now like to hand it over to Nathalie, our Head of Development, who will share additional context and also take us through other pipeline updates. Nathalie.