Sangamo Therapeutics, Inc.

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 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, Pratisha Durrababu, 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 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 and operating expense guidance, the anticipated closing of the announced underwritten offering, 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 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 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 filing for 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 10Q for the fiscal quarter ended March 31, 2025, and subsequent filings and reports that Sangam 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. 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 were 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 licensed 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 ST503, our investigational epigenetic regulator for the treatment of chronic neuropathic pain. We're preparing for a Phase 1-2 study to assess the safety, tolerability, and preliminary efficacy of a one-time dose of ST503, 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 enrollment and dosing for the ST503 study in mid 2025 and anticipate having preliminary proof of efficacy data in the fourth quarter of 2026. We also continue to advance clinical trial authorization, or CTA, enabling activities for ST506, our epigenetic regulator for the treatment of prion disease, to be delivered intravenously using our own STAT BBB. We are extremely proud to have been selected to present during the prestigious Presidential Symposium at this week's ASGCT Annual Meeting in New Orleans. We look forward to showcasing our potent combination of epigenetic regulation and capsid delivery technology in prion disease and describing the profound survival benefits we observe when administered to post symptomatic mice. We will also describe the sustained brain-wide suppression of prion protein expression in both mouse and non-human primate models, supporting its potential as a one-time therapeutic approach for prion disease. We plan to begin clinical trial enrollment and dosing for ST506 in mid-2026 and expect to have preliminary clinical data in the fourth quarter of 2026. In addition to the prion presentation, we have had eight abstracts accepted by ASGCT. I'm very proud of our scientists and look forward to showcasing the progression of our neurology pipeline, including advances in zinc finger epigenetic regulation, the latest innovations in capsid delivery engineering and developments in our modular integrase technology. Moving to our late stage Fabry programme, last week we were excited to announce a number of important de-risking milestones in the pathway to the anticipated BLA submission for ST920. All dose patients in the Phase 1-2 STAR study have now completed at least 52 weeks of follow-up, a key milestone required by the FDA for an accelerated approval regulatory pathway for ST920. Importantly, preliminary analysis of the clinical data collected as of this 52 week milestone date across all 32 dose patients indicated that the mean EGFR slope continued to remain positive. The product candidate continues to be well tolerated and a pivotal data readout is now expected by the end of this quarter. Furthermore, in April of this year, Sangamo held a productive type B meeting with the FDA, providing us with a clear chemistry, manufacturing and controls or CMC pathway to the planned BLA submission. We were encouraged by the productive nature of the discussions and engagement from those FDA representatives in attendance and are happy to have clarity on these important activities from the agency. With the newly agreed CMC pathway, we believe we have a clear line of sight to an anticipated BLE submission as early as the first quarter of 2026, which would facilitate a potential approval and commercial launch of ST920 as early as the second half of that year. As you can imagine, this clinical and regulatory progress has been well received by our potential commercial partners, and we are hopeful that these de-risking events may accelerate our ongoing negotiations. We remain committed to securing an anticipated partnership that is best suited to bring SC920 to Fabry patients upon potential approval, and that provides near-term capital for Sangamo to advance our core neurology pipeline. As I've said before, in order to execute on our plans and deliver on this promising neurology genomic medicine pipeline, Sangamo must be sufficiently capitalised. We must have the resources to fund us through proof of concept in both our chronic neuropathic pain and prion disease programmes while operating a lean, efficient and focused organisation. In support of this strategy, today we announced the pricing of an equity offering to extend our immediate cash runway. We're optimistic these funds will provide us with the bridge that we believe is necessary to secure a fabric commercialisation agreement. We believe that this modest infusion of equity capital alongside the recent positive fabric clinical and regulatory de-risking events will allow us to secure the right commercial partner for the company and for our shareholders. We also continue to engage in promising business development discussions across our technology platforms, We have opportunities for potential funding through both new and existing STAC BBB collaboration partners with our ZincFinger platform and through collaborative research agreements related to our modular integrase or MINT platform, all of which demonstrate the ongoing interest in our technology. We look forward to sharing more information when we can. With that overview of our business progress and strategy, I'd now like to hand it over to Prathusha Durai-Babu, our Chief Financial Officer, who will provide more context on long-term vision and financial strategy to support our neurology-focused mission. Prathusha.