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The call highlights meaningful clinical and regulatory progress for ST-920 (rolling BLA, FDA affirmation of 2-year confirmatory endpoint, durable long-term patient data) and continued advancement across neurology programs and platform technologies. However, material near-term risks center on capital availability and a NASDAQ delisting determination that forced a transition to the OTCQB market. Progress on regulatory and scientific fronts is balanced by significant financing and market-listing challenges that could impact execution and timing.
Sangamo said the rolling BLA for ST‑920 is in progress with the first two modules submitted, the clinical module (1‑year data) already filed, management characterizing the filing as roughly 3/5 complete and anticipating BLA completion “as early as this summer” subject to securing additional funding; the FDA agreed accelerated approval can be based on mean annualized eGFR slope at 52 weeks across all 32 dosed patients, affirmed no additional confirmatory trial is required, and indicated 104‑week (2‑year) data across those 32 patients may be used for full approval (the last patient reached the 2‑year time point around April). Management also highlighted durability and safety — four patients beyond five years with supraphysiological alpha‑Gal and 17 patients off ERT (some >3 years) — and operational progress in neurology with six sites activated for the Phase I/II STAND (ST‑503), GLP toxicology for ST‑506 completed with analysis ongoing, and MHRA alignment on key matters.
First 2 modules of the rolling BLA for ST-920 submitted; company states the rolling submission is ~3/5 complete and anticipates completing the BLA submission as early as this summer, subject to adequate funding. FDA affirmed accelerated approval pathway using mean annualized eGFR slope at 52 weeks across all 32 dosed patients.
FDA recently affirmed that 104-week (2-year) eGFR data can serve as confirmatory evidence and indicated an additional confirmatory study will not be required, potentially accelerating path to traditional approval if 104-week data are submitted.
Company reports durability of benefit: 4 patients beyond 5 years with supraphysiological alpha‑Gal levels and 17 patients off enzyme replacement therapy (ERT), some over 3 years off ERT; team reports safety profile remains remarkable.
Six clinical sites activated for Phase I/II STAND study (ST-503 for small fiber neuropathy); GLP toxicology study for ST-506 (prion disease) completed and analysis ongoing; ongoing CTA-enabling activities and productive MHRA interactions.
3 abstracts accepted at ASGCT and 4 presentations at the WORLD Symposium; showcased advances in zinc finger epigenetic regulation (Nav1.7, prion disease) and Modular Integrase (MINT) technology with reported impressive targeted integration levels across cell types.
1-year clinical module already submitted; company notes processes underway to clean and analyze full 2-year data and mentions seeing patients with up to 5 years’ follow-up, supporting long-term durability claims.
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.
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...