Sangamo targets Q1 2026 BLA submission for Fabry gene therapy as FDA affirms accelerated pathway

Earnings Call Insights: Sangamo Therapeutics (SGMO) Q3 2025

MANAGEMENT VIEW

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CEO Alexander Macrae highlighted continued advancement of the clinical and preclinical pipeline, with a focus on managing cash resources. Macrae stated, "In September, we presented promising detailed clinical data from our registrational STAAR study in Fabry disease, demonstrating the potential for ST-920 as a onetime durable treatment...for all types of Fabry disease patients." He also noted a productive FDA meeting in October that reaffirmed use of eGFR slope as an endpoint for accelerated approval.

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Macrae emphasized patient engagement, sharing his participation at the 15th Annual Fabry Family Education Conference and reinforcing the company's responsibility to "bring this medicine to the Fabry community."

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In neurology, Macrae announced the commencement of patient enrollment in the Phase I/II STAND study for chronic neuropathic pain and progress in the prion program with a planned CTA submission next year.

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Nathalie Dubois-Stringfellow, Senior VP & Chief Development Officer, reported, "a positive mean annualized estimated glomerular filtration rate or eGFR slope of almost 2 was observed at 52 weeks across all 32 patients dosed in this study." She also highlighted new cardiac data indicating stability in morphology and function, and described ongoing strong durability in the study with up to 4.5 years of follow-up.

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Interim Principal Financial Officer Prathyusha Duraibabu stated, "As of today, we believe our cash and cash equivalents, including the license fee received from Pfizer and proceeds from sales of common stock under our at-the-market offering program since September 30 will be sufficient to fund our planned operations into the first quarter of 2026."

OUTLOOK

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Management reiterated plans to submit a BLA for ST-920 under the accelerated approval pathway as early as the first quarter of 2026. Dubois-Stringfellow said, "We continue to prepare for our anticipated BLA submission...planned for as early as the first quarter of 2026."

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The company is also advancing its prion disease program toward a CTA submission as early as mid-2026 and expects to dose the first patient in the STAND neuropathic pain study in the coming months.

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No explicit changes to guidance or targets were announced compared to last quarter.

FINANCIAL RESULTS

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Duraibabu reported receiving $6 million from Pfizer's exercise of a buyout option related to a 2008 license. She reiterated that current cash and equivalents, including this license fee and proceeds from stock sales, will support operations into the first quarter of 2026.

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The company is actively seeking additional capital and business development partnerships, with a continued focus on non-dilutive funding opportunities.

Q&A

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Hang Hu, Barclays: Asked about progress in partnership negotiations for Fabry and whether recent regulatory actions affecting peer companies had any read-through for Sangamo. Macrae responded that "our interactions with the agency and the written meeting minutes...confirm our ability to use the eGFR data for 1 year to get accelerated approval." He added, "now that the FDA has reaffirmed both the CMC and the clinical pathway, it can only be helpful in those discussions."

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Maurice Raycroft, Jefferies: Inquired about the need for an additional pre-BLA meeting with FDA and regulatory clarity for business development partners. Dubois-Stringfellow replied, "We are exploring at this point if there are further topics that require a pre-BLA meeting." Macrae noted, "If you feel all the questions have been answered," a pre-BLA meeting may not be necessary.

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Kuan-Hung Lin, Wells Fargo: Asked about potential for a Priority Review Voucher and sharing FDA meeting minutes with partners. Dubois-Stringfellow said, "No, we have not discussed specifically that, but we are looking into it." Macrae confirmed that partners have access to regulatory correspondence as part of business development deals.

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Luis Santos, H.C. Wainwright: Asked about recruitment challenges for the STAND study and platform partnership opportunities. Dubois-Stringfellow stated, "We've also broadened the population to SFN...which really allows a broader scope in terms of finding patients." Macrae and Gregory Davis highlighted ongoing interest and negotiations around the MINT platform and STAC-BBB capsid.

SENTIMENT ANALYSIS

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Analysts focused on partnership progress, regulatory clarity, and clinical trial execution, with a tone that was generally neutral and sought specifics on regulatory and partnership developments.

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Management maintained a confident and proactive tone, frequently expressing optimism about regulatory progress, patient engagement, and partnership potential, supported by statements such as "we are very confident on that progress" and "we believe both of them independently offer significant potential value."

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Compared to last quarter, management's tone remains consistently confident, with a slight increase in emphasis on regulatory clarity and business development momentum. Analyst tone has shifted from operational curiosity to a more pointed focus on regulatory and commercial strategy.

QUARTER-OVER-QUARTER COMPARISON

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The current quarter included the presentation of detailed clinical data for ST-920, with new cardiac data and more follow-up on durability, compared to last quarter's focus on top-line results and study completion.

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Regulatory progress has moved from post-topline planning to explicit FDA reaffirmation of the accelerated pathway and CMC clarity.

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Cash runway outlook improved, supported by the Pfizer license fee and stock sales, extending the runway into Q1 2026 versus Q4 2025 previously.

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Business development strategy continues, but current discussions appear more advanced, with regulatory clarity cited as a catalyst.

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Management tone remains confident, but current remarks more strongly stress the importance of partnership and long-term funding.

RISKS AND CONCERNS

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The company highlighted ongoing efforts to secure additional capital as the "#1 priority," indicating continued funding risk.

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Duraibabu emphasized a "balanced approach" to bridging funding needs, including non-dilutive opportunities and capital options.

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No specific new risks or operational setbacks were disclosed; regulatory clarity and business development remain focal points for future risk mitigation.

FINAL TAKEAWAY

Sangamo Therapeutics reported substantial clinical progress in its Fabry disease and neurology pipelines, underscored by detailed data presentations and FDA affirmation of its accelerated approval strategy. With cash runway extended into Q1 2026 and ongoing partnership negotiations, management remains focused on securing long-term funding to support anticipated regulatory submissions and continued pipeline advancement.

Read the full Earnings Call Transcript [https://seekingalpha.com/symbol/sgmo/earnings/transcripts]

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