SANGAMO THERAPEUTICS, INC Q3 FY2025 earnings call

Sandy Macrae noted the quarter saw advancing the clinical and preclinical pipeline while managing cash carefully. In September, promising clinical data from the STAAR study in Fabry disease was presented. In October, a meeting with the FDA reaffirmed use of eGFR slope as an endpoint for an accelerated approval pathway. Attended the Fabry Family Education Conference. Nathalie Dubois-Stringfellow provided updates on the STAAR study for ST-920 in Fabry disease, including positive eGFR slopes and cardiac data. Commenced patient enrollment in the Phase I/II STAND study for chronic neuropathic pain. Advanced the prion program with ST-506, including CTA-enabling activities and preclinical data presentations. Prathyusha Duraibabu mentioned $6 million received from Pfizer, cash runway sufficient to fund operations into Q1 2026, and focus on long-term funding. Sandy Macrae also noted the Nobel Prize related to Tregs and CAR-Tregs in kidney transplantation.

Cash and cash equivalents, including the Pfizer license fee and proceeds from at-the-market offering, are sufficient to fund planned operations into Q1 2026. Anticipated BLA submission for ST-920 in Fabry disease in Q1 2026. Anticipated CTA submission for ST-506 in prion disease as early as mid-2026.

Forward-looking statements subject to risks and uncertainties discussed in SEC filings. All forward-looking statements about future plans and expectations are subject to ability to secure adequate additional funding.

Q: Just a couple of questions on Fabry disease. What's the latest progress in partnership deal negotiation and any read-through to Fabry disease drug program from uniQure news?

A: Can't comment on uniQure's discussions, but FDA interactions reaffirmed use of eGFR data for accelerated approval, and overall data, safety, etc., are compelling.

Q: Wondering if you're planning to have any additional meetings with FDA prior to a pre-BLA meeting and what additional clarification is needed?

A: Pleased with clarity on clinical/safety package and CMC strategy for BLA submission; exploring if further topics require a pre-BLA meeting.

Q: Has the topic of Commissioner's National Priority Review Voucher been mentioned in engagement with FDA and could it affect BD discussions?

A: Not specifically discussed, but regulatory correspondence will be shared with partners in BD deals.

Q: Any additional challenges in recruiting for the STAND study and what would be a clear win for moving the program forward?

A: Sites activated, population broadened to SFN; efficacy monitored via PI-NRS in blinded fashion during dose escalation.

Q: Any ongoing partner interest in the STAC-BBB capsid and MINT platform progress?

A: Always talking to new people about the capsid, MINT platform is doing well with minimal spend and ongoing BD negotiations