SANGAMO THERAPEUTICS, INC Q2 FY2025 earnings call

• Positive top line results from the registrational STAAR study in Fabry disease, with a positive mean annualized eGFR slope at 52 weeks across all dose patients, and FDA agreement to use mean eGFR slope as primary approval basis under accelerated pathway. Key secondary endpoints positive, including stabilization in cardiac endpoints and clinical benefit in quality of life scores. • Initiation of the first clinical site in the Phase 1/2 STAND study in chronic neuropathic pain, expecting to dose first patient in fall 2025 and have preliminary efficacy data by Q4 2026. • Productive meeting with MHRA for prion disease program, on track for CTA submission as early as mid-2026. • Completed equity offering to bridge to Fabry commercialization, cash runway expected to fund operations into Q4 2025, focused on securing Fabry commercialization partner and advancing business development across pipeline.

No detailed breakdown of product segment financial performance in terms of absolute revenue and contribution % provided in the transcript.

• Cash runway expected to fund operations into Q4 2025. • Highly focused on securing a Fabry commercialization partner in the near term and advancing business development negotiations for that potential agreement. • Engaging in broader business development discussions across the pipeline and platforms, including the MINT platform.

• Forward-looking statements are subject to risks and uncertainties discussed in SEC filings, including those related to securing adequate additional funding, clinical trial outcomes, regulatory approvals, and commercialization challenges.

Q: Has the team held a pre-BLA meeting with the FDA regarding the path to approval using 1-year eGFR data as a predictor for 2-year benefit?

A: No pre-BLA meeting held yet; FDA agreed on accelerated approval with 1-year data and may receive yearly updates as patients progress.

Q: What additional insights to anticipate from SSIEM presentation?

A: Will present top line data with additional details compared to press release, including more on endpoints.

Q: How does ST-503's efficacy in Nav1.7 compare to Nav1.8 inhibitors?

A: Sangamo remains convinced Nav1.7 is the right target as seen in mouse studies and human mutation evidence.

Q: Have surveys been done on potential adoption rate of Fabry treatment?

A: Patient advocacy groups and physicians are enthusiastic about the treatment, with patients reporting life-changing benefits.

Q: How will STAND trial cash burn be weighed?

A: Intention is to continue Nav1.7 program, with focus on Fabry commercialization partner to solve funding needs.

Q: What data to expect from STAND trial by end of 2026?

A: Safety data from patients and early efficacy data for dose escalation trial.