SANGAMO THERAPEUTICS, INC Q1 FY2025 earnings call

• Capsid engineering platform: Announced third capsid license agreement with Lilly for STAC-BBB, $18M upfront, potential for $1.4B+ in fees and royalties. Engaging with new collaborators for STAC-BBB. • Neurology pipeline: Advancing ST-503 clinical study prep for chronic neuropathic pain, plan to start enrolment mid-2025, preliminary efficacy data Q4 2026. Advancing ST-506 CTA enabling activities for prion disease, selected to present at ASGCT Presidential Symposium. • Fabry program: All dosed patients in Phase 1/2 STAAR study completed 52 weeks follow-up, mean eGFR slope positive, pivotal data readout end of quarter. Held Type B meeting with FDA for CMC pathway to BLA submission, anticipates BLA as early as Q1 2026. • Finances: Announced equity offering to extend cash runway, aim to secure Fabry commercialization agreement for funding neurology pipeline. Engaging in business development discussions across technology platforms.

No distinct product segments with revenue contribution percentages provided. Key financial related to capsid engineering: Received $18M upfront license fee from Lilly for STAC-BBB with potential to earn up to $1.4B in additional fees and milestone payments across targets and tiered royalties on net sales. Equity offering announced to extend cash runway.

• Announced equity offering to extend immediate cash runway, expecting funds to bridge and secure Fabry commercialization agreement. • Anticipate BLA submission for ST-920 as early as Q1 2026, potentially leading to approval and commercial launch in H2 2026. • Plan to continue advancing neurology pipeline programs with secured capital.

• Dependence on securing adequate additional funding to execute plans. • Regulatory uncertainties related to clinical trials, submissions, and approvals. • Uncertainty in securing the right commercial partner for Fabry program, which could impact neurology pipeline funding.

Q: Could you provide more color on eGFR data, quality of life endpoints, and statistical plan for Fabry?

A: Nathalie mentioned they'll share top line eGFR slope data, 19 patients have two years data, and they have agreement with FDA on path forward but not commenting on statistical analysis yet.

Q: How many potential partners for Fabry are you in conversations with?

A: Sandy said there are multiple potential partners, Type B meeting was helpful for CMC path.

Q: Regarding Fabry, are patients on ERT still off ERT and details on eGFR slope for BLA submission?

A: 18 patients on ERT are still off ERT, mean eGFR slope remains positive, all 32 patients' data will be used for BLA submission with 32 at one year and 19 at two years data.