SANGAMO THERAPEUTICS, INC Q4 FY2025 earnings call

Sangam will continue to make significant pipeline progress in 2025 and into the first quarter of 26. In June, positive top-line results from the Registrational STAR study in Fabry disease were announced. Rolling submission of a BLA to the FDA for ST920 is in progress. Transitioned to become a clinical stage neurology company with six clinical sites activated in the Phase 1-2 STAMP study in chronic neuropathic pain. Announced a third neurology capsid license agreement with Eli Lilly. Raised over $130 million in funding since the start of 2025. Natalie provided updates on the Registrational Phase 1-2 STAR study for ST920, including rolling submission of BLA, submission of companion diagnostic, completion of CMC activities. Also updated on neurology pipeline, including fast track designation for SC503, activation of additional clinical sites for SC503 study, publication of manuscript on SC503. Advancing clinical trial application-enabling activities for ST506.

Not applicable as no specific financial performance per product segment details provided

Need to solve long-term cash runway. Securing a commercial partner for Fabry remains number one focus, continue to engage in Fabry business development discussions with multiple potential partners. Seek ways to raise additional capital, including assessment of all strategic options for each of our assets, and are in discussions with multiple potential partners, including alongside focused efforts to secure a fabric commercialization partner.

All forward-looking statements about future plans and expectations are subject to ability to secure adequate additional funding. Regulatory and market uncertainty. Discussions for partnerships are complex and take time as potential partners assess regulatory environment for gene therapies.

Q: Provide more color on revised timing for the FABRI BLA submission, PPQ and other CMC-related activities, and status of FABRI partnership discussions.

A: CMC has been on critical path, had helpful interactions with FDA CMC group, timeline for CMC longer due to careful cash management. Been talking to people for 18 months, speaking to multiple partners now.

Q: Any additional interactions with FDA recently relating to acceptance of EGFR slope as primary endpoint?

A: Spoke to agency last October, feel they reiterated EGFR at one year could be used for accelerated approval, not planning to go back to agency now but would address if changes occur.

Q: On prion disease, timeline for first-in-human trial for STAC-BBB capsid and aspects of approach for different indications?

A: STAC BBB program, eminent neurological companies choosing it, AV9 used for neuropathic pain as program started before STAC-BVB capsid known, AV9 effective intrathecally at dosing dorsal root ganglion