SANGAMO THERAPEUTICS, INC Q4 FY2024 earnings call

• Sangamo's pipeline advanced with neurology therapies towards clinic, first neurology IND secured, non-clinical proof of concept in prion disease, and blue-chip pharma agreements for STAC-BBB capsid.
• Reduced non-GAAP operating expenses by nearly half year-over-year and raised over $100 million in funding in 2024.
• Clear regulatory pathway for Fabry disease with pivotal data readout expected in mid-2025; engaged in Fabry business development negotiations aiming for partnership in Q2 2025.
• Advanced contract negotiations for third STAC-BBB license agreement, hopeful of news near end of current quarter.
• ST-503 ready for clinic with plans to begin patient enrollment and dosing in mid-2025; prion disease program advancing towards clinical trial enrollment in 2026; Fabry gene therapy data showed sustained benefit in kidney function and favorable safety profile.

No detailed financial performance by product segment with absolute revenue and contribution % provided in the transcript.

• Aim to secure Fabry partnership in Q2 2025.
• Hopeful of news on third STAC-BBB license agreement near end of current quarter.
• Guided to keep operating expenses level for 2025 focused on advancing neurology pipeline (Nav1.7 and prion).

• Time-consuming process of Fabry business development negotiations due to complexity and extensive due diligence required by potential partners.
• Uncertainty around timing of finalizing Fabry partnership and data releases.
• Complexity of clinical trial and partnership discussions for neurology programs.

Q: Luis Santos asked about Fabry data, partnerships, and data later in the year and its impact on partnerships.

A: Sandy Macrae stated Fabry partnership discussions are in late phase, data from WORLDSymposium remains positive, and one year data for the last patient expected soon; hemophilia program had termination notice, but incoming interest exists.

Q: Yanan Zhu inquired about Fabry data seen by partners beyond WORLDSymposium and eGFR consistency.

A: Nathalie Dubois-Stringfellow said partners haven't seen efficacy data beyond WORLDSymposium, but have seen other data; eGFR data remains encouraging.

Q: Maury Raycroft asked about STAC-BBB deal timeline and OpEx going forward.

A: Sandy Macrae said hoped to have STAC-BBB deal in place by end of quarter, Prathyusha Duraibabu mentioned OpEx guided to same level as last year focused on neurology pipeline.

Q: Nicole Germino asked about hypotension concerns with ST-503 and patient enrollment for Nav1.7 study.

A: Nathalie Dubois-Stringfellow said no hypotension seen in animal studies, Nav1.7-hypertension relationship not well established; patient enrollment targets iSFN with specific inclusion/exclusion criteria.

Q: Gena Wang asked about Fabry deal delay reasons and ST-503 data expectations.

A: Sandy Macrae said Fabry discussions going well, energy increases as pre-BLA meeting nears; Nathalie Dubois-Stringfellow mentioned ST-503 hopes for near-term efficacy within 12 weeks, placebo effect wanes over time.