Sangamo Therapeutics(SGMO) - 2025 Q3 - Earnings Call Presentation

Neurology Pipeline and Programs - Sangamo is focused on developing genomic medicines for debilitating neurological diseases, leveraging its zinc finger epigenetic regulation technology and AAV capsid discovery platform[3, 4] - ST-503 for small fiber neuropathy (SFN) is in Phase 1/2 clinical trials, with preliminary efficacy data expected in Q1 2027, targeting a market impacting 650,000 people across the US, Europe and Japan[12, 18, 19, 64, 65] - ST-506 for prion disease is advancing towards a CTA submission expected as early as mid-2026, with preliminary efficacy data anticipated from mid-2027, addressing a condition resulting in 1,500 deaths per year across the US, Europe and Japan[12, 21, 22, 33, 87] - The STAC-BBB capsid has demonstrated industry-leading CNS tropism in NHPs, with 700-fold higher transgene expression than benchmark capsid AAV9, and is the subject of license agreements with Genentech, Astellas and Lilly[25, 27, 92] Financial Highlights and Partnerships - Sangamo has received $88 million in cash from STAC-BBB partners to date, with up to $46 billion in potential future milestones and exercise fees assuming exercise of all options and targets, plus additional potential product royalties[26] - As of September 30, 2025, Sangamo had approximately $296 million in cash and cash equivalents, which is expected to fund planned operations into Q1 2026[34, 36] - Sangamo anticipates non-GAAP operating expenses for 2025 to be in the range of $125 million to $145 million[36] - Pfizer exercised a buyout option for a license to use certain zinc finger modified cell lines, resulting in $6 million payment to Sangamo[34] Fabry Disease Program - Positive clinical data from the registrational STAAR study in Fabry disease showed a positive mean annualized eGFR slope of 1965 mL/min/173m2/year (95% CI: -0153, 4083) at 52-weeks across all 32 dosed patients[33] - Sangamo is preparing for an anticipated BLA submission for Isaralgagene civaparvovec as early as the first quarter of 2026, while continuing business development discussions for a Fabry commercialization agreement[28, 33, 124]