SANGAMO THERAPEUTICS, INC Q3 FY2024 earnings call

Clinical and Regulatory Progress

• Fabry disease program: Aligned with FDA on accelerated approval pathway for ST-920, avoiding additional costly registrational study, with BLA submission expected in second half of 2025. Long-term treated patients show positive eGFR slope and sustained alpha-Gal production.
• Hemophilia A: Pfizer to present Phase III AFFINE trial data at ASH, with potential $220M milestones from regulatory and commercial achievements, and ongoing discussions with regulators.
• Neurology: Signed neurology epigenetic regulation and capsid delivery license with Genentech, receiving $50M upfront; submitted first IND for neurology indication (ST-503 for intractable pain), with Phase I/II study expected in mid-2025; prion program CTA submission expected in fourth quarter of 2025.

Partnerships

• Secured $50M upfront from Genentech for neurology license, enhancing cash runway and attracting potential collaborations for STAC-BBB capsid.

No specific product segment financial performance with revenue contribution % detailed. Key programs highlighted include Fabry disease, hemophilia A, neurology epigenetic regulation with Genentech, and neurology IND submission.

Forward-Looking Statements

• Anticipate BLA submissions for up to 2 gene therapy programs in 2025.
• Plan to dose patients in first neurology epigenetic regulation study and submit IND for STAC-BBB neurotropic capsid in 2025.
• Cash runway sufficient to fund planned operations into first quarter of 2025, pending potential funding from Fabry partnerships, hemophilia A milestones, or STAC-BBB collaborations.

Risks

• Uncertainties in regulatory approvals for Fabry and hemophilia programs.
• Dependence on securing additional funding for continued operations.
• Risks related to clinical trial outcomes and successful commercialization of products.

Q: Regarding FDA comments on eGFR slope at 52 weeks and 104 weeks for Fabry program, and strategy for the program.

A: Sandy Macrae passes to Nathalie Dubois-Stringfellow who explains FDA's acceptance of eGFR slope at 52 weeks for accelerated approval and plans to share 2-year data in second half of 2025. On strategy, ongoing partnership discussions are underway with focus on getting the treatment to patients quickly.

Q: Next data update for the Fabry program.

A: Nathalie states data from the Phase I/II study will be available in second quarter of 2025, with top line data shared then.

Q: FDA endpoint requirements for Fabry program and confidence in Pfizer's support for hemophilia A.

A: Sandy Macrae discusses FDA's evolving approach to rare diseases and Pfizer's engagement with regulators regarding the hemophilia A program, noting Pfizer's ongoing discussions with regulatory authorities.

Q: FDA's requirements for Fabry program regarding female patients or cardiac variant, and commercial tool for cardiac involvement.

A: Sandy Macrae and Nathalie Dubois-Stringfellow explain FDA's focus on eGFR as the primary endpoint but mention other data in the submission will cover various patient aspects, and the importance of addressing patient needs including cardiac involvement in future commercial efforts