Fabry disease partnership and data presentation, Fabry disease partnership and collaboration timeline, Fabry partnering status, and eGFR data and statistical analysis are the key contradictions discussed in
Therapeutics' latest 2025Q1 earnings call.
Fabry Disease Program Progress:
-
announced that all dosed patients in the Phase 1/2 STAAR study have completed at least 52 weeks of follow-up, meeting a key milestone for an accelerated approval regulatory pathway for ST-920.
- The product candidate continues to be well tolerated, and a pivotal data readout is expected by the end of this quarter, driven by positive eGFR slope and support from the FDA Type B meeting.
Capsid Engineering Platform Success:
- Sangamo signed its third capsid license agreement, this time with
, granting Lilly a worldwide exclusive license to STAC-BBB for up to five potential disease targets of the central nervous system.
- The success of these agreements demonstrates Sangamo's status as a collaborator of choice for neurotropic capsids, showcasing the value and effectiveness of their capsid engineering platform.
Funding and Financial Strategy:
- Sangamo priced an equity offering to extend its immediate cash runway, with proceeds expected to provide financial stability until late in the third quarter of this year.
- The funding strategy is aimed at securing a Fabry commercialization agreement and supporting the neurology-focused mission, with a focus on achieving clinical proof of concept across chronic neuropathic pain and prion disease programs.
Neurology Pipeline Advancements:
- Sangamo continues preparations for a Phase 1/2 study of ST-503 for the treatment of chronic neuropathic pain, with patient enrolment and dosing expected to begin in mid-2025.
- The company has also advanced clinical trial authorization enabling activities for ST-506, with plans to begin clinical trial enrollment and dosing in mid-2026. These advancements are driven by the potential of epigenetic regulation and capsid delivery technology in these fields.
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