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Sangamo Therapeutics Announces Evidence of Clinical Benefit in Phase 1/2 STAAR Study in Fabry Disease
- Sustained, elevated expression of alpha-galactosidase A (α-Gal A) activity observed in 13 patients for over two years for the longest treated patient as of
About this update from Sangamo Therapeutics, Inc.
[{"type":"text","content":"\n- Sustained, elevated expression of alpha-galactosidase A (α-Gal A) activity observed in 13 patients for over two years for the longest treated patient as of cutoff date.\n\n- Achieved 78% globotriaosylceramide (Gb3) substrate clearance at 6-months and 77% reduction in urine podocyte loss in one of the first kidney biopsies.\n\n- All dose escalation patients had been withdrawn from enzyme replacement therapy (ERT) and remain off ERT today.\n\n- Reported a clinically meaningful and statistically significant increase in mean general health scores, as measured by the SF-36 General Health survey.\n\n- Since the cutoff date, four additional patients have been dosed in the expansion phase, and a further two patients have been withdrawn from ERT.\n\n- The Phase 1/2 STAAR study expansion phase is ongoing and preparations for a potential Phase 3 trial actively progress, with a trial start anticipated by the end of 2023.\n\n BRISBANE, Calif.--(BUSINESS WIRE)--\nSangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced updated preliminary data as of the October 20, 2022 cutoff date from the Phase 1/2 STAAR clinical study evaluating isaralgagene civaparvovec, or ST-920, a wholly owned gene therapy product candidate for the treatment of Fabry disease. These data, which present new biomarker data and results from the first kidney biopsies in this study, indicate evidence of clinical benefit for isaralgagene civaparvovec in Fabry disease.\n\nAs of the November 15, 2022 supplemental cutoff date, 13 patients across the dose escalation and expansion phases exhibited supraphysiological levels of α-Gal A activity, sustained for over two years for the patient with the longest follow-up. All five patients who began the dose escalation phase on ERT had been successfully withdrawn from ERT and continued to exhibit supraphysiological levels of α-Gal A activity following withdrawal. No patient has required the resumption of ERT treatment to date.\n\nImportantly, there is evidence of significant Gb3 substrate reduction at six months in one of the first kidney biopsies taken from this study, along with a significant reduction in urine podocyte loss. In addition, the study observed a clinically meaningful and statistically significant increase in mean general health scores, as measured by the SF-36 General Health survey.\n\nTh...