Sangamo presents detailed data from STAAR study

Sangamo (SGMO) Therapeutics announced detailed data from the registrational Phase 1/2 STAAR study evaluating isaralgagene civaparvovec, or ST-920. Efficacy: A positive mean annualized eGFR slope of 1.965 mL/min/1.73m2/year at 52-weeks was observed across all 32 dosed patients. Furthermore, a mean annualized eGFR slope at Week 104 of 1.747 mL/min/1.73m2/year was observed for the 19 patients who have achieved 104-weeks of follow-up. Stable cardiac function was observed, including left ventricular mass, left ventricular mass index, left ventricular myocardial global longitudinal strain, T1 and T2 mapping, end-diastolic and end-systolic volumes that remained stable over at least one year. Durability of effect was demonstrated with elevated expression of alpha-galactosidase A activity maintained for up to 4.5 years for the longest treated patient. All 18 patients who began the study on Enzyme Replacement Therapy had been withdrawn from ERT and remained off ERT as of the data cutoff date. Of the 10 patients who had measurable titers of total antibodies or neutralizing antibodies against alpha-Gal A associated with ERT at baseline, TAb or NAb titers decreased markedly in nine patients and became undetectable in eight following treatment. Improvements in disease severity were reported in the Fabry Outcome Survey adaptation of the Mainz Severity Score Index age-adjusted score. Statistically and clinically significant improvements in the short form-36 quality of life scores were observed. Statistically significant improvements were seen in the gastrointestinal symptom rating scale compared to baseline. Safety: The majority of adverse events were grade 1-2 in nature. The most common treatment-emergent adverse events were pyrexia, COVID-19, headache, and nasopharyngitis. All TEAEs resolved in response to clinical management and there were no safety-related study discontinuations and no deaths.