Sangamo Therapeutics reports positive topline results from Fabry gene therapy trial

Sangamo Therapeutics (NASDAQ:SGMO), a $108.63 million market cap biotech company trading at $0.46 per share, announced positive topline results Tuesday from its registrational Phase 1/2 STAAR study evaluating isaralgagene civaparvovec (ST-920), an investigational gene therapy for adults with Fabry disease. According to InvestingPro data, the company’s stock has seen significant pressure recently, though analysts maintain a consensus buy rating with potential upside.

According to the press release statement, a single dose of isaralgagene civaparvovec produced a mean annualized estimated glomerular filtration rate (eGFR) slope of 1.965 mL/min/1.73m² per year (95% CI:-0.153, 4.083) at 52 weeks across all 32 dosed patients. While developing these promising treatments, InvestingPro analysis shows the company generated $63.76 million in revenue over the last twelve months, though it continues to invest heavily in R&D, which is typical for clinical-stage biotech companies. For the 19 patients with 104 weeks of follow-up, the mean annualized eGFR slope was 1.747 mL/min/1.73m² per year (95% CI:-0.106, 3.601). The U.S. Food and Drug Administration (FDA) has agreed that eGFR slope will serve as an intermediate clinical endpoint under the Accelerated Approval pathway.

Sangamo stated it will compare the annualized mean eGFR slope of isaralgagene civaparvovec with approved Fabry disease treatments using a meta-analysis of published studies. Observational data for other treatments, including Replagal (agalsidase alfa), Fabrazyme (agalsidase beta), and Galafold (migalastat), show estimated mean annualized eGFR slopes ranging from -2.2 to -0.4 mL/min/1.73m² per year.

The STAAR study enrolled male and female patients who were either on enzyme replacement therapy (ERT), ERT pseudo-naïve, or ERT-naïve, with a median age of 42 and a median follow-up of 24 months. The longest treated patient has reached 4.5 years of follow-up.

Key secondary endpoints were positive, with sustained elevated alpha-galactosidase A activity for up to 4.5 years in the longest treated patient. All 18 patients who began on ERT have been withdrawn from ERT and remain off it as of Tuesday, with generally stable plasma lyso-Gb3 levels and stabilized cardiac endpoints.

Additional findings included improvements in disease severity, statistically significant gains in quality of life (SF-36) scores at week 52, and improvements in gastrointestinal symptoms. Some patients reported reduced or eliminated pain medication use and resumption of sweating.

The therapy demonstrated a favorable safety profile, with most adverse events being grade 1 or 2. The most common were fever, COVID-19, headache, and nasopharyngitis. All events resolved with clinical management, and no patients discontinued due to safety issues.

This summary is based on a statement from the company’s SEC filing. While Sangamo’s clinical results show promise, InvestingPro analysis indicates the company faces financial challenges with a WEAK overall financial health score. Investors seeking deeper insights into Sangamo’s financial position and growth prospects can access comprehensive Pro Research Reports, available exclusively to InvestingPro subscribers, covering over 1,400 US stocks with expert analysis and actionable intelligence.

In other recent news, Sangamo Therapeutics announced positive results from its Phase 1/2 STAAR study of isaralgagene civaparvovec, a gene therapy for Fabry disease. The study revealed a positive mean annualized eGFR slope, which contrasts with the declining slopes typically seen with existing treatments, and all patients have discontinued enzyme replacement therapy. The company plans to submit a Biologics License Application to the FDA in 2026. Meanwhile, H.C. Wainwright maintained a Buy rating with a $10 price target on Sangamo, expressing optimism about the company’s upcoming milestones, including a potential BLA submission in early 2026. Jefferies, however, lowered its price target to $1.50 but kept a Buy rating, citing recent financing efforts aimed at extending Sangamo’s financial runway. The company recently secured $23 million in financing and is in discussions with potential partners for its Fabry disease program. Additionally, Sangamo’s collaboration with Eli Lilly (NYSE:LLY) could result in milestone payments up to $1.4 billion, highlighting the company’s strategic partnerships in the gene therapy market.

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