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RICHMOND, Calif. - Sangamo Therapeutics, Inc. (NASDAQ:SGMO), a clinical-stage biotechnology company with a market capitalization of $161 million and trading near its 52-week low of $0.41, announced detailed results from its Phase 1/2 STAAR study of isaralgagene civaparvovec (ST-920), an investigational gene therapy for adults with Fabry disease, showing positive kidney function outcomes and durable treatment effects.
The data, presented at the International Congress of Inborn Errors of Metabolism 2025 in Kyoto, demonstrated a positive mean annualized estimated glomerular filtration rate (eGFR) slope of 1.965 mL/min/1.73m²/year at 52 weeks across all 32 dosed patients. This kidney function measure remained positive at 104 weeks for the 19 patients with longer follow-up. According to InvestingPro data, while Sangamo maintains more cash than debt on its balance sheet, the company is quickly burning through its resources, with an EBITDA of -$58.27 million in the last twelve months.
The therapy also showed stable cardiac function and maintained elevated alpha-galactosidase A enzyme activity for up to 4.5 years in the longest-treated patient. All 18 patients who began the study on enzyme replacement therapy (ERT) were able to discontinue it following treatment.
"The positive mean eGFR slope at both one and two years, which compares favorably to approved Fabry treatments, alongside stable cardiac function, are exciting developments," said Dr. John Bernat, study investigator from the University of Iowa, in the company’s press release.
The gene therapy demonstrated a favorable safety profile without requiring preconditioning. Most adverse events were mild to moderate, with pyrexia, COVID-19, headache, and nasopharyngitis being the most common. No safety-related study discontinuations occurred.
Patients also reported improvements in quality of life measures and disease severity scores. The therapy has received multiple regulatory designations, including Fast Track and RMAT from the FDA.
Sangamo intends to submit a Biologics License Application to the FDA under the Accelerated Approval pathway in 2026, while continuing to engage in business development negotiations for a potential commercialization agreement. With current gross profit margins at -27.89% and analysts not anticipating profitability this year, investors seeking deeper insights can access comprehensive analysis through InvestingPro, which offers exclusive financial health scores and detailed valuation metrics among its 10+ additional ProTips for Sangamo.
Fabry disease is a rare genetic disorder where deficient alpha-galactosidase A enzyme activity leads to harmful substrate accumulation in organs throughout the body. Despite challenging market conditions, Sangamo’s revenue grew significantly to $81.71 million in the last twelve months, though InvestingPro’s Fair Value analysis suggests the stock may be currently undervalued.
In other recent news, Sangamo Therapeutics disclosed its Q2 2025 earnings, which showed a notable shortfall in both earnings per share (EPS) and revenue compared to forecasts. The company reported an EPS of -$0.08, which was below the expected -$0.02. Revenue also fell short, coming in at $18.3 million, missing the anticipated $29.9 million by 38.8%. These results highlight a significant gap between the company’s performance and analysts’ projections. The earnings miss is a critical development for investors to consider. Additionally, the company’s stock experienced a decline in premarket trading following the announcement. This earnings report reflects recent developments that investors are closely monitoring.
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