RICHMOND, Calif. - Sangamo Therapeutics, Inc. (NASDAQ: SGMO), a genomic medicine company with a market capitalization of $263 million, has announced promising updated results from its Phase 1/2 STAAR study of isaralgagene civaparvovec (ST-920) for treating Fabry disease. The data, presented at the 21st Annual WORLDSymposium in San Diego, indicates sustained enzyme activity and improvements in kidney function in patients. According to InvestingPro data, the company's stock has shown remarkable resilience with a 142% return over the past year, despite operating with significant cash burn rates.
The study showed that the longest treated patient maintained elevated alpha-galactosidase A activity for nearly four years. Furthermore, the 23 patients with at least one-year follow-up demonstrated a positive mean estimated glomerular filtration rate (eGFR) slope, suggesting notable kidney function improvements, a key factor in Fabry disease prognosis. While the clinical results are promising, InvestingPro analysis indicates the company faces financial challenges with negative EBITDA of $117 million in the last twelve months. Get access to 11 more exclusive InvestingPro Tips and comprehensive financial analysis through the Pro Research Report.
Significantly, all 18 patients who started the study on enzyme replacement therapy (ERT) have been withdrawn from and remain off ERT. This suggests that ST-920 could provide a durable, one-time treatment alternative to the ongoing ERT regimen currently used for managing the disease.
Sangamo anticipates that the data will support an Accelerated Approval pathway, with a potential Biologics License Application (BLA) submission to the U.S. Food and Drug Administration (FDA) expected in the latter half of 2025. The company is also engaged in discussions for a potential collaboration on ST-920. With an InvestingPro Financial Health Score rated as 'FAIR' and analyst consensus showing potential upside, investors seeking detailed analysis can access the comprehensive Pro Research Report available on InvestingPro, which covers this and 1,400+ other US stocks.
The updated data from the STAAR study did not report any adverse events leading to study discontinuation or deaths, and no liver function test elevations post-dosing requiring steroids occurred. The majority of adverse events were grade 1-2 in nature, suggesting a strong safety profile for the gene therapy.
In addition to safety and enzyme activity, the study reported improvements in patients' quality of life measures and a decrease in total antibodies or neutralizing antibodies associated with ERT in most patients following ST-920 treatment.
The Phase 1/2 STAAR study is now complete in terms of enrollment and dosing. The FDA has agreed that data from this study can serve as the primary basis for approval under the Accelerated Approval Program, using the eGFR slope at 52 weeks as an intermediate clinical endpoint.
Sangamo's announcement is based on a press release statement and is subject to the further detail provided in the company's Form 8-K filing. The company continues to advance its genomic medicine pipeline, with a focus on serious neurological diseases and other conditions with limited treatment options.
In other recent news, Sangamo BioSciences has been the focus of several analyst adjustments following Pfizer (NYSE:PFE)'s decision to terminate their collaboration on a gene therapy for hemophilia A. Truist Securities lowered its price target for Sangamo from $7 to $5, maintaining a Buy rating, while H.C. Wainwright reiterated a Buy rating and a $10 price target. Wells Fargo (NYSE:WFC), on the other hand, revised its price target to $2 from $3, maintaining an Equal Weight rating.
These adjustments were influenced by Pfizer's decision to not seek regulatory submissions or pursue commercialization of the gene therapy, giroctocogene fitelparvovec, despite the therapy meeting its primary and key secondary endpoints in the Phase 3 AFFINE clinical trial. This termination will take effect on April 21, 2025, and it has led Sangamo to explore other partnership opportunities.
Sangamo's financial situation has been highlighted, with a cash and cash equivalents balance of approximately $39 million as of the third quarter of 2024. The company also received a $20 million upfront payment from a recent collaboration with Astellas Pharma. However, the future of Sangamo's cash reserves largely depends on securing a partnership for its Fabry disease treatment.
In addition to the Fabry disease program, Sangamo's diverse pipeline includes isaralgagene civaparvovec (ST-920 or isa-vec), a gene therapy program, and STAC-BBB, an innovative approach targeting the central nervous system. These recent developments could have a significant impact on Sangamo's financial prospects and share value.
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