Wednesday, Sangamo BioSciences (NASDAQ:SGMO) received a reaffirmed Buy rating from TD Cowen, following the company's announcement of FDA alignment on an accelerated Biologics License Application (BLA) filing for its Fabry disease gene therapy candidate, '920. The FDA's agreement is based on the ongoing Phase 1/2 Open Label STAAR study of estimated Glomerular Filtration Rate (eGFR) data.
SGMO has outlined plans to compile full one-year data from 32 participants, with two-year follow-up data from 19 participants, by the second quarter of 2025. This comprehensive data set is expected to support a BLA submission in the second half of 2025. Interim analysis of the study has indicated positive results in eGFR preservation and slopes, which are considered key indicators of kidney health and function.
The analyst from TD Cowen expressed optimism about the development, noting the news as encouraging and anticipating a straightforward FDA review process for '920. This sentiment is based on the interim analysis suggesting favorable outcomes in preserving kidney function among patients with Fabry disease, a genetic condition that can lead to significant organ damage.
SGMO's commitment to a 2H25 BLA submission marks a significant milestone for the company and for patients affected by Fabry disease. The therapy, if approved, could offer a new treatment option for managing the disease and its complications.
The FDA's alignment with Sangamo BioSciences on an expedited pathway for '920 underscores the potential of the therapy and the importance of bringing new treatments to market for rare genetic disorders.
In other recent news, Sangamo Therapeutics has made significant strides in its Fabry disease treatment program. The U.S. Food and Drug Administration (FDA) has outlined an Accelerated Approval pathway for Sangamo's gene therapy product candidate, isaralgagene civaparvovec, which could potentially expedite the therapy's availability by about three years. The therapy has shown promising safety and efficacy data, including evidence of improved kidney function in patients with Fabry disease.
In legal developments, Sangamo has filed an application with the Delaware Court of Chancery to validate a recent amendment to its certificate of incorporation, which increased its authorized common stock. This move was made to address a class action complaint challenging the validity of the amendment.
In financial updates, Sangamo has reported a significant licensing agreement with Genentech, potentially worth up to $1.9 billion in milestone payments. This deal, along with near-term payments, extends Sangamo's financial stability into the first quarter of 2025. The company has also reported positive results from their Phase 3 AFFINE trial for hemophilia A.
Continuing on a positive trajectory, Sangamo is actively pursuing additional funding opportunities and partnerships to further their neurology and gene therapy programs. The company is also seeking partners for their STAC BBB capsid technology, which has shown promise in neuron transduction and liver de-targeting.
InvestingPro Insights
Sangamo BioSciences' recent FDA alignment for its Fabry disease gene therapy candidate has sparked investor interest, reflected in the company's recent stock performance. According to InvestingPro data, SGMO has seen a remarkable 201.52% price return over the last three months, indicating strong market optimism surrounding the company's developments.
However, investors should approach with caution. An InvestingPro Tip highlights that SGMO is quickly burning through cash, which could be a concern as the company progresses towards its BLA submission in 2025. Additionally, with a market cap of $251.95 million and a revenue of just $12.28 million in the last twelve months, SGMO is trading at a high revenue valuation multiple.
Despite these challenges, another InvestingPro Tip notes that SGMO operates with a moderate level of debt, which may provide some financial flexibility as it advances its pipeline. For investors seeking a comprehensive analysis, InvestingPro offers 11 additional tips for SGMO, providing a deeper understanding of the company's financial health and market position.
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