Wells Fargo analyst Yanan Zhu maintained a Hold rating on Sangamo Biosciences (SGMO – Research Report) yesterday and set a price target of $2.00.
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Yanan Zhu’s rating is based on a series of promising yet uncertain developments in Sangamo Biosciences’ Fabry disease program. The updated data from the STAAR study, presented at the WORLDSymposium conference, indicated a positive eGFR slope for the first 23 patients after one year. This is a promising sign, as the FDA has agreed that this could serve as an intermediate endpoint for potential accelerated approval. However, full data for all 33 patients, which is critical for a potential Biologics License Application (BLA) filing, will not be available until the first half of 2025.
Despite these positive indicators, the absence of individual patient data leaves questions about the underlying drivers of the improvement in the eGFR slope. Factors such as the increased number of patients in the study, the inclusion of higher dose cohorts, and other patient characteristics could have influenced the results. Additionally, while all patients who were on enzyme replacement therapy (ERT) at the baseline have successfully withdrawn and maintained stable biomarker levels, the sustainability of these outcomes remains to be verified. Consequently, while the developments are encouraging, the Hold rating reflects the need for further clarity and data confirmation before making a more definitive investment recommendation.