Sangamo Therapeutics (SGMO -1%) is down again, albeit modestly on average volume. Shares have sold off over 30% since touching $12.33 on Monday.
Investors appear to be reacting to preliminary results from a Phase 1/2 clinical trial, THALES, evaluating cell therapy ST-400 in beta thalassemia patients. The data were presented at ASH in Orlando on that day.
Two patients had been infused. One experienced a serious adverse event, hypersensitivity, deemed related to ST-400’s cryoprotectant DMSO that resolved by the end of infusion. On the efficacy front, the patient was transfusion-free for only six weeks. More time will be needed to assess the treatment effect on the second patient.
For comparison purposes, results from a Phase 1/2 study evaluating bluebird bio’s (BLUE -1.7%) LentiGlobin gene therapy in transfusion-dependent beta-thalassemia patients showed a median transfusion-free period of 33 months in eight patients. Preliminary data on seven subjects in a Phase 3 trial showed transfusion-free periods of 4.7 – 15.1 months.
#ASH19
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