Sangamo Touts Additional Positive Data For Fabry Gene Therapy Candidate

Sangamo Therapeutics Inc SGMO has announced updated preliminary results from the Phase 1/2 STAAR study evaluating isaralgagene civaparvovec (ST-920), a gene therapy candidate for Fabry disease.
Recently, Sangamo and Pfizer Inc PFE reopened enrollment in phase 3 AFFINE study hemophilia A gene therapy trial after pausing it due to some patients experiencing blood clotting protein factor VIII activity greater than 150%.
These latest data show that all nine patients treated in the dose escalation phase across the four dose cohorts sustained elevated α-Gal A activity ranging from nearly 2-fold to 30-fold of mean normal at the last measurement date.
The gene therapy was generally well tolerated. All treatment-related adverse events were mild except for one instance of moderate pyrexia. No treatment-related serious adverse events were reported.
Related: Sangamo's Fabry Disease Gene Therapy Continues To Show Encouraging Safety Profile.
Four patients were withdrawn from enzyme replacement therapy (ERT) and demonstrated significantly elevated levels of α-Gal A activity up to 28 weeks post-withdrawal.
Since the cutoff date, one additional patient was withdrawn from ERT.
The Phase 1/2 STAAR study has progressed into the expansion phase, with four patients dosed. Sangamo is currently planning for a potential Phase 3 clinical trial.
Price Action: SGMO shares closed at $4.75 on Tuesday.