Sangamo Therapeutics (SGMO) recently reported that the FDA has granted Fast Track Designation (FTD) to isaralgagene civaparvovec, or ST-920, a wholly owned gene therapy product candidate for the treatment of Fabry disease. The company is poised to deliver shareholder value in 2023, maintains John McCamant, editor of The Medical Technology Stock Letter.
The goal of FTD is to facilitate development and expedite the review of new therapeutics that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs. FTD provides for more frequent interactions with the FDA and also may become eligible to apply for Accelerated Approval and Priority Review if relevant criteria are met.
The FDA has previously granted ST-920 Orphan Drug Designation. In our view, the Fabry data in Feb. was proof-of-concept and the program has since been significantly de-risked.
ST-920 is currently being evaluated in the Phase 1/2 STAAR study, with a total of 20 patients dosed to date. In February 2023, the company announced promising results from the STAAR study at the 19th Annual WORLDSymposium, showing sustained, elevated expression of alpha-galactosidase A (α-Gal A) activity in the 13 dosed patients as of the data cutoff, 78% globotriaosylceramide (Gb3) substrate clearance at 6-months and 77% reduction in urine podocyte loss in one of the first kidney biopsies, and a clinically meaningful and statistically significant increase in mean general health scores, as measured by the SF-36 General Health survey.
SGMO is currently preparing for a potential Phase III trial and plans to meet with the FDA on the proposed trial design in the summer, with a trial start anticipated by the end of 2023, depending on FDA feedback.
The positive POC Fabry data also provided POC/de-risking for SGMO’s early-stage pipeline. Wall Street is barely assigning any value to the Fabry program despite the progress, Phase III by year end, and zero value for the pipeline.
Recommended Action: Buy SGMO under $7.
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