May. 15 at 7:42 PM
$LRMR setup: key catalyst this quarter.
Downside ~
$2/share in a renewed safety-risk scenario. Upside driven by strong mechanism (directly addressing frataxin deficiency) with prior efficacy signals; if approved, could become standard of care for this rare disease, with analyst targets mostly
$20+.
Valuation is mainly held back by safety concerns (7/39 anaphylaxis cases). Company added test-dose and antihistamine premedication, with FDA alignment.
FDA granted Breakthrough Therapy Designation and remains aligned on BLA pathway, including FXN as a potential surrogate endpoint and exposure-response analysis; safety dataset will be reviewed at filing.
If upcoming data shows improved safety, accelerated approval path becomes more likely, with near-term share potential into the teens.
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