Sep. 29 at 7:02 PM
Leerink reit'd
$LRMR Outperform/
$25
$BIIB $LXEO $PTCT
Leerink said:
Bottom Line: The stock is under pressure on the 7 anaphylaxis events that occurred in the open label study. However, we believe that the efficacy and clinical data are quite compelling, especially given Friedreich's ataxia (FA) is a devastating disorder, making the risk/reward profile favorable.
This morning, LRMR provided an update on their Phase 2 open label (OL) trial.
As anticipated in our preview, key focus areas included safety, frataxin levels, and enrollment progress to assess whether the company remains on track to file their BLA in 2Q26 (here).
On skin frataxin, LRMR reported impressive results. 100% (10 out of 10) of patients achieved levels >50% of the median frataxin concentration found in healthy volunteers by day 180 which we believe is striking. For context, heterozygous patients who exhibit no signs of disease typically have frataxin levels at ~50% of those in unaffected healthy individuals.
Leerink added:
