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Investor2014

07/21/24 3:11 PM

#464582 RE: Rubyred77 #464581

If provides solid support to existing claims and / or new ones, then yes chances can be updated/adjusted.

It’d be fun to see the updated view when that peer review finally does get released.


As always it will be key not to get carried away and to spent time thoroughly reviewing the data and reading the limitations, like these from the P2a peer reviewed paper:

There are some limitations in this study:
• Small sample size, with 32 patients entering the study and
21 patients having available genomic data, which limited the power
of the study.
• Lack of adjustment for multiple comparisons in the analyses of relations
between markers and therapeutic response.
• Genomic analysis results reported for only a subset panel of
243 genes; however, an analysis of 39,974 DNA variants (Table S5
in supporting information) showed SIGMAR1 p.Gln2Pro and COMT
p.Leu146fs in the top 0.2% association rules linked to outcome.
• Standard of care measure of change in MMSE and change in ADCSADL
for calculating effect size were obtained from the literature at
48, 52, and 82 weeks61-64 and extrapolated to 57 weeks, which was
the time point used in this study.
• RNA analysis was limited due to collection time (103 to 135 weeks)
and lack of baseline transcriptome data for comparison. Nonetheless,
an exploratory analysis did find that high SIGMAR1 expression
was associated with better therapeutic response (Table S6 in supporting
information).
• Newly identified patient selection biomarkers were longitudinally
confirmed for change in ADCS-ADL, but not for change in MMSE,
over 148 weeks. This result may be due to a higher variability in
MMSE scores.

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Steady_T

07/21/24 3:48 PM

#464586 RE: Rubyred77 #464581

It doesn't really matter what our opinions are on the approval possibilities. That decision is in the hands of the regulators.

It only matters to an individual investor in how they choose to position their portfolio.