Replies to post #175424 on Biotech Values

[biomaven0]

My understanding of Hy's Law is that the concern arises with ALT plus bilirubin elevations that are not accompanied by ALP elevations.

Here is the FDA's Temple:

What about a combination of hepatocellular injury
(elevated AT) and evidence of obstruction (elevated
AP), with elevated bilirubin?
In that case we do not think you can conclude that
the elevated bilirubin has resulted from the massive
damage that identifies the drug as one with the ability
to cause death and need for transplantation.

He is not saying to give the drug a pass in these cases, just that they are not Hy's Law cases.

Peter

[researcher59]

GERN analyst downgrade per briefing.com -

MLV & Co downgrades GERN to Hold from Buy and lowers their tgt to $2 from $9. Firm is still positive on imetelstat's potential in myelofibrosis (MF) given disease-modifying effects that were seen at ASH last year, but it believes potential liver toxicity issues are always to be taken seriously. On a positive note, what has been seen thus far appears to be lower grade in severity and nature and, importantly, there have not been patient deaths due to liver toxicity. But that said, the FDA's choice to implement a full clinical hold (instead of a partial) throws into serious question how long it ultimately might take GERN to adequately resolve the issue.

[biopearl]

jq, thanks for posting slide, I note the bilirubin elevation appears to be unconjugated does this not imply a cell lysis effect rather than hepatocellular failure? Also most lft abn classified as low grade and none met Hy's law. Also a board of expert hepatologists were said to have reviewed and passed on the data in question. Low grade lft abn seen w statins which usually resolve. FDA seems pretty concerned here even w hundreds of patients who have received drug (don't hold me to that number). Where's the red flag esp since patients have cancer some side effects might be outweighed by benefit esp if PR or CR achieved. TIA for the board's thoughts. Regards bp