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Whalatane

12/15/17 12:06 PM

#216065 RE: mcbio #216048

MDGL. ...mcbio ...At end of Q3 they had $62 m in cash , marketable securities etc and had operating expenses of only $8.6m in the last Qt. They are running 105 and 125 patient trials that are fully enrolled and will read out late Jan 2018 and late April 2018 .

So they can easily wait until the results from the HeFH trial results ..only 4-6 wks away .

The results of the He FH trial are important , for many reasons that many here may not recognize .
HeFH patients ( of which I am one ) will often have LDL cholesterol levels over 300. ( my highest was 312 ) .
Max dose of the strongest Statin ( Crestor ) ..may cut that LDL cholesterol level in half ...to around 150 and adding Zetia ( Etizimbe ) may get you down closer to 100 . ( mine has been below 100 only once )

This is a high risk heart disease population . Life expectancy typically falls off a cliff once past 60 unless you have very aggressive LDL lowering , no smoking , med diet , lots of exercise etc etc.
So we ( the he FH population ) are targeted to LDL 70 ...which probably few of us achieve ...UNLESS we get a PCSK9......HOWEVER these ( expensive ) drugs are so far reserved only for the very high risk with usually prior intervention ( ie already had a non fatal MI ...heart attack )

So if MGL-3196 can lower our LDL cholesterol another 28% ( P1 trial ...p<0.001 ) at 80 mg strength ...this becomes an alternative to the PCSK9's ( as well as the drug ESPR is developing ) ...for the he FH patients denied access to the PCSK9's.
MGL-3196 also dramatically lower Tg's ( triglycerides )...very important to those with the lipid profile of low HDL with high TG as well as diabetics at risk of amputations.

The he Fh ( hetero familia hypercholestremia ) patient population is about 1 in 400 in the US and as low as 1 in 100 in some areas like Quebec . Its inherited on the male side of families.
JMO
Kiwi