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Post# of 252311
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Monday, 06/11/2018 4:18:04 PM

Monday, June 11, 2018 4:18:04 PM

Post# of 252311
ONCE Hemophilia A GT Data:

Spark's upcoming Hemophilia A data was brought to my attention and I got to thinking can anything good come of this? From the prior data cut
https://twitter.com/MauriceOnTW/status/1006259636876521472
there were a lot of questions/concerns. At the time I actually thought there is a good chance the program gets terminated (at some future date not based on just the 4 patients)
1-The expression levels have wide variations at different time points for the individuals and amongst the 4 patients presented.
2-No sign of dose-response at all!
3-Steriods were needed (not to control ALT elevations) but to stop loss of expression!
4-The competition (BMRN) has people concerned because mean/median expression level is 50% after 2-3 years yet Spark is nowhere near that in the presented data.

Curious what others think. I actually don't dislike the company and cheap enough (much much lower price) would consider a position.

Also, what would be the best way to play this if I wanted to limit risk and yet gain more potential exposure? I thought to buy puts (but they are a bit pricey) so I'd need to speculate on how low it could go 40's (if they terminate the program or becomes more apparent that will happen) or perhaps somewhere in the 50's/60's is more realistic. Maybe someone more experienced in options knows a better way and can comment? I'd appreciate any ideas!

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