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Re: RockRat post# 219178

Wednesday, 05/23/2018 1:13:00 PM

Wednesday, May 23, 2018 1:13:00 PM

Post# of 252727
myok - I am not as confident as the buyers wrt HOCM trial design. One positive is that FDA, for now, thinks 30 weeks is enough to get long term data compare to one year minimum which was more my thinking. For BB therapy, it takes a few months to figure out optimal dosing/combo and 6 months or longer to tell whether it is working or have bad side effects. There will probably be further modifications to time line depending on time of dosing changes and interaction with existing meds (those will probably get adjusted too). All that will add to the difficulty of analyzing results unless the positive (or negative) effects of 461 are big.

Enrollment may present challenges as well just due to small patient pool. Although I understand from HOCM friend that the company has been reaching out to leaders of non-profits and social media groups since last August. I don't know how much financial "aid" has been provided to publicise enrollment.
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