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Re: biomaven0 post# 216765

Tuesday, 01/23/2018 5:22:59 PM

Tuesday, January 23, 2018 5:22:59 PM

Post# of 252302
FGEN DMD Video's

For people interested the clip is from a PPMD webcast with Elias Kouchakji VP Clinical Development and Drug Safety at Fibrogen. The whole video link is here:
(From October 2017)

Early PPMD FGEN Video's are here:
(May 2016)
(February 2017)
(July 2017)

I took a less optimistic view (from the video) in that the treatment effect wasn't obvious at 1 year and that is why they extended it to two years. Overall I am (hopeful) it will show a beneficial effect in various combinations like Peter said. Its noteworthy that Jerry Mendel has pointed out GT can't penetrate muscle where there is a lot of fibrosis. My criticism is of the company enrolling this trial and Elias seems to have done a terrible job (in one video he states about wanting to give up after a year of not enrolling ANY patients and Neff talked him into pushing on!). This is one of the very few trials for NON-ambulatory given the nature of the disease (and comments from some parents on the webcasts) you'd think they should have enrolled very rapidly!

I imagine a company strong in the space (i.e. SRPT) could add a lot of value such as doing a (much) better job enrolling in the DMD community. But for that to happen I would think FGEN would have to commit to not partnering FG-3019 (as a package) to a big Pharma/Bio.

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