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Re: TopelRoad post# 2416

Thursday, 01/18/2018 2:09:23 AM

Thursday, January 18, 2018 2:09:23 AM

Post# of 3353
The phase II data shows clinical efficacy that lasts 4 weeks.

Your post referred to the overall data, including Fast and Slow progressors, where statistical efficacy was shown for 2 weeks.
https://prnewswire2-a.akamaihd.net/p/1893751/sp/189375100/thumbnail/entry_id/0_aey9hdpc/def_height/400/def_width/400/version/100012/type/1

But for the Phase III trial, Brainstorm stated they are only going to be recruiting Fast Progressors. These had 4 weeks statistical benefit
https://prnewswire2-a.akamaihd.net/p/1893751/sp/189375100/thumbnail/entry_id/0_r883qntw/def_height/400/def_width/400/version/100012/type/1


Brainstorm saw this and so is using only the fast progressors only for the Phase III to maximise the chances of Phase III success.

Also what you need to understand is that the '100% Improvement' in the ALSFRS-R slope effectively means no worsening of the situation or even potential improvement. So this means that in these Fast Progressors, NurOwn appears to be stopping the disease progression in it's tracks.

responders were defined using a very high threshold of 100% improvement in the slope post-treatment compared to the pre-treatment slope, meaning a subject needed to have stable disease or improve to be defined as a responder.



Also, clearly a treatment that would require a once every 2 weeks injection of new cells would not be seen as convenient or maybe even economically possible for patients. Thus also the benefit of the efficacy lasting 4 weeks. A nice once a month injection to potentially stop ALS dead in it's track in these fast progressors. That indeed will be a very fortunate treatment for these patients.

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