Nextblockbuster wrote: "Could the existing BETONMACE trial results be that compelling to the FDA at this point that maybe the FDA will allow the existing data to file an NDA in the US?  my rose coloured glasses are getting fogged up........"

I personally do not like this added ambiguity concerning USA patient involvement in BETonMACE. In my opinion, this casts much uncertainty as to how US patient enrollment may affect the ability to file an NDA with the FDA, the timing of the NDA application to the FDA and the general pathway to FDA approval. Of course, if the BETonMACE trial results hit a home run, then likely none of this really matters and the share price will see an enormous boost regardless of FDA/NDA timelines.

How do we interpret the January 11, 2018 news release in light of today's news? To many of us, this January 2018 news seemed like too little (only a 100 or so US patients out of 2400) and too late (US patients would be treated for less than a year) according to original BETonMACE trial design. From an objective point of view, what value would 100 or so USA patients treated for well under the median treatment duration add to the credibility of BETonMACE and the statistical strength of the data? Not much in my opinion. 

My hunch is that the sample size re-estimation analysis (SSRA) is key. If the DSMB/CSC recommends increasing the sample size, then this will be the green light for inclusion of USA patients. However, if the DSMB/CSC do not recommend increasing the sample size, and there is no halt for futility, then it becomes a voluntary choice of not including any USA patients, or increasing the sample size and including USA patients. The results of this SSRA may not only represent a critical decision point in patient number and USA patient involvement, but may also be a key determinant in any financing arrangements/deals/letters of intent/etc, which are in turn key to providing funds for other planned trials (i.e. renal trial, Fabrys trial). Kind of like an SSRA domino effect. Just my two cents.

BearDownAZ