The speculation is on what the data will demonstrate after being analyzed by Chiltren International. The data hinges on two possibly important findings remembering that the rest period was decreased from 56 days to 28 days, which was different than the Phase IIb trial:
1. That the primary endpoint of FEV1 is statistically significantly maintained or improved. This is associated with the liposome formulation which mimics the natural lubricant of the respiratory conducting airways; and
2. Whether the secondary endpoint of two log reduction or eradication of Pa infection is achieved within the 28 day treatment period. Either of these outcome would be signinficant, particularly in light of the probability that most (if not all) of the patients in this trial would have had previous treatments with TOBI.
Then it comes down to time to exacerbation of any Pa re-infection, which is also important. Pa is in the biofilm. Not in the fixed macrophages like NTM.
So all things being equal, if Arikace maintains or improves FEV1 and kills a significant number of Pa bacteria that do not come back that fast . . . what national regulatory agency would push back against that kind of data and outcome NOT being clinically significant for treating patients in the US? Particularly when the FDA indicated they only wanted a dog inhalation study to close the safety file.
Also, remember the NTM study (which is strictly a US clinical trial) represents the clinical safety and (probably efficacy) of 84 straight days of Arikace. That data, which will be reviewed by the QA department within NIH (they have their own QA) because of the huge number of clinical trials performed on the NIH Campus, so that data will almost certainly be considered depending upon the outcome of that trial. I still believe the NTM trial is 60% plus of INSM's future because of the recent orphan status granted and the pan-global interest that will follow from that trial.
The FDA is obligated to review any and all data that is presented in an IND, or a NDA. They have to review the file in its entirity and assess safety and efficacy. Case in point, the EAP IND for Iplex treatment of ALS was reviewed by the FDA and approved for "compassionate use" (based upon the Italian data) even though INSM did not file the requested Phase IIa/Phase IIb IND as requested by the FDA. This is an example of the FDA reviewing data that did not conform to Phase I criteria (defined primary and secondary endpoints). The EAP IND was facilitated by the approved safety and efficacy master file for short stature. The endpoints were never agreed upon or attained.