$10M for the NTM study and he said the PH3 or PH4elf-imposed) would only be 3 months (as Pianoman stated) so the cost will not be major. This was a very good presentation and he answered questions quite thorough and honestly.
The cost is not the limitation. What dose, duration of treatment and endpoint will be agreed upon with the FDA so that INSM meets their primary and secondary endpoints? Hopefully Dr. Olivier has some insights that will be written into the Phase III.
If you look at the one Primary and the other Secondary endpoints, they all say "Safety" after the criteria. I think the NTM study was written as a Phase IIa with the hope that the therapeutic index would be so outstanding that FDA would expedite them to submit an NDA. That is not going to happen as a result of this study. But they did achieve safety.
We shall see what appears on the horizon in the next 3-4 months.
Will Lewis said that the primary endpoint the FDA will be looking at is culture conversion. The other endpoints will mainly be for safety. The dosage is unchanged, 560mg once per day. The duration is 3 months. The arms of the study that he mentioned are (1) treatment naive (2) recalcitrant, and (3) placebo. There would be stratification on CF/non-CF, and MAC/Mabscessus
My own opinion is that they should try it on some other NTM bugs (other than MAC and M abcsessus), such as M kansasii or M chelonae.
"I think the NTM study was written as a Phase IIa"
It was Phase 3 and "pivotal" (in insmed's words) before they accidentally caused the clinical hold (for most of a year), trashed the stock, diluted us (again) and then said "opps, only 2 of the overdosesed dogs were affected afterall).