[ Unless we see cures in the Ph2/3, we think immediate US filing is unlikely, no matter what KOLs agitate FDA for. ]
Err ..... and what if we see a cure rate of a monster 25%?
Worth remembering that the analyst set his price target with the assumption that NTM results which didn't include cures would not qualify for accelerated approval -
[ Reiterate BUY, raising PT to $30 on Arikace's increased potential in nontuberculous mycobacteria (NTM).
INSM's lead drug Arikace is an inhaled liposomal form of potent, FDA-approved antibiotic amikacin. We expect positive data from a Ph2 US NTM trial in March.
Our higher pNPV-based $30 target is driven by a higher chance of success.
Based on proprietary analysis/KOL talk, we think INSM's Ph2/3 NTM trial has a good chance of success with late March top-line data. The trial is powered to show a 1-point change on the 7-point semiquantitative scale of NTM infection burden. We think a 1-point benefit in these severe trial patients (whose baseline score we think will be ~5) would correlate with a benefit in QoL and be clinically meaningful.
More confidence on data variance and noise: interim look gives us comfort on "neatness" of data and powering.
INSM conducted an interim blinded look at the variance of the data to determine if the Ph2 was adequately powered. The look indicated data dispersion/ variance fell within trial powering assumption. We think it is unlikely INSM was able to see graphical representation of the data dispersion during variance calculation, which could have provided a "tell" on data.
Unless we see cures in the Ph2/3, we think immediate US filing is unlikely, no matter what KOLs agitate FDA for.
Based on our KOL conversations, we think FDA still has a lot to learn about NTM, having been previously hung up on TB-like "cure" endpoints. Given trial patients are end of the line, hard to treat, cures with only 3-mo Tx are unlikely. ]
I wonder what the WHO and CDC will think as well - considering the major non-compliance issue associated with the currently-recommended 18-24 month antibiotic regimen for drug-resistant mycobacterial infection.