Biotech Values

[iwfal]

AKAO -

However, the statement below is something of a concern regarding the sample size. ... This will be a non-inferiority trial comparing plazomicin to meropenem with a 15% non-inferiority (NI) margin. As such, the trial will have a sample size of approximately 530 patients, which is significantly smaller than prior industry-sponsored Phase 3 cUTI trials.

On the one hand, it is certainly true that the regulatory agencies are trying to be less demanding wrt antibiotics trial requirements. So smaller might be possible. E.g. has the FDA relaxed the Non Inferiority Bounds from previous trials? (although 15% hardly seems lax) Lesser safety database required? (Or is the company, like many small biotech, just assuming less safety database needed)


But OTOH Dew is right, they are missing key powering assumptions.


And, more globally, on the topic of AKAO, my concerns are:

a) The ph2 data they have in their chart package has the appearance of serious mining - e.g. they show mITT with 35% of patients missing.

b) the drug you are talking about above is an aminoglycoside, with, apparently, the typical significant side effects (e.g. permanent hearing problems). Not very high rate (1% class), but still a very limiting AE.

All told, it has the appearance of a small biotech with a marginal drug. Might get approved, but high chance of some go backs (e.g. get a larger safety database) and, at best, niche market.