NorthWest Biotherapeutics Inc (NWBO)

[HyGro]

JAMA Oncology paper is done independently of the FDA filing. FDA would accept the manuscript version as it fails to reference key deficiencies in the trial -- FDA partial hold, failure to report PFS clinical progression endpoint, confounding of naïve OS endpoint and the statistical manipulation of the endpoint, the post hoc data dredged nature of the recurrent OS endpoint (this was a naïve GBM trial), etc.

Patients in compassionate use don't meet clinical trial screening requirements. It can be submitted, but won't be useable for the drug label, it is just added reference that may or may not have any value. Usually it doesn't add value. NWBO hasn't every reported any compassionate use data -- many drugs do publications from that dataset, but not NWBO.

It may get approved, but this extensive set of trial deficiencies for the ONLY Phase 3 trial make the odds of rejection much higher than a properly run set of two trials used for approval. NWBO doesn't have a choice but to file -- no more cash and can't wait 16 years for the second Phase 3 trial that is normally required by the FDA.