Replies to post #524628 on NorthWest Biotherapeutics Inc (NWBO)

[exwannabe]

the simple answer is Liau never said this:

The part attributed to LL is that NWBO does not have patient level data. It is a fact that they do not. I have no idea if and when LL said it.

It is a fact that Medicenna used patient level data for their P2, and for the P3
See this paper

Using propensity score weighting, we compared rGBM patients from the Phase 2b trial (NCT02858895) (2017-2019) to patients from rGBM registries who had received standard of care therapies (2011-2019) and met eligibility requirements. Propensity scores were estimated using a logistic regression model with 11 covariates. We compared the propensity score weighted groups according to demographic and disease attributes before and after weighting and compared overall survival between the two groups.

In advance of a Phase 3 trial, comparison of Phase 2b trial results to an ECA suggests that MDNA55 may be efficacious in rGBM. In view of the known challenges associated with drug development for rGBM, these results provided a proof-of-concept for the design of a novel hybrid Phase 3 trial. This planned Phase 3 trial incorporates propensity score weighting to create a composite hybrid randomized and external control arm, an approach preferred by the FDA over full replacement of a randomized control with an external control.

Note: propensity scoring requires patient level data.

[biosectinvestor]

Not to pick, I get that you were correcting the other guy. She did not say when they have not responded “after 3 months.” That’s not actually how it works. That implies that they actually respond at all when you are “approved” or that they were waiting 3 months and then when they did not respond they just kind of said, “I guess it’s OK”. If you are good to go, they do not respond ever at that point, and their silence is clear approval after 30’days. You don’t need to wait 3 months to see if they might yet respond, and just guess. They only have a set time period and the FDA has set that time period. That’s it. They give no one a latter of approval under the standard initial process and never after that, during a trial. It’s a well articulated process. They will tell you if you are not good to proceed they don’t want to create rights that can be litigated to suggest they awarded something without the full information. So they are very careful not to do that any time, to create any false expectations.

She simply explained you apply, they don’t respond after a period and you’re free to proceed. But I do not think she implied anything unusual or ambiguous. That is just the standard process always.

The actual approval process for IND’s, meaning every new clinical trial that is approved, is a “non-response” response, after 30 days. That is an actual and firm approval. The same goes for changes to the IND. Why would it be different for any approval of a change to the same trial if the first approval is always a firm non-response after a set period of time? If they do not want you to proceed, they tell you.