Replies to post #416895 on NorthWest Biotherapeutics Inc (NWBO)

[biosectinvestor]

How do you know they were not using statistical models using the blinded data and their synthetic placebo arm all along and that all they need to do is lay in what are likely better numbers from the unblinded data whenever that is or was ready? They may likely have worked on it for some time. I think you and similar might wonder what there were doing all of these years. Maybe that is what they were doing? Maybe it was all set and ready to go, with revisions.

Hard to know, but I do not think it is easy to say otherwise or either way.

[skitahoe]

I've not found a link to Les G's appearances on the investment show, but I believe it was there that he indicated that he expected a BLA would be filed by the end of this year. Those webcasts were last year, they were prior to the quiet period being declared, and I could be wrong, but that's what I believe he said.

As for developing the BLA, many months ago it was a hot topic here. Several much more knowledgeable posters than me brought up the various ways that development and approval could be expedited, many included the rolling BLA in which completed parts of the BLA are submitted separately. I gather that those that are submitted undergo immediate review, and if revisions are deemed necessary the company is notified of the changes deemed necessary. I may be wrong about this, but I don't know of another advantage to submitting the BLA in sections if those sections aren't being reviewed and the company advised of questions or omissions noted in the review.

It's my belief that the company has worked closely with the four regulators in establishing the SAP which changed the trial goals as documented in the European versions of Clinical Trials. I believe they continue working closely in developing the BLA that each of the regulators ultimately will act on. Clearly changes in the way the regulators operate are keys to the acceptance of the data using historical data rather than a control group in trials. These changes aren't specifically for the DCVax-L Trial, I believe the regulators are recognizing that for deadly diseases a trial can be designed where all who volunteer for the trial receive the experimental treatment. In the future I believe we'll see many trials that are run in this way.

Gary

[flipper44]

https://jamanetwork.com/journals/jamainternalmedicine/article-abstract/2734629

Of the 249 applications (188 NDAs and 61 BLAs), 222 (89.2%) were disclosed in at least 1 public medium (Figure), including in a press release for 196 applications (78.7%) (Table). Of the press releases, 196 (86.2%) were found on applicants’ websites. Of the applications, 157 (63.1%) were disclosed in SEC filings, including 136 (54.6%) in press releases and SEC filings. The overall rates of public disclosures increased from 87.5% in 2010 to 97.6% in 2016. Publicly traded companies were more likely to disclose applications in any medium compared with nonpublic companies (relative risk, 1.43; 95% CI, 1.09-1.90); private companies were less likely to disclose applications (relative risk, 0.70; 95% CI, 0.53-0.92) (Table). The median time to disclosure was 6 days (IQR, 1-63 days) for press releases, 55 days (IQR, 9-86 days) for SEC filings, and 6 days (IQR, 1-63 days) for any public media.

Current regulations prohibit the US Food and Drug Administration (FDA) from publicly disclosing the existence of pending New Drug Applications (NDAs) or Biologics License Applications (BLAs) unless previously publicly disclosed or acknowledged.1 Applicants may disclose applications in press releases, Securities and Exchange Commission (SEC) filings, or in other media; how frequently they do so is not known.

[antihama]

Any takers?

Not me.