Hey mate, I appreciate the reply.I had a quick look online for...

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raisinsareheresy

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05/11/21

16:28

Post #: 57353541
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Originally posted by BoomCrashOffera: ↑

I appreciate the considered post shyaam. To your last point - I agree - and take responsibility for some less than respectful rhetoric - especially considering my "newbie" status compared to the what appears to be some knowledgeable long term contributors here. I should stick to a rule that nothing good comes from posting after 10pm. Apologies to Itsa.

I am not concerned with the CR - although it could have been handled better imo. I am concerned with reinforcing my conviction on what has become a largish position for me in a binary outcome biotech investment.

Appreciate you looking into the PUL 2.0 results like I did. It would be handy to know what the normal six month deterioration in PUL 2.0 results for non-ambulant boys is - just to determine margin of error on efficacy. For instance - if the placebo scores average minus 4.0 on the scale, it would make 0.125 or 0.9 look a lot better than if the normal deterioration is minus 1.0. The fact that the scores are in integers as well, means there is wider margin of error in scores. The lymphocyte reduction data in my mind looked more conclusive, but I understand that is not an endpoint for the P2b trial (?).

The main reason for the point about the PUL 2.0 results, was to see the statistical robustness of the testing parameters and getting a better idea of the probability of success in P2b. The grip and pinch test results seemed to be less convincing - but I'm no expert here and would be glad to be told otherwise.

P2b and P3 trials with promising prior P2a results have been known to fail because of factors such as:

- changing the trial parameters from the prior trial (I understand we are trialing both 25mg and 50mg so either dose being successful, or both, would be a tick).
- having less control over the clinical application in the larger trial
- results not reproducable

The costs of failure are high obviously and reasonably binary in nature.

The Ionis licensing deal terms will be hard to get imo as it is probably going to be kept under wraps.

Yes - there are some very informative/knowledgeable posters here that I have learned a lot from in a short time. I am happy that you are open to critical questioning as it coincides with my ethos of investment - seeking the truth.

cheers, Boom

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Hey mate, I appreciate the reply.

I had a quick look online for some research into the expected decrease in PUL 2.0 scores for a non ambulant DMD population, and found the following study: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6010252/

The study tested a population of both ambulant and non ambulant boys (total pop = 187) and measured their PUL 2.0 scores over 24 months. The results found decreases for both ambulant (mean = -2.07) and non-ambulant (mean = -4.36) for PUL 2.0+ scores over this period. The regression used for the data for both were linear, so let’s presume the average decrease for the relevant non-ambulant population for comparison is -4.36/4 for 6 months of the study, yielding a -1.09 average PUL 2.0 decrease. I would think this is still a significant distance from the conservative +0.125 value we get to from the P2A results (removing patient 8’s +7.0 score).

I’ll have to look into the lymphocyte data in the P2A trial results to really comment, but I think we can only hope that the 25mg and 50mg stratums yield good results in P2B (for both us as shareholders and for these poor boys).

ANP Price at posting: 22.5¢ Sentiment: Hold Disclosure: Held
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