Hello science friends. Back from some drinks. Wow, seems like there's a lot of flaming and accusations that I somehow got owned. I think that's probably not that case since my actual work is in clinical trials and health economics. Being my work and passion compelled me to comment on here for a discussion but ended up getting childishly attacked. I haven't actually dissed any investors and have only giving my thoughts on the DATA as it stands ATM and how the future of ATL1102 (not ANP itself) hinges on the successful of the phase 2b and subsequent trials/real-world data as given the rare disease nature/orphan designation, real-world data/phase 4 will be required for ongoing label approval.
I'll respond to posts which have had some substance and ignore the trolls who cannot be objective or state anything that shows any substance
@mephistophles So your comment on the mean PUC 2.0 change on 0.89. The SD is 2.89. That means, based on of an n=9, 68% of the values are between (-2, 3.78). This means that, out of a max score of 42, the mean was 0.89 (2.1% improvement) but for some patients, it was less beneficial or beneficial. This does mean the uncertainty is high and makes sense since n=9, not 20,30 or 50 depending on which reference you prefer. When the mean is < SD, this is concerning as you cannot outright say it's 'beneficial' or 'positive'. At 2.1%, in an non-ambulant population, it may potentially be good from a individual perspective (I mean, what isn't given the disease, any benefit is positive for an individual) but as a regulator/payer (such as PBS), a listing recommendation would not be given based on this. Which is why the PUL 2.0 is promising at best, meaningless at worse.
When I'm referring to price, I'm not referring to the stock price, but the drug itself relative to the buy. A price commercially viable to to make ATL1102 and ANP a success while acceptable to the payer (HTA markets like being Aus i.e PBS and other markets like US being private) will be based on how strong the data is. ATL1102 is not at that point.
@imperatore While many drugs are welcomed by experts in their relative field, it doesn't mean it's the next blockbuster. These experts are investigators so it's their job to talk about data. Orphan designation by EMEA or FDA DOESN'T MEAN the regulators are giving the green light to treat the disease. It means that they have confirmed the status of the drug as an orphan drug, meaning that they have given the green light to develop/research it to see if it can treat a rare disease. You have totally missed interpreted what ODD means. ODD are granted but many ODDs do not make it to market.
Many drugs have strong data but sit in development limbo, regulatory approval limbo or reimbursement limbo. Just look at the list of PBAC recommendations that are stuck in Cabinet limbo and you'll get a better idea
I'll ignore your other attacks as that they are quite childish.
The data is not excellent, it's promising at best. Which is the purpose of my post. Seems like the hyp train of non-science people believe the data is astounding. It's not. Which is why I said the phase 2b needs to deliver astounding results or it's not gonna make it or requires further trials, thus delaying any approval. Don't forget patent expiry
Re: Exondys 51 - Mainly US market, a unique private and capitalistic with exorbitant prices being the norm. It took at least 11 years with 7 in-human trials to get to where it is with conclusive data. ATL1002 has a 'long' way to go to even compare, which is my point. To note, patent for ATL1102 currently is expires in 2029.
I've never disputed the potential benefit of ATL1102 or any drug for that matter. The question is what will be the data. I work in the field for a living and it's my daily job to get patients novel therapies. My post has been about the data and what is needed to bring it to the next level but emotions get in the way of a discussion for some (or most). From non-ambulant to playing video games would be remarkable but that's what needs to be determined, my point all along. The hype atm is as though the data is already superior. It might be, it might not but doesn't look great at the moment, nowhere near excellent.
TL;DR: I wanted to provide some insight as someone who works in the field to balance out the hype but I've come to realise that people have invested too much to accept a balance discussion. I congratulate anyway who is won so far on ANP and my only message (to people who are happy to read without being childish) is be careful around the hype surrounding the data as astounding, as it is currently misleading. You don't have to believe me but ask someone who works in pharma, bioscience, conducts trials, provides medical information, works in Market Access etc.. that knows how to interpret the data and see what they think. I mean it would be wise to ask someone you know personally that knows better right?
Since I'm gonna get flamed anyway, I will say goodbye
(20min delay)
