Replies to post #137323 on Biotech Values

[iwfal]

ISIS -

Part of the reason that ISIS has programs against targets that pharma does not is intentional - many have proven to be undruggable with small molecules (both diabetes and other therapeutic areas). SGLT2 was a notable exception, and proved to be a mistake.

Do you have any idea how assess this? What is small molecule accessible and what is not?

I also be interested in people's take on the Factor XI program - the preclinical data is pretty impressive to me. But I am not so thrilled that they are planning to run the phase 2 program themselves.

I'll look - but if they are wise they will keep a few promising drugs through ph ii for themselves. In particular I would suggest that they should keep the drugs which have such phenomenal benefit over SoC that even a small randomized ph ii should knock it out of the park. They may still need BP to take it through a huge safety clinical program.

PS Thanks for the R&D day slides.

[iwfal]

ISIS - FWIW - Just finished listening to their R&D Day and my initial response is that it was the best biotech R&D Day I've ever seen. (And I have seen 10's). All the science data was relevant and meaningful. Little chest thumping about the size of the market or other items deserving of only a line or two. And the CEO's discussion on balancing the portfolio was absolutely crystal clear and on target.

They even brought up some of the concerns and addressed them head on - e.g. as soon as I saw the CRP target mentioned somewhere early in the presentation I asked of myself the obvious question of whether CRP is a legitimate target or only a marker. And before the end of the presentation the CEO answered head on - yes, it is a risk as to whether it is an actor or just a bystander. But the market is large enough that it is a risk worth taking. A legitimate and considered position. And given their willingness to cancel even successful programs because they look too difficult or they will be a me-too I'd tend to trust them to design a perceptive set of trials and kill it early if appropriate.

PS Do you know what size royalties they typically mean when they say 'double digit'?

PPS Do you know of any background in anti-sense cancer risks? (it is the obvious question when it comes to playing with nucleotides)

PPPS Do you know the kind/size of injection used for their drugs? IM? SC? ...

PPPPS ?How the heck are they planning to make an oral anti-sense?

PPPPPS ?How are they using antisense to fix a splicing problem? (I'd hunt through the patents for this and the previous question - but I do not have the time to filter through 1500 patents so am taking the lazy way out and hoping someone has the answer at hand -g-)