ARC-AAT part B

[trader87]

Any guess on which metric Arrowhead hit? One patient at 60% KD or three at 30%.
would love to have Hparch comment.
Jtrader

[Think2Succeed]

It's difficult to guess but personally I'd prefer 1 patient 60% KD since that would be more meaningful in terms of efficiency (closer to 1 log), especially since part A of the study ended faster than anticipated which means the other patients have time to catch up if the drug is active. Three patients achieving 30% is a better scenario if most patients achieved at least 30% KD AND the KD curve continues to be pointing downwards. We should find out soon what scenario played out...

T2S

[christopher2206]

T2S,

I am hoping the activity / tolerability of AAT is inline with what 520 has shown us. If the drug is safe and 100% active in all patients at some level we have something to build on. I am curious what the yardstick for success will be in AAT. There was an article a month or so ago that had JM and BG both talking about AAT and both had a different outlook as testing for success in trial patients, and screening protocol. The need for an effective standard of care drug in AAT is huge.

Thanks for the new board. I wasn't able to add my _'s to my username.

[Think2Succeed]

Chris,

Currently there is no treatment for AAT (I remember in one of the presentations C. Anzalone mentioning that he and the FDA had some troubles when trying to establish a clear endpoint for ARC-AAT) so ANY type of benefit ARC-AAT can provide with a good safety profile will make the drug approvable.

PS: I sent you a PM with instructions on how to add the number of shares to your profile.

T2S

[trader87]

I was an owner of ALNY, and rode the stock from 19 down to 6, the real catalyst was the ALN-TTRsc phase 1 data. See linked chart and press release. I think ARWRs AAT phase 1 data will create a similar catalyst.
Jtrader

investors.alnylam.com/releasedetail.cfm?ReleaseID=734139

ALNY Chart

[mudbug021]

Its not a question if RNAi therapeutics work anymore. It has become a question of making sure the correct gene is/has been targeted and reacts as intended. 

The only doubt at this stage of ARWR research is the not knowing if the immune system can take over after a specific gene has been turned off or not.  Once this becomes a realization is when ARWR success comes to pass. Until then, investors will have to wait for results.