New crohns drug heading to phase 3 clinical trial

Celgene recently bought ged-0301 from Nogra pharma for $710 million after seeing unpublished phase 2 results. The phase 2 results should be published end of this month. Celgene plans to start a phase 3 trial soon.

It is a new method of treatment - a smad7 knockdown antisense oligoneucteotide. And apparently, it had a great safety profile and even better remission rates. So, I'm cautiously hopeful!

Here are some details:
http://omicsonline.org/open-access/smad-sustains-inflammation-in-the-gut-from-bench-to-bedside-2155-9899.1000236.pdf

http://www.nature.com/mt/journal/v20/n4/full/mt2011290a.html

Anyone have any experience or knowledge about this drug?

Thanks PV,

Always apprecieate reading about potential new treatments. I read through the phase 1 trial results. It really seems promising. They showed a very high success rate, but didn't do follow-up colonoscopys, they only used the CDAI scores for analysis.

Also, it seems most of the participants hadn't had a surgery, and may not have presented on the severe side.

Still, this does indeed look very hopeful, and thanks again for posting.

That's great!!! I hope that if the Humira wears off on me that this drug can replace Humira for me...now if they could just make a med to clear up irritable bowel syndrome too then I'd finally be free from using the toilet multiple times a day still, considering my crohn's is in clinical remission...now I have to get a handle on my IBS.

More data released today. 72% remission rate at 4 weeks, 67% at 12 weeks with no significant safety issues. This is extremely exciting news. Anything can happen in phase III, but sometimes it's nice for us to have a little hope.

news.investors.com/technology/102114-722719-celg-stock-nears-high-on-mongersen-data.htm

That is very exciting. They mentioned that the great PII results make this less likely to be a fluke and more likely to pass through PIII. Like mentioned in the first post though, cautious optimism is probably the way to go.

I'm really surprised to see a drug like this already in development, however. I was reading about RNA interference as a method of treating disease a few weeks ago and it seemed like something in the distant future. While this isn't technically RNAi, the basic idea is still the same (interfering with the RNA that makes a protein that causes inflammation). I guess my point is that it's really awesome to see this technology already be so close to being available. Really hoping that it turns out to be as effective as these phase 2 trials are saying. It seems to work very quickly too. This could definitely be a game changer.