Hello All,Has Summit presented any new info regarding the next phase for SMT C1100?

Views: 661

Reply to This

Replies to This Discussion

Hi, in short not that I know of, but I did post recently that they have raised $34m to fund phase 2.  Timeline looks like this:

DMD Top-line results from Phase 1b trial Q2 2014

Submission of clinical trial application for Phase 2 trial (CTA and/or IND) Q3 2014
Complete recruitment for Phase 2 trial H1 2015
Top-line data from Phase 2 trial H1 2016
Select clinical candidate from next generation utrophin modulators H2 2014



Hi Steven

What do we say,all seems so far but we have to stay strong,and keep our eyes on the whole range of products that are being worked on.And hope.Thanks.

Dates provided in company press releases are as reliable as a campaign pledge from a politician. In fact that may too harsh on politicians

Worse than a politician's pledge?Now that must be really bad,David.And also pretty scary and gets one's optimism gauge heading south.

Don't mean to be a downer...I'm optimistic about other programs. I'm probably jaded from following the treatment since Biomarin and we heard all the same garbage from them too. They all know that they CAN move faster ... and yet they consistently miss their targets.

If I gave my boss an absurdly vague estimate like '2nd half of the year' and then didn't deliver. ..I would be looking for a new job

David, well you certainly sound jaded and I don;t know if you work in pharma, but as we all know, and are learning to the communities cost, getting drugs safely through trials to treatment is a horribly long and fraught process.

At least the Summit utrophin program is up and running and happening. I can't see it's in the companies interest to delay anything any longer than is needed (especially as they just raised over $35m to fund the next phase) but it needs to be safe for the boys and then to see if it actually works. For a good portion of the DMD community it is the best or only hope as it;s one of the few potential treatments that should be effective on all boys, not just based on exons. However, if they take an extra few months but get the trial right so it doesn't hit FDA roadblocks then good.  I'm sure Sarepta, PTC and Halo might agree with that and in retrospect might have done a few things differently! 

Note that those dates are without any suspension for the trial,so it might takes 10 years!

I am so disappointed and I feel if any treatment will be as mirage or dream!!!!!!

My point is that any trial will take however long it takes. The dates they put out in the press releases which Steven noted are always best case scenario. Campaign promises.

Yes we should hope for the best. I would love nothing more than to eat my words.

I guess we got to stick in their n just hope for the best,but again i also think it is good not to get my hopes too high that we will have anything in the market in three years.Defence mechanism against disappointment for us and our loved ones.

Best not to get ones hopes up, although we all need some hope in our lives, especially where DMD is concerned. I keep a close eye on most of the biotechs in the sector (as do many here), and Summit are hoping - as I understand it - to get accelerated approval post phase 2 IF it proves effective and as such are intending to run a large enough and well designed trial to make that a real possibility, so it could be available in 2016, but it might be 10 years, I don't know, but they are doing this in a logical way and without much charity funding and I think they should be applauded and supported.

I said 10 years as my expectation based on failures and suspensions we used to hear!

According to me the best way to get treatment if any is to recruit my son in a non-specific mutation approach trial,but I don't know what is the most promising,is it Utrophin booster,HT-100,...?

Can't answer that nor if trials will be in your area or not.

Reply to Discussion

RSS

Need help using this community site? Visit Ning's Help Page.

Members

Events

© 2020   Created by PPMD.   Powered by

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service