GSK trial of exon 51 skipping on Ambulatory Boys

Does anyone know what is going on with GSK and the trial on exon 51 skipping?  They said the trial will not be conducted this year. Does that mean next year, or not at all?  Have they pulled the plug on this trial?  If they have, why the heck are they moving forward with the non-ambulatory trial which the FDA has green lighted.  
Insight????

Views: 691

Reply to This

Replies to This Discussion

 

I came across this note where a bunch of AVI stock holders chat...  Please, go ahead and question GSK's drug and their potential reasons for hurrying up with the EU trial.

 

"GSK's motives for investing were not that noble. They chose a drug that they knew gad troubled potential ( inferior to AVII) and would jump through all the hoops knowing it would lead to failure, this way they could ultimately get the tax relief they need."

Just lovely!

I have been checking the yahoo board for AVII for almost 2 years now. There are a few folks who appear to have a full time job of positng on yahoo boards.

 

 They have 3 pet topics

1. Shouting at each other and calling names :-)

2. Telling the world that AVII has anti viral contracts from defense

3. AVII has patents on DMD and their drug is better than that of Prosensa

 

Personally I feel that they are more intrested in fooling people to buy or sell as per their wishes.

 

However these guys are well informed, both with the science and the industry. I remember one guy even predicted the when the last AVII CEO will be fired. I would still read the board but take everything wiht lots of salt.

 

 

Yes, they can get fairly animated... Definitely taken with a grain of salt!

Unfortunately, what they say about GSK is more likely true. (GSK) Prosensa's drug is old and at the dosing levels needed for it to do anything, it is just too toxic.  I'm very glad the FDA repeatedly fails to approve it in the US.  AVI did have a patent on their drug, but lost it.  If GSK really wants to go with a winner, they should snap up AVI's expired patent and help make a real difference in the DMD world.  

Looking to clear things up here......If the Prosensa's {GSK} drug is too toxic and inaffective, why are they doing phase III studies in Europe???  My son falls into this exon skipping category and I was planning on putting him in the trials when they start in the US. Please fill me in on what I'm not getting! Thanks  

I am not aware of any serious issues with Prosensa drug.

 

The length of Prosensa compound is 20 units while that of AVI is 30 units (dont remeber what these units are but it was in one of the papers). From a lay person's perspective,Mili gram to Mili gram comparision, AVII should be more specifc and also toxic. Prosensa drug should be easy to go in and out of the cell more easily hence less toxic but because it is smaller it will be less specifc and hence would require more dosage.

 

It can be argued that to achieve the same end results more of Prosensa drug is required hence dosage amounts are high with Prosensa and effective toxicity may be more. This however can be prooven only after both the compounds have been through a trial and a dosing regeme has been established.

 

Unfortunately not allowing trials is killing something which we dont have. Time.

 

I agree!!!!!!!!!!!!!!!   Thanks for the info!!!!!!

Tulika said:

I am not aware of any serious issues with Prosensa drug.

 

The length of Prosensa compound is 20 units while that of AVI is 30 units (dont remeber what these units are but it was in one of the papers). From a lay person's perspective,Mili gram to Mili gram comparision, AVII should be more specifc and also toxic. Prosensa drug should be easy to go in and out of the cell more easily hence less toxic but because it is smaller it will be less specifc and hence would require more dosage.

 

It can be argued that to achieve the same end results more of Prosensa drug is required hence dosage amounts are high with Prosensa and effective toxicity may be more. This however can be prooven only after both the compounds have been through a trial and a dosing regeme has been established.

 

Unfortunately not allowing trials is killing something which we dont have. Time.

 

Before you criticize avii listen to yesterdays call

 

I get the feeling they are busting there balls to get the trials started

Music to my ears!  Wish the waiting was over and skipping was available for everyone. 

arnold said:

I am CERTAINLY NOT CRITICIZING AVI - I deem them as the only current hope of proceeding with exon 51 skipping in a way that can actually help my child and others like him.  Posted results do not sugar coat and AVI's drug is far superior to that of Prosensa/GSK.  It has been proven to be effective.  GSK's has proven to be toxic - thus repeated denials by the FDA for human ambulatory trials.

I was commenting on GSK's trial. The Prosensa drug is old and has shown to be ineffective at getting to the muscles at the dosages they currently use.  When the dose is raised, toxicity flags are raised.  EU is far more liberal than the US when it comes to testing drugs on humans - and I personally feel they are correct to deny GSK trial options here.

I hate to paint the bleak picture, but it really boils down to greed.  GSK presses on because their stock holders would have a fit if they pulled out of a phase III trial and if they did, no tax relief.  I find it difficult at best to believe that GSK has the patients as their primary concern here.  Last year they paid out the largest fine in pharma history.  What makes it worse is how they knew years before they were going to be fined, but did not put an end to the drug tainting. They had been putting funds away for a while in anticipation of this fine of nearly a billion dollars. In the world of drugs, money does all the talking. 

 

I hope to God that AVI stockholders do not put an end to their DMD research - especially exon 51 skipping.  They have come up with a very plausible treatment - certainly not a cure, but a good treatment to help extend the loves of our children.  Maybe they will press on with antibiotics or other drugs that are far more lucrative for them - I do not know, but I can only hope they stick with DMD and help our children.

 

 

 

I hope that whatever happens, it happens quickly for all our boys.

Reply to Discussion

RSS

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

Members

Events

© 2019   Created by PPMD.   Powered by

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