I have gotten several google alerts on the Aceleron and Shire arrangement that came down this last week.  Shire, a drug company, has agreed to pour in $490mil into Aceleron for the commercial drug rights.  The initial deposit is 45mil. 

I know that Aceleron has completed their phase II clinical trials and are now recruiting for DMD boys in Canada.  Just wondering if someone can give me a time line on when this drug may come into commercial use.  If a company like Shire is willing to invest 490mil, I would think that they must be very certain that the drug will come together. 

Next question would be, will it be affordable to us DMD-ers.  Hopefully, there will be other markets - other diseases or conditions that will benefit from this drug that increases uptropin and builds muscle and reduces fibrosis.

Thanks for your imput.
Char Burke 

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Still years away from market approval if proven effective. The trial in Canada is dose ranging trying to find a therapeutic dose level. They plan to start trials in the US next year. If approved it will be covered by insurance so hopefully not incredibly expensive ... but who knows. It' too early to talk about approval until we see some data in DMD boys, drugs worked in animals but the results did not translate in DMD boys by now, we need to wait and see. With trials starting in 2011 I don't think it can hit the market earlier than 3 years or so from now.

Also, I do not know how certain Shire is that this can work. Both Biomarin and GSK made similar deals and paid money upfront, this is the way these deals are made, no guarantee that the durgs will work though.
It is interesting that Shire is putting in $45 million in upfront money, and they're only getting US/Canada commercialization rights for that. Link.

The GSK / Prosensa deal was $25 million upfront.

Does that indicate anything about Shire's confidence level? Don't know. Maybe they just want to be first to market, so they put more money in up front.
Ofelia Marin said:
Still years away from market approval if proven effective. The trial in Canada is dose ranging trying to find a therapeutic dose level. They plan to start trials in the US next year. If approved it will be covered by insurance so hopefully not incredibly expensive ... but who knows. It' too early to talk about approval until we see some data in DMD boys, drugs worked in animals but the results did not translate in DMD boys by now, we need to wait and see. With trials starting in 2011 I don't think it can hit the market earlier than 3 years or so from now.
Ofelia - You indicate that the "drug works in animals but results did not translate in DMD"...I do know that the drug was tested in menopasal women in Canada with good results - albeit, it's not the DMD boys. So, yes it still has to do through many years of testing. Just happy there is something to look forward to. It sure beats nothing - if your son does not have the exon 51 deletion that seems to be the target of the exon skipping. Char

Also, I do not know how certain Shire is that this can work. Both Biomarin and GSK made similar deals and paid money upfront, this is the way these deals are made, no guarantee that the durgs will work though.
I think GSK paid less upfront b/c the % of potential patients is much lower.

I think they said Acceleron gets rights in US/Canada and Shire rest of the World.

"Acceleron will hold U.S. and Canadian commercialization rights, while Shire will hold commercialization rights to the rest of the world"

Keith Van Houten said:
It is interesting that Shire is putting in $45 million in upfront money, and they're only getting US/Canada commercialization rights for that. Link.

The GSK / Prosensa deal was $25 million upfront.

Does that indicate anything about Shire's confidence level? Don't know. Maybe they just want to be first to market, so they put more money in up front.
Right, it was tested with good results in post-menopausal women...I do not know how it will work in dystrophic muscles before I see some data. Wyatt Pharma tested a myostatin inhibitor in a clinical trial a couple of years ago, but that did not work; however ACE-031 is supposedly more efficient and does not inhibit only myostatin so I am also hopeful! Even if exon skipping is approved, I think a combination of exon skipping and myostatin inhibitor (ace-031) would give much better results than exon skipping alone. The results obtained so far with PMOs for exon skipping did not look that good, produced very inconsistent results. There is a lot more work to be done with that. ACE-031 looks promising ...I pray it works and gets approved as soon as possible.

Char Burke said:
Ofelia Marin said:
Still years away from market approval if proven effective. The trial in Canada is dose ranging trying to find a therapeutic dose level. They plan to start trials in the US next year. If approved it will be covered by insurance so hopefully not incredibly expensive ... but who knows. It' too early to talk about approval until we see some data in DMD boys, drugs worked in animals but the results did not translate in DMD boys by now, we need to wait and see. With trials starting in 2011 I don't think it can hit the market earlier than 3 years or so from now.
Ofelia - You indicate that the "drug works in animals but results did not translate in DMD"...I do know that the drug was tested in menopasal women in Canada with good results - albeit, it's not the DMD boys. So, yes it still has to do through many years of testing. Just happy there is something to look forward to. It sure beats nothing - if your son does not have the exon 51 deletion that seems to be the target of the exon skipping. Char Also, I do not know how certain Shire is that this can work. Both Biomarin and GSK made similar deals and paid money upfront, this is the way these deals are made, no guarantee that the durgs will work though.
In the first site listed, they say how the ACE-031 trial can benefit more than just DMD. They say, "Over the past few years, pharmaceutical companies have been racing to develop ways to mimic myostatin gene mutations in the hope of treating everything from the muscle loss that accompanies muscular dystrophy, cancer, and aging to obesity and other metabolic disorders." This is probably why there are others investing in this. It is able to serve more than one medical purpose.

http://www.technologyreview.com/biomedicine/19589/?a=f
http://www.checkorphan.org/grid/news/treatment/acceleron-pharma-ini...
http://www.medicalnewstoday.com/articles/187960.php
http://www.acceleronpharma.com/content/products/ace-031/duchenne.jsp
Yes, many forms of MD, ALS, lose of muscle due the some forms of cancer, even due to advanced age. The market is very large, unlike the other treatments tested for DMD at this point.

Joshua's mom said:
In the first site listed, they say how the ACE-031 trial can benefit more than just DMD. They say, "Over the past few years, pharmaceutical companies have been racing to develop ways to mimic myostatin gene mutations in the hope of treating everything from the muscle loss that accompanies muscular dystrophy, cancer, and aging to obesity and other metabolic disorders." This is probably why there are others investing in this. It is able to serve more than one medical purpose.

http://www.technologyreview.com/biomedicine/19589/?a=f
http://www.checkorphan.org/grid/news/treatment/acceleron-pharma-ini...
http://www.medicalnewstoday.com/articles/187960.php
http://www.acceleronpharma.com/content/products/ace-031/duchenne.jsp
You're right, Ofelia - I read it wrong.

Shire's getting access to everything but the US and Canada, which is about 95% of the world's population, and thus 95% of the DMD population. (or close enough for arguement's sake. there's differences in the numbers based on age)

The 5 most commonly needed exon skips account for something like 30% of the DMD population. (assuming those are the 5 compounds they'd develop first)

So, Shire paid $45 M upfront for the potential of access to 95% of the DMD population, GSK paid $20 M upfront for the potential of access to about 30% of the DMD population.

Of course, the economics of who will be able to afford it factor in as well. Quality of insurance coverage will play a big part. These drugs are clearly going to be extraordinarily expensive, based on the kinds of investments these companies are making and the size of the markets.

Joshua's Mom - I wasn't aware of the other potential uses of myostatin inhibitors, for cancer and such. That only helps bring more money and focus on it, because of the exponential increase in the size of the market. Thanks for pointing that out.

Hi,

I always read about concerns that ACE 031 may use up the storage of stem cells to reach a point that there are no stem cells left.

Do you have any idea about this,please!

Yes, there is concern about stem cell depletion ... no one knows exactly what will happen at this time.

Peter Novak said:

Hi,

I always read about concerns that ACE 031 may use up the storage of stem cells to reach a point that there are no stem cells left.

Do you have any idea about this,please!

Ofelia and Peter - I read a huge article on stem cells.  I read about a researcher that developed stem cells from other sources because of the current administration at the time (Bush) banned usage of them.  What was interesting about the scientist was that he has two kids that are type 1 diabetics and he did indeed figure out a way by developing another type of stem cell to use.  It seems to me that scientists are coming up with more and more ways of reproducing stem cells from different areas of the body.  I guess, I don't share your concern of stem cell depletion at this time.  Further, I thought that the drug that Acceleron is developing isn't coming from stem cells.  I thought it is something that is manufactured from chemicals much like the exon skipping ones.  Char Burke

Char, stem cell depletion is a concern for any myostatin inhibitor. Only time will tell, they have not been tested/used long term so no one knows what's going to happen...

 

http://en.wikipedia.org/wiki/Myostatin

 

"It remains unclear whether long term treatment of muscular dystrophy with myostatin inhibitors is beneficial: the depletion of muscle stem cells could worsen the disease later on."



Char Burke said:

Ofelia and Peter - I read a huge article on stem cells.  I read about a researcher that developed stem cells from other sources because of the current administration at the time (Bush) banned usage of them.  What was interesting about the scientist was that he has two kids that are type 1 diabetics and he did indeed figure out a way by developing another type of stem cell to use.  It seems to me that scientists are coming up with more and more ways of reproducing stem cells from different areas of the body.  I guess, I don't share your concern of stem cell depletion at this time.  Further, I thought that the drug that Acceleron is developing isn't coming from stem cells.  I thought it is something that is manufactured from chemicals much like the exon skipping ones.  Char Burke

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