Prosensa and GlaxoSmithKline form alliance to fight Duchenne Muscular Dystrophy

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PRESS RELEASE

Prosensa and GlaxoSmithKline form alliance to fight Duchenne Muscular Dystrophy


Leiden and London, October 13, 2009 – Prosensa, the Dutch based biopharmaceutical company focusing on RNA modulating therapeutics, and GlaxoSmithKline (LSE: GSK) announce that they have entered into an exclusive worldwide collaboration for the development and commercialization of RNA based therapeutics for Duchenne Muscular Dystrophy (DMD). DMD is a severely debilitating childhood neuromuscular disease that affects one in 3,500 newborn boys. Currently, there is no treatment to prevent the eventual fatal outcome.
The alliance was established under GSK’s Centre of Excellence for External Drug Discovery (ceedd) which seeks to collaborate with companies at the leading edge of highly innovative and transformative science. The scope of the alliance includes four RNA-based products intended to treat specific, but different, subpopulations of patients suffering from DMD.
Under the terms of the agreement, GSK will obtain an exclusive worldwide license to develop and commercialize Prosensa’s lead compound, PRO051, intended to treat DMD by skipping exon 51 of the dystrophin gene. Mutations in the dystrophin gene result in the absence of normal dystrophin protein, which is necessary for proper muscle cell function. GSK’s Neurosciences Medicines Development Centre will continue to progress the further development of PRO051 in collaboration with Prosensa. Both parties have begun preparations for a Phase III study which is intended to start in early 2010. GSK will fund all costs associated with the further clinical development of PRO051. In addition, GSK has exclusive options to license three more RNA-based compounds targeting additional DMD exons. One such option includes Prosensa’s second lead compound, PRO044, which targets the skipping of exon 44 and for which Prosensa expects to initiate a Phase I/II study before the end of 2009. In this case, GSK’s option rights will be triggered by a successful completion of this study.
The financial terms include a GBP 16 million (USD 25 million) upfront payment. Furthermore, Prosensa is eligible to receive up to GBP 412 million (USD 655 million) in milestones payments if all four compounds are successfully developed and is also entitled to double-digit royalties on product sales. Prosensa will retain commercial participatory rights, and has an option to expand its commercial rights, in certain European countries on products arising under the collaboration.
Hans Schikan, CEO of Prosensa said: “We are delighted by GSK’s commitment to develop and commercialize our promising lead compound, PRO051. This alliance will not only speed-up the further development of PRO051, but will also accelerate the progress of our complementary DMD therapeutics, allowing us to reach a broader patient population. Our joint commitment to serve Duchenne patients provides a solid basis to achieve our goal to improve the lives of these boys and their families.”
“We are always looking for inspiring science to take forward and are incredibly excited at the prospect of working with Prosensa to bring a vital new treatment to patients with
DMD,” said Dr. Christoph Westphal, CEO of Sirtris, a GSK company, and SVP and Head of ceedd, GSK.
“PRO051 has generated a great deal of interest from the pharmaceutical industry. This strategic alliance with a premier pharmaceutical company is an acknowledgement of our business strategy and of the strength of our technology platform, which was jointly developed with Leiden University Medical Center. It is a transformative event for Prosensa that will enable us to accelerate the development of our pipeline of RNA modulating therapeutics and to broaden our development scope into other therapeutic areas with an unmet medical need,” said Luc Dochez, Vice President Business Development at Prosensa.

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This also shows how ridiculous it is that FDA holds the start of AVI-4658 clinical trials in the US. If they would lift the hold AVI will be able to move faster as well.
Thank God, finally a big pharma bite!! Good to see this one Ofelia, no, BETTER THAN GOOD!!

Something must be working, somewhere, with exon skipping or GSK wouldn't jump in right on the cusp of our finding out if it is effective. Part of the FDA dilema, I think, is the new administration STILL hasn't filled all positions and just barely filled others. FDA is still playing by the old rules, hasn't gotten up to speed. Not sure if they will with regard to exon skipping now since the science is moving very, very fast, much faster than our government. But, not everybody has to wait for FDA. Not everybody can. And so I remain hopeful other avenues will present themselves in due time.

If no other options come about in due time we can always exit the US.
If that isn't an option then we get out the pitchforks and go medieval on DC.
Either way, our sons will live.
I think the same thing Cheryl! I do not think GSK would partner with Prosensa and pay $25 MM upfront if they did not believe in exon skipping. Plus, the saw the results and those should look pretty good!



cheryl cliff said:
Thank God, finally a big pharma bite!! Good to see this one Ofelia, no, BETTER THAN GOOD!!

Something must be working, somewhere, with exon skipping or GSK wouldn't jump in right on the cusp of our finding out if it is effective. Part of the FDA dilema, I think, is the new administration STILL hasn't filled all positions and just barely filled others. FDA is still playing by the old rules, hasn't gotten up to speed. Not sure if they will with regard to exon skipping now since the science is moving very, very fast, much faster than our government. But, not everybody has to wait for FDA. Not everybody can. And so I remain hopeful other avenues will present themselves in due time.

If no other options come about in due time we can always exit the US.
If that isn't an option then we get out the pitchforks and go medieval on DC.
Either way, our sons will live.
This is so cool, it's hard for me to avoid violating the use of language policy for this family friendly site. Not that they will, but GSK could take its ad budget for Tums for one days worth college bowl games on January 1 and probably fund parallel trials for exon skipping drugs targeting the twenty most common mutation categories. Now, AVI needs to find a rich uncle to beat down the door at the FDA
This is awesome news!!!!!! It made my day.
Wow, that's a lot of money. I think it says 2 things. $25 million down says they think it's promising. $655 million + royalties for 4 approved treatments means they think they can recover that much down the road....
Translated to pharma speak...ITS WORKING!!

Keith Van Houten said:
Wow, that's a lot of money. I think it says 2 things. $25 million down says they think it's promising. $655 million + royalties for 4 approved treatments means they think they can recover that much down the road....
This is GREAT news!!
We need to keep up advocacy and show GSK they made the right choice...they need to know we are in a hurry!

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