Cure Duchenne Funds Pre-Clinical Research for Gene Therapy

June 22, 2010 by CureDuchenne
Filed under Press Releases

The following is a press release sent out today:

Non-Profit Pledges $600,000 to fund Seattle Doctor’s Research aimed at finding a Cure for Duchenne Muscular Dystrophy

SEATTLE, Wash. – June 22, 2010 – CureDuchenne, the Orange County non-profit focused on finding a cure for Duchenne muscular dystrophy (DMD) is one step closer to that goal through the work of Dr. Jeff Chamberlain.

Dr. Chamberlain, of the University of Washington, in partnership with Dr. Stephen Tapscott, of the Fred Hutchinson Cancer Research Center, has identified a promising gene therapy that could lead to a treatment for DMD. The method delivers a new gene to the body’s muscles to replace the defective, disease-causing gene.

As the leading genetic killer of young boys, DMD affects more than 1 in 3,500 boys worldwide and is the most common and lethal form of Muscular Dystrophy. Boys with DMD are usually diagnosed by the age of five, in a wheelchair by age 12, and may be completely paralyzed by their late teens. Historically, most patients with DMD do not live to see adulthood. Duchenne can occur in any family, from any race and from any background.

Debra and Paul Miller, co-founders of CureDuchenne will allocate the $250,000 raised during last weekend’s 2010 Climb to CureDuchenne: Pick Your Peak, where teams across the country summitted mountains from coast to coast to raise awareness for DMD, along with the $165,000 raised by Mack and Sally Brown of Austin, TX during CureDuchenne’s annual “Dealing for Duchenne” fundraiser, to fund Drs. Chamberlain and Tapscott’s pre-clinical research.

“The focus of the trial will be on how the patient’s immune system will respond to the gene delivery,” said Dr. Chamberlain. Immune related issues have emerged as a major limitation on applying gene therapy, and the Seattle team has identified methods that appear to overcome the immune issues that have hampered many previous attempts at gene therapy. Another arm of this research will study the delivery of another gene, Utrophin, through AAV delivery. “I believe we’re on the precipice of an amazing breakthrough with this research. We’re seeing more and more that in order to get this kind of research to the human clinical trial stage, the funding has to come from non-profit organizations like CureDuchenne. Without their support of our work, we’d never have gotten this far,” Chamberlain continued.

If successful, the studies will lead to clinical trials aimed at improving strength in patients with DMD. CureDuchenne is playing a key role in funding not only the pre-clinical research but in providing funds to obtain regulatory approval from the FDA, which regulates all gene therapy and drug trials.

“This is what we’re all about,” said CureDuchenne co-founder Debra Miller. “CureDuchenne is dedicated to focusing on research that is en route to the human clinical trial phase. It’s this level of research that has true potential to save this generation of boys.”

For more information, please visit www.cureduchenne.org

About CureDuchenne

CureDuchenne is a nonprofit organization that raises awareness and funds specifically aimed at taking on Duchenne Muscular Dystrophy (DMD). By working closely with the world’s leading DMD scientists CureDuchenne works to determine the most viable research projects that will accelerate the clinical trial process and bring potential life saving drugs to help this generation of young boys living with the deadly disease.

Tags: Cure, DMD, Gene Therapy, Jeff Chamberlain

Prosensa and GlaxoSmithKline Form Alliance to Fight Duchenne Muscular Dystrophy

October 26, 2009 by CureDuchenne
Filed under Press Releases, Research Articles

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CureDuchenne is very pleased to forward this announcement from Prosensa. Almost six years ago we began a partnership with this biotech company when we funded their pre-clinical work on exon skipping, a method to skip over the genetic mutation that causes Duchenne muscular dystrophy. The founding CEO of Prosensa stated recently that CureDuchenne’s support was vital to the growth of the company, and we couldn’t have done it without the support of so many of you.

GSK is the first large pharmaceutical company to invest substantially in Duchenne. They have committed between $25 million to $650 million in Prosensa. It’s not time to celebrate yet, but with Prosensa’s positive phase 2 results announced last month and this commitment from GSK, we can say that a major milestone has been met.

Exon skipping may only be therapeutic for a sub-set of Duchenne patients. Now is the time to really work hard to get enough potential therapies into the pipeline so all Duchenne boys can be saved.

Thanks to all of you that had faith in CureDuchenne as we have grown over the years.

Debra Miller

President & Founder

***

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.

About DMD and exon skipping
DMD is a severely debilitating childhood neuromuscular disease that affects one in 3,500 newborn boys. These young patients suffer from progressive loss of muscle strength due to the absence of the protein dystrophin, often making them wheelchair-bound before the age of 12, and most die in early adulthood due to respiratory and cardiac failure. Today, there is no treatment to prevent the eventual fatal outcome. The disease is caused by mutations in thedystrophingene, resulting in the absence of the dystrophin protein, which is crucial for the integrity of muscle fiber membranes.

RNA-based therapeutics, specifically antisense oligonucleotides inducing exon skipping, are currently amongst the most promising therapies for DMD. More specifically, antisense oligonucleotides have the capacity to skip an exon and thereby correct the reading frame of DMD transcripts aiming at the synthesis of a largely functional dystrophin protein. Different mutations in the gene require different oligonucleotide drugs. PRO051, the first of its kind, will be suitable for approximately 13% of all DMD patients.

About GlaxoSmithKline
GSK is enhancing the way it discovers and develops new medicines by increasing external alliances. The Centre of Excellence for External Drug Discovery (ceedd) seeks out alliances with world class biotech companies with cutting edge technologies and scientific platforms that can support drug discovery and development. For information about the ceedd, visit the group’s website at www.ceedd.com.

GlaxoSmithKline – one of the world’s leading research-based pharmaceutical and healthcare companies – is committed to improving the quality of human life by enabling people to do more, feel better and live longer. For further information please visit www.gsk.com.

About Prosensa
Prosensa is a highly innovative Dutch biopharmaceutical company focused on the discovery, development and commercialization of nucleic acid based therapeutics correcting gene expression in diseases with large unmet medical needs, in particular neuromuscular disorders. Prosensa is focused on developing a treatment for DMD. Prosensa’s lead compound PRO051 has shown great promise in an advanced phase I/II clinical trial, and will enter a phase III trial early next year. For more information about Prosensa, please visit www.prosensa.eu.

Contact details Prosensa
Hans Schikan, CEO, h.schikan@prosensa.nl
Luc Dochez, VP Business Development, l.dochez@prosensa.nl

Prosensa Therapeutics BV
Wassenaarseweg 72
2333 AL Leiden
The Netherlands
T: +31 71 3322100
W: www.prosensa.eu

Contact details GSK

UK Media enquiries: Philip Thomson (020) 8047 5502
Claire Brough (020) 8047 5502
Stephen Rea (020) 8047 5502
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European Analyst/Investor enquiries: David Mawdsley (020) 8047 5564
Sally Ferguson (020) 8047 5543
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US Analyst/ Investor enquiries: Tom Curry (215) 751 5419
Jen Hill Baxter (215) 751 7002