Prosensa and GlaxoSmithKline Form Alliance to Fight Duchenne Muscular Dystrophy
October 26, 2009 by CureDuchenne
Filed under Press Releases, Research Articles
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
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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.
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
Alexandra Harrison (020) 8047 5502
Gwenan White (020) 8047 5502
US Media enquiries: Nancy Pekarek (919) 483 2839
Mary Anne Rhyne (919) 483 2839
Kevin Colgan (919) 483 2839
Sarah Alspach (919) 483 2839
European Analyst/Investor enquiries: David Mawdsley (020) 8047 5564
Sally Ferguson (020) 8047 5543
Gary Davies (020) 8047 5503
US Analyst/ Investor enquiries: Tom Curry (215) 751 5419
Jen Hill Baxter (215) 751 7002
Prosensa to Present Data at the World Muscle Society Congress
September 8, 2009 by CureDuchenne
Filed under Press Releases, Research Articles
At CureDuchenne, it goes without saying that we’re extremely interested in all the latest findings in the field of Duchenne Muscular Dystrophy and the possibility of finding life enhancing treatments and a cure.
Prosensa just released an announcement stating they’ll be sharing data at the upcoming World Muscle Society Congress in Geneva Switzerland.
Please read the Press Release Posted Here:
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Ibero-American Muscular Dystrophy Community Form Collaboration Network, UPA! Establishes Alliance with CureDuchenne
June 12, 2008 by CureDuchenne
Filed under Press Releases
Leading organizations in Latin America, Spain and Portugal have launched a collaborative network called UPA! to accelerate awareness, information management, and fundraising for neuromuscular disorders. UPA! will focus on improving the quality of life of children already diagnosed with these conditions, as well as, the development of promising treatments and a cure for neuromuscular disorders, with initial emphasis on Duchenne muscular dystrophy, the most lethal genetic killer of young boys worldwide. UPA! has also formed a strategic alliance with California-based CureDuchenne to facilitate shared resources for increased awareness, information management and fundraising for Duchenne muscular dystrophy.
“Upa! is an expression in Spanish used often by parents to encourage their children to get up,” said Leslie Guzman, founder of UPA! and mother of Diego (diagnosed with Duchenne). Upa! reflects the love of a father when he carries his son and encourages him to go forward. It embodies loving relationships and denotes happiness among our families and communities. By using this name, we want to inspire the values of optimism, hope, determination and love.
”We’re excited to partner with UPA! and work with the Latin American and Spanish communities in a common goal. If we’re to succeed in saving boys that have Duchenne today, we must develop partnerships that raise public awareness and focus on funding Duchenne specific research,” said Debra Miller, founder of CureDuchenne and parent of a Duchenne boy. ”Duchenne research is severely underfunded. In 2006, the National Institutes of Health allocated only $18 million for Duchenne specific research, substantially below the amount of funding allocated to other childhood diseases with a much lower mortality rate.”
About Duchenne Muscular Dystrophy (DMD)
One in every 3,500 boys worldwide is afflicted with Duchenne muscular dystrophy. They will be in wheelchairs by 12 and, historically, most have not survived their teenage years. Duchenne knows no boundaries and crosses all cultures and races. The gene mutation that causes Duchenne is usually passed from mother to son, but more than one-third of cases are spontaneous. It can happen in any family. There currently is no cure, but there are now promising therapies on the horizon.
About UPA!
The formation of UPA!is an important first step to facilitate collaboration and effectively mobilize resources against Duchenne muscular dystrophy. The UPA! alliance stands strong and committed to improving today’s quality of life, accelerating the advancement of treatment options, and ultimately finding a cure for Duchenne boys.
UPA! leaders have established the following key priorities:
- Increased public awareness;
- improved quality of life for Duchenne patients;
- Increased fundraising efforts;
- Broader collaboration with global/international networks and programs;
- Improved access to information and treatment options;
- Advancement of basic and applied research
- Primary focus on Duchenne muscular dystrophy
Members of this network of collaboration include:
- ARGENTINA
- Asociación de la Distrofia Muscular de Argentina: www.fundacionfavaloro.org/IN_neurociencias.htm
- Familia Serra Argentina: www.dmdargentina.com.ar/
- BRAZIL
- Associação Brasilera de Distrofia Muscular A.C.: www.abdim.org.br/
- Associação Amigos dos Portadores de Distrofia Muscular: www.aadm.com.br/
- ECUADOR
- MEXICO
- UPA! México
Guadalupe Franco: www.dmdla.org, upamexico@dmdla.org, upaguadalupe@dmdla.org
- Distrofia Muscular Progresiva Ave Sin Vuelo: www.avesinvuelo.org
- Enlace Distrofia Muscular Duchenne Becker: www.enlaceac.org
- Sociedad Mexicana de Distrofia Muscular, Rosa Elena Escobar: rescobarmx@yahoo.com.mx
- Asociación de Distrofia Muscular de Occidente: www.admo.org.mx
- Ricardo Rojas Caballero: www.distrofia-mexico.org
- Asociación Leonesa para la Distrofia Muscular: www.admo.org.mx
- PERU
- Asociación de Distrofia Muscular del Perú: www.admperu.org
- PORTUGAL
- APN: www.apn.pt
- SPAIN
- ASEM: www.asem-esp.org
- Duchenne Parent Project España: duchennepp@orange.es
- Proyecto Deusto de Enfermedades Neuromusculares: www.neuromuscular.deusto.es
- VENEZUELA
- Fundación Paso a Paso: www.pasoapaso.com.ve
- Center Centro Terapéutico de Neurodesarrollo: center01@cantv.net
- UNITED STATES OF AMERICA
- UPA! Leslie Guzman: upa@dmdla.org, upaleslie@dmdla.org, www.dmdla.org, http://groups.google.com/group/DMDLA,
- OTHER MEMBERS
- Families in Bolivia, Chile, Colombia, El Salvador, Republica Dominicana,
Uruguay and Panama.
- Scientists, Clinicians and Friends.
For more information on UPA!, visit www.dmdla.org, or email upa@dmdla.org.
About CureDuchenne
CureDuchenne is a non-profit organization founded in 2003 by Debra and Paul Miller, parents of a Duchenne boy. CureDuchenne’s vision is its name to cure Duchenne muscular dystrophy. CureDuchenne aggressively seeks the most promising, leading edge research breakthroughs and expedites them to the clinical trial process. The ultimate goal…give the boys that have Duchenne now a chance to live a normal life by expediting the cure and/or the availability of therapies that can give quality of life to Duchenne boys, much like insulin does for diabetics.
For more information on Duchenne muscular dystrophy and CureDuchenne, 3334 E. Coast Hwy. #157, Corona del Mar, CA 92625, visit www.cureduchenne.org or call (949) 872-2552. CureDuchenne is a 501(c)(3) organization. Federal Tax ID #: 20-0299958.
