Prosensa and GSK add to muscular dystrophy alliance
pharmafile | June 23, 2010 | News story | Research and Development | GlaxoSmithKline, Prosensa, muscular dystrophy
GSK has extended its research alliance with biotech company Prosensa in the field of muscular dystrophy.
Netherlands-based Prosensa focuses on RNA modulating therapeutics, and has added two extra programmes to its GSK alliance covering novel RNA-based treatments for Duchenne Muscular Dystrophy (DMD).
The companies first agreed an alliance last year, in a deal which could yield up to £437 million plus double-digit royalty payments for Prosensa.
The alliance focuses on Prosensa’s ‘exon skipping’ platform, a technology which helps overcome the genetic problems seen in DMD.
Four new compounds in all will be researched, and will target different subpopulations of patients suffering from DMD. GSK has made two initiation payments to Prosensa and the biotech company could receive further pre-option milestone payments based on research progress.
Prosensa and GSK will focus on the skipping of four exons (i.e. exon 45, 52, 53, and 55), in addition to their existing programmes which target skipping of exon 51 and 44 (PRO051/GSK2402968 and PRO044).
GSK has an option to select two of these additional four compounds for development and commercialisation. Prosensa will retain some limited European marketing rights alongside GSK for the two compounds selected by GSK. For the two compounds not selected by GSK, Prosensa will retain full rights.
“The expansion of our DMD portfolio with four additional compounds is a great step forward in our efforts to develop safe and effective treatments for this severely debilitating disease and to help as many patients as possible,” said Hans Schikan, chief executive of Prosensa
“With Prosensa retaining full commercialisation rights to two of these exon skipping compounds and any other products which are not part of the alliance with GSK, this set-up enables us to develop into a fully integrated specialty-pharma company.”
DMD and exon skipping
Duchenne Muscular Dystrophy is a severely debilitating childhood neuromuscular disease that affects 1 in 3,500 newborn boys.
It is caused by mutations in the DMD gene, resulting in the absence of the dystrophin protein, which is crucial for the integrity of muscle fibre membranes.
The 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.
Most patients die in early adulthood due to respiratory and cardiac failure. Today, there is no treatment to halt the disease.
Elizabeth Vroom is chair of the United Parent Project Muscular Dystrophy, which unites different parent project organisations set up by parents of children with DMD in many countries all over the world.
She said: “We are very pleased with this news. Prosensa and GSK’s commitment to progress further developments of additional products that can provide for a solution in DMD is encouraging and welcomed by all of us.”
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.
Andrew McConaghie
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