GlaxoSmithKline Scraps Rights to Failed Muscular Dystrophy Drug


Pharmaceutical giant GlaxoSmithKline (NYSE:GSK) has turned over the rights to drisapersen, a drug in development for the treatment of Duchenne muscular dystrophy, back to Prosensa Holding NV (NASDAQ:RNA), a Dutch biopharmaceutical company that specializes in medicines for neuromuscular diseases, Reuters reports.

The British pharmaceutical firm is terminating a 2009 partnership with Prosensa after the drug failed to meet expected goals in a critical phase III clinical trial in late September. The drug didn’t significantly outperform the placebo in its final stage trial, and as a result, the company’s hopes for the drug began to dwindle.

GlaxoSmithKline on Monday announced that Prosensa will now have full, exclusive rights to the drug. The analysis of the failed trial results as well as an assessment of what the next steps for the drug might be are still ongoing, and results of the analysis can be expected early this year, according to a press release per The Wall Street Journal.

Prosensa CEO Hans Schikan said in a statement: “Given the devastating impact of DMD on boys and their families, it is of critical importance that this robust dataset is understood in its entirety and no stone is left unturned. The drisapersen program represents one of the largest datasets in this disease to date, and we continue to collaborate with patient groups and other stakeholders to help patients with DMD.”

Schikan also indirectly responded to GlaxoSmithKline’s decision to hand back rights to the drug, saying: “Prosensa is now in a favorable strategic position to advance the DMD portfolio, which includes drisapersen and five additional compounds, three of which are currently in clinical development. We will continue to work closely with patient groups, investigators, academia, and regulators to ensure that we do everything we can to bring treatments to boys affected by DMD.”

The drug, drisapersen, formerly GSK2402968/PRO051, is an antisense oglionucleotide that uses a strategy called “exon skipping” — exons being sections of DNA that code for protein — to treat the disease. According to Prosensa’s website, drisapersen is the company’s lead RNA-based product.

Duchenne musucular dystrophy is a debilitating childhood neuromuscular disease that affects as many as one in 3,500 male births. The disease, which is caused by a defective gene for a muscular protein, worsens quickly and results in a severely shortened lifespan. Most boys diagnosed with disease die before the age of 25, usually from lung problems.