GlaxoSmithKline amends accord with Isis Pharma; gets FDA panel backing for raxibacumab

USA-based Isis Pharmaceuticals (Nasdaq: ISIS) says it has agreed to amend the clinical development plan and financial terms relating to ISIS-TTRRx with UK pharma giant GlaxoSmithKline (LSE: GSK) to reflect the accelerated development plan for the drug.

ISIS-TTRRx is an antisense drug in development with GSK for the treatment of TTR amyloidosis, a severe and rare genetic disease characterized by progressive dysfunction of peripheral nerves and/or heart tissue, under an agreement that, according to the original deal, was worth a potential $1.5 billion to the US firm (The Pharma Letter April 1, 2010). The revised development plan anticipates initiation of a Phase II/III, registration-directed, clinical study later this year. ISIS-TTRRx is one of several promising drugs in Isis' pipeline that the firm expects will begin registration-directed studies within the next several years.

"In our Phase I study, we demonstrated that ISIS-TTRRx was generally well tolerated and produced dose-dependent significant reductions in TTR protein with several subjects reaching TTR protein levels that were below the limit of assay detection," said Brett Monia, senior vice president, antisense drug discovery at Isis, adding: "We anticipate initiating the next clinical study this year, which will evaluate the effects of ISIS-TTRRx on neurological dysfunction and on quality-of-life in patients with familial amyloid polyneuropathy."