October 9, 2014
By Krystle Vermes, BioSpace.com Breaking News Staff
Today, Isis Pharmaceuticals, Inc. , announced that it has received a $18 million milestone payment from GlaxoSmithKline for advancing ISIS-TTR Rx, a drug used to treat patients living with familial amyloid polyneuropathy in a phase 2/3 study.
“ISIS-TTR Rx is the most advanced drug in our collaboration with GSK, and aside from KYNAMRO, it is also the furthest advanced in our late-stage clinical pipeline,” said B. Lynne Parshall, chief operating officer at Isis. “We have patients who have completed fifteen months of therapy and are now receiving ISIS-TTRRx in our open-label extension study. The rapid development of ISIS-TTR Rx from a research-stage program to a drug in a late-stage clinical trial is a testament to the efficiency and productivity of our drug discovery technology platform. As this program advances, we will continue to benefit from GSK’s resources and expertise as well as earn additional milestone payments.”
ISIS-TTR Rx is a drug that is in development with GlaxoSmithKline to treat transthyretin amyloidosis. This is a rare, severe genetic disease that can negatively impact the peripheral nerve or heart tissue.
The phase 2/3 study of the drug is a randomized, double-blind, placebo-controlled, international study that will take place over the course of 15 months. Researchers will be monitoring participants to see if there is a change in their quality of life with or without ISIS-TTR Rx. The investigational drug is designed to inhibit the productions of all forms of TTR.
This is not the first time that Isis has received a milestone payment from GlaxoSmithKline for one of its drugs. The most recent instance took place on Oct. 1 when the company rewarded Isis with a payment of $1.5 million. This was in recognition of Isis’ progression on its ISIS-GSK4 Rx drug, designed to treat an undisclosed ocular disease.
“Our collaboration with GSK has been very productive resulting in four drugs in development,” said B. Lynne Parshall. “We look forward to advancing ISIS-GSK4Rx into clinical development and providing more information on the target and disease opportunity, which we are very excited about.”
Isis has a pipeline of 32 drugs that treat a wide range of diseases, including cardiovascular, metabolic, severe and rare conditions. Isis’ lead partner, Genzyme , is responsible for commercializing Kynamro in the U.S. and other countries for the treatment of homozygous FH. This is currently Isis’ leading product.