Isis Pharmaceuticals updated the SMA community on phase 2 clinical trial results of the drug ISIS-SMNRx.
Isis Pharmaceuticals, developer of the experimental spinal muscular atrophy (SMA) drug ISIS-SMNRx, revealed positive results from phase 2 clinical studies of the drug focusing on infants and children with SMA. The new data, which was shared on October 10 by ISIS representatives attending the 19th International World Muscle Society Congress in Berlin, included the following highlights:
“We’re greatly encouraged by the data suggesting prolonged survival for infants with SMA, the signs of increased muscle function in both age groups, as well as the absence so far of adverse reactions for the infants and children being tested,” said Valerie A. Cwik, M.D., MDA’s Executive Vice President & Chief Medical and Scientific Officer. “All of these results point strongly to the need to test and develop ISIS-SMNRx further, hopefully culminating in a lifesaving treatment for babies and children with SMA.”
The drug was created using a technology called antisense, a method of altering genetic instructions, with the goal of increasing levels of the needed SMN protein. Although MDA is not directly sponsoring this trial, MDA supported laboratory development of ISIS-SMNRx through a 2007-2010 grant to molecular biologist Adrian Krainer at Cold Spring Harbor (New York) Laboratory.
For more information read: SMA: ISIS-SMNRx Shows Benefit in Infants, Children or ISIS-SMNRx to Be Tested in Phase 3 Trial in Infants With SMA.