SMA: Further Data on ISIS-SMNRx Shows Promising Results

Isis Pharmaceuticals updated the SMA community on phase 2 clinical trial results of the drug ISIS-SMNRx.

Article Highlights:
  • ISIS-SMNRx is a drug focused on infants and children with SMA that uses a method of altering genetic instructions to increase levels of the needed SMN protein.
  • Babies taking ISIS-SMNRx in either 6 mg or 12 mg doses experienced increases in muscle function and fewer negative outcomes, while children being tested on multiple dosages showed increases in muscle function.
  • Further study to establish safety and tolerability is needed. One phase 3 study is already underway, and another will begin later this year.
by Phil Ivory on October 17, 2014 - 8:00am

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:

  • Babies taking ISIS-SMNRx in either 6 mg or 12 mg doses experienced fewer negative outcomes, including early death or the need for permanent ventilation as compared to an untreated population in a separate natural history study. Motor assessment testing also showed increases in muscle function for infants on the drug.
  • Older children being tested on multiple dosages of ISIS-SMNRx showed increases in muscle function compared to baseline data.
  • While no serious adverse reactions have been observed in either age group due to the drug, further study to establish safety and tolerability is needed.
  • A phase 3 study called ENDEAR focusing on infants is already underway. Another phase 3 study called CHERISH will target children with SMA and begin later this year.

“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.

Your rating: None Average: 5 (3 votes)
MDA cannot respond to questions asked in the comments field. For help with questions, contact your local MDA office or clinic or email publications@mdausa.org. See comment policy