Isis Pharmaceuticals Reports Data On Spinal Muscular Atrophy

Isis Pharmaceuticals, Inc.  recently revealed its latest data on the Phase 2, open-label and multiple-dose study of ISIS-SMN_Rx in children diagnosed with spinal muscular atrophy (SMA) who are still receiving treatment in an open-label extension (OLE) study.

Muscle function scores and motor function tests were preformed in children receiving ISIS-SMN_Rx treatment, and data proved consistent with previous clinical information. Isis is collaborating with Biogen to advance and market ISIS-SMN_Rx to address those with SMA.

Darryl De Vivo, who is a Professor of Neurology and Pediatrics, from the Columbia University Medical Center, stated, “The natural course for children with untreated type II or type III SMA typically experience loss of muscle function that develops slowly and continually over time, a sustained increase of three or more points in HMFSE scores represents a significant departure from the natural course and is unusual for these children.”

The OLE study enrolled 30 children who suffered with Type II or Type III SMA and were administered with 12 mg of ISIS-SMN_Rx dosed intrathecally every 6 months. Children who enrolled the OLE study had concluded the open-label Phase 2 study of ISIS-SMN_Rx and were treated with multiple doses of 3, 6, 9 or 12 mg of ISIS-SMN_Rx.

Clinical endpoints were assessed every 3 months along with the Phase 2 baseline scores for each patient, such as measurements of muscle function (using the Hammersmith Functional Motor Scale-Expanded test), the 6 minute walk test (6MWT) for ambulatory patients, and the upper limb module (ULM) test for non-ambulatory patients.

The results from this trial revealed continued and durable increases in muscle function. “In these studies using multiple measurements of muscle and motor function changes, we observed encouraging results that were consistent with earlier results from our open-label Phase 2 study.  Taken together, these data suggest that ISIS-SMN_Rx could provide benefit to patients with SMA beyond halting their disease progression. The safety and tolerability profile that we have observed across all of our ISIS-SMN_Rx studies support the ongoing Phase 3 programs in both infants and children with SMA,” said C. Frank Bennett from Isis Pharmaceuticals.