At last week’s Annual SMA Conference, Isis Pharmaceuticals, Inc. provided an update on children with spinal muscular atrophy (SMA) who have completed the open-label, Phase 2 multiple-dose study of ISIS-SMNRx and are continuing to receive treatment in an open-label extension (OLE) study. Consistent with earlier observations, increases in muscle function scores and additional motor function tests were observed in children treated with ISIS-SMNRx. Isis is currently collaborating with Biogen to develop and commercialize ISIS-SMNRx to treat patients with SMA.
An analysis on May 15, 2015 at the nine month evaluation in the OLE study showed a mean increase of 3.8 points in HFMSE score (n=22), with 57% of children with SMA achieving increases in HFSME scores of at least three points.
Based on the results shown in this most recent report:
- ISIS-SMNRx is safe and well tolerated in children with SMA type II or III. This means that the clinical trials should continue to move ahead as currently planned.
- The next step for this and the other ongoing trials is to prove the efficacy of SMNRx against a placebo control.
- All signs show that this program should continue to move ahead at a rapid pace.
Cure SMA Seed Funding
From 2003 to 2006, Cure SMA provided the seed funding needed to begin investigation into this therapeutic approach.
Several Cure SMA grants totaling over $500,000 were given to U Massachusetts, to help develop the intellectual property needed to investigate antisense oligonucleotides for SMA. The intellectual property generated with our funding was then licensed to Isis Pharmaceuticals to create ISIS-SMNRx.
Currently, Isis and Biogen are conducting four ongoing trials of ISIS-SMNRx:
- ENDEAR, a Phase 3 clinical trial testing infants with SMA type I.
- CHERISH, a Phase 3 clinical trial testing children age 2-12, with onset of symptoms at age six months or later.
- NURTURE, a Phase 2 clinical trial testing very young infants who are genetically predisposed to SMA but not yet showing symptoms.
- EMBRACE, a Phase 2 clinical trial testing other population groups.
There are currently two ongoing controlled phase 3 studies that must remained focused on to provide concrete proof of efficacy.
It is important to focus on completing the two pivotal studies ENDEAR & CHERISH to advance potential regulatory filings as quickly as possible and, equally important, honor the sacrifice of those participating in sham controlled clinical trials, who are contributing to the future of the entire community of families living with SMA.