Isis Pharmaceuticals Announces Results From Phase 2 Study of ISIS-SMNRx In Infants With SMA Type I

Isis Pharmaceuticals, a Ribonucleic acid-targeted (RNA-targeted) drug discovery and development company, recently updated the results from an ongoing open-label Phase 2 trial of ISIS-SMNRx in infants with a diagnosis of spinal muscular atrophy (SMA) type I.

The company had previously reported results from this trial which included assessments of muscle function, event-free survival and evaluations of developmental milestones.

Results from ISIS-SMNRx tolerability and safety profile further support the drug development. The company is working alongside Biogen to develop and commercialize ISIS-SMNRx as a treatment option for SMA patients.

During the study, 20 infants with a diagnosis of SMA received treatment of either 6 mg or 12 mg of ISIS-SMNRx. The study efficacy endpoints were set as event-free survival (time to permanent ventilation or death); CHOP-INTEND motor function results; and evaluations of developmental milestones.

The available trial results showed the drug is well tolerated and safe in SMA Type 1 infants, which means other studies can continue as expected. The subsequent step for this ongoing study is to effectively demonstrate SMNRx’s efficacy when compared to a placebo control.

Between 2003 and 2006, Cure SMA provided the seed funding required to begin research on this treatment strategy.

Ongoing Trials

Isis and Biogen are currently working on four different ongoing trials of ISIS-SMNRx. These include:

• ENDEAR, a Phase 3 clinical trial testing infants with SMA type I.
• CHERISH, a Phase 3 study assessing children aged between 2 and 12 years, with  symptom’s onset at age six months or later.
• NURTURE, a Phase 2 study assessing very young infants who are genetically predisposed to SMA but not yet showing symptoms.
• EMBRACE, a Phase 2 study assessing other population groups.

There are currently two ongoing controlled phase 3 trials to determine the drug efficacy. During the SMA Annual Conference, Isis will be providing additional updates and information on these studies.