Isis Pharmaceuticals announced that follow-up preliminary data from a single dose, open-label Phase 1 study of ISIS-SMNRx in children with spinal muscular atrophy (SMA), show that most SMA children receiving the two highest doses of the drug (6 mg and 9 mg) continued to show improvements in muscle function tests up to 14 months after a single injection of the drug.
The Phase 1 data, including these preliminary follow-on data, will be presented at theInternational Congress of the World Muscle Society by Dr. Kathy Swoboda on Oct. 3 , 2013. SMA is a severe and rare genetic neuromuscular disease characterized by muscle atrophy and weakness and is the most common genetic cause of infant mortality. ISIS-SMNRx is an antisense drug designed to treat all types of SMA.
The preliminary data is from a follow-up analysis of 24 children with SMA who participated in a Phase 1 single-dose, open-label study of ISIS-SMNRx. Analysis of motor function was performed in these children nine to 14 months following a single dose of ISIS-SMNRx using the Hammersmith Functional Motor Scale-Expanded (HFMSE). The improvements in HFSME scores were dose dependent with the largest improvements observed in children in the highest dose cohort (9 mg, mean = 5.75). Most children in the 9 mg dose cohort showed continuing improvements during follow up, with no children declining.
The Phase 1 data, including these preliminary follow-on data, will be presented at the
The preliminary data is from a follow-up analysis of 24 children with SMA who participated in a Phase 1 single-dose, open-label study of ISIS-SMNRx. Analysis of motor function was performed in these children nine to 14 months following a single dose of ISIS-SMNRx using the Hammersmith Functional Motor Scale-Expanded (HFMSE). The improvements in HFSME scores were dose dependent with the largest improvements observed in children in the highest dose cohort (9 mg, mean = 5.75). Most children in the 9 mg dose cohort showed continuing improvements during follow up, with no children declining.
How long before we can actually use it? Estimate?
ReplyDeleteIt would probably take another 5 - 7 years to finish phase II and III and get the approval, even for an orphan disease like SMA.
ReplyDeleteOlesoxime is a bit further along the way in the trials as its phase II results should already be out by the end of this year. The preliminary results published in March were positive.
http://sma-daily.blogspot.com/2013/03/positive-results-for-sma-drug-candidate.html