A number of important discoveries, therapeutic developments, and events related to spinal muscular atrophy (SMA) were reported daily by SMA News Today throughout 2016. Now that the year is over, it is time to briefly review the articles that appealed most to our readers. Here are the 10 most-read articles of 2016, with a brief description of what made them interesting and relevant to SMA patients, family members, and caregivers.
Several companies presented progress reports on six treatment options for SMA at the 2016 Annual SMA Conference, in Anaheim, CA. Four of the six treatment programs — AVXS-101, nusinersen, RG7800/RG7916, and LMI070 — target the genetic cause of SMA. Other options for treatments that improve muscle performance and protect nerve cells were also presented.
Biogen and Ionis Pharmaceuticals issued a series of updates on the clinical development of nusinersen, including completion of the enrollment goals for both Phase 3 trials starting at the time, CHERISH (NCT02292537) and ENDEAR (NCT02865109), as well as a Phase 2 study called EMBRACE (NCT02462759).
Cure SMA released an update on its SMA drug pipeline that included increased coverage of potential treatment approaches to SMA. After this update, the drug pipeline included 18 active programs, 14 pharmaceutical partners, six programs in clinical trials, and 28 programs in total, including 10 failures.
Roche announced that a new medicine for the treatment of SMA, RG7916, would soon advance to its first clinical trial, to investigate its safety, tolerability and properties in healthy individuals. RG7916 is an SMN2 splicing modifier, meaning it targets the SMN2 gene.
Biogen and Ionis Pharmaceuticals reported that nusinersen met the primary endpoints for an interim analysis of its Phase 3 ENDEAR clinical trial, evaluating the therapy as a treatment for infantile-onset SMA (consistent with Type 1 SMA). Results from the interim analysis showed that infants treated with nusinersen experienced a statistically significant improvement in the achievement of motor milestones in comparison with those given placebo. The safety profile of the treatment was considered acceptable.
At the 2016 World Muscle Society Congress, Biogen and Ionis Pharmaceuticals presented important data from clinical trials for their co-developed therapy candidate, nusinersen, for the treatment of SMA. Nusinersen showed potential to increase the amount of functional SMN protein in infants and children with SMA.
AveXis completed patient enrollment for its AVXS-101 Phase 1 clinical trial, evaluating the safety, effectiveness and tolerability in treating SMA Type 1. After AveXis presented positive interim data for AVXS-101, the U.S. FDA granted the candidate its Breakthrough Therapy Designation.