AveXis presents updated STRONG data at WMS demonstrating a higher mean increase in Hammersmith Functional Motor Scale-Expanded (HFMSE) scores among older SMA Type 2 patients following one-tim
- Older patients (= 2 years and < 5 years) achieved a mean increase of 5.9 points from baseline in HFMSE scores, nearly double the clinically meaningful threshold (at a mean duration of follow-up time of 9.3 months)
- Untreated SMA Type 2 patients historically experience declining HFMSE scores over time, will never walk without support and often need a wheelchair.[1] More than 30% of patients with SMA Type 2 will die by age 25[2]
- The primary efficacy endpoint is the ability to stand without support for = 3 seconds
- The secondary efficacy endpoint is the ability to walk independently for = 5 steps, according to the Bayley-III Gross Motor Milestone Scale
- Since treatment, 18 motor milestones were achieved among the 16 patients who received Dose A or Dose B, including two patients who gained the ability to stand independently, one of whom went on to walk alone
- The primary efficacy endpoint is change in HFMSE score from baseline
- The secondary efficacy endpoint is the ability to walk independently for = 5 steps according to the Bayley-III Gross Motor Milestone Scale
- Patients showed a clinically meaningful improvement in motor function, having a mean 5.9-point increase from baseline in HFMSE scores at their most recent visit, at a mean duration of follow-up time of 9.3 months
- In a responder analysis, half of patients (6/12) had a = 3-point increase, which was observed starting at one month post-treatment
- Since treatment, four motor milestones have been achieved among 12 patients in the Dose B group, including one patient who gained the ability to walk with assistance
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References
???????[1]?? Farrar MA, et al.?Ann Neurol.?2017;81(3):355-368. [2]?? Darras BT, Finkel RS Spinal Muscular Atrophy. Chapter 25 - Natural History of Spinal Muscular Atrophy. October 2017. [3]?? Anderton RS and Mastaglia FL.?Expert Rev Neurother. 2015;15(8):895-908. [4]?? National Organization for Rare Disorders (NORD). Spinal Muscular Atrophy.?http://rarediseases.org/rarediseases/spinal-muscular-atrophy/. Accessed?October 9, 2018. [5]?? Finkel RS, et al.?Neurology. 2014;83(9):810-7. [6]?? National Center for Biotechnology Information. Revised Hammersmith Scale for spinal muscular atrophy: A SMA specific clinical outcome assessment tool.?https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5319655/. Accessed October 2, 2019. Novartis Media Relations E-mail:?media.relations@novartis.comEric Althoff Novartis External Communications +1 646 438 4335 (mobile) eric.althoff@novartis.com | Farah Bulsara Speer VP, Corporate Communications, AveXis +1 312 543 2881 (mobile) fSpeer259@avexis.com |