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B.06 Safety and efficacy of nusinersen in infants/children with spinal muscular atrophy (SMA): part 1 of the phase 2 EMBRACE study

Published online by Cambridge University Press:  27 June 2018

PB Shieh
Affiliation:
(Los Angeles)
G Acsadi
Affiliation:
(Hartford)
W Mueller-Felber
Affiliation:
(Munich)
TO Crawford
Affiliation:
(Baltimore)
R Richardson
Affiliation:
(St. Paul)
N Natarajan
Affiliation:
(Seattle)
D Castro
Affiliation:
(Dallas)
S Gheuens
Affiliation:
(Cambridge)
I Bhan
Affiliation:
(Cambridge)
G Gambino
Affiliation:
(Maidenhead)
P Sun
Affiliation:
(Cambridge)
W Farwell
Affiliation:
(Cambridge)
SP Reyna
Affiliation:
(Cambridge)
J Vajsar
Affiliation:
(Toronto)
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Abstract

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Background: EMBRACE (NCT02462759) Part 1 is a randomized, double-blind, sham-procedure controlled study assessing safety/tolerability of intrathecal nusinersen (12-mg equivalent dose) in symptomatic infants/children with SMA who were not eligible to participate in ENDEAR or CHERISH. Methods: Eligible participants had onset of SMA symptoms at ≤6 months with 3 SMN2 copies; onset at ≤6 months, age >7 months and 2 copies; or onset at >6 months, age ≤18 months, and 2/3 copies. Safety/tolerability was the primary endpoint. Exploratory endpoints included Hammersmith Infant Neurological Examination Section 2 (HINE-2) motor milestone attainment, change in ventilator use, and growth. Results: EMBRACE Part 1 was terminated early based on positive results from ENDEAR. Safety/tolerability was similar to previous trials. More nusinersen-treated (11/14;79%) vs. sham–treated individuals (2/7;29%) were HINE-2 motor milestone responders. Between Day 183 and 302, mean (SD) hours of ventilator use changed by +1.236 (3.712) hours in nusinersen-treated (n=12) and +2.123 (3.023) hours in sham–treated individuals (n=7). Similar increases in weight and body length were observed in nusinersen-treated and sham–treated individuals by Day 183. Conclusions: In EMBRACE Part 1, nusinersen demonstrated a favorable benefit-risk profile. These results add to the aggregated efficacy, safety/tolerability data of nusinersen in SMA.

Study Supported by: Ionis and Biogen

Type
PLATFORM PRESENTATIONS
Copyright
© The Canadian Journal of Neurological Sciences Inc. 2018