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Abstract Details

Safety Profile of Nusinersen in Presymptomatic and Infantile-Onset Spinal Muscular Atrophy (SMA): Interim Results From the NURTURE and ENDEAR-SHINE Studies
General Neurology
P6 - Poster Session 6 (12:00 PM-1:00 PM)
6-010

Several clinical trials have demonstrated a favorable benefit:risk profile for nusinersen.
 

To report the safety profile of nusinersen using interim data from the ongoing NURTURE (NCT02386553) and ENDEAR-SHINE (NCT02594124) studies.

NURTURE participants were ≤6 weeks at first dose, clinically presymptomatic, and genetically diagnosed with SMA. ENDEAR participants were ≤7 months at screening and had symptomatic infantile-onset SMA, and following completion of ENDEAR were eligible to transition to the SHINE open-label extension study. Nusinersen 12 mg was administered every 4 months.

At the 29 March 2019 data cut, the median (range) time on study in NURTURE was 33.9 (25.3–45.1) months. The adverse events (AEs) with the greatest incidence in NURTURE were: pyrexia, 21/25 (84%); upper respiratory tract infection, 19/25 (76%); cough, 13/25 (52%); nasopharyngitis, 13/25 (52%); and vomiting, 10/25 (40%). As of 15 October 2018, nusinersen-treated ENDEAR-SHINE participants had been on study (ENDEAR and SHINE) for a median (range) of 997.5 (6–1517) days. The most common AEs during SHINE in participants continuing nusinersen treatment from ENDEAR were: pyrexia, 43/65 (66%); upper respiratory tract infection, 25/65 (38%); pneumonia, 20/65 (31%); nasopharyngitis, 19/65 (29%); and ear infection, 17/65 (26%). There were no clinically relevant trends relating to thrombocytopenia or proteinuria in either study cohort, nor any cases meeting Hy’s Law criteria. There were no cases of hydrocephalus in either population.

After up to ~3.75 years of treatment in NURTURE and ~4 years in SHINE, safety findings were consistent with those reported previously. AEs associated with other antisense oligonucleotides (thrombocytopenia and proteinuria) were low in NURTURE and ENDEAR-SHINE, with no clinically relevant trends, and there were no safety concerns relating to liver function.

Study Supported by: Biogen

Authors/Disclosures
Basil T. Darras, MD (Children'S Hosp Boston Harvard Med School)
PRESENTER
The institution of Dr. Darras has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amicus. Dr. Darras has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Amicus. The institution of Dr. Darras has received research support from National Institutes of Health/National Institute of Neurological Disorders and Stroke,. The institution of Dr. Darras has received research support from Slaney Family Fund for SMA. The institution of Dr. Darras has received research support from Spinal Muscular Atrophy Foundation. The institution of Dr. Darras has received research support from CureSMA. The institution of Dr. Darras has received research support from Working on Walking Fund . The institution of Dr. Darras has received research support from CHERISH, CS2/CS12 . The institution of Dr. Darras has received research support from Biogen for CS11. The institution of Dr. Darras has received research support from AveXis. The institution of Dr. Darras has received research support from Sarepta Pharmaceuticals. The institution of Dr. Darras has received research support from PTC Therapeutics. The institution of Dr. Darras has received research support from Roche. The institution of Dr. Darras has received research support from Santhera. The institution of Dr. Darras has received research support from Scholar Rock. The institution of Dr. Darras has received research support from Fibrogen. The institution of Dr. Darras has received research support from Summit. Dr. Darras has received publishing royalties from a publication relating to health care. Dr. Darras has received publishing royalties from a publication relating to health care.
Darryl C. De Vivo, MD, FAAN (Columbia University) Dr. De Vivo has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen and Novartis. Dr. De Vivo has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Aspa Therapeutics.
Michelle A. Farrar Michelle A. Farrar has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Michelle A. Farrar has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Michelle A. Farrar has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Michelle A. Farrar has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Biogen. Michelle A. Farrar has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche.
Eugenio Mercuri Eugenio Mercuri has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta, Santhera, PTC, Pfizer, Roche, Biogen, Avexis Novartis, Scholar Rock, Cytokinetics. Eugenio Mercuri has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Sarepta, Santhera, PTC, Pfizer, Roche, Biogen, Avexis Novartis. The institution of Eugenio Mercuri has received research support from biogen.
Richard S. Finkel, MD, FAAN (St. Jude Children's Research Hospital) Dr. Finkel has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for AveXis. Dr. Finkel has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Finkel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Catabasis. Dr. Finkel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Capricor. Dr. Finkel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for ReveraGen. Dr. Finkel has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Dr. Finkel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Scholar Rock. Dr. Finkel has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Elsevier. The institution of Dr. Finkel has received research support from AveXis. The institution of Dr. Finkel has received research support from Biogen. The institution of Dr. Finkel has received research support from Capricor. The institution of Dr. Finkel has received research support from Catabasis. The institution of Dr. Finkel has received research support from ReveraGen. The institution of Dr. Finkel has received research support from Roche. The institution of Dr. Finkel has received research support from Scholar Rock. Dr. Finkel has received intellectual property interests from a discovery or technology relating to health care. An immediate family member of Dr. Finkel has received intellectual property interests from a discovery or technology relating to health care. Dr. Finkel has received personal compensation in the range of $0-$499 for serving as a Speaker in workshop with National Academy of Sciences. Dr. Finkel has a non-compensated relationship as a advisor with n-Lorem Foundation that is relevant to AAN interests or activities. Dr. Finkel has a non-compensated relationship as a Board Member with EveryLife Foundation that is relevant to AAN interests or activities.
Richard Foster Richard Foster has received personal compensation for serving as an employee of Biogen.
Wildon Farwell, MD Dr. Farwell has received personal compensation for serving as an employee of Biogen. Dr. Farwell has received stock or an ownership interest from Biogen.
Ishir Bhan No disclosure on file