Log In

Forgot Password?

OR

Not a member? Continue as a nonmember.

Become a Member

By becoming a member of the AAN, you can receive exclusive information to help you at every stage of your career. Benefits include:

Join Now See All Benefits

Loading... please wait

Abstract Details

Longer-term Treatment With Nusinersen: Results in Later-onset Spinal Muscular Atrophy From the SHINE Study
General Neurology
P6 - Poster Session 6 (12:00 PM-1:00 PM)
6-007

Several clinical trials have demonstrated a favorable benefit:risk profile for nusinersen and established clinically meaningful efficacy on motor function.

To present results from the SHINE open-label extension study (NCT02594124) for participants with later-onset SMA.

Participants from CS2/12, CHERISH and EMBRACE could enroll in SHINE. Following protocol amendment, all participants transitioned to the Modified Maintenance Dosing Regimen (MMDR; 12mg nusinersen every 4 months). Participants initiated the MMDR at the end of the loading dose period or 120 days after date of last visit. Endpoints were assessed from MMDR Day 1.

83 participants from the CHERISH nusinersen and 42 from the sham-procedure group transitioned to SHINE; 24 transitioned from CS2/12. Using the SHINE 15 October 2018 interim data cut, mean (±SD) Hammersmith Functional Motor Scale-Expanded (HFMSE) score at MMDR Day 1 (for participants with a MMDR Day 240 visit) was 26.0 (11.01) for those who received nusinersen in CHERISH/SHINE (n=61), and 21.2 (7.75) for those randomized to sham-procedure in CHERISH/nusinersen in SHINE (n=36). Mean (±SD) Revised Upper Limb Module (RULM) scores at MMDR Day 1 for these groups were 23.4 (5.54; n=61), and 21.2 (4.31; n=36), respectively. Mean (±SD) HFMSE at MMDR Day 1 (for participants with a MMDR Day 240 visit) was 33.2 (12.26) for those with SMA Type II who transitioned from CS2/12 (n=9) and 56.2 (6.85) for those with SMA Type III (n=13). Mean (±SD) RULM at MMDR Day 1 were 26.4 (4.81; Type II; n=8) and 36.7 (0.58; Type III; n=3), respectively. Data from the 2019 SHINE data cut for these cohorts and those who transitioned from EMBRACE, as well as an updated safety profile, will be presented.

Continued analysis of SHINE data will increase the information available on the long-term safety/tolerability and efficacy of repeated nusinersen doses in patients with later-onset SMA.

Study Supported by: Biogen

Authors/Disclosures
Claudia A. Chiriboga, MD, FAAN (Columbia University)
PRESENTER
Dr. Chiriboga has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Genentec. Dr. Chiriboga has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. The institution of Dr. Chiriboga has received research support from Roche. The institution of Dr. Chiriboga has received research support from Avexis/Novartis. The institution of Dr. Chiriboga has received research support from Biogen. The institution of Dr. Chiriboga has received research support from NIH. The institution of Dr. Chiriboga has received research support from Biohaven. The institution of Dr. Chiriboga has received research support from Genentec. Dr. Chiriboga has received publishing royalties from a publication relating to health care.
Basil T. Darras, MD (Children'S Hosp Boston Harvard Med School) 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.
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.
Jan Kirschner Jan Kirschner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche. Jan Kirschner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Jan Kirschner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Jan Kirschner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. Jan Kirschner has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Genethon. Jan Kirschner has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Jan Kirschner has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Pfizer. The institution of Jan Kirschner has received research support from Biogen. The institution of Jan Kirschner has received research support from Novartis. The institution of Jan Kirschner has received research support from Roche.
Nancy L. Kuntz, MD, FAAN (Ann & Robert H Lurie Children'S Hospital of Chicago) Dr. Kuntz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Dr. Kuntz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Affinia Therapeutics. Dr. Kuntz has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenyx. Dr. Kuntz has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Audentes Therapeutics. Dr. Kuntz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Kuntz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Dr. Kuntz 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. Dr. Kuntz has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Sarepta.
Gyula Acsadi, MD, FAAN (Connecticut Children'S Medical Center/UConn) Dr. Acsadi has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Biogen. Dr. Acsadi has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Avexis. Dr. Acsadi has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Serepta. Dr. Acsadi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Genentech. Dr. Acsadi has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Biogen.
Mar Tulinius Mar Tulinius has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for PTC Therapeutics.
Jacqueline Montes, PT, EdD, NCS (Columbia University Medical Center) Ms. Montes 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. Ms. Montes has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for F. Hoffman LaRoche. Ms. Montes 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. Ms. Montes has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. The institution of Ms. Montes has received research support from NIH/NICHD. The institution of Ms. Montes has received research support from Muscular Dystrophy Association. The institution of Ms. Montes has received research support from Cure SMA.
Giulia Gambino Giulia Gambino has nothing to disclose.
Richard Foster Richard Foster has received personal compensation for serving as an employee of Biogen.
Ishir Bhan No disclosure on file
Janice Chun Yee Wong, MD Dr. Wong has received personal compensation for serving as an employee of Janssen Research & Development. Dr. Wong has received personal compensation for serving as an employee of Biogen. Dr. Wong has stock in Biogen. Dr. Wong has stock in Janssen Research & Development.
Boris Kandinov No disclosure on file
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.