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Spinal Muscular Atrophy Drug Successfully Meets Primary Endpoint in Interim Analysis of Phase 3 Study for Infants with SMA

MDA funded foundational research and contributed to early-stage development of nusinersen

Aug. 1, 2016


In a joint statement today, Biogen and Ionis Pharmaceuticals announced encouraging interim results from the late-stage ENDEAR clinical trial to test the experimental drug nusinersen in infants with type 1 spinal muscular atrophy (SMA). Based on the results, investigators have stopped the trial to allow all participants to transition into an open-label trial called SHINE, in which all infants will be treated with the drug.

In addition, Biogen plans to file in the coming months for regulatory approval for nusinersen in the United States and the European Union, and is working to put in place a global expanded access program (EAP) for eligible patients with infantile-onset SMA in the coming months.

The Muscular Dystrophy Association has funded foundational work in SMA, and invested nearly $750,000 toward early-stage development of nusinersen via a grant to Adrian Krainer, Ph.D. at Cold Spring Harbor Laboratory in New York. Since its inception, MDA has invested more than $45 million in SMA research.

Statement from MDA Scientific Program Officer Amanda Haidet-Phillips, Ph.D.: 

“Today marks an important day for the Spinal Muscular Atrophy (SMA) community in our collective pursuit of a meaningful treatment for this life-threatening disease. These encouraging results are an important step, not only in making nusinersen available as quickly as possible as an approved therapy for infants with type 1 SMA, but also in allowing eligible SMA infants not currently participating in the ongoing ENDEAR clinical trial to receive nusinersen this fall.

MDA is proud to have been part of early-stage research that helped lay the groundwork for this development that’s brought us to this meaningful moment and closer to our ultimate goal of helping all those with SMA live longer and grow stronger.”

Statement from Adrian R. Krainer, Ph.D.

“The latest news from the ENDEAR trial are extremely encouraging, and it’s very heartening that the infants in the trials who were getting sham injections can now be administered the actual drug. In addition, many new patients will very soon have access to nusinersen through an expanded-access program. I remain extremely grateful to MDA for their generous support for the research in my lab at Cold Spring Harbor Laboratory; this crucial funding, from 2007-2010, made possible the preclinical development of nusinersen, in a very productive collaboration with Ionis Pharmaceuticals.”

* (Dr. Krainer is an MDA Grantee, St. Giles Professor of molecular genetics and Program Chair of Cancer & Molecular Biology, Cold Spring Harbor Laboratory in Cold Spring Harbor, NY.)

For more information on today’s news:

About MDA
MDA is leading the fight to free individuals — and the families who love them — from the harm of muscular dystrophy, ALS and related muscle-debilitating diseases that take away physical strength, independence and life. We use our collective strength to help kids and adults live longer and grow stronger by finding research breakthroughs across diseases; caring for individuals from day one; and empowering families with services and support in hometowns across America.

Learn how you can fund cures, find care and champion the cause at mda.org.

Contact: Kelli Park | MDA Manager, Public Relations | 312.260.5923 | kpark@mdausa.org