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Biogen plays up spinal muscular atrophy pipeline

By Brian Buntz | March 14, 2022

BiogenAs Biogen Inc. (Nasdaq: BIIB) continues to struggle to capitalize on the Alzheimer’s drug Aduhelm (aducanumab), the company is highlighting new data from its Spinraza (nusinersen) and spinal muscular atrophy (SMA) research program.

First approved in 2016, Spinraza is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide indicated to treat spinal muscular atrophy (SMA).

At the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference held March 13-16, 2022), Biogen will describe the Phase 3b ASCEND trial design. The company recently announced that it had enrolled the first patient in ASCEND and continues to enroll participants in that study.

Biogen is also sharing data from the Phase 4 RESPOND trial, which is testing the safety and efficacy of Spinraza in infants and toddlers with unmet clinical needs after receiving Zolgensma (onasemnogene abeparvovec).

The company also will provide an update from the Phase 2 NURTURE trial, which focuses on infants treated in the presymptomatic stage of SMA. The study that early and sustained Spinraza therapy for up to 5.7 years helped participants maintain and make progressive improvements in motor function.

“Spinraza has demonstrated significant benefit in individuals with SMA, from presymptomatic infants to adults with later-onset SMA. Biogen is working to address remaining unmet needs and answer critical questions for the SMA community through our new and ongoing research, including studies like ASCEND and RESPOND, as well as digital solutions to advance clinical care and patient empowerment,” said Dr. Maha Radhakrishnan, chief medical officer at Biogen, in a statement.

Biogen shares dropped almost 2% today to close at $193.77.


Filed Under: Neurological Disease
Tagged With: Aduhelm, Biogen, SMA, spinal muscular atrophy, Spinraza
 

About The Author

Brian Buntz

As the pharma and biotech editor at WTWH Media, Brian has almost two decades of experience in B2B media, with a focus on healthcare and technology. While he has long maintained a keen interest in AI, more recently Brian has made making data analysis a central focus, and is exploring tools ranging from NLP and clustering to predictive analytics.

Throughout his 18-year tenure, Brian has covered an array of life science topics, including clinical trials, medical devices, and drug discovery and development. Prior to WTWH, he held the title of content director at Informa, where he focused on topics such as connected devices, cybersecurity, AI and Industry 4.0. A dedicated decade at UBM saw Brian providing in-depth coverage of the medical device sector. Engage with Brian on LinkedIn or drop him an email at [email protected].

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