NURTURE study shows pre-symptomatic Spinal Muscular Atrophy (SMA) infants treated with SPINRAZA™ (nusinersen injection) attained positive results

• Treatment with SPINRAZA™ resulted in survival of 100 percent of infants and none requiring permanent ventilation
• Recently published outcomes include motor milestones achieved in 100 percent of infants sitting without support and 88 percent walking independently

MISSISSAUGA, ON, Oct. 3, 2019 /CNW/ - Biogen Canada is pleased to announce results from the NURTURE study, which demonstrated that infants who initiated treatment with SPINRAZA™ (nusinersen injection) prior to the onset of clinical symptoms of spinal muscular atrophy (SMA) attained positive results compared to the natural history of the disease. These results have been published in the journal Neuromuscular Disorders. NURTURE is the first study investigating a treatment targeting the underlying cause of spinal muscular atrophy (SMA) in infants treated pre-symptomatically.

NURTURE is an ongoing, Phase 2, open-label study of 25 infants with the genetic diagnosis of SMA (most likely to develop SMA Type 1 or 2) who received their first dose of SPINRAZA™ in the pre-symptomatic stage and before six weeks of age. The study, conducted at 15 sites in seven countries, has close to four years of data (up to 45.4 months). When compared with the natural history of the disease, the results are dramatic in their impact on changing the course of SMA. As of March 2019, all participants were alive, without the need for permanent ventilation and experienced continuous improvements, with the majority achieving motor milestones in timelines consistent with normal development. The results also demonstrated durability of effect with children making progress throughout the study. 

"By showing dramatic clinical benefit in very young SMA patients, the NURTURE study throws into sharp relief the new therapeutic SMA era the world has entered," said Ottawa pediatrician and SMA newborn screening advocate, Dr. Alex MacKenzie. "The results make a compelling case for the pre-symptomatic treatment of SMA, underlining the critical importance of newborn screening for all Canadian infants."

As of March 2019, all infants in the study were 25 months or older, past the typical age of symptom onset for SMA Type 1 and Type 2 and were alive without the need for permanent ventilation. In comparison to the natural history of SMA, many of these infants would likely have passed away or require permanent ventilation on average by 13.5 months. In both the children with two and three copies of SMN2, treatment with SPINRAZA™ demonstrated rapid onset of improvement and durability of effect with their mean Children's Hospital of Pennsylvania Infant Test of Neuromuscular Disorders (CHOP-INTEND) score of motor function reaching the maximum mean score of 64 for all participants with three copies of SMN2 (n=10) and a mean of 62.1 for those with two copies of SMN2 (N=15).  

"We are pleased to share this very exciting data with the SMA community in Canada because it adds to our understanding of what optimal disease management with SPINRAZA™ could be with young patients with SMA," said Marina Vasiliou, Vice President and Managing Director of Biogen Canada. "Ultimately what we want for patients is to change the course of their disease which is why we are committed to ongoing research in SMA. This study, and others we are conducting are part of Biogen's commitment to helping Canadians affected by SMA."

Reaction from the SMA community

Susi Vander Wyk, Executive Director, Cure SMA Canada: "SPINRAZA™ is a life-changing medicine and these eye-opening results in infants with SMA has brought hope for the future that these patients never had. SMA babies deserve a chance to meet motor milestones and to function independently as long as they can."

Barbara Stead-Coyle, CEO, Muscular Dystrophy Canada: "We welcome the growing body of evidence with SPINRAZA™ and believe that research like this is critical for the SMA community. With this unparalleled evidence in newborn infants, it is critical that newborn screening for SMA gets adopted in every province in Canada. NURTURE is evidence that shows the sooner the infant is treated, the better the outcomes for the infant and the family."

The Health Canada approval of SPINRAZA™ in June 2017 for pre-symptomatic, Type I, II and III SMA patients regardless of age is based on Biogen's extensive clinical development program for this treatment, including data from over 300 patients across a broad range of SMA populations. Biogen continues to partner with the medical community in Canada and worldwide for the ongoing evaluation of the effectiveness and tolerability of this treatment in SMA.

About Spinal Muscular Atrophy (SMA)

SMA is a rare, debilitating neurodegenerative condition that is characterized by loss of motor neurons in the spinal cord and lower brain stem, resulting in severe and progressive muscular atrophy and weakness.

SMA is the leading genetic cause of death among infants. It is estimated that 1 in 10,000 live births are affected by SMA. Untreated, children with the most severe form of SMA (Type 1 or infantile-onset SMA) rarely live to see their second birthday.

Due to a deletion of, or mutation in, the SMN1 gene, people with SMA do not produce enough survival motor neuron (SMN) protein, which is critical for the maintenance of motor neurons. The severity of SMA correlates with the amount of SMN protein an individual has. Ultimately, individuals with SMA can become paralyzed and have difficulty performing the basic functions of life, like breathing and swallowing.

People with Type I SMA, the form that requires the most intensive and supportive care, produce very little SMN protein and do not achieve the ability to sit without support or typically live beyond two years without respiratory support. People with Type II and Type III SMA produce greater amounts of SMN protein and have less severe, but still life-altering forms of SMA.

About SPINRAZA™ (nusinersen injection)

SPINRAZA™ (nusinersen injection) is the first and only approved treatment in Canada for spinal muscular atrophy (SMA). It is the only SMA therapy with a sustained efficacy and manageable safety profile and is supported by the largest clinical data set and real-world evidence, with more than 8,400 patients treated worldwide for up to nearly six years. Approved in more than 44 countries, it has been shown to improve survival for those with the most severe form of SMA (Type 1 or infantile-onset SMA) and has enabled children, teens and adults with SMA to maintain or improve motor function, transforming the course of the disease.

About Biogen 
At Biogen, our mission is clear: we are pioneers in neuroscience. Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases as well as related therapeutic adjacencies. One of the world's first global biotechnology companies, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Kenneth Murray, and Nobel Prize winners Walter Gilbert and Phillip Sharp, and today has the leading portfolio of medicines to treat multiple sclerosis, has introduced the first approved treatment for spinal muscular atrophy, and is focused on advancing neuroscience research programs in MS and neuroimmunology, Alzheimer's disease and dementia, movement disorders, neuromuscular disorders, acute neurology, neurocognitive disorders, pain, and ophthalmology. Biogen also commercializes biosimilars of advanced biologics.

We routinely post information that may be important to investors on our website at www.biogen.ca . To learn more, please visit www.biogen.ca and follow us on social media – Twitter, LinkedIn, Facebook, YouTube. 

SOURCE Biogen Canada

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