Biogen Presents New ENDEAR Data Showing SPINRAZA (nusinersen) Significantly Reduces Risk of Death or Permanent Ventilation

Biogen presented new data from the Phase 3 ENDEAR study of SPINRAZA™ (nusinersen), which demonstrated a statistically significant reduction in the risk of death or permanent ventilation in SPINRAZA-treated infants with spinal muscular atrophy (SMA), compared to untreated infants. The data was presented at the British Pediatric Neurology Association (BPNA) annual conference in Cambridge, UK, 11-13 January 2017.

In August 2016, Biogen reported that ENDEAR met its pre-specified primary endpoint at the interim analysis, the proportion of motor milestone responders as measured by the Hammersmith Infant Neurological Examination (HINE). Following the positive interim analysis, Biogen ended the study early so that all participants could have the option to receive SPINRAZA in an open-label extension study. Today, Biogen provided the first presentation of the pre-specified primary endpoint, time to death or permanent ventilation, from the end of (EOS) analysis. The EOS results presented at BPNA include data from patients’ final study visit, which occurred after the announcement that the study was being stopped and was not part of the interim analysis.

“Although ENDEAR was stopped early based on positive interim results, the study still demonstrated that a significantly greater number of infants treated with SPINRAZA survived and did not require permanent ventilation. These data further underscore the impact SPINRAZA may have on individuals living with this devastating disease,” said Wildon Farwell, M.D., M.P.H., senior medical director, Clinical Development, Biogen. “We are very encouraged that individuals with SMA have already started treatment with SPINRAZA this week in the U.S., and we continue to work closely with regulatory agencies to bring this therapy to patients around the world as quickly as possible.”

SPINRAZA met the pre-specified primary endpoint at the ENDEAR EOS, demonstrating a statistically significant 47% reduction in the risk of death or permanent ventilation (p

More About Spinraza

On December 23, the FDA announced that it had approved SPINRAZA™ (nusinersen) to treat spinal muscular atrophy, making it the first-ever FDA-approved therapy for SMA.

From 2003 to 2006, Cure SMA provided the very first research funding needed to begin investigation into this therapeutic approach. We thank Drs. Ravindra Singh and Elliot Androphy of the University of Massachusetts Medical School for their work funded by Cure SMA in orginally indentifying the ISSN1 gene sequence, which is the sequence targeted by Spinraza. We acknowledge Drs. Adrian Krainer, Yimin Hua, and colleagues at Cold Spring Harbor Laboratory for generating the critical intellectual property. All this work was then licensed to Ionis Pharmaceuticals to created the antisense therapy, SPINRAZA.

The program was then licensed to Biogen. Together with Ionis, Biogen worked to develop and implement a comprehensive clinical testing program that would provide both the quickets route to approval and the high quality data necessary to support a broad label and access.

Next Steps

Biogen also announced yesterday that the first-ever patient has received the first commerical SPINRAZA dose. This marks the true start of a key stage to ensure that more patients are able to gain access to SPINRAZA.

We are excited that patients are now gaining real commercial access to the first-ever approved therapy for SMA, and know that more work remains to ensure the treatment is broadly available as quickly as possible.

For more information, please visit our Spinraza page.


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