Ionis’ SPINRAZA Recognized with Prestigious Prix Galien Award for Best Biotechnology Product

Highlight Video of CEO Dr. Stanley Crooke Accepting Award Now Posted

In October 2017, the Ionis team, along with partner Biogen, was honored to receive the prestigious Prix Galien USA Award for the Best Biotechnology Product in 2017 for SPINRAZA® (nusinersen), which is approved globally for the treatment of spinal muscular atrophy, or SMA. This award, which recognizes extraordinary achievement in scientific innovation that improves the state of human health, is considered the industry’s equivalent of the Nobel Prize and is the highest accolade for pharmaceutical research and development.

Commenting on the award, Stanley Crooke, CEO of Ionis said, “Along with our colleagues at Biogen, we are honored to receive this prestigious award for SPINRAZA, a medical innovation that has already changed the lives of many patients and families suffering from the devastating consequences of SMA. We believe that SPINRAZA and its potential to help children and adults represents the very best of what our industry can achieve.”

To see a video of Dr. Crooke accepting the prestigious Prix Galien USA Award for the Best Biotechnology Product in 2017 for SPINRAZA click here.

ABOUT SMA
Spinal Muscular Atrophy (SMA) is characterized by loss of motor neurons in the spinal cord and lower brain stem, resulting in severe and progressive muscular atrophy and weakness. Ultimately, individuals with the most severe type of SMA can become paralyzed and have difficulty performing the basic functions of life, like breathing and swallowing.

Due to a loss of, or defect 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. People with Type 1 SMA, the type that requires the most intensive and supportive care, produce very little SMN protein and do not achieve the ability to sit without support or live beyond two years without respiratory support. People with Type 2 and Type 3 produce greater amounts of SMN protein and have less severe, but still life-altering, forms of SMA.

ABOUT SPINRAZA (nusinersen)
SPINRAZA is being developed globally for the treatment of spinal muscular atrophy (SMA).

SPINRAZA is an antisense oligonucleotide (ASO), using Ionis Pharmaceuticals’ proprietary antisense technology, that is designed to treat SMA caused by mutations or deletions in the SMN1 gene located in chromosome 5q that leads to SMN protein deficiency. SPINRAZA alters the splicing of SMN2 pre-mRNA in order to increase production of full-length SMN protein. It was discovered and co-developed by Ionis, a leader in antisense therapeutics, and Biogen. ASOs are short synthetic strings of nucleotides designed to selectively bind to target RNA and regulate gene expression. Through use of this technology, SPINRAZA has the potential to increase the amount of full-length SMN protein in individuals with SMA.

SPINRAZA must be administered via intrathecal injection, which delivers therapies directly to the cerebrospinal fluid (CSF) around the spinal cord, where motor neurons degenerate in patients with SMA due to insufficient levels of SMN protein.

Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals in August 2016 and is now responsible for all development, regulatory and commercialization activities and costs for SPINRAZA. SPINRAZA was first approved by the U.S. Food and Drug Administration (FDA) on December 23, 2016, within three months of regulatory filing. SPINRAZA is also approved in the EU, Japan, Canada, Brazil, and Switzerland. Biogen has also submitted regulatory filings in additional countries and plans to initiate additional filings in other countries.

For complete SPINRAZA U.S. prescribing information please visit www.SPINRAZA.com.