(Nogas1974, Wikimedia Commons)
9 April 2018. A biotechnology enterprise developing gene therapies for rare inherited diseases and neurological disorders is being acquired by pharmaceutical company Novartis AG for $8.7 billion. Stockholders of AveXis Inc. in Chicago are expected to receive $218 per share, 88 percent higher than the stock’s closing price on Friday, 6 April and 72 percent more than the volume-adjusted average for the past 30 days.
AveXis develops treatments for genetic diseases that transfer healthy genes to replace proteins from missing or mutated genes affecting the brain and nervous system. The company’s lead product, code-named AVXS-101, is designed as a therapy for spinal muscular atrophy, an inherited condition in infants where specialized motor nerve cells in spinal cord and brain stem are missing, leading to wasting away of muscles for crawling, walking, sitting up, and head movements. In severe cases, muscles for breathing and swallowing can also be affected.
The company is testing AVXS-101 in clinical trials among children with types 1 and 2 of spinal muscular atrophy. Type 1 of the disease is a severe form of the disease that is evident in a baby’s first few months, which can also cause difficulties in breathing and swallowing, leading to choking, while type 2 usually affects children age 6 to 12 months and can make actions like sitting up or standing difficult. The advocacy organization CureSMA says spinal muscular atrophy affects 1 in 11,000 infants, with about 1 in 50 Americans being genetic carriers of the disorder. The group also says spinal muscular atrophy is the leading genetic cause of death among infants.
AveXis says its technology delivers healthy genes to motor nerve cells to produce proteins needed for proper functioning of those cells. In the case of spinal muscular atrophy, the survival of motor neuron or SMN1 gene is delivered, which provides instructions for producing the SMN protein. The gene is contained in an engineered protein shell called a capsid delivered by an adeno-associated virus, a benign, naturally occurring microbe that infects cells, but does not integrate with the cell’s genome, and generates at most a mild immune response.
This delivery capsid, says the company, is also one of the few that can cross the blood-brain barrier, the tightly-packed cells lining blood vessels that allow nutrients to pass through, but keeping out foreign substances, including most drug molecules. In addition to the healthy gene, the capsid also delivers a complimentary DNA strand to enable immediate coding and production of SMN proteins, as well as engineered enhancer and promoter sequences that allow the delivered gene to continuously produce the SMN proteins needed.
AVXS-101 is currently in a late-stage clinical trial in the U.S. with children having type 1 spinal muscular atrophy, and a similar study in Europe set to begin recruiting patients. Early results from the U.S. trial, reported in November 2017, show all 15 participants were alive and event-free some 20 months after treatment, compared to historical averages of 8 percent.
AveXis says FDA designated AVXS-101 an orphan drug, and assigned the treatments Breakthrough Therapy and Fast Track tags to accelerate their regulatory reviews. The company is also developing gene therapies for Rett syndrome, a genetic disease affecting mainly girls affecting their language and coordination functions between 6 and 18 months of age, and an inherited form of amyotrophic lateral sclerosis, or ALS, also known as Lou Gehrig’s disease.
Novartis says the acquisition of AveXis supports its goal of becoming a leader in neurodegenerative treatments, as well as acquiring AveXis’s manufacturing capabilities of adeno-associated viruses for neurological disorders.
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