Novartis has entered an agreement to acquire AveXis Inc in an $8.7 billion deal. Under the terms, the Swiss drug maker will gain AveXis’ gene therapy platform and pipeline treatments for spinal muscular atrophy (SMA).
The deal, which will be funded through available cash and short-term borrowing, is expected to be completed in mid-2018.
AveXis is a US-based, Nasdaq-listed gene therapy company with a number of ongoing clinical trials for SMA treatments. Its gene therapy platform, AAV9, has been proven to effectively assist in the treatment of neurological disorders.
AveXis’s lead product candidate AVXS-101 will be adopted by Novartis. AVXS-101 has so far given promising clinical data in the treatment of SMA Type 1, the most common genetic cause of death in infants. The condition causes death or permanent ventilator dependence in nine out of 10 children below the age of two, and estimates indicate that one out of every 6,000 to 10,000 children born is affected by some form of SMA.
If approved, AVXS-101 could be the first one-time gene replacement therapy for SMA. It works by replacing the defective survival motor neuron (SMN1) in patients with SMA, targeting the root genetic cause of the condition.
A clinical study in November 2017 demonstrated the therapy’s life-saving efficacy, with the 15 infants enrolled being event-free at 20 months compared with an event-free survival rate of 8% in an historical cohort. Findings from the study were published in The New England Journal of Medicine.
AVXS-101 has already received Orphan Drug designation from the US Food and Drug Administration (FDA) for the treatment of SMA, as well as Breakthrough Therapy designation for SMA Type 1. PRIME and Sakigake designations have also been granted in Europe and Japan, respectively.
A Biologic License Application (BLA) filing with the FDA for AVXS-101 is expected in the latter half of 2018, with its launch in the US anticipated in 2019. AveXis is also due to present its two-year data to the American Academy of Neurology at the end of this month.
“The proposed acquisition of AveXis offers an extraordinary opportunity to transform the care of SMA,” Novartis CEO Vas Narasimhan said.
“We believe AVXS-101 could create a lifetime of possibilities for the children and families impacted by this devastating condition.
“The acquisition would also accelerate our strategy to pursue high-efficacy, first-in-class therapies and broaden our leadership in neuroscience. We would gain with the team at AveXis another gene therapy platform, in addition to our CAR-T platform for cancer, to advance a growing pipeline of gene therapies across therapeutic areas."
The agreement marks Novartis’s second large gene therapy deal this year, following its acquisition of the rights to Spark Therapeutic’s drug Luxturna—a treatment for blindness—outside of the US. The drugmaker paid an initial installment of $105 million, with a further $65 million in milestone payments.
Novartis’s acquisition of the AveXis platform will help to secure its position as a forerunner in the burgeoning field of gene therapy and neuroscience.
"Bringing AveXis on board would support both our ambition to be a leader in neurodegenerative diseases and our Neuroscience franchise priorities to strengthen our position in devastating pediatric neurological diseases such as SMA,” Novartis Pharmaceuticals CEO Paul Hudson said.
SMA is a genetic neurodegenerative disease caused by a defect in SMN1. It causes a weakening of muscles and impairs motor skills. Typical symptoms include twitching muscles, joint problems such as scoliosis, breathing difficulties and problems swallowing. There are currently no treatment options for the condition, though often exercises and equipment are used to help movement and breathing, with surgery as a potential option for spine and joint problems.
AveXis has a number of other gene therapy candidates in its pipeline, including one for Rett Syndrome and a genetic form of amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene.