To transform care in SMA and expand its position as a gene therapy and Neuroscience leader, Novartis is set to acquire AveXis Inc. for $8.7 billion.
AVXS-101, AveXis lead product candidate, has the potential to be the first ever one time gene replacement therapy for spinal muscular atrophy (SMA). SMA is a disease that results in either early death or lifelong disability with considerable healthcare costs.
AVXS-101 expects US patient availability by 2019 and has breakthrough therapy designation in the US and PRIME designation in the EU and Sakigake in Japan.
Acquiring clinical stage gene therapy company for $218 per share for a total of $8.7 billion in cash, Novartis is all set to acquire AveXis Inc. which has been unanimously approved by the Boards of directors of both companies.
Several ongoing clinical studies is being performed by AveXis for SMA and AveXis’s AVXS-101 has highly compelling clinical data in treating SMA Type 1, which is the number one genetic cause of death in infants, where 9 out of 10 infants do not live to their second birthday or are permanently ventilator dependent. Out of every 6000 to 10000 children born 1 is affected by some form of SMA.
According to Vas Narasimhan, CEO of Novartis:
“The proposed acquisition of AveXis offers an extraordinary opportunity to transform the care of SMA. 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. We look forward on the closing of the deal to a smooth transition for AveXis employees and welcoming them to Novartis.”
AVXS-101 would be a first-in-class one-time therapy that addresses the root genetic cause of SMA by effectively replacing the defective SMN1 gene and it has shown lifesaving efficacy with all the 15 infants treated event free at 20 months compared with an event-free survival rate of 8 percent in an historical cohort (NEJM, November 2017). AveXis will also present two year data to the American Academy of Neurology on April 25, 2018.
Paul Hudson, CEO Novartis Pharmaceuticals added the following about the latest investment:
“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. We relish the opportunity to leverage our expertise, our 70-plus year heritage in neuroscience and our global footprint to help AVXS-101 benefit high-need SMA patients around the world.”
The transaction is expected to be completed close to mid-2018 and AveXis also offers other state of the art AAV9 gene therapy manufacturing capabilities and valuable R&D capabilities includes other pipeline products for Rett Syndrome (RTT) and a genetic form of amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene.
Headquartered in Switzerland, Novartis manufactures pharmaceutical and consumer healthcare products. Novartis specializes in pharmaceuticals for cardiovascular, respiratory and infectious diseases, oncology, neuroscience, transplantation, dermatology, gastrointestinal and urinary conditions, arthritis, vaccines and diagnostics, vision, and animal health products.
Founded in 2010, AveXis Inc. is a clinical stage gene therapy company that engages in developing and commercializing treatments for patients suffering from rare and life-threatening neurological genetic diseases. Headquartered in the US, AveXis initial product candidate is AVXS-101, a gene therapy product candidate that has completed Phase I clinical trial for the treatment of spinal muscular atrophy Type 1. Employing around 219 employees, AveXis also intends to identify, acquire, develop, and commercialize gene therapy product candidates for the treatment of other rare and life-threatening neurological genetic diseases.