09 Apr 2018 --- Novartis has entered into an agreement and plan of merger with AveXis, Inc. to acquire the US-based Nasdaq-listed clinical-stage gene therapy company for US$8.7 billion in cash (US$218 per share). The transaction was unanimously approved by the Boards of both companies. The move comes just days after Novartis agreed to sell its stake in a consumer healthcare joint venture for $13 billion to GlaxoSmithKline.
AveXis has several ongoing clinical studies for the treatment of SMA, an inherited neurodegenerative disease caused by a defect in a single gene, the survival motor neuron (SMN1). The lead AveXis gene therapy candidate, 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. It is estimated that one out of every 6,000-10,000 children born is affected by some form of SMA.
Vas Narasimhan, CEO of Novartis, said: "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 to the closing of the deal to a smooth transition for AveXis employees and welcoming them to Novartis."
The US Food and Drug Administration (FDA) has granted AVXS-101 Orphan Drug designation for the treatment of SMA as well as Breakthrough Therapy designation for SMA Type 1. A BLA filing with the FDA for AVXS-101 is expected in the second half of 2018 and approval and launch in the US is expected in 2019. PRIME and Sakigake designations have been secured in Europe and Japan, respectively.
If approved, 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. In a clinical study, AVXS-101 showed life-saving efficacy, with all 15 infants treated event-free at 20 months compared with an event-free survival rate of 8 percent in a 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, said: "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."
AveXis also offers state of the art AAV9 gene therapy manufacturing capabilities and valuable R&D capabilities, which in addition to AVXS-101, 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. AAV9 is considered to be a clinically proven gene delivery platform for diseases of the central nervous system (CNS).
Assuming mid-2018 completion, the acquisition impact would be slightly negative to Core Operating Income in 2018 and 2019, mainly due to R&D investments. As of 2020, Novartis would expect the acquisition impact to strongly contribute to Core Operating Income and Core EPS accretion driven by a significant increase in sales.
The transaction is expected to close in mid-2018, pending the successful fulfillment of the tender offer and all other closing conditions. On completion, Novartis said that it plans a smooth transition of operations and “the integration of AveXis' talented and dedicated employees to continue the mission of bringing AVXS-101 to patients worldwide.”