Novartis announced that it has entered into an agreement and plan of merger with AveXis, Inc. to acquire the US-based clinical stage gene therapy company for $8.7 billion.
AveXis has several ongoing clinical studies for the treatment of spinal muscular atrophy (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. AVXS-101 has Breakthrough therapy designation in the US, PRIME designation in the EU and Sakigake in Japan; expected US patient availability in 2019. 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.
AveXis also offers state of the art adeno-associated virus serotype 9 (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). AAV9 is considered to be a clinically proven gene delivery platform for diseases of the central nervous system.
The transaction, approved by the boards of both companies, is expected to close in mid 2018.