Novartis to acquire AveXis to expand its gene therapy portfolio

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Swiss pharma giant Novartis has entered into an agreement with AveXis to acquire the US-based clinical stage gene therapy company. With its latest acquisition plan, Novartis aims to become a major gene therapy player in the industry.

Novartis will acquire AveXis for USD 218 per share or a total of USD 8.7 billion in cash. The transaction was unanimously approved by the boards of both companies and is expected to close in mid-2018, pending the successful fulfilment of all closing conditions.

AveXis is developing adeno-associated virus (AAV) vector-based gene therapies for neurological genetic diseases. It has several ongoing clinical studies for the treatment of spinal muscular atrophy (SMA), a severe neuromuscular disease characterized by the loss of motor neurons leading to progressive muscle weakness and paralysis. The lead AveXis gene therapy candidate, AVXS-101 is currently being investigated in two trials: a pivotal trial for treating patients with type 1 SMA and a Phase 1 trial in patients with type 2 SMA. If approved, it would be the first gene therapy to treat SMA.

AVXS-101 holds FDA’s Orphan Drug Designation for the treatment of all types of SMA and Breakthrough Therapy Designation and Fast Track Designation for the treatment of 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. It was also granted access to EMA’s PRIority Medicines (PRIME) program and Japan’s Sakigake designation for the treatment of SMA Type 1.

In addition to AVXS-101, Novartis will also gain access to AveXis’ AAV9 gene therapy manufacturing capabilities and R&D capabilities for other pipeline products for Rett Syndrome and a genetic form of amyotrophic lateral sclerosis caused by mutations in the superoxide dismutase 1 (SOD1) gene.

Dr Vasant Narasimhan, Novartis’ new CEO is hopeful that in addition to the company’s CAR-T platform for cancer, the acquisition will enable Novartis to become a major gene therapy player for neurodegenerative diseases and thus broaden its leadership in neuroscience.

Paul Hudson, CEO Novartis Pharmaceuticals commented: “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.”

Source: Novartis enters agreement to acquire AveXis Inc. for USD 8.7 bn to transform care in SMA and expand position as a gene therapy and Neuroscience leader; Press Release