Novartis and AveXis Merge Spells Opportunity for SMA Research

Today, it was announced that Novartis and AveXis, Inc have entered into a plan and agreement of merger so that Novartis may acquire the US-based Nasdaq-listed clinical stage gene therapy company for $218 per share or a total of $8.7 billion in cash. The boards of both companies unanimously approved the transaction.

AveXis has several ongoing clinical studies for the treatment of spinal muscular atrophy (SMA); the company’s lead product is AVXS-101.

SMA is a genetic neurodegenerative disease caused by a defect in a single gene, the survival motor neuron (SMN1). Approximately 9 out of 10 infants with SMA Type 1 do not live to see their second birthdays, and those who do are permanently ventilator dependent.

AVXS-101 is a non-replicating recombinant AAV9 containing the complimentary deoxyribonucleic acid (cDNA) of the human SMN gene under the control of the cytomegalovirus (CMV) enhancer/chicken-β-actin-hybrid promoter (CB). The AAV inverted terminal repeat (ITR) has been modified to promote intramolecular annealing of the transgene, thus forming a double-stranded transgene ready for transcription.²

CEO of Novartis, Vas Narasimhan, 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.”¹

“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."

While the Phase 3 study has only recently commenced (September 2017) and is not due to reach completion until March 31, 2020, AVXS-10 currently has highly compelling clinical data in treating SMA Type 1. In its Phase 1 clinical study, AVXS-10 proved to have life-saving efficacy in al 15 participating infants that were treated event free at 20 months. (This is in comparison to an event-free survival rate of 8 percent in an historical cohort.) On April 25, 2018, AveXis will present its two-year data to the American Academy of Neurology.

If the drug is approved, it will be a first-in-class one-time therapy that will address the root genetic cause of SMA through its effective replacement of the defective SMN1 gene.

In addition, the US Food and Drug Administration (FDA) has previously 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.

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References:

1. 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. GlobeNewswire. 9, Apr. 2018.
2. Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 (STR1VE). National Institute of Health. 28, Feb. 2018.