AveXis and Its Potential ALS Gene Therapy to Be Purchased by Novartis for $8.7B

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by Grace Frank |

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AveXis, which is running clinical trials of a promising gene therapy for spinal muscular atrophy (SMA) — and developing a potentially similar therapy for a genetic form of amyotrophic lateral sclerosis (ALS) — announced plans to be acquired by Novartis for $8.7 billion in cash.

AVXS-101 is the company’s gene therapy now in five ongoing or soon-to-start Phase 3 clinical trials in infants and children with SMA, including type 1, the disease’s most severe form.

The company’s potential ALS therapy, called AVXS-301, is still in preclinical testing. It is being advanced as a possible treatment for a form of ALS caused by mutations in the superoxide dismutase 1 (SOD1) gene, which accounts for between 15 and 20 percent of all known cases of this disease.

Studies in ALS mouse models found that a one-time administration of AVXS-301 extended survival from 130 days to more than 200 days — the longest lifespan extension seen in this animal model, AveXis executives said in a February earnings conference call reported by MSN.

AVXS-301 also delayed motor impairment and maintained motor function in the treated mice over 200 days, compared to an untreated control group of diseased mice, Brian Kaspar, the company’s chief scientific officer, said in the call. It also showed a good safety profile.

The merger, to be completed by mid-year, met with the unanimous approval of the boards of directors of both companies, AveXis said in a press release.

Novartis’ per-share acquisition price for AveXis stock is $218, a premium of 88 percent on the company’s closing price as of April 6.

“The commitment, drive and expertise of the entire AveXis team has created significant stockholder value, and we are pleased that Novartis recognizes that value in the potential of AVXS-101, our first in class manufacturing capabilities, and our gene therapy pipeline, all of which serve to transform the lives of people devastated by rare and life-threatening neurological diseases such as SMA, Rett syndrome, and genetic ALS,” Sean Nolan, president and CEO of AveXis, said in its release.

“With worldwide reach and extensive resources, Novartis should expedite our shared vision of bringing gene therapy to these patient communities across the globe as quickly and safely as possible,” Nolan added.

As an SMA gene therapy, AVXS-101 is designed to be a safe, one-time treatment with the potential to prevent muscle degeneration throughout a person’s life, Sukumar Nagendran, AveXis’ chief medical officer, said in a February interview with SMA News Today that focused on the treatment and its potential.

SMA children in a Phase 1 trial who were treated with AVXS-101 once — and as long as four years ago — still show no signs of “losing any motor milestones,” Kaspar said in the interview.

“We look forward on the closing of the deal to a smooth transition for AveXis employees and welcoming them to Novartis,” Vas Narasimhan, Novartis’ CEO, said in a separate press release.

Under a 2017 licensing agreement, AveXis obtained worldwide rights to REGENXBIO’s gene-delivery platform, NAV AAV9 vector, to help it develop AVXS-201 and AVXS-301.

The NAV AAV9 vector delivers a healthy gene, placed inside the shell of an adeno-associated virus (AAV), directly to a patient’s cell nucleus.

The agreement follows preclinical studies showing that gene therapy using the NAV AAV9 delivery platform is effective against ALS and Rett syndrome. AveXis plans new studies in these diseases as it continues to advance these potential treatments.