SMA Day: AveXis Gene Therapy Creeps Forward as Biogen Drug’s Sales Slow

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the magnitude of nusinersen’s benefit nor its long-term impact on patients is clear. And it comes with a $750,000 first-year list price followed by a $375,000 price tag each following year.

On Tuesday, Biogen updated results from a long-term nusinersen study called SHINE. This study includes patients who had first gotten a placebo in an earlier trial, ENDEAR, as well as those in that trial who first got—and have since stayed on—nusinersen. Biogen reported interim results from 89 patients with what it calls infantile-onset SMA, “most likely to develop Type 1.”

Both groups had seen benefits as of June 30, 2017, Biogen reported. In ENDEAR, placebo patients died or needed a ventilator after a median of 22.6 weeks. By comparison, the median time to death or needing a ventilator for those who started on nusinersen in ENDEAR and have continued on the drug is 73 weeks. The patients that started on placebo and transitioned to nusinersen—and hadn’t yet needed a ventilator—were event-free for a median of 9.2 months.

Separately, Biogen also reported that nusinersen sales, which were Biogen’s main source of growth last year, were $188 million in the U.S. for the first quarter. Those numbers were 14 percent lower than the same quarter last year and missed consensus analyst estimates by 14 percent. The biggest reason: a decline in the number of new patients starting up treatment. Jefferies analyst Brian Abrahams dubbed the disclosure “somewhat concerning,” given Biogen started by getting the most urgent patients on drug and is now moving to more “challenging areas,” like adults whose more moderate forms of SMA may make it more challenging to get reimbursement from insurers.

Biogen bought full nusinersen rights from San Diego-based Ionis Pharmaceuticals (NASDAQ: IONS) in 2016, just before the FDA approved the drug. It has been pleased enough with the results to expand its Ionis neurology partnership earlier this week with a new 10-year, $1 billion deal.

Biogen R&D chief Michael Ehlers told Xconomy last year that the company wanted to pursue more RNA-interference drugs like nusinersen, which interrupt the production of disease-causing proteins, to address more neurological diseases that other drug-making methods can’t touch. But an advance of one-time gene therapies, like AVXS-101, could upend that strategy.

The Novartis acquisition of AveXis is expected to close mid-year. If all goes well, Novartis expects to begin selling AVXS-101 in the U.S. in 2019. It would become a high-profile test case of competition among rare disease drugs and its effect on prices. Unlike nusinersen, AVXS-101 is a gene therapy that could provide long-lasting, if not permanent effects with a single treatment.

Alex Lash contributed to this report.

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