Drug Development

Biogen, Ionis declare an early victory for PhIII spinal muscular atrophy study, triggering $75M payout

Two months after a clinical hold on a top Phase III program tripped up Ionis and sent its shares into a tailspin, the antisense RNA specialist has bounced back with a late-stage win for spinal muscular atrophy, setting up a $75 million payout from Biogen, which will now race toward regulatory approvals for their drug.

Battered shares of Ionis–which is now contemplating its first new drug approval–raced up 39% on the news, while Biogen clocked a 4% gain.

Ionis $IONS says that they hit the primary endpoint at the interim analysis of a Phase III study that enrolled patients with infantile-onset SMA. Biogen $BIIB will now take control of the therapeutic after handing over the cash as it looks for a boost of its own as CEO George Scangos heads for the exits.

Patients will now be enrolled in an ongoing open-label study, allowing study participants on a placebo to get the drug. And Biogen is working on expanding an open access program for the therapy.

“Results are in, earlier than expected, and nusinersen appears to have done what few other CNS antisense oligos have done before — shown a statistically significant effect on a key functional endpoint,” noted Baird’s Brian Skorney. “This success in treating SMA, the first ever for the most common genetic cause of infant death, is a major achievement.”

Back in May Ionis was sent reeling after it revealed that evidence of thrombocytopenia—severe platelet declines — had derailed one of its late-stage efforts. That makes this success particularly critical. Biogen also has a lot to gain from regulatory approvals, as analysts look for evidence that it can advance important new products into the market as sales of Tecfidera slide.

Ionis CEO Stan Crooke

Ionis CEO Stan Crooke

Infants and young children who suffer from rare cases of SMA don’t live long. Defects in the survival motor neuron 1 gene, a key player in promoting neuromuscular growth, cause them to grow progressively weaker and suffer from a series of harsh events ending in a very early death. But a year ago Ionis CEO Stan Crooke was praising evidence that their antisense RNA approach was working.

“They are living longer, achieving milestones that are simply unheard of,” Crooke told me back then. Five of the Type 1 patients in a study were able to able to sit unassisted. “What we hoped to see (in this study) were good safety data. We never dreamt we’d see this.”

“Nusinersen is the first antisense drug from our neurological disease franchise to advance to regulatory review, and it illustrates the potential of our antisense technology to address severe diseases that other therapeutic modalities are unable to address adequately,” says B. Lynne Parshall, chief operating officer at Ionis Pharmaceuticals.

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Sr. Manager, Regulatory Affairs, CMC
CytomX Therapeutics San Francisco, CA
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