Biogen has announced interim results from a small Phase 1 study of its ASO therapy, salanersen, in spinal muscular atrophy (SMA). Salanersen was licensed from Ionis.
The results are so good that Biogen is planning to move straight to a registrational trial.
Twenty-four children aged 5 months to 12 years, who had previously been treated with Novartis’ gene therapy, Zolgensma, were administered either a 40mg or 80mg once-a-year dose of salanersen.
There were no serious adverse events linked to the treatment.
A mean reduction of 70% in Nfl – a biomarker of neurodegeneration – was seen at six months and this reduction was sustained at one year.
Further, half of the 40mg subgroup for whom one year of data was available achieved motor function milestones that they had previously not achieved, despite receiving gene therapy. These included walking, crawling, standing or sitting. The Principal Investigator of the trial commented
“To see a child dosed with gene therapy at one year of age and still unable to sit without support at age five then gain the ability to sit independently just three months after initiating salanersen—that is unexpected.”
Biogen is hoping to position salanersen as a more potent and less-frequently-dosed follow-up drug for its blockbuster ASO therapy for SMA, Spinraza.
Spinraza, a quarterly-dosed treatment was approved by the FDA in 2016, and reached peak sales of US$2.1 m in 2021. Since then, sales have come under pressure from the gene therapy, Zolgensma, first approved by the FDA in 2019, and Roche’s oral therapy, Evrysdi, which was first approved by the FDA in 2020.
I view these latest results as broadly positive for the antisense class of therapeutics, especially in light of continuing negative publicity for gene therapies.
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