by Biogen’s Antisense Oligonucleotide (ASO), Salanersen, continues to Phase III studies after the benefit had shown with a lower dosing schedule than Spinraza (Nusinersen) in patients with spinal muscle atrophy (SMA).
Delivering for the same mechanism of action as Spinraza but designed to achieve greater potential, Biogen believes that Salanersen has the potential to achieve high efficacy and at the same time make an annual dose possible. In the meantime, Spinraza is dosed once every four months.
Interim analysis of the I-study phase (NCT05575011) showed that both tested dose levels, 40 mg and 80 mg, once a year, were generally well tolerated and led to a substantial delay of neurodegeneration. This was demonstrated by an average reduction of 70% in the Light Chain of Neurophilament (NFL) after six months.
Half of the patients who were dosed with Salanersen achieved new WHO mile poles that they could not reach in themselves or required help to do, such as walking, crawling, standing or sitting. These patients also experienced clinically meaningful improvements in the motor function of basic line up to one year, including an average improvement of 3.3 points compared to the basic line on the Hammersmith Functional Scale Expanded (HFMSE) and a 5.3-point improvement on the revised upper limb module (rulm).
Salanersen was generally well tolerated in both doses, with most side effects (AEs) mild to moderate in severity. The most common AEs were pyrexia and infection of the upper respiratory tract.
The interim analysis comes from part B of the study, an open label segment that Pediatric SMA patients registered who previously received Novartis’ Zlensma (OnaSemnogen Abparvovec) and had suboptimal clinical status reported by researcher.
The data will be presented at the SMA Research & Clinical Care meeting organized by Cure SMA in Anaheim, California, on 25 June.
Biogen is now working on global health authorities to initiate phase III studies. BIOGEN has a permit for global development, production and commercialization rights for Salanersen from Ionis Pharmaceuticals. This is the second SMA collaboration between the couple, where Spinraza also originally comes from Ionis.
Globaldata analysis shows that Spinraza achieved $ 1.57 billion in sale in 2024, with 2030 turnover expected $ 1.19 billion. This fall in sales is partly due to the therapy that loses his American exclusivity in December 2023.
Globaldata is the parent company of Clinical Trials Arena.
Main researcher of the Salanersen Phase I Test and Clinical Center Nemo’s clinical and scientific director Dr. Valeria a sansone said: “For me, it is neurophilament and the WHO mile poles that are the easiest to interpret, since these children have previously received only three months to be in the ability to sit in the state to sit, only three months. After initiating Salanersen, that is unexpected.”
