Biogen-Ionis Partnered Drug Shows Benefit In Children With Spinal Muscular Atrophy Previously Treated With Novartis' Zolgensma

Biogen Inc. BIIB on Wednesday announced in a press release the topline results from the Phase 1 study of salanersen (BIIB115/ION306), an antisense oligonucleotide (ASO) being developed for spinal muscular atrophy (SMA).

Biogen licensed the global development, manufacturing and commercialization rights for salanersen from Ionis Pharmaceuticals, Inc. IONS. Salanersen was discovered by Ionis.

Leveraging the same mechanism of action as Spinraza (nusinersen) but designed to achieve greater potency, salanersen can achieve high efficacy and enable once-a-year dose.

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An interim analysis of the phase one study in participants with SMA who were previously treated with Novartis AG’s NVS gene therapy, Zolgensma (onasemnogene abeparvovec), was conducted to inform the decision on whether to move salanersen forward into registrational studies.

Both dose levels tested, 40 mg and 80 mg, given once a year, were generally well-tolerated and led to substantial slowing of neurodegeneration, as shown by reductions in neurofilament.

Exploratory clinical outcome data show clinically meaningful improvements in function and attainment of new World Health Organization (WHO) milestones over one year.

Interim results are from part B (n=24) in individuals who received either 40 mg or 80 mg salanersen once a year.

In participants with elevated baseline concentrations of neurofilament light chain (NfL), indicating ongoing neurodegeneration, initiation of salanersen led to mean reductions in NfL of 70% at six months, which were sustained through the one-year dosing interval.

Exploratory clinical outcome data were evaluated for the subgroup of participants with at least one year of follow-up during the interim analysis (n=8 participants aged 2-12 who received 40 mg of salanersen). Half (four out of eight) of these participants achieved new WHO motor milestones they could not previously achieve on their own or required assistance, such as walking, crawling, standing or sitting.

Furthermore, these participants experienced clinically meaningful improvements in motor function from baseline to one year, including a 3.3-point (SD 4.46) mean improvement from baseline on the Hammersmith Functional Motor Scale – Expanded (HFMSE) and a 5.3 point (SD 4.75) improvement on the Revised Upper Limb Module (RULM).

Biogen is currently engaging with global health authorities regarding the design of the phase 3 studies.

Earlier in June, Biogen initiated dosing in the BRAVE phase 3 study to evaluate the efficacy and safety of omaveloxolone in children with Friedreich ataxia (FA) between the ages of 2 to <16.

Both non-ambulatory and ambulatory participants may qualify for the study.

Currently, omaveloxolone is commercialized under the brand name Skyclarys in over 40 countries, including the U.S. and the European Union, and is the only approved product for FA in adults and adolescents aged 16 years and older.

BIIB Price Action: Biogen stock is up 0.58% at $127.04 at publication on Wednesday.

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