Biogen's Spinraza For Muscle Movement Disorder Shows Improved Motor Function In Infants, Toddlers

Biogen Inc BIIB released interim 6-month biomarker data from the initial 29 participants in the open-label RESPOND study.

The Phase 4 study evaluates clinical outcomes and safety following treatment with SPINRAZA (nusinersen) over two years in infants and toddlers with spinal muscular atrophy (SMA) who have unmet clinical needs after treatment with Novartis AG’s NVS Zolgensma (onasemnogene abeparvovec). 

Spinal muscular atrophy (SMA) is a disorder affecting the motor neurons-nerve cells that control voluntary muscle movement. These cells are located in the spinal cord.

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The new data show that plasma neurofilament light chain (NfL) levels, an objective biomarker of axonal injury and neurodegeneration, were reduced in nearly all study participants treated with SPINRAZA. 

Among participants with 2 SMN2 copies:

• All participants had elevated baseline NfL levels relative to healthy children of similar age.
• In infants (n=11) who were nine months or younger at the first SPINRAZA dose (mean baseline NfL: 148.3 pg/mL), NfL levels decreased by a mean of 70% from baseline.
• In children (n=11) over nine months of age at first SPINRAZA dose (mean baseline NfL: 121.8 pg/mL), NfL levels decreased by a mean of 78% from baseline.

Among participants with 3 SMN2 copies:

• Baseline NfL levels were elevated in 2 of 3 children (mean: 60.6 pg/mL).
• NfL reductions were observed in those with elevated levels at baseline and remained stable in the participant without an elevated level at baseline.
• SPINRAZA is approved in more than 71 countries to treat infants, children, and adults with spinal muscular atrophy. More than 14,000 individuals have been treated with SPINRAZA worldwide.

Biogen licensed the global rights to develop, manufacture, and commercialize SPINRAZA from Ionis Pharmaceuticals Inc BIIB.

Price Action: BIIB shares closed higher by 0.15% at $217.56 on Wednesday.