Success for Biogen, Ionis as spinal muscular atrophy drug performs in Phase III
pharmafile | August 2, 2016 | News story | Manufacturing and Production, Research and Development | Biogen, Ionis, phase III, spinal muscular atrophy
Biogen and Ionis Pharmaceuticals have announced that their spinal muscular atrophy treatment, nusinersen, met its primary endpoint at an interim analysis in a Phase III trial, paving the way for marketing authorisation applications in the coming months.
Spinal muscular atrophy is a rare disease characterise by loss of motor neurons in the spinal cord and lower brain stem. It can cause patients to become paralysed and have difficulty performing basic tasks such as breathing and swallowing.
Nusinersen is an antisense oligonucleotide that is designed to alter the splicing of a gene called SMN2, a near identical gene to SMN1 where defects or losses in this gene can cause spinal muscular atrophy.
In the Phase III ENDEAR trial, infants receiving nusinersen experienced a statistically significant improvement in the achievement of motor milestones compared to those who did not receive treatment. Due to these positive results, Biogen has exercised its option to commercialise nusinersen globally and has paid Ionis a $75 million milestone fee. Under the terms of their agreement, Ionis is eligible to receive a further $150 million in milestone payments based on regulatory approvals, as well as tiered royalties on any potential sales.
B. Lynne Parshall, chief operating officer at Ionis, says: “We are hopeful that nusinersen, if approved, will make a meaningful difference in the lives of patients and families affected by spinal muscular atrophy. We look forward to working with Biogen on completing the clinical program and preparing for what we hope is a positive regulatory review. Nusinersen is the first antisense drug from our neurological franchise to advance to regulatory review, and it illustrates the potential of our antisense technology to address severe disease that other therapeutic modalities are unable to address adequately.”
Additionally, Biogen is working on a global expanded access program for eligible patients with infantile-onset spinal muscular atrophy in the coming months. From here on, Ionis will complete the Phase III studies and work with Biogen on regulatory filings while the latter will be responsible for all nusinersen development, regulatory and commercialisation activities and costs.
Sean Murray
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