Biogen is strengthening its grip on the spinal muscular atrophy (SMA) treatment space.
The company unveiled results from a series of clinical trials at the 2017 CURE SMA conference over the weekend. The investigations were geared towards evaluating the safety and efficacy of Spinraza (nusinersen), which is the first and only therapy approved by the U.S. Food and Drug Administration for SMA.
SMA is a genetic condition characterized by loss of motor neurons in the spinal cord and lower brain stem inducing severe and progressive muscular atrophy and weakness. Nusinersen is an antisense oligonucleotide designed to increase production of SMN proteins that are crucial for repairing damaged motor neurons.
Two phase III studies of the drug revealed promising data regarding its efficacy when administered to patients early whereas an interim analysis from a phase II trial indicated the treatment was able to achieve a number of clinical and safety milestones.
Drug Discovery & Development asked Wildon Farwell, M.D., M.P.H., Biogen’s senior medical director of clinical development about his thoughts on this research as well as what the next steps are for this drug.
DD&D: What encouraged you about these findings?
Farwell: “The breadth of data being presented at the Cure SMA annual conference reinforces the significant and clinically meaningful efficacy of Spinraza on the achievement of motor milestones and measures of motor function across a broad range of individuals with spinal muscular atrophy (SMA), as well as on survival in infantile-onset SMA. We are also encouraged to see the unprecedented improvement in motor function among infants who required permanent ventilation and no increase in risk of adverse events in individuals with scoliosis.”
DD&D: Please explain why diagnosing and treating SMA earlier can improve outcomes? Does earlier administration of SPINRAZA yield better outcomes?
Farwell: “Data from the Spinraza clinical development program demonstrate early treatment may lead to better outcomes. The NURTURE study evaluated the potential benefit of early treatment initiation with Spinraza in infants under six weeks old with genetically diagnosed SMA who were presymptomatic at treatment initiation. Interim results showed presymptomatic infants treated with Spinraza continued to achieve motor milestones generally consistent with normal development. The results demonstrate clinically meaningful improvements that are inconsistent with the natural history of SMA disease progression and support the early initiation of treatment as soon as possible after an SMA diagnosis.”
DD&D: What are your predictions for the neuromuscular treatment overall?
Farwell: “The development and approval of Spinraza exemplifies Biogen’s mission to improve the lives of patients with devastating neurological diseases through groundbreaking science. We recognized the potential of using one of the first antisense oligonucleotides to treat SMA, and we are now investigating a gene therapy for SMA. Biogen is dedicated to being a leader in SMA research and to developing therapies for diseases with high unmet medical need. The pace of discovery and innovation in neuroscience is accelerating rapidly, bringing the promise of incredible advances to patients. We remain deeply committed to the SMA community and incredibly grateful for the collaboration we have with families, advocacy organizations and physicians, to help provide access to Spinraza for those who may benefit.”
DD&D: What are the next steps for the treatment?
Farwell: “Biogen has submitted regulatory filings in Japan, Canada, Australia, Switzerland, and Brazil, and we plan to initiate additional filings in other countries in 2017. We are continuing to work closely with healthcare professionals, advocacy groups and government agencies around the world to help provide access to individuals who may benefit from Spinraza as quickly as possible. Additionally, as part of our mission to make a meaningful difference in the lives of those affected by SMA, we continue to collect and evaluate data to provide a deeper understanding of the efficacy and safety of Spinraza across SMA populations and share those results with the SMA community.
Now that we have an approved therapy to treat SMA in the United States, newborn screening is a critical component for diagnosing and treating SMA as early as possible. We will continue to work closely with the SMA community and state legislatures across the U.S. to make newborn screening available, as we believe early treatment provides the greatest potential benefit to individuals with SMA.”
Filed Under: Drug Discovery
