Biogen presents robust efficacy and safety data from phase 2 and 3 Spinraza (nusinersen) studies at the Cure SMA 2017 Annual SMA Conference in Orlando, Fl, June 29 – July 2, 2017. The breadth of data presented 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 endpoints in infantile-onset SMA.

“Data presented at the Cure SMA 2017 Annual SMA Conference further demonstrate the significant impact of Spinraza and the benefits of early treatment initiation. We are encouraged to see unprecedented motor function gains in infants on permanent ventilation and a continued favorable benefit-risk profile across a broad population including no increase in risk of adverse events in children who have developed scoliosis.” said Wildon Farwell, M.D., M.P.H., senior medical director, Clinical Development, Biogen. “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 impact of Spinraza across SMA populations and share those results with the SMA community.”

New Spinraza Data Show Robust Efficacy and Safety Across Broad Range of Individuals with SMA

In an analysis of the phase 3 ENDEAR end of study results, a greater proportion of infants with SMA on permanent ventilation treated with Spinraza demonstrated clinical benefits compared to untreated infants.

End of study data from both the Phase 3 ENDEAR and CHERISH studies further demonstrate that earlier Spinraza treatment in individuals with SMA may lead to improved outcomes. In individuals with shorter disease durations (i.e., generally younger at symptom onset), infants in ENDEAR demonstrated a lower risk of death or permanent ventilation and children in CHERISH demonstrated greater motor function improvement from baseline to 15 months compared to untreated individuals.

In addition, further results from the interim analysis of the phase 2 NURTURE study highlight the clinically meaningful efficacy of Spinraza on event-free survival, measures of motor function and achievement of motor milestones when administered to infants with genetically-diagnosed SMA before symptom onset.

“New Spinraza data continue to reinforce the positive results seen in clinical studies and in my own practice,” said Thomas Crawford, M.D., co-director, Muscular Dystrophy Association Clinic at Johns Hopkins Medicine. “The Spinraza clinical development program demonstrates the impact of early treatment. The additional NURTURE data extends this finding by showing substantial improvements in motor milestones, generally consistent with normal development among infants with SMA who have yet to manifest symptoms before they were treated with Spinraza.”

Spinraza demonstrated a favorable benefit-risk profile, with commonly reported adverse events consistent with those expected in the general SMA population or related to a lumbar puncture procedure. Safety data involving the intrathecal administration of Spinraza showed the incidence and nature of the most common lumbar puncture-related adverse events were similar in children with later-onset SMA with or without scoliosis in the clinical studies.

Spinraza is being developed globally for the treatment of SMA. Spinraza is an antisense oligonucleotide (ASO), using Ionis Pharmaceuticals’ proprietary antisense technology, that is designed to treat SMA caused by mutations or deletions in the SMN1 gene located in chromosome 5q that leads to SMN protein deficiency. Spinraza alters the splicing of SMN2 pre-mRNA in order to increase production of full-length SMN protein.  ASOs are short synthetic strings of nucleotides designed to selectively bind to target RNA and regulate gene expression. Through use of this technology, Spinraza has the potential to increase the amount of full-length SMN protein in individuals with SMA.

Spinraza must be administered via intrathecal injection, which delivers therapies directly to the cerebrospinal fluid (CSF) around the spinal cord, where motor neurons degenerate in individuals with SMA due to insufficient levels of survival motor neuron (SMN) protein.

Spinraza demonstrated a favorable benefit-risk profile. The most common adverse reactions reported for Spinraza were upper respiratory infection, lower respiratory infection and constipation. Serious adverse reactions of atelectasis were more frequent in Spinraza-treated patients. Coagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Individuals may be at increased risk of bleeding complications. Renal toxicity has been observed after administration of some antisense oligonucleotides. Spinraza is present in and excreted by the kidney.