Liz Andersson

New SPINRAZA® (NUSINERSEN) SOCIETY DEMONSTRATE BENEFITS IN TREATING PRESYMPTOMATIC INFANTS WITH SPINAL MUSCULAR ATROPHY

CAMBRIDGE, Mass., Oct. 06, 2018 (GLOBE NEWSWIRE) — Biogen Inc. (Nasdaq: BIIB) today announced new interim results from NURTURE, an ongoing open-label, single-arm efficacy and safety study of SPINRAZA® (nusinersen) in 25 presymptomatic infants with SMA. The data are being presented today in a late-breaking session at the 23rd Annual Congress of the World Muscle Society (WMS) held in Mendoza, Argentina.

“The NURTURE study results demonstrate that early diagnosis and treatment with SPINRAZA has the potential to dramatically change the course of SMA,” said Wildon Farwell, M.D., senior medical director, clinical development at Biogen. “This is the longest available span of data on infants with SMA who began treatment in a presymptomatic period and indicates that children treated early with SPINRAZA can achieve motor milestones they would likely not attain without treatment.”

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  1. Liz Andersson
    • NURTURE study participants were alive and did not require permanent ventilation, in contrast to natural history of spinal muscular atrophy (SMA)
    • Study participants achieved motor milestones with 100 percent sitting independently and 88 percent able to walk
    • Additional data feature biomarkers as an indicator for clinical development work in SMA

    The interim analysis evaluated survival and respiratory intervention rates in infants (n=25) who were genetically diagnosed with SMA and began treatment in the presymptomatic stage of the disease. As of May 2018 all patients in the study were alive and none required tracheostomy or permanent ventilation. Additionally, 22 of the 25 participants were able to walk either with assistance or independently according to the motor milestone standard of the World Health Organization and all 25 were able to sit without support.

    The motor skills of study participants were also evaluated using the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND), an assessment which considers 16 different types of movement to create an overall score between zero and 64. The mean CHOP INTEND scores were 62.6 for study participants with three copies of the SMN2 gene and 61.0 for those with two copies of the gene.

    All NURTURE study participants were 14 months or older at the time of the analysis. Participants included infants with two copies of the SMN2 gene (n=15) who are likely to develop a fatal, early-onset form of SMA known as Type 1, and infants with three copies of the SMN2 gene (n=10) who typically develop SMA Type 2 or 3. People living with SMA Types 2 and 3 may never be able to walk or will lose that ability over time. No new safety concerns were identified.

    Additional research presented at WMS compared levels of phosphorylated neurofilament heavy chain (pNF-H) in plasma in more than 300 patients from SPINRAZA clinical trials, including those in the NURTURE study, and a control group of people without SMA. The data demonstrated that treatment with SPINRAZA is associated with a rapid decline followed by stabilization of pNF-H in plasma at levels close to those of healthy controls. The results are part of Biogen’s ongoing work to identify and validate biomarkers that could provide insight on the disease progression of SMA.

    “We continue to develop tools to inform our clinical research and are encouraged by the potential of neurofilament as a biomarker for SMA, how it could further expand the scientific understanding of this rare disease and, more importantly, its potential impact on those living with SMA,” continued Farwell.

    SPINRAZA Program Status

    SPINRAZA is the first and only approved medicine for the treatment of spinal muscular atrophy (SMA) and is currently approved in the U.S., the European Union, Japan and Brazil, among other countries. Biogen has submitted regulatory filings in additional countries and plans to initiate additional filings in other countries. As of June 30, 2018, more than 5,000 individuals with SMA are being treated with SPINRAZA worldwide, based on patients across the post-marketing setting, Expanded Access Program (EAP) and clinical trial participants.

    To support the urgent need for treatment for the most severely affected individuals living with SMA, Biogen initiated one of the largest, global, pre-approval EAPs in any rare disease, providing access to therapy free of charge. From its launch to June 30, 2018, the EAP has provided treatment access to more than 750 patients across 29 countries. Biogen also supports a Named Patient Sales Program (NPP), which allows access to SPINRAZA in countries where it is not commercially available.

    Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals (Nasdaq: IONS), a leader in antisense therapeutics. Biogen and Ionis conducted an innovative clinical development program, the largest of its kind in SMA, that moved SPINRAZA from its first dose in humans in 2011 to its first regulatory approval in five years.

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