Biogen has reported the release of interim results from the ongoing NURTURE study.
This open-label, single-arm clinical trial is designed to assess the safety and efficacy of Spinraza in presymptomatic infants with spinal muscular atrophy (SMA).
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Spinraza is an antisense oligonucleotide (ASO) designed for the treatment of SMA caused due to mutations or deletions in the survival motor neuron 1 (SMN1) gene.
The interim analysis examined survival and respiratory intervention rates in 25 patients that were genetically diagnosed with the condition.
As of May this year, no participants required tracheostomy or permanent ventilation. In addition, 22 of the patients were able to sit without support and walk either with assistance or independently based on the motor milestone standard of the World Health Organization (WHO).
The study also examined the effects of Spinraza on motor skills in infants using Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) scores.
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By GlobalDataMean CHOP INTEND scores were 62.6 for patients with three copies of the survival motor neuron 2 (SMN2) gene and 61 for those with two copies.
The results were presented at the 23rd Annual Congress of the World Muscle Society (WMS) held in Mendoza, Argentina.
Biogen clinical development senior medical director Wildon Farwell said: “The NURTURE study results demonstrate that early diagnosis and treatment with Spinraza has the potential to dramatically change the course of SMA.
“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.”
The drug changes the SMN2 pre-messenger ribonucleic acid (mRNA) splicing to increase production of full-length SMN protein.
Spinraza is currently approved in multiple countries, including the US, Japan, the EU and Brazil. Biogen has submitted regulatory applications in additional markets.
