Biogen has announced that the journal Neuromuscular Disorders has published positive data from the phase 2 NURTURE study of nusinersen (Spinraza) for preventing or delaying the need for respiratory intervention or death in infants with genetically diagnosed and presymptomatic spinal muscular atrophy (SMA).

NURTURE is an ongoing, open-label, multinational study evaluating 25 infants with the genetic diagnosis of SMA (most likely to develop SMA Type 1 and 2) who received their first dose of nusinersen in the presymptomatic stage and before 6 weeks old. The primary end point of the study is time to death or respiratory intervention, defined as invasive or noninvasive ventilation for ≥6 hours/day continuously for ≥7 days or tracheostomy.

Results showed that as of March 2019 all patients were alive and were at least 25 months of age without the need for permanent ventilation and continued to experience improvements consistent with normal development. 

Key secondary end points demonstrated all patients with 3 copies of SMN2 (N=10) reached a maximum mean score of 64 on the CHOP-INTEND (Children’s Hospital of Pennsylvania Infant Test of Neuromuscular Disorders) score of motor function and a mean score of 62.1 for those with 2 copies of SMN2 (N=15). Additionally, 100% of patients were sitting independently and 88% of patients were walking independently consistent with the World Health Organization (WHO) standards. Motor function also improved based on the Hammersmith Infant Neurologic Examination, Section 2 (HINE-2) in all patients with scores nearing 26 points at the last assessment.

Regarding safety, nusinersen was well-tolerated with no new concerns. The most common adverse reactions were respiratory infection, fever, constipation, headache, vomiting, and back pain.

The published results were previously presented at the 2019 Cure SMA Annual SMA Conference and the 5th Congress of the Euopean Academy of Neurology.

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“This pioneering study has far surpassed what we thought was possible and redefined our expectations of how early treatment helps individuals with SMA achieve optimal outcomes,” said Darryl De Vivo, MD, Sidney Carter Professor of Neurology and Pediatrics, Columbia University Irving Medical Center in New York. “The extraordinary results achieved in this study underline the critical importance of newborn screening and early treatment of the clinically normal infants with SMA before the onset of any clinical symptoms.”

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