3 November 2017 - - Results of the joint clinical development programme of Cambridge, Massachusetts-based neurological and autoimmune disease therapies specialist Biogen (NASDAQ: BIIB) and Carlsbad, California-based RNA-targeted therapeutics developer Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) of Spinraza (nusinersen) for the treatment of spinal muscular atrophy (SMA), have been published in The New England Journal of Medicine (NEJM), the company said.
End of study results from the Phase 3 study ENDEAR, a randomized, double-blind, sham-procedure controlled 13-month study in individuals with infantile-onset spinal muscular atrophy, appear in the November 2 issue of The New England Journal of Medicine.
The end of study efficacy analysis included all patients (n=121) who had their final study visit after the interim analysis and had the opportunity to attend the six-month study visit assessment.
The two pre-specified ENDEAR primary endpoints were percentage of motor milestone responders, defined as improvements in motor milestone categories in the Hammersmith Infant Neurological Examination, and time to death or permanent ventilation.
The final analysis demonstrated that a greater proportion of infants treated with Spinraza were motor milestone responders, compared to untreated infants (51% vs. 0%, P