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Biogen Announces New Interim Phase 2 Results From NURTURE

Biogen (BIIB) announced new interim Phase 2 results from NURTURE, the ongoing open-label, single-arm study evaluating the efficacy and safety of SPINRAZA or nusinersen among pre-symptomatic infants with spinal muscular atrophy SMA.

In NURTURE, all infants treated with SPINRAZA were alive, did not require permanent ventilation and showed improvement in motor function and motor milestone achievements as of July 5, 2017, compared to the disease's natural history.

In the NURTURE study, SPINRAZA was administered to infants six weeks old or younger (n=25), who were in the pre-symptomatic stage, genetically-diagnosed with SMA and had two or three copies of the SMN2 gene (n=15 for two copies (most likely to develop Type 1 SMA); n=10 for three copies (most likely to develop Type 2 SMA)).

At the time of this interim analysis, infants had been followed for up to 25.6 months - well beyond the typical timeframe when most infants with Type 1 SMA would have required permanent ventilation or died. The interim analysis showed that all infants were alive and none required tracheostomy or permanent ventilation.

NURTURE participants also achieved a mean CHOP INTEND score, which measures general motor function among infants with SMA, of 58.4 at last visit (out of a maximum score of 64). Many continue to improve and maintain these scores beyond a point in time at which untreated individuals with Type 1 SMA would experience a significant decline. Overall, the study showed that SPINRAZA was well-tolerated and no new safety concerns were identified.

by RTT Staff Writer

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