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Novartis Takes Huntington's Path With SMA Therapy Branaplam

Priority Has Shifted After Rival Roche's Success

 
• By Kevin Grogan

With Roche's oral spinal muscular atrophy therapy Evrysdi off to a strong start after its recent US launch, Novartis is switching its priorities for rival RNA-splicing drug branaplam and putting it into a Phase IIb trial for Huntington's disease.

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Huntington's is a progressive brain disorder caused by a defective gene • Source: Shutterstock

Novartis AG is planning to evaluate its investigational oral spinal muscular atrophy (SMA) drug branaplam as a treatment for Huntington's disease (HD), suggesting that the future prospects for the therapy may be best served by focusing on this other devastating inherited neurodegenerative condition instead.

Branaplam, previously known as LMI070, is a once-weekly, orally administered, small molecule RNA splicing modulator that Novartis has been developing...

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