Scholar Rock Sees Phase III Path For SMA Drug

Apitegromab Being Studied As Monotherapy And In Combination

 
• By Alaric DeArment
  
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While the drug may be well-positioned for registrational development, some important caveats remain after mixed data in Phase II study cohorts.

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Scholar Rock announced 12-month results from its Phase II TOPAZ study of apitegromab • Source: Shutterstock

Scholar Rock, LLC’s SRK-015 (apitegromab) appears well-positioned to move into Phase III development in spinal muscular atrophy (SMA) based on 12-month topline results from the Phase II TOPAZ study with Biogen, Inc.’s Spinraza (nusinersen) as background therapy despite two of the three cohorts showing declines relative to a prior interim analysis and one showing an absolute decline.

The Cambridge, MA-based company said 6 April that the longer-term data from TOPAZ bolstered six-month results that were reported in...

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