Capricor Stock Is Moving Higher As Duchenne Dystrophy Cell Therapy Slows Disease Progression By 71%

  • Capricor Therapeutics Inc CAPR has announced final data from Phase 2 HOPE-2 trial of CAP-1002 for advanced stages of Duchenne muscular dystrophy (DMD). 
  • The trial met its primary efficacy endpoint of mid-level performance of upper limb (mid-PUL v1.2) and various skeletal and cardiac function endpoints. 
  • Young men in the advanced stages of DMD experienced improvements in skeletal and cardiac measurements after receiving four doses of CAP-1002 over one year. 
  • The results showed that CAP-1002 demonstrated slowed disease progression by 71%.
  • PUL is a tool designed to assess high (shoulder), mid (elbow), and distal (wrist & hand) function.
  • Additional notable endpoints of full PUL v2.0 (p=0.04) and cardiac endpoint of ejection fraction (p=0.002). The final data efficacy analysis shows a change of 3.2 points for full PUL (v1.2) in CAP-1002 versus placebo at 12 months.
  • CAP-1002 was generally safe and well-tolerated. 
  • Except for two hypersensitivity reactions early in the clinical trial, which were mitigated with a common pre-medication regimen, the HOPE-2 Data and Safety Monitoring Board (DSMB) identified no serious safety signals.
  • Capricor will host a conference call and webcast today at 8:30 a.m. ET to discuss the final data.
  • Price Action: CAPR stock is up 17.1% at $5.88 during the premarket session on the last check Friday.
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Posted In: BiotechLong IdeasNewsHealth CareMoversTrading IdeasGeneralBriefsDuchenne Muscular DystrophyPhase 2 Trial
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