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Capricor preps phase 3 for Duchenne cell therapy as it seeks a partner

September 27, 2021

Initial results from the 20-patient HOPE-2 trial reported last year showed signs of activity in older boys and young men with advanced DMD, prompting Capricor to ask the FDA about the possibility of filing for approval based on the mid-stage trial.

The regulator is adamant it wants to see phase 3 data however, and the updated results – which showed that CAP-1002 reduced the decline in performance of the upper limb (PUL) function by 71% compared to placebo over the course of a year – could encourage a partner to come forward.

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