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FDA Opens Door to Reconsider Capricor’s Duchenne Cell Therapy Based on HOPE-3 Data

Regulators told Capricor they will review topline HOPE-3 results as the additional study requested after July’s rejection.

Overview

  • Capricor said the FDA indicated it would keep the current deramiocel application under review and evaluate new HOPE-3 data for potential approval.
  • HOPE-3 enrolled 105 patients with a primary endpoint in upper-limb function and secondary cardiac measures, with topline readout expected in mid–fourth quarter.
  • The July Complete Response Letter cited deficiencies, including manufacturing issues that Capricor now says it has addressed.
  • Capricor reported approximately $122.8 million in cash, cash equivalents and marketable securities as of June 30 to support next steps.
  • Shares of Capricor rose after the update, as investors reacted to the clearer regulatory pathway for deramiocel.