Clinical Trial Progress
Completed enrollment for cohort 1 in CONNECT1-EDO51 evaluating 5 mg per kg dose in DMD patients in the Phase II clinical trial. Clearance received from MHRA in the U.K. to initiate CONNECT2-EDO51, a Phase II study of EDO51.
Financial Position
Successful underwritten stock offering raised approximately $80 million, extending projected cash runway into 2026.
FDA Fast Track Designation
EDODM1 granted fast track designation by FDA, facilitating development and review of therapies for serious conditions with unmet medical needs.
Dystrophin Production Expectations
At 10 mg per kg dose cohort, potential to achieve greater than 9% of normal levels of dystrophin protein, highest level achieved by a DMD exon-skipping therapy to date.