Encell: EN001 CMT1A Phase 1b safety confirmed, FDA orphan drug designation, pipeline progress

EN001 CMT1A repeat-dose Phase 1b completed: zero DLT, safety confirmed
FDA orphan drug designation for EN001-CMT (Feb 2025) and EN001-DMD (Feb 2026)
EN001-DMD single-dose Phase 1 completed; repeat-dose Phase 1/2 early terminated, switched to advanced regenerative medicine study
EN001-SP (sarcopenia) Phase 1/2a IND approved (Jun 2024), ongoing
License-out to Lucy Biotech: USD 1.5M upfront + up to USD 18M milestones + royalties
Q1 2026 revenue ₩308.9M (down 71.7% YoY), operating loss ₩4,869.8M, net loss ₩5,260.5M
Cash and equivalents ₩24,435.8M, short-term financial instruments ₩10,000M
Financial burdens: convertible bonds (₩22.5B) and derivative liabilities (₩8.1B)
R&D expenses ₩2,167.6M (680.7% of revenue), CDMO order backlog ₩7.68B