Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.
Phase 2 · ActiveModality: oligonucleotide (EDO peptide-conjugated PMO)
Target DMPK (CUG repeat expansion in DMPK mRNA)
Partnerships Oxford University Innovation Ltd / UK Medical Research Council (exclusive worldwide license to cell-penetrating peptide technology)
Indications
Myotonic dystrophy type 1 (DM1)(Phase 2)
Catalysts
— 2026-11
— first half of 2027
Status note Lead product candidate; company substantially dependent on its success. Phase 1 FREEDOM study completed; Phase 2 FREEDOM2 MAD study ongoing. FDA granted orphan drug and Fast Track designation for DM1; EMA orphan medicinal product designation.
PGN-EDO51
Phase 2 · TerminatedModality: oligonucleotide (EDO peptide-conjugated PMO)
Target DMD (dystrophin)
Partnerships Oxford University Innovation Ltd / UK Medical Research Council (exclusive worldwide license to cell-penetrating peptide technology)
Indications
Duchenne muscular dystrophy (DMD)(Phase 2)
Status note Company voluntarily discontinued development of PGN-EDO51 on May 28, 2025 and has substantially completed wind-down of DMD-related R&D activities.
Per-asset detail extracted from the 10-Q filed 2026-08-06. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.
Sponsor-matched active trials from ClinicalTrials.gov (2 as sponsor, 0 as collaborator); "Sponsor" = company is the lead sponsor, "Collaborator" = company is a collaborator. The BP column flags big-pharma involvement as lead sponsor or collaborator (excluding the company itself). The Mgmt guide column shows what management guided for readout timing when a guided program matches the trial (hover for the quote, click for the source). Trial listings are not a company pipeline slide.
Institutional holder positions from 13F filings. Q/Q change is vs the prior quarter's filing; % of portfolio is the fund's own reported portfolio weight.
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