Drugs / PGN-EDO51
Regulatory milestones approvals, filings & regulatory actions · 1 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Clinical hold | US (FDA) | — | DMD-related muscular dystrophy | — | “pause the Phase 2 CONNECT2-EDO51 study of PGN-EDO51 in patients with Duchenne muscular dystrophy (DMD)” pepgen.com ↗ “As previously reported, the Company received a clinical hold notice from the U.S.” pepgen.com ↗ |
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 2 | NCT06833931 | Dec 2024 → May 2025 | Duchenne muscular dystrophy | PepGen Inc | Withdrawn | No outcome recorded |
| Phase 2 | NCT06079736 | Jan 2024 → Aug 2025 | Duchenne muscular dystrophy | PepGen Inc | Terminated | Missed primary |
News releases announcing trial results or a regulatory action · 13
| Date | Issuer | Release |
|---|---|---|
| 2025-05-28 | PepGen Inc | Results PepGen to Focus on Development of Promising DM1 Program Following 10 mg/kg PGN-EDO51 Update pepgen.com ↗
In the 10 mg/kg cohort (n=4) of the CONNECT1 study, PGN-EDO51 increased exon 51 skipped transcripts to 4.26% (a mean increase of 3.5%); however, total dystrophin only increased to 0.59% of normal levels (a mean increase of 0.36%). |
| 2025-01-29 | PepGen Inc | Results PepGen Announces CONNECT Program Updates pepgen.com ↗
Magnesium levels in two of the participants in the 10 mg/kg cohort, who were previously reported as having asymptomatic hypomagnesemia, have returned to baseline levels with administration of ongoing oral magnesium supplementation. |
| 2024-12-16 | PepGen Inc | Regulatory PepGen Announces Clinical Hold in the U.S. on IND Application to Initiate CONNECT2-EDO51 Phase 2 Study of PGN-EDO51 for Duchenne Muscular Dystrophy pepgen.com ↗
today announced that the Company received a clinical hold notice from the U.S. Food and Drug Administration (FDA) regarding its Investigational New Drug (IND) application to initiate the CONNECT2-EDO51 clinical trial in patients with Duchenne muscular dystrophy (DMD). |
| 2024-10-08 | PepGen Inc | Results PepGen Announces Presentations at the 29th Annual Congress of the World Muscle Society pepgen.com ↗
There have been no serious adverse events, and all treatment-related adverse events have been mild and have resolved. |
| 2024-07-30 | PepGen Inc | Results PepGen Announces Positive Data from Low-Dose Cohort of PGN-EDO51 in Ongoing CONNECT1-EDO51 Phase 2 Clinical Trial for Treatment of Duchenne Muscular Dystrophy pepgen.com ↗ |
| 2024-03-13 | PepGen Inc | Regulatory PepGen Receives U.S. FDA Orphan Drug and Rare Pediatric Disease Designations for PGN-EDO51 for the Treatment of Duchenne Muscular Dystrophy pepgen.com ↗
PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today announced that the U.S. Food and Drug Administration (FDA) granted both orphan drug and rare pediatric disease designations for PGN-EDO51, an investigational therapeutic for Duchenne muscular dystrophy (DMD) patients whose mutations are amenable to an exon 51 skipping approach. |
| 2024-03-04 | PepGen Inc | Regulatory PepGen Announces Clearance of CTA by UK Medicines & Healthcare Products Regulatory Agency to Begin CONNECT2-EDO51, a Phase 2 Clinical Trial designed to support potential accelerated approval of PGN-EDO51 for the Treatment of Duchenne Muscular Dystrophy pepgen.com ↗
PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today announced that the UK Medicines & Healthcare products Regulatory Agency (MHRA) has authorized its Clinical Trial Application (CTA) to initiate the CONNECT2-EDO51 Phase 2 clinical trial of PGN-EDO51 in patients with Duchenne muscular dystrophy (DMD) amenable to an exon 51-skipping approach. |
| 2023-05-18 | PepGen Inc | Regulatory PepGen Announces Clearance by Health Canada of CTA for PGN-EDO51 to Begin the Phase 2 Clinical Trial, CONNECT1-EDO51, for the Treatment of Duchenne Muscular Dystrophy pepgen.com ↗
today announced that the company has received a No Objection Letter (NOL) for its Clinical Trial Application (CTA) from Health Canada for its Phase 2 CONNECT1-EDO51 study |
| 2023-03-22 | PepGen Inc | Results PepGen Presents Clinical and Nonclinical Data at the 2023 Annual Muscular Dystrophy Association Clinical and Scientific Conference pepgen.com ↗ |
| 2022-10-13 | PepGen Inc | Results PepGen Presents Data from its Duchenne Muscular Dystrophy Program at World Muscle Society Congress pepgen.com ↗
In animal models of Duchenne muscular dystrophy, the EDO technology mediated very high levels of exon skipping and dystrophin restoration. |
| 2022-09-28 | PepGen Inc | Results PepGen Reports Positive Data from Phase 1 Trial of PGN-EDO51 for the Treatment of Duchenne Muscular Dystrophy pepgen.com ↗
PGN-EDO51 exhibited the highest levels of oligonucleotide delivery and exon skipping in a clinical study following a single dose when compared to publicly available clinical data for other exon 51 skipping approaches |
| 2022-03-15 | PepGen Inc | Regulatory PepGen Announces Approval by Health Canada of CTA to Begin First in Human Trials of PGN-EDO51 to Treat Duchenne Muscular Dystrophy pepgen.com ↗ |
| 2021-03-15 | PepGen Inc | Results PepGen to Present Late-Breaking Data from Novel Enhanced Delivery Oligonucleotide (EDO) Pipeline at 2021 Muscular Dystrophy Association Virtual Clinical and Scientific Conference pepgen.com ↗
We are encouraged by strong preclinical results demonstrating well-tolerated in vivo efficacy that surpasses levels of exon skipping seen with other approaches to DMD, and the achievement of long-term correction of splicing defects in DM1 with a single treatment. |
All press releases naming this drug 15 releases
Evidence & citations 2 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | PGN-EDO51 | “A Study of PGN-EDO51 or Placebo in People With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment” NCT06833931 ↗ |
| Route | Intravenous | “IV infusion” NCT06079736 ↗ |