Drugs / PGN-EDODM1
Regulatory milestones approvals, filings & regulatory actions · 1 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Clinical hold | US (FDA) | — | adult-onset Steinert myotonic dystrophy | 2023-05-30 | “clinical hold notice from the FDA regarding its Investigational New Drug (IND) application to initiate a Phase 1 study of PGN-EDODM1 in patients with DM1” pepgen.com ↗ “including release of the partial clinical hold placed by the FDA and/or clearance to commence planned clinical studies” pepgen.com ↗ “We are disappointed to receive a clinical hold notice on our planned PGN-EDODM1 study in the U.S.” pepgen.com ↗ |
Trials 3
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT06204809 | Dec 2023 → Oct 2025 | myotonic dystrophy type 1 | PepGen Inc | Completed | No outcome recorded |
| Phase 22 trials | ||||||
| Phase 2 | NCT07220603 | Dec 2025 → Jan 2029 expected | myotonic dystrophy type 1 | PepGen Inc | Recruiting | No outcome recorded |
| Phase 2 | NCT06667453 | Dec 2024 → Mar 2027 expected | myotonic dystrophy type 1 | PepGen Inc | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 18
| Date | Issuer | Release |
|---|---|---|
| 2026-08-06 | PepGen Inc | Results PepGen to Advance PGN-EDODM1 Into Highest Dose Cohort in Phase 2 FREEDOM2-DM1 Study Following DSMB Review pepgen.com ↗
“The DSMB's recommendation to advance FREEDOM2 into the highest planned dose level in the study and escalate dosing in the OLE supports the encouraging safety profile of PGN-EDODM1 following multiple months of treatment,” said James McArthur , PhD, President and Chief Executive Officer of PepGen . |
| 2026-05-21 | PepGen Inc | Results PepGen Announces Presentations at the 15th International Myotonic Dystrophy Consortium Highlighting Clinical Data from the PGN-EDODM1 Program pepgen.com ↗
The FREEDOM-DM1 clinical trial demonstrated strong splicing correction with single doses of PGN-EDODM1, with an acceptable safety profile |
| 2026-03-30 | PepGen Inc | Results PepGen Announces Topline Results from Lowest Dose (5 mg/kg) MAD Cohort in the Ongoing Phase 2 FREEDOM2 Study Demonstrating Favorable Safety, Splicing and vHOT Data pepgen.com ↗ |
| 2026-03-04 | PepGen Inc | Regulatory PepGen Announces Regulatory Updates on FREEDOM2 pepgen.com ↗
The U.S. Food and Drug Administration (FDA) has placed a partial clinical hold on the FREEDOM2-DM1 Phase 2 multiple ascending dose (MAD), randomized, placebo-controlled clinical trial of PGN-EDODM1 in patients with myotonic dystrophy type 1 (DM1). |
| 2025-11-12 | PepGen Inc | Regulatory PepGen Announces Issuance of U.S. Patent for Proprietary PGN-EDODM1 Molecule pepgen.com ↗ |
| 2025-09-24 | PepGen Inc | Results PepGen Announces Highest Mean Splicing Correction Reported in DM1 Patients pepgen.com ↗
These latest results demonstrated a mean splicing correction of 53.7% following a single 15 mg/kg dose of PGN-EDODM1, substantially higher than any previously reported splicing correction in DM1 patients. |
| 2025-02-24 | PepGen Inc | Results PepGen Reports Fourth Quarter and Year-End 2024 Financial Results and Recent Corporate Highlights pepgen.com ↗
Today, we reported initial results from FREEDOM-DM1, which showed robust splicing correction in patients with DM1 following a single dose of PGN-EDODM1. |
| 2025-02-24 | PepGen Inc | Results PepGen Announces Positive Initial Results, Including Robust Splicing Correction, from Ongoing FREEDOM-DM1 Trial in Patients with DM1 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 initial positive clinical data from the 5 and 10 mg/kg dose cohorts in the ongoing FREEDOM-DM1 Phase 1 trial investigating PGN-EDODM1 in myotonic dystrophy type 1 (DM1). |
| 2024-10-08 | PepGen Inc | Results PepGen Announces Presentations at the 29th Annual Congress of the World Muscle Society pepgen.com ↗
Nonclinical data for PGN-EDODM1 demonstrated nuclear delivery, mechanistic and meaningful activity for the potential treatment of DM1 |
| 2024-08-08 | PepGen Inc | Regulatory PepGen Reports Second Quarter 2024 Financial Results and Recent Corporate Highlights pepgen.com ↗
FREEDOM2-DM1 clinical trial cleared by Health Canada and U.K. Medicines and Healthcare products Regulatory Agency. |
| 2024-02-20 | PepGen Inc | Regulatory PepGen Receives U.S. FDA Fast Track Designation for PGN-EDODM1 for the Treatment of Myotonic Dystrophy Type 1 pepgen.com ↗ |
| 2023-10-12 | PepGen Inc | Regulatory PepGen Inc. Announces FDA has Lifted the Clinical Hold on its Investigational New Drug Application for FREEDOM-DM1 Phase 1 Study of PGN-EDODM1 for Myotonic Dystrophy Type 1 (DM1) pepgen.com ↗
We have worked closely with the FDA to resolve their questions expeditiously and are pleased that the clinical hold on our DM1 program in the United States has been lifted. |
| 2023-09-06 | PepGen Inc | Results PepGen Inc. Presents PGN-EDODM1 Preclinical Data Supporting the Company’s Enhanced Delivery Oligonucleotide Platform and PGN-EDODM1 Program at Two Medical Conferences pepgen.com ↗
Also in the current DM1 HSA LR mouse model study reported here, 4 doses of 30 mg/kg of PGN-EDODM1 were generally well-tolerated and reversed 99% of myotonia, as measured by pinch test and corrected 99% of mis-splicing. |
| 2023-09-06 | PepGen Inc | Regulatory PepGen Inc. Announces Clearance of CTA by Health Canada to Begin the FREEDOM-DM1 Phase 1 Study of PGN-EDODM1 in Patients with Myotonic Dystrophy Type 1 pepgen.com ↗ |
| 2023-06-13 | PepGen Inc | Regulatory PepGen Inc. Provides Update on Planned Initiation of Phase 1 Study of PGN-EDODM1 in Myotonic Dystrophy Type 1 pepgen.com ↗ |
| 2023-05-30 | PepGen Inc | Regulatory PepGen Inc. Announces Clinical Hold in the U.S. on IND Application to Initiate a Phase 1 Study of PGN-EDODM1 for Myotonic Dystrophy Type 1 (DM1) 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 Company received a clinical hold notice from the U.S. Food and Drug Administration (FDA) regarding their Investigational New Drug Application (IND) to initiate a Phase 1 study of PGN-EDODM1 in patients with Myotonic Dystrophy Type 1 (DM1). |
| 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 ↗ |
| 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 ↗
Data to be presented at MDA 2021 will show that a single dose of PGN-EDODM1 in mice was well-tolerated and demonstrated sustained correction of mRNA mis-splicing and complete amelioration of the DM1 myotonia phenotype for up to three months. |
All press releases naming this drug 20 releases
Evidence & citations 2 cited values
Every value below carries the sentence it was read from. 3 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | PGN-EDODM1 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT06204809 ↗ |
| Route | Intravenous | “by intravenous (IV) infusion” NCT06204809 ↗ |