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# Partial Hold on Myotonic Dystrophy Type 1 Therapy
- URL: https://www.fdaweb.com/partial-hold-on-myotonic-dystrophy-type-1-therapy/
- Published: 2026-03-04T12:00:00.000Z
- Updated: 2026-09-14T13:34:53.000Z
- Author: David McFarland
- Tags: FDA Policy/General, #legacy-id-D5160764

FDA has placed a partial clinical hold on a mid-stage U.S. trial of PepGen Inc.’s experimental therapy for myotonic dystrophy Type 1\. The action affects the Phase 2 FREEDOM2-DM1 multiple ascending dose study of PGN-EDODM1 in patients with myotonic dystrophy Type 1\. According to PepGen, the FDA’s questions relate to previously submitted preclinical pharmacology and toxicology data, not to blinded clinical results from the earlier Phase 1 FREEDOM study.

The company said the agency did not cite concerns about Phase 1 clinical data that had been submitted to support initiation of the U.S. Phase 2 trial. PepGen is providing additional analyses to regulators, including recently unblinded data from the FREEDOM study, and said it is working to address the agency’s questions “as quickly as possible.”

No patients in the U.S. have been enrolled in the FREEDOM2 trial to date.

PGN-EDODM1 is an investigational oligonucleotide therapy designed to treat DM1, a rare genetic neuromuscular disorder caused by a cytosine-uracil-guanine repeat expansion in the DMPK gene. The therapy uses PepGen’s Enhanced Delivery Oligonucleotide (EDO) platform, which employs cell-penetrating peptides to deliver a therapeutic oligonucleotide intended to restore normal RNA splicing by freeing the MBNL1 protein from toxic RNA repeat expansions, according to the company.