The FDA placed a partial clinical hold on PepGen’s Freedom2 Phase II trial of PGN‑EDODM1 for myotonic dystrophy type 1 (DM1), citing concerns in preclinical pharmacology and toxicology data rather than the company’s human safety dataset. PepGen said it is providing additional analyses, including recently unblinded Phase I data, and will continue trials in non‑U.S. jurisdictions while engaging regulators in the U.S. Market reaction was immediate: PepGen shares fell sharply after the agency’s action. Analysts note the hold appears to stem from re‑examination of earlier mouse data rather than new human safety signals; the company and independent analysts expect the hold to be resolvable with additional data and discussion. The move underscores continued FDA caution for novel oligonucleotide delivery platforms targeting neuromuscular diseases.
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