PepGen shares almost double as DM1 oligonucleotide achieves 'best in class' splicing
PepGen's experimental drug for myotonic dystrophy type 1 achieved a record splicing correction of 46.5% in a mid-stage clinical trial, outperforming competitors and causing its stock price to nearly double. The positive safety profile and potential for higher doses offer new hope for treating this rare genetic disease.
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PepGen announced that its experimental oligonucleotide PGN-EDODM1 achieved a mean splicing correction of 46.5% at week 13 in a Phase 2 trial for myotonic dystrophy type 1 (DM1). This result significantly outperforms competitors like Dyne Therapeutics, Novartis, and Ionis, which have typically achieved less than 30% correction. Analysts praised the outcome as a best-case scenario, and the news sent PepGen's stock soaring 92% to $4.61.
Beyond genetic metrics, investigators noted meaningful clinical improvements in patients' muscle control, mobility, and grip strength. The data came from the lowest dose group of 10 mg/kg, and a higher 12.5 mg/kg dose arm is currently being evaluated, with results expected in the first half of 2027. Despite a lingering partial clinical hold from the FDA regarding animal safety data, PepGen successfully advanced trials internationally and launched a $125 million public offering to fund further development.
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