PepGen’s stock has risen by more than 120% after its investigational myotonic dystrophy type 1 (DM1) candidate reported the highest mean splicing correction in a Phase I trial.
In the FREEDOM-DM1 single ascending dose (SAD) study (NCT06204809), the 15mg/kg dose cohort of PGN-EDODM1 demonstrated a mean splicing correction of 53.7%, which PepGen states is substantially higher than any previously reported splicing correction in DM1 patients.
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This comes after PepGen previously reported mean splicing correction at 5mg/kg and 10mg/kg of 12.3% and 29.1%, respectively, demonstrating greater than dose-proportional increases in splicing correction.
Greater than dose-proportional increases in muscle tissue concentrations of PGN-EDODM1 were also observed across all three cohorts; however, specific data have not been released.
PepGen saw its stock jump 121.8% on the data, from a 24 September market close of $2.66 to a 25 September market close of $5.90. The company has a market cap of $192.86m.
PepGen R&D executive vice president Dr Paul Streck said: “Since mis-splicing is the underlying cause of DM1, we believe high levels of splicing correction have the potential to reverse the underlying molecular defects, and produce functional improvements in multiple outcome measures, including myotonia and muscle weakness, in repeat dose studies.”
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By GlobalDataPGN-EDODM1 was generally well-tolerated at 15mg/kg, with no serious treatment-related adverse events (AEs). All treatment-related AEs at 15mg/kg were mild or moderate. There was an event in one patient that required oral OTC antihistamines, but no further intervention.
PGN-EDODM1 is a phosphorodiamidate morpholino oligonucleotide (PMO) that acts by inhibiting myotonic dystrophy protein kinase (DMPK), a protein that plays a critical role in muscle contraction and relaxation, as a mutation in the DMPK gene results in downstream mis-splicing events. In February 2024, the candidate received fast track designation from the US Food and Drug Administration (FDA).
PepGen is also investigating PGN-EDODM1 in the Phase I FREEDOM2-DM1 multiple ascending dose (MAD) study (NCT06667453), with data from the 5mg/kg cohort and dosing in the 10mg/kg cohort expected in Q1 2026.
DM1 landscape
DM1 is a genetic disorder characterised by muscle weakness and wasting. It is caused by a triplet repeat in the DMPK gene, resulting in a toxic gain of functional mRNA and is the most prevalent muscular dystrophy in adults.
The current treatment landscape includes supportive treatments to manage symptoms. No curative therapies are available. There are, however, a number of disease-modifying therapies in clinical trials.
Avidity Biosciences is running a Phase III trial (NCT06411288) of del-desiran, an intravenous antibody oligonucleotide conjugate (AOC), that showed promise in a Phase II open-label extension (OLE) trial.
GlobalData predicts sales of del-desiran will reach $1.31bn in 2031. Meanwhile, the company predicts that PGN-EDODM1 will bring in $283m in global sales in 2031.
GlobalData is the parent company of Clinical Trials Arena.
