PepGen is pausing the initiation of a new Phase II Duchenne muscular dystrophy (DMD) study as it awaits data from an ongoing Phase II trial.
The US-based biotech said it made the voluntary decision to pause the Phase II CONNECT2-EDO51 trial (NCT06833931) of PGN-EDO51 until it has properly reviewed data from the 10mg cohort in the ongoing Phase II CONNECT1-EDO51 study (NCT06079736).
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On the Nasdaq market, PepGen’s stock dropped 18% on the news, from a market close of $2.18 on 3 March to a close of $2.28 on 4 March.
PepGen president Dr James McArthur said: “This will allow us to gather additional safety data, assess the impact of this dose of PGN-EDO51 on dystrophin levels, and potentially improve the design of CONNECT2. This decision enables us to focus resources on completing CONNECT1, as well as rapidly advancing our FREEDOM studies in myotonic dystrophy type 1 with PGN-EDODM1, in which we recently reported encouraging initial clinical data from the Phase I FREEDOM-DM1 study.”
The biotech said that the two cohorts on the CONNECT1 study are fully enrolled and data from the 10mg/kg cohort is expected during the third quarter of 2025. Data from the 5mg/kg cohort, released in July 2024, showed the therapy to be well-tolerated, with patients showing early signs of efficacy, including a mean exon skipping level of 2.15% and a 0.26% increase from baseline of absolute dystrophin after three months of dosing.
The company added that no new safety issues related to PGN-EDO51 have been observed since the company’s last safety update on 23 January.
PGN-EDO51 acts by targeting exon 51 in the dystrophin gene. The therapy binds to RNA and efficiently interferes with gene expression in a sequence-specific manner. A functional form of the dystrophin protein is produced by skipping exon 51, which should improve the disease condition. If approved, GlobalData projects the drug’s sales will reach $136m by 2030.
GlobalData is the parent company of Clinical Trials Arena.
On 16 December 2024, the US Food and Drug Administration (FDA) placed a clinical hold on the company’s investigational new drug (IND) application for the CONNECT2 trial, but the reason for this pause was not declared. This came a year after the FDA released a clinical hold on PGN-EDODM1, which was placed during the FREEDOM-DM1 study (NCT06204809) in myotonic dystrophy type 1 (DM1).
CONNECT1 is an open-label study evaluating two dose levels of PGN-EDO51, being conducted in Canada. The trial has enrolled a number of boys and young men with DMD who are amenable to exon-51 skipping. CONNECT2 is a 25-week, multinational, double-blind, placebo-controlled trial with multiple ascending doses. The study has opened at sites in the UK.
The FDA granted both orphan drug and rare pediatric disease designations on 13 March 2024 for PGN-EDO51 in patients with DMD amenable to an exon-51 skipping approach. Three weeks before, the company earned fast track designation for PGN-EDODM1.
Elsewhere in the DMD research field, Solid Biosciences stock recently soared on a positive data readout from a Phase I/II study. In addition, Entrada received UK Medicines and Healthcare Products Regulatory Agency (MHRA) authorisation last month to initiate a Phase I/II study of its DMD therapy.
