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PepGen announces new preclinical data on progression of PGN-EDODM1
The Fly

PepGen announces new preclinical data on progression of PGN-EDODM1

PepGen announced new preclinical data supporting the progression of PGN-EDODM1, PepGen’s product candidate in development for the treatment of myotonic dystrophy type 1, into clinical trials. PGN-EDODM1 leverages PepGen’s Enhanced Delivery Oligonucleotide technology and consists of the Company’s proprietary cell-penetrating peptide conjugated to a steric blocking oligonucleotide cargo. "We have previously shown that PGN-EDODM1 can achieve greater than 60% correction of the mis-splicing events in a murine model of DM1, resulting in a complete reversal of myotonia. This correction in mis-splicing was observed to be highly durable, with activity sustained through 24 weeks following a single dose. Furthermore, results from our recent Phase 1 clinical trial of PGN-EDO51 in healthy volunteers suggest that our EDO technology has the potential to deliver therapeutic levels of the PGN-EDODM1 oligonucleotide to human muscle," stated James McArthur, Ph.D., President and CEO of PepGen. "We are now pleased to provide an update on the safety profile of PGN-EDODM1, having completed IND-enabling preclinical studies in multiple models. Notably, we observed that this product candidate was well-tolerated in acute GLP studies conducted in rodents and NHPs at what we believe are clinically relevant doses. In addition, while we have observed that PGN-EDODM1 corrects mis-splicing in cells with both long and short CTG repeats, our studies indicate that this product candidate does not mediate the degradation of DMPK transcripts. Finally, no off-target effects were observed in other transcripts containing more than 10 CUG repeats in a DM1 mouse model of disease." Dr. McArthur continued, "We are highly encouraged by the results seen in these IND-enabling studies, and are excited by the potential of PGN-EDODM1 to address the root cause of this debilitating disease without degrading DMPK. We believe that PGN-EDODM1 offers a targeted approach for the treatment of DM1, without the risk of potential confounding effects due to the knockdown of DMPK. There are no approved, disease-modifying therapeutics currently available to those living with DM1, and we look forward to advancing PGN-EDODM1 for this patient population. We anticipate initiating a single ascending clinical trial in DM1 patients in the first half of 2023."

Published first on TheFly

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