PepGen Presents Data from its Duchenne Muscular Dystrophy Program at World Muscle Society Congress
PepGen Inc. (Nasdaq: PEPG) presented new data at the 27th International Hybrid Annual Congress of the World Muscle Society regarding its Duchenne muscular dystrophy (DMD) program, focusing on its lead candidate, PGN-EDO51. The data showed significant exon skipping and dystrophin restoration in both non-human primates and mouse models. Highlights include up to 93.1% exon skipping in murine models with PGN-EDO23 and 78% exon skipping in non-human primates with PGN-EDO51. The technology aims to improve oligonucleotide delivery for treating severe neuromuscular diseases.
- PGN-EDO23 achieved up to 93.1% exon skipping and 99.7% dystrophin expression in skeletal muscle.
- PGN-EDO51 showed up to 78% exon skipping in non-human primates, indicating effective delivery.
- Data supports the potential of the Enhanced Delivery Oligonucleotide (EDO) platform.
- None.
BOSTON, Oct. 13, 2022 (GLOBE NEWSWIRE) -- PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, announced the presentation of data from its Duchenne muscular dystrophy program. The data are featured in a poster at the 27th International Hybrid Annual Congress of the World Muscle Society taking place in Halifax, Nova Scotia, Canada from October 11-15, 2022.
PepGen’s Senior Vice President, Head of Clinical Development, Michelle Mellion, M.D., will present data from PepGen’s Duchenne muscular dystrophy (DMD) candidate, PGN-EDO51, including data evaluating the potential of PGN-EDO51 in non-human primates and evaluating PGN-EDO23 (mouse equivalent of PGN-EDO51) in mdx and wild-type mice.
“Our Enhanced Delivery Oligonucleotide (EDO) technology enables delivery of oligonucleotide levels in human muscle. In animal models of Duchenne muscular dystrophy, the EDO technology mediated very high levels of exon skipping and dystrophin restoration. We believe the combined results in human volunteers and animal models supports the potential of our platform,” Dr. Mellion said.
“We are excited to present data at World Muscle Society Annual Conference on the heels of our recent announcement of data from our Phase 1 Healthy Normal Volunteer trial of PGN-EDO51, our lead candidate targeted to treat Duchenne muscular dystrophy, where we observed the highest levels of oligonucleotide delivery and exon skipping in a clinical study following a single dose. We remain committed to our mission to develop transformative therapies for people living with devastating neuromuscular diseases,” said James McArthur, Ph.D., President and CEO of PepGen.
Poster Details:
Title: Unlocking the potential of oligonucleotide therapeutics for Duchenne muscular dystrophy through enhanced delivery
- A single dose of PGN-EDO23 in the murine mdx DMD model achieved up to
93.1% exon skipping and99.7% dystrophin expression in skeletal muscle, and62.3% exon skipping and25.7% dystrophin expression in the heart - Three doses of the clinical product candidate, PGN-EDO51, in non-human primates achieved up to
78% exon skipping in skeletal muscle and24% exon skipping in the left ventricle of the heart - Repeat dosing of the clinical product candidate, PGN-EDO51, in non-human primates demonstrated accumulation of exon skipped transcript with each dose in quadriceps and biceps
Presenter: Michelle Mellion, M.D., Senior Vice President, Head of Clinical Development
Date & Time: Poster presentations available throughout the conference from October 12-15, 2022
Additional details can be found on the conference website. The poster will be available in the “Events & Presentations” section on PepGen’s website following the meeting.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is an X-linked recessive muscle-wasting disease that predominantly affects males. This debilitating disease is caused by genetic mutations in the gene encoding dystrophin, a protein critical for healthy muscle function, and is one of the most prevalent rare genetic diseases, with an incidence rate of approximately one in every 3,500 to 5,000 male births. DMD is characterized by progressive muscle weakness, which leads to patients losing the ability to walk, a loss of upper body function, cardiac issues and difficulties breathing. DMD is invariably fatal by young adulthood. Despite significant advances in treatments for this devastating disease, current therapies are limited by poor delivery to muscle tissue and have yet to establish meaningful clinical benefit for DMD patients.
About PepGen
PepGen Inc. is a clinical-stage biotechnology company advancing the next-generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases. PepGen’s Enhanced Delivery Oligonucleotide (EDO) platform is founded on over a decade of research and development and leverages cell-penetrating peptides to improve the uptake and activity of conjugated oligonucleotide therapeutics. Using these EDO peptides, we are generating a pipeline of oligonucleotide therapeutic candidates that are engineered to target the root cause of serious diseases. For more information, visit www.pepgen.com or follow PepGen on Twitter and LinkedIn.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by words such as “aims,” “anticipates,” “believes,” “could,” “estimates,” “expects,” “forecasts,” “goal,” “intends,” “may,” “plans,” “possible,” “potential,” “seeks,” “will,” and variations of these words or similar expressions that are intended to identify forward-looking statements. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. These forward-looking statements include, without limitation, statements about our clinical and preclinical programs, product candidates, including their planned development and therapeutic potential, plans for future development and clinical trials in our programs, including the planned initiation of a Phase 2a MAD trial of PGN-EDO51 in DMD patients, achievement of milestones, and corporate and clinical/preclinical strategies.
Any forward-looking statements in this press release are based on current expectations, estimates and projections only as of the date of this release and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to that we may fail to successfully complete preclinical studies and clinical trials of our product candidates or to obtain regulatory approval for marketing of such products; initial clinical trial results for one or more of our product candidates may not be predictive of future trial results for such candidates; our product candidates may not be safe and effective; there may be delays in regulatory clearance or changes in regulatory framework that are out of our control; we may not be able to nominate new drug candidates within the estimated timeframes; our estimation of addressable markets of our product candidates may be inaccurate; we may need additional funding before the end of our expected cash runway and may fail to timely raise such additional required funding; more efficient competitors or more effective competing treatments may emerge; we may be involved in disputes surrounding the use of our intellectual property crucial to our success; we may not be able to attract and retain key employees and qualified personnel; earlier-stage trial results may not be predictive of later stage trial outcomes; and we are dependent on third parties for some or all aspects of our product manufacturing, research and preclinical and clinical testing. Additional risks concerning PepGen’s programs and operations are described in its most recent quarterly report on Form 10-Q on file with the SEC. PepGen explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.
Investor Contact
Laurence Watts
Gilmartin Group
Laurence@gilmartinir.com
Media Contact
Gwendolyn Schanker
LifeSci Communications
gschanker@lifescicomms.com
FAQ
What was presented by PepGen at the World Muscle Society Congress?
What are the results of PGN-EDO23 in murine models?
What is the significance of the data for PGN-EDO51?
When was the World Muscle Society Congress held?
