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, today announced that the Company will be
presenting a short oral presentation as well as five posters at the
29th Annual Congress of the World Muscle Society (WMS), being held
October 8-12, 2024 in Prague, Czech Republic.
“We are pleased to be presenting preclinical and clinical data
on our Duchenne muscular dystrophy and myotonic dystrophy type 1
programs at this premier global muscle meeting,” said James
McArthur, PhD, President and CEO of PepGen. “Based on the totality
of data in both our 5 mg/kg cohort and the ongoing 10 mg/kg cohort
in the CONNECT1-EDO51 trial as of October 3, we believe PGN-EDO51
has a favorable emerging safety profile. There have been no serious
adverse events, and all treatment-related adverse events have been
mild and have resolved. Hypomagnesemia was observed in one patient
and resolved with oral supplementation. All participants continue
in the study as planned, with no discontinuations, dose
interruptions or reductions.”
PGN-EDO51: Duchenne Muscular Dystrophy
(DMD) Short Oral
Title: CONNECT1-EDO51: A 12-week open-label Phase 2
study to evaluate PGN-EDO51 safety and efficacy in people with
Duchenne amenable to exon 51 skipping Presentation Number: #403P Session: Short Oral Presentations 6 - Terrace
2B Date & Time: October 9th
at 6:15-6:45pm CEST Presenter:
Michelle Mellion, MD, Chief Medical Officer
Poster Title:
CONNECT1-EDO51: A 12-week open-label Phase 2 study to evaluate
PGN-EDO51 safety and efficacy in people with Duchenne amenable to
exon 51 skipping Poster Number:
#403P Session: Poster Session 2
- Forum Hall Date & Time:
October 9th at 5:15-6:15pm CEST Presenter: Michelle Mellion, MD, Chief Medical
Officer
Poster Title:
CONNECT2-EDO51: A Phase 2 placebo-controlled study to evaluate
PGN-EDO51 safety and efficacy in people with Duchenne amenable to
exon 51 skipping Poster Number:
#404P Session: Poster Session 2
- Forum Hall Date & Time:
October 9th at 5:15-6:15pm CEST Presenter: Michelle Mellion, MD, Chief Medical
Officer
Poster Title: Single- and
repeat-dose nonclinical data for PGN-EDO51 demonstrated favorable
pharmacology and safety profiles for the treatment of DMD
Poster Number: #405P
Session: Poster Session 2 -
Forum Hall Date & Time:
October 9th at 5:15-6:15pm CEST Presenter: Ashling Holland, PhD, Director,
Research & Preclinical Development
PGN-EDODM1: Myotonic Dystrophy Type 1
(DM1) Poster Title:
Nonclinical data for PGN-EDODM1 demonstrated nuclear delivery,
mechanistic and meaningful activity for the potential treatment of
DM1 Poster Number: #440P
Session: Poster Session 3 -
Forum Hall Date & Time:
October 11th at 2:15-3:15pm CEST Presenter: Ashling Holland, PhD, Director,
Research & Preclinical Development
Poster Title: Evaluation of
PGN-EDODM1: FREEDOM-DM1 and FREEDOM2-DM1 clinical trials in
myotonic dystrophy type 1 Poster
Number: #461P Session: Poster Session 3 - Forum Hall
Date & Time: October 11th
at 2:15-3:15pm CEST Presenter:
Jane Larkindale, DPhil, Vice President, Clinical Science
Following the conference, the presentations presented at the
29th Annual Congress of the WMS will be available on the Investors
page of PepGen’s website under Scientific Publications.
About PGN-EDO51 PGN-EDO51, PepGen's lead clinical
candidate for the treatment of Duchenne muscular dystrophy (DMD),
utilizes the Company's proprietary Enhanced Delivery
Oligonucleotide (EDO) technology to deliver a therapeutic
phosphorodiamidate morpholino oligomer (PMO) that is designed to
target the root cause of this devastating disease. PGN-EDO51 is
designed to skip exon 51 of the dystrophin transcript, an
established therapeutic target for approximately 13% of DMD
patients, thereby aiming to restore the open reading frame and
enabling the production of a truncated, yet functional dystrophin
protein. The U.S. Food and Drug Administration (FDA) has granted
PGN-EDO51 both Orphan Drug and Rare Pediatric Disease Designations
for the treatment of patients with DMD amenable to an exon-51
skipping approach.
About PGN-EDODM1 PGN-EDODM1, PepGen's second
investigational candidate in development for the treatment of DM1,
utilizes the Company's proprietary EDO technology to deliver a
therapeutic oligonucleotide that is designed to restore the normal
splicing function of MBNL1, a key RNA splicing protein. DM1 is a
progressively disabling, life-shortening genetic disorder. DM1 is
estimated to affect 40,000 people in the United States, and over
74,000 people in Europe. The U.S. FDA has granted PGN-EDODM1 both
Orphan Drug and Fast Track Designations for the treatment of
patients with DM1.
About PepGen PepGen 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 designed to
target the root cause of serious diseases.
For more information, please visit PepGen.com. Follow PepGen on
LinkedIn and X.
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 regarding the emerging safety profile of our
product candidates, including, based on early data, PGN-EDO51, and
our plans to continue to advance the CONNECT1 study.
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 risks related to: delays or failure to
successfully initiate or complete our ongoing and planned
development activities for our product candidates, including
PGN-EDO51; our ability to enroll patients in our clinical trials,
including CONNECT1; that our interpretation of clinical and
preclinical study results may be incorrect, or that we may not
observe the levels of therapeutic activity in clinical testing that
we anticipate based on prior clinical or preclinical results,
including for PGN-EDO51; our product candidates, including
PGN-EDO51, may not be safe and effective or otherwise demonstrate
safety and efficacy in our clinical trials; adverse outcomes from
our regulatory interactions, including delays in regulatory review,
clearance to proceed or approval by regulatory authorities with
respect to our programs, including clearance to commence planned
clinical studies of our product candidates, or other regulatory
feedback requiring modifications to our development programs,
including with respect to our CONNECT1 clinical trial; changes in
regulatory framework that are out of our control; unexpected
increases in the expenses associated with our development
activities or other events that adversely impact our financial
resources and cash runway; and our dependence 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 our most recent
annual report on Form 10-K and quarterly report on Form 10-Q that
are filed with the SEC. PepGen explicitly disclaims any obligation
to update any forward-looking statements except to the extent
required by law.
This release discusses PGN-EDO51, an investigational therapy
that has not been approved for use in any country, and is not
intended to convey conclusions about its efficacy or safety. There
is no guarantee that PGN-EDO51 or any other investigational therapy
will successfully complete clinical development or gain regulatory
authority approval.
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version on businesswire.com: https://www.businesswire.com/news/home/20241008707151/en/
Investor Contact Dave Borah,
CFA SVP, Investor Relations and Corporate Communications
dborah@pepgen.com
Media Contact Julia Deutsch
Lyra Strategic Advisory Jdeutsch@lyraadvisory.com
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