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 U.S. Food and Drug
Administration (FDA) granted both orphan drug and rare pediatric
disease designations for PGN-EDO51, an investigational therapeutic
for Duchenne muscular dystrophy (DMD) patients whose mutations are
amenable to an exon 51 skipping approach. PepGen is evaluating
PGN-EDO51 for the treatment of DMD in the ongoing CONNECT1 Phase 2
trial, and expects to begin enrolling patients in the CONNECT2
Phase 2 trial later this year.
“Receiving these FDA designations for PGN-EDO51 emphasizes the
critical demand for novel and enhanced therapeutic options for
people living with DMD,” said James McArthur, Ph.D., President and
CEO of PepGen. “With our CONNECT1 Phase 2 trial underway, we look
forward to reporting preliminary data this year.”
Orphan designation is granted by the FDA to advance the
evaluation and development of new treatments intended to treat a
rare disease or condition, which is generally a disease or
condition that affects either (i) fewer than 200,000 individuals in
the United States, or (ii) more than 200,000 individuals on the
basis of certain facts and circumstances. Under the Orphan Drug
Act, the FDA may provide sponsors incentives including tax credits
for qualified clinical trials, FDA user-fee benefits, and seven
years of market exclusivity in the United States after
approval.
The FDA defines a "rare pediatric disease" as a serious or
life-threatening disease in which the serious or life-threatening
manifestations primarily affect individuals aged from birth to 18
years. Under the Rare Pediatric Disease program, the FDA may award
priority review vouchers to sponsors who have been granted rare
pediatic disease designation for its drug. A sponsor who has
been granted the designation and receives an approval for a drug
for a rare pediatric disease may qualify for a voucher that can be
redeemed to receive a priority review of a subsequent marketing
application for a different product.
For more information on these designations, please visit the FDA
website’s Orphan Drug and Rare Pediatric Disease webpages.
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 oligonucleotide 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.
In preclinical studies, PepGen observed that administration of
PGN-EDO51 to NHPs resulted in greater levels of exon-skipping when
compared in head-to-head studies against a molecule that we believe
is structurally equivalent to the most clinically-advanced
peptide-conjugated oligonucleotide therapeutic candidate. Higher
levels of exon skipping may translate to higher levels of
dystrophin production in patients. PGN-EDO51 also exhibited the
highest level of exon 51 skipping in nonhuman primate skeletal
muscles, including the diaphragm, reported for any approved
therapeutic or known development candidate at tolerable target dose
levels, based on cross-trial comparisons of publicly available
data.
In a Phase 1 Healthy Volunteer single ascending dose study,
PGN-EDO51 exhibited six-times higher mean exon 51 skipping than the
naked oligonucleotide based on cross-trial comparisons of publicly
available data.
About Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy (DMD) is an X-linked recessive,
progressive, muscle-wasting disease that predominantly affects
males. This debilitating disease is caused by genetic mutations in
the gene encoding dystrophin, a protein necsessary for normal
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 exon skipping therapies are thought to have
limited impact on disease progression due to low levels (<5%) of
dystrophin production.
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, or 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 designed to target the root cause
of serious diseases.
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 therapeutic
potential and safety profile of our product candidates, including
PGN-EDO51, our technology, including our EDO platform, the design,
initiation and conduct of clinical trials, including the
CONNECT1-EDO51 and CONNECT2-EDO51 trials, expected timelines and
preliminary data reports from our clinical trials, including the
CONNECT1-EDO51 and CONNECT2-EDO51 trials, regulatory interactions,
and our financial resources and cash runway.
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-EDO51 and CONNECT2-EDO51; 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, such as PGN-EDO51, or
other regulatory feedback requiring modifications to our
development programs, including in each case with respect to
CONNECT1-EDO51 and CONNECT2-EDO51; 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.
Investor Contact
Laurence Watts
Gilmartin Group
Laurence@gilmartinir.com
Media Contact
Sarah Sutton
Argot Partners
pepgen@argotpartners.com
PepGen (NASDAQ:PEPG)
Historical Stock Chart
From Nov 2024 to Dec 2024
PepGen (NASDAQ:PEPG)
Historical Stock Chart
From Dec 2023 to Dec 2024