PepGen Announces Clearance by Health Canada of CTA for PGN-EDO51 to Begin the Phase 2 Clinical Trial, CONNECT1-EDO51, for the Treatment of Duchenne Muscular Dystrophy
2023年5月18日 - 8:00PM
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 has
received a No Objection Letter (NOL) for its Clinical Trial
Application (CTA) from Health Canada for its Phase 2 CONNECT1-EDO51
study to initiate an open label, multiple ascending dose (MAD)
clinical trial of PGN-EDO51 in patients with Duchenne muscular
dystrophy (DMD) amenable to an exon 51 skipping approach. The
Company expects to begin dosing patients with PGN-EDO51 in 2H
2023.
“Today is the next step in our development of PGN-EDO51, a
potentially transformative treatment candidate for people living
with DMD. Building upon the encouraging safety, tolerability, and
exon skipping data from our Phase 1 healthy volunteer (HV) study
reported last year showing promising tolerability and exon-skipping
activity, we are pleased to continue our work with the DMD
community to develop a therapy that we hope will produce meaningful
levels of a functional, skipped dystrophin protein,” said James
McArthur, Ph.D., President and CEO of PepGen. “In addition to
our most advanced program, PGN-EDO51, we eagerly anticipate
providing the community with updates on our progress to develop
transformational therapies for myotonic dystrophy type one (DM1),
and other exon skippable mutations for people living with DMD,
including 53, 45 and 44. We are leveraging the power of our EDO
technology to work to change the future for people living with
these devastating diseases.”
“PGN-EDO51 exhibited the highest levels of oligonucleotide
delivery and exon 51 skipping in a clinical study following a
single dose of 5, 10 and 15 mg/kg in healthy volunteers when
compared to publicly available clinical data for other exon 51
skipping approaches,” said Michelle Mellion, M.D., SVP and Head of
Clinical Development at PepGen. “At these dose levels, the majority
of treatment emergent adverse events (TEAE) were assessed as mild
and resolved without any intervention. Looking ahead, and based on
our nonclinical data, we believe CONNECT1-EDO51 may support a
differentiated profile for PGN-EDO51 relative to other
investigational and approved therapies based on previously observed
meaningful and durable data on dystrophin production, as well as
clinical assessments. In summary, we believe PGN-EDO51 offers the
hope of a more complete correction of the disease pathology and
look forward to sharing updates on the progress of our planned
trial.”
PepGen plans to evaluate PGN-EDO51 in approximately 3 cohorts of
ambulatory and non-ambulatory boys and young men in the
CONNECT1-EDO51 study, an open label, MAD clinical trial starting at
5 mg/kg, with plans to escalate to 10 mg/kg and potentially other
doses following Drug Safety Monitoring Board (DSMB) review. The
Phase 2 study will evaluate safety, tolerability, dystrophin
levels, pharmacokinetics and clinical assessments.
About PepGenPepGen 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 StatementsThis 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 potential therapeutic benefits
and safety profile of our candidates, expected outcomes, initiation
and timeline of the Phase 2 studies in PGN-EDO51, our
interpretation of results from the Phase 1 study in PGN-EDODM1 and
other nonclinical studies, and statements about our programs and
product candidates.
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 experience delays or fail to
successfully initiate, enroll, or complete our planned clinical
trials for PGN-EDO51 and PGN-EDODM1 and preclinical studies of
other product candidates or to obtain regulatory approval before
commercialization for marketing of such products; our
interpretation of clinical and preclinical study results may be
incorrect; our product candidates may not be safe and effective; we
may encounter adverse safety signals, such as in our Phase 1
clinical trial where we observed a non-life threatening serious
adverse event in one patient dosed with PGN-EDO51 at 15 mg/kg;
there may be delays in regulatory review, clearance to proceed or
approval 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 take
advantage of certain accelerated regulatory pathways; we may not be
able to attract and retain key employees and qualified personnel;
earlier study results may not be predictive of later stage study
outcomes; we may encounter liquidity distress due to failure of
financial institutions with which we maintain relationship;
disruption in financial markets may interfere with our access to
cash, including our cash deposited in financial institutions, 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 our most recent annual report on Form
10-K on file with the SEC and quarterly report on Form 10-Q to be
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 WattsGilmartin
GroupLaurence@gilmartinir.com
Media ContactSarah Sutton Argot Partners
pepgen@argotpartners.com
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