4DMT Announces FDA Clearance of IND Application for 4D-175 Genetic Medicine for the Treatment of Geographic Atrophy
2024年6月24日 - 9:00PM
4D Molecular Therapeutics (Nasdaq: FDMT, 4DMT or the Company),
a leading clinical-stage genetic medicines company focused on
unlocking the full potential of genetic medicines to treat large
market diseases, today announced U.S. Food and Drug Administration
(FDA) clearance of the Investigational New Drug Application (IND)
for 4D-175, an R100 vector-based intravitreal genetic medicine, for
the treatment of patients with GA.
The Phase 1 GAZE clinical trial will assess 4D-175 in patients
with GA secondary to AMD. The study design consists of an
open-label, sequential cohort Dose Exploration stage, in which
patients will receive a single intravitreal injection of 4D-175 at
one of three dose levels. Clinical trial objectives include safety
and tolerability, definition of the Phase 2 trial dose level(s),
transgene expression and biological activity. The IND clearance
enables the initiation of GAZE clinical study sites, and 4DMT
expects to begin enrollment in H2 2024.
“GA is a leading cause of irreversible vision loss for over 5
million people globally and while current bolus complement
inhibitor treatments reduce the rate of growth in GA lesions, they
require burdensome monthly or bimonthly intravitreal injections and
do not demonstrate functional vision benefit,” said David Kirn,
M.D., Co-founder and Chief Executive Officer of 4DMT. “4D-175 has
the potential for durable clinical benefit with a single
intravitreal injection, greatly reducing the current treatment
burden for patients, which may lead to better long-term vision
outcomes. In Phase 1, we aim to explore safety and transgene
expression levels to select doses for Phase 2. We look forward to
beginning enrollment in the Phase 1 GAZE clinical trial in the
second half of 2024.”
sCFH is an engineered and optimized version of CFH that can fit
into adeno associated virus (AAV) vectors with robust expression
and full functionality confirmed in human cells in vitro, as well
as in multiple preclinical animal models and species in vivo. The
construct was co-invented by Wenchao Song, Ph.D., Professor of
Pharmacology at the Perelman School of Medicine at the University
of Pennsylvania. Dr. Song has extensive experience researching
complement-mediated inflammatory, autoimmune and thrombotic
vasculopathy disorders. Restoring CFH function through targeted
delivery of a therapeutic sCFH transgene could restore normal
complement regulation and reduce retinal injury that manifests as
progressive GA. Preclinical proof-of-concept for this approach
using 1) human sCFH delivered systemically using an AAV vector in a
mouse model of atypical hemolytic uremic syndrome (aHUS) and 2) a
mouse version of sCFH delivered using an AAV vector in mouse models
of C3 glomerulopathy and aHUS each demonstrated recovery from
complement dysregulation, reduced organ damage and improved
survival.
Preclinical data from 4D-175 in vitro and in vivo
characterization studies were presented at the 2024 ARVO Annual
Scientific Meeting in May; the presentation can be found on the
4DMT website here.
About Geographic Atrophy
Geographic atrophy (GA) is a highly prevalent disease with a
significant unmet medical need. It is estimated that there are
approximately 2.5 million individuals with GA in the United States
and major European markets, and an estimated 5 million individuals
globally. GA is an advanced and severe form of age-related macular
degeneration (AMD) that leads to irreversible vision loss. GA is
characterized by atrophic lesions in the outer retina that affect
central vision and lead to irreversible vision loss. Progressive
loss of central vision leads to difficulties driving, reading, and
completing basic daily tasks. As a result, GA has a major impact on
quality of life. Complement-mediated inflammation is recognized as
a main contributor to the development and worsening of GA. The only
FDA approved treatments for GA are complement inhibitors
administered by intravitreal (IVT) injection once every 4-9
weeks.
About 4D-175 for Geographic Atrophy
4D-175 combines the customized and evolved intravitreal vector,
R100, and a codon-optimized transgene encoding a highly functional
shortened form of human complement factor H (sCFH). The genetic
medicine candidate is designed to provide durable transgene
expression in the retina without significant inflammation following
a single, low-dose intravitreal injection. The sCFH payload is
designed to restore normal complement regulation, which has the
potential to slow progression of disease.
About 4DMT
4DMT is a leading clinical-stage genetic medicines company
focused on unlocking the full potential of genetic medicines to
treat large market diseases in ophthalmology and pulmonology.
4DMT’s proprietary invention platform, Therapeutic Vector
Evolution, combines the power of the Nobel Prize-winning
technology, directed evolution, with approximately one billion
synthetic AAV capsid-derived sequences to invent customized and
evolved vectors for use in our wholly owned and partnered product
candidates. Our product design, development, and manufacturing
engine helps us efficiently create and advance our diverse product
pipeline with the goal of revolutionizing medicine with potential
curative therapies for millions of patients. Currently, 4DMT is
advancing six clinical-stage and one preclinical product candidate,
each tailored to address rare and large market diseases in
ophthalmology, pulmonology and cardiology. In addition, 4DMT is
also advancing programs in CNS through a gene editing partnership.
4D Molecular Therapeutics™, 4DMT™, Therapeutic Vector Evolution™,
and the 4DMT logo are trademarks of 4DMT.
All of our product candidates are in clinical or preclinical
development and have not yet been approved for marketing by the
U.S. Food and Drug Administration (FDA) or any other regulatory
authority. No representation is made as to the safety or
effectiveness of our product candidates for the therapeutic uses
for which they are being studied.
Learn more at www.4DMT.com and follow us on 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, including, without limitation, implied and
express statements regarding the therapeutic potential, and
clinical benefits of 4DMT’s product candidates, as well as the
plans, announcements and related timing for the clinical
development of 4D-175. The words "may," “might,” "will," "could,"
"would," "should," "expect," "plan," "anticipate," "intend,"
"believe," “expect,” "estimate," “seek,” "predict," “future,”
"project," "potential," "continue," "target" and similar words or
expressions are intended to identify forward-looking statements,
although not all forward-looking statements contain these
identifying words. Any forward looking statements in this press
release are based on management's current expectations and beliefs
and are subject to a number of risks, uncertainties and important
factors that may cause actual events or results to differ
materially from those expressed or implied by any forward-looking
statements contained in this press release, including risks and
uncertainties that are described in greater detail in the section
entitled "Risk Factors" in 4D Molecular Therapeutics’ most recent
Quarterly Report on Form 10-Q as well as any subsequent filings
with the Securities and Exchange Commission. In addition, any
forward-looking statements represent 4D Molecular Therapeutics'
views only as of today and should not be relied upon as
representing its views as of any subsequent date. 4D Molecular
Therapeutics explicitly disclaims any obligation to update any
forward-looking statements. No representations or warranties
(expressed or implied) are made about the accuracy of any such
forward-looking statements.
Contacts:
Media:
Katherine SmithInizio Evoke
CommsKatherine.Smith@inizioevoke.com
Investors:
Julian PeiHead of Investor Relations and Corporate
FinanceInvestor.Relations@4DMT.com
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