Ongoing Phase 1/2 clinical trial for NGN-401 to
include additional patients in Cohort 1 and a dose-escalation
cohort
Both previously planned updates are expected to
provide a more robust dataset to inform future registrational trial
design
Removal of staggered dosing in Cohort 1
expected to enable the anticipated completion of Cohort 1 dosing in
the second half of 2024
Third patient dosed in Cohort 1
Neurogene Inc. (NASDAQ: NGNE) (“Neurogene” or “the Company”), a
clinical-stage company founded to bring life-changing genetic
medicines to patients and families affected by rare neurological
diseases, today announced the expansion of its ongoing Phase 1/2
gene therapy clinical trial for NGN-401 for female pediatric
patients with Rett syndrome and updates to enable more rapid
enrollment in the trial.
“We are excited to share that we have met our first 2024 program
milestones, including dosing the third patient in the NGN-401 Phase
1/2 trial for Rett syndrome and expansion of the trial to include
more patients in the current dosing cohort and the addition of a
high dose cohort,” said Founder and Chief Executive Officer, Rachel
McMinn, Ph.D. “Our clinical development strategy has been to build
flexibility and optionality early in the program with two
concurrent dose cohorts designed to generate a more complete data
package, which we expect will inform future registration
discussions with global health authorities. We expect that
expansion of the clinical trial and the removal of staggered dosing
in Cohort 1 will enable us to treat more patients in a shorter
period of time. Based on this update, we expect to complete
enrollment of Cohort 1 in the second half of 2024.”
Rett Syndrome Program Update
The U.S. Phase 1/2 clinical protocol for NGN-401 has been
amended as follows:
- Cohort 1, which specifies a total dose of 1×1015 total vector
genomes delivered via intracerebroventricular (ICV) administration,
was expanded from five patients to eight patients. The dosing
stagger has been removed from Cohort 1, enabling the remaining
patients to be dosed in parallel.
- Cohort 2, which specifies a total dose of 3×1015 total vector
genomes delivered via ICV administration, was added and is expected
to include a total of eight patients.
- The first three patients in Cohort 2 will be dosed in a
staggered manner, with first patient dosing expected in the second
quarter of 2024; pending Data and Safety Monitoring Board review of
the safety data for the first three patients, the protocol will
allow parallel enrollment for the remaining patients in Cohort
2.
- In addition, the protocol includes a targeted immunosuppression
regimen for Cohort 2, designed as a preventative measure to aid in
avoiding potential adeno-associated virus (AAV)-related immune
responses that have been observed with other AAV-based products in
this dose range. The immunosuppression regimen includes the use of
rituximab and sirolimus, along with a shortened course of
corticosteroids. Cohort 1 immunosuppression remains unchanged with
corticosteroids alone.
A similar protocol amendment was submitted to the UK regulatory
authorities. These changes are consistent with the Company’s
guidance issued in January 2024. Importantly, in comprehensive
nonclinical studies, the EXACT transgene regulation technology
embedded in NGN-401 was shown to mechanistically constrain MECP2
transgene expression levels, allowing for the potential to dose
escalate and enhance biodistribution to the brain, without the
commensurate increase in MECP2 transgene expression observed with
conventional gene therapy. Both doses in the updated protocol are
below the “no observed adverse effect” level established in rodent
and nonhuman primate models.
A third patient was dosed in the trial early in the first
quarter. NGN-401 has been generally well-tolerated and there have
been no treatment-emergent or procedure-related serious adverse
events, or signs of overexpression-related toxicity observed in any
patient.
Neurogene remains on track to report interim clinical data from
Cohort 1 in the fourth quarter of 2024 and additional data,
including from Cohort 2, in the second half of 2025.
About EXACT
Neurogene’s novel and proprietary EXACT gene regulation platform
technology is a self-contained transgene regulation platform that
can be tuned to deliver a desired level of transgene expression
within a narrow and therapeutically relevant range, with the goal
of avoiding transgene-related toxicities associated with
conventional gene therapy. EXACT is compatible with viral and
non-viral delivery platforms.
About NGN-401
NGN-401 is an investigational AAV9 gene therapy being developed
as a one-time treatment for Rett syndrome. It is the first clinical
candidate to deliver the full-length human MECP2 gene under the
control of Neurogene’s EXACT technology. The EXACT technology
utilized in NGN-401 is an important advancement in gene therapy for
Rett syndrome, specifically because the disorder requires a
treatment approach that enables targeted levels of MECP2 transgene
expression without causing overexpression-related toxic effects
associated with conventional gene therapy. The robust nonclinical
data package for NGN-401 provides evidence of a potentially
compelling efficacy and safety profile in Rett syndrome.
About Neurogene
Neurogene’s mission is to treat devastating neurological
diseases to improve the lives of patients and families impacted by
these rare diseases. Neurogene is developing novel approaches and
treatments to address the limitations of conventional gene therapy
in central nervous system disorders. This includes selecting a
delivery approach to maximize distribution to target tissues and by
designing products to maximize potency and purity for an optimized
efficacy and safety profile. The Company’s novel and proprietary
EXACT transgene regulation platform technology allows for the
delivery of therapeutic levels while limiting transgene toxicity
associated with conventional gene therapy. Neurogene has
constructed a state-of-the-art gene therapy manufacturing facility
in Houston, Texas. GMP production of NGN-401 was conducted in this
facility and will support pivotal clinical development activities.
For more information, visit www.neurogene.com.
Cautionary Note Regarding Forward-Looking Statements
Statements in this press release which are not historical in
nature are intended to be, and hereby are identified as,
forward-looking statements within the meaning of the Private
Securities Litigation Reform Act of 1995. These statements may
discuss goals, intentions and expectations as to future plans,
trends, events, results of operations or financial condition, or
otherwise, based on current expectations and beliefs of the
management of Neurogene, as well as assumptions made by, and
information currently available to, management of Neurogene,
including, but not limited to, statements regarding the therapeutic
potential and utility, efficacy and clinical benefits of NGN-401,
the safety and tolerability profile of NGN-401, trial designs,
clinical development plans and timing for NGN-401, including
completion of Cohort 1 dosing, first patient dosing of Cohort 2 and
anticipated clinical data results in NGN-401 Phase 1/2 trial for
Rett syndrome and anticipated impact of expansion of Phase 1/2
trial and removal of staggered dosing in Cohort 1. Forward-looking
statements generally include statements that are predictive in
nature and depend upon or refer to future events or conditions, and
include words such as “may,” “will,” “should,” “would,” “expect,”
“anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,”
“intend,” “on track,” and other similar expressions or the negative
or plural of these words, or other similar expressions that are
predictions or indicate future events or prospects, although not
all forward-looking statements contain these words. Forward looking
statements are subject to risks, uncertainties, and assumptions
that are difficult to predict with regard to timing, extent,
likelihood, and degree of occurrence, which could cause actual
result to differ materially from anticipated results and many of
which are outside of Neurogene’s control. Such risks, uncertainties
and assumptions include, among other things, risks related the
timing and success of enrolling patients in the expanded cohort of
our Phase 1/2 clinical trial of NGN-401 for the treatment of Rett
syndrome, the expected timing and results of dosing of patients in
our clinical trials, including NGN-401, the potential that we may
not be able to expand our Phase 1/2 clinical trial of NGN-401 for
the treatment of Rett syndrome into the UK based on a variety of
factors, including but not limited to any decisions of regulatory
authorities, costs of expanding the trial in the UK, the
availability of suitable clinical test sites, the ability to enroll
patients in the UK or other reasons, the potential for negative
impacts to patients resulting from using a higher dose of NGN-401
in Cohort 2 of the Phase 1/2 clinical trial for the treatment of
Rett syndrome, the risk that we may not be able to report our data
on the predicted timeline, our limited operating history; the risk
that we may not be able to raise adequate additional capital to
finance our operations, complete our clinical trials and
commercialize our products, risks related to our ability to obtain
regulatory approval for, and ultimately commercialize, our product
candidates, including NGN-401; risks related to the outcome of
non-clinical testing and early clinical trials for our product
candidates, including the ability of those trials to satisfy
relevant governmental or regulatory requirements; risks related to
our limited experience in designing clinical trials and lack of
experience in conducting clinical trials; expectations regarding
the market and potential for Neurogene’s current product
candidates, including NGN-401; the substantial competition we face
in discovering, developing, or commercializing products, including
NGN-401; expectations regarding the potential tolerability, safety
or efficacy for our current product candidates, including NGN-401;
our ability to attract, hire, and retain skilled executive officers
and employees; our ability to protect our intellectual property and
proprietary technologies; risks related to our reliance on third
parties, contract manufacturers, and contract research
organizations and legislative, regulatory, political and economic
developments and general market conditions. These and other risks
and uncertainties are identified under the heading "Risk Factors"
included in our periodic reports that we file with the Securities
and Exchange Commission.
Nothing in this communication should be regarded as a
representation by any person that the forward-looking statements
set forth herein will be achieved or that the contemplated results
of any such forward-looking statements will be achieved.
Forward-looking statements in this communication speak only as of
the day they are made and are qualified in their entirety by
reference to the cautionary statements herein. Except as required
by applicable law, we do not undertake any obligation to revise or
update any forward-looking statement, or to make any other
forward-looking statements, whether as a result of new information,
future events or otherwise.
This communication contains hyperlinks to information that is
not deemed to be incorporated by reference into this
communication.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240304960271/en/
Neurogene Contacts: Investor Relations: Melissa Forst
Argot Partners Neurogene@argotpartners.com Media: David Rosen Argot
Partners david.rosen@argotpartners.com
Neurogene (NASDAQ:NGNE)
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