Neurogene Announces First Patient Dosed in High-Dose Cohort of NGN-401 Gene Therapy Clinical Trial for Rett Syndrome
2024年6月18日 - 10:26PM
ビジネスワイヤ（英語）
High-dose NGN-401 has been well-tolerated, and
low-dose NGN-401 continues to show a favorable safety profile
Interim safety data presented at the
International Rett Syndrome Foundation (IRSF) ASCEND Summit
Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded
to bring life-changing genetic medicines to patients and families
affected by rare neurological diseases, today announced that the
first patient in Cohort 2 received high-dose NGN-401 gene therapy
in the Phase 1/2 trial for female pediatric patients with Rett
syndrome, and high-dose NGN-401 has been well-tolerated following
dosing in May 2024. The Company also provided an update on interim
safety data on the first three patients in the low-dose cohort
during the IRSF ASCEND 2024 Rett Syndrome National Summit, which
shows that NGN-401 has continued to have a favorable safety profile
with no new treatment-related adverse events (AEs).
“We have met an important program milestone of initiating dosing
in the high-dose cohort of our Rett syndrome gene therapy trial,
and we are pleased to share that high-dose NGN-401 has been
well-tolerated thus far with an early favorable safety profile,”
said Rachel McMinn, Ph.D., Founder and Chief Executive Officer of
Neurogene. “We intentionally designed NGN-401 with the goal of
creating a best-in-class treatment option by leveraging what we
believe to be the optimal route of administration to deliver
consistent and tightly controlled full-length MECP2 expression to
key areas of the brain and nervous system, and today’s safety
update underscores NGN-401’s potential to deliver on that profile.
We remain on track to share interim efficacy data from the low-dose
cohort in the fourth quarter of 2024 and look forward to working
with the FDA as part of its START Pilot Program to accelerate the
development of NGN-401.”
During an oral and poster presentation, Bernhard Suter, M.D.,
Medical Director of the Blue Bird Circle Rett Center at Texas
Children’s Hospital, Associate Professor of Pediatrics and
Neurology at Baylor College of Medicine, and principal investigator
in the NGN-401 clinical trial, will present an updated safety
presentation on the first three low-dose patients. These data
show:
- NGN-401 continues to have a favorable safety profile
- There have been no new treatment-related adverse events (AEs)
since the last safety update during the American Society of Gene
and Cell Therapy (ASGCT) Annual Meeting; all treatment-related AEs
have been mild/Grade 1, and transient or resolving, and most AEs
are known potential risks of AAV
- No signs or symptoms indicative of MeCP2 overexpression
toxicity have been reported, including in the patient with a mild
genetic variant predicted to result in residual MeCP2
expression
- No treatment-emergent or intracerebroventricular (ICV)
procedure-related serious AEs
Dr. Suter will be joined at the podium by Neurogene’s Chief
Scientific Officer, Stuart Cobb, Ph.D., who will present an
overview of Neurogene’s EXACT™ transgene regulation technology.
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.
NGN-401 was one of the Center for Biologics Evaluation and
Research programs selected by the U.S. Food and Drug Administration
(FDA) for its START Pilot Program. NGN-401 previously received
orphan drug designation, Fast Track designation and rare pediatric
designation from the FDA. Neurogene was previously granted an
INTERACT meeting with the FDA regarding the EXACT technology.
NGN-401 also received orphan designation and advanced therapy
medicinal product designation from the European Medicines Agency
and the Innovative Licensing and Application Pathway designation
from the United Kingdom’s Medicines and Healthcare products
Regulatory Agency.
About Neurogene
The mission of Neurogene 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
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. CGMP production of NGN-401 was conducted in this
facility and is expected to support pivotal clinical development
activities. For more information, visit www.neurogene.com.
Cautionary Note Regarding Forward-Looking Statements
Statements in this press release that 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 of the presentation
of clinical trial data for NGN-401, and the anticipated benefits of
participation in the FDA’s START program. 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 based on current beliefs and assumptions that 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 results 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 to the timing and
success of enrolling patients in either or both of the cohorts of
Neurogene’s Phase 1/2 clinical trial of NGN-401 for the treatment
of Rett syndrome; the expected timing and results of dosing of
patients in Neurogene’s NGN-401 clinical trial; 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, including the risk of more significant
or more severe adverse events; the risk that the Company may not be
able to report its data on the predicted timeline; risks related to
Neurogene’s ability to obtain regulatory approval for, and
ultimately commercialize, NGN-401;and other risks and uncertainties
identified under the heading “Risk Factors” included in the
Company’s Annual Report on Form 10-K for the year ended December
31, 2023, filed with the Securities and Exchange Commission (“SEC”)
on March 18, 2024, or its Quarterly Report on Form 10-Q for the
quarter ended March 31, 2024 filed with the SEC on May 10, 2024,
and other filings that the Company has made and may make with the
SEC in the future. 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, Neurogene undertakes no 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20240618924853/en/
Company Contact: Cara Mayfield Vice President, Corporate
Affairs cara.mayfield@neurogene.com
Investor Contact: Melissa Forst Argot Partners
Neurogene@argotpartners.com
Neurogene (NASDAQ:NGNE)
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Neurogene (NASDAQ:NGNE)
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