NGN-401 has been generally well-tolerated by
first three patients dosed, with three to nine months of
follow-up
No signs or symptoms of overexpression
toxicity, including in one patient with a mild variant predicted to
result in residual MeCP2 expression
Neurogene remains on track to provide interim
efficacy data from the trial in 4Q:24
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 initial
safety and tolerability data from its ongoing Phase 1/2 gene
therapy clinical trial for Rett syndrome, which showed that NGN-401
was generally well-tolerated by all three patients dosed, with
follow-up of approximately nine, six and three months post-dosing.
These data were presented during the American Society of Gene and
Cell Therapy (ASGCT) Annual Meeting.
“We designed NGN-401 to overcome the limitations of conventional
gene therapy for Rett syndrome by incorporating our EXACTTM
transgene regulation technology, which we believe provides
tolerable and therapeutic levels of protein expression to the key
areas of the brain and nervous system that drive disease,” said
Rachel McMinn, Ph.D., Founder and Chief Executive Officer of
Neurogene. “The NGN-401 data presented at ASGCT demonstrate a
favorable tolerability profile in the first three pediatric
patients, including one with a mild variant predicted to result in
residual MeCP2 expression, with no signs or symptoms of
overexpression-related toxicity reported in any patient. We remain
on track to share interim efficacy data from the first cohort of
the trial in the fourth quarter of 2024.”
The Phase 1/2 open-label trial is evaluating the safety,
tolerability, and preliminary efficacy of two dose levels of
NGN-401 delivered via one-time intracerebroventricular (ICV)
infusion. Enrollment of female patients ages 4-10 years old with
classic Rett Syndrome and a Clinical Global Impression-Severity
(CGI-S) score of 4-6 is ongoing in low-dose Cohort 1 and high-dose
Cohort 2.
The baseline demographics of the first three patients who
received NGN-401 in Cohort 1 (1E15 vector genomes) include:
Patient 1
Patient 2
Patient 3
Age at Dosing
7 years old
4 years old
6 years old
Race
Asian
White
White
MECP2 mutation
Mild
Severe
Severe
Time post-NGN-401
administration
~9 months
~6 months
~3 months
NGN-401 has been generally well-tolerated by all three patients.
All adverse events (AEs) related to NGN-401 have been mild, or
Grade 1, and transient or resolving. Most AEs are known potential
risks of adeno-associated virus (AAV), including asymptomatic
laboratory value changes.
There have been no signs or symptoms indicative of MeCP2
overexpression toxicity reported in any of the three patients,
including the patient with a mild variant that is predicted to
result in residual MeCP2 expression. There have been no
treatment-emergent or ICV procedure-related serious AEs (SAEs).
“There is a high unmet need for new treatments that can address
the incredible disease burden people with Rett syndrome and their
families face on a daily basis,” stated Bernhard Suter, M.D.,
Medical Director of the Blue Bird Circle Rett Center at Texas
Children’s Hospital, Associate Professor of Pediatrics at Baylor
College of Medicine, and principal investigator in the NGN-401
clinical trial. “Gene therapy has the potential to address the
underlying cause of Rett syndrome with a one-time treatment, and
these interim safety data from the NGN-401 trial provide an
important milestone on the path to realizing its potential for
patients. I look forward to the continued work in conducting the
trial as we evaluate NGN-401’s safety and efficacy.”
The presentation by Dr. Suter is available as an ePoster on the
ASGCT Annual Meeting platform and https://ir.neurogene.com. The
data cut-off date for this presentation was April 19, 2024.
Neurogene continues to expect to provide interim clinical data,
including efficacy data, from Cohort 1 in the fourth quarter of
2024 and additional interim data, including from Cohort 2, in the
second half of 2025.
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 will 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; and
anticipated timing of interim clinical trial results from the
Company’s NGN-401 Phase 1/2 trial for Rett syndrome.
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 the Company’s clinical trials,
including NGN-401; 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; Neurogene’s limited operating
history; the risk that Neurogene may not be able to raise adequate
additional capital to finance its operations, complete its clinical
trials and commercialize its products; risks related to Neurogene’s
ability to obtain regulatory approval for, and ultimately
commercialize, its product candidates, including NGN-401; risks
related to the outcome of non-clinical testing and early clinical
trials for the Company’s product candidates, including the ability
of those trials to satisfy relevant governmental or regulatory
requirements; risks related to Neurogene’s limited experience in
designing clinical trials and lack of experience in conducting
clinical trials; 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, 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.
This communication contains hyperlinks to information that is
not deemed to be incorporated by reference into this
communication.
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version on businesswire.com: https://www.businesswire.com/news/home/20240507803994/en/
Company Contact: Cara Mayfield Vice President, Corporate
Affairs cara.mayfield@neurogene.com
Investor Contact: Melissa Forst Argot Partners
Neurogene@argotpartners.com
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