Neurogene Announces NGN-401 Gene Therapy for Rett Syndrome Selected by FDA for START Pilot Program
June 03 2024 - 7:30AM
Business Wire
NGN-401 is one of only three CBER programs
chosen by FDA
NGN-401 was selected based on potential for
clinical benefits and clinical development program readiness
START Program provides sponsors enhanced
communications with FDA to accelerate development of rare disease
therapies
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 its
NGN-401 gene therapy for Rett syndrome has been selected to
participate in the U.S. Food and Drug Administration (FDA) Support
for Clinical Trials Advancing Rare Disease Therapeutics (START)
Pilot Program. As part of the START Program, Neurogene will have
opportunities for enhanced communications with the FDA, with the
aim to further accelerate the pace of NGN-401’s development. These
opportunities are designed to provide frequent advice and regular
ad-hoc conversations to address product-specific development
issues, including, but not limited to, clinical study design,
choice of control group and fine-tuning the choice of patient
population.
“We are honored that NGN-401 gene therapy for Rett syndrome has
been chosen as one of only three CBER programs for FDA’s START
Pilot Program and are grateful that the FDA has committed to
investing significant Agency resources to accelerate development of
NGN-401,” said Rachel McMinn, Ph.D., Founder and Chief Executive
Officer of Neurogene. “We are pleased our application demonstrated
the potential clinical benefits of NGN-401, one of the factors
evaluated by the FDA in making its selection. We look forward to
participating in this landmark effort with the FDA as we seek to
rapidly advance NGN-401 toward a potential registrational study for
the patients and families who live with this devastating
disease.”
Two dose levels of NGN-401 are being evaluated in a Phase 1/2
clinical trial. The trial is assessing safety, tolerability, and
preliminary efficacy of a one-time intracerebroventricular (ICV)
administration of NGN-401 in female pediatric patients with Rett
syndrome. Neurogene recently presented favorable safety data from
the first three patients dosed with NGN-401, and the Company
remains on track to report interim efficacy data from the trial in
the fourth quarter of 2024.
The milestone-driven START Program was launched to help further
accelerate development of novel drug and biological product
candidates intended to address rare diseases through more frequent
communication and rapid interactions with the FDA. START Program
applications required clinical, CMC (chemistry, manufacturing and
controls), and non-clinical development plans as well as an update
on the current status of development. The FDA reported that it
would be considering multiple factors in selecting participants,
including potential clinical benefits of the product candidate, the
alignment of clinical development and CMC plans, and the ability to
move the program forward toward a marketing application. Selected
sponsors are expected to receive more frequent advice from FDA
staff with the goal of facilitating program development and an
expectation of generating high quality and reliable data to support
a potential future marketing application.
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 three Center for Biologics Evaluation and
Research (CBER) 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 (EMA) and the Innovative Licensing and Application
Pathway (ILAP) designation from the United Kingdom (UK) Medicines
and Healthcare products Regulatory Agency (MHRA).
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 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 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 its advancement into a registrational study,
and the anticipated benefits of participation in the FDA’s START
Program. 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 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 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 NGN-401 clinical; the risk that we may
not be able to report our data on the predicted timeline; risks
related to our ability to obtain regulatory approval for, and
ultimately commercialize, our product candidates, including
NGN-401; and other risks and uncertainties identified under the
heading "Risk Factors" included in our 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 our Quarterly
Report on Form 10-Q for the quarter ended March 31, 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/20240603610436/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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