All participants experienced a 2-point
improvement in the clinician-rated Clinical Global
Impression-Improvement (CGI-I) scale from baseline
All participants improved in the
caregiver-completed Rett Syndrome Behavior Questionnaire (RSBQ),
ranging from 28 to 52 percent improvement from baseline
All participants with disruptions in sleep,
constipation, and dysphagia at baseline demonstrated objective
improvements
Gains in skill and developmental milestones
were consistent, durable, deepened over time and demonstrated
improvements not expected based on natural history data
Low-dose NGN-401 well-tolerated with favorable
safety profile
Company plans to provide an update of
registrational trial design in the first half of 2025
Company to host investor/analyst webcast today,
November 11, 2024, at 4:30 p.m. ET
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 positive
interim clinical data in the first four participants in the
low-dose cohort of its ongoing Phase 1/2 open-label trial designed
to evaluate NGN-401 gene therapy for the treatment of female
pediatric patients with Rett syndrome. Low-dose NGN-401 has
demonstrated a favorable safety profile.
“Today marks an important day for Neurogene and the Rett
syndrome community as we share positive interim data for NGN-401
from our low-dose cohort that shows the first four participants
demonstrated meaningful gains of skills and developmental
milestones in core clinical domains of Rett syndrome, which are not
expected to occur when compared to and contextualized against the
natural history of Rett syndrome. Data were also concordant across
multiple scales and show consistency of effect across patients,
despite their unique clinical presentations at baseline,” said
Rachel McMinn, Ph.D., Founder and Chief Executive Officer of
Neurogene. “We are incredibly thankful to the participants,
caregivers and Rett syndrome trial sites who are participating in
our study.”
“Rett syndrome is a devastating neurodevelopmental disease that
is incredibly challenging for patients and their caregivers given
there are no treatment options available to address the underlying
cause of the disease,” said Aleksandra Jacobs, M.D., Ph.D.,
Professor of Pediatric Neurology, Albert Einstein College of
Medicine and Director of the Center for Rett Syndrome in the
Children’s Hospital at Montefiore Medical Center. “The totality of
the outcomes shared today with NGN-401 gene therapy have never been
seen before in the treatment of Rett syndrome. Notably, these
initial participants acquired developmental skills post-treatment
during the period in which the natural history of Rett syndrome
indicates girls would not. I look forward to the continued progress
in this program and additional data to come.”
Interim Clinical Data as of Data Cut-Off Date of October 17,
2024
Interim Safety Data (N=7)*
Low-dose (1E15 vg) and high-dose (3E15 vg) NGN-401 have been
well-tolerated with a favorable safety profile in the first seven
pediatric participants (N=5 low-dose; N=2 high-dose):
- No treatment-related serious adverse events (SAEs)
- No signs or symptoms indicative of MeCP2 overexpression
toxicity
- Most treatment-related adverse events (AEs) are known potential
risks of adeno-associated virus (AAV), have been responsive to
steroids, and are resolved or are resolving
- No intracerebroventricular (ICV)-related AEs
- No seizures for any participants following NGN-401
treatment
*Today, Neurogene became aware of an emerging treatment-related
SAE consistent with known risks of AAV gene therapy in the third
high-dose participant who was recently dosed.
Low-Dose Interim Efficacy Data (N=4)
The first four participants (age range 4-7 years old, efficacy
assessments at 15, 12, 9, and 3 months post-dosing) in low-dose
Cohort 1 showed consistent, concordant and durable improvements
across key Rett syndrome assessments:
- All participants achieved a rating of “much improved,” or a
score of 2, on the clinician-rated Clinical Global Impression Scale
of Improvement (CGI-I) from baseline; a score of < 3 is considered clinically meaningful
- All participants improved in the caregiver-completed Rett
Syndrome Behavior Questionnaire (RSBQ), ranging from 28 to 52
percent improvement from baseline
- All participants acquired skills and/or developmental
milestones in one or more core clinical domains of Rett syndrome -
hand function/fine motor, language/communication and
ambulation/gross motor
- These improvements include complex skills that are rarely
learned in this population and skills that are rarely relearned
after developmental regression when compared to the NIH-sponsored
Rett syndrome natural history
- New skills and milestones have increased and deepened over
time
Initiation of Adolescent/Adult Cohort in NGN-401 Clinical
Trial
Neurogene announced today that it has initiated an
adolescent/adult Cohort 3 to gain initial data on the potential of
NGN-401 to treat a broader patient population. This cohort is
designed to enroll three participants ages 16 and above at the high
dose.
FDA Alignment on CMC Requirements to Initiate Future
Registrational Trial and Support Potential Product Launch
Neurogene also announced today that it has gained alignment with
the FDA on its potency assay strategy for NGN-401, which is
necessary to have in place prior to initiating a registrational
trial. In addition, the FDA is aligned with Neurogene’s
manufacturing scale-up plans for NGN-401, which is important to
support a future commercial product launch.
Completed and Upcoming Milestones for the NGN-401
Program
- Expect to complete enrollment in the low-dose pediatric Cohort
1 (N=8) in the fourth quarter of 2024
- Plans to provide an update of registrational trial design in
the first half of 2025
- Plans to announce additional interim Phase 1/2 clinical data in
the second half of 2025
CLN5 Batten Disease Program Update
Neurogene announced today that the Company does not expect to
move forward with the NGN-101 CLN5 Batten disease gene therapy
program at this time. Given the rarity of the disease, continued
investment in the program was predicated on alignment on a
streamlined registrational pathway with FDA. To support a
streamlined pathway, Neurogene submitted a Regenerative Medicine
Advance Therapy (RMAT) application to the FDA. Despite the
Company’s belief that the application met the standard of
preliminary clinical evidence required to obtain an RMAT
designation, the RMAT application was denied. Neurogene is
currently evaluating options for the program.
Investor/Analyst Webcast Details
Management will host a live webcast and conference call today,
November 11, 2024, at 4:30 p.m. ET to review the interim data from
the NGN-401 clinical trial. Access information is available in the
Investor Relations section of Neurogene’s website under Events,
where the webcast replay will also be available for a limited
time.
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™ transgene regulation technology.
EXACT technology 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 selected by the U.S. Food and Drug Administration
(FDA) for its START Pilot Program and has also received
Regenerative Medicine Advance Therapy (RMAT) designation, 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 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
and efficacy results of NGN-401; anticipated future improvements
for participants in the NGN-401 Phase 1/2 trial for the treatment
of Rett syndrome trial designs, clinical development plans and
timing for NGN-401, including anticipated timing of enrollment in
and clinical trial results from the Company’s NGN-401 Phase 1/2
trial for Rett syndrome and expansion of that clinical trial to a
third cohort for adolescent/adult patients; expected benefits of
RMAT designation and participation in the FDA’s START pilot program
for NGN-401, including future interactions with the FDA; the timing
and success of Neurogene’s plans for scale-up of commercial
production of NGN-401; any potential alternatives for the future
development of NGN-101; and our expected cash resources and
liquidity. 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 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 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 potential
for unexpected results or negative impacts to adolescent or adult
patients in Cohort 3 of the Phase 1/2 clinical trial for NGN-401;
the risk that we may not be able to report additional 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
June 30, 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20241111403774/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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