Data from Phase 1/2 trial of NGN-401 for Rett syndrome showed 100% of participants improved in CGI-I score and gained >=1 developmental milestone, with an average of 4.7 milestones per participant; no plateau and no milestone loss observed as of data cutoff date of June 16, 2026
Milestones gained in a progressive, stepwise sequence, suggesting a restart of development post-treatment
Topline data from Embolden$(TM)$ registrational trial anticipated in 2H 2027
NGN-401 at the 1E15 vg dose remains generally well-tolerated (N=35) as of data cutoff date of August 10, 2026
Initiated PPQ campaign to support BLA submission for NGN-401; completion of PPQ runs expected by end of 2026
Extended cash runway into 1Q 2029 with oversubscribed financing of approximately $144 million gross proceeds
NEW YORK--(BUSINESS WIRE)--August 11, 2026--
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 second quarter 2026 financial results and highlighted recent corporate updates.
"The second quarter and recent months marked important progress across our NGN-401 program, highlighted by positive long-term Phase 1/2 data showing durable, multidomain developmental gains that have not been observed in the natural history of Rett syndrome," stated Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. "The clinically meaningful benefit observed across all participants, including 100% of treated participants improving on CGI-I and gaining an average of 4.7 developmental milestones, strengthens our confidence in the program as we advance toward topline Embolden(TM) registrational data, which are expected in the second half of 2027. With initiation of our PPQ campaign, ongoing preparations for a potential BLA submission and a strengthened balance sheet supporting commercial-readiness activities, we believe we are well positioned to bring NGN-401 to patients and families as quickly as possible, if approved."
Second Quarter 2026 and Recent Highlights
NGN-401 Gene Therapy for the Treatment of Rett Syndrome
-- Reported positive long-term Phase 1/2 data as of the data cutoff date
of June 16, 2026, which demonstrated developmental milestones were gained
in a progressive, stepwise sequence, suggesting a restart of development
post-treatment
-- 100% of participants (N=10) improved on the Clinical Global
Impression-Improvement (CGI-I) scale and gained >=1 developmental
milestone, consistent with the composite endpoint used to evaluate
efficacy in Embolden
-- 47 total developmental milestones were gained for an average of
4.7 per participant; improvement continued across follow-up
through 30 months with no plateau or loss of milestone observed
-- 7 of 10 participants gained >=2 developmental milestones and
demonstrated improvements across >=2 core Rett syndrome domains;
both of these improvements were observed in pediatric and
adolescent/adult participants
-- Participants experienced clinically meaningful improvements
across additional validated Rett syndrome scales, including the
Rett Syndrome Gross Motor Scale (RSGMS) and Rett Syndrome Hand
Function Scale (RSHFS) (p<0.001)
-- Presented on the Rett Syndrome Natural History Study analysis conducted
to assess treatment effect in Embolden, which demonstrated that the
likelihood of milestone gains >=3 years of age is rare
-- Completed successful dosing of 25 participants in Embolden; topline
data anticipated in the second half of 2027
-- Announced today the Process Performance Qualification (PPQ) campaign
was initiated in July 2026 to support planned Biologics License
Application (BLA) submission; completion of PPQ runs expected by the end
of 2026
-- NGN-401 at the 1E15 vg dose has been generally well-tolerated in the
Phase 1/2 trial and Embolden (N=35) as of August 10, 2026
Additional Corporate Updates
-- Executed an oversubscribed public follow-on offering of approximately
$144 million in gross proceeds, including full exercise of the
underwriters' option to purchase additional shares, extending cash runway
into the first quarter of 2029
Key Anticipated NGN-401 Milestones
-- Report topline data from Embolden registrational trial in the second
half of 2027
-- Complete BLA-enabling PPQ runs by year-end 2026
-- Continue commercial-readiness activities
Second Quarter 2026 Financial Results
-- Cash, Cash Equivalents and Short-Term Investments: Cash, cash
equivalents and short-term investments as of June 30, 2026 were $225.4
million. Together with approximately $134.8 million of net proceeds
received from our July 2, 2026 public offering, Neurogene had pro forma
cash, cash equivalents and short-term investments of approximately $360.2
million and expect its current cash resources to fund planned operations
into the first quarter of 2029.
-- Research & Development (R&D) Expenses: R&D expenses were $25.5 million
for the three months ended June 30, 2026, compared to $19.4 million for
the three months ended June 30, 2025. The increase in R&D expenses for
the three months ended June 30, 2026 was primarily driven by higher costs
associated with the development of NGN-401, including Rett syndrome
clinical trial activities and chemistry, manufacturing and controls $(CMC)$
activities supporting the NGN-401 program. The increase also reflected
higher employee-related expenses, primarily due to increased non-cash
stock-based compensation expenses. The increase was partially offset by
lower spending on the CLN5 Batten disease program and early discovery
activities.
-- General & Administrative (G&A) Expenses: G&A expenses were $11.2
million for the three months ended June 30, 2026, compared to $6.7
million for the three months ended June 30, 2025. The increase in G&A
expenses was primarily driven by higher non-cash stock-based compensation
expense, including a one-time catch-up charge associated with
performance-based stock unit awards following achievement of specified
corporate milestones. Additional increases were driven by corporate and
pre-commercial activities, higher employee-related expenses and
professional fees.
-- Net Loss: Net loss was $34.5 million for the three months ended June
30, 2026, compared to $22.0 million for the three months ended June 30,
2025.
About Neurogene
Neurogene $(NGNE)$ is a clinical-stage biotechnology company focused on developing life-changing genetic medicines for people and their families impacted by devastating neurological diseases. The Company is using a biology-first approach paired with optimized delivery to develop purpose-built genetic medicines, including programs powered by its novel and proprietary EXACT(TM) transgene regulation technology. Neurogene is advancing its lead gene therapy program, NGN-401, as a potential best-in-class, one-time treatment for Rett syndrome. For more information, visit neurogene.com or follow on LinkedIn.
About NGN-401
NGN-401 is an investigational AAV9 gene therapy in late-stage clinical development as a potential best-in-class, one-time treatment for Rett syndrome. It is the only clinical candidate to deliver the full-length human MECP2 gene and includes Neurogene's EXACT(TM) transgene regulation technology, which is designed to deliver consistent, tightly controlled MeCP2 protein expression on a cell-by-cell basis. NGN-401 is delivered through intracerebroventricular administration to achieve the broadest targeting directly to the brain and nervous system based on nonclinical biodistribution data. NGN-401 is being evaluated in the Embolden(TM) registrational clinical trial. Data from the Phase 1/2 trial (as of June 16, 2026) have shown that participants experienced multidomain, durable gains with continued developmental milestone acquisition observed over time, and NGN-401 at the 1E15 vg dose has been generally well-tolerated. NGN-401 has received Breakthrough Therapy, Regenerative Medicine Advanced Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations and selection for the START Pilot Program from the U.S. Food and Drug Administration, Advanced Therapy Medicinal Product, Orphan and Priority Medicines designations from the European Medicines Agency and Innovative Licensing and Application Pathway designation from the United Kingdom Medicines and Healthcare products Regulatory Agency.
Cautionary Note Regarding Forward-Looking Statements
Statements in this press release are made as of the date of this press release. Neurogene does not undertake any obligation to make any updates to these statements to reflect events that occur or circumstances that arise after the date of this press release, except as may be required under applicable U.S. securities law.
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