Neurogene Reports Third Quarter 2025 Financial Results and Provides Comprehensive Clinical and Corporate Updates

a clinical-stage biotechnology company dedicated to delivering transformative genetic medicines for patients with rare neurological disorders, announced its financial results for the third quarter of 2025 along with several significant clinical, operational, and strategic advancements. The quarter marked substantial progress in the company’s lead gene therapy program, NGN-401 for Rett syndrome, and positioned Neurogene for continued momentum as it moves toward late-stage development and potential commercialization.

Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene, emphasized the importance of recent achievements, stating, “We significantly advanced our NGN-401 gene therapy clinical program for Rett syndrome over the past quarter, culminating in the recent dosing of the first participant in the Embolden registrational trial after initiating 12 of 13 sites that enable our plans to rapidly enroll the trial in the next three to six months. The positive interim clinical data from the pediatric cohort of our NGN-401 Phase 1/2 trial shared yesterday, which showed multidomain, durable gains with continued developmental/milestone skill acquisition, underscore NGN-401’s therapeutic promise. With cash runway through the first quarter of 2028, we are in a strong financial position to bring a much-needed gene therapy to people with Rett syndrome.”

Clinical Program Progress: NGN-401 for Rett Syndrome

NGN-401, Neurogene’s lead investigational gene therapy, continues to gain momentum based on new interim findings and the advancement of the pivotal-stage Embolden™ trial. The program represents one of the most advanced gene therapy efforts in Rett syndrome, a rare and severely debilitating neurodevelopmental disorder caused by mutations in the MECP2 gene.

Updated Interim Data from the Phase 1/2 Pediatric Cohort

Neurogene reported updated interim clinical data from the pediatric cohort (ages 4–10) of the ongoing Phase 1/2 trial. As of the October 30, 2025 data cutoff, participants demonstrated:

Multidomain and durable clinical improvements across key functional areas typically impaired in Rett syndrome.
A cumulative total of 35 developmental milestones and skills acquired across the eight pediatric participants.
Improvements spanned hand function and fine motor abilities, language and communication, and ambulation and gross motor function, indicating broad therapeutic impact.
Durable improvements in Clinical Global Impression–Improvement (CGI-I) scores among participants previously reported in the November 2024 interim update.
A favorable safety and tolerability profile with NGN-401 administered at the 1E15 vg dose in both the pediatric cohort and the adolescent/adult cohort (ages 11 and older).

These interim findings reinforce earlier observations that NGN-401 has the potential to address core neurological deficits in Rett syndrome and drive sustained functional progression that is uncommon in this patient population.

Advancement of the Embolden™ Registrational Trial

One of Neurogene’s most significant milestones in the quarter was the dosing of the first participant in the Embolden registrational trial, representing a major step toward late-stage development and potential future approval.

Enrollment is expected to be fully completed within the next three to six months, supported by rapid site activation.
12 of 13 sites for the trial have already been initiated, and Neurogene noted that these centers could be efficiently transitioned into commercial gene therapy delivery sites in the future.

The Embolden trial is designed to evaluate the efficacy and safety of NGN-401 in a broader Rett syndrome population and will serve as the foundation for potential regulatory submissions.

Preclinical Data on Administration Routes Presented at ESGCT

In addition to clinical advancements, Neurogene presented new preclinical data at the ESGCT (European Society of Gene and Cell Therapy) Congress. These findings explored alternative routes of administration for NGN-401 and provided valuable insights into biodistribution and potential clinical implications.

Key Preclinical Findings

Intracerebroventricular (ICV) delivery demonstrated superior AAV biodistribution across brain regions relevant to Rett syndrome compared with intrathecal lumbar (IT-L) administration.
These findings offer a mechanistic rationale supporting the multidomain functional improvements observed in the Phase 1/2 trial.
The data also showed that ICV and IT-L routes produced comparable peripheral organ transduction, meaning IT-L does not provide a liver-sparing advantage over ICV.
Collectively, the results reinforce ICV administration as the optimal approach for delivering NGN-401 to the central nervous system, where MECP2 restoration is most essential.

This emerging body of evidence strengthens the scientific foundation of the NGN-401 program and supports Neurogene’s strategy for clinical development and commercialization.

Third Quarter 2025 Financial Results

Neurogene delivered financial results in line with expectations and maintained a strong balance sheet to support ongoing and upcoming clinical milestones.

Cash, Cash Equivalents, and Short-Term Investments

As of September 30, 2025, the company reported $265.4 million in cash, cash equivalents, and short-term investments.
This financial position is expected to fund planned operations through the first quarter of 2028, providing stability during late-stage development and preparatory commercial activities.

Research & Development (R&D) Expenses

R&D expenses for the quarter were $17.2 million, compared with $16.3 million in the same period of 2024.
The increase was driven primarily by:
- Higher costs associated with ongoing Rett syndrome clinical trials.
- Expanded R&D headcount and employee-related expenses.
These increases were partially offset by reduced investment in other program areas, including Batten disease and early discovery research.

General & Administrative (G&A) Expenses

G&A expenses were $6.6 million for the quarter, up from $5.9 million in Q3 2024.
The rise reflects:
- Higher stock-based compensation.
- Increased staffing in support of broader corporate and operational activities.

Net Loss

Neurogene reported a net loss of $21.0 million for the quarter, compared with $20.2 million in the same period last year.
The year-over-year increase primarily reflects expanded investment supporting the growth of the NGN-401 program and overall company scale.

Looking Ahead: Anticipated Milestones

With a strong financial foundation and considerable progress in its lead clinical program, Neurogene is positioned for a productive upcoming period. Key anticipated milestones include:

Completion of enrollment in the Embolden registrational trial within three to six months.
Continued maturation of clinical data from ongoing trials.
Expanded engagement with regulatory authorities to align on pathways toward approval.
Preparatory work to establish commercial infrastructure leveraging activated Embolden clinical sites.

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.

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