Neurogene Announces Q4 & Full-Year 2024 Results, Updates
Neurogene Inc., a clinical-stage company dedicated to developing life-changing genetic medicines for patients and families affected by rare neurological diseases, has announced its fourth quarter and full-year 2024 financial results along with recent corporate updates.
Significant Progress in 2024
Neurogene made notable advancements in 2024, particularly in the NGN-401 Phase 1/2 trial for Rett syndrome. Interim efficacy data indicated encouraging results, with NGN-401 being well-tolerated at the 1E15 vg dose in a pediatric cohort. Looking ahead, Neurogene plans to update its registrational trial strategy in the first half of 2025 and share additional interim clinical data from the Phase 1/2 trial, including findings from its cohort of patients aged 11 years and older in the latter half of 2025.
Key Updates and Milestones for NGN-401 Phase 1/2 Trial
NGN-401 is currently under evaluation in an open-label Phase 1/2 clinical trial. The company has outlined several crucial updates and anticipated milestones:
- Protocol Amendment and Dose Selection: Neurogene has amended the protocol and submitted it to the U.S. Food and Drug Administration (FDA), selecting the 1E15 vector genomes (vg) dose for all future participants. This dosage is expected to be evaluated in a future registrational trial based on positive interim efficacy results from November 2024.
- Expanded Age Range: The trial now includes an expanded age range, with patients aged 11 years and older in the older pilot cohort (n=3), in addition to the original 4-to-10-year-old cohort (n=8).
- CMC Scale-Up Plans: The company has aligned with the FDA on chemistry, manufacturing, and controls (CMC) scale-up strategies to support a potential commercial launch of NGN-401 and established a potency assay strategy for the program.
- Regulatory Milestones: Neurogene remains on track to provide an update on its registrational trial plans in the first half of 2025 and anticipates reporting further interim clinical data in the second half of 2025, including results from additional participants expected to be dosed in early 2025.
- Priority Medicines (PRIME) Designation: Neurogene has received PRIME designation from the European Medicines Agency (EMA), a status granted to medicines demonstrating potential to address unmet medical needs by significantly improving clinical outcomes.
Financial Results for Fourth Quarter and Full Year 2024
Cash, Cash Equivalents, and Marketable Securities: As of December 31, 2024, Neurogene reported cash, cash equivalents, and marketable securities of $312.4 million. This funding is expected to sustain the company into the second half of 2027, allowing for:
- Completion of enrollment for a future registrational trial of NGN-401 for Rett syndrome.
- CMC scale-up activities to support NGN-401 registrational requirements.
- Further development of the company’s EXACT™ gene therapy pipeline.
Research & Development (R&D) Expenses: R&D expenses were $15.3 million for the fourth quarter of 2024 and $60.9 million for the full year, compared to $12.2 million and $44.4 million for the same periods in 2023. The increase in R&D expenses was driven primarily by:
- Expanded clinical trial costs for Rett syndrome.
- Higher employee-related expenses due to increased headcount.
- Increased preclinical expenses related to Neurogene’s EXACT gene therapy pipeline.
General & Administrative (G&A) Expenses: G&A expenses for Q4 2024 were $6.2 million, and full-year expenses totaled $22.6 million. This reflects an increase from $2.5 million and $11.2 million in the corresponding periods of 2023, primarily due to:
- Higher employee-related costs from headcount expansion.
- Increased professional service fees.
- Higher office-related and corporate operational costs.
Net Income and Net Loss:
- Net loss for Q4 2024 was $19.5 million, while full-year net loss reached $75.1 million.
- Comparatively, net income for Q4 2023 was $2.4 million, while full-year net loss for 2023 was $36.3 million.
- The net income in 2023 included a one-time $16.4 million bargain purchase gain related to the reverse merger.
Neurogene’s Vision for 2025 and Beyond
Neurogene remains committed to its mission of bringing innovative genetic therapies to patients with rare neurological disorders. The company’s progress in 2024 provides a solid foundation for its 2025 initiatives, including advancing NGN-401 toward registrational trials, expanding its clinical reach, and continuing to refine its gene therapy pipeline.
Neurogene’s advancements in regulatory approvals, clinical research, and financial sustainability reinforce its potential to transform genetic medicine. As the company navigates the next phases of development, stakeholders can expect continued progress in addressing the unmet medical needs of patients with Rett syndrome and beyond.
