Neurogene Inc. Reports $75.1 Million Net Loss for 2024 Amid Rising Operating Expenses

Neurogene Inc. reported a net loss of $75.1 million for the year ended December 31, 2024, compared to a net loss of $36.3 million in the prior year. The increase in net loss is primarily attributable to a significant rise in operating expenses, particularly research and development expenses, which increased by $16.5 million to $60.9 million, and general and administrative expenses, which increased by $11.4 million to $22.6 million. The company generated $0.9 million in licensing revenue during 2024, stemming from agreements related to the disposition of legacy assets from a prior business combination.

Significant changes compared to 2023 include the completion of a reverse merger with Neurogene OpCo on December 18, 2023, resulting in a name change and a 1-for-4 reverse stock split. Concurrently, a pre-closing financing raised approximately $95 million. A subsequent private placement in November 2024 generated gross proceeds of $200 million, with net proceeds of approximately $189.5 million after deducting fees and expenses. The company also announced the completion of enrollment in a Phase 1/2 clinical trial for NGN-101 for CLN5 Batten disease, but subsequently stated that they do not expect to move forward with this program due to the denial of an RMAT application by the FDA.

Key operational developments include the ongoing Phase 1/2 clinical trial of NGN-401 for Rett syndrome, which has dosed participants in the United States, the United Kingdom, and Australia. Positive interim efficacy data were announced in November 2024, showing improvements across multiple clinical assessments. However, the company also reported a death in a participant in the high-dose cohort, leading to the removal of that dose from the trial protocol. The company also established a fully operational cGMP manufacturing facility in Houston, Texas, and as of March 20, 2025, employed 107 full-time employees, 81 of whom were engaged in research, development, and technical operations.

The company's financial statements show an accumulated deficit of $262.3 million as of December 31, 2024, and cash, cash equivalents, and short-term investments totaling $312.4 million. Management plans to fund future operations with existing cash and the sale and issuance of securities. The company highlights several risk factors, including its limited operating history, dependence on NGN-401's success, substantial capital requirements, and the inherent uncertainties of clinical trials and regulatory approvals. The company also notes the complexities and potential delays associated with obtaining regulatory approvals in the United States and other jurisdictions, including the EU. The company expects to provide further updates on safety and efficacy data for NGN-401 in the second half of 2025.