Neurogene Advances Rett Program as Spending Rises
The gene-therapy developer raised pro forma liquidity to $360.2 million after a July offering.
Neurogene Inc. (NGNE), a clinical-stage gene-therapy developer, completed dosing of all 25 participants in its Embolden Rett syndrome study and set the second half of 2027 for topline data.
The enrollment milestone capped a steady acceleration for the pivotal study. Neurogene dosed the first participant in the third quarter of 2024, reached more than half of enrollment by March and about 90% by May before completing dosing.
Earlier-stage results also broadened as follow-up lengthened. Ten children in the Phase 1/2 study gained 47 developmental milestones or skills through June, averaging 4.7 each, and all improved on the Clinical Global Impression-Improvement scale. Seven gained at least two milestones and improved across at least two core Rett domains, with no plateau or milestone loss through as long as 30 months of follow-up.
The larger clinical program pushed research and development expense up 31% to $25.5 million from $19.4 million a year earlier, mainly because of higher NGN-401 trial, manufacturing and employee costs. Spending was nearly unchanged from $25.2 million in the first quarter after rising from $17.2 million in the third quarter of 2024.
General and administrative expense climbed 67% to $11.2 million, reflecting higher stock compensation, including a one-time performance-award catch-up charge, as well as pre-commercial, personnel and professional-fee costs. The net loss widened 57% to $34.5 million from $22.0 million a year earlier.
Cash, equivalents and short-term investments declined to $225.4 million at June 30 from $243.2 million at the end of March. An approximately $144 million gross follow-on offering in July extended Neurogene’s projected cash runway to the first quarter of 2029, a year beyond the outlook carried in the preceding three releases.
Neurogene also began its process-performance-qualification manufacturing campaign in July after previously targeting a midyear start. The company expects to complete the runs needed for a biologics-license application by year-end, while the expanded runway carries the program through the planned Embolden readout.