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Neurogene (NGNE) investor relations material
Neurogene Q3 2025 earnings summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.Executive summary
NGN-401 for Rett syndrome advanced to a registrational trial, with positive interim Phase 1/2 data showing multidomain, durable gains and a favorable safety profile at the 1E15 vg dose; higher dose discontinued after a fatal SAE.
First participant dosed in the Embolden trial, with full enrollment expected in 3–6 months across 13 sites and FDA alignment on design and endpoints.
NGN-101 for CLN5 Batten disease deprioritized after FDA denied RMAT designation; options for the program are being evaluated.
In-house cGMP manufacturing facility in Houston is operational, supporting clinical and preclinical supply.
NGN-401 received multiple regulatory designations, including RMAT, orphan drug, Fast Track, rare pediatric, PRIME, and ILAP.
Financial highlights
Net loss of $65.6 million for the nine months ended September 30, 2025, and $21.0 million for Q3 2025; net loss per share was $(3.11) for nine months and $0.99 for Q3.
Cash, cash equivalents, and short-term investments totaled $265.4 million as of September 30, 2025.
R&D expenses increased to $54.4 million for nine months and $17.2 million for Q3 2025, mainly due to NGN-401 clinical and CMC costs.
G&A expenses rose to $21.4 million for nine months and $6.6 million for Q3 2025, driven by higher headcount and stock-based compensation.
Licensing revenue of $0.9 million recognized in the nine months and Q3 2025.
Outlook and guidance
Cash runway expected to fund operations through at least Q1 2028; additional capital will be needed for commercialization and pipeline expansion.
Embolden trial for NGN-401 expected to complete enrollment in 3–6 months.
Ongoing evaluation of options for NGN-101 and early discovery programs.
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