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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.
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Q3 2025 earnings summary13 Nov, 2025

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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Neurogene Inc. (NGNE) is a clinical-stage biotechnology company focused on developing genetic medicines for rare neurological diseases. Utilizing its proprietary transgene regulation technology, known as Expression Attenuation via Construct Tuning (EXACT), Neurogene aims to address high unmet medical needs that are not effectively treated by conventional gene therapies. The company is headquartered in New York, New York, and its shares are listed on the NASDAQ.

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