NGNE
Neurogene Inc.19.53
-0.73-3.6%
Dec 16, 4:00:01 PM EST
Earnings Call Transcripts
This Quarter (Q1 '26)
No earnings call transcript available yet
Last Quarter (Q4 '25)
No earnings call transcript available
Key Stats
Market Cap
302.52MP/E (TTM)
-Basic EPS (TTM)
-4.10Dividend Yield
0%Recent Filings
8-K
FDA grants Breakthrough to NGN-401
Neurogene's NGN-401 gene therapy for Rett syndrome snagged FDA Breakthrough Therapy Designation on February 26, 2026, based on interim Phase 1/2 data through October 30, 2025, showing durable, multidomain functional gains. Dosing in the Embolden registrational trial wraps Q2 2026; more Phase 1/2 data drops mid-2026. FDA fast-tracks it. Designation speeds approval but hinges on trial risks.
8-K
NGN-401 trial dosing advances
Neurogene dosed multiple participants in its Embolden registrational trial for NGN-401 Rett syndrome gene therapy during Q4 2025, expecting dosing completion in Q2 2026. Mid-2026 brings interim Phase 1/2 data with 12+ months follow-up for all 10 participants, building on prior multidomain gains. Early commercial activities advance. Cash lasts through Q1 2028. Catalysts stack up fast.
10-Q
Q3 FY2025 results
Neurogene posted a Q3 net loss of $21.0M, up 3.7% y/y from $20.2M, driven by R&D expenses climbing to $17.2M (up 5.9% y/y) on higher Rett syndrome trial costs while Batten spending plunged. Operating loss widened to $23.8M (up 7.4% y/y), softened by $2.7M interest income. Cash burn accelerated, with $59.7M used in operations YTD (up 12.4% y/y) and $15.6M net in investing; quarter-end liquidity stood at $265.4M across cash and short-term investments, bolstered by $9.8M ATM proceeds. No debt. Legacy CVR liabilities fell to $1.1M. Clinical momentum builds in Rett. Competition shadows rare disease gene therapies.
8-K
NGN-401 interim data shines
Neurogene reported positive interim Phase 1/2 data for NGN-401 in Rett syndrome, with all eight pediatric participants gaining 35 developmental milestones across hand function, communication, and ambulation as of October 30, 2025. Dosed first participant in the Embolden registrational trial; enrollment completes in 3-6 months. Cash at $265.4M funds operations through Q1 2028. NGN-401 well-tolerated.
8-K
NGN-401 interim data shines
Neurogene unveiled positive interim Phase 1/2 data for NGN-401 gene therapy in Rett syndrome's pediatric cohort on November 12, 2025. All 8 participants gained functional skills across hand function, communication, and gross motor domains—35 milestones total—with durable multidomain improvements up to 24 months and no HLH evidence. All showed gains. Embolden registrational trial enrollment nears completion in 3-6 months.
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