NGNE

Dosed ~90% of participants in Embolden™ registrational trial of NGN-401 for Rett syndrome; on track to complete dosing in the second quarter of 2026 NGN-401 has been generally well-tolerated, with no cases of HLH at the 1E15 vg dose; additional interim Phase 1/2 data expected mid-2026 Presentation at ASGCT Meeting highlighted therapeutic rationale for ICV delivery in CNS-targeted gene therapy, including NGN-401 Strong cash position provides runway through first quarter of 2028 NEW YORK, May 12, 2026--(BUSINESS WIRE)--Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced first quarter 2026 financial results and highlighted recent corporate updates. "The significant unmet need in Rett syndrome and strong interest among physicians and caregivers continue to drive momentum for NGN-401, with approximately 90% of participants dosed in the Embolden™ registrational trial," stated Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. "We continue to be encouraged that NGN-401 at the 1E15 vg dose has been generally well-tolerated. We are laser-focused on completing dosing in Embolden in the coming weeks and look forward to providing an update on this important clinical development milestone. In parallel, we are continuing to strengthen the organization and execute on early commercial-readiness activities to support a successful potential launch of NGN-401." First Quarter 2026 and Recent Highlights NGN-401 Gene Therapy for the Treatment of Rett Syndrome Announced today that ~90% of participants have been dosed in the Embolden registrational trial, and the Company is on track to complete dosing in the second quarter of 2026 NGN-401 at the 1E15 vg dose has been generally well-tolerated, with no cases of hemophagocytic lymphohistiocytosis (HLH) in the Phase 1/2 trial or Embolden as of May 11, 2026 Presented on the therapeutic rationale for intracerebroventricular (ICV) administration of central nervous system (CNS)-targeted gene therapy, including NGN-401, at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting Presentation highlighted the biological need to deliver gene therapy to the key regions of the brain underlying disease, supported by preclinical data demonstrating broader CNS biodistribution to brain...

NEW YORK (AP) — NEW YORK (AP) — Neurogene Inc. (NGNE) on Tuesday reported a loss of $30.9 million in its first quarter. The New York-based company said it had a loss of $1.39 per share. The results fell short of Wall Street expectations. The average estimate of five analysts surveyed by Zacks Investment Research was for a loss of $1.35 per share. _____ This story was generated by Automated Insights (http://automatedinsights.com/ap) using data from Zacks Investment Research. Access a Zacks stock report on NGNE at https://www.zacks.com/ap/NGNE

NEW YORK (AP) — NEW YORK (AP) — Neurogene Inc. (NGNE) on Tuesday reported a loss of $24.7 million in its fourth quarter. On a per-share basis, the New York-based company said it had a loss of $1.12. The results surpassed Wall Street expectations. The average estimate of six analysts surveyed by Zacks Investment Research was for a loss of $1.20 per share. For the year, the company reported a loss of $90.4 million, or $4.24 per share. _____ This story was generated by Automated Insights (http://automatedinsights.com/ap) using data from Zacks Investment Research. Access a Zacks stock report on NGNE at https://www.zacks.com/ap/NGNE

100% of participants enrolled in Embolden™ registrational trial and >50% of participants dosed; on track to complete dosing in second quarter of 2026 NGN-401 has been generally well-tolerated in Phase 1/2 trial and Embolden, with no cases of HLH at the 1E15 vg dose; additional interim Phase 1/2 data expected mid-2026 FDA Breakthrough Therapy designation granted to NGN-401 based on positive interim safety and efficacy data from Phase 1/2 trial PPQ campaign planned to begin mid‑2026, supporting BLA readiness Strong cash position provides runway through first quarter of 2028 NEW YORK, March 24, 2026--(BUSINESS WIRE)--Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced full year 2025 financial results and highlighted recent corporate updates. "We continue to demonstrate strong execution, with completion of enrollment in the Embolden registrational trial, dosing more than half of participants, and receipt of Breakthrough Therapy designation for NGN-401," stated Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. "NGN-401 continued to be generally well-tolerated at the 1E15 vg dose, and we remain on track to complete dosing in the second quarter of 2026. With PPQ planned to begin mid-year and early commercial-readiness activities well underway, we believe NGN-401 is increasingly de-risked as we advance toward a BLA submission and potential launch of NGN-401." Recent Corporate and NGN-401 Program Highlights NGN-401 Gene Therapy for the Treatment of Rett Syndrome Announced today 100% of participants are enrolled in the Embolden™ registrational trial and >50% of participants have been dosed, and the Company is on track to complete dosing in the second quarter of 2026 NGN-401 at the 1E15 vg dose has been generally well-tolerated, with no cases of hemophagocytic lymphohistiocytosis (HLH) in the Phase 1/2 trial or Embolden as of March 23, 2026 Received Breakthrough Therapy designation for NGN-401 from the U.S. Food and Drug Administration (FDA), which was based on the FDA’s review of interim efficacy and safety data from the Phase 1/2 trial as of the data cutoff date of October 30, 2025, including patient-level data and supporting video documentation In preparation for the planned NGN-401 Biologics License Application...

Reported positive interim data from NGN-401 Phase 1/2 gene therapy trial for Rett syndrome demonstrating multidomain, durable gains, with 35 total developmental milestones/skills acquired across eight participants First participant dosed in Embolden™ registrational trial of NGN-401 for Rett syndrome, with complete enrollment expected in three to six months Strong cash position provides runway through first quarter of 2028 NEW YORK, November 13, 2025--(BUSINESS WIRE)--Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced third quarter 2025 financial results and highlighted recent corporate updates. "We significantly advanced our NGN-401 gene therapy clinical program for Rett syndrome over the past quarter, culminating in the recent dosing of the first participant in the Embolden registrational trial after initiating 12 of 13 sites that enable our plans to rapidly enroll the trial in the next three to six months," stated Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. "The positive interim clinical data from the pediatric cohort of our NGN-401 Phase 1/2 trial shared yesterday, which showed multidomain, durable gains with continued developmental/milestone skill acquisition, underscore NGN-401's therapeutic promise. With cash runway through the first quarter of 2028, we are in a strong financial position to bring a much-needed gene therapy to people with Rett syndrome." Third Quarter 2025 and Recent Highlights, and Anticipated Milestones NGN-401 Gene Therapy for the Treatment of Rett Syndrome Reported updated interim clinical data with NGN-401 from the pediatric cohort (ages 4-10 cohort) of the Phase 1/2 trial, which continue to demonstrate multidomain, durable gains with continued developmental milestone/skill acquisition as of the data cutoff date of October 30, 2025 Functional improvements have been shown across all eight pediatric participants, with an aggregate 35 developmental milestones/skills gained across core clinical domains of Rett syndrome – hand function/fine motor, language/communication and ambulation/gross motor Improvements in the Clinical Global Impression-Improvement (CGI-I) have been durable for participants included in the November 2024 interim data report NGN-401 at the 1E15 vg dose has...

NEW YORK (AP) — NEW YORK (AP) — Neurogene Inc. (NGNE) on Thursday reported a loss of $21 million in its third quarter. On a per-share basis, the New York-based company said it had a loss of 99 cents. The results beat Wall Street expectations. The average estimate of five analysts surveyed by Zacks Investment Research was for a loss of $1.26 per share. _____ This story was generated by Automated Insights (http://automatedinsights.com/ap) using data from Zacks Investment Research. Access a Zacks stock report on NGNE at https://www.zacks.com/ap/NGNE

NEW YORK (AP) — NEW YORK (AP) — Neurogene Inc. (NGNE) on Monday reported a loss of $22 million in its second quarter. On a per-share basis, the New York-based company said it had a loss of $1.05. The results topped Wall Street expectations. The average estimate of five analysts surveyed by Zacks Investment Research was for a loss of $1.15 per share. _____ This story was generated by Automated Insights (http://automatedinsights.com/ap) using data from Zacks Investment Research. Access a Zacks stock report on NGNE at https://www.zacks.com/ap/NGNE

Announced design of Embolden™ registrational clinical trial of NGN-401 gene therapy for Rett syndrome; trial initiation activities underway Completed dosing in Phase 1/2 NGN-401 trial, and remains on track to report updated clinical efficacy and safety data in the second half of 2025 Cash runway into early 2028 NEW YORK, August 11, 2025--(BUSINESS WIRE)--Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced second quarter 2025 financial results and highlighted recent corporate updates. "In the first half of 2025, we made significant progress in our NGN-401 program for Rett syndrome. We completed dosing of the last five participants in the Phase 1/2 trial and received written agreement from the U.S. FDA on the key elements of the Embolden™ registrational trial, in which we have already begun initiation activities," stated Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. "We are pleased to be moving forward with Embolden, which was purposefully and rigorously designed to differentiate NGN-401 by evaluating participants as young as three years of age with a primary endpoint that incorporates measures that KOLs, caregivers and payors believe to be clinically meaningful. Our previously announced reallocation of capital provides us runway into early 2028, enabling us to focus our resources on advancement of this potential therapy for the patients and families who urgently need new treatment options." Dr. McMinn continued, "In leveraging the continual dialogue under the START program, the FDA encouraged the analysis of the Embolden primary endpoint to remain at 12 months as the basis for full approval, noting that a 6-month endpoint may not be considered clinically meaningful. In further maintaining the rigorous design of the Embolden trial, we are electing to dose the last planned participant from the Phase 1/2 trial as part of the registrational Embolden trial and add one more participant to complete the proposed sample size at 20 patients." Second Quarter 2025 and Recent Highlights, and Anticipated Milestones NGN-401 Gene Therapy for Treatment of Rett Syndrome Received written agreement from the U.S. Food and Drug Administration (FDA) on key elements of the Embolden registrational trial of NGN-401 and refined...

Welcome to this week’s installment of “The Short Interest Report” – The Fly’s weekly recap of short interest trends among some of the most widely followed high-short-float stocks. Using the data from our partner Ortex.com, which utilizes the latest information from stock lenders to estimate short interest changes for thousands of publicly traded companies, this report will screen for some of biggest changes in short interest as a percentage of free float and days-to-cover ratios while also considering the short interest data on some of the more volatile and heavier-traded names of the week. Based on the availability of data from Ortex, the report tracks the trading period that covers prior Friday through Thursday of this week, excluding holidays. As a basis of comparison for stocks discussed below, the S&P 500 index was down 1.3%, the Nasdaq Composite was down 1.0%, the Russell 2000 index was down 2.4%, the Russell 2000 Growth ETF (IWO) was down 1.1%, and the Russell 2000 Value ETF (IWN) was down 3.0% in the five-day trading session range through May 22. Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter SHORT INTEREST GAINERS Otex-reported short interest in Krispy Kreme (DNUT) continues to soar as its stock continues to sink following another disappointing earnings report by the company on May 8th. Shorts as a percentage of free float jumped five percentage points to a record high of 32.2, with days to cover also increasing from 5.1 to 5.8 despite the spike in trading volume post-earnings. The company missed on the top line in Q1, offered below-consensus Q2 guidance, and also pulled its FY25 guidance due to “macroeconomic softness and the uncertainty around the McDonald’s deployment schedule”. The stock was 8% in the five-day period covered through Thursday, but has fallen 33% overall from levels just prior to Q1 earnings and is now down over 70% year-to-date. Shares of Rocket Companies (RKT) treaded water this week as the run-up in interest rates appeared to slow, though – as highlighted last week – the propulsion in Ortex-reported short interest on the stock is persisting despite the absence of pronounced pressure in price. Shorts as a percentage of free float on the stock was up another six percentage points...

On track to provide registrational trial plan update for NGN-401 gene therapy for Rett syndrome in the first half of 2025 Continues to plan to provide clinical data update from ongoing NGN-401 Phase 1/2 trial in the second half of 2025 Cash runway into the second half of 2027 NEW YORK, May 09, 2025--(BUSINESS WIRE)--Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced first quarter 2025 financial results and highlighted recent corporate updates. "We have made significant progress in planning for our future registrational trial with NGN-401 gene therapy for Rett syndrome and expect to provide an update on our plans during the first half of this year," said Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. "As we work to finalize our proposed trial design to differentiate and position the product candidate as potentially best-in-class for future commercialization, we appreciate the continued collaboration with the FDA. We also advanced the Phase 1/2 trial of NGN-401, and we look forward to sharing updated interim clinical data from the trial in the second half of 2025." First Quarter 2025 and Recent Highlights, and Anticipated Milestones Phase 1/2 Trial of NGN-401 Gene Therapy for Treatment of Rett Syndrome NGN-401 is currently being evaluated in an open-label Phase 1/2 trial. Key updates include: Remains on track to provide a regulatory update on registrational trial plans in the first half of 2025 Enrolling additional participants in the Phase 1/2 trial, and continues to expect to report additional interim clinical data from the trial in the second half of 2025; the trial includes a pediatric cohort of females aged 4-10 years old (N=8) and an older cohort of females aged 11 years and older (N=3) Announced a peer-reviewed publication in Science Translational Medicine providing proof-of-concept for Neurogene’s EXACT™ transgene regulation technology and showing promising tolerability and efficacy in nonclinical studies compared to conventional gene therapy; the publication details EXACT’s ability to regulate transgene expression to a tolerable and therapeutic level in non-clinical studies, which led to the development of NGN-401 Upcoming Events American Society of Gene and Cell Therapy (ASGCT) Annual Meeting:...

Remains on track to provide regulatory update on registrational trial plans with NGN-401 for Rett syndrome in the first half of 2025 Continues to expect to share additional interim clinical data from NGN-401 Phase 1/2 trial in the second half of 2025 Received PRIME designation for NGN-401 from the European Medicines Agency Cash runway into the second half of 2027 NEW YORK, March 24, 2025--(BUSINESS WIRE)--Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced fourth quarter and full year 2024 financial results and highlighted recent corporate updates. "We believe 2024 was a year of significant progress for our NGN-401 Phase 1/2 trial in Rett syndrome, as we reported encouraging interim efficacy data and NGN-401 was generally well-tolerated at the 1E15 vg dose in a pediatric cohort," said Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. "We remain on track to provide an update on the registrational trial plans in the first half of 2025, and as we look ahead to the second half of 2025, we plan to share additional interim clinical data from the Phase 1/2 trial, including from our 11 years and older cohort." Fourth Quarter 2024 and Recent Highlights, and Anticipated Milestones Phase 1/2 Trial of NGN-401 Gene Therapy for Treatment of Rett Syndrome NGN-401 is currently being evaluated in an open-label Phase 1/2 trial. Key updates include: Protocol amended and submitted to U.S. Food and Drug Administration (FDA) with the 1E15 vector genomes (vg) dose for all future participants in the trial, and enrollment is ongoing; 1E15 vg is the dose Neurogene expects to evaluate in a future registrational trial of NGN-401 based on positive interim efficacy results presented in November 2024 Expanded the age range in the trial to evaluate NGN-401 in a broader population, including ages > 11 years in the older pilot cohort (n=3), from the previous design of > 16 years; the pilot cohort is in addition to the 4 to 10 years cohort (n=8) Previously shared the Company gained alignment with the U.S. Food and Drug Administration (FDA) on chemistry, manufacturing and controls (CMC) scale-up plans to support NGN-401 potential commercial launch and potency assay strategy for the program Remains on track to provide a regulatory update on...