NGNE

Data from Phase 1/2 trial of NGN-401 for Rett syndrome showed 100% of participants improved in CGI-I score and gained ≥1 developmental milestone, with an average of 4.7 milestones per participant; no plateau and no milestone loss observed as of data cutoff date of June 16, 2026 Milestones gained in a progressive, stepwise sequence, suggesting a restart of development post-treatment Topline data from Embolden™ registrational trial anticipated in 2H 2027 NGN-401 at the 1E15 vg dose remains generally well-tolerated (N=35) as of data cutoff date of August 10, 2026 Initiated PPQ campaign to support BLA submission for NGN-401; completion of PPQ runs expected by end of 2026 Extended cash runway into 1Q 2029 with oversubscribed financing of approximately $144 million gross proceeds NEW YORK, August 11, 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 second quarter 2026 financial results and highlighted recent corporate updates. "The second quarter and recent months marked important progress across our NGN-401 program, highlighted by positive long-term Phase 1/2 data showing durable, multidomain developmental gains that have not been observed in the natural history of Rett syndrome," stated Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. "The clinically meaningful benefit observed across all participants, including 100% of treated participants improving on CGI-I and gaining an average of 4.7 developmental milestones, strengthens our confidence in the program as we advance toward topline Embolden™ registrational data, which are expected in the second half of 2027. With initiation of our PPQ campaign, ongoing preparations for a potential BLA submission and a strengthened balance sheet supporting commercial-readiness activities, we believe we are well positioned to bring NGN-401 to patients and families as quickly as possible, if approved." Second Quarter 2026 and Recent Highlights NGN-401 Gene Therapy for the Treatment of Rett Syndrome Reported positive long-term Phase 1/2 data as of the data cutoff date of June 16, 2026, which demonstrated developmental milestones were gained in a progressive, stepwise sequence, suggesting a restart of development post-treatment Presented on the Rett Syndrome Natural History Study analysis conducted to assess treatment effect in Embolden, which demonstrated that the likelihood of milestone gains ≥3 years of age is rare Completed successful dosing of 25 participants in Embolden; topline data anticipated in the second half of 2027 Announced today the Process Performance Qualification (PPQ) campaign was initiated in July 2026 to support planned Biologics License Application (BLA) submission; completion of PPQ runs expected by the end of 2026 NGN-401 at the 1E15 vg dose has been generally well-tolerated in the Phase 1/2 trial and Embolden (N=35) as of August 10, 2026 Additional Corporate Updates Executed an oversubscribed public follow-on offering of approximately $144 million in gross proceeds, including full exercise of the underwriters’ option to purchase additional shares, extending cash runway into the first quarter of 2029 Key Anticipated NGN-401 Milestones Report topline data from Embolden registrational trial in the second half of 2027 Complete BLA-enabling PPQ runs by year-end 2026 Continue commercial-readiness activities Second Quarter 2026 Financial Results Cash, Cash Equivalents and Short-Term Investments: Cash, cash equivalents and short-term investments as of June 30, 2026 were $225.4 million. Together with approximately $134.8 million of net proceeds received from our July 2, 2026 public offering, Neurogene had pro forma cash, cash equivalents and short-term investments of approximately $360.2 million and expect its current cash resources to fund planned operations into the first quarter of 2029. Research & Development (R&D) Expenses: R&D expenses were $25.5 million for the three months ended June 30, 2026, compared to $19.4 million for the three months ended June 30, 2025. The increase in R&D expenses for the three months ended June 30, 2026 was primarily driven by higher costs associated with the development of NGN-401, including Rett syndrome clinical trial activities and chemistry, manufacturing and controls (CMC) activities supporting the NGN-401 program. The increase also reflected higher employee-related expenses, primarily due to increased non-cash stock-based compensation expenses. The increase was partially offset by lower spending on the CLN5 Batten disease program and early discovery activities. General & Administrative (G&A) Expenses: G&A expenses were $11.2 million for the three months ended June 30, 2026, compared to $6.7 million for the three months ended June 30, 2025. The increase in G&A expenses was primarily driven by higher non-cash stock-based compensation expense, including a one-time catch-up charge associated with performance-based stock unit awards following achievement of specified corporate milestones. Additional increases were driven by corporate and pre-commercial activities, higher employee-related expenses and professional fees. Net Loss: Net loss was $34.5 million for the three months ended June 30, 2026, compared to $22.0 million for the three months ended June 30, 2025. About Neurogene Neurogene (NASDAQ: NGNE) is a clinical-stage biotechnology company focused on developing life-changing genetic medicines for people and their families impacted by devastating neurological diseases. The Company is using a biology-first approach paired with optimized delivery to develop purpose-built genetic medicines, including programs powered by its novel and proprietary EXACT™ transgene regulation technology. Neurogene is advancing its lead gene therapy program, NGN-401, as a potential best-in-class, one-time treatment for Rett syndrome. For more information, visit neurogene.com or follow on LinkedIn. About NGN-401 NGN-401 is an investigational AAV9 gene therapy in late-stage clinical development as a potential best-in-class, one-time treatment for Rett syndrome. It is the only clinical candidate to deliver the full-length human MECP2 gene and includes Neurogene’s EXACT™ transgene regulation technology, which is designed to deliver consistent, tightly controlled MeCP2 protein expression on a cell-by-cell basis. NGN-401 is delivered through intracerebroventricular administration to achieve the broadest targeting directly to the brain and nervous system based on nonclinical biodistribution data. NGN-401 is being evaluated in the Embolden™ registrational clinical trial. Data from the Phase 1/2 trial (as of June 16, 2026) have shown that participants experienced multidomain, durable gains with continued developmental milestone acquisition observed over time, and NGN-401 at the 1E15 vg dose has been generally well-tolerated. NGN-401 has received Breakthrough Therapy, Regenerative Medicine Advanced Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations and selection for the START Pilot Program from the U.S. Food and Drug Administration, Advanced Therapy Medicinal Product, Orphan and Priority Medicines designations from the European Medicines Agency and Innovative Licensing and Application Pathway designation from the United Kingdom Medicines and Healthcare products Regulatory Agency. Cautionary Note Regarding Forward-Looking Statements Statements in this press release are made as of the date of this press release. Neurogene does not undertake any obligation to make any updates to these statements to reflect events that occur or circumstances that arise after the date of this press release, except as may be required under applicable U.S. securities law. Statements in this press release which are not historical in nature are intended to be, and hereby are identified as, forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current expectations and beliefs of the management of Neurogene, as well as assumptions made by, and information currently available to, management of Neurogene, including, but not limited to, statements regarding: the therapeutic potential and utility, efficacy and clinical benefits of NGN-401; the safety and tolerability profile of NGN-401; the applicability of reported interim results from the NGN-401 Phase 1/2 clinical trial to other participants; the potential for NGN-401 to be a best-in-class gene therapy for Rett syndrome; trial designs and clinical development plans for the Company’s Embolden registrational clinical trial of NGN-401 for Rett syndrome, including the expected timeline of its PPQ activities for its CMC requirements; the response rate, expected durability and deepening of clinical data results from our NGN-401 clinical trial; the potential for future approval for commercialization of NGN-401 as a treatment for Rett syndrome; commercial launch readiness for NGN-401; expected timing for release of topline data from the Company’s registrational trial of NGN-401; the potential for success of the Embolden registrational clinical trial of NGN-401 for Rett syndrome; the clinical benefit of delivering NGN-401 via intracerebroventricular administration; expected future interactions with or positions of the FDA, including the timing and outcome of any such interactions and anticipated benefits of any regulatory designation for NGN-401, including the FDA’s Breakthrough Therapy designation, Rare Pediatric Disease designation, RMAT designation and participation in the FDA’s START program; expected timing and plans for a BLA submission for NGN-401; and the time period over which existing cash resources may be sufficient to fund the Company’s operations. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as "may," "will," "should," "would," "expect," "anticipate," "plan," "likely," "believe," "estimate," "project," "intend," "on track," and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Forward-looking statements are based on current beliefs and assumptions that are subject to risks, uncertainties and assumptions that are difficult to predict with regard to timing, extent, likelihood, and degree of occurrence, which could cause actual results to differ materially from anticipated results and many of which are outside of Neurogene’s control. Such risks, uncertainties and assumptions include, among other things: the potential for negative impacts to participants in the Phase 1/2 clinical trial of NGN-401 for the treatment of Rett syndrome; the risk that the Company may not be able to report data on the predicted timeline; risks related to the Company’s ability to obtain regulatory approval for, and ultimately commercialize, its product candidates, including NGN-401; and other risks and uncertainties identified under the heading "Risk Factors" included in Neurogene’s Annual Report on Form 10-K for the year ended December 31, 2025, filed with the Securities and Exchange Commission ("SEC") on March 24, 2026, the Quarterly Report on Form 10-Q for the quarter ended March 31, 2026, filed with the SEC on May 12, 2026, and other filings that the Company has made and may make with the SEC in the future. Nothing in this communication should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that the contemplated results of any such forward-looking statements will be achieved. Forward-looking statements in this communication speak only as of the day they are made and are qualified in their entirety by reference to the cautionary statements herein. Except as required by applicable law, Neurogene undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise. This communication contains hyperlinks to information that is not deemed to be incorporated by reference into this communication. - Financial Tables Follow - View source version on businesswire.com: https://www.businesswire.com/news/home/20260811540370/en/ Contacts Media Contact: Mike DevineExecutive Director, Corporate [email protected] Investor Contact: Lina LiExecutive Director, Investor [email protected]

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 regions central to Rett syndrome pathophysiology, as compared with intrathecal lumbar (IT-L) administration Additional Corporate Updates Strengthened the leadership team with the appointment of Christy Shafer as Chief Commercial Officer and Christine Mikail, J.D., President and CFO of Neurogene, to the Board of Directors Ms. Shafer brings more than 20 years of industry experience building and leading high-performing commercial organizations and launching therapies for rare neurological diseases Ms. Mikail brings to the Board deep expertise in strategic transactions, capital formation, and corporate development, with a track record of building and positioning companies for long-term value creation Key Anticipated NGN-401 Milestones in 2026 Complete dosing of participants in the Embolden registrational trial in the second quarter of 2026 Present updated interim safety and efficacy data on the pediatric and adolescent/adult cohorts from the Phase 1/2 trial, including at least 12 months of follow-up for all 10 participants, in mid-2026 Initiate Process Performance Qualification (PPQ) campaign in mid-2026 Continue early commercial-readiness activities Upcoming Events H.C. Wainwright 4th Annual BioConnect Investor Conference: Management will participate in a fireside chat at 11:30 a.m. ET on May 19 and participate in investor meetings Goldman Sachs 47th Annual Global Healthcare Conference: Management will participate in a fireside chat at 2:40 p.m. ET on June 8 and participate in investor meetings First Quarter 2026 Financial Results Cash, Cash Equivalents and Short-Term Investments: Cash, cash equivalents and short-term investments as of March 31, 2026 were $243.2 million and are expected to fund planned operations through the first quarter of 2028. Research & Development (R&D) Expenses: R&D expenses were $25.2 million for the three months ended March 31, 2026, compared to $17.8 million for the three months ended March 31, 2025. The increase in R&D expenses for the three months ended March 31, 2026 was primarily driven by an increase in Rett syndrome clinical trial costs of NGN-401, chemistry, manufacturing and controls (CMC) costs to support NGN-401 and employee-related expenses due to an increase in R&D headcount. The increase was partially offset by decreases in spending on the CLN5 Batten disease program and early discovery. General & Administrative (G&A) Expenses: G&A expenses were $8.2 million for the three months ended March 31, 2026 and the three months ended March 31, 2025. Higher costs related to corporate and pre-commercial activities, along with increased employee-related expenses and professional fees, were offset by a decrease in non-cash stock-based compensation expense. Net Loss: Net loss was $30.9 million for the three months ended March 31, 2026, compared to $22.6 million for the three months ended March 31, 2025. About Neurogene Neurogene (NASDAQ: NGNE) is a clinical-stage biotechnology company focused on developing life-changing genetic medicines for people and their families impacted by devastating neurological diseases. The Company is using a biology-first approach paired with optimized delivery to develop purpose-built genetic medicines, including programs powered by its novel and proprietary EXACT™ transgene regulation technology. Neurogene is advancing its lead gene therapy program, NGN-401, as a potential best-in-class, one-time treatment for Rett syndrome. For more information, visit neurogene.com or follow on LinkedIn. About NGN-401 NGN-401 is an investigational AAV9 gene therapy in late-stage clinical development as a potential best-in-class, one-time treatment for Rett syndrome. It is the only clinical candidate to deliver the full-length human MECP2 gene and includes Neurogene’s EXACT™ transgene regulation technology, which is designed to deliver consistent, tightly controlled MeCP2 protein expression on a cell-by-cell basis. NGN-401 is delivered through intracerebroventricular administration to achieve the broadest targeting directly to the brain and nervous system based on nonclinical biodistribution data. NGN-401 is being evaluated in the Embolden™ registrational clinical trial. Interim data from the Phase 1/2 trial (as of October 30, 2025) have shown that participants experienced multidomain, durable gains with continued skill acquisition observed over time, and NGN-401 at the 1E15 vg dose has been generally well-tolerated. NGN-401 has received Breakthrough Therapy, Regenerative Medicine Advanced Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations and selection for the START Pilot Program from the U.S. Food and Drug Administration, Advanced Therapy Medicinal Product, Orphan and Priority Medicines designations from the European Medicines Agency and Innovative Licensing and Application Pathway designation from the United Kingdom Medicines and Healthcare products Regulatory Agency. Cautionary Note Regarding Forward-Looking Statements Statements in this press release are made as of the date of this press release. Neurogene does not undertake any obligation to make any updates to these statements to reflect events that occur or circumstances that arise after the date of this press release, except as may be required under applicable U.S. securities law. Statements in this press release which are not historical in nature are intended to be, and hereby are identified as, forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current expectations and beliefs of the management of Neurogene, as well as assumptions made by, and information currently available to, management of Neurogene, including, but not limited to, statements regarding: the therapeutic potential and utility, efficacy and clinical benefits of NGN-401; the safety and tolerability profile of NGN-401; the applicability of reported interim results from the NGN-401 Phase 1/2 clinical trial to other participants or potential participants, including adolescent or adult patients; the potential for NGN-401 to be a best-in-class gene therapy for Rett syndrome; trial designs and clinical development plans for the Company’s Embolden registrational clinical trial of NGN-401 for Rett syndrome, including timing of anticipated dosing and completion of participant dosing in the Company’s clinical trial and the expected timeline of its PPQ activities for its CMC requirements; the response rate, expected durability and deepening of clinical data results from our NGN-401 clinical trial; the potential for future approval for commercialization of NGN-401 as a treatment for Rett syndrome; commercial launch readiness for NGN-401; expected timing for release of additional data from the Company’s Phase 1/2 clinical trial of NGN-401; the potential for success of the Embolden registrational clinical trial of NGN-401 for Rett syndrome; the clinical benefit of delivering NGN-401 via ICV administration; expected future interactions with or positions of the FDA, including the timing and outcome of any such interactions and anticipated benefits of any regulatory designation for NGN-401, including the FDA’s Breakthrough Therapy designation, Rare Pediatric Disease designation, RMAT designation and participation in the FDA’s START program; and the time period over which existing cash resources may be sufficient to fund the Company’s operations. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as "may," "will," "should," "would," "expect," "anticipate," "plan," "likely," "believe," "estimate," "project," "intend," "on track," and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Forward-looking statements are based on current beliefs and assumptions that are subject to risks, uncertainties and assumptions that are difficult to predict with regard to timing, extent, likelihood, and degree of occurrence, which could cause actual results to differ materially from anticipated results and many of which are outside of Neurogene’s control. Such risks, uncertainties and assumptions include, among other things: the potential for negative impacts to participants in the Phase 1/2 clinical trial of NGN-401 for the treatment of Rett syndrome; the risk that the Company may not be able to report data on the predicted timeline; risks related to the Company’s ability to obtain regulatory approval for, and ultimately commercialize, its product candidates, including NGN-401; risks related to timing of completing enrollment in the Embolden trial of NGN-401 for Rett syndrome; and other risks and uncertainties identified under the heading "Risk Factors" included in Neurogene’s Annual Report on Form 10-K for the year ended December 31, 2025, filed with the Securities and Exchange Commission ("SEC") on March 24, 2026, the Quarterly Report on Form 10-Q for the quarter ended March 31, 2026, filed with the SEC on May 12, 2026, and other filings that the Company has made and may make with the SEC in the future. Nothing in this communication should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that the contemplated results of any such forward-looking statements will be achieved. Forward-looking statements in this communication speak only as of the day they are made and are qualified in their entirety by reference to the cautionary statements herein. Except as required by applicable law, Neurogene undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise. This communication contains hyperlinks to information that is not deemed to be incorporated by reference into this communication. - Financial Tables Follow - View source version on businesswire.com: https://www.businesswire.com/news/home/20260512420099/en/ Contacts Media Contact: Mike Devine Executive Director, Corporate Communications [email protected] Investor Contact: Lina Li Executive Director, Investor Relations [email protected]

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 (BLA) submission, confirmed with the FDA that commercial manufacturing scale is the same as current clinical manufacturing scale, removing the need for comparability studies Initiated all 13 U.S. clinical trial sites conducting Embolden, with ongoing activities to convert these sites to commercial sites at launch Completed rigorous market payor research confirming strong reimbursement potential of NGN-401 Reported positive interim data from the pediatric cohort (ages 4-10 cohort) of the Phase 1/2 trial, which demonstrated multidomain, durable gains with continued developmental milestone acquisition as of the data cutoff date of October 30, 2025 Key Anticipated Milestones in 2026 Complete dosing of participants in the Embolden registrational trial in the second quarter of 2026 Present updated interim safety and efficacy data on the pediatric cohort and the adolescent/adult cohort from the Phase 1/2 trial in mid-2026, including at least 12 months of follow-up for all 10 participants Initiate Process Performance Qualification (PPQ) campaign in mid-2026 Initiate additional early commercial-readiness activities Full Year 2025 Financial Results Cash, Cash Equivalents and Short-Term Investments: Cash, cash equivalents and short-term investments as of December 31, 2025 were $269.0 million and are expected to fund planned operations through the first quarter of 2028. Research & Development (R&D) Expenses: R&D expenses were $75.0 million for the twelve months ended December 31, 2025, compared to $60.9 million for the twelve months ended December 31, 2024. The increase in R&D expenses for the twelve months ended December 31, 2025 was primarily driven by an increase in Rett syndrome clinical trial costs and employee-related expenses due to an increase in R&D headcount, and it was partially offset by decreases in spending on the CLN5 Batten disease program and early discovery. General & Administrative (G&A) Expenses: G&A expenses were $28.3 million for the twelve months ended December 31, 2025, compared to $22.6 million for the twelve months ended December 31, 2024. The increase in G&A expenses for the twelve months ended December 31, 2025 was primarily driven by an increase in employee-related expenses due to an increase in non-cash stock-based compensation and headcount. Net Loss: Net loss was $90.4 million for the twelve months ended December 31, 2025, compared to $75.1 million for the twelve months ended December 31, 2024. About Neurogene Neurogene (NASDAQ: NGNE) is a clinical-stage biotechnology company focused on developing life-changing genetic medicines for people and their families impacted by devastating neurological diseases. The Company is using a biology-first approach paired with optimized delivery to develop purpose-built genetic medicines, including programs powered by its novel and proprietary EXACT™ transgene regulation technology. Neurogene is advancing its lead gene therapy program, NGN-401, as a potential best-in-class, one-time treatment for Rett syndrome. For more information, visit neurogene.com or follow on LinkedIn. About NGN-401 NGN-401 is an investigational AAV9 gene therapy in late-stage clinical development as a potential best-in-class, one-time treatment for Rett syndrome. It is the only clinical candidate to deliver the full-length human MECP2 gene and includes Neurogene’s EXACT™ transgene regulation technology, which is designed to deliver consistent, tightly controlled MeCP2 protein expression on a cell-by-cell basis. NGN-401 is delivered through intracerebroventricular administration to achieve the broadest targeting directly to the brain and nervous system based on nonclinical biodistribution data. NGN-401 is being evaluated in the Embolden™ registrational clinical trial. Interim data from the Phase 1/2 trial (as of October 30, 2025) have shown that participants experienced multidomain, durable gains with continued skill acquisition observed over time, and NGN-401 at the 1E15 vg dose has been generally well-tolerated. NGN-401 has received Breakthrough Therapy, Regenerative Medicine Advanced Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations and selection for the START Pilot Program from the U.S. Food and Drug Administration, Advanced Therapy Medicinal Product, Orphan and Priority Medicines designations from the European Medicines Agency and Innovative Licensing and Application Pathway designation from the United Kingdom Medicines and Healthcare products Regulatory Agency. Cautionary Note Regarding Forward-Looking Statements Statements in this press release are made as of the date of this press release. Neurogene does not undertake any obligation to make any updates to these statements to reflect events that occur or circumstances that arise after the date of this press release, except as may be required under applicable U.S. securities law. Statements in this press release which are not historical in nature are intended to be, and hereby are identified as, forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current expectations and beliefs of the management of Neurogene, as well as assumptions made by, and information currently available to, management of Neurogene, including, but not limited to, statements regarding: the therapeutic potential and utility, efficacy and clinical benefits of NGN-401; the safety and tolerability profile of NGN-401; the applicability of reported interim results from the NGN-401 Phase 1/2 clinical trial to other participants or potential participants, including adolescent or adult patients; the potential for NGN-401 to be a best-in-class gene therapy for Rett syndrome; trial designs and clinical development plans for the Company’s EmboldenTM registrational clinical trial of NGN-401 for Rett syndrome; trial designs and clinical development plans for the Company’s Embolden registrational clinical trial of NGN-401 for Rett syndrome, including timing of anticipated dosing and completion of participant dosing in our clinical trial and the expected timeline of our process performance qualification (PPQ) activities for our chemistry, manufacturing and controls (CMC) requirements; the response rate, expected durability and deepening of clinical data results from our NGN-401 clinical trial; the potential for future approval for commercialization of NGN-401 as a treatment for Rett syndrome, including the potential for third-party payor approval of reimbursement as well as the potential to convert existing clinical trial sites to commercial sites in the event NGN-401 does receive commercial approval; expected timing for release of additional data from our Phase 1/2 clinical trial of NGN-401; the potential for success of the Embolden registrational clinical trial of NGN-401 for Rett syndrome; the clinical benefit of delivering NGN-401 via intracerebroventricular administration; expected future interactions with or positions of the FDA, including the timing and outcome of any such interactions and anticipated benefits of any regulatory designation for NGN-401, including the FDA’s Breakthrough Therapy designation, Rare Pediatric Disease designation, RMAT designation and participation in the FDA’s START program; and the time period over which existing cash resources may be sufficient to fund the Company’s operations. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as "may," "will," "should," "would," "expect," "anticipate," "plan," "likely," "believe," "estimate," "project," "intend," "on track," and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Forward-looking statements are based on current beliefs and assumptions that are subject to risks, uncertainties and assumptions that are difficult to predict with regard to timing, extent, likelihood, and degree of occurrence, which could cause actual results to differ materially from anticipated results and many of which are outside of Neurogene’s control. Such risks, uncertainties and assumptions include, among other things: the potential for negative impacts to participants in the Phase 1/2 clinical trial of NGN-401 for the treatment of Rett syndrome; the risk that the Company may not be able to report data on the predicted timeline; risks related to the Company’s ability to obtain regulatory approval for, and ultimately commercialize, its product candidates, including NGN-401; risks related to timing of completing enrollment in the Embolden trial of NGN-401 for Rett syndrome; and other risks and uncertainties identified under the heading "Risk Factors" included in Neurogene’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2025, filed with the Securities and Exchange Commission ("SEC") on November 13, 2025, and other filings that the Company has made and may make with the SEC in the future. Nothing in this communication should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that the contemplated results of any such forward-looking statements will be achieved. Forward-looking statements in this communication speak only as of the day they are made and are qualified in their entirety by reference to the cautionary statements herein. Except as required by applicable law, Neurogene undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise. This communication contains hyperlinks to information that is not deemed to be incorporated by reference into this communication. - Financial Tables Follow - View source version on businesswire.com: https://www.businesswire.com/news/home/20260324872399/en/ Contacts Company Contact: Mike Devine Executive Director, Corporate Communications [email protected] Investor Contact: Argot Partners [email protected]

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 been generally well-tolerated in the pediatric cohort and the adolescent/adult cohort (ages ≥ 11 cohort) Dosed the first participant in the Embolden registrational trial, with complete enrollment expected in three to six months Initiated 12 of 13 Embolden sites; these sites could be rapidly converted into future commercial sites Presented new preclinical route of administration data at the ESGCT Congress highlighting that intracerebroventricular (ICV) delivery of NGN-401 achieved superior AAV biodistribution across brain regions relevant to Rett syndrome, compared to intrathecal lumbar (IT-L) delivery Findings support the best-in-class potential of NGN-401 and add to the body of evidence that ICV administration is superior to IT-L in reaching key brain regions underlying Rett syndrome; these data provide potential rationale for the multidomain skill acquisition and durable improvements observed in the Phase 1/2 NGN-401 trial These data provide evidence-based confirmation that ICV and IT-L administration resulted in comparable peripheral organ transduction, showing no liver-sparing benefit to IT-L when compared to ICV Third Quarter 2025 Financial Results Cash, Cash Equivalents and Short-Term Investments: Cash, cash equivalents and short-term investments as of September 30, 2025 were $265.4 million. We expect cash, cash equivalents and short-term investments to fund planned operations through the first quarter of 2028. Research & Development (R&D) Expenses: R&D expenses were $17.2 million for the three months September 30, 2025 compared to $16.3 million for the three months ended September 30, 2024. The increase in R&D expenses for the three months ended September 30, 2025 was primarily driven by an increase in Rett syndrome clinical trial costs and employee-related expenses due to an increase in R&D headcount, and was partially offset by decreases in spending on Batten disease and Early discovery. General & Administrative (G&A) Expenses: G&A expenses were $6.6 million for the three months ended September 30, 2025 compared to $5.9 million for the three months ended September 30, 2024. The increase in G&A expenses for the three months ended September 30, 2025 was primarily driven by an increase in employee-related expenses due to an increase in stock-based compensation and headcount. Net Loss: Net loss was $21.0 million for the three months ended September 30, 2025 compared to $20.2 million for the three months ended September 30, 2024. About Neurogene The mission of Neurogene is to treat devastating neurological diseases to improve the lives of patients and families impacted by these rare diseases. Neurogene is developing novel approaches and treatments to address the limitations of conventional gene therapy in central nervous system disorders. This includes selecting a delivery approach to maximize distribution to target tissues and designing products to maximize potency and purity for an optimized efficacy and safety profile. The Company’s novel and proprietary EXACT™ transgene regulation platform technology allows for the delivery of therapeutic levels while limiting transgene toxicity associated with conventional gene therapy. Neurogene has constructed a state-of-the-art gene therapy manufacturing facility in Houston, Texas. CGMP production of NGN-401 was conducted in this facility and will support pivotal clinical development activities. For more information, visit www.neurogene.com. About NGN-401 NGN-401 is an investigational AAV9 gene therapy being developed as a one-time treatment for Rett syndrome. It is the first clinical candidate to deliver the full-length human MECP2 gene under the control of Neurogene’s EXACT™ transgene regulation technology. EXACT technology is an important advancement in gene therapy for Rett syndrome, specifically because the disorder requires a treatment approach that enables targeted levels of MECP2 transgene expression without causing overexpression-related toxic effects associated with conventional gene therapy. NGN-401 was selected by the U.S. Food and Drug Administration (FDA) for its START Pilot Program and has also received Regenerative Medicine Advance Therapy (RMAT) designation, orphan drug designation, Fast Track designation and rare pediatric designation from the FDA. Neurogene was previously granted an INTERACT meeting with the FDA regarding the EXACT technology. NGN-401 also received Priority Medicines (PRIME) designation, orphan designation and advanced therapy medicinal product designation from the European Medicines Agency (EMA) and the Innovative Licensing and Application Pathway (ILAP) designation from the United Kingdom (UK) Medicines and Healthcare products Regulatory Agency (MHRA). Cautionary Note Regarding Forward-Looking Statements Statements in this press release are made as of the date of this press release. Neurogene does not undertake any obligation to make any updates to these statements to reflect events that occur or circumstances that arise after the date of this press release, except as may be required under applicable U.S. securities law. Statements in this press release which are not historical in nature are intended to be, and hereby are identified as, forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current expectations and beliefs of the management of Neurogene, as well as assumptions made by, and information currently available to, management of Neurogene, including, but not limited to, statements regarding: the therapeutic potential and utility, efficacy and clinical benefits of NGN-401, including the potential benefits of ICV administration as compared to IT-L administration for gene therapy in the treatment of Rett syndrome; trial designs, clinical development plans and timing for NGN-401, including expected timing of the completion of enrollment, the potential for future approval for commercialization of NGN-401 as a treatment for Rett syndrome, as well as the potential to convert existing clinical trial sites to commercial sites in the event NGN-401 does receive commercial approval; expected future interactions with or positions of the FDA, including the potential acceleration of submission of a BLA for NGN-401; and the time period over which existing cash resources may be sufficient to fund the Company’s operations. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as "may," "will," "should," "would," "expect," "anticipate," "plan," "likely," "believe," "estimate," "project," "intend," "on track," and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Forward-looking statements are based on current beliefs and assumptions that are subject to risks, uncertainties and assumptions that are difficult to predict with regard to timing, extent, likelihood, and degree of occurrence, which could cause actual results to differ materially from anticipated results and many of which are outside of Neurogene’s control. Such risks, uncertainties and assumptions include, among other things: the potential for negative impacts to participants in the Phase 1/2 clinical trial of NGN-401 for the treatment of Rett syndrome; the risk that the Company may not be able to report data on the predicted timeline; risks related to the Company’s ability to obtain regulatory approval for, and ultimately commercialize, its product candidates, including NGN-401; risks related to timing of completing enrollment in the Embolden trial of NGN-401 for Rett syndrome; and other risks and uncertainties identified under the heading "Risk Factors" included in Neurogene’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2025, filed with the Securities and Exchange Commission ("SEC") on November 13, 2025, and other filings that the Company has made and may make with the SEC in the future. Nothing in this communication should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that the contemplated results of any such forward-looking statements will be achieved. Forward-looking statements in this communication speak only as of the day they are made and are qualified in their entirety by reference to the cautionary statements herein. Except as required by applicable law, Neurogene undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise. This communication contains hyperlinks to information that is not deemed to be incorporated by reference into this communication. - Financial Tables Follow - View source version on businesswire.com: https://www.businesswire.com/news/home/20251113825408/en/ Contacts Company Contact: Mike Devine Executive Director, Corporate Communications [email protected] Investor Contact: Melissa Forst Argot Partners [email protected]

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 the sample size to propose 20 participants Initiated Embolden clinical trial activities to support the conversion of the Phase 1/2 trial to a registrational trial Completed enrollment in the Phase 1/2 trial, with the last 5 additional participants dosed in the first half of 2025 Remains on track to report updated clinical efficacy and safety data from the Phase 1/2 trial in the second half of 2025 Presented at scientific conferences the hemophagocytic lymphohistiocytosis (HLH) monitoring and treatment algorithm incorporated into the NGN-401 clinical trial, which has been acknowledged as valuable information by the Rett syndrome and gene therapy communities There has been no evidence of HLH/hyperinflammatory syndrome in any NGN-401 trial participant at the 1E15 vg dose level, as of the date of this press release Upcoming Events Stifel Biotech Summer Summit: Management will participate in a fireside chat at 12:00 p.m. ET on August 12 (not webcast) H.C. Wainwright Annual Global Investment Conference: Management will participate in a fireside chat at 2:30 p.m. ET on September 8 and participate in 1x1 meetings Second Quarter 2025 Financial Results Cash, Cash Equivalents and Short-Term Investments: Cash, cash equivalents and short-term investments as of June 30, 2025 were $274.5 million. We currently expect cash, cash equivalents and short-term investments to fund planned operations into early 2028. Research & Development (R&D) Expenses: R&D expenses were $19.4 million for the three months June 30, 2025 compared to $15.7 million for the three months ended June 30, 2024. The increase in R&D expenses for the three months ended June 30, 2025 was primarily driven by an increase in Rett syndrome clinical trial costs and employee-related expenses due to an increase in R&D headcount. General & Administrative (G&A) Expenses: G&A expenses were $6.7 million for the three months ended June 30, 2025 compared to $5.3 million for the three months ended June 30, 2024. The increase in G&A expenses for the three months ended June 30, 2025 was primarily driven by an increase in employee-related expenses due to an increase in stock-based compensation, headcount and other corporate expenses. Net Loss: Net loss was $22.0 million for the three months ended June 30, 2025 compared to $18.5 million for the three months ended June 30, 2024. About Neurogene The mission of Neurogene is to treat devastating neurological diseases to improve the lives of patients and families impacted by these rare diseases. Neurogene is developing novel approaches and treatments to address the limitations of conventional gene therapy in central nervous system disorders. This includes selecting a delivery approach to maximize distribution to target tissues and designing products to maximize potency and purity for an optimized efficacy and safety profile. The Company’s novel and proprietary EXACT™ transgene regulation platform technology allows for the delivery of therapeutic levels while limiting transgene toxicity associated with conventional gene therapy. Neurogene has constructed a state-of-the-art gene therapy manufacturing facility in Houston, Texas. CGMP production of NGN-401 was conducted in this facility and will support pivotal clinical development activities. For more information, visit www.neurogene.com. About NGN-401 NGN-401 is an investigational AAV9 gene therapy being developed as a one-time treatment for Rett syndrome. It is the first clinical candidate to deliver the full-length human MECP2 gene under the control of Neurogene’s EXACT™ transgene regulation technology. EXACT technology is an important advancement in gene therapy for Rett syndrome, specifically because the disorder requires a treatment approach that enables targeted levels of MECP2 transgene expression without causing overexpression-related toxic effects associated with conventional gene therapy. NGN-401 was selected by the U.S. Food and Drug Administration (FDA) for its START Pilot Program and has also received Regenerative Medicine Advance Therapy (RMAT) designation, orphan drug designation, Fast Track designation and rare pediatric designation from the FDA. Neurogene was previously granted an INTERACT meeting with the FDA regarding the EXACT technology. NGN-401 also received Priority Medicines (PRIME) designation, orphan designation and advanced therapy medicinal product designation from the European Medicines Agency (EMA) and the Innovative Licensing and Application Pathway (ILAP) designation from the United Kingdom (UK) Medicines and Healthcare products Regulatory Agency (MHRA). Cautionary Note Regarding Forward-Looking Statements Statements in this press release are made as of the date of this press release. Neurogene does not undertake any obligation to make any updates to these statements to reflect events that occur or circumstances that arise after the date of this press release, except as may be required under applicable U.S. securities law. Statements in this press release which are not historical in nature are intended to be, and hereby are identified as, forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current expectations and beliefs of the management of Neurogene, as well as assumptions made by, and information currently available to, management of Neurogene, including, but not limited to, statements regarding: the therapeutic potential and utility, efficacy and clinical benefits of NGN-401; trial designs, clinical development plans and timing for NGN-401, including elements of the registrational clinical study trial design subject to final approval of the FDA, such as the proposed number of participants in the Embolden trial, and the timing of the conversion of the NGN-401 Phase 1/2 clinical trial to a registrational clinical trial, anticipated timing of additional updates for the Embolden registrational trial of NGN-401 for Rett syndrome; expected timing for additional interim data from the Company’s NGN-401 Phase 1/2 trial for Rett Syndrome, expected future interactions with or positions of the FDA; and the time period over which existing cash resources may be sufficient to fund the Company’s operations. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as "may," "will," "should," "would," "expect," "anticipate," "plan," "likely," "believe," "estimate," "project," "intend," "on track," and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Forward-looking statements are based on current beliefs and assumptions that are subject to risks, uncertainties and assumptions that are difficult to predict with regard to timing, extent, likelihood, and degree of occurrence, which could cause actual results to differ materially from anticipated results and many of which are outside of Neurogene’s control. Such risks, uncertainties and assumptions include, among other things: the expected timing of additional results from the NGN-401 clinical trial; the potential for negative impacts to participants in the Phase 1/2 clinical trial of NGN-401 for the treatment of Rett syndrome; the risk that the Company may not be able to report data on the predicted timeline; risks related to the Company’s ability to obtain regulatory approval for, and ultimately commercialize, its product candidates, including NGN-401; risks related to timing of initiating the Embolden trial of NGN-401 for Rett syndrome; and other risks and uncertainties identified under the heading "Risk Factors" included in Neurogene’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, filed with the Securities and Exchange Commission ("SEC") on August 11, 2025, and other filings that the Company has made and may make with the SEC in the future. Nothing in this communication should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that the contemplated results of any such forward-looking statements will be achieved. Forward-looking statements in this communication speak only as of the day they are made and are qualified in their entirety by reference to the cautionary statements herein. Except as required by applicable law, Neurogene undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise. This communication contains hyperlinks to information that is not deemed to be incorporated by reference into this communication. - Financial Tables Follow - View source version on businesswire.com: https://www.businesswire.com/news/home/20250811382019/en/ Contacts Company Contact: Cara Mayfield Vice President, Corporate Affairs [email protected] Investor Contact: Melissa Forst Argot Partners [email protected]

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 to hit a four-year high above 44% and days-to-cover extended its increase by a decimal to 7.2. Shares, meanwhile, were down just 1.5% in the five-day period covered, and the year-to-date tally on Rocket is still a robust 13%. Ortex reported short interest in Neurogene (NGNE) had trended largely sideways from the last week of April through the middle of May just shy of the 60% level, though this past week has seen a spike all the way up to 87%. Volume has receded, though days-to-cover on the stock slipped from 17.3 to 15.3. The stock has had a positive couple of weeks, with a higher thrust on May 15th as Baker Bros Advisors disclosed a 10% stake and then earning a higher price target from BMO after an oral presentation at the American Society of Gene and Cell Therapy annual meeting describing a monitoring / treatment algorithm intended to reverse hyperinflammatory syndrome hemophagocytic lymphohistiocytosis. In the five-day period covered, Neurogene was down 6.9%, though relative to its April 8 low, the stock has more than doubled. Gains in Ortex-reported short interest in 1-800-Flowers (FLWS) have accelerated while the stock continued to track lower after reporting its Q3 results on May 8. Shorts as a percentage of free float rose sharply this week, increasing from 56% to about 67% – a record high. The stock, meanwhile, was up 2% in the five-period covered through Thursday, but including Friday’s 4% loss, shares of 1-800-Flowers are now down 19% post earnings and 43% year-to-date. SHORT INTEREST DECLINERS Ortex-reported short interest on Madrigal Pharmaceutical (MDGL) receded from about 40% to a 10-month low below 32% and days-to-cover slipped from 6.6 to 6.1 as bears chose to book profits in the stock following this month’s pullback. Madrigal shares slipped 12% over the 4-day period after its Q1 results on May 1 and are now down 20% from levels heading into those earnings, even though its loss was narrower than expected and several sell-side firms boosted their price targets due to strong Rezdiffra product sales. In the five-day period covered through Thursday, the stock was down 13%. Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>> See the top stocks recommended by analysts >> Read More on DNUT: Disclaimer & DisclosureReport an Issue Krispy Kreme (DNUT) Hit with Class Action Lawsuit Over McDonald’s Deal Fallout Krispy Kreme Faces Investor Lawsuit Over McDonald’s Sales JPMorgan downgrades Krispy Kreme on strategy reset, execution risk Krispy Kreme downgraded to Neutral from Overweight at JPMorgan Krispy Kreme price target lowered to $3.60 from $4.75 at Citi

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: Fireside chat: Reviving Hope in Deprioritized Cell and Gene Therapy Programs at 3:45 p.m. CT on May 14 Panel presentation: FDA’s START Pilot Program in Action: Insights from Year One at 3:45 p.m. CT on May 15 Oral presentation: Hemophagocytic Lymphohistiocytosis (HLH)/ Hyperinflammatory Syndrome Following High Dose AAV9 Therapy at 3:00 p.m. CT on May 16 H.C. Wainwright 3rd Annual BioConnect Investor Conference: Management will participate in a fireside chat at 9:30 a.m. ET on May 20 and participate in 1x1 meetings Goldman Sachs 46th Annual Global Healthcare Conference: Management will participate in a corporate presentation at 8:00 a.m. ET on June 11 and participate in 1x1 meetings First Quarter 2025 Financial Results Cash, Cash Equivalents and Short-Term Investments: Cash, cash equivalents and short-term investments as of March 31, 2025 were $292.6 million and are expected to provide runway into the second half of 2027, which would allow for the completion of enrollment of a future registrational trial for NGN-401 for Rett syndrome, chemistry, manufacturing and controls (CMC) scale-up to support NGN-401 registrational activities and further development of Neurogene’s EXACT gene therapy pipeline. Research & Development (R&D) Expenses: R&D expenses were $17.8 million for the three months ended March 31, 2025 compared to $13.5 million for the three months ended March 31, 2024. The increase in R&D expenses for the three months ended March 31, 2025 was primarily driven by an increase in Rett syndrome clinical trial costs and employee-related expenses due to an increase in headcount. General & Administrative (G&A) Expenses: G&A expenses were $8.2 million for the three months ended March 31, 2025 compared to $5.2 million for the three months ended March 31, 2024. The increase in G&A expenses for the three months ended March 31, 2025 was primarily driven by an increase in employee-related expenses due to an increase in headcount and other corporate expenses. Net Loss: Net loss was $22.6 million for the three months ended March 31, 2025 compared to $16.9 million for the three months ended March 31, 2024. About Neurogene The mission of Neurogene is to treat devastating neurological diseases to improve the lives of patients and families impacted by these rare diseases. Neurogene is developing novel approaches and treatments to address the limitations of conventional gene therapy in central nervous system disorders. This includes selecting a delivery approach to maximize distribution to target tissues and designing products to maximize potency and purity for an optimized efficacy and safety profile. The Company’s novel and proprietary EXACT™ transgene regulation platform technology allows for the delivery of therapeutic levels while limiting transgene toxicity associated with conventional gene therapy. Neurogene has constructed a state-of-the-art gene therapy manufacturing facility in Houston, Texas. CGMP production of NGN-401 was conducted in this facility and will support pivotal clinical development activities. For more information, visit www.neurogene.com. Cautionary Note Regarding Forward-Looking Statements Statements in this press release are made as of the date of this press release. Neurogene does not undertake any obligation to make any updates to these statements to reflect events that occur or circumstances that arise after the date of this press release, except as may be required under applicable U.S. securities law. Statements in this press release which are not historical in nature are intended to be, and hereby are identified as, forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current expectations and beliefs of the management of Neurogene, as well as assumptions made by, and information currently available to, management of Neurogene, including, but not limited to, statements regarding: the therapeutic potential and utility, efficacy and clinical benefits of NGN-401; trial designs, clinical development plans and timing for NGN-401, including anticipated timing of additional updates for a registrational trial of NGN-401 for Rett syndrome; expected timing for additional interim data from the Company’s NGN-401 Phase 1/2 trial for Rett Syndrome, expected future interactions with or positions of the FDA; and the time period over which existing cash resources may be sufficient to fund the Company’s operations. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as "may," "will," "should," "would," "expect," "anticipate," "plan," "likely," "believe," "estimate," "project," "intend," "on track," and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Forward-looking statements are based on current beliefs and assumptions that are subject to risks, uncertainties and assumptions that are difficult to predict with regard to timing, extent, likelihood, and degree of occurrence, which could cause actual results to differ materially from anticipated results and many of which are outside of Neurogene’s control. Such risks, uncertainties and assumptions include, among other things: risks related to the timing and success of enrolling patients in the Company’s Phase 1/2 clinical trial of NGN-401 for the treatment of Rett syndrome; the expected timing and results of dosing of patients in the NGN-401 clinical trial; the potential for negative impacts to participants in the Phase 1/2 clinical trial of NGN-401 for the treatment of Rett syndrome; the risk that the Company may not be able to report data on the predicted timeline; risks related to the Company’s ability to obtain regulatory approval for, and ultimately commercialize, its product candidates, including NGN-401; and other risks and uncertainties identified under the heading "Risk Factors" included in Neurogene’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, filed with the Securities and Exchange Commission ("SEC") on May 9, 2025, and other filings that the Company has made and may make with the SEC in the future. Nothing in this communication should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that the contemplated results of any such forward-looking statements will be achieved. Forward-looking statements in this communication speak only as of the day they are made and are qualified in their entirety by reference to the cautionary statements herein. Except as required by applicable law, Neurogene undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise. This communication contains hyperlinks to information that is not deemed to be incorporated by reference into this communication. - Financial Tables Follow - View source version on businesswire.com: https://www.businesswire.com/news/home/20250509094802/en/ Contacts Company Contact: Cara Mayfield Vice President, Corporate Affairs [email protected] Investor Contact: Melissa Forst Argot Partners [email protected]