NGNE — what changed in the latest 10-Q

Text added vs the prior filing · source: 10-Q · 2026-05-12

• Our first clinical-stage program, NGN-401, is in development for the treatment of Rett syndrome, a severe and progressive neurodevelopmental disease with substantial neurological and physical impairments and significant unmet need. NGN-401 is purposefully designed to utilize the EXACT platform and a…

• We received clearance of our Investigational New Drug (“IND”) application by the U.S. Food and Drug Administration (“FDA”) in January 2023. We completed enrollment in the Phase 1/2 open-label, multi-center clinical trial evaluating NGN-401 for the treatment of female patients with classic Rett syndr…

• In November 2025, we announced updated positive interim clinical data from the Phase 1/2 NGN-401 trial in the pediatric cohort (ages 4-10) receiving the 1E15 vg dose (n=8 for efficacy data; n=10 for safety data, including pediatric and adolescent/adult participants) with a data cutoff date of Octobe…

• All pediatric participants, regardless of baseline disease severity, experienced functional gains, with an aggregate 35 developmental milestones gained across core clinical domains of Rett syndrome, including hand function/fine motor, language/communication and ambulation/gross motor. Participants w…

• As of October 30, 2025, four out of five participants with at least 12 months of follow-up met the responder definition of the primary endpoint planned for assessment at Month 12 in the Embolden trial. The three participants with six months of follow-up have also showed early clinical activity, cons…

Text removed vs the prior filing · source: 10-Q · 2025-11-13

• Our first clinical-stage program to utilize the EXACT platform is NGN-401, which is in development for the treatment of Rett syndrome, a disease with a patient population that has a significant unmet need, and that ultimately progresses to substantial neurological and physical impairment and prematu…

• We received clearance of our Investigational New Drug (“IND”) application by the U.S. Food and Drug Administration (“FDA”) in January 2023.

• In June 2025, we first announced written agreement from the FDA on key elements of the NGN-401 Embolden registrational trial design, and we confirmed these elements and the trial design in September 2025. The primary endpoint is a responder-based composite endpoint that will assess an improvement in…

• The Embolden trial is expected to enroll across 13 sites and completion of enrollment in the trial is expected in three to six months. We previously gained alignment with the FDA on our potency assay strategy and chemistry, manufacturing and control (“CMC”) scale-up planning for the program.

• In November 2025, we announced updated positive interim clinical data from the Phase 1/2 NGN-401 clinical trial in the pediatric cohort (ages 4-10) of participants receiving the 1E15 vg dose (n=8 for efficacy data; n=10 for safety data which includes both pediatric and adolescent/adult participants)…

Text added vs the prior filing · source: 10-Q · 2026-05-12

• •NGN-401 is in clinical development and our other programs are in early stages of development and may fail in development or suffer delays that materially and adversely affect their commercial viability. If we are unable to complete development of, or commercialize, our product candidates, or experi…

• We are substantially dependent on the success of our most advanced product candidate, NGN-401, and our clinical trial of NGN-401 may not be successful.

• Our future success is substantially dependent on our ability to timely obtain marketing approval for, and then successfully commercialize, our most advanced product candidate, NGN-401. We are investing a majority of our efforts and financial resources into the research and development of this produc…

• NGN-401 will require additional clinical development, evaluation of clinical, preclinical and manufacturing activities, marketing approval in multiple jurisdictions, substantial investment and significant marketing efforts before we generate revenues from product sales, if any. We are not permitted …

• The success of NGN-401 will depend on a variety of factors. We do not have complete control over many of these factors, including certain aspects of clinical development and the regulatory submission process, potential threats to our intellectual property rights and the manufacturing, marketing, dis…

Text removed vs the prior filing · source: 10-Q · 2025-11-13

• •NGN-401 is in clinical development and our other programs are in early stages of development and may fail in development or suffer delays that materially and adversely affect their commercial viability. If we or our current or future collaborators are unable to complete development of, or commercia…

• If safety concerns develop with respect to our product candidates or clinical trial designs, we may be delayed in our development plans as we may need to pause our enrollment in a clinical trial, revise our trial designs, investigate potential safety developments, or take other measures that may inc…

• We are substantially dependent on the success of our most advanced product candidate, NGN-401, and our clinical trial of NGN-401 may not be successful.

• Our future success is substantially dependent on our ability to timely obtain marketing approval for, and then successfully commercialize, our most advanced product candidate, NGN-401. We are investing a majority of our efforts and financial resources into the research and development of this produc…

• NGN-401 will require additional clinical development, evaluation of clinical, preclinical and manufacturing activities, marketing approval in multiple jurisdictions, substantial investment and significant marketing efforts before we generate revenues from product sales, if any. We are not permitted …

Text added vs the prior filing · source: 10-Q · 2026-05-12

• During the quarter ended March 31, 2026, no director or Section 16 officer adopted, modified or terminated any Rule 10b5-1 trading arrangement or non-Rule 10b5-1 trading arrangement.

Text removed vs the prior filing · source: 10-Q · 2025-11-13

• On November 7, 2025, we entered into a Second Amendment to Attorned Sublease (the “Sublease Amendment”) between our wholly owned Nevada subsidiary, Neurogene Inc., and GTM Associates, LLC, effective as of November 1, 2025. The Sublease Amendment extends the term on our New York office space until Ja…

• The foregoing is a summary description of certain terms of the Sublease Amendment and does not purport to be complete, and is qualified in its entirety by reference to the full text of the Sublease Amendment, which will be filed as an exhibit to our Annual Report on Form 10-K for the fiscal year end…

• During the quarter ended September 30, 2025, no director or officer (as defined in Section 16 of the Exchange Act) adopted, modified or terminated any contract, instruction or written plan for the purchase or sale of our securities that was intended to satisfy the affirmative defense conditions of R…

• Aggregate Number of Shares of Common Stock to be Purchased or Sold Pursuant to Trading Arrangement

• (1) Except as indicated by footnote, each trading arrangement marked as a “Rule 10b5-1 Trading Arrangement” is intended to satisfy the affirmative defense of the Rule.