Neurogene Inc 2025年度报告

NGN-401 is an investigational AAV9 gene therapy in development as a potentialbest-in-class disease-modifying treatmentfor Rett syndrome.It is purposefully designed to deliver the full-lengthMECP2gene andincorporates Neurogene’s proprietaryEXACT™ transgene regulationtechnologyto achieve target therapeutic levels of MeCP2 protein. NGN-401is delivered through intracerebroventricular (ICV) administration, a commonneurosurgical procedure, which has been established in multiple preclinicalstudies to provide thebroadest gene therapy biodistributionto the keyareas of the brain and nervous system underlying Rett syndrome. Rachel McMinn, Ph.D.Founder and'LMIJ)\IGYXMZI3JɚGIV TO OUR SHAREHOLDERS, ZDVDWUDQVIRUPDWLRQDO\HDUIRU1HXURJHQHGHˋQHGE\clinical execution with NGN-401 gene therapy for Rett syndromeand the transition into a late-stage clinical development companyZLWKDGHˋQHGUHJXODWRU\SDWK2XUDFKLHYHPHQWVVLJQLˋFDQWO\advanced our mission to deliver life-changing medicines forSHRSOHDQGIDPLOLHVLPSDFWHGE\GHYDVWDWLQJQHXURORJLFDOGLVHDVHV ,Q1RYHPEHUZHUHSRUWHGXSGDWHGLQWHULP3KDVHFOLQLFDOGDWDIURPWKHSHGLDWULFFRKRUW DJHVQ  VKRZLQJDRQHWLPHtreatment of NGN-401 led to clinically meaningful, multidomainLPSURYHPHQWVWKDWZHUHGXUDEOHDQGFRQWLQXHGWRGHHSHQRYHUWLPH 2XUFOLQLFDOSURJUHVVZLWK1*1ZDVKLJKOLJKWHGE\WKHLQLWLDWLRQRIWKH(PEROGHQɐUHJLVWUDWLRQDOWULDODPDMRUPLOHVWRQHWKDWUHˌHFWVWKHVWUHQJWKRIRXU3KDVHGDWDDQGWKHHQWKXVLDVPIURPWKH5HWWV\QGURPHFRPPXQLW\(PEROGHQLVDVLQJOHDUPEDVHOLQHFRQWUROOHGRSHQODEHOWULDOHYDOXDWLQJNGN-401 in females with classic Rett syndrome ages three andROGHU%DVHGRQDULJRURXVDQDO\VLVRIWKH1,+VSRQVRUHG5HWW6\QGURPH1DWXUDO+LVWRU\6WXG\LWLVUDUHIRUDIHPDOHZLWK5HWWV\QGURPHWRJDLQRUUHJDLQDGHYHORSPHQWDOPLOHVWRQHDWRUDERYHDJHWKUHH7KHWULDOȢVSULPDU\HQGSRLQWLVDFRPSRVLWHRID&OLQLFDO*OREDO,PSUHVVLRQ,PSURYHPHQW &*,, VFRUHRI”3, which isa clinician-assessed measure of improvement, and a gain fromEDVHOLQHRIDQ\RQHGHYHORSPHQWDOPLOHVWRQHIURPDSUHVSHFLˋHGOLVWDWPRQWKV7KHOLVWRIGHYHORSPHQWDOPLOHVWRQHVZDVGHHPHGFOLQLFDOO\PHDQLQJIXOE\5HWWV\QGURPHFDUHJLYHUVDQGZHDSSUHFLDWHWKH5HWWV\QGURPHFRPPXQLW\ȢVRQJRLQJLQYROYHPHQWLQDGYLVLQJWKHGHVLJQRIRXUFOLQLFDOSURJUDP • 100% showed functional improvements across core diseaseHSQEMRWȦɚRIQSXSVLERHJYRGXMSRKVSWWQSXSVambulation and communication • 35 total developmental milestones gained• No plateau, including out to 24 months• Multidomain gains enable increasingly complex activities,enhancing independence and health-related quality of life %\DOLJQLQJZLWKWKH86)RRGDQG'UXJ$GPLQLVWUDWLRQ )'$ RQNH\HOHPHQWVRIWKHWULDOZHEHOLHYHZHKDYHDFOHDUSDWKWRUHJLVWUDWLRQ:HDUHWKHRQO\FRPSDQ\ZLWKDVLQJOHJHQHWKHUDS\WULDOGHVLJQHGWRJHQHUDWHERWKHIˋFDF\DQGVDIHW\GDWDGRZQWRDJHWKUHHWRVXSSRUWDEURDGODEHOLQ5HWWV\QGURPH:HEHOLHYHthese data will provide key stakeholders, including physicians,caregivers and payors, the necessary information to informtreatment at an age when families may want to intervene toPD[LPL]HEHQHˋW • 88% achieved improved CGI-I score• 1E15 vg dose continues to be generally well-tolerated As of data cutoff date of October 30, 2025 7KHVHGDWDUHLQIRUFHWKHWKHUDSHXWLFSRWHQWLDORI1*1WRPHDQLQJIXOO\DOWHUWKHGLVHDVHWUDMHFWRU\DQGLPSURYHGDLO\OLYLQJIRUIHPDOHVZLWK5HWWV\QGURPHDQGWKHLUFDUHJLYHUV 'XULQJZHDOVRPDGHLPSRUWDQWSURJUHVVZLWKOD\LQJWKHIRXQGDWLRQIRU1*1ȢVFRPPHUFLDOL]DWLRQ:HFRPSOHWHGUR-EXVWPDUNHWSD\RUUHVHDUFKFRQˋUPLQJWKHVWURQJUHLPEXUVHPHQWSRWHQWLDOIRU1*1DQHQFRXUDJLQJYDOLGDWLRQRIRXUEHOLHIWKDWZHDUHZHOOSRVLWLRQHGWRWUDQVIRUPDPXOWLELOOLRQGROODUPDUNHWWKDWFXUUHQWO\KDVQRGLVHDVHPRGLI\LQJWUHDWPHQW :HFRQWLQXHWRKDYHUHJXODURQJRLQJFRPPXQLFDWLRQZLWKWKH)'$WKURXJKRXUSDUWLFLSDWLRQLQWKH67$57 6XSSRUWIRU&OLQLFDO7ULDOV$GYDQFLQJ5DUH'LVHDVH7KHUDSHXWLFV 3LORW3URJUDP,QDGGLWLRQWKH)'$KDVJUDQWHG%UHDNWKURXJK7KHUDS\GHVLJQDWLRQIRU1*1EDVHGRQLWVUHYLHZRILQWHULPHIˋFDF\DQGVDIHW\GDWDIURPWKH3KDVHWULDOZKLFKZHEHOLHYHYDOLGDWHVWKHVWUHQJWKRI1*1ȢVFOLQLFDOSURˋOH :HDUHDOVRSUHSDULQJWKH5HWW6\QGURPH&HQWHUVRI([FHOOHQFHWKDWDUHSDUWLFLSDWLQJLQ(PEROGHQIRUIXWXUHFRQYHUVLRQWRFRPPHUFLDOVLWHV :HFRQWLQXHWREHZHOOFDSLWDOL]HGZLWKDQH[SHFWHGFDVKUXQZD\WKURXJKWKHˋUVWTXDUWHURI7KLVVWURQJˋQDQFLDOfoundation is anticipated to fund operations through keyPLOHVWRQHVLQFOXGLQJWKH(PEROGHQGDWDUHDGRXW%/$VXEPLVVLRQDQGNH\SUHODXQFKDFWLYLWLHV Looking ahead, we remain laser-focused on rapidly advancing1*1WRZDUGVFRPPHUFLDOL]DWLRQ,Q0DUFKZHDQQRXQFHGWKDWRISDUWLFLSDQWVKDYHEHHQHQUROOHGLQWKH(PEROGHQUHJLVWUDWLRQDOWULDOZLWKPRUHWKDQRIparticipants dosed, and