Neurogene Inc. · NASDAQ:NGNE
Neurogene — Neoleukin Therapeutics — Merger or Acquisition (July 2023)
34 slides from Neurogene's merger presentation, Jul 18, 2023
Slide 1: Exhibit 99.2
Exhibit 99.2
Slide 2: Disclaimer
Disclaimer Forward Looking Statements cuties lows, ching ne US. Piva Secuis Litgation Retr Act of 195, Soncornng NeclevtnThetpeutes ine, (NeotuAr] Newogene ne. [Nectogere’} s Erope posed busi Torsactio’ and other mates, these forwarclecking stalemens include, but eno! ted, expres or imped sclement r
Slide 3: Disclaimer (Cont.)
Disclaimer (Cont.) Transaction. In connection withthe Proposed Transaction. cs oe etevant motercls wih ne Sec. nung aregsrotion sctement on Frm hol wil contain optonysiclement/prspecus of Neslovdh, NEOLEUEIN URGES INVESTORS AND STOCIHOLDERS TO READ IME REGITRATON STATEMENT. PFOXY STATEMENT/PROSPECTS
Slide 4: Merger Creates Clinical-Stage Genetic Medicines Company
Merger Creates Clinical-Stage Genetic Medicines Company with Multiple Value Creating Events ‘a ‘ JN neoleukiry Juevrocene 7 * Provides Neoleukin shareholders the opportunity to participate in the potential Neurogene Opportunity upside of the evolving Neurogene growth story Differentiated Genetic Med
Slide 5: Merger of Neurogene and Neoleukin
Merger of Neurogene and Neoleukin + Neurogene to merge with Neoleukin via an all stock transaction which is samen to close in Aor 23, + Combined company expected to have a cash balance of ~$ into 2H:26 + Includes $95 million from oversubscribed concurrent private financing by new ‘and existing healt
Slide 6: Neurogene is a Differentiated Clinical Stage Company Utilizing EXACT Technology
Neurogene is a Differentiated Clinical Stage Company Utilizing EXACT Technology to Treat Complex Neurological Diseases Novel EXACT technology designed to overcome key limitations of conventional gene therapy Pipeline addresses attractive market opportunities including Rett syndrome Internal manufact
Slide 7: Neurogene Pipeline
Neurogene Pipeline HD transgene Reguiction J Cns + Ocular Delivery Product : Reo lerny Indication Discovery IND Enabling Phase 1/2 Expected Milestones Candidate Preliminary Data NGN-401 Rett Syndrome Early Discovery Unalsclosed | o- Additional Data 2H:25 Preliminary Data 2H:24 NGN-101 CLN5 Batten Di
Slide 8: EXACT Developed to Solve the Limitations of Conventional Gene Therapy
EXACT Developed to Solve the Limitations of Conventional Gene Therapy in Complex Neurological Disorders day’s Gene Therapy Limited By: Neurogene’s Solutions: ox Variable Gene Expression Safety Limitations Inefficient Gene Delivery Novel, modular EXACT gene regulation technology and aS other regulato
Slide 9: Wholly-Owned and Fully Integrated In-House AAV Manufacturing
Wholly-Owned and Fully Integrated In-House AAV Manufacturing GMP Manufacturing Analytical Toxicology Batch Development Manufacturing NGN-401 Produced and Ready For Dosing Quality Process Control Development + Flexibility to manufacture AAV product at low cost + Manufacturing capability from research
Slide 10: Experienced Leadership Team Backed by Top Tier Investors
Experienced Leadership Team Backed by Top Tier Investors Management Team Rachel McMinn, Ph.D. Christine Mikail, J.D. Stuart Cobb, Ph.D. Founder and CEO. President and CFO cso Intercept] triste = geyant Leg, Dendrfi Cree Oesar— Albena Patroneva, M.D. Effie Albanis, M.! SVP, Clinical Development D. An
Slide 11: Leveraging EXACT gene regulation technology
Leveraging EXACT gene regulation technology
Slide 12: Rett Syndrome — Devastating Disorder with High Unmet Need
Rett Syndrome — Devastating Disorder with High Unmet Need Genetics =) + X-Linked disorder causing mutations in the gene encoding for methyl-CpG binding protein 2 (MeCP: One of the most common ede — of developmental and intellectual impairment in fem: + Unknown incidence in boys, but pity lethal by ~
Slide 13: Cardinal Clinical Features of Rett Syndrome
Cardinal Clinical Features of Rett Syndrome Inability to Impaired Fine and Autonomic Additional Disease Communicate Gross Motor Skills Dysfunction Manifestations * Loss of purposeful * Loss of hand function hand use & * Gait abnormalities involuntary hand + Ambulation requiring movements assistance
Slide 14: Rett Syndrome Treatment Requires Tight Gene Regulation
Rett Syndrome Treatment Requires Tight Gene Regulation MeCP2 Duplication Disorder** treatment goal gene expression drives disease + Rett syndrome (RTT) is a severe neurological disorder caused by mosaic mutations in X-linked MeCP2 gene + Mice modeling RTT recapitulate many neurological phenotypes ob
Slide 15: EXACT Acts As a Genetic Thermostat, Limiting Transgene Expression
EXACT Acts As a Genetic Thermostat, Limiting Transgene Expression d w ATW n EXACT miRNA controls fc i iii io transgene levels to targeted range [ener Regulatory elements 4) designed to avoid off- anu. TNT. target effects i ATINWUATIY. : ATTN. AIM Ze } EXACT enables gene Toy ™ < therapy for Rett rw u
Slide 16: EXACT Widens Therapeutic Window to Enable Gene Therapy for Rett Syndrome
EXACT Widens Therapeutic Window to Enable Gene Therapy for Rett Syndrome Variable AAV EXACT Dampens Wide Therapeutic Window Transduction Overexpression § QQu QQ» 0 ~50% of cells express WT levels of MeCP2 » ~50% are MeCP2 deficient Well tolerated in animal models Endogenous+NGN-401 Deficient [___] d
Slide 17: NGN-401 Demonstrates Efficacy and Safety in Mecp2 Mouse Models
NGN-401 Demonstrates Efficacy and Safety in Mecp2 Mouse Models ICV Delivery of NGN-401 Delivers Targeted MeCP2 Levels AAV? capsid preety RACI Full length human MECP2 Survival in Male Knockout Survival in Female Het a NGN-401 (regulated) 400: 15: ee ee = 15 = = = E so 3 50. unregulated 2 2 5 5 a. a D
Slide 18: NGN-401 Preclinical Data Enabled Pediatric Clinical Approach
NGN-401 Preclinical Data Enabled Pediatric Clinical Approach Strong efficacy, favorable safety DEMONSTRATED CONTROLLED MeCP2 LEVELS Robust MeCP2 levels to key brain areas PROVIDES TRANSLATIONAL FOUNDATION FOR HUMANS te ¥ N fe] has ived FDA cl Delivery of full-length MeCP2 MAXIMIZES THERAPEUTIC POTEN
Slide 19: Clinical Study For NGN-401 Designed to Evaluate Pediatric Population
Clinical Study For NGN-401 Designed to Evaluate Pediatric Population Dosing Initiates + Starting dose bracketed by two efficacious mouse doses, 2H:23 [ae > with > 4x safety margin from GLP tox study + Preparing for dose escalation, and cohort expansion to generate additional clinical data N=5S + Key
Slide 20: Treating both CNS and vision through dual route of administration
Treating both CNS and vision through dual route of administration
Slide 21: CLN5 Batten Disease - Fatal Neurodegenerative With No Disease Specific Treatment Options
CLN5 Batten Disease - Fatal Neurodegenerative With No Disease Specific Treatment Options Recurrent seizures or epilepsy Decline in intellectual function Vision deterioration over time cognition leading to blindness and Loss of language involuntary, 9p, and rhythmic sees ‘Speech impairment, loss of a
Slide 22: NGN-101 Dual Delivery Supported by Compelling Preclinical data
NGN-101 Dual Delivery Supported by Compelling Preclinical data Dual route of administration NGN-101 dosing (ICV+IVT) in CLN5 knockout sheep First clinical gene therapy study targeting both Combination dosing leads to halting of disease neurodegeneration and vision loss progression 30: = g$ ay @ 20:
Slide 23: Clinical Study Design For NGN-101 Addresses Vision and CNS
Clinical Study Design For NGN-101 Addresses Vision and CNS Enrolling High dose Fully enrolled » Dose selection based on sheep studies showing significant Low dose treatment effects N=4 » Key assessments every 6 and 12 months N=2 Key Eligibility Criteria Efficacy Endpoints/Markers of Interest + Age 2
Slide 24: NGN-101 — Defining a Registration Path
NGN-101 — Defining a Registration Path Regulatory meeting focused on finalizing CMC Regulatory interactions to define a registration path planned for 1H:24 plans expected toward end of 2023 Sheep model shows vision loss precedes motor deterioration Potency Assay Potency assay alignment necessary 6 p
Slide 25: Key Value Creation Events
Key Value Creation Events
Slide 26: Key Upcoming Anticipated Milestones and Pipeline Developments
Key Upcoming Anticipated Milestones and Pipeline Developments Rett syndrome (NGN-401) Q First patient dosing 2H:23 Preliminary Phase 1/2 clinical data 4Q:24 Q_ Additional Phase 1/2 clinical data 2H:25 CLN5 Batten disease (NGN-101) Q FDA feedback on CMC (toward end of 2023) and clinical trial design
Slide 27: Why Neurogene?
Why Neurogene? Unlocking multi-billion dollar neurological disease markets Proprietary and t enable complex diseases Strategy focused on efficiency and maximizing probability of success x) Leadership team with deep ional, logical and clinical Leading life sciences investor syndicate Strong balance s
Slide 28: Appendix
Slide 29: EXACT Technology Mechanism of Action
EXACT Technology Mechanism of Action + When high vector G 9 wv aT 7S uptake occurs, miRNA € aS) iE Lk. more robustly Lu binds excess transcripts v Te EON preventing translation wv * Transgene derived — protein expression TINUTTIN, remains at targeted oes levels a eu TRANSLATION Protein @ 6° Expresse
Slide 30: NGN-401 Demonstrates Tight MECP2 Regulation in Knockout Mouse Model
NGN-401 Demonstrates Tight MECP2 Regulation That Translates to Compelling Outcomes in a Knockout Mouse Model ICV Delivery of NGN-401 Delivers Targeted MECP2 Levels AAV? iS) Survival Clinical Score* a0. Wr venice > Mecp2” + Vehicle 3 a Mecpo + v Aan = gz Sf ee eS Key domains improved: q 3 15. + Motor
Slide 31: NGN-401 Via ICV Delivery Well Tolerated in Multiple Studies
NGN-401 Via ICV Delivery Well Tolerated in Multiple Studies While Conventional Unregulated Gene Therapy is Toxic NGN-401 Well Tolerated in Female Tight mRNA Levels in NHPs for NGN-401 Well Tolerated in NH ciel=e Mouse Model, Unregulated NGN-401, While Unregulated Has While Unregulai MeCP2 Highly Tox
Slide 32: NGN-401 Distribution and Expression Levels in NHPs Support Encouraging Profile
NGN-401 Distribution and Expression Levels in NHPs Support Encouraging Profile for Human Testing NGN-401 distributes to key regions underlying RTT pathophysiology in WT non-human primates Degree of mRNA expression tracks vector genome biodistribution of AAV9 across key brain regions Aggregate transg
Slide 33: GLP Toxicology in NHPs Support Favorable Safety Profile
GLP Toxicology in NHPs Support Favorable Safety Profile * NGN-401 evaluated in GLP NHP toxicology study with 90-day and 180-day cohorts No signs or symptoms of MeCP2 overexpression observed >4x safety margin relative to NGN-401 clinical starting dose in Phase 1/2 Overall toxicology profile consisten
Slide 34: Clinical Study For NGN-401 — FDA Authorization to Pursue a Pediatric-First Approach
Clinical Study For NGN-401 — FDA Authorization to Pursue a Pediatric-First Approach » The FDA typically requires therapies to demonstrate safety in adults prior to advancing into children » In order to get authorization for a pediatric-first approach, a therapy must demonstrate a strong safety packa
Neoleukin Therapeutics — Merger or Acquisition (July 2023) on 6ix