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CAMP4 Analyst Event September 28, 2026
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This presentation contains forward‐looking statements that are based on the beliefs and assumptions and on information currently available to CAMP4’s management. All statements other than statements of historical fact contained in this presentation are forward-looking statements. Forward‐looking statements include information concerning the company, corporate strategy and operational and development plans; plans and expectations regarding the design of, and potential endpoints for, the company’s clinical trials; the timing, progress and results of preclinical and clinical trials of CAMP4’s product candidates; the timing or outcome of regulatory interactions, filings and approvals for any of its product candidates; expectations regarding the clinical and therapeutic potential of CAMP4’s product candidates; estimates with respect to addressable patient populations and markets for the company’s product candidates; and estimates regarding CAMP4’s expenses, future revenues, and future capital requirements. In some cases, you can identify forward-looking statements because they contain words such as “may,” “might,” “will,” “would,” “shall,” “should,” “expects,” “plans,” “anticipates,” “could,” “intends,” “target,” “projects,” “contemplates,” “believes,” “estimates,” “looks,” “seeks,” “predicts,” “potential,” “ongoing,” or “continue” or the negative of these words or other similar terms or expressions that concern our expectations, strategy, plans or intentions, although not all forward-looking statements are accompanied by such words. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause CAMP4’s actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. This information was factually accurate on the date it was published. CAMP4 assumes no duty to update the information to reflect subsequent developments, except as required by law. The safety and efficacy of CAMP4’s product candidates and/or uses under investigation have not been established. There is no guarantee that any of our product candidates will receive regulatory authority approval or become commercially available in any country for the uses being investigated or that any such product candidate will achieve a particular revenue level. In particular, CAMP4’s expectations could be affected by, among other things, uncertainties involved in the development of new therapeutic products; unexpected clinical trial results or unexpected new clinical data; unexpected regulatory actions or delays or government regulation generally; CAMP4’s ability to obtain or maintain patent or other proprietary intellectual property protection; competition in general; CAMP4’s ability to establish and maintain collaborations, strategic relationships and supply arrangements, or to realize the intended benefits from such relationships or arrangements; whether CAMP4’s cash resources will be sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; CAMP4’s ability to raise additional funding on favorable terms, or at all; the rate and degree of market acceptance and clinical utility of CAMP4’s product candidates; the ability and willingness of our third-party collaborators to continue research, development and manufacturing activities relating to our product candidates; the accuracy of our data analyses or estimates for the potential and market for our products; and government, industry, and general public pricing and other political pressures. The actual results may vary from the anticipated results and the variations may be material. Other factors that may cause the Company’s actual results to differ from current expectations are discussed in the Company’s filings with the SEC, including the sections titled “Risk factors,” “Management’s discussion and analysis of financial condition and results of operations” and “Special note regarding forward-looking statements and market and industry data” in the Company’s most recent Annual Report on Form 10-K for the year ended December 31, 2025 and quarterly report on Form 10-Q for the quarter ended June 30, 2026. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date this presentation is given. Except as required by law, CAMP4 undertakes no obligation to publicly update any forward-looking statements, whether as a result of new information, future events or otherwise. 2 Forward-Looking Statements
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Agenda 3 • Welcome & Opening remarks | Josh Mandel-Brehm, CEO • Patient story: hearing from a SYNGAP1 parent | Beata Tarasiuk, CURE SYNGAP1 State Ambassador (AZ) • ASCEND Phase 1/2 clinical trial | Dr. Dan Tardiff, CSO, and Dr. Yuri Maricich, CMO • Patient advocacy | Mike Graglia, Co-Founder and Managing Director, CURE SYNGAP1 • KOL Fireside chat | Dr. Yuri Maricich & Dr. Stéphane Auvin, Epileptologist & Pediatric Neurologist • Emerging pipeline and closing remarks | Josh Mandel-Brehm • Analyst Q&A
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4 Solving genetic diseases of the brain through upregulation Lead Program Focused on SYNGAP1 >20k patients1 Broad-based pipeline of CNS-focused upregulation therapeutics in areas of high unmet need Today Future RAP Platform® Addressing the root cause of genetic disease by restoring healthy protein levels 1 Scaled annual incidence to prevalence using country specific live births and adjusted for mortality estimates; incidence ra tes based on Lopez Rivera et al. (2020)
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Video: Patient story 5
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ASCEND clinical trial in SYNGAP1 ASO therapy for SYNGAP1 Clinical Evaluation in Neuro Development 6
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Activators and repressors bind to regRNAs to control the expression of protein-coding genes regRNAs originate from enhancers and promoters ASOs disrupt the interactions between transcription factors and regRNAs enabling increases in gene expression 1 2 Increased mRNA addresses root cause of disease by returning targeted protein levels toward a healthy range 3 7 Source: Sharp et al. (2022) RNA 28: 52-57; Henninger et al. (2021) Cell 184:207-225; Sartorelli & Lauberth (2020) Nat. Struct. Mol. Biol. 27: 521-8; Oksuz et al. (2023) Molecular Cell 83: 2449-63. regRNAs play a central role in the regulation of every gene’s expression
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8 CMP-002 CAMP4 aims to increase SYNGAP protein levels, restore SYNGAP1 function, and improve disease symptoms Mutations in SYNGAP1 lead to decreased SYNGAP protein, causing increased synaptic firing CMP-002 binds to a SYNGAP-specific regRNA to increase SYNGAP1 expression, aiming to restore SYNGAP towards wild-type levels and normalize synaptic function
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CMP-002 restored SYNGAP levels in models of haploinsufficiency (SYNGAP-ZBTB9)1Bpro/Mmjax mouse strain obtained from the Mutant Mouse Resource and Research Centers (originally deposited by Benjamin Prosser, Ph.D., University of Pennsylvania). Dose-dependent increase in SYNGAP approaching wild-type levels → both ASO activity and regRNA function translated from human patient-derived neurons in a dish to neurons in an intact animal brain SYNGAP1 Patient iPSC-derived neurons “Humanized” SYNGAP1 mouse SYNGAP1 patient neurons + CMP-002 Familial control neurons + NTC SYNGAP1 patient neurons + NTC NTC: nontargeting control SYNGAP1 patient mutation: K1185X [Lysine → “stop”] • ICV dosing in at P21 • Takedown 2-wks post-dose 9 SYNGAP1 mRNA FC Normalized to Familial Control SYNGAP Protein FC Normalized to Homozygous Control Homozygous Control HaploinsufficientCMP-002 (nM)
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CMP-002 broadly improved neurological phenotypes in humanized SYNGAP1 mice 10 Motor FunctionLearning & Memory Hyperactivity Seizures Broad benefit of CMP-002 supports potential translation across multiple phenotypes in SYNGAP1 patients Healthy Haplo + ASO Haplo
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SYNGAP Protein Normalized to aCSF Frontal Cortex Intrathecal administration of CMP-002 to cynomolgus monkeys achieved broad brain distribution to increase SYNGAP protein levels 11 Summary IT administration in NHPs was well-tolerated Broad ASO distribution throughout disease- relevant brain regions ↑ SYNGAP protein throughout brain aCSF • N=4/group • Dose Q2w x3 • Take down 2 wks post last dose CMP-002 (3 dose levels) aCSF Low Mid High SYNGAP Protein Normalized to aCSF Hippocampus aCSF = artificial CSF *, p <0.05 **, p<0.01 ***, p<0.001 ****, p<0.0001 ASO Concentrations SYNGAP Protein Levels aCSF low mid high
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SYNGAP1 is a devastating disease with significant unmet need for a targeted disease modifying therapy Complex Symptoms No Approved Therapy Non-specific treatments have limited impact on SYNGAP1 symptoms • Anti-seizure medications • Cannabinoids • Sleep medications Polypharmacy is common – Patient regimen 6 example: • Epidiolex • Ravicti • Sodium bicarb • Amantadine Constant patient care needed • Caregivers vigilant at all times • Significant lifelong cost of care Developmental delay and/or intellectual disability • 100% of patients 1,2,3 Generalized epilepsy • ~85% of patients 3,4,5 Severe behavioral problems • ~70% of patients 1,5 Sleep problems • ~60% of patients 2,5 Limited communication • ~30% non-verbal, single words 4 12 1 Wiltrout, et al., Epilepsia, 2024; 2 Jimenez-Gomez, et al. J Neurodev Disord, 2019; 3 Holder et al., GeneReviews, 2019; 4 SYNGAP-Related Epilepsy, Epilepsy Foundation (Accessed May 2025); 5 Vlaskamp et al. Neurology, 2019; 6 CURE SYNGAP1 JAELI, 16
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Natural history studies and registries inform ASCEND clinical trial N = 1,806 SYNGAP1 Census as of Q2 2026 Advocacy-group collaboration ~200 new patients/year since 2020 N = 159 Prospective natural history as of Q2 2026 Ongoing study 2023–2028; Children’s Hospital Colorado, Stanford University, and CHOP N = 350 Retrospective EMR cohort as of Q3 2026 Curated real-world records, cohort still expanding N = 569 European registry (PATRE) as of Q3 2026 Broadens geographic representation Sources: SYNGAP1 Census Q2 2026 (curesyngap1.org); McKee et al.,2026; PATRE Q3 2026 (euras-project.eu).13 Sponsored 2,500+ patients characterized across census, prospective, real-world, and registry cohorts EURAS project, PATRE SYNGAP1 registry
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Majority of SYNGAP1 patients have severe protein-truncating variants 14 Multiple natural history studies have replicated highest burden of disease across multiple domains in PTV 5-19 genotypes SYNGAP1 GENE Exons 1 – 4 Exons 5 – 19 PTV exons 1 – 4 PTV exons 5 – 19 MILDER Intermediate (>50%) SYNGAP protein levels • Verbal responses • Milder epilepsy in some patients MOST SEVERE True haploinsufficiency: 50% SYNGAP protein levels • most homogeneous • highest seizure burden Missense variants / VUS — distributed across the gene Sources: Graglia et al, 2025: Variant types across 195 SRD patients; Vlaskamp et al, 2019: Table 1: SYNGAP1 Phenotypes in Patients with SYNGAP1 mutations and microdeletions. Ph1/2 Clinical Trial target population Natural history studies have replicated highest burden of disease across multiple domains in PTV 5-19 genotypes
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SYNGAP1-related disorder impacts many aspects of neurological function 15 Phenotypic features of SYNGAP1-Related Disorder by prevalence All patients display ID, majority with epilepsy, behavioral/autistic features and sleep problems Phenotypic feature Prevalence Intellectual disability 100% Epilepsy 84% Autism 68% Behavior 68% Sleeping 61% Feeding problems 47% Gait 33% Anxiety 24% Sensory 22% Ataxia 21% Patients, n=147 Source: Wiltrout et al., 2024
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SYNGAP1 has similar or higher severity relative to analogue conditions Source: McKee et al., 2025 16 Seizures among highest frequency Intellectual disability: neuro-arrest / developmental plateau (avg cognitive age of 2) Difficult to manage autism-like behavior Communication impairment: many patients are non- verbal or have few words Age (years) 0 10 20 0 10 20 0 10 20 Age (years) 0 10 20 Age (years)Age (years) Age (years) SYNGAP1 Epilepsy (G40+) Rett Syndrome Angelman Syndrome
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Majority of patients have epilepsy with high frequency of seizures Sources: Vlaskamp et al, 2019; Galer et al., 2025; McKee et al., 2026 (reproduced with author permission). Seizure Burden 17 One of highest seizure burdens among genetic epilepsies, consisting of multiple types 85% of patients receive epilepsy diagnosis Compared to other DEEs, SYNGAP has some of the highest frequency of seizures Genotype of focus (PTV 5-19) have highest rates of daily seizures among SYNGAP1 patients Multiple seizure types – with non-motor seizures being common (ex. absence seizures) Seizure type can vary by age SYNGAP1-specific EEG signatures include changes in alpha-theta wave Percent of Observed Cohort Age (months)
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ASCEND clinical trial designed to assess safety & tolerability across key domains of SYNGAP1 using validated measures 18 ASCEND exploratory endpoints assessed Communication Sleep Motor & Gait Seizure Behavior Development & Cognition CLINICAL TRIAL GOALS • Identify relevant endpoints applicable to SYNGAP1 patients to chart path for regulatory approval • Demonstrate safety and tolerability • Characterize pharmacokinetics and select optimal biological dose
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ASCEND Phase 1/2 Clinical Trial Summary 19 Overview Design • Phase 1/2, randomized, double-blind, controlled, multiple ascending dose study • 3:1 randomization • IT administration or a sham pinprick • Open-Label Extension Study population • Minimum 8 participants per cohort • Participants ≥2 to <18 years of age with genetically confirmed SYNGAP1-Related Disorder (SRD) • Required daily seizures, impaired sleep, and inability to speak in phrases Study periods • Screening and baseline period • Study treatment period with 3 doses • Post-treatment follow-up Study length • ~10 months per participant
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20 ASCEND eligibility criteria Key Inclusion Criteria ✓ Age 2 to <18 years Male or female participants ✓ Genetically confirmed SRD Clinical diagnosis of SRD with genetic confirmation of a protein- truncating mutation in SYNGAP1 exons 5–19 ✓ Haploinsufficiency phenotype Intellectual disability, inability to speak in full phrases, and sleep disturbance consistent with haploinsufficiency ✓ Refractory epilepsy Daily seizures despite ≥1 ongoing anti-seizure medication and prior trial of ≥2 ASMs ✓ Stable ASM regimen at baseline Stable anti-seizure medication regimen prior to baseline Key Exclusion Criteria ✕ Non-truncating variants SYNGAP1 variants that either are non-truncating (missense, microdeletions), located in exons 1–4, or of uncertain significance ✕ Other CNS disease History of central nervous system disease unrelated to SRD ✕ Unsafe for intrathecal dosing Spinal abnormalities, implanted CSF shunts, or intolerance to lumbar puncture ✕ Clinically significant lab abnormalities
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ASCEND clinical trial schema 21 MAD Cohort 1 TREATMENT PERIOD 3 doses over 3 months FOLLOW-UP PERIOD multiple timepoints over 6 mosR TREATMENT FOLLOW-UP MAD Cohort 2 R TREATMENT FOLLOW-UP MAD Cohort 3 R SCREENING: 28 days ASSESSMENTS : ≥4 weeks Baseline Period After 2 doses in 8 participants After 2 doses in 8 participants Open-Label Extension Daily seizure diary, side effect and medication review Actigraphy prior to each study visit Confirmation of Eligibility Randomization 3:1 Safety and functional assessments across multiple domains throughout
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Five validated scales being utilized in ASCEND: Behavior, development, communication, and motor function 22 Scale Communication Motor Behavior Development Bayley Scales of Infant and Toddler Development Bayley-4 Vineland Adaptive Behavior Scales, 3rd Edition Vineland-3 Aberrant Behavior Checklist, 2nd Edition ABC-2 Observer-Reported Communication Ability Measure ORCA Gross Motor Function Measure GMFM Leveraging validated scales used in DEE clinical trials and ProMMiS Natural History Study Scale to be utilized to assess domain in ASCEND trial
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Assessing seizure and sleep: multiple methods to measure change, with vEEG common to both domains 23 Seizure • Seizure diaries • Seizure-Related Impact Assessment Scale (SERIAS) Sleep • Children’s Sleep Habits Questionnaire (CSHQ) • Actigraphy EEG 24hr video- EEG for seizure and sleep detection: • REM, SOL, WASO, TST • EEG informs both seizure and sleep endpoints EEG measures multiple parameters Clinical signatures spikes, seizures, slowing Spectral features alpha-theta wave Spatial / topographic measures Event-related potentials VEP / AEP Sleep / state-based measures Functional connectivity Temporal / nonlinear dynamics entropy, temporal correlations
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Planned global footprint of ASCEND Phase 1/2 clinical trial 24 UK Approved by MHRA EU Under regulator and ethics committee review ARG Approved by ANMAT AUS Approved by HREC; acknowledged by TGA EU sites under review US Future expansion 11+ Countries Regions with sites planned approved: UK, ARG, AUS 5 4 7 9 Total sites
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CAMP4 is pioneering SYNGAP1 development with clearly defined Phase 1/2 study goals 25 Signal-seeking across all domains of disease using validated measures Partnering with leading experts and patient community Expect first patient dosed by year end. Topline read-out targeted first-half of 2028 Severe, life-long disease with consistent natural history data across multiple studies to guide development
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Video: Co-Founder and Managing Director, CURE SYNGAP1 26
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KOL fireside chat 27
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Emerging pipeline & closing remarks 28
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Source: 1Levy et al., Autism Research (2026)29 Early pipeline focused on haploinsufficient neurodevelopmental disorders to expand on expertise gained through SYNGAP1 program Program Indication Target Discovery & Preclinical Development Phase 1/2 Anticipated Milestone Commercial Rights CMP-002 SYNGAP1-related disorder SYNGAP1 SHANK3 Phelan-McDermid Syndrome SHANK3 Development Candidate in 2027. CNS Program 1 CNS haploinsufficiency CNS Program 2 CNS DISEASES Haploinsufficiency due to a mutation in SHANK3, a central scaffolding protein in the postsynapse Clear unmet need with no approved treatments Autism, developmental delay, intellectual disability, motor impairment, milestone regression 45,000+ patients in the US 1 with a well-organized and sophisticated patient advocacy group Overlapping KOLs & physician network with SYNGAP1 SHANK3 – Phelan-McDermid Syndrome CNS haploinsufficiency Clinical initiation in Q4 2026. Topline data 1H 2028.
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Analyst Q&A 30