Camp4 Therapeutics Corporation · NASDAQ:CAMP
Camp4 Therapeutics — Investor Presentation (September 2026)
26 slides from Camp4 Therapeutics's investor presentation, Sep 28, 2026
Slide 1: Corporate Overview
CIAMP4 Corporate Overview Pioneering a new class of RNA medicines to increase targeted gene expression. SEPTEMBER 2026
Slide 2: Forward-Looking Statements
Forward-Looking Statements 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 stat
Slide 3: CAMP4: Targeted ASO therapeutics that selectively upregulate gene expression by modulating regulatory RNA
CAMP4: Targeted ASO therapeutics that selectively upregulate gene expression by modulating regulatory RNA Developing a targeted disease modifying therapy to address dire unmet need in SYNGAP'1-related disorder » SYNGAP1 is a haploinsufficient CNS disorder, and an optimal target for CAMP4 + CMP-002 i
Slide 4: regRNAs play a central role in the regulation of every gene's expression
regRNAs play a central role in the regulation of every gene’s expression Increased mRNA addresses root cause of disease by returning targeted protein levels toward a healthy range REPRESSOR @ regRNAs originate from enhancers and promoters ACTIVATOR Activators and repressors bind to regRNAs to contro
Slide 5: CAMP4's proprietary RAP Platform catalogs thousands of regRNA targets and generates ASO candidates
CAMP4’s proprietary RAP Platform® catalogs thousands of regRNA targets and generates ASO candidates to increase gene expression Genome-wide analyses of chromatin & RNA oe Map candidate regRNAs * Generate large scale genomic datasets for cells & tissues TF and Cofactor Binding * Deploy proprietary ML
Slide 6: Our pipeline of CNS-focused upregulation programs
Our pipeline of CNS-focused upregulation programs Progam Inieation Target Pooven Ect Prose 2 Poses | Atta erat cm-o02 groirietins sca cisions cRMpa disorder Topline data 1H 2028. Prelan-MeDemis Hans Development Candidate a SHANKS conte GD Deiaonereceeain C/AMP4 Es Progen as CAMP4 haploinsutfficiency
Slide 7: SYNGAP1 patient journey: Tony and his family's experience
SYNGAP'1 patient journey: Tony and his family’s experience highlights the dire unmet need for disease modifying therapy PATIENT CAREGIVER + FAMILY BURDEN Tony, 11 Years Old Immense Caregiver Burden TONY, 3 TONY, 8 “His spontaneous aggression leads to bruises and scary moments for family . members an
Slide 8: SYNGAP1 is a haploinsufficiency with >10,000 US patients in need of therapy
SYNGAP1 is a haploinsufficiency with >10,000 US patients in need of therapy Haploinsufficiency >10,000 High unmet need for results in 50% of SYNGAP1 patients in the US disease-modifying therapy normal protein levels 6.1-10 per 100K ICD-10 code incidence rate 12 assigned in F78.A1 SYNGAP1 Haploinsuff
Slide 9: SYNGAP1 is a devastating disease with significant unmet need for a targeted disease modifying therapy
SYNGAPT 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 Developmental delay and/or intellectual disability + 100% of patients
Slide 10: Expanding awareness and testing is enabling faster diagnosis
10 Expanding awareness and testing is enabling faster diagnosis from ~5 years to ~1 year from time of first symptom or missed milestone Historical ~5 year Diagnostic Journey Chromosomal Microarray @ @ (Often sit in limbo calc after neg. test) WES, on Diagnosis Severe behavioral Panels Seizures probl
Slide 11: ~21K patients across key global geographies (US + EU5)
1 ~21K patients across key global geographies (US + EU5); SYNGAP1 remains highly underdiagnosed Major Market Prevalence ~21K" Patients * Scaled annual incidence to prevalence * * Prevalence may be larger; diagnosis rates may increase significantly with genetic testing awareness and utilization 2 * T
Slide 12: CAMP4 aims to increase SYNGAP protein levels, restore SYNGAP1 function and improve disease symptoms
42 CAMP4 aims to increase SYNGAP protein levels, restore SYNGAP1 function and improve disease symptoms CMP-002 binds to a SYNGAP-specific regRNA to increase SYNGAP1 expression, aiming to restore SYNGAP towards wild-type levels and normalize synaptic function Mutations in SYNGAP7 lead to decreased SY
Slide 13: SYNGAP1 represents an ideal target for CAMP4
SYNGAP'1 represents an ideal target for CAMP4; restoring SYNGAP protein has the potential to meaningfully improve patient outcomes Opportunity in SYNGAP1 driven by unmet need and compelling preclinical data * Ultra-rare SYNGAP1 sub population with intermediate SYNGAP levels equate to milder disease
Slide 14: CMP-002 restored SYNGAP levels in models of haploinsufficiency
14 CMP-002 restored SYNGAP levels in models of haploinsufficiency SYNGAP1 mRNA FC Normalized to Familial Control SYNGAP1 Patient iPSC-derived neurons “Humanized” SYNGAP1 mouse SNe 2.0 SYNGAP1 patient mutation: K1185X [Lysine — “stop”] 18 SYNGAP1 patient neurons + CMP-002 10 Familial control neurons
Slide 15: Improved behavioral phenotypes in SYNGAP1 humanized haploinsufficient mice given CMP-002
15 Improved behavioral phenotypes in SYNGAP1 humanized haploinsufficient mice given CMP-002 Learning & Memory Motor Function ns ns so * * * * s 100. mm t 2 a) 5 a0. © E 3 0 < 2 5 20 AD 2 2 A 3 z= Ro ° HOM HET HET HOM HET HET aCSF aCSF CMP-002 aCSF aCSF CMP-002 60000. 3 3 3 3 Distance (pixel) Hyperac
Slide 16: CMP-002 reduced PTZ-induced seizures in SYNGAP1 humanized mouse
CMP-002 reduced PTZ-induced seizures in SYNGAP1 humanized mouse * Chemically-induced seizure model shown to be sensitized PND21 PND4g PND5O by SYNGAP1 haploinsufficiency * Repeated IP administration of GABA antagonist with | | Terminate / protein itori i omni i CMP-002 (50 1g) or aCSF | monitoring o
Slide 17: Intrathecal administration of CMP-002 to cynomolgus monkeys achieved broad brain distribution
Intrathecal administration of CMP-002 to cynomolgus monkeys achieved broad brain distribution to increase SYNGAP protein levels ASO Concentrations SYNGAP Protein Levels Summary aCsF + Nea/group Frontal Cortex Hippocampus ITadministration in NHPs cMP-002 | = Dose Qi x8 see was well-tolerated “ * Take
Slide 18: ASCEND clinical trial designed to assess safety & tolerability across key domains
ASCEND clinical trial designed to assess safety & tolerability across key domains of SYNGAP1 using validated measures CLINICAL TRIAL GOALS A iN Demonstrate safety and tolerability * Characterize Identify relevant endpoints pharmacokinetics and select applicable to SYNGAP1 optimal biological dose pat
Slide 19: ASCEND Phase 1/2 Clinical Trial Summary
ASCEND Phase 1/2 Clinical Trial Summary Overview + Phase 1/2, randomized, double-blind, controlled, multiple ascending dose study * 3:1 randomization * |T administration or a sham pinprick * Open-Label Extension Design * Minimum 8 participants per cohort Surely eee * Participants =2 to <18 years of
Slide 20: ASCEND eligibility criteria
20 ASCEND eligibility criteria Key Inclusion Criteria e 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
Slide 21: ASCEND clinical trial schema
21 ASCEND clinical trial schema MAD Cohort 1 R») TREATMENT PERIOD FOLLOW-UP PERIOD as 3 doses over 3 months multiple timepoints over 6 mos Randomization 3:1 After 2 doses in Baseline Period Spacers MAD Cohort 2 SCREENING: ASSESSMENTS @-— qe ener 28 days :24 weeks = After 2 doses in Confirmation 8 pa
Slide 22: Five validated scales being utilized in ASCEND
Five validated scales being utilized in ASCEND: Behavior, development, communication, and motor function sete Communication — Behavior eae nce Bayley Scales of Infant and Toddler Development V Bayley-4 Vineland Adaptive Behavior Scales, 3rd Edition Aberrant Behavior Checklist, 2nd Edition ABC-2 VY V
Slide 23: Assessing seizure and sleep: multiple methods to measure change
23 Assessing seizure and sleep: multiple methods to measure change, with VEEG common to both domains 24hr video- EEG for “ seizure and Seizure Sleep sleep detection: Seizure diaries EEG * Children’s Sleep * REM, SOL, oe ee Habits WASO, TST P Questionnaire Assessment Scale (SERIAS) (CSHQ) ° EEG infor
Slide 24: Planned global footprint of ASCEND Phase 1/2 clinical trial
Planned global footprint of ASCEND Phase 1/2 clinical trial a) Countries Approved by MHRA _J EU Q Under regulator and ethics committee review us Future expansion Regions with sites planned approved: UK, ARG, AUS Q a) Re Approved by HREC; acknowledged by TGA Approved by ANMAT EU sites under review 2
Slide 25: CAMP4 team has been pioneering the field of regRNAs
CAMP4 team has been pioneering the field of regRNAs 2 Board of Directors Steven Holtzman Josh Mandel-Brehm Josh Mandel-Brehm Kelly Gold Dan Tardiff, PhD Yuri Maricich, MD President & CEO Chief Financial Officer Chief Scientific Officer Chief Medical Officer Biogen polaris arome —oiagan: [Z]rmmtenne
Slide 26: Building momentum and a unique value proposition
26 Building momentum and a unique value proposition @ CAMP4 is advancing the first potentially disease modifying therapy for SYNGAP1 into a Phase 1/2 clinical study in Q4 ‘26 and well positioned to deliver long-term pipeline value @ SYNGAP1 represents a major market opportunity and is the cornerston
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