Camp4 Therapeutics Corporation · NASDAQ:CAMP
Camp4 Therapeutics — Investor Day (September 2026)
30 slides from Camp4 Therapeutics's investor day presentation, Sep 28, 2026
Slide 1: CAMP4 Analyst Event
CAMP4 Analyst Event September 28, 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: Agenda
Agenda • 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 Manag
Slide 4: Solving genetic diseases of the brain through upregulation
Solving genetic diseases of the brain through upregulation Future Broad-based pipeline of CNS-focused upregulation therapeutics in areas of high unmet need Lead Program Focused on SYNGAP1 Today >20k patients1 RAP Platform® Addressing the root cause of genetic disease by restoring healthy protein lev
Slide 5: Video: Patient story
Video: Patient story 5
Slide 6: ASCEND clinical trial in SYNGAP1
ASCEND clinical trial in SYNGAP1 ASO therapy for SYNGAP1 Clinical Evaluation in Neuro Development 6
Slide 7: 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 1 regRNAs originate from enhancers and promoters Activators and repressors 2 bind to regRNAs to control the expression o
Slide 8: CAMP4 aims to increase SYNGAP protein levels, restore SYNGAP1 function, and improve disease symptoms
CAMP4 aims to increase SYNGAP protein levels, restore SYNGAP1 function, and improve disease symptoms CMP-002 binds to a SYNGAP-specific regRNA to increase Mutations in SYNGAP1 lead to decreased SYNGAP protein, SYNGAP1 expression, aiming to restore SYNGAP towards causing increased synaptic firing wil
Slide 9: CMP-002 restored SYNGAP levels in models of haploinsufficiency
CMP-002 restored SYNGAP levels in models of haploinsufficiency SYNGAP1 Patient iPSC-derived neurons “Humanized” SYNGAP1 mouse FC Normalized to Homozygous FC Normalized to Familial Control SYNGAP1 patient mutation: K1185X [Lysine → “stop”] SYNGAP Protein SYNGAP1 patient SYNGAP1 mRNA neurons + CMP-002
Slide 10: CMP-002 broadly improved neurological phenotypes in humanized SYNGAP1 mice
CMP-002 broadly improved neurological phenotypes in humanized SYNGAP1 mice Learning & Memory Motor Function Hyperactivity Seizures Healthy Haplo + ASO Haplo Broad benefit of CMP-002 supports potential translation across multiple phenotypes in SYNGAP1 patients 10
Slide 11: 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 • N=4/group Frontal Cortex Hippocampus IT administration in NHPs aCSF • Dose Q2w x3 was well-tolerated CMP-002 • Take down
Slide 12: SYNGAP1 is a devastating disease with significant unmet need
SYNGAP1 is a devastating disease with significant unmet need for a targeted disease modifying therapy Complex Symptoms No Approved Therapy Developmental delay and/or intellectual Non-specific treatments have limited disability impact on SYNGAP1 symptoms • 100% of patients 1,2,3 • Anti-seizure medica
Slide 13: Natural history studies and registries inform ASCEND clinical trial
Natural history studies and registries inform ASCEND clinical trial N = 1,806 N = 159 Sponsored N = 350 N = 569 Prospective natural history Retrospective EMR SYNGAP1 Census European registry (PATRE) as of Q2 2026 cohort as of Q2 2026 as of Q3 2026 as of Q3 2026 Ongoing study 2023–2028; Advocacy-grou
Slide 14: Majority of SYNGAP1 patients have severe protein-truncating variants
Majority of SYNGAP1 patients have severe protein-truncating variants Natural history studies Multiplehave replicated natural historyhighest studiesburden of diseasehighest have replicated acrossburden multipleofdomains disease in PTV 5-19 genotypes across multiple domains in PTV 5-19 genotypes Ph1/2
Slide 15: SYNGAP1-related disorder impacts many aspects of neurological function
SYNGAP1-related disorder impacts many aspects of neurological function Phenotypic features of SYNGAP1-Related Disorder by prevalence Phenotypic feature Prevalence Intellectual disability 100% Epilepsy 84% Autism 68% Behavior 68% Sleeping 61% Feeding problems 47% Gait 33% Anxiety 24% Sensory 22% Atax
Slide 16: SYNGAP1 has similar or higher severity relative to analogue conditions
SYNGAP1 has similar or higher severity relative to analogue conditions Seizures among highest frequency Difficult to manage autism-like behavior 0 10 20 0 10 20 0 10 20 Age (years) Age (years) Age (years) Age (years) Intellectual disability: neuro-arrest / developmental Communication impairment: man
Slide 17: Majority of patients have epilepsy with high frequency of seizures
Majority of patients have epilepsy with high frequency of seizures One of highest seizure burdens among genetic epilepsies, consisting of multiple types Seizure Burden Percent of Observed Cohort Age (months) 85% of patients receive epilepsy diagnosis Compared to other DEEs, SYNGAP has some of th
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 • Demonstrate safety • Characterize • Identify relevant endpoints and tolerability pharmacokinetics and select applicable to SYNGAP1 optimal biological dose pati
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 Design • IT administration or a sham pinprick • Open-Label Extension • Minimum 8 participants per cohort Study population • Participants ≥2 to <18 yea
Slide 20: ASCEND eligibility criteria
ASCEND eligibility criteria Key Inclusion Criteria Key Exclusion Criteria ✓ Age 2 to <18 years ✕ Non-truncating variants Male or female participants SYNGAP1 variants that either are non-truncating (missense, microdeletions), located in exons 1–4, or of uncertain significance ✓ Genetically confirmed
Slide 21: ASCEND clinical trial schema
ASCEND clinical trial schema MAD Cohort 1 TREATMENT PERIOD FOLLOW-UP PERIOD R 3 doses over 3 months multiple timepoints over 6 mos Open-Label Extension Randomization 3:1 After 2 doses in Baseline Period 8 participants MAD Cohort 2 SCREENING: ASSESSMENTS R TREATMENT FOLLOW-UP 28 days : ≥4 weeks After
Slide 22: Five validated scales being utilized in ASCEND
Five validated scales being utilized in ASCEND: Behavior, development, communication, and motor function 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
Slide 23: Assessing seizure and sleep: multiple methods to measure change
Assessing seizure and sleep: multiple methods to measure change, with vEEG common to both domains EEG measures multiple parameters 24hr video- EEG for seizure and Seizure Sleep Clinical signatures spikes, seizures, slowing sleep Spectral features detection: alpha-theta wave • Seizure diaries • Child
Slide 24: Planned global footprint of ASCEND Phase 1/2 clinical trial
Planned global footprint of ASCEND Phase 1/2 clinical trial 11+ Total sites 9 UK Countries Approved by MHRA EU Under regulator and ethics committee review US Future expansion 5 Regions with sites planned 4 approved: UK, ARG, AUS ARG AUS Approved by HREC; acknowledged by TGA 7 Approved by ANMAT EU si
Slide 25: CAMP4 is pioneering SYNGAP1 development with clearly defined Phase 1/2 study goals
CAMP4 is pioneering SYNGAP1 development with clearly defined Phase 1/2 study goals Severe, life-long disease with consistent natural history data across multiple studies to guide development Partnering with leading experts and patient community Signal-seeking across all domains of disease using vali
Slide 26: Video: Co-Founder and Managing Director, CURE SYNGAP1
Video: Co-Founder and Managing Director, CURE SYNGAP1 26
Slide 27: KOL fireside chat
KOL fireside chat CAMP4
Slide 28: Emerging pipeline & closing remarks
Emerging pipeline & closing remarks 28
Slide 29: Early pipeline focused on haploinsufficient neurodevelopmental disorders
Early pipeline focused on haploinsufficient neurodevelopmental disorders to expand on expertise gained through SYNGAP1 program Discovery & Preclinical Anticipated Commercial Program Indication Target Development Phase 1/2 Milestone Rights CNS DISEASES SYNGAP1-related Clinical initiation in Q4 2026.
Slide 30: Analyst Q&A
Analyst Q&A CAMP4