Camp4 Therapeutics Corporation · NASDAQ:CAMP
Camp4 Therapeutics — Investor Presentation (March 2026)
22 slides from Camp4 Therapeutics's investor presentation, Mar 5, 2026
Slide 1: Pioneering a new class of RNA medicines to increase targeted gene expression
C/AMP4 Pioneering a new class of RNA medicines to increase targeted gene expression. MARCH 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
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 SYNGAP1-related disorder + SYNGAP1 isa 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 olonce of activating and repressive toctors at the enhancer (1) regRNAs originate from enhancers and promoters ACTIVATOR
Slide 5: CAMP4's proprietary RAP Platform catalogs thousands of regRNA targets
CAMP4’s proprietary RAP Platform® catalogs thousands of regRNA targets and generates ASO candidates to increase gene expression Genome-wide analyses of chromatin & RNA (1) Map candidate regRNAs * Generate large scale genomic datasets for cells & tissues TF and Cofactor Binding * Deploy proprietary M
Slide 6: Our pipeline of CNS-focused upregulation programs
Our pipeline of CNS-focused upregulation programs Progam pan Target Discovery & Prectinical eee ieee’ ‘edeipated ‘Commercial i SYNGAPT-related jiiaa cz GLP tox studies ongoing a CON, disorder * Clinical initiation as early as H2 2026 CAMP4 New Di . Pe Discovery ons Numerous EB Active ciscovery and d
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 and
Slide 8: SYNGAP1 is a true haploinsufficiency with >10,000 US patients in need of therapy
SYNGAP‘1 is a true haploinsufficiency with >10,000 US patients in need of therapy Haploinsufficiency results in 50% of normal protein levels SYNGAP17 Haploinsufficiency 50% SYNGAP Protein lo >10,000 SYNGAP'1 patients in the US 6.1-10 per 100K incidence rate '? 100% have intellectual disability, ~85%
Slide 9: Dire unmet need for a targeted disease modifying therapy
Dire unmet need for a targeted disease modifying therapy to alter SYNGAP'1’s devasting disease course Developmental delay and/or intellectual disability 100% of patients +> Generalized epilepsy ~85% of patients+ Severe behavioral problems ~70% of patients ** Sleep problems ~60% of patients?> Limited
Slide 10: Expanding awareness and testing is enabling faster diagnosis
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 sitin limbo Confirmed after neg. test) / WES,WGS Diagnosis Severe behavioral se Panels as < Sei
Slide 11: ~21K patients across key global geographies; SYNGAP1 remains highly underdiagnosed
~21K patients across key global geographies (US + EU5); SYNGAP1 remains highly underdiagnosed Major Market Prevalence Increasing Diagnosis Rate ~85% =21K" Patients ~25% * Sealed annual incidence to prevalence * * Predict notable increase in diagnosis: * Prevalence may be larger; diagnosis rates may
Slide 12: CAMP4 aims to increase SYNGAP protein levels, restore SYNGAP1 function
CAMP4 aims to increase SYNGAP protein levels, restore SYNGAP1 function and improve disease symptoms CMP-002 binds to aSYNGAP-specific regRNA to increase SYNGAP1 expression, aiming to restore SYNGAP towards wild-type levels and normalize synaptic function Mutations in SYNGAP1 lead to decreased SYNGAP
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 ee 'e Opportunity in SYNGAP1 driven by unmet need and compelling preclinical data (presented at ASGCT May ‘25) e * Ultra-rare SYNGAP1 sub population with intermediate SY
Slide 14: CMP-002 restores SYNGAP levels in models of haploinsufficiency
CMP-002 restores SYNGAP levels in models of haploinsufficiency Ke SYNGAP1 Patient iPSC-derived neurons )2{ “Humanized” SYNGAP1 mouse Sane 20: g SYNGAP1 patient mutation: & z K1185X [Lysine > “stop”] cs SE 15 SYNGAP1 patient SE me neurons + CMP-002 Lis E2s Qos q gE 10156-4—_5— Familial control neuron
Slide 15: Improved behavioral phenotypes in SYNGAP1 humanized haploinsufficient mice
Improved behavioral phenotypes in SYNGAP1 humanized haploinsufficient mice given CMP-002 Learning & Memory Motor Function Hyperactivity ns ns ns * * + + wee a < 60: aCSF= £ as — artificial CSF Fs 3 = *,p<0.05 i 40: = 40000. **,ps0.01 E = 60. "=", p<0.001 < 2 3 2 40. 2” g es o i + 20. + HOM - aC SF (
Slide 16: Intrathecal administration of CMP-002 to cynomolgus monkeys achieves broad brain distribution
Intrathecal administration of CMP-002 to cynomolgus monkeys achieves broad brain distribution to increase SYNGAP protein levels ASO Concentrations SYNGAP Protein Levels N=4/group Frontal Cortex Hippocampus Dose Q2wx3 ee ** * Take down 2 wks + postlast dose 3 ° aCSF * cMP-002 (3 dose levels) Decor Ho
Slide 17: CAMP4 positioned to be first in the clinic for SYNGAP1
CAMP4 positioned to be first in the clinic for SYNGAP1 Standard-of-Care Natural History Study Center of Excellence Path to Clinic Path to Approval No disease modifying therapies available Patients currently managed symptomatically, with complications of polypharmacy Consortium-driven, funded, multi-
Slide 18: Multiple regulatory precedents and validated measures to demonstrate disease-modifying effect
Multiple regulatory precedents and validated measures to demonstrate disease-modifying effect for PoC in First-in- Human yo 4 Seizure Sleep Motor & Gait Communication Seizure + Seizure as primary with secondary neurodevelopmental endpoints: fits with SYNGAP1 high seizure burden Multiple methodologie
Slide 19: Development path and design to maximize speed and success
Development path and design to maximize speed and success Ph 1/ 2 Optimized for: First-in- Human Clinical Trial Design Approach * Rapid enrollment * Straight to MAD * Efficacy assessment across * Population: enriched genotype and phenotype population multiple domains of disease + Aimto start in pedi
Slide 20: CAMP4 team has been pioneering the field of regRNAs
20 CAMP4 team has been pioneering the field of regRNAs h a Josh Mandel-Brehm Kelly Gold Dan Tardiff, PhD Yuri Maricich, MD President & CEO Chief Financial Officer Chief Scientific Officer Chief Medical Officer ° aereyre ° J] ons “1 aie iumety = Corixa PEAR Caleb Moore Michelle Gates Alla Sigova, PhD
Slide 21: Building momentum and a unique value proposition
Building momentum and a unique value proposition @ Werecently repositioned our pipeline to focus on SYNGAP1 and have funded the program to accelerate the path to the clinic as early as H2 2026 @ SYNGAP1 represents a major market opportunity and is the cornerstone program for CAMP4 + Future opportuni
Slide 22: Thank You
@ Corporate OVERVIEW MARCH 2026 Thank You CAMP4
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