Camp4 Therapeutics Corporation · NASDAQ:CAMP
Camp4 Therapeutics — Investor Presentation (May 2026)
21 slides from Camp4 Therapeutics's investor presentation, May 14, 2026
Slide 1: CAMP4 Corporate Overview
CAMP4 Corporate Overview Pioneering a new class of RNA medicines to increase targeted gene expression. MAY 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 is a haploinsufficient CNS disorder, and an optimal target for CAMP4 * CMP-002i
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 Balance of activating ond repressive factors at the enhancer REPRESSOR @ regRNAs originate from enhancers and promoters
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 C1] 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 Discovery & Preciinical Anticipated Commercial Program Indication Target 2 ” Phase 72 Phase 3 sneceed a CNS DISEASES srnoaet ete LP isucesongeg = ‘MP-002 ae) A disorder Clinical initiation as early as H2 2026 CAM P4 New Discovery ; a New Diep ons tam
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
Slide 8: SYNGAP1 is a haploinsufficiency with >10,000 US patients in need of therapy
SYNGAP‘1 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 Haploinsuf
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 Non-specific treatments have limited disability impact on SYNGAP1 symptoms 100% of patients *25 Anti-seizure medications Cannabinoids Generalized epilepsy Sle
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 al Chromosomal Microarray @ (Often sit in timbo Confirmed after neg test) WES,WGS Diagnosis Severe behavioral Panels ry Seizures
Slide 11: ~21K patients across key global geographies; SYNGAP1 remains highly underdiagnosed
his ~21K patients across key global geographies (US + EU5); SYNGAP1 remains highly underdiagnosed Major Market Prevalence Increasing Diagnosis Rate ~85% ~21K1 Patients ~25% * Scaled annual incidence to prevalence * * Predict notable increase in diagnosis: * Prevalence may be larger; diagnosis rates
Slide 12: CAMP4 aims to increase SYNGAP protein levels, restore SYNGAP function
12 CAMP4 aims to increase SYNGAP protein levels, restore SYNGAP7 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 restores SYNGAP levels in models of haploinsufficiency
14 CMP-002 restores SYNGAP levels in models of haploinsufficiency SYNGAP1 mRNA SYNGAP1 Patient iPSC-derived neurons Control = & 2 3 g a E & 2 2 “ rt ee Humanized” SYNGAP1 mouse = -S_. \_, 20 g SYNGAP1 patient mutation: & K1185X [Lysine > “stop”] £f 1s SYNGAP1 patient ge neurons + CMP-002 ate a 22 10
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 * * ” * ee g@ aCsF = g 7 | = uM artificial OSF & 5 a0. = 40000. 5 +*,p<0.01 E — 3 = z a -- +44! 90.001 & a + 2 3 E F 6. °. 0. HOM HET HET HOM HET HET HOM H
Slide 16: CMP-002 reduces PTZ-induced seizures in SYNGAP1 humanized mouse
CMP-002 reduces PTZ-induced seizures in SYNGAP1 humanized mouse * Chemically-induced seizure model shown to be sensitized ‘ wah PND21 PND49 PNDSO by SYNGAP1 haploinsufficiency | | + Repeated IP administration of GABA antagonist with Terminate protein monitoring of seizures over 5-minute window CMP-0
Slide 17: Intrathecal administration of CMP-002 to cynomolgus monkeys achieves broad brain distribution
17 Intrathecal administration of CMP-002 to cynomolgus monkeys achieves broad brain distribution to increase SYNGAP protein levels ASO Concentrations SYNGAP Protein Levels Summary Frontal Cortex Hippocampus IT administration in NHPs, CSF * Ne4/group “ } Dose Q2w x3 eK ** was well-tolerated cMP-002 (
Slide 18: CAMP4 positioned to be first in the clinic for SYNGAP1
CAMP4 positioned to be first in the clinic for SYNGAP1 * No disease modifying therapies available Standard-of-Care Patients currently managed symptomatically, with complications of polypharmacy * Collaborating with CURE SYNGAP1 to support ongoing, multi-site study with multiple Natural History Study
Slide 19: Phase 1/2 study will assess key domains of SYNGAP1
19 Phase 1/2 study will assess key domains of SYNGAP‘1, utilizing validated measures to demonstrate PoC in First-in-Human Seizure Sleep Motor & Gait Communication Behavior Development & Cognition Validated measures: Bayley-4, Vineland, Clinical Global Impression, others Ph 1/2 key features: First-in
Slide 20: CAMP4 team has been pioneering the field of regRNAs
20 CAMP4 team has been pioneering the field of regRNAs Josh Mandel-Brehm President & CEO Caleb Moore Chief Business Operations Officer 3 BE Gomes eye 7 gereyme Kelly Gold Chief Financial Officer ’ 74) Michelle Gates Chief People Officer ‘< Dan Tardiff, PhD Chief Scientific Officer 3 Yumanty Alla Sig
Slide 21: Building momentum and a unique value proposition
2 Building momentum and a unique value proposition @ CAMP4is advancing the first potentially disease modifying therapy for SYNGAP1 into a Phase 1/2 clinical study in 2H ‘26 and well positioned to deliver long-term pipeline value @ SYNGAP1 represents a major market opportunity and is the cornerstone
CAMP4 Therapeutics — Investor Presentation (May 2026) on 6ix