Camp4 Therapeutics Corporation · NASDAQ:CAMP
Camp4 Therapeutics — Investor Presentation (May 2025)
35 slides from Camp4 Therapeutics's investor presentation, May 16, 2025
Slide 1: Corporate Overview: Pioneering a new class of RNA medicines
Pioneering a new class of RNA medicines to increase targeted gene expression May 2025 C O R P O R A T E O V E R V I E W
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 overview agenda
CAMP4 overview RAP Platform: regRNAs are master controllers of gene regulation Lead metabolic program: Urea Cycle Disorders Lead CNS program: SYNGAP1-related Disorders 3 Agenda
Slide 4: CAMP4 is the leader in regRNA discovery and regRNA-targeting ASO therapies
CAMP4 is the leader in gene regulatory RNA (regRNA) discovery and regRNA-targeting antisense oligonucleotide (ASO) therapies to upregulate gene expression to restore healthy protein levels 4 Our proprietary RAP Platform was built for the discovery of novel regRNAs that regulate the expression of eve
Slide 5: World-class management team, experienced board and advisors
5 World-class management team, experienced board and advisors Josh Mandel-Brehm President & CEO David Bumcrot, PhD Chief Scientific Officer Michelle Gates Chief People Officer Caleb Moore Chief Business Operations Officer Yuri Maricich, MD Chief Medical Officer Kelly Gold Chief Financial Officer Boa
Slide 6: Advancing a pipeline in metabolic and CNS genetic diseases
Program Indication Target Discovery & Preclinical Phase 1 Phase 2 Phase 3 Anticipated Milestones Commercial Rights Metabolic diseases CMP-CPS-001 Urea Cycle Disorders CPS1 Phase 1 MAD data in Q4’25 CMP-CPS-001 Expansion Urea Cycle Disorders- OTC heterozygotes CPS1 CNS diseases CMP-SYNGAP-01 SYNGAP1-
Slide 7: RAP Platform agenda
CAMP4 overview RAP Platform: regRNAs are master controllers of gene regulation Lead metabolic program: Urea Cycle Disorders Lead CNS program: SYNGAP1-related Disorders 7 Agenda
Slide 8: regRNAs play a central role in the regulation of every gene's expression
8 regRNAs play a central role in the regulation of every gene’s expression mRNA Activators and repressors bind to regRNAs to control the transcriptional state regRNAs originate from enhancers and promoters to control the expression of protein-coding genes Active Enhancer Nucleus Transcription Factor
Slide 9: CAMP4's proprietary RAP Platform catalogs thousands of regRNAs
9 1. Map candidate regRNAs 2. Generate ASO leads 3. Optimize leads CAMP4’s proprietary RAP Platform catalogs thousands of regRNAs in any tissue and generates ASO leads to increase gene expression Next-gen sequencing and machine learning algorithms map regRNAs controlling every expressed gene. One of
Slide 10: RAP Platform applied to genetic diseases where modest increases in expression matter
CAMP4 applies its RAP Platform to genetic diseases where modest increases in gene expression can be clinically meaningful 10 Clinical, Regulatory and CMC • Defined patient population • Efficacy can be evaluated in Ph 1/2 based on availability of biomarkers • Established manufacturing & supply chain
Slide 11: Urea Cycle Disorders agenda
CAMP4 overview RAP Platform: regRNAs are master controllers of gene regulation Lead metabolic program: Urea Cycle Disorders Lead CNS program: SYNGAP1-related Disorders 11 Agenda
Slide 12: Urea Cycle Disorders are life-threatening inherited metabolic diseases
Urea Cycle Disorders (UCDs) are a set of life-threatening inherited metabolic diseases characterized by the accumulation of toxic ammonia 12 OTC ASS1 ASL ARG1 CPS1 Dysfunctional Urea Cycle NAGS^ OTC mutated retaining only partial activity UREA AMMONIA Mutation in one of several urea cycle enzymes or
Slide 13: CMP-CPS-001 is a GalNAc-conjugated ASO that increases CPS1 expression
CMP-CPS-001 is a GalNAc-conjugated ASO that binds to a CPS1-specific regRNA to increase CPS1 expression and upregulate the expression of multiple urea cycle enzymes to amplify the conversion of ammonia to urea, potentially addressing more than 90% of patients with late onset UCDs. 13 CAMP4 is target
Slide 14: Modest increases in ureagenesis can significantly reduce disease severity
14 Clinical observations and prior studies have shown that modest increases in ureagenesis can significantly reduce disease severity Early onset (Mortality at birth) Severe UCDs (~90% patients) OTC female heterozygotes (Range of symptoms) WT Late onset patients (>1 month) CMP-CPS-001 target therapeu
Slide 15: CMP-CPS-001 has the potential to be the first disease modifying therapy
CMP-CPS-001 has the potential to be the 1st disease modifying therapy 15 Increase ureagenesis via GalNAc-conjugated ASO *CMP-CPS-001 has been granted Rare Pediatric Disease Designation and Orphan Drug Designation 1x monthly (titratable), subcutaneous • ~90% of severe UCD patients (pediatric and adul
Slide 16: CMP-CPS-001 targets the key regRNA controlling CPS1 expression
16 CMP-CPS-001 targets the key regRNA controlling CPS1 expression RAP Platform identified CPS1 regRNA CMP-CPS-001 increases CPS1 mRNA in primary WT liver cells CPS1 promoter CPS1 enhancers: Target of CMP-CPS-001 CPS1 gene Activity confirmed in OTC deficient donor cells with CMP-CPS-001 analog (clini
Slide 17: ASO targeting mouse Cps1 regRNA reduces ammonia in Otc-deficient mice
ASO targeting mouse Cps1 regRNA in Otc-deficient mice reduces ammonia and supports once-monthly dosing 17 Onset Maintenance Return (1-2 wks) (~4 wks) (~1-2 wks) • The Otcspf-ash mouse model carries a patient mutation in Otc that reduces mRNA levels • Otc activity is 5%-10% of wild-type1 • Model disp
Slide 18: CMP-CPS-001 increases ureagenesis in NHPs
18 Using 13C-Sodium Acetate for ureagenesis in WT NHP NHP administered drug once-monthly. Ureagenesis assessed up to 30 days after final dose. Data shown is one week after a second dose. Error bars represent standard error of the mean; PBS denotes phosphate-buffered saline; * denotes p<.05; ** denot
Slide 19: Clinical Program Update: expansion into Ph 1b in OTC heterozygotes
19 Clinical Program Update: CMP-CPS-001 expansion into Ph 1b in OTC heterozygotes MAD* HV - data expected in Q4 2025 Cohort 2 Cohort 3 Cohort 4 Dosing complete PRIMARY FOCUS: safety, inform MAD PRIMARY FOCUS: safety and pharmacokinetics / pharmacodynamics (URT) Cohort 1 (4 mg / kg) (1.8 mg / kg) (0.
Slide 20: Phase 1 SAD safety and PK summary
20 Phase 1 SAD safety and PK summary (all cohorts 1 – 4) SAD portion of study completed. Conducted planned interim analysis of safety data for 48 normal healthy volunteer participants. 4 Cohorts of 12 participants each, randomized 3:1 investigational product to placebo; 36 individuals received a sin
Slide 21: Ongoing trial has the potential to support a combined Phase 2/3 study
21 Ongoing trial has the potential to support a combined Phase 2/3 study Safety Pharmacokinetics Ureagenesis rate test (PD) • Vitals • Cardiac monitoring • Liver function tests • Immunogenicity • Plasma and urine measurements • Compare human pharmacokinetic behavior to pre-clinical data observations
Slide 22: SYNGAP1 agenda
CAMP4 overview RAP Platform: regRNAs are master controllers of gene regulation Lead metabolic program: Urea Cycle Disorders Lead CNS program: SYNGAP1-related Disorders 22 Agenda
Slide 23: SYNGAP1 is a haploinsufficiency with 10,000+ US patients
SYNGAP1 is a haploinsufficiency with 10,000+ US patients in need of therapy 23 Haploinsufficiency results in 50% of normal protein levels 10,000+ SYNGAP1 patients in the US High unmet need for disease-modifying therapy 6.1–10 per 100K incidence rate 1,2 ~85% have seizures, potentially experiencing 1
Slide 24: Dire unmet need for a targeted disease modifying therapy
Dire unmet need for a targeted disease modifying therapy to alter SYNGAP1 disease course 24 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
Slide 25: CAMP4 aims to increase SynGAP protein levels to restore SynGAP at the synapse
CAMP4 aims to increase SynGAP protein levels to restore SynGAP at the synapse and improve disease symptoms 25 Mutations in SYNGAP1 lead to decreased SynGAP protein, causing increased synaptic firing Neuronal Excitability 1 Illustrative depiction of Electroencephalogram EEG1 Reduced SynGAP-PSD95 comp
Slide 26: Targeting SYNGAP1 regRNA increases transcriptional machinery and nascent transcription
Targeting SYNGAP1 regRNA increases transcriptional machinery and nascent transcription 26 ASO binding to SYNGAP1 regRNA increases RNA Pol II recruitment at SYNGAP1 promoter RegRNA-targeting ASO increases nascent RNA A B C SYNGAP1 promoter regions Human SYNGAP1 regulatory regions • Increased RNA Poly
Slide 27: Targeting SYNGAP1 regRNA restores wild-type levels in patient iPSC-derived neurons
27 Targeting SYNGAP1 regRNA restores wild-type SYNGAP1 levels in patient iPSC-derived neurons compared to familial control NTC = non-targeting control Patient with a heterozygous nonsense mutation in Syngap1 (K1185X) and their healthy familial control 6. 25 12 .5 25 50 10 0 20 0 6. 25 12 .5 25 50 10
Slide 28: CMP-SYNGAP-01 restores near-normal protein levels in humanized mouse model
28 CMP-SYNGAP-01 restores near-normal protein levels in Humanized Mouse Model Haploinsufficient for SynGAP Haploinsufficient Mouse • No copies of mouse Syngap1 • Single copy of Human SYNGAP1 Homozygous Mouse • No copies of mouse Syngap1 • Two copies of Human SYNGAP1 D1 D15 ICV Terminal Protein Mouse
Slide 29: CMP-SYNGAP-01 rescues motor function deficits and increased activity
CMP-SYNGAP-01 rescues motor function deficits and increased activity of Humanized Mouse Model Haploinsufficient for SynGAP 29 Hyperactivity & anxietyMotor function Increased activity is rescued (PalmReader) Motor defects are rescued (Rotarod) Spatial learning defect is rescued (Y-Maze) Cognition & m
Slide 30: Nonhuman Primate PK/PD Study to Assess CMP-SYNGAP-01 Pharmacology
30 Nonhuman Primate PK/PD Study to Assess CMP-SYNGAP-01 Pharmacology aC SF Q2w x3 low Q2w x3 mid Q2w x3 high Q2w x3 C M P- SY N G AP -0 1 TA Dose Dosing frequency Cynomolgus monkey Study Design Key Takeaways • CMP-SYNGAP-01 was clinically well-tolerated at all dose levels • IT administration achieve
Slide 31: Dose-linear increases of CMP-SYNGAP-01 levels across brain regions
Dose-linear increases of CMP-SYNGAP-01 levels across brain regions following repeat IT administration 31 Highest ASO levels observed in brain regions primarily involved in SYNGAP1-related disorders • Dose-linear CMP-SYNGAP-01 concentrations achieved with repeat IT administration • Generally, even di
Slide 32: Intrathecal CMP-SYNGAP-01 resulted in ~50% increase in SynGAP protein in monkeys
Intrathecal administration of CMP-SYNGAP-01 to monkeys resulted in a ~50% increase in SynGAP protein 32 0 1 2 3 Fo ld c ha ng e N or m al iz ed to a C SF ns ✱ ✱✱✱✱ 0 1 2 3 Fo ld c ha ng e N or m al iz ed to a C SF ns ✱✱✱ ✱✱ 0.0 0.5 1.0 1.5 2.0 2.5 Fo ld c ha ng e N or m al iz ed to a C SF ✱ ns ✱ 0 1
Slide 33: CAMP4 positioned to be first in the clinic for SYNGAP1
33 CAMP4 positioned to be first in the clinic for SYNGAP> 1 yr, 100 pts of data obtainable from natural history study Standard-of-Care Natural History Study No disease modifying therapies available Patients currently treated using a polypharmacy approach of symptomatic treatments Third party S
Slide 34: CAMP4 is the leader in regRNA discovery and ASO therapies
CAMP4 is the leader in gene regulatory RNA (regRNA) discovery and regRNA-targeting antisense oligonucleotide (ASO) therapies to upregulate gene expression to restore healthy protein levels 34 Our proprietary RAP Platform was built for the discovery of novel regRNAs that regulate the expression of ev
Slide 35: Thank you
Thank you May 2025 C O R P O R A T E O V E R V I E W
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