Camp4 Therapeutics (CAMP) 43rd Annual J.P. Morgan Healthcare Conference summary

Platform and Scientific Approach

• Utilizes antisense oligonucleotides to upregulate gene expression by targeting regulatory RNAs from enhancer and promoter regions, with the RAP Platform cataloging regRNAs and their corresponding protein-coding genes across multiple cell types.

• Next-gen sequencing and machine learning create an in silico database for efficient target and ASO candidate selection, enabling rapid identification and optimization of ASOs for diverse genetic targets.

• Platform allows for disease-specific delivery methods, such as subcutaneous for metabolic and intrathecal for CNS disorders.

• Focuses on diseases where modest gene upregulation can yield significant clinical impact, especially haploinsufficiencies and partial loss-of-function disorders.

• Platform is applicable to a broad array of rare and prevalent diseases, including metabolic, CNS, inflammatory, and fibrotic conditions.

Pipeline and Clinical Development

• Lead program CMP-CPS-001 targets urea cycle disorders by increasing CPS1 expression, with a Phase 1 SAD/MAD trial in healthy volunteers ongoing; MAD data expected in 2H 2025.

• SAD portion of the study is complete; MAD portion is ongoing with positive safety data and no maximum tolerated dose reached.

• Preclinical data demonstrate dose-dependent increases in CPS1 expression and ureagenesis, with significant reductions in toxic ammonia in animal models.

• SYNGAP1 program for CNS disorders is nearing GLP tox studies, with strong preclinical data showing restoration of gene function in patient-derived cells and animal models.

• GBA1 program for Parkinson’s disease is advancing, leveraging existing biomarkers and clinical scales, with new discovery programs expanding into other indications.

Regulatory and Business Development Strategy

• Orphan drug and rare pediatric designations secured for lead program; regulatory interactions planned for the year.

• Ammonia reduction and ureagenesis rate test (URT) are key endpoints for approval, with potential for full approval based on clinically meaningful outcomes.

• Business development is a priority, with a recent collaboration with BioMarin and ongoing efforts to partner on larger indications while retaining rare disease programs.

• Platform enables rapid generation of ASO candidates for partner-nominated targets, supporting both internal pipeline and external collaborations.

• IPO completed in October 2024; company is listed on NASDAQ as CAMP with cash runway into Q2 2026.