Camp4 Therapeutics (CAMP) Piper Sandler 36th Annual Healthcare Conference summary

Scientific platform and technology

• Focuses on regulatory RNAs (regRNAs) transcribed from non-coding genome regions, which control local gene expression by forming structures that attract transcription factors and other regulators.

• Uses a proprietary RNA-activating platform combining next-generation sequencing and machine learning to map regRNAs in human cell types, creating detailed gene expression maps.

• Antisense oligonucleotides (ASOs) are designed to target regRNAs, enabling specific upregulation of protein-coding genes, a unique approach compared to traditional downregulation.

• The platform’s specificity allows for targeted gene expression changes without affecting the entire genome.

Lead program: CMP-CPS-001 for urea cycle disorders

• CMP-CPS-001 targets urea cycle disorders, rare genetic diseases caused by mutations in enzymes that convert toxic ammonia to urea.

• Upregulates CPS-1, the first enzyme in the urea cycle, which increases downstream enzyme activity and enhances ammonia clearance.

• Most patients with urea cycle disorders could benefit, except those with specific rare mutations or extremely low enzyme activity.

• Ongoing phase I study in healthy volunteers; single ascending dose (SAD) completed, with safety data expected in Q1 and multiple ascending dose (MAD) data in the second half of next year.

• Ureagenesis rate test will be used as a biomarker to assess drug effect, with plans to move rapidly into patient studies and potential registrational trials.

Pipeline expansion and CNS focus

• Advancing CMP-SYNGAP for SYNGAP1 haploinsufficiency, a severe epilepsy and neurodevelopmental disorder with no approved disease-modifying therapies.

• Intrathecal delivery of oligonucleotides is feasible for CNS targets, leveraging established delivery methods from other approved drugs.

• Preclinical data show upregulation of SYNGAP1, with IND-enabling studies planned as the next step.

• Over 30 haploinsufficiency diseases in the brain are potential future targets.