Camp4 Therapeutics Corp
For the period ended May 16, 2025. Filed May 16, 2025.
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May 16, 2025 · Filed May 16, 2025
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Contents
EX-99.1 · UCD program
UCD program
Preclinical data
o In Otc-deficient mice, treatment with CMP-CPS-001 resulted in dose-dependent reductions in ammonia levels, which persisted for approximately 4 weeks
Increases in mRNA levels of additional enzymes of the urea cycle were observed, suggesting increased metabolic activity
o In mice with humanized livers, administration of CMP-CPS-001 following an ammonia challenge resulted in increased ureagenesis and decreased ammonia levels
o Administration of CMP-CPS-001 in NHPs resulted in up to 40% increase in ureagenesis, supporting the MOA to convert ammonia to urea
Phase 1 clinical data
o 48 healthy adult participants were enrolled across four SAD cohorts and randomized 3:1 to a single subcutaneous dose of CMP-CPS-001, with 36 participants randomized to CMP-CPS-001
o CMP-CPS-001 was well tolerated, with no evidence of a maximum tolerated dose and no safety trends of concern
o All treatment-emergent adverse events (TEAEs) were Grade 1 (mild) or Grade 2 (moderate) with no serious or severe adverse events (AEs) or TEAEs and no participants discontinued study drug due to a TEAE
Most common TEAEs were headache (n=6) followed by nausea (n=4)
o Pharmacokinetics
Dose-dependent increase in exposure (Cmax and AUC) with clear separation between dose levels
Greater than dose-proportional increase in exposure (Cmax and AUC0-24)
Study Update
o Dosing complete in MAD Cohort 1 through Cohort 3
o Anticipate expansion into OTC heterozygotes to assess safety and CMP-CPS-001 effect on ureagenesis in patients with evidence of reduced urea cycle function
The presentations can be accessed on the CAMP4 website athttps://investors.camp4tx.com/news-events/presentations after the presentations.
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