CAMP4 Therapeutics Corporation provided corporate updates and outlined key objectives for 2025, including positive safety data for its lead program. The Single Ascending Dose (SAD) portion of the Phase 1 study of CMP-CPS-001 for urea cycle disorders was completed, with the drug demonstrating good tolerability.
The company anticipates reporting Multiple Ascending Dose (MAD) safety and key biomarker data in the second half of 2025. These results are expected to enable the advancement of the CMP-CPS-001 program into a registrational Phase 2/3 trial in 2026.
CAMP4 also reiterated the significance of its strategic partnership with BioMarin and the Rare Pediatric Disease Designation and Orphan Drug Designation granted to CMP-CPS-001. These achievements, combined with the proceeds from the IPO, position the company to progress its pipeline development and execute on key milestones throughout the year.
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