CalAmp(US:CAMP) Globenewswire·2025-01-07 21:05Core Insights - CAMP4 Therapeutics Corporation is a clinical-stage biotechnology company focused on developing regRNA-targeting antisense oligonucleotide (ASO) therapies to enhance gene expression and restore healthy protein levels [1][2] 2024 Key Highlights - 2024 was a transformative year for CAMP4, marked by a successful IPO, with gross proceeds of approximately $75.0 million from the sale of 6,820,000 shares at an initial price of $11.00 per share [5] - The company received Rare Pediatric Disease Designation and Orphan Drug Designation for its lead program CMP-CPS-001, aimed at treating urea cycle disorders (UCDs) [4][5] - The Phase 1 study of CMP-CPS-001 demonstrated favorable safety results, with all treatment emergent adverse events being Grade 1 (mild) or Grade 2 (moderate) [5] - CAMP4 was added to the Russell 2000® Index, enhancing its market visibility [4][9] - A new discovery program targeting a GBA1 regRNA for Parkinson's disease was initiated [4][9] Expected Milestones in 2025 - The company anticipates reporting Multiple Ascending Dose (MAD) safety and key biomarker data in the second half of 2025, which could facilitate the advancement of CMP-CPS-001 into a registrational Phase 2/3 trial in 2026 [2][4][9] - Initiation of GLP toxicity studies for the lead ASO candidate targeting neurodevelopmental disorders caused by SYNGAP1 mutations is planned for this year [9] - CAMP4 aims to expand its strategic partnerships to maximize the value of its RAP platform [9]