CAMP4 Therapeutics Gains Rare Pediatric Disease Designation for CMP-CPS-001 to Treat Urea Cycle Disorders

CAMP4 Therapeutics, a clinical-stage biotechnology company headquartered in Cambridge, Massachusetts, has announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to its lead investigational drug candidate CMP-CPS-001. This drug is being developed for the treatment of urea cycle disorders (UCDs), a group of rare and severe inherited metabolic conditions affecting protein metabolism. UCDs can lead to excessive ammonia accumulation in the blood, potentially resulting in irreversible brain damage, disability, seizures, and even death.

The safety, tolerability, and pharmacokinetics of CMP-CPS-001 are currently under evaluation in a randomized, placebo-controlled Phase 1 trial in Australia. According to Josh Mandel-Brehm, CEO of CAMP4, the FDA's designation highlights the seriousness of UCDs and the urgent need for new therapeutic options. Current treatments for UCDs are primarily supportive and do not address the underlying cause of the disease. CMP-CPS-001 aims to be a groundbreaking, disease-modifying treatment by increasing the expression of a key enzyme in the urea pathway, thereby restoring healthy protein levels.

The FDA's Rare Pediatric Disease Designation program incentivizes the development of treatments for rare pediatric diseases that primarily affect individuals from birth to 18 years old. This designation may allow the company to receive a voucher for priority review of a future marketing application for a different product, should CMP-CPS-001 receive FDA approval.

CMP-CPS-001 is an antisense oligonucleotide (ASO) therapeutic candidate specifically targeting carbamoyl phosphate synthetase 1 (CPS1). CPS1 is the first enzyme in the metabolic cycle that converts ammonia to urea. The drug is designed to boost CPS1 gene expression by binding to a specific regulatory RNA sequence, ultimately increasing CPS1 protein levels. CMP-CPS-001 is currently being evaluated in a Phase 1 clinical study.

CAMP4 Therapeutics is focused on developing disease-modifying treatments for a variety of genetic diseases by amplifying healthy protein production through a novel mechanism of gene regulation. The company’s ASO drug candidates target regulatory RNAs (regRNAs), which are critical in controlling gene expression locally at the transcription level. CAMP4's proprietary RAP Platform™ allows for the mapping of regRNAs and the design of optimal chemistry to create potent therapeutic candidates. This approach has the potential to address over 1,200 genetic diseases across multiple tissues.

CAMP4’s innovative strategies and technologies aim to revolutionize the treatment landscape for genetic diseases, providing hope for patients with conditions that currently have limited or no effective treatment options.