Potential first-in-class therapeutic for urea cycle disorders enters trial

A strip of 8 PCR tubes on the thermal cycler

CAMP4 Therapeutics has dosed the first participant in its Phase I clinical study of CMP-CPS-001, a potential first-in-class therapeutic for the treatment of urea cycle disorders (UCDs).

UCDs are a group of rare, severe, inherited metabolic diseases impacting protein metabolism. Mutations in genes encoding urea cycle enzymes result in insufficient levels of these important proteins.

CMP-CPS-001 is an antisense oligonucleotide (ASO) designed to amplify CPS1 mRNA by harnessing fundamental cellular gene expression control mechanisms.

CMP-CPS-001 targets carbamoyl phosphate synthetase 1 (CPS1), a key enzyme that catalyses the first step of the urea cycle.

The study is currently active in Australia and anticipates enrolling a total of 96 participants across single- and multiple-ascending dose cohorts.

People with urea cycle disorders accumulate excessive ammonia in their blood, which may cause irreversible brain damage, disability, and seizures, and may be fatal.

These disorders occur across all age groups, from infants to adults, and mild symptoms may go unnoticed until a stressor – such as illness, protein consumption, or environmental stress – overwhelms compensatory functions, resulting in an acute metabolic crisis.

No approved, disease-modifying therapeutics exist for the most prevalent forms of UCD, so the only current treatment options are nitrogen scavengers, strict diet, lifestyle constraints, and monitoring with supportive care during crises.

“This trial initiation is an exciting milestone, representing CAMP4’s first investigational drug candidate to be studied in a clinical trial and one of the first mRNA-amplifying therapeutics in the clinic,” said Josh Mandel-Brehm, CEO of CAMP4. “Most importantly, the clinical development of CMP-CPS-001 is a step toward potentially bringing a new, disease-modifying treatment to individuals living with UCDs.”

Diana Spencer, Senior Digital Content Editor, DDW

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