CAMP4 Therapeutics has received Australian regulatory and ethics clearance to begin a Phase 1/2 trial of its investigational SYNGAP1 therapy CMP-002.

CAMP4 Therapeutics has received clearance from Australia’s Therapeutic Goods Administration (TGA) and a local Human Research Ethics Committee to begin a Phase 1/2 clinical trial of CMP-002, an investigational therapy for SYNGAP1-related disorder.

The regulatory milestone allows CAMP4 to open an Australian clinical trial site, which is expected to be among the initial locations to enrol patients in the first-in-human study.

SYNGAP1-related disorder is a rare genetic condition caused by mutations in the SYNGAP1 gene. The disorder affects the central nervous system and is associated with intellectual disability, epilepsy, behavioural difficulties, sleep problems and limited communication.

There are currently no approved disease-modifying therapies for people living with SYNGAP1-related disorder.

CMP-002 is an antisense oligonucleotide (ASO) designed to increase expression of the SYNGAP1 gene and raise levels of SYNGAP protein.

The therapy is administered intrathecally, meaning it is delivered into the fluid surrounding the spinal cord.

CAMP4 said its preclinical studies have shown dose-dependent increases in SYNGAP protein expression in patient-derived neurons. The company has also reported improvements in disease-related behavioural and seizure measures in mouse models, alongside evidence of brain distribution and increased SYNGAP protein expression in non-human primates.

These findings are preclinical and will need to be assessed in humans as the programme moves into clinical development.

Josh Mandel-Brehm, president and CEO of CAMP4 Therapeutics, said: “For patients living with SYNGAP1-related disorder, a condition with no approved treatments, this clearance means a potentially disease modifying therapy is now one step closer to moving into human studies for the first time.”

The Phase 1/2 trial is expected to initially enrol patients in Australia, with CAMP4 continuing regulatory activities to support expansion of the study to additional countries and sites.

The company said Australia was selected as its first regulatory filing jurisdiction because of regional expertise in diagnosing and treating people with SYNGAP1-related disorder, alongside established clinical trial infrastructure.

The Australian clearance also triggers a financing milestone under an agreement CAMP4 announced in September 2025. The company is now eligible to receive up to $50 million in additional gross proceeds through a second closing of a private placement.

The financing is expected to support development of CMP-002 and CAMP4’s wider pipeline of investigational therapies designed to increase gene expression in genetic diseases.

The second closing is expected to take place within five business days of the announcement, subject to customary conditions, according to CAMP4.

The company is developing CMP-002 using its regulatory RNA-targeting approach, which aims to increase the production of proteins affected by genetic disorders rather than directly replacing defective genes.

The move into first-in-human clinical development represents a significant step for the programme, although the safety, tolerability and potential clinical benefit of CMP-002 have yet to be established in patients.