Camp4 Therapeutics Corporation (Nasdaq: CAMP) said it received clearance from Australia's Therapeutic Goods Administration (TGA) and a local Human Research Ethics Committee (HREC) to initiate a Phase 1/2 clinical trial of CMP-002, an investigational antisense oligonucleotide (ASO) therapy targeting SYNGAP1-related disorder.

SYNGAP1-related disorder is a rare genetic condition affecting more than 10,000 individuals in the United States. It is characterized by intellectual disability in all patients, epilepsy in approximately 85%, and severe behavioral problems in approximately 70%. No approved disease-modifying therapies currently exist for the condition.

CMP-002 is designed to bind to a SYNGAP1-specific regulatory RNA to increase gene expression and restore SYNGAP protein levels. The drug is administered intrathecally.

The TGA clearance satisfies a regulatory milestone under a Securities Purchase Agreement dated September 9, 2025, under which Camp4 previously received approximately $50 million in gross proceeds. The clearance now allows the company to raise up to an additional $50 million through a second closing, in exchange for up to 32,721,172 shares of common stock or pre-funded warrants. The closing is expected within five business days, subject to customary conditions.

Investors committed to the second closing include Coastlands Capital, Janus Henderson Investors, Balyasny Asset Management, Vivo Capital, 5AM Ventures, Adage Capital Management LP, Trails Edge Capital Partners, and CURE SYNGAP1.

Leerink Partners is acting as lead placement agent. Piper Sandler & Co., Cantor Fitzgerald & Co., and Wedbush Securities Inc. are serving as co-placement agents.

Australia was selected as the first regulatory filing jurisdiction due to regional expertise in SYNGAP1 diagnosis and treatment, established clinical trial infrastructure, and the TGA's review process. Camp4 said it is continuing to pursue additional regulatory filings to expand the trial to other international sites.