CAMP4 Therapeutics Corporation (CAMP), a clinical-stage biopharmaceutical company, on Monday reported receiving clearance from the Australian Therapeutic Goods Administration (TGA) and the Human Research Ethics Committee (HREC) to initiate the Phase 1/2 trial for CMP-002 in treating SYNGAP1-related disorder.
SYNGAP-1 related disorder is a rare, haplodeficient central nervous system (CNS) disorder resulting in a decrease of 50% in production of the SYNGAP protein due to mutations in the SYNGAP1 gene. The disease may lead to intellectual disability in 100% of patients, epilepsy in approximately 85%, severe behavioral problems in approximately 70%, sleep problems in approximately 60%, and limited communication, with approximately 30% of patients being non-verbal.
The company's lead therapeutic, CMP-002, is an investigational antisense oligonucleotide (ASO), designed to target SYNGAP-specific RNA to increase SYNGAP protein levels. The drug previously showed success in a humanized haplodeficient mouse model and non-human primates dosed intrathecally during preclinical studies.
With the clearance for the trial, CAMP4 is eligible to receive $50 million as a milestone payment, supplementing the initial $50 million provided via a Securities Purchase Agreement signed in September 2025.
The company will, in turn, issue 32,721,172 shares of its common stock to investors participating in the agreement, including Coastland Capital, 5AM Ventures, and CURE SYNGAP1, which is a global initiative to support research into the disease. The closing of the milestone provision is expected within 5 business days.
The proceeds will be utilized in advancing CMP-002 through clinical evaluation. The Phase 1/2 trial is anticipated to begin dosing at a clinical testing site in Australia.
CAMP closed Tuesday at $3.42.
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