CAMP4 Therapeutics is set to initiate a clinical trial for its therapy aimed at treating SYNGAP1-related disorders, a rare genetic disease characterized by epilepsy and neurodevelopmental delays. This marks a pivotal moment as it will be the first time an antisense oligonucleotide treatment is tested in humans.
The trial is expected to assess the safety, efficacy, and dosing of the therapy, which aims to address the underlying cause of SYNGAP1 by increasing the production of the SynGAP protein, crucial for brain development.
Currently, over 1,800 patients with SYNGAP1 have been identified globally, but the actual number may be higher, as mutations in the SYNGAP1 gene are estimated to account for 1%-2% of all intellectual disabilities. Early preclinical studies have shown promising results, with increased protein expression in primates and improved seizure measures in mouse models.
The trial will involve at least 30 patients aged 2-18 and will utilize a double-blind model, where half of the participants will receive a placebo. This approach, while sometimes controversial in rare disease trials, is intended to provide robust data for regulatory approval.
CAMP4's CEO, Josh Mandel-Brehm, emphasized the importance of allowing the data to guide the definition of success for the trial, which aims to ultimately bring the therapy to market to benefit patients. The company is also in discussions to potentially initiate trials in the U.S. and plans to treat patients through 2027