Cerecin Inc. has announced the publication of a phase 1/2a clinical study evaluating the investigational drug CER-0001 (tricaprilin) for the treatment of refractory infantile epileptic spasms, a rare and devastating form of early-onset epilepsy.
The study, “Ketogenic drug tricaprilin (CER-0001) for the treatment of refractory infantile epileptic spasms: a phase 1/2a study,” was published in Frontiers in Pediatrics.
Infantile epileptic spasms, also known as West syndrome, affect infants typically between three and 24 months of age and are associated with developmental delay and long-term neurological disability. Despite available treatments, approximately 40–60% of patients do not respond adequately to first-line therapies, highlighting the urgent need for new treatment options.
CER-0001 is an investigational ketogenic drug designed to induce therapeutic ketosis without requiring strict dietary restrictions. Unlike traditional ketogenic diets, CER-0001 can be administered as a prescription oral therapy, potentially expanding access to metabolic treatment approaches.
The phase 1/2a open-label study enrolled eight infants with drug-resistant infantile epileptic spasms across clinical sites in Australia and Singapore. The study demonstrated that CER-0001 may provide a novel pharmacological approach to ketogenic therapy without the burden of strict dietary intervention.
“Rare epilepsies such as infantile epileptic spasms represent a major unmet medical need,” said Samuel Henderson, chief scientific officer of Cerecin.
“These results support the potential of CER-0001 as a practical metabolic therapy that could provide the benefits of ketosis without the challenges of a ketogenic diet. On Rare Disease Day, we reaffirm our commitment to developing metabolic therapies that address the needs of patients with rare neurological disorders.”
The results support further clinical development, and a larger randomised controlled study is planned to confirm the therapeutic potential of CER-0001 in developmental and epileptic encephalopathies.