Cerecin Announces Promising Results for Rare Pediatric Epilepsy Drug

Study shows investigational ketogenic drug CER-0001 was generally well-tolerated and demonstrated seizure reduction in infants with drug-resistant epileptic spasms.

Published on Feb. 26, 2026

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Cerecin Inc., a clinical-stage biotechnology company, 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 demonstrated that CER-0001 may provide a novel pharmacological approach to ketogenic therapy without the burden of strict dietary intervention, supporting further clinical development.

Why it matters

Infantile epileptic spasms, also known as West Syndrome, affect infants typically between 3 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 like CER-0001.

The details

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 showed that CER-0001, an investigational ketogenic drug designed to induce therapeutic ketosis without requiring strict dietary restrictions, was generally well tolerated and demonstrated seizure reduction in the participants.

  • The study, titled "Ketogenic drug tricaprilin (CER-0001) for the treatment of refractory infantile epileptic spasms: a phase 1/2a study," was published in Frontiers in Pediatrics on February 20, 2026.
  • The Phase 1/2a study was conducted across clinical sites in Australia and Singapore.

The players

Cerecin Inc.

A clinical-stage biotechnology company developing metabolic drugs for neurological disorders.

CER-0001 (tricaprilin)

An investigational ketogenic drug designed to induce therapeutic ketosis without requiring strict dietary restrictions.

Samuel Henderson, PhD

Chief Scientific Officer of Cerecin.

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What they’re saying

“Rare epilepsies such as infantile epileptic spasms represent a major unmet medical need. 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.”

— Samuel Henderson, PhD, Chief Scientific Officer of Cerecin (PRNewswire)

What’s next

A larger randomized controlled study is planned to confirm the therapeutic potential of CER-0001 in developmental and epileptic encephalopathies.

The takeaway

This study highlights the potential of CER-0001, an investigational ketogenic drug, to provide a practical metabolic therapy for rare and devastating forms of early-onset epilepsy like infantile epileptic spasms, which currently have limited treatment options.