Study showed that the investigational ketogenic drug was generally well tolerated and demonstrated seizure reduction in infants with rare drug-resistant epileptic spasms.

Phase 1/2a study supports further development as a metabolic therapy for rare developmental and epileptic encephalopathy (DEE).

DENVER, Feb. 26, 2026 /PRNewswire/ -- Cerecin Inc., a clinical-stage biotechnology company developing metabolic drugs for neurological disorders, today 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, 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.

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.

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.

Promising Early Clinical Results

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

Commitment to Rare Disease Patients

Rare Disease Day highlights the importance of innovation for patients living with uncommon and often severe conditions. Cerecin is developing metabolic medicines designed to address the underlying energy deficits present in a range of neurological diseases, including rare epilepsies.

About CER-0001

CER-0001 (tricaprilin) is an investigational oral ketogenic drug designed to elevate ketone bodies and provide an alternative energy source for the brain. CER-0001 is being developed as a prescription therapy for neurological disorders including rare epilepsies, Alzheimer's disease, and migraine.

CER-0001 has not been approved by any regulatory authority.

About Cerecin

Cerecin is a biopharmaceutical company focused on the development and commercialization of drugs to improve brain health. Cerecin's product development program leverages its extensive experience in lipid science and the potential therapeutic benefits of ketosis. Cerecin is led by an expert executive management team with strong global expertise in central nervous system drug development and supported by two partners, Nestlé and Wilmar, one of the world's leading manufacturers and traders of fatty acids and lipids. Bringing together the deep industry expertise of its leadership team and a highly differentiated portfolio, Cerecin is growing its global presence to serve the brain health needs of patients and people across the world.

Media Contact

Cerecin Inc.
info@cerecin.com 
www.cerecin.com

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SOURCE Cerecin Inc.